Epigenetic drugs witness 375% growth in series A venture financing in 2024YTD, reveals GlobalData

New Delhi: Advances in gene editing, such as the FDA approval of Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) in December 2023 for the treatment of beta thalassemia and sickle cell disease, have fueled significant interest in innovative epigenetic drugs.

Against this backdrop, the total value of series A venture financing for epigenetic drugs almost doubled from $172 million in 2019 to $342 million in 2024 year-to-date (YTD) and observed a 375% increase from 2023, reveals GlobalData, a leading data and analytics company.

GlobalData’s Pharma Intelligence Center Deals Database reveals that companies developing epigenetic drugs received over $1 billion in total series A venture financing from 2019 to 2024YTD. Over half of these companies are headquartered in the US.

Epigenetic drugs modify genetic material, resulting in heritable changes in gene expression without altering the DNA sequence. Current marketed epigenetic drugs in blood cancers include Celgene’s Vidaza (azacitidine), Eisai’s Dacogen (decitabine) and Merck & Co’s Zolinza (vorinostat). However, these drugs act in a genome- and tissue-wide manner, resulting in off-target effects and subsequent toxicity.

Alison Labya, Business Fundamentals Analyst at GlobalData, comments: “Biotech startups are developing a new generation of epigenetic drugs with improved gene specificity to enhance efficacy, tolerability, and target indications beyond oncology. These drugs aim to provide safer alternatives to other methods of gene editing, such as clustered regularly interspaced short palindromic repeats (CRISPR), by not cutting into the DNA.”

The 2024YTD already witnessed most of series A venture financing for epigenetic drugs within the last five years. In March 2024, Avenzo Therapeutics raised $150 million in the largest round of series A financing for epigenetic drugs in the last five years to develop its CDK2 inhibitor ARTS-021, currently in Phase I/II for solid tumors and HR+/HER2- metastatic breast cancer.

In January 2024, Moonwalk Biosciences secured $57 million in seed and series A financing to develop its epigenetic discovery platform technology. In July 2022, Epicrispr Biotechnologies raised $55 million in series A financing to advance its preclinical pipeline and further drug discovery, using its CRISPR-based epigenetic platform technology with adeno-associated virus (AAV) as a delivery vector.

Labya concludes: “Epigenetic drugs are key to precision medicine, offering tailored treatments with improved patient outcomes and reduced off-target effects. Investors are demonstrating a focus on precision medicine, leading to an increase in early-stage investment in epigenetic drug development.

“However, companies developing innovative epigenetic drugs must overcome challenges including maintaining durable effects on gene expression, delivery of large drug components to target tissues, and demonstrating safety and efficacy in clinical trials to enable market success.”