Alcyone Therapeutics Secures Funding to Develop Pediatric Neurological Therapies

Alcyone Therapeutics has announced a significant advancement in its mission to transform pediatric care, focusing on precision CNS therapeutics. The company recently secured strategic funding from Nationwide Children's Hospital, which further solidifies their collaboration. This partnership now includes a promising gene therapy, CLN-301, aimed at treating CLN3 Batten disease, a rare and fatal neurological disorder primarily affecting children.

CLN-301 is currently in clinical development and represents a potential breakthrough for those affected by CLN3 Batten disease. This condition, also known as juvenile neuronal ceroid lipofuscinosis, is a genetic disorder causing progressive neurological decline due to an enzyme deficiency. Symptoms typically include rapid vision loss, cognitive and motor deterioration, and behavioral challenges. Unfortunately, there are no approved treatments for this devastating disease.

The gene therapy, utilizing AAV9 vectors, is designed to deliver the missing CLN3 gene to central nervous system cells, addressing the enzyme deficiency that leads to the disorder’s progression. Early results from a Phase 1/2 clinical trial have shown encouraging outcomes. In this trial, four patients receiving CLN-301 exhibited safety and therapeutic benefits when compared to the natural progression of the disease. Patients in the trial remained stable on the Unified Batten Disease Rating Scale, with far less impairment over three years than expected according to natural history data.

Dr. Emily De Los Reyes, Director of the Batten Disease Center of Excellence at Nationwide Children's, emphasized the importance of continued research and clinical trials in providing care and support for families dealing with Batten disease. The ongoing trial results suggest that CLN-301 has the potential to significantly impact the quality of life for patients and their families by altering the course of the disease.

Kathrin Meyer, Ph.D., Chief Scientific Officer at Alcyone Therapeutics, highlighted the transformative potential of CLN-301 in addressing the underlying causes of CLN3 Batten disease. Similarly, Amy Fenton Parker, President & CEO of the BDSRA Foundation, expressed enthusiasm about the program moving forward, acknowledging the significant unmet need it addresses.

The collaboration with Nationwide Children's Hospital enriches Alcyone’s pipeline of CNS therapeutics. In addition to CLN-301, Alcyone’s portfolio includes XGR-101, a genetic medicine for Rett Syndrome, and Efralex, a preclinical small molecule targeting neurodegeneration. These assets leverage Alcyone’s FalconTM platform, which enhances drug delivery to the central nervous system through advanced modeling and bioengineering.

PJ Anand, CEO of Alcyone Therapeutics, expressed gratitude for the expanded partnership with Nationwide Children's Hospital. The integration of CLN-301 into Alcyone’s portfolio exemplifies the company's commitment to developing first-in-class, disease-modifying therapies with significant commercial potential.

Batten disease encompasses a range of inherited neurological disorders characterized by cellular recycling defects due to genetic mutations. Among the 13 known forms, CLN3 Batten disease is caused by mutations in the CLN3 gene, leading to severe neurological impairments and early death.

Nationwide Children's Hospital, renowned for its comprehensive pediatric care and research, plays a pivotal role in advancing treatments for neurological diseases. Its Abigail Wexner Research Institute is a leader in pediatric research, supporting innovative therapies and groundbreaking scientific discoveries.

Alcyone Therapeutics, through its strategic collaborations and advanced therapeutic platforms, is at the forefront of developing precision treatments for pediatric neurological conditions, aiming to improve outcomes and enhance the quality of life for affected children and their families.