Companies Harness Gene Therapy Technology in Hopes of Developing Lasting Treatments for Disease

Companies Harness Gene Therapy Technology in Hopes of Developing Lasting Treatments for Disease
February 1, 2016
By Alex Keown, Breaking News Staff

A number of pharmaceutical companies are blazing a trail to develop of treatment for an assortment of cancers and rare diseases using gene therapy techniques.

Gene therapy treatment is a high risk, high reward venture, but the promise of therapies developed through these programs could be life changing for patients. The goal of gene therapy is to develop treatments that only have to be administered once a decade or longer, Tim Miller, chief executive officer Abeona Therapeutics, told BioSpace in an exclusive interview.

Gene therapy essentially transforms patient’s cells to harness their immune system to fight an invading disease on its own. Miller said he was excited about the possibility of developing lasting, and hopefully curative treatments for rare diseases such as Sanfilippo syndrome or Batten disease—two diseases that rarely allow those stricken with them to live past the second decade of life. Miller said he hopes new therapies being developed by Abeona are able to increase the survivability rates of these genetic diseases.

“Being able to do that, to correct that survivability, is of critical importance to families,” Miller said.

Miller predicted 2016 will be an “exciting year for gene therapy” companies. He said about half dozen gene therapy companies are conducting trials and he is hoping to see plenty of positive results.

Abeona, like many other gene therapy companies, uses CRISPR-Cas9 gene editing technology for the development of its treatments. "CRISPR" refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the "molecular scissors" that cut and edit, or correct, disease-associated DNA in a cell. The technology was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier. Doudna went on to found Caribou Biosciences. Charpentier sold her part of the rights of the CRISPR-Cas9 platform to CRISPR Therapeutics, a company making big moves in the gene therapy field.

CRISPR Therapeutics struck a $335 million deal with Bayer in December 2015 to develop treatments for genetic disorders of the blood, blindness, and congenital heart disease through CRISPR-Cas9 gene editing technology. CRISPR is also working with Vertex on developing therapies for sickle cell disease.

CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions. Other companies that are directly controlling the technology include Caribou Biosciences, which has formed a strategic alliance with DuPont and Novartis . Caribou also formed Intellia Therapeutics, which is active in gene therapy. In 2015, Juno Therapeutics struck a $727 million deal with Editas Medicine, the gene editing company, for a partnership that will forge three research programs marrying Editas’ technologies, including CRISPR-Cas9, to Juno’s CAR-T and TCR technologies.

Elsewhere, preclinical data from a Cedars-Sinai study of inherited retinitis pigmentosa, a degenerative eye disease with no known cure and can lead to blindness, is making headway in animal models. The researchers used CRISPR/Cas9 to remove a genetic mutation that causes the blinding disease. Cedars-Sinai said that CRISPR/Cas9 has been in use for about five years, but over that time “it has transformed the science of genome editing by making the process easier, more dependable and less expensive.”

While there is great reward through gene therapy, it’s not uncommon for a promising therapy to see a setback, such as recently occurred with bluebird. In December, the company stock plunged after bluebird released mixed data for its gene therapy sickle cell treatment revealed at the American Society of Hematology. The company announced its gene therapy treatment, LentiGlobin, works differently for each patient, which means single conclusive results cannot be achieved. This was something hinted at in November when bluebird released preliminary information.

But it wasn’t all bad news for bluebird. Company data showed that some patients who required needed frequent and regular blood transfusions have responded positively to the therapy and have been transfusion free for up to two years.

As gene therapy continues to make rapid advances, Miller’s prediction of 2016 being the year for gene therapy may be on the money.

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