28 January 2025 — Atalanta Therapeutics, a Boston-based biotech, has announced the successful close of a $97 million Series B financing round, an oversubscribed raise that highlights the growing enthusiasm for RNA interference (RNAi) approaches in neurological disease. With this fresh capital, the company plans to advance two of its lead programs into Phase 1 clinical trials by the end of 2025—one targeting KCNT1-related epilepsy and the other focusing on Huntington’s disease.

A Focus on Rare and Devastating Neurological Conditions

KCNT1-related epilepsy, though rare, is a severe paediatric condition caused by mutations in the KCNT1 gene that lead to early-onset, drug-resistant seizures. Families affected by this condition face limited treatment options and significant quality-of-life challenges. Huntington’s disease, on the other hand, is a well-known genetic neurodegenerative disorder that progressively robs patients of movement, cognition, and independence. Despite years of research, effective disease-modifying therapies remain elusive.

By targeting the genetic roots of these conditions, Atalanta’s RNAi programs aim not just to manage symptoms but to alter the course of disease itself—something no current treatment can achieve.

Innovative Delivery Platform

Atalanta’s programs rely on its proprietary di-siRNA platform, which consists of double-stranded interfering RNAs designed to achieve potent, durable, and widely distributed gene silencing in the central nervous system. Historically, delivering RNA-based therapies to the brain has been one of the biggest technical hurdles. Atalanta’s technology claims to overcome these barriers, enabling consistent distribution after intrathecal administration and potentially reducing the need for frequent dosing.

The company believes this approach could transform the treatment landscape for central nervous system (CNS) disorders, where precision targeting and long-lasting efficacy are critical.

A Significant Financing Round

The $97 million Series B was described as oversubscribed, a testament to investor confidence in the company’s leadership and platform. This financing follows a $110 million Series A announced in 2021, bringing Atalanta’s total funding close to a quarter of a billion dollars in just a few years. The new capital will be directed toward advancing the KCNT1 and Huntington’s programs into the clinic, while also supporting earlier pipeline efforts in other CNS diseases.

In an environment where biotech fundraising has become more challenging, such a substantial raise underscores how select platforms with strong translational potential continue to attract significant attention.

The Broader Industry Context

RNA interference therapies have already proven their worth in conditions like transthyretin amyloidosis and acute hepatic porphyrias, but their applications in neurology remain relatively new territory. Recent advances in drug delivery to the brain have reignited interest in RNA-based approaches for CNS diseases. Atalanta is part of a growing group of companies—including Wave Life Sciences, Ionis Pharmaceuticals, and Alnylam—that are pushing the frontiers of gene-silencing modalities in neurology.

If successful, Atalanta’s therapies could not only provide life-changing options for patients but also validate RNAi as a mainstream strategy for CNS drug development. The coming years will be crucial

Future prospects

Atalanta expects to file IND applications for both KCNT1-related epilepsy and Huntington’s disease later in 2025, with first-in-human dosing anticipated soon after. These trials will test the safety, tolerability, and initial signals of efficacy for the company’s di-siRNA constructs. Success at this stage would not only advance treatments for two high-need indications but also open the door to broader applications across the CNS disease spectrum.

As RNAi edges further into neurological disorders, Atalanta’s work could mark a turning point in how genetic conditions of the brain are treated—offering hope to patients who have long been underserved by traditional drug development.