BioSpace News Archive

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  Rare diseases

  CRISPR Success in Single Baby Highlights Rare Disease ‘Crisis’

  Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.

  May 20, 2025

   · 

  3 min read

   · 

  Dan Samorodnitsky
• 

  FDA

  FDA’s New Risk-Based Approach to COVID-19 Vaccines Aims to Ease ‘Public Distrust’: Makary, Prasad

  The new framework was well-received by biopharma analysts, who say it “largely formalizes” current COVID-19 vaccination trends.

  May 20, 2025

   · 

  4 min read

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  Heather McKenzie
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  Collaboration

  CRISPR Expands Focus, Pays Up To $95M in Sirius’ siRNA Pact

  The partnership with Sirius expands CRISPR Therapeutics’ modality toolkit, especially in the cardiovascular space.

  May 20, 2025

   · 

  2 min read

   · 

  Tristan Manalac
• 

  Opinion

  Understanding What Trump’s MFN Executive Order Can and Can’t Do

  The Most Favored Nation order is unlikely to deliver broad, sustained savings without triggering legal challenges, administrative friction and unintended consequences for both the healthcare sector and patient access.

  May 20, 2025

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  6 min read

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  Robert Albarano
• 

  Cancer

  Pfizer Puts $6B on the Line for 3SBio’s PD-1/VEGF Antibody

  The deal comes three months after Pfizer inked a PD-1/VEGF partnership with Summit Therapeutics, leading BMO Capital Markets to express confusion regarding the pharma’s overall strategy.

  May 20, 2025

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  2 min read

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  Tristan Manalac
• 

  Psychedelics

  Makary Backs Psychedelics for Neuropsych, Promises Speedy Review

  In an interview on Friday, FDA Commissioner Marty Makary threw his weight behind psychedelic therapies, noting that patients taking these substances experience significant benefits for various neuropsychiatric conditions.

  May 20, 2025

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  2 min read

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  Tristan Manalac
• 

  Cardiovascular disease

  Ionis Builds Triglyceride-Lowering Case for RNA-Targeting Tryngolza

  The late-stage results come in advance of pivotal data that Ionis expects to provide for its antisense oligonucleotide Tryngolza in the third quarter, building up toward a regulatory submission in hypertriglyceridemia by year-end.

  May 20, 2025

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  2 min read

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  Tristan Manalac
• 

  China

  Pfizer’s China Deal Worth up to $6B Eclipses Peers in a Frenzied Market

  The largest Chinese licensing deal behind Pfizer’s is Novartis’ partnership with Shanghai Argo Biopharma, worth potentially more than $4 billion.

  May 20, 2025

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  1 min read

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  Annalee Armstrong
• 

  Drug pricing

  New Details From Trump’s Drug Pricing Effort Put Branded Drugs in the Crosshairs

  Drugmakers will be expected to commit to aligning U.S. prices with the lowest price set in a group of peer nations for all brand products across all markets that do not currently have generic or biosimilar competition.

  May 20, 2025

   · 

  1 min read

   · 

  Annalee Armstrong
• Press Releases

  ITM and Radiopharm Sign Supply Agreement for n.c.a. Lutetium-177

  May 20, 2025

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  3 min read