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BioWorld - Saturday, June 20, 2026
Home » Topics » Disease categories and therapies » Genetic/congenital

Genetic/congenital
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Respiratory

Idorsia Pharmaceuticals discloses new CFTR modulators

Oct. 10, 2022
Idorsia Pharmaceuticals Ltd. has described cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
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Human aging illustration
Genetic/Congenital

Genetic influences on longevity depend on sex and age

Oct. 6, 2022
By Mar de Miguel
Researchers have gained new insights into both genetic and nongenetic factors affecting life span, and how they differ between males and females. Several genes were correlated with longevity, according to a study by the École Polytechnique Fédérale de Lausanne (EPFL). However, some did not affect life span until males reached a certain age. Early access to nutrients during growth also affected longevity, as they saw in their study of more than 3,000 mice and verified with human data. “This study is one of the biggest studies on mass longevity. We were looking for genetic determinants of longevity but there are non-genetic determinants affecting longevity,” the first author Maroun Bou Sleiman, researcher at EPFL, told Bioworld.
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Svante Pääbo with skull
Genetic/Congenital

From ancient DNA, a Nobel Prize, and perhaps modern drug targets

Oct. 3, 2022
By Anette Breindl and Mar de Miguel
The Nobel Prize in Physiology or Medicine 2022 was awarded to Svante Pääbo today "for his discoveries concerning the genomes of extinct hominins and human evolution." Pääbo, who is currently the director of the Max Planck Institute for Evolutionary Anthropology in Leipzig, Germany, and his colleagues overcame extreme technical challenges to sequence the DNA of ancient hominids – because after tens of thousands of years, there is no such thing as aging well for DNA.
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RNA strand
Endocrine/Metabolic

Wave Life Sciences reports data on WVE-006 in AATD mouse model

Sep. 29, 2022
Wave Life Sciences Ltd. has highlighted progress with WVE-006, its preclinical Aimer (A-to-I RNA base editing) oligonucleotide candidate for the treatment of alpha-1 antitrypsin deficiency (AATD).
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Mitochondria illustration
Genetic/congenital

Pretzel Therapeutics launches with focus on mitochondrial biology

Sep. 13, 2022
Pretzel Therapeutics Inc. has launched with a $72.5 million series A financing to pioneer novel therapies to modulate mitochondrial function to treat rare genetic diseases and common diseases of aging.
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