Next-generation therapeutics

The earliest medicines were mostly serendipitous discoveries from nature. Then, in the late 20th century, we entered a new era of targeted therapeutics based on proteins or small molecules. Although modern medicine has vastly improved human health, small-molecule drug discovery takes an enormous amount of time and money. Moreover, many conditions—from rare genetic conditions to common neurodegenerative disorders—remain untreatable.

Broad scientists have also catalyzed a major paradigm shift in therapeutic development by pioneering breakthrough gene-editing technologies, such as CRISPR-Cas9 and base editing, that strike at the root causes of diseases. Broad researchers are also working to engineer efficient vehicles for safely and efficiently delivering gene therapies to specific cells and organs. These revolutions promise to both dramatically accelerate the speed at which medicines are created and open new therapeutic avenues for difficult-to-treat diseases.

At the same time, the Broad’s Center for the Development of Therapeutics has spearheaded new methods to accelerate the pace at which new medicines are developed. They partner closely with investigators from across the Broad community to take on ideas that might be considered too risky or early for industry, combining deep biological knowledge with state-of-the-art technologies to pursue drug targets.

Philanthropic partnerships are critical in supporting the rigorous research needed to bring these innovations closer to the clinic. We are seeking philanthropic partners in the following ongoing projects:

  • Engineering modular, programmable therapeutics that accurately and consistently strike at the root causes of diseases.

  • Discovering and harnessing systems that can be programmed to safely deliver gene therapies to specific cells and organs.

  • Developing the next wave of groundbreaking therapeutic modalities, including finding novel methods and tools to accelerate the early-stage drug discovery process.

News coverage on our next-generation therapeutic research