StemCell Therapy

HAMBURG, GERMANY / ACCESSWIRE / April 6, 2020 / Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) today announced that the Company has established a dedicated site for research and development of gene therapy-based projects: Evotec Gene Therapy (Evotec GT) which will start operations with a strong team of gene therapy experts at an R&D site in Orth/Donau, Austria.

Evotec GT is an integral part of Evotecs integrated drug discovery platform and complements the Companys existing expertise. This strategic addition marks an important step towards Evotecs long-term vision of becoming a fully modality-agnostic drug discovery and development partnership company.

The team in Austria have worked together for many years and applied their research within gene therapy to different gene therapy-related technologies as well as various indications. The scientists have deep expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases. Evotec GTs fully operational site will enable the Company to perform dedicated services in the field of gene therapy along the value chain of its customers from Pharma and biotech as well as foundations and academia.

Dr Werner Lanthaler, Chief Executive Officer of Evotec, commented: We are delighted to initiate our new gene therapy platform and step into this field, which perfectly fits into our business strategy going forward. In recent years, precision medicines based on cell and gene therapies have emerged and are predicted to grow significantly. Gene therapy is a promising approach in the development of genetic medicines for patients, especially for inherited and rare diseases. Finding the best candidate agnostic of modality for any given disease biology will ultimately bring forward the best medicine for patients.

Dr Friedrich Scheiflinger, EVP Head of Gene Therapy at Evotec, said: We are proud to join the growing Evotec team to add the highly promising modality of gene therapy to drug discovery projects. Our team has performed research in the field across different technologies and therapeutic areas for many years and we look forward to leveraging our expertise as part of the truly impressive, modality-agnostic Evotec platform.

About Gene Therapy

Gene therapy is a technique that modifies a persons genes to treat or prevent disease by introduction, removal or editing of genetic material, specifically DNA or RNA, within the cells of a patient. Gene therapies aim to replace a disease-causing gene with a healthy copy, inactivate a disease-causing gene, introduce a new or modified gene or interfere on an expression-regulatory level to support treatment of a disease. Through this modification of gene expression, gene therapies can increase levels of disease-fighting proteins or reduce levels of disease-causing proteins within the cell. Since direct insertion of genes into cells is still very inefficient, gene delivery is facilitated by vehicles which are most often of viral origin. The structure of these viral vectors has been modified to accommodate for the therapeutic gene and to render the vector non-infectious. Depending on the indication and the affected tissue, the technique can be either applied ex-vivo or in-vivo, i.e. with or without removing the cells from the patients body for the therapeutic procedure.

According to various analyst reports, the gene therapy market was valued at approx. $ 500 m in 2018 and the market is expected to reach > $ 5 bn by 2025 with an impressive CAGR of ~34% over the forecast period. Furthermore, rapid and significant progress in the molecular and cellular biology arena, driven by technological advancements in genomics and gene-editing tools, has contributed to an increasing number of approved gene therapies as well as an expanding pipeline. According to the Alliance for Regenerative Medicine (ARM), by the end of the second quarter of 2019, there were more than 700 clinical trials ongoing globally.[1]

ABOUT EVOTEC SE Evotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,000 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industrys need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and womens health. On this basis, Evotec has built a broad and deep pipeline of approx. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to http://www.evotec.com and follow us on Twitter @Evotec.

FORWARD LOOKING STATEMENTS Information set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

Contact Evotec SE:

Gabriele Hansen, SVP Corporate Communications, Marketing & Investor Relations, Phone: +49.(0)40.56081-255,

[1] Sources: https://www.grandviewresearch.com/industry-analysis/gene-therapy-market; https://www.marketwatch.com/press-release/at-339-cagr-gene-therapy-market-size-to-surpass-usd-518-billion-by-2025-2019-09-16; https://www.prnewswire.com/news-releases/gene-therapy-market-to-garner-6-21-bn-globally-by-2026-at-34-8-cagr-says-allied-market-research-300975194.html

SOURCE: Evotec AG via EQS Newswire

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Evotec Expands into Gene Therapy - Associated Press

Evotec has allied with Takeda to expand into gene therapy research. The move sees Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.

Takeda acquired a gene therapy center in Orth an der Donau, Austria, through its acquisition of Shire, which picked up the site two years earlier in its takeover of Baxalta. Throughout the series of changes in ownership, which began when Baxter spun out Baxalta in 2014, a team at the site has worked on gene therapies.

Now, Evotec is set to start playing a role in those efforts. The German drug discovery shop has set up a gene therapy unit, Evotec GT, staffed by a team of more than 20 scientists in Orth an der Donau who have worked together for many years.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

Evotec disclosed news of the move into gene therapies alongside details of a multiyear partnership with Takeda. The deal, which features an undisclosed upfront fee and other payments over time, tasks Evotec with applying its new gene therapy capabilities and broader drug discovery platform to Takedas cancer, rare disease, neuroscience and gastroenterology programs.

Neither Evotec nor Takeda referred directly to a transfer of employees in the statement to disclose the deal. However, Evotec did reveal that Friedrich Scheiflinger is leading its gene therapy unit. Until recently, Scheiflinger headed up drug discovery for Takeda in Austria, with a particular focus on gene therapies. In light of Evotecs comments about its new gene therapy team having worked together for years, it is likely that other gene therapy researchers made the move from Takeda with Scheiflinger.

The agreement gives Evotec a beachhead in the fast-growing gene therapy sector. In explaining(PDF) the rationale for moving into the space, Evotec expressed a desire to be "modality agnostic" and develop wholly and co-owned candidates.

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Evotec allies with Takeda to move into gene therapy R&D - FierceBiotech

German biotech giant Evotec has moved into gene therapy and partnered with Takeda to develop new therapies in oncology, rare diseases, neuroscience, and gastroenterology.

The two companies did not reveal financial details of the collaboration, but did say that it will be a multi-year alliance that adds to Evotecs existing deal with the big pharma that stretches back more than 10 years. The four therapy areas they will concentrate on align with Takedas core therapeutic focus.

The alliance with Takeda gives us a head start, Evotec CEO Werner Lanthaler told me. Takeda has strong experience in gene therapy and we are proud that they have chosen us as a partner.

Evotec also announced it will be opening a 20-person R&D site in Austria, in Orth an der Donau, to work on its gene therapies. While this will be run independently from the Takeda deal, according to Lanthaler, he said the discussions around the collaboration happened at the same time as the gene therapy site was being set up.

The site known as Evotec GT will be led by Friedrich Scheiflinger, who worked for Takeda before moving to Evotec. According to a statement from Evotec, the team has worked together for many years and as well as a focus on gene therapy techniques, has expertise in virology and blood disorders, as well as metabolic and muscle diseases.

Gene therapy has undergone a revolution in recent years and has changed dramatically since the 90s. With approvals of therapies such as the eye gene therapy Luxturna in 2018, many companies are now investing in and working to develop similar therapies.

One reason Evotec cites for moving into this area is the potential for rapid market growth. At present, there are only a few gene therapies on the market, but there are many in development. The value of the market is expected to reach 4.6B by 2025, a dramatic increase from 459M in 2018.

Evotec is also working on various other advanced therapies, including cell therapy with its EVOcells platform. The company announced last week that it made a licensing and investment agreement with Canadian biotech panCELLa, a company modifying cell therapies to evade the immune system and prevent rejection. Evotec additionally has a PanOmics platform that combines transcriptomics and proteomics data to find new drug targets.

After expanding our biologics capabilities through the acquisition of Just Evotec Biologics in 2019, Evotec GT is yet another important piece to complete the multimodality puzzle, explained Lathaler.

The timing was ideal, as Evotec GT brings our existing expertise in the gene therapy field to a new level. It also has close technological ties especially to our induced pluripotent stem cells and PanOmics platforms, so these platforms can grow and gain traction together.

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Evotec Makes Move into Gene Therapy with Takeda... - Labiotech.eu

German drug discovery company Evotec which has a thriving rolodex of biopharma partners such as Bayer, Boehringer Ingelheim, Novartis, Novo Nordisk, Pfizer, Sanofi, and Takeda is now venturing into gene therapies.

The company swallowed Seattle-based Just Biotherapeutics, a company focused on reducing the cost of manufacturing protein therapies last year. It is now setting up a dedicated R&D site for gene therapies in Austria, in an effort to achieve a modality-agnostic repertoire small molecules, biologics and now gene therapies.

The site will be run by a team of twenty researchers led by Friedrich Scheiflinger, who worked for Baxalta, Shire and Takeda. The scientists have expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases.

In recent years, precision medicines based on cell and gene therapies have emerged and are predicted to grow significantly. Gene therapy is a promising approach to the development of genetic medicines for patients, especially for inherited and rare diseases, said Evotec chief Werner Lanthaler in a statement.

Cell and gene therapies are not easy to develop and are prohibitively expensive to manufacture.A combined $2 billion has been set aside by nearly a dozen drugmakers including Pfizer and Novartis for gene therapy manufacturing since 2018, according to an analysis by Reuters in November.

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Small molecules, biologics and now gene therapies: Germany's Evotec adds another feather to its R&D cap - Endpoints News

Base editors, which convert one nucleotide to another without a double-strand DNA break, have the potential to treat diseases caused by mutant genes. One drawback, though, is that the DNA that encodes CRISPR base editors is longtoo long to fit in the adeno-associated viruses (AAVs) most commonly used for gene therapy. In a study published in Molecular Therapy on January 13, researchers split the DNA encoding a base editor into two AAV vectors and injected them into a mouse model of inherited amyotrophic lateral sclerosis (ALS). The strategy disabled the disease-causing gene, improving the animals symptoms and prolonging their lives.

Wed like to be able to make gene editing tools that can fit inside an AAV vector. Unfortunately, some of the tools are so big that they cant fit inside, so in this study, they were able to come up with a solution to that by using a split protein, says David Segal, a biochemist at the University of California, Davis, who was not involved in the work. Its not the first time that that system has been used, but its the first time its been applied to this kind of base editor.

Pablo Perez-Pinera, a bioengineer at University of Illinois at Urbana-Champaign, and colleagues developed a strategy to split the base editor into two chunks. In a study published in 2019, they generated two different AAV vectors, each containing a portion of coding DNA for an adenine-to-thymine base editor. They also included sequences encoding so-called inteinsshort peptides that when they are expressed within proteins stick together and cleave themselves out, a bit like introns in RNA. The researchers built the inteins into the vectors such that when the inteins produced by the two vectors dimerized, bringing the two base editor parts together, and then excised themselves, they left behind a full-length, functional base editor.

When Perez-Pinera told Thomas Gaj, also a bioengineer at the University of Illinois at Urbana-Champaign, about the strategy, Gaj tells The Scientist,they immediately set out to test it in a mouse model of ALS. The transgenic mice have about 25 copies of the human gene, superoxide dismutase 1(SOD1), with mutations that cause ALS in people. The animals display motor neuron loss and muscle atrophy, plus their neurons accumulate inclusionsdense spots in the gray and white matter of their spinal cords that include SOD1 proteinbefore dying at about four months of age on average. The symptoms and life expectancy in the 20 percent of ALS patients with mutations in SOD1vary based on which mutation they have, but most have muscle weakness and motor neuron death, as well as inclusions containing SOD1 protein.

Instead of using the adenine-to-thymine base editor, the researchers developed a cytosine-to-thymine converter using the coding sequence of Streptococcus pyogenes Cas9 and a guide RNA that targets both wild type and mutant human SOD1 to create an early stop codon. This doesnt affect the mouse SOD1. In human cells, the split base editor seemed to be even more efficient than when the editor was transfected at full length, hitting about 29 percent of the target sites, compared to the full-length editors 19 percent.

Next the authors packaged their split base editor into two AAV backbones and injected them or a control AAV into the animals lumbar cerebrospinal fluid when they were around two months old. The vectors ended up primarily in astrocytes, as well as in neurons and microglia. While the researchers didnt see a difference in symptom onset at around three months, the mice that received the base editor maintained their weight and lived about 10 percent longer than controls. The treated mice also had fewer SOD1-positive inclusions and healthier motor neurons.

In this cross section of the spinal cord of a mouse model of amyotrophic lateral sclerosis (ALS), researchers delivered a CRISPR base editing system (yellow) to astrocytes (red) in order to disable the expression of a mutant gene and reduce symptoms.

Colin Lim, University of Illinois

Using base editors to disable the mutant SOD1 gene in astrocytes (a cell type that normally supports healthy nervous system function but in SOD1-ALS exerts toxicity onto motor neurons) led to a marked slowing in disease progression, Gaj writes in an email to The Scientist. Since many persons with ALS are diagnosed following the onset of symptoms, pre-clinical strategies that can meaningfully slow the disease are especially important and should be further studied.

This is a good indication that base editing actually can be used to treat ALS, says Baisong Lu, a gene therapy researcher at Wake Forest School of Medicine who did not participate in the work. He cautions that off-target effectsthe base editor can edit both DNA and RNAand how long the AAV delivery method lasts are both in need of more work before this technique would be safe for people.

The dual AAV strategy could also be expensive, says Mimoun Azzouz, a neuroscientist at the University of Sheffield in the United Kingdom. Thinking about the clinical development and marketing and the commercialization of this product, you need to manufacture two viruses, and you need to assess these two viruses for safety, so the cost can be extremely high.

Despite the challenges, the strategy shows promise for translation to humans, Perez-Pinera writes in an email to The Scientist.AAVs are already approved by the Food and Drug Administration for gene therapy, he explains. Plus, using a humanized model of the diseasea mouse that contains the human sequence of the target genemeans that the method validated in mouse models can be translated to people without adapting them to target a different sequence. People who develop ALS due to a mutation in SOD1also have one good copy of the gene, just like the mice, which have a functioning mouse copy.

We injected animal models shortly before disease onset. While injecting the animals earlier could improve the outcome of the disease as demonstrated in other studies, the reality is that ALS is not typically diagnosed until the patient experiences symptoms. Our study predicts what can be expected from treating a patient recently diagnosed with the disease, Perez-Pinera writes.

We still have some distance to travel before the results in our current study can benefit ALS patients, Gaj acknowledges. The researchers are working on minimizing off target effects and on developing new delivery methods that could improve efficacy. We still have a number of important questions to answer and technological hurdles to address before we begin thinking about clinical translation.

C.K.W. Lim et al., Treatment of a mouse model of ALS by in vivo base editing,Molecular Therapy,doi:10.1016/ j.ymthe.2020.01.005, 2020.

Abby Olena is a freelance journalist based in Alabama. Find her on Twitter@abbyolena.

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Symptoms in ALS Mouse Model Improve with CRISPR Base Editing - The Scientist

Niche players in the global gene therapy market must leverage greater government expenditure and upgrade their existing infrastructure along with expanding their gene therapy centers for sustaining their market hegemony.

ROCKVILLE, MD / ACCESSWIRE / April 8, 2020 / Global gene therapy market is poised for robust growth with net revenue pool set to exceed approximately US$ 5 Bn by 2026 end. The market is receiving tailwinds from advancements in synthetic biology. On that premise, the gene therapy market will expand 3X through over the forecast period, projects Fact.MR (2020-2026).

"Certain types of cancer such as Diffuse Large B-cell Lymphoma (DLBCL) and lymphoblastic leukemia are contributing to high mortality rates across the world. Gene therapy is gaining increasing recognition in its immense potential for treating rare diseases. Continued research and development in the area of gene therapy is supporting market growth as well," states Fact.MR.

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Gene Therapy Market - Key Findings

Gene Therapy Market - Key Driving Factors

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Gene Therapy Market - Key Restraints

Impact of COVID-19 on Gene Therapy Market

In view of ongoing onslaught of COVID-19 pandemic, the focus of major healthcare authorities of the world has gravitated towards developing vaccines for the deadly respiratory disease. Gene therapy is one such area of research which could help boost antibodies required to treat patients infected with Coronavirus. For instance, Generation Bio is exploring the potential role of gene therapy in treating COVID-19 patients. Hence, the global gene therapy market will benefit from the outbreak in that market players are rushing to develop multiple therapeutic approaches for SARS-CoV-2. Growing fears of similar Coronavirus outbreaks in the future will continue accelerating the development of gene therapy as well.

Competitive Landscape

Prominent players profiled in this Fact.MR study include, but are not limited to, Orchard Therapeutics Limited, CELGENE CORPORATION, Spark Therapeutics, Inc., Sibiono GeneTech Co. Ltd., Spark Therapeutics Inc., Gilead Sciences Inc., and Novartis AG. Developed regions remain the key focus area of major stakeholders in the global gene therapy market. Existing gene therapy centers are being prioritized by market players in order to utilize the full extent of their resources. Moreover, they are benefitting from success rates associated with gene therapy and faster drug approvals. Gilead Sciences Inc. expanded their gene therapy centers to a total of 90 recently.

About the Report

This 170-page study offers in-depth commentary on the gene therapy market. The study provides compelling insights on the gene therapy market on the basis of product (yescarta, kymriah, luxturna, strimvelis, and gendicine), application (ophthalmology, oncology, Adenosine Deaminase Deficiency- Severe Combined Immunodeficiency) across three regions (The United States, Europe, and Rest of the World).

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Gene Therapy Market Set for 3X Expansion Between 2020 and 2026; COVID-19 Stimulating Development of Multiple Therapeutic Approaches: Fact.MR -...

The genetic code to alllife on Earth, both simple and complex, comes down to four basic letters: A, C,T and G.

Untangling the role thatthese letters play in lifes blueprint has allowed scientists to understandwhat makes everything from bacteria to people the way they are. But as researchershave learned more, they have also sought ways to tinker with this blueprint,bringing ethical dilemmas into the spotlight. The Gene, a two-part PBS documentary from executive producer Ken Burnsairing April 7 and 14, explores the benefits and risks that come withdeciphering lifes code.

The film begins with oneof those ethical challenges. The opening moments describe how biophysicist HeJiankui used the gene-editing tool CRISPR/Cas9 to alter the embryos of twin girls who were born in China in 2018 (SN: 12/17/18). Worldwide, criticscondemned the move, claiming it was irresponsible to change the girls DNA, asexperts dont yet fully understand the consequences.

This moment heraldedthe arrival of a new era, narrator David Costabile says. An era in whichhumans are no longer at the mercy of their genes, but can control and evenchange them.

Headlines and summaries of the latest Science News articles, delivered to your inbox

The story sets the stagefor a prominent theme throughout the documentary: While genetics holdsincredible potential to improve the lives of people with genetic diseases,there are always those who will push science to its ethical limits. But thedriving force in the film is the inquisitive nature of the scientistsdetermined to uncover what makes us human.

The Gene, based on the book of the same name by Siddhartha Mukherjee (SN:12/18/16), one of the documentarys executive producers, highlights many ofthe most famous discoveries in genetics. The film chronicles Gregor Mendels classicpea experiments describing inheritance and how experts ultimately revealed inthe 1940s that DNA a so-called stupid molecule composed of just four chemicalbases, adenine (A), thymine (T),cytosine (C) and guanine (G) is responsible for storing geneticinformation. Historical footage, inBurns typical style, brings to life stories describing the discovery of DNAshelical structure in the 1950s and the success of the Human Genome Project indecoding the human genetic blueprint in 2003.

The film also touches ona few of the ethical violations that came from these discoveries. The eugenicsmovement in both Nazi Germany and the United States in the early 20th century aswell as the story of the first person to die in a clinical trial for genetherapy, in 1999, cast a morbid shadow on the narrative.

Interwoven into thistimeline are personal stories from people who suffer from genetic diseases.These vignettes help viewers grasp the hope new advances can give patients asexperts continue to wrangle with DNA in efforts to make those cures.

In the documentarysfirst installment, which focuses on the early days of genetics, viewers meet a family whose daughter is grappling with arare genetic mutation that causes her nerve cells to die. The family searchesfor a cure alongside geneticist Wendy Chung of Columbia University. The secondpart follows efforts to master the human genome and focuses on AudreyWinkelsas, a molecular biologist at the National Institutes of Health studyingspinal muscular atrophy, a disease she herself has, and a family fighting tosave their son from a severe form of the condition.

For science-interested viewers, the documentary does not disappoint. The Gene covers what seems to be every angle of genetics history from the ancient belief that sperm absorbed mystical vapors to pass traits down to offspring to the discovery of DNAs structure to modern gene editing. But the stories of the scientists and patients invested in overcoming diseases like Huntingtons and cancer make the film all the more captivating.

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The PBS documentary The Gene showcases genetics promise and pitfalls - Science News

A group of Korean researchers said they have confirmed a gene responsible for inherited retinal disorders (IRD) among Koreans.

IRD is a combination of several rare diseases that usually develops at a young age and progresses slowly over the lifetime. The patients gradually lose their sight, and most of them eventually lose their vision entirely due to continuous retinal cell degeneration.

The Seoul National University Bundang Hospital (SNUBH) Department of Ophthalmology and Seoul National University Hospital (SNUH) Department of Laboratory Medicine jointly conducted the study.

Currently, antioxidant therapy, artificial retinal transplantation, and stem cell therapy are being used to treat the disorder regardless of mutations, but the only viable treatment is gene therapy. Even when gene therapy is possible, only less than 1 percent of all IRD patients can be treated with it.

In the West, genetic abnormalities of these retinal diseases have been studied and known well. However, researches on Korean cases are still lacking, and the joint research team tackled the subject to find the causative gene for IRDs with 86 domestic patients, the team said in a news release on Wednesday.

The team studied and identified the gene responsible for the disorders by using the latest technique of gene analysis with the most number of patients who have been reported so far.

The study revealed that only 44 percent of the patients, 38 out of 86, possessed the causal gene for IRDs. Even among the patients with retinitis pigmentosa, the most common disorder among the IRDs, only 41 percent had the causative gene.

The causative genes could be quite diverse even in the same disorder. The patients can find a responsible gene only when they receive genetic counseling very actively and can receive gene counseling, too, the research team explained.

Differences were found in the type and frequency of causal gene mutations between Korean and Western cases. However, there were similarities between those of Korean and other Asian nations, including Japan.

The research and diagnosis environment for IRDs has been very poor until now, and our study has significance as a basic data for diagnosis and treatment for Korean patients with IRDs, SNUH Department of Ophthalmology Professor Woo Se-joon said.

Patients need to receive causal gene tests actively to provide the domestic medical communities with sufficient data, and a list of patients who can be treated. By doing so, clinical trials and new drug development in gene therapy will progress smoothly, he added.

Previously, only a few hospitals could diagnose the causative gene for IRDs and afford to test and treat IRD patients due to the high cost of genetic testing. Recently, however, the chance of diagnosis has increased as more hospitals are conducting genetic tests amid the lowered cost thanks to insurance benefits.

Also, the therapeutic opportunity for IRD patients is likely to get broadened, as the retinal pigment epithelium 65 gene (RPE65) therapy won approval from the U.S. Food and Drug Administration for the first time in the world.

Although we do not have a clear way to prevent IRDs at the moment, the prediction of risk and their early detection are developing through the discovery of family history and causative genes, Professor Woo said. Early diagnosis can prevent impaired vision by gene therapy and vision correction, and the patients will be able to choose appropriate jobs with social activities.

Also taking part in the research team were Professors Joo Kwang-sic and Park Kyu-hyung of SNUBH and Professors Seong Moon-woo and Park Sung-sup of SNUH.

The results of this study were published in the Journal of Korean Medical Science.

shim531@docdocdoc.co.kr

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SNUH team finds causal gene of inherited retinal disorder - Korea Biomedical Review

LOS ANGELES, April 09, 2020 (GLOBE NEWSWIRE) -- Enochian Biosciences, a company focused on gene-modified cellular therapy in infectious disease and cancer,announces that all three submissions to the 23rd Annual Meeting of the ASCGT were accepted for presentation. The Conference will be held virtually May 12-15, 2020.

Dr. Serhat Gumruku, inventor and co-founder, will have two presentations: Hijacking HBV Pol to Induce Apoptosis Specifically in Infected HepatocytesIn Vivo: A Novel Approach for Potential Treatment or Cure; and Increased Engraftment of Gene Modified HSPCs Overexpressing ALDH1 UsingIn VivoChemoselection. Dr. Ramesh Halder, Senior Staff Scientist, will present: Gene Modified CD34+Cells With Increased ALDH1 Expression ConfersIn VitroProtection Against Cyclophosphamide.

Forward-Looking StatementsStatements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties, including but not limited to the success or efficacy of our pipeline. All statements other than historical facts are forward-looking statements, which can be identified by the use of forward-looking terminology such as believes, plans, expects, aims, intends potential, or similar expressions. Actual events or results may differ materially from those projected in any of such statements due to various uncertainties, including as set forth in Enochians most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Enochian undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

Contact: ir@enochianbio.com

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Enochian Biosciences Announces Scientific Presentations at the American Society of Cell and Gene Therapy (ASCGT) Annual Meeting - Yahoo Finance

Global Gene Therapy Market report underlines the specific study of the Biotechnology industry which explains what the market definition, classifications, applications, engagements, and global industry trends are. The market data analyses and evaluated in this Gene Therapy market report makes you achieve the business goals and objectives in preset time frame. It highlights a wide-ranging evaluation of the markets growth prospects and restrictions. This report is very useful to all sizes of business which makes it simpler to take informed decisions regarding different facets of industry. Gene Therapy market report truly acts as a backbone to the business.

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MARKET INTRODUCTION

Gene therapy is introduction of DNA into a patient to treat a genetic disease or a disorder. The newly inserted DNA contains a correcting gene to correct the effects of disease causing mutations. Gene therapy is promising treatment for the genetic diseases and also includes cystic fibrosis and muscular dystrophy. Gene therapy is suitable treatment for the infectious diseases, inherited disease and cancer.

Key Competitors In Market are Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure N.V., AveXis, Vineti, Solid Biosciences., Spark Therapeutics, Inc., CHIMERON BIO, RENOVA THERAPEUTICS, and HORAMA S.A.

MARKET SCOPE

The Global Gene Therapy Market Analysis to 2025 is a specialized and in-depth study of the gene therapy industry with a focus on the global market trend. The report aims to provide an overview of global gene therapy market with detailed market segmentation by cell type, application, and geography. The global gene therapy market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading market players and offers key trends and opportunities in the market.

Market segmentation:

By Cell Type (Somatic Gene Therapy, Germline Gene Therapy);

By Application (Genetic Disorder, Cancer, Neurological Disorder, and Others)

By Geography North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. And 13 countries globally along with current trend and opportunities prevailing in the region.

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Gene Therapy Market 2020: Competitive Landscape and Recent Industry Development Analysis by Sangamo Therapeutics, Inc., bluebird bio, Inc., uniQure...

University of Georgia Honors student Landon Clark is among 396 undergraduates across the nation to be recognized as Barry Goldwater Scholars, earning the highest undergraduate award of its type for the fields of the natural sciences, mathematics and engineering.

Clark, from Leesburg, is a third-year CURO Honors Scholar who is triple majoring in biochemistry and molecular biology, genetics and biology.

Since 1995, 61 UGA students have received the Goldwater Scholarship, which recognizes exceptional sophomores and juniors across the United States.

The University of Georgia is proud of Landon and pleased that he is extending our record of success in this prestigious competition, said President Jere W. Morehead. Already as an undergraduate student, he is helping UGA fulfill our commitment to conduct research that helps people lead longer, healthier lives.

This year, recipients were selected from a field of more than 5,000 college sophomores and juniors who were nominated by 461 academic institutions nationwide. Each awardee will receive up to $7,500 toward the cost of tuition, fees, books, and room and board.

Of this years Goldwater Scholars, 50 are mathematics and computer science majors, 287 are majoring in the natural sciences and 59 are majoring in engineering.

I am thrilled for Landon, who is absolutely one of our very best students, said David S. Williams, associate provost and director of the Honors Program, who serves as the UGA campus faculty representative for the Goldwater Scholarship. As a CURO Honors Scholar, Landon was recruited to come to UGA because of his promise as a researcher. This Goldwater award and recognition proves that his hard work is paying off. I look forward to watching his career continue to blossom in the future.

Clarks long-term plans include pursuing an MD/PhD in immunology, performing translational research on immune diseases using gene and immunotherapy techniques, and teaching at a university. As a translational researcher specializing in immunology, he plans to use gene therapy techniques to treat and cure immune disorders in humans.

Since his first weeks as a UGA freshman in August 2017, Clark has conducted research in the lab of Michael Terns, Distinguished Research Professor of Biochemistry and Molecular Biology. He researches the complex processes of CRISPR-Cas immune systems through a model archaeal organism, Pyrococcus furiosus. Now a junior, he has collaborated on three different projects, co-authored a paper, and mentors other undergraduates in this field.

Last summer, he worked in the translational research lab of H. Trent Spencer, a professor of pediatrics and director of the gene and cell therapy program at Emorys Winship Cancer Institute. There, Clark researched immune diseases and potential gene therapy techniques. In 2017, he also conducted research on sociological variables influencing educational inequality with Dawn Robinson, a professor of sociology at UGA.

Clark is a Crane Leadership Scholar, director of academic outreach for the Student Government Association, director of internal communications for UGA Red Cross, an exam writer for UGA Science Olympiad Outreach, co-president of the Biochemistry Undergraduate Society, co-president of the Omicron Delta Kappa National Honor Society, and treasurer of the Honors Program Student Council. He also volunteers in the neuroscience and cardiology units at Piedmont Athens Regional Medical Center.

Clark has presented his research at 10 conferences. He also studied neurology and cancer biology through UGAs Studies Abroad Program in Cortona, located in Tuscany, Italy.

The scholarship honoring Sen. Barry Goldwater was created to encourage outstanding students to pursue careers in the fields of mathematics, natural sciences and engineering. Since its first award in 1989, the Barry Goldwater Scholarship and Excellence in Education Foundation has bestowed 8,628 scholarships worth more than $68 million.

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UGA's Landon Clark named 2020 Goldwater Scholar - University of Georgia

Detailed market survey on the Global Hemophilia Gene Therapy Market Research Report 2020-2026. It analyses the vital factors of the Hemophilia Gene Therapy market supported present business Strategy, Hemophilia Gene Therapy market demands, business methods utilised by Hemophilia Gene Therapy market players and therefore the future prospects from numerous angles well. Business associatealysis could be a market assessment tool utilized by business and analysts to grasp the quality of an business. Hemophilia Gene Therapy Market report It helps them get a sense of what is happening in an industry, i.e., demand-supply statistics, Hemophilia Gene Therapy Market degree of competition within the industry, Hemophilia Gene Therapy Market competition of the business with different rising industries, future prospects of the business.

NOTE: Hemophilia Gene Therapy reports include the analysis of the impact of COVID-19 on this industry. Our new sample is updated which correspond in new report showing impact of Covid-19 on Industry trends. Also we are offering 20% discount

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The Global Hemophilia Gene Therapy Market report is a fully analyzed and intelligent study of the international industry that focuses on a wide range of significant elements such as market size in terms of value and volume, regional growth analysis, competition and segmentation. It is considered as extraordinary findings that accountable to offer insightful details into some essential attributes related to the global Hemophilia Gene Therapy Market 2020. The detailed investigation of this report has been carried out by the list of skillful researchers and investigators with a deep analysis of current industry trends, availability of distinct opportunities, drivers, openings and limitation that influence the Hemophilia Gene Therapy Market on the global scale.

The Global Hemophilia Gene Therapy market worth about xx billion USD in 2020 and it is expected to reach xx billion USD in 2026 with an average growth rate of x%. United States is the largest production of Hemophilia Gene Therapy Market and consumption region in the world, Europe also play important roles in global Hemophilia Gene Therapy market while China is fastest growing region.

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Geographically, Hemophilia Gene Therapy market report is segmented into several key Regions, with production, consumption, revenue. The major regions involved in Hemophilia Gene Therapy Market are (United States, EU, China, and Japan).

Leading companies reviewed in the Hemophilia Gene Therapy report are:

Spark TherapeuticsFreeline TherapeuticsSangamo TherapeuticsUltragenyxuniQureShire PLCBioMarinBioverativ

Hemophilia Gene Therapy Market Product Type Segmentation As Provided Below:The Hemophilia Gene Therapy Market report is segmented into following categories:

The product segment of the report offers product market information such as demand, supply and market value of the product.

The application of product in terms of USD value is represented in numerical and graphical format for all the major regional markets.The Hemophilia Gene Therapy market report is segmented into Type by following categories;Hemophilia AHemophilia B

The Hemophilia Gene Therapy market report is segmented into Application by following categories;Hemophilia A Gene TherapyHemophilia B Gene Therapy

Reportedly, the massive growth graph in the research and development sectors will be liable to generate plenty of excellent opportunities in the upcoming years. The Hemophilia Gene Therapy market is a valuable resource of insightful information for specific business strategists. Apart from this, it also offers an in-depth summary of the Hemophilia Gene Therapy Market along with growth assessment, revenue share, demand & supply data, historical as well as futuristic amount etc. A group of research analysts offers a detailed description of the value chain and its distributors info. Moreover, the Hemophilia Gene Therapy market study report delivers comprehensive information regarding the global industry that enhances the scope, understanding and application of the same.

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Industry analysis, for an entrepreneur or a company, is a method that helps it to understand its position relative to other participants in the Hemophilia Gene Therapy Market. It helps them to identify both the opportunities and threats coming their way and gives them a strong idea of the present and future scenario of the Hemophilia Gene Therapy industry. The key to extant during this changing business setting is to know the variations between yourself and your competitors within the Hemophilia Gene Therapy Market. The deep research study of Hemophilia Gene Therapy market based on development opportunities, growth limiting factors and feasibility of investment will forecast the Hemophilia Gene Therapy market growth.

Finally, The global research document on the Hemophilia Gene Therapy Market discovers a large set of information regarding the competitive business environment and other substantial components. The prime aim of these major competitors is to focus on improved technologies and newer innovations.

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Research on Hemophilia Gene Therapy Market (impact of COVID-19) 2020-2026 Spark Therapeutics, Freeline Therapeutics, Sangamo Therapeutics, Ultragenyx...

Welcome to the latest edition of our weekly EuroBiotech Report. We start this week with AstraZeneca, which revealed it aims to have a COVID-19-neutralizing antibody in the clinic in the next three to five months. AstraZeneca's compatriot GlaxoSmithKline also shared an update on its COVID-19 activities. GSK added Xiamen Innovax Biotech to the list of organizations it is working with on a COVID-19 vaccine. While those are just two of the many initiatives targeting COVID-19, Wellcome is worried about a funding shortfall. Wellcome wants businesses to commit $8 billion this month to keep programs moving forward quickly. Researchers working outside of COVID-19 are faring worse still, with Cancer Research UK responding to the pandemic with deep cuts. Away from COVID-19, Evotec entered the gene therapy space through a deal with Takeda. And more. Nick Taylor1. AstraZeneca targets summer start for COVID-19 antibody trial

AstraZeneca has outlined its multipronged approach to the development of antibodies against the pandemic SARS-CoV-2 virus. The Big Pharma is applying its own discovery capabilities to the task and helping to evaluate candidates identified by academic partners in China and the U.S.2. GSK allies with Innovax for COVID-19 vaccine R&D project

GlaxoSmithKline has teamed up with Xiamen Innovax Biotech to evaluate a vaccine against the novel coronavirus behind the COVID-19 pandemic. The agreement gives Innovax access to a GSK adjuvant to enhance the immune response triggered by its recombinant protein-based vaccine.3. Wellcome targets $8B raise in weeks to fix COVID-19 funding gap

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

Wellcome is calling on businesses to invest in an $8 billion (7 billion) fund focused on reducing cases of COVID-19 to zero as soon as possible. Almost half the money is earmarked for the development of drugs and vaccines that Wellcome sees as the worlds best exit strategy from the pandemic.4. COVID-19 claims Cancer Research UK funding cuts, forecasting major shortfall

Cancer Research UK (CRUK) said it has been forced to take the difficult decision to make deep funding cuts across its grants and institutes as the fallout from the pandemic continues to affect all areas of biomedical research.

5. Evotec allies with Takeda to move into gene therapy R&D

Evotec has allied with Takeda to expand into gene therapy research. The move sees Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.And more articles of note>>

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EuroBiotech ReportAZ, GSK in COVID-19, $8B funding call, CRUK cuts and Evotec - FierceBiotech

Global Cancer Gene Therapy Market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

The report titled Global Cancer Gene Therapy Market is one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Cancer Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Cancer Gene Therapy market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Cancer Gene Therapy market is carefully analyzed and researched about by the market analysts.

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The Essential Content Covered in the Global Cancer Gene Therapy Market Report:

In terms of region, this research report covers almost all the major regions across the globe such as North America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. While Cancer Gene Therapy Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets. Cancer Gene Therapy Market in South, America region is also expected to grow in near future.

The key players covered in this studyAdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene

Market segment by Type, the product can be split intoOncolytic VirotherapyGene TransferGene-Induced Immunotherapy

Market segment by Application, split intoHospitalsDiagnostics CentersResearch Institutes

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

Make An EnquiryAbout This Report @ https://www.researchmoz.com/enquiry.php?type=E&repid=2081748&source=atm

Key questions answered in the report

*What will be the market size in terms of value and volume in the next five years?

*Which segment is currently leading the market?

*In which region will the market find its highest growth?

*Which players will take the lead in the market?

*What are the key drivers and restraints of the markets growth?

We provide detailed product mapping and analysis of various market scenarios. Our analysts are experts in providing in-depth analysis and breakdown of the business of key market leaders. We keep a close eye on recent developments and follow latest company news related to different players operating in the global Cancer Gene Therapy market. This helps us to deeply analyze companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study that will help you to stay on top of the competition.

Table of Contents

1 Cancer Gene Therapy Market Overview

1.1 Product Overview and Scope of Cancer Gene Therapy

1.2 Cancer Gene Therapy Segment by Type

1.2.1 Global Cancer Gene Therapy Production Growth Rate Comparison by Type 2020 VS 2026

1.3 Cancer Gene Therapy Segment by Application

1.3.1 Cancer Gene Therapy Consumption Comparison by Application: 2020 VS 2026

1.4 Global Cancer Gene Therapy Market by Region

1.4.1 Global Cancer Gene Therapy Market Size Estimates and Forecasts by Region: 2020 VS 2026

1.4.2 North America Estimates and Forecasts (2015-2026)

1.4.3 Europe Estimates and Forecasts (2015-2026)

1.4.4 China Estimates and Forecasts (2015-2026)

1.4.5 Japan Estimates and Forecasts (2015-2026)

1.5 Global Cancer Gene Therapy Growth Prospects

1.5.1 Global Cancer Gene Therapy Revenue Estimates and Forecasts (2015-2026)

1.5.2 Global Cancer Gene Therapy Production Capacity Estimates and Forecasts (2015-2026)

1.5.3 Global Cancer Gene Therapy Production Estimates and Forecasts (2015-2026)

2 Market Competition by Manufacturers

2.1 Global Cancer Gene Therapy Production Capacity Market Share by Manufacturers (2015-2020)

2.2 Global Cancer Gene Therapy Revenue Share by Manufacturers (2015-2020)

2.3 Market Share by Company Type (Tier 1, Tier 2 and Tier 3)

2.4 Global Cancer Gene Therapy Average Price by Manufacturers (2015-2020)

2.5 Manufacturers Cancer Gene Therapy Production Sites, Area Served, Product Types

2.6 Cancer Gene Therapy Market Competitive Situation and Trends

2.6.1 Cancer Gene Therapy Market Concentration Rate

2.6.2 Global Top 3 and Top 5 Players Market Share by Revenue

2.6.3 Mergers & Acquisitions, Expansion

3 Production Capacity by Region

4 Global Cancer Gene Therapy Consumption by Regions

5 Production, Revenue, Price Trend by Type

5.1 Global Cancer Gene Therapy Production Market Share by Type (2015-2020)

5.2 Global Cancer Gene Therapy Revenue Market Share by Type (2015-2020)

5.3 Global Cancer Gene Therapy Price by Type (2015-2020)

5.4 Global Cancer Gene Therapy Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End

6 Global Cancer Gene Therapy Market Analysis by Application

6.1 Global Cancer Gene Therapy Consumption Market Share by Application (2015-2020)

6.2 Global Cancer Gene Therapy Consumption Growth Rate by Application (2015-2020)

Read More..

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Cancer Gene Therapy Market Scope Analysis 2019-2025 - Science In Me

Located on the Tufts University Medford, MA campus, this new donor center will enable delivery of fresh blood, leukopaks and buffy coats for COVID-19, cell and gene therapy research within hours of collection

WESTBURY, NY - Apr 6, 2020 - BioIVT, a leading provider of research models and services for drug and diagnostic development, today announced the opening of its new blood donor center on the Tufts University campus in Medford, MA to support academic and pharmaceutical researchers involved in COVID-19, cell and gene therapy research.

BioIVT wants to play a leading role in supporting COVID-19 research efforts and blood donations are a vital resource for the research and development of new therapies, vaccines, and diagnostics. We have many years experience developing blood products, including blood-derived immune cells for cell and gene therapy research, and we want to make that expertise count, said BioIVT CEO Jeff Gatz. Researchers recognize and appreciate BioIVTs rapid response and commitment to high quality, fresh blood products and this new donor center will allow us to offer those attributes and services to additional US clients.

BioIVTs new Boston blood donor center is its seventh. The company has similar facilities located in California, Tennessee and Pennsylvania to serve US clients and in London, UK for EU-based clients.

While the initial focus at our Boston donor center will be on delivering fresh blood, leukopaks and buffy coats within hours of collection, we plan to add more capabilities and donors over time, said Jeff Widdoss, Vice President of Donor Center Operations at BioIVT.

Leukopaks, which contain concentrated white blood cells, are used to help identify promising new drug candidates, assess toxicity levels, and conduct stem cell and gene therapy research. They are particularly useful for researchers who need to obtain large numbers of leukocytes from a single donor.

BioIVT blood products can be supplied with specific clinical data, such as the donor age, ethnicity, gender, BMI and smoking status. Its leukopaks are also human leukocyte antigen (HLA), FC receptor and cytomegalovirus typed. HLA typing is used to match patients and donors for bone marrow or cord blood transplants. FC receptors play an important role in antibody-dependent immune responses.

COVID-19-related Precautions

Blood donor centers are considered essential businesses and will remain open during the COVID-19 quarantine. BioIVT is taking additional safety measures to protect both blood donors and its staff during this difficult time. It has instituted several social distancing measures, including increasing the space between chairs in the waiting room and between donor beds, and limiting the entrance of non-essential personnel. The screening rooms are disinfected between donors and all areas of the center continue to be cleaned at regular intervals.

As soon as each blood donor signs their informed consent form, their temperature is taken. If they have a fever, their appointment is postponed, and they are referred to their physician. Any donor who develops COVID-19 symptoms after donating blood is required to inform the center immediately.

All BioIVT blood collections are conducted under institutional review board (IRB) oversight and according to US Food and Drug Administration (FDA) regulations and American Association of Blood Banks (AABB) guidelines.

Those who would like to donate blood at BioIVTs new Boston-area donor center should call 1-833-GO-4-CURE or visit http://www.biospecialty.com to make an appointment.

Further information about the products available from BioIVTs new donor center can be found at https://info.bioivt.com/ma-donor-ctr-req.

About BioIVT

BioIVT is a leading global provider of research models and value-added research services for drug discovery and development. We specialize in control and disease-state biospecimens including human and animal tissues, cell products, blood and other biofluids. Our unmatched portfolio of clinical specimens directly supports precision medicine research and the effort to improve patient outcomes by coupling comprehensive clinical data with donor samples. Our PHASEZERO Research Services team works collaboratively with clients to provide target and biomarker validation, phenotypic assays to characterize novel therapeutics, clinical assay development and in vitro hepatic modeling solutions. And as the premier supplier of hepatic products, including hepatocytes and subcellular fractions, BioIVT enables scientists to better understand the pharmacokinetics and drug metabolism of newly-discovered compounds and their effects on disease processes. By combining our technical expertise, exceptional customer service, and unparalleled access to biological specimens, BioIVT serves the research community as a trusted partner in elevating science. For more information, please visit http://www.bioivt.com or follow the company on Twitter @BioIVT.

Excerpt from:
BioIVT Opens New Blood Donor Center to Support Boston-area Research into COVID-19 Therapies, Vaccines and Diagnostics - Bio-IT World

While the world is working to slow the COVID-19 pandemic by social isolation and quarantine, numerous companies globally are working to develop a vaccine against the virus. The most likely timelinesat bestfor a viable vaccine against SARS-CoV-2, the coronavirus causing COVID-19, are 12 to 18 months. And thats if everything goes right.

As a recent article in Bloomberg Businessweek notes, For that to happen in the next year or so, an almost equally implausible set of circumstances has to occur: flawless scientific execution, breakneck trials and a military-style manufacturing mobilization unlike any the pharmaceutical industry has put in place before.

Typically, it takes 10 to 15 years of deliberate and careful work to develop a commercial vaccineand as both the common cold and HIV have demonstrated, sometimes its not possible even then. The infrastructure for the seasonal flu vaccines that come out every year has been in place for decades.

One of the companies that is gaining a lot of attention is Cambridge, Massachusetts-based Moderna. They have a head-start and are using a novel technology to develop their COVID-19 vaccine, mRNA-1273, which is already in clinical trials. mRNA-1273 is a mRNA vaccine that encodes for a prefusion stabilized form of the Spike (S) protein. Whats different about the vaccine is that it is almost like a form of gene therapyit codes for the genetic sequence for the spike protein, and when injected into the body, causes the patients own cells to produce the protein (not the virus), which triggers an immune reaction that will prep itself to battle the virus.

The technology is interesting, but unproven. There are no approved RNA therapies. And Moderna has never brought a product to the market before.

One thing thats of note is how early a start Moderna got on the vaccine. On January 11, 2020, Chinese researchers posted the genetic sequence of the coronavirus, well before most of the world was paying any attention. Moderna scientists had been working on a novel vaccine for a different coronavirus disease and jumped on this sequence and began working on a vaccine against it.

Bloomberg writes, By late February, when President Trump was still downplaying the risk of coronavirus, Modernas scientists had already delivered the first batch of candidate vaccines to researchers at the U.S. National institutes of Health. When the coronavirus was starting to explode in the U.S. in mid-March, the first healthy patient received a dose in a small, government-sponsored safety trial.

Moderna may be the leader, but they are not the only company employing this technology. Pfizer and a few others are as well. Pfizer is working with a German company, BioNTech, hoping to get their experimental RNA vaccine into human trials sometime this month.

Johnson & Johnson is using technology it employed for its experimental Zika and Ebola vaccine. Paris-based Sanofi is modifying technology it uses for its flu shots. There are more than two dozen companies working on coronavirus vaccines that are already in early-stage testing.

There is no precedent for the speed at which we are moving, said Clement Lewin, an associate vice president at Sanofi. I cant think of a parallel, in his thirty years of vaccine work.

The RNA vaccine technology, despite the apparent speed with which Moderna, Pfizer and BioNTech are moving, has some risks. Not as much is known about its efficacy. Early-stage data suggests it should be safe, but there are concerns about unwanted immune responses. And all vaccines run a risk of what is called disease enhancement, where the immune system responds in an unusual way, which can cause the illness to be worse.

It's possible that any vaccines just wont be effective, or the side effects will be such that they wont be considered safe for use.

Scale-up is also a major roadblock. As of April 7, there are 1,362,936 confirmed cases of COVID-19 around the globe. But there are another 8 billion possible people who would possibly benefit from a vaccine.

Under a scenario where Modernas vaccine turns out to be effective and safe, no one has experience in commercial production of RNA vaccines, let alone in the type of scale necessary. The company indicates it has the manufacturing capability to produce millions of doses of the vaccine per month at a factory that was already scaling up for a different vaccine. They are apparently also in discussions with possible partners. But millions is a very long way from producing vaccines for billions of peopleor even a few hundred million people.

If a vaccine were to become available in the next year, an optimistic timeline, the first candidates for the drug would be first-line respondersdoctors, nurses, paramedics, as well as infants, toddlers and pregnant women. Thats a figure that exceeds 26 million people in the U.S. alone.

The next round would be essential personnel and children with preexisting conditions, higher-risk patients and people over the age of 65. Once you add all those, just in the U.S., youre talking more than 100 million who would be considered high-priority for a vaccine.

Mark Feinberg, a former Merck & Co. executive in vaccines who now runs the International AIDS Vaccine Initiative, told Bloomberg that the real question wasnt just efficacy, but how quickly can you ramp up manufacturing to meet global need.

There arent a lot of drugs in the industry that are filled at these scales, period, said Stephen Hoge, president of Moderna. Even large pharma companies dont usually operate on this kind of scale. No one entity or one company will be able to do it by themselves.

With any luck, social distancing and quarantine will slow the spread of the virus. Perhaps the coming summer months will also decrease its spread, although whether there is a seasonality component to the disease is still unknown. This would give biopharma companies a little more room to develop and distribute vaccines or to determine which drugs are best-suited to preventing or treating the disease.

Meanwhile, biopharma companies worldwide are working desperately to develop solutions.

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The Hopes and Challenges of a COVID-19 Vaccine - BioSpace

The Food and Drug Administration has quietly closed out an investigation of data violations tied to Novartis' gene therapy Zolgensma, indicating it will not penalize the Swiss drugmaker for submitting manipulated testing results last year.

The violations, documented in an August 2019 inspection of Novartis' AveXis unit, were at the center of a scandal which last year engulfed the pharma and marred a landmark approval for its high-profile spinal muscular atrophy treatment.

In securing the FDA's blessing for the therapy, Novartis had knowingly filed an application containing altered data from preclinical tests in mice. At the time, the FDA warned it would consider civil or criminal penalties against the company, indicating its displeasure that Novartis was aware of the issue but proceeded to ask for approval anyway.

The statement was highly unusual and a signal, former FDA officials said, to other companies in the fast-growing gene therapy field. After seven months of review, however, the FDA has decided against leveling such sanctions, a spokesperson for the agency confirmed to BioPharma Dive.

Instead, the FDA has classified its inspection review as "Voluntary Action Indicated,"or VAI, which means violations were found during the inspection but didn't cross the threshold for regulatory action.

"FDA has completed its review of the information and records of the inspection, the evidence collected, and the firm's responses as well as the corrective actions to the inspectional observations, and the agency has classified the inspection as Voluntary Action Indicated," the FDA spokesperson said.

"Based on its review of the information available, FDA continues to find Zolgensmato be safe and effective for its intended use," added the spokesperson. Throughout its investigation, the FDA maintained that it found no evidence human data were changed.

FDA inspections, such as the one conducted of AveXis last August,result in what are known as Form 483s, technical documents that detail potential violations of the Food Drug and Cosmetic Act. Companies are allowed to respond, after which the FDA decides whether to proceed with a formal warning.

Novartis submitted its responseto the FDA's inspection in late August last year,pledging to retrain employees and improve quality control oversight. The company placed much of the blame for the manipulation on two senior AveXisexecutives, Brian and Allan Kaspar, who were fired in mid August. Through his lawyer, Brian Kasparhas publicly denied any wrongdoing.

Novartis also committed to informing the FDA within five business days of any credible allegations related to data integrity questions with any pending drug application in the future.

Novartis disclosed the FDA's Form 483 classification in a presentation Monday of new clinical data for Zolgensma, briefly noting the FDA's decision.

"We are pleased with this positive outcome and reiterate our firm commitment to data integrity and transparency in our engagements with regulators," a Novartis spokesperson said in a statement.

Zolgensma was only the second gene therapy for an inherited disease to win U.S. approval, showing in clinical studies it could keep alive infants who otherwise were likely to die from spinal muscular atrophy, or SMA. The neuromuscular disease robs newborns of a protein needed for muscle development, usually leading in its most severe form to death or permanent ventilation by age two.

Some 200 infants received Zolgensma in 2019, earning Novartis $361 million last year. The one-time therapy costs $2.1 million per patient, making it the most expensive drug in the world on a per-dose basis.

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No sanctions for Novartis as FDA ends review of gene therapy violations - BioPharma Dive

DUBLIN--(BUSINESS WIRE)--The "Innovations in Bioreactors, Gene Therapy, Vermifiltration, and Membranes for Desalination Processes" report has been added to ResearchAndMarkets.com's offering.

This edition of the Industrial Bioprocessing TOE features trends and innovations in fixed bed and disposable bioreactors used for processing viral vaccines and for the production of recombinant proteins used in gene therapy. The TOE also provides intelligence on the transformation of cellulose waste and other plant waste in to bio-char, levulinic acid, and formic acid that can be used for production of biofuels. The TOE features innovations based on the novel gasification systems that can be vital in converting biomass in to renewable energy. Additionally, the TOE provides intelligence on the use of innovative membrane technologies that can enhance water permeability and selectivity to generate more freshwater from desalination processes. The TOE also focuses on innovations that are based on the use of modular vermifiltration processes for wastewater treatment, and the generation of bioplastics and biofertilizers from plant and food waste.

The Industrial Bioprocessing TOE provides intelligence on technologies, processes and strategic insights of industries involving bioprocessing, including innovations in the development and production of chemicals, pharmaceuticals, nutraceuticals, alternative fuels, chemical feedstocks, food and beverages, and consumer products.

Key Topics Covered:

Advancements in Bioreactors, Gene Therapy, Vermifiltration, and Membranes for Desalination Processes

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Novartis is on the final straight to approval of its spinal muscular atrophy (SMA) gene therapy Zolgensma in the EU, after getting a recommendation for approval from the CHMP.

The positive opinion gets Novartis marketing application for Zolgensma (onasemnogene abeparvovec) back on track after the company revealed last year that regulators in Europe and Japan had asked for more information on manufacturing of the gene therapy which delayed approval by some months.

At the time, Novartis AveXis unit which developed Zolgensma said the questions raised by the regulators werent related to a data manipulation scandal that emerged with the therapy last year, shortly after it was approved in the US. It was approved in Japan on 19 March.

The delay to its approval in Europe prompted Novartis to set up a managed access programme for some patients, which operated like a lottery and is expected to provide free access to the therapy for 100 patients, although it has come in for criticism as being an unfair way to determine access.

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Approximately 550 to 600 infants are born with SMA in Europe each year.

Now, the CHMP has backed conditional approval of Zolgensma for babies and young children with SMA, which could lead to full EMA approval within the next couple of months.

Novartis says it will offer a day one access programme to EU governments and reimbursement agencies to enable immediate access if the EMA gives it the nod.

The recommendation covers children with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1; or patients with 5q SMA with a bi-allelic mutation in SMN1 and up to three copies of the SMN2 gene.

Zolgensma is the worlds most expensive drug, with a price tag of more than $2.1 million per one-shot intravenous infusion, which Novartis argues is justified given the lifelong benefits the therapy can bring.

The company says SMA costs EU healthcare systems between 2.5 million and 4 million within the first 10 years of life alone.

The data scandal doesnt appear to have affected its uptake in the US sales in 2019 reached $361 million, more than half of that total from the last quarter of the year alone.

The quick uptake has come despite the established presence on the market of Biogens Spinraza (nusinersen), an antisense drug given by injection into the spine, which became the first treatment option for SMA when it was approved in 2016 and reached $2.1 billion in sales last year.

GlobalData recently predicted however that Zolgensma will generate global sales of $2.5 billion by 2025, overtaking Spinraza and leading the SMA market.

The CHMP recommendation is another shot in the arm for Novartis gene therapy, coming shortly after it reported additional safety and efficacy data with intrathecal delivery of the gene therapy i.e. delivery into the spinal column rather than intravenously.

Intrathecal administration is considered key to extending the use in therapy for children with milder forms of the disease who tend to exhibit symptoms later in childhood. Novartis also reported new data demonstrating long-term efficacy with Zolgensma, with patients showing benefits for up to five years after they were dosed with the therapy

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Novartis closes on EU approval for SMA gene therapy Zolgensma - - pharmaphorum

WALTHAM, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies and a mission to develop transformative medicines for devastating diseases, today announced it has closed an oversubscribed $60 million Series A financing. Seed venture investors F-Prime Capital and New Enterprise Associates (NEA) co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza and Partners Innovation Fund.

The proceeds will be used to advance the companys platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

The company is led by recently appointed Chief Executive Officer Rick Modi, who has a proven track record of building value at companies including AveXis, InterMune, MedImmune and Centocor. Joining the board of directors as part of the financing are Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, NEA; and Robert Weisskoff, Ph.D., Partner, F-Prime Capital. Industry veteran and gene therapy leader Sean Nolan will chair the board.

At Affinia Therapeutics, were setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors and gene therapies that have remarkable targeting properties, said Modi, CEO of Affinia. We are pleased to partner with such a distinguished syndicate to advance our platform and investigational product candidates toward the clinic for patients in need.

The companys technology was licensed from Lonza and Massachusetts Eye and Ear. It was developed at the Grousbeck Gene Therapy Center and further advanced under a sponsored research agreement with Lonza led by Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

In addition to Vandenberghe, joining Affinia Therapeutics as scientific co-founders are Botond Roska, M.D., Ph.D., Director, Institute of Molecular and Clinical Ophthalmology Basel; Aaron Tward, M.D., Ph.D., Assistant Professor, University of California, San Francisco; and Eric Zinn, Ph.D. student, Mass. Eye and Ear and Harvard University. Together, these scientists have authored more than 200 papers and filed more than 20 patents in the field of gene therapy.

Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies. By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine, said Vandenberghe, Director, Grousbeck Gene Therapy Center at Mass. Eye and Ear.

The Series A financing comes after Vandenberghe and his team successfully developed AAVSmartLibraries comprising thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vectors structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. Affinia Therapeutics has potentially the worlds largest library of patented functional AAV vectors.

Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines, said Mathers, General Partner at NEA. We are pleased to accelerate the impact of this exciting field.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need.www.affiniatx.com.

Link:
Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies - GlobeNewswire