Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

The companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the genetic defects in the BBS1 gene.

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side effect experienced by one participant on a high dose of its Rett therapy.

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...

Take a look overseas, the U.S. market currently counts 43. However, since 2020, the European Medical Agency (EMA) has made notable progress in approving advanced therapy medicinal products (ATMPs) – ...

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of ...

Although these diseases were initially treated by transplantation of allogeneic hematopoietic stem cells, the past 20 years has shown that these conditions are correctable by gene therapy.

Roger Hajjar, ’86, is the inaugural director of the Gene and Cell Therapy Institute at Mass General Brigham and the president, chief medical officer, and co-founder of Medera, a clinical-stage ...

MORE GENETIC MODIFICATIONS: Gene Therapy at Duke Improves NC Dad’s Failing Vision Just in Time for His Baby’s Birth “In my opinion, I’m perfect. I never felt fine before—before ...

Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...

Understand the critical steps involved in translating laboratory research into clinical-grade cell and gene therapies. Explore how innovations in cell and gene therapy are paving the way for more ...