StemCell Therapy

Abstract on Rett syndrome treatment through X-reactivation receives Excellence in Research Award

Updated preclinical safety and efficacy data will be presented for ACTX-401, a gene replacement therapy currently in a Phase 1/2 study for the treatment of IGHMBP2-related disorders

LOWELL, Mass., May 2, 2022 /PRNewswire/ -- Alcyone Therapeutics Inc. ("Alcyone"), a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions, today announced the acceptance of four abstracts at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, an event being held May 16-19, 2022, at the Walter E. Washington Convention Center in Washington, D.C. and virtually.

Accepted abstracts include preclinical data from its X-reactivation gene therapy platform, an approach to correct X-linked dominant genetic disorders by reactivating the silenced X chromosome; preclinical safety and efficacy data from its gene replacement platform, which utilizes viral vectors to deliver a functional gene to compensate for a cell's missing or mutated gene; and improved efficiency of AAV gene therapy manufacturing through its platform upstream process, which is scalable and easily transferrable between multiple systems.

"The oral presentations will highlight the potential of Alcyone's discrete gene therapy platforms, X-reactivation for treatment of Rett syndrome and gene replacement for IGHMBP2-related disorders, spinal muscular atrophy with respiratory distress type 1 and Charcot Marie Tooth disease type 2S, which we are advancing in partnership with the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children's Hospital," said PJ Anand, Chief Executive Officer of Alcyone Therapeutics. "We also will have a poster presentation demonstrating the scalability and multi-system transferability of our AAV production process, which is a critical piece in our gene therapy platform development."

Anandcontinued, "Alcyone's multidisciplinaryapproach to addressing complex neurological conditionsis comprised of our partnered novel gene-based therapeutic platform combined with our proprietary CNS precision drug delivery and dosing technology platform, FalconTM,and leverages a scalable CMC process optimized to produce high-quality clinical material. Our three-pronged approach is designed to better address the current challenges of treating people living with severe neurological disorders."

The presentations are listed below, and the full preliminary program is available online on the ASGCT website.

Oral Presentation: A Novel Gene Therapy for Rett Syndrome through Reactivation of the Silent X Chromosome

Oral Presentation Details:

Presenting Author: Kathrin Meyer, Ph.D., Principal Investigator, Nationwide Children's Hospital and Chief Scientific Advisor, AlcyoneSession Title: Novel Therapeutic Targets to Treat CNS DisordersSession Date/Time: Wednesday, May 18, 3:45 5:30 p.m. ETPresentation Time: 4:45 5:00 p.m. ETRoom: Room 202Abstract #: 837

Samantha Powers, Ph.D., from the Center for Gene Therapy at Nationwide Children's Hospital, received the Excellence in Research Award in recognition for presenting one of the top 18 abstracts submitted for the ASGCT 25th Annual Meeting by a postdoctoral fellow or student.

Oral Presentation: Multicenter AAV Gene Therapy Studies for SMARD1/CMT2S Establish Safety and Efficacy in Multiple Animal Models and Pave the Way for Initiation of a Phase I/II Clinical Trial

Oral Presentation Details:Presenting Author: Kathrin Meyer, Ph.D., Principal Investigator, Nationwide Children's Hospital and Chief Scientific Advisor, AlcyoneSession Title: Musculo-skeletal DiseasesSession Date/Time: Monday, May 16, 10:15 a.m. 12:00 p.m. ETPresentation Time: 11:15 11:30 a.m. ETRoom: Salon GAbstract #: 33

Poster Presentation: Development of an Upstream Process and Analytics for AAV Manufacturing

Poster Presentation Details:Presenting Author: Desyree Jesus, Ph.D., Associate Director, CMC Analytics, AlcyoneSession Title: Vector Product Engineering, Development or Manufacturing IIISession date/time: Wednesday, May 18, 5:30 6:30 p.m. ETRoom: Hall DPoster Board #: W-286Abstract #: 1160

Poster Presentation: Evaluation of AAV9 Gene Therapy for SMARD1/CMT2S in Different Mouse Models Reveal Differences in Efficacy Dependent on Promoter Choice

Poster Presentation Details:Presenting Author: J. Andrea Sierra Delgado, M.D., M.Sc., Chief Research Associate in Dr. Kathrin Meyer's Lab, Nationwide Children's HospitalSession Title: Musculo-skeletal DiseasesSession Date/Time: Wednesday, May 18, 5:30 6:30 p.m. ETRoom: Hall DPoster Board #: W-198Abstract #: 1072

About Alcyone TherapeuticsAlcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The Company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the Company's proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone's lead programs utilize X-chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMPB-2 related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). For more information, visit http://www.alcyonetx.com.

About Alcyone's Strategic Collaboration with the Abigail Wexner Research Institute at Nationwide Children's HospitalAlcyone works closely with scientists from the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI) in Columbus, Ohio, where four discrete gene therapy platform technologies, each with novel and differentiated mechanisms of action, including X-reactivation, conventional transgene replacement, vectorized exon skipping, and promoter modulation were designed, developed, and are being advanced towards the clinic. Alcyone has optioned the four programs and is funding research to explore the potential for the clinical application of these therapeutics using FalconTM, its proprietary CNS precision drug delivery and dosing technology platform, to improve the lives of people impacted by severe neurological conditions. The research is led by Kathrin Meyer, Ph.D., and Nicolas Wein, Ph.D., Principal Investigators in the Center for Gene Therapy at AWRI. Both Dr. Meyer and Dr. Wein sit on Alcyone's Scientific Advisory Board (SAB), with Dr. Meyer serving as Chief Scientific Advisor and Chair of Alcyone's SAB.

SOURCE Alcyone Therapeutics

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Alcyone Announces Two Oral Presentations on its Gene Therapy Platforms at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting -...

BOSTON, May 02, 2022 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, announced today that it will present at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting, being held virtually and in Washington D.C. May 15-19, 2022.

The Company will present on its lead investigational gene therapy, DB-OTO, being developed to restore hearing to individuals with a mutation in the otoferlin gene. Decibel will also present two posters featuring its AAV.104 program, a gene therapy designed to restore hearing to individuals with a mutation in the stereocilin gene, and its AAV.103 program, a gene therapy designed to restore hearing to individuals with a GJB2 deficiency, the most common cause of congenital hearing loss.

Details for the oral presentation are as follows:

Development of an AAV-Based Gene Therapy for Children with Congenital Hearing Loss Due to Otoferlin Deficiency (DB-OTO)Oral Abstract Session: Pharmacology/Toxicology Studies or Assay Development IIPresenter: Orion Keifer Jr, M.D., Ph.D., Senior Medical Director, Decibel TherapeuticsDate & Time: Thursday, May 19, 2022 at 10:45 am ET

Details for the poster presentations are as follows:

M-185 | Dual Vector Mediated Gene Therapy for Restoration of STRC-Related Hearing LossPoster Session: Ophthalmic and Auditory DiseasesDate & Time: Monday, May 16, 2022 at 5:30 pm ET

M-183 | Identification of GJB2s Upstream Regulatory Elements Facilitates Design of Safe, Precision AAVs and Recovery of Hearing in a GJB2-Deficient Mouse ModelPoster Session: Ophthalmic and Auditory DiseasesDate & Time: Monday, May 16, 2022 at 5:30 pm ET

About Decibel TherapeuticsDecibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibels pipeline, including its lead gene therapy product candidate, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of creating a world of connection for people with hearing and balance disorders. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow us on Twitter.

Investor Contact:Julie SeidelStern IR, Inc.212-362-1200Julie.seidel@sternir.com

Media Contact:Chris RaileyTen Bridge Communications617-834-0936chris@tenbridgecommunications.com

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Decibel Therapeutics to Present at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting - GlobeNewswire

One of the most important systems in our bodies is the immune system. It defends our bodies from germs, infections, bacteria, viruses, and more. But, would you have imagined that about 1 in 58,000 babies are born with little to no Immune system? This is a very rare genetic disorder called Severe Combined Immunodeficiency (SCID). A baby can be diagnosed with SCID before birth through screenings, but most babies are diagnosed with SCID within the first six months of life.

When a child has SCID, it means they lack T cells, Natural Killer Cells, and Functional B cells. T Cells help protect the body from infection and help fight cancer. Natural Killer cells destroy cells infected with a virus. Functional B cells produce antibodies to fight bacteria and viruses.

The process of treating or curing a disease by altering a persons genes is known as gene therapy. In two previous studies conducted, SCID gene therapy consisted of two generations, both using viruses to deliver the genes.The first generation of treatment worked, but patients unfortunately developed leukemia, a cancer of the white blood cells that makes them abnormally large. The research community did deliver a second generation of gene therapies that were safer, but they did not completely restore the immune system.

To treat infants with SCID, stem cells are taken from the bone marrow of siblings, parents, or unrelated donors. Then, a bone marrow transplant introduces these healthy infection-fighting cells into the SCID infants body. The idea is that this will provide a new immune system for the patient. Generally, bone marrow stem cell transplants from family donors are effective but unavailable for more than 80% of patients in the world. This means a higher risk of the non-family donors T cells attacking and damaging the patients healthy cells.

In this study, the researchers used a type of gene therapy involving a lentiviral vector. A lentiviral vector is a type of virus called a lentivirus that inserts its RNA into the hosts cells. They took advantage of this viruss action to insert a corrected gene sequence, as an RNA strand, into the patients own bone marrow stem cells to both fix the genetic error and reduce the chance of rejection. The gene of interest is IL2RG, which instructs the body to make certain immune proteins in the bone marrow. When this gene is broken, SCID results.

The research team had successfully tried this type of treatment before in children and young adults with SCID. They combined the lentiviral vector gene therapy with a chemotherapy agent called nonmyeloablative busulfan, typically given to patients before a stem cell transplant. This drug destroys a patients bone marrow cells in preparation for new stem cells. The researchers hypothesized that lentiviral gene therapy, after a low dose of this drug busulfan, would be a safe and effective treatment for infants with recently diagnosed SCID.

First, the infants bone marrow was collected. The correct gene was inserted into the patients blood stem cells using the lentivirus vector or carrier. The cells were then frozen and went through quality testing in order to detect, reduce, and correct any problems that may have occurred. Importantly, the lentiviral vector contained protectors that prevented the gene therapy from accidentally causing leukemia. The protectors work by blocking the virus from turning on certain oncogenes (or cancer-linked genes) that happen to sit next to the IL2RG gene on the chromosome.

They recruited a group of eight infants newly diagnosed with SCID. The researchers conducted their experiment by giving the infants one to two daily doses of busulfan by injection. They customized the initial dose based on the weight and age of the patient and previous knowledge on how this drug typically moves through the body.

The results of the experiment supported the research teams prediction. Natural Killer cells were restored within the first four months in seven of the eights infants as the T cells. The eighth infant initially developed a low T cell count but improved after a boost of gene-corrected cells without needing additional busulfan pre-treatment. Several types of blood cells such as T, B and natural killer cells made in the bone marrow seemed active within 3-4 months after infusion with the viral lentiviral vector.

The combination of lentiviral gene therapy with busulfan conditioning appeared safe in all eight infants. These results aligned with what the researchers expected. Patients were followed for a range of 6-24 months after the study to assess whether their new immune system remained stable. After the 24 month period, they concluded that this treatment was more effective than current treatments for SCID patients with fewer side effects.

Originally posted here:
New treatment for infants with weakened immune systems - Sciworthy

Selecta Biosciences, Inc.

Data to be featured in two oral presentations and four poster presentations

WATERTOWN, Mass., May 02, 2022 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases, power gene therapies and mitigate unwanted immune responses to biologics, today announced six upcoming presentations, including three joint presentations with our partner AskBio, at the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually and in-person from May 16-19, 2022 in Washington, D.C. These presentations demonstrate the power of Selectas immune tolerance platform, ImmTOR, in mitigating unwanted immune responses to AAV capsids and potentially enabling gene therapy re-dosing for patients with severe genetic disorders.

We are thrilled to present data demonstrating ImmTORs ability to inhibit anti-AAV antibody formation and potentially enable re-dosing of gene therapies. Excitingly, in a first-in-human clinical trial conducted with our partner AskBio, we demonstrated the ability of ImmTOR to blunt the early immune response to empty AAV8 capsids said Dr. Kei Kishimoto, Ph.D., Chief Scientific Officer of Selecta. Furthermore, we are pleased to showcase results indicating an evolution of our ImmTOR platform is close at hand. Combining ImmTOR with engineered T-reg specific IL-2 mutein (ImmTOR-IL) synergistically enhances the magnitude and durability of mitigation of anti-AAV antibody formation in preclinical studies. We look forward to initiating our Phase 1 clinical trial for the treatment of methylmalonic acidemia, building on these data, and continuing our efforts to help patients overcome autoimmunity and immunogenicity with our precision immune tolerance therapies.

Details and key takeaways from ASGCT presentations are as follows:

Oral Presentations:

Selecta Presentation Title: Mechanisms, Monitoring, and Mitigation of Host Immune Responses to AAV Gene Therapy VectorsSession Title: Immune Responses to Gene TherapyPresenter: Kei Kishimoto, Ph.D., Selecta BiosciencesPresentation Date and Time: Monday, May 16, 2022, 2:20 - 2:45 p.m. ETKey takeaways: General overview of the various ways that the immune response affects the efficacy and safety of AAV gene therapy and development of ImmTOR to mitigate these effects.

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Selecta & AskBio Presentation Title: Functional Assessment of T Cell Responses to AAV8 Empty Capsids in Healthy VolunteersSession Title: Immune Responses to AAV VectorsAbstract Number: 37Presenter: Shari Gordon, AskBioPresentation Date and Time: Monday, May 16, 2022, 10:30 - 10:45 a.m. ET Key takeaways: This study demonstrates for the first time that empty AAV capsids, a hidden component of all AAV gene therapies, are highly immunogenic in humans.

Poster Presentations:

Selecta Presentation Title: ImmTOR Combined with B Cell-Targeted Therapies Provides Synergistic Activity in Mitigating Anti-AAV Capsid Antibody Responses and Enables Repeated Vector DosingSession Title: Immunological Aspects of Gene Therapy and Vaccines IIPoster: W-255Abstract Number: 1129Presenter: Petr Ilyinskii, Selecta BiosciencesPresentation Date and Time: Wednesday, May 18, 2022, 5:30 - 6:30 p.m. ETKey takeaways: The combination of ImmTOR and B cell-targeted therapies act synergistically to inhibit anti-AAV antibody response to enable repeated dosing, including vectors doses up to 5E13 vg/kg.

Selecta Presentation Title: Combination of ImmTOR Tolerogenic Nanoparticles and IL-2 Mutein Synergistically Inhibits the Formation of Anti-AAV AntibodiesSession Title: Immunological Aspects of Gene Therapy and Vaccines IIPoster: W-256Abstract Number: 1130Presenter: Kei Kishimoto, Ph.D., Selecta BiosciencesPresentation Date and Time: Wednesday, May 18, 2022, 5:30 - 6:30 p.m. ETKey takeaways: ImmTOR-IL, the combination of ImmTOR and Treg-selective IL-2 mutein, show profound synergistic effects on inducing antigen-specific Treg and enable more durable inhibition of anti-AAV antibody response at vectors doses up to 5E13 vg/kg.

Selecta & AskBio Presentation Title: Effect of Tolerogenic ImmTOR Nanoparticles on the Formation of Anti-AAV8 Antibodies in Mice, Nonhuman primates, and Healthy Human VolunteersSession title: Immunological Aspects of Gene Therapy and Vaccines IPoster: Tu-216Abstract Number: 711Presenter: Peter Traber, M.D., Selecta BiosciencesPresentation Date and Time: Tuesday, May 17, 2022, 5:30 - 6:30 p.m. ETKey takeaways: A single dose of ImmTOR is shown to mitigate the early anti-AAV neutralizing antibody response in humans and preclinical studies indicate that antibody inhibition can be sustained with multiple doses of ImmTOR.

Selecta & Askbio Presentation Title: ImmTOR Blunts AAVrh32.33 Capsid-specific Immune Responses in C57BL/6 Albino MiceSession Title: Immunological Aspects of Gene Therapy and Vaccines IPoster: Tu-213Abstract Number: 708Presenter: Md Mahmudul Hasan, AskBioPresentation Date and Time: Tuesday, May 17, 2022, 5:30 - 6:30 p.m. ETKey takeaways: ImmTOR is shown to inhibit capsid-specific CD8 T cell responses, which in human clinical trials have been associated with serum transaminase elevation and loss of transgene expression.

Following the conference, the presentations will be available in the Resources section of Selectas website at http://www.selectabio.com/resources/.

About Selecta Biosciences, Inc.Selecta Biosciences, Inc. is a clinical stage biotechnology company leveraging its ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the completion and anticipated proceeds of the proposed offering. All such forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties related to fluctuations in Selectas stock price, changes in market conditions and satisfaction of customary closing conditions related to the offering, as well as the other factors discussed in the Risk Factors section in Selectas most recently filed Annual Report on Form 10-K as well as other risks detailed in Selectas subsequent filings with the Securities and Exchange Commission. There can be no assurance that Selecta will be able to complete the proposed offering on the anticipated terms. All information in this press release is as of the date of the release, and Selecta undertakes no duty to update this information unless required by law.

For Investors:Bruce MackleLifeSci Advisors, LLCPhone: +1-929-469-3859Email: bmackle@lifesciadvisors.com

For Media:Brittany Leigh, Ph.D.LifeSci Communications, LLCPhone: +1-646-751-4366Email: bleigh@lifescicomms.com

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Selecta Biosciences Announces Six Presentations at the Upcoming 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) - Yahoo...

Research details assessment and selection of AAV regulatory cassettes that drive optimal expression of Company's novel engineered ligand-gated ion channel for the treatment of focal epilepsy

Findings inform selection process of final development candidate

SOUTH SAN FRANCISCO, Calif., May 5, 2022 /PRNewswire/ -- CODA Biotherapeutics, Inc. ("CODA"), a preclinical-stage biopharmaceutical company developing a gene therapy-mediated chemogenetic platform to treat intractable neurological disorders, today announced that preclinical data from the Company's focal epilepsy program will be featured as a poster presentation at the American Society of Gene & Cell Therapy 25th Annual Meeting being held virtually and in person, May 16-19, 2022, in Washington, D.C.

CODA Biotherapeutics, Inc. (PRNewsfoto/CODA Biotherapeutics, Inc.)

CODA's approach to treat focal epilepsy is adeno associated virus (AAV)-mediated delivery of a novel engineered ligand-gated ion channel (LGIC) that can subsequently respond to an exogenous ligand. Oral administration of this small molecule ligand, which is designed to only interact with CODA's chimeric LGIC, can be finely tuned to control the aberrant activity of neurons and suppress seizures without adverse effects. To create an effective gene delivery approach for focal epilepsy, the Company generated a series of AAV expression cassettes that could drive optimal expression of its LGIC in target cells of the hippocampus.

Michael Narachi, president and CEO of CODA, said, "Focal epilepsy is a chronic, debilitating neurologic disorder that is characterized by unpredictable seizures initiated from a specific location in the brain and affects millions of people around the world. Recurrent seizures can result in cognitive and emotional deficits, with current interventions offering limited efficacy and multiple side effects. This research details the analysis and selection process of AAV regulatory cassettes that should drive optimal expression of our LGIC for the treatment of focal epilepsy. We look forward to advancing our assessment of these two selected cassettes in epilepsy models to inform the final decision of our development candidate in focal epilepsy."

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Title: Assessment of AAV Regulatory Cassettes with Optimal Hippocampal Neuron Expression for the Treatment of Focal EpilepsyDate: May 16, 2022 5:30-6:30 PM ETSession: Neurological Diseases IPoster Board Number: M-158Abstract Number: 277Location: Hall D, Walter E. Washington Convention CenterPresenter: Edward Yeh

The first round of selection was performed in vitro with 23 unique expression cassettes containing various combinations of regulatory elements including, enhancer, promoter, intron, 5' untranslated region, 3' untranslated region, and polyA tail. Plasmids with inverted terminal repeats (ITRs) flanking the regulatory cassettes driving LGIC expression were transfected in SKNAS neuroblastoma cells, as well as HeLa cells to determine which cassettes were able to target the cells more efficiently and provide high levels of LGIC expression. Analysis by Droplet Digital PCR (ddPCR) and ELISA showed varying levels of expression, with the highest observed in cassettes containing the cytomegalovirus (CMV) promoter followed by those with various tissue-specific promoters. For the second round of selection, nine expression cassettes chosen from the first round were packaged into AAV9 vectors and transduced in neonatal rat hippocampal mixed cultures. Analysis by ddPCR revealed high levels of LGIC mRNA expression from CaMKII- and Syn-driven cassettes, which were comparable to the strong, ubiquitous CAG promoter. CaMKII and Syn are both excitatory neuron-specific promoters.

Finally, the highest expressing cassettes from round two were assessed in vivo in male Sprague Dawley rat hippocampi by AAV9-mediated directed injection. mRNA analysis using ddPCR and immunofluorescence for cellular tropism demonstrated high levels of expression from human versions of the Syn- and CaMKII-driven cassettes. Further assessment of the two selected cassettes, one driven by the pan-neuronal hSyn promoter and the other by CaMKII promoter, will be performed in an animal model of focal epilepsy to finalize the development candidate.

Abstracts can be accessed via the conference website at annualmeeting.asgct.org

First Applications of the CODA Platform: Focal Epilepsy and Chronic Neuropathic PainEpilepsy is one of the most common chronic neurological diseases and, according to the Centers for Disease Control, affects more than 65 million people around the world of which 3.4 million are in the U.S. Epilepsy is characterized by unpredictable seizures and the term "focal" epilepsy is used to describe seizures that initiate from a specific location in the brain, typically in one hemisphere. Focal epilepsy represents approximately 60 percent of all epilepsy (National Institute of Neurological Disorders and Strokes). According to the World Health Organization, recurrent seizures disrupt normal brain functions, lead to neuronal loss, and result in cognitive and emotional deficits. Patients suffer from stigmatization, social isolation, combined with disability, educational underachievement, and poor employment outcomes. The Epilepsy Foundation estimates that one-third of people with epilepsy live with uncontrollable seizures because no available treatments are effective.

According to a study published in the Journal of Pain Research, 10 percent of the U.S. population suffers from neuropathic pain an estimated 30 million Americans. Neuropathic pain is caused by damage or disease of the sensory system, leading to chronic debilitation and loss of quality of life. Current pharmacological therapies for chronic neuropathic pain, such as opioids, anticonvulsants, and tricyclic anti-depressants, are not always effective and can have side effects, including the potential for addiction.

About the CODA PlatformCODA Biotherapeutics is developing a paradigm-shifting gene therapy approach by deploying a chemogenetic strategy for treating neurological disorders. The Company's innovative treatment aims to modulate specific neuronal circuits via adeno-associated virus (AAV)-mediated delivery of an engineered, inhibitory receptor by standard-of-care neurosurgical procedures. The receptor is designed to be quiescent in the transduced cells but will specifically and dose-dependently inhibit neurons when exposed to a novel, orally bioavailable small-molecule agonist. CODA expects this treatment will produce substantially improved and durable results while potentially avoiding off-target/adverse effects of currently available treatments.

About CODA Biotherapeutics CODA Biotherapeutics, Inc., is a preclinical-stage biopharmaceutical company developing an innovative gene therapy platform to treat intractable neurological disorders, with an initial focus on epilepsy, movement disorders, and neuropathic pain. The Company is creating the ability to control neurons with its revolutionary chemogenetics-based technology. CODA is located in South San Francisco, CA. For more information, please visit http://www.codabiotherapeutics.com.

Cision

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SOURCE CODA Biotherapeutics, Inc.

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CODA Biotherapeutics Announces Preclinical Data From Epilepsy Program to be Presented at American Society of Gene & Cell Therapy 25th Annual...

Gene Therapy Cell Culture Media Market research report is the new statistical data source added by Research Cognizance.

Gene Therapy Cell Culture Media Market is growing at a High CAGR during the forecast period 2022-2029. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

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Gene Therapy Cell Culture Media Market Scope and overview, To Develop with Increased Global Emphasis on Industrialization 2029 Fujifilm Holdings...

By now, the complexity of COVID-19 is well known, if not well understood. One of the mysteries is so-called brain fog, or cognitive issues such as confusion or forgetfulness that occur as a result of Long Covid. New research is quantifying this. Heres a look at that story and other research news.

COVID-19 Cognitive Impairment Equal to 20 Years of Aging

A study from theUniversity of CambridgeandImperial College Londonfoundthat cognitive impairment caused by severe COVID-19 is similar to that sustained between 50 and 70 years of age. Another way of putting it is that it was equal to losing 10 IQ points. The research came out of the NIHR COVID-19 BioResource and found the effects were still detectable more than six months after acute illness, and recovery is at best gradual. The research was published inThe Lancet'spublicationeClinicalMedicine.

Cognitive impairment is common to a wide range of neurological disorders, including dementia, and even routine aging, but the patterns we saw the cognitive fingerprint of COVID-19 was distinct from all of these, said Dr. David Menon, M.D. from the Division of Anaesthesia at the University of Cambridge, the studys senior author. We followed some patients up as late as ten months after their acute infections, and were able to see a very slow improvement. While this was not statistically significant, it is at least heading in the right direction, but it is very possible that some of these individuals will never fully recover.

The reason behind the cognitive deficits seen with COVID-19 is not well understood. One theory is that direct viral infection may be to blame, but scientists dont believe it is a major cause. They think it is more likely that a combination of factors is involved, including poor oxygen and blood supply to the brain, blockage of large or small blood vessels from clotting and microscopic bleeds. Emerging evidence suggests the most significant factor may be damage from the bodys own inflammatory and immune response.

Repairing Tendons with Silk Proteins

Tendon injuries take a long time and often dont heal completely. Some of this is related to the relative lack of blood flow to the tendons and the fact they are soft tissues connected to stiff bones. Damage often changes the connective tissue from a linear to a kinked formation.

Investigators with the Terasaki Institute for Biomedical Innovationtestedthe use of biomaterial scaffolds to generate new tendon tissue. They started with silk fibroin, a silk protein generated by the Bombyx mori silkworm. It is used in silk fabrics and optical and electrical devices, and in several biomedical applications, including sutures and bioengineered ligaments, bone and corneal tissue.

They paired silk fibroin with GelMA, a gelatin-based, water-retaining gel. The mixtures were seeded with mesenchymal stem cells. They found that cell viability and proliferation and MSC gene activity were significantly increased, showing more than an 80% attachment rate. They also tested them on live rats with injured Achilles tendons, and the new technology accelerated healing with reduced injury sites and the formation of well-aligned, densely packed tendon fibers and remodeled muscle components.

Decreased Sedentary Time Mitigates Risk of Type 2 Diabetes & CV Disease

Research from the University of Turku in Finlandfoundthat decreasing the amount of time youre sedentary can mitigate the risk of type 2 diabetes and cardiovascular diseases. This is a slightly different lens than the association of exercise with decreased risk of diabetes and CV disease in that it looked at reducing the intervention groups time sitting by one hour per day by increasing standing and light-intensity physical activity.

The control group was to keep their typical habits and sedentary lifestyle. The groups were measured by wearing accelerometers for the entire three-month period. The intervention group decreased sedentary time by 50 minutes per day on average, typically by increasing the amount of light- and moderate-intensity physical activity. The study observed benefits in health outcomes associated with blood sugar regulation, insulin sensitivity and liver health.

Nanotechnology Visualizes RNA Structure at Near-Atomic Resolution

Scientists at the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical Schooldevelopeda new approach, ROCK, that uses an RNA nanotechnological technique to visualize RNA structure at near-atomic resolution. ROCK stands for RNA oligomerization-enabled cryo-EM via installing kissing loops. It allows for assembly of identical RNA molecules into a highly organized structure. This decreases the individual RNA molecules flexibility and increases their molecular weight.

It allows for structural analysis of the contained RNA subunits using the cryo-electron microscopy (cryo-EM) technique. For proof-of-principle, they focused on a large intron RNA from Tetrahymena, a single-celled organism, and a small intron RNA from Azoarcus, a nitrogen-fixing bacterium in addition to the FMN riboswitch.

Autism-Linked Mutation Reversed in Brain Organoids with Gene Therapy

A study out of the University of California San Diego School of Medicineutilizedhuman brain organoids to test a gene therapy. A number of neurological and neuropsychiatric diseases, including autism spectrum disorders (ASD) and schizophrenia, are linked to Transcription Factor 4 (TCF4) mutations. TCF4 is an essential gene in brain development. The researchers focused on Pitt-Hopkins Syndrome, an ASD caused by mutations in TCF4.

Existing mouse models of the syndrome dont accurately mimic patients neural characteristics. As a result, the research team created a human research model of the disorder using stem cells to convert patients skin cells to develop into three-dimensional brain organoids. They found that the TCF4 mutation led to downstream dysregulation of SOX genes and the Wnt pathway. These molecular signals guide embryonic cells to multiply, mature into neurons, and migrate to the appropriate brain location.

They then tested two different gene therapies for recovering the functional gene in brain tissue. Both effectively increased TCF4 levels and corrected Pitt-Hopkins Syndrome characteristics at molecular, cellular and electrophysiological levels.

The fact that we can correct this one gene and the entire neural system reestablishes itself, even at a functional level, is amazing, Alysson R. Muotri, Ph.D. said, professor at UC San Diego School of Medicine, director of the UC San Diego Stem Cell Program and member of the Sanford Consortium for Regenerative Medicine.

The work took place at a prenatal stage of brain development. Children diagnosed with this syndrome do so typically several years later in the clinic. Clinical trials would be required to confirm if a later intervention would be safe and effective. The research group is currently optimizing its licensed gene therapy tools for a trial, which would use spinal injections of the genetic vector in hopes of recovering TCF4 brain function.

Originally posted here:
Research Roundup: COVID-19 Cognitive Impairment Equal to 20 Years of Aging - BioSpace

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

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...

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

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.

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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