Global Gene Therapy: The Panacea for All the Ills

Global Gene Therapy: The Panacea for All the Ills

The global gene therapy is estimated to be the billion-dollar market by 2021. The gene therapy market is projected to grow at a higher CAGR with the launch of potential therapies during the forecast period from 2016-2021 in the rare indications as reported by the Polaris Market Research Report “Gene Therapy-Competitive Analysis and Market Forecast- [2016-2021]”


There are many gene therapies which have been launched in the market. The European Union has approved two gene therapies until now namely Glybera from UniQure for the treatment of lipoprotein lipase deficiency in the year 2012. Also, in the year 2016, the European Union approved Strimvelis for severe combined immune deficiency in children. While the FDA until now has approved three gene therapies namely Yescarta for adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. On December 19, 2017 FDA approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Moreover, around 12 to 14 new gene therapies are in the development pathway for various common conditions and rare conditions and are expected to reach FDA approval stages in the next 2 to 3 years.


What is Gene Therapy?

Gene therapy is a novel method, which has the potential to treat, cure and even prevent disease by means of altering the gene expression, which in turn alters the protein in the body. Most of the diseases are caused by damaged; dysfunctional genes which when rectified can restore the normal function of the gene and give us the “long life”. Genetic disorders cause around 4000 diseases, which theoretically can be corrected by gene therapy, thus bringing the ray of hope especially for the terminally ill patients.


How it is done? The genetics ABC of Gene Therapy.

Genes are made of DNA, which carry information to produce the correct protein, which is quintessential for normal functioning of the human body. However, genetic mutations cause the DNA sequences to change leading to faulty protein sequences or non at all. To correct the faulty gene sequence, a carrier vector in the form of virus is used to deliver the modified gene. It is interesting to note here that the virus is modified as it might cause a disease on its own. The delivery of modified gene containing virus is done through injection (intravenous, subcutaneous, intraperitoneal or intramuscular). Once inside the virus integrates with the cell and produces the protein for the gene encoded, thereby eliminating the problem at the genetic level. Therefore, theoretically, gene therapy could be the proverbial “magic bullet”.


The milestones reached by Gene Therapy so far.

There are number of gene therapies for different disease segments, with cancer leading the chart with 693, followed by rare diseases with 501, besides several neurological, cardiovascular, metabolic diseases, infectious diseases, blood and clotting, musculoskeletal and others.


Opportunities and Challenges associated with Gene Therapy.

Gene therapy can address the unmet clinical needs for the patients suffering from life threatening diseases, who for now have no other options except for supportive care and the prescribed treatment includes serious side effects besides being costly. However, given the fact that gene therapy is challenging to develop, it could prove difficult for small biotech companies owing to associated set-backs in the various development stages and the unforeseen side-effects of the gene therapy. To make the gene therapy available to the patients, financial viability studies are to be conducted by the manufacturers. Perhaps, new model of pricing and payment could be adopted to address the concerns.


Given the small population segment, which suffers from genetic disorders, it might affect the generation of robust clinical evidence. Also, the use of viral vectors to transfer the therapeutic gene into the target area could raise long-term safety issues.


The biggest challenge to be addressed for gene therapy is regarding the entire regime of pricing and payment as the affordability of gene therapy is one of the primary concern. To address this challenge, their needs to be collaboration for effective policy development in order to create a stable pricing, financing, and health insurance structure for gene therapy.

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