Rarebase, a public benefit biotechnology company, has launched the Function therapeutic discovery platform specifically for rare neurological diseases, including Batten disease.
Led by scientists, engineers, patients, and advocates, Function combines large-scale therapeutic screening with genetic screens, all conducted in nerve cells (neurons) grown in a dish.
The combination of these strategies will inform the therapeutic potential of thousands of small molecules on the activation pattern of genes linked to rare neurological diseases. The potential hits are then tested in patient cell models for their therapeutic effect. The results from these validation approaches are then used to inform and improve Function’s predictions.
The goal is to have a streamlined approach to identify likely effective therapies to be further tested in patients.
“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,” Onno Faber, co-founder and CEO of Rarebase, said in a press release.
In total, 15 rare disease patient organizations have partnered with Rarebase in the Function platform, including the Charlotte and Gwenyth Gray Foundation, which advocates and raises funds to find a cure for Batten disease.
But Rarebase is collaborating with over 100 patient organizations.
“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,” Faber said.
Since rare neurological diseases may share common yet unknown genes and paths, the joint efforts may identify therapies that have broader therapeutic effects across the different diseases.
“There are many common elements shared between neurological diseases. Instead of each of us working in silos, partnering on specific projects is critical,” said Nasha Fitter, the co-founder and CEO of the FOXG1 Research Foundation. “With Rarebase, we can screen more drugs, we can share costs, and we have more opportunity to learn from one another.”
FOXG1 syndrome is a rare, pediatric, neurological disorder that impairs brain development and causes severe physical and cognitive disabilities. It is caused by a mutation in the FOXG1 gene, which is critical in brain development.
“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, another partner in Function. STXBP1 encephalopathy is a rare neurodevelopmental condition and genetic epilepsy.
Other involved patient organizations include the ADNP Kids Research Foundation, SynGAP Research Fund, Hereditary Neuropathy Foundation, Hope4Harper, Lightning and Love Foundation, Cure ATRX, CureMito Foundation, SHANK2 Foundation, and multiple funds associated with the Rare Village and Rare Crossroads networks.
“A single strand of spider silk can be easily broken. However, add thousands together and they become stronger than steel,” said Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation. “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.”
Rarebase has an innovative financial scheme in which patient organizations can directly fund and support research that can make a direct impact on patients’ lives.
“Rarebase is pushing the limits of science and innovating a financial structure that allows patient organizations to directly support drug discovery,” said Mike Graglia, founder and managing director of SynGAP Research Fund.