Around the Helix: Cell and Gene Therapy Company Updates – December 13, 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. New Clinical Data Presented at ASH’s 65th Annual Meeting

Catch up on CGTLive’s coverage of the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California. Our coverage will continue throughout the week.

2. FDA Approves Exa-cel, Vertex and CRISPR Therapeutics’ Gene Therapy, for Sickle Cell Disease

The FDA approved Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel), marketed under the name Casgevy, for the treatment of severe sickle cell disease (SCD) in patients aged 12 years and older with recurrent vaso-occlusive crises. It is the first CRISPR-based gene therapy to be approved in the United States.

3. FDA Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease

Alongisde exa-cel, the FDA also approved bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia, as a treatment for SCD in patients aged 12 years and older. The therapy consists of autologous CD34+ hematopoietic stem cells collected by plerixa for mobilization and apheresis, transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene.

4. Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Trial Doses First Patient

Benitec Biopharma’s phase 1b/2a clinical trial evaluating BB-301, its investigational adeno-associated virus vector-based silence and replace gene therapy for the treatment of oculopharyngeal muscular dystrophy-related dysphagia, has dosed its first patient.

5. FDA’s New GeMDAC AdComm Will Provide Advice and Recommendations on Treatment Options Under Evaluation for Genetic Metabolic Diseases

The FDA’s newly established Genetic Metabolic Diseases Advisory Committee (GeMDAC), which will operate within the oversight of the Division of Rare Diseases and Medical Genetics and ultimately have 9 voting members, is currently seeking interested experts. “Genetic metabolic diseases include very rare diseases that individually affect a limited number of patients,” Janet Maynard, MD, MHS, the director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at the FDA, said in a statement. “Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.”

6. Newly Launched Company Tome Biosciences to Focus Efforts on Programmable Genomic Integration

Tome Biosciences, which launched with $213 million in Series A funding from investors such as ARCH Venture Partners and FUJIFILM Corporation, will specialize in developing treatments that use programmable genomic integration, a method they anticipate will allow any DNA sequence, regardless of size, to be inserted in any programmed location in the genome.

7. Kamau Therapeutics Picks Up Graphite Bio’s Nula-cel

Following Graphite Bio’s discontinuation of development on SCD gene-edited cell therapy nulabeglogene autogedtemcel (nula-cel) after the first patient dosed experienced an unexpected serious adverse event, the investigational product, along with all of Graphite’s genome editing assets, has now been sold to newly launched company Kamau Therapeutics. This move comes after Graphite’s recent strategic agreement to merge with Lenz Therapeutics.

8. ViroCell to Support UCL CAR-T Research By Providing Manufacturing Services

ViroCell, a contract development and manufacturing organization, will produce lentiviral vectors for use during University College London (UCL)’s research into a novel chimeric antigen receptor T-cell (CAR-T) approach to treating relapsed acute lymphoblastic leukaemia in children. UCL’s approach, developed by Persis Amrolia, a professor of transplantation immunology at UCL, uses CAR T-cells targeted at both CD19 and CD22 and also seeks to preserve “stem cell memory” T-cells.

9. Modalis and JCR Pharmaceuticals Bring Technologies Together In Pursuit of CNS Disease Gene Therapies

Under a new agreement, the 2 companies will leverage Modalis’ CRISPR-GNDM (Guide Nucleotide-Directed Modulation) and JCR’s J-Brain Cargo for the development of novel gene therapies intended to treat central nervous system (CNS) diseases. “JCR Pharmaceuticals’ proprietary blood-brain barrier penetrating technology, J-Brain Cargo, has successfully and efficiently delivered biopharmaceutical candidates of various modalities to the CNS," Haru Morita, MS, CEO of Modalis, said in a statement. "As a pioneer in developing CRISPR based epigenome editing technology (CRISPR-GNDM), we have demonstrated long-term drug efficacy and functional improvement in animal disease model studies, including demonstration of target engagement and safety in nonhuman primates."