Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
An investigational allogeneic cell therapy consisting of side population adipose-derived mesenchymal stem cells (SPADMSCs) obtained from volunteer donors. Developed by the Tehran University of Medical Sciences, this therapy is being evaluated for the treatment of spinal muscular atrophy type 1 (SMA type 1), also known as Werdnig-Hoffman disease. SMA type 1 is a severe autosomal recessive neurodegenerative disorder caused by mutations in the SMN1 gene, leading to the loss of alpha motor neurons in the spinal cord. The therapy is administered via intrathecal injection, where the stem cells are hypothesized to provide neurotrophic support and paracrine signaling to protect degenerating motor neurons, potentially slowing disease progression and improving motor function in affected infants.
Beyond the preview
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Follow clinical development from study design and recruitment through results.
Explore development by indication, patient population, and geography.
Trace asset ownership, licensing agreements, and commercial partnerships.
Explore the patent landscape and regulatory exclusivity around an asset.
Compare development programs by target, modality, and indication.
Connect source evidence and development news to your research questions.
See how Gosset can support your research on allogeneic side population adipose-derived mesenchymal stem cells.