‘A missed opportunity’: The biggest takeaways from the FDA’s underwhelming gene therapy hearing

September 7, 2021 07:14 AM EDT

'A missed opportunity': The biggest takeaways from the FDA's underwhelming gene therapy hearing

Jason Mast

Editor

Researchers and executives hoping that the FDA’s two-day hearing on gene therapy would offer a clear direction for the field’s future came away disappointed.

For 16 hours, the agency listened to presentations and debated everything from AAV-driven cancers in mice to how concerned we should be about strange MRI results in patients given direct injections in the brain. But few firm conclusions or recommendations ever arrived.

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Why it Works: Manufacturing a Vaccine in a Multi-Product Facility.

Grand River Aseptic Manufacturing

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Alexander Lefterov/Endpoints News

September 27, 2021 07:12 AM EDTUpdated 09:08 AM

Coronavirus

In Focus

The coronavirus vaccine that the world forgot could still help save it

Jason Mast

Editor

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Habib Dable, Acceleron CEO

September 24, 2021 10:09 PM EDTUpdated September 27, 07:15 AM

Deals

R&D

Days of heated rumors culminate in a report that Acceleron is in advanced buyout talks

John Carroll

Editor & Founder

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 118,600+ biopharma pros reading Endpoints daily — and it's free.

ENDPOINTS CAREERS

Senior Analyst, Oncology

Brass Tacks Health

Remote

view job offer post your job now

September 28, 2021 10:49 AM EDT

R&D

Cell/Gene Tx

Safety fears force Pfizer to change pivotal DMD gene therapy trial protocol

Kyle Blankenship

Managing Editor

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 118,600+ biopharma pros reading Endpoints daily — and it's free.

Merck CEO Rob Davis

September 27, 2021 06:04 PM EDTUpdated 14 hours ago

Deals

Pharma

Merck emerges as lead bidder in potential Acceleron buyout with deal possible this week — report

Kyle Blankenship

Managing Editor

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

September 28, 2021 10:57 AM EDTUpdated 03:06 PM

R&D

In Focus

Contract research is having a moment right now. Will M&A splashes drive the industry to even greater heights?

Nicole DeFeudis

Editor

Contract research organizations are a fairly mysterious bunch. They’re typically considered the skilled laborers behind big drug development — the stage crews who run the trials behind some of the most (and least) successful data reveals in biopharma history.

But all that is changing.

This year, a couple of huge, out-of-the-blue M&A deals sounded the alarm on just how much money is flying around in this corner of the industry.

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

September 28, 2021 10:17 AM EDTUpdated 10:39 AM

Coronavirus

Covid-19 roundup: Pfizer/BioNTech submit vaccine data to FDA for younger children; Doctors kept prescribing hydroxychloroquine

Zachary Brennan

Senior Editor

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.

ENDPOINTS CAREERS

Senior Scientific Research Analyst

American Association for Cancer Research

Philadelphia, PA, USA

view job offer post your job now

From left to right: Mark Springel, Kristina Wang, Lin Ao, Soufiane Aboulhouda

September 28, 2021 08:00 AM EDTUpdated 10:07 AM

Startups

George Church, his students, and top VCs go nationwide with a biotech training camp

Paul Schloesser

Editorial Intern

One night last fall, Floris Engelhardt sat down in her Boston apartment and logged onto a Zoom call, armed with a comic and a vague idea about starting a biotech.

Engelhardt was joining a student-run “match night.” A postdoc at MIT’s Bathe BioNanoLab, where researchers use DNA and RNA like Lego blocks for nanometer-sized structures, Engelhardt wanted to find real-world applications for her work. She sketched out — literally — a plan to use DNA origami, a decade-old technique for precisely folding DNA, to make therapies.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 118,600+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

September 28, 2021 07:23 AM EDT

R&D

Coronavirus

Sanofi calls it quits on mRNA Covid-19 shots, scrapping vaccine from $3.2B Translate Bio buyout

Max Gelman

Editor

Sanofi is throwing in the towel on mRNA-based Covid-19 vaccines.

The French drugmaker will halt development on its unmodified mRNA Covid-19 shot despite what it said were positive Phase I/II results, a spokesperson told Endpoints News on Tuesday morning. Sanofi said the reason it’s stopping the Covid-19 mRNA program, developed in partnership with its new $3.2 billion acquisition Translate Bio, is because the market is too crowded.