Around the Helix: Cell and Gene Therapy Company Updates – December 18, 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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social and use #AroundTheHelix!

1. PepGen’s IND for Duchenne Muscular Dystrophy PMO PGN-EDO51 Placed on Clinical Hold by the FDA

PepGen’s investigational new drug (IND) application for PGN-EDO51, an investigational phosphorodiamidate morpholino oligomer (PMO) intended to treat Duchenne muscular dystrophy (DMD), has been placed on hold by the FDA. As such, the phase 2 CONNECT2-EDO51 clinical trial for PGN-EDO51, which is already active in the United Kingdom, may not yet go forward to with activities in the United States.

2. Pros and Cons of Various Reimbursement Models for Cell and Gene Therapies

Payers have demonstrated increased interest in innovative payment models for these high-cost treatments to improve affordability, decrease exposure to shock claims, and help protect their investments if the treatments do not provide the outcomes they promise.

3. Cilta-Cel Boosts MRD Negativity Rates Compared to Standard of Care Therapy in New Multiple Myeloma Data

Johnson & Johnson subsidiary Janssen’s and Legend Biotech's ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti) an FDA-approved BCMA-directed autologous chimeric antigen receptor T-cell (CAR-T) therapy, has produced higher rates of minimal residual disease (MRD) negativity in comparison to patients treated with standard-of-care (SOC) therapy for lenalomide-refractory multiple myeloma (MM) in newly updated data from the phase 3 CARTITUDE-4 clinical trial (NCT04181827).

4. Bristol Myers Squibb and 2seventy bio's CAR-T Ide-Cel Effects Responses in CNS-Involved R/R Multiple Myeloma

Bristol Myers Squibb and 2seventy bio's idecabtagene vicleucel (ide-cel, marketed as Abecma), an FDA-approved CAR-T therapy, brought about responses in patients with relapsed/refractory (r/r) multiple myeloma (MM) affecting the central nervous system (CNS).

5. Editas Axes Reni-Cel Development, Fully Shifts Focus to In Vivo Gene Therapy

Editas Medicine has made the decision to discontinue development of renizgamglogene autogedtemcel (previously EDIT-301), its clinical-stage ex vivo gene editing therapy for hemoglobinopathies, after its search for a partner to carry on development of the product did not bear fruit. The company is now turning its efforts to fully focus on the development of approaches based on in vivo CRISPR-editing.

6. SpliceBio's Protein Splicing Gene Therapy SB-007 Cleared for US Trial in Stargardt Disease

The FDA has granted IND clearance to SpliceBio's SB-007, an investigational protein splicing gene therapy, enabling the company to move forward with plans for the phase 1/2 ASTRA clinical trial (NCT identifier pending), which it anticipates launching recruitment activities for in the first half of next year. Notably, this constitutes the first IND clearance for a protein splicing gene therapy product. “Stargardt disease has been a challenge for the development of gene therapies due to the large size of the ABCA4 gene, and currently has no approved therapies available,” Paul Yang, MD, PhD, the chief of the Paul H. Casey Ophthalmic Genetics Division at Casey Eye Institute at Oregon Health & Science University, said in a statement. “This new therapy utilizes a unique approach to replace the full-size, normal ABCA4 protein at high efficiency, which addresses the root cause of Stargardt disease across any pathogenic variant in the ABCA4 gene."