Amylyx
Drug Repurposing for ALS
In 2013, two Brown undergrads hypothesized that a combination of drugs that supported mitochondrial function while reducing endoplasmic reticulum stress might slow the motor neuron death that causes the fatal progression of ALS.
Amylyx has recently completed a pivotal phase 2 trial in ALS that showed a clinically significant slowing of disease progression and are testing the drug combination in other neurodegenerative conditions.
ApicBio
Dual-Acting Gene Therapy for ALS
Many patients with inherited ALS have mutations in the SOD1 gene. Apic Bio has developed an AAV-based gene therapy vector that not only replaces the errant SOD1 gene, but also inhibits the basal levels of mutant SOD1 protein. This dual effect therapy has been administered to patients on a compassionate use basis and is showing signs of efficacy.
Apic Bio is also working on gene therapy treatments for other disorders, including α-1 antitrypsin deficiency.
Cognito
Non-Invasive Alzheimer’s Therapy
Cognito leverages insights from optogenetics work done by Li-Hui Tsai and Ed Boyden at MIT. When exposed to gamma frequencies, microglial cells in the brain activate and clear amyloid plaques and tau tangles, two hallmarks of Alzheimer’s.
Cognito has developed a device that administers visual and audio stimulation in this frequency range that is being tested in clinical trials for the symptoms of Alzheimer’s disease.
Kuzani Pharmaceuticals
A Drug Treatment for Autism
Based on the hypothesis of the cell danger response underlying the autistic brain, an approved drug which is a purinergic receptor antagonist was used to treat autistic children in an investigator-initiated study. The children exhibited dramatic improvement in their emotional engagement after only a single dose of the drug.
Kuzani was formed to further develop this treatment and is preparing to initiate clinical studies in the new year.
Neuroelectrics
Transcranial Electric Stimulation for Epilepsy and Depression
Neuroelectrics has developed a proprietary algorithm that uses both EEG data and MRI scans to map individualized brain circuits and deliver customized transcranial electric stimulation therapy to affected regions. The technology is in clinical trials for treatment-resistant epilepsy, of which there are 500,000 patients in the US alone.
The Neuroelectrics headset and software platform is also being tested in trials for depression, ADHD, and Alzheimer’s Disease.
Pinteon Therapeutics
Ultra-Specific Anti-Tau Antibodies for Tauopathies
Diseases such as traumatic brain injury and progressive supranuclear palsy share the characteristic of elevated levels of misfolded tau protein in the brain. Pinteon has developed an antibody that is ultra-specific for the pathological form of phospho-tau and has commenced phase 1 studies in humans. By removing this pathological species, Pinteon aims to create a disease modifying therapy in multiple tauopathies.
Prilenia Therapeutics
Treating Huntington’s and ALS
Prilenia is a late-stage clinical company that is developing pridopidine, an extremely selective S1R agonist, as a potential treatment for Huntington’s Disease and amyotrophic lateral sclerosis (ALS), two diseases which currently do not have a cure.
Pridopidine positively influences S1R regulated pathways across neurodegenerative and neurodevelopmental indications, including protection against axonal and neuronal injury, restoring spine impairments, enhancing BDNF secretion and restoring mitochondrial function.