Rarebase launches a neuroscience drug discovery platform collaborating with STXBP1 Foundation and 14 other rare disease patient organizations

The STXBP1 Foundation is excited to share news that we are partnering with Rarebase and 14 other rare disease patient organizations on the launch of the Function platform, a neuroscience drug discovery platform. The Function platform is being developed to support high-throughput assays to accelerate identification of potential therapies.

"There is an urgent, unmet need for effective therapies for STXBP1 and other neurological diseases. We are excited to partner with Rarebase and other patient organizations to accelerate drug discovery across multiple conditions, and to do so through a novel collaborative model."

-Charlene Son Rigby, Co-Founder and President of the STXBP1 Foundation

Read on for the press release:

Rarebase launches a neuroscience drug discovery platform collaborating with 15 rare disease patient organizations.

The Function platform enables drug and target discovery for rare genetic diseases. 

September 14, 2021

Rarebase, a public benefit biotechnology company, launched a tech-enabled drug discovery platform called Function. Function’s 15 patient organization collaborators represent genes that cause epilepsy, autism, developmental delay and neurodegeneration. Learn more about the capabilities of Function here.

Function platform collaborators include: ADNP Kids Research Foundation, SynGAP Research Fund, FOXG1 Research Foundation, STXBP1 Research Foundation, Hereditary Neuropathy Foundation, Hope4Harper, Lightning and Love Foundation, Cure ATRX, Charlotte and Gwenyth Gray Foundation, the SHANK2 Foundation and multiple funds associated with the Rare Village and Rare Crossroads networks. 

“We’re thrilled to be actively collaborating with rare disease patient organizations. In our first year, we have built relationships with more than 100 patient organizations who are seeking to find therapies for their loved ones. We see the potential of applying our technology at scale to the discovery of drugs and targets for thousands of diseases that currently have no treatments.” said Onno Faber, Co-Founder and CEO of Rarebase.

“Rarebase is pushing the limits of science and innovating a financial structure that allows patient organizations to directly support drug discovery,” said Mike Graglia, the Founder and Managing Director of the SynGAP Research Fund. 

“There are many common elements shared between neurological diseases. Instead of each of us working in silos, partnering on specific projects is critical. With Rarebase, we can screen more drugs, we can share costs, and we have more opportunity to learn from one another,” said Nasha Fitter, the Co-Founder and CEO of the FOXG1 Research Foundation. 

"There is an urgent, unmet need for effective therapies for STXBP1 and other neurological diseases. We are excited to partner with Rarebase and other patient organizations to accelerate drug discovery across multiple conditions, and to do so through a novel collaborative model," said Charlene Son Rigby, Co-Founder and President of the STXBP1 Foundation.

“Rarebase is stepping outside the traditional drug development pipeline and harnessing new technology and science that patient organizations like ours would never be able to utilize alone,” said Sandra Sermone, the Founder and CEO of the ADNP Kids Research Foundation. 

“We want our children to be independent in life and, honestly, just be able to live another day. Function is finally doing justice to how we need to approach finding answers for rare diseases,” said Penny Howard, the Founder and CEO of Hope4Harper. 

“A single strand of spider silk can be easily broken. However, add thousands together and they become stronger than steel. The same goes for our rare disease communities; there is undeniable strength in numbers and exponential power in supporting each other towards our common goal,” said Allison Moore, the Founder and CEO of the Hereditary Neuropathy Foundation.

Rare diseases represent a major unmet medical need as millions of people globally suffer without treatment options. The number of identified rare diseases is rapidly growing due to advances in genetic testing. Nearly 10,000 rare diseases affect an estimated 400 million people worldwide, but only 5% of them have an FDA approved treatment. 

About Rarebase, PBC

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

Press release on Cision PR Newswire: https://www.prnewswire.com/news-releases/rarebase-launches-a-neuroscience-drug-discovery-platform-collaborating-with-15-rare-disease-patient-organizations-301376383.html

Learn more at the Rarebase website: https://www.rarebase.org/