FDA OKs Second Gene Therapy for Hemophilia B
(MedPage Today) -- The FDA has approved a second gene therapy for adults with hemophilia B, drugmaker Pfizer announced on Friday.
The approval of fidanacogene elaparvovec (Beqvez) stipulates use in adult men with moderate to severe hemophilia... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - April 26, 2024 Category: American Health Source Type: news
FDA Approves Second Gene Therapy for Hemophilia B FDA Approves Second Gene Therapy for Hemophilia B
Pfizer reported a list price of $3.5 million for its new gene therapy, Beqvez, for adults with this rare bleeding disorder.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - April 26, 2024 Category: Consumer Health News Tags: Internal Medicine News Alert Source Type: news
FDA approves Pfizer ’s first gene therapy for rare inherited bleeding disorder
The Food and Drug Administration on Friday approved Pfizer 's treatment for a rare genetic bleeding disorder, making it the company's first-ever gene therapy to win clearance in the U.S. The agency greenlit the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B…#pfizer #beqvez #hemophilia #adamcuker #comprehensiveand #sparktherapeutics #cslbehring #fda #hemgenix #hemophiliaa (Source: Reuters: Health)
Source: Reuters: Health - April 26, 2024 Category: Consumer Health News Source Type: news
Walgreens to open cell and gene therapy pharmacy center in Pittsburgh
It's part of the growing market share of specialty drugs. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 26, 2024 Category: Pharmaceuticals Authors: Paul J. Gough Source Type: news
U.S. FDA approves Pfizer & #039;s BEQVEZ ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with Hemophilia B
Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. (Source: World Pharma News)
Source: World Pharma News - April 26, 2024 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news
NxGen founder fires back at Penn and Dr. James Wilson in lawsuit over 'decade-long nightmare'
The one-time postdoctoral researcher alleges her "life ’s work is being held hostage" by Penn and Dr. James Wilson, director of the university's gene therapy program. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 25, 2024 Category: Pharmaceuticals Authors: John George Source Type: news
Cell and Gene Therapy Development: Characterization of Cellular Source Material, Upcoming Webinar Hosted by Xtalks
In this free webinar, gain insights into characterizing cellular source material for developing and manufacturing cell and gene therapy-based products. Attendees will learn about intrinsic and external factors that influence cell variability in donors with the desired target profile, and... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - April 25, 2024 Category: Pharmaceuticals Source Type: news
AskBio receives FDA Fast Track Designation for AB-1002 investigational gene therapy program in congestive heart failure
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1. (Source: World Pharma News)
Source: World Pharma News - April 25, 2024 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
Researchers publish final results of key clinical trial for gene therapy for sickle cell disease
In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) - a blockage that results in lack of oxygen and painful episodes - for at least one year, and 100% were able to remain hospitalization-free for the same length of time. (Source: World Pharma News)
Source: World Pharma News - April 24, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Auchincloss to keynote rare disease innovation forum
The Massachusetts congressman has been an advocate for biotech investment, R&D, and broader access to gene therapies. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 19, 2024 Category: Pharmaceuticals Authors: Mary Serreze Source Type: news
Gene Therapy: Non-Viral Vectors
Modern medicine constantly searches for safer and more efficient ways to treat genetic disorders. One cutting-edge solution that has emerged is the use of non-viral vectors. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - April 18, 2024 Category: Pharmaceuticals Authors: William A. Haseltine, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
Gene Therapy Methods Explained
Gene delivery systems are critical for gene therapy and can be classified as viral or non-viral vectors. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - April 16, 2024 Category: Pharmaceuticals Authors: William A. Haseltine, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
AskBio presents 18-month Phase Ib trial results of AB-1005 gene therapy for patients with Parkinson & #039;s disease
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, on Sunday April 14 presented results from the 18-month Phase Ib clinical trial for AB-1005, an investigational gene therapy for treating patients with Parkinson's disease (PD).(1,2) The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA.
The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. (Source: World Pharma News)
Source: World Pharma News - April 16, 2024 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
Mayo Clinic RegenBio Summit 2024 registration opens, keynotes announced
Gene editing, gene therapy and tissue engineering are emerging regenerative biotherapies that are on the cusp of transforming medical care. RegenBio Summit: Transforming Next-Gen Biotherapeutics 2024, will focus on accelerating technologies from concept to reality for patients in need of new healing options. Register now to join physicians, scientists, general healthcare staff, industry leaders and students from Dec. 9 to 11 at the Ponte Vedra Inn& Club in Ponte Vedra Beach, Florida. Regenerative biotherapies… (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - April 15, 2024 Category: Databases & Libraries Source Type: news
AskBio co-founder Jude Samulski steps down, successor named
The groundbreaking co-founder of one of North Carolina's most well-known gene therapy companies has left his longtime role in the C-suite. He's replaced by a prominent executive from Eli Lilly and Co. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 9, 2024 Category: Pharmaceuticals Authors: Zac Ezzone Source Type: news
