• Leading precision medicine and nucleic acid therapy (NAT) innovators will harness their platforms to develop NAT drug candidates for novel gene targets with strong genetic association to sporadic ALS
• The programme will be supported by mechanism-based patient stratification biomarkers to identify likely responders and accelerate and derisk clinical development

OXFORD and HARWELL, UK – 26 March 2024 – The pioneering precision medicine company, PrecisionLife, and the MRC nucleic acid therapy research unit, NATA, today announce a collaboration to co-develop novel oligonucleotide therapies for sporadic ALS. The collaboration combines NATA’s expertise in NAT design and optimization with PrecisionLife’s deep 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 partnership builds on PrecisionLife’s unique insights into the mechanisms driving ALS and NATA’s translational research expertise and exploits the teams’ wide range of collaborations with patient charities, academic researchers and the biopharma industry.

“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”.

“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.