Pharmaceutical Technology

AUGUST 25, 2022

The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s gene therapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

APRIL 19, 2024

Paolo Morgese, vice president of European public affairs at the Alliance for Regenerative Medicine, discusses the barriers affecting access to gene therapies.

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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

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

JULY 28, 2023

The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.

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Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Personalised medicines will also be developed at the centre.

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Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

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Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Bio Pharma Dive

AUGUST 17, 2022

The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a very high cost of $2.8 million in the U.S.

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Pharmaceutical Technology

MARCH 10, 2023

Cell One Partners has announced a strategic collaboration with the Center for Breakthrough Medicines (CBM) to expedite the development and commercialisation of cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.

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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

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Bio Pharma Dive

MARCH 24, 2021

A group of drug companies, including Novartis, Takeda and Roche's Genentech, have agreed to collaborate with Vineti to usher in new identification standards for complex medicines.

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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

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.

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Bio Pharma Dive

JUNE 22, 2023

The cost makes Sarepta’s treatment, called Elevidys, among the most expensive medicines in the world. But company executives said they don’t expect significant insurer pushback.

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Bio Pharma Dive

DECEMBER 9, 2021

The amount of blood clotting protein found in patients who received the medicine was nearly the same at six months and 18 months post-treatment, according to results disclosed Thursday.

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Pharmaceutical Technology

MAY 12, 2023

Editas Medicine will release new efficacy and safety results of its gene therapy EDIT-301 in severe sickle disease as part of an oral presentation at the European Hematology Association’s (EHA) Hybrid Congress. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Bio Pharma Dive

APRIL 6, 2022

Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.

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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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Bio Pharma Dive

DECEMBER 8, 2023

Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1

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Bio Pharma Dive

AUGUST 19, 2021

Developers of the complex treatments raised $14 billion between January and June, nearly three-quarters of last year's record-setting total, according to numbers compiled by the Alliance for Regenerative Medicine.

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Pharmaceutical Technology

APRIL 9, 2025

The potential for cell and gene therapies is growing, offering groundbreaking treatments for rare genetic disorders and promising to reshape the future of precision medicine.

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Bio Pharma Dive

JULY 9, 2021

Zynteglo sales have been on hold since February, when a patient in a clinical trial of another, related Bluebird medicine developed leukemia.

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Bio Pharma Dive

DECEMBER 15, 2020

The acquisition marks a significant bet by Lilly on gene therapy's potential to address tough-to-treat neurological disorders like Parkinson's disease.

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Bio Pharma Dive

AUGUST 24, 2022

The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.

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Bio Pharma Dive

JANUARY 24, 2024

An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.

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Pharmaceutical Technology

FEBRUARY 4, 2025

USC has received a $6m grant from the California Institute for Regenerative Medicine (CIRM) to advance a new gene therapy for glioblastoma.

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Bio Pharma Dive

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

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Bio Pharma Dive

FEBRUARY 12, 2021

Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental genetic medicines, a research boom that recalls biotech's roots.

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Bio Pharma Dive

OCTOBER 24, 2022

The biotech is the latest in a growing list of genetic medicine developers to sell themselves near record stock lows amid a challenging funding environment.

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Camargo

DECEMBER 20, 2021

Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies. The increase is designed to strengthen CBER’s capacity and capabilities for regulating cell and gene therapies. 2024: 48 staffers. 2025: 29 staffers.

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Bio Pharma Dive

DECEMBER 14, 2021

But gene therapies and other new medicines might provide competition. Fitusiran, which Sanofi licensed from Alnylam, could finally get to market after trial delays and safety concerns slowed its progress.

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Pharmaceutical Technology

DECEMBER 23, 2022

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.

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Pharmaceutical Technology

JANUARY 16, 2023

CARsgen Therapeutics has announced a col labor ation with Huadong Medicine to commercialise zevorcabtagene autoleucel (zevor-cel), CT053, in mainland China. Under the collaboration, Huadong Medicine will have the exclusive right to commercialise CARsgen’s CT053 in mainland China. By Cytiva Thematic.

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BioSpace

JANUARY 25, 2021

Gene therapies offer great reward in the form of treating various devastating diseases, but there are also significant risks.

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