Gene Therapies For Rare Diseases Market Report 2025 (Global Edition)

Global Gene Therapies For Rare Diseases Market Report 2025 Market Size Split by Drug (Approved Drugs, Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), ...

Gene Therapies For Rare Diseases Market Report Description

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Impact of Tariff Trumph on Pharmaceuticals Industry

The reintroduction of tariffs under President Trump’s “Liberation Day Tariffs” in April 2025 has created significant disruption in global pharmaceutical trade. These tariffs, ranging from 10% to 46%, specifically target pharmaceutical products, APIs (active pharmaceutical ingredients), medical packaging, and machinery from key manufacturers in China and India. This has led to increased production costs, supply chain delays, and regulatory challenges for U.S.-based pharmaceutical companies.

The tariffs have particularly impacted generic drug manufacturers, who rely heavily on APIs from China and India. Biopharmaceuticals and vaccines are also feeling the pressure, with tariffs on critical materials like bioproduction media and cold-chain packaging raising costs by up to 25%. Similarly, contract manufacturing organizations (CMOs) are grappling with the need to identify new suppliers and adjust production plans to mitigate rising costs.

Foreign governments, including the European Union, have retaliated with tariffs on U.S. pharmaceutical exports, further complicating global trade. Clinical trials are also impacted, as tariffs on drugs and diagnostic kits delay trial timelines.

Market research is increasingly crucial for navigating these challenges. By providing real-time data on API pricing, supplier reliability, and tariff impact, pharmaceutical firms can make informed decisions on sourcing, compliance, and investment strategies. This data-driven approach helps mitigate risks, optimize supply chains, and identify alternative manufacturing hubs to offset tariff-related disruptions.

Overall, market research is essential for pharmaceutical companies to adapt to the new tariff landscape, enabling them to maintain competitive advantage and navigate global trade dynamics effectively.

Gene Therapies For Rare Diseases Market Insights Video

Competitive Landscape of the Gene Therapies For Rare Diseases Market

The competitive landscape of the Gene Therapies For Rare Diseases market is characterized by a diverse range of players competing to gain market share through product innovation, strategic partnerships, and aggressive marketing efforts. Key players in the market include established electronics manufacturers, specialized Gene Therapies For Rare Diseases companies, and emerging start-ups aiming to disrupt the market with innovative solutions.

Top Companies Market Share in Gene Therapies For Rare Diseases Industry: (In no particular order of Rank)

• Novartis AG
• Pfizer Inc.
• Roche Holding AG
• Sanofi SA
• Johnson & Johnson
• AbbVie Inc.
• Vertex Pharmaceuticals Incorporated
• Amgen Inc.
• AstraZeneca PLC
• Bristol Myers Squibb Company
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Regional Analysis of the Gene Therapies For Rare Diseases Market

Which Region Dominates the Gene Therapies For Rare Diseases Market in 2024?

According to Cognitive Market Research, North America dominated the market in 2024. North America is a leading location within the boom of gene treatments for uncommon sicknesses, driven by using robust research infrastructure, superior biotechnology talents, and supportive regulatory frameworks. Key elements encompass huge investments from biotech companies, educational establishments, and authorities investment companies. The place's strong healthcare system and patient advocacy also contribute to accelerating the improvement and adoption of those progressive remedies.

Which Region is Expanding at the Fastest CAGR?

Europe stands out as the fastest-growing region in the Gene Therapies For Rare Diseases market due to several compelling reasons. ?Europe is witnessing a huge increase in gene treatment options for rare diseases, buoyed by collaborative research efforts, supportive regulatory environments just like the European Medicines Agency (EMA), and sturdy healthcare infrastructure. Biotech corporations and academic establishments across Europe are actively advancing gene remedy innovations, supported by way of investment initiatives and affected person advocacy companies that endorse stepping forward to get admission to these transformative treatments.

 

The current report Scope analyzes Gene Therapies For Rare Diseases Market on 5 major region Split (In case you wish to acquire a specific region edition (more granular data) or any country Edition data then please write us on info@cognitivemarketresearch.com

• North America (United States, Canada, Mexico)
• Europe (United Kingdom, France, Germany, Italy, Russia, Spain, Sweden, Denmark, Switzerland, Luxembourg, Rest of Europe)
• Asia Pacific (China, Japan, South Korea, India, Australia, Singapore, Taiwan, South East Asia, Rest of APAC)
• Latin America (Brazil, Argentina, Colombia, Peru, Chile, Rest of South America)
• Middle East (Saudi Arabia, Turkey, UAE, Egypt, Qatar, Rest of Middle East)
• Africa (Nigeria, South Africa, Rest of Africa)

The above graph is for illustrative purposes only.

North America Gene Therapies For Rare Diseases Market Analysis

According to Cognitive Market Research, the global Gene Therapies For Rare Diseases market size was estimated at USD 25.6 Million, out of which North America held the major market of more than 40% of the global revenue with a market size of USD 10.24 million in 2024 and will grow at a compound annual growth rate (CAGR) of 25.00% from 2024 to 2031. North America leads in advancing gene therapies for neurological disorders, leveraging its strong research infrastructure, biotechnology expertise, and supportive regulatory environment. The region hosts numerous clinical trials and research initiatives focused on addressing conditions like ALS, Parkinson's disease, and rare genetic neuropathies. Biotech firms and academic institutions collaborate closely to develop innovative treatments, supported by substantial investments and patient advocacy. With a robust pipeline of promising therapies in development, North America stands at the forefront of revolutionizing treatment options for these challenging neurological conditions, aiming to improve patient outcomes and quality of life.

Europe Gene Therapies For Rare Diseases Market Analysis

According to Cognitive Market Research, the global Gene Therapies For Rare Diseases market size was estimated at USD 25.6 Million out of which Europe held the market of more than 30% of the global revenue with a market size of USD 7.68 million in 2024 and will grow at a compound annual growth rate (CAGR) of 25.3% from 2024 to 2031. Europe is a key hub for advancing gene therapies targeting neurological disorders, benefiting from collaborative research efforts, well-established biotech infrastructure, and supportive regulatory frameworks such as the European Medicines Agency (EMA). Clinical trials and research initiatives across the continent focus on innovative treatments for conditions like multiple sclerosis, Alzheimer's disease, and rare genetic neurodegenerative disorders. With strong academic-industry partnerships and proactive patient advocacy, Europe is poised to lead in developing transformative therapies that address unmet medical needs in neurological care, aiming to enhance treatment options and outcomes for patients across the region.

Asia Pacific Gene Therapies For Rare Diseases Market Analysis

According to Cognitive Market Research, the global Gene Therapies For Rare Diseases market size was estimated at USD 25.6 Million, out of which Asia Pacific held the market of around 23% of the global revenue with a market size of USD 5.89 million in 2024 and will grow at a compound annual growth rate (CAGR) of 28.8% from 2024 to 2031. Asia Pacific is increasingly becoming a pivotal region for advancing gene therapies targeting neurological disorders, driven by growing healthcare investments, expanding biotech capabilities, and a rising prevalence of these conditions. Clinical research and trials focus on disorders such as stroke, epilepsy, and rare genetic neurodegenerative diseases. The region's diverse patient populations and collaborative research networks contribute to a dynamic landscape for innovation in neurological care. With supportive regulatory frameworks evolving and increasing industry participation, Asia Pacific is poised to play a significant role in developing and commercializing transformative gene therapies for neurological disorders, improving patient outcomes regionally and globally.

South America Gene Therapies For Rare Diseases Market Analysis

According to Cognitive Market Research, the global Gene Therapies For Rare Diseases market size was estimated at USD 25.6 Million out of which Latin America market of more than 5% of the global revenue with a market size of USD 1.28 million in 2024 and will grow at a compound annual growth rate (CAGR) of 26.2% from 2024 to 2031. Latin America is emerging as a promising region for advancing gene therapies targeting neurological disorders, bolstered by increasing healthcare investments and a growing emphasis on biotech innovation. Clinical research and trials are focusing on conditions such as cerebral palsy, neurogenetic disorders, and rare neurological conditions prevalent in the region. Challenges include access to advanced therapies and infrastructure limitations. However, with supportive regulatory developments and collaborative efforts between local researchers and global biotech firms, Latin America is poised to expand its role in developing effective gene therapies that address the specific healthcare needs of its diverse population, improving neurological care outcomes.

Middle East Gene Therapies For Rare Diseases Market Analysis

According to Cognitive Market Research, the global Gene Therapies For Rare Diseases market size was estimated at USD 25.6 Million, out of which the Middle East and Africa held the major market of around 2% of the global revenue with a market size of USD 0.51 million in 2024 and will grow at a compound annual growth rate (CAGR) of 26.5% from 2024 to 2031. The Middle East and Africa (MEA) region is beginning to explore gene therapies for neurological disorders, spurred by increasing healthcare investments and partnerships with global biotech firms. Clinical research and trials are focusing on conditions such as epilepsy, neurodegenerative diseases, and rare genetic disorders prevalent in the region. Challenges include infrastructure limitations and access to advanced therapies. Despite these hurdles, ongoing regulatory advancements and collaborations between local healthcare providers and international researchers are paving the way for MEA to emerge as a significant player in advancing gene therapies, aiming to address critical unmet medical needs in neurological care.

Gene Therapies For Rare Diseases Market Analysis

Gene Therapies For Rare Diseases Market Drug Segment Analysis

According to Cognitive Market Research, Approved Drugs stands out as the dominating category. The variety of accepted gene remedies for rare illnesses is growing steadily, reflecting improvements in biotechnology and regulatory approvals. These treatment plans provide promising results with the aid of addressing underlying genetic causes rather than signs and symptoms. Pharmaceutical corporations are expanding their pipelines to encompass gene remedy treatments, responding to rising demand and demonstrating the potential for healing procedures in formerly untreatable uncommon diseases.

Pipeline Drugs emerges as the fastest-growing category in the Gene Therapies For Rare Diseases market. The pipeline for gene therapies targeting uncommon illnesses is increasing swiftly, with several promising candidates in diverse ranges of clinical improvement. Biotech firms are that specialize in novel techniques to address genetic issues, leveraging improvements in CRISPR-Cas9, viral vectors, and gene editing technology. This robust pipeline reflects ongoing funding and innovation aimed toward meeting the pressing clinical wishes of sufferers with uncommon illnesses.

The above Chart is for representative purposes and does not depict actual sale statistics. Access/Request the quantitative data to understand the trends and dominating segment of Gene Therapies For Rare Diseases Industry. Request a Free Sample PDF!

Gene Therapies For Rare Diseases Market Therapeutic Application Segment Analysis

According to Cognitive Market Research, the dominating category is Oncology. Oncology is rising as a massive region for gene treatment plans targeting rare sicknesses, mainly in addressing uncommon kinds of cancer with particular genetic mutations. These treatment options intend to supply centered remedies that may potentially cure or substantially enhance effects for sufferers with rare oncological conditions. Advances in precision medicine and gene-modifying technology are riding innovation in this swiftly evolving discipline.

The fastest-growing category in the Gene Therapies For Rare Diseases market is Neurological Disorders. Neurological disorders are increasingly turning into a focal point for gene cures targeting rare diseases, aiming to address conditions like Huntington's ailment, spinal muscular atrophy (SMA), and certain forms of inherited neuropathies. These remedies goal to correct genetic defects or provide neuroprotective outcomes, potentially presenting transformative remedies where traditional treatment plans are limited. Advancements in gene modifying and transport technologies are riding development in this promising vicinity.

The above Graph is for representation purposes only. This chart does not depict actual Market share.

Author's Detail

Manjiri Kanhere

Senior Research Associate at Cognitive Market Research

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Manjiri Kanhere is an experienced market researcher focused on the Pharma & Healthcare industry. With over 3+ years of experience, She has worked with major pharmaceutical companies and healthcare providers, helping them to understand market trends, identify new business opportunities, and develop effective sales & marketing strategies. Her expertise lies in the ability to analyze complex data sets and deliver insights that drive business decisions. At Cognitive Market Research, she handles the market research related to Pharma and healthcare industry.

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