The biotechnology company Wave Life Sciences strengthens their efforts in Huntington’s disease. In February they announced their collaboration with Takeda, a Japanese pharmaceutical company with long and solid traditions.
The partnership enables Wave to continue their work in an even larger scale. The Japanese pharma company is listed with around 7000 employees and a paid in capital of over 65 billion USD. With this new partner, the global HD research community has got another strong player.
The collaboration also implies a global strategy in the development and commercialization of possible treatment. This is good news for Huntington families all over the world and our worldwide community.
Nucleic acid therapies
For quite some time, Wave has developed a potential medicine for HD and other neurological and genetic diseases. They call their method stereopure nucleic acid therapies.
What’s really unique and exciting is that the therapy only target the mutant Huntingtin (mHTT) – the protein that causes Huntington’s disease. The function of the healthy Huntingtin, the so called wild type Huntingtin (wHTT), is potentially not altered in any way.
If the therapy works as anticipated, it will reduce the presence of the sick Huntingtin (mHTT) and leave the healthy one (wHTT) relatively untouched and able to do it’s job.
Wave’s steropure nucleic acid therapies can be thought of as “precision medicine”. That’s why their ongoing trials are called Precision HD 1 and Precision HD 2.
The “precision medicine” is studied through two Phase 1b/2a trials. This is mainly to test the safety of the drug in a smaller number of patients – but the potential effects on symptom progression will also be measured.
The first patient got the injection in the spine (just like IONIS’ Huntingtin Lowering trial) in July 2017.
The trials are not yet fully recruited. In the end around 120 participants with early stage HD will participate. Participants are currently recruited in Canada and Poland. Scotland will follow soon.
Another very interesting strategy from Wave is that they have opened a big (90,000 Square feet) manufacturing venue in Massachusetts, US.
This gives them the ability to control the quality and precision of the therapy not only in the developing and testing but also in the manufacturing process.
You can read more about Wave’s methods here.