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EXG 001 307 - Exegenesis Bio

Drug Profile

EXG 001 307 - Exegenesis Bio

Alternative Names: EXG001-307 - Exegenesis Bio; Spinal muscular atrophy gene therapy - Exegenesis Bio

Latest Information Update: 02 May 2024

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At a glance

  • Originator Exegenesis Bio
  • Class Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference; Survival of motor neuron 1 protein replacements
  • Orphan Drug Status

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

  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Phase I Spinal muscular atrophy

Most Recent Events

  • 25 Apr 2024 Efficacy and adverse events data from a phase I trial in Spinal muscular atrophy released by Exegenesis Bio
  • 30 Jan 2023 Exegenesis Bio plan to submit two IND applications for Spinal muscular atrophy worldwide in 1H of 2022
  • 23 Jan 2023 Spinal muscular atrophy gene therapy - Exegenesis Bio is available for licensing as of 23 Jan 2023. (https://exegenesisbio.com/partnering-business-development/#)
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