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Rarebase, a utility biotechnology company, 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 screenings, all performed 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. Potential hits are then tested in patient cell models for their therapeutic effect. The results of these validation approaches are then used to inform and improve Function’s predictions.

The goal is to have a simplified approach to identifying effective therapies that can be tested further in patients.

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“We see the potential to apply our technology at scale to drug and target discovery for thousands of diseases that currently have no cure,” Rarebase co-founder and CEO Onno Faber said in a statement. Press release.

A total of 15 rare disease patient organizations have partnered with Rarebase in the Function platform, including the Charlotte and Gwenyth Gray Foundation, which advocates and funds to find a cure for Batten disease.

But Rarebase works with more than 100 patient organizations.

“We are delighted to actively collaborate with rare disease patient organizations. In our first year, we’ve built relationships with over 100 patient organizations looking to find therapies for their loved ones,” Faber said.

Because rare neurological diseases may share common but unknown genes and pathways, joint efforts can identify therapies that have broader therapeutic effects on the different diseases.

“There are many common elements between neurological diseases. Instead of each of us working in silos, partnership on specific projects is essential,” said Nasha Fitter, co-founder and CEO of the FOXG1 Research Foundation. “With Rarebase, we can screen more drugs, we can share costs, and we have more opportunities to learn from each other.”

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, essential for brain development.

“We are thrilled to partner with Rarebase and other patient organizations to accelerate drug discovery across multiple conditions, and to do so through a new model of collaboration,” said Charlene Son Rigby, Co-Founder and President. of the STXBP1 Foundation, another Function partner. . STXBP1 encephalopathy is a rare neurodevelopmental disease and genetic epilepsy.

Other patient organizations involved include ADNP Kids Research Foundation, SynGAP Research Fund, Hereditary Neuropathy Foundation, Hope4Harper, Lightning and Love Foundation, Cure ATRX, CureMito Foundation, SHANK2 Foundation and several funds associated with 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 Neuroopathy Foundation. even for our rare disease communities; there is undeniable strength in numbers and exponential power to support each other towards our common goal.

Rarebase has an innovative financial system where patient organizations can directly fund and support research that can directly impact the lives of patients.

“Rarebase pushes the boundaries of science and innovates with a funding structure that enables patient organizations to directly support drug discovery,” said Mike Graglia, Founder and CEO of SynGAP Research Fund.