Families of Spinal Muscular Atrophy Awards awarded 150.000 USD to Dr. Lee Rubin at Harvard to advance a new spinal muscular atrophy (SMA) drug discovery program. The objective of this program is to To identify novel SMA therapeutics by using patient-derived motor neurons for drug discovery and preclinical testing.
By using human motor neurons to screen for new SMA drugs, the researchers hope to identify compounds that can increase functional SMN levels in the cell type most affected by the disease. They will prioritize compounds that are already approved for use so they can move through the development pipeline more quickly.
The researchers will test the candidate compounds across a panel of SMA patient-derived cells so that they can identify drugs that are effective across many genetic backgrounds and disease severities. The researchers hope that this innovative approach will lead to the discovery of drugs that are mechanistically unique so that they may be used in addition to the therapies that are currently being developed. These cell lines can also help assess existing SMA drug candidates.
The SMA drug pipeline now has 13 SMA drug programs in development, including 3 in clinical trials. This pipeline has expanded from just 4 programs 5 years ago. Families of SMA has funded half of all the ongoing drug programs for SMA.
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