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FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease
FDA approves Ultragenyx's gene therapy Fayuvi for Sanfilippo syndrome at a price of $3.95 million.
Gene Therapy
15 trends tracked about this subject — the full history, oldest to newest below.
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‘Curved Air’ Review: A Mutation’s Painful Legacy
Medical advancements in gene therapy are transforming the lives of sickle cell patients, highlighted by new treatments and cultural reflections.
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Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findings
2 news sources are covering this Business story right now — PULSE is tracking how fast it spreads.
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FDA pauses Regenxbio gene therapy trial again due to safety concerns
The FDA has placed a clinical hold on Regenxbio's gene therapy for Hunter Syndrome following safety concerns related to spine MRI findings.
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Exclusive: Third death in China's popular but opaque trials revealed, this time in CAR-T
Coverage reveals a third death in opaque Chinese clinical trials involving CAR-T therapy, intensifying global scrutiny.
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The ethical perils of gene therapy
The death of a child in a gene editing trial exposes the ethical perils and limits of scientific advancement.
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6-year-old girl dies after parents paid $860K for experimental therapy that ‘shouldn’t have gone to trial’
3 news sources are covering this Health story right now — PULSE is tracking how fast it spreads.
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Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients
Personalized gene therapy using bespoke ASOs has enabled a teen with SCN2A-related epileptic encephalopathy to walk independently for the first time.
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$160 million ‘moonshot’ backs Boston effort to revolutionize rare disease treatment
A $160 million non-profit initiative in Boston aims to revolutionize the development of genetic medicines for rare and ultra-rare diseases.
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FDA’s top gene therapy regulator is leaving his role
11 news sources are covering this Business story right now — PULSE is tracking how fast it spreads.
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Louisiana Notches a Sickle Cell First
8 news sources are covering this Health story right now — PULSE is tracking how fast it spreads.
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Scientists begin first trial to reverse human aging
Researchers have initiated the first human clinical trial focused on reversing cellular aging through experimental gene therapy.
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Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says
The FDA is allowing Regenxbio to resubmit its gene therapy for Hunter syndrome, marking a reversal of a previous rejection.
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'I've never been this good'
A lupus treatment borrowed from cancer therapy is putting patients into remission—with one calling it life-changing.
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What’s the Future of Gene Editing?
The gene editing landscape is expanding rapidly through next-generation CRISPR tools and significant projected market growth through 2028.