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First CRISPR In-Vivo Gene Therapy Poised for FDA Approval

Intellia's lonvoguran ziclumeran (lonvo-z), a CRISPR-based in-vivo gene therapy for hereditary angioedema, cut attack rates by 87% in a Phase 3 trial, and the company has filed for FDA approval. If cleared, it would be the first gene-editing medicine that edits a patient's cells inside their own body. In other news, physicists measured the residual antineutrino 'glow' from shut-down nuclear reactors for the first time, and astronomers found the strongest evidence yet for a companion star to Betelgeuse.

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CRISPR therapy lonvo-z cuts hereditary angioedema attacks by 87% in Phase 3 trial Intellia Therapeutics has filed for FDA approval of lonvoguran ziclumeran, a single-infusion in-vivo CRISPR therapy that disables the KLKB1 gene in liver cells. If approved, it would be the first gene-editing medicine that edits a patient's cells inside their own body. 1

Physicists measure eerie antineutrino glow from shut-down nuclear reactors for first time Using data from the Double Chooz experiment in France, researchers detected the residual electron antineutrino flux from spent fuel and cooling pools after reactor shutdown. This validates a long-theorized signal and could improve non-intrusive nuclear safeguards. 2

Astronomers capture clearest image yet of Betelgeuse's hidden companion star Using ESO's Very Large Telescope, a team led by Miguel Montargès imaged Betelgeuse B, a faint stellar companion orbiting the famous red supergiant. The discovery concludes a century-long search and was published in Astronomy & Astrophysics. 3

Magnetar 1E 1547.0−5408 provides strongest evidence yet for vacuum birefringence NASA's IXPE telescope found X-ray polarization from the magnetar that matches the 1936 Heisenberg-Euler prediction that strong magnetic fields make the quantum vacuum refract light differently by polarization. The result is a powerful test of quantum electrodynamics. 4

Researchers observe optical Magnus effect for first time using trapped calcium ion Scientists at PSI, ETH Zurich, and the University of Amsterdam found that a tightly focused laser beam's interaction with a single trapped ion is shifted sideways, mirroring the classical Magnus effect. The finding could improve precision control of qubits in quantum computers. 5

UCSF awarded $46M to develop precision medicines from largest autism gene map The grant from ARIA builds on a Science study by UCSF's QBI that mapped how hundreds of autism risk genes converge on a small number of shared protein complexes. The funding will support the Protein-Protein Interactions Hub to develop mechanism-based small molecule therapies. 67

New electrically controlled molecule recovers gold from e-waste with less chemistry University of Illinois researchers designed a multifunctional extraction molecule with a permanent built-in charge, allowing electricity to drive metal recovery without intermediate chemical reagents. The advance could make recycling valuable metals cleaner and more energy efficient. 8

Laser internet breakthrough hits 98% fidelity over hundreds of meters using skyrmions Scientists used magnetic 'skyrmions' to shield data from distortion in a free-space laser communication system, achieving 98% fidelity over hundreds of meters. The approach could offer an alternative to fiber-optic cables. 9

Chemists develop simpler method to make molecular building block for drug development Researchers at Scripps Research have devised a more straightforward synthesis for a key molecular building block used in drug development. The method could streamline the production of pharmaceuticals. 10

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