Alcyone Therapeutics Opens, Focus on Gene Therapy for Rett Syndrome

Alcyone Therapeutics, a new company focused on developing gene therapies for disorders that affect the nervous system, announced its official launch.

One of its lead candidates, ACTX-101, is an investigational gene therapy for Rett syndrome now in preclinical testing with a goal of bringing into clinical trials in patients

“Our mission at Alcyone Therapeutics is to provide life-changing therapies for children and their families impacted by severe neurological conditions,” PJ Anand, founder, president, and CEO of Alcyone, said in a press release.

Gene therapies work to correct the underlying genetic defect that causes a particular disease. Alcyone reports that it is developing a dozen different gene therapies across four platforms.

ACTX-101 is part of Alcyone’s X-reactivation platform. Almost all cases of Rett syndrome are caused by mutations in the gene MECP2. This gene is on the sex-determining X-chromosome — females inherit two copies, while males inherit one (biologic males have one X and one Y chromosome).

Having two active X-chromosomes inside the same cell can actually be deadly for the cell. Because of this, in female cells, one of the two X-chromosomes gets randomly inactivated.

In Rett syndrome, this can mean a woman having an active X-chromosome with a mutated version of the MECP2 gene, and an inactive X-chromosome with a healthy version of the gene. The overall goal of X-reactivation is to activate the healthy version of the gene in such cases.

All of the Alcyone’s gene therapies use a modified version of adeno-associated virus (AAV) — a common gene therapy vector — to deliver a genetic payload to cells in the body.

According to Alcyone, the company’s therapy platforms allow for more precise and versatile delivery of AAV-based gene therapies within the central nervous system (CNS), which includes the brain and spinal cord. Delivering therapies into the CNS requires overcoming obstacles that are not present in other tissues. One prime example is the blood-brain barrier that, as its name suggests, tightly controls what substances from the blood can get into the brain.

“Our next-generation precision delivery platform shows strong potential in overcoming the fundamental challenge in CNS therapy development,” Anand said.

Alcyone’s gene therapies are currently being investigated through a collaboration with researchers at Abigail Wexner Research Institute at Nationwide Children’s Hospital in Ohio. The company’s launch was supported by $23 million in funding from funds affiliated with RTW Investments.

“Coupled with multiple cutting-edge gene therapy technologies and research approaches at the Abigail Wexner Research Institute … 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,” Anand said.

Added Piratip Pratumsuwan, managing director at RTW, who will join Alcyone’s board of directors: “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.”