BioCentury | Sep 10, 2020
Finance

Casma to advance muscle dystrophy candidate into the clinic with $50M series B

...In the case of muscular dystrophy, missing dystrophin...
...Rock Ventures Eventide Asset Management Schroder Adveq Mucolipin 1 (TRPML1) (MCOLN1) Dystrophin Myeloid...
BioCentury | Jun 8, 2020
Product Development

Sarepta heading toward pivotal trial of first limb-girdle muscular dystrophy gene therapy

...study of SRP-9001; Roche (SIX:ROG; OTCQX:RHHBY) has its ex-U.S. rights (see “Deal Could Extend Beyond Micro-Dystrophin”...
...its market cap is above $12 billion. Targets SGCB - sarcoglycan β Paul Bonanos, Associate Editor MYO-101 SRP-9001, rAAVrh74.MHCK7.micro-Dystrophin Sarepta...
BioCentury | May 15, 2020
Product Development

12-month data set up Pfizer DMD gene therapy for Phase III, but SAEs may give Sarepta safety edge

...high-dose cohort and efficacy data out to 12 months. Biopsy data measuring the expression of mini-dystrophin...
...Roche (SIX:ROG; OTCQX:RHHBY), because the companies use different methodologies for measuring mini-dystrophin expression. Pfizer measured mini-dystrophin...
...for the recombinant AAV9 gene therapy that carries a truncated human dystrophin gene, known as mini-dystrophin...
BioCentury | Jan 25, 2020
Product Development

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

...Zioncheck cited Roche’s December co-development deal with Sarepta as an example, which involved the biotech’s micro-dystrophin-targeting...
...and Vyondys 53 golodirsen (see “Roche’s ex-U.S. Deal for Sarepta DMD Programs Could Extend Beyond Micro-Dystrophin”...
BioCentury | Jan 23, 2020
Company News

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

...as justification for their decisions. FDA’s approval of Exondys, based on the surrogate endpoint of dystrophin...
...System Drugs Advisory Committee that Sarepta had not shown substantial evidence that the therapy induces dystrophin...
...approval letter. “From a regulatory perspective, this precedent establishes that FDA considered the change in dystrophin...
BioCentury | Jan 4, 2020
Finance

All’s well that ends well

...dystrophy because interim results of a Phase I trial showed no change from baseline in dystrophin...
BioCentury | Jan 4, 2020
Product Development

More Waves to come on stereopure antisense oligo tech

...dystrophy (DMD) candidate suvodirsen, a stereopure antisense oligonucleotide that induces skipping of exon 51 in dystrophin...
...mRNA, as well as preclinical candidate WVE-N531, which skips exon 53. Suvodirsen failed to change dystrophin...
BioCentury | Dec 23, 2019
Company News

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

...this fall, Roche broadened its commitment to gene therapy by obtaining ex-U.S. rights to Sarepta’s micro-dystrophin-targeting...
...another $1.7 billion in milestones, plus royalties. The parties will share costs of developing SRP-9001 (rAAVrh74.MHCK7.micro-Dystrophin...
...products including casimersen, SRP-5051 and gene therapies that express dystrophin or act on a dysfunctional dystrophin...
BioCentury | Dec 16, 2019
Product Development

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

...NASDAQ:WVE) fell $20.81 (55%) to $16.79 on Monday after announcing that suvodirsen failed to change dystrophin...
...dropping its programs for suvodirsen, a stereopure antisense oligonucleotide that induces exon 51 skipping in dystrophin...
...Exondys 51 eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin...
BioCentury | Dec 13, 2019
Company News

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

...per 10 ml vial. The accelerated approval is based on the surrogate endpoint of increased dystrophin...
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