Rare Daily Staff

Alcyone Therapeutics launched with $23 million in funding to develop next-generation gene therapies for severe neurological conditions.

Funds affiliated with RTW Investments provided the capital and Managing Director Piratip Pratumsuwan will join Alcyone’s Board of Directors.

Alcyone says its precision CNS delivery technology platform allows for unprecedented control and versatility for biodistribution of genetic medicines to CNS regions of interest, including deep brain regions and spare off-target areas.

A collaboration with Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) leverages Alcyone’s delivery platform and AWRI’s research and technologies to advance broad pipeline that utilizes four gene therapy platforms—X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation—and 12 optioned AAV gene therapy programs targeting severe CNS disorders.

The lead programs target the treatment of Rett syndrome and spinal muscular atrophy with respiratory distress type 1.

“Our mission at Alcyone Therapeutics is to provide life-changing therapies for children and their families impacted by severe neurological conditions,” said P.J. Anand, founder, president and CEO of Alcyone. “Our next-generation precision delivery platform shows strong potential in overcoming the fundamental challenge in CNS therapy development. Coupled with multiple cutting-edge gene therapy technologies and research approaches at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, we aim to transform the efficiency of CNS therapeutics.”

Photo: PJ Anand, founder, president and CEO of Alcyone

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