Enzyvant, a subsidiary of Sumitovant Biopharma, received a long-awaited FDA approval for its tissue based regenerative therapy for treatment of congenital athymia last week after almost 30 years of development. The treatment, which is known as Rethymic, is a single use therapy that is inserted as small slices of tissue…

Pfizer saw positive topline results of its investigational gene therapy for adults with hemophilia A in a Phase III study (AFFINE). The therapy, giroctocogene fitelparvovec, reduced patients’ bleeds for at least 15 months and was generally well tolerated. It comprises a bio-engineered AAV6 capsid and a modified B-domain deleted human…

Updated interim data from a Phase I/II trial of uniQure’s “one shot” gene therapy in Huntington’s disease were promising, suggesting AMT-130 is effective and safe. Huntington’s is one of the most common rare diseases. It is caused by the expansion of CAG trinucleotide in exon 1 of a multifunctional gene…

A team of researchers from the University of Basel, Switzerland, and colleagues in Germany has demonstrated the benefit of specific exercise that can reduce a common side-effect of cancer treatment, chemotherapy-induced peripheral neuropathy (CIPN). As many as 90% of patients receiving therapies such as oxaliplatin or vinca alkaloids experience a…

Promising new data from a trial of Vertex’s early phase islet cell therapy VX-880 for type 1 diabetes (T1D) trial showed three patients, with 12 months of follow-up, reached insulin independence. All of the dozen patients in the trial showed improvement over all—they received the full dose of VX-880 and…

Over the last thirty years, protein-based injections and DNA therapeutics accounted for the majority of newly introduced treatments. However, these medicines remain out of reach for many around the world due to high pricing, with some therapies ranging from thousands to several millions of dollars to purchase. [caption id=”attachment_147257″ align=”alignright”…

Researchers at the University of California, San Francisco (UCSF) have begun enrollment for a clinical trial that uses a new, powerful version of CAR T as a potential therapy for glioblastoma, the most common and deadly adult brain cancer. The clinical trial is funded by an $11 million grant to…

The Food and Drug Administration (FDA) has granted accelerated approval to lifileucel (Amtagvi, Iovance Biotherapeutics), a tumor-derived autologous T cell immunotherapy, for adult patients with unresectable or metastatic melanoma. It is the first, and the only one-time, T cell therapy to receive FDA approval for a solid tumor cancer. The…

Life is like the changing of seasons, each uniquely beautiful. Just as autumn leaves fall to the ground and make way for winter’s gentle snow, life has transformational cycles. The cycle of life continues through the changing seasons, like how the autumn winds strip the trees of their leaves and…

Immusoft has dosed the first patient with an engineered B cell therapy—ISP-001 for MPS I (Mucopolysaccharidosis type I). This approach has several potential advantages over current gene and cell therapies. It does not require preconditioning (myeloablative chemotherapy) or immunosuppression, is administered as an IV on an outpatient basis, and it…

Entering the gene therapy field, Ajinomoto is acquiring Forge, a leading manufacturer of genetic medicines in an all-cash deal for about $554M. Forge provides end-to-end manufacturing services for gene therapy programs from preclinical through clinical and commercial manufacturing. “Forge’s unparalleled expertise in gene therapy development and manufacturing will be a transformative…

Research from Tongji University in Shanghai shows promising results from a Phase I human study of a cell therapy to treat chronic obstructive pulmonary disease (COPD). A group of 17 patients had notable improvements in breathing, walking distance, and quality of life after receiving the therapy, which is created using…

A multidisciplinary team at UC San Diego Health, participating in a clinical trial sponsored by Neurona Therapeutics, recently injected regenerative brains cells into the brain of a patient in an effort to treat their epileptic seizures. The regenerative brain cell therapy uses interneurons derived from human stems cells and has…

Ray Therapeutics, an optogenetics start-up founded in 2021, has announced a $100M Series A financing round led by Novo Holdings A/S. The funding will advance multiple programs targeting blinding diseases of the eye through clinical development. Ray’s lead candidate is RTx-015 in retinitis pigmentosa, a degenerative retinal disease. It is…

Bluebird bio has submitted to the FDA for approval of its sickle cell gene therapy. It’s a one-time treatment that adds a functional β-globin gene into the patient’s own hematopoietic (blood) stem cells. Bluebird’s move comes soon after Vertex and CRISPR Therapeutics submitted for their gene editing therapy in the…