Startups are applying innovative techniques to cure the common cold, re-engineer the human microbiome, and fight against cancer.
Biotechnology is a broad sector encompassing technologies that take advantage of cellular or biomolecular processes to develop products across healthcare, agriculture, and industrials. Specific examples include vaccines, genetically modified seeds, and yeast-derived biofuels.
The past decade has seen tremendous advances in synthetic biology, and gene editing is nearing the inflection point of becoming a clinically viable therapy.
Led by former Biogen CEO George Scangos and conceived of by ARCH Venture Partners co-founder Robert Nelson, Vir Biotechnology launched in January 2017. Vir Biotechnology will be developing treatments and methods of prevention for viral and bacterial conditions based on the concept of immune programming. They will adopt a broad technology portfolio, including methods of drug delivery using viruses which were obtained through the acquisition of TomegaVax, Inc. Potential targets include Hepatitis B, influenza, and tuberculosis.
Focus: Viral and bacterial diseases
Select Investors: ARCH Venture Partners, Bill & Melinda Gates Foundation
Total Disclosed Funding: $150M
2. Forty Seven
A Stanford spin-out, Forty Seven is developing an antibody-based therapy aimed at treating two types of non-Hodgkin lymphoma, a form of blood cancer. The therapy will target the protein CD47, which normally sits on the surface of a cell and instructs the immune system not to attack it. Many cancers increase production of this protein in order to evade the immune system. However, Forty Seven’s antibody therapy (Hu5F9-G4) will attack these proteins, exposing cancerous cells to the body’s natural immune system.
Select Investors: Google Ventures, Lightspeed Venture Partners, Sutter Hill Ventures, Clarus
Total Disclosed Funding: $75M
C4 Therapeutics, spun out of research conducted at the Dana-Farber Cancer Institute, is built on a novel method of targeting disease-causing proteins for destruction. Their technology, called Degronimid, is capable of tagging disease-causing proteins for recognition by the body’s natural cell destruction mechanism, the ubiquitin/proteasome system. In March of this year, Google’s anti-aging spin-out Calico entered into a 5-year agreement with C4 Therapeutics to develop therapies that treat diseases of aging, including cancer.
Focus: Protein degradation
Select Investors: Novartis Venture Funds, Roche Venture Fund, The Kraft Group, Cobro Ventures, Cormorant Asset Management, EG Capital Group
Total Disclosed Funding: $73M
Nohla Therapeutics, based in Seattle, WA, is developing “off-the-shelf” universal cellular therapies for the treatment of patients with blood cancers, sepsis, chemotherapy-induced low white blood cells counts, and other life-threatening conditions. For example, in some types of cancer such as leukemia, patients receive bone marrow transplants in order to replenish the body’s ability to produce white blood cells. However, this method requires using bone marrow with proteins, called human leukocyte antigens (HLA), that match the patients’. Nohla’s therapy will be able to be administered without the need for HLA matching, making it an “off the shelf” universal donor product.
Focus: Stem cell transplantation
Select Investors: 5AM Ventures, ARCH Venture Partners, Jagen Group
Total Disclosed Funding: $63.5M
Launched in July 2016 with funding from Third Rock Ventures, Fulcrum Therapeutics is focused on developing small molecule drugs that modulate genetic on/off switches. The company is currently working on treatments for Fragile X Syndrome (genetic condition causing learning disabilities and cognitive impairment) and facioscapulohumeral muscular dystrophy (progressive skeletal muscle weakness).
Focus: Gene Therapy
Select Investors: Google Ventures, Third Rock Ventures
Total Disclosed Funding: $60M
Founded in the laboratory of Dr. Timothy A. Springer at Harvard Medical School, Morphic Therapeutics is developing oral integrin-based treatments with applications in oncology, immunology, vascular disease, and fibrotic conditions. Integrins are proteins that function in cell signaling by attaching to the outside of a cell and transmitting information both into, and out of, the cell. Abnormal cell signaling plays a role in many diseases due to its importance in cell survival, cell cycle progression, and cell differentiation.
Focus: Integrin-based therapies
Select Investors: AbbVie Biotech Ventures, Pfizer Venture Investments, Polaris Partners, SR One, ShangPharma
Total Disclosed Funding: $51.5M
Vedanta Biosciences is working on modulating the human microbiome in order to produce specific therapeutic effects. Vedanta initially isolated a collection of human-associated bacterial strains to determine how the human immune system responds to the microbes and through which biological pathways. And now Vedanta is applying these findings to develop a microbiome-based therapeutic pipeline. The company was recently awarded a patent for therapies, based on human microbiome-derived bacteria, targeting inflammatory bowel disease (IBD) and graft-versus-host disease, among other conditions.
Focus: Human microbiome
Select Investors: Invesco, PureTech, Rock Springs Capital, Seventure Partners
Total Disclosed Funding: $50M
8. Petra Pharma
NY-based Petra Pharma is working on small molecule therapies that target novel enzymes key to various disease processes such as cancer. While the company has released little concerning the mechanism of their proposed therapy, it will be based on the research of its founders Dr. Lewis Cantley of Cornell Medical College and Dr. Nathanael Gray of Harvard Medical School.
Focus: Oncology and metabolic diseases
Select Investors: Johnson & Johnson Innovation, Pfizer Venture Investments, IBM Watson Group, ARCH Venture Partners, Eli Lilly & Co., WuXi PharmaTech, AbbVie Biotech Ventures
Total Disclosed Funding: $48M
eGenesis, co-founded by Dr. George Church of Harvard, is developing genetically-edited pigs from which organs can be safely transplanted into humans. This is not currently possible due to the human immune reaction to foreign DNA. The solve this, the company is hoping to remove key pig genes using CRISPR-Cas9 gene editing. This will allow pig organs, such as livers or kidneys, to go undetected by the immune system when transplanted into humans.
Select Investors: ARCH Venture Partners, Khosla Ventures, Alta Partners, Biomatics Capital Partners, Heritage Provider Network
Total Disclosed Funding: $45M
Homology Medicines is developing both gene therapies and gene editing techniques to treat genetic conditions such as cystic fibrosis, Duchenne muscular dystrophy, lysosomal storage disorders, and inborn errors of metabolism. The company’s gene therapies are based on delivering functional genes, carried within viruses, to a cell where the gene is either missing or mutated. Homology Medicines is unique in its application of homologous recombination (naturally occurring method of DNA repair) to gene therapy. Typical gene therapies, while also delivered via viruses, enter the cell, express a chosen therapeutic protein, and then stop when the cell dies. Using homologous recombination, however, the cell will be able to spread the new gene every time it divides.
Focus: Gene Therapy
Select Investors: 5AM Ventures, ARCH Venture Partners, Deerfield Management, Temasek Holdings
Total Disclosed Funding: $43.6M
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