Alcyone’s Next Level CNS Gene Therapy Initiative

As reported in PR Newswire, Alcyone Therapeutics is launching a new set of precise gene therapies for CNS disorders with the help of a new $23 million investment from RTW Investments. To reach the biotech company’s goal of improving the lives of patients living with intensive neurological conditions, the company is teaming with researchers at Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) to draw together expertise, technologies, and production tools into a collaboration.
Their collaboration will draw on the expertise of physicians Kathrin Meyer, Ph.D., and Nicolas Wein, Ph.D at AWRI. The team will utilize Alcyone’s ultra-precise gene therapy platform in efforts to create treatment options for the central nervous system.

The Collaboration

They’ll be teaming with researchers at the AWRI who have been studying and applying gene therapies for rare patients. The hospital will bring to the toolkit their own set of four gene therapy technologies, as well as capabilities for advanced discovery, research, and development. Together, AWRI and Alcyone are powerful team, joining technological might and a team of genetic experts.
The Next-Generation CNS Delivery Platform
The biotech company has the most modern precision tool kit to create CNS treatments. They plan to use a next-generation CNS delivery platform, which has the capacity to deliver and dose a gene therapy with extreme precision. The technology’s versatility gives it an edge, enabling researchers to have much more control over their therapy and CNS bio-distribution.
 Alcyone’s mission to reduce the burden of severe neurological diseases means top level science, so they come with the top resources. They have four gene therapy platforms that can preform X-reactivation, conventional transgene replacement, vectorized exon skipping, and promotor modulation. They already have 12 adeno-associated virus gene therapy programs in development.
“Our mission at Alcyone Therapeutics is to provide life-changing therapies for children and their families impacted by severe neurological conditions,”
said Alcyone’s Founder, President, and CEO, PJ Anand.
“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. As we advance our broad pipeline towards the clinic, we are grateful to the investors who have supported us and pleased to have the financial and strategic support of RTW.”
Alcyone’s Board of Directors member, Piratip Pratumsuwan, commented,
“We are excited by the potential for Alcyone’s CNS precision delivery platform to generate a pipeline of drug candidates aimed at improving the lives of patients affected by neurological disease. I look forward to working with Alcyone’s Board and team of experienced gene therapy leaders as they advance towards the clinic.”

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