Around the Helix: Cell and Gene Therapy Company Updates – February 15, 2023

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.

1. Wet AMD Gene Therapy Trial Demonstrates Consistent Safety and Efficacy With New Manufacturing Process

REGENXBIO’s RGX-314, an investigational adeno-associated virus vector-based gene therapy intended to treat wet age-related macular degeneration, has demonstrated consistent results between product manufactured with the company’s new NAVXpress bioreactor platform process and product produced with an adherent cell culture process.

2. Focal Epilepsy Cell Therapy Trial Cleared to Expand

The FDA has cleared an amendment for the ongoing phase 1/2 clinical trial (NCT05135091) for Neurona Therapeutics’ NRTX-1001, an allogeneic regenerative neural cell therapy intended to treat drug-resistant mesial temporal lobe epilepsy, expanding the enrollment criteria to include both patients with dominant-lobe disease and patients with non-dominant lobe disease.

3. Phase 3 Data Garners RMAT Designation for Chronic Low Back Pain Cell Therapy

Mesoblast’s rexlemestrocel-L, an investigational allogeneic human bone marrow-derived mesenchymal precursor cell therapy, has received regenerative medicine advanced therapy designation from the FDA’s Office of Tissues and Advanced Therapies for the treatment of chronic low back pain associated with disc degeneration in combination with hyaluronic acid.

4. Ide-cel Beats SOC in Relapsed/Refractory Multiple Myeloma PFS

Idecabtagene vicleucel (ide-cel; Bristol Myers Squibb, 2seventybio), the chimeric antigen receptor T-cell therapy marketed as Abecma, improved progression-free survival compared with standard combination regimens in patients with relapsed/refractory multiple myeloma after 2 to 4 prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, and whose disease was refractory to their last regimen, according to results from the phase 3 KarMMa-3 trial (NCT03651128).

5. BioRestorative Therapies Gets Notice of Allowance from European Patent Office for Cell Therapy Manufacturing Process Patent Application

The application is related to implantable 3-dimensional scaffolds and brown adipocytes derived from human brown adipose-derived stem cells, which are elements of ThermoStem, an investigational cell therapy being developed for obesity and metabolic disorders including type 2 diabetes.

6. Gritstone bio, National Cancer Institute to Collaborate on Clinical Trial Evaluating Cell Therapy, Investigational KRAS-directed Vaccine Combination

The planned phase 1 trial is expected to include patients with metastatic solid tumors with G12V or G12D KRAS mutations.

7. AcuraBio to Integrate Cytiva's 2-Step Purification Protocol Into its Plasmid DNA Manufacturing Process

AcuraBio noted that Cytiva's protocol utilizes single-use purification technology.

8. Replay, MD Anderson Launch New TCR-NK Cell Therapy Company

Syena, the newly formed company, will be focused on developing T-cell receptor natural killer cell therapies for oncology indications.

9. Magenta Therapeutics Halts Development of Programs

The company, whose programs are focused on improving stem cell transplantation, has halted development to conduct a review of alternative strategic options, potentially including an acquisition or merger.