The Genes That Could Cancel Out a Fatal Diagnosis

Abraham Lincoln, for one, may have had the syndrome—and are at very high risk of a kind of fatal rupture in the heart.
Medical Xpress

ALG6 acts as a modifier gene in the inherited genetic eye disease retinitis pigmentosa 59

Modifier genes add complexity to precision medicine. A modifier gene is one that has genetic variants such as single nucleotide changes that differ from the most common sequence in the population.
Nasdaq

Ocugen, Inc. to Showcase Innovative Modifier Gene Therapy Platform at Upcoming ARVO and Retina World Congress Meetings

Ocugen, Inc., a biotechnology company focused on gene therapies for blindness diseases, announced its participation in two significant upcoming conferences: the ARVO 2025 Annual Meeting in Salt Lake ...
Nasdaq

Ocugen, Inc. Announces Signing of Binding Term Sheet for the License of OCU400 Modifier Gene Therapy for Retinitis Pigmentosa in Korea

MALVERN, Pa., June 05, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the ...
The American Journal of Managed Care

Splicing Modifiers and Gene Therapy for SMA

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...
Seeking Alpha

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2024 Annual Meeting

MALVERN, Pa., April 26, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN) (Ocugen or the Company), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell ...
Kaleido Scope

ALG6 acts as a modifier gene in the inherited genetic eye disease retinitis pigmentosa 59

Steven Pittler, Ph.D., at the University of Alabama at Birmingham has sought to find modifier genes for the hereditary eye disorder retinitis pigmentosa type 59. After onset in one’s late teens, RP59 ...