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Drug Development Is Cheap And Easy, Said No One Ever

March 15, 2018Research

The AveXis announcement ushers in a new and exciting era of clinical trials that attack the root cause of Rett Syndrome. Without a lot of precedent with gene therapy just yet, it’s hard to know what FDA will require and what the development timelines will look like. In my last…

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Making It Count

March 12, 2018Fundraising, Research

I’ve blogged about how RSRT solicits and reviews proposals. But what happens once an award is made? For most funding organizations making the award is the last step in the process. The MO (modus operandi) is “congratulations on the award, good luck and send us a report in a year…

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A Rigorous Peer Review: Why You Should Care

March 6, 2018Research

Peer review, it’s a term you’ve probably heard numerous times. But what exactly is it? As the name implies, peer review is the evaluation of work by others in the same field as a means to ensure high quality standards. Two main examples are: 1) peer review used by the…

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Vision and Guts

March 4, 2018Research

Our recent announcement that AveXis is planning to initiate a gene therapy clinical trial within a year (pending FDA approval) is exciting and worthy of celebration. Getting us to this point took years of hard work and millions of dollars raised and thoughtfully awarded. It is truly a moment worthy…

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AveXis Reports on Rett Gene Therapy Program: AVXS-201

February 28, 2018In The News, Research

AveXis Reports on the Rett Syndrome Gene Therapy Program – Clinical Trial Likely to Start Within a Year In June of 2017 AveXis announced that based on encouraging data generated by RSRT’s Gene Therapy Consortium it would advance the lead MECP2 gene therapy candidate toward clinical trials. Yesterday the company…

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Funding Collaboration Not Competition

February 26, 2018Research, Roadmap

Andrew Carnegie, the 19th century business magnate and philanthropist, once said, “It is more difficult to give money away intelligently than to earn it in the first place”. I wholeheartedly agree and here’s why. Making wise and effective funding decisions requires a rigorous process and unwavering attention on multiple fronts:…

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The Road to FDA Approval

February 7, 2018Research

First, what is the FDA and what do they do? Many people assume that the FDA develops treatments, or that they choose treatments or indications for development, but that’s not true. They do not develop treatments, they do not fund treatment development, and they do not choose the patient groups…

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