DelveInsight’s, “Hemophilia Pipeline Insight 2023” report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in the Hemophilia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Key takeaways from Hemophilia Pipeline Report
DelveInsight’s Hemophilia Pipeline Report depicts a robust space with 80+ active players working to develop 80+ pipeline therapies for Hemophilia.
Leading hemophilia companies such as Intellia tx, Amarna therapeutics, Expression Therapeutics, GC Pharma, Chameleon Biosciences, Pfizer, UBI Pharma, GeneVentiv, Chia Tai Tianqing Pharmaceutical Group, Bayer, ASC Therapeutics, Catalyst Biosciences, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, CSL Behring, Sanofi, Novo Nordisk, Centessa Pharmaceuticals, OPKO Health, Freeline Therapeutics, Spark Therapeutics, Novo Nordisk, Asklepios BioPharmaceutical, Belief Biomed, ASC Therapeutics, uniQure, Sanofi, Bioverativ, and others are evaluating novel hemophilia treatment drugs candidate to improve the treatment landscape.
Key hemophilia pipeline therapies in various stages of development include Hemophilia B Research Project, Hemophilia A Research Project, AMA006, Hemophilia ET3 Research Project, Hemophilia ET8 Research Project, Hemophilia ET9 Research Project, MG1113A, Hemophilia EVADER Research Project, PF-06741086, Giroctocogene fitelparvovec, PF06838435, UB-854, GENV-HEM, GENV-001, TQG203, BAY1093884, ASC618, Marzeptacog Alfa, STSP-0601, SPK-8011, Fitusiran, Concizumab, SerpinPC, MOD-5014, FLT180a, SPK-8011, Mim 8, AskBio009, BBM-H901, PF 06838435, ASC618 and Etranacogene dezaparvovec, Efanesoctocog alfa, and others.
The Hemophilia companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia R&D. The Hemophilia therapies under development are focused on novel approaches to treat/improve Hemophilia.
In the Hemophilia pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, Hemophilia NDA approvals (if any), and product development activities comprising the technology, Hemophilia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
To explore more information on the latest breakthroughs in the Hemophilia Pipeline treatment landscape of the report, click here @ Hemophilia Pipeline Outlook
Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. Blood contains many proteins called clotting factors that can help to stop bleeding. People with hemophilia have low levels of either factor VIII (8) or factor IX (9). The severity of hemophilia that a person has is determined by the amount of factor in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems. In rare cases, a person can develop hemophilia later in life. The majority of cases involve middle-aged or elderly people, or young women who have recently given birth or are in the later stages of pregnancy.
Recent Breakthroughs of the Hemophilia Treatment Landscape
In September 2022, Pfizer and Sangamo Therapeutics announced that the Phase III AFFINE study evaluating giroctocogene fitelparvovec, investigational gene therapy for patients with moderately severe to severe hemophilia A, has re-opened recruitment. All trial sites are anticipated to be active by the end of 2022, and a pivotal readout is expected in the first half of 2024.
In June 2022, Freeline Therapeutics Holdings announced that it had begun dosing the second cohort in its Phase I/II B-LIEVE dose confirmation trial of FLT180a in people with hemophilia B.
In May 2022, uniQure announced that the FDA accepted for priority review the Biologics License Application (BLA) submitted for etranacogene dezaparvovec. Priority review of a BLA is reserved for medicines that, if approved, would be potentially significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
In March 2022, The Food and Drug Administration (FDA) granted Fast Track designation to ASC618 for the treatment of hemophilia A.
In January 2022, 2seventy bio announced that it has entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for hemophilia A. This agreement builds upon a successful existing multi-year research collaboration between the two companies.
In December 2021, Belief BioMed Group (BBM) announced that it had successfully dosed the first subject in the registrational gene therapy clinical trial by intravenous (IV) infusion of BBM-H901, an adeno-associated virus (AAV) vector expressing factor IX gene for treatment of adult male hemophilia B patients.
Request a sample and discover the recent advances in Hemophilia Ongoing Clinical Trial Analysis and Medications, click here @ Hemophilia Treatment Landscape
Hemophilia Emerging Drugs Profile
SerpinPC: Centessa Pharmaceuticals
SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.
Fitusiran: Alnylam Pharmaceuticals
Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs) by their collaborators at Sanofi Genzyme. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Hemophilia.
ASC 618: ASC Therapeutics
ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant; in preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells. ASC Therapeutics will conduct a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program received IND clearance from the U.S. Food and Drug Administration.
Hemophilia Pipeline Therapeutics Assessment
There are approx. 80+ key companies which are developing the therapies for Hemophilia. The companies which have their Hemophilia drug candidates in the most advanced stage, i.e. Phase III include, Alnylam Pharmaceuticals.
DelveInsight’s Hemophilia Pipeline Report covers around 80+ products under different phases of clinical development like
Late stage products (Phase III)
Mid-stage products (Phase II)
Early-stage product (Phase I) along with the details of
Pre-clinical and Discovery stage candidates
Discontinued & Inactive candidates
Scope of the Hemophilia Pipeline Report
Hemophilia Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal
Hemophilia Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule, Gene Therapy
Hemophilia Therapeutics Assessment By Mechanism of Action: Activated protein C receptor modulators, Serine protease inhibitors, Antithrombin III expression inhibitors, Haemostasis stimulants, RNA interference, Factor VIII replacements, Gene transference, Factor IX replacements, Factor X stimulants
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