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Cell Therapy Biotech Raises $75.5M in Venture Funds

Platelets in Red Cross Box

(Jason Scragz, Flickr. https://www.flickr.com/photos/scragz/4900742158)

4 Nov. 2021. A developer of off-the-shelf genetically engineered platelet cells to treat a range of diseases is raising $75.5 million in its second venture round. Platelet BioGenesis Inc. in Watertown, Massachusetts is a seven year-old spin-off business from the cell biology lab of Joseph Italiano at Boston Children’s Hospital, affiliated with Harvard Medical School.

Platelet Bio designs and develops therapeutics based on properties of platelets for autoimmune diseases, blood disorders, and inflammation. Platelets are blood cells whose function is to stop bleeding from broken blood vessels. When platelets are working properly and at normal levels, they respond to signals of a damaged blood vessel from a protein known as thrombin and accumulate at the site forming a clot, which slows the blood flow.

But as the company notes, platelets perform other useful functions beyond control of bleeding. Platelets also accumulate proteins and antibodies for delivery to tumors and other disease sites. In addition, platelets are cells without nuclei, which means they function without DNA, making platelets less likely to generate an immune reaction. The company says its technology produces engineered platelet-like cells that perform blood coagulation functions like natural platelets, as well as deliver therapeutic payloads and clear unwanted antibodies from disease sites.

Platelet Bio says its technology engineers induced pluripotent stem cells, also known as adult stem cells since they’re derived from mature rather than embryonic tissue, that can transform into almost any human cell or tissue. In this case, the company reprograms adult stem cells to become megakaryocytes that produce platelets in bone marrow. In that process, Platelet Bio genetically alters the stem cells to transform into megakaryocytes for producing platelet-like cells with desired therapeutic properties.

Three therapy candidates in preclinical stages

The company’s lead product is a treatment for immune thrombocytopenia, an autoimmune disorder where the body produces low levels of platelets, leading to excessive bruising that appears as purple or red dots on the skin and bleeding. In people with immune thrombocytopenia, the immune system attacks rather than produces platelets. For children, immune thrombocytopenia is usually a short-term acute condition, but in adults, it can become a long-term chronic disease. Most patients with immune thrombocytopenia are treated with corticosteroids, but in severe cases that do not respond to medications, patients may need to have their spleens removed to prevent production of harmful antibodies.

Platelet Bio says its as-yet unnamed treatment candidate for immune thrombocytopenia is in preclinical development in preparation for clinical trials, and expects to file an investigational new drug application with FDA in the first half of next year. The company has two other therapies in earlier preclinical stages for osteoarthritis and liver fibrosis.

Platelet BioGenesis is raising $75.5 million in its second venture funding round, with contributions from current investors Ziff Capital Partners and Qiming Venture Partners, and new investors SymBiosis, K2 HealthVentures, and Oxford Finance. According to Crunchbase, the company previously raised $47.3 million in its first venture round delivered in three installments between June 2017 and September 2019. In addition, Platelet Bio received a number of research grants and a contract in October 2019 with the U.S. Biomedical Advanced Research and Development Authority for platelet-like cells to treat nuclear or radiological illnesses, which could bring the company as much as $56.6 million.

“This is a major milestone for Platelet Bio,” says CEO Sam Rasty in a company statement released through BusinessWire, “adding capital and resources needed to advance our innovative platelet-like cell therapy science and manufacturing platform and support key corporate initiatives over the next 18 to 24 months.”

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