Around the Helix: Cell and Gene Therapy Company Updates – August 24, 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. Homology Trades in Gene Delivery for Gene Editing to Treat Phenylketonuria

Homology Medicines has paused enrollment in its phase 1/2 pheNIX clinical trial (NCT03952156) of the gene therapy HMI-102 for the treatment of phenylketonuria (PKU), the company revealed 2 months after announcing that a clinical hold had been lifted from the program. The company is deprioritizing the HMI-102 gene therapy in favor of its gene editing therapy, HMI-203, which is being evaluated in the phase 1/2 pheEDIT trial (NCT05222178) that initiated in June.

2. Genprex Patents REQORSA Immunogene Therapy for NSCLC

The US Patent and Trademark Office (USPTO) has granted a patent to Genprex for quaratusugene ozeplasmid (REQORSA), the company’s Immunogene therapy being evaluated for the treatment of late-stage non-small cell lung cancer (NSCLC).

3. Dystrophic Epidermolysis Bullosa Gene Therapy BLA Accepted With Priority Review

The FDA has accepted for filing a biologics license application (BLA) for Krystal Biotech's B-VEC, an investigational, redosable gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB).

4. Verismo Looks to Assess KIR-CAR Cell Therapy in Solid Tumors

Verismo has submitted an investigational new drug application (IND) to initiate a first-in-human phase 1 clinical trial, dubbed STAR-101, to assess SynKIR-110 in patients with mesothelin-expressing ovarian cancer, mesothelioma and cholangiocarcinoma.

5. Resurrecting ALS Mesenchymal Cell Therapy for Regulatory Approval

In an unexpected turnaround, BrainStorm Cell Therapeutics is submitting a BLA against previous FDA advice after announcing a corrected statistical analysis of a phase 3 clinical trial (NCT03280056) of the mesenchymal cell therapy NurOwn that revealed statistically significant improvements in participants with early-stage amyotrophic lateral sclerosis (ALS).

6. IND for NMOSD CAR T-Cell Therapy Approved by Chinese Health Authorities

An investigational BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy from IASO Biotherapeutics, CT103A (equecabtagene autoleucel), received IND application approval from China's National Medical Products Administration (NMPA) for an expanded indication in neuromyelitis optica spectrum disorder (NMOSD).

7. Metastatic Colorectal Cancer CAR-T Clinical Trial Enrolls First Patient

The first patient has been enrolled in the phase 1 clinical trial (CARAPIA-1; NCT05319314) of Innovative Cellular Therapeutics' GCC19CART, an investigational autologous CAR-T therapy for the treatment of relapsed/refractory metastatic colorectal cancer (r/r mCRC).

8. Lacerta Therapeutics, Prevail Therapeutics Will Collaborate on Development of Novel AAV Capsids for CNS Targets

Lacerta Therapeutics will be responsible for discovery, screening, and validation of novel capsids for the treatment of central nervous system (CNS) diseases, and Prevail Therapeutics will carry out preclinical and clinical studies, manufacturing, and commercialization.

9. Noile-Immune Biotech’s PRIME CAR-T Technology Focus of New Licensing Agreement With Chugai

The PRIME platform is designed to enhance the anti-tumor effect of both CAR-T cells and normal immune cells by enabling increased release of IL-7 and CCL19 from CAR-T cells.

10. ElevateBio to Provide iPSC lines to California Institute for Regenerative Medicine's Discovery and Translational Grant Awardees in New Partnership

In addition to induced pluripotent stem cell (iPSC) cell lines, ElevateBio will also provide viral vector technology, process development, analytical development, and GMP manufacturing capabilities to the academic institutions and biopharmaceutical companies in question.

11. Sonoma Biotherapeutics to Create a Center for Treg Cell Research, Development, and Manufacturing in Seattle

The approximately 83,000 square-foot facility will advance the continued development of Sonoma's treatments for autoimmune and inflammatory diseases.

12. Thermo Fisher Scientific Launches Viral Vector Manufacturing Facility in Plainville, Massachusetts

The newly opened 300,000 square foot facility will aid pharmaceutical companies in developing, testing, and manufacturing viral vectors for cell and gene therapies.