Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

The technology was announced at the JP Morgan Healthcare Conference in San Francisco. In this project, the stakeholders aim to use a human reference genome to combine with patient data in order to try ...

Gemini Bioproducts, LLC ("GeminiBio"), a biopharma and advanced therapy raw materials supplier, and a portfolio company of BelHealth Investment Partners, LLC ("BelHealth"), a Fort Lauderdale-based ...

The early stage company is developing a new approach to gene editing to treat rare genetic diseases. It does this using mRNA, ...

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

New gene editing methods have been introduced in the country, bringing new hope to couples who tested positive for certain ...

Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys for ...

Research continues in this field of precision oncology and there has been an explosion of novel drugs in this field ...

Taysha Gene Therapies offers hope for Rett syndrome with innovative treatment, strong trial results, and promising regulatory ...