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Live FundingHarvard Quantum Lab: $142M Grant
IPO AlertYale Biotech Spin-off Valued at $1.4B
PolicyColumbia Law Review Analysis on SEC Ruling
AlumniPenn Wharton Grad Appointed to Fed Board
ResearchPrinceton PPPL Fusion Q +41s
EndowmentYale FY24 +14.2%
VentureCornell Lattice Power Series A $80M
RankingsBrown Climbs to #7 in Research Output Index

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Penn Medicine breakthrough in CRISPR-based neuro-regeneration

The Perelman School of Medicine announces human trials for a novel gene-editing vector targeting ALS.

By Sarah ChenHealth & Science Correspondent·October 13, 2024·6 min read
Penn Medicine vector preparation lab.
Penn Medicine vector preparation lab.

The Perelman School of Medicine has received conditional FDA clearance to begin Phase I trials of a new CRISPR-Cas13 vector targeting the SOD1 mutation implicated in familial ALS. Early animal studies showed a 41% reduction in motor neuron degeneration.

"The Perelman School of Medicine announces human trials for a novel gene-editing vector targeting ALS."

Sarah Chen
HealthcareResearchPenn

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