Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. The transplant is associated with serious risks and potential complications, including death, resulting in few CALD patients ever being treated.

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Gene Therapy FDA Approval Gene Genetic Disease 289

Rethinking Clinical Trials

APRIL 27, 2023

Tags #pctGR, @Collaboratory1 The post Grand Rounds April 21, 2023: Personalised Cooler Dialysate for Patients Receiving Maintenance Haemodialysis (MyTEMP): A Pragmatic, Cluster-randomised Trial (Amit Garg, MD, MA, FRCPC, FACP, PhD; Stephanie N. degrees Celsius.

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Trials Clinical Trials Clinical Trials Medicine 130

Pharmaceutical Technology

APRIL 13, 2023

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. While stem cell transplants can cure the condition, their use is limited due to some of the risks that come with them. Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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Gene Therapy Gene FDA Approval Licensing 147

pharmaphorum

JUNE 12, 2022

Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8 Haematopoietic stem cell transplantation (HSCT) offers a chance of a cure, but is only possible when a donor with a matching human leukocyte antigen (HLA) signature, within the correct age range, is available.

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Gene Therapy Gene Marketing Trials 58

pharmaphorum

APRIL 14, 2022

million price tag. Haematopoietic stem cell transplantation (HSCT) offers a chance of a cure, but is only possible when a donor with a matching human leukocyte antigen (HLA) signature, within the correct age range, is available.

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Gene Therapy Gene Marketing Clinical Trials 63

XTalks

DECEMBER 13, 2023

A bone marrow transplant from a donor can offer a potential cure, but it is often difficult to find a matching donor and the procedure carries the risk of rejection by the immune system. Current treatments for sickle cell disease include blood transfusions but patients often have episodes of intense pain and an increased risk of stroke.

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Gene Therapy FDA Approval Gene In-Vivo 98

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. Currently, all approved CAR-T therapies are autologous, where T cells taken from patients are modified and then re-infused.

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Gene Therapy Gene Engineer Production 147