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.

1. New Clinical Data Presented at WORLDSymposium 2023

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

2. Graphite Bio Drops Lead Sickle Cell Gene-Edited Cell Therapy

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.

3. Upcoming FDA Decisions for Rare Diseases in 2023

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.

4. Latest News in Gene Therapy for Rare Disease Day 2023

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

5. AviadoBio, Neurgain Technologies Enter Exclusive License and Collaboration Agreement For Subpial Delivery Technology for Gene Therapies

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

6. AllCells Expands Apheresis Network with 2 New Centers

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

7. Eye Disease Genetic Medicines the Focus of Expanded Collaboration Agreement Between AbbVie, Capsida Biotherapeutics

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.

8. Moderna, Life Edit Therapeutics Announce Collaboration on mRNA Gene Editing Therapies for Various Indications

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.

9. SonoThera Gains Rights to Use GE HealthCare's Optison and Sonazoid Microbubble Products in its Development of Ultasound-guided Gene Therapies

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.

10. Peter MacCallum Cancer Centre, Cartherics Enter Collaborative Development Program Agreement Focused on Ovarian Cancer CAR-T

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.