BioCentury | Jan 25, 2020
Product Development

A milestone year for Roche’s CNS gambit could kick off its plan for the decade

A ramped up focus on CNS that has been long in the making could start to pay off this year for Roche, which has two programs slated for launch and four others with late-stage readouts...
BioCentury | Jan 23, 2020
Company News

Disparities in FDA decisions on Sarepta DMD therapy raise questions about integrity of review process

Documents released Wednesday by FDA reveal unexplained discrepancies between an August complete response letter for Vyondys 53 golodirsen and December’s surprising accelerated approval of the therapy. The differences between FDA’s skepticism about the development program...
BioCentury | Dec 23, 2019
Company News

Roche’s ex-U.S. deal for Sarepta DMD programs could extend beyond micro-dystrophin gene therapy

After weathering one clinical failure in DMD this fall, Roche broadened its commitment to gene therapy by obtaining ex-U.S. rights to Sarepta’s micro-dystrophin-targeting program in a deal worth more than $1.1 billion up front. Roche...
BioCentury | Dec 16, 2019
Product Development

Wave crashes on DMD data as investors await Huntington’s readout

Wave’s decision to discontinue its two Duchenne muscular dystrophy programs raised broader investor concerns about the company’s stereopure oligonucleotide platform and sent shares down more than 50% on Monday. Another readout for the platform in...
BioCentury | Dec 13, 2019
Company News

Sarepta wins approval for second exon-skipping DMD therapy after disputing FDA decision

FDA reversed an August decision that rebuffed Sarepta’s Vyondys 53 golodirsen, granting accelerated approval to the therapy to treat Duchenne muscular dystrophy in patients amenable to exon 53 skipping. The decision came as a surprise...
BioCentury | Nov 23, 2019
Politics, Policy & Law

Debating the fate of innovation

No one believes that H.R. 3, the Democratic House leadership’s drug pricing bill, will be enacted into law , but that hasn’t stopped it from precipitating frenzied debates. The bill has become a strawman for advocates...
BioCentury | Oct 5, 2019

Seeking bright spots in 4Q19 amid continued underperformance

Biotech watchers are hoping a busy fourth quarter of data readouts, regulatory actions and IPOs will provide some bright spots to counteract the sector’s continued overall underperformance, which is not expected to improve in 2020....
BioCentury | Oct 4, 2019
Clinical News

Limb-girdle muscular dystrophy gene therapy data lift Sarepta

Early data from a Phase I/II study of SRP-9003 to treat a form of limb-girdle muscular dystrophy showed the gene therapy led to functional improvements in all three patients in a low-dose cohort at nine...
BioCentury | Sep 26, 2019
Emerging Company Profile

Edgewise: breaking away from dystrophin for muscular dystrophy

With a lead compound that staves off use-induced muscle damage, Edgewise thinks it can compete with dystrophin replacement therapies for muscular dystrophy. Edgewise Therapeutics emerged from stealth this month with a $50 million series B...
BioCentury | Aug 19, 2019
Company News

Sarepta sinks after FDA rejects DMD therapy

Sarepta dropped $15.61 (13%) to $104.70 in after hours trading Monday, losing over $1.1 billion in market cap, after FDA issued a complete response letter for golodirsen, the biotech's second exon skipping therapy for Duchenne...
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