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Home » Topics » Drugs » Antisense

Antisense
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Dyne seeks accelerated path on positive dystrophy data; shares down

Jan. 10, 2025
By Lee Landenberger
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.
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Euro symbol in piggy bank
Neurology/psychiatric

Series A at Neumirna supports RNA therapies for neurological disorders

Jan. 8, 2025
A €20 million (US$20.6 million) series A financing round at Neumirna Therapeutics ApS is set to support the company’s development of RNA therapies for epilepsy, Parkinson’s disease and other neurological disorders, and enable the company to advance its lead asset into the clinic.
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Hepatitis B virus rendering

Ausperbio bags $73M series B for hep B-targeting RNA therapy

Dec. 31, 2024
By Marian (YoonJee) Chu
Ausperbio Therapeutics Inc. raised $110 million from two financing rounds in 2024 to advance its lead antisense oligonucleotide candidate as a functional cure for chronic hepatitis B.
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Hepatitis B virus rendering

Ausperbio bags $73M series B for hep B-targeting RNA therapy

Dec. 30, 2024
By Marian (YoonJee) Chu
Ausperbio Therapeutics Inc. raised $110 million from two financing rounds in 2024 to advance its lead antisense oligonucleotide candidate as a functional cure for chronic hepatitis B.
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Red blood cells
Hematologic

Vanda’s VGT-1849A gets orphan designation for polycythemia vera

Dec. 23, 2024

Vanda Pharmaceuticals Inc.’s VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor, has been awarded orphan drug designation by the FDA for the treatment of polycythemia vera.


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FDA Approved stamp

Ionis notches Arrowhead win; Tryngolza cleared for FCS

Dec. 20, 2024
By Randy Osborne
Ionis Pharmaceuticals Inc. CEO Brett Monia predicted that diagnoses of familial chylomicronemia syndrome (FCS) will “accelerate fairly quickly” now that the firm has gained U.S. FDA clearance for Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with the rare form of severe hypertriglyceridemia (sHTG) that can lead to life-threatening acute pancreatitis (AP).
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Neurology/psychiatric

Trace Neuroscience launches with focus on genomic medicines for neurodegenerative diseases

Nov. 13, 2024
Trace Neuroscience Inc. has launched with a $101 million series A financing and a focus on genomic medicine for neurodegenerative diseases.
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Man measuring waist

Sanofi bets on Resalis’ oligonucleotide RES-010 for obesity

Oct. 28, 2024
By Nuala Moran
After missing out on the glucagon-like peptide 1 obesity market, Sanofi SA is prospecting for next-generation drugs and is making a strategic equity investment in Resalis Therapeutics Srl, providing the Italian biotech with funding to take its lead program RES-010 through to phase II.
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Illustration of motor neuron connecting to muscle fiber
Musculoskeletal

ENTR-601-45 restores dystrophin in Duchenne muscular dystrophy models

Oct. 10, 2024
Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.
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Lungs anatomy
Respiratory

Splisense’s SPL5AC restores mucus viscoelasticity and lung clearance in cystic fibrosis

Oct. 4, 2024
As in other muco-obstructive diseases, the airways in cystic fibrosis (CF) are characterized by goblet cell and glandular hyperplasia, with overproduction of mucins MUC5 and MUC5AC, resulting in viscous mucus, respiratory blockade and recurrent infections and inflammation.
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