#ASH20: David Altshuler and the Vertex/CRISPR team hit a historic milestone in the hunt for a curative gene editing approach to sickle cell disease, beta thalassemia

David Altshuler (Endpoints file photo)

December 5, 2020 12:30 PM ESTUpdated December 6, 03:36 PM

#ASH20: David Altshuler and the Vertex/CRISPR team hit a historic milestone in the hunt for a curative gene editing approach to sickle cell disease, beta thalassemia

John Carroll

Editor & Founder

Back in the mid-80s, David Altshuler worked in Richard Mulligan’s famous Harvard lab, focused on hemoglobinopathies and the glimmering promise of a gene therapy for sickle cell disease.

Today, 35 years later, the Broad co-founder and chief scientist at Vertex and his partners at CRISPR Therapeutics have reached a sign post on the world’s most advanced gene editing program that points to a potential cure for both sickle cell disease and beta thalassemia. And Altshuler can see the finish line ahead.

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Data Literacy: The Foundation for Modern Trial Execution

Kristin Mauri

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

January 27, 2021 10:11 AM EST

R&D

Pfizer's big blockbuster Xeljanz flunks its post-marketing safety study, renewing harsh questions for JAK class

Jason Mast

Editor

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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January 27, 2021 07:02 AM ESTUpdated 11:05 AM

Coronavirus

Covid-19 roundup: EU and AstraZeneca trade blows over slowdowns; Unusual unions pop up to test antibodies, vaccines

Amber Tong

Senior Editor

Max Gelman

Associate Editor

Nicole DeFeudis

Associate Editor

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Pascal Soriot, AP

January 27, 2021 09:21 AM EST

Bioregnum

AstraZeneca CEO Pascal Soriot severs an unusual board connection, steering clear of conflicts while retaining important alliances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

January 25, 2021 04:54 PM ESTUpdated January 26, 07:11 PM

In Focus

After 3 deaths rock the field, gene therapy researchers contemplate AAV's future

Jason Mast

Editor

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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January 25, 2021 06:45 AM ESTUpdated 07:07 AM

Coronavirus

Merck scraps Covid-19 vaccine programs after they fail to measure up on efficacy in another major setback in the global fight

John Carroll

Editor & Founder

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Anthony Fauci, NIAID director (AP Images)

January 27, 2021 03:40 PM EST

Coronavirus

As new Covid-19 task force gets underway, threat looms of vaccine, monoclonal antibody-resistant variants

Jason Mast

Editor

Hours before President Biden’s Covid-19 team gave their first virtual press conference, the famed AIDS researcher David Ho delivered concerning news in a new pre-print: SARS-CoV-2 B.1.351, the variant that emerged in South Africa, is “markedly more resistant” to antibodies from convalescent plasma and vaccinated individuals.

The news for several monoclonal antibodies, including Eli Lilly’s bamlanivimab, was even worse: Their ability to neutralize was “completely or markedly abolished,” Ho wrote. Lilly’s antibody cocktail, which was just shown to dramatically reduce the risk of hospitalizations or death, also became far less potent.

Jackie Fouse, Agios CEO

January 26, 2021 04:55 PM ESTUpdated 05:23 PM

R&D

Agios scores its second positive round of data for its lead pipeline drug — but that won't answer the stubborn questions that surround this program

John Carroll

Editor & Founder

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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January 26, 2021 10:58 AM ESTUpdated 06:09 PM

Manufacturing

Vir's CMO says he's surprised that a low dose of their hepatitis B drug appears promising in early slice of data — shares soar

Conner Mitchell

Associate Editor

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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