Inside a heated tent on the Children’s Hospital of Philadelphia campus, 17-month-old KJ Muldoon wiggled around in his mother’s arms, smiling and clinging to a multicolored toy. This time, the toddler ...

Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just ...

SAYRE, PA, UNITED STATES, January 13, 2026 /EINPresswire.com/ -- Bookspert is excited to announce the launch of Lawless ...

For the first time in history, doctors have successfully used a personalized gene-editing therapy to treat a rare genetic disorder. The patient, a baby named KJ, was born with a condition called CPS1 ...

In a medical breakthrough, a child diagnosed with a rare genetic disorder has received a bespoke CRISPR gene editing therapy to correct the mutation, and is already showing signs of thriving. The ...

US biotech start-up Aurora Therapeutics has launched with $16 million in seed funding from Menlo Ventures, aiming to turn one-off CRISPR gene-editing successes into a scalable model for rare diseases.

Clinical and commercial quality controls are eased as the FDA looks to expedite therapy approvals in areas of unmet need.

After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D ...

Stephanie Innes Dec 23, 2016 Dec 23, 2016 Updated Jun 14, 2018 University of Arizona researchers, buoyed by support from dog lovers and a California pharmaceutical startup, say a canine vaccine ...