Around the Helix: Cell and Gene Therapy Company Updates - February 16, 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. RNA Therapy Fails to Show Benefit in Leber Congenital Amaurosis 10

ProQR Therapeutics announced that the phase 2/3 ILUMINATE trial (NCT03913143) of sepofarsen failed to meet its primary end point of best corrected visual acuity compared to sham. Secondary endpoints of full-field stimulus test and mobility were also not met.

2. Thermo Fisher Scientific Launches Patheon Commercial Packaging Services for Cell and Gene Therapies

The integrated services are designed to seamlessly transition therapies from clinic to commercial use. The service, which combines GMP storage, serialization, ultracold and cryogenic packaging, and global distribution, has been launched for patients across the US and Europe.

3. New Collaboration to Develop Next-Generation AAV Vectors for Diabetes Gene Therapy

SIRION Biotech and Centre for Genomic Regulation in Spain are teaming up to develop adeno-associated virus vectors for gene therapies for types 1 and 2 diabetes. The collaboration will combine SIRION’s AAV platform with the Centre’s research into genetic regulatory mechanisms to develop pancreatic cell-targeted vectors.

4. IND Cleared for Friedreich Ataxia Cardiomyopathy Gene Therapy

LEXEO Therapeutics has announced that the FDA has approved its investigational new drug application for the gene therapy candidate LX2006 for cardiac manifestations of FA, which are the leading cause of death in this population. The company plans to initiate a phase 1/2 clinical trial in mid-2022.

5. Repligen Launches New AAV Purification Resins

Avitide, a Repligen company, has launched 3 advanced affinity chromatography resins for use in gene therapy manufacturing workflows, AVIPure® - AAV9, AVIPure® - AAV8, and AVIPure®.

6. Artemis-SCID Cell Therapy Granted Fast Track Review

The California Institute for Regenerative Medicine is developing the investigative cell therapy, which is being evaluated in a phase 1/2 trial (NCT03538899). Encouraging early data from 13 treated participants has also warranted regenerative medicine advanced therapy designation by the FDA.

7. New Collaboration to Develop Allogeneic CRISPR-Edited NK Cell Therapies

Intellia Therapeutics is combining their genome editing platform and lipid nanoparticle technologies with ONK’s optimized natural killer (NK) cell therapy platform to develop up to 5 CRISPR natural killer cell therapies for oncologic indications.

8. SpliceBio to Expand Gene Therapy Platform

The company has raised 50 million euros to advance their unique protein splicing platform. The money will help advance their lead program in Stargardt disease into the clinic and expand their gene therapy pipeline to other currently untreatable genetic diseases.

9. CAR T-Cell Therapy Demonstrates Encouraging Data in mCRPC

Mustang Bio’s MB-105 CAR T-cell therapy is being evaluated in a phase 1 trial (NCT03873805) for PSCA-positive metastatic castration-resistant prostate cancer. A dose-limiting toxicity of cystitis has been identified and the study continues to assess the recommended phase 2 dose. Efficacy results will be presented at the ASCO Genitourinary Cancers Symposium on February 17.

10. Adrenomyeloneuropathy Gene Therapy Gets Fast Track Designation

The FDA has granted fast track designation to SwanBio Therapeutics’s SBT-101, an investigational adeno-associated virus gene therapy candidate for adrenomyeloneuropathy. The therapy’s investigational new drug application was cleared earlier this month and a phase 1/2 clinical trial is expected to initiate in the second half of 2022.

11. T-Cell Lymphoma CAR T-Cell Therapy Trial Placed on Clinical Hold

The FDA has placed a clinical hold on the phase 1 trial (NCT04712864) of LB1901, an autologous CAR T-cell product developed by Legend Biotech for the potential treatment of relapsed or refractory T-cell lymphoma after low CD4-positive T-cell counts were observed in the peripheral blood of the only patient dosed so far.

12. Canavan Disease Gene Therapy Shows Promising Data

Myrtelle’s recombinant adeno-associated virus vector-based investigational gene therapy being developed with Pfizer has shown favorable safety and tolerability in the 3 patients dosed so far in the phase 1/2 first in-human clinical trial (NCT04833907) in Canavan disease. No treatment-related adverse events have been observed.