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Quark Pharmaceuticals, SUNY, Opko, UMMS, RXi Pharmaceuticals

Quark Extends Hearing Loss Drug Alliance with SUNY
Quark Pharmaceuticals said last month that it has extended a research agreement with the State University of New York at Buffalo’s Center for Hearing & Deafness, which is currently conducting preclinical studies of the company’s RNAi-based hearing loss drug candidate, AHLi-11.
Specific terms of Quark’s arrangement with the center were not disclosed.
AHLi-11, an siRNA that targets p53, is being developed as a therapy for acute hearing loss associated with acoustic trauma or ototoxic drugs such as aminoglycoside antibiotics.
“We are encouraged by the advancement of AHLi-11 for acute hearing loss and look forward to filing an IND by year-end for the prevention of chemotherapy-induced hearing loss,” Danny Zurr, CEO of Quark, said in a statement.

Opko Begins Dosing Patients in Phase III AMD Trial
Opko Health said last month that it has dosed the first patient in a phase III trial of the company’s siRNA-based wet age-related macular degeneration drug bevasiranib.
The international study — the first phase III trial of an RNAi drug — will assess in more than 330 wet AMD patients whether bevasiranib administered every 8 or 12 weeks is safe and as effective in preventing vision loss as Genentech's top-selling AMD drug Lucentis, which is administered every four weeks.
RNAi News first reported on the phase III study’s design in late June (see RNAi News, 6/28/2007).
Opko acquired the rights to bevasiranib when it merged with Acuity Pharmaceuticals in March (see RNAi News, 3/29/2007).

UMMS siRNA Patent, Licensed to RXi, Issues in Australia
RXi Pharmaceuticals said this week that an Australian patent it has exclusively licensed in certain fields from the University of Massachusetts Medical School was granted.
The patent covers siRNA molecules and their use in methods for inducing RNA interference of a target gene in mammalian cells in vivo, according to RXi. The company holds exclusive rights to the patented technology for inhibiting human cytomegalovirus, the mutant SOD1 gene associated with familial amyotrophic lateral sclerosis, and gene targets associated with type 2 diabetes and obesity.

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.