Q4 2024 Earnings Call Transcript March 20, 2025 Abeona Therapeutics Inc. beats earnings expectations. Reported EPS is $-0.24, ...

Treatment shows potential to slow the progression of human degenerative eye diseases, including retinitis pigmentosa.

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

The discovery could pave the way for a groundbreaking RNA-based multi-cancer treatment. Alternative RNA splicing functions ...

Beam Therapeutics reported promising Phase 1/2 data for BEAM-302, demonstrating durable gene correction for AATD. See why I ...

Alnylam's Amvuttra enters ATTR-CM drug race, and Expedition Therapeutics seeks to bring Chinese drugs to the U.S.

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

Preclinical data with the investigational gene therapy INS1202 show improvements in survival and motor performance in ALS.

For 14 years, the Hillenberg family has been involved with Lowell-based Fitness for a Cure, a charity that supports St. Jude ...

A baby boy with a rare illness is making “incredible” progress after he became the first ever patient to receive a new gene ...