Around the Helix: Cell and Gene Therapy Company Updates – July 20, 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. Cell Therapy Trial Fails Primary Endpoint in Treating Muscle Injury After Arthoplasty for Hip Fracture

Pluristem’s phase 3 trial (NCT03451916) of allogeneic PLX-PAD cells for the treatment of muscle injury following arthroplasty for hip fracture has failed to meet its primary endpoint of improvements in Short Physical Performance Battery test at week 26 compared to placebo, although other improvements were seen.

2. Angelman Syndrome Gene Therapy Shows Promising Efficacy in Higher-Dose Cohorts

Ultragenyx has acquired GeneTx after promising interim data, from a phase 1/2 trial (NCT04259281) on GTX-102, the companies’ joint gene therapy program for the treatment of Angelman syndrome, were released.

3. Gene Therapy Fails to Improve Survival in Platinum-Resistant Ovarian Cancer

The phase 3 OVAL trial (NCT03398655) of ofranergene obadenovec (VB-111; VBL Therapeutics) in combination with paclitaxel has failed to meet its primary endpoint in improving progression-free survival or overall survival in patients with platinum-resistant ovarian cancer.

4. Ixo-Vec Back in Phase 2 Trials, Now for Wet Age-Related Macular Degeneration

After submitting an amended IND, Adverum’s gene therapy is set to be evaluated in the phase 2 LUNA trial.

5. MPSII Gene Therapy Granted Orphan Drug Designation

The FDA has granted orphan drug designation to AVROBIO’s gene therapy AVR-RD-05 for the potential treatment of mucopolysaccharidosis type II (MPSII) also known as Hunter syndrome.

6. First Patient With Mesial Temporal Lobe Epilepsy Dosed With Neural Cell Therapy

Neurona Therapeutics’ phase 1/2 clinical trial (NCT05135091) for NRTX-1001, a regenerative neural cell therapy derived from human pluripotent stem cells, has dosed its first patient with Mesial Temporal Lobe Epilepsy (MTLE).

7. Bone to Optimize Phase 2b Trial of High-Risk Tibial Fracture Cell Therapy

Bone Therapeutics has made adjustments to optimize their phase 2b trial (NCT04432389) of their allogeneic osteoblast cell therapy, ALLOB, for high-risk tibial fractures. The adjustments come after the company announced a reprioritization to focus on ALLOB in September 2021.

8. Purespring's FunSel Gene Therapy Screening Platform to be Used by SwanBio in New Licensing Agreement

SwanBio will use the platform to identify new targets for gene therapies for neurological diseases.

9. Strategic Cooperation MOU for the Development of Antibody and Cell Treatments Signed Between GenScript ProBio and DAAN Bio Therapeutics

The two companies will cooperate in discovery, manufacturing, clinical trials, and related research/service projects.

10. AAV Gene Therapy Vectors for Ocular Diseases to be Developed in Avista Therapeutics Partnership with Roche

Avista's scAAVengr platform will be used to develop AAV capsids, which will be evaluated and potentially licensed by Roche for use in gene therapy programs.

11. Combination of Antegene's Bispecific Antibody and Celularity's Natural Killer Cell Platform to be Evaluated for Potential Therapeutic Synergy

A collaboration has been announced between Antegene and Celularity which will focus on researching potential combination therapies for the treatment of hematological and solid tumor cancers. Notably, this collaboration will be Antegene's entrance into the field of cell therapy development.

12. Novartis seeking partner to continue development of retinitis pigmentosa gene therapy CPK850

The phase 2 (NCT03374657) sub-retinally delivered therapy candidate is in development for the treament of patients with retinitis pigmentosa due to mutations in the RLBP1 gene, and functions by delivering a working copy of the RLBP gene.

13. Biopharma Manufacturing Learning Facility Launched to Support Innovation in Cell and Gene Therapy Manufacturing

The New Jersey facility was launched by McKinsey & Company in partnership with New Jersey Innovation Institute and will give companies the opportunity to see technologies in practice and engage in hands-on educational activities.

14. Hemogenyx Pharmaceuticals Opens New Cell Therapy Manufacturing Facility

The New York City facility will enable the company to manufacture its cell therapies in-house.