Tag Archives: Friedreich’s ataxia

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AbbVie Walks From Voyager R&D Pact Spanning Alzheimer’s, Parkinson’s

A partnership between Voyager Therapeutics and AbbVie developing gene therapies for Alzheimer’s and Parkinson’s disease has ended without a drug for either disease reaching human testing. Voyager (NASDAQ: VYGR) announced the termination of the research pact after market close Monday. The Cambridge, MA-based biotech is keeping full rights to its technology for using engineered viruses […]

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A Baby’s Milestones and the Next Steps for Novartis in Gene Therapy

[Updated 4/10/2020, 3:24 p.m. See below.] Cameron Hewitt’s family celebrates the anniversary of her life not once, but twice yearly: Besides her September birthday, they commemorate a day in March—the anniversary of two-year-old Cameron receiving the gene therapy that gave her a new shot at life. Cameron was born with spinal muscular atrophy (SMA), a […]

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Zafgen’s Path Forward Is Reverse Merger With Chondrial Therapeutics

Zafgen stumbled in clinical trials testing its experimental therapies for metabolic diseases. Going forward, it will have a new name, new management, and a mitochondrial disorder drug from another company. Boston-based Zafgen (NASDAQ: ZFGN) has entered a merger agreement with Chondrial Therapeutics, a rare disease drug developer just entering the clinic. Chondrial shareholders will own […]

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Reata’s Kidney Drug Hits Goals of Key Study, Paves Way for FDA Filing

About half of all patients with the most severe form of Alport syndrome, a rare, genetic type of chronic kidney disease, will need dialysis or a transplant by age 25. On Monday, Reata Pharmaceuticals (NASDAQ: RETA) announced that its investigational drug for those with Alport syndrome, bardoxolone methyl (bard), met the main goal of a […]

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Bio Roundup: Ichnos Emerges, Beyond CAR-T, BridgeBio Bails & More

New brand-name drugs typically grab the life science headlines. A venture capital firm will invest tens of millions to develop one and a pharmaceutical company hopes to recoup hundreds of millions by selling it. But a shortage of one particular chemotherapy is showing the crucial role generic drugs play in healthcare. This week, The New […]

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PTC Dives Into Gene Therapy with $50M Agilis Biotherapeutics Deal

PTC Therapeutics is looking to gene therapy in a bid to broaden its pipeline, scooping up privately held Agilis Biotherapeutics in a cash and stock deal. South Plainfield, NJ-based PTC (NASDAQ: PTCT) will pay $50 million up front, plus $150 million in PTC shares, for Agilis. Agilis, based in Cambridge, MA, could get up to […]

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Sanofi Pulls Back from Voyager Gene Therapy Pact in Parkinson’s

A key study for Voyager Therapeutics and its experimental gene therapy for Parkinson’s disease will have to move ahead without help from pharmaceutical giant Sanofi, which has walked away from the deal. Global rights to Parkinson’s treatment VY-AADC01 are now squarely in the hands of Cambridge, MA-based Voyager (NASDAQ: VYGR). Two years ago, the company […]

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RaNA Expands, Joins Messenger RNA Drug Race With Shire Deal

RaNA Therapeutics is branching out. Today, the Cambridge, MA, startup is acquiring some assets from pharmaceutical giant Shire that immediately make it a player in an emerging, yet unproven field of drugmaking—messenger RNA therapeutics. Cambridge, MA-based RaNA has acquired a small, recently shuttered unit of Shire (NASDAQ: SHPG) devoted to making drugs with synthetic mRNA. […]

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Seeking A Fresh Start in Gene Therapy, Avalanche Merges With Annapurna

Avalanche Biotechnologies went public during a renaissance for gene therapy, but it’s been searching for a way forward ever since a disappointing trial led to an executive overhaul. The Menlo Park, CA, company has turned to a privately held startup with roots at Weill Cornell Medical College to turn things around. This morning, Avalanche (NASDAQ: […]

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WaVe, Voyager Get $170M Combined in Biotech’s Latest IPO Tests

Two privately held biotechs in the Boston area priced IPOs on Tuesday with similar estimates about their value. One of them hit its mark, thanks to some help from a few insiders, but the other fell a bit short. WaVe Life Sciences, of Cambridge, MA, priced 6,375,000 shares at $16 apiece, raising $102 million. That’s […]

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