PlateletBio is a company that develops new cell therapies based on platelet biology. $75.5 million raised Advance its drug pipeline, including a lead candidate for a rare bleeding disorder, which is expected to reach clinics next year.
Platelets are a component of blood and are known for their ability to form hemostatic clots. But PlateletBio, based in Watertown, Massachusetts, points out that platelets have other properties, including their role in transporting growth factors and proteins throughout the body. PlateletBio is developing therapies that take advantage of these properties, but instead of using platelets from patients or healthy donors, the startup makes them.
In the body, platelets are formed in the bone marrow. PlateletBio produces platelet-like cells or PLC in a bioreactor that simulates bone marrow conditions. The raw material of its PLC is stem cells, which can become almost any cell or tissue in the body.
Platelets are not technically cells. They do not have a nucleus, but this is an advantage for therapeutic applications. Since PlateletBio therapy does not introduce DNA into the patient’s body, it avoids the potential risk of introducing foreign genetic material. PlateletBio said it can produce PLCs with new functions and therapeutic payloads, including antibodies, signaling proteins, therapeutic proteins and nucleic acids.
PlateletBio’s main cell therapy drug candidates are being developed for treatment Immune Thrombocytopenia, A blood disease in which the immune system mistakenly treats the patient’s platelets as a foreign body and destroys them. People with immune thrombocytopenia have dangerously low platelet counts, which makes them prone to bleeding.
There is no FDA-approved treatment for the underlying cause of immune thrombocytopenia, but corticosteroids are used to try to suppress the immune system’s attack on platelets. Platelet transfusion is another option, but the National Rare Disease Organization states that these treatments are usually used in emergency situations because platelets may be destroyed by antibodies produced by the patient.
There are two FDA-approved small molecule options for patients who do not respond to early treatment: Tavalisse from Rigel Pharmaceuticals and Swedish orphan Biovitrum drug DopteletSanofi’s goal is to treat this disease with a small molecule called rilzabrutinib. The drug is designed to block Bruton’s tyrosine kinase, a protein that plays a role in the development of B cells (a type of immune cell). Sanofi acquired the molecule last year by acquiring Principia Biopharma for US$3.7 billion.
The main disease target of the Principia drug is multiple sclerosis. In September, Sanofi reported on Rizabutinib Failure The third phase of research. A separate phase 3 trial for immune thrombocytopenia is underway, and another mid-term clinical trial for an autoimmune disease called IgG4-related disease is also underway.
PlateletBio is not the only company trying to transform blood components into new cell therapies.Cambridge, Massachusetts Rubius Therapeutics is developing cell therapy based on red blood cellsAfter the disappointing results of early clinical trials for the rare disease phenylketonuria last year, Rubius Shift Its focus is cancer and immune system diseases. PlateletBio’s PLC will represent a new approach to the treatment of immune thrombocytopenia. According to PlateletBio’s website, the company plans to submit an investigational new drug application for its therapeutic candidates in the first half of next year.
PlateletBio is based on the research of Harvard University scientist Joseph Italiano, who co-founded the company under the name Platelet BioGenesis. when. . .when The start-up company appeared in 2017 and it is developing platelets that can solve the platelet shortage problem faced by blood donation centers.. Two years ago, the start-up company expanded its Series A financing with $26 million. Additional financing And plans to use its platelets for cell therapy. In addition to immune thrombocytopenia, other diseases that the biotechnology aims to treat include osteoarthritis and liver fibrosis.
PlateletBio’s latest round of Series B financing added new investors SymBiosis, K2 HealthVentures and Oxford Finance. Earlier investors Ziff Capital Partners and Qiming Venture Capital also participated in a new round of financing.
“This is an important milestone for PlateletBio, increasing the capital and resources required to advance our innovative platelet-like cell therapy science and manufacturing platform, and supporting key corporate plans for the next 18 to 24 months,” said the startup’s president and chief executive. Executive Officer Sam Rasty said in a prepared statement.
Photos of Flickr users Marco Wilch Through knowledge sharing license
