Atalanta Therapeutics Secures $97M Series B Financing to Advance RNAi Therapies for KCNT1-Related Epilepsy and Huntington’s Disease into Clinical Trials

"This financing validates Atalanta's leading di-siRNA platform's transformative potential in delivering oligonucleotide therapies to the central nervous system, as well as the immense promise of our broad wholly-owned pipeline," said Alicia Secor, President and CEO of Atalanta. "Importantly, this Series B financing will support advancing two programs into the clinic for severe neurological diseases that currently lack disease-modifying therapies – KCNT1-related epilepsy and Huntington’s disease – and will bolster our growing pipeline of investigational drugs for Huntington’s disease. We are aggressively advancing these programs with plans to file INDs this year to initiate Phase 1 clinical trials, bringing hope to those waiting for treatment.""Patients."

ATL-201 is Atalanta Therapeutics' investigational therapy for KCNT1-related epilepsy, a form of early-onset epilepsy and encephalopathy driven by gain-of-function variants in the KCNT1 gene. Infants and children with KCNT1-related epilepsy experience severe and frequent seizures that are resistant to anti-seizure medications, and they often also face developmental delays and intellectual disabilities. ATL-201 aims to reduce KCNT1 levels and restore normal neuronal excitability. Preclinical studies have shown that ATL-201 significantly reduces seizure frequency and improves behavior, demonstrating excellent durability and tolerability.

The company's second development candidate, ATL-101, is a di-siRNA designed to silence the HTT gene for the treatment of Huntington's disease. Huntington's disease is a progressive neurodegenerative disorder caused by the expansion of the HTT gene, leading to the gradual deterioration of patients' physical, cognitive, and psychiatric abilities. Preclinical studies have shown that a single dose of ATL-101 can significantly reduce HTT expression, including in deep brain regions, with six months of durability and good tolerability.

AboutAtalanta Therapeutics

Atalanta TherapeuticsAtalanta Therapeutics is a biotechnology company that utilizes RNA interference technology to develop treatments for refractory diseases of the central nervous system. Atalanta's unique bivalent small interfering RNA (di-siRNA) platform enables durable, selective gene silencing in the brain and spinal cord. Atalanta is advancing a series of wholly owned disease-modifying programs, covering Huntington's disease,Hereditary Epilepsy, severe chronic pain and other neurological diseases, while being associated withGenentechThe strategic cooperation is also underway. Atalanta Therapeutics is headquartered in Boston, Massachusetts.