NEW YORK – Alcyone Therapeutics on Thursday said it has expanded a collaboration with Nationwide Children's Hospital and received an investment from the Columbus, Ohio-based hospital system and other existing investors.
Lowell, Massachusetts-based Alcyone did not disclose the other investors or the dollar amount it received in the financing round. Alcyone will use the recently raised funds to continue to develop its ThecaFlex DRx intrathecal drug delivery system, which is part of an ongoing codevelopment and copromotion collaboration with Biogen.
The latest financing also coincides with the recent addition to Alcyone's pipeline of a clinical-stage gene therapy program for Batten disease driven by CLN3 gene mutations that Nationwide Children's was developing. CLN-301 is now the third therapeutic candidate for pediatric central nervous system (CNS) diseases in Alcyone's pipeline.
"With the addition of CLN-301, Alcyone has enriched its clinical-stage pipeline of precision CNS therapeutics with a program exhibiting promising safety and preliminary efficacy data and significant commercial potential as a first-in-class disease-modifying therapy," Alcyone CEO PJ Anand said in a statement.
CLN-301 uses an adeno-associated virus 9 vector to deliver the coding sequence of the CLN3 gene to cells of the CNS. It is currently being evaluated in a Phase I/II trial for Batten disease, specifically a form of the rare and fatal inherited neurological disorder caused by CLN3 mutations, which result in progressive cell damage and neurodevelopmental decline. Initial results from four patients who have received CLN-301 suggest they're maintaining motor and cognitive function.