Gene Therapy: The Breakthrough Treatment Approach Poised to Disrupt Multibillion Dollar Existing Markets

New York City, 14 Jan 2019: Gene therapy continues to advance with several programs demonstrating proof of concept and well positioned to be competitive products. Current clinical development of gene therapy is predominantly centered on hereditary diseases, for which repeated drug administration could be burdensome for patients.

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The approval of Luxturna, developed by Spark Therapeutics for inherited retinal disease is a watershed event facing this space. Luxturna gained the U.S. FDA approval in December 2017 and is priced at a whopping USD 850,000. Gene therapy market in late 2017 also witnessed the approvals of Gilead/Kite Pharma’s Yescarta and Novartis’ Kymriah in the cancer therapeutic area.

Currently Approved Gene Therapy Products

        Source - Polaris Market Research

In addition, the commercialization of BMN270, hemophilia A treatment, in Phase III clinical trials, developed by BioMarin and Lenti-D by bluebird bio, gene therapy for childhood cerebral adrenoleukodystrophy (CCALD), in Phase III clinical trials, are gaining visibilities, implying that gene therapy will be used to treat a wider range of diseases. After some minor setbacks, bluebird’s LentiGlobin appears poised to deliver strong clinical results in beta-Thalassemia and potentially for sickle cell patients as well. Renova Therapeutics’ lead product candidate RT-100 entered Phase III trials early this year for congestive heart failure, the first gene therapy product that has advanced to late-stage trials in a cardiovascular indication. The FDA has fast tracked RT-100 AC6 gene transfer therapy for treatment of patients with heart failure with reduced ejection fraction (HFrEF). RT-100 fulfills significant clinical unmet need in the CHF patient population while serving an addressable market opportunity of over USD 3 billion.

There are a number of frontier companies emerging as important players in the field. AveXis’ AVXS-101 for Spinal Muscular Atrophy (SMA) is the highest-visibility gene therapy program, and continues to rapidly advance with potential for early approval. Similarly, Gene therapy for Parkinson’s by Voyager Therapeutics is demonstrating clinical benefit in a 5-patient mid-dose cohort and Hemophilia by UniQure and Spark Therapeutics continues to be an area of focus for the field.

Setting aside some setbacks and disappointments, the number of patients treated successfully and safely with gene therapy continues to grow and the consistent clinical evidence points to a robust platform with utility in both Orphan and ultra-Orphan diseases and broader indications.

Large Pharmaceuticals and Biotechnology companies’ exhibit strong interest in this field and notable among them include Allergan, Shire, Biomarin, Pfizer and GSK. In particular, GSK has a meaningful presence in gene therapy with the approved Strimvelis for ADASCID in Europe and a deep pipeline of programs. Moreover, this space is abuzz with a wave of partnerships and alliances. Pfizer has recently expanded its presence in gene therapy with the acquisition of Bamboo Therapeutics and Allergan entered the field, with the acquisition of RetroSense and its Phase I/II optogenetic program.

Gene Therapy- Late-Stage Pipeline (Select Companies)

    Source - Polaris Market Research

Gene Therapy represents the leading edge of Biotechnology’s promise to change disease paradigms. Although gene therapy space is still in its infancy, the field continues to offer promise. About $12bn investment is riding on a growing universe of small cap public gene therapy companies. The evidence of success in Spinal Muscular Atrophy, Hemophilia A & B, sickle cell disease & beta thalassemia and a rare genetic cause of blindness have ignited investor interest in the field and expanded the number of clinical and preclinical development programs all targeting a large addressable market and serving strong unmet need.

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