BioCentury | Mar 6, 2021
Product Development

The pace of new modality approvals is picking up

...debate; with approvals based on the biomarker dystrophin...
BioCentury | Feb 5, 2021
Product Development

Despite miss in biomarker study, PTC to discuss FDA approval pathway based on totality of data

...approval.PTC Therapeutics Inc. (NASDAQ:PTCT) reported Thursday after market close that Translarna ataluren failed to raise dystrophin...
...20 patients ages 2-7 in the intent-to-treat population of Study 045. On the primary endpoint, dystrophin...
...to present the results, CEO Stuart Peltz pointed out that after excluding two non-evaluable patients, IHC-measured dystrophin...
BioCentury | Jan 9, 2021
Finance

Targeted oncology is having a moment, again

...although SRP-9001 met the biological endpoint of micro-dystrophin...
BioCentury | Jan 9, 2021
Product Development

Possible factors behind Sarepta’s DMD miss

...of a Phase III study of its micro-dystrophin...
...met the primary biological endpoint of increasing micro-dystrophin...
...vary, but data from animal models suggest micro-dystrophin...
BioCentury | Jan 8, 2021
Product Development

Jan. 7 Quick Takes: Sarepta tumbles after hours on DMD miss; plus InnoCare, AstraZeneca, Astellas and Autolus

...41 patients with Duchenne muscular dystrophy. The study did meet the primary biological endpoint of micro-dystrophin...
...also will cut headcount by 20%.TARGETSBtk – Bruton’s tyrosine kinaseSGLT2 – Sodium-glucose cotransporter 2 Stephen Hansen RG6356 (SRP-9001, rAAVrh74.MHCK7.micro-Dystrophin) InnoCare...
BioCentury | Dec 12, 2020
Product Development

Dec. 11 Quick Takes: AZ to run combo trial with Sputnik; plus Phase III hits for Lilly, AbbVie and TG, data from Immutep, NVS, Mycovia, Sarepta and Greenwich

...SRP-5051 to treat DMD patients amenable to exon 51 skipping showed higher tissue exposure and dystrophin...
BioCentury | Nov 21, 2020
Product Development

Could an approval of Biogen’s Alzheimer’s therapy still be an option?

...Exondys approval on the surrogate endpoint of dystrophin...
BioCentury | Nov 20, 2020
Deals

After three-target Lilly deal, Precision BioSciences hunting for more partnerships

...in the EU to treat nonsense mutation DMD.Sarepta is also developing a gene therapy targeting micro-dystrophin...
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...
...Asset Management Schroder Adveq Calporta Therapeutics Inc. 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...
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