Around the Helix: Cell and Gene Therapy Company Updates – March 15, 2023

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. FDA Accepts BLA Resubmission for Remestemcel-L in Steroid-Refractory aGVHD

The FDA has accepted the resubmission of a biologic license application for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease under the age of 12 years, according to an announcement by the therapy’s developer, Mesoblast Limited.

2. Estrella Biopharma’s CD19-Targeted T-cell Therapy for B-Cell Lymphomas Gets IND Clearance

Estrella Biopharma’s EB103, an investigational CD19-targeted T-cell therapy intended to treat B-cell lymphomas, has received clearance for its investigational new drug (IND) application from the FDA.

3. FDA Greenlights Phase 1/2 Trial of Vertex’s Type 1 Diabetes Cell Therapy

The FDA has cleared an IND application from Vertex Pharmaceuticals to proceed with a clinical trial of its stem cell-derived, fully differentiated pancreatic islet cell therapy for type 1 diabetes. The treatment is currently known as VX-264.

4. Hemgenix Displays Superiority to Factor IX Prophylaxis for Hemophilia B in HOPE-B Trial Data

Data from the open-label phase 3 HOPE-B trial (NCT03569891) demonstrate that the treatment of patients with hemophilia B with etranacogene dezaparvovec (Hemgenix; CSL Behring) is superior to prophylactic factor IX treatment on annualized bleeding rate, with a favorable safety profile.

5. Porton Advanced Solutions to Provide Manufacturing Services for Bennu Biotherapeutics' Cell and Gene Therapy Pipelines

Bennu Biotherapeutics is focused on the development of treatments for various indications including solid tumors and autoimmune diseases. The partnership aims to accelerate the development of T-cell therapies, specifically.

6. Gracell Biotechnologies Plans to Initiate Multiple Clinical Trials in 2023

The company announced that it anticipates the intiation of clinical trials for FasTCAR-T GC012F, which is intended to treat relapsed/refractory (r/r) multiple myeloma (MM), in both the US and China. It also announced that it is on track to initiate an investigator-initiated trial for SMART CAR-T GC506 for patients with Claudin18.2-positive solid tumors in China.

7. ICER Announces Plans to Evaluate Orchard Therapeutics' Arsa-cel

Arsa-cel is a gene therapy intended to treat metachromatic leukodystrophy. ICER has published a Draft Scoping Document and welcomes comments and suggestions on the assessment's scope until March 30, 2023.

8. Catalent Announces Expansion of AAV Platform Process

The UpTempo platform, which the company originally launched in 2022, has been expanded to include a clonal HEK293 cell line and off-the-shelf pHelper and rep/cap plasmids plasmids.

9. Caribou Biosciences Provides Updates on Pipeline

The coprorate update included the announcement that the phase 1 CaMMouflage clinical trial for CB-011, an allogeneic CAR-T therapy being evaluated in patients with r/r MM, is now recruiting patients for its first dose level.

10. Ring Therapeutics Obtains Funding for Anellogy Platform for Gene Therapy Delivery

The $86.5 million raised in the Series C funding round will support the company's continued development of the platform, which utilizes annelloviruses as delivery vectors for DNA and RNA payloads.

11. Adaptimmune and TCR2 Therapeutics Announce Merger

The combined company will focus initial efforts on the development of engineered T-cell therapies for solid tumor indications. The lead clinical franchises are therapies targeting MAGE-A4 and mesothelin.