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BridgeBio Pharma Gene Therapy Subsidiaries Present Data Demonstrating Potential in Two Rare Disease Indications at the European Society of Gene and Cell Therapy Conference
SAN FRANCISCO, Oct. 22, 2019 /PRNewswire/ -- BridgeBio Pharma, Inc. (NASDAQ: BBIO) today announced that promising preclinical data from two of its gene
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"SAN FRANCISCO, Oct. 22, 2019 /PRNewswire/ -- BridgeBio Pharma, Inc. (NASDAQ: BBIO) today announced that promising preclinical data from two of its gene therapy-focused subsidiaries – Adrenas Therapeutics and Aspa Therapeutics – will be presented at the European Society of Gene and Cell Therapy (ESGCT) Conference, occurring in Barcelona from October 22 to 25. In addition, the company announced that Aspa Therapeutics is currently conducting a natural history study in children with Canavan disease, CANinform.\n\n \nExperimental Gene Therapy for Congenital Adrenal Hyperplasia Shows Durable Transgene Expression Researchers from BridgeBio subsidiary Adrenas Therapeutics presented new preclinical results from gene therapy candidate BBP-631 in a poster entitled \"Durable CYP21A2 Gene Therapy in Non-Human Primates for Treatment of Congenital Adrenal Hyperplasia.\" Throughout the study, a total of 20 non-human primates (NHPs) were treated with BBP-631 at one of three intravenous (IV) doses. Vector copy number and transgene mRNA expression in the adrenal glands were analyzed at 4 and 12 weeks post-dosing in the low- and medium-dose arms and at 12 and 24 weeks post-dosing in the high-dose arm. No dose-related adverse events were observed at any of the doses tested at any time point.\nOverall, treatment with BBP-631 resulted in high vector copy number (VCN) and mRNA expression in the adrenal gland, suggesting strong tropism and uptake of BBP-631 for the adrenal gland. In the high-dose arm, VCNs were maintained between 12 and 24 weeks. Furthermore, mRNA levels increased between 4 and 12 weeks for the medium dose arm and were consistent between 12 and 24 weeks for the high dose arm. Researchers also saw dose-dependent increases in both VCNs and mRNA levels across the three doses tested.\n\"Durable expression of a vector in adrenal tissue has presented challenges for the development of gene therapies for adrenal indications,\" said Clayton Beard, Ph.D., Senior Vice President, Research and Development at BridgeBio Gene Therapy. \"The observed maintenance of the BBP-631 AAV5 vector for as long as 24 weeks in NHPs represents an important step in validating a gene therapy approach to treat 21-hydroxylase deficiency, and we anticipate filing our IND for this indication in 2020.\"\nCanavan Disease Experimental Therapy Amenable to IV DeliveryIn a post...