Around the Helix: Cell and Gene Therapy Company Updates – March 2, 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. Cilta-Cel Approved for Relapsed/Refractory Multiple Myeloma

Ciltacabtagene autoleucel (cilta-cel; Carvykti) has been granted FDA approval for the treatment of adult patients with relapsed/refractory multiple myeloma following 4 or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

2. Celyad Pauses CAR T-Cell Therapy Trial

Celyad Oncology has voluntarily paused their phase 1b KEYNOTE-B79 clinical trial (NCT04991948) following the report of 2 patient deaths with similar findings. The trial is evaluating the investigational CAR T-cell therapy CYAD-101 administered with pembrolizumab in refractory metastatic colorectal cancer with microsatellite stable/mismatch-repair proficient disease.

3. CRISPR Therapy Yields Deep and Sustained Improvements in ATTR Amyloidosis

Updated data on NTLA-2001, Intellia and Regeneron’s investigational CRISPR-Cas9 therapy, showed deep and sustained reductions of transthyretin protein in patients with hereditary transthyretin (ATTR) amyloidosis with polyneuropathy in the phase 1 study (NCT04601051).

4. Advanced Ovarian Cancer Immunotherapy Trial Continues

The phase 1/2 OVATION trial (NCT033393884) will continue to treat patients with advanced ovarian cancer with GEN-1, a novel gene-mediated immunotherapy after a positive recommendation from the Data Safety Monitoring Board. Treatment will continue at a dose of 100 mg/m².

5. Collaboration Will Focus on Developing Gene Therapy for Congenital Muscular Dystrophy

Santhera Pharmaceuticals and SEAL Therapeutics have entered into an agreement to develop gene therapy approaches for the treatment of LAMA2-deficient congenital muscular dystrophy. SEAL will help build off of preclinical research Santhera previously engaged with in previous agreements with University Basel as well as Rutgers, The State University of New Jersey.

6. Muscular Dystrophy Gene Therapy Receives Orphan Drug Designation

The FDA has granted orphan drug designation to ATA-100, Atamyo Therapeutics’ gene therapy candidate for the potential treatment of fukutin-related protein limb-girdle muscular dystrophy Type 2I/R9. The company also announced Denmark’s approval of a clinical trial application for the therapy as well as public financing of 2 million euros by Bpifrance to support a first in-human trial.

7. Manufacturing Partnership to Advance Retinitis Pigmentosa Gene Therapy Program

Ray Therapeutics is advancing Ray-001, their gene therapy candidate for the potential treatment of retinitis pigmentosa, into clinical trials with the help of Forge Biologics’ development and manufacturing capabilities, using their proprietary HEK 293 suspension Ingition Cells and pEMBR adenovirus helper plasmid.

8. Allogene Back On Track With CAR T trials Following Clinical Hold Resolution

After resolving questions around a reported chromosomal abnormality in a patient involved in their clinical trial, Allogene announced that its AlloCAR T trails are back on track to assess their allogeneic cell therapy products.

9. New CRISPR Approach May Reduce Off-Target Gene-Editing Effects

Researchers at UT Austin have published work in Nature, demonstrating that a refined CRISPR-based gene-editing approach -- dubbed SuperFi-Cas9 -- may significantly reduce the risk of off-target gene editing, which remains a serious concern with the original CRISPR Cas9 enzyme approach.

10. Patent Ruling on CRISPR IP Boosts Broad Institute, Spurns Intellia and Others

A ruling by the US Patent and Trademark Office came in in favor of Harvard and MIT's Broad Institute, ruling that they were the first to create CRISPR/Cas9 -- not the group collectively known as 'CVC', which includes Nobel Prize winners Emmanuelle Charpentier, PhD, and Jennifer Doudna, PhD, the University of California and the University of Vienna.