Pipeline – VISUMPharma

Alzheimer’s Disease

Aberrant activation of microglia plays a central role in the pathogenesis of a broad spectrum of neurodegenerative diseases including Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), schizophrenia, and other conditions. In these diseases, overactive microglia engulf and eliminate synaptic connections leading to impairments in cognitive and motor function. Finding ways to pharmacologically mitigate this process is the subject of many drug discovery efforts. To date, there are no FDA approved therapeutics that addresses this key aspect of neurodegenerative diseases. Visum is taking an entirely novel approach to reduce both microglial activation and the scavenging of synapses.

Amyotrophic Lateral Sclerosis (ALS)

ALS is the leading cause of adult-onset motor neuron disease, characterized by rapid progression and, in the vast majority of cases, death within 2-5 years of diagnosis. There are no disease-modifying therapeutics available. Although targeted approaches in development for specific genetic causes of ALS hold significant promise, new insights and new strategies are needed to target downstream points of convergence in ALS pathogenesis that are common to most or all forms of the disease. Visum is exploring new cellular signaling abnormalities in ALS to develop broadly acting therapeutics for the disease.

Discovery Programs: Oncology

About 15% of all cancers – including a majority of the most prevalent types – and potentially most cancer stem cells re-express the protein LIN28, a pluripotency factor that endows cancers with stem cell-like qualities, enhances resistance to chemo and radiation therapies, and promotes metastasis. Many of these effects are due to the inhibition of microRNAs of the let-7 family which would otherwise inhibit LIN28 itself and have effects on cancer cells that oppose LIN28. Despite its wide recognition as a key target in oncology, there are no FDA approved or clinical stage therapies targeting this protein. Visum is developing a novel pharmacological strategy to downregulate LIN28, which has the potential to enhance the effectiveness of standard of care therapies and reduce cancer recurrence.