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Home » Topics » Drugs » Gene therapy

Gene therapy
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Close up of eye and vision test
Ocular

Avirmax begins IND-enabling studies with gene therapy for dry AMD

March 3, 2025
Avirmax Biopharma Inc. has begun IND-enabling studies of ABI-201, a gene therapy for dry age-related macular degeneration (AMD). ABI-201 is an AAV vector that delivers three genes to correct the dysregulation of complement activation, to protect retinal pigment epithelia and photoreceptors, as well as to block retinal neovascularization.
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Leqembi gets another thumbs-up amid CHMP February recommendations

Feb. 28, 2025
By Nuala Moran
The EMA’s Committee for Medicinal products for Human Use (CHMP) is standing by its opinion on Leqembi (lecanemab) after the European .mission pushed back against a recommendation in November 2024 that the Alzheimer’s disease drug be approved
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Child cupping hand to ear

Regeneron gene therapy improves children’s hearing

Feb. 25, 2025
By Lee Landenberger
In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss, 10 out of 11 have shown improvements after being treated with a gene therapy. “What is really remarkable about this type of therapeutic approach is that the first people who are going to see the impact are not actually the physicians – it’s the families,” Jonathon Whitton, vice president and Regeneron’s auditory global program head, told BioWorld.
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Close-up of toddler eyes

Meiragtx’s gene therapy shows ‘exceptional’ data for blind children

Feb. 21, 2025
By Nuala Moran
Gene therapy specialist Meiragtx Holdings plc is heading for its first marketing approval following the successful treatment of 11 children with Leber amaurosis, a severe form of congenital retinal dystrophy that rendered them blind at birth. The 11 children, aged between 1 and 4 years old, all gained visual acuity following a single delivery of a correct version of the AIPL1 (aryl-hydrocarbon interacting protein-like1) gene, which codes for a photoreceptor protein in the cones and rods.
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Close up of man wearing nebulizer

Trial begins for inhaled gene therapy for cystic fibrosis

Feb. 20, 2025
By Nuala Moran
The longstanding ambition of developing an inhaled gene therapy for cystic fibrosis has taken a step forward, with the start of a phase I/II trial of a product using a novel pseudotyped viral vector that it is hoped will circumvent problems encountered in previous studies with other vectors.
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3D Euro symbol

EG 427 adds €27M series B for neurogenic bladder program

Feb. 20, 2025
By Nuala Moran
EG 427 SAS has closed a €27 million (US$28.3 million) series B round, which will fund it to completion of the first clinical trial of the lead gene therapy program, opening the way for its herpes simplex viral-vectored products to be developed in a range of chronic neuro-urology disorders.
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Cardiovascular

TLT-101 cBIN1 gene therapy reverses heart failure in animal models

Feb. 20, 2025
Researchers from Tikkunlev Therapeutics Inc. and the University of Utah have presented preclinical data on TLT-101, a gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector encoding cardiac bridging integrator 1 (cBIN1) designed for the treatment of heart failure.
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Solid rocks DMD space with phase I/II gene therapy data

Feb. 18, 2025
By Randy Osborne
Solid Biosciences Inc. is preparing for a sit-down with the U.S. FDA this year to discuss the firm’s results with the next-generation gene therapy SGT-003 for Duchenne muscular dystrophy (DMD).
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Endocrine/metabolic

scAAV9/SUMF1 shows safety and efficacy as gene replacement therapy for the treatment of multiple sulfatase deficiency

Feb. 13, 2025
Multiple sulfatase deficiency is an autosomal recessive lysosomal storage disorder caused by homozygous or compound heterozygous loss-of-function mutations in the SUMF1 gene, which encodes formylglycine generating enzyme (FGE), which catalyzes the post-translational modification of all known 17 sulfatases.
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Neurology/psychiatric

Voyager to evaluate alternate payloads for ALS gene therapy program

Feb. 12, 2025
Voyager Therapeutics Inc. has announced its decision to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). Emerging preclinical data indicate the siRNA payload component of VY-9323 does not meet its standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window.
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