Pharma R&D Today

Ideas and Insight supporting all stages of Drug Discovery & Development

Select category
Search this blog

Advances in Gene & Cell Therapy

Posted on November 6th, 2017 by in Pharma R&D


I would like to applaud the recent news about the Advisory Panel to the USA FDA’s unanimous (16-0) recommendation for allowing to further develop therapy by Spark Therapeutics Inc. It happened on October 12, a bright day for the research and development of cellular, tissue and gene therapies, which have been waiting in the wings for so many years. This will help the public and patients with many unmet medical needs. Patients are the real beneficiaries to what is taking place at this time. Members of the committee carefully looked at what Spark Therapeutics has been able to do for a patient who had a genetic disposition for being blind.

The therapy is called Luxturna, for which the FDA is hoping to decide on approval within the next four to six months. The mechanism of Luxturna’s efficacy is through delivery of the functional copy of a gene known as RPE65, which can restore visual function in patients who are prone to total blindness. It’s a real—literal—ray of hope for this category of patients. This is an unbelievable gift of vision for those who have been considered unfortunate until this date. Now they can have a ray of light which can shine in their eyes and a hope for seeing this beautiful world. Spark Therapeutics did a small Phase III trial, which showed that the treatment improved sight in almost all study participants.

The entire sector in research now has high hopes. Even earlier in the year, we saw Novartis AG’s CAR-T therapy receiving such an approval from the regulatory agency. Mike Lee, Jeffries analyst, rightfully said that “Spark’s gene therapy treatment, once approved, would thematically mark a watershed moment for the entire field.” Now it remains to be seen as to how these therapies would be paid for or covered by health insurance companies or Medicare. The cost per therapy is still something that needs to be debated amongst the manufacturing companies and the payers. For an average patient, the cost would be beyond means if it has to be borne by out-of-pocket coverage.

Fortunately, Luxturna is designed to be a single-time treatment for patients with vision loss, which could be total or partial. It is inherited retinal disease that is generally diagnosed in young children. I really liked what Dr. Wilson Bryon of the FDA said about this therapy. He said, “This is exactly the type of disease that we hoped that gene therapy would someday treat.” He is currently holding the post of Director, Office of Tissues & Advanced Therapies, at the FDA. The retinal disease that we are discussing here is caused due to a mutated RPE 65 gene, which was the target of Spark Therapeutics. Many of the patients who received therapies had night blindness and an inability to see in dim lighting. One thing that one needs to look at is the ‘gene delivery’ system. The delivery was done through sub retinal injection, which seems to cause treatment emergent adverse events. About 15% events were severe in nature.

My thinking at this time is to address such a gene delivery through application of some of the novel delivery systems, which came out of Prof. Bob Langer’s labs in MIT or through usage of ‘nano-bubble’ technology that I have seen come through Prof. Harendra Parekh’s labs in Queensland University. Someday, the delivery of genes has to be done in a more efficient and painless fashion. Nano-bubbles may be a solution, but it remains to be tested. We look forward to further long-term effects of such a therapy and its sustainability beyond a single-dose delivery.

In my opinion, gene therapy is here to stay, provided it is made sustainable through a better, affordable ‘cost of goods’ equation and sustainable delivery with built-in efficiency. Most of the inherited diseases should require only single-time administration of genes, which is a welcome news. However, the cost of such single administration needs to be further optimized so that it becomes affordable to the patient and sustainable to the pharma/biotech companies. I surely see benefits significantly outweighing the risks. Therefore, let us hope that in the next decade we see the emergence of many more such therapies.

Novartis AG’s recently approved CAR-T cell therapy, Kymriah, is also classified as gene or cell therapy. It costs about half a million US dollars for one-time treatment. I would like to see the treatment costs optimized so that payers and patients see some equilibrium, and pharma/biotech companies see commercial sustainability in their pursuit to be in the market and continuing R&D on such novel therapies! Let us hope this is the beginning of many such novel gifts to the patiently-awaiting patients.

Elsevier Recommends:

Read the white paper to learn more: Precision Medicine.

All opinions shared in this post are the author’s own.

R&D Solutions for Pharma & Life Sciences

We're happy to discuss your needs and show you how Elsevier's Solution can help.

Contact Sales