BioSpace News Archive

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Regenxbio Fails To Best Sarepta’s Elevidys With New Data for DMD Gene Therapy

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according to BMO Capital Markets analysts.

June 5, 2025

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2 min read

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Dan Samorodnitsky

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Autoimmune disease

Cullinan Bets $700M+ in Back-Heavy China Deal for Autoimmune T Cell Engager

Genrix’s velinotamig complements Cullinan’s own pipeline, according to William Blair, which added that the deal will put Cullinan in a better position to target autoimmune diseases.

June 5, 2025

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2 min read

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Tristan Manalac

Gene therapy

Sarepta Wins FDA’s Platform Technology Designation, Marking ‘Critical Shift’ in Gene Therapy Development

The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug development and review process, particularly for rare diseases.

June 5, 2025

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2 min read

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Tristan Manalac

Government

Fired HHS Staff Sue RFK Jr., Musk, Claiming Terminations Were Based on ‘Error-Ridden’ Information

The lawsuit alleges that HHS leadership knew the records they used to guide their layoff decisions were inaccurate and contained errors.

June 5, 2025

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2 min read

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Tristan Manalac

IN PARTNERSHIP WITH IQVIA

AI’s Role in Decoding the FDA’s New Regulatory Communications

In this episode of Denatured, presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses how AI transformation can help organizations navigate a rapidly evolving regulatory environment with senior director of regulatory innovation and technology, Michelle Gyzen.

June 5, 2025

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1 min read

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Lori Ellis

Huntington’s disease

uniQure’s Regulatory Progress Sparks Optimism for ‘Desperate’ Huntington’s Space

Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on target for a Q1 2026 submission for its one-time gene therapy for Huntington’s disease—but patients have been here before.

June 5, 2025

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6 min read

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Heather McKenzie