Alcyone Therapeutics picks up $23M in hopes of being a star in treating CNS disorders

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Alcyone Therapeutics has $23 million to fuel its AAV gene therapies for severe central nervous system disorders. (efetova/iStock/Getty Images Plus/Getty Images)

Alcyone Therapeutics hopes to be a standout star and rising goddess in the central nervous system (CNS) treatment landscape.

The Massachusetts biotech unveiled itself Wednesday with $23 million in funding from RTW Investments with a pipeline of 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone lead programs include ACTX-101, a Rett syndrome gene therapy in pre-IND enabling studies, and ACTX-401, an IND-stage gene therapy for spinal muscular atrophy with respiratory distress type 1.

Alcyone, a name derived from Greek mythology as well as one that shares its name with a star, is coming out of the gates with four gene therapy platform technologies in a collaboration with the Abigail Wexner Research Institute at Nationwide Children’s Hospital. The technologies include X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation.

The company, which said it launched today but has been in the works for many years, also inked a “broad strategic collaboration” with Roche in December 2018, according to Alcyone’s website. The collaboration is meant to “optimize intrathecal therapies and develop novel antisense oligonucleotide delivery treatment options for patients with neurological disorders,” Alcyone says on its website.  

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

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Alcyone is going up against recent biotechs to emerge with a focus on CNS disorders. Taysha Gene Therapies is one. The Dallas-based pivotal-stage company provided preclinical data showing statistically significant survival extension in mouse models of Rett syndrome. Taysha moved quickly last year, uncloaking in April, picking up a $95 million series B in August and an initial public offering in September.

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Atalanta Therapeutics is another recent startup to come out of stealth this year to focus on the CNS, with a focus on silencing genes in the brain. The biotech was infused with a $110 million series A in January and has partnerships with Biogen and Roche’s Genentech.

In conjunction with Alcyone’s funding, RTW Managing Director Piratip Pratumsuwan joins the board.

Alcyone Therapeutics emerges from device company Alcyone Lifesciences, which in March 2019 received breakthrough device designation from the FDA for its implantable subcutaneous port and intrathecal catheter. The system is for patients 3 years or older who need chronic bolus intrathecal treatment for life threatening CNS disorders. In May 2015, Alcyone Lifesciences had also partnered with DNAtrix on a clinical collaboration for brain cancer.