XTalks

AUGUST 19, 2022

Along with beti-cel, there is one being developed for sickle cell (approved in Europe as Skysona or eli-cel) and another being jointly developed with Bristol Myers Squibb for melanoma. But bluebird bio has said such setbacks are expected for a company at the frontier of developing gene therapies for rare diseases.

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Gene Therapy FDA Approval Gene Clinical Trials 95

pharmaphorum

JUNE 12, 2022

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8

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Gene Therapy Gene Marketing Trials 58

pharmaphorum

JANUARY 11, 2021

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. The gene therapy firm will be focused on its most important therapies in beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US And Europe.

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Gene Therapy Gene Genetic Disease Manufacturing 61

XTalks

SEPTEMBER 27, 2022

The drug took over the world’s most expensive drug title from fellow bluebird bio gene therapy Zynteglo, which received FDA approval last month, by just a couple of hundred grand as Zynteglo costs $2.8 The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. Related: Bluebird’s $2.8M

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FDA Approval Gene Therapy Drugs Gene 52

pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. bluebird has opted to launch it at a cost of $2.8 million, which compares to the $1.7

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Gene Therapy FDA Approval Gene Clinical Trials 52

pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. The post Roctavian okay sets up another gene therapy test for Europe appeared first on. million spread over five years.

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Gene Therapy Gene Antibody Marketing 110

pharmaphorum

MARCH 27, 2022

The decision comes less than a month after Carvykti was approved by the FDA as a fifth-line therapy for multiple myeloma, teeing up a tussle in the market with Bristol-Myers Squibb and bluebird bio spinout 2seventy’s Abecma (idecabtagene autoleucel) which was approved for a similar indication last year.

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Antibody Clinical Trials Clinical Trials Sales 98