Around the Helix: Cell and Gene Therapy Company Updates – October 19, 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. Val-Rox up for Review in Hemophilia A 2 Years After Initial BLA Submission

The FDA has accepted BioMarin’s resubmitted biologics license application (BLA) for valoctocogene roxaparvovec (val-rox) for review in treating severe hemophilia A.

2. Axi-cel Approved by European Commission for Second-Line Treatment of Adults With DLBCL/HGBL

Axicabtagene ciloleucel (axi-cel; Yescarta) has been approved by the European Commission for the treatment of adult patients with diffuse large B-cell lymphoma (DLBCL) or high-grade B-cell lymphoma (HGBL) who are refractory to or have relapsed within 12 months 6of first-line chemoimmunotherapy.

3. Otoferlin-Related Hearing Loss Gene Therapy Gets IND Clearance

Decibel Therapeutics’ DB-OTO, an investigational adeno-associated virus (AAV) dual-vector-based gene therapy intended to treat otoferlin-related hearing loss being developed in collaboration with Regeneron Pharmaceuticals, has received clearance from the FDA for its investigational new drug (IND) application, which was submitted last month.

4. Allogeneic Natural Killer Cell Therapy Given Go-Ahead for Solid Tumors

The FDA has given investigational new drug application (IND) clearance to NKGen’s SNK02, an allogeneic natural killer (NK) cell therapy for treating solid tumors.

5. Eli Lilly Announces Acquisition of Akouos

Akouos' lead investigational gene therapy, AK-OTOF, which is intended to treat otoferlin-gene related hearling loss, is a focal point of the acquisition.

6. Xcell Biosciences, aCGT Vector Announce Cell and Gene Therapy Manufacturing and Analysis Collaboration Focused on Solid Tumors

The collaboration will aim to combine aCGT Vector's manufacturing platform with Xcell Biosciences' AVATAR AI platform for use in manufacturing and analyzing the potency of cell therapies directed at solid tumors.

7. Gamma Biosciences, Lonza Announce Collaboration Focused on Cell Selection Reagents

BioMagnetic Solutions, a subsidiary of Gamma Biosciences, will supply its FerroSelect reagents to be utilized with Lonza's Coccoon manufacturing platform, with the aim of advancing the development of new CAR-T and other immunotherapeutic products.

8. Charles River Laboratories, Nanoscope Therapeutics to Collaborate on Manufacturing for Retinal Gene Therapies

Charles River Laboratories will provide its viral vector and plasmid DNA manufacturing services to support the advancement of Nanoscope's Multi-Characteristic Opsin optogenetic therapies.

9. Alpha Teknova to Provide Early Access to its Anion Exchange Buffer Screening Kit and Functional Reporter Assay

The 2 technologies are still in early-stage development and are intended to address existing inefficiencies in the development of AAV vector-based gene therapies.

10. Minaris to Develop Manufacturing Process for Cellaris's Bullous Keratopathy Cell Therapy

CLS001 conists of Corneal Endothelial Cell Substitute derived from induced Pluripotent Stem Cells and is intended to address the limited availability of corneal transplants for bullous keratopathy.

11. Shenzhen Pregene Biopharma Backs Out of Agreement For Bone Cell Therapy Platform Rights with Bone Therapeutics and Link Health Pharma

Bone Therapeutics has regained worldwide rights to the ALLOB platform, but Link Health has expressed continued interested in an agreement for the rights to ALLOB in Hong Kong, Macau, Taiwan, Singapore, South Korea, and Thailand.

12. Josep Carreras Leukaemia Research Institute Gains Rights to use Elicera Therapeutics' iTANK Platform in CAR-T for Ewing Sarcoma

The iTANK platform is intended to address difficulties with targeting solid tumors with CAR-T therapies. While it has intitially been used in Elicera's own CAR-T products, it was designed to be applicable to other CAR-T products as well.