Our member @rosebudbiosci is rethinking how rare disease drugs are discovered by starting where biology is most predictive, in human systems. Learn how their approach to disease modeling is helping generate more relevant data earlier in development by reading the full Member Spotlight: https://t.co/RmKbvKHKRk
#RareDiseaseAwareness
@sulemanali Appreciate the shoutout @sulemanali. We are incredibly excited about the future for new therapies and targets discovered with our platform!
@yumatanaka@sulemanali Thanks for asking, Yuma. Funding is the biggest challenge for us and many other startups in biotech. The tech itself works beautifully.
I've been blown away by the progress of medicine over the past decade
And one startup doing gene therapy really got my attention
You may be asking "What's gene therapy?"
Gene therapy uses genes to treat diseases
If you've got a genetic disease like Parkinson's, cancer, heart disease, etc., gene therapy can help in correcting it - straight Sci-fi shit
However gene therapy is a fairly recent field, but also fast-growing:
- until 2017 the FDA had approved 2 gene therapies
- in 2022 alone 3 therapies were approved
But that doesn't seem like that many. Why haven't we gotten 100's of gene therapies yet?
Because it can take years or even decades to find cures for certain diseases using traditional methods. And 90% of clinical trials fail, leading to wasted time and resources.
A company called Rosebud Biosciences is seeking to change that
@rosebudbiosci has developed an innovative approach for screening drugs and their effectiveness for children with rare diseases
Children account for 50% of all people with genetic diseases, and only 5% of genetic diseases have approved gene therapies.
Rosebud is seeking to find solutions for the other 95%
And how they do this is mind-blowing
In their lab they use stem cells to grow 1000s of "micro-organs" (mini organs) that have the target gene mutations they want to treat
This allows for testing a large number of therapies across these organs and seeing which ones work, saving a ton of time and money, while also leading to better probability of getting through clinical trials
More gene therapies getting through clinical trials quickly = more diseases that can be treated
Gene therapy as a whole seems to be having a breakthrough moment and I'm excited to see how this space continues to grow!
A video related to the work shows emergence of thousands of cardiac and vascular cells which form cardiac vascularized organoid (cVOs), where cardiomyocytes are green, endothelial cells are orange, and smooth muscle cells are blue.
Organoids, like real organs, need vascularization (veins/arteries) as they develop and grow. Think nutrients, signaling, oxygen, drug delivery. Honored to take part in the Abilez lab's quest for specific factors that do just this in human heart and liver organoids.