Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

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HAYWARD, Calif. - Benitec Biopharma Inc. (NASDAQ: BNTC), a clinical-stage biotechnology company with a market capitalization of $225 million, announced today that it will present interim data from its ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Boehringer will leverage ExpressionEdits' Genetic Syntax Engine which generates optimized introns to increase protein expression.

New gene therapy modalities, such as CRISPR guide RNA (single guide ribonucleic acid [sgRNA]) and messenger RNA (mRNA), ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...