Unlocking EpigeneticS to Transform Disease Treatment
Despite major advances in biology and technology, small-molecule drug development remains slow, costly, and inefficient. We believe this is driven by outdated models and an incomplete understanding of disease. Traditional “one-drug, one-target” strategies, including receptor and kinase inhibitors, often fail because of toxicity, drug resistance, and limited long-term benefit.
As Einstein said, “We cannot solve our problems with the same level of thinking that created them.” Guided by this idea, Parkside Scientific Inc., based in New York City, is redefining drug discovery. Instead of targeting a single defect, we reprogram the epigenome and transcriptome to reset disease cells and treat disease at its source.
Rethinking Disease Through Gene Expression
Cell function is controlled by gene expression, which is regulated by the epigenome. While genetic mutations and abnormal signaling can trigger disease, widespread changes in gene expression across many cell types often drive disease progression.
This points to a new therapeutic strategy: restore healthy gene-expression programs to return cells to a healthy state.
Among the most important targets are bromodomain (BrD) proteins. These epigenetic histone readers recognize acetylation marks and regulate gene transcription. When dysregulated, they drive abnormal gene-expression programs in cancer, inflammation, and neurodegenerative diseases.
Parkside’s Breakthrough
The BET family of BrD proteins, especially BRD4, is a validated therapeutic target. However, first-generation BET inhibitors such as pelabresib[1](Novartis), were designed under the “one drug, one target” model. Their clinical benefit has been limited by dose-limiting toxicity and short-lived responses.
Parkside’s proprietary STAMP™ (Single Therapeutic Acts on Multiple Proteins) uses a different strategy. Rather than blocking one protein, STAMP™ molecules modulate transcriptional networks. They reprogram acetylation-driven gene-expression circuits, creating a wider therapeutic window, longer-lasting responses, and broader clinical benefit.
Our lead candidate, PS1132, has shown strong safety, favorable pharmacokinetics, and early efficacy in Phase 1 studies of hematologic cancers. Responses have been seen in patients with relapsed or refractory T- and B-cell lymphomas after chemotherapy, immunotherapy, and targeted therapies, including JAK1, PI3Kδ, and BTK inhibitors. PS1132 offers meaningful clinical promise and a differentiated partnering opportunity.
A Platform with Broad Potential
The STAMPTM platform is designed to treat many diseases caused by abnormal gene-expression programs. Beyond hematologic cancers, our pipeline includes solid tumors, such as prostate and breast cancer, and chronic inflammatory and neurodegenerative diseases, including inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis, and Alzheimer’s disease.
Join the Epigenetic Revolution
Parkside Scientific is building the next generation of epigenetic medicines. By reprogramming gene-expression networks, we aim to deliver safer, more durable therapies for patients with serious diseases.
For investment and partnership opportunities, contact info@pksci.com.
07/2026
[1] Novartis is seeking EMA and FDA approval for Pelabresib + Ruxolitinib combination for myelofibrosis.