Around the Helix: Gene and Cell Therapy Company Updates - February 2, 2022

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

1. X-Linked SCID Gene Transfer Therapy Placed on Clinical Hold

The FDA has placed a clinical hold on the investigational new drug application (IND) for MB-207, Mustang Bio’s gene transfer program for X-linked severe combined immunodeficiency (XSCID). The phase 2 IND was previously submitted in December 2021 and the clinical hold is pending Chemistry, Manufacturing and Controls clearance.

2. Taysha Announces Positive Giant Axonal Neuropathy Data Amid GM2 Gangliosidosis Study Death

Taysha Gene Therapies announced positive data from the high-dose cohort of a phase 1 clinical trial (NCT02362438) evaluating TSHA-120, an investigational gene therapy for the potential treatment of giant axonal neuropathy. The company also announced positive data in GM2 gangliosidosis but this was tempered by a study death deemed unrelated to treatment.

3. Axi-Cel Label Updated to Include Prophylactic Corticosteroids

The FDA has approved a label update for CAR T-cell therapy axicabtageneciloleucel to include use of prophylactic corticosteroids across all approved indications. The decision was based on data from a safety management cohort (cohort 6) of the phase 1/2 ZUMA-1 trial (NCT02348216). 

4. TCR Bispecific Therapy Approved for Unresectable or Metastatic Uveal Melanoma

The FDA has approved Immunocore’s KIMMTRAK® (tebentafusp-tebn), a T cell receptor bispecific immunotherapy for the treatment of unresectable or metastatic uveal melanoma, marking the first approved therapy for the indication. The approval was based on results from the phase 3 IMCgp100-202 clinical trial (NCT03070392).

5. Chimeric Therapeutics Granted CAR T Patent, Enters Research Agreement With UPenn

Chimeric Therapeutics has been granted a patent covering their chlorotoxin chimeric antigen receptor (CAR) T technology used in their candidates CHM1101, which is being evaluated in a phase 1 clinical trial (NCT04214392) for recurrent or progressive glioblastoma, andCHM 2101, currently in preclinical studies for recurrent or progressive glioblastoma. The company also announced a 3-year sponsored research agreement with the University of Pennsylvania to support and develop the CHM 2101 candidate.

6. Phase 1/2 Trial Initiated forHIV CRISPR Therapy

Excision BioTherapeutics has initiated a phase 1/2 trial (NCT05144386) to evaluate the safety and efficacy of EBT-101, an in-vivo, CRIPSR-based therapeutic in approximately 9 adults with aviremic human immunodeficiency virus.

7. IND Cleared for Adrenomyeloneuropathy Gene Therapy

SwanBio Therapeutics' investigational new drug application for gene therapy SBT101 has been cleared by the FDA for the potential treatment of adrenomyeloneuropathy. SwanBio plans to initiate a randomized, placebo-controlled, phase 1/2 clinical trial to evaluate the safety and efficacy of the therapy in the second half of 2022.

8. LogicBio’s Methylmalonic Acidemia Gene Therapy On Hold After Serious Adverse Event

The FDA has placed LogicBio’s phase 1/2 SUNRISE clinical trial of LB-001 on clinical hold following the report of another drug-related serious adverse event in a patient in the trial’s youngest cohort. The AAV genome-editing therapy is being studied for the treatment of methylmalonic acidemia in pediatric patients.

9. Sarepta and GenEdit Developing Gene Editing Therapeutics for Neuromuscular Diseases

The new agreement will combine GenEdit’s NanoGalaxy platform and Sarepta’s gene editing technology to develop novel therapies for neuromuscular diseases. The agreement adds to the companies’ existing collaborationinitiated in December 2020 researching genetic medicine delivery with GenEdit’s polymer nanoparticles.

10. Sio Gene Therapies Walks Away From Parkinson Disease Gene Therapy Program

Sio intends to end their license agreement with Oxford Biomedica for the latter’s Parkinson disease gene therapy program, AXO-Lenti-PD. Sio is currently conducting the phase 2 SUNRISE-PD clinical trial (NCT03720418) of AXO-Lenti-PD which has previously demonstrated promising safety and efficacy data. Sio decided to deprioritize the program citing a constraint on resource requirements.

11. Gamida Cell Seeking Omidubicel Commercialization Partners

Gamida Cell is evaluating commercialization strategies for omidubicel, their cell therapy for bone marrow transplant in patients with hematologic malignancies, including licensing agreements and partnerships. The company is also addressing FDA concerns that placed their GDA-201 natural killer (NK) cell therapy for follicular and diffuse large B-cell lymphomas on clinical hold and is advancing other genetically modified NK cell therapy programs.

12. CRISPR Therapeutics Doses First Patient in Diabetes Cell Therapy Trial

The first patient has been dosed with VCTX210, a cell therapy for the potential treatment of type 1 diabetes developed by CRISPR and ViaCyte, in a phase 1 trial (NCT05210530) evaluating the safety and efficacy of the novel, allogeneic, gene-editedcell therapy.