Summary analysis of the top ten precision medical companies

Introduction to "Personalized Medicine"

Precision medicine is also called “personalized medicine”. Conceptually “individualized medicine” is not an independent industry. It is a modern medical concept that runs through the whole process of medical treatment; its technical basis is “gene sequencing” and related diagnostic services. (At present, the application in tumor diagnosis and prenatal screening is relatively mature), the follow-up treatment and medication are different according to genetic characteristics, age, gender, personality, etc. The cost is the doctor's brain power and experience to achieve more accurate, The damage is smaller.

The development of genetic technology originated from the study of hematological diseases, and its "model" can be extended to the fields of solid tumors, genetic diseases, infectious diseases, etc. Therefore, gene sequencing, diagnosis and clinical application of blood diseases have absolute advantages in "personalized medicine". First mover advantage.

Precision Medicine is a new medical concept and medical model based on personalized medicine, combined with genome sequencing technology and the cross-application of biological information and big data science. As early as 2011, the American medical community first proposed the concept of "precise medicine". In early 2015, Obama proposed the "precise medical plan" in the US State of the Union address, hoping that precision medicine can lead a new era of medicine.

Since then, precision medicine has sprung up. In 2016, precision medicine is the best time to develop. Boston, USA, has gathered some of the world's top universities, such as the Massachusetts Institute of Technology (MIT) and Harvard University. These scientific research institutions have made Boston a global precision medical company. One of the hot spots gathered.

EditasMedicine: The first IPO company in the field of CRISPR gene editing

Founded in 2013, EditasMedicine (NASDAQ:EDIT) is a biotechnology company that is co-founded by MIT's Zhang Feng and others. EditasMedicine is committed to applying gene editing technology to human therapy and performing precise molecular modifications at the genetic level to develop treatments that directly alter disease-causing genes.

EditasMedicine is a leader in gene editing, protein engineering, molecular and structural biology, and CRISPR/Cas9 and TALENs technologies. As of now, EditasMedicine has 21 patents in related fields, and 200 patents are pending.

EditasMedicine is a company that has made rapid progress in clinical trials in the field of genetic editing. It is planned to conduct a CRISPR experiment in 2017 to conduct human experiments on Lebercongenitalamaurosis (LCA; a hereditary vision loss disease). This will be the first time that CRISPR gene editing technology has been used in clinical trials.

In addition, Editas's projects include cancer CAR-T treatment programs in collaboration with Juno, as well as benign blood diseases, hereditary muscle diseases, hereditary lung diseases, hereditary and infectious liver diseases.

Summary analysis of the top ten precision medical companies

IntelliaTherapeuTIcs: Exclusive access to CRISPR/Cas9 intellectual property

Founded in 2014, IntelliaTherapeuTIcs focuses on the development of CRISPR/Cas9 technology in the therapeutic field, such as leukemia and cancer. Intellia enjoys an extremely important intellectual property right in the field of CRISPR/Cas9 technology treatment and received the first round of financing from AtlasVenture and Novartis.

The future direction of IntelliaTherapeuTIcs is to better integrate CRISPR and CAR-T. In January 2015, IntelliaTherapeuTIcs launched a five-year R&D cooperation program with Novartis, a leader in the CAR-T field. The collaboration between Novartis and Intellia Therapeutics will enable the integration of CAR-T technology with the CRISPR/Cas9 genome editing technology. Develop a more powerful cancer treatment.

In addition, Novartis will work with Intellia Therapeutics to study diseases related to hematological stem cells (HSCs) using CRISPR genome editing techniques, including sickle cell disease and beta-thalassemia. In 2015, Intellia Therapeutics received $70 million in Series B financing.

Summary analysis of the top ten precision medical companies

BluebirdBio: Focus on developing rare disease gene therapy

Founded in 1992, BluebirdBio (NASDAQ: BLUE) is a pharmaceutical company that provides genetic testing and gene therapy for children. It provides targeted early treatment for polio, child malnutrition, and anemia through genetic research in children. .

BluebirdBio has a number of clinical programs, such as Lenti-D for the treatment of childhood cerebral adrenal leukodystrophy, and the treatment of beta-aerita anemia and sickle cell anemia drug LentiGlobin in the first patient.

BluebirdBio is a "unicorn" in the field of gene therapy and currently has a market capitalization of $3.6 billion. Of course, this is closely related to the fact that it is studying the treatment of high-risk monogenic genetic diseases, thalassemia.

Summary analysis of the top ten precision medical companies

ModernaTherapeutics: Creating an mRNA treatment platform

2015 was the year of the outbreak of mRNA therapy, as four mRNA treatment companies, including Moderna Therapeutics, attracted more than $4 billion.

Founded in 2010, ModernaTherapeutics is a biotechnology company that specializes in the study of messenger RNA (mRNA) for the treatment of cancer, infectious diseases and rare diseases. The core technology of ModernaTherapeutics is to help people make drugs in their own cells, rather than configuring drugs for taking or injecting in the lab (this is the practice of all other biotech companies).

The specific method is to implant mRNA into the patient, which then stimulates the human cells to make the protein needed for treatment without triggering a common human immune response.

In January 2015, the company completed the largest financing of biotech companies in history - $450 million. This financing has made Moderna the most valuable private company with venture capital support in the world's drug development industry.

"Fortune" has written that: Moderna may be the only revolutionary pharmaceutical company born in at least a decade, or at least equal to Google's support of Calico, which aims to delay cell aging.

Summary analysis of the top ten precision medical companies

SQZBiotech: Leading Cell Therapy Platform

Founded in 2013, SQZBiotech was founded by ArmonSharei of the Massachusetts Institute of Technology. On December 7, pharmaceutical giant Roche reached a $500 million cooperation order with startup SQZBiotech. The two sides will rely on SQZ's technology platform to jointly carry out research on cancer immunotherapy.

This cell therapy will use SQZBiotech's CellSqueeze technology to introduce tumor-associated proteins into patient beta cells, thereby activating T cells against cancer. CellSqueeze technology is a microfluidic chip that allows the presented material to enter almost all cell types. SQZBiotech has an exclusive global license for the MITCellSqueeze technology platform.

SQZ was selected by FierceBiotech as one of the “15 Most Popular Companies” in 2015; its technology platform was selected as one of the “Top Ten Creative Ideas in the World” by the famous American popular science magazine Science American in 2014.

Summary analysis of the top ten precision medical companies

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