Genomic Medicine: Catch The Gene Therapy Wave

Brief about the Genomic Medicine:
– The first two U.S.-approved autologous T-cell immunotherapies, Kymriah and Yescarta, are commercially available in 2018.
– Three leading gene therapy companies have been acquired in the last nine months for a combined $30 billion.
– Catalysts over the next year are poised to drive group valuations.

The Genomic medicine is being considered as the future of the healthcare industry. This precise and personal approach to medicine is an emerging discipline with enormous disruptive potential, is an initiation by the Human Genome Project (“HGP”). Although sought by Silicon Valley and DC politicians for many years; the genomic medicine is showing signs of being the transformative force. This article is about the cells and gene therapy and genome (aka gene) editing. The companies that fall under this category would be targetted as follows:

Big Pharma-
Representing the pharmaceutical and large-cap biotechnology companies that are actively looking to get engaged in acquiring or collaborating with gene therapy companies. Companies referenced later in this article are Celgene (NASDAQ:CELG), Gilead (NASDAQ:GILD), Novartis AG (NYSE:NVS) and Pfizer (NYSE:PFE).

Gene and Cell Therapy-
Considered as the superset representing companies whose primary purpose is pursuing cell therapies, gene therapies, gene regulation or genome editing. The companies tracked include Abeona Therapeutics (NASDAQ:ABEO), bluebird bio (NASDAQ:BLUE), Audentes Therapeutics (NASDAQ:BOLD), AveXis (NASDAQ:AVXS), Cellectis S.A. (NASDAQ:CLLS), CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT), Homology Medicines (NASDAQ:FIXX), Intellia Therapeutics (NASDAQ:NTLA), Spark Therapeutics (NASDAQ:ONCE), uniQure N.V. (NASDAQ:QURE), REGENXBIO (NASDAQ:RGNX), Sangamo Therapeutics (NASDAQ:SGMO) and Voyager Therapeutics (NASDAQ:VYGR).

Genome Editing-
It is a subset of the Gene and Cell Therapy group. The five companies tagged with Genome Editing are Cellectis, CRISPR, Editas, Intellia and Sangamo. Both bluebird bio and Homology Medicines are likely to be included in this group in the next six months. Bluebird acquired privately held Precision Genome Engineering “Pregenen” in 2014, and has been quietly in early research mode. This will be explored further in a future article. Homology is also pursuing genome editing, but further research needs to be done to assess its status.

The Healthcare Spending
Out of the U.S. Gross Domestic Product is enlightening, the healthcare spending accounts for 17.9%, yet the realization of this economic mix was only 5% in 1960 is staggering. They stated that we increasingly allocate healthcare funding for treating the alleviate pain and slow disease progression, furthermore, occasionally finding the cure. This makes an increase in average lifespan, that helps in adding years of therapeutic treatments. The current costs of such treatments are untenable yet unyielding. Whereas, the Genomic medicine is poised for changing this equation by providing one-time curative therapeutics which will eliminate a lifetime of pharmaceuticals.

Genes are the building blocks of heredity
Every human has two copies of each gene, in which one is inherited from each of the parents. So far, the HGP has investigated that humans have between 20,000 and 25,000 genes. Our genes provide an instruction manual to the manufacture protein molecule, as they are made up of DNA. However, the gene mutations are never found causing any disease in the human body. Where the genetic disorders are caused due to the gene mutations that disturb the functional role of the gene.
Here, it is important to note that not all mutations are ever found leading to any of the disorders. They are, in fact, found with only a small percentage of them causing the actual genetic disorders. However, they also have some positive effects that assist an evolutionary change as soon as the genes adapt to the environment.

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