header logo image


Page 229«..1020..228229230231..240250..»

Transforming Outcomes in Advanced CSCC with Immunotherapy – LWW Journals

February 4th, 2021 1:51 pm

Are there updated data for LIBTAYO in advanced CSCC? What do they show?

Longer-term data from EMPOWER-CSCC-1 were presented at the 2020 American Society of Clinical Oncology (ASCO) virtual meeting. These results showed an ORR of 46% (95% CI: 39%-53%) following treatment with LIBTAYO, with a median time to response of 2 months (interquartile range: 2-4 months) across the three treatment groups, which were metastatic CSCC and locally advanced CSCC dosed at 3mg/kg every 2 weeks and metastatic CSCC dosed at 350mg every 3 weeks. The median time to CR was 11 months (interquartile range: 7.4-14.8months) among those who achieved a CR in any group. The median DoR hadyet to be reached for any treatment group (range for groups combined: 1.9-34.3 months).4,10

Updated response rates arein the table below.4,10

Safety was generally consistent with previous data. The most common adverse reactions reported were fatigue (35%), diarrhea (28%) and nausea (24%). The most common Grade 3 or higher adverse reactions were pneumonitis (3%), autoimmune hepatitis (2%), anemia, colitis and diarrhea (each 1%).

Warnings and Precautions

Severe and Fatal Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue at any time after starting treatment. While immune-mediated adverse reactions usually occur during treatment, they can also occur after discontinuation. Immune-mediated adverse reactions affecting more than one body system can occur simultaneously. Early identification and management are essential to ensuring safe use of PD-1/PD-L1 blocking antibodies. The definition of immune-mediated adverse reactions included the required use of systemic corticosteroids or other immunosuppressants and the absence of a clear alternate etiology. Monitor closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

No dose reduction for LIBTAYO is recommended. In general, withhold LIBTAYO for severe (Grade 3) immune-mediated adverse reactions. Permanently discontinue LIBTAYO for life-threatening (Grade 4) immune-mediated adverse reactions, recurrent severe (Grade 3) immune-mediated adverse reactions that require systemic immunosuppressive treatment, or an inability to reduce corticosteroid dose to 10 mg or less of prednisone equivalent per day within 12 weeks of initiating steroids.

Withhold or permanently discontinue LIBTAYO depending on severity. In general, if LIBTAYO requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroids.

Immune-mediated pneumonitis:LIBTAYO can cause immune-mediated pneumonitis. In patients treated with other PD-1/PD-L1 blocking antibodies, the incidence of pneumonitis is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.7% (22/591) of patients receiving LIBTAYO, including fatal (0.3%), Grade 4 (0.3%), Grade 3 (1.0%), and Grade 2 (1.9%). Pneumonitis led to permanent discontinuation in 1.9% of patients and withholding of LIBTAYO in 1.9% of patients. Systemic corticosteroids were required in all patients with pneumonitis. Pneumonitis resolved in 59% of the 22 patients. Of the 11 patients in whom LIBTAYO was withheld, 7 reinitiated after symptom improvement; of these 1/7 (14%) had recurrence of pneumonitis. Withhold LIBTAYO for Grade 2, and permanently discontinue for Grade 3 or 4. Resume in patients with complete or partial resolution (Grade 0 to 1) after corticosteroid taper. Permanently discontinue if no complete or partial resolution within 12 weeks of initiating steroids or inability to reduce prednisone to less than 10 mg per day (or equivalent) within 12 weeks of initiating steroids.

Immune-mediated colitis: LIBTAYO can cause immune-mediated colitis. The primary component of immune-mediated colitis was diarrhea. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis treated with PD-1/PD-L1 blocking antibodies. In cases of corticosteroid-refractory immune-mediated colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.2% (7/591) of patients receiving LIBTAYO, including Grade 3 (0.3%) and Grade 2 (0.7%). Colitis led to permanent discontinuation in 0.2% of patients and withholding of LIBTAYO in 0.7% of patients. Systemic corticosteroids were required in all patients with colitis. Colitis resolved in 71% of the 7 patients. Of the 4 patients in whom LIBTAYO was withheld, none reinitiated LIBTAYO. Withhold LIBTAYO for Grade 2 or 3, and permanently discontinue for Grade 4. Resume in patients with complete or partial resolution (Grade 0 to 1) after corticosteroid taper. Permanently discontinue if no complete or partial resolution within 12 weeks of initiating steroids or inability to reduce prednisone to less than 10 mg per day (or equivalent) within 12 weeks of initiating steroids.

Immune-mediated hepatitis:LIBTAYO can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 1.9% (11/591) of patients receiving LIBTAYO, including fatal (0.2%), Grade 4 (0.2%), and Grade 3 (1.5%). Hepatitis led to permanent discontinuation of LIBTAYO in 0.8% of patients and withholding of LIBTAYO in 0.8% of patients. Systemic corticosteroids were required in all patients with hepatitis. Additional immunosuppression with mycophenolate was required in 9% (1/11) of these patients. Hepatitis resolved in 64% of the 11 patients. Of the 5 patients in whom LIBTAYO was withheld, none reinitiated LIBTAYO.

For hepatitis with no tumor involvement of the liver: Withhold LIBTAYO if AST or ALT increases to more than 3 and up to 8 times the upper limit of normal (ULN) or if total bilirubin increases to more than 1.5 and up to 3 times the ULN. Permanently discontinue LIBTAYO if AST or ALT increases to more than 8 times the ULN or total bilirubin increases to more than 3 times the ULN.

For hepatitis with tumor involvement of the liver: Withhold LIBTAYO if baseline AST or ALT is more than 1 and up to 3 times ULN and increases to more than 5 and up to 10 times ULN. Also, withhold LIBTAYO if baseline AST or ALT is more than 3 and up to 5 times ULN and increases to more than 8 and up to 10 times ULN. Permanently discontinue LIBTAYO if AST or ALT increases to more than 10 times ULN or if total bilirubin increases to more than 3 times ULN. If AST and ALT are less than or equal to ULN at baseline, withhold or permanently discontinue LIBTAYO based on recommendations for hepatitis with no liver involvement.

Resume in patients with complete or partial resolution (Grade 0 to 1) after corticosteroid taper. Permanently discontinue if no complete or partial resolution within 12 weeks of initiating steroids or inability to reduce prednisone to less than 10 mg per day (or equivalent) within 12 weeks of initiating steroids.

Immune-mediated endocrinopathies: For Grade 3 or 4 endocrinopathies, withhold until clinically stable or permanently discontinue depending on severity.

Immune-mediated nephritis with renal dysfunction: LIBTAYO can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.5% (3/591) of patients receiving LIBTAYO, including Grade 3 (0.3%) and Grade 2 (0.2%). Nephritis led to permanent discontinuation in 0.2% of patients and withholding of LIBTAYO in 0.3% of patients. Systemic corticosteroids were required in all patients with nephritis. Nephritis resolved in all 3 patients. Of the 2 patients in whom LIBTAYO was withheld, none reinitiated LIBTAYO. Withhold LIBTAYO for Grade 2 or 3 increased blood creatinine, and permanently discontinue for Grade 4 increased blood creatinine. Resume in patients with complete or partial resolution (Grade 0 to 1) after corticosteroid taper. Permanently discontinue if no complete or partial resolution within 12 weeks of initiating steroids or inability to reduce prednisone to less than 10 mg per day (or equivalent) within 12 weeks of initiating steroids.

Immune-mediated dermatologic adverse reactions: LIBTAYO can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson Syndrome (SJS), toxic epidermal necrolysis (TEN), and Drug Rash with Eosinophilia and Systemic Symptoms (DRESS) has occurred with PD-1/PD-L1 blocking antibodies. Immune-mediated dermatologic adverse reactions occurred in 2.0% (12/591) of patients receiving LIBTAYO, including Grade 3 (1.0%) and Grade 2 (0.8%). Immune-mediated dermatologic adverse reactions led to permanent discontinuation in 0.3% of patients and withholding of LIBTAYO in 1.4% of patients. Systemic corticosteroids were required in all patients with immune-mediated dermatologic adverse reactions. Immune-mediated dermatologic adverse reactions resolved in 42% of the 12 patients. Of the 8 patients in whom LIBTAYO was withheld for dermatologic adverse reaction, 5 reinitiated LIBTAYO after symptom improvement; of these 60% (3/5) had recurrence of the dermatologic adverse reaction. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-exfoliative rashes. Withhold LIBTAYO for suspected SJS, TEN, or DRESS. Permanently discontinue LIBTAYO for confirmed SJS, TEN, or DRESS. Resume in patients with complete or partial resolution (Grade 0 to 1) after corticosteroid taper. Permanently discontinue if no complete or partial resolution within 12 weeks of initiating steroids or inability to reduce prednisone to less than 10 mg per day (or equivalent) within 12 weeks of initiating steroids.

Other immune-mediated adverse reactions: The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% in 591 patients who received LIBTAYO or were reported with the use of other PD-1/PD-L1 blocking antibodies. Severe or fatal cases have been reported for some of these adverse reactions.

Infusion-related reactions

Severe infusion-related reactions (Grade 3) occurred in 0.2% of patients receiving LIBTAYO. Monitor patients for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion for Grade 1 or 2, and permanently discontinue for Grade 3 or 4.

Complications of Allogeneic HSCT

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1/PD-L1 blocking antibody. Transplant-related complications include hyperacute graft-versus-host-disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease (VOD) after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1/PD-L1 blockade and allogeneic HSCT. Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with a PD-1/PD-L1 blocking antibody prior to or after an allogeneic HSCT.

Embryo-fetal toxicity

LIBTAYO can cause fetal harm when administered to a pregnant woman due to an increased risk of immune-mediated rejection of the developing fetus resulting in fetal death. Advise women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with LIBTAYO and for at least 4 months after the last dose.

Adverse reactions

Use in specific populations

Please click here for full Prescribing Information.

INDICATIONAND USAGE

LIBTAYO is indicated for the treatment of patients with metastatic cutaneous squamous cell carcinoma (mCSCC) or locally advanced CSCC (laCSCC) who are not candidates for curative surgery or curative radiation.

++++

References:

1. LIBTAYO (cemiplimab-rwlc) injection full U.S. prescribing information. Regeneron Pharmaceuticals, Inc., and sanofi-aventis U.S. LLC. Available at: https://www.regeneron.com/sites/default/files/Libtayo_FPI.pdf

2. Mansouri B, Housewright C. The treatment of actinic keratosesthe rule rather than the exception. J Am Acad Dermatol 2017; 153(11):1200. doi:10.1001/jamadermatol.2017.3395.

3.Schmults CD, et al. High-Risk Cutaneous Squamous Cell Carcinoma A Practical Guide for Patient Management. Springer. ISBN 978-3-662-47081-7 (eBook).DOI 10.1007/978-3-662-47081-7.

4. Data on File. Regeneron Pharmaceuticals Inc. 2020.

5. Data on File. Regeneron Pharmaceuticals Inc. 2018.

6. Migden M, Rischin D, Schmults C, Guminski A, Hauschild A, Lewis K et al. PD-1 Blockade with Cemiplimab in Advanced Cutaneous Squamous-Cell Carcinoma. New England Journal of Medicine. 2018;379(4):341-351.

7. NCCNClinical Practice Guidelines in Oncology (NCCN Guidelines) forSquamous Cell Skin Cancer V.2.2020. National Comprehensive CancerNetwork, Inc. 2020.

8. Califano JA, Lydiatt WM, Nehal KS, et al. Cutaneous squamous cell carcinoma of the head and neck. In: Amin MB, Edge SB, Greene FL, et al, eds. AJCC Cancer Staging Manual. 8th ed. Springer; 2017:171-181.

9. Jennings L, Schmults CD. Management of high-risk cutaneous squamous cell carcinoma. J Clin Aesthet Dermatol. 2010;3(4):39-48.

10. RischinD, Khushalani NI, Schmults CD, et al. Phase 2 study of cemiplimab in patients with advanced cutaneous squamous cell carcinoma (CSCC): longer follow-up. Poster presented at: American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program; May 29-31, 2020.

LIB.20.04.0063 1/21

Read more:
Transforming Outcomes in Advanced CSCC with Immunotherapy - LWW Journals

Read More...

Ashley Cain is living his worst nightmare as his baby daughter battles leukaemia in hospital – The Sun

February 4th, 2021 1:51 pm

ASHLEY Cain praised his sick daughter for 'smiling everyday' as she battles leukaemia.

The former footballer's baby daughter Azaylia underwent a stem cell transplant 10 days ago after her leukaemia had returned.

9

He told The Sun that four-month old is at high risk of complications and that the transplant has likely a lower success outcome after her leukaemia returned.

But he has since opened up about how the tot is doing after the transplant, admitting Azaylia was in a lot of pain.

Taking to Instagram to share some adorable pictures of his smiling daughter, Ashley let his fans know how was she was going.

He captioned the series of pics: "It has now been 10 days since Azaylias Stem Cell transplant and she still gives Daddy bright eyes and big smiles everyday despite her pain to help me through my worries. "

9

9

9

9

"She is going through quite some pain at the moment and has ran into a few side effects over the last 10 days.

Ashley added: "But, she is doing well and her love for life seems to outshine any trouble she is going through. She is a remarkable little girl and her strength fuels my soul every single day!

"LETS GO CHAMP! I BELIVE IN YOU! "

His fans rushed to send words of encouragement to him, Azaylia and his partner, Safiyya Vorajee.

9

"Keep going champs," wrote model, Brandon Myers.

MMA fighter Kane Mousah wrote: "Shes Amazing Brother Shes Has Got This LETS GO CHAMP !!! "

Another Instagram user wrote: "What an inspirational little baby girl you have ! Strongest human Ive known yet ... just an inspiring family all in all."

Azaylia had undergone a second round of chemotherapy to clear the cancer ahead of the stem cell transplant.

However doctors informed the couple that despite their best efforts, the leukaemia had returned quickly.

9

Azaylia, who is battling the rarest form of the disease, will undergo the transplant tomorrow at Birmingham Childrens Hospital.

Speaking to The Sun, Ashley, 30, explained: All we wanted for Christmas was a match for our daughter. We have been told that we have found a match for our daughter. It is amazing. We have had our routes of ups and downs and this is the next stage."

Ashley and Safiyya have been staying at in theHoliday Inn at Snow Hill, next to the hospital, to be close to Azaylia at all times.

9

Speaking ahead of the transplant, Ashley said the news Azaylias leukaemia had returned was hard to comprehend.

He said: It came as a massive blow to us and brought us to our knees. It means she will have to go into transplant with Leukaemia which is not ideal at all.

9

Exclusive

KATIE CALLS COPSKatie Price calls in police after thieves steal her personal photographs

Exclusive

PRICEY PAYDAYKatie Price makes 2,500 in just five days selling old stained tops online

Latest

NEW TURNBeverley Turner hits back at Piers & says hes 'manipulating people' in clap feud

life lessonHolly Willoughby insists Whitty abuser shouldn't be haunted by mistake for life

CIRCLE OF LIFECharlotte Dawson in tears as fan photoshops son and dad Les Dawson together

TRAINSPOTTINGKatie Price takes Harvey to watch trains after documentary lands him job

We were told that because of her age and the aggressiveness of her leukaemia she is in the high risk category for transplant complications and the poor risk category for transplant success.

This news was incredibly hard to hear and has made every day even more difficult to face since.

Ashley said: Were remaining strong and positive for our daughter, she has got past the first stage of treatment against the odds and we truly believe that she will do the same this time around.

She is a fighter, she is a warrior and she will do this and we will be by her side every step of the way!

Read more here:
Ashley Cain is living his worst nightmare as his baby daughter battles leukaemia in hospital - The Sun

Read More...

Biobanks Market | Prominent Factors Analysis that will Help in Reshaping the Market Growth – BioSpace

February 4th, 2021 1:50 pm

Biobanks are essentially repositories for storing biological substances derived from humans, which may include organs, bio-specimens, plasma, saliva, and blood. With a vast rise in the number of organ replacement surgeries globally and significant advancements made in the fields of transfusion and replacement technologies, the role of the biobanks market has become more concrete in the healthcare sector in recent years.

The global biobanks market has witnessed expansion at a significant pace in the recent years owing to the vast rise in prevalence of a number of chronic diseases and the increased demand for personal medicine. The massive rise in incidence rate of conditions such as diabetes, cancer, neurovascular diseases, cardiovascular diseases, and respiratory diseases has compelled government bodies to take stronger actions in terms of investments in biobanks and increasing awareness about them. The global market for biobanks works in coherence with these efforts by adding pace to the process of drug discovery and the treatment of chronic diseases that are caused by activities such as smoking, consumption of alcohol, obesity, and unhealthy lifestyles.

Get Brochure of the Report @ https://www.tmrresearch.com/sample/sample?flag=B&rep_id=2321

Heavy investment by a proactive government has boosted the North America biobanks market to the leading position in the global biobanks market, followed closely by Europe. Both regions are home to several bioscience companies and host a large number of drug discovery and research activities. Asia Pacific still remains the region with the most promising growth potential as the region shown high promise owing to an increase in investments from both government and non-government organizations, along with a large and increasingly affluent population base that is gaining awareness about the market.

Global Biobanks Market: Overview

The global biobanks market owes its conception to the rising need for replacement organs and the steady advancement in the replacement and transfusion technologies regarding a number of bodily substances. Though the technology and need to store organs and other bio-entities had been available for a long time, the global biobanks market took on a more important role in the healthcare sector following the increasing research in genomics. In the new millennium, the development of the personalized medicine field has been the vital driver for the global biobanks market. The likely advancement of the latter, thanks to helpful government regulations, is likely to make the crucial difference for the global biobanks market in the near future.

The steady technological advancement in the healthcare sector in the last few decades has now led to a scenario where the full potential of biobanks can be harnessed. As a result, the global biobanks market is projected to exhibit steady growth over the coming years.

Global Biobanks Market: Key Trends

The rising interest in personalized medicine is the prime driver for the global biobanks market. Personalized medicine has only become a viable branch of modern medicine after steady research in genomics and the way various patients react to various medicines. The biobanks market has thus come into the spotlight thanks to their role as a steady supplier of human biomaterials for research as well as direct application. The increasing research in genomics following the findings of the Human Genome Project is thus likely to remain a key driver for the global biobanks market in the coming years.

Buy this Premium Report @ https://www.tmrresearch.com/checkout?rep_id=2321&ltype=S

The utilization of biobanks in stem cell research has been hampered in several regions by ethical concerns regarding the origin of stem cells. Nevertheless, the potential of stem cells in the healthcare sector is immense, and is likely to have a decisive impact on the trajectory of the global biobanks market in the coming years. Many countries have, in recent years, adopted a supportive stance towards stem cell research, aiding the growth of the biobanks market. Continued government support is thus likely to remain vital for the global biobanks market in the coming years.

Global Biobanks Market: Market Potential

The leading role of the U.S. in the global biobanks market is unlikely to change in the coming years. The easy availability of government-supported healthcare infrastructure and the presence of several industry giants in the region has driven the biobanks market in the U.S.

Northwell Health, the largest healthcare provider in New York State, earlier in 2017 initiated a new biobank aimed at creating precision therapies against various types of cancer. Launched in collaboration with Indivumed, the biobank will provide catalogued biomaterials for research into lung, colorectal, breast, and pancreatic cancer. This would enable targeted, gene-specific studies of a variety of cancer samples, leading to a more comprehensive understanding of cancer. Such well-funded collaboration efforts are crucial for the developing biobanks market.

Global Biobanks Market: Geographical Dynamics

Led by the fertile healthcare research scenario in the U.S., North America is likely to retain a dominant share in the global biobanks market in the coming years. Steady support from institutes such as the NIH is likely to be vital for the North America biobanks market.

Emerging Asia Pacific economies such as India and China could emerge with a significant share in the global biobanks market in the coming years. The healthcare sector in both countries has received steady public or private funding in the last few years. India is also a global leader in medical tourism and is likely to receive an increasing number of patients in the coming years, leading to promising prospects for the global biobanks market in the region.

Global Biobanks Market: Competitive Dynamics

Due to the dynamic nature of the global biobanks market, with advancements in diagnostic fields often determining the direction of the market, the market is heavily fragmented. It is likely to retain a significant degree of fragmentation in the coming years thanks to the diversity in the application segments of the biobanks market. The leading players in the global biobanks market include BioCision, Tecan Group, VWR, Beckman Coulter Inc., and Thermo Fisher Scientific.

Get Table of Content of the Report @ https://www.tmrresearch.com/sample/sample?flag=T&rep_id=2321

About TMR Research

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

Contact:

Rohit Bhisey

TMR Research,

3739 Balboa St # 1097,

San Francisco, CA 94121

United States

Tel: +1-415-520-1050

Visit Site: https://www.tmrresearch.com/

Excerpt from:
Biobanks Market | Prominent Factors Analysis that will Help in Reshaping the Market Growth - BioSpace

Read More...

Biotechnology | Encyclopedia.com

February 4th, 2021 1:48 pm

Biotechnology is a set of techniques by which human beings modify living things or use them as tools. In its modern form, biotechnology uses the techniques of molecular biology to understand and manipulate the basic building blocks of living things. The earliest biotechnology, however, was the selective breeding of plants and animals to improve their food value. This was followed in time by the use of yeast to make bread, wine, and beer. These early forms of biotechnology began about ten thousand years ago and lie at the basis of human cultural evolution from small bands of hunter-gatherers to large, settled communities, cities, and nations, giving rise, in turn, to writing and other technologies. It is doubtful that, at the outset, the first biotechnologists understood the effects of their actions, and so the reason for their persistence in pursuing, for example, selective breeding over the hundreds of generations necessary to show much advantage in food value, remains something of a mystery.

The world's historic religions emerged within the context of agriculture and primitive biotechnology, and as one might expect they are at home in that context, for instance through their affirmation of agricultural festivals. In addition, Christianity took the view that nature itself has a history, according to which, nature originally was a perfectly ordered garden, but as a result of human refusal to live within limits, nature was cursed or disordered by its creator. The curse makes nature at once historic, disordered, both friendly and hostile to human life, and open to improvement through human work. These effects fall especially on human agriculture and childbirth, both of which are focal areas of biotechnology.

By the time of Charles Darwin (18091882), plant and animal breeders were deliberate and highly successful in applying techniques of selective breeding to achieve specific, intended results. Darwin's theory of evolution is built in part on his observation of the ability of animal breeders to modify species. The work of human breeders helped Darwin see that species are variable, dynamic, and subject to change. Inspired by the success of intentional selective breeding, Darwin proposed his theory of natural selection, by which nature unintentionally acts something like a human breeder. Nature, however, uses environmental selection, which favors certain individuals over others in breeding. The theory of natural selection, of course, led to a profound shift in human consciousness about the fluidity of life, which in turn fueled modern biotechnology and its view that life may be improved. While Christianity struggled with other implications of Darwinism, it did not object to the prospect that human beings can modify nature, perhaps even human nature.

In the twentieth century, as biologists refined Darwin's proposal and explored its relationship to genetics, plant breeders such as Luther Burbank (18491926) and Norman Borlaug (1914) took selective breeding to new levels of success, significantly increasing the quality and quantity of basic food crops. But it was the late twentieth-century breakthroughs in molecular biology and genetic engineering that established the technological basis for modern biotechnology. The discovery that units of hereditary information, or genes, reside in cells in a long molecule called deoxyribonucleic acid (or DNA) led to an understanding of the structure of DNA and the technology to manipulate it. Biotechnology is no longer limited to the genes found in nature or to those that could be moved within a species by breeding. Bioengineers can move genes from one species to another, from bacteria to human beings, and they can modify them within organisms.

The discovery in 1953 of the structure of DNA by Francis Crick (b. 1916) and James Watson (b. 1928) is but one key step in the story of molecular biology. Within two decades, this discovery opened the pathway to the knowledge of the socalled genetic alphabet or code of chemical bases that carry genetic information, an understanding of the relationship between that code and the proteins that result from it, and the ability to modify these structures and processes (genetic engineering). The decade of the 1980s saw the first transgenic mammals, which are mammals engineered to carry a gene from other species and to transmit it to their offspring, as well as important advances in the ability to multiply copies of DNA (polymerase chain reaction or PCR). The Human Genome Project, an international effort begun around 1990 to detail the entire DNA information contained in human cells, sparked the development of bioinfomatics, the use of powerful computers to acquire, store, share, and sort genetic information. As a result, not only is a standard human DNA sequence fully known (published in February 2001), but it is now possible to determine the detailed code in any DNA strand quickly and cheaply, a development likely to have wide applications in medicine and beyond.

Biotechnology is also dependent upon embryology and reproductive technology, a set of techniques by which animal reproduction is assisted or modified. These techniques were developed largely for agricultural purposes and include artificial insemination, in vitro fertilization, and other ways of manipulating embryos or the gametes that produce them. In 1978, the first in vitro human being was born, and new techniques are being added to what reproductive clinics can do to help women achieve pregnancy. These developments have been opposed by many Orthodox Christian and Roman Catholic theologians, by the Vatican, and by some Protestants, notably Paul Ramsey. Other faith traditions have generally accepted these technologies. In addition, some feminist scholars have criticized reproductive medicine as meeting the desires of men at the expense of women and their health.

Reproductive medicine, however it may be assessed on its own merits, does raise new concerns when it is joined with other forms of biotechnology, such as genetic testing and genetic engineering. In the 1990s, in vitro fertilization was joined with genetic testing, allowing physicians to work with couples at risk for a genetic disease by offering them the option of conceiving multiple embryos, screening them for disease before implantation, and implanting only those that were not likely to develop the disease. This technique, known as preimplantation diagnosis, is accepted as helpful by many Muslim, Jewish, and Protestant theologians, but is rejected by Orthodox Christians and in official Catholic statements. The ground for this objection is that the human embryo must be shown the respect due human life, all the more so because it is weak and vulnerable. It is permissible to treat the embryo as a patient, but not to harm it or discard it in order to treat infertility or to benefit another. The usual counterargument is to reject the view that the embryo should be respected as a human life or a person.

Developments in cloning and in the science and technology of stem cells offer additional tools for biotechnology. In popular understanding, cloning is usually seen as a technique of reproduction, and of course it does have that potential. The birth of Dolly, the cloned sheep, announced in 1997, was a surprising achievement that suggests that any mammal, including human beings, can be created from a cell taken from a previously existing individual. Many who accept reproductive technology generally, including such techniques as in vitro fertilization, found themselves opposing human reproductive cloning, but they are not sure how to distinguish between the two in religiously or morally compelling ways. With few exceptions, however, religious institutions and leaders from all faith traditions have opposed human reproductive cloning, if only because the issues of safety seem insurmountable for the foreseeable future. At the same time, almost no one has addressed the religious or moral implications of the use of reproductive cloning for mammals other than human beings, although it has been suggested that it would not be wise or appropriate to use the technique to produce large herds of livestock for food because of the risk of a pathogen destroying the entire herd.

The technique used to create Dollythe transfer of the nuclear DNA from an adult cell to an egg, thereby creating an embryo and starting it through its own developmental processcan serve purposes other than reproduction, and it is these other uses that are especially interesting to biotechnology. Of particular interest is the joining of the nuclear transfer technique with the use of embryonic stem cells to treat human disease. In 1998, researchers announced success in deriving human embryonic stem cells from donated embryos. These cells show promise for treating many diseases. Once derived, they seem to be capable of being cultured indefinitely, dividing and doubling in number about every thirty hours. As of 2002, researchers have some confidence that these cells can be implanted in the human body at the site of disease or injury, where they can proliferate and develop further, and thereby take up the function of cells that were destroyed or impaired.

Stem cells, of course, can be derived from sources other than the embryo, and research is underway to discover the promise of stem cells derived from alternative sources. There are two advantages in using these other sources. First, no embryos are destroyed in deriving these cells. For anyone who sets a high standard of protection for the human embryo, the destruction of the embryo calls into question the morality of any use of embryonic stem cells. Second, the use of stem cells from sources other than an embryo may mean that in time, medical researchers will learn how to derive healing cells from the patient's own body. The advantage here is that these cells, when implanted, will not be rejected by the patient's immune system. Embryonic stem cells, which may have advantages in terms of their developmental plasticity, are decidedly problematic because of the immune response.

One way to eliminate the immune response is to use nuclear transfer to create an embryo for the patient, harvesting stem cells from that embryo (thereby destroying it) and implanting these cells in the patient. Because they bear the patient's DNA, they should not be rejected. This approach is medically complicated, however, and involves the morally problematic step of creating an embryo to be destroyed for the benefit of another.

As a result of the developments in the underlying science and technologies, biotechnology is able to modify any form of life in ways that seem to be limited only by the imagination or the market. Biotechnology has produced genetically modified microorganisms for purposes ranging from toxic waste clean-up to the production of medicine. For example, by inserting a human gene into a bacterium that is grown in bulk, biotechnology is able to create a living factory of organisms that have been engineered to make a specific human protein. Such technologies may also be used to enhance the virulence of organisms, to create weapons for bioterrorism, or to look for means of defense against such weapons. Aside from obvious concerns about weapons development, religious institutions and scholars have not objected to these uses of biotechnology, although some Protestant groups question the need for patents, especially when sought for specific genes.

Plants, perhaps the first organisms modified by the earliest biotechnology, remain the subject of intense efforts. Around the year 2000, major advances were made in plant genome research, leading to the possibility that the full gene system of some plant species can be studied in detail, and the ways in which plants respond to their environment may be understood as never before. Some attention is given to plants for pharmaceutical purposes, but the primary interest of biotechnology in plants is to improve their value and efficiency as sources of food. For instance, attempts have been made to increase the protein value of plants like rice. The dependence of farm plants on fertilizer and pesticides may also be reduced using biotechnology to engineer plants that, for instance, are resistant to certain insects.

In the 1990s, the expanding use of genetically modified plants in agriculture was met with growing concerns about their effects on health and on the environment. Adding proteins to plants by altering their genes might cause health problems for at least some who consume the plants, perhaps through rare allergic reactions. Genes that produce proteins harmful to some insects may cause harm to other organisms, and they might even jump from the modified farm plant to wild plants growing nearby. Furthermore, some believe that consumers have a right to avoid food that is altered by modern biotechnology, and so strict segregation and labeling must be required. Deeply held values about food and, to some extent, its religious significance underlie many of these concerns. In Europe and the United Kingdom, where public opposition to genetically modified food has been strong, some churches have objected to excessive reliance upon biotechnology in food production and have supported the right of consumers to choose, while at the same time recognizing that biotechnology can increase the amount and the value of food available to the world's neediest people.

Animals are also modified by biotechnology, and this raises additional concerns for animal welfare. Usually the purpose of the modification is related to human health. Biotechnologists may, for example, create animals that produce pharmaceuticals that are expressed, for instance, in milk, or they may create animal research models that mimic human disease. These modifications usually involve a change in the animal germlinethat is, they are transmissible to future generations and they affect every cell in the body. Such animals may be patented, at least in some countries. All this raises concern about what some see as the commodification of life, the creation of unnecessary suffering for the animals, and a reductionistic attitude toward nature that sees animals as nothing but raw materials that may be reshaped according to human interest.

It is the human applications of biotechnology, however, that elicit the most thorough and intense religious responses. As of 2002, genetic technologies are used to screen for a wide range of genetic conditions, but treatments for these diseases are slow to develop. Screening and testing of pregnancies, newborns, and adults have become widespread in medicine, and the resulting knowledge is used to plan for and sometimes prevent the development of disease, or to terminate a pregnancy in order to prevent the birth of an infant with foreseeable health problems. Some religious bodies, especially Roman Catholic and Orthodox Christian, vigorously criticize this use of genetic testing. One particular use of prenatal testingto identify the sex of the unborn and to abort femalesis thought to be widespread in cultures that put a high priority on having sons, even though it is universally criticized. It is believed that the uses of testing will grow, while the technologies to treat disease will lag behind.

Attempts at treatment lie along two general pathways: pharmaceuticals and gene therapy. Biotechnology offers new insight into the fundamental processes of disease, either by the creation of animal models or by insight into the functions of human cells. With this understanding, researchers are able to design pharmaceutical products with precise knowledge of their molecular and cellular effects, with greater awareness of which patients will benefit, and with fewer side effects. This is leading to a revolution in pharmaceutical products and is proving to be effective in treating a range of diseases, including cancer, but at rapidly increasing costs and amidst growing concerns about access to these benefits, especially in the poorest nations.

Gene therapy, begun in human beings in 1990, tries to treat disease by modifying the genes that affect its development. Originally the idea was to treat the classic genetic diseases, such as Tay Sachs or cystic fibrosis, and it is expected that in time this technique will offer some help in treating these diseases. But gene therapy will probably find far wider use in treating other diseases not usually seen as genetic because researchers have learned how genes play a role in the body's response to every disease. Modifying this response may be a pathway to novel therapies, by which the body treats itself from the molecular level. For instance, it has been shown that modified genes can trigger the regeneration of blood vessels around the heart. In time these approaches will probably be joined with stem cell techniques and with other cell technologies, giving medicine a range of new methods for modifying the body in order to regenerate cells and tissues.

Religious opinion has generally supported gene therapy, seeing it as essentially an extension of traditional therapies. At the same time, both religious scholars and bioethicists have begun to debate the prospect that these technologies will be used not just to treat disease but to modify traits, such as athletic or mental ability, that have nothing to do with disease, perhaps to enhance these traits for competitive reasons. Many accept the idea of therapy but reject enhancement, believing that there is a significant difference between the two goals. Many scholars, however, are skeptical about whether an unambiguous distinction can be drawn, much less enforced, between therapy and enhancement. Starting down the pathway of gene therapy may mean that human genetic enhancement is likely to follow. This prospect raises religious concerns that people who can afford to do so will acquire genetic advantages that will lead to further privilege, or that people will use these technologies to accommodate rather than challenge social prejudices.

It is also expected that these techniques will be joined with reproductive technologies, opening the prospect that future generations of humans can be modified. The prospect of such germline modification is greeted with fear and opposition by many, usually for reasons that suggest religious themes. In Europe, germline modification is generally rejected as a violation of the human rights of future generations, specifically the right to be born with a genome unaffected by technology. In the United States, the opposition is less adamant but deeply apprehensive about issues of safety and about the long-term societal impact of what are popularly called "designer babies." Religious bodies have supported these concerns and have called either for total opposition or careful deliberation.

How far biotechnology can go is limited by the complexities of life processes, in particular in the subtleties of interaction between DNA and the environment. Biotechnology itself helps researchers discover these subtleties, and as much as biotechnology depends upon the sciences of biology and genetics, it must be noted that the influence between technology and science is reciprocal. The Human Genome Project, for instance, opened important new questions about human evolution and about how DNA results in proteins. Knowledge of the genomes of various species reveals that the relationship between human beings and distant species, such as single-celled or relatively simple organisms, turns out to be surprisingly close, suggesting that evolution conserves genes as species diverge.

Perhaps even more surprising is the way in which the Project has challenged the standard view in modern genetics of the tight relationship between each gene and its protein, the so-called dogma of one gene, one protein. It turns out that human beings have about one hundred thousand proteins but only about thirty-three thousand genes, and that genes are more elusive and dynamic than once thought. It appears that DNA sequences from various chromosomes assemble to become the functional gene, the complete template necessary to specify the protein, and that these various sequences can assemble in more than one way, leading to more than one protein. Such dynamic complexity allows some thirty-three thousand DNA coding sequences to function as the templates for one hundred thousand proteins. But this complexity, in view of the limited understanding of the processes that define it, means that the ability to modify DNA sequences may have limited success and unpredictable consequences, which should lower confidence in genetic engineering, especially when applied to human beings.

Biotechnology is further limited by financial factors. Most biotechnology is pursued within a commercial context, and the prospect of near-term financial return must be present to support research. Biotechnology depends upon access to capital and upon legal protection for intellectual property, such as the controversial policy of granting patent protection on DNA sequences or genes and on genetically modified organisms, including mammals. This financial dependence is itself a matter of controversy, giving rise to the fear that life itself is becoming a mere commodity or that the only values are those of the market.

There is no reason, however, to think that biotechnology has reached the limits of its powers. On the contrary, biotechnology is growing not just in the scope of its applications but in the range and power of its techniques. Biotechnology's access to the whole genomes of human beings and other species means that the dynamic action and interaction of the entire set of genes can be monitored. In one sense, the completion of full genomes ushers in what some have called post-genomic biotechnology, characterized by a new vantage point of a systematic overview of the cell and the organism. This is proving valuable, for instance, in opening new understandings of cancer as a series of mutation events within a set of cells in the body. Attention is turning, however, from the study of genes to the study of proteins, which are more numerous than genes but also more dynamic, coming quickly into and out of existence in the trillions of cells of the human body according to precise temporal and spatial signals. Most human proteins are created only in a small percentage of cells, during a limited period of human development, and only in precisely regulated quantities. Studying this full set of proteins, in all its functional dynamism, is a daunting task requiring technologies that do not exist at the beginning of the twenty-first century. The systematic study of proteins, called proteomics, may in fact become a new international project for biology, leading in time to a profound expansion of the powers of biotechnology.

In time, researchers will develop powerful new methods for modifying DNA, probably with far higher precision and effectiveness than current techniques allow, and perhaps with the ability to transfer large amounts of DNA into living cells and organisms. Computer power, which is essential to undertakings like the Human Genome Project and to their application, continues to grow, along with developments such as the so-called gene chip, using DNA as an integrated part of the computing device. Advances in engineering at the very small scale, known as nanotechnology (from nanometer, a billionth of a meter), suggest that molecular scale devices may someday be used to modify biological functions at the molecular level. For instance, nanotechnology devices in quantity may be inserted into the human body to enter cells, where they might modify DNA or other molecules. In another area of research, scientists are exploring the possibility that DNA itself may be used as a computer or a data storage device. DNA is capable of storing information more efficiently than current storage media, and it may be possible to exploit this capacity.

It is impossible to predict when new techniques will be developed or what powers they will bring. It is clear, however, that new techniques will be found and that they will converge in their effectiveness to modify life. Precisely designed pharmaceutical products will be available to treat nearly every disease, often by interrupting them at the molecular level and doing so in ways that match the specific needs of the patient. Stem cells, whether derived from embryos or from patients themselves, will probably be used to regenerate nearly any tissue or cell in the body, perhaps even portions of organs, including the brain. The genes in patients' bodies will be modified, either to correct a genetic anomaly that underlies a disease or to trigger a special response in specific cells to treat a disease or injury. It is more difficult to foresee the full extent of the long-term consequences of biotechnology on nonhuman species, on the ecosystem, on colonies of life beyond Earth, and on the human species itself; estimates vary in the extreme. Some suggest that through these means, human beings will engineer their own biological enhancements, perhaps becoming two or more species.

The prospect of these transformations has evoked various religious responses, and scholars from many traditions have been divided in their assessments. Those who support and endorse biotechnology stress religious duties to heal the sick and feed the hungry. Most hold the view that nature is to be improved, perhaps within limits, and that human beings are authorized to modify the processes of life. Some suggest that creation is not static but progressive, and that human beings are co-creators with God in the achievement of its full promise.

Others believe biotechnology will pervert nature and undermine human existence and its moral basis. They argue, for instance, that genetic modifications of offspring will damage the relationship between parents and children by reducing children to objects, products of technology, and limit their freedom to grow into persons in relationship with others. Some warn that saying yes to biotechnology now will make it impossible to say no in the future. Still others suggest that the point is not to try to stop biotechnology but to learn to live humanely with its powers, and as much as possible to steer it away from selfish or excessive uses and toward compassionate and just ends.

See also Cloning; Darwin, Charles; DNA; Evolution; Eugenics; Gene Patenting; Gene Therapy; Genetically Modified Organisms; Genetic Engineering; Genetics; Genetic Testing; Human Genome Project; In Vitro Fertilization; Reproductive Technology; Stem Cell Research

american association for the advancement of science. "human inheritable genetic modifications: assessing scientific, ethical, religious, and policy issues." washington, d.c.: aaas, 2000

bruce, donald, and bruce, ann, eds. engineering genesis: the ethics of genetic engineering in non-human species. london: earthscan publications, 1998

chapman, audrey r. unprecedented choices: religious ethics at the frontiers of genetic science. minneapolis, minn.: fortress press, 1999

cole-turner, ronald. the new genesis: theology and the genetic revolution. louisville, ky.: westminster and john knox press, 1993.

cole-turner, ronald. beyond cloning: religion and the remaking of humanity. harrisburg, pa.: trinity press international, 2001.

dorff, elliot n. matters of life and death: a jewish approach to modern medical ethics. philadelphia, pa.: jewish publication society, 1998

dorff, elliot n. "jewish views on technology in health care." in claiming power over life: religion and biotechnology policy, ed. mark j. hanson. washington, d.c.: georgetown university press, 2001.

evans, john h. playing god? human genetic engineering and the rationalization of public bioethical debate. chicago: university of chicago press, 2002.

genome sequencing consortium. "initial sequencing and analysis of the human genome." nature 409 (2001): 860921.

kilner, john f.; pentz, rebecca d.; and young, frank e., eds. genetic ethics: do the ends justify the genes? grand rapids, mich.: eerdmans, 1997.

national council of churches, panel on bioethical concerns. genetic engineering: social and ethical consequences. new york: pilgrim press, 1984.

peacocke, arthur r. god and the new biology. san francisco: harper, 1986.

peters, ted. playing god? genetic determinism and human freedom. new york: routledge, 1997.

peters, ted, ed. genetics: issues of social justice. cleveland, ohio: pilgrim press, 1998.

peterson, james c. genetic turning points: the ethics of human genetic intervention. grand rapids, mich.: eerdmans, 2001.

rahner, karl. "the problem of genetic manipulation." in theological investigations, vol. 9, trans. g. harrison. new york: seabury, 1966.

rahner, karl. "the experiment with man: theological observations on man's self-manipulation." in theological investigations, vol. 9, trans. g. harrison. new york: seabury, 1966.

ramsey, paul. fabricated man: the ethics of genetic control. new haven, conn.: yale university press, 1970.

shinn, roger lincoln. the new genetics: challenges for science, faith and politics. london and wakefield, r.i.: moyer bell, 1996.

venter, j. craig, et. al. "the sequence of the human genome." science 291 (2001): 13041351.

willer, roger a., ed. genetic testing and screening: critical engagement at the intersection of faith and science. minneapolis, minn.: kirk house, 1998.

world council of churches, church and society. manipulating life: ethical issues in genetic engineering. geneva: world council of churches, 1982.

world council of churches, church and society. biotechnology: its challenges to the churches and the world. geneva: world council of churches, 1989.

ronald cole-turner

See the original post:
Biotechnology | Encyclopedia.com

Read More...

PDS Biotechnology Moves Forward With Full Enrollment In PDS0101 Trial In HPV-Associated Cancers, Shares Retreat – Yahoo Finance

February 4th, 2021 1:47 pm

TipRanks

Lets talk about risk and the big picture. Its an appropriate time, as the big risk presented by the COVID-19 pandemic is finally receding thanks to the ongoing vaccination program. COVID is leaving behind an economy that was forced into shutdown one year ago while in the midst of a great expansion, boosted by the deregulation policies. While the new Biden Administration is busy reversing many Trump policies, at least for now the economy is rebounding. And this brings us to risk. A time of economic growth and rebound is a forgiving time to move toward risk investments, as general economic growth tends to lift everything. Two strategists from JPMorgan have recently chimed in, promoting the view that the markets fundamentals are still sound, and that small- to mid-cap sector is going to keep rising. First, on the general conditions, quant strategist Dubravko Lakos-Bujas wrote, Although the recent technical selloff and short squeeze is receiving a lot of attention, we believe the positive macro setup, improving fundamentals and COVID-19 outlook, strength of the US consumer, as well as the reflation theme remain the bigger forces at play. Not only should this drive further equity upside, but it remains favorable for continued rotation into economic reopening Building on this, Eduardo Lecubarr, chief of the Small/Mid-Cap Strategy team, sees opportunity for investors now, especially in the smaller value stocks. We stick to our view that 2021 will be a stockpickers paradise with big money-making opportunities if you are willing to go against the grain Many macro indicators did fall in January but SMid-Caps and equities in general continued to edge higher, Lecubarr noted. And if you are prone to look at high-risk, small- to mid-cap stocks, youll find yourself drawn to penny stocks. The risk involved with these plays scares off the faint hearted as very real problems like weak fundamentals or overwhelming headwinds could be masked by the low share prices. So, how should investors approach a potential penny stock investment? By taking a cue from the analyst community. These experts bring in-depth knowledge of the industries they cover and substantial experience to the table. Bearing this in mind, we used TipRanks database to find two compelling penny stocks, according to Wall Street analysts. Both tickers boast a Strong Buy consensus rating and could climb over 200% higher in the year ahead. CNS Pharmaceuticals (CNSP) We will start with CNS Pharmaceuticals, a biotechnology company with a focus on the treatment of glioblastomas, a class of aggressive tumors that attack the braid and spinal cord. These cancers, while rare, are almost always terminal, and CNS is working a new therapy designed to more effectively cross the blood-brain barrier to attack glioblastoma. Berubicin, CNSs flagship drug candidate, is an anthracycline, a potent class of chemotherapy drugs derived from the Streptomyces bacteria strains, and used in the treatment of a wide variety of cancers. Berubicin is the first drug in this class to show promise against glioblastoma cancers. The drug candidate has completed its Phase 1 clinical trial, in which 44% of patients showed a clinical response. This number included one patient who showed a Durable Complete Response, defined as a demonstrated lack of detectable cancer. Following the success of the Phase 1 study, CNS applied for, and received, FDA approval of its Investigational New Drug application. This gives the company the go-ahead to conduct a Phase 2 study on adult patients, an important next step in the development of the drug. CNS plans to start the mid-stage trial in 1Q21. Based on the potential of the companys asset in glioblastoma, and with its share price at $2.22, several analysts believe that now is the time to buy. Among the bulls is Brooklines 5-star analyst Kumaraguru Raja who takes a bullish stance on CNSP shares. Until now, the inability of anthracyclines to cross the blood brain barrier prevented its use for treatment of brain cancers. Berubicin is the first anthracycline to cross the blood-brain barrier in adults and access brain tumors Berubicin has promising clinical data in a Phase 1 trial in recurrent glioblastoma (rGBM) and has Orphan drug designation for treatment of malignant gliomas from the FDA. We model approval of Berubicin for treatment of recurrent glioblastoma in 2025 based on the Phase 2 data with 55% probability of success for approval. We model peak sales of $533 million in 2032, Raja opined. CNS pipeline also includes WP1244 (novel DNA binding agent) that is 500x more potent than daunorubicin in inhibiting tumor cell proliferation is expected to enter the clinic in 2021 In vivo testing in orthotopic models of brain cancer showed high uptake of WP1244 by brain and subsequent antitumor activity, the analyst added. To this end, Raja rates CNSP a Buy, and his $10 price target implies room for a stunning 350% upside potential in the next 12 months. (To watch Rajas track record, click here) What does the rest of the Street have to say? 3 Buys and 1 Hold add up to a Strong Buy consensus rating. Given the $8.33 average price target, shares could climb ~275% in the year ahead. (See CNSP stock analysis on TipRanks) aTyr Pharma (LIFE) The next stock were looking at, aTyr Pharma, has a focus on inflammatory disease. Its leading drug candidate, ATYR1923, is a Neuropilin-2 (NRP2) agonist, working through the receptor proteins expressed by the NRP2 gene. These pathways are important for cardiovascular development and disease, and play a role in the inflammatory lung disease pulmonary sarcoidosis. In December, the company reported that the drug candidate had completed enrollment of 36 patients in a Phase 1b/2a clinical trial, testing the drug in the treatment of pulmonary sarcoidosis. Results of the current study are expected in 3Q21, and will inform further trials of ATYR1923, including against other forms of inflammatory lung disease. On a more immediate note, in early January the company announced top-line results of another Phase 2 clinical involving ATRY1923 this time in the treatment of patients hospitalized with severe respiratory complications from COVID-19. The results were positive, showing that a single dose of ATYR1923 (at 3 mg/kg) resulted in a 5.5-day median recovery time. Overall, of the patients dosed in this manner, 83% saw recovery in less than one week. Covering LIFE for Roth Capital, 5-star analyst Zegbeh Jallah noted, We like the risk profile here, with two shots on goal, and updated data details from the COVID study is expected in the coming months. Also announced recently, is that data from aTyr's Pulmonary Sarcoidosis program, will be reported in 3Q21 the success of either of these studies could result in a doubling or more of the market cap as these opportunities appear to barely be accounted for by investors. In line with his optimistic approach, Jallah gives LIFE shares a Buy rating and his $15 price target suggests an impressive 277% potential upside for the coming year. (To watch Jallahs track record, click here) Other analysts are on the same page. With 2 additional Buy ratings, the word on the Street is that LIFE is a Strong Buy. On top of this, the average price target is $13.33, suggesting robust growth of ~236% from the current price of $3.97. (See LIFE stock analysis on TipRanks) To find good ideas for penny stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

Read more from the original source:
PDS Biotechnology Moves Forward With Full Enrollment In PDS0101 Trial In HPV-Associated Cancers, Shares Retreat - Yahoo Finance

Read More...

Nano Biotechnology Market 2021, Size, Share, Global Industry Growth, Business Statistics, Top Leaders, Competitive Landscape, Forecast To 2027 KSU |…

February 4th, 2021 1:47 pm

Nano Biotechnology Market:Global Size, Trends, Competitive, Historical & Forecast Analysis, 2020-2025. Increasing demand of new therapies for various tissues of lungs and cardiac as well as rising organizations investment in research and development for new drugs are some important factors driving the growth of Nano biotechnology market.

Get Sample Copy of This Premium Report https://brandessenceresearch.com/requestSample/PostId/1259

Nano biotechnology is application of nanotechnology which is applied in biological field. The concept nanotechnology means a designed, development and applications of material which functional make up is on a nanometer scale. Technically nanotechnology is developing material or other structured material which is size from 1 to 100 nanometers. And biotechnology means the study of biological subjects including microorganism. Nano biotechnology is intersection of biology and nanotechnology. Nano biotechnology is a study of biological and biochemical applications. Nano biotechnology is used to study the nature of fabricating new Nano devices and elements which exists in living organisms. The use of Nano biotechnology in commercial applications in biomedical field is direction towards development of new techniques in biosciences and drug delivery systems.

The global nano biotechnology is segmented on the basis of applications, therapeutics and by regional basis. Based on application the global Nano biotechnology is segmented as medical research, medical devices, pharmaceuticals and others. On the basis of therapeutics the global Nano biotechnology market is divided as orthopedic therapies, dental therapies, cardiac therapies and others.

The regions covered in global Nano biotechnology report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, Global Nano biotechnology sub divided in U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Nano Biotechnology Companies:

Global Nano biotechnology reports cover prominent players like,

Dabur Pharma

cellix

InanoBio

Marina Biotech

dermaCM

Ablynx

Xantac Bioanalytics

AC Serndip

WaveSense

Nanobiotech Pharma

Celgene

Thermo Fisher Scientific

merck KGaA

Bionano Genomics

Others

New Nano drug candidate kills aggressive breast cancer cells

News: July 20, 2020 The researchers at the University of Arkansas have developed a new Nano drug candidate that kills triple-negative breast cancer cells. Triple-negative breast cancer is one of the most aggressive and fatal types of breast cancer. The research will help clinicians target breast cancer cells directly while avoiding the adverse and toxic side effects of chemotherapy. The American Cancer Society estimated 268,600 new cases of invasive breast cancer in 2019 and 41,760 deaths. Researchers in Beyzavis laboratory focus on developing new targeted photodynamic therapy drugs, as alternative to chemotherapy and significantly fewer side effects.

Global Nano Biotechnology Dynamics

The key factor for growth of global Nano biotechnology is increasing advancements in medical science to improve healthcare practices around the world for better drug delivery system which drives the growth of Nano biotechnology market. The product of Nano biotechnology is under trial but scientist have confident that this trail will give positive result to increase the prevention process, diagnosis and treatment of various diseases. Nano biotechnology is also used in areas like electronics, biomaterial and energy production. The public and private companies are also showing keen interest in Nano biotechnology which gives commercial platform for the product of Nano biotechnology.

More detailed research is required to introduce diverse components of Nano biotechnology in medical applications which restrain the growth of global Nano biotechnology market. The effect of toxicity of Nano material on environment and economy affect the growth of Nano biotechnology market. However the increase in government initiative for research and development in Nano biotechnology will increase opportunities of Nano biotechnology market in future.

The National Nanotechnology Initiatives (NNI) funded approximately USD 170 billion in 2018. Currently there are 60 countries which is member of National Nanotechnology Initiative and conducted program on National Nanotechnology. According to National institute of standard there are 1700 nanotechnology companies worldwide.

Nano BiotechnologyRegional Analysis

North America is dominating the growth nano biotechnology due to increase in investment in research and development projects and implementing flexible policies. The National Nanotechnology Initiatives which was established in 2001 which develop nanotechnology within United States. There is 3729 patent registered in USA on Nanotechnology in 2018. European Union is expected to emerge the growth of share in global Nano biotechnology market. To increase in research and development spending and investment in public and private sectors of Nano biotechnology. The European Union has published the publications on Nano biotechnology.

Asia Pacific is also emerging in the field of nano biotechnology. India has introduced Nano biotechnology based chemotherapy. The Nano biotechnology have introduced polymer free drug for treatment of cardiac disease. The drug name is Sirolimous which shows extreme excellent result which reduces the blockages of in the arteries. China has duplicated research paper and publication about 3592 and 30.479 respectively. The patent on Nano biotechnology of china is 5030 in 2019.

Key Benefits forNano biotechnologyreports

Global Nano biotechnology report covers in depth historical and forecast analysis.

Global Nano biotechnology Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market opportunities, Competitive Analysis, Regional and Country Level.

Global Nano biotechnology report helps to identify opportunities in market place.

Global Nano biotechnology report covers extensive analysis of emerging trends and competitive landscape.

Nano BiotechnologySegmentation

by Application:Medical Devices, Pharmaceuticals, Medical research, Others

by Therapeutics:Cardiac Therapies, Orthopedic Therapies, Dental therapies, Others

Regional & Country AnalysisNorth America, U.S., Mexico, Canada , Europe, UK, France, Germany, Italy , Asia Pacific, China, Japan, India, Southeast Asia, South America, Brazil, Argentina, Columbia, The Middle East and Africa, GCC, Africa, Rest of Middle East and Africa

Continued

Get Full Report: https://brandessenceresearch.com/biotechnology/nano-biotechnology-market-industry-analysis

https://www.marketwatch.com/press-release/cell-culture-industry-size-industry-analysis-report-statistics-application-development-price-trends-competitive-market-share-forecast-20212027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/europe-construction-equipment-market-by-growth-trends-opportunities-and-industry-forecast-to-2026-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/asia-mobile-generator-market-trends-size-2021-growth-strategies-opportunity-challenges-rising-trends-and-forecast-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/organic-poultry-market-expected-to-reach-usd-3102-billion-by-2025-cagr-101-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/us-pigmentation-disorder-market-size-2021-growth-types-trends-size-share-and-top-key-players-by-forecast-research-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/plant-extracts-market-their-uses-types-size-share-trends-global-industry-report-2021-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/uk-porcine-vaccine-market-2021-size-share-industry-growth-business-statistics-top-leaders-competitive-landscape-forecast-to-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/uk-potato-protein-market-size-2021-share-growth-rate-and-gross-margin-development-trends-industry-forecast-report-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/us-pressure-ulcer-treatment-market-share-size-by-stage-products-sales-shares-challenges-opportunities-industry-revenue-and-forecast-to-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/radioimmunoassay-market-by-type-application-share-size-trends-growth-and-industry-report-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/us-telehealth-market-size-2020-opportunities-key-players-revenue-emerging-trends-and-business-strategy-till-2027-2021-02-03?tesla=y

https://www.marketwatch.com/press-release/wireless-brain-sensor-market-size-2021-share-region-wise-analysis-of-top-players-growth-rate-application-and-forecast-by-2027-2021-02-03?tesla=y

Read more from the original source:
Nano Biotechnology Market 2021, Size, Share, Global Industry Growth, Business Statistics, Top Leaders, Competitive Landscape, Forecast To 2027 KSU |...

Read More...

Is Ovid Therapeutics Inc (OVID) The Right Choice in Biotechnology? – InvestorsObserver

February 4th, 2021 1:47 pm

Ovid Therapeutics Inc (OVID) is near the middle in its industry group according to InvestorsObserver. OVID gets an overall rating of 40. That means it scores higher than 40 percent of stocks. Ovid Therapeutics Inc gets a 39 rank in the Biotechnology industry. Biotechnology is number 26 out of 148 industries.

Searching for the best stocks to invest in can be difficult. There are thousands of options and it can be confusing on what actually constitutes a great value. Investors Observer allows you to choose from eight unique metrics to view the top industries and the best performing stocks in that industry. A score of 40 would rank higher than 40 percent of all stocks.

These rankings allows you to easily compare stocks and view what the strengths and weaknesses are of a given company. This lets you find the stocks with the best short and long term growth prospects in a matter of seconds. The combined score incorporates technical and fundamental analysis in order to give a comprehensive overview of a stocks performance. Investors who then want to focus on analysts rankings or valuations are able to see the separate scores for each section.

Ovid Therapeutics Inc (OVID) stock is lower by -3.34% while the S&P 500 is higher by 0.66% as of 12:01 PM on Thursday, Feb 4. OVID has fallen -$0.11 from the previous closing price of $3.29 on volume of 1,234,638 shares. Over the past year the S&P 500 has risen 15.62% while OVID has fallen -20.30%. OVID lost -$1.39 per share the over the last 12 months.

Click Here to get the full Stock Score Report on Ovid Therapeutics Inc (OVID) Stock.

Subscribe to our daily morning update newsletter and never miss out on the need-to-know market news, movements, and more.

You May Also Like

See the article here:
Is Ovid Therapeutics Inc (OVID) The Right Choice in Biotechnology? - InvestorsObserver

Read More...

Is Geron Corporation (GERN) the Top Pick in the Biotechnology Industry? – InvestorsObserver

February 4th, 2021 1:47 pm

A rating of 73 puts Geron Corporation (GERN) near the top of the Biotechnology industry according to InvestorsObserver. Geron Corporation's score of 73 means it scores higher than 73% of stocks in the industry. Geron Corporation also received an overall rating of 61, putting it above 61% of all stocks. Biotechnology is ranked 26 out of the 148 industries.

Searching for the best stocks to invest in can be difficult. There are thousands of options and it can be confusing on what actually constitutes a great value. Investors Observer allows you to choose from eight unique metrics to view the top industries and the best performing stocks in that industry. A score of 61 would rank higher than 61 percent of all stocks.

These rankings allows you to easily compare stocks and view what the strengths and weaknesses are of a given company. This lets you find the stocks with the best short and long term growth prospects in a matter of seconds. The combined score incorporates technical and fundamental analysis in order to give a comprehensive overview of a stocks performance. Investors who then want to focus on analysts rankings or valuations are able to see the separate scores for each section.

Geron Corporation (GERN) stock is lower by -6.67% while the S&P 500 is higher by 0.66% as of 11:47 AM on Thursday, Feb 4. GERN is lower by -$0.14 from the previous closing price of $2.10 on volume of 5,084,909 shares. Over the past year the S&P 500 has risen 15.62% while GERN is higher by 48.48%. GERN lost -$0.35 per share the over the last 12 months.

Click Here to get the full Stock Score Report on Geron Corporation (GERN) Stock.

Subscribe to our daily morning update newsletter and never miss out on the need-to-know market news, movements, and more.

You May Also Like

See the rest here:
Is Geron Corporation (GERN) the Top Pick in the Biotechnology Industry? - InvestorsObserver

Read More...

Is PDS Biotechnology Corp (PDSB) Stock Near the Top of the Biotechnology Industry? – InvestorsObserver

February 4th, 2021 1:47 pm

PDS Biotechnology Corp (PDSB) is near the top in its industry group according to InvestorsObserver. PDSB gets an overall rating of 77. That means it scores higher than 77 percent of stocks. PDS Biotechnology Corp gets a 94 rank in the Biotechnology industry. Biotechnology is number 24 out of 148 industries.

Finding the best stocks can be tricky. It isnt easy to compare companies across industries. Even companies that have relatively similar businesses can be tricky to compare sometimes. InvestorsObservers tools allow a top-down approach that lets you pick a metric, find the top sector and industry and then find the top stocks in that sector.

This ranking system incorporates numerous factors used by analysts to compare stocks in greater detail. This allows you to find the best stocks available in any industry with relative ease. These percentile-ranked scores using both fundamental and technical analysis give investors an easy way to view the attractiveness of specific stocks. Stocks with the highest scores have the best evaluations by analysts working on Wall Street.

PDS Biotechnology Corp (PDSB) stock is down -11.08% while the S&P 500 is higher by 0.18% as of 11:54 AM on Wednesday, Feb 3. PDSB is down -$0.43 from the previous closing price of $3.88 on volume of 1,803,239 shares. Over the past year the S&P 500 is higher by 16.24% while PDSB is higher by 36.90%. PDSB lost -$1.57 per share the over the last 12 months.

Click Here to get the full Stock Score Report on PDS Biotechnology Corp (PDSB) Stock.

Subscribe to our daily morning update newsletter and never miss out on the need-to-know market news, movements, and more.

You May Also Like

More here:
Is PDS Biotechnology Corp (PDSB) Stock Near the Top of the Biotechnology Industry? - InvestorsObserver

Read More...

Biotechnology Crop Seeds Market Analysis and Demand with Forecast Overview to 2026 – AlgosOnline

February 4th, 2021 1:47 pm

A comprehensive research study on Biotechnology Crop Seeds market available at MarketStudyReport.com provides insights into the market size and growth trends of this industry over the forecast timeline. The study evaluates key aspects of Biotechnology Crop Seeds market in terms of the demand landscape, driving factors and growth strategies adopted by market players.

The study on Biotechnology Crop Seeds market provides thorough insights pertaining to major growth drivers, challenges, and opportunities that will influence the industry expansion in the forthcoming years.

Request a sample Report of Biotechnology Crop Seeds Market at:https://www.marketstudyreport.com/request-a-sample/3221257?utm_source=algosonline.com&utm_medium=SHR

According to the research document, the industry is expected to register XX% CAGR over the forecast period (2021-2026), subsequently accruing significant gains by the end of analysis timeframe.

The market is in a disarray due to the lockdowns imposed for limiting the spread of COVID-19 outbreak. In addition to the sudden slump of revenue, some businesses are expected to tackle a plethora of challenges post the pandemic.

Most of the businesses across various sectors have renewed their budgets to focus on profit gains in upcoming years. Our comprehensive analysis of the business sphere can help stakeholders in making sound decisions to compensate for market uncertainties and support them in building strong contingency plans.

Further, the report provides a granular assessment of the several industry segmentations to convey a deeper understanding of the areas with strong profit potential.

Key inclusions of the Biotechnology Crop Seeds market report:

Ask for Discount on Biotechnology Crop Seeds Market Report at:https://www.marketstudyreport.com/check-for-discount/3221257?utm_source=algosonline.com&utm_medium=SHR

Biotechnology Crop Seeds Market segments covered in the report:

Regional segmentation: North America, Europe, Asia-Pacific, South America, Middle East and Africa

Product types: Herbicide Tolerant and Insect Tolerant

Applications spectrum: Corn, Soybean, Cotton and Others

Competitive outlook: Bayer, Corteva, KWS SAAT, Limagrain and China National Chemical

For More Details On this Report: https://www.marketstudyreport.com/reports/global-biotechnology-crop-seeds-market-2021-by-manufacturers-regions-type-and-application-forecast-to-2026

Related Reports:

1. Global Cabrio Greenhouses Market 2021 by Company, Regions, Type and Application, Forecast to 2026Cabrio Greenhouses market research report provides the newest industry data and industry future trends, allowing you to identify the products and end users driving Revenue growth and profitability. The industry report lists the leading competitors and provides the insights strategic industry Analysis of the key factors influencing the market.Read More: https://www.marketstudyreport.com/reports/global-cabrio-greenhouses-market-2021-by-company-regions-type-and-application-forecast-to-2026

2. Global Greenhouse Heating Systems Market 2021 by Manufacturers, Regions, Type and Application, Forecast to 2026Greenhouse Heating Systems Market report begins from overview of Industry Chain structure, and describes industry environment, then analyses market size and forecast of Greenhouse Heating Systems by product, region and application, in addition, this report introduces market competition situation among the vendors and company profile, besides, market price analysis and value chain features are covered in this report.Read More: https://www.marketstudyreport.com/reports/global-greenhouse-heating-systems-market-2021-by-manufacturers-regions-type-and-application-forecast-to-2026

Read More Reports On: https://www.marketwatch.com/press-release/Explosion-Proof-Equipment-Market-Size-2027-Industry-Sales-Revenue-Price-trends-and-more-2021-02-03

Read More Reports On: https://www.marketwatch.com/press-release/Neoprene-Market-Size-2025-Industry-Sales-Revenue-Price-trends-and-more-2021-02-04

Contact Us:Corporate Sales,Market Study Report LLCPhone: 1-302-273-0910Toll Free: 1-866-764-2150 Email: [emailprotected]

More here:
Biotechnology Crop Seeds Market Analysis and Demand with Forecast Overview to 2026 - AlgosOnline

Read More...

Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 – Arizton – PRNewswire

February 3rd, 2021 8:53 pm

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- In-depth analysis and data-driven insights on the impact of COVID-19 included in this Europe cell and gene therapy market report.

The Europe cell and gene therapy market is expected to grow at a CAGR of over 23% during the period 20202026.

Key Highlights Offered in the Report:

Key Offerings:

Get your sample today! https://www.arizton.com/market-reports/europe-cell-gene-therapy-market-size-analysis

Europe Cell and Gene Therapy Market Segmentation

Europe Cell and Gene Therapy Market by Product

Europe Cell and Gene Therapy Market by End-user

Europe Cell and Gene Therapy Market by Application

Europe Cell and Gene Therapy Market Dynamics

Cell and gene therapy is revolutionizing the global healthcare segment. Although various new cell and gene therapies are approved, there are various hurdles that limit the penetration of new therapies, such as high cost, multiple regulatory hurdles, and other manufacturing challenges. These cell and gene therapy developers need reliable, efficient, and cost-effective manufacturing services with the flexibility to scale up production as the demand increases. Cell and gene therapy products are very complex, and their manufacturing requires skilled labor, developed infrastructure for limited patients. Such huge investments will affect vendors and contract manufacturing organizations (CMOs) work with companies to overcome these challenges.

Key Drivers and Trends fueling Market Growth:

Europe Cell and Gene Therapy Market Geography

European countries such as Germany, France, the UK, Italy, and Spain play a significant role in the cell and gene therapy market. However, clinical trials and the number of manufacturing facilities are increasing slowly in Europe. Europe has become a major R&D destination for many vendors as the funding for cell and gene therapies is increasing across many European countries. Europe stands next to North America in the global cell and gene therapy market. Initially, Europe led the cell and gene therapy market due to first product approvals. France, Germany, and Italy had a greater contribution globally and in Europe. However, from the past decade, the US has competed and increased its market share globally. Europe stands second in the market, with the increasing prevalence of cancer and rare genetic disorders that are not effectively solved by the conventional therapies are increasing in the region. This increased target population is driving the demand for cell and gene therapy in the region.

Get your sample today! https://www.arizton.com/market-reports/europe-cell-gene-therapy-market-size-analysis

Europe Cell and Gene Therapy Market by Geography

Major Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

Explore our healthcare & lifesciencesprofile to know more about the industry.

Read some of the top-selling reports:

About Arizton:

AriztonAdvisory and Intelligence is an innovation and quality-driven firm, which offers cutting-edge research solutions to clients across the world. We excel in providing comprehensive market intelligence reports and advisory and consulting services.

We offer comprehensive market research reports on industries such as consumer goods & retail technology, automotive and mobility, smart tech, healthcare, and life sciences, industrial machinery, chemicals and materials, IT and media, logistics and packaging. These reports contain detailed industry analysis, market size, share, growth drivers, and trend forecasts.

Arizton comprises a team of exuberant and well-experienced analysts who have mastered in generating incisive reports. Our specialist analysts possess exemplary skills in market research. We train our team in advanced research practices, techniques, and ethics to outperform in fabricating impregnable research reports.

Mail: [emailprotected] Call: +1-312-235-2040 +1-302-469-0707

SOURCE Arizton Advisory & Intelligence

See the original post here:
Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire

Read More...

Paragon Biosciences Launches CiRC Biosciences to Expand Cell and Gene Therapy Platform – PRNewswire

February 3rd, 2021 8:53 pm

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, today announced the launch of CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye.

"The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer of Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."

CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.

The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform.

"Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer of CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.

CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020)discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."

The abstract in Nature is available here: https://www.nature.com/articles/s41586-020-2201-4

Access to the NEJM article is available here: https://www.nejm.org/doi/full/10.1056/NEJMcibr2027602

About CiRC Biosciences CiRC Biosciences is a privately held cell therapy company dedicated to developing treatments for serious diseases with high, unmet needs with an initial focus on the eye. Currently it is pre-clinical phase for Geographic Atrophy Age-Related Macular Degeneration (Dry AMD) and advanced Retinitis Pigmentosa (RP). CiRC Biosciences is a portfolio company of Paragon Biosciences. Visit our website: https://circbiosciences.com/.

About Paragon Biosciences Paragon is a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the replicable Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contact:

Evelyn M. O'Connor Paragon Biosciences 312-847-1335 [emailprotected]

SOURCE Paragon Biosciences

Home

See the article here:
Paragon Biosciences Launches CiRC Biosciences to Expand Cell and Gene Therapy Platform - PRNewswire

Read More...

Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market – BioSpace

February 3rd, 2021 8:53 pm

Global Retinal Gene Therapy Market: Overview

The retinal gene therapy market is estimated to expand at an exponential growth rate. For the use of gene therapy, retina is considered a highly desirable target as it an irreplaceable part of a body. The global retinal gene therapy market is likely to be influenced by the promise its holds for the treatment of various forms of inherited and non-inherited blindness. Furthermore, this therapy can also be used in the treatment of rare genetic retinal diseases, such as Leber's congenital amaurosis, which is likely to augur well for the development of the global retinal gene therapy market during the forecast period, from 2020 to 2030. It is expected that the global retinal gene therapy market is anticipated to witness the entry of new players, with the presence of promising candidates in the phases of drug approval process.

Read Report Overview - https://www.transparencymarketresearch.com/retinal-gene-therapy-market.html

Type, application, and region are the three important parameters based on which the global retinal gene therapy market has been classified. Such detailed analysis of the market comes with the sole purpose to provide stakeholders with a detailed and clear analysis of the global retinal gene therapy market.

Read Report Overview - https://www.transparencymarketresearch.com/retinal-gene-therapy-market.html

Global Retinal Gene Therapy Market: Notable Developments

One of the important market developments that give a quick view of the dynamics pertaining to the global retinal gene therapy market is mentioned as below:

There is only one player in this global retinal gene therapy market, which is mentioned as below:

Request for Analysis of COVID-19 Impact on Retinal Gene Therapy Market- https://www.transparencymarketresearch.com/sample/sample.php?flag=covid19&rep_id=78059

Global Retinal Gene Therapy Market: Key Trends

The global retinal gene therapy market is characterized by the presence of the following restraints, drivers, and opportunities.

Advent of High-end Technologies to Support Development of the Market

Mostly in the cases of inherited retinal diseases, retinal gene therapy is performed. Gene therapy is capable of bettering vision impairment through mutation in RPE65 gene. Luxturna, a recently introduced gene therapy is utilized for the treatment of patients suffering from type 2 Leber's congenital amaurosis. This disease is a form of inherited disease that causes impairment in vision at the time of birth, which leads to a highly progressive degeneration. At present, there are many retinal gene therapy at the clinical trial phase and those are utilizing recombinant viruses. This factor is likely to increase the scope of growth for the global retinal gene therapy market over the period of assessment, from 2020 to 2030.

Request for Custom Research - https://www.transparencymarketresearch.com/sample/sample.php?flag=CR&rep_id=78059

In addition to that, the emergence of new market players together with the advent of high-end technological developments is likely to encourage growth of the global retinal gene therapy market during the forecast period. It is estimated that retinal gene therapy is likely to come up as a standard form of treatment for such retina-related diseases.

Pre Book Retinal Gene Therapy Market Report - https://www.transparencymarketresearch.com/checkout.php?rep_id=78059&ltype=S

Global Retinal Gene Therapy Market: Geographical Analysis

North America is clearly at the forefront of the growth of the global retinal gene therapy market at the very moment. It is estimated that the region will continue to retain its dominance over the period of forecast, from 2020 to 2030. So far, the product that has been approved for use is from a manufacturer from this region. Europe is likely to emerge as another lucrative region in the global retinal gene therapy market over the period of forecast.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through ad hoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.

ContactMr. Rohit BhiseyTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY - 12207United StatesUSA - Canada Toll Free: 866-552-3453Email: sales@transparencymarketresearch.comWebsite: https://www.transparencymarketresearch.com/

Go here to see the original:
Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace

Read More...

The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -…

February 3rd, 2021 8:53 pm

London, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Roots Analysis has announced the addition of Gene Therapy Market (4th Edition), 2020-2030 report to its list of offerings.

Success of approved gene therapies has resulted in a surge in interest of biopharmaceutical developers in this rapidly evolving domain. Presently, the ability of gene therapies to treat diverse disease indications is considered among the most prominent drivers of this market. In addition, promising clinical results of pipeline candidates are anticipated to draw in more investments to support product development initiatives.

To order this 720+ page report, which features 220+ figures and 375+ tables, please visit this link

Key Market Insights

Around 800 gene therapies are currently being developed across different stages Apart from 10 approved products, most of the aforementioned therapies (65%) are in the early stages of development (discovery / preclinical), while the rest are being evaluated in clinical trials. It is worth mentioning that more than 40% of clinical stage candidates are intended for the treatment of oncological disorders.

Over 65% of innovator companies focused on gene therapy development, are based in North AmericaInterestingly, more than 75 players based in the same region, are start-ups, while over 35 are mid-sized players, and 10 are large and very large firms. Since the majority of gene therapy developers are headquartered in the US, it is considered a key R&D hub for such advanced therapy medicinal products.

There are 400+ registered gene therapy focused clinical trials, worldwideClinical research activity, in terms of number of trials registered, is reported to have increased at a CAGR of 12% during the period 2015-2020. Of the total number of trials, close to 25% have already been completed, and 35% claim to be actively recruiting.

USD 25.4 billion has been invested by both private and public investors, since 2015So far, a significant proportion of the capital raised has been through secondary offerings (USD 12.9 billion). On the other hand, around USD 5 billion was invested by venture capital investors, representing 20% of the total amount.

Close to 20,000 patents have been filed / published related to gene therapies, since 2016Around 30% of the total number of applications were related to gene editing-based therapies, while the remaining were associated with gene therapies. Further, majority of the patent assignees were industry players, however, the contribution of non-industry players in the overall patent filing activity has increased considerably (CAGR of 16%), over the past few years.

There have been several mergers and acquisitions in this market during the period 2015-2019 In fact, M&A activity is reported to have increased at a CAGR of more than 40%. Key drivers of the acquisitions mentioned in the report include, therapeutic area expansion, access to a novel technology / platform, drug class consolidation and drug class expansion.

North America and Europe are anticipated to capture over 90% of the market share, in terms of sales revenues, in 2030In vivo gene therapies currently represent a significant share of the market, and this trend is unlikely to change in the foreseen future, as several such candidates are being evaluated in late stages. In addition, more than 130,000+ patients are projected to use gene therapies in 2030 and the demand for gene therapies is expected to grow at an annualized rate of 29% and 31% during the periods 2020-2025 and 2025-2030, respectively.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 14.6 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom, gene therapies exhibit the potential to become a promising alternative for the treatment of genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research includes brief profiles of key players (listed below) engaged in the development of gene therapies; each profile features an overview of the therapy, current development status, clinical trials and its results (if available), target indication, route of administration, and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapies-market/268.html or email sales@rootsanalysis.com

You may also be interested in the following titles:

View post:
The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -...

Read More...

Gene Therapy Market to Reach US$ 20.9 Billion by 2027, Globally |CAGR: 29.7%|UnivDatos Market Insights – PR Newswire India

February 3rd, 2021 8:53 pm

NOIDA, India, Jan. 29, 2021 /PRNewswire/ -- A comprehensive overview of the gene therapy market is recently added by UnivDatos Market Insights to its humongous database. The gene therapy market report has been aggregated by collecting informative data of various dynamics such as market drivers, restraints, and opportunities. This innovative report makes use of several analyses to get a closer outlook on the gene therapy market. The gene therapy market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth. Furthermore, this statistical market research repository examines and estimates the gene therapy market at the global and regional levels. The Global Gene therapy Market is expected to grow at a CAGR of 29.7% from 2021-2027 to reach US 20.9 billion by 2027.

Market Overview

Gene therapy is the next trend of curative transformation in the life sciences industry. Globally, around 2,600 clinical trials in gene therapy have been performed, are underway, or have been approved to date. More than ever, the field of gene therapy seeks to identify a route to the clinic and the market. Approximately 20 gene therapies have now been licensed and over two thousand clinical trials of human gene therapy have been published globally. Aging populations worldwide and socio-economic risk factors are among the primary influences driving this surge.

As per Alliance for Regenerative Medicine (ARM) Quarterly Regenerative Medicine Global Data Report Q12019, 372 gene therapy clinical trials were in progress as of the end of Q1. Remarkably, a margin (217 or 58%) were studies in Phase II, followed by Phase I (123 or 33%), and Phase III (32 or 9%). The number of gene therapy clinical trials edged up by 10 from the 362 recorded as of the end of 2018.

Request Sample Copy of this Report @ https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

Covid-19 Impact:

The COVID-19 pandemic has dislocated global management attempts across gene therapies. The manufacture and delivery of treatments, research and clinical development, and commercial operations are the three areas within the gene therapy sector that have been most interrupted amid the COVID-19 crisis. The development of gene therapies has been less affected. For instance, Peter Marks, Director of FDA's Center for Biologics Evaluation and Research (CBER) stated that with the arrival of therapies for cell and gene therapies over the last five years, it should have doubled in size while it is only modestly larger, 15-20% larger in size.

Ask for Price & Discounts @ https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

Global Gene therapy Market report is studied thoroughly with several aspects that would help stakeholders in making their decisions more curated.

By Vector, the market is primarily bifurcated into

The viral vector segment dominated the gene therapy vector market in 2019 and will grow at 29.2% CAGR to reach US$ 17.9 billion by the year 2027.

By Viral Vector, the market is primarily sub-segmented into

Amongst viral vector types, adeno-associated virus accounted for the largest share and is expected to grow at 29.3% CAGR during the forecast period 2021-2027. In 2019, the adeno-associated virus segment accounted for a revenue share of almost 34%.

By Gene Type, the market is primarily studied into

In 2019, the antigen segment dominated the global gene therapy market with nearly 19.2% of the market share and it is anticipated by 2027, the segment will garner US$ 3.9 billion of the market.

By Indication, the market is primarily studied into

In 2019, the oncology segment dominated the global gene therapy market by indication with nearly 48.6% of the market share and it is anticipated to grow at 27.6% CAGR during the forecast period 2021-2027.

By Delivery Method, the market is primarily segmented into

Amongst delivery method, In vivo accounted for the largest share and is expected to grow at 28.6% CAGR during the forecast period 2021-2027. In 2019, the ex vivo segment accounted for a revenue share of 12.5%.

Gene therapy Market Geographical Segmentation Includes:

Based on the estimation, the North America region dominated the gene therapy market with almost US$ 1.7 billion revenue in 2019. At the same time, the Asia-Pacific region is expected to grow remarkably with a CAGR of 28.7% over the forecast period on account of owing increasing government initiative to improve healthcare infrastructure and rise in healthcare expenditure and surging cancer incidence rate in the region.

Ask for Report Customization @ https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

The major players targeting the market includes

Competitive Landscape

The degree of competition among prominent global companies has been elaborated by analyzing several leading key players operating worldwide. The specialist team of research analysts sheds light on various traits such as global market competition, market share, most recent industry advancements, innovative product launches, partnerships, mergers, or acquisitions by leading companies in the gene therapy market. The leading players have been analyzed by using research methodologies for getting insight views on global competition.

Key questions resolved through this analytical market research report include:

We understand the requirement of different businesses, regions, and countries, we offer customized reports as per your requirements of business nature and geography. Please let us know If you have any custom needs.

For more informative information, please visit us @ https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

Contact:UnivDatos Market Insights Pvt. Ltd. 4th & 5th Floor, C-80B, Sector 8, Noida, Pin code- 201301, UP, India Ph: +91 7838604911 Email: [emailprotected]

Logo : https://mma.prnewswire.com/media/1225049/UnivDatos_Logo.jpg

SOURCE UnivDatos Market Insights

Visit link:
Gene Therapy Market to Reach US$ 20.9 Billion by 2027, Globally |CAGR: 29.7%|UnivDatos Market Insights - PR Newswire India

Read More...

Delay aging and extend our lifespans? Gene therapy might be able to do that – Genetic Literacy Project

February 3rd, 2021 8:53 pm

How many aging-promoting genes are there in the human genome? What are the molecular mechanisms by which these genes regulate aging? Can gene therapy alleviate individual aging? Recently, researchers from the Chinese Academy of Sciences have shed new light on the regulation of aging.

In this study, the researchers conducted genome-wide CRISPR/Cas9-based screens in human premature aging stem cells and identified more than 100 candidate senescence-promoting genes. They further verified the effectiveness of inactivating each of the top 50 candidate genes in promoting cellular rejuvenation using targeted sgRNAs.

Among them, KAT7 encoding a histone acetyltransferase was identified as one of the top targets in alleviating cellular senescence. It increased in human mesenchymal precursor cells during physiological and pathological aging. KAT7 depletion attenuated cellular senescence whereas KAT7 overexpression accelerated cellular senescence.

Altogether, this study has successfully expanded the list of human senescence-promoting genes using CRISPR/Cas9 genome-wide screen and conceptually demonstrated that gene therapy based on single-factor inactivation is able to delay individual aging. This study not only deepens our understanding of aging mechanism but also provides new potential targets for aging interventions.

Read the original post

Originally posted here:
Delay aging and extend our lifespans? Gene therapy might be able to do that - Genetic Literacy Project

Read More...

Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease – BioSpace

February 3rd, 2021 8:53 pm

First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006

Enrollment of approximately 20 total study participants is ongoing

PHILADELPHIA, Feb. 01, 2021 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral (AAV) vector gene therapy for late-onset Pompe disease (LOPD), a rare, inherited lysosomal storage disorder.

Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and first step to what we hope will ultimately allow us to bring an innovative gene therapy to these patients, said Gallia G. Levy, M.D., Ph.D., chief medical officer of Spark Therapeutics. We are deeply appreciative of the ongoing collaboration of the Pompe disease community as we continue to enroll participants in this Phase 1/2 study.

The RESOLUTE trial is an open-label Phase 1/2, dose-escalation gene transfer study designed to evaluate the safety, tolerability and efficacy of a single intravenous infusion of investigational SPK-3006, an AAV vector-based gene therapy, developed in collaboration with Genethon, in adults with clinically moderate LOPD currently receiving enzyme replacement therapy. The study is expected to enroll approximately 20 participants receiving the investigational gene therapy in sequential, dose-level cohorts. Additional details are available on ClinicalTrials.gov (NCT04093349).

We are honored to have the first participant dosed in this clinical trial, which we hope will lead us to introduce a novel therapeutic option for patients living with late-onset Pompe disease, said Principal Investigator Tahseen Mozaffar, M.D., University of California Irvine Health.

The International Pompe Association has been proud to collaborate with Spark Therapeutics to enhance the Pompe disease communitys understanding of gene therapy research, said Tiffany House, International Pompe Association Board Chairman. We look forward to the progress in the Phase 1/2 RESOLUTE trial, as well as the ongoing work aimed at developing gene therapies that have the potential to help individuals living with genetic diseases.

Pompe disease is a rare, inherited lysosomal storage disorder. It is a progressive, often life-limiting disease caused by the buildup of a complex sugar, glycogen, in the bodys cells. Mutations in the gene encoding acid alpha-glucosidase (GAA) result in deficiencies of the GAA enzyme and limit the breakdown of glycogen. For patients living with LOPD, the respiratory system, locomotion and maintenance of gait are the most critically impacted. These symptoms commonly result in patients becoming wheelchair bound and requiring respiratory support, which may result in reduced life-expectancy.

About SPK-3006 for Pompe diseaseSPK-3006is an investigational liver-directed AAV gene therapy for the potential treatment of late-onset Pompe disease (LOPD).SPK-3006has been engineered to produce a modified enzyme (secretable GAA) that is produced by the liver, which may result in sustained GAA plasma levels and could potentially provide greater uptake in muscle tissue. The transgene integrates technologies designed at and licensed from Genethon, where the in-vivo proof of concept in pre-clinical models was demonstrated. Spark Therapeutics retains global commercialization rights toSPK-3006.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanocommunications@sparktx.com(215) 294-9942

Read more here:
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace

Read More...

Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult – PharmiWeb.com

February 3rd, 2021 8:52 pm

Nottingham, UK 2nd February 2021 Life Science Newswire Albumedix Ltd. (Albumedix), an enabler of advanced therapies and the world leader in recombinant human albumin (rAlb), announced today that they have entered into a collaboration agreement with the Cell and Gene Therapy Catapult (CGT Catapult) to investigate the use of Albumedix proprietary albumin-based solutions for advanced therapy applications, including viral vectors manufacturing.

This agreement reflects the continued efforts of Albumedix to engage with the industry and expand upon its knowledge in the field, and the CGT Catapults mission to drive the growth of the UK cell and gene therapy industry by helping cell and gene therapy organisations across the world translate early-stage research into commercially viable and investable therapies.

Albumedix Chief Executive Officer; Jonas Skjdt Mller commented on the collaboration:

With a mission to empower excellence in advanced therapies, we are committed to continuously playing an integral part in enabling our customers to advance in a fast-moving industry. For us to do so, we continuously look at other industry leaders to establish collaborations. Continuing to learn from each other allows innovation in the market to advance, and Albumedix to support our customers with in-depth knowledge of how rAlb can uniquely benefit their therapies. Cell and Gene Therapy Catapult is the ideal partner; located in our own backyard here in the UK and with incredible skills, knowledge and drive to advance the cell and gene therapy industry we are excited about this collaboration.

Matthew Durdy, Chief Executive Officer at Cell and Gene Therapy Catapult commented:

The opportunity to collaborate with a leading company like Albumedix Ltd in order to assess and drive the potential of their latest technology is something that we embrace. The prospect of improving manufacturing of viral vectors such as Adeno-associated virus (AAV) through this exciting technology is something which could significantly benefit and advance the wider cell and gene therapy field.

Activities under this agreement will be carried out both at the CGT Catapult facility in Braintree and at Albumedix new R&D center, with state-of-the-art laboratories specifically designed for the process optimization, characterization and formulation development of advanced therapies.

Get in touch with Albumedix today by emailing communications@albumedix.com to learn more about their Recombumin range of world leading recombinant human albumin products. Reach out to Cell and Gene Therapy Catapult by emailing communications@ct.catapult.org.uk to learn more about how they can help your organisation to translate early stage research into commercially viable and investable therapies.

About Albumedix Dedicated to Better Health

Albumedix is a science-driven, life-science company focused on enabling the creation of superior biopharmaceuticals utilizing our recombinant human albumin products. We believe in empowering excellence to enable advanced therapies and facilitate otherwise unstable drug candidates reach patients worldwide. We are proud to be recognized as the world leader in recombinant human albumin with products and technologies used in clinical and marketed drugs by pharmaceutical and medical device companies worldwide. Headquartered in Nottingham, England with more than 100 people all committed to improving patient quality of life. We are just as passionate about albumin and albumin-enabled therapies today as we were when we started more than 35 years ago. For more information, please reach out to Albumedix at communications@albumedix.com or visit http://www.albumedix.com

About Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

See original here:
Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult - PharmiWeb.com

Read More...

Method may improve safety of gene therapies targeting the brain – Spectrum

February 3rd, 2021 8:52 pm

Gene silencer: Monkeys injected with a modified virus that includes the binding sequence of miRNA183 (bottom) express significantly lower levels of a gene delivered for gene therapy than do those injected with an unmodified virus, with or without steroid treatment (top).

A novel method for delivering gene therapies to the brain and spinal cord reduces nerve damage in primates, a new study shows. The approach could improve the safety of gene therapies under development for conditions related to autism, such as Angelman syndrome, Rett syndrome and fragile X syndrome.

Gene therapy typically involves replacing or repairing a faulty gene with a functional version, using a harmless adeno-associated virus (AAV). In primates, however, AAV-based gene therapies can damage the axons neuronal projections that transmit signals to other cells of neurons in the dorsal root ganglion, a bundle of nerves close to the spinal cord that relay information to the brain. In severe cases, degeneration in these cells leads to poor motor coordination.

Cell death in the dorsal root ganglion is associated with high levels of expression of the artificially introduced gene, the new work shows. To prevent it, the researchers devised a way to limit this gene expression only in the dorsal root ganglion nerves and not where it is needed.

The method takes advantage of short regulatory RNAs, known as microRNAs, that dampen gene expression by binding to messenger RNA, the intermediary between genes and proteins. The team identified four microRNAs miRNA96, miRNA145, miRNA182 and miRNA183 that are mainly expressed in the dorsal root ganglion. For each microRNA, they added its binding sequence to an AAV carrying the gene for a green fluorescent protein and injected it into mice. After 21 days, they imaged tissue slices from the spinal cord and various organs, including the brain, to gauge levels of the protein.

Viruses carrying the binding sequence of miRNA183 most effectively prevented expression of the green protein in the dorsal root ganglion, imaged tissue slices revealed. The findings were published in November in Science Translational Medicine.

Next, the team tested the viruses in macaques. In one experiment, they injected two animals with a virus carrying only the gene for the fluorescent protein, and another four with a version carrying miRNA183s binding sequence.

The researchers also made a virus carrying hIDUA, a gene that codes for an enzyme lacking in people with the condition mucopolysaccharidosis. They injected this virus into six macaques, half of which also received steroids to lower immune responses, a common practice in gene therapy trials. Three additional macaques received a modified version of the hIDUA virus that included the miRNA183 binding sequence.

After 14, 60 or 90 days, the team examined tissue slices from the animals spinal cord, brain and other organs. Adding miRNA183s binding sequence to the virus significantly prevented expression of either the fluorescent protein or hIDUA in the dorsal root ganglion, the researchers report. Elsewhere in the body, however, the genes expression levels were unaltered, or even increased.

The modified viruses that included miRNA183s binding sequence also caused less toxicity: Macaques injected with these versions had few, if any, damaging lesions in the dorsal root ganglion. By contrast, the steroid treatment did not reduce toxicity, suggesting that the immune response does not explain the damage.

The microRNA technique could be used to mitigate toxicity for a variety of gene therapies that target the central nervous system, the researchers say. It may also enable scientists to further investigate gene therapies for autism.

See the rest here:
Method may improve safety of gene therapies targeting the brain - Spectrum

Read More...

Prototype Gene Therapy for Long QT Heart Condition Developed – Clinical OMICs News

February 3rd, 2021 8:52 pm

A gene therapy targeting the inherited electrical heart disorder long QT syndrome has been developed by researchers at the Mayo Clinic and has shown early success in lab-based studies.

The therapy involves a two-stage process of silencing the genetic error that causes the electrical dysfunction and then replacing it with a functional protein using a short hairpin RNA vector.

As reported in the journal Circulation, the research is still at an early stage, but shows promise in a lab-based cardiac cell model.

Although the first gene therapy trials happened more than 30 years ago, several deaths and adverse effects from these original therapies, combined with sometimes limited efficacy, led to delays in further research and development in this area.

However, things have changed over the last few years. Since the eye gene therapy Luxturna was approved by the FDA in 2017, more than double the number of new therapies for different genetic diseases have gone into development than before and there have been notable successes in developing therapies for various retinal, neurological and immune disorders.

The possibility of developing gene therapies for heart disease has not been explored in depth until recently and is still in its early stages. More widespread heart diseases with polygenic causes are complicated to target with gene therapies and incur high costs. But many rarer arrythmias, such as long QT syndrome, have a genetic cause and are therefore potential gene therapy targets.

Gene therapy is an emerging area of interest for treating a variety of genetic heart diseases in general and long QT syndrome in particular, says Michael Ackerman, M.D. Ph.D., a Mayo Clinic cardiologist and director of Mayo Clinics Windland Smith Rice Comprehensive Sudden Cardiac Death Program, who led the research.

Long QT syndrome is caused by an electrical heart problem that can cause irregular heartbeat, and under conditions of stress, or exertion, can cause the heart to stop beating suddenly. It occurs in approximately 1 in 7,000 people and most often has a genetic cause, although in some cases it can be acquired as a side effect of certain medications or as a result of other illnesses.

Ackerman and team designed and developed the first suppression and replacement KCNQ1 gene therapy approach for the potential treatment of patients with type 1 long QT syndrome. The KCNQ1 protein is involved in potassium channel function and mutations in this gene are common in people with long QT syndrome.

The therapy designed by the Mayo Clinic team involved cloning two short hairpin RNAs one for silencing purposes and one to replace the faulty protein into a single gene therapy construct.

When tested in beating cardiac cells from long QT patients and controls in the lab, the therapy successfully normalized the electrical function of the long QT cells, providing proof of principle to continue development of the therapy.

If the therapeutic efficacy of this disease-in-the-dish gene therapy trial with KCNQ1 can be replicated in a nonhuman, animal model of long QT syndrome, then suppression-replacement gene therapy may be a promising strategy for long QT syndrome in general and in theory almost any sudden death-predisposing autosomal dominant genetic heart disease, says Ackerman.

Of course, we still have a long way to go from nearly curing a patients heart cells in the dish to effectively treating the whole person. Nevertheless, we are excited by this first critical milestone and look forward to the next step.

Read the rest here:
Prototype Gene Therapy for Long QT Heart Condition Developed - Clinical OMICs News

Read More...

Page 229«..1020..228229230231..240250..»


2025 © StemCell Therapy is proudly powered by WordPress
Entries (RSS) Comments (RSS) | Violinesth by Patrick