Around the Helix: Cell and Gene Therapy Company Updates – November 16, 2022

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. Data From Various Clinical Studies Presented at SITC 2022

Catch up on CGTLive's coverage of the Society for Immunotherapy of Cancer’s (SITC) 37th Annual Meeting, November 8-12, 2022, in Boston, Massachusetts

2. ALS Cell Therapy BLA Refused for Filing by the FDA

BrainStorm Cell Therapeutics has announced that the biologics license application (BLA) for NurOwn, its investigational autologous mesenchymal stem cell (MSC) neurotrophic factor–secreting (NFS) cell therapy product intended to treat amyotrophic lateral sclerosis (ALS), has been refused for filing by the FDA.

3. ALS Gene Therapy Momentum Continues With New Orphan Drug Designation

The FDA has granted orphan drug designation (ODD) to ET-101 (Eikonoklastes Therapeutics), an investigational gene therapy for the potential treatment of ALS.

4. Lupus Nephritis CAR-T Therapy Cleared for Phase 1/2 Clinical Trial

Kyverna Therapeutics’ KYV-101, an investigational chimeric antigen receptor (CAR) T-cell therapy intended to treat lupus nephritis, has received clearance of its investigational new drug (IND) application, which was submitted to the FDA in October of this year.

5. High-risk Leukemia and MDS Expanded Cell Therapy Trial Completes Phase 2 Enrollment

The phase 2 clinical trial (NCT04103879) for ExCellThera’s ECT-001, an investigational cell therapy intended to treat patients with high-risk leukemias and myelodysplastic syndromes (MDS), has completed patient enrollment.

6. ElevateBio, Affini-T Partner to Bring KRAS-directed TCR-T Therapies to the Clinic

ElevateBio's new LentiPeak Lentiviral Vector platform production platform, previously covered by CGTLive, will be utilized in the advancement of Affini-T Therapeutics' investigational T-cell receptor (TCR)-engineered therapies that target oncogenic driver mutations in the KRAS gene.

7. Redpin Acquired by Kriya Therapeutics

Kriya intends to advance Redpin Therapeutics' existing gene therapy progams for epilepsy and trigeminal neuralgia.

8. BioViva Sciences Announces Intention to Initiate Series A Financing Push Next Year

The company intends to facilitate the advancement of gene therapies that utilize its patent-pending CMV vector for indications related to aging.

9. Takara Bio Regains Rights to NY-ESO-1 siTCR Gene Therapy After Mutually Terminating Agreement With Otsuka

It was noted that the termination of the agreement for the investigational synovial sarcoma gene therapy was unrelated to safety or efficacy concerns.

10. FDA Provides New Guidance for Clinical Trials Evaluating Multiple Versions of the Same Cell Therapy or Gene Therapy

Issued earlier this month, the guidance includes recommendations covering IND application structure and reporting of adverse events for clinical trial sponsors.

11. Voyager, Touchlight Partner on Novel Capsids

Touchlight is licensing its doggybone DNA technology to Voyager Therapeutics for use in the creation of novel capsids to support Voyager's genetic therapy pipeline, allowing for the rapid and scalable amplification of DNA that can be applied to mRNA, AAV, lentiviral, genome editing, and vaccine-based therapeutics.