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.
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 ...
Nature

Modifier variants in metabolic pathways are associated with an increased penetrance of Leber’s Hereditary Optic Neuropathy

Leber’s hereditary optic neuropathy (LHON) is the most common disease associated with pathogenic variants in the mitochondrial DNA (mtDNA) with an approximate prevalence of about 1 in 27,000–31,000 [1 ...
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 ...
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 ...
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 ...
Seeking Alpha

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress

MALVERN, Pa., April 29, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN) (Ocugen or the Company), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the ...