The choice of a particular AAV to use as a gene transfervector is heavily reliant on several critically important criteria:
(1) Which cell/tissue types are being targeted?
(2) The safety profile associated with the delivered gene;
(3) The choice of systemic versus local delivery; and
(4) The use of tissue-specific or constitutively active promoters.
There are currently two classes of recombinant AAVs (rAAVs)in use: single-stranded AAV (ssAAV) and self-complementary AAV (scAAV). rAAVgene therapy strategies include Gene replacement, Gene silencing, Gene additionand Gene editing.
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The first AAV-based gene therapy drug, Glybera, was approvedby the European Medicines Agency (EMA) in 2012 but later in 2017, it waswithdrawn from the market mainly due to commercial failure. Only two AAV-basedgene therapies are currently FDA-approved, Luxturna was approved in 2017 for arare inherited retinal dystrophy, and Zolgensma approved in 2019 for spinal muscular atrophy.
Adeno-Associated Virus Vectors in Gene Therapy Market is expected to grow with asignificant high rate and mainly driven by increase in approval of growingnumber of gene therapies and readily adoption on approval, ability to treatbroad array of conditions, increase in number of cases, expected one time dosingapproach and curative treatment options.
Development of Adeno-Associated Virus (AAV) vector basedgene therapy across the various therapeutic areas are analyzed and 11indications have been taken forward for the forecast. In the perspective of theunmet medical needs, majority of rAAV gene therapy programs are centered aroundthe liver, striated muscles and the CNS. Essentially all characteristic of AAV capsids can transduce liver productively following systemic administration.
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Hence, rAAVs offers a superior liver-targeting platform totreat a variety of diseases such as hemophilia A and hemophilia B, familialhypercholesterolemia, and other. Capsids, for example, AAV8 and AAV9 can targetmultiple muscle types throughout the body, enabling rAAV gene therapies to be developedfor multiple muscle diseases, especially those afflicting muscles of the entire body, such as Duchenne muscular dystrophy (DMD).
