Around the Helix: Cell and Gene Therapy Company Updates - February 23, 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. Phenylketonuria Gene Therapy Study Put on Clinical Hold

The FDA has placed a clinical hold on Homology Medicines’ pheNIX trial (NCT03952156), which is investigating the gene therapy HMI-102 for the potential treatment of phenylketonuria (PKU) following elevated liver function test scores. Homology has not yet received a formal letter from the FDA.

2. BioMarin’s Phenylketonuria Gene Therapy Program Stalls

BioMarin’s PHEARLESS study (NCT04480567) of BMN-307 for the potential treatment of PKU will likely be on pause for several quarters, as the FDA has requested additional nonclinical studies of the therapy. The program was previously placed on clinical hold after oncogenesis was observed in mice.

3. Enrollment Paused in Mesothelioma CAR T-Cell Therapy Trial

Atara Biotherapeutics and Memorial Sloan Kettering have paused enrollment in their phase 1 trial (NCT04577326) of ATA2271, a next-generation, autologous, mesothelin-targeted investigational chimeric antigen receptor (CAR) T-cell therapy for the potential treatment of malignant pleural mesothelioma following a fatal, serious adverse event (AE). The company is evaluating the death and the extent of its relationship to treatment.

4. Liso-Cel Gets Priority Review for Second-Line R/R Large B-Cell Lymphoma

The FDA has accepted and granted priority review to lisocabtagene maraleucel's (liso-cel; Breyanzi) supplemental biologics license application for the treatment of adult patients with relapsed or refractory large B-cell lymphoma in whom frontline therapy has failed. Positive data from the phase 3 TRANSFORM trial (NCT03575351) informed the decision.

5. Memory T Cell CAR T Therapy Showcases Anti-Tumor Activity in Resistant Prostate Cancer

P-PSMA-101, Poseida Therapeutics’ autologous CAR T-cell candidate, has shown promising anti-tumor activity in metastatic castration-resistant prostate cancer, with significant declines in prostate-specific antigen. Data from the phase 1 SPOTLIGHT trial (NCT04249947) were presented at ASCO GU.

6. Gracell Advances CAR T Platform

Gracell Biotechnology is advancing 2 CAR T-cell therapy candidates developed with its allogeneic TruUCAR, dual-targeted platform. These are GC502, in development for multiple indications including B-cell acute lymphoblastic leukemia (ALL), and GC027, in development for T-cell ALL. Data from GC02’s first in-human study will be presented at the American Association for Cancer Research Annual Meeting 2022.

7. New Collaboration to Develop Gene Therapies for Rare Diseases

Code Biotherapeutics and Takeda are partnering to develop gene therapies for liver-directed and central nervous system-directed rare diseases, with Takeda optioning Code’s targeted 3DNA® non-viral genetic medicine delivery platform. Any selected candidates will be developed and commercialized by Takeda.

8. Multiple Myeloma CAR T-cell Therapy Granted Orphan Drug Designation

The FDA has granted orphan drug designation to CT103A, an investigational anti-BCMA CAR T-cell therapy for the potential treatment of relapsed/refractory multiple myeloma. The therapy is developed by IASO Biotherapeutics and Innovent Biologics.

9. Eli Lilly Launches Institute for Genetic Medicine

Eli Lilly is advancing their RNA-based therapeutics pipeline with the launch of the Lilly Institute for Genetic Medicine in Boston, which will develop genetic medicines. The site, which thr company has invested $700 million in, is scheduled to be occupied in 2024, will also house a shared space to support biotech startups in the area.

10. Synthego Advancing Cell and Gene Therapy Pipeline

The company has raised $200 million to accelerate their cell and gene therapy discovery and development ecosystem, to expand their Halo and Eclipse genome engineering platforms, and to invest in next-generation technologies including CRISPROff, a light-based CRISPR editing system.

11. Discovery Life Sciences Launches New Cellular Materials

Discovery Life Sciences is expanding its portfolio with the launch of Discovery CryoLeukopaks. The new cryopreservation human cell material products are intended to streamline the transition from research to manufacturing of cell and gene therapies.

12. Mesenchymal Stromal Cells Improves Colitis Healing

Direct injection of remestemcel-L, a mesenchymal stromal cell therapy, demonstrated rapid mucosal healing and disease remission in patients with medically refractory ulcerative colitis or Crohn’s colitis at high risk of progression to surgery. Mesoblast Limited presented these results from an interim analysis of the phase 1/2 study (NCT04543994).