hemophilia A

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The Future is Here: BioMarin’s Roctavian First Hemophilia A Gene Therapy Paving the Way for a Cure

Hemophilia A is a rare genetic bleeding disorder that primarily affects males. Hemophilia A is the most common type of hemophilia, accounting for approximately 80% of all cases. It is estimated to occur in about 1 in 5,000 to 10,000 male births. As per the Centers for Disease Control (CDC) (2020), the estimated pre...

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Pharma News for BioMarin, CellTrans, Genenta, GSK
First Gene Therapy for Severe Hemophilia A; FDA Approves CellTrans’s Type 1 Diabetes Cellular Therapy; FDA Approve Amneal’s Parkinson’s Drug Over Safety Concerns; NLS Pharmaceutics’s Phase III Clinical Trials of Quilience; Genenta Science’s Temferon for the Treatment of Glioma; FDA Fast Track Designation to GSK’s Gonorrhea Drug

FDA Approves First Gene Therapy for Severe Hemophilia A BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the US Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the tr...

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Cell and Gene Therapies in Rare Disorders: From Rarity to Recovery

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

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Hemophilia A Market Forecast and Assessment
Roche’s HEMLIBRA: A Game Changer in Hemophilia A Treatment Landscape

Hemophilia A treatment scenario before the launch of HEMLIBRA Despite the ups and downs throughout history, the management of hemophilia A patients substantially improved over the past 40 years. The groundbreaking discovery of cryoprecipitate in 1964 marked the beginning of the modern progression of hemophilia t...

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Pharma News for Sangamo, Santen, and Eli Lilly
Daiichi Sankyo’s Ezharmia; Pfizer & Sangamo Hemophilia A Gene Therapy Trial; Approval for Fennec’s Pedmark; FDA Approves UBE and Santen’s OMLONTI; EC Approves AstraZeneca’s Tezspire; FDA Approves Selpercatinib; FDA Grants Accelerated Approval to Eli Lilly’s Retevmo; GSK & Spero Announce Exclusive License Agreement

Daiichi Sankyo Receives the First Approval for its Blood Cancer Drug Ezharmia Daiichi Sankyo has received the first global approval for Ezharmia, a first-in-class dual EZH1 and EZH2 inhibitor for the treatment of patients with relapsed or refractory adult T-cell leukemia/lymphoma (ATL). The Japanese Ministry of ...

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AAV Vectors in Gene Therapy: How Recent Clinical Advances are Unraveling New Potentials?

There has been a renaissance in gene therapy attempts, spurred partly by the discovery and understanding of novel gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that may be designed to carry DNA to target cells and has sparked considerable interest in the area, particularly in clinical...

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Pharma News for AbbVie and Sobi
Novo Nordisk’s Concizumab for Hemophilia; AbbVie Ends its Alliance with Alector; ADC Therapeutics and Sobi Enters in Exclusive Licensing Deal; BMS’ Opdivo Gets NHS Use; Merck to Acquire Seagen; FDA Priority Review to Roche’s Lunsumio; AstraZeneca to Acquire TeneoTwo; FDA Orphan Drug Designation to PBI-200

Novo Nordisk Reports Phase III Results of Concizumab Drug for Hemophilia A or B Novo Nordisk has announced Phase III results for their concizumab drug for hemophilia A or B, demonstrating efficacy in preventing bleeding events and paving the way for regulatory filings later this year. Concizumab, an anti-tissue ...

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Pharma News for Ipsen, BioMarin and AbbVie
Ipsen to Buy Epizyme; BioMarin’s Gene Therapy for Hemophilia; AbbVie’s Qulipta for Chronic Migraine; FDA Approves Breyanzi for R/R LBCL; NDA Filed for Elacestrant in ER+/HER2- Breast Cancer; EU Approval to Enhertu for Breast Cancer; FDA Approves Novartis’ BRAF-positive Tumor Combination; GSK’s Hepatitis B Vaccine, Bepirovirsen

Ipsen to Buy Biopharmaceutical Company Epizyme for USD 247 Million The restructuring of French manufacturer Ipsen has progressed with a takeover agreement for US competitor Epizyme and its cancer drug Tazverik in a deal for slightly less than USD 250 million. Ipsen is proposing USD 1.45 per share for Epizyme, va...

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Introducing a New Era of Hemophilia Management Tools
Role of Mobile Technology in Hemophilia Management

The universality of mobile technology has proven to be extremely beneficial, specifically in recent years. The potential of improving medical care delivery, and the potential for instantaneous and two-way transfer of information has turned out to be a revolutionary step. The following article indicates the importan...

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The Evolved Gene Therapy for Hemophilia

Hemophilia is an inherited rare disorder where blood doesn’t clot in the regular way because the person affected doesn’t make enough blood-clotting proteins (clotting factors). Without these factors, patients cannot stop bleeding when they are injured. Patients with more severe forms of the disease can experience s...

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