Pipeline / BPC-509

rare disease · gene therapy

BPC-509 — Phenylketonuria (PAH deficiency)

A liver-directed AAV gene therapy designed to restore phenylalanine hydroxylase expression in patients with classical phenylketonuria. Discovery-stage research is ongoing.

discovery Target: Phenylalanine hydroxylase (PAH) Sample data

Investigational candidate. BPC-509 has not been approved by any regulatory authority.

Mechanism of action

How the molecule works.

BPC-509 delivers a functional PAH transgene to hepatocytes via a liver-tropic AAV capsid, restoring conversion of phenylalanine to tyrosine.

Milestones

Program timeline.

  1. 2024 Capsid screen done
  2. 2025 Expression cassette optimization current
  3. 2026 Lead capsid selected planned
  4. 2027 GLP toxicology planned

Publications

2 related publications.

  • Cell Chemical Biology 2022

    A structure-guided platform for the discovery of selective kinase inhibitors

    Raman A, Fischer L, Becker T, et al.

    An overview of the structure-guided discovery platform underlying the BPC-101 and BPC-305 programs.

    DOI: 10.1016/j.chembiol.[sample]
  • PNAS 2022

    Antibody and antibody-drug conjugate discovery using engineered Fc libraries

    Hoffmann P, Shah N, Watanabe K, et al.

    Description of the Fc engineering library used to discover BPC-203 and to assemble the BPC-402 linker-payload.

    DOI: 10.1073/pnas.[sample]
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