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buyout

Novartis ups investment in gene therapy for the eye with $800M buyout

Novartis’ offer comes three months after Gyroscope presented at a medical meeting early data from 13 patients who had been dosed with the gene therapy, …

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Biotech

A high-profile gene therapy biotech takes aim at Huntington’s

Huntington’s was the first illness ever traced back to changes in a specific chromosome. And yet, almost 40 years since that discovery, no drug has …

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Bluebird

Bluebird, after delays, gets speedy FDA review for beta thalassemia gene therapy

The FDA’s acceptance of Bluebird’s application, which comes two months after the company submitted it to the agency, marks the final stretch of a long …

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gene therapy

Pfizer gene therapy research delayed by trial changes, safety questions

In both hemophilia and Duchenne, unexpected findings have led Pfizer to redraw its development plans. The company recently paused patient screening and dosing in a …

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biological medicines

SGS Increases Biosafety Testing Capacity at Its Glasgow Facility

SGS has just completed a significant expansion project at its Glasgow facility, meaning it can provide increased biosafety testing for the biologics, drug development, and …

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Boehringer Ingelheim

Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy

Boston biotech Vertex is the leading developer of cystic fibrosis medicines. Its four approved drugs can treat most patients. Trikafta, the one most recently approved …

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ALS

Gene therapy developer gets chance at a comeback with Pfizer deal

Voyager’s first generation of gene therapies for ALS, Parkinson’s and Huntington’s disease, all now discontinued, relied on a direct infusion into brain tissue, an invasive …

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Biotech

Sarepta embarks on late-stage clinical trial of Duchenne gene therapy

Although Pfizer beat Sarepta to start a Phase 3 trial of its gene therapy, the smaller biotech is claiming a milestone in initiating the study …

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Angelman

FDA lifts hold on GeneTx, Ultragenyx study of Angelman therapy

While the clinical hold lifting is encouraging news for the companies’ partnership, questions still loom about safety in the genetic medicine field. Earlier this month, …

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BioMarin

FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

The theoretical risk that gene therapy may cause cancer in humans has hung over the field for decades. Those worries resurfaced last year when a …

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Bluebird

FDA clears Rocket to resume testing gene therapy for a rare heart disease

Rocket’s program has been in limbo since early May, when the company surprised investors and analysts with news of the trial’s stoppage. That announcement stalled …

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