Around the Helix: Cell and Gene Therapy Company Updates – March 1, 2023

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.

Catch up on CGTLive's coverage of WORLDSymposium 2023, held February 22-26, in Orlando, Florida.

Graphite Bio is discontinuing development of its lead program, the sickle cell disease (SCD) gene therapy nulabeglogene autogedtemcel (nula-cel; formerly known as GPH-101), and will instead seek a partnership agreement for external development of the therapy.

The company also anonunced its intent to restructure folowing the decision, with the Board of Directors approved actions including a reduction of the company's workforce in order to reduce costs while exploring strategic alternatives.

Tuesday, February 28, 2023, was Rare Disease Day, recognizing the more than 300 million people worldwide affected by the more than 7000 conditions designated as rare diseases. CGTLive took a look at some of the upcoming FDA decisions on selected gene and cell therapies for rare diseases.

In observance of Rare Disease Day, the CGTLive team gathered some of the latest updates on rare disease research.

AviadoBio has integrated use of subpial delivery into its pipeline of amyotrophic lateral sclerosis gene therapies.

The new centers in Texas and Alabama will add to the company's existing presence in the San Francisco Bay and Boston Metropolitan areas.

As part of the agreement, which expands on a previous partnership established in 2021, Capsida will receive $70 million upfront and may be eligible to receive up to $595 million in option fees and research and development milestones.

The companies will collaborate on initial research and preclinical studies and Moderna will take on further development, manufacturing, and commercialization if it chooses to exercise an option for a target.

SonoThera's gene therapy platform, which is currently in pre-clinical development, utilizes a microbubble-mediated biophysical process with the intention of delivering genetic payloads without the use of a viral vector.

Cartherics' CTH-004, an investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy, will be evaluated in a clinical trial carried out by Peter MacCallum Cancer Centre.