A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice.
Vanda Pharmaceuticals Inc. will get to take its argument for twice-rejected jet lag disorder drug Hetlioz (tasimelteon) before the U.S. FDA in a formal evidentiary public hearing, a rare move by the agency that the firm claims underscores the “gravity of the legal and scientific issues” it has raised.
A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.
CHA University has identified chalcone derivatives acting as acetylcholinesterase (AChE) and butyrylcholinesterase (BuChE) inhibitors potentially useful for the treatment of Alzheimer’s disease.
Ono Pharmaceutical Co. Ltd. has entered into an agreement to expand its drug discovery collaboration agreement with Congruence Therapeutics Inc. for the discovery of novel small-molecule modulators against multiple protein targets in the areas of neurology and immunology.
Cure Rare Disease has entered into a multiyear partnership with the LGMD2L Foundation to develop a gene replacement therapy for anoctamin 5 (ANO5)-related disease, a rare genetic disorder.
The busy Prader-Willi syndrome (PWS) space took another hit as did shares of Aardvark Therapeutics Inc. (NASDAQ:AARD), which closed March 2 at $5.47, down $7.02, or 56%, after the company disclosed a voluntary pause of the phase III Hunger Elimination or Reduction Objective (HERO) trial testing ARD-101 as a treatment for hyperphagia, or intense hunger, in patients with the disease.
Uniqure NV is the latest firm to get caught between the FDA’s shifting demands for “gold standard” science and regulatory flexibility for rare disease therapies. The company disclosed in its latest earnings report that U.S. regulators are calling for a sham-controlled study before they will consider approval of gene therapy AMT-130 in Huntington’s disease, a requirement that could set the program back by two to three years and raises potential ethical issues.
Sumitomo Pharma Co. Ltd. has patented aminomethylcyclohexane derivatives acting as muscarinic M4 receptor antagonists. As such, they are described as potentially useful for the treatment of neurodegenerative diseases, cerebrovascular disorders, brain injury, movement and psychiatric disorders.
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