BridgeBio Enters into Rare Disease Collaborations with Johns Hopkins, University of Florida

Rare Daily Staff

BridgeBio Pharma entered into two separate collaborations with universities to support the translation of academic innovations in genetically driven diseases into commercial products for patients in need.

BridgeBio is focused on building relationships with academic partners to smooth and speed up the transition from the lab to the patient. The new collaborations are with Johns Hopkins University and the University of Florida.

Under its collaboration with Johns Hopkins, BridgeBio will focus on opportunities to support the early-discovery research around genetically validated targets underway at the university. Where possible, BridgeBio will invest in programs to accelerate promising genetic-disease therapies to the clinic, from gene therapy to small molecule. It will also lend its expertise to science designed to help develop a promising molecule or approach into a viable preclinical program, including medicinal chemistry for small-molecule hit optimization, strategies to modify or formulate a potential biologic therapy or approaches for testing non-optimized viral vectors. Additionally, BridgeBio may conduct proof-of-concept studies for lead therapeutic compounds in relevant mammalian models.

“Our hope is that this relationship will allow us to build on the significant advances being made in Johns Hopkins’ labs and support work to translate them as quickly and as safely as possible into meaningful medicines for patients in need,” said BridgeBio CEO and founder Neil Kumar.

The partnership with the University of Florida also combines academic prowess in studying genetically driven disease, including its capabilities in gene therapy, with BridgeBio’s expertise in efficiently advancing therapeutics from the academic laboratory through preclinical studies and into human testing.

BridgeBio will provide sponsorship to select University of Florida research programs around diseases with a genetic basis, including gene therapies and large and small molecules. The company will provide guidance for sponsored programs around medicinal chemistry for small-molecule hit optimization, strategies to modify or formulate a potential biologic therapy or approaches for testing non-optimized viral vectors. BridgeBio may conduct proof-of-concept studies for lead therapeutic compounds in relevant mammalian models.

“The scientists at University of Florida are recognized as leaders in research dedicated to genetically driven diseases, especially in the area of gene therapy,” said Kumar.

Photo: Neil Kumar, BridgeBio CEO and founder