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Bone marrow transplantation drug: Magenta Therapeutics, Inc. (MGTA)

Magenta Therapeutics, Inc. (NASDAQ:MGTA) was founded on June 17, 2015, formerly known as HSCTCo Therapeutics, Inc., and changed to its current name on February 16, 2016. Headquartered in Cambridge, Massachusetts, USA, full-time employees 48 people (4/30/2018), focusing on the development and commercialization of transplant drugs such as bone marrow transplantation. Investors include Third Rock Ventures and Atlas Venture, Alexis A. Borisy, Bruce Booth, D. Phil., and the co-founder is Jason Gardner, D. Phil. and David T. Scadden, MD, etc.

Magenta Therapeutics

Magenta Therapeutics, Inc. (MGTA):

For more than 50 years, doctors and patients have made the difficult choice of bone marrow transplantation: surgery can save lives and cure patients, but the risk of toxicity and death often hinders its use. At Magenta, the company believes that it can refocus this concern on treatment and enable more patients with devastating diseases of severe autoimmune diseases (including multiple sclerosis), blood cancers, including leukemia, and sickle cell Diseases and other genetic diseases have benefited from advances in transplant medicine.

Transplantation is an effective and often cured medical treatment. The emerging data of stem cell gene therapy (that is, the use of genetically modified stem cells for bone marrow transplantation) show that this new type of transplantation method may bring meaningful benefits. Bone marrow transplantation and stem cell gene therapy use the same extensive transplantation process as decades old. Bone marrow transplantation is a huge market opportunity, and improvements to existing methods can expand bone marrow transplantation to more patients. The ability of bone marrow transplantation therapy is limited by how to obtain enough cells for surgery, and current methods for patients preparing for transplantation and post-transplant complications lead to inherent morbidity and mortality.

Magenta Therapeutics is a clinical-stage biotechnology company that develops new drugs to bring the effects of bone marrow transplantation to more patients. At Magenta, the company believes that it has unique advantages to overcome these challenges and lead transplant medicine into a new era.

Magenta Therapeutics’ product portfolio includes biologics, small molecules and cell therapies, designed to solve the shortcomings of existing methods and extend the healing power of bone marrow transplantation to more patients with multiple diseases. Currently, only a small percentage of patients with these diseases have undergone transplantation because the risks and challenges outweigh the possibility of cure. These include: diseases for which bone marrow transplantation is the standard treatment for the disease (such as blood cancers, including acute myelogenous leukemia, myelodysplastic syndromes, multiple myeloma and non-Hodgkin’s lymphoma), which can be adapted to transplant treatment but limited use Diseases (such as hemoglobinopathy, including sickle cell disease and β-thalassemia) and autoimmune diseases.

Emerging clinical data indicate that bone marrow transplantation may be a breakthrough method with therapeutic potential for patients with severe autoimmune diseases. For example, the latest results of multiple clinical trials show that patients with autoimmune diseases, including multiple sclerosis and scleroderma can be cured by transplantation. However, according to Magenta Therapeutics’ epidemiological analysis, currently only about 1% to 2% of patients with multiple sclerosis or scleroderma in the United States and Europe receive bone marrow transplantation.

In order to solve the main unmet medical needs in the existing bone marrow transplantation process, Magenta Therapeutics is developing a stem cell biology discovery platform and a comprehensive combination of new therapies. Magenta’s plan will improve stem cell volume (expansion), stem cell collection (mobilization), preparation for transplant patients (conditioning), and potential post-transplant complications to address the key limitations of the bone marrow transplant process , In order to benefit more patients.

1. In the expansion project of Magenta Therapeutics, the company’s most advanced clinical candidate product is MGTA-456 , a cell therapy, which has achieved clinical proof of concept in 36 patients with blood cancer and is currently working on the treatment of fatal genetic metabolism. Study on patients with sexual diseases. MGTA-456 is an expanded cord blood product that may give more patients a better chance of successful stem cell transplantation.

2. In the mobilization project of Magenta Therapeutics, MGTA-145 focuses on helping doctors more easily collect more hematopoietic stem cells (HSCs) from patients and donors to improve patient prognosis.

3. Magenta Therapeutics’ targeted transplant conditioning program for transplantation of patients is designed to selectively remove transplanted stem cells and/or immune cells from patients before transplantation, and the toxicity is much lower than that of a single radiation used for decades And chemotherapy.

4. Magenta Therapeutics’ post-transplant complications program aims to target donor immune cells that cause graft-versus-host disease (GvHD) in patients, which may be a fatal complication of transplantation.

Magenta Therapeutics Portfolio

Magenta Therapeutics project & plan portfolio

Magenta Therapeutics, Inc. (MGTA) investment:

Magenta Therapeutics, Inc. (NASDAQ:MGTA) submitted its IPO prospectus on 5/24/2018 and listed on NASDAQ on 6/21/2018 with an issue price of US$15.00. It issued 6,666,667 shares and raised US$100 million.

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