Boost for pursuit of novel genetics-based treatment for Huntington’s

By Published On: 29 January 2025
Boost for pursuit of novel genetics-based treatment for Huntington’s

A life sciences firm aiming to harness the RNA molecule that carries genetic instructions to treat Huntington’s has been boosted by a major fundraise.

Atalanta Therapeutics, from Boston, US, has raised Us$75m from investors to help fund its groundbreaking search for genetics-based treatments for neurological diseases.

RNA is a molecule that carries genetic instructions from DNA, guiding cells in protein production and serving as a blueprint for cellular processes.

A method called RNAi enables the altering of these instructions, allowing the targeting of diseases at the molecular level by potentially silencing harmful genes.

Atalanta has developed a proprietary RNAi platform called di-siRNA, which, for the first time, enables RNAi to be deployed as a therapeutic approach throughout the brain and spinal cord.

With its new funding, the firm aims to advance its investigational RNAi therapies for KCNT1-related epilepsy and Huntington’s disease to Phase 1 clinical trials.

CEO and president Alicia Secor said: “[The funding] will support a path to the clinic for two programmes for serious neurological diseases that today lack disease-modifying therapies: KCNT1-related epilepsy and Huntington’s disease.

“We’re diligently progressing these medicines toward IND submissions next year so that we can start our Phase 1 trials and reach patients who are waiting.”

Arno de Wilde, of EQT Life Sciences, which led the new investment, said: “Atalanta’s di-siRNA technology has shown promising ability to durably and evenly silence disease-promoting genes throughout previously inaccessible regions of the brain and spinal cord – opening a wide range of treatment possibilities for devastating neurological diseases.”

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