Harvard Startups

Selective, small-molecule histone deacetylase (HDAC) inhibitors

Infectious disease detection and genotyping

In 2012, the Botswana Harvard AIDS Institute Partnership — a decades-long collaboration chaired by Prof. Max Essex — was conducting a study of HIV transmission in newly pregnant women in Gabarone, Botswana. It was critical for clinicians to identify patients with drug-resistant strains of HIV in order to prescribe the medication that would be most effective — but the existing test was slow and expensive. A team of Harvard researchers developed a novel qPCR genotyping platform, called Pan-Degenerate Amplification and Adaptation (PANDAA), that can rapidly identify variations in clinically relevant genetic sequences. In 2014, the team launched a startup, Aldatu Biosciences, to make the technology widely available at low cost, and set Aldatu up as a public benefit corporation.

Precision medicines, using base editing to correct genes

The majority of disease-associated human DNA variations consist of point mutations — locations in the genome where a single base pair has been replaced by another. Innovations developed in the Harvard lab of Prof. David R. Liu enable the correction of single-base mutations in DNA with unprecedented precision and efficiency. “We developed programmable molecular machines that go to a target site of our choosing in the genomic DNA of a cell and directly convert one base to another base without making a double-stranded break in the DNA,” said Liu, who is a cofounder of Beam. The licensed technology opens up a wide range of human genetic conditions to the therapeutic promise of genome editing.

Understanding the diversity of the human microbiome

Harvard innovations in “lab-on-a-chip” technology enable researchers to manipulate and analyze a vast number of cells by capturing them within millions of tiny droplets of fluid. Biomillenia launched to commercialize this technology platform to study bacterial cultures from the human microbiome, in order to identify undiscovered strains and biological products that may be harnessed to promote health and well-being.

Human therapeutics through CRISPR genome editing

Researchers at Harvard made foundational contributions to the field of genome editing, enabling the use of technologies like CRISPR in the development of therapeutics to treat and prevent disease.

Regenerative medicines for stroke recovery and aging-related diseases

Stem cell researchers at Harvard, studying models of disease in mice, demonstrated that a protein called GDF11 plays a role in restoring function to tissues in the aging heart, muscle, and brain. Elevian was founded to pursue the potential for GDF11 to treat and prevent multiple aging-related diseases.

Oral delivery of therapeutics

i2O Therapeutics is exploiting the versatile properties of ionic liquids for the development of oral biologic therapeutics, with a focus on metabolic diseases, inflammatory diseases, and other indications. The ionic liquid platform technology was developed in the lab of Prof. Samir Mitragotri with support from the Blavatnik Biomedical Accelerator and can support oral solutions of a broad range of drugs including peptides, proteins, nucleic acids, and full-length antibodies.

Proteomic analysis for drug discovery and development

Treating blood diseases and immune disorders

With support from the Blavatnik Biomedical Accelerator over two years, Prof. David Scadden developed a milder method to carry out the bone marrow conditioning necessary in advance of hematopoetic stem cell transplantation. The portfolio licensed by Magenta Therapeutics has the potential to transform blood stem cell transplants from a “treatment of last resort” into a safer, more efficient therapy for patients with blood diseases and immune disorders.

Medications to treat cough, pain, itch, and inflammation

With support from the Blavatnik Biomedical Accelerator, researchers at Harvard and Boston Children’s Hospital led by Profs. Bruce Bean and Clifford Woolf identified novel sodium channel blockers that locally and selectively inhibit pain and itch signaling in activated nociceptors but spare other neurons. They founded Nocion Therapeutics to develop novel pharmaceuticals that may provide targeted, robust, and sustained relief for the treatment of serious medical conditions including cough, itch, pain, and inflammation.

Cell therapy for diabetes

Basic stem cell research over the course of a decade in the lab of Prof. Doug Melton led to a groundbreaking approach to treating diabetes. His lab developed methods of generating pancreatic beta cells in vitro. "In type 1 diabetes,” Prof. Melton told Harvard Magazine in 2019, “you have a situation where patients have to prick their finger and measure the sugar in their blood, and then inject the right amount of insulin. The beta cell does both of those things. It measures blood sugars, and injects the right amount of insulin.” Semma Therapeutics launched to advance that work toward the clinic.

Small-molecule therapies for cancer and other diseases

Within cells, non-coding RNA (ncRNA) plays a key role in regulating the expression of oncogenes and other proteins that affect disease. Harvard researchers identified one such helpful ncRNA called Let-7; it suppresses the translation of oncogenes, reducing the development of tumors. But a troublesome protein called Lin28 can reduce levels of Let-7, and a lower level of Let-7 correlates with greater cancer metastasis. Building upon the researchers’ initial innovations, Twentyeight-Seven Therapeutics aims to discover and develop small-molecule therapies to target pathways like Lin28/Let-7 to treat cancer and other diseases.

Developing single-course gene editing medicines to treat patients with cardiovascular disease

Verve Therapeutics has licensed foundational CRISPR gene-editing technologies, including the use of Cas9 and Cas12a (Cpf1), from Harvard University and the Broad Institute of MIT and Harvard, for human therapeutic applications against certain cardiovascular targets.

Delivering biologics to targets within cells

With support from the Blavatnik Biomedical Accelerator, Prof. Quan Lu harnessed the capabilities of newly discovered microvesicles called ARMMs to package and deliver large molecules (such as gene-editing complexes, RNA molecules, or other large proteins) to disease targets within cells. The innovations were licensed to Vesigen Therapeutics, a startup company which launched with investment led by Morningside Ventures and Leaps by Bayer. The company aims to build out the ARMMs platform as well as to advance numerous therapeutic agents into preclinical and clinical development.