On 26 May, the EHA, in collaboration with Roche, organised an informative webinar to present POINT-HD, the new Phase I clinical trial that the company is conducting in 12 countries.
Roche has been researching new therapies for Huntington’s disease since 2013, and this new study represents a further step in the development of treatments specifically targeting the genetic cause of the disease.
The POINT-HD trial, which began in December 2025, is aimed at people aged between 25 and 65 with early-stage Huntington’s disease who meet certain clinical and genetic criteria.
The study is evaluating RG6496, an experimental treatment based on an allele-specific antisense oligonucleotide (ASO), designed to reduce the production of mutant huntingtin (mHTT) whilst preserving normal huntingtin (HTT). The treatment is administered via intrathecal injection following a lumbar puncture.
During the webinar, Dr Peter McColgan and Dr Patrik Brundin explained the scientific basis of this new therapeutic strategy and helped the Huntington’s disease community understand complex concepts, such as the role of SNPs and their importance in the development of precision therapies for the disease. The speakers pointed out that the specific SNP targeted by this treatment is present in approximately 40% of the population, making the genetic identification of candidates through epidemiological screening a key aspect of the study.
