Pfizer’s DMD gene therapy rises

Today’s Big News

Nov 1, 2023

ESMO: GLP-1s have taken over the cultural zeitgeist. 33,000 oncologists have something to say about that 


FDA advisers wave through first CRISPR-based therapy, satisfied with off-target tests


After Sarepta miss, Pfizer’s DMD gene therapy is ‘main game in town,’ CSO says 


GSK loses its STING as part of phase 1 cleanout


AstraZeneca takes 22% stake in Cellectis as part of collab for 10 cell and gene therapies


Bioluminescence enters biotech venture spotlight with almost $500M under management 


Sanofi pays $30M for first look at MeiraGTx's data and a shot to brew up deals

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Featured

ESMO: GLP-1s have taken over the cultural zeitgeist. 33,000 oncologists have something to say about that

If you’ve been living under a rock for the past several years, you probably still know what a GLP-1 inhibitor is—at least by brand name. But the 33,000 attendees at the European Society for Medical Oncology would like to remind you that there’s plenty of breakthroughs in cancer worthy of the same kind of fanfare.
 

Top Stories

FDA advisers wave through first CRISPR-based therapy, satisfied with off-target tests

FDA advisers appear satisfied with CRISPR Therapeutics and Vertex Pharmaceuticals’ assessment of potential off-target effects from their CRISPR-based gene therapy exa-cel, quelling the top concern from the FDA as an approval deadline nears. 

After Sarepta miss, Pfizer’s DMD gene therapy is ‘main game in town,’ CSO says

With a confirmatory trial for Sarepta’s Duchenne muscular dystrophy trial ending in failure, Pfizer’s chief scientist says the pharma giant’s gene therapy is now “the main game in town.”

Debunking 7 Common Myths About Data Quality in Clinical Trials

The clinical trial landscape has seen a seismic shift in recent years, spurred by technology-driven data collection. Yet there are some opportunities…

GSK loses its STING as part of phase 1 cleanout

GSK has dropped its in-house STING agonist only a day after a similar drug the British Big Pharma reserved the rights was finally lifted from clinical hold limbo.

AstraZeneca takes 22% stake in Cellectis as part of collab for 10 cell and gene therapies

Despite dropping its multiple myeloma CAR-T only five months ago, Cellectis has secured over $100 million in upfront cash and equity investment from AstraZeneca for a 10-candidate deal.

Bioluminescence enters biotech venture spotlight with almost $500M under management

Bioluminescence is rocketing out of stealth, looking to warm a frigid funding environment with the embrace of fresh funding. 

Sanofi pays $30M for first look at MeiraGTx's data and a shot to brew up deals

Sanofi is making an initial $30 million investment in MeiraGTx to gain a first peek at data from the biotech’s riboswitch platform, all for a chance to someday brew up some new deals.

Tonix halts major depression disorder drug after phase 2 failure

Tonix Pharmaceuticals’ pipeline has taken another hit as the biotech drops a major depression disorder therapy after the drug flamed out of a phase 2 trial.

Mersana's phase 1 ADC cancer trial breaks free of FDA hold

In a year riddled with obstacles and adverse events, Mersana Therapeutics finally has some good news: The FDA has lifted a clinical hold on a phase 1 trial for the antibody-drug conjugate XMT-2056.

C4 Therapeutics discontinues cancer program, waits for Betta payment

C4 Therapeutics is discontinuing one of its cancer assets after a phase 1/2 trial proved the drug ineffective.   

Calling all astronauts: NASA, Defender to test motion sickness gel in new trials

NASA and Defender Pharmaceuticals will test an anti-nausea gel in two clinical trials, recruiting astronauts to undergo motion simulation exercises to test sensorimotor performance. 

Already nearing blockbuster status, GSK's RSV vaccine Arexvy outdueled Pfizer rival in first round of clash

Aside from Pfizer's declining COVID-19 sales, GSK is giving the New York drugmaker another reason to worry. In the first round of their RSV vaccine showdown, GSK's Arexvy pulled down $860 million, more than doubling the haul from Pfizer's Abrysvo.

US government to launch AI Safety Institute, develop standards for biotech R&D

The Biden administration is launching a federal outfit to evaluate the safety of AI, and to address its impact in healthcare, R&D and the broader societal concerns that have recently come to the fore.

After patent settlement, Amgen scores FDA nod for its biosimilar version of J&J's Stelara

The FDA has approved Amgen’s biosimilar version of Johnson & Johnson’s autoimmune standout Stelara. But the new product will not launch until 2025 because of a settlement the companies made earlier this year, which delayed the launch in exchange for J&J dropping a patent infringement lawsuit.

Powered by $5M investment, startup Yuzu Health takes on insurers’ ‘brittle’ technology

Third-party administrator Yuzu Health says it can help insurance companies and brokers provide small businesses up to 40% in savings.
 
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