Around the Helix: Cell and Gene Therapy Company Updates – August 3, 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. Editas' Gene Therapy Trial for Sickle Cell Initiates After Hold Lifted

The FDA has removed a partial clinical hold on Editas Medicine’s phase 1/2 clinical trial of experimental cell therapy EDIT-301 for the treatment of severe sickle cell disease (SCD; RUBY; NCT04853576) and the first patient in the trial has been dosed.

2. Clinical Hold Lifted on Celyad's Phase 1b Trial in Refractory Metastatic Colorectal Cancer

Celyad Oncology's phase 1b clinical trial, KEYNOTE-B79 (NCT04991948) in refractory metastatic colorectal cancer (mCRC) is back on track after the FDA lifted its clinical hold which was placed back in March 2022.

3. Omidubicel BLA Accepted With Priority Review in Hematological Malignancies

The FDA has accepted a biologics license application (BLA) with priority review for omidubicel, an allogeneic hematopoietic stem cell transplant candidate developed by Gamida Cell for the treatment of hematological cancers.

4. IND Halted for T-Cell Malignancy Multiplex-Edited Cell Therapy

The FDA has placed a clinical hold on Beam Therapeutics’ investigational new drug application for BEAM-201, a chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T cell lymphoblastic lymphoma (T-LL).

5. FDA Accepts IND Application for Cystic Fibrosis Gene Therapy KB407

The FDA has accepted the investigational new drug application for Krystal Biotech’s KB407, a gene therapy candidate being evaluated for the treatment of cystic fibrosis (CF).

6. IND Cleared for Dual-Targeted CAR T Therapy for B-Cell Malignancies

Cellectis has received investigational new drug clearance for their dual-targeted allogeneic CAR T-cell therapy UCART2022 for the potential treatment of B-cell malignancies.

7. Altheia Science's Gene Therapy Programs Supported by Development Partnership With AGC Biologics

AGC Biologics' Milan-based facility will help push forward Altheia Science's autoimmunity treatments which focus on the modulation of PD-L1 expression in hematopoietic stem cells and progenitor cells.

8. Certara to Collaborate With Memorial Sloan Kettering Cancer Center on CAR-T Therapy Biosimulation Software

The collaboration intends to use the software to learn more about optimal CAR-T therapy doses and regimens for multiple myeloma.

9. Wendy Li, MD, Appointed Chief Medical Officer of Gracell Biotechnologies

Li's previous experience includes providing leadership for cell therapy pipelines as chief medical officer of EXUMA Biotech.

10. Over $120 Million Raised by OriCell Therapeutics for Cancer Immunology Cell Therapy Pipeline Development

The money will additionally help fund OriCell's proprietary discovery platform and a new manufacturing plant's construction.

11. Ottawa Heart Institute Announced as First Clinical Site for BioCardia's Cell Therapy Heart Failure Trial

A formal Data Safety Monitoring Board Review for the CardiAMP Cell Therapy Heart Failure Trial is scheduled for August 30, 2022, to evaluate the trial's safety and futility.

12. Roche and Poseida Therapeutics to Collaborate on CAR T-Cell Therapies for Hematological Malignancies

The strategic global collaboration and license agreement will focus on researching and developing allogeneic, "off-the-shelf" treatments for indications including multiple myeloma and B-cell lymphoma.