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まれな遺伝疾患治療の世界市場

Rare Genetic Disorders Treatment

出版日
ページ情報
英文 170 Pages
納期
即日から翌営業日
適宜更新あり
価格
価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=148.34円
まれな遺伝疾患治療の世界市場
出版日: 2025年08月27日
発行: Global Industry Analysts, Inc.
ページ情報: 英文 170 Pages
納期: 即日から翌営業日
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概要

まれな遺伝疾患治療の世界市場は2030年までに3,052億米ドルに達する見込み

2024年に1,553億米ドルと推定されるまれな遺伝疾患治療の世界市場は、2030年には3,052億米ドルに達し、分析期間2024-2030年のCAGRは11.9%で成長すると予測されます。本レポートで分析したセグメントの一つである生物製剤とバイオシミラーは、CAGR10.2%を記録し、分析期間終了時には1,716億米ドルに達すると予測されます。非生物製剤セグメントの成長率は、分析期間中CAGR 14.4%と推定されます。

米国市場は408億米ドルと推定、中国はCAGR 11.3％で成長予測

米国のまれな遺伝疾患治療市場は、2024年に408億米ドルと推定されます。世界第2位の経済大国である中国は、2030年までに478億米ドルの市場規模に達すると予測され、分析期間2024-2030年のCAGRは11.3%です。その他の注目すべき地域別市場としては、日本とカナダがあり、分析期間中のCAGRはそれぞれ10.7%と10.4%と予測されています。欧州では、ドイツがCAGR約8.9%で成長すると予測されています。

世界のまれな遺伝疾患治療市場- 主要動向と促進要因まとめ

希少遺伝性疾患がようやく市場で注目されるようになったのはなぜか？

希少遺伝子疾患は、患者数が少なく、商業的インセンティブが限られているため、かつては軽視されてきたが、現在ではバイオ医薬品イノベーションの最前線にあります。複雑な病態を特徴とし、有効な治療法がないことが多いこれらの疾患は、個々の疾患が希少であるにもかかわらず、合わせて世界中で3億人以上が罹患しています。ゲノミクス、CRISPR-Cas9による遺伝子編集、ハイスループット・ドラッグスクリーニングの進歩は、標的治療をデザインする前例のない機会を生み出し、商業的パラダイムを希少疾患への投資にシフトさせています。

希少疾病用医薬品の指定や、米国FDA、EMA、日本の医薬品医療機器総合機構（PMDA）などが提供する市場独占権、税額控除、規制緩和措置などの優遇措置により、製薬会社はますますこの市場に引き寄せられるようになっています。こうした優遇措置により、デュシェンヌ型筋ジストロフィー（DMD）、脊髄性筋萎縮症（SMA）、ファブリー病、ゴーシェ病、レット症候群などの疾患に対する治療薬の開発が活発に行われています。ゾルゲンスマ（SMAの遺伝子治療）やエブリスディ（SMN2スプライシングモジュレーター）のような画期的な承認は、標的を絞った技術革新が、以前は治療不可能であった疾患にどのように対処できるかを例証するものです。

新しい治療法と精密アプローチはどのように治療を変えるのか？

希少遺伝性疾患の治療法は、遺伝子治療、アンチセンス・オリゴヌクレオチド（ASO）、酵素補充療法（ERT）によって再構築されつつあります。AAVを介したデリバリーシステムなどの遺伝子治療は、単発性疾患に対して1回限りの、治癒の可能性のある介入を提供するものです。これらの治療法は、スケーラブルな製造プラットフォームと遺伝子発現のより深いメカニズム理解に支えられ、血友病A/Bや代謝症候群のような幅広い適応症に拡大しつつあります。

SMAやバッテン病の治療に用いられるようなASOは、個々の変異に合わせた個別化治療を提供します。これらの薬剤は、スプライシングや遺伝子転写を改変し、欠損したタンパク質産生を回復させるもので、従来の低分子が失敗した病態に希望を与えるものです。一方、ライソゾーム貯蔵障害では、ERTが引き続き主流であり、新しい製剤は生体内分布の改善と免疫原性の低下を示しています。

もう一つの注目すべき進歩は、患者由来の人工多能性幹細胞（iPSC）やオルガノイドモデルを前臨床試験に用いることです。これらにより、多くの希少疾患の変異特異的病因に沿った個別化医療アプローチが可能になります。臨床試験デザインもまた、希少疾患集団の小規模で分散した性質に合わせたバスケット試験、n-of-1試験、自然史データの統合によって変革されつつあります。

どのような治療領域と利害関係者が牽引しているのか？

神経疾患と代謝性遺伝性疾患は、技術革新と投資が最も集中している疾患です。ハンチントン病、SMA、リーバー先天性黒内障などの治療が遺伝子治療パイプラインの標的である一方、ゴーシェ病、ポンペ病、ファブリー病の治療ではERTが引き続き主流を占めています。早期診断と早期介入は疾病負担の軽減とQOLの向上に極めて重要であるため、小児への適応は特に顕著です。

バイオマリン社、サレプタ社、ウルトラジェニックス社、スパーク・セラピューティクス社などの希少疾患を専門とするバイオ製薬企業が臨床および商業的進歩を主導しています。これらの企業は、非営利財団、学術研究ネットワーク、患者擁護団体の支援を受けていることが多く、臨床試験の募集や規制当局への働きかけを後押ししています。世界の患者登録や希少疾患コンソーシアムは、データ収集の標準化、エンドポイントの検証、早期承認の正当化にますます利用されるようになっています。

地域的には、成熟したヘルスケアシステム、資金調達エコシステム、有利な政策枠組みにより、北米と欧州が医薬品開発と規制活動において優位を保っています。しかし、アジア太平洋地域は、特に遺伝子編集研究と生物製剤製造能力において急速な勢いを見せており、希少遺伝子治療の将来の成長フロンティアとして位置づけられています。

まれな遺伝疾患治療市場の成長の原動力は？

まれな遺伝疾患治療市場の成長は、科学的イノベーション、規制上のインセンティブ、ゲノム検査によって可能になる診断率の上昇の収束によってもたらされます。全ゲノムシーケンスがより身近で安価になるにつれ、以前は診断されていなかった、あるいは誤診されていた疾患が正確に分類されるようになり、対象となる治療人口が拡大しています。このような診断の明確化は、ASOや遺伝子治療のような精密医療アプローチにとって極めて重要です。

希少疾病用医薬品法（米国）、PRIME（EU）、サキガケ指定（日本）などの規制的枠組みは、新規治療薬の市場投入までの時間を早めています。これらの政策は、開発リスクを軽減し、投資を呼び込み、ファスト・トラック指定、より少ない治験要件、より長い独占期間を通じてイノベーションを促進します。同時に、高コストでインパクトの大きい治療（多くの場合、長期的なコスト・オフセットによって正当化される）に対する支払者の償還意欲は、これらの治療の商業的実行可能性を強化しています。

バイオテクノロジー企業、患者擁護団体、規制機関を含む協力的なエコシステムは、研究が患者中心でアウトカム志向であることを保証しています。デジタル・ヘルス・プラットフォームと遠隔医療が拡大し、特に承認後の患者モニタリングと実臨床でのエビデンス創出が進むにつれて、まれな遺伝疾患治療市場はニッチケアからノーマライズドケアへと進化し、個別化医療の未来を再定義する立場にあります。

セグメント

医薬品タイプ（生物学的製剤＆バイオシミラー、非生物学的製剤）、流通チャネル（病院薬局、小売薬局、オンライン薬局）、アプリケーション（がんアプリケーション、神経疾患アプリケーション、心血管疾患アプリケーション、代謝疾患アプリケーション、血液疾患アプリケーション、その他のアプリケーション）

調査対象企業の例

  • 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インテグレイションズ

Global Industry Analystsは、有効な専門家コンテンツとAIツールによって、市場情報と競合情報を変革しています。

Global Industry Analystsは、一般的なLLMや業界別SLMのクエリに従う代わりに、ビデオ記録、ブログ、検索エンジン調査、大量の企業、製品/サービス、市場データなど、世界中の専門家から収集したコンテンツのリポジトリを構築しました。

関税影響係数

Global Industry Analystsは、本社の国、製造拠点、輸出入（完成品とOEM）に基づく企業の競争力の変化を予測しています。この複雑で多面的な市場力学は、売上原価（COGS）の増加、収益性の低下、サプライチェーンの再構築など、ミクロおよびマクロの市場力学の中でも特に競合他社に影響を与える見込みです。

目次

第1章 調査手法

第2章 エグゼクティブサマリー

  • 市場概要
  • 主要企業
  • 市場動向と促進要因
  • 世界市場の見通し

第3章 市場分析

  • 米国
  • カナダ
  • 日本
  • 中国
  • 欧州
  • フランス
  • ドイツ
  • イタリア
  • 英国
  • その他欧州
  • アジア太平洋
  • その他の地域

第4章 競合

目次
Product Code: MCP39262

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

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TARIFF IMPACT FACTOR

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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