Around the Helix: Cell and Gene Therapy Company Updates – December 11, 2024

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. New Clinical Data Presented at ASH’s 66th Annual Meeting

Catch up on CGTLive®’s coverage of the 2024 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 7-10, in San Diego, California. Our coverage will continue throughout the week.

2. Cartesian Therapeutics’ mRNA CAR-T Descartes-08 Produces Durable Responses in Myasthenia Gravis

Cartesian Therapeutics’ Descartes-08, an investigational autologous mRNA-engineered chimeric antigen receptor T-cell therapy (CAR-T) therapy for the treatment of autoimmune diseases including myasthenia gravis (MG), has produced durable responses in some patients with MG treated in the phase 2b portion of a clinical trial (NCT04146051).

3. HIV/AIDS Awareness Month 2024: Looking Back at Progress in Gene Therapy and Gene Editing

In honor of HIV/AIDS Awareness Month, observed annually in December by the patient and clinician communities, CGTLive® took a look back at some of our coverage of gene therapy and gene editing approaches in HIV over the past year.

4. Gene Therapy RP-A501 Shows Promising Phase 1 Results in Danon Disease

Rocket Pharmaceuticals has announced the long-term safety and efficacy results from the phase 1 RP-A501 study (NCT03882437) of its investigational gene therapy RP-A501, noting that the treatment was well tolerated on the whole, and that all the patients with Danon disease demonstrated sustained LAMP2 protein expression after 1 year and up to 60 months, among other positive indicators.

5. CMS Aligns With Vertex Pharmaceuticals and bluebird bio for Outcomes Based Agreements for Sickle Cell Gene Therapy

The Centers for Medicare & Medicaid Services (CMS) has come to an agreement with both Vertex and bluebird regarding outcomes-based agreements for the provision of each company's respective sickle cell disease gene therapy product to Americans with Medicaid under the Cell and Gene Therapy Access Model. “Cell and gene therapies hold significant potential to improve patient outcomes and transform lives, ultimately reducing long-term health care spending..." Liz Fowler, PhD, JD, the deputy administrator and director of the CMS Innovation Center, said in a statement. “This model will afford state Medicaid agencies more budget predictability while helping improve access to these innovative therapies for people with Medicaid with sickle cell disease.”

6. Amarna Therapeutics and Phenocell Secure Grant for Dry AMD Gene Therapy Project

The 2 companies have received a grant from Eureka's Eurostars program intended to support AMD-HALT, a joint project aimed at evaluating Amarna's Nimvec gene delivery vector platform using Phenocell’s induced pluripotent stem cell-derived age-related macular degeneration (AMD) disease model. “Partnering with Phenocell on this Eurostars-funded project is a critical step in advancing our proprietary gene therapy technology," Henk Streefkerk, MD, the CEO and medical director of Amarna, said in a statement. "The 3-dimensional in vitro cellular disease models will enable us to evaluate the therapeutic potential of our gene therapy candidates with unprecedented accuracy, accelerating progress toward effective treatments for diseases such as Dry AMD.”

7. uniQure and FDA Reach Accord on Approval Pathway for Huntington Disease Gene Therapy

During a Type B meeting, the FDA and uniQure came into agreement regarding the use of an accelerated approval pathway for AMT-130, the company's adeno-associated virus (AAV) vector-based gene therapy currently being evaluated for the treatment of Huntington disease in 2 phase 1/2 clinical trials (NCT04120493 and NCT05243017). Notably, the agency agreed that data from the ongoing phase 1/2 studies will be sufficient to support a biologics license application, negating the need to launch another trial before submission.

8. Krystal Biotech Hits Turbulence in Plans to Bring Dystrophic Epidermolysis Bullosa Gene Therapy to Europe

The European Medicines Agency (EMA)'s Committee for Medicinal Products for Human Use has cancelled an Oral Explanation that had been set for Krystal's marketing authorization application for dystrophic epidermolysis bullosa (DEB) gene therapy beremagene geperpavec-svdt, citing the need for written responses to certain outstanding issues. Despite this, Krystal still expects to remain on track for its planned launch of the therapy in Germany in the second quarter of next year. “We are confident in our ability to address the remaining postmarketing issues, and we believe that this additional exchange with EMA will ultimately maximize benefit and convenience to patients suffering from DEB,” Suma Krishnan, MS, MBA, the president of research and development at Krystal Biotech, said in a statement.