There are currently no available treatment options for more than 90% of the approximately 7,000 rare diseases identified to ...

EXTON, PA, Aug. 14, 2025 (GLOBE NEWSWIRE) -- As the first wave of gene therapies for transfusion-dependent β-thalassemia (TDT) continues to roll out in the U.S., a new conversation is emerging within ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Gene therapy company Affinia Therapeutics has raised $40m in a Series C funding round, led by New Enterprise Associates (NEA) ...

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

Recently, Taysha Gene Therapies announced it has regained full rights to its lead gene therapy candidate TSHA-102 for Rett syndrome after the expiration of an option agreement with Astellas, following ...

StemRIM Inc. ( ($JP:4599) ) has shared an announcement. StemRIM Inc. has announced the patent registration for its stem cell gene therapy ...

This report by Towards Healthcare, a sister firm of Precedence Research, provides an in-depth analysis of the global cell and ...

The inaugural Genetic Agency Technology Conference (GATC) on November 11th, 2025 will convene technical thought leaders, gene therapy developers, patients, their families, and patient advocates across ...

When she was born, doctors informed Opal Sandy’s parents that their child would never hear. At the age of 11 months, she had ...