Bio Pharma Dive

JUNE 27, 2023

The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.

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Bio Pharma Dive

MARCH 24, 2022

At least nine biotechs working in cell or gene therapy have announced layoffs, cost cuts or restructured their research since December — restructurings that have coincided with a stock market downturn.

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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

APRIL 13, 2023

Takeda has announced that it will be pivoting away from its discovery and preclinical programmes in adeno-associated virus (AAV) gene therapies. Takeda’s announcement underlines the risk associated with gene therapy R&D at the preclinical stage and the fact that many current AAV programs are unlikely to reach late-stage trials.

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BioSpace

MAY 8, 2024

As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.

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Bio Pharma Dive

JULY 20, 2022

The Swiss drugmaker, which already owns rights to a marketed gene therapy for inherited vision loss, will work with startup Avista Therapeutics to develop better delivery tools for the complex treatments.

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Bio Pharma Dive

JUNE 24, 2022

The decision brings what could be the first approved hemophilia gene therapy, Roctavian, closer to market, after a series of regulatory setbacks that have delayed its arrival.

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Pharmaceutical Technology

APRIL 3, 2023

The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and gene therapies.

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Pharmaceutical Technology

JUNE 19, 2023

Indian pharmaceutical company Laurus Labs has signed a memorandum of agreement (MoA) with the Indian Institute of Technology, Kanpur (IIT Kanpur) to bring new gene therapy products to the market. Laurus Labs will be responsible for launching these products in India and emerging markets.

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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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Bio Pharma Dive

JULY 21, 2021

The approval of Skysona for a genetic brain disease is a milestone for one of gene therapy's pioneering companies, but isn't expected to turn Bluebird's financial fortunes around.

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BioSpace

APRIL 21, 2024

From gene-corrected cell therapies to a new CAR-T, the cell and gene therapy space looks to expand its reach into the market.

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Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Bio Pharma Dive

MAY 21, 2021

The treatment, for a progressive, often deadly brain disease, could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world.

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pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.

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Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

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Pharma Mirror

MAY 7, 2021

The leading Australian CRO for biotechs and Frost & Sullivan Asia-Pacific CRO Market Leadership Award winner, Avance Clinical, has expanded its gene technology clinical trial services to meet the increasing global demand predicted to reach $17.4 The post Avance Clinical Expands Gene Technology Clinical Trial Services to Meet $17.4

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Bio Pharma Dive

MAY 17, 2022

The treatment, which is designed to treat Sanfilippo syndrome, could become Ultragenyx's first gene therapy to reach market in the U.S., according to the biotech's CEO.

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Bio Pharma Dive

OCTOBER 21, 2021

The biotech won EU approval for two gene therapies, Zynteglo and Syksona, both of which it's now said it will pull from market after difficulties negotiating reimbursement.

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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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Drug Discovery World

FEBRUARY 5, 2024

Mark Treherne will talk about advanced therapies and the development of RNA gene therapy for aggressive cancers which is being carried out at Spliceor. Doug Danison will discuss transitioning innovative science into commercial success in the areas of CGTs.

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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. By Cytiva Thematic.

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Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.

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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. The NHSBT hopes the CBC will increase the UK’s competitiveness within the market.

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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

AUGUST 18, 2022

Its market launch is likely to be watched carefully by other gene therapy developers. Approved Wednesday for severe beta thalassemia, Zynteglo will test insurers’ willingness to pay for expensive one-time treatments.

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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 ($1.5 million ($1.8

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Drug Discovery World

MAY 2, 2024

Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. Perhaps most significantly, Casgevy (or exa-cel) from Vertex Pharmaceuticals and CRISPR Therapeutics, was approved to treat sickle cell disease and became the first gene therapy that uses CRISPR gene editing.

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Bio Pharma Dive

OCTOBER 25, 2022

Treatment is now mostly of infants newly diagnosed with spinal muscular atrophy, as many of those previously eligible either already received Zolgensma or other drugs from Biogen and Roche.

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BioSpace

APRIL 10, 2023

Gene Editing Therapeutics Could Hit the Market in 2023 4/10/2023

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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

DECEMBER 20, 2022

The European Commission (EC) has granted marketing authorization for Atara Biotherapeutics’ Ebvallo (tabelecleucel) as a monotherapy to treat Epstein‑Barr virus positive post‑transplant lymphoproliferative disease (EBV+ PTLD) patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery World

FEBRUARY 13, 2024

In this webinar, hosted by DDW and sponsored by Astrea Bioseparations, you will learn about how market growth can be sustained to maximise on opportunities in cell and gene therapy (CGT). The post Cell & gene therapies: How can market growth be sustained to maximise on opportunities?

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Pharmaceutical Technology

JULY 7, 2022

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research. Cold chain logistics, meanwhile, have never been so important.

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Pharmaceutical Technology

SEPTEMBER 8, 2023

In this issue: Digital jobs in UK life sciences, generative AI changes drug discovery, and how robotics is impacting gene therapy manufacturing.

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BioPharma Reporter

MAY 30, 2024

Cellular Origins and the Cell and Gene Therapy Catapult (CGT Catapult) have announced a new collaboration to demonstrate universal automation of CGT manufacturing.

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BioSpace

NOVEMBER 16, 2023

s conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel raises some potential safety concerns with the risk of off-target effects.

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