Pharmaceutical Technology

MAY 26, 2023

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene FDA Approval Manufacturing 264

Pharmaceutical Technology

JANUARY 17, 2024

The US FDA has approved Vertex's CASGEVY, a gene-edited cell therapy, to treat transfusion-dependent beta thalassemia.

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Gene Editing Gene FDA Approval 245

Bio Pharma Dive

MAY 28, 2021

For decades, scientists have tried unsuccessfully to target the KRAS gene, which is often mutated in lung, colon and pancreatic cancers. Lumakras is the first drug proven effective.

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FDA Approval Drugs Gene Scientist 361

Bio Pharma Dive

APRIL 24, 2023

Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.

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Gene Editing Gene Gene Therapy FDA Approval 256

Pharmaceutical Technology

DECEMBER 11, 2023

The FDA has approved Vertex/CRISPR’s Casgevy and bluebird bio’s Lyfgenia gene therapies for the red blood cell disorder.

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Gene Therapy Gene FDA Approval 246

Pharmaceutical Technology

MARCH 19, 2024

The US FDA has granted approval to Orchard Therapeutics' Lenmeldy for specific forms of metachromatic leukodystrophy (MLD).

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Gene Therapy FDA Approval Gene 246

Pharmaceutical Technology

MARCH 10, 2023

The US Food and Drug Administration (FDA) has granted approval for Pfizer ’s Zavzpret (zavegepant) for the acute treatment of migraine in adult patients with or without aura. Zavzpret is claimed to be the first and only calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray approved to treat migraine.

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FDA Approval Pharmacy Trials Clinical Development 246

Bio Pharma Dive

SEPTEMBER 16, 2021

Exkivity follows Johnson & Johnson's Rybrevant on the market for treating lung tumors harboring specific mutations in the EGFR gene. Unlike J&J's drug, however, Takeda's is given orally.

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FDA Approval Drugs Gene Marketing 273

XTalks

MARCH 19, 2025

A 16-year-old patient died after treatment with Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy (DMD). Liver injury is a known risk with Elevidys and other gene therapies that use adeno-associated viral (AAV) vectors. Sarepta is reviewing all available data.

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Pharmaceutical Technology

SEPTEMBER 19, 2022

A one-time gene therapy, Skysona leverages ex-vivo transduction with the Lenti-D lentiviral vector for adding the ABCD1 gene’s functional copies into the hematopoietic stem cells of the patient. The post FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy appeared first on Pharmaceutical Technology.

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FDA Approval In-Vivo Gene Therapy Clinical Trials 246

Bio Pharma Dive

AUGUST 10, 2020

The test, developed by Guardant Health, combines next-generation sequencing with a cancer blood test.

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Gene Sequencing FDA Approval Gene Development 246

XTalks

DECEMBER 19, 2024

Ensartinibs approval, granted under the commercial name Ensacove, was awarded to Xcovery Holdings, a subsidiary of Chinas Betta Pharmaceuticals. ALK-positive NSCLC is a subset of lung cancer driven by genetic rearrangements in the ALK gene. ALK-positive NSCLC is estimated to account for about five percent of all NSCLC cases.

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FDA Approval Marketing Trials Drugs 98

Pharmaceutical Technology

JULY 4, 2023

The gene therapy prevents neovascularisation from a single, intravitreally delivered injection.

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FDA Approval Gene Therapy Gene Trials 243

Pharmaceutical Technology

FEBRUARY 10, 2023

4D Molecular Therapeutics (4DMT), the California-based biotechnology company focused on developing gene therapies for rare and large market diseases, has had the FDA place a clinical hold onto its Fabry disease (FD) gene therapy program (4D-310).

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Gene Therapy Gene Trials Marketing 264

Bio Pharma Dive

MAY 22, 2023

Cleared to treat dystrophic epidermolysis bullosa, Vyjuvek is the sixth gene therapy for an inherited disease to gain clearance in the U.S.

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Gene Therapy Gene FDA Approval 171

Bio Pharma Dive

JUNE 30, 2023

The agency granted a long-awaited clearance on Thursday, but unexpected aspects of the hemophilia treatment’s label had some investors worried about its commercial prospects.

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Gene Therapy FDA Approval Gene 263

BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

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Gene Therapy FDA Approval Gene Genetics 114

Bio Pharma Dive

FEBRUARY 23, 2022

Jeffrey Marrazzo has led Spark since its founding in 2013, through the historic FDA approval of Luxturna and the company's $4.3 billion buyout by Roche.

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Gene Therapy FDA Approval Gene 264

Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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Gene Therapy Gene Trials Clinical Trials 264

Bio Pharma Dive

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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Gene Therapy Gene FDA Approval Development 164

Pharmaceutical Technology

AUGUST 7, 2023

HuidaGene claims its gene therapy candidate was superior to FDA-approved Luxturna in preclinical studies.

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Gene Therapy FDA Approval Gene 246

pharmaphorum

DECEMBER 19, 2022

Ferring Pharma ’s Adstiladrin is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy. Dr Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said: “This approval provides healthcare professionals with an innovative treatment option for [these] patients.”.

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Gene Therapy FDA Approval Gene Drugs 119

Fierce Pharma

DECEMBER 8, 2023

| Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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Gene Therapy FDA Approval Gene Gene Editing 123

Fierce Pharma

NOVEMBER 14, 2024

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries, where it's branded as Upstaza.

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Gene Therapy Gene FDA Approval Branding 131

BioPharma Reporter

APRIL 30, 2024

million gene therapy for hemophilia B just got FDA approval. Pfizer's groundbreaking $3.5 Hereâs what it means for patients and the future of medicine

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Gene Therapy FDA Approval Gene Medicine 97

BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

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Gene Therapy FDA Approval Gene Genetics 99

XTalks

FEBRUARY 25, 2025

Mirum Pharmaceuticals, a biotech innovator known for developing therapies for rare metabolic disorders, now has FDA approval for its new treatment, Ctexli (chenodiol) tablets the first and only medication approved for cerebrotendinous xanthomatosis (CTX) in adults.

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FDA Approval Gene Therapy Gene Genetics 59

Fierce Pharma

APRIL 26, 2024

Ten years after dipping its toes into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained FDA approval for the acquired treatment. | It becomes the first FDA-approved gene therapy for Pfizer, which will charge $3.5 million for the single-dose drug.

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Gene Therapy Gene FDA Approval Licensing 128

STAT News

NOVEMBER 22, 2022

The Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder. The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company.

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Gene Therapy Gene FDA Approval Marketing 139

XTalks

JANUARY 21, 2025

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Date of FDA approval : March 29, 2024 Approved for : To detect exposure to human parvovirus B19. TriClip size comparison photo. Photo courtesy of Abbott Medical.

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FDA Approval Manufacturing Development RNA 105

Fierce Pharma

DECEMBER 8, 2023

Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell | Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival (..)

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Gene Editing Gene Gene Therapy FDA Approval 126

Pharmaceutical Technology

OCTOBER 24, 2024

There are already two FDA-approved gene therapies for haemophilia B courtesy of Pfizer and CSL Behring.

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Gene Therapy Gene FDA Approval Trials 130

Pharma Mirror

NOVEMBER 24, 2020

Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced that it had entered into a commercial supply agreement with Blueprint Medicines following FDA approval of GAVRETO™ (pralsetinib).

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pharmaphorum

MARCH 26, 2024

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of underlying genetic mutations.

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Gene Therapy Gene FDA Approval Genetics 128

XTalks

JANUARY 6, 2025

This approval marks a significant milestone for Vertex Pharmaceuticals, making Alyftrek their fifth CFTR modulator to secure FDA approval, offering hope to an expanded group of CF patients. The CFTR gene itself is complex, with over 2,000 known mutations.

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Pharmaceutical Technology

JUNE 21, 2023

The combination therapy is indicated to treat homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC) in adult patients. It is the first and only PARP inhibitor to receive approval for use along with the current standard of care (Xtandi) for HRR gene-mutated mCRPC.

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BioSpace

APRIL 25, 2024

Pfizer will go toe-to-toe with CSL Behring following the FDA’s Friday approval of its hemophilia B gene therapy Beqvez and will launch a warranty program based on the durability of response.

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Gene Therapy Gene FDA Approval 105