Beyond AAV 1.0
The gene therapy boom was built on AAV delivery tech. And recent clinical work has highlighted a variety of major hurdles; payload limitations, safety threats on high doses, fears associated with waning durability for drugs that intended to be priced once-and-done. What are the leaders in the field doing to break down these barriers — and what could the future hold for AAV and beyond?
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Flattening the 'valley of death:' New tools in the hunt for better preclinical R&D
We all know the stats on preclinical programs. Precious few will survive the rigors of clinical trials. Endpoints talks with some of the star investigators doing the translational work behind some of the industry’s most ambitious pipeline efforts. What have they learned about setting up a solid preclinical strategy that gives them better odds of success in humans.go to website
Advancing Surrogate Endpoints in R&D
Surrogate endpoints are a hot topic in R&D. As an agreed substitute for clinical endpoints, they may make it more efficient to do research or allow for additional measures on patient satisfaction and more. We’ll discuss crafting surrogates as well as the use of digital tech and other aspects that will make it possible to track these endpoints more effectively in clinical trials.go to website
Reengineering for solid tumors
CAR-Ts revolutionized the way blood cancers can be treated, but still haven’t really caught on in the market as physicians and patients consider vein-to-vein times, cost and manufacturing snafus. Now a large group of scientists are leading a well-funded drive to adapt this technology for solid tumors, while making the whole process much more economical for patients and payers. The winners will help revolutionize the field. And the race to be first has inspired a furious sprint to the finish line.go to website
The big platform plays that have been in vogue. But for how long?
Moderna offers the kind of big upside even the largest institutional investors dream of. And now, you can already hear execs discussing who the next Moderna will be, and whether they can catch that wave themselves. We’ll analyze the numbers on venture capital and IPOs, looking at how platforms are performing compared to smaller groups of drug assets. And how will that influence financing trends for years to come?go to website
The neurosciences comeback: After a lengthy chill, Big Pharma is warming back up to neuro.
Neurosciences became the stepchild of the pharma industry after a series of ugly setbacks in the clinic drove away the major players. But now, considering the advances in genetics and some new work on old ideas, some of the biggest outfits are driving a comeback of sorts as they team up or buy out some of the upstarts in this space. Endpoints examines the trend, and where it appears we’re headed now.go to website
After the pandemic, antibody R&D remains key
The onslaught of a global pandemic triggered a frenzy of R&D activity in the industry. But where we saw a revolution in vaccine development in record time, finding new drugs to effectively fight the virus remained difficult and fraught with failure and half-measures of success. Nevertheless, a group of players is still actively engaged in developing a new generation of antibodies to fight this virus, and others to come. We’ll talk to the key leaders in the field about where this is headed, and how it may more broadly affect everyone doing antibody work.go to website
After a decade of consolidation, who rules the CRO roost (Top 10 & Trends)
Over the past decade, we’ve seen a group of well-financed CROs backed by private equity consolidate and then set out to build new hubs around the world. More recently, we’ve also seen new CROs join the game in China; and both the opportunities in a booming drug development industry and the competition have never been more pronounced. Nicole DeFeudis finds out who the top 10 players and what the key trends are here.
A new class of startups is scouting for deals - on both sides of the Pacific
It used to be that executing a global strategy meant finding a partner for the European market. Now, China has completely changed the way the world works. Startups have been in-licensing for years in a growing set of diseases. And now they are outlicensing to the US and Europe. What happens as more sophisticated R&D organizations go after novel drug targets? Lets find out.go to website
The FDA in a post-Covid world
Covid-19 changed the R&D world. And it’s not going back to what it was before the pandemic. That’s particularly true at the FDA, which worked with developers to make it possible to continue trials when sites were shuttered around the world. We’ll explore what’s likely to stick now that the pandemic is winding down.go to website