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市場調査レポート
商品コード
1355370
血友病A市場 - KOLの考察Haemophilia A - KOL Insight |
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価格
| 血友病A市場 - KOLの考察 |
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出版日: 2023年05月16日
発行: FirstWord Group
ページ情報: 英文
納期: 即日から翌営業日
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- 全表示
- 概要
- 目次
概要
当レポートでは、血友病A市場について調査分析し、上市済みの治療法とパイプラインにある治療法に対するKOLの考察を提供しています。
目次
エグゼクティブサマリー
調査目的
上市済みの治療法
- 血液凝固因子刺激剤
- 重要な考察のまとめ
- Hemlibra (emicizumab; Roche)
- 組換え第VIII因子療法
- 新しい長時間作用型組換え第VIII因子療法
- Altuviiio (efanesoctocog alfa; Amunix/Bioverativ [Sanofi])
パイプライン製品
非因子補充療法
- 遺伝子治療
- 重要な考察のまとめ
- ASC618 (CRISPR/cas9-edited hFVIII gene therapy; ASC Therapeutics)
- Peboctocogene camaparvovec (DTX201; Bayer HealthCare/Dimension Therapeutics)
- Giroctocogene fitelparvovec (PF-07055480/SB-525; Sangamo Therapeutics/Pfizer)
- Dirloctocogene samoparvovec (SPK-8011) and SPK-8016 (Spark Therapeutics)
- TAK-754 (SHP654; Takeda)
- Roctavian (valoctocogene roxaparvovec; BioMarin Pharmaceutical)
- 抗組織因子経路インヒビターモノクローナル抗体
- 重要な考察のまとめ
- Concizumab (anti-TFPI mAb; Novo Nordisk)
- Marstacimab (anti-TFPI mAb; Pfizer)
- FVIII模倣抗体
- 重要な考察のまとめ
- Mim8 (NNC0365-3769 A; Novo Nordisk)
- アンチトロンビンIIIインヒビター/RNA干渉
- 重要な考察のまとめ
- Fitusiran (Alnylam Pharmaceuticals/Sanofi)
- 活性化プロテインC受容体モジュレーター
- 重要な考察のまとめ
- SerpinPC (Centessa Pharmaceuticals)
- その他の初期段階のプログラム
- 重要な考察のまとめ
- BT200 (RNA modulator/vWF inhibitor; Band Therapeutics)
- IDO 8 (tolerogenic dendritic cell therapy; Idogen)
付録
ニュース
目次
Altuviiio? Despite gaining European approval, experts are uncertain about the prospects for BioMarin's Roctavian - why? Could Novo Nordisk's next-generation FVIII mimetic pipeline antibody Mim8 be superior to Roche's SoC Hemlibra? KOLs critically assess the prospects of launched and pipeline therapies.
In addition to the full report, licensed users have access to the Report Highlights slide deck and the following KOL Bulletins via the Attachments area.
- 16 Jun 23 KOL Bulletin -- Expert opinion on the Phase III BASIS data for Pfizer's marstacimab
- 16 Jun 23 KOL Bulletin -- Expert perspective on Phase III ATLAS-PPX data for Alnylam and Sanofi's fitusiran
- 11 Jul 23 KOL Bulletin -- Leading haematologist opines on US approval of BioMarin's haemophilia A gene therapy Roctavian
Table of Contents
Executive summary
- Current and future treatment algorithm
Research objectives
Marketed therapies
- Blood coagulation factor stimulant
- Key insights summary
- Hemlibra (emicizumab; Roche)
- Recombinant Factor VIII therapies
- Newer long-acting recombinant Factor VIII therapies
- Altuviiio (efanesoctocog alfa; Amunix/Bioverativ [Sanofi])
Pipeline products
Non-factor replacement therapies
- Gene therapy
- Key insights summary
- ASC618 (CRISPR/cas9-edited hFVIII gene therapy; ASC Therapeutics)
- Peboctocogene camaparvovec (DTX201; Bayer HealthCare/Dimension Therapeutics)
- Giroctocogene fitelparvovec (PF-07055480/SB-525; Sangamo Therapeutics/Pfizer)
- Dirloctocogene samoparvovec (SPK-8011) and SPK-8016 (Spark Therapeutics)
- TAK-754 (SHP654; Takeda)
- Roctavian (valoctocogene roxaparvovec; BioMarin Pharmaceutical)
- Anti-tissue factor pathway inhibitor mAbs
- Key insights summary
- Concizumab (anti-TFPI mAb; Novo Nordisk)
- Marstacimab (anti-TFPI mAb; Pfizer)
- FVIII mimetic antibody
- Key insights summary
- Mim8 (NNC0365-3769 A; Novo Nordisk)
- Antithrombin III inhibitors/RNA interference
- Key insights summary
- Fitusiran (Alnylam Pharmaceuticals/Sanofi)
- Activated protein C receptor modulator
- Key insights summary
- SerpinPC (Centessa Pharmaceuticals)
- Other early-stage programmes
- Key insights summary
- BT200 (RNA modulator/vWF inhibitor; Band Therapeutics)
- IDO 8 (tolerogenic dendritic cell therapy; Idogen)
Appendix
- KOL details
- KOLs from the USA
- KOLs from Europe
