Research programme: motor neuron disorders gene therapy - Sanofi
Alternative Names: AAV2/8-CBA-hSMN1; AAV4-IGF-1; AAV4-IGF-I; AAV4-VEGF; ALS gene therapy - Sanofi; SMA gene therapy - SanofiLatest Information Update: 31 Mar 2022
At a glance
- Originator Genzyme Corporation
- Developer Sanofi
- Class Gene therapies
- Mechanism of Action Gene transference; Insulin-like growth factor I stimulants; Survival of motor neuron 1 protein stimulants; Vascular endothelial growth factor A expression stimulants
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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
Highest Development Phases
- No development reported Amyotrophic lateral sclerosis; Spinal muscular atrophy
Most Recent Events
- 16 Jul 2016 No recent reports of development identified for preclinical development in Amyotrophic-lateral-sclerosis in USA (Parenteral, Injection)
- 16 Jul 2016 No recent reports of development identified for preclinical development in Spinal-muscular-atrophy in USA (Parenteral)
- 08 Apr 2011 Genzyme Corporation has been acquired by sanofi-aventis