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BioWorld - Friday, February 20, 2026
Home » Topics » Genetic/congenital, BioWorld Science

Genetic/congenital, BioWorld Science
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Human SCNT oocyte with visible spindle before fertilization.
Gynecology/obstetrics

Fertilizable egg-like cells generated with DNA from skin cells

Oct. 1, 2025
By Mar de Miguel
No Comments
Generating gametes from nonreproductive tissues could help overcome infertility. Previous studies have successfully transformed stem cells into viable oocytes through cellular reprogramming. Scientists at Oregon Health & Science University (OHSU) developed a method to derive them from skin cells via somatic cell nuclear transfer (SCNT), unlocking a mechanism that blends mitosis and meiosis. Now, the researchers have taken another step forward by generating fertilizable eggs from human skin cells.
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Respiratory

New CFTR modulators disclosed in Idorsia Pharmaceuticals patent

Sep. 29, 2025
Idorsia Pharmaceuticals Ltd. has divulged macrocyclic compounds acting as cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
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Illustration of hemoglobin structure
Genetic/congenital

Preclinical data on renizgamglogene autogedtemcel in models of SCD and TDT

Sep. 29, 2025
No Comments
Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are severe monogenic blood disorders caused by mutations in the β-globin gene (HBB), resulting in abnormal or insufficient production of adult hemoglobin (HbA). Among emerging therapeutic approaches, the reactivation of fetal hemoglobin (HbF) represents one of the most promising strategies for both conditions.
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Neurology/psychiatric

Cure Rare Disease’s CRD-003 designated orphan drug for LGMD2i/R9

Sep. 25, 2025
No Comments
The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic mutations in the FKRP gene.
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Illustration of chromosomes, red blood cells.
Endocrine/metabolic

Genfit prioritizes VS-01 development in urea cycle disorder

Sep. 22, 2025
No Comments
Genfit SA is discontinuing its VS-01 program in acute-on-chronic liver failure (ACLF), and has decided to reprioritize development of VS-01 for urea cycle disorder.
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Illustration of damaged nerve cell and amyloid plaques
Biomarkers

pTau217 could change how Alzheimer’s is diagnosed

Sep. 22, 2025
By Mar de Miguel
No Comments
Experts agree that the earlier Alzheimer's disease is detected, the sooner action can be taken. And so, the key to preventing deterioration is identifying the most effective early biomarkers, those that can spot the disorder and help halt its progression. Recent advances in the field have pushed a new era of early detection through blood-based biomarkers and personalized medicine strategies based on each patient’s genetic, immunological and clinical profile.
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Syringe in spotlight
Drug design, drug delivery & technologies

Cancer vaccines face collateral damage of mRNA funding cuts

Sep. 17, 2025
By Mar de Miguel
No Comments
When Robert Kennedy Jr. announced the cancellation of 22 projects related to mRNA vaccines and the end of new investments in that technology, the U.S. Secretary of Health only mentioned their use against respiratory viruses, without referring to other applications. The vaccines whose safety and effectiveness Kennedy is questioning are based on the same molecular principles as cancer vaccines under development. “Continued investment in mRNA technology is essential to fully realize its potential in oncology and ensure that promising strategies like neoantigen-based vaccines reach clinical application.” Kazuhiro Kakimi, professor at the Department of Immunology at Kindai University Faculty of Medicine, told BioWorld.
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Vial and syringe in row of dominoes
Drug design, drug delivery & technologies

The domino effect of cutting mRNA vaccine research

Sep. 15, 2025
By Mar de Miguel
No Comments
In August, a press release from HHS announced the cancellation of 22 vaccine research projects based on mRNA, the latest available technology aimed at developing therapies for viral infections, cancer, and genetic conditions. What happens to mRNA innovation when funding dries up? This series explores how reductions in funding could impact mRNA technology, affecting innovation, research and future therapies.
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Lasker award winners 2025
Drug design, drug delivery & technologies

Lasker Awards honor the fine compartmentalization of life

Sep. 12, 2025
By Mar de Miguel
A tangle of DNA can look like a knotted ball in the cell nucleus. However, the genetic machinery has a complex and regulated structure. Its long repetitive sequences also seemed to have no function. They were called junk DNA, although they were not. The same happened with proteins and low-complexity domains, disordered chains of amino acids that were poorly understood. Nevertheless, that protein noise has turned into music for the 2025 Lasker Awards. These prizes have recognized the work of scientists who were able to see order in chaos.
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Human body skeleton with DNA science background art
Genetic/congenital

Vantictumab reverses bone defects in mice with osteopetrosis

Sep. 9, 2025
No Comments
At the American Society for Bone and Mineral Research (ASBMR) in Seattle, researchers from Ashibio Inc. reported preclinical efficacy data on vantictumab, a human monoclonal IgG2 lambda antibody that binds to multiple frizzled (FZD) receptors.
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