A型血友病市场 - KOL的考察

Haemophilia A - KOL Insight

出版日期: 2023年05月16日 | 出版商:

FirstWord Group | 英文 | 商品交期: 最快1-2个工作天内

价格

简介目录

本报告提供A型血友病市场相关调查分析，对已上市的治疗方法和开发平台有的治疗方法的KOL的考察。

基因治疗
- 重要考虑因素摘要
- ASC618（CRISPR/cas9 编辑的 hFVIII 基因疗法；ASC Therapeutics）
- Peboctocogene camaparvovec（DTX201；拜耳医疗保健/Dimension Therapeutics）
- Giroctocogene fitelparvovec（PF-07055480/SB-525；Sangamo Therapeutics/辉瑞）
- Dirloctocogene samoparvovec (SPK-8011) 和 SPK-8016 (Spark Therapeutics)
- TAK-754（SHP654；武田）
- Roctavian（valoctocogene roxaparvovec；BioMarin Pharmaceutical）
抗组织因子途径抑制剂单株抗体
- 重要考虑因素摘要
- Concizumab（抗 TFPI 单株抗体；诺和诺德）
- Marstacimab（抗 TFPI mAb；辉瑞）
FVIII 模拟抗体
- 重要考虑因素摘要
- Mim8（NNC0365-3769 A；诺和诺德）
抗凝血? III 抑制剂/RNA 干扰
- 重要考虑因素摘要
- Fitusiran（Alnylam 製药公司/赛诺菲）
活化蛋白 C 受体调节剂
- 重要考虑因素摘要
- SerpinPC（Centessa 製药公司）
其他早期项目
- 重要考虑因素摘要
- BT200（RNA 调节剂/vWF 抑制剂；Band Therapeutics）
- IDO 8（耐受性树突细胞疗法；Idogen）

附录

新闻

简介目录

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

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)