Around the Helix: Cell and Gene Therapy Company Updates – October 23, 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. Wave Life Sciences Achieves RNA Editing in Trial for WVE-006 in Alpha-1 Antitrypsin Deficiency

Wave Life Sciences’ WVE-006, an investigational RNA editing oligonucleotide intended to treat alpha-1 antitrypsin deficiency (AATD), has demonstrated the ability to edit targeted RNA molecules in the first 2 patients treated in the phase 1b/2a RestorAATion-2 clinical trial (NCT06405633).

2. Adicet's T-Cell Therapy ADI-100 Snags Clearance for Evaluation in Idiopathic Inflammatory Myopathy and Stiff Person Syndrome

Adicet Bio’s ADI-100, an investigational allogeneic chimeric antigen receptor (CAR)-engineered gamma delta T-cell therapy currently being evaluated in a phase 1 clinical trial (NCT06375993) for lupus nephritis (LN), has received clearance from the FDA of an amendment to its investigational new drug (IND) application, allowing for the recruitment of patients with idiopathic inflammatory myopathy (IIM) and stiff person syndrome (SPS) into the trial.

3. First Patient Dosed in Trial for uniQure’s SOD1-ALS Gene Therapy AMT-162

UniQure has dosed the first patient in its phase 1/2 EPISOD1 clinical trial (NCT06100276), which is evaluating AMT-162, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (SOD1-ALS).

4. Tenaya Therapeutics Cleared to Move Onto Higher Dose Cohort in Trial of Hypertrophic Cardiomyopathy Gene Therapy TN-201

Tenaya Therapeutics has received clearance from the independent Data and Safety Monitoring Board (DSMB) to move onto a higher dose cohort for the MyPeak-1 phase 1b clinical trial (NCT05836259) evaluating TN-201, an investigational adeno-associated virus (AAV)-vector based gene therapy intended to treat hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.

5. OcuPhire and Opus Genetics Merge

OcuPhire Pharma has purchased Opus Genetics in an all-stock transaction and formed a new combined company, which will operate under the name Opus Genetics, Inc., with the ticker symbol $IRD. The company announced that the former Opus Genetics' investigational AAV vector-based gene therapy products, which are in development for inherited retinal diseases (IRDs), will form a major part of the newly combined' company's pipeline.

6. Sangamo Reaches Accord With FDA on Plans for Accelerated Approval Pathway for Fabry Disease Gene Therapy ST-920

Sangamo Therapeutics has emerged from a Type B interaction with the FDA having come into alignment with the agency on plans to pursue an accelerated approval pathway for isaralgagene civaparvove (ST-920), an investigational AAV vector-based gene therapy product intended to treat Fabry disease. The company noted that based on the interaction it intends to use 1 year posttreatment estimated glomerular filtration rate (eGFR) slope data from patients treated in the ongoing phase 1/2 STAAR clinical trial (NCT04046224) as an intermediate clinical end point for a planned 2025 biologics license application submission.

7. Editas Medicine Demonstrates Proof-of-Concept for In Vivo Hemoglobinopathy Gene Editing Therapy

Editas Medicine has reported preclinical data demonstrating proof-of-concept for an in vivo gene editing approach intended to treat sickle cell disease and beta thalassemia. Alongside the announcement, Editas also noted that it is seeking a partner to continue development of renizgamglogene autogedtemcel (previously EDIT-301), its clinical-stage ex vivo gene editing product for hemoglobinopathies, in order to focus its efforts on in vivo approaches.

8. AvenCell Rakes in $112 Million for Switchable CAR-T Tech

AvenCell Therapeutics has garnered $112 million in a Series B round of financing led by Novo Holdings, which will be put towards clinical validation of its switchable CAR T-cell therapy platform, which is designed with a safety mechanism intended to allow the CAR T-cells to be switched "Off" or "On" within a patient's body. “AvenCell’s switchable CAR-T platform represents a paradigm shift in cell therapy, offering unprecedented control over treatment dynamics,” Nihal Sinha, MB BChir, a partner at F-Prime Capital, said in a statement. “The ability to modulate CAR-T cell activity post-infusion could address critical safety and efficacy challenges in current therapies."