Around the Helix: Cell and Gene Therapy Company Updates – May 24, 2023

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.

Catch up on CGTLive’s coverage of the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California. Our coverage will continue throughout the week.

The FDA has approved beremagene geperpavec, a topical and redosable gene therapy from Krystal Biotech, for the treatment of dystrophic epidermolysis bullosa in patients 6 months or older. The gene therapy, the first for this indication, will be marketed under the name Vyjuvek.

The FDA has informed Sarepta Therapeutics that it needs more time to discuss final label negotiations and postmarketing commitment for the company’s investigational gene therapy delandistrogene moxeparvovec for the treatment of ambulant individuals with Duchenne muscular dystrophy, pushing back the PDUFA date from May 29 to June 22, 2023.

The FDA has approved AbbVie’s epcoritamab-bysp, a T-cell engaging bispecific antibody marketed as Epkinly, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B–cell lymphoma, after 2 or more lines of systemic therapies, under the accelerated approval pathway, making it the first and only T-cell engaging bispecific antibody for this indication.

VectivBio is investigating treatments for acute Graft versus Host Disease (aGvHD), a condition which can occur in patients receiving allogeneic hemopoietic stem cell transplantation. AGvHD commonly occurs in the gastrointestinal system, which is the focus of Ironwood Pharma’s activities.

AVR-RD-04 is an investigational gene therapy currently being evaluated in a phase 1/2 clinical trial (NCT03897361) for the treatment of cystinosis. The deal between the 2 companies also includes an exclusive license to some specific assets and intellectual property associated with AVROBIO’s gene therapy platform.

The panel is intended to streamline identification of the genetic basis for patients’ neuromuscular disorders in order to inform treatment options. “Getting a timely, accurate diagnosis for a neuromuscular disorder and identifying its causative variants is key to starting a patient on a precision treatment plan and, in some cases, preventing disease progression or the onset of secondary conditions,” Linyan Meng, PhD, the senior division director at Baylor Genetics, said.

As part of a strategic prioritization, the company will suspend the development of several of its preclinical and early research gene therapy programs. PTC Therapeutics’ CEO noted that the company will seek to identify third parties to continue the development of these programs to the extent that it is feasible to do so.

Bespoke Gene Therapy Consortium is managed by the National Institutes of Health and focused on streamlining gene therapy development processes in order to make the pursuit of bringing these therapies to market within the current drug development framework more cost-effective for companies.

As part of the agreement, Life Edit, a subsidiary of Elevate Bio, has the option for global profit share for 1 program related to the collaboration, which is focused on rare disease and cardiometabolic indications. “The advancements we’ve made to our next-generation gene editing platform and base editing capabilities are opening the next frontier of treating disease through DNA editing,” Mitchell Finer, PhD, the CEO of Life Edit Therapeutics and the president of research and development of ElevateBio said.

Adolore Biotherapeutics has incorporated the property portfolio, entitled "Non-Toxic Herpes Simplex Virus Vectors for Efficient Gene Delivery Applications and Complementing Cells for their Production,” into its a development of its CA8* gene-therapy, which is intended to treat chronic pain indications including erythromelalgia and knee osteoarthritis.