by The global Gene Therapy For Rare Disease Market is estimated to be valued at US$ 193.56 Bn in 2023 and is expected to exhibit a CAGR of 11.% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Market Overview:
Gene therapy involves modifying or manipulating genes to treat or prevent genetic disorders. Gene therapy aims to introduce a normal gene into cells to compensate for an abnormal gene that is causing disease. This technology has proven successful in treating numerous rare genetic disorders by delivering therapeutic genes into patient’s cells and tissues to compensate for abnormal genes that are causing disease.
Market key trends:
One of the key trends in the gene therapy for rare disease market is the increasing number of clinical trials evaluating diverse disease targets. Gene therapy holds immense potential in treating various monogenic disorders stemming from a single defective gene. Currently, there are over 2,000 rare diseases identified and majority lack any approved treatment options. To address this unmet need, biopharma companies are actively conducting clinical trials evaluating various rare diseases like hemophilia, cystic fibrosis, muscular dystrophy etc. Additionally, several academic research institutes are also evaluating potential gene therapy candidates for a variety of orphan conditions. This rising clinical research focus on rare diseases is expected to drive the market growth during the forecast period.
Market key trends:
With the rise in rare diseases and genetic disorders, gene therapy is emerging as one of the promising treatment options. It aims to treat genetic diseases by delivering working copies of genes into the patient’s cells and tissues to compensate for mutated genes which are responsible for the disease. The gene therapy for rare disease market is witnessing significant growth with the advancement in gene editing technologies like CRISPR, development of novel viral and non-viral gene delivery vectors. Several clinical trials demonstrating safety and efficacy of gene therapies for rare diseases like spinal muscular atrophy, retinal disorders are further fueling the market growth.
SWOT Analysis
Strength: Gene therapy offers long term functional cure by correcting the genetic root cause of rare diseases. It provides a one-time treatment opportunity without the need for repeated medications/interventions.
Weakness: High cost associated with the R&D and manufacturing of new gene therapies. Complex delivery methods and potential side effects are major challenges.
Opportunity: Rapid expansion in clinical research for various rare genetic disorders. Rise in collaboration between pharma companies and research institutes globally.
Threats: Stringent regulatory approval process. Complications related to viral vectors limiting their use. Doubts over the effectiveness and safety of newer gene editing tools.
Key Takeaways
The Global Gene Therapy For Rare Disease Market Size is expected to witness high growth at a CAGR of 11% over the forecast period of 2023 to 2030. The market size for 2023 is estimated to be US$ 193.56 Bn.
Regional analysis: North America dominated the gene therapy for rare disease market in 2022 with the highest number of approved gene therapies and ongoing clinical research. Asia Pacific is expected emerge as the fastest growing regional market over the forecast period owing to improving healthcare infrastructure and increasing healthcare investments in China and India.
Key players operating in the Gene Therapy For Rare Disease market include Amgen Inc., Biomarin Pharmaceuticals, Bayer AG. Gene therapies for inherited retinal dystrophies from Spark Therapeutics and Novartis, spinal muscular atrophy from AveXis acquired by Novartis, hemophilia from uniQure are some of key commercial gene therapies.Multiple other players are also engaged in developing therapies for rare neuromuscular, immunological and metabolic disorders which are anticipated to enter the market in coming years.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
