Around the Helix: Cell and Gene Therapy Company Updates – April 28, 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. FDA Clears Pfizer to Resume Phase 3 Gene Therapy Trial for Duchenne Muscular Dystrophy

The CIFFREO trial is underway in 11 countries but was halted in December 2021 amid safety concerns, including the death of a participant in a phase 1b study in the non-ambulatory cohort.

2. FDA Grants SQZ-PBMC-HPV Fast Track Designation for HPV+ Cancers

The FDA has granted a Fast Track Designation to the therapeutic vaccine SQZ-PBMC-HPV as a potential treatment for patients with advanced or metastatic HPV16+ solid tumors.

3. Gene Therapy for Frontotemporal Dementia Granted Orphan Drug Status

The investigational, AAV-based gene therapy delivers a functional copy of the progranulin GRN gene, helping to restore normal progranulin levels in patients who have an autosomal dominant mutation in the gene, which is one of 3 linked to familiar cases of the neurodegenerative disease.

4. Obe-Cel Granted RMAT Designation in R/R B-Acute Lymphocytic Leukemia

The US FDA has granted regenerative medicine advanced therapy (RMAT) to Autolus Therapeutics’ investigational gene-edited cell therapy, obecabatagene autoleucel (obe-cel), an autologous CD19-directed CAR T-cell therapy currently being evaluated in the phase 2 FELIX clinical trial in patients with relapsed or refractory B-acute lymphocytic leukemia (B-ALL).

5. Editas Receives FDA Rare Pediatric Disease Designation For EDIT-301 for Beta Thalassemia

EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia, was previously granted rare pediatric disease designation for the treatment of sickle cell disease.

6. Solid Bio to Cut Headcount Following DMD Program Refocus

Solid Bio will reduce its headcount by 35% in the wake of a corporate refocus on its 2 leading DMD gene therapy programs, SGT-001 and SGT-003.

7. Cytovia Merges to Help Bring NK Cell Therapies to the Clinic

Cytovia announced that it has entered into a definitive business combination agreement with Isleworth Healthcare Acquisition Corp., , which will help which will help provide Cytovia with capital for up to 2 years to further develop its gene-edited iNK and Flex-NK cell engager technologies.

8. Allogene's ALLO-605 Gets Orphan Drug Designation for Multiple Myeloma

Allogene's TurboCAR T-cell product ALLO-605 has been granted orphan drug designation from the FDA for the treatment of multiple myeloma, following a fast track designation recieved in Q2 2021.

9. Ocugen Gets Green Light to Move Forward With Retinitis Pigmentosa Gene Therapy Trial

Ocugen received a positive review from a data safety monitoring board, clearing them to continue enrollment of patients in their phase 1/2 trial evaluating OCU400, a gene therapy for retinitis pigmentosa.