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Home » Topics » Drugs » Gene therapy

Gene therapy
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Silhouette of child and brain

Orchard-Kyowa scores US approval for gene therapy Lenmeldy

March 18, 2024
By Marian (YoonJee) Chu
The U.S. FDA approved Orchard Therapeutics plc’s BLA for gene therapy atidarsagene autotemcel, making it the first treatment option for metachromatic leukodystrophy in the U.S. The one-time treatment, branded Lenmeldy, is indicated for children with presymptomatic late infantile, presymptomatic early juvenile or early symptomatic early juvenile disease.
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Green strand of DNA

Pulsesight advancing eye therapies delivered via electroporation

Feb. 29, 2024
By Nuala Moran
Ocular gene therapy specialist Pulsesight Therapeutics SAS has launched with seed funding and is raising a series A to take forward two programs using electroporation to deliver plasmid DNA in the treatment of wet age-related macular degeneration and geographic atrophy.
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Green strand of DNA

Pulsesight advancing eye therapies delivered via electroporation

Feb. 28, 2024
By Nuala Moran
Ocular gene therapy specialist Pulsesight Therapeutics SAS has launched with seed funding and is raising a series A to take forward two programs using electroporation to deliver plasmid DNA in the treatment of wet age-related macular degeneration and geographic atrophy.
Read More
Monica Bertagnolli, NIH director

NIH’s Bertagnolli vows to improve data gaps in US health system

Feb. 23, 2024
By Nuala Moran
The new director of the U.S. NIH, Monica Bertagnolli, has set out the prospectus for her tenure, saying she intends to apply the agency’s $47 billion per annum budget to reverse the decline in health and life expectancy in the U.S.
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Hepatitis B virus rendering
Infection

Precision Biosciences advances PBGENE-HBV toward clinic

Feb. 15, 2024
Precision Biosciences Inc. has received pre-IND regulatory feedback from the FDA and ex-U.S. agencies providing alignment and clarity on Precision's final IND/CTA-enabling preclinical plans and clinical strategy for PBGENE-HBV.
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Biogen’s ataxia drug, Crispr’s sickle cell therapy cleared in EU

Feb. 13, 2024
By Caroline Richards
The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).
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Regenxbio’s Hunter syndrome gene therapy hits pivotal trial goals

Feb. 8, 2024
By Jennifer Boggs
Regenxbio Inc. plans to file a BLA this year seeking accelerated approval for gene therapy candidate RGX-121 to treat young children with mucopolysaccharidosis type II, also known as Hunter syndrome, based on positive data from the phase I/II/III Campsiite trial, which not only hit the biomarker endpoint but also indicated potential systemic benefits.
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Eye and DNA illustration

Street likes Luna wet AMD phase II but Adverum wanes

Feb. 8, 2024
By Randy Osborne
With Adverum Biotechnologies Inc.’s preliminary safety and efficacy data made public from the ongoing Luna phase II trial testing gene therapy ixoberogene soroparvovec (ixo-vec) in wet age-related macular degeneration (AMD), Wall Street promptly began stacking the results against those of competitors. CEO Laurent Fischer pointed out that ixo-vec boasts the “highest rate of injection-free patients of any study of any program at any dose.”
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DNA illustration

Ultragenyx’s gene therapy UX-111 significant for Sanfilippo

Feb. 6, 2024
By Karen Carey
A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
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Sangamo’s early phase I/II Fabry Staar data shine but funds needed for late-stage advancement

Feb. 6, 2024
By Caroline Richards
Sangamo Therapeutics Inc.’s gene therapy for Fabry disease, isaralgagene civaparvovec (ST-920), is continuing to show promising efficacy and safety in the phase I/II study, Staar, but further ahead, the company needs to either attract a partner or secure financing to move to a registrational trial.
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