Around the Helix: Cell and Gene Therapy Company Updates – May 15, 2024

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Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

CGTLive Around the Helix

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.

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