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The ongoing evolution in the field of genomics and
biotechnology, has catalyzed innovations and advancements of gene therapies
across a wide spectrum of disorders. The global gene
therapy market is estimated to grow from $2.4 billion in 2024 to $16.5
billion by 2035, representing a CAGR of 19.25% during the forecast period till
2035. Genetic aberration / mutation is well known to cause several
systemic and localized (organ system specific) clinical conditions resulting in
severe and hard-to-treat inherited disorders. Such diseases are often treated
by modifying the abnormal gene / allele responsible for coding a dysfunctional
/ non-functional protein. This has essentially led to the foundation of novel
class of therapeutics known as gene therapies. According to the USFDA, gene
therapy is defined as a the method introducing genetic material in order to
modify or manipulate the expression of either disease-causing or suppressor
genes. Till date, a variety of
gene-focused corrective strategies have been developed for the treatment of a
wide range of genetic conditions, such as adenosine deaminase severe combined
immunodeficiency (ADA-SCID), β-thalassemia, biallelic RPE65 mutation-associated
retinal dystrophy, critical limb ischemia, head and neck squamous cell
carcinoma, melanoma, peripheral artery disease and spinal muscular atrophy. It is worth mentioning that as of now, around 20 gene
therapies have received approval; examples of recently USFDA approved gene
therapies include (arranged in alphabetical order) Casgevy™ (developed by Vertex Pharmaceuticals,
approved in 2024), Lenmeldy™ (developed by Orchard Therapeutics, approved in
2024), Lyfgenia™ (developed by bluebird bio, approved in 2023), Elevidys®
(developed by Sarepta Therapeutics, approved in 2023), Roctavian® (developed by
BioMarin Pharmaceutical, approved in 2023) and Vyjuvek® (developed by Krystal
Biotech, approved in 2023). Considering that such therapies are designed to
address the root cause (at the genetic level) of a disease, a number of
contemporary drug / therapy development initiatives are focused on gene
correction and genome editing-based therapeutic interventions. Further, the
growing need for treatment options that can cure a clinical condition, as
opposed to those intended for treatment of disease-related symptoms, has
created a demand for more effective gene manipulation approaches. Types of Gene Therapies Gene augmentation therapy: In this type of therapy, a
DNA segment representing the functional copy of a gene is inserted into cells
(or tissues) that either lack or have dysfunctional copies of the same gene.
Such an alteration usually results in restoration of normal function of the
target cell / cell population. Gene inhibition therapy: In this type of therapy, a
DNA segment that is capable of either inhibiting the expression of a gene, or
interfering with the activity of another gene, is introduced into a target cell
/ cell population. Gene replacement therapy: In this type of therapy, a
defective gene within a cell is replaced with a therapeutic gene by using an
appropriate delivery vehicle (vector). The Future of Gene Therapy Market In recent years, gene therapy has been demonstrated to be a
promising solution for patients suffering from severe inherited disorders.
These therapies have shown remarkable outcomes in enhancing the life expectancy
of patients suffering from severe conditions, such as AIDS, cancer, cystic
fibrosis and diabetes. Further, with the rise in the R&D activity in this
domain along with advancements in gene editing technologies, an increasing
number of novel gene therapies are likely to be commercialized in the coming
years. It is worth highlighting that, according to Robert M. Califf, the former
commissioner of the USFDA, more than ten gene therapies are anticipated to
receive approval annually, by 2025. Thank you for reading our report. Kindly get in touch with
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team will ensure the report is tailored according to your needs. To view more details on this report, click on the link https://www.rootsanalysis.com/reports/gene-therapies-market/268.html
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