3 ms·

I have been a supporter of Cures Within Reach, a nonprofit that focuses on repurposing drugs, especially for rare diseases. https://www.cureswithinreach.org htt

by jawns 4mo ago

I have been a supporter of Cures Within Reach, a nonprofit that focuses on repurposing drugs, especially for rare diseases. https://www.cureswithinreach.org https://www.cureswithinreach.org They have funded some important repurposed-drug studies for Huntingtons Disease, which runs in my family. For a disease like this, it's never going to make sense for major pharmaceutical companies to invest the effort to develop entirely new drugs, but by repurposing existing drugs, it gives people living with rare diseases a chance to ease symptoms.

dmichulke 4mo ago
I'm on my cell phone and I couldn't figure out what diseases can be treated on the site. I'd say it's donor focused and not patient focused
- notRobot 4mo ago
  Not exactly what you asked for, but this page lists their impact: https://www.cureswithinreach.org/about-us-repurposing-research-leader/our-impact/ https://www.cureswithinreach.org/about-us-repurposing-resear...
unsupp0rted 4mo ago
Have you looked into UniQure / Clearpoint Neuro ? Exciting stuff, if it gets FDA approved.
petesergeant 4mo ago
> For a disease like [Huntingtons], it's never going to make sense for major pharmaceutical companies to invest the effort to develop entirely new drugs This is ... not correct. Roche, Regeneron, and Novartis all have novel HD drugs under development in tandem with smaller labs (Ionis, Alnylam, and PTC respectively), and then smaller labs like uniQure and Wave Life Sciences do too. Novartis have already dropped $1bn on the partnership with a committed $2b more. In addition, there are a bunch of incentive schemes for diseases like HD: both the FDA and EMA have offered orphan-drug designation to therapies for HD, the FDA does expedited programmes and can offer RMAT designation for drugs like AMT-130. With some luck (which is always in short supply for HD treatments, sadly), people with the disease might be able to get a single-injection treatment in the next 12 months[0]. 0: https://en.hdbuzz.net/the-other-shoe-drops-uniqure-shares-plans-to-submit-licensing-application-with-the-fda-for-amt-130/ https://en.hdbuzz.net/the-other-shoe-drops-uniqure-shares-pl...

by jawns 4mo ago

dmichulke 4mo ago

I'm on my cell phone and I couldn't figure out what diseases can be treated on the site. I'd say it's donor focused and not patient focused

notRobot 4mo ago
Not exactly what you asked for, but this page lists their impact: https://www.cureswithinreach.org/about-us-repurposing-research-leader/our-impact/ https://www.cureswithinreach.org/about-us-repurposing-resear...

unsupp0rted 4mo ago

Have you looked into UniQure / Clearpoint Neuro ? Exciting stuff, if it gets FDA approved.

petesergeant 4mo ago

> For a disease like [Huntingtons], it's never going to make sense for major pharmaceutical companies to invest the effort to develop entirely new drugs This is ... not correct. Roche, Regeneron, and Novartis all have novel HD drugs under development in tandem with smaller labs (Ionis, Alnylam, and PTC respectively), and then smaller labs like uniQure and Wave Life Sciences do too. Novartis have already dropped $1bn on the partnership with a committed $2b more. In addition, there are a bunch of incentive schemes for diseases like HD: both the FDA and EMA have offered orphan-drug designation to therapies for HD, the FDA does expedited programmes and can offer RMAT designation for drugs like AMT-130. With some luck (which is always in short supply for HD treatments, sadly), people with the disease might be able to get a single-injection treatment in the next 12 months[0]. 0: https://en.hdbuzz.net/the-other-shoe-drops-uniqure-shares-plans-to-submit-licensing-application-with-the-fda-for-amt-130/ https://en.hdbuzz.net/the-other-shoe-drops-uniqure-shares-pl...