pharma


September 2017 with Nathan and Laura: Venter Blunder, RNAi Returns, and Monthly Science Moments

To honor Laura's pentametric thirst,

We write the summary today in verse.

 

Was it a quake that had no epicenter,

That silly paper out by J. Craig Venter?

 

And after years of silencing the market

Has RNAi at last knocked out its target?

 

Then Nathan gives to yuppies devil's choice.

Which one libs: gluten dough or GMOs?

 

The First In-Human Gene Editing Trial in the U.S. - And It’s Not with CRISPR

The challenge for the first ever in-human gene editing trial, according to today’s guest, is with the delivery to the body.

“At the moment, the easiest place to deliver your gene or genome editing is to the liver, using AAV which are viruses that seek out and go to the liver cells," says Sandy Macrae, the CEO of Sangamo Therapeutics.

Sangamo is known for two things: They have pioneered the commercialization of an older gene editing technology called Zinc Fingers. And they have done a lot of work in the area of HIV.

Today, Sangamo is enrolling patients in a new trial which they say will be the first "in-vivo" trial using their Zinc Fingers for patients with hemophilia B, Hunter syndrome, and Hurler syndrome. The former gene editing work with the T cells of HIV patients, Sandy says, was done “ex-vivo”, or outside the body.

So why is Sangamo still using Zinc Fingers in the age of CRISPR? Sandy says that the older technology is much better developed for medical applications and is safer. The company has been able to get their off target effects to below the level of detection.

“When I was doing my postdoc, I would have used CRISPR. It’s better if you’re just wanting an easy experiment that isn’t about making a medicine but just getting a quick answer,” he says.

Because Sangamo has been the sole commercial developer of Zinc Fingers with not a lot of intellectual property dispute, the technology didn’t make the big PR splash that CRISPR has—nor, at the same time, did it generate all the fear.

We finish the interview with a question about what was the result of all Sangamo's work in HIV over the years.

The Last Major Disease To Be Studied? Ron Davis of Stanford Thinks So

Let’s say you’re a biomedical researcher looking for a place to make your mark. You find out that there is still a major disease that affects more than 2 million people in the US, and we still know virtually nothing about this disease at the molecular level. Wouldn't that stand out?

It certainly has to today's guest, Ron Davis, who is also a father searching for answers for his son. Ron has been the Director of the Stanford Genome Technology Center for decades. He collaborated on the first DNA microarray and made a major contribution to the Human Genome Project. For five years now, Ron has directed his comprehensive skill set in bioengineering--and his vast connections--to work on a cure for ME/CFS, or Chronic Fatigue Syndrome, a disease which has ravished his son, Whitney Dafoe.

With no funding from the NIH so far (he says they're not good at starting things), Ron is working to characterize the disease at the molecular level. A new device developed at his center that he calls a "nano needle" could enable the first definitive diagnostic test for patients with CFS.

The history of this disease is of patients desperate with hope but always facing a major stigma. Many medical professionals are still not on board with diagnosing a patient with CFS. Ron says this stigma and lack of interest by the research community has created a big chance.

“This is a tremendous opportunity. Here’s a major disease which at the molecular level you don’t know anything about. This has got to be the last disease like this."

Find an extensive recent written interview with Ron here.

Proposed NIH Cuts, Undermining GINA, and Game Changing Drugs: March 2017 with Nathan and Laura

The largest cut to NIH budget ever, rolling back genetic non-discriminatory law—the bad news continues to roll from Washington. But there was great news this month as well.

Both Nathan and Laura are fuming about HR 1313, or a Republican bill to roll back GINA protections. Laura points out that the proposed law builds on an exemption in GINA for wellness programs—a category difficult to define. And Nathan reminds us that families and children could be hurt by the new bill. Theral asks since when did privacy become partisan? GINA passed in ’08 with a vote of 95-0 in the Senate, 414-1 in the House (Ron Paul playing the weirdo there), and it was signed by George Bush.

Then on to some “game changing” drugs for multiple sclerosis and eczema and a successful gene therapy trial for severe sickle cell anemia. Not only are there new therapies, drug manufacturers seem to be getting the message on pricing.

The Story of Geisinger and Doing Genomic Medicine at the Right Pace

Mike Murray and the crew over at Geisinger are making the implementation of genomic medicine look down right easy.

In today’s interview, Mike explains GenomeFIRST Medicine, a program at the Geisinger Health System in Pennsylvania to offer care “that is based on an individual’s DNA sequence.” The healthcare provider boasts its own biobank and has partnered up with Regeneron’s Genome Center to offer exome screening to self selected patients. As of DNA Day last year, April 25th 2016, 100,000 recruits had signed up.

What has made Geisinger, who was selected to join the nation Precision Medicine Initiative, so successful with genomics? Mike points to the leadership.

“We have incredible support from the highest levels of the organization. As we’ve rolled out genomics, they are supportive and interested. As long as we’re there to explain what we’re doing and why we’re doing it, we have them on our side,” he says.

Has there been any pushback from doctors or patients?

Mike says one of the challenges they hadn’t really considered has been a “naming issue.” Sometimes one of the variants a patient tests positive for “puts their clinical story together.” But other patients may test positive for something like lynch syndrome, for example, who haven’t really had any problems.

“They really don’t have lynch syndrome, “ he says, "they have a genetic variant that goes with it. Until they have problems associated with it, they just have risk for lynch syndrome. So the problem is how do you keep something like that high enough on the radar that people and their providers know what to look for, but not so high that insurers or other entities might say, we’re going to treat them like our standard approach to lynch syndrome?”

In fact, Mike and his team have thought quite far through this challenge of how to report genomic findings back to patients. He explains what they’ve come up with in this beautifully clear interview about one of America’s most genomically experienced and progressive health systems.

One Less Voice on the BioBeat - Luke Timmerman Ends Stint with Xconomy

Monday mornings won’t be the same in biotech.

Luke Timmerman announced last week that he’ll no longer be writing his Monday morning BioBeat column or serving as National Biotech Editor at Xconomy.

Timmerman’s voice has stood out in our industry for consistency, optimism, pragmatism . . .  and great sports analogies.  

 

Source: Xconomy

Clinical Trials and Social Media with Craig Lipset, Pfizer

Podcast Sponsor: ScienceExchange.com- The Scientific Services Marketplace

Guests:

Craig Lipset, Pfizer Bio and Contact Info

Bridging Listen (5:48) Bridging clinical research with eHealth

<img src="/images/speaker.jpg" alt=What happens when trial patients connect with each other online?"/> Listen (2:09) What happens when trial patients connect online?

How have patients changed Listen (4:01) How have patients changed?

What has Pfizer changed Listen (2:51) What has Pfizer changed?

Challenges with mobile health Listen (4:09) Challenges with mobile health?

How is Pfizer using social media Listen (4:58) How is Pfizer using social media?

Social media guidance Listen (9:44) Social media guidance

Pfizer and innovation Listen (1:28) Pfizer and innovation

Clinical trials in 5 years Listen (5:14) Clinical trials in 5 years

We talk with Craig Lipset of Pfizer about how social media is changing clinical trials. Craig has good support at Pfizer to launch innovative trial programs. At Pfizer he is bridging clinical research with eHealth technologies and exploring the opportunity to further inform and empower both patients and providers in the age of social media. Craig served as Vice President of Program Management at Adnexus Therapeutics and was part of the founding management team for Perceptive Informatics. He is the 2010 recipient of the DIA's Outstanding Service Award.

Start-up 'Helps Out' FDA: Brian Overstreet with AdverseEvents.com

(Read our blog and watch a video about visiting Brian in Healdsburg, CA "Having Your Wine and Drinking It Too")

Podcast Sponsor:

Open Science Summit, Oct 22-23, Mountain View, CA Register Today

Guest:

Brian Overstreet, President, Adverse Events, Inc. Bio and Contact Info

FDA site offers no easy way to research side effects Listen (5:39) FDA site offers no easy way to research side effects

What does RxFilter(TM) do? Listen (3:42) What does RxFilter do

Site free for patients Listen (4:21) Site free for patients

Why Healdsburg Listen (3:56) Why Healdsburg?

Living a dream Listen (0:47) Living a dream

Over 500,000 adverse events are reported for medications yearly to the FDA; estimated to be only 10% of all actual adverse events. If you experience a negative side effect taking a drug and want to report it, or if you are a drug developer and you want to research negative side effects, you can at FDA.gov in a section called Adverse Event Reporting System. There you will find tens of thousands of adverse events listed and connected to thousands of drugs. Or you can go to AdverseEvents.com, a new website that provides accurate, real-time information on adverse drug events that are reported to the FDA. Using a unique data sourcing method, the site is a user-friendly, fully searchable, database of over 4,000 approved medications. Joining us today is the President of AdverseEvents, Brian Overstreet. Brian just launched his new company at Health 2.0 on Sept 26-27.



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