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

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

JUNE 29, 2023

After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

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Pharmaceutical Technology

JUNE 30, 2023

BioMarin’s Roctavian, a one-time therapy for severe haemophilia A, has secured FDA approval following previous rejections and delays.

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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). The patient suffered acute liver failure several months after receiving the therapy in December. Sarepta is reviewing all available data.

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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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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. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

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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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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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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

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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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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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

JULY 4, 2023

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

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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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Gene Therapy FDA Approval Gene Genetics 114

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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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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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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Pharmaceutical Technology

AUGUST 7, 2023

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

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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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Fierce Pharma

DECEMBER 8, 2023

Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. Groundbreaking? Game-changing? Transformational?

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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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Gene Therapy FDA Approval Gene Genetics 99

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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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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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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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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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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Fierce Pharma

JULY 25, 2024

Three months after bagging FDA approval, Pfizer’s hemophilia B gene therapy has been cleared to expand its reach across the pond. | The company's first gene therapy will debut in Europe as Durveqtix. the drug is approved under the brand name Beqvez. In the U.S.,

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XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. thalassemia (TDT) in the field of gene therapy.

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BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

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Gene Therapy Gene FDA Approval Genetics 105

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient.

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Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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Gene Therapy Gene FDA Approval Licensing 147

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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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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