Picture
SEARCH
What are you looking for?
Need help finding what you are looking for? Contact Us
Compare
Free Samples
In Vitro Diagnostics

PUBLISHER: Global Industry Analysts, Inc. | PRODUCT CODE: 1799184

Cover Image

PUBLISHER: Global Industry Analysts, Inc. | PRODUCT CODE: 1799184

Rare Genetic Disorders Treatment

PUBLISHED:
PAGES: 170 Pages
DELIVERY TIME: 1-2 business days
REQUEST FREE SAMPLE
REQUEST CUSTOMIZATION
SELECT AN OPTION
PDF & Excel (Single User License)
USD 5850
PDF & Excel (Global License to Company and its Fully-owned Subsidiaries)
USD 17550

Add to Cart

Description

Table of Contents

Global Rare Genetic Disorders Treatment Market to Reach US$305.2 Billion by 2030

The global market for Rare Genetic Disorders Treatment estimated at US$155.3 Billion in the year 2024, is expected to reach US$305.2 Billion by 2030, growing at a CAGR of 11.9% over the analysis period 2024-2030. Biologics & Biosimilars, one of the segments analyzed in the report, is expected to record a 10.2% CAGR and reach US$171.6 Billion by the end of the analysis period. Growth in the Non-Biologics segment is estimated at 14.4% CAGR over the analysis period.

The U.S. Market is Estimated at US$40.8 Billion While China is Forecast to Grow at 11.3% CAGR

The Rare Genetic Disorders Treatment market in the U.S. is estimated at US$40.8 Billion in the year 2024. China, the world's second largest economy, is forecast to reach a projected market size of US$47.8 Billion by the year 2030 trailing a CAGR of 11.3% over the analysis period 2024-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 10.7% and 10.4% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 8.9% CAGR.

Global Rare Genetic Disorders Treatment Market - Key Trends & Drivers Summarized

Why Are Rare Genetic Disorders Finally Receiving Market Attention?

Rare genetic disorders-once neglected due to small patient populations and limited commercial incentives-are now at the forefront of biopharmaceutical innovation. Characterized by complex pathologies and often lacking effective treatments, these disorders collectively affect over 300 million people worldwide, despite each condition being individually rare. Advances in genomics, CRISPR-Cas9 gene editing, and high-throughput drug screening have created unprecedented opportunities to design targeted therapies, shifting the commercial paradigm in favor of rare disease investment.

Pharmaceutical companies are increasingly drawn to this market by orphan drug designations and incentives such as market exclusivity, tax credits, and accelerated regulatory pathways offered by agencies like the U.S. FDA, EMA, and Japan’s PMDA. These incentives have stimulated significant activity in the development of therapies for conditions such as Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), Fabry disease, Gaucher disease, and Rett syndrome. Breakthrough approvals like Zolgensma (gene therapy for SMA) and Evrysdi (SMN2 splicing modulator) exemplify how targeted innovation can address previously untreatable conditions.

_How Are Novel Modalities and Precision Approaches Transforming Treatment?_

The treatment landscape for rare genetic disorders is being reshaped by gene therapy, antisense oligonucleotides (ASOs), and enzyme replacement therapies (ERTs). Gene therapies-such as AAV-mediated delivery systems-offer one-time, potentially curative interventions for monogenic disorders. These therapies are expanding into broader indications like hemophilia A/B and metabolic syndromes, supported by scalable manufacturing platforms and deeper mechanistic understanding of genetic expression.

ASOs, such as those used in treating SMA and Batten disease, provide personalized treatments tailored to individual mutations. These agents modify splicing or gene transcription to restore deficient protein production, offering hope in conditions where traditional small molecules have failed. Meanwhile, ERTs continue to dominate in lysosomal storage disorders, with newer formulations exhibiting improved biodistribution and reduced immunogenicity.

Another notable advancement is the use of patient-derived induced pluripotent stem cells (iPSCs) and organoid models for preclinical drug testing. These enable personalized medicine approaches that align with the mutation-specific etiology of many rare diseases. Clinical trial design is also being revolutionized through basket trials, n-of-1 studies, and natural history data integration-tailored to the small, dispersed nature of rare disease populations.

Which Therapeutic Areas and Stakeholders Are Leading the Push?

Neurological and metabolic genetic disorders represent the highest concentration of innovation and investment. Conditions such as Huntington’s disease, SMA, and Leber congenital amaurosis are targets for gene therapy pipelines, while ERTs continue to dominate the treatment of Gaucher, Pompe, and Fabry diseases. Pediatric indications are particularly prominent, as early diagnosis and intervention are crucial for reducing disease burden and improving quality of life.

Biopharmaceutical companies specializing in rare diseases-such as BioMarin, Sarepta, Ultragenyx, and Spark Therapeutics-are leading clinical and commercial progress. These firms are often supported by nonprofit foundations, academic research networks, and patient advocacy groups that help drive trial recruitment and regulatory engagement. Global patient registries and rare disease consortia are increasingly being used to standardize data collection, validate endpoints, and justify accelerated approvals.

Geographically, North America and Europe remain dominant in drug development and regulatory activity, thanks to mature healthcare systems, funding ecosystems, and favorable policy frameworks. However, Asia-Pacific is seeing rapid momentum, particularly in gene editing research and biologics manufacturing capabilities, positioning it as a future growth frontier for rare genetic therapies.

What Is Driving Growth in the Rare Genetic Disorders Treatment Market?

The growth in the rare genetic disorders treatment market is driven by the convergence of scientific innovation, regulatory incentives, and increasing diagnosis rates enabled by genomic testing. As whole-genome sequencing becomes more accessible and affordable, previously undiagnosed or misdiagnosed conditions are being accurately classified, expanding the eligible treatment population. This diagnostic clarity is critical for precision medicine approaches like ASOs and gene therapy.

Regulatory frameworks such as the Orphan Drug Act (USA), PRIME (EU), and Sakigake designation (Japan) are accelerating time-to-market for novel therapies. These policies reduce development risk, attract investment, and foster innovation through fast-track designations, smaller trial requirements, and longer exclusivity windows. Simultaneously, payer willingness to reimburse high-cost, high-impact treatments-often justified by long-term cost offsets-has strengthened the commercial viability of these therapies.

Collaborative ecosystems involving biotech firms, patient advocacy organizations, and regulatory bodies are ensuring that research is patient-centered and outcome-oriented. As digital health platforms and telemedicine expand, especially in post-approval patient monitoring and real-world evidence generation, the rare genetic disorders treatment market is positioned to evolve from niche to normalized care-redefining the future of personalized medicine.

SCOPE OF STUDY:

The report analyzes the Rare Genetic Disorders Treatment market in terms of units by the following Segments, and Geographic Regions/Countries:

Segments:

Drug Type (Biologics & Biosimilars, Non-Biologics); Distribution Channel (Hospitals Pharmacy, Retail Pharmacy, Online Pharmacy); Application (Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application, Metabolic Disorders Application, Hematology Diseases Application, Other Applications)

Geographic Regions/Countries:

World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; and Rest of Europe); Asia-Pacific; Rest of World.

Select Competitors (Total 34 Featured) -

  • Alexion (AstraZeneca Rare Disease)
  • Amicus Therapeutics
  • Arcturus Therapeutics
  • Beacon Therapeutics
  • BioMarin Pharmaceutical
  • Bluebird Bio
  • CRISPR Therapeutics
  • Genomic startups (e.g., GeneQuine)
  • PTC Therapeutics
  • ProQR Therapeutics
  • Rocket Pharmaceuticals
  • Sarepta Therapeutics
  • Spark Therapeutics
  • Translate Bio (Sanofi)
  • Tune Therapeutics
  • Ultragenyx Pharmaceutical
  • Vertex Pharmaceuticals
  • Zygnema/Airna etc. (RNA-editing)
  • Glycomine
  • Calliditas Therapeutics
  • Ipsen

AI INTEGRATIONS

We're transforming market and competitive intelligence with validated expert content and AI tools.

Instead of following the general norm of querying LLMs and Industry-specific SLMs, we built repositories of content curated from domain experts worldwide including video transcripts, blogs, search engines research, and massive amounts of enterprise, product/service, and market data.

TARIFF IMPACT FACTOR

Our new release incorporates impact of tariffs on geographical markets as we predict a shift in competitiveness of companies based on HQ country, manufacturing base, exports and imports (finished goods and OEM). This intricate and multifaceted market reality will impact competitors by increasing the Cost of Goods Sold (COGS), reducing profitability, reconfiguring supply chains, amongst other micro and macro market dynamics.

Show more
Product Code: MCP39262

TABLE OF CONTENTS

I. METHODOLOGY

II. EXECUTIVE SUMMARY

  • 1. MARKET OVERVIEW
    • Influencer Market Insights
    • World Market Trajectories
    • Tariff Impact on Global Supply Chain Patterns
    • Rare Genetic Disorders Treatment - Global Key Competitors Percentage Market Share in 2025 (E)
    • Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2025 (E)
  • 2. FOCUS ON SELECT PLAYERS
  • 3. MARKET TRENDS & DRIVERS
    • Acceleration of Gene and Cell Therapy Approvals Throws the Spotlight On Rare Genetic Disorder Treatments
    • Rising Orphan Drug Designations Drive R&D Investment and Market Exclusivity Opportunities
    • Innovation in mRNA, CRISPR, and AAV-Based Therapies Expands the Genetic Treatment Toolbox
    • Surge in Patient Advocacy and Rare Disease Registries Enhances Disease Awareness and Diagnosis Rates
    • Partnerships With Biotech Startups Strengthen Clinical Pipelines in Niche Genetic Indications
    • Growth in Precision Medicine Initiatives Supports Development of Mutation-Specific Therapies
    • Expansion of Newborn Screening and Genomic Panels Promotes Early Intervention in Rare Conditions
    • Increased Payer Willingness to Reimburse High-Cost Therapies Strengthens Commercial Viability
    • Focus on Single-Dose and Long-Term Cure Modalities Drives Differentiation in Product Profiles
    • Rising Incidence of Ultra-Rare and Undiagnosed Disorders Expands Addressable Patient Subsets
    • Strategies for Companion Diagnostic Integration Improve Treatment Matching and Patient Selection
    • Growth in Global Regulatory Harmonization for Orphan Drugs Speeds Time-to-Market in Emerging Economies
    • Use of Expanded Access and Compassionate Use Programs Builds Clinical Experience and Evidence
    • Increased Investment From Venture Capital and Nonprofit Consortia Boosts Innovation Funding
    • Development of AI Tools for Variant Interpretation and Therapy Matching Enhances Discovery
    • Surge in Academic-Industry Collaborations Supports Translational Research Across Rare Pathologies
    • Participation in Basket and Umbrella Trials Enables Efficient Testing Across Multiple Mutations
    • Rising Role of Cell Banks and Biorepositories Enhances Research Infrastructure and Target Validation
    • Commitment to Lifecycle Management and Post-Market Data Collection Supports Long-Term Safety Monitoring
    • Policy Support for Personalized and Value-Based Reimbursement Models Expands Market Access Pathways
  • 4. GLOBAL MARKET PERSPECTIVE
    • TABLE 1: World Rare Genetic Disorders Treatment Market Analysis of Annual Sales in US$ Million for Years 2014 through 2030
    • TABLE 2: World Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 3: World 6-Year Perspective for Rare Genetic Disorders Treatment by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2025 & 2030
    • TABLE 4: World Recent Past, Current & Future Analysis for Biologics & Biosimilars by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 5: World 6-Year Perspective for Biologics & Biosimilars by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 6: World Recent Past, Current & Future Analysis for Non-Biologics by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 7: World 6-Year Perspective for Non-Biologics by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 8: World Recent Past, Current & Future Analysis for Hematology Diseases Application by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 9: World 6-Year Perspective for Hematology Diseases Application by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 10: World Recent Past, Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 11: World 6-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 12: World Recent Past, Current & Future Analysis for Cancer Application by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 13: World 6-Year Perspective for Cancer Application by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 14: World Recent Past, Current & Future Analysis for Neurological Disorders Application by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 15: World 6-Year Perspective for Neurological Disorders Application by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 16: World Recent Past, Current & Future Analysis for Cardiovascular Diseases Application by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 17: World 6-Year Perspective for Cardiovascular Diseases Application by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 18: World Recent Past, Current & Future Analysis for Metabolic Disorders Application by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 19: World 6-Year Perspective for Metabolic Disorders Application by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 20: World Recent Past, Current & Future Analysis for Hospitals Pharmacy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 21: World 6-Year Perspective for Hospitals Pharmacy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 22: World Recent Past, Current & Future Analysis for Retail Pharmacy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 23: World 6-Year Perspective for Retail Pharmacy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 24: World Recent Past, Current & Future Analysis for Online Pharmacy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 25: World 6-Year Perspective for Online Pharmacy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030

III. MARKET ANALYSIS

  • UNITED STATES
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United States for 2025 (E)
    • TABLE 26: USA Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 27: USA 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 28: USA Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 29: USA 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 30: USA Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 31: USA 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • CANADA
    • TABLE 32: Canada Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 33: Canada 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 34: Canada Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 35: Canada 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 36: Canada Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 37: Canada 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • JAPAN
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Japan for 2025 (E)
    • TABLE 38: Japan Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 39: Japan 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 40: Japan Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 41: Japan 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 42: Japan Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 43: Japan 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • CHINA
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in China for 2025 (E)
    • TABLE 44: China Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 45: China 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 46: China Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 47: China 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 48: China Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 49: China 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • EUROPE
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Europe for 2025 (E)
    • TABLE 50: Europe Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 51: Europe 6-Year Perspective for Rare Genetic Disorders Treatment by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2025 & 2030
    • TABLE 52: Europe Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 53: Europe 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 54: Europe Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 55: Europe 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 56: Europe Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 57: Europe 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • FRANCE
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in France for 2025 (E)
    • TABLE 58: France Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 59: France 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 60: France Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 61: France 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 62: France Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 63: France 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • GERMANY
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Germany for 2025 (E)
    • TABLE 64: Germany Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 65: Germany 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 66: Germany Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 67: Germany 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 68: Germany Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 69: Germany 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • ITALY
    • TABLE 70: Italy Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 71: Italy 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 72: Italy Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 73: Italy 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 74: Italy Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 75: Italy 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • UNITED KINGDOM
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United Kingdom for 2025 (E)
    • TABLE 76: UK Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 77: UK 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 78: UK Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 79: UK 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 80: UK Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 81: UK 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • REST OF EUROPE
    • TABLE 82: Rest of Europe Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 83: Rest of Europe 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 84: Rest of Europe Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 85: Rest of Europe 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 86: Rest of Europe Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 87: Rest of Europe 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • ASIA-PACIFIC
    • Rare Genetic Disorders Treatment Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Asia-Pacific for 2025 (E)
    • TABLE 88: Asia-Pacific Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 89: Asia-Pacific 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 90: Asia-Pacific Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 91: Asia-Pacific 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 92: Asia-Pacific Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 93: Asia-Pacific 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030
  • REST OF WORLD
    • TABLE 94: Rest of World Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Drug Type - Biologics & Biosimilars and Non-Biologics - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 95: Rest of World 6-Year Perspective for Rare Genetic Disorders Treatment by Drug Type - Percentage Breakdown of Value Sales for Biologics & Biosimilars and Non-Biologics for the Years 2025 & 2030
    • TABLE 96: Rest of World Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Application - Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 97: Rest of World 6-Year Perspective for Rare Genetic Disorders Treatment by Application - Percentage Breakdown of Value Sales for Hematology Diseases Application, Other Applications, Cancer Application, Neurological Disorders Application, Cardiovascular Diseases Application and Metabolic Disorders Application for the Years 2025 & 2030
    • TABLE 98: Rest of World Recent Past, Current & Future Analysis for Rare Genetic Disorders Treatment by Distribution Channel - Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 99: Rest of World 6-Year Perspective for Rare Genetic Disorders Treatment by Distribution Channel - Percentage Breakdown of Value Sales for Hospitals Pharmacy, Retail Pharmacy and Online Pharmacy for the Years 2025 & 2030

IV. COMPETITION

Show more
Have a question?
Picture

Jeroen Van Heghe

Manager - EMEA

+32-2-535-7543

Picture

Christine Sirois

Manager - Americas

+1-860-674-8796

Questions? Please give us a call or visit the contact form.
Contact Us +1-866-353-3335
Hi, how can we help?
Contact us!