Atalanta Therapeutics Advances RNA-Based Epilepsy Treatment Towards Clinical Trials

Atalanta Therapeutics, a Boston-based biotech company backed by major pharmaceutical players, is making significant strides in the development of a novel RNA therapy for a rare form of genetic epilepsy. The company's lead candidate, ATL-201, has shown promising results in preclinical studies, paving the way for its anticipated entry into clinical trials.

Preclinical Success in KCNT1-Related Epilepsy

ATL-201, a small interfering RNA (siRNA) molecule, has demonstrated remarkable efficacy in suppressing seizures in mouse models of KCNT1-related epilepsy. In a recent study published in the journal Epilepsia, a single injection of ATL-201 into the cerebrospinal fluid resulted in an average 84% reduction in seizures compared to control mice, three days post-treatment.

The therapy targets the KCNT1 gene, which codes for a potassium transport protein. Mutations in this gene can lead to hyperactive potassium channels, disrupting neuronal activity and causing frequent seizures in affected children, sometimes beginning within days of birth.

Dr. Aimee Jackson, Chief Scientific Officer at Atalanta, emphasized the significance of these results, stating, "We are working diligently to prepare ATL-201 to enter the clinic in the hopes of bringing the first disease-modifying therapy for KCNT1-related epilepsy to the families who urgently need it."

Strategic Partnerships and Financial Backing

Atalanta Therapeutics has garnered substantial support from the pharmaceutical industry since its inception in 2021. The company launched with a $110 million Series A funding round, securing deals with Genentech (a Roche subsidiary) and Biogen. Although the collaboration with Biogen has since concluded, Atalanta has regained full rights to two preclinical programs that were part of the initial partnership.

In a recent development, Atalanta successfully raised $97 million in a Series B funding round in January. This latest financial boost, which included investments from Sanofi and Novartis through their respective venture funds, is earmarked to support the clinical advancement of ATL-201 and another siRNA asset targeting Huntington's disease.

Looking Ahead: Clinical Trial Preparations

With the preclinical phase showing promising results, Atalanta is now setting its sights on the clinical stage. The company has announced plans to submit an Investigational New Drug (IND) application for ATL-201 to the FDA this year, marking a crucial step towards human trials.

As Atalanta prepares for this next phase, the potential of ATL-201 as the first disease-modifying therapy for KCNT1-related epilepsy represents a significant advancement in the field of rare genetic disorders. The coming months will be critical as the company transitions from preclinical success to the challenges and opportunities of clinical development.