00:47 , Jun 29, 2019 |  BioCentury  |  Finance

All eyes on new modality launches, cancer and rare disease data in 3Q19

While the typical summer lull means fewer catalysts in the third quarter, investors will be keeping a close eye on launches of new modality drugs. Of special interest to multiple buysiders is the launch of...
22:53 , Jun 28, 2019 |  BC Extra  |  Clinical News

Pfizer planning Phase III for DMD gene therapy, close on Sarepta's heels

Undeterred by two patient hospitalizations revealed in Phase Ib data Friday for its DMD gene therapy, Pfizer is moving forward with plans for a Phase III trial start in 1H20, setting up a tight race...
03:11 , Jun 7, 2019 |  BC Extra  |  Company News

Exonics takeout, expanded CRISPR deal vault Vertex into neuromuscular diseases

Via a pair of deals totaling $420 million in upfront payments late Thursday, Vertex expanded into neuromuscular diseases and bolstered its gene editing capabilities by acquiring Exonics, broadening its partnership with CRISPR Therapeutics and hiring...
22:34 , May 14, 2019 |  BC Extra  |  Clinical News

May 14 Clinical Quick Takes: Myovant, Solid, Minerva, Modus, Zealand

Myovant tumbles after Phase III uterine fibroid data  Myovant Sciences Ltd. (NYSE:MYOV) lost $4.39 (25%) to $12.83 Tuesday despite reporting that relugolix (TAK-385) met the primary endpoint of a better response rate than placebo in...
21:58 , Apr 26, 2019 |  BioCentury  |  Product Development

Rise of the independents, biotechs go to market

While product launch and commercialization has largely been the province of big biotech and big pharma, with smaller companies partnering to get their products over the finish line, BioCentury’s analysis of recent FDA approvals finds...
21:34 , Apr 16, 2019 |  BC Extra  |  Clinical News

Wave down on Phase I safety data for DMD therapy

Following a setback last week for its Huntington disease program, Wave lost $9.55 (28%) to $24.47 on Tuesday after the company announced plans to test two lower doses of suvodirsen in a Phase II/III trial...
20:46 , Apr 15, 2019 |  BC Extra  |  Company News

$1.2B Catalent-Paragon deal latest in race to lock up gene therapy manufacturing

The 6x multiple Catalent is paying for gene therapy manufacturer Paragon Bioservices suggests buyers believe gene therapy companies will remain heavily dependent on outsourcing as new therapies are approved in the next few years. Catalent...
22:42 , Apr 8, 2019 |  BC Extra  |  Company News

Audentes to develop vectorized antisense therapies with Nationwide

Audentes partnered with Nationwide Children's Hospital to develop antisense oligonucleotides delivered with the biotech's adeno-associated viral (AAV) vectors to treat muscular dystrophies. Audentes Therapeutics Inc. (NASDAQ:BOLD) Chairman and CEO Matt Patterson told BioCentury the decision...
18:47 , Apr 5, 2019 |  BC Week In Review  |  Clinical News

Sarepta planning NDA for DMD candidate casimersen

Sarepta said it plans to submit an NDA to FDA in mid-2019 for casimersen (SRP-4045) to treat Duchenne muscular dystrophy amenable to exon 45 skipping. The company will seek approval for casimersen based on a...
14:23 , Mar 19, 2019 |  BC Innovations  |  Distillery Therapeutics

Gene therapies targeting dystrophin exons for DMD

DISEASE CATEGORY: Musculoskeletal INDICATION: Muscular dystrophy Cell culture and mouse studies suggest CRISPR-Cas9-based gene therapies targeting exons 43 or 45 of dystrophin could help treat Duchenne muscular dystrophy (DMD) caused by dystrophin exon 44 deletion....