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Drug Discovery

Drug Discovery with Rarebase

The TBCD non-profit LandOn A Cure Foundation has sponsored phase 1 of a drug discovery with the biotech company Rarebase

What is Rarebase?

Rarebase is a public benefit biotech led by scientists, engineers, patients and advocates. They leverage innovative technologies to build platforms that help them discover and develop treatments for the millions of people worldwide affected by the long tail of rare genetic disorders.

The Function Platform

The Function platform combines two unique data sets: large scale drug screens and genetic screens performed in neurons. The drug screen tests the effects of thousands of small molecules  on gene expression. The genetic screen measures changes in expression when disease causing genes are suppressed.

The combined data sets will be used to match potentially effective drugs to TBCD.

How Can I Help?

Rarebase has Landon's samples, which is an amazing start - but more participants would get us the best data possible. There are a few ways you can participate.

RNA Study

RNA Study

Rarebase will do an RNA study on your blood sample for free! This will strengthen their data around TBCD Disorder. If you've already done an RNA study via GeneDx or another genetics program, you can send them the data you have.

Send Your Sample

Send Your Sample

While a Phase 1 is already being conducted with Landon's sample, TBCD Disorder has a broad spectrum, largely based on where the mutations lie on the gene. A larger sample size will make it more likely for them to find treatments that are safe and effective for more people with TBCD.

Finance Research

Finance Research

Phase 1 was $50k. Phase 2 will be even more costly - $75k. Consider helping defray costs for LandOn A Cure by making a donation to their foundation. No amount is too small to make a difference.