Around the Helix: Cell and Gene Therapy Company Updates – February 19, 2025

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 (@CGT_Live on X, or CGTLive on LinkedIn) and use #AroundTheHelix!

1. Phase 2 Trial for BMS's Liso-Cel Meets Primary End Point in R/R Marginal Zone Lymphoma Cohort

Bristol Myers Squibb (BMS)’s phase 2 TRANSCEND FL clinical trial (NCT04245839) evaluating lisocabtagene maraleucel (liso-cel, marketed as Breyanzi), an autologous CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy, has met its primary end point in its cohort for adult patients with relapsed/refractory (r/r) marginal zone lymphoma (MZL).

2. CareDx’s AlloHeme Shows Accuracy in Detecting Relapse Following AlloHSCT in AML and MDS

CareDx’s AlloHeme, a next-generation sequencing (NGS)-based assay, has demonstrated the ability to detect early relapse following allogeneic hematopoietic stem cell transplantation (alloHSCT) in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), according to interim data from the prospective ACROBAT study (NCT04635384).

3. Keys to a Successful Cell and Gene Therapy Launch

The obstacles to affordability and accessibility are complex and multifactorial; they include the complex, high-cost manufacturing processes for personalized living therapies, long vein-to-vein times, complicated logistics, their often-curative nature, and high prices. Addressing these challenges effectively requires therapy developers to consider affordability and accessibility from early development alongside the traditional focus on safety and efficacy, which drive approvability.

4. Solid Biosciences’ Gene Therapy SGT-003 Produces Microdystrophin Expression in Patients With DMD

Solid Biosciences SGT-003, an investigational next-generation adeno-associated virus (AAV) vector-based gene therapy, has produced expression of microdystrophin in patients being treated for Duchenne muscular dystrophy (DMD) in the phase 1/2 INSPIRE DUCHENNE clinical trial (NCT06138639).

5. Biogen Purchases Certain International Commercial Rights for Stoke Therapeutics' Dravet Syndrome Gene Therapy

Biogen has purchased the rights to develop and commercialize Stoke Therapeutics' zorevunersen (also known as STK-001), an investigational antisense oligonucleotide (ASO) intended to treat Dravet syndrome, in all territories outside the United States, Canada, and Mexico. Notably, Stoke expects to initiate EMPEROR, a planned global phase 3 registrational clinical trial for zorevunersen, in the second quarter of this year.

6. AskBio Snags RMAT Designation for Parkinson Disease Gene Therapy AB-1005

Asklepios BioPharmaceutical (AskBio)'s AB-1005 (AAV2-GDNF), an investigational AAV vector serotype 2 (AAV2)-based gene therapy intended to treat Parkinson disease (PD), has received regenerative medicine advanced therapy (RMAT) designation from the FDA. Notably, a sham-controlled phase 2 clinical trial (REGENERATE-PD; NCT06285643) evaluating AB-1005, recently began the process of randomly assigning enrolled participants in January 2025.

7. FDA Accepts BLA for Ultragenyx’s MPSIII Gene Therapy UX111 With Priority Review

The FDA has accepted the biologics license application (BLA) that Ultragenyx submitted for UX111 (ABO-102), an AAV vector-based gene therapy intended to treat mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome), with priority review. The Prescription Drug User Fee Act (PDUFA) action date for the BLA has been set at August 18, 2025, and the FDA is not planning to hold an advisory committee meeting regarding the application.

8. Patient Treated in Bahrain Becomes First to Receive Vertex and CRISPR's SCD Gene Therapy Outside the US

The patient received Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel, marketed under the name Casgevy) for the treatment of sickle cell disease (SCD) at The Bahrain Oncology Centre. Bahrain originally approved exa-cel for the treatment of SCD and transfusion-dependent beta-thalassaemia on December 2, 2023, becoming the first country in the Middle East and the second in the world to do so.