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

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

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

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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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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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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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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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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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

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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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). Topic sponsors are not involved in the creation of editorial content.

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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

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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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. Download this In Focus eReport to gain a full understanding of the current market for CGTs and the areas of greatest commercial potential for the future.

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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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Fierce Pharma

MAY 7, 2024

After a 2023 approval from the FDA, Krystal Biotech has collected more than $95 million from its launch of Vyjuvek, the first treatment for the rare skin disease dystrophic epidermolysis bullo | The topical gene therapy has pulled in nearly $100 million during its first three quarters on the U.S.

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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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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

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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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

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.

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XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

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Pharmaceutical Technology

JULY 22, 2022

Pipeline therapies within the diabetic macular oedema (DME) space have recently gathered interest following the American Society of Retina Specialists (ASRS) Annual Meeting, which took place on 13–16 July.

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Bio Pharma Dive

MARCH 20, 2024

million list price, the highest of any genetic medicine to come to market. Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25

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Drug Discovery World

APRIL 30, 2024

Download this In Focus eReport to gain a full understanding of the current market for CGTs and the areas of greatest commercial potential for the future.

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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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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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Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

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Pharma Mirror

JANUARY 30, 2022

Ben Beckley, Global Lead at EmerGENE explores the market challenges holding back Cell and Gene Therapy (C&GT) from taking its place as an established treatment area. The global market is projected to reach $13.8 He explains how we can navigate obstacles to ensure C&GT can achieve its full potential.

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Pharmaceutical Technology

DECEMBER 14, 2022

Merck and Synplogen have signed a non-binding Memorandum of Understanding (MoU) to expedite the development and manufacturing of viral vector-based gene therapy applications. The firms intend to merge their expertise to provide simplified viral vector gene therapy development, production and testing in Japan.

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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. It is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

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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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pharmaphorum

DECEMBER 16, 2022

CSL’s gene therapy for haemophilia B has been recommended for approval by the EMA’s human medicine committee, setting up a decision by the European Commission early next year. The post CSL closes on EU approval for haemophilia B gene therapy appeared first on.

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BioPharma Reporter

AUGUST 24, 2023

Cell and Gene Therapy Catapult, Rentschler Biopharma, and Refeyn have announced a new partnership to develop and apply âinnovativeâ process analytical technologies (PAT) to improve the process and efficiency of AAV manufacture.

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