FDA posed a sprawl­ing set of ques­tions for two-day gene ther­a­py hear­ing. Here's what com­pa­nies and ex­perts will be watch­ing for

Gene therapy is having a moment. Once confined to academic labs and a couple of companies, the field now encompasses hundreds of trials, dozens of companies and billions of investment dollars.

With greater power, though, comes greater responsibility. The FDA is asking a panel of outside experts to try and define those responsibilities as they debate a lengthy list of 15 questions Thursday and Friday, in a two-day marathon meeting on the future of a field that has promised one-time treatments for some of the most famous and fatal diseases known to medicine.

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

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

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

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.

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Habib Dable, Acceleron CEO

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

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.

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

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.

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Merck CEO Rob Davis

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

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.

Peter Marks (Jim Lo Scalzo/Pool via AP Images)

Tur­moil at CBER: Pe­ter Marks grabs con­trol of FDA's Of­fice of Vac­cines ahead of 2 key ca­reer leader de­par­tures

FDA’s top vaccine official Peter Marks is pulling the plug on a months-long transition for two top career vaccine officials who abruptly called it quits in late August.

Marion Gruber, director of the FDA’s Office of Vaccines Research & Review and 32-year veteran of the agency, her deputy director Phil Krause, announced their departures and then raised concerns with Covid-19 booster shots ahead of and during a recent Covid-19 booster vaccine advisory committee.

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

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.

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

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

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.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

Seiz­ing Aduhelm prece­dent, Bio­gen, Ei­sai get the ball rolling on an­oth­er ac­cel­er­at­ed Alzheimer's ap­proval

Biogen — together with partners at Eisai — will be the first to breeze down that Alzheimer’s trail it blazed with the FDA’s historic and controversial accelerated approval of Aduhelm.

Eisai disclosed it’s initiated a rolling BLA submission for lecanemab, or BAN2401, the anti-amyloid beta antibody that Biogen has long touted as a follow-on to Aduhelm (then aducanumab). Following the precedent that the agency has now set, the two companies are gunning for an OK based on data suggesting that the drug could lower amyloid beta plaques.