The U.S. Food and Drug Administration approved Soleno Therapeutics' drug to treat a rare genetic disorder on Wednesday, ...

Researchers from the J. Craig Venter Institute (JCVI), the Friedrich-Loeffler-Institut (FLI), and the International Livestock Research Institute (ILRI) have developed a reverse genetics system for ...

Mutations in BRCA2—a gene known to repair damaged DNA and suppress tumor formation—can predict an individual's predisposition ...

Tenaya Therapeutics (TNYA) announced the publications of positive preclinical data for TN-201, the company’s gene therapy candidate for Myosin-Binding Protein C3-associated hypertrophic cardiomyopathy ...

The goal of genome editing technology is to develop strategies for the correction of those mutations that are responsible of ...

Studies conducted in human induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs) compared various cassette configurations and informed the final design of TN-201, which incorporates a ...

A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...

The injection will test a new gene therapy that ... arthritis, and heart disease. And it is only one of a growing suite of potential treatments designed to address aging, as scientists race to reverse ...

Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics. Medera operates via ...

"The completion of enrollment in the Phase 1/2a trial marks a crucial milestone for this cardiac gene therapy trial in patients with heart failure with reduced ejection fraction (HFrEF).

An urgent need exists for disease modifying therapies, such as SRD-001, which aim to reverse the pathophysiology ... AAV-based cardiac gene therapy clinical trials targeting Heart Failure with ...

Double Oscar-winning actor Gene Hackman's tragic ... evidence of advanced Alzheimer's disease. He was in a very poor state of health. He had significant heart disease, and I think ultimately ...