Explore the Science
Behind our Novel Therapeutics
Asha's Priority Disease Areas
Explore the Biology of Key Disease Indications Below
Alzheimer's Disease, Parkinson's Disease, and ALS
Neurodegenerative diseases represent one of the most poorly defined therapeutic areas with respect to the availability of disease modifying medicines... our goal is to change that. At Asha we are exploring disease-associated pathways to restore normal cellular function and mitigate pathology.
Alzheimer's Disease (AD)
A Comprehensive Approach to Solving AD Pathology
At Asha we are taking a multifaceted approach to therapeutic intervention in AD. This includes addressing key metabolic pathways and mitochondrial dysfunction as well as targeting leading genetic risk factors for AD including ApoE4.
A key feature of most neurodegenerative diseases including AD is the hyper-fragmentation of mitochondria. This promotes neuroinflammation, neuronal dysfunction, and ultimately neurodegeneration. We have developed a first-in-class highly specific inhibitor of mitochondrial fragmentation that restores normal cellular function, ASHA-091.
Parkinson's Disease (PD)
Targeting Dysfunctional Mitochondria
Mitochondrial hyper-fragmentation and dysfunction is the key pathological driver of PD. By restoring normal mitochondrial dynamics and function, curative outcomes are now a probable reality.
As a multi-indication therapeutics, ASHA-091 has demonstrated robust efficacy in preclinical models of PD by restoring normal mitochondrial homeostasis, limiting neurodegeneration, and bolstering dopamine levels.
Amyotrophic Lateral Sclerosis (ALS)
Preventing Axonal Degeneration
ALS pathology is driven by the dysfunction and ultimate loss of motor neurons. Axonal degeneration is a key driving mechanism of ALS and other neuropathies and motor function disorders.
SARM1 is the primary regulator of
axon degeneration and is activated in ALS.
Using our novel intra-molecular glue technology and the PRISM™ platform, we have developed a robust SARM1 inhibitor, ASHA-624 that ameliorates ALS pathology in preclinical models in not only an effective but more importantly safe manner.
Infectious Disease & Post-Infection Illnesses
ME/CFS, Post-viral Illness, and Pathogen Resistance
Millions of people live with chronic post-infection illnesses with currently no therapeutic interventions. Furthermore, anti-microbial and anti-bacterial resistant pathogens are ever increasing with only marginal future therapies on the horizon. These two aspects of infectious disease are at the heart of Asha's efforts to enable health security and provide hope to millions dealing with chronic post-infection illness.
Post-viral Illness (Long COVID & ME/CFS)
Mitochondrial Fragmentation is the Commonality
Post-viral illnesses are a set of chronic pathologies with multiple symptomatic profiles including ME/CFS and Long COVID. To date no cures, let alone effective therapies exist.
As observed in the neurodegenerative diseases described above, mitochondrial hyper-fragmentation has been identified as a driving component to post-viral illness.
Similar to approaches for AD and PD, ASHA-091 intervention in preclinical models of post-viral illness is proving efficacious. Interestingly, ASHA-091 was designed specifically as the first targeted therapy for ME/CFS and post-viral illness and has since proven to have a host of other therapeutic indications including AD and PD as highlighted above.
Cancer & Immuno-oncology
Targeted Therapies for Key Oncology Pathways
Over the past two decades significant advances in cancer treatments have been made, still cancer remains a primary life-threatening disease. We are leveraging our PRISM™ Design Platform to develop medicines to some of the most difficult to drug pathways that are driving cancer including Beta-Catenin and RAS.
Colorectal Cancer (CRC)
Restoring Normal Beta-Catenin Signaling
Wnt/Beta-Catenin signaling drives over 70% of CRC tumors and contributes to oncogenesis in a variety of other cancer types. Targeting Beta-Catenin has long been held as the holy grail of CRC treatment, but has proven challenging.
ASHA-044, the Beta-Catenin Solution
To safely target Beta-Catenin, we looked to normal biology. Beta-Catenin signaling is controled by the turnover of the protein through a "destruction complex". This complex is dysfunctional in CRC. Therefore, we developed a novel protein degrader in ASHA-044 to create a "synthetic destruction complex" and restore normal Beta-Catenin homeostasis, providing both efficacy and safety.
In preclinical studies ASHA-044 mitigated tumor induced mortality and promoted the immunological conversion of the tumors from immune excluded "cold" tumors to T-cell infiltrated "hot" tumors.