As the fairness of orphan drug exclusivities is debated in Congress, the U.S. Food and Drug Administration granted Orphan Drug designations to Editas Medicine and Neurocrine Biosciences.

Some of the best medicines happen by accident, according to BioSpace. The discovery of insulin began with two doctors removing the pancreas of a healthy dog in an attempt to understand its impact on digestion. Penicillin grew out of an unrelated scientific experiment involving Staphylococcus bacteria colonies. Will someone one day write the same of Novartis’ branaplam in correlation with Huntington’s disease?  

Three companies – Intellia Therapeutics, Editas Medicine and uniQure – are each anticipating a bright 2022 as the promises of gene therapy continue to be realized.

Ionis Pharmaceuticals announced that Roche, the company’s partner firm, is designing a new Phase II trial that would assess the effectiveness of tominersen in younger adult patients diagnosed with Huntington’s disease. 

Ipsen, a global biopharmaceutical firm that specializes in neuroscience, oncology and rare diseases, partnered with Exicure, a clinical-stage biotech company that develops treatments for neurological, inflammatory and genetic disorders, to create new therapies for Angelman syndrome (AS) and Huntington’s disease (HD).

Roche is calling it quits on a late-stage trial of the company’s Huntington’s disease hopeful tominersen, the Swiss drugmaker said on March 22, a blow for sufferers of the rare, debilitating disease with no treatments to stop or reverse it.

RNAi-focused Atalanta Therapeutics launched with $110 million in combined Series A funding and collaboration deals with Genentech and Biogen to address diseases related to the central nervous system, including Huntington’s, Alzheimer’s and Parkinson’s diseases.

Drugmakers agreed to cut prices by around 50% on average for more than 100 medicines in order to have them included in China’s state medical insurance scheme from March, potentially heralding a massive leap in sales.

Novartis is seeking to repurpose the company’s investigational oral spinal muscular atrophy (SMA) medicine branaplam to treat Huntington’s disease, as the Swiss drugmaker plans a clinical trial after winning U.S. orphan drug designation.

Vaccinex announced topline data from the early manifest treatment arm of the company’s Phase II SIGNAL trial of pepinemab in patients with early manifest and prodromal Huntington’s disease (HD).