Around the Helix: Cell and Gene Therapy Company Updates – December 7, 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. OTOF Hearing Loss Gene Therapy Granted Orphan Drug Designation

Sensorion’s OTOF-GT, an investigational gene therapy intended for the treatment of otoferlin (OTOF) gene-mediated hearing loss, has been granted orphan drug designation (ODD) by the FDA.

2. Multiplex-Base Edited CAR T Therapy Cleared for Trial in T-ALL After Hold

The FDA has lifted the clinical hold on and cleared the investigational new drug application (IND) of Beam Therapeutics’ multiplex-base edited chimeric antigen receptor (CAR) T-cell therapy BEAM-201 for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T cell lymphoblastic lymphoma (T-LL).

3. Enhanced Cell Therapy Gets Fast Track Designation for HPV16+ Solid Tumors

SQZ Biotechnologies’ SQZ-eAPC-HPV, an investigational cell therapy intended to treat patients with HPV16+ advanced or metastatic solid tumors, has been granted fast track designation by the FDA.

4. BioCardia Follows up CardiAMP With CardiALLO Clearance for Heart Failure

The FDA has cleared BioCardia’s IND to initiate a phase 1/2 trial of its CardiALLO neurokinin-1 receptor positive (NK1R+) allogeneic human mesenchymal stem cell (MSC) therapy for the treatment of patients with ischemic heart failure with reduced ejection fraction (ischemic HFrEF).

5. Celularity Receives Halal Certification of its Cell Therapy Products from Circle H International

The certification will support the company's expansion of its operations in the Middle East North Africa region.

6. SonoThera Closes Round of Series A Financing for Ultrasound-Guided Gene Therapy Development

The $60.75 million raised will support the advancement of SonoThera's technology platform, which the company intends to provide an alternative to viral vector-based delivery methods for gene therapies.

7. Renovacor Acquired by Rocket Pharmaceuticals

Renovacor's REN-001, an investigational gene therapy intended to treat BAG3-associated dilated cardiomyopathy, is a focal point of the deal, which was originally agreed to by the companies on September 19, 2022.

8. Innoforce Launches New Manufacturing Facility in Hangzhou, China

The Contract Development and Manufacturing Organization's GMP facility, which will offer process development and manufacturing for RNA, plasmid DNA, viral vectors, and cell therapy products, and double up as the company's corporate headquarters, has begun operations.

9. King Abdulaziz City for Science and Technology, Pfizer to Collaborate on Gene Therapy Research

The Saudi Arabian organization signed a memorandum of understanding with Pfizer related to cooperation on research, including research and development of treatments for genetic diseases.

10. MaxCyte Licenses Flow Electroporation technology and ExPERT platform to Curamys

The strategic platform license provides non-exclusive rights to Curamys, which intends to utilize them in the development of cell and gene therapies for rare diseases.

11. Solid Biosciences Acquires AavantiBio

The combined company's focus will include advancing the development of investigational gene therapies for Duchenne muscular dystrophy and Friedreich’s ataxia. Solid Biosciences also announced the simultaneous completion of a $75 million private placement.

12. European Organizations to Collaborate on Regenerative Gene Therapy for Lung Cancer

The €12.8 million INSPIRE Project, launched by OmniSpiran Limited and EVerZom, includes collaboration with Aerogen Limited, Trinity College Dublin, RemedyBio, Myriad Associates, Biopharma Excellence (Pharmalex), Deutsches Krebsforschungszentrum, and Lung Cancer Europe.

13. AGTC Acquired by Syncona Limited Portfolio Company

Applied Genetic Technologies Corporation (AGTC), which is focused on the development of gene therapies for indications including inherited retinal diseases and Cental Nervous System disorders, is now a wholly owned subsidiary of Syncona.

14. BioRestorative Therapies Receives NIH Grant to Support Cell Therapy Research for Polycystic Ovary Syndrome

The Small Business Innovation Research Phase 1 grant from the National Institue of Health (NIH)'s Eunice Kennedy Shriver National Institute Of Child Health & Human Development is intended advance the company's ThermoStem program.

15. Cimeio Therapeutics Granted US Patent for its SCIP Platform

The Shielded-Cell & Immunotherapy Pairs (SCIP) platform involves a method for selective depletion of edited or non-edited primary hematopoietic cells in vivo.