BridgeBio Pharma Gene Therapy Subsidiaries Present Data Demonstrating Potential in Two Rare Disease Indications at the European Society of Gene and Cell Therapy Conference
Experimental 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.
Overall, 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.
"Durable expression of a vector in adrenal tissue has presented challenges for the development of gene therapies for adrenal indications," said
Canavan Disease Experimental Therapy Amenable to IV Delivery
In a poster entitled "A Route of Administration Study of BBP-812, an AAV9-based Gene Therapy for the Treatment of Canavan Disease, in Juvenile Cynomolgus Macaques," scientists from Aspa Therapeutics examined the uptake of and resulting DNA and mRNA expression for BBP-812, an experimental AAV9 vector, in three different administration routes: intravenous (IV), intrathecal (IT) or intracerebroventricular (ICV). Biodistribution of vector genomes and of transgene mRNA was evaluated throughout the central nervous system (CNS) at both 3 and 8 weeks post-administration. No dose-related adverse events were observed at any tested doses at either time period.
Using an IV route of administration, vector copy number and mRNA expression were not only detected broadly throughout the brain and spinal cord, but were generally detected at levels higher than when administered ICV or IT.
"Developing therapies for diseases like Canavan presents particular delivery challenges – namely getting the therapy into the central nervous system," said
Aspa Therapeutics is currently enrolling a natural history study of Canavan disease called CANinform. Interested individuals can find information about the study at TreatCanavan.com.
About Adrenas Therapeutics
Adrenas Therapeutics is a
About Aspa Therapeutics
Aspa Therapeutics is a
About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio's pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit www.bridgebio.com.
BridgeBio Pharma Forward-Looking Statements
This press release contains forward-looking statements. Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to the clinical and therapeutic benefits of BBP-631 and BBP-812, the potential of BBP-631 to be a meaningful therapy for patients with congenital adrenal hyperplasia with 21-hydroxylase deficiency and the potential for BBP-812 to overcome the challenges associated with other gene therapies' inability to target the CNS, Adrenas Therapeutics' and Aspa Therapeutics' clinical development plans, including their plans to file their respective INDs in 2020, and timing and completion of preclinical studies and regulatory submissions, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, Adrenas Therapeutics' and Aspa Therapeutics' ability to continue its planned development and regulatory submissions for BBP-631 and BBP-812, respectively, and the timing and success of any such continued clinical development and planned regulatory submissions, as well as those set forth in the Risk Factors section of
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Carolyn Hawley, Canale Communications, firstname.lastname@example.org, 619-849-5382