The autosomal dominant form of osteopetrosis, referred to as autosomal dominant osteopetrosis type 2 (ADO2), is caused by single allele dominant negative mutations of the CLCN7 gene. In a recent paper, researchers from Sisaf Ltd. detailed the development and preclinical evaluation of novel silicon stabilized hybrid lipid nanoparticles (sshLNPs), SIS-101-ADO, designed to deliver small interfering RNA (siRNA) specific against the human CLCN7 G215R mRNA.
How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder Sept. 27 as it looks at the data for Brainstorm Cell Therapuetics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS. Nurown is going into the adcom with a bit of a checkered history that includes a refuse-to-file letter and a single phase III trial that failed to demonstrate efficacy for the primary endpoint and all key secondary efficacy endpoints, according to the FDA briefing document.
Researchers from the University of Florida have published preclinical data for the novel indoleamine 2,3-dioxygenase (IDO) and galectin-3 (Gal3) fusion protein (IDO-Gal3), being evaluated for the treatment of osteoarthritis (OA).
Long bones, vertebrae and skull bones have distinct types of stem cells, and new insights into those stem cells could lead to new ways to treat both rare developmental disorders of skull formation and the all-too-common phenomenon of bone metastases. Scientifically, the work, which was published in two papers by Matthew Greenblatt and colleagues in Nature, adds to the increasing understanding of bone’s complexities. “Bone may serve as an endocrine organ that is secreting factors throughout the body,” Greenblatt said.
“We’re going to battle,” PTC Therapeutics Inc. CEO Matthew Klein said, responding to a surprise negative opinion from the EMA’s Committee for Medicinal Products for Human Use on converting the conditional marketing authorization to full status for Translarna (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy. The opinion applies to the renewal of the existing conditional authorization, too.
Di-cyclopropyl based interleukin-17A (IL-17A) production inhibitors have been reported in a Dice Alpha Inc. patent as potentially useful for the treatment of psoriasis, radiographic axial spondyloarthritis (ankylosing spondylitis), hidradenitis suppurativa, spondyloarthritis, psoriatic and rheumatoid arthritis.
Verge Genomics Inc. has entered a second big AI deal with a large drug company. Privately held Verge will receive up to $42 million, including up-front, equity and near-term payments from Alexion, Astrazeneca Rare Disease, to identify multiple targets for rare neurodegenerative and neuromuscular diseases. The deal could top out at $840 million. There also is potential for downstream royalties.
The interleukin-17 (IL-17) family is involved in innate immunity regulation and plays a crucial role in inflammatory and autoimmune disorders by sustaining inflammation during the onset and progression of diseases such as rheumatoid arthritis (RA), psoriasis or inflammatory bowel disease (IBD). Researchers from the University of Naples have reported on a new biological entity, Ab-IPL-IL-17, that displays potent anti-inflammatory activity in preclinical testing.
While an “unexpected placebo effect” marred its primary endpoint, the pivotal phase II/III study testing AMO-02 (tideglusib) showed clinically significant benefits across a range of functional and objective assessments, according to developer Amo Pharma Ltd., which is prepping to meet with regulators to discuss potential approval for use in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), an ultra-rare subtype of myotonic dystrophy type 1 for which no treatment options are available.
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