Around the Helix: Cell and Gene Therapy Company Updates – May 15, 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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social and use #AroundTheHelix!

1. New Clinical Data Presented at ASGCT's 2024 Meeting

Catch up on CGTLive's coverage of the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 11, 2024, in Baltimore, MD. Expert insights will continue to be posted throughout the week.

2. Patient With DMD Dies in Pfizer’s Phase 2 Gene Therapy Trial

A patient with Duchenne muscular dystrophy (DMD) has died in Pfizer’s phase 2 DAYLIGHT study (NCT05429372) evaluating fordadistrogene movaparvovec (PF-06939926). In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial (NCT04281485) currently evaluating the gene therapy against standard of care in boys with DMD aged 4 to less than 8 years. DAYLIGHT’s participants are aged 2 to less than 4 years.

3. First Patient Set to Receive bluebird’s Approved Sickle Cell Gene Therapy, Lyfgenia

The first patient with sickle cell disease (SCD) is set to receive bluebird bio’s lovotibeglogene autotemcel (lovo-cel) after completing a cell collection at Children’s National Hospital in Washington. Lovo-cel was approved as Lyfgenia by the FDA in December 2023.

4. Verve Therapeutics Doses First Patient in Phase 1b Trial for Gene Therapy VERVE-102

Verve Therapeutics has dosed the first patient in Heart-2, a phase 1b clinical trial (NCT06164730) evaluating VERVE-102, an investigational in vivo base editing therapy that uses a lipid nanoparticle (LNP) delivery system, for the treatment of heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD).

5. BioMarin Lets Go of 5% of Employees

The company, which recently brought its gene therapy product valoctocogene roxaparvovec (val-rox) to market for the treatment of adults with severe hemophilia A, has laid off 170 employees, amounting to about 5% of its workforce, in relation to a decision to downsize and refocus its pipeline, a move which also led to the halting of 4 programs.

6. Verismo Cleared to Bring Its KIR-CAR Platform to Clinical Trials for B-NHL

Verismo Therapeutics has received clearance of an investigational new drug application for the evaluation of SynKIR-310, a chimeric antigen receptor T-cell therapy based on the company's KIR-CAR platform developed at the University of Pennsylvania. The platform is intended to enable sustained antitumor activity of the CAR T-cells in tumor microenvironments. SynKIR-310 will be evaluated in the phase 1 CELESTIAL-301 clinical trial.