The global gene therapy market was value at USD 500 million in 2016 and is estimated to grow at a CAGR of 43.1% during the forecast period. Despite setbacks and disappointments, gene therapy offers promise and have begun to demonstrate clinical benefit for cancer, Parkinson, hemophilia and a range of orphan and ultra-orphan indications. The number of patients treated successfully and safely continues to grow and the progress thus far is exciting and encouraging.
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.The number of gene therapy programs in clinical development continues to expand driven by rising proof of concept and recent evidence of success in Spinal Muscular Atrophy, Hemophilia A, beta thalassemia and inherited retinal disease.
After the year 2015, the gene therapy has become the most lucrative part of the pharmaceutical industry specifically in cancer segment. Majority of the gene therapies are getting researched and expected to get launched in rare indications. The increasing trend has been seen through the increase in number of clinical trials, increase in government funding for Cell and Gene based therapies and favourable environment created by EMA and FDA regulatory bodies to execute the faster approvals process through Breakthrough and Fast Track Designations.
Furthermore, rising partnerships and alliances, and increasing R&D spends in platform technologies by the players across the continuum are some of the factors that would drive the growth of the gene therapy market. Majority of the biopharmaceutical companies are focused on developing new drugs for the treatment of incurable diseases. Recently, Spark Therapeutics received approval of Luxturna, a one-time treatment for children and adults with retinal dystrophy caused due to the mutation of RPE65 gene leading to permanent vision loss.
Gene Transfer Techniques:
Gene therapy determines the gene causing the disease of interest and replacing it through administration of a vector in the form of a viral carrier system. Vector based method is one of the most preferred mode of transfer and is expected to experience healthy growth in the future. In this method, the foreign DNA is transferred with the help of vectors like adenoviral vectors, adeno-associated virus vectors (AAV), retro viral vectors, and plasmid DNA. Plasmid DNA is the most common type of transfer vector used while AAV mode of transfer is now gaining strong attention in the recent years. The use of AAV is by far the safest method of gene transfer, which is a versatile vector that can be engineered for very specific functionality with wide range applications in various disease setting.
Gene therapy offers transformative benefits and allows for a wide range of genetic and non-genetic diseases to be treated. Gene therapy in oncology and rare blood disorders offers strong commercial prospects. The growth in the treatment of oncology is majorly driven by the rising prevalence of cancer patients globally and favorable regulatory environment. However, high cost of treatment and potential reimbursement hurdles can stifle growth of the gene therapy market through the forecast period.
Gene Therapy represents the leading edge of Biotechnology’s promise to change disease paradigms. North America holds major share for gene therapy market driven by large number of ongoing clinical trials, market presence of leading private and public companies and strong investor interests in the field due to favorable risk/benefit opportunities.
Europe holds the second largest market share for gene therapy. European Medicines Agency (EMA) recently granted marketing authorization for Glybera in the treatment for lipoprotein lipase deficiency (LPLD). The U.K. is also witnessing strong growth in the gene therapy market supported by rising number of clinical trial in the last five years. Asia Pacific market is not expected to show signs of strength in new product and commercial development through the forecast period.