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판코니 빈혈 : 파이프라인 리뷰

Fanconi Anemia (Hematology) - Drugs in Development, 2021

리서치사 Global Markets Direct
발행일 2021년 09월 상품 코드 361627
페이지 정보 영문 93 Pages
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판코니 빈혈 : 파이프라인 리뷰 Fanconi Anemia (Hematology) - Drugs in Development, 2021
발행일 : 2021년 09월 페이지 정보 : 영문 93 Pages

본 상품은 영문 자료로 한글과 영문목차에 불일치하는 내용이 있을 경우 영문을 우선합니다. 정확한 검토를 위해 영문목차를 참고해주시기 바랍니다.

판코니 빈혈(FA)은 골수 부전으로 연결되는 유전성 질환입니다. FA는 재생 불량성 빈혈(무형성성 빈혈)이며, 골수는 세 종류의 혈액세포(혈구)를 만들지 않거나 충분히 만들지 못하게 됩니다. 세 종류의 혈구수가 감소함으로써 체내 각 기관과 조직이 손상을 입습니다. 치료는 헤모글로빈 8g/dL 미만, 혈소판 500/μL 미만과 같은 중증 혈구 감소증의 경우에 추천됩니다.

판코니 빈혈(Fanconi Anemia) 치료제 개발 상황에 대해 조사 분석했으며, 파이프라인 제품 개요, 임상시험 단계별 제품 개요, 주요 기업 개요, 약제 프로파일, 파이프라인 제품의 최신 동향, 최신 뉴스와 프레스 릴리스 등에 대해 전해드립니다.

서론

  • 조사 범위

판코니 빈혈 개요

치료제 개발

  • 파이프라인 제품 개요
  • 기업별 파이프라인
  • 대학/연구기관에서 개발된 파이프라인
  • 기업에서 개발중인 제품
  • 대학/연구기관에서 개발중인 제품

치료제 평가

  • 표적별
  • 작용기전별
  • 투여 경로별
  • 분자 종류별

판코니 빈혈 치료제 개발 참여 기업

  • Abeona Therapeutics, Inc.
  • Genethon

약제 프로파일

휴지 상태인 프로젝트

제품 개발 마일스톤

  • 주요 뉴스와 프레스 릴리스

부록

도표

LSH 17.07.31

List of Tables

List of Tables

  • Number of Products under Development for Fanconi Anemia, 2021
  • Number of Products under Development by Companies, 2021
  • Number of Products under Development by Universities/Institutes, 2021
  • Products under Development by Companies, 2021
  • Products under Development by Universities/Institutes, 2021
  • Number of Products by Stage and Target, 2021
  • Number of Products by Stage and Mechanism of Action, 2021
  • Number of Products by Stage and Route of Administration, 2021
  • Number of Products by Stage and Molecule Type, 2021
  • Fanconi Anemia - Pipeline by Bellicum Pharmaceuticals Inc, 2021
  • Fanconi Anemia - Pipeline by Boehringer Ingelheim International GmbH, 2021
  • Fanconi Anemia - Pipeline by Elixirgen Therapeutics Inc, 2021
  • Fanconi Anemia - Pipeline by Foresee Pharmaceuticals Co Ltd, 2021
  • Fanconi Anemia - Pipeline by Novartis AG, 2021
  • Fanconi Anemia - Pipeline by Rimedion Inc, 2021
  • Fanconi Anemia - Pipeline by Rocket Pharmaceuticals Inc, 2021
  • Fanconi Anemia - Dormant Projects, 2021

List of Figures

List of Figures

  • Number of Products under Development for Fanconi Anemia, 2021
  • Number of Products under Development by Companies, 2021
  • Number of Products under Development by Universities/Institutes, 2021
  • Number of Products by Top 10 Targets, 2021
  • Number of Products by Stage and Top 10 Targets, 2021
  • Number of Products by Top 10 Mechanism of Actions, 2021
  • Number of Products by Stage and Top 10 Mechanism of Actions, 2021
  • Number of Products by Routes of Administration, 2021
  • Number of Products by Stage and Routes of Administration, 2021
  • Number of Products by Molecule Types, 2021
  • Number of Products by Stage and Molecule Types, 2021

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fanconi Anemia - Drugs In Development, 2021, provides an overview of the Fanconi Anemia (Hematological Disorders) pipeline landscape.Fanconi anemia (fan-KO-nee uh-NEE-me-uh), or FA, is a rare, inherited blood disorder that leads to bone marrow failure. The disorder also is called Fanconi's anemia. FA is a type of aplastic anemia. In aplastic anemia, the bone marrow stops making or doesn't make enough of all three types of blood cells. Low levels of the three types of blood cells can harm many of the body's organs, tissues, and systems. Treatment is recommended for significant cytopenias, such as hemoglobin less than 8 g/dL, platelets fewer than 500/μL.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fanconi Anemia - Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Fanconi Anemia (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.The Fanconi Anemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fanconi Anemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 4, 1, 2 and 2 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 3 and 2 molecules, respectively.

Fanconi Anemia (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope:

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Fanconi Anemia (Hematological Disorders).
  • The pipeline guide reviews pipeline therapeutics for Fanconi Anemia (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Fanconi Anemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Fanconi Anemia (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Fanconi Anemia (Hematological Disorders)

Reasons to Buy:

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Fanconi Anemia (Hematological Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Fanconi Anemia (Hematological Disorders) pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

Table of Contents

List of Tables

List of Figures

  • Introduction
  • Global Markets Direct Report Coverage
  • Fanconi Anemia - Overview
  • Fanconi Anemia - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
  • Fanconi Anemia - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Fanconi Anemia - Companies Involved in Therapeutics Development
  • Bellicum Pharmaceuticals Inc
  • Boehringer Ingelheim International GmbH
  • Elixirgen Therapeutics Inc
  • Foresee Pharmaceuticals Co Ltd
  • Novartis AG
  • Rimedion Inc
  • Rocket Pharmaceuticals Inc
  • Fanconi Anemia - Drug Profiles
  • afatinib dimaleate - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • eltrombopag olamine - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • EXG-34217 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Fancalen - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • FP-045 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapies to Activate FANCC and FANCG for Fanconi Anemia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy 1 for Fanconi Anemia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate FANCA for Fanconi Anemia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Gene Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • JP-4039 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • mozafancogene autotemcel - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • rivogenlecleucel - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Small Molecules for Fanconi Anemia - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Fanconi Anemia - Dormant Projects
  • Fanconi Anemia - Product Development Milestones
  • Featured News & Press Releases
  • May 13, 2021: Rocket Pharmaceuticals presents positive clinical data of mozafancogene autotemcel at 24th Annual Meeting of the American Society of Gene and Cell Therapy
  • Dec 07, 2020: Rocket Pharmaceuticals presents positive clinical data from its Fanconi Anemia Program at the 62nd American Society of Hematology annual meeting
  • May 12, 2020: Rocket Pharmaceuticals presents positive updates on RP-L102 program at the 23rd annual meeting of the American Society of Gene and Cell Therapy
  • Feb 24, 2020: Rocket Pharmaceuticals announces publication of manuscript evaluating mosaicism in Fanconi anemia
  • Dec 16, 2019: Rocket Pharmaceuticals receives the European Medicines Agency PRIME eligibility for RP-L102 gene therapy for Fanconi anemia
  • Dec 08, 2019: Rocket Pharmaceuticals presents promising preliminary results from phase 1 trial of commercial-grade RP-L102 "Process B" for Fanconi Anemia at 61st American Society of Hematology Annual Meeting
  • Dec 06, 2019: Rocket pharmaceuticals announces first patient treated in global registrational phase 2 study of RP-L102 "process B" for Fanconi anemia
  • Nov 06, 2019: Rocket Pharmaceuticals to present preliminary phase 1 data of RP-L102 "Process B" for Fanconi Anemia at the 61st American Society of Hematology Annual Meeting
  • Oct 24, 2019: Rocket Pharmaceuticals presents first evidence of long-term improvement and stabilization in blood counts and durable mosaicism in RP-L102 "Process A" for Fanconi Anemia
  • Oct 15, 2019: Rocket Pharmaceuticals announces upcoming presentation on Gene Therapy 1 for Fanconi Anemia at the European Society of Gene and Cell Therapy Annual Congress
  • Sep 10, 2019: Rocket Pharmaceuticals announces publication of data from phase 1/2 trial of first-generation RP-L102 for Fanconi Anemia in Nature Medicine
  • Sep 04, 2019: Rocket Pharmaceuticals announces registration-enabling phase 2 plans for RP-L102 gene therapy for Fanconi anemia following a supportive end-of-phase 1 FDA meeting
  • Aug 22, 2019: Rocket Pharmaceuticals announces clearance from the Spanish Agency for Medicines and Health Products for the phase 2 registration-enabling FANCOLEN-II study of RP-L102 for Fanconi Anemia
  • Apr 15, 2019: Rocket Pharmaceuticals announces upcoming presentations at the American Society of Gene and Cell Therapy 2019 Annual Meeting
  • Mar 12, 2019: Rocket Pharmaceuticals announces first patient dosed in phase 1 clinical trial of RP-L102 in the U.S.
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact Us
  • Disclaimer
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