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Applegate
Columbus, Ohio, United States
- Ownership
- Private
- Development stage
- Other
01
Overview
Applegate Therapeutics is a biotechnology company focused on the development of small molecule therapeutics for rare genetic disorders, with a primary emphasis on Spinal Muscular Atrophy (SMA). Founded by industry veterans including Brian Kaspar, the company leverages expertise in RNA splicing modulation to increase the production of functional Survival Motor Neuron (SMN) protein. The company's approach aims to provide oral alternatives or adjuncts to existing gene therapies and antisense oligonucleotides. Applegate Therapeutics operates primarily in the pre-clinical stage, targeting the underlying genetic causes of neurodegenerative conditions.
- Therapeutic areas
- Neurology
- Modalities
- Small molecule
- Industry
- Biotech
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