StemCell Therapy

Five percent of the human population is carrier of some form of disorder of Hemoglobin (Hb) (Hemoglobinopathy) affecting its oxygen carrying capacity in blood, as per to WHO. Prevalence rate for Thalassemia (a type of hemoglobinopathy) mutations have been reported to be as high as 17% by studies from Indian subcontinent. Thalassemia and Sickle cell disease are the most common of Hemoglobinopathies and are inherited in an Autosomal recessive manner, meaning that if both partners are carrier of a mutation in the Hb gene, then the risk of having an child affected with Thalassemia major is about 25%. This risk becomes even more significant in view of the high prevalence of Hb gene mutation carrier status in general population (tribal belts, Sindhis, Parsis, Gujratis and specific pockets in south India), marriages within closed communities and consanguinity (marriages within relation)

Treatment of Thalassemia major is lifelong and includes regular blood transfusions, chelation medicines to tackle the issue of increasing iron content in body, managing any complications such as infections, endocrine resulting hormonal imbalances. While Hematopoietic stem cell transplantation (HSCT) is the only curative therapy available currently for patients with -thalassemia major, it is limited by feasibility, cost and availability of suitable matched donor. HSCT is also associated with potential risk of immune-mediated rejection and graft-versus-host disease (GVHD) in few cases. Gene therapy trails have provided a new impetus in this field.

Routine screening for Hemoglobinopathies is done by Hb electrophoresis of HPLC (high performance liquid chromatography). This helps identify the particular disorder and institute prompt treatment and follow up. However such HPLC has major have limitations as all Hb variants may not be detected by HPLC and when screening the neonates or doing prenatal testing (specific testing done during pregnancy to know if the fetus is affected) as the pattern of functioning Hb gradually shifts from fetal type to adult type by around one year of age. Also blood transfusions may influence the HPLC results. Herein comes the importance of Molecular genetic testing. Hundreds of Hemoglobinopathies causing alterations in the HBB gene have been reported, curated and catalogued in various databases. It must be noted that about 5 common mutations in the HBB gene account for over 90% cases of Thalassemia. Making them the first line of mutations to be tested if suspecting Thalassemia. If these are negative then we proceed with the HBB gene sequencing.

Dr. AnupKumar Rawool, Associate Director, Clinical Genomics, SRL Diagnosticssaid,"Living with Thalessemia is not an easy lifestyle and if not diagnosed correctly or at the right time, can lead to other health problems. We know that Thalessemia and Beta Thalessemia is prevalent in India and there is research underway for better cure, treatment and therapy of the illness. While there are other forms of treatment available, the newest treatment now making waves all over the world is Gene Therapy. We now have studies to prove that Gene Therapy is a viable cure for beta thalessemia. It has therapeutic potential and we are excited to have with us this indispenasable expertise that is is key to good health for countless patients in our country.

"Timely diagnosis of genetic disorders with appropriate Molecular genetic tests provides an optimum window for offering prenatal diagnosis and decision making for the family. It is recommended that the person with thalassemia or any Hemoglobinopathy undergo HBB sequencing test to identify the disease causing alterations in the HBB gene. Once these are identified and confirmed then diagnostic testing during pregnancy can be offered by either Chorionic villi sampling between 11-13 weeks or amniocentesis after 16 completed weeks of gestation, leaving ample time for the molecular genetic lab to perform and report the test and for the clinician, medical geneticist and the family for appropriate pre and post test genetic consultation and decision making accordingly. It is advisable that Planning for prenatal testing to be done prior to pregnancy. Advances in molecular testing techniques also provide option for testing embryos on day 5 by pre-implantation genetic diagnosis (PGD) if availing an option of IVF/ART. So prior screening of embryo can be done for any known genetic disorder in the family. However PGD services are available at limited centres. A prior Genetic consultation with a Medical Geneticist for the families in such situation is strongly recommended to smoothen the entire process.

Timely diagnosis of genetic disorders with appropriate Molecular genetic tests provides an optimum window for offering prenatal diagnosis and decision making for the family. It is recommended that the person with thalassemia or any Hemoglobinopathy undergo HBB sequencing test to identify the disease causing alterations in the HBB gene. Once these are identified and confirmed then diagnostic testing during pregnancy can be offered by either Chorionic villi sampling between 11-13 weeks or amniocentesis after 16 completed weeks of gestation, leaving ample time for the molecular genetic lab to perform and report the test and for the clinician, medical geneticist and the family for appropriate pre and post test genetic consultation and decision making accordingly. It is advisable that Planning for prenatal testing to be done prior to pregnancy. Advances in molecular testing techniques also provide option for testing embryos on day 5 by pre-implantation genetic diagnosis (PGD) if availing an option of IVF/ART. So prior screening of embryo can be done for any known genetic disorder in the family. However PGD services are available at limited centres. A prior Genetic consultation with a Medical Geneticist for the families in such situation is strongly recommended to smoothen the entire process.

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International Thalassaemia Day 2022: The genetic perspective of Thalassemia - Free Press Journal

OTTAWA, Ontario--(BUSINESS WIRE)--Virica Biotech Inc. (Virica), a leading developer of solutions for scaling of viral medicines, today announced it has received $400,000 from Innovation, Science and Economic Development Canada (ISED) through the Innovative Solutions Canada (ISC) program. This funding supports Viricas collaboration with the National Research Council of Canadas (NRC) Cell and Gene Therapy Challenge program to enhance the manufacturing of an affordable version of AAV-LPL, a gene therapy being developed for people with debilitating lipoprotein lipase deficiency (LPLD).

NRC researchers are re-engineering AAV-LPL using new adeno-associated viral (AAV) derived vectors to advance the LPLD gene therapy originally developed at the University of British Columbia. AAV vectors are the delivery vehicle of choice for inserting therapeutic genes into cells, but increasing production yields of these biopharmaceuticals in sufficient quantities to meet demand is challenging.

Virica is deploying its custom VSE formulation, in collaboration with the NRC, to design a more robust AAV vector manufacturing process. The VSE formulation is tailor-made to optimize the scalability of the NRCs AAV manufacturing process and produce the next-generation gene therapy for LPLD.

The NRC offers Canadian businesses access to unique expertise in this area, as well as outstanding research and manufacturing facilities. It will be exciting to see the NRC use our VSEs to produce higher yields of their AAV vectors and ultimately contribute to lowering the cost of a life-saving gene therapy for people with LPLD.- Dr. Jean-Simon Diallo, CEO of Virica Biotech

The funding from ISC supports our commitment to partnering with gene therapy developers and manufacturers, as we push for more scalable and cost-effective processes, in order for new cell and gene therapies to be affordable to all.- Ella Korets-Smith, Chief Business Officer of Virica Biotech

Our government is proud to support companies like Virica Biotech as they work to make medical therapies more affordable through innovative solutions. The work Virica is undertaking can offer real hope to those suffering from a rare disease for which there are no treatments, and more so, could make it more affordable for Canadians.- The Honourable Franois-Philippe Champagne, Minister of Innovation, Science and Industry

The NRCs collaboration with Virica Biotech will help expand Canadas capacity to domestically manufacture AAV gene therapy vectors. It brings us one step closer to accelerating the development of affordable and accessible made-in-Canada cell and gene therapies, through innovative partnerships with government, academia and industry.- Iain Stewart, President of the National Research Council of Canada

About LPLDLipoprotein lipase deficiency (LPLD) is a rare, inherited disease for which no treatment exists. People with LPLD have a faulty gene and are unable to breakdown fat particles carried in their blood. The accumulation of fat turns their blood white and leads to recurring and potentially fatal pancreatitis attacks. LPLD affects about two people out of a million, but in the Saguenay region of Quebec, as many as one in 50 people carry the gene mutation.

About Virica BiotechVirica optimizes the manufacturing of viral medicines which allows developers to economically deploy their products at scale. Viricas Viral Sensitizer (VSE) platform reduces production inefficiencies caused by innate anti-viral defenses in manufacturing cells. Customized VSE formulations substantially increase manufacturing yields and reduce the cost of goods for a range of products, including vaccines, cell and gene therapies, and anti-cancer therapies. Visit viricabiotech.com for more information.

Photos available upon request.

This news release contains forward-looking statements, which reflect the current expectations of the Companys management for future growth, results of operations, performance and business prospects. Forward-looking statements involve significant known and unknown risks, uncertainties and assumptions.

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Virica Biotech Announces Collaboration with the Government of Canada - Business Wire

Retinal regions of preserved photoreceptors identified as targets for subretinal delivery of AAV8-based gene therapy to address mutations in genes that cause forms of Leber congenital amaurosis

Subretinal injection well tolerated in preclinical dose-ranging studies

Data presented at Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022

RESEARCH TRIANGLE PARK, N.C., May 04, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced promising new preclinical data from studies evaluating the potential of its gene therapies OPGx-001 and OPGx-002 to address forms of Leber congenital amaurosis (LCA), a group of rare inherited retinal diseases characterized by photoreceptor degeneration, due to mutations ofLCA5orRDH12genes, respectively.

In preparation for IND-enabling trials of OPGx-001 and OPGx-002, studies were conducted to determine eligibility, therapeutic window, and possible outcome measures for gene therapy for LCA5 and RDH12 inherited retinal diseases. In addition, safety evaluations for the subretinal delivery of an AAV8 vector containing LCA5 or RDH12 were performed in non-human primates (NHP).

The data demonstrated that despite severe retinal dysfunction, LCA patients exhibited detectable photoreceptor regions that may be targets for gene augmentation, identified in the central and midperipheral retina of LCA5-LCA patients and in the pericentral and peripapillary retina of RDH12-LCA patients. In two dose-ranging studies in NHPs, subretinal delivery of OPGx-001 and OPGx-002 was well tolerated, with mild inflammatory changes observed at the higher dose. The data support the therapeutic potential and tolerability of gene augmentation to address LCA5-LCA and RDH12-LCA and provide guidance for formal preclinical toxicology studies and future human clinical trials.

"Patients with Leber congenital amaurosis due to mutations of the LCA5 or RDH12 genes experience rapid retinal degeneration, resulting in vision loss in early childhood," said Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus. "The detection of preserved photoreceptors in LCA patients signals a therapeutic opportunity to target the mutation and potentially restore structure and function through gene augmentation. In addition, the encouraging dose-ranging results in the primate model suggest subretinal delivery of Opus' AAV8-based gene therapies are safe and inform the doses to be used in our toxicology studies, a key step on our path toward the clinic for OPGx-001 and OPGx-002."

The data were presented today at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022 in Denver, Colo., by Jean Bennett, M.D., Ph.D., Scientific Co-founder, Opus Genetics, and University of Pennsylvania Perelman School of Medicine; and Tomas Aleman, M.D., University of Pennsylvania Perelman School of Medicine.

Also at ARVO 2022, Dr. Jayagopal was awarded the title of ARVO Fellow, an honor established to recognize current ARVO members for their individual accomplishments, leadership and contributions to the ARVO Association.

About Opus GeneticsOpus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness's venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit http://www.opusgenetics.com.

Media Contact: Heather Anderson6 Degrees919-827-5539handerson@6degreespr.com

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Opus Genetics Announces Promising New Data Highlighting Potential of AAV-based Gene Therapies for the Tre - Benzinga

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Tenaya Thearpeutics Reports Will Present Preclinical Data On Gene Therapy Programs, Platform Capabilities - Benzinga

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced that four abstracts, including one from a research and license collaboration, were accepted by the American Society of Gene & Cell Therapy (ASGCT) and will be presented as posters and oral presentations at the upcoming annual meeting on May 16-19, 2022 at the Walter E. Washington Convention Center in Washington, D.C.

Details for the poster and oral presentations can be accessed on the ASGCT website at https://annualmeeting.asgct.org/abstracts.

Oral Presentations:

Title: Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In VivoDate and Time: Tuesday, May 17, 2022, 3:45 PM 4:00 PMSession Title: Gene Editing in Cancer and Complex DiseasesLocation: Hall EAbstract #: 447

Title: AAV-Meganuclease-Mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver1Date and Time: Wednesday, May 18, 2022, 3:00 PM 3:15 PMSession Title: Presidential Symposium and Presentation of Top AbstractsLocation: Hall EAbstract #: 811

Poster Presentations:

Title: Optimization of Hydroxyacid Oxidase 1 (HAO1) Targeting ARCUS Nucleases for the Treatment of Primary Hyperoxaluria Type 1 (PH1)Date and Time: Monday, May 16, 2022, 5:30 PM - 6:30 PMSession Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases ILocation & Poster Board Number: Hall D, M-120Abstract #: 239

Title: ARCUS Gene Editing to Eliminate MELAS-associated m.3243A>G Mutant Mitochondrial DNADate and Time: Tuesday, May 17, 2022, 5:30 PM - 6:30 PMSession Title: Gene Targeting and Gene Correction IILocation & Poster Board Number: Hall D, Tu-66Abstract #: 561

1 University of Pennsylvanias Gene Therapy Program presentation sponsored by iECURE.

About Precision BioSciences, Inc.

Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS genome editing platform. ARCUS is a highly precise and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Companys pipeline consists of multiple ex vivo off-the-shelf CAR T immunotherapy clinical candidates and several in vivo gene editing candidates designed to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit http://www.precisionbiosciences.com.

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Precision BioSciences to Present Preclinical In Vivo Gene Editing Research at Upcoming American Society of Gene & Cell Therapy 25th Annual Meeting...

Sharing the news on Thursday (15th April), the company said the investment has enabled part of the facility to be dedicated to the safe handling of cell and gene therapy samples, including the installation of cryogenic storage, allowing biological materials to be preserved in liquid nitrogen vapor at temperatures of around -180C.

The facility also has the ability to package, label and distribute cryogenic materials, ensuring the integrity of the therapies being prepared for clinical trials, and has been designed so that capacity can be rapidly expanded further to meet growing clinical supply needs, as well as future commercial demand.

Establishing robust clinical supply chain services for cell and gene therapies is complex and challenging, and Catalent has undertaken an in-depth strategic review to evaluate how it can establish a safe, efficient and flexible approach to support this fast-growing area of the industry, said Ricci Whitlow, President, Catalent Clinical Supply Services.

The solution we have implemented at Philadelphia not only meets current needs, but also provides a template for us to easily replicate at other facilities in our global network, allowing incremental capacity expansion within the new infrastructure as demand grows.

With sites in the US, UK, Germany, Singapore, Japan and China, and an extended network of over 50 depots, Catalents clinical supply services can handle a broad range of international compliance and distribution requirements to support global clinical trials.

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Catalent introduces cryogenic capabilities to support growing demand for cell and gene therapy - gasworld

Cell and gene therapies hold great promise for treating human diseases, for which current therapeutics are lacking. However, the research and development process for delivering these therapies to the clinic is complex, comprising various bioprocessing and bioproduction steps that must be fulfilled and optimized to ensure the safety and efficacy of the final product. The process continues to evolve to meet increasing demand and to overcome unexpected obstacles, such as the impact of the COVID-19 global pandemic on supply chains and manufacturing efforts in the biotech and biopharma industries.In this interview, Technology Networks spoke with Neal Goodwin, PhD, Chief Scientific Officer at Teknova, to learn more about current bioprocessing and bioproduction trends in the cell and gene therapy space. Goodwin also discusses the impact of the COVID-19 pandemic on the biopharmaceutical sector and highlights how the industry has united to contribute to the development of vaccines at this critical time.

Molly Campbell (MC): You have 20 years of experience working in biotechnology and therapeutics. Can you talk about the evolution of the field through this time?Neal Goodwin (NG):It has been a great 20 years! The human genome release is one of the most dramatic events during those 20 years. Once we had the human genome at our disposal, the field burgeoned. We've seen targeted therapeutics make a significant impact in personalized healthcare imatinib, erlotinib, and the new KRAS inhibitors, among other targeted therapeutics for oncology, come to mind. Also, the first immuno-oncology therapeutics are now positively affecting large numbers of patients which is terrific.

The ability to move into gene therapy and address severe genetic disorders and conduct gene replacement therapy is fantastic. The progress in gene delivery viral vectors and non-viral nanoparticle delivery contributed to the rapid advancement of prominent COVID-19 vaccines. The mRNA-based vaccines and non-viral nanoparticle-based gene delivery technologies have validated a promising approach to gene therapy that is being expanded to rare genetic diseases. The ability to mass-produce mRNA vaccines demonstrated the feasibility of mRNA treatments and gene therapies for more widespread use for treating broader therapeutic targets and conditions.

MC: Can you talk to us about some of the current trends you are seeing in the bioprocessing and bioproduction space?NG: First of all, there is a considerable demand for bioprocessing and commercial production of viral and non-viral vectors. These efforts must support what is now well over 1,000 clinical development programs in gene therapy alone. There is a need to develop better individualized processes that are readily scalable for GMP production matched to specific therapeutics. This effort will require the implementation of new strategies, new technologies, and increased global manufacturing capacity. It is an exciting place to be, but there is a great deal of development left to achieve streamlined processes and optimized capacity.

MC: Cell and gene therapies are key emerging markets. What are some of the key challenges that exist when developing these products from the lab, through to clinical testing and eventually authorization?NG:There is a wide breadth of requirements I will try to narrow them down to a few of the essential points. Some autologous cellular therapies require cells to be genetically modified ex vivo. This genetic modification is usually done with viral vectors, and a crucial aspect is the delivery of the gene, i.e., the active pharmaceutical ingredient, to the cells. There is a need for more advanced lentiviral vector systems that are safer for use in systemic gene delivery.

Among the non-cellular gene delivery segment, adeno-associated virus (AAV) vector gene delivery is the most widely used viral gene delivery platform. AAV production relies on the efficient expression in human cell lines of plasmids that encode viral packaging, capsid and replication genes, and expression plasmids encoding the therapeutic gene. One of the drawbacks of AAV vectors employed for gene therapy is they generally cannot deliver larger gene fragments required for optimal treatments. So, better viruses or non-viral gene delivery systems are needed.

In turn, choosing the best cell manufacturing conditions that provide the most efficient viral vector production is essential. A key challenge is how to scale up with reproducibility and efficiency.

MC: How can we look to overcome the challenge of scale-up in cell and gene therapies?NG: Because therapeutic programs are each different, having adaptable processes to individual programs is vital. Some strategies focus on gene delivery into autologous cells or allogeneic cells that become expanded and transplanted into patients other strategies require direct delivery of viral or non-viral gene therapy products into patients.In viral gene delivery, matching the most suitable virus to achieve the best target tissue tropism is essential. To date, AAV has been successfully used in two FDA-approved gene therapies, with many in advanced clinical trials. Traditionally, there have been few available AAV serotypes available for gene delivery. However, now we can use a directed evolution approach to manipulate and generate AAV capsids that have better target tissue tropism with reduced host immunogenicity.

Concerning AAV production, the purification processes of viruses need optimized, which is a focus of Teknova. We are barcoding different AAV serotypes with specific processes to achieve higher purity and viral vector yield.

MC: How has the COVID-19 impacted the biotech sector?NG: COVID-19 motivated and united the biotech sector. It remains a worldwide effort where enormous challenges are being addressed rapidly and collaboratively. The field appears more collaborative, almost cheering once competitive firms and laboratories. Teknova experienced this by retooling and supplying GMP viral transport media for COVID-19 testing and providing the global research community with research reagents and custom GMP manufactured products. This transformation is something that instils pride.

MC: How do you envision the biotech sector will change over the next few years? Are there any areas, aside from cell and gene therapies, that excite you?NG: The biotech sector's attitude is positive and more confident than 20 years ago, and this spirit enables us to tackle problems once considered unsolvable.

One of the most exciting biotech advancement areas is precision therapeutics. For example, a difficult hurdle to overcome was treating cancers linked to specific mutant KRAS alleles in a high percentage of cancers. Multiple firms have recently advanced precision therapeutics to late-stage clinical trials to treat this historically untreatable cancer type precisely. I think this can-do attitude will continue.

Another area where I expect to see progress is gene therapy delivery that will increasingly evolve non-viral delivery technologies. This evolution will allow precise targeting of therapeutics delivery and undoubtedly improve human health through this process.

Neal Goodwin was speaking to Molly Campbell, Science Writer for Technology Networks.

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Bioprocessing and Bioproduction Trends in Cell and Gene Therapies - Technology Networks

STAMFORD, Conn., April 16, 2021 /PRNewswire-PRWeb/ --April 29 marks a pivotal moment in cancer research when the world's brightest minds come together to discuss how to translate the success of CAR T-cell therapies for blood cancers into successful cell and gene therapies for the most complex and deadly solid tumor cancers. The scientists and companies driving the latest advances in cancer cell and gene therapy will gather online for a virtual Summit with the Alliance for Cancer Gene Therapy, who envision a cancer free future and want to change the C-word from Cancer to Cure. The Summit is open to the public, to medical professionals, scientists and companies interested in cell and gene therapy to fight cancer. To register for the Summit, visit acgtfoundation.org.

Summit 2021 is being held online on Thursday, April 29, 2021, from 10:00 a.m. until 6:00 p.m (ET), and features eight (8) panel discussions with leading researchers developing the next generation cancer cell and gene therapies, biotech companies who are bringing new treatments through the clinic, and investors who are funding this burgeoning pipeline of solid tumor breakthroughs.

The panels include:

Keynote: Does Cancer Have a Future? What's Next? Where Will We Be in 2025?

A conversation with Nobel Laureate James P. Allison, PhD, executive director of the Immunotherapy Platform at MD Anderson Cancer Center, and Carl H. June, MD, director of the Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania. The conversation will be moderated by Siddhartha Mukherjee, MD, founder, Myeloid Therapeutics, Pulitzer-prize winning author of "The Emperor of All Maladies."

Evolving Cell and Gene Regulatory and Manufacturing Processes to Match the Speed of Innovation

Panel discussion with: Rafael Amado, MD, executive vice president, head of R&D and chief medical officer, Allogene Therapeutics; Andre Choulika, PhD, chairman, CEO and founder, Cellectis; Amy DuRoss, founder and CEO, Vineti; Bruce Levine, PhD, Barbara and Edward Netter professor in Cancer Gene Therapy, University of Pennsylvania; Ke Liu, MD, PhD, senior vice president and head of Regulatory Affairs & Strategy at Sana Biotechnology, former FDA Oncology Center of Excellence associate director for Cell and Gene Therapy; and moderated by Asthika Goonewardene, managing director, Equity Research - Biotech, Truist Securities.

Innovating Science, From the Lab to the Clinic

Panel discussion with: Brian Brown, PhD, professor and associate director of the Precision Immunology Institute, Icahn School of Medicine, Mount Sinai; Yvonne Chen, PhD, associate professor of Microbiology, Immunology & Molecular Genetics, UCLA, Founder Kalthera; Franco Marincola, PhD, senior vice president and global head of Cell Therapy Research at Kite, a Gilead Company; Clodagh O'Shea, PhD, professor, Molecular and Cell Biology Laboratory, Salk Institute, scientific founder IconOVir Bio; and moderated by Jenna Foger, senior vice president, Science & Technology, Alexandria Venture Investments/Alexandria Real Estate Equities.

Tackling the Toughest Challenges Pancreatic Cancer

Panel discussion with: Sidi Chen, PhD, associate professor, Yale Cancer Center; Joseph A. Fraietta, PhD, director, Solid Tumor Immunotherapy Laboratory, Center for Advanced Cellular Therapeutics, University of Pennsylvania; Michael T. Lotze, MD, professor of Surgery and Bioengineering, University of Pittsburgh, chief cellular officer, Nurix, chair of Alliance for Cancer Gene Therapy's Scientific Advisory Council; Mark H. O'Hara, PhD, assistant professor of Medicine at the Hospital of the University of Pennsylvania; and moderated by Andrew Rakeman, PhD, vice president of Research, Lustgarten Foundation.

Tackling the Toughest Challenges Glioblastoma

Panel discussion with: Nduka Amankulor, MD, department of Neurosurgery, University of Pittsburgh Medical Center; Samantha Bucktrout, PhD, senior director of Research, Parker Institute of Cancer Immunotherapy; Noriyuku Kasahara, MD, PhD, principal investigator, Brain Tumor Center, UCSF; Crystal Mackall, MD, founding director, Stanford Center for Cancer Cell Therapy; and moderated by Klaus Veitinger, MD, PhD, venture partner OrbiMed.

New Approaches to Solid Tumor Breakthroughs

Panel discussion with: Ken Drazan, MD, chief executive officer, ArsenalBio; Daniel Getts, PhD, chief executive officer & co-founder, Myeloid Therapeutics; Garry E. Menzel, PhD, president and chief executive officer at TCR Therapeutics; and moderated by Luke Timmerman, founder and editor, The Timmerman Report.

Patients, Caregivers, Doctors, Oncologists, Advocates, Researchers, You, Me We all Have Cancer in Common

Panel discussion with: Caroline Corner, PhD, managing director, Westwicke; Robert Levis, director CLL Society; Callum Miller, CAR-T recipient, patient advocate; Tom Whitehead, co-founder, The Emily Whitehead Foundation; and moderated by Gregory C. Simon, former president of the Biden Cancer Initiative.

Innovating Finance, Non-Traditional Funding Sources

Panel discussion with: Jay Campbell, managing director of Cancer Research Institute Clinical Accelerator and Venture Fund; Marc Hurlbert, PhD, chief science officer, Melanoma Research Alliance; Ken Schaner, partner Schaner & Lubitz; Anna Turetsky, PhD, principal, Venture Investments, The Mark Foundation for Cancer Research; moderated by Luke Evnin, PhD, co-founder and managing director MPM Capital.

"It's so rare to have the opportunity to hear from the world's top cancer researchers, biotechs, investors and patients all in the same room," noted Barbara Lavery, chief program officer for Alliance for Cancer Gene Therapy. "We're excited to have Alliance for Cancer Gene Therapy be a catalyst for these important conversations that will help not only other scientists working on new cell and gene therapy approaches, but companies seeking investment to advance their clinical pipelines, and patients and their families looking for potentially life changing therapeutic options in their fight against cancer."

To learn more, see the list of speakers and panels and register for the event, visit the website Alliance for Cancer Gene Therapy Summit 2021.

Alliance for Cancer Gene Therapy

For 20 years, Alliance for Cancer Gene Therapy has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Alliance for Cancer Gene Therapy funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. 100% of all public funds raised by Alliance for Cancer Gene Therapy directly support research and programs. For more information, visit acgtfoundation.org, call 203-358-5055, or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube @acgtfoundation.

Media Contact

Jenifer Howard, J. Howard Public Relations, +1 (203) 273-4246, jhoward@jhowardpr.com

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Alliance for Cancer Gene Therapy Summit 2021 Features World Renowned Cancer Researchers Advancing Solid Tumor Breakthroughs - WFMZ Allentown

RESEARCH TRIANGLE PARK StrideBio, an emerging startup focusing on gene therapies which closed on a big $81.5 million round of venture capital in March, is partnering with Duke University as it expands efforts to create next-generation therapies.

The agreement announced Wednesday is described as a multi-technology collaboration.

Specific financial terms were not disclosed but includeequity, upfront and milestone-driven payments, and sponsored research commitments from StrideBio to Duke University. Also included are royalties on future product sales.

We are very excited to partner with Duke University to advance these technologies that can improve and expand on the potential benefits of gene therapies for patients who desperately need them, stated Sapan Shah, StrideBios CEO. We look forward to working together with a fantastic group of Duke researchers and clinicians to bring next-generation AAV-based gene therapies to patients with rare CNS diseases and beyond, starting with Alternating Hemiplegia of Childhood.

StrideBio is focused on what it calls engineered viral vectors, or AAV, for gene therapy. The firm has already announced it has struck a deal with Crispr Therapeutics to develop in vivo gene delivery applications. As part of the deal, StrideBio will receive development funding, milestones and royalties on licensed vectors, and retain certain rights to use the novel AAV vectors for gene therapy applications.

Heres how StrideBio describes the agreement and the tecnologies involved:

The agreements announced today provide StrideBio an exclusive license to multiple technologies that will enable best-in-class next-generation gene therapies developed at Duke University. Included are novel engineered AAV vectors which complement StrideBios existing STRIVETMcapsid engineering platform, having been selected through a cross-species evolution that results in significantly enhanced tropism and potency versus AAV9 across a wide range of tissues such as CNS, skeletal and cardiac muscle. Data on these novel vectors were presented by Duke researcher and StrideBio co-founder, Aravind Asokan, Ph.D., at the American Society of Gene & Cell Therapy 23rdAnnual Meeting in an abstract titled Cross Species Evolution of Synthetic AAV Strains for Clinical Translation (Gonzalez et al., ASGCT 2020, Abstract 24). In addition, StrideBio has licensed exclusive rights covering a novel use of IgG-degrading enzyme IdeZ to clear neutralizing antibodies in conjunction with AAV gene therapy administration. This innovative approach was recently published by members of the Asokan Lab in a manuscript titled Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme (Elmore et al., JCI Insight, 2020, 5(19): e139881). Finally, StrideBio obtained license rights to a novel gene therapy approach for the treatment of AHC recently published by Duke researcher Mohamad Mikati, M.D., in a manuscript titled AAV Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood (Hunanyan et al., Human Gene Therapy, February 12, 2021).

Gene therapy startup StrideBio lands $81.5M in capital, new investors

This License and Master Sponsored Research Agreement will ensure that these innovative technologies receive the resources and expertise necessary to develop treatments that can ultimately benefit patients. We are delighted to have StrideBio as a partner on this important effort in the gene therapy area, said Robin Rasor, Executive Director of the Office of Licensing and Ventures at Duke, in the announcement.

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Gene therapy startup StrideBio signs collaboration deal with Duke University - WRAL Tech Wire

Roots Analysis has released its latest overview on theCell and Gene Therapy CROs Market (2nd Edition), 2021-2030, covering key aspects of the industry and identifying areas for potential future growth.

The opportunity is likely to be well distributed across therapeutic areas, scales of operation, types of therapies and key geographical regions, said the analysts.

The advanced therapy medicinal products (ATMPs) sector, including cell and gene therapy (CGTs) developers. is a relatively niche area, with the presence of several start-ups/small-sized developers dominating the development landscape. About 52% of cell and gene therapy developers are small players.

And, over time, said the analysts, outsourcing has become a preferred operating model for product development activity in this domain, with CROs making significant investments towards acquiring advanced technologies and the capacity to accommodate their growing clientele.

The R&D of biologics is complex and time intensive process, requiring sophisticated techniques and dedicated infrastructure, which is readily available with contract service providers. Further, these service providers adhere to the rapidly evolving regulatory environment, acting as a key enabler to the broader industrys evolution in the coming years.

The study noted that, currently, over 100 industry players claim to have the necessary capabilities to offer contract research and clinical trial support for ATMPs.

Close to 38% of the CROs engaged in this domain are large companies, having required capabilities to provide services for both types of therapies. Examples of new entrants those established since 2018 - that claim to offer clinical services include Geistek Pharma, The Discovery Labs, KCT and Longmore 60 Biotech, reported the market specialists.

Around 45% of cell and gene therapy CROs offer research related services across both, preclinical and clinical scales of operations, while some 74% of players offer CRO players offer regulatory and data management services for cell and gene therapies, according to the analysis.

Both established players and new entrants have forged several partnerships in the recent past; most of the deals were focused on cell therapy, said the analysts, citing examples of the companies that have signed multiple international deals: Accelovance, Charles River, Laboratories, and CitoxLab.

In pursuit of a competitive edge and to eventually establish themselves as one-stop shops, stakeholders are also actively consolidating their capabilities related to cell and gene therapies through mergers and acquisitions, commented the authors.

Some are pursuing such activity for geographical consolidation reasons as shown by the acquisition of Synteract by Syneos Health in December 2020, some for portfolio addition, as seen in Comparative Biosciences being bought by Genesis Biotechnology in August 2020. Others have gone down the M&A route to expand their portfolios: the acquisition of Absorption Systems by Pharmaron in December last year is a case in point, while geographic expansion was the rationale behind the acquisition of Veristat by TCTC Group in January 2020, according to the market specialists.

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Cell and gene therapy services market forecast to see significant growth over the next decade - BioPharma-Reporter.com

AGTC Transfers Clinical Trial Materials and Phase 1/2 Data To TeamedON

ROCKVILLE, Md., April 13, 2021 (GLOBE NEWSWIRE) -- TeamedOn International, Inc., a biotechnology company dedicated to advancing gene therapies for rare diseases, including ophthalmic indications, and Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced a licensing agreement to advance gene therapy to treat X-linked retinoschisis (XLRS), an inherited disease that causes loss of vision due to degeneration of the retina in males. Under the terms of the agreement, AGTC will provide TeamedOn with the clinical trial material, pre-clinical and clinical data generated for the development of AGTCs investigational intravitreal gene therapy candidate, rAAV2tYF-CB-hRS1.

The license between AGTC and TeamedOn re-opens the possibility of a much-needed treatment for individuals with XLRS, said Peter Mu, CEO of TeamedOn. TeamedOn is very pleased to be able to build on AGTCs efforts, carry on their investment in XLRS to bring potential benefit to patients.

Under the agreement, TeamedOn will conduct all activities required to reinitiate clinical development of the program. AGTC will be eligible to receive milestones and royalties based on clinical progress.

There is no cure for XLRS, and patients living with this disease have an urgent need for disease-modifying therapies that have the potential to stabilize and/or improve their long-term vision outcomes, said Dr. Paul Yang, Assistant Professor of Ophthalmology at the Oregon Health and Sciences University, who was a clinical investigator for AGTCs prior XLRS Phase 1/2 clinical trial and recently engaged by TeamedOn. Intravitreal injection of this gene therapy for XLRS previously demonstrated a reasonable safety profile. When administered through subretinal injection as TeamedOn is planning, this investigational gene therapy may have an increased likelihood of producing detectable biological activity.

AGTC discontinued its XLRS clinical program and development of rAAV2tYF-CB-hRS1 in 2018 because defined efficacy endpoints were not met using intravitreal injection.

We are excited at the prospect of TeamedOn taking a license for this program to explore the opportunity for subretinal injection of rAAV2tYF-CB-hRS1 to provide potential clinical benefit to mitigate the devastating impact on the vision of individuals with XLRS for whom there are no effective treatments, said Sue Washer, President and CEO of AGTC.

Forward-Looking StatementsThis release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs, including statements regarding the licensing agreement between AGTC and TeamedOn International, Inc. for AGTC to provide TeamedOn with the clinical trial material, pre-clinical and clinical data generated for the development of AGTCs investigational intravitreal gene therapy candidate, rAAV2tYF-CB-hRS1. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions, and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

About TeamedOnTeamedOn International, Inc. (TeamedOn) (https://www.TeamedOn.com) is advancing gene therapies for rare diseases and currently focused on ophthalmic indications. We are developing innovative medicine to treat inherited retinal disorders (IRDs) and other eye diseases with significant unmet medical need. In addition to our internal R&D programs, we also seek partners for collaboration or co-development. Our goal is to restore sight and prevent vision loss for patients all around the world. TeamedOn is privately funded and located in Rockville, MD.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTCs most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, an innovator in the emerging field of optogenetics and retinal coding.

TeamedOn PR CONTACTS:Peter Mu, PhD, MBACEOTeamedOn International, Inc.(301) 461-1617Peter_Mu@teamedon.com

AGTC IR/PR CONTACTS: David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

AGTC Corporate Contacts:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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TeamedOn and AGTC Announce a Licensing Agreement Advancing X-Linked Retinoschisis Gene Therapy Program - BioSpace

LAKE FOREST -- Jaguar Gene Therapy said it has raised $139 million through its most recent round of investments, led by Eli Lilly and Co. and Deerfield Management.

Also participating in the round were Arch Venture Partners, co-founded by Robert Nelsen and one of the largest early-stage technology venture firms in the United States; Goldman Sachs; and Nolan Capital, the investment fund of former AveXis CEO and current Jaguar Executive Chairman Sean P. Nolan.

Jaguar Gene Therapy is a company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including galactosemia, genetic causes of autism spectrum disorder, Type 1 diabetes and Bardet-Biedl syndrome.

"We are thrilled to have attracted this prestigious group of visionary investors, which affirms our strategic approach, promising pipeline programs, and unique collaboration with Deerfield Management," said Joe Nolan, chief executive officer of Jaguar Gene Therapy. "With this capital infusion, we are well positioned to execute on our mission to accelerate breakthroughs in gene therapy and bring them to children and adults with severe genetic diseases."

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Jaguar Gene Therapy gains $139 million from investors - Daily Herald

This is an excerpt from the April 13, 2021 edition of Medically Necessary, a health care supply chain newsletter.Subscribe here.

The trend: The number of biologic drugs and gene therapies is growing, and logistics companies are increasing their capacity for temperature-controlled transportation to support them.

Biologics are drugs created from biological sources, such as blood or other cells. Theyre usually much more complex than drugs produced chemically.

Gene therapies, which can be biologics, are drugs that replace or modify a patients gene to treat a disease.

Drugs in both categories require temperature to be carefully controlled during transportation.

The future: About a quarter of all drugs currently require some kind of temperature control during transportation, according to RanjeetBanerjee, CEO of the pharmaceutical logistics company Cold Chain Technologies.

He expects that number to jump to about a third of all drugs within the next two years.

With the launch of biologics, with the launch of personalized medicine and certain gene therapy formulations, that are happening now, the need for temperature and other condition monitoring is becoming even more acute, he said.

Biologics: The market for temperature-sensitive biologics is growing faster than that of other types of drugs.

Spending on biologics through Medicare Part B, which covers drugs that patients recieve at a doctors office or hospital, jumped from about 56% of total drug spending in 2006 to 77% in 2017, according to a Health and Human Services Department report.

In large part, that growth is because biologic drugs tend to be very expensive. The report found that biologics accounted for almost all of the programs drug spending growth during those years.

The number of patients receiving biologics also appears to be increasing.

Data from Medicare Part D, which covers prescription drugs, shows that the number of beneficiaries receiving a group of seven common biologics increased by more than 75% from 2011 to 2018.

A 2017 report from The Biosimilars Council predicts that the growth of biosimilars cheaper alternatives that have the same clinical effects as biologics will further expand access to these types of treatments.

Like biologics, many biosimilars require temperature control during transportation and storage.

Gene therapies: The cold chain logistics company Cryoport expects the value of the gene therapy market to increase sevenfold between 2019 and 2024.

FDA approved the first gene therapy in the U.S. in 2017 and has approved six more treatments since then.

The agency approved two of those seven treatments in the first quarter of 2021 alone.

In 2019, former FDA Commissioner Scott Gottlieb said he expected the agency to be approving 10 to 20 cell and gene therapy products every year by 2025.

This trend has a couple of logistics implications, Cedric Picaud, CEO of the Cryoport company CRYOPDP told FreightWaves. The value of each shipment is very high. The cost of managing such medication is high. In terms of logistics, the lead time requirement is very challenging.

The response: As the number of biologic drugs and gene therapies continues to grow, logistics companies are increasing their capacity for temperature-controlled transportation.

In March, Cold Chain Technologies, which previously focused on packaging, expanded the scope of its business.

The company will now offer last-mile delivery services, warehousing and point-of-care storage for temperature-controlled drugs. The goal is to provide logistics from the manufacturer to the patient, Banerjee said.

Over the past two years, Cryoport has acquired several cold chain logistics companies to expand its reach.

Cryoport acquired an Australian logistics company in April. That follows the acquisition of two cold chain logistics companies in August 2020 and another acquisition in May 2019.

The company grew from 57 employees at the end of 2018 to more than 625 by the end of 2020.

Tribe Transportation, a temperature-controlled trucking company based in Georgia, recently added more than 100 trailers to its fleet to meet demand from the life sciences industry.

There are still life sciences customers out there looking for capacity to move shipments, Executive Vice President Matt Handte told FreightWaves. Post-vaccine, weve seen that continue to rise.

The past three years have been the companys best years for growth of both revenue and fleet size, Handte said.

The virus: The coronavirus vaccine rollout has put the cold chain in the spotlight, but companies dont expect this to be a flash in the pan.

Cold Chain Technologies provides packaging for COVID-19 vaccines made by Moderna and Johnson & Johnson. The company opened a new manufacturing plant in Tennessee last year to meet the needs of the vaccine rollout, but Banerjee doesnt expect that capacity to go to waste.

If we plan this carefully, we could use this capacity to support COVID vaccinations outside the U.S., which will have a longer ramp down as well as taking care of the new drugs and biologics, he said.

Whats next? Banerjee said he hopes the vaccine rollout will help logistics companies do a better job of transporting temperature-controlled drugs after the pandemic.

How do we make sure we transform the cold chain so that the next time this happens we are way ahead? he said. And use all that learning for the day-to-day shipments of drugs and pharmaceuticals.

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Medically Necessary: Growth of biologics and gene therapies means more cold chain - FreightWaves

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced new data for multiple preclinical programs and a planned R&D Day, which will be held in June 2021.

Collectively, these new preclinical data highlight Tayshas next wave of novel gene therapies that have the potential to impact meaningful patient populations. The promising data underscore our ability to rapidly and reproducibly investigate disease biology, design innovative gene therapies and efficiently advance the development of these drug candidates, said RA Session II, President, Founder and Chief Executive Officer of Taysha. Among the compelling new data, for the first time, we have shown that TSHA-113, an AAV9 gene therapy that utilizes AAV-mediated gene silencing, reduced tau expression in mouse models of human tauopathies. The potential implications of these data are far reaching, and we intend to further evaluate TSHA-113 in additional preclinical studies. The totality of the preclinical data presented today support the fundamental elements of our scientific approach of coupling validated technology with novel targeted payload design while utilizing a proven HEK293 suspension manufacturing process. We believe our deep pipeline and innovative scientific engine hold tremendous potential, and we are poised to continue delivering meaningful value to patients with monogenic CNS diseases.

Todays data demonstrate the breadth, depth and velocity of our development engine as a sustainable pivotal-stage gene therapy company. There are no approved disease modifying therapies for any of the programs in our portfolio and we are encouraged by the results of our gene therapy approach of vectorized RNA and gene replacement therapies across our portfolio, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. We are very excited to further develop TSHA-113 in tauopathies, including Alzheimers disease, MAPT-associated frontotemporal dementia and progressive supranuclear palsy, based on the significant reduction in tau expression demonstrated in transgenic mouse models of human tauopathies. In addition, to date, we have advanced five programs into IND/CTA-enabling studies, including TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN in Lafora disease, TSHA-111-MALIN in Lafora disease, TSHA-112 in APBD and TSHA-119 in GM2 AB variant. We intend to file an IND/CTA for one of these five named programs by the end of 2021. By mid-year, we intend to select a development candidate for Angelman syndrome and obtain interim expression and safety data from confirmatory non-human primate studies by year-end. We remain on track to report Phase 1/2 biomarker data for TSHA-101 in GM2 gangliosidosis in the second half of this year and to provide a clinical and regulatory update for TSHA-120 in giant axonal neuropathy by year-end. Finally, in the second half of the year, we continue to expect dosing of the first patient with CLN1 disease in a Phase 1/2 trial for TSHA-118 under an already open IND, filing an IND/CTA for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome, and filing an IND for TSHA-101 in GM2 gangliosidosis in the U.S. These anticipated clinical and regulatory milestones are expected to be followed by the initiation of Phase 1/2 clinical trials for each of these indications. We look forward to providing additional updates at our R&D Day in June.

TSHA-113 for Tauopathies

Taysha is developing tau-specific microRNA (miRNA) shuttles designed to target tau mRNA for all six isoforms found in the human brain and/or mouse brain. TSHA-113 is an AAV9 capsid that packages these miRNA shuttles and is delivered in the CSF for the treatment of tauopathies.

TSHA-105 for SLC13A5 deficiency

TSHA-105 is a recombinant self-complementary AAV9 vector that expresses the human SLC13A5 protein under the control of a ubiquitous promoter. The drug candidate is being developed for the treatment of SLC13A5 deficiency.

TSHA-106 for Angelman syndrome

TSHA-106 is an intrathecally delivered AAV9 viral vector designed for shRNA-mediated knockdown of UBE3A-ATS, the antisense transcript governing the expression of UBE3A through the paternal allele.

TSHA-112 for Adult Polyglucosan Body Disease (APBD)

TSHA-112 is an intrathecally delivered AAV9 viral vector designed for miRNA-mediated knockdown of the GYS1 gene to treat APBD.

TSHA-111-LAFORIN for EPM2A and TSHA-111-MALIN for EPM2B for Lafora disease

TSHA-111-LAFORIN and TSHA-111-MALIN are intrathecally delivered AAV9 viral vectors designed for miRNA-mediated knockdown of the GYS1 gene to treat Lafora disease.

TSHA-119 for GM2 AB variant

TSHA-119 is a self-complementary AAV9 vector designed to deliver a functional copy of the GM2A gene to treat GM2 AB variant.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210414005278/en/

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Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day - BioSpace

By Abbey Slattery, WRAL Digital Solutions

This article was written for our sponsor, the North Carolina Biotechnology Center.

For decades, North Carolina has been known as a nerve center for the life sciences industry. In fact, after the creation of the Biotech Center in the 1980s, North Carolina became the first state in the country to have a state-supported entity dedicated to growing the industry.

N.C. Biotech runs a number of programs to support technology and company development, to recruit companies and to connect North Carolina residents to high-paying jobs.

In the years since then, numerous life sciences companies have established a base in North Carolina, including major names like Biogen, BASF, LabCorp and Merck, among others. By committing to the industry early and cultivating a strong university system, the state established itself as a hub and prepared a workforce that rivals that of anywhere else in the country. Now, it's the site of global scientific breakthroughs and game-changing medical advancements.

"North Carolina has really done a good job with creating a flow from the workforce to the industry, and we've continued to grow and expand because of that. We now have a lot of research and development tools, and we have a tremendous amount of manufacturing here," said Laura Gunter, executive vice president at NCBIO. "All of the amenities of North Carolina and climate and education attract people, but then we've done a good job of educating folks and giving North Carolinians an opportunity to work and thrive here."

With a highly trained workforce of over 67,000, 775 diverse bioscience companies and nearly 2,500 companies, the state has created a thriving ecosystem for the industry. In the Triangle region alone, agricultural and biopharma manufacturing companies are developing exciting new advancements with potential for global impacts.

"A company called Pairwise in Durham is focused on using the gene-editing technology called CRISPR, but with plants. They're working on developing more nutrients or better growing capabilities with plants using that CRISPR technology," said Gunter. "They've developed a new variety of leafy green called brassica that's related to Brussels sprouts, cauliflower and kale. It's nutrient-dense and naturally resistant to pests, but apparently, it has a very pungent flavor. What they've done is work on the flavor, while keeping the nutrients and resistance."

"Another major company is Novozymes, and they specialize in industrial enzymes and microorganisms. If you've ever toured any of these facilities where they're doing fermentation, like a beer brewing facility, Novozymes does that on a massive scale with industrial enzymes," continued Gunter. "Those enzymes can be used in everything from pharmaceuticals to food to detergent. They're also working on some things on the agriculture side to improve crop yields through microbes."

Biogen founded in 1978 and one of the world's first global biotechnology companies is another major player that finds a home in Durham. While Pairwise and Novozymes are more focused on the agricultural side of biotech, Biogen is making advancements in pharmaceutical manufacturing and medical research programs. Its active research includes treatments for conditions like multiple sclerosis, spinal muscular atrophy and other serious neurological conditions.

The company announced a $200 million expansion in March to produce gene therapies and other treatments in their project pipeline. It joins Novartis Gene Therapies, which is producing its Zolgensma therapy for spinal muscular atrophy in Durham, alongside Adverum, Audentes, Beam Therapeutics, bluebird bio, Cellectis, Pfizer, Taysha Gene Therapies and more.

The benefit of cell and gene therapies is the treatments are potentially curative. Early applications are for diseases caused by single-gene mutations, and those that are life-threatening. Thanks to these companies, North Carolina will be the place these treatments are produced. The treatments would likely have a global impact on improving the lives of those who suffer.

One specific example is spinal muscular atrophy, which is the loss of nerve cells and the weakening of muscles near the spine. This degenerative disease has previously been a fatal diagnosis for infants.

"The fascinating thing about spinal muscular atrophy treatments, in the case of Biogen and Novartis, they're both game-changing and potentially curative treatments versus just trying to alleviate a symptom," said Gunter. "With the gene replacement therapy that Novartis is using, if it works, then that child could potentially no longer have the disease. If it ends up being a long-term fix, it's pretty miraculous and it's made right here."

The list of companies that develop products and treatments in North Carolina goes on from endoscopy devices at Cook Medical to in vitro products at bioMerieux to injectable drugs at Hospira. Thanks to forward-thinking from the state and organizations like N.C. Biotech, North Carolina is well-equipped to continue being the site of life sciences products and advancements.

"North Carolina has paid attention to reinforcing the entire pipeline starting with idea and proof of concept by a university researcher. Small companies are created from the research, and we help them navigate a bumpy road to success. Then, we work with companies all around the world looking for the right place to manufacture their products," said Doug Edgeton, president and CEO of N.C. Biotech.

"We continue to invest in this innovation infrastructure, and train the talent to do these important jobs. We think it's why North Carolina is a global leader in this field."

This article was written for our sponsor, the North Carolina Biotechnology Center.

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From gene therapies to medical treatments, cutting-edge research finds a home in NC - WRAL.com

Promising research at the University of Cambridge suggests gene therapies could help repair some of the damage caused in chronic neurodegenerative conditions such as glaucoma and dementia, writes editor Paul Brackley.

Their studies in animals offer hope that such therapies could be effective in polygenic conditions, which are complex and do not have a single genetic cause.

Gene therapies involve replacing a missing or defective gene with a healthy version.

They have become increasingly common, but typically for rare and monogenic conditions those caused by a single defective gene, such as Lebers congenital amaurosis, spinal muscular atrophy and Lebers hereditary optic neuropathy.

There have been limited applications of such therapies to polygenic conditions, which include the majority of neurodegenerative conditions.

In their new research, Cambridge scientists delivered two candidate molecules simultaneously to nerve cells using a single virus to achieve a strong effect on axonal growth.

Dr Tasneem Khatib, from the John van Geest Centre for Brain Repair at the University of Cambridge, the studys first author, explained: The axons of nerve cells function a bit like a railway system, where the cargo is essential components required for the cells to survive and function.

In neurodegenerative diseases, this railway system can get damaged or blocked. We reckoned that replacing two molecules that we know work effectively together would help to repair this transport network more effectively than delivering either one alone, and that is exactly what we found.

This combined approach also leads to a much more sustained therapeutic effect, which is very important for a treatment aimed at a chronic degenerative disease.

Rather than using the standard gene therapy approach of replacing or repairing damaged genes, we used the technique to supplement these molecules in the brain.

Axons are long fibres that transmit electrical signals, allowing nerve cells to communicate with one another and with muscles.

Axonal transport is a cellular process that moves key molecules and cellular building blocks such as mitochondria, lipids and proteins to and from the body of a nerve cell.

It has been suggested that stimulating this process by enhancing intrinsic neuronal processes in the diseased central nervous system could be a way of repairing damaged nerve cells.

The Cambridge team explored this idea using two candidate molecules for improving axonal function brain-derived neurotrophic factor (BDNF) and its receptor, tropomyosin receptor kinase B (TrkB).

They tested the idea in two models of neurodegenerative disease known to be associated with reduced axonal transport glaucoma and tauopathy, a degenerative disease associated with dementia.

Glaucoma involves damage to the optic nerve and is typically associated with abnormally high pressure in the eye.

Using rats, researchers deployed a tracer dye to show axonal transport between the eye and brain was impaired in glaucoma, while a reduction in electrical activity in the retina in response to light suggested vision was also impaired.

Dr Khatib and colleagues then used viral vectors which are gene therapy delivery systems to deliver the two molecules to the retina of rats. The movement of the dye showed this restored axonal transport between the retina and the brain and the retinas also showed an improved electrical response to light, which is a prerequisite for visual restoration.

Next they used transgenic mice bred to model tauopathy, which is the build-up of tangles of tau protein in the brain.

Tauopathy is seen in neurodegenerative diseases, including Alzheimers disease and frontotemporal dementia.

Injecting the dye again enabled the team to show that axonal transport was impaired between the eye and the brain and that it was restored using the viral vectors.

They even found preliminary evidence of possible improvement in the mices short-term memory using an object recognition task.

Before the treatment, a mouse was placed at the start of a Y-shaped maze and left to explore two identical objects at the end of the two arms.

Shortly after, the mouse was once again placed in the maze, but this time one arm contained a new object, while the other contained a copy of the repeated object.

The researchers measured the amount of the time the mouse spent exploring each object to see whether it had remembered the object from the previous task.

After the viral vector had been injected into the mouses brain, the test was repeated and suggested a small improvement in short-term memory. The result here was not deemed statistically significant, but the researchers were encouraged and plan a larger study to confirm it.

Professor Keith Martin, from the Centre for Eye Research Australia and the University of Melbourne, who led the study while at Cambridge, said: While this is currently early stage research, we believe it shows promise for helping to treat neurodegenerative diseases that have so far proved intractable.

Gene therapy has already proved effective for some rare monogenic conditions, and we hope it will be similarly useful for these more complex diseases which are much more common.

In the study, published in Science Advances, the researchers write: We feel that these findings do have implication for clinical practice.

The research was supported by Fight for Sight, Addenbrookes Charitable Trust, the Cambridge Eye Trust, the Jukes Glaucoma Research Fund, Quethera Ltd, Alzheimer's Research UK, Gates Cambridge Trust, Wellcome and the Medical Research Council.

Read more

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Gene therapy: Discussing the worlds most expensive treatments at Health Horizons Future Healthcare Forum

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University of Cambridge research suggests gene therapies offer hope in treating glaucoma and dementia - Cambridge Independent

Genetic diseases were thought to be incurable letters carved in stone (or on a double helix). But gene therapy could change all that.

From treating Duchenne muscular dystrophy, autism, or glaucoma, there has been a surge in the news surrounding gene therapy (type "gene therapy" into the Freethink search tool, and you'll see endless stories about medical breakthroughs).

The total number of completed or ongoing gene therapy clinical trials reached 4000 in February 2020. This type of treatment is on its way toward addressing some of the most severe and complicated ailments known to man. Gene therapy even shows promise for treating diseases like cancer or Huntington's disease longstanding mysteries of medical science.

Instead of using medications or surgery, doctors can use gene therapy to fight diseases at their source. Gene therapy attempts to cure a disease, or boost the body's ability to combat a disease, by replacing a defective gene or adding a new gene.

The success of gene therapy all hinges on the ability to deliver that precious genetic cargo into the patient's cells safely. To do that, scientists use a "vector" as a delivery vessel for the genetic material. In most cases, that vector is a virus.

Scientists engineer an unwitting virus to act as a vector to deliver a therapeutic genetic payload.

We don't typically think fondly of viruses. Usually, they don't enter the human body to help. Instead, viruses like the flu or coronavirus make humans sick by inserting their genetic material into human cells and hijacking them. The cell reads the viral DNA (or RNA) and produces virus offspring, infecting other cells, repeating the process.

But, since viruses naturally deliver genetic material into a human cell, they are the most widely used vectors in gene therapy.

Scientists engineer an unwitting virus to act as a vector to deliver a therapeutic genetic payload. They strip the virus of its own genes and replace it with new genes. Once the virus transports its genetic cargo into tissues, the altered genes get to work producing further instructions to treat the disease.

But the human body can recognize the virus as a foreign invader and launch an immune response attempting to target and reject the virus.

Enter the adeno-associated viruses (AAV) a small virus that has emerged as the most promising platform for gene therapy.

These small, versatile viruses have several advantages over other gene therapy vectors.

First, despite their small size, they can carry a sizeable genetic payload. They are not known to cause many human diseases, and they can be designed to target particular types of cells or tissues. They cannot replicate on their own, so scientists can control how much gene therapy is delivered to the body.

The best part: they don't trigger a very large immune response like other viruses. A gene therapy trial in the 1990s that used an adenovirus (a kind of cold virus, different from AAVs) caused a deadly immune overreaction in one patient and effectively ended gene therapy research in humans for a decade.

After that, researchers went back to the drawing board, looking for safer vectors. AAVs, because they don't cause disease and can't replicate, generally fly under the immune system's radar. These characteristics make them perfect vessels for gene therapy. And clinical trials have since shown that AAV gene therapy is safe and, for some diseases, effective.

Now that AAVs are established as gene therapy vectors, scientists are working to improve their delivery and create the next generation of AAV gene therapy.

Researchers have been successful with AAV gene therapy in the clinical setting. Currently, two FDA-approved gene therapy treatments use AAVs: Luxturna, which was approved in 2017 for a rare vision disorder, and Zolgensma, which was approved last year for spinal muscular atrophy.

But there are challenges to overcome. Because AAVs are viruses that people are naturally exposed to, many people have already developed antibodies against them. When a patient who has been previously exposed to an AAV receives AAV gene therapy, these antibodies could block the therapy.

Recently, a team of researchers figured out how to cloak the AVV to sneak past the immune system and deliver its gene therapy payload without triggering any immune response.

With the recent surge in AAVs as viral vectors and more clinical studies in the pipeline, we can expect to see new gene therapies that are safe and effective.

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What is AAV Gene Therapy and What Makes it Safe? - Freethink

The collaboration will improve visibility and reporting of cell and gene therapy manufacturing through a pre-integrated data solution

London, UK and Woodcliff Lake, NJ, US, April 14, 2021 ATMPS Ltd, a leader in blockchain-based vein to vein cell orchestration platforms for advanced therapies, and Ori Biotech, an innovator in cell and gene therapy (CGT) manufacturing platforms, today announce a global collaboration to create seamless integration between their respective data platforms to support the development of Advanced Therapies.

The non-exclusive partnership will result in the integration of Oris proprietary CGT manufacturing platforms data architecture with ATMPS HataaliTM, a secure data sharing platform, specially designed as a scalable system for advanced therapies. The combination of the two proprietary platforms will provide improved product tracking, scheduling as well as live monitoring of the manufacturing status across the entire CGT supply chain from vein to vein.

Ori selected ATMPS Hataali technology for integration with our platform to allow the secure sharing of relevant process data with therapy developers, contract development and manufacturing organizations (CDMOs) and even clinicians or patients in real-time. This collaboration will provide increased visibility into the treatment process for those involved at each stage of the treatment process, said Jason C. Foster, CEO, Ori Biotech. The addition of established technologies from companies like ATMPS allows Ori to further its mission of enabling partners across the CGT industry to bring therapies as safely, cost effectively and quickly as possible to large numbers of patients with few alternative therapeutic options.

Hataali encrypts and stores data using distributed ledger technology through a process known as hashing. This process ensures data security is protected through a system of private keys and cryptography. Hataali was the first cell orchestration platforminstalled in a hospital globally and ATMPS the first blockchain company to integrate with the UK National Health Service for the delivery of advanced therapies.

Ori is an ideal partner for ATMPS, as both companies share a common mission to lower the costs and improve processes for cell and gene therapies. Oris work in helping innovators scale from preclinical to commercial is a massive step towards reducing complexity and bringing these vital therapies to patients more quickly, said Raja Sharif, CEO, ATMPS. We all know how challenging and expensive the development and manufacturing of CGTs can be. With the help of Hataali, Ori can securely leverage vein-to-vein data to advance their manufacturing solutions much more efficiently. This will enable real-time therapy tracking, improved patient trust and expediated clinical development.

Ori continues to experience significant growth since closing a $30 million Series A round of funding as it prepares to bring its platform to market. The company is actively recruiting the best talent in cell and gene therapy technology through its networks and through http://www.oribiotech.com.

###

About ATMPS

ATMPS Ltd provides patent pending advanced technical solutions for healthcare companies in the Advanced Therapy Medicines Products sector, including CAR-T treatments, stem cell and gene treatments. ATMPS have a unique distributed ledger solution which provides immutable and incorruptible data and automation capabilities. The solution is operational, configurable and can provide data analytics modules. It can integrate with all LIMS, MES and legacy solutions. It provides full chain of custody, identity and condition of samples, treatments and assets which it tracks. It also schedules and co-ordinates the supply chain participants, as well as giving them information at the same time, in real time, from a "single source of truth. For more information, please visit ATMPS website athttps://www.atmps.net/.

About Ori Biotech

Ori is a London and New Jersey based cell and gene therapy (CGT) manufacturing technology company. Ori has developed a proprietary, flexible manufacturing platform that closes, automates and standardizes CGT manufacturing allowing therapeutics developers to further develop and bring their products to market at commercial scale. The promise of the full stack Ori platform is to fully automate CGT manufacturing to increase throughput, improve quality and decrease costs by combining hardware, software and data services.

Ori was founded by Professor Chris Mason (AVRO) and Dr. Farlan Veraitch (UCL) and has brought together a seasoned Board and executive management team with over 100 years of pharmaceutical, CGT and venture building experience including CEO Jason C. Foster (Indivior), CBO Jason Jones (Miltenyi Biotec), VP Tom Heathman (Minaris), VP David Smith (Minaris) alongside expert advisers like Bruce Levine, Anthony Davies and Annalisa Jenkins. For more information, visit http://www.oribiotech.com.

For media enquiries, please contact:

Alex Heeley or Abdul Khalifeh

De Facto Communications

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ATMPS and Ori Biotech Collaborate on an Integrated Digital Platform for Cell and Gene Therapy Developers - BioSpace

Los Angeles, USA, April 12, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapies Pipeline Insights 2021: Analysis of Key Companies, Emerging Therapies, Recent Happenings and Futuristic Trends

The leading gene-editing companies looking at commercializing CRISPR-based therapeutics are CRISPR Therapeutics, Intellia Therapeutics, and Editas Medicine. CRISPR Therapeutics has the largest market cap of the three, at $10.9B, with a clinical development program that is more advanced than those of Intellia and Editas. Editas Medicine has the smallest market cap of the three companies. Intellia has established high profile collaborations with Regeneron and Novartis.

DelveInsights CRISPR Therapies Pipeline Insight report offers a broad view of available CRISPR therapies in the market, pipeline CRISPR therapies, their MoA, RoA, key companies working in the domain and competitive assessment.

Some of the key takeaways from the CRISPR Therapies Pipeline Report:

Interested in knowing more? Request for the sample @ CRISPR Therapies in the Pipeline

The report underlines the present unmet needs in the market, driving factors and market constraints, along with the holistic view of the inactive therapeutics (comprising dormant and terminated products) with the reasons behind their downfall, detailed insights into the structure and gene editing tool of the pipeline CRISPR therapies to help clients gauge the opportunities and risks in the market.

In the News

Know what is happening in the CRISPR Pipeline Therapies @ CRISPR Pipeline Recent Happenings

What is CRISPR?

CRISPRs (Clusters of Regularly Interspaced Short Palindromic Repeats) are specialized stretches of DNA and are a shorthand for CRISPR-Cas9, which are transcribed by the bacteria to RNA stretches during viral infections. The same CRISPR technology can be leveraged to identify, alter and modify the DNA sequences and genomes.

The technique is used to correct genetic defects, prevent the spread of disease by altering the genetic sequence, improving crop viability and durability, and so on without affecting the functions of other genes.

Want to learn more about the leading candidates in different clinical stages of trials? Reach out @ CRISPR Emerging Therapies and Key Companies

At a Glance: Emerging CRISPR Therapies, RoA, MoA and Companies

Know more about budding CRISPR therapies projected to transform the landscape @ Emerging CRISPR Therapeutics and Market Scenario

CRISPR Therapeutic Assessment The CRISPR Therapies Pipeline report proffers comprehensive insights into active pipeline assets segmented by Stage, Product Type, Route of Administration, Molecule Type, Target and Indications of various drugs.

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By Stage

By Route of Administration

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By Stage and Product Type

To know more, Visit CRISPR CAS-9 Technology and Emerging Trends

Scope of the report

Learn more about the scope and highlights of the report @ CRISPR Pipeline Emerging Drug Pipeline

Key Questions Answered in the Report

Got queries? Get in touch @ CRISPR Technology and Pipeline Therapies

Table of Contents

Know more about report offerings @ CRISPR Pipeline Insights

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CRISPR Gene-Editing and Stem-Cell Technology

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

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CRISPR Therapies Pipeline Insights 2021: Analysis of Key Companies, Emerging Therapies, Recent Happenings and Futuristic Trends - GlobeNewswire

FOSTER CITY, Calif. & PARIS--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Vivet Therapeutics (Vivet) announced today an agreement whereby Mirum has the exclusive option to develop and subsequently commercialize Vivets two proprietary AAV gene therapy programs for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. The two programs, VTX-803 and VTX-802, are currently being evaluated in preclinical studies by Vivet, a privately-held gene therapy biotechnology company.

Under the terms of the agreement, Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs. Until that time, Mirum will provide funding to support the continued research and development costs associated with the two gene therapy programs.

This gene therapy collaboration will work to address the root cause of PFIC3 and PFIC2 and provide an option for patients who do not respond to ASBT inhibition, said Chris Peetz, president and chief executive officer at Mirum. We are encouraged by the expertise and dedication of the Vivet team to develop next-generation gene therapies that have the potential to transform the lives of patients and their families. There are clear synergies in our combined missions to help the people who need it the most.

Mirums dedication to the treatment of cholestatic liver diseases and those rare diseases for which there are limited medications make them a great partner to potentially develop and bring to market VTX-803 and VTX-802, said Jean-Philippe Combal, chief executive officer and co-founder of Vivet. Their leadership in rare liver disease and in particular PFIC, is important as they not only understand the urgent need for patients but also that todays treatment options will never be enough. Gene therapy is a transformative approach, potentially benefiting more patients.

VTX-803 and VTX-802 are two proprietary AAV gene therapy programs of Vivet currently being evaluated in preclinical studies for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. It is thought that successful correction by gene therapy of the defective MDR3 transporter and bile salt export pump (BSEP) functions for PFIC3 and PFIC2, respectively, may ultimately provide a cure for patients living with these rare liver diseases. Such an approach carries the potential to overcome the main limitations of current standard of care for PFIC3 and PFIC2. It may provide long-lasting benefits by restoring physiological bile secretion and preventing severe hepatic complications of the diseases and outweighing its related significant costs.

Vivet has received Orphan Drug Designation for VTX-803 by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Additionally, preclinical proof-of-concept studies highlighting VTX-803 were published in Nature Communications in 2019. The data demonstrated sustained and significant reversal of PFIC3 disease biomarkers in a model of PFIC3.

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirums lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirums marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND/CTA-enabling studies.

Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

About Vivet Therapeutics

Vivet Therapeutics is a clinical stage emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundacin para la Investigacin Mdica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigacin Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain.

Vivets lead program, VTX-801, currently under IND clinical development with the GATEWAY clinical trial, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damages, neurologic symptoms and potentially death.

Vivets second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020.

Vivet is supported by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners.

Please visit us on http://www.vivet-therapeutics.com and follow us on Twitter at @Vivet_tx and LinkedIn.

About PFIC

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe. Six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease The PFIC2 patient population accounts for approximately 60% of the PFIC patient population. PFIC2 is caused by a mutation in the ABCB11 gene, which normally encodes a bile salt export pump protein that moves bile acids out of the liver.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the option and license agreement between Mirum and Vivet and the potential development of VTX-802 and VTX-803. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as plans, will, may, anticipates, expects, potential and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirums current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirums business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirums filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on managements assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Mirum Pharmaceuticals and Vivet Therapeutics Enter into Exclusive Worldwide Option and License Agreement for Vivet's Gene Therapy Programs Targeting...