The Tachi Yamada legacy tree continues to grow with a new gene editing biotech from Jim Wilson

September 9, 2021 07:00 AM EDTUpdated September 10, 02:18 PM

The Tachi Yamada legacy tree continues to grow with a new gene editing biotech from Jim Wilson

Max Gelman

Editor

Jim Wilson has been busy lately.

Back in May, the AAV pioneer from the University of Pennsylvania launched a second-generation gene therapy biotech, G2 Bio Companies, and just last week unveiled a nonprofit aiming to serve rare disease populations where the pharma model provides little economic incentive. Both efforts saw significant involvement from the late Tachi Yamada, who continues to inspire those working at his legacy startups.

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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.

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.

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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.

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Brass Tacks Health

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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.

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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.

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Brian Hubbard, Anji Pharmacetuticals CEO

September 28, 2021 06:30 AM EDTUpdated 09:50 AM

Financing

R&D

Looking to rewrite the rules of drug licensing, startup Anji is on the hunt for 'dynamic equity' joint ventures

Kyle Blankenship

Managing Editor

Licensing is one of the most common ways big drugmakers leverage biotech innovation to drive gains across their pipelines — and the structure of those deals is pretty well established. But one biotech with home bases in China and the US thinks it may have a better way.

On Tuesday, Cambridge-based biotech Anji Pharma closed a $70 million Series B with two late-stage molecules in the fold and a mission to rewrite the rules of drug licensing through what it calls “dynamic equity” deals and a joint venture-heavy game plan. The round was funded in whole by Chinese hedge fund CR Capital.

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.

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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.

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.