Aptose’s Tuspetinib Exceeds Expectations When Combined with Standard of Care Treatment Across Diverse Populations of Newly Diagnosed AML

Addition of TUS to VEN+AZA achieves CR/CRh responses in all (6/6, 100%) patients treated at the higher dose levels of 80 mg and 120 mg TUS, ...
BioWorld

AAV-delivered PPT1 gene therapy shows promise for CLN1 disease

Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...
Pharmabiz

AAVantgarde receives US FDA orphan drug designation & UK CTA approval for AAVB-039 to treat Stargardt disease

AAVantgarde receives US FDA orphan drug designation & UK CTA approval for AAVB-039 to treat Stargardt disease: Milan Saturday, October 4, 2025, 17:00 Hrs [IST] AAVantgarde Bio (AA ...
TMCnet

AAVantgarde Announces FDA Orphan Drug Designation and UK CTA approval for AAVB-039 for the Treatment of Stargardt Disease

AAVB-039 addresses the root cause of the disease, benefitting patients with any ABCA4 mutation AAVB-039 is currently being assessed in the CELESTE interventional clinical trial and we continue ...
Toronto Star

AAVantgarde Announces FDA Orphan Drug Designation and UK CTA approval for AAVB-039 for the Treatment of Stargardt Disease

the company’s investigational gene therapy for the treatment of Stargardt disease secondary to biallelic mutation in ABCA4, Orphan Drug Designation (ODD). The company has also received Clinical Trial ...
Ophthalmology Times

Positive pediatric data emerge from the OPGx-LCA5 phase 1/2 trial of Leber congenital amaurosis type 5

Opus Genetics today announced the positive 3-month pediatric data from its Phase 1/2 open-label, ascending-dose study of the safety and efficacy of OPGx-LCA5, an investigational gene therapy for Leber ...
Precision Medicine Online

Opus Genetics Hoping to Solidify LCA5 Gene Therapy Development Plan With FDA Later This Year

In the meeting, the firm will discuss with the agency new, positive three-month data from the pediatric cohort of the Phase I/II OPGx-LCA5-1001 trial.
The Manila Times

Opus Genetics Reports Positive Pediatric Data from OPGx-LCA5 Phase 1/2 Trial in Leber Congenital Amaurosis Type 5 (LCA5)

Pediatric participants demonstrated large gains in cone-mediated vision; therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicitiesLasting, durable responses ...
BioWorld

Cure Rare Disease’s CRD-003 designated orphan drug for LGMD2i/R9

The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic ...
Phys.org

Key mutation for later flowering of barley discovered

An international research team led by the Leibniz Institute of Plant Genetics and Crop Plant Research (IPK) has discovered a gene mutation (PPD-H1) that causes barley to flower later in regions with ...
Variety

Busan Titles ‘The Mutation,’ ‘Time of Cinema,’ ‘Boss,’ ‘Journey There’ Picked Up by Finecut

Korean sales agent Finecut has acquired international rights to three films — “The Mutation,” “Time of Cinema” and “Wrangler” — and is presenting two additional titles at the Busan International Film ...