Around the Helix: Cell and Gene Therapy Company Updates – October 26, 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. GvHD Leads to Death in FCR001 Living Donor Kidney Transplant Trial

A patient that received a living donor kidney transplant and was treated with FCR001 as part of the phase 3 FREEDOM-1 trial (NCT03995901) has died after being hospitalized with grade 4 graft-vs-host disease complicated by serious infections leading to respiratory and renal failure.

2. NIH Awards Grant for Engineered Mechanosensitive Channel Glaucoma Gene Therapy

The NIH has awarded a $1.5 million SBIR Direct-to-Phase 2 grant to Nanoscope Therapeutics to support advancement of its Engineered Mechanosensitive Channel glaucoma therapy.

3. Celyad Discontinues CYAD-101 for Metastatic Colorectal Cancer

Celyad Oncology is no longer developing its investigational chimeric antigen receptor T-cell therapy CYAD-101 for the treatment of unresectable metastatic colorectal cancer.

4. New Process Could Drastically Reduce CAR-T Manufacturing Time

A new manufacturing process developed by investigators at University Hospitals Seidman Cancer Center in collaboration with a biotech start-up company could reduce the time to manufacture chimeric antigen receptor T-cell therapies to approximately 24 hours.

5. Astellas Pharma Invests in Taysha Gene Therapies

The strategic investment is aimed at advancing the development of investigational gene therapies for Rett syndrome and giant axonal neuropathy.

6. AVROBIO's Gaucher Disease Gene Therapy AVR-RD-02 Receives Innovative Licensing and Access Pathway Designation in UK

The investigational gene therapy was previously granted FDA fast track designation and FDA and EU orphan drug designations.

7. Tabelecleucel Receives Recommendation for Approval in R/R Epstein-Barr Virus-Positive Post-Transplant Lymphoproliferative Disease from EMA's CHMP

The recommendation was informed by data from ALLELE, a phase 3 clinical trial.

8. China's NMPA Accepts NDA for Zevor-cel for the Treatment of R/R Multiple Myeloma

The decision was informed by safety and efficacy results from the LUMMICAR STUDY 1, a phase 1/2 clinical trial.

9. LEXEO Therapeutics' Gene Therapy Gets EC Orphan Drug Designation in CLN2 Batten Disease

LX1004 previously received FDA orphan drug designation and rare pediatric disease designation.

10. Kite Gains Rights to Use Refuge Biotechnology's Gene Expression Platform for Hematological Cancer Indications

Via the exclusive license agreement, Kite aims to leverage the platform in the development of new CAR-T therapies.

11. AGTC Announces Upcoming Acquisition By Syncona

Applied Genetic Technologies Corporation believes the acquisition will allow for the continued development of AGTC-501, an investigational gene therapy intended for the treatment of X-linked retinitis pigmentosa.

12. Candel Therapeutics and University of Pennsylvania’s Center for Cellular Immunotherapies to Tackle Solid Tumors With Combination Therapies

The new research collaboration will explore the potential of combining the University of Pennsylvania’s CAR-T therapies with Candel's viral immunotherapies.