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. Securing the supply chain.

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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

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

JULY 22, 2022

The spotlight was placed on many up-and-coming pharmacotherapies for retinal diseases, one of which was AbbVie’s/Regenxbio’s gene therapy RGX-314. The drug is of paramount interest due to addressing vital unmet needs for anti-vascular endothelial growth factor (VEGF) treated retinal diseases, including DME.

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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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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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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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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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Bio Pharma Dive

DECEMBER 10, 2023

While new gene therapies for the blood disease grabbed headlines at ASH, Pfizer presented fresh data for an oral drug meant to build on its marketed therapy Oxbryta.

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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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Drug Discovery World

JANUARY 31, 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. Ian Scanlon will discuss bottlenecks in advanced therapeutic delivery and how novel technologies can improve patient access and deliver sustainable processes.

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Drug Discovery World

FEBRUARY 15, 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. Ian Scanlon will discuss bottlenecks in advanced therapeutic delivery and how novel technologies can improve patient access and deliver sustainable processes.

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Drug Discovery World

NOVEMBER 27, 2023

This 24-page eBook sponsored by Sartorius explores the latest innovations in cell and gene therapies, where the challenges lie and the possible solutions to overcoming these obstacles.

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Drug Discovery World

FEBRUARY 22, 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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BioPharma Reporter

APRIL 22, 2024

SK pharmteco has signed an agreement with Ferring Pharmaceuticals, to scale up commercial manufacturing capacity for the drug substance of Ferringâs intravesical gene therapy Adstiladrin.

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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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Drug Discovery World

NOVEMBER 27, 2023

Hailed as a revolution in the treatment of many diseases, cell and gene therapy is the fastest growing area of therapeutics. The post New eBook: The latest innovations in cell and gene therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

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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. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. How do Casgevy and Lyfgenia Work? million after discounts.

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.

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pharmaphorum

SEPTEMBER 26, 2022

Pfizer and Sangamo have restarted recruitment of patients into their registration trial for haemophilia A gene therapy giroctocogene fitelparvovec, after the FDA lifted a clinical hold on the study. The post Pfizer, Sangamo resume haemophilia gene therapy trial appeared first on.

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. As such, rare disease patients and their families often face little hope for effective treatments.

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BioPharma Reporter

MARCH 19, 2024

Food and Drug Administration has cleared AGC Biologicsâ Milan site to begin manufacturing of Orchard Therapeuticsâ Lenmeldy (atidarsagene autotemcel), a gene therapy for early-onset metachromatic leukodystrophy (MLD).

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Drug Discovery World

JULY 3, 2023

Cytiva and BioCentriq, a New Jersey-based cell and gene therapy contract development and manufacturing organisation (CDMO), have been awarded funding from the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), a public-private partnership whose mission is to accelerate biopharmaceutical innovation.

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Drug Discovery World

JANUARY 25, 2023

Cell and gene therapies are transforming the way diseases are treated. As research progresses it is hoped that cell and gene therapies will offer innovation for the future. autologous therapies/solid tumours/ indications outside of oncology?) Register for free now.

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Drug Discovery World

FEBRUARY 8, 2024

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

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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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Drug Discovery World

FEBRUARY 21, 2024

We are delighted to be further backing MIP as they are making significant technical progress.” The post £7M financing to drive commercialisation in cell and gene therapy appeared first on Drug Discovery World (DDW).

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Bio Pharma Dive

DECEMBER 14, 2021

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

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pharmaphorum

MAY 18, 2022

SwanBio Therapeutics has raised $56 million in a second-round financing that will be used to advance its lead gene therapy candidate for adrenomyeloneuropathy (AMN) into clinical testing later this year. The post SwanBio raises $56m for rare neurological disease gene therapy appeared first on.

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BioPharma Reporter

APRIL 24, 2023

bluebird bio has announced the submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD).

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pharmaphorum

JUNE 10, 2022

The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks. Thirteen of them said no, with one abstention and one vote to the contrary. That is a much more common disorder than CALD.

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STAT News

MARCH 20, 2023

DALLAS — A top Food and Drug Administration official said Monday that the agency needs to start using accelerated approval , a much-debated path commonly used for advancing cancer drugs, to advance gene therapies for rare disease. ” Continue to STAT+ to read the full story…

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pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Regardless of cohort, eligible participants are scheduled to receive the investigational gene therapy, either at the start of the study or after one year following treatment with placebo.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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