Bio Pharma Dive

SEPTEMBER 17, 2022

The therapy, called Skysona and cleared to treat cerebral adrenoleukodystrophy, is the product of more than a decade of work by Bluebird. It will cost $3 million.

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Bio Pharma Dive

MAY 20, 2022

The brain-infused treatment for a rare neurological disease would be one of only three gene therapies commercially available in the EU. PTC plans to ask for FDA approval later this year.

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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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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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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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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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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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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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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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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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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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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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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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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

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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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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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 173

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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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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Pharmaceutical Technology

NOVEMBER 9, 2022

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. An approval decision on the gene therapy, also known as EtranaDez, is expected by the end of this month. million ($1.8 million price tag.

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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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XTalks

APRIL 1, 2025

Clinical Trials Backing Qfitlias Approval Two Phase III studies involving adult and pediatric male patients with hemophilia A or B supported Qfitlias FDA approval. Qfitlia is the second RNA interference therapy to receive a highly anticipated FDA approval recently. Among these are three gene therapies.

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Pharmaceutical Technology

APRIL 27, 2023

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) to expand the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to treat cystic fibrosis (CF) in children aged between two and five years with certain mutations.

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Pharmaceutical Technology

AUGUST 7, 2023

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

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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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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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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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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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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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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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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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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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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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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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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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