AAV gene editing therapy for Angelman syndromeUniversity of North Carolina at Chapel Hill scientists have created a CRISPR gene therapy for Angelman syndrome that ameliorated motor function deficits in a mouse model of the maternally...

The stain of “too high-risk for investment” that plagued neuroscience for years has now worn off, according to a BioCentury analysis of early stage venture investments. Neurology sits second only to cancer...

With a spate of new and expanded deals this year, Takeda is not letting its $62 billion dollar takeout of Shire slow its transformation from a traditional small molecule company to a home for edgier,...

New Therapeutic Targets and Biomarkers: November 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during November. Therapeutic targets are defined as any protein, gene or other molecule...

Cadent Therapeutics Inc. (Cambridge, Mass.) emerged from stealth with news of a $40 million tranched series B round to bring its lead compound through Phase II trials to treat essential tremor and spinocerebellar ataxia (SCA)....

Cadent Therapeutics Inc. (Cambridge, Mass.) emerged from stealth with news of a $40 million tranched series B round to bring its lead compound through Phase II trials to treat essential tremor and spinocerebellar ataxia (SCA)....

INDICATION: Blindness Mouse studies suggest inhibiting ATXN7 could help treat vision loss in spinocerebellar ataxia type 7 (SCA7), which is caused by gain-of-function mutations in the gene. In a genetic mouse model of SCA7, intravitreal...

INDICATION: Liver disease In vitro and mouse studies identified a peripherally restricted purine-based CNR1 antagonist that could help treat alcoholic liver steatosis. Chemical synthesis of analogs of the CNR1 antagonist otenabant and in vitro binding...

New Therapeutic Targets and Biomarkers: March 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during March 2018. Therapeutic targets are defined as any protein, gene or other...

INDICATION: Ataxia Cell culture, fruit fly and mouse studies suggest inhibiting KPNA3 could help treat spinocerebellar ataxia type 3 (SCA3), which is caused by mutant ATXN3 containing polyglutamine repeats that accumulates in the nucleus. In...