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September 13, 2017

Today's Rundown

  1. Amicus ditches skin disorder drug after late-stage failure

  2. Rocket to merge with Inotek to bag Nasdaq listing, plots gene therapy trials

  3. BridgeBio Pharma gains $135M for genetic disease work

  4. [Sponsored] Leveraging Social Listening to Advance Alzheimer's Disease Research 

  5. ProQR spins out CNS assets to form RNA rare disease biotech

  6. Blood test could mean earlier diagnosis, better treatment for macular degeneration

  7. EASD: Sanofi's Toujeo bests Lantus at holding off hypoglycemia, studies show

Featured Story

Amicus ditches skin disorder drug after late-stage failure

A drug for rare skin disease epidermolysis bullosa that Amicus Therapeutics acquired in a $847 million deal two years ago has failed a key phase 3 trial.

Top Stories

Rocket to merge with Inotek to bag Nasdaq listing, plots gene therapy trials

Rocket Pharmaceuticals has landed a Nasdaq listing through a reverse merger. The deal will see the low-profile gene therapy startup merge with floundering Inotek Pharmaceuticals before embarking on a clutch of clinical trials.

BridgeBio Pharma gains $135M for genetic disease work

Early-stage California biotech BridgeBio Pharma has completed a $135 million financing round as it looks to push deeper into genetic disease R&D.

[Sponsored] Leveraging Social Listening to Advance Alzheimer's Disease Research

When it comes to healthcare we know that consumers actively participate in social media to seek information and find support. More than 40% say that information from social media affects how they cope with a chronic condition, their approach to diet and exercise, the physician they select and even their decision to seek a second opinion.

ProQR spins out CNS assets to form RNA rare disease biotech

ProQR has spun out its CNS assets to form Amylon Therapeutics. The offshoot starts life with seed funding and an RNA-based program targeting a rare genetic disease that causes strokes.

Blood test could mean earlier diagnosis, better treatment for macular degeneration

Age-related macular degeneration is a leading cause of vision loss in older adults but is challenging to catch early. Researchers at Massachusetts Eye and Ear have discovered a group of metabolites that could be used to improve diagnosis and treatment for AMD.

EASD: Sanofi's Toujeo bests Lantus at holding off hypoglycemia, studies show

Sanofi’s longer-acting basal insulin Toujeo, a follow-up to its own long-dominant product Lantus, beat the older drug at holding off hypoglycemia, both by day and by night. It’s an advantage Sanofi could push as it works to build up Toujeo scripts—and fights off biosimilar versions of Lantus.

Resources

[Webinar] Five ways to make your patient support program a success

Join this webinar to learn how to design, deliver, and measure a successful patient support program. Presented by Nareda Mills, Ashfield's SVP of Patient Services in the US, you’ll discover how to improve adherence and improve patient outcomes using 5 key methods.

[Whitepaper] The Impact of Contraceptive Requirements on Pediatric Clinical Trials

Children participating in pediatric clinical trials are included in the contraceptive requirements applied to women of child-bearing potential, creating significant challenges for sponsors, parents, investigators and study staff.

[Webinar] Within-Subject Clinical Trials: Intro to New Methods & Stat. Models

In this webinar, we will introduce the rapidly-evolving within-subject clinical trial (WSCT) methods for early phase evidence generation that require fewer people, less time, and usually less funding than traditional early phase methods.

Events

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