Fierce Pharma

NOVEMBER 7, 2023

Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Vertex and CRISPR Therapeutics are up first with a Dec. |

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Gene Therapy Gene 86

Fierce Pharma

JULY 14, 2023

The potentially curative promise of gene therapies often carries a steep price tag. | The potentially curative promise of gene therapies often carries a steep price tag. But for a pair of personalized medicine prospects in sickle cell disease (SCD), the cost could be worth it, at least as far as ICER is concerned.

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Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene FDA Approval Licensing 147

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. It also affects Hispanic Americans, but at a lower prevalence.

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Gene Therapy FDA Approval Gene In-Vivo 98

STAT News

OCTOBER 6, 2022

… Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease , Bloomberg News tells us. Even

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Gene Therapy Gene Insulin Drugs 98

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. The cells are prepared outside of the body ( ex vivo ) and then infused into the patient. Zynteglo’s hefty price tag of $2.8

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Gene Therapy FDA Approval Gene Clinical Trials 95

pharmaphorum

JANUARY 11, 2021

While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA. 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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