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表紙：ファンコニー貧血の世界市場-2023年～2030年
市場調査レポート
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1352115

ファンコニー貧血の世界市場-2023年～2030年

Global Fanconi Anemia Market - 2023-2030

出版日
ページ情報
英文 186 Pages
納期
即日から翌営業日
カスタマイズ可能
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=148.90円
ファンコニー貧血の世界市場-2023年～2030年
出版日: 2023年09月27日
発行: DataM Intelligence
ページ情報: 英文 186 Pages
納期: 即日から翌営業日
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  • 概要
  • 目次
概要

概要

ファンコニー貧血はまれな遺伝性疾患であり、典型的には比較的若い年齢で血球産生不全が小児に現れ、骨髄不全とがん傾向を特徴とします。健康なドナーからの造血幹細胞移植は、他の緩和的治療に加えて、現在これらの患者にとって望ましい治療法です。

この種の移植が最近大きく進歩したとはいえ、すべての患者に適切なドナーがいるわけではありません。さらに、この治療法には移植前の化学療法が必要であり、拒絶反応の問題やその他の長期的な副作用も生じる可能性があります。

ダイナミクス

積極的な主要企業

2022年5月、スタンフォード大学医学部のCenter for Definitive and Curative Medicine（CDCM）は、造血細胞移植療法に特化したバイオテクノロジー企業であるJasper Therapeutics, Inc.のスポンサーシップのもと、JPS191をファンコニー貧血治療のコンディショニング剤として使用する研究を実施しています。同社は、この試験の最初の患者の投与を発表しました。

スタンフォード大学医学部では、骨髄不全を有し、非同胞ドナーからの同種移植を必要とするファンコニー貧血患者を対象に、JSP191を用いた第1/2相臨床試験（NCT04784052）を受託研究契約に基づき実施しています。

研究活動の活発化

造血幹細胞移植の生存率は通常80％以上であるにもかかわらず、この治療法には100日死亡率や悪性腫瘍のリスク上昇といった重大な副作用が伴います。調査チームは、ファンコニー貧血A群CD34+濃縮造血幹細胞および前駆細胞を用いた遺伝子治療を試験しています。現在、ロケット・ファーマシューティカルズが創製した薬剤RP-L102の第1/2相臨床試験が進行中です。

RP-L102の表現型補正、生着、臨床反応、安全性を評価するため、2022年10月のデータカットオフ時点で、HLA適合の兄弟ドナーを持たない1歳以上の患者12人を治療し、モニターしています。コンディショニングを行わないことで、骨髄コロニー形成細胞（BM CFC MMC）抵抗性の持続的な増加、遺伝的同時修正、血液学的安定化が可能となり、研究者らは少なくとも1年間の追跡調査を行った患者10人のうち少なくとも6人に認めました。

治療オプションの増加

血液および骨髄幹細胞移植：骨髄不全のFA患者は通常、治療の一環として血液および骨髄幹細胞移植を受けます。これはFAの血液関連合併症を回復させる唯一の治療法です。輸血：これは治療法ではありませんが、支持療法とはなります。

アンドロゲン療法：合成男性ホルモンを使用することで、赤血球や血小板の数を増やし、幹細胞の増殖を改善します。血液および骨髄幹細胞移植：骨髄不全のFA患者は通常、治療の一環として血液および骨髄幹細胞移植を受けます。これはFAの血液関連合併症を回復させる唯一の治療法です。

技術の進歩

FA患者における血液学的機能の回復とMDS/AML形成の予防のために、技術進歩により、治癒の可能性のある新しいDNA指向性遺伝子治療が生み出されつつあります。FAの原因となる遺伝子変異は、遺伝子治療を用いて修正することが意図されています。調査チームは、変異遺伝子の機能的コピーを患者の細胞に挿入することによって、血液細胞の前駆体である造血幹細胞（HSC）の正常な作動を回復させようとしています。

さらに自家幹細胞移植では、患者自身の骨髄や血液の幹細胞を採取し、研究室で遺伝子の誤りを修正し、修正した細胞を患者に移植します。その結果、健康な血液細胞が作られ、MDS/AMLの増殖が止まる可能性があります。

希少疾患の課題

希少疾患はその希少性から、医療界で十分に理解されていないことがあります。医療関係者は特定の疾患の正確な症状や特徴に精通していない可能性があり、その結果、診断が遅れたり、誤った診断が下されたりする可能性があります。これらの疾患の正確な診断は、その希少性ゆえに難しいかもしれません。正しい診断を得るまでに、患者は多くの医師の診察を受け、長い診断の旅に出ることになるかもしれません。

このような診断の遅れにより、患者にとって大きな損失となる可能性があります。希少疾患の希少性ゆえに、大規模な臨床研究を実施し、治療法の有効性を証明するために必要な情報を得ることは困難です。そのため、新規の治療法が規制当局の承認を得ることが難しくなる可能性があります。

医薬品開発の課題

希少疾患の研究は様々な機関や国に分散しているため、データが断片化し、連携が不十分です。研究の改善や治療法の創出のためには、データや知見の共有が不可欠です。研究や治療開発のための資金が不十分なのは、希少疾患に対する一般市民の認識や擁護が低いためかもしれません。このことが、これらの病気の根本的な原因を解明し、新しい治療法を生み出す努力を妨げているのかもしれません。

遺伝子治療や幹細胞治療を含め、徹底的な調査や新しい治療法の開発には長い時間がかかります。患者の命を救う可能性のある治療法へのアクセスは、開発の遅れによって影響を受けるかもしれません。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

  • 影響要因
    • 促進要因
      • 活発な主要企業
      • 研究活動の活発化
      • 治療オプションの増加
      • 技術進歩
    • 抑制要因
      • 希少疾患の課題
      • 医薬品開発の課題
    • 機会
    • 影響分析

第5章 産業分析

  • ポーターのファイブフォース分析
  • サプライチェーン分析
  • 価格分析
  • 規制分析
  • ロシア・ウクライナ戦争の影響分析
  • DMIの見解

第6章 COVID-19分析

第7章 薬剤タイプ別

  • 造血成長因子
  • 遺伝子治療
  • アンドロゲン療法
  • その他

第8章 年齢層別

  • 小児
  • 成人

第9章 エンドユーザー別

  • クリニック
  • 病院
  • その他

第10章 地域別

  • 北米
    • 米国
    • カナダ
    • メキシコ
  • 欧州
    • ドイツ
    • 英国
    • フランス
    • イタリア
    • スペイン
    • その他欧州
  • 南米
    • ブラジル
    • アルゼンチン
    • その他南米
  • アジア太平洋
    • 中国
    • インド
    • 日本
    • オーストラリア
    • その他アジア太平洋
  • 中東・アフリカ

第11章 競合情勢

  • 競合シナリオ
  • 市況/シェア分析
  • M&A分析

第12章 企業プロファイル

  • Rocket Pharmaceuticals
    • 企業概要
    • 製品ポートフォリオと説明
    • 財務概要
    • 主な動向
  • Forty-Seven, Inc.
  • Jasper Therapeutics, Inc.
  • Genethon
  • Abeona Therapeutics Inc.
  • Others

第13章 付録

目次
Product Code: PH6872

Overview

Fanconi anemia is a rare hereditary disorder that typically manifests in children with inadequate blood cell production at relatively young ages and is characterized by bone marrow failure and cancer propensity. Hematopoietic stem cell transplantation from a healthy donor, in addition to other palliative treatments, is currently the preferred course of action for these patients.

Even though this kind of transplant has undergone significant advancements recently, not all patients have a suitable donor. Additionally, pre-transplant chemotherapy is necessary for this procedure, and rejection issues as well as other long-term side effects are possible.

Dynamics

Active Major Players

In May 2022, the Center for Definitive and Curative Medicine (CDCM) at the Stanford University School of Medicine is conducting research using JPS191 as a conditioning agent to treat Fanconi anemia under the sponsorship of Jasper Therapeutics, Inc., a biotechnology company that specializes in hematopoietic cell transplant therapies. The company announced the dosing of the first patient in this study.

Stanford Medicine is conducting a Phase 1/2 clinical trial (NCT04784052) using JSP191 to treat patients with Fanconi anemia who have bone marrow failure and need an allogeneic transplant from a non-sibling donor in compliance with the sponsored research agreement.

Increasing Research Activities

Despite the fact that hematopoietic stem cell transplant normally has a survival rate of over 80%, the therapy is linked to substantial side effects, such as 100-day mortality and an elevated risk of malignancies. Researchers are testing a gene treatment that uses autologous Fanconi anemia Group A CD34 + enriched hematopoietic stem and progenitor cells that engraft without the need for antecedent conditioning to address this. Phase 1/2 clinical studies for the Rocket Pharmaceuticals-created medication, RP-L102, are now underway.

To evaluate phenotypic correction, engraftment, clinical response, and safety of RP-L102, 12 patients at least 1 year of age with no HLA-matched sibling donor have been treated and monitored as of the data cutoff in October 2022. The absence of conditioning allowed for the sustained increase in bone marrow colony-forming cell (BM CFC MMC) resistance, concurrent genetic correction, and hematologic stabilization that the researchers saw in at least six out of ten patients with at least a year of follow-up.

Increasing Treatment Options

Blood and bone marrow stem cell transplant: FA patients with bone marrow failure typically have blood and bone marrow stem cell transplants as part of their treatment. It is the sole therapy that could reverse FA's blood-related complications. Transfusion of blood: While not a curative procedure, blood transfusions can offer supportive care.

Androgen therapy: The use of synthetic male hormones occasionally increasing the number of red blood cells and platelets while improving stem cell growth, Trusted Source is supportive rather than curative. Blood and bone marrow stem cell transplant: FA patients with bone marrow failure typically have blood and bone marrow stem cell transplants as part of their treatment. It is the sole therapy that could reverse FA's blood-related complications.

Technological advances

For the restoration of hematological function and the prevention of MDS/AML formation in FA patients, new, potentially curative DNA-directed gene treatments are being created thanks to technological advancements. The genetic mutations that cause FA are intended to be fixed using gene therapy. Researchers seek to restore the proper operation of hematopoietic stem cells (HSCs), which are the precursors of blood cells, by inserting a functional copy of the mutant gene into the patient's cells.

In addititon, autologous stem cell transplantation, the patient's own bone marrow or blood stem cells are taken, the genetic error is fixed in a lab, and the corrected cells are then transplanted back into the patient. This might result in the creation of healthy blood cells and stop the growth of MDS/AML.

Rare Disease Challenges

Due to their rarity, rare diseases are sometimes poorly understood by the medical community. Healthcare personnel might not be familiar with the precise symptoms and traits of certain illnesses, which might result in delayed or incorrect diagnoses. Accurate diagnosis of these disorders might be difficult due to their rarity. Before acquiring a correct diagnosis, patients may go through a protracted diagnostic odyssey that involves seeing numerous doctors.

The results for patients may suffer significantly as a result of this delay. Due to the rarity of rare diseases, it is challenging to conduct extensive clinical studies and obtain the necessary information to prove a treatment's effectiveness. This may make it difficult for novel therapies to receive regulatory approval.

Drug Development Challenges

Rare disease research may be spread out over various institutions and nations, resulting in fragmented data and insufficient collaboration. For the purpose of improving research and creating therapies, sharing data and findings can be essential. Inadequate funding for research and treatment development may be the result of low public awareness of and advocacy for uncommon diseases. This may impede efforts to uncover the underlying causes of these illnesses and create novel treatments.

It can take a long time to conduct thorough research and create new therapeutics, including gene therapies and stem cell therapies. Access to potentially life-saving therapies for patients may be impacted by development delays.

Segment Analysis

The global fanconi anemia market is segmented based on drug type, age group, end-user and region.

Hematopoietic Growth Factors Accounted for Dominant Market share during the Forecast period

In the bone marrow, hematopoietic growth factors (HGFs) are chemicals that promote the development of blood cells. To support and improve the development of particular blood cell types, they are frequently employed in medical practice. Hematopoietic growth factors can be used as part of the overall care plan for Fanconi Anemia (FA), a rare hereditary condition that compromises bone marrow function.

A hematopoietic growth factor called erythropoietin (EPO) promotes erythropoiesis or the creation of red blood cells. Anemia can occur in patients with Fanconi Anemia because red blood cell synthesis is compromised. To treat this anemia and enhance the patient's general level of energy and quality of life, EPO therapy may be recommended.

Geographical Penetration

Increasing Research Activities in North America

North America has been a dominant force in the global fanconi anemia market. Fanconi anemia is an extremely rare disease. About 31 children are born with FA per year in the United States, according to the Fanconi Anemia Research Fund. The incidence rate, or the likelihood of a child being born with FA, is about 1 in 136,000 births.

Researchers estimate that the incidence of people carrying an abnormality in an FA gene in the United States is 1 in 181. Research, information exchange, and the creation of viable Fanconi Anemia remedies have all been made possible through partnerships between academic institutions, healthcare facilities, pharmaceutical firms, and patient advocacy organizations.

Technologies and advances in gene therapy have shown promise in the treatment of genetic diseases like Fanconi anemia. Gene therapy research and development are concentrated in North America, the region which may have an impact on FA treatment options.

COVID-19 Impact Analysis

The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global fanconi anemia market. Patients with fanconi anemia frequently have impaired immunological function and dysfunctional bone marrow. They may be more susceptible as a result to serious respiratory infections like COVID-19. Therefore, strong attention to preventive measures, such as social isolation, mask use, and proper hand cleanliness, is essential to lower the chance of viral exposure.

FA patients must undergo routine checkups and medical appointments in order to maintain good health. However, there may have been delays in healthcare services during the pandemic, including as canceled appointments, fewer in-person visits, and a switch to telemedicine consultations. To ensure continuing care and necessary modifications to treatment plans, patients and their carers should stay in touch with their healthcare providers.

By Drug Type

  • Hematopoietic Growth Factors
  • Gene Therapy
  • Androgen Therapy
  • Others

By Age Group

  • Pediatric
  • Adults

By End-users

  • Clinics
  • Hospitals
  • Others

By Region

  • North America
    • U.S.
    • Canada
    • Mexico
  • Europe
    • Germany
    • UK
    • France
    • Italy
    • Spain
    • Rest of Europe
  • South America
    • Brazil
    • Argentina
    • Rest of South America
  • Asia-Pacific
    • China
    • India
    • Japan
    • Australia
    • Rest of Asia-Pacific
  • Middle East and Africa

Competitive Landscape

The major global players in the market include: Rocket Pharmaceuticals, Forty-Seven, Inc., Jasper Therapeutics, Inc., Genethon, Abeona Therapeutics Inc., and others.

Why Purchase the Report?

  • To visualize the global fanconi anemia market segmentation based on drug type, age group, end-user and region as well as understand key commercial assets and players.
  • Identify commercial opportunities by analyzing trends and co-development.
  • Excel data sheet with numerous data points of fanconi anemia market-level with all segments.
  • PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
  • Product mapping available as excel consisting of key products of all the major players.

The global fanconi anemia market report would provide approximately 61 tables, 58 figures and 186 Pages.

Target Audience 2023

  • Manufacturers/ Buyers
  • Industry Investors/Investment Bankers
  • Research Professionals
  • Emerging Companies

Table of Contents

1. Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

  • 3.1. Snippet by Drug Type
  • 3.2. Snippet by Age Group
  • 3.3. Snippet by End-user
  • 3.4. Snippet by Region

4. Dynamics

  • 4.1. Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Active Major Players
      • 4.1.1.2. Increasing Research Activities
      • 4.1.1.3. Increasing Treatment Options
      • 4.1.1.4. Technological Advances
    • 4.1.2. Restraints
      • 4.1.2.1. Rare Disease Challenges
      • 4.1.2.2. Drug Development Challenges
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Industry Analysis

  • 5.1. Porter's Five Force Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Pricing Analysis
  • 5.4. Regulatory Analysis
  • 5.5. Russia-Ukraine War Impact Analysis
  • 5.6. DMI Opinion

6. COVID-19 Analysis

  • 6.1. Analysis of COVID-19
    • 6.1.1. Scenario Before COVID
    • 6.1.2. Scenario During COVID
    • 6.1.3. Scenario Post COVID
  • 6.2. Pricing Dynamics Amid COVID-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During Pandemic
  • 6.5. Manufacturers Strategic Initiatives
  • 6.6. Conclusion

7. By Drug Type

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
    • 7.1.2. Market Attractiveness Index, By Drug Type
  • 7.2. Hematopoietic Growth Factors*
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.3. Gene Therapy
  • 7.4. Androgen Therapy
  • 7.5. Others

8. By Age Group

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 8.1.2. Market Attractiveness Index, By Age Group
  • 8.2. Pediatric*
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.3. Adults

9. By End-user

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-user
    • 9.1.2. Market Attractiveness Index, By End-user
  • 9.2. Clinics*
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 9.3. Hospitals
  • 9.4. Others

10. By Region

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
    • 10.1.2. Market Attractiveness Index, By Region
  • 10.2. North America
    • 10.2.1. Introduction
    • 10.2.2. Key Region-Specific Dynamics
    • 10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
    • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-user
    • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 10.2.6.1. U.S.
      • 10.2.6.2. Canada
      • 10.2.6.3. Mexico
  • 10.3. Europe
    • 10.3.1. Introduction
    • 10.3.2. Key Region-Specific Dynamics
    • 10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
    • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-user
    • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 10.3.6.1. Germany
      • 10.3.6.2. UK
      • 10.3.6.3. France
      • 10.3.6.4. Italy
      • 10.3.6.5. Spain
      • 10.3.6.6. Rest of Europe
  • 10.4. South America
    • 10.4.1. Introduction
    • 10.4.2. Key Region-Specific Dynamics
    • 10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
    • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-user
    • 10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 10.4.6.1. Brazil
      • 10.4.6.2. Argentina
      • 10.4.6.3. Rest of South America
  • 10.5. Asia-Pacific
    • 10.5.1. Introduction
    • 10.5.2. Key Region-Specific Dynamics
    • 10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
    • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-user
    • 10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 10.5.6.1. China
      • 10.5.6.2. India
      • 10.5.6.3. Japan
      • 10.5.6.4. Australia
      • 10.5.6.5. Rest of Asia-Pacific
  • 10.6. Middle East and Africa
    • 10.6.1. Introduction
    • 10.6.2. Key Region-Specific Dynamics
    • 10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
    • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-user

11. Competitive Landscape

  • 11.1. Competitive Scenario
  • 11.2. Market Positioning/Share Analysis
  • 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

  • 12.1. Rocket Pharmaceuticals*
    • 12.1.1. Company Overview
    • 12.1.2. Product Portfolio and Description
    • 12.1.3. Financial Overview
    • 12.1.4. Key Developments
  • 12.2. Forty-Seven, Inc.
  • 12.3. Jasper Therapeutics, Inc.
  • 12.4. Genethon
  • 12.5. Abeona Therapeutics Inc.
  • 12.6. Others

LIST NOT EXHAUSTIVE

13. Appendix

  • 13.1. About Us and Services
  • 13.2. Contact Us