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BioWorld - Friday, December 12, 2025
Home » Newsletters » BioWorld

BioWorld

March 5, 2018

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Bench Press: BioWorld looks at translational medicine

Synthetic biologists have managed to design multipass transmembrane proteins whose structures were stable, and very close to their computationally predicted structures. The authors said their work "pave[s] the way for the design of multispan membrane proteins with new functions." Read More

Earnings

Sarepta Therapeutics Inc., of Cambridge, Mass., reported net product sales of Duchenne muscular dystrophy drug Exondys 51 (eteplirsen) of $57.3 million in the fourth quarter of 2017. Read More

In the clinics

Ra Pharmaceuticals Inc., of Cambridge, Mass., plans to present the design of its phase II trial testing RA-101495 SC, an inhibitor of complement component 5, in patients with generalized myasthenia gravis at the 70th Annual American Academy of Neurology in Los Angeles next month. Read More

Other news to note

Sanofi SA, of Paris, said the FDA has accepted for review the supplemental biologics license application of Dupixent (dupilumab) as an add-on maintenance treatment in certain adults and adolescents (12 and older) with moderate to severe asthma. The PDUFA target action date is Oct. 20. Read More

Financings

Carsgen Therapeutics Co. Ltd., of Shanghai, said it entered a framework agreement under which it would receive a $60 million equity investment from existing and new investors. Read More

Indian pharma companies urged to enter ties-ups with digital tech, data firms

HYDERABAD, India – Indian pharmaceutical companies are taking baby steps toward a greater collaboration with digital tech firms and data analytics, but they may not be moving fast enough. Delegates at the BioAsia 2018 conference said such collaborations should speed up to tap into that significant innovation potential. Read More

Glioblastoma CAR T cell can induce expression of its target

In the Feb. 28, 2018, issue of Science Translational Medicine, researchers from the University of North Carolina at Chapel Hill describe an antigen that might enable the cells' use in the majority of cases of glioblastoma multiforme. Read More

U.K. Prime Minister willing to seek associate membership to maintain post-Brexit EMA ties

LONDON – In a long-awaited speech, Prime Minister Theresa May has committed to the U.K. remaining part of the EMA's system of drug regulation and to making a financial contribution to the agency after Brexit. Read More

Verona stock rises on preliminary cystic fibrosis data

DUBLIN – Shares in Verona Pharma plc rose as much as 21 percent Friday as the company reported that a single dose of its dual phosphodiesterase (PDE) 3 and 4 inhibitor, RPL-554, improved lung function in cystic fibrosis (CF) patients. Read More

Biogen and Abbvie, unable to assess risks to relapsed MS patients, withdraw Zinbryta

Planning for a global market withdrawal of the multiple sclerosis (MS) drug Zinbryta (daclizumab) is underway after reports of seven cases of serious inflammatory brain disorders, including encephalitis and meningoencephalitis, triggered "an urgent review" by the EMA of the medicine. Read More

Roche to take IONIS-HTTRx pivotal 'quickly' following early efficacy signal in HD

Stanley Crooke, chairman and CEO of Ionis Pharmaceuticals Inc., was buoyant on a conference call Friday as the company reported details from the phase I/II study of IONIS-HTTRx (RG-6042) in individuals with Huntington's disease (HD). Read More

Biosimilars on the rise in India; technology, capital remain challenges

HYDERABAD, India – Companies are increasingly willing to invest in biosimilar development and manufacturing in India, despite a spate of regulatory hurdles and a shortage of capital that remain visible. Read More

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