Tag Archives: Doug Ingram

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Sarepta Inks Codiak Deal to Explore Combining Exosomes, Muscle Meds

Tiny, naturally occurring particles called exosomes shuffle molecular payloads between cells, facilitating intercellular communication. Codiak Biosciences’ focus is using those nanoparticles to more safely deliver a range of medicines to specific cell types. Now the Cambridge, MA-based biotech has teamed up with neuromuscular disease drug developer Sarepta Therapeutics (NASDAQ: SRPT) to explore using its engineered […]

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Sarepta Eyes Key Test for Gene Therapy in Rare Muscular Dystrophy

The $165 million bet that Sarepta Therapeutics placed on the gene therapy programs of Myonexus Therapeutics is starting to pay off with more early but encouraging clinical data from the deal’s most advanced program—a potential treatment for a type of limb-girdle muscular dystrophy. Currently no available treatments exist for any of the many subtypes of […]

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In an About-Face, FDA Approves Second Sarepta Muscular Dystrophy Drug

Sarepta Therapeutics received an early holiday gift Thursday—approval of golodirsen (Vyondys 53), its second Duchenne muscular dystrophy treatment, which the FDA rejected in August. The agency had dinged the application over potential side effects, namely a risk of infections from the “ports” used to infuse the drug, and kidney problems observed in animals in which […]

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The Finalists for the 2019 Xconomy Awards Are …

After a robust discussion, the editors and judges have narrowed down an impressive list of nearly 300 nominees for the 2019 Xconomy Awards in Boston to an all-star group of almost 60 finalists in 11 categories. Given the overall caliber of our nominees, we know it will be a tight race for the winners. Thankfully, we […]

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How an Ohio Kids’ Hospital Quietly Became Ground Zero for Gene Therapy

[Updated, 4/22/19, see below] If a once-modest regional hospital and its new biotech allies have their way, the capital of Ohio could one day rival America’s other biomedical hubs. “Our goal is to make Columbus the center of the universe for gene therapy,” says Doug Ingram, CEO of Sarepta Therapeutics. Sarepta (NASDAQ: SRPT) is based […]

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Notes from the JPM19 Vortex: Price Apocalypse, Celgene Fallout & More

[Editor’s note: Ben Fidler and Sarah de Crescenzo coauthored this report.] It is here, and then it is gone. But by the time everyone staggers to the airport or back home to sleep in their own beds, the annual few days of the J.P. Morgan Healthcare Conference and everything that mushrooms around it seem just […]

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Sarepta Touts New Data and a “Signal” With Duchenne Gene Therapy

For the first time, a gene therapy—a one-time, long-lasting treatment—has shown it may improve the lives of kids with Duchenne muscular dystrophy, a deadly genetic disease with no cure. Patients in a small trial showed improved motor function, such as walking and climbing stairs, and near-normal levels of a key muscle protein. But the data […]

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Europe Advisory Panel Rejects Sarepta’s Duchenne Drug Once Again

Sarepta Therapeutics won one of the most controversial drug approvals in recent memory, when the FDA in September 2016 approved the Duchenne muscular dystrophy drug eteplirsen (Exondys 51) off a slim set of data. The Cambridge, MA, company isn’t having the same luck in Europe. Sarepta (NASDAQ: SRPT) said today that the Committee for Medicinal […]

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New Faces, Big Impact: Meet the Finalists For Xconomy’s Newcomer Award

Being a newcomer can mean a few different things: being new to the area (or newly back after being away) or new to an industry. The finalists for Xconomy’s Newcomer Award this year cover these definitions. One is a serial biotech entrepreneur who has now made an impact in multiple cities, most recently Boston. Another […]

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The 2018 Xconomy Awards Finalists Are…

[Updated 6/28/18 5:20pm ET; see note below] There was discussion. There was debate. After much wrangling, the editors and judges have narrowed down the impressive list of nearly 300 nominees for the 2018 Xconomy Awards to an all-star group of 64 finalists in 11 categories. There is no doubt that the overall caliber of the […]

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