Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

CEO Ron Cooper said the company’s focus is advancing its DDX platform, a proprietary non-viral gene therapy technology, with its lead program detalimogene being developed for non-muscle invasive ...

UB’s new Genetic Counseling Graduate Program has received accreditation from the Accreditation Council for Genetic Counseling (ACGC), the field’s national accrediting organization. The two-year Master ...

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene ...

What if the darkness that enveloped a child's world suddenly… vanished? What if hope, once a whisper, roared into life with a ...

A new chapter is unfolding for gene therapies in Europe. With retinal programs advancing into Phase I/II trials and ...

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...

Oppenheimer initiated coverage of Ocugen (NASDAQ:OCGN) with an Outperform rating and a $10 price target on Wednesday, ...

After a multi-year process of development and garnering approvals, the new Genetic Counseling Graduate Program at UB has received accreditation from the Accreditation Council for Genetic Counseling ...