Drug Profile

BBP 821

Alternative Names: BBP-821

Latest Information Update: 06 Sep 2023

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At a glance

Originator BridgeBio Pharma
Class Gene therapies
Mechanism of Action Tuberous sclerosis complex 1 protein replacements; Tuberous sclerosis complex 2 protein replacements

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

27 Jun 2023 Preclinical trials in Tuberous sclerosis in USA (Parenteral) (BridgeBio Pharma Pipeline, June 2023)
27 Jun 2023 BridgeBio Pharma announces intention to submit IND application for Tuberous sclerosis in 2025 (BridgeBio Pharma Pipeline, June 2023)

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