Research programme: amyotrophic lateral sclerosis gene therapies - Beacon Therapeutics
Latest Information Update: 29 Jun 2023
At a glance
- Originator Applied Genetic Technologies Corporation
- Developer Beacon Therapeutics
- Class Gene therapies
- Mechanism of Action C9orf72 protein modulators; Gene transference
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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
- Preclinical Amyotrophic lateral sclerosis
Most Recent Events
- 13 Jul 2022 Amyotrophic lateral sclerosis gene therapies is available for licensing as of 13 Jul 2022. https://agtc.com/programs/
- 07 Jul 2022 Preclinical trials in Amyotrophic lateral sclerosis in USA (Parenteral) (Applied Genetic Technologies Corporation pipeline, July 2022)