Around the Helix: Cell and Gene Therapy Company Updates – December 27, 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.

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1. BrainStorm Cell Therapeutics Seeks Special Protocol Assessment for New NurOwn Trial After Meeting With FDA

BrainStorm Cell Therapeutics has met with the FDA to discuss a possible regulatory path forward for debamestrocel (NurOwn), its investigational autologous mesenchymal stem cell neurotrophic factor–secreting cell therapy product intended to treat amyotrophic lateral sclerosis.

2. Some Dose-Dependent Neurological Benefits Seen With AMT-130 Huntington Disease Gene Therapy

Interim data from uniQure's phase 1/2 clinical trials of AMT-130 gene therapy in patients with Huntington disease has shown evidence of preserved neurological function and dose-dependent clinical benefits.

3. The Lupus Approach: CAR-T Begins the Jump from Blood Cancers to B-Cell Driven Autoimmune Disease

Throughout 2023, interest in the application of chimeric antigen receptor T-cell (CAR-T) and other cell therapies to B-cell-driven autoimmune disease skyrocketed, with a multitude of clinical trials being initiated by various companies. As part of a larger review of the year’s highlights, CGTLive took a closer look at this new trend.

4. Robert M Califf, MD, MACC, on 2023’s Landmark FDA Approvals of Cell and Gene Therapies

CGTLive’s sister publication HCPLive™ sat down with Robert M Califf, MD, MACC, the commissioner of food and drugs at the FDA, to discuss the progress made in gene therapy and cell therapy this year.

5. AstraZeneca to Take Gracell Biotechnologies Into the Fold

Under a newly announced definitive agreement, AstraZeneca will acquire Gracell Biotechnologies, adding the latter’s GC012F to its repertoire of cell therapies. GC012F is an investigational autologous CAR-T therapy being evaluated for multiple myeloma, other hematological malignancies, and autoimmune diseases such as systemic lupus erythematosus.

6. ImmunoScape and Singapore’s EDDC Join Forces on Development of TCR-based Bispecific Molecules

Singapore’s Experimental Drug Development Centre (EDDC), part of its Agency for Science, Technology and Research (A*STAR), and ImmunoScape, a company spun out from A*STAR in 2016, are working collaboratively to develop T-cell receptor (TCR)-based bispecific molecules intended to help treat cancer. “By combining the power of our machine learning-augmented TCR discovery platform with EDDC’s broad expertise in the design of multifunctional constructs, we will aim to rapidly develop novel off-the-shelf TCR-based bispecific molecules that could overcome widespread challenges in cell therapy approaches against solid tumors,” Choon-Peng Ng, MBA, the CEO of ImmunoScape, said in a statement.

7. Janssen Snags Interests in MeiraGTx’s X-Linked Retinitis Pigmentosa Gene Therapy

Under a new asset purchase agreement, potentially worth up to $415 million, Jannssen Pharmaceuticals will acquire remaining interests in MeiraGTx’s X-linked retinitis pigmentosa gene therapy botaretigene sparoparvovec. “The initial $130 million upfront and near-term milestone payments, combined with the $30 million investment we received from Sanofi in October, increases our cash runway to mid-2026, without including the additional $285 million in potential payments associated with this transaction,” Alexandria Forbes, PhD, the president and CEO of MeiraGTx, said in a statement.

8. Takeda Pharmaceuticals Pulls Out of Gene Therapy R&D Collaboration With JCR Pharmaceuticals

As part of a larger strategic move to cease work on AAV-based gene therapy programs, Takeda has terminated its participation in a collaboration agreement with JCR, originally signed in March 2022, on gene therapies for use in conjunction with the latter's J-Brain Cargo delivery technology. "We will continue to advance our own research and development in the field of gene therapy with a focus on collaborating with other companies thereby expanding outside of lysosomal storage disorders into additional neurological diseases," Shin Ashida, the chairman and president of JCR, said in a statement.