Around the Helix: Cell and Gene Therapy Company Updates – July 5, 2023

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Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

CGTLive Around the Helix

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive™’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. FDA Approves Lantidra Cell Therapy for Type 1 Diabetes

The FDA has approved CellTrans’ donislecel allogeneic pancreatic islet cell therapy under the name Lantidra, for treating adults with type 1 diabetes (T1D) who are unable to approach target glycated hemoglobin because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. Donislecel is designed to secrete insulin, removing the need to take insulin to control blood sugar levels in some patients with T1D.

2. FDA Approves BioMarin’s Hemophilia A Gene Therapy Roctavian

The FDA has approved BioMarin’s valoctocogene roxaparvovec (val-rox) for the treatment of adults with severe hemophilia A defined as congenital factor VIII (FVIII) deficiency with FVIII activity of less than 1 IU/dL who do not have antibodies to adeno-associated virus serotype 5 (AAV5) according to an FDA-approved test. Val-rox will be marketed in the United States under the name Roctavian; it is the first gene therapy for hemophilia A to be approved in the US. BioMarin noted that it will offer an outcomes-based warranty for val-rox to insurance companies in the United States that includes a provision for full reimbursement in the event that a patient's disease does not respond to the treatment.

3. Silence and Replace Gene Therapy Cleared for Oculopharyngeal Muscular Dystrophy Clinical Trial

Benitec Biopharma has received FDA clearance for its investigational new drug (IND) application for BB-301, an investigational AAV vector-based silence and replace gene therapy intended to treat oculopharyngeal muscular dystrophy (OPMD)-related dysphagia. Benitec Biopharma is planning to initiate a phase 1b/2a clinical trial to evaluate BB-301. The clinical trial will enroll patients from a natural history study of patients with OPMD that is currently being conducted by the company.

4. Parkinson Disease Neuronal Cell Therapy Demonstrates Safety, Cell Survival

BlueRock Therapeutics has announced that its phase 1/2 clinical trial (NCT04802733) of bemdaneprocel (BRT-DA01) cell therapy has yielded positive topline results in treated participants with Parkinson disease. The detailed data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders taking place in Copenhagen from August 27 to 31, but BlueRock has so far reported that bemdaneprocel was well-tolerated in all 12 patients treated to date and no serious adverse events have been observed, meeting the study’s primary endpoints.

5. Forge Biologics to Provide CDMO Services to New Hope Research Foundation 

Forge Biologics will serve as a contract development and manufacturing organization (CDMO) for New Hope Research Foundation’s NHR01, an investigational AAV vector-based gene therapy intended to treat patients with GM2 gangliosidosis, including Tay-Sachs disease. AAV process development, analytical services, and current good manufacturing practice manufacturing for NHR01’s clinical development will be among the tasks carried out by Forge in the new partnership.

6. Cambrian Bio Aims to Improve CAR-T Persistence With New Pipeline Company Telos Biotech

Telos Biotech, a company launched by Cambrian Bio, is developing a novel recombinant protein referred to as Telovance that is intended to be applied during ex-vivo manufacturing of chimeric antigen receptor T-cell (CAR-T) therapy products to lengthen telomeres in the CAR T-cells. Telovance is expected to help overcome issues with efficacy and persistence in CAR-T products derived from T-cells with critically shortened telomeres.

7. Sigilon Therapeutics to Become Subsidiary of its Partner Eli Lilly 

Sigilon Therapeutics, which is engaged in collaboration with Eli Lilly and Company on cell therapy products, including SIG-002, has entered into a new agreement to be acquired by its collaborator. Following the acquisition, Eli Lilly will continue to advance the development of SIG-002, which is intended to treat T1D. "By combining Sigilon's talent and expertise in cell therapy with the knowledge and skills of Lilly's research and development teams, we will enhance opportunities to create innovative islet cell therapy solutions to improve the care of people living with diabetes,” Ruth Gimeno, PhD, the group vice president in diabetes, obesity, and cardiometabolic research at Eli Lilly, said in a statement.

8. CytoMed and MD Anderson Join Forces to Develop Gamma Delta T-cell Therapies

CytoMed Therapeutics and the University of Texas MD Anderson Cancer Center will evaluate the former’s allogenic gamma-delta T-cells in preclinical studies for their potential to treat acute myeloid leukemia and breast cancer indications. "Interest in the potential of our allogeneic immunotherapy platform to provide additional treatment options has been strong and international collaborations, like the current collaboration with MD Anderson Cancer Center, are a key part of our strategy to maximize our impact,” Tan Wee Kiat, PhD, the chief operating officer of Singapore-based CytoMed, said in a statement.

9. Sarepta Therapeutics Sells Priority Review Voucher Obtained From Elevidys Approval

Sarepta Therapeutics announced that it has sold the rare pediatric disease priority review voucher obtained via the recent approval of Duchenne muscular dystrophy gene therapy delandistrogene moxeparvovec, which will be marketed under the name Elevidys, to an undisclosed buyer. The funds from the sale, which resulted in a $102 million payment to Sarepta, will be used to support the development of other therapies by the company.

10. Bespoke Gene Therapy Consortium Will Financially Support Development of Gene Therapy Candidates at UT Southwestern and Children’s Health

Three clinical trials evaluating treatments for spastic paraplegia type 50, multiple sulfatase deficiency, and Charcot-Marie-Tooth disease type 4J, respectively, will be the targets of the funding. “By creating an open-access playbook for how to initiate and carry out gene therapy clinical trials for these types of rare diseases, Bespoke Gene Therapy Consortium will make this process substantially easier,” Steven Gray, PhD, an associate professor of pediatrics, molecular biology, and neurology in the Eugene McDermott Center for Human Growth and Development and director of UT Southwestern’s Translational Gene Therapy Core, said in a statement.

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Maria Escolar, MD, the chief medical officer of Forge Biologics
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies
Erika Fullwood Augustine, MD, MS, the associate chief science officer of the Kennedy Krieger Institute
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Casey Maguire, PhD, associate professor of neurology and associate investigator of neurology, Harvard Medical School
Evan Weber, PhD, an assistant professor of pediatrics at Children's Hospital of Philadelphia
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