Around the Helix: Cell and Gene Therapy Company Updates – February 7, 2024

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.

The Biden-Harris Administration’s new Cell and Gene Therapy (CGT) Access Model, which is led by the Centers for Medicare & Medicaid Services' Innovation Center, will initially focus its efforts on improving access to CGT treatment options for patients with sickle cell disease.

Adaptimmune Therapeutics’ biologics license application for afamitresgeneautoleucel (afami-cel, formerly ADP-A2M4), an investigational T-cell receptor (TCR) T-cell therapy intended to treat synovial sarcoma, has been accepted by the FDA with priority review. The Prescription Drug User Fee Act target action date has been set for August 4, 2024.

World Cancer Day, observed annually on February 4 by the patient and clinician communities, is organized by the Union for International Cancer Control and intended to improve awareness, education, and action towards ending preventable deaths from cancer.

Encoded Therapeutics’s ETX101, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat SCN1A+ Dravet syndrome, has received clearance from the FDA and the Australia Therapeutic Goods Administration for 2 separate clinical trials in each agency’s respective jurisdiction.

Florida Biotechnologies, which is focused on the development of gene therapies for mitochondrial diseases, collaborated with the University of Miami’s Bascom Palmer Eye Institute on an AAV vector-based gene therapy intended to treat Leber hereditary optic neuropathy that is now in clinical development. NAYA intends to push forward the development of this gene therapy, which uses an AAV capsid targeted at mitochondria.

BridgeBio, which obtained the new funding from Blue Owl Capital and Canada Pension Plan Investment Board Credit Investments, will put the finances towards advancing both its gene therapy programs and the launch of acoramidis, a key small molecule program for the company.

The new product line, referred to as Rep/Cap Plasmids, consists of off-the-shelf plasmids intended to speed up production of AAV gene therapy products. “The launch of Charles River’s off-the-shelf Rep/Cap offering is the latest in a series of portfolio enhancements designed to streamline adeno-associated virus (AAV)-based gene therapy programs,” Kerstin Dolph, the senior vice president of manufacturing at Charles River, said in a statement. “The reduction in production time and improved efficacy of the supply chain will help accomplish our ultimate goal of delivering safe, effective therapies to patients, faster.”

The pharmacy accelerator’s newly launched model is intended to simplify the process of incorporating and offering newly approved gene therapy treatments for hospitals and health systems. "Many health systems are not set up to coordinate the procurement of a novel gene therapy,” Eric Manuel Balmir, BS, MS, PharmD, CIM, the vice president of clinical ancillary services and chief pharmacy officer of Children's National Hospital, said in a statement. “There are an incredible amount of steps and considerations involved in the process. Clearway Health's framework allowed us to quickly and efficiently address these concerns, positioning our hospital to provide the latest pharmaceutical advancement."