Tag: genomics
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Gene Therapy Stimulates Missing Enzymes in Rare Disease
Results from a clinical trial show an experimental gene therapy for Krabbe disease is safe and produces enzymes missing in children with the rare disease.
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Drug Licensed for Precision Autism Spectrum Therapies
A company making precision therapies for neurological disorders is acquiring rights to a compound first designed as a treatment for cancer, inflammation, and oxidative stress.
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RNA Cancer Immunotherapy Shown Feasible in Preclinical Test
Tests with lab animals show synthetic viral RNA particles, encased in natural oils, can safely invoke an immune response to attack solid tumors.
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Nasal Spray Vaccines Get CEPI, NIH Funding
A developer of infectious disease vaccines is gaining funds for new nasal sprays protecting against a range of coronaviruses and the sexually-transmitted disease gonorrhea.
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Algae-Grown Non-Needle Malaria Vaccine in Preclinical Test
A vaccine grown in genetically-altered algae, designed as a nasal-spray and edible drug, is shown to protect lab mice against parasite infections causing malaria.
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Patent Issued for Crispr-Edited Viruses in Microbiome
A biotechnology company developing gene-edited therapies for bacterial infections in the gut received a U.S. patent for viruses engineered as treatments.
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Start-Up Developing Efficient RNA Process, Raises $12M
A company formed earlier this year is commercializing a more efficient process for making synthetic RNA molecules and raising $12 million in seed funds.
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Start-Up Licenses Gene-Edited Drug Discovery Tech.
A new company is acquiring a technology using genome-edited tadpoles that finds current drugs addressing precise genetic conditions underlying rare diseases.
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NIH Funds Decentralized Rare Disease Data Network
A start-up company is building a secure decentralized network of genomic and other medical data on rare diseases, funded by a fast-track award from an NIH agency.
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Trial Underway Assessing Gut Microbiome-Cancer Links
A clinical trial began collecting stool and blood samples to find connections between microbial communities in the gut and four types of solid-tumor cancer.