RARE Daily

BridgeBio Enters Multi-Year Partnership with Resilience to Advance its Gene Therapy Treatments

October 3, 2023

Rare Daily Staff

BridgeBio Pharma and National Resilience entered into a strategic collaboration to manufacture and advance two of BridgeBio’s experimental gene therapies for rare diseases: BBP-812, an adeno-associated virus 9 gene therapy for Canavan disease, and BBP-631, an investigational AAV 5 gene therapy for congenital adrenal hyperplasia, or CAH.

The two companies have developed a novel manufacturing and aligned incentive business model to drive these gene therapies forward with an emphasis on sustainability and capital-efficiency. Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites. As part of an innovative cost and risk-sharing framework, Resilience will provide in-kind manufacturing services and will receive future development and approval milestones and low-to-mid single digit royalties on BBP-631 and BBP-812. Resilience will support the ongoing clinical development manufacturing needs and will serve as the primary commercial manufacturer for both programs if successful.

“Our partnership with BridgeBio seeks to accelerate development of innovative therapeutic options for patients in need,” said Rahul Singhvi, CEO of Resilience.

Beyond BBP-812 and BBP-631, Resilience will also be the primary manufacturer for future clinical projects across BridgeBio’s gene therapy portfolio. The agreement will reduce manufacturing uncertainty for these programs and is expected to help BridgeBio expedite development of gene therapies going forward.

“Manufacturing is the most critical and costly aspect of developing gene therapy for patients with a serious unmet need. We conduct process development, analytical development and optimization in our own labs, and with this partnership we can now hand these programs off to one of the most trusted partners in the industry for scale up and commercial manufacturing. This allows us to accelerate the development of our gene therapy portfolio in a capital-efficient and sustainable way with the hope of providing medicines more quickly,” said Eric David, CEO of BridgeBio Gene Therapy.

BBP-812 is an investigational AAV9 gene therapy for Canavan disease. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, potentially correcting the disease at its source. Preclinical proof-of-concept results have shown the approach restores survival and normal motor function in Canavan disease models. BBP-812 was granted Fast Track, Rare Pediatric Drug, and Orphan Drug designations by the U.S. Food and Drug Administration. BBP-812 was also granted Orphan Drug designation by the European Medicines Agency.

BBP-631 is an AAV5 gene therapy developed to treat CAH due to 21-hydroxylase deficiency at its source. BBP-631 is designed to deliver a functional copy of the 21-hydroxylase gene and has been shown through multiple preclinical studies to result in efficient and persistent delivery to the adrenal gland, where hormones are naturally made. If successful, BBP-631 may restore the body’s hormone and steroid balance by enabling people with CAH to naturally make their own cortisol and aldosterone. It could also allow for people with CAH to eliminate or significantly reduce their daily glucocorticoid or mineralocorticoid doses, which is the current standard of care for patients.

Photo: Rahul Singhvi, CEO of Resilience

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