Around the Helix: Cell and Gene Therapy Company Updates – September 4, 2024

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.

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1. Amarna Therapeutics Secures Regulatory Guidance From FDA for Diabetes Gene Therapy AM510

Amarna Therapeutics has received regulatory guidance from the FDA regarding AM510, its investigational gene therapy product intended to treat type 1 diabetes, in the context of an Initial Targeted Engagement for Regulatory Advice (INTERACT) meeting.

2. Orgenesis’ CAR-T ORG-101 Demonstrates Efficacy and Safety in Acute Lymphoblastic Leukemia in Real-World Study in China

Orgenesis’ ORG-101, a CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy, has demonstrated efficacy and safety in patients with CD19+ B-cell acute lymphoblastic leukemia treated in real-world data that came from 233 patients treated at a leading hematology center in China.

3. Abata Therapeutics' Treg Therapy ABA-101 Garners FDA Fast Track Designation

Abata Therapeutics’ ABA-101, an autologous regulatory T-cell (Treg) therapy for the treatment of patients with progressive multiple sclerosis, has received fast track designation from the FDA.

4. Grit Biotechnology’s TIL Therapy GT201 Cleared for US Trial in Advanced Solid Tumors

Grit Biotechnology has received clearance of an investigational new drug (IND) application from the FDA for GT201, an investigational genetically-engineered tumor infiltrating lymphocyte (TIL) therapy, for a clinical trial in patients with advanced solid tumors.

5. Bayer and NextRNA Team Up to Tackle lncRNA-Driven Disease

Bayer and NextRNA have entered a strategic collaboration agreement focused on using small molecule drugs to disrupt the pathological activity of long noncoding RNAs (lncRNAs) in oncology indications. “With NextRNA’s exceptional expertise and lncRNA platform, we aim to advance novel small molecule therapeutics against a new class of targets in oncology,” Juergen Eckhardt, MD, the head of business development and licensing at Bayer’s Pharmaceuticals Division, said in a statement.

6. Ractigen Snags Orphan Drug Designation for saRNA Therapy RAG-18

Ractigen Therapeutics has received orphan drug designation from the FDA for RAG-18, its investigational small activating RNA (saRNA) that is currently in preclinical development for the treatment of cases of Duchenne muscular dystrophy and Becker muscular dystrophy caused by any mutation in the DMD gene. Notably, the therapy previously received rare pediatric disease designation from the FDA in July 2024.

7. TC Biopharm Obtains EPO Patent Related to Gamma Delta T-cell Therapy

TC BioPharm (TCBP) has obtained a patent from the European Patent Office (EPO) that covers the utilization of modified gamma delta cells T-cells for cancer and virus treatment. "TCBP continues to focus on our immediate applications for TCB008 while developing future high value gamma delta assets for other indications such as solid tumors," Bryan Kobel, the chief executive officer of TCBP, said in a statement. "We believe gamma deltas can be a high impact therapeutic when modified with the potential to outpace current CAR and modified T-cells in safety and efficacy, patent protecting these approaches gives us a competitive moat and further value in a potential acquisition scenario."

8. Arsenal Biosciences Garners $325 Million for Solid Tumor Cell Therapy

Arsenal Biosciences has closed an oversubscribed round of Series C funding, raking in $325 million from a myriad of new and existing investors. The company plans to put the cash towards continued advancement of its investigational therapeutic programs, which include T-cell therapies for solid tumors, and towards the development of new tools and processes for further cell therapy product candidate identification.