Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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. FDA Approves PTC Therapeutics’ Gene Therapy Kebilidi for AADC Deficiency
PTC Therapeutics’ eladocagene exuparvovec, a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, has been approved by the FDA for the treatment of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency. Notably, the indication covers the full spectrum of disease severity and the approval constitutes the first gene therapy for direct administration to the brain to be approved in the United States.
2. FDA Accepts Abeona Therapeutics' BLA for Epidermolysis Bullosa Gene Therapy Pz-Cel
Abeona Therapeutics' biologics license application (BLA) for prademagene zamikeracel (pz-cel; EB-101), an investigational autologous gene-corrected epidermal sheet therapy for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB), has been accepted for filing by the FDA.
3. Patient Treated With Neurogene’s Investigational Rett Syndrome Gene Therapy NGN-401 in Critical Condition After Developing Life-Threatening Immune Response
A patient treated with Neurogene’s NGN-401, an investigational AAV vector-based gene therapy intended to treat Rett syndrome, in the context of a phase 1/2 pediatric clinical trial (NCT05898620) has developed a life-threatening immune response to the treatment and is now in critical condition.
4. First Patient Dosed in Trial for Adicet's T-Cell Therapy ADI-100 in Autoimmune Disease
The first patient, who has lupus nephritis (LN), has been dosed in Adicet Bio’s phase 1 clinical trial (NCT06375993) evaluating ADI-100, its investigational allogeneic chimeric antigen receptor (CAR)-engineered gamma delta T-cell therapy, for the treatment of autoimmune diseases.
5. CSL Behring Shuttering Pasadena Facility
CSL Behring has stated that it will be closing down a research & development facility located in Pasadena, California, that is focused on cell and gene therapy. The announcement comes after the company revealed that it would be moving away from ex vivo gene therapy approaches and after noted slow patient uptake of its marketed gene therapy etranacogene dezaparvovec (Hemgenix; EtranaDez), an AAV serotype 5 vector-based product approved by the FDA for the treatment of some patients with hemophilia B.
6. Anixa Biosciences Moves Onto Third Cohort in Trial for Ovarian Cancer CER-T
Anixa Biosciences and its partner Moffitt Cancer Center have dosed the first patient in the third cohort of their ongoing phase 1 clinical trial (NCT05316129) evaluating Anixa's novel follicle stimulating hormone receptor (FSHR)-targeting chimeric endocrine receptor (CER) T-cell therapy for the treatment of ovarian cancer. Notably, the dose used for the third cohort is tenfold greater than that used in the study's initial cohort.
7. ALS Research Collaborators Make Large Data Repository Available for Therapeutic Research
Answer ALS and Cedars-Sinai have made a repository of data that includes biological and clinical data from almost 1,000 patients with amyotrophic lateral sclerosis (ALS) completely available as a resource for research being conducted by companies and institutions around the world. “We reprogrammed blood cells into iPSCs and differentiated them into motor neurons, which die in ALS patients at an unprecedented scale,” Dhruv Sareen, PhD, the executive director of the Cedars-Sinai Biomanufacturing Center, said in a statement. “This has established the world’s largest bank of specialized neurons for ALS research to date and offers a comprehensive and advanced model for disease research and drug discovery.”
8. MyoGene Snags ODD and RPD FDA Designations for Preclinical Gene Editing Therapy
MyoGene has received orphan drug designation (ODD) and rare pediatric disease (RPD) designations from the FDA for MyoDys45-55, its a preclinical gene editing therapy targeted at a DNA region where mutations are found in approximately half of patients with Duchenne muscular dystrophy. "MyoDys45-55 targets the patient’s own DNA to permanently correct the mutation and restore gene function,” Courtney Young, PhD, the cofounder and chief executive officer of MyoGene Bio.
