R&D partnership to jointly develop new ALS drugs

Biochemists working in the lab

PrecisionLife and the Nucleic Acid Therapy Accelerator (NATA) have announced a collaboration to co-develop novel oligonucleotide therapies for sporadic motor neurone disease/ALS.

The collaboration combines NATA’s expertise in NAT design and optimisation with PrecisionLife’s understanding of complex disease biology and the mechanisms, drug targets and treatments that are relevant to specific ALS patient subgroups.

The target and mechanism of the first programme is predicted to have a prevalence amongst ALS patients of 15-30x that of SOD1 (the gene target for tofersen).

“The ALS patient community is rightly impatient for a range of new disease modifying approaches for such a devastating disease. Co-development with NATA brings a rapid, clear development path for one such novel drug programme,” said Steve Gardner, CEO of PrecisionLife. “We are particularly excited about the opportunity to explore NATs as modern AAV technologies have shown excellent potential for delivering oligonucleotides across the blood brain barrier.”

The partnership builds on PrecisionLife’s insights into the mechanisms driving ALS and NATA’s translational research expertise and exploits a wide range of collaborations with patient charities, academic researchers and the biopharma industry.

NATA is a UK Research & Innovation (UKRI)/Medical Research Council funded research unit, based at the Harwell Science and Innovation Campus. NATA’s mission is to advance the development of nucleic acid therapies and associated technologies through high-quality science, collaborations and interdisciplinary research.

“We are excited to be working with PrecisionLife, bringing together world-renowned experts in precision neuroscience with unique deep insights into ALS disease biology and design of nucleic acid therapies. This disease needs multiple innovative approaches for different disease mechanisms,” said Professor Nick Lench, Executive Director, NATA.

Diana Spencer, Senior Digital Content Editor, DDW

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