Around the Helix: Cell and Gene Therapy Company Updates – May 11, 2022

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. FDA Lifts Hold on LogicBio’s Phase 1/2 SUNRISE Trial of LB-001 for MMA

The US Food and Drug Administration (FDA) has lifted its clinical hold on LogicBio Therapeutics’ phase 1/2 SUNRISE trial (NCT04581785), which is evaluating LB-001 for the potential treatment of methylmalonic acidemia (MMA) in pediatric patients.

2. REGENXBIO DMD Gene Therapy Trial Delayed Due to Manufacturing Issue

During its Q1 earnings call, REGENXBIO disclosed that it uncovered an “unexpected and isolated observation” during the vial-filling stage of the manufacturing process for its Duchenne muscular dystrophy gene therapy at a third-party manufacturing facility.

3. AGTC-501 Efficacy Maintained in 18-Month Follow Up Data for X-linked Retinitis Pigmentosa

The RPGR gene-delivering rAAV-based therapy AGTC-501 (rAAV2tYF-GRK1-RPGR) demonstrated sustained efficacy and safety after 18 months of follow-up for patients with X-linked retinitis pigmentosa (XLRP), which is caused by RPGR mutations, according to findings from an ongoing, phase 1/2, open-label, dose escalation trial presented at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting.

4. ICER Will Asses Hemophilia A and B Gene Therapies

The Institute for Clinical and Economic Review (ICER) announded that it will assess the comparative clinical effectiveness and value of etranacogene dezaparvovec (EtranaDez; UniQure and CSL Behring) for the treatment of hemophilia B, and also update its assessment of BioMarin's valoctocogene roxaparvovec (Roctavian).

5. Ohio State University Opens Gene Therapy Institute

Ohio State’s Gene Therapy Institute will coordinate existing strengths to accelerate the expansion of gene therapies studied at the university, which holds one of the largest first in human clinical trial portfolios.

6. Google Will Permit Ads for Gene, Cell Therapies if FDA-Approved or Educational

Google announced that it was relaxing its strict no-ad policy around cell and gene therapies, stating that beginning in July, it would allow ads for FDA-approved products and ads that are purely educational or informational in nature, regardless of regulatory status.

7. Mass General Brigham Names 'Disruptive Dozen' in Gene and Cell Therapy Tech

The New England-based health system named its annual "Disruptive Dozen", a list of 12 emergine gene and cell therapy technologies slated to have the greatest impact on healthcare.

8. CARsgen Therapeutics Publishes Interim Phase 1 Data in Gastro Cancers

The publication, titled "Claudin18.2-specific CAR T cells in gastrointestinal cancers: phase I trial interim results" includes results from a multicenter, open-label phase I clinical trial conducted in China to explore the safety, efficacy, and cellular pharmacokinetics of CT041 in patients with advanced CLDN18.2-positive gastrointestinal cancers.

9. Arcellx Publishes CART-ddBCMA Phase 1 Data in Multiple Myeloma

Clinical data from Arcellx's dose escalation cohorts in its ongoing phase 1 study of CART-ddBCMA for the treatment of patients with relapsed or refractory multiple myeloma were published in Blood Advances.

10. Gene Therapy May Help Reduce Neuropathy in Spinal Cord Injuries, Research Shows

New research published in Molecular Therapy by scientists from the University of California San Diego, and colleagues, demonstrates that gene therapy can be used to inhibit nerve cell signaling associated with neuropathy in models of spinal cord or peripheral nerve injuries.