Certa Therapeutics Pty Ltd. has acquired Occurx Pty Ltd. in a move to strengthen its pipeline to target multiple fibrotic diseases as both companies share a focus on targeting GPR68, a defined G protein-coupled receptor (GPCR) receptor that mediates signaling pathways associated with inflammation and fibrosis and is thought to be a master switch of fibrosis.
Vigeneron GmbH has received IND approval from the FDA for VG-801, a novel mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene. IND clearance enables initiation of a phase I/II trial, with a clinical trial application (CTA) submission to the EMA also planned in the coming months.
Lento Bio Inc. has synthesized cysteine derivatives reported to be useful for the treatment of presbyopia. An exemplified compound (Cpd in row 4 in tbl1 pg 20, claim 18) decreased in lens stiffness in C57BL/6 mice (at 20 mM; as eye drops).
Splicebio S.L. has gained IND clearance from the FDA for its lead program SB-007, a protein splicing gene therapy to address the root genetic cause of Stargardt disease.
Outlook Therapeutics Inc. is forging onward with plans to resubmit the BLA for ONS-5010, an ophthalmic version of Avastin (bevacizumab, Roche AG), after a missed endpoint in phase III with the VEGF binder for wet age-related macular degeneration (AMD).
Celltrion Inc. said it would acquire Iqone Healthcare Switzerland SA in the fourth quarter of 2024 for about ₩30 billion (US$21.34 million) to expand its European business. “This move represents a strategic shift in our growth strategy,” Taehun Ha, Celltrion vice president and Europe head, said in a statement Nov. 15.
The U.S. FDA issued a complete response letter Nov. 15 for Izervay’s (avacincaptad pegol intravitreal solution, ACP) supplemental NDA, which sought to include positive two-year data for the Astellas Pharma Inc. therapy, previously approved for use in a treatment for geographic atrophy secondary to age-related macular degeneration.
Researchers from the U.K. have analyzed whole-genome sequencing data from 7,276 cases and 236,741 controls in the UK Biobank to perform gene-level and a variant-level exome-wide association study analysis to identify variants related to retinal detachment.
Celltrion Inc. said it would acquire Iqone Healthcare Switzerland SA in the fourth quarter of 2024 for about ₩30 billion (US$21.34 million) to expand its European business. “This move represents a strategic shift in our growth strategy,” Taehun Ha, Celltrion vice president and Europe head, said in a statement Nov. 15.
WDR45 is located on the X chromosome. Its pathogenic variants are associated with various neurodegenerative disorders that are predominantly reported in females and present a wide range of clinical phenotypes, from early-onset developmental delay to neurodegeneration and multiple epileptic syndromes.
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