Gene Therapy

UPDATED: Gene therapy for 'bubble boy disease' sets stage for cure

Discovery

Penn scientists correct genetic flaw in mice fetuses, expanding in utero CRISPR reach to lung diseases

Startups, Venture

Serial biotech entrepreneur Michael Gilman takes his key to the $100M-plus club and goes all-in on drugging RNA
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calendar Protocols

Quick hits from around the biopharma web

Apr 18

Genfit wins BTD status at FDA for its lead drug for liver disease; Idorsia outlines $530M '19 spending plans on late-stage R&D

Apr 17

Turning Point unveils $166M IPO; Aquestive's riluzole film product is accepted for FDA review

Apr 16

NICE backs Akcea's Tegsedi; EMA restricts Lemtrada use while it probes safety concerns

Apr 15

Insys promotes CFO to the helm as Saeed Motahari hits the exit; Gelesis gets OK to market weight loss treatment

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person Peer Review

Weekly biopharma careers news report

Apr 18

Peer Review: KSQ woos Beni Wolf from Blueprint with CRISPR platform; AstraZeneca exec jumps to CNS biotech spun out of Takeda

Apr 12

Peer Review: Leaving Cerecor after a year, Peter Greenleaf starts a new CEO chapter at Aurinia — out to slay Restasis

Apr 5

Peer Review: Plotting US moves, Zealand Pharma poaches Alnylam's Emmanuel Dulac as new CEO

Mar 29

Sunao Manabe takes top job at Daiichi Sankyo in wake of AstraZeneca deal; CAR-T player Celyad promotes Filippo Petti to CEO

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Dollar Premium

Going gangbusters: The top 20 biotech platform deals by upfront cash

Another player in the gene therapy field is gunning for the Duchenne MD crown — and here’s what they're betting on

The top 20 preclinical biotech deals ranked by cash upfront reveals 3 big players — and some major league stinkers

Top 20 cancer drug deals: AstraZeneca just changed the global landscape for dealmaking in oncology

Team building. Pipeline revamp. Forward thrust. What does a decade of deals at Gilead tell us about Daniel O’Day’s first M&A moves?

After a slate of small, tack-on acquisitions, is Merck ready to pull the trigger on a big M&A deal?

By the numbers: Top M&A deals, VC investments and pacts point to a growing hunger for gene therapy deals

The $100M biotech club: Who's in, and what’s it take to join the top-performing group of biopharmas?

The high rollers: The top 20 blockbuster biopharma deals of the past decade

bullhorn Bioregnum

The view from John Carroll

Apr 17

Ned Sharpless is not playing with the FDA’s gold standard — any more than Scott Gottlieb did

Apr 15

SCOTUS just turned its back on Allergan’s legal maneuver to take a blow at inter partes review. What did you expect?

Apr 12

Going viral? J&J tries to spark a Twitterstorm for the latest data on its HIV med

Apr 8

Another player in the gene therapy field is gunning for the Duchenne MD crown — and here’s what they're betting on

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twitter Marco

Top biopharma links on Twitter - last updated ago

arrow-thick Most Viral

A startup's goal: Kill cancer with pills targeting RNA, not proteins - STAT

STAT a day

Xconomy: It's Tough to Find Drugs to Bind to RNA: Arrakis Nabs $75M to Help

Xconomy Frank Vinluan a day

ASCO 2019 - not a Marker of excellence

Evaluate.com a day

Genfit bags FDA breakthrough tag on route to phase 3 in PBC

FierceBiotech Nick Paul Taylor a day

Gene therapy for 'bubble boy disease' sets stage for cure

ENDPOINTS NEWS Natalie Grover a day

As Syphilis Invades Rural America, A Fraying Health Safety Net Is Failing To Stop It

Kaiser Health News khnterryb a day

Wotton replaces Gilman as CEO of cell, gene therapy startup Obsidian

FierceBiotech Nick Paul Taylor a day

Serial biotech entrepreneur Michael Gilman takes his key to the $100M-plus club and goes all-in on drugging RNA

ENDPOINTS NEWS John Carroll a day

HIV drugmakers ViiV, Gilead top 2018 pharma reputation survey

FiercePharma Beth Snyder Bulik a day

Google Has Warned Some Employees About A Measles Case On Its Campus

BuzzFeed News a day

timer Latest

FiercePharma

HIV drugmakers ViiV, Gilead top 2018 pharma reputation survey

ENDPOINTS NEWS

Serial biotech entrepreneur Michael Gilman takes his key to the $100M-plus club and goes all-in on drugging RNA

Apple

"Bubble Boys" cured in medical breakthrough using gene therapy - Bloomberg

FierceBiotech

Genfit bags FDA breakthrough tag on route to phase 3 in PBC

FierceBiotech

Wotton replaces Gilman as CEO of cell, gene therapy startup Obsidian

Pharmaphorum

Alexa: Tell me about Amazon's ambitions in healthcare - Pharmaphorum

Kaiser Health News

As Syphilis Invades Rural America, A Fraying Health Safety Net Is Failing To Stop It

Thefly

Ultragenyx Analyst Day saw positive DTX701, DTX301 updates, says Piper Jaffray RARE - The Fly

STAT

A startup's goal: Kill cancer with pills targeting RNA, not proteins - STAT

Seeking Alpha

CorMedix: Infected With Hype, Valuation Swollen

fire Hot This Week

1

Fast Edgar

Marker Therapeutics Form 8-K

2

NPR.org

First U.S. Patients Treated With CRISPR As Gene-Editing Human Trials Get Underway

3

STAT

A study of cancer test seemed like a triumph. But data were missing - STAT

4

STAT

It's just in mice! This scientist is calling out hype in science reporting - STAT

5

STAT

A startup's goal: Kill cancer with pills targeting RNA, not proteins - STAT

6

STAT

New research hints at the biology behind ketamine's antidepressant effects

7

Xconomy

Xconomy: It's Tough to Find Drugs to Bind to RNA: Arrakis Nabs $75M to Help

8

investors-hub

Biotech Values Post # 224656: Recent Biotech Bankruptcies/Liquidations/Dissolutions

9

STAT

Stanford clears three faculty members of 'CRISPR babies' involvement

10

STAT

Most people pay $5 per month or less for the drug. But some pay $11,002

Contact Endpoints News

You can also contact John Carroll, Editor at john@endpointsnews.com

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