The gene therapy for rare disease market involves the development and application of treatments that modify or manipulate a person's genes to treat rare diseases. Gene therapy aims to correct defective genes that are responsible for many rare genetic disorders by adding a normal copy of the gene to replace or supplement the function of the mutated gene. Gene therapies are showing great promise for treating various rare diseases like hemophilia, muscular dystrophy, and retinal degeneration for which conventional therapies are ineffective. The global gene therapy market for rare diseases is gaining traction due to increasing government funding for research and development activities as well as rising healthcare expenditure across the globe.
The Global Gene Therapy For Rare Disease Market is estimated to be valued at Us$ 222.99 Mn in 2024 and is expected to exhibit a CAGR Of 5.9% over the forecast period 2024 to 2030.
Key Takeaways
Key players operating in the Gene Therapy For Rare Disease are Topcon Corporation, Agfa-Gevaert N.V., Sonomed Escalon, Carl Zeiss Meditec AG, EyePACS, VersaSuite, Heidelberg Engineering GmbH, ScImage, Inc., IBM Corporation, and Visbion Limited. The increasing incidence of rare genetic disorders and lack of effective treatment options are propelling the demand for gene therapy. Technological advancements in gene delivery vectors such as AAV and lentivirus are also augmenting the market growth.
The growing demand for gene therapy to treat hemophilia, retinal dystrophies, and neurological disorders is fueling market expansion. Hemophilia A and B are the major targets for gene therapies owing to their high prevalence and potential of gene therapy to provide lifelong cure. Technological advancements in vector engineering, design, and production have increased targeting efficiency and safety of various gene delivery platforms. Advancements such as self-complementary AAV vectors and engineered capsid variants are enhancing transduction efficiency in gene therapies.
Market Trends
Personalized Gene Therapy - Advances in genetic sequencing and gene editing technologies are enabling development of personalized gene therapies tailored to individual patient's genetic makeup. This trend allows development of precise and safe treatments.
Innovation in Delivery Vectors - Innovation in delivery vectors such as engineered AAV vectors with improved tissue targeting and ability to evade immune response is a key trend. Development of non-viral delivery vectors is also gaining interest.
Market Opportunities
Neurological Rare Diseases - Gene therapies for neurological rare diseases like spinal muscular atrophy (SMA) and Batten disease remain major opportunities owing to high unmet needs.
Gene Therapy Combinations - Combination of gene therapy with other modalities like small molecule medications offers opportunities by delivering augmentation effects or overcoming resistance.
Impact of COVID-19 on Gene Therapy for Rare Disease Market Growth
The COVID-19 pandemic impacted the growth of the gene therapy market for rare diseases. The lockdowns imposed affected research activities and clinical trials. Several clinical trials were either halted or delayed to focus healthcare resources on fighting the pandemic. Recruitment of new patients for trials was also slowed down. However, the remote working capabilities allowed some research activities to continue albeit at a slower pace. Telemedicine solutions helped clinical monitoring of ongoing trials to an extent. The pandemic also highlighted the need for advanced therapies like gene therapy for rare diseases which otherwise do not have effective treatment options. Governments and private organizations increased funding for COVID-19 as well as other disease research which would benefit the gene therapy market in the longer run. With vaccines rollout and policies for safe resumption of activities, the market is expected to bounce back as new drug approvals and clinical trial activities recover lost time in 2021 and beyond. Additional funding and focus on rare disease treatment post pandemic may give a boost to market growth as well.
Geographical Concentration of Gene Therapy for Rare Disease Market
North America accounts for the largest share of the global gene therapy market for rare diseases in terms of value, followed by Europe. This is due to the presence of major pharmaceutical players, widespread medical insurance coverage and reimbursement policies favorable to advanced therapies in countries like the US. Increased funding from both public and private sources towards gene therapy research especially for pediatric rare diseases also drives regional market growth. However, Asia Pacific region is emerging as the fastest growing market as countries like China invest heavily in building local research infrastructure and improving accessibility of high-cost specialty therapies. Growing pharmaceutical industry, rising healthcare spending capacity and initiatives to make diagnostics and treatment available even in remote areas are supporting the regional market expansion.
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Rare Disease Gene Therapy Market: SnapshotThe global rare disease gene therapy is expected to witness a significant growth in the forecast period, 2020-2030 on account of the increasing cases of genetic diseases worldwide.
These kids are expected to experience complex clinical issues in the wake of getting live-sparing bone marrow transfers to treat the condition.Request Brochure – https://www.transparencymarketresearch.com/sample/sample.php?flag=B_id=80404The report offers insights into the market emphasizing on factors boosting, repelling, challenging, and creating opportunities for the market in the forecast period.
In terms of indication, the market is grouped into inflammatory diseases, neurological diseases, infectious diseases, hematology diseases, oncology diseases, genetic diseases, and others.
What's more, clinical-stage improvement organizations are allotting critical assets towards the advancement of the uncommon illness quality treatment market.For example, in August 2020, the U.S. based Ovid Therapeutics teamed up with Italian firm Angelini Pharma to create, make and popularize drug gaboxadol for treatment of Angelman disorder (uncommon hereditary sickness) in European market.Some of the players of the global rare disease gene therapy market include Amgen Inc., AbbVie Inc., F.Hoffmann-La Roche Ltd., Merck & Co., Johnson & Johnson, Baxter, Novo Nordisk A/S, Alexion, Pfizer Inc., Bristol-Myers Squibb Company, Novartis AG, Sanofi S.A., Teva Pharmaceutical Industries Ltd., Bayer AG, Allergen plc., Vertex Pharmaceuticals, and others.Request Customization on Rare Disease Gene Therapy Market Report – https://www.transparencymarketresearch.com/sample/sample.php?flag=CR_id=80404Rare Disease Gene Therapy Market: Interesting InsightsThere has been an intense competition among biopharmaceutical players to create Covid treatment that is postponing on account of innovative work exercises in uncommon infection quality treatment market.
Stop of assembling exercises and flexibly tie disturbance because of exchanging across outskirts will have sway on uncommon illness quality treatment market development rate.
Also, clinical-stage advancement organizations are allotting critical assets towards the improvement of the uncommon infection quality treatment market.Pre Book Rare Disease Gene Therapy Market Report –https://www.transparencymarketresearch.com/checkout.php?rep_id=80404=S Rare Disease Gene Therapy Market: Regional CategorizationNorth America will keep on overwhelming the uncommon illness quality treatment market in the coming decade with a high pace of endorsements and commercialization of genomic medication.
