Source https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.026#utm_source=ETOC&utm_medium=email&utm_campaign=hum Corneal diseases are among the most prevalent causes of blindness worldwide. The transparency and clarity of the cornea are guaranteed by a delicate physiological, anatomic, and functional balance. For this reason, all the disorders, including those of genetic origin, that compromise this state of harmony can lead to opacity and eventually vision loss.

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Patients are about to be enrolled in the first study to test a gene-editing technique known as CRISPR inside the body to try to cure an inherited form of blindness. People with the disease have normal eyes but lack a gene that converts light into signals to the brain that enable sight. The experimental treatment aims to

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Source Human Gene Therapy Clinical Development Background: Cell and gene therapy products belong to a diverse class of biopharmaceuticals known as advanced therapy medicinal products. Cell and gene therapy products are used for the treatment and prevention of diseases that until recently were only managed chronically. The objective of this study was to examine the characteristics

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Sangamo Therapeutics and Pfizer have reported additional findings from the Phase I/II Alta clinical trial of investigational gene therapy SB-525 to treat patients with severe haemophilia A. SB-525 is made up of a recombinant adeno-associated virus serotype 6 vector (AAV6) that encodes the complementary deoxyribonucleic acid for B domain deleted human Factor VIII (FVIII). During

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We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these expensive treatments are entering a market structure that was not built to price them. Congress will likely need to play a part in developing a new paradigm for financing such

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Bluebird bio Inc on Friday set a price for its gene therapy, Zynteglo, at 1.58 million euros ($1.78 million) over five years, after winning conditional approval in Europe this month to treat a rare genetic blood disorder. The company proposed an installment plan, with 315,000 euros paid up front and four additional annual payments due

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