Gene therapy is defined as “the prologue genetic material, either DNA or RNA into cells for therapeutic use.” Conventionally, the aim is to introduce a functional gene to repair or substitute an aberrant mutant gene or to selectively constrain the expression of a gene by knocking out the phrase.
Some vector-based gene therapies use a virus to redeem genetic material directly into a cell to replace a missing or defective gene for treatment of single-gene disorders.
As part of their replication cycle, all viruses introduce genetic material into the host cell by binding to their hosts this is the advantage that gene therapies have.
Glybera (alipogene tiparvovec) of uniQure, is the first gene therapy to receive approval was for lipoprotein lipase (LPL) deficiency.
The drug makes use of a viral vector that introduces the wild-type lipoprotein lipase (LPL)-encoding gene in patients with mutated gene/genes, making the encoded protein to be non-functional.
The FDA recently approved Luxturna, a kind of vector-based gene therapy that is injected directly into the retina of the patient to help reverse vision loss in pediatric and adult patients with a specific type of progressive blindness caused due to a mutated gene (RPE65).
Gene therapies (Vector-based) are also in development for the treatment of other diseases like ocular and immune disorders, hemophilia, cystic fibrosis, certain forms of brain cancer and sickle cell disease.
The marketing approvals of Kymriah, Luxturna, and Yescarta added to this growing class of products. However, due to various restraints, progress in developing this technology and attaining commercial uptake over the past few decades has been lagging.
In short-term, there is a shortage in global gene and cell therapy manufacturing capacity, and in the long-term, the currently available therapies may not be able to meet the centralized biopharmaceutical manufacturing model, this is attributed to personalized nature of many gene therapies.
Also, the minimal number of patients in orphan genetic diseases that these therapies are uniquely well suited for has caused financial challenges.
The high pricing of these therapies versus the low patient number presented strong reimbursement difficulties. However, the pipeline for gene therapies is robust; there are about 985 gene therapies (76%) that are in being tested in animals.
There are about 23 gene therapy programs in Phase III development. Gene therapy pipeline is dominated by gene silencing and gene insertion, which comprise 53% and 36% of the pipe, respectively. Also, aptamers, splice modification therapies, and microRNA-mimicking therapies are the other common gene therapies being developed. Oncology is the active therapy area for gene therapy developments primarily due to its high prevalence and genetically driven pathophysiology.
The overall market is expected to validate significant growth in opportunities for a variety of stakeholders in the coming decade.
Designing and introduction of advanced platforms for the engineering of the vectors are already on the path which is developed and supported by the technology providers who are interested in this platform. The focus of the developers includes the provision of safe and effective gene therapies.
The capability to target diverse therapeutic areas is amongst the most prominent growth drivers of this market. Innovation in this circle has also led to the discovery of new molecular targets and fortified the research pipelines of companies focused on this space.
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