NEW YORK, NY — Myrtelle Inc. (“Myrtelle” or the “Company”), a pioneering clinical-stage gene therapy company dedicated to revolutionizing treatment for neurodegenerative diseases, today announced the official launch of commercial-stage manufacturing for its first-in-class oligotrophic recombinant adeno-associated virus (rAAV) gene therapy product, developed specifically for Canavan disease (CD). This landmark achievement propels...
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NEW YORK, NY — Myrtelle Inc. (“Myrtelle” or the “Company), a pioneering clinical-stage gene therapy company developing transformative treatments for neurodegenerative diseases, today announced the publication of interim results from its Phase 1/2 clinical trial of rAAV-Olig001-ASPA (MYR-101) in Nature Medicine. The published article, entitled, “Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2...
NEW YORK, NY — Myrtelle Inc., a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation to the Company’s lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of...
Westmead, Australia – Scientists at Children’s Medical Research Institute (CMRI) have solved a big mystery in cancer research – why cells die in different ways following radiotherapy. This surprising finding opens up new opportunities to improve treatment and increase cure rates. The findings were published in Nature Cell Biology by...
CULVER CITY, Calif. – ImmunityBio, Inc (NASDAQ: IBRX), a clinical-stage immunotherapy company, today announced data from a Phase 1 pilot study showed N-803 combined with natural killer cells could have the potential to reduce viral load in people living with HIV. Published in The Journal of Infectious Diseases, researchers at...
SAN DIEGO, Calif. — n-Lorem, a nonprofit foundation, has announced the dates for its second annual Nano-rare Patient Colloquium, which will be held Oct. 30 & 31 in Cambridge, Mass. The Nano-rare Patient Colloquium will bring together a Community of Care for Nano-rare that consists of treating physicians, industry leaders,...
SAN DIEGO – n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, today announced a new partnership with Ultragenyx Pharmaceutical (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases. n-Lorem Foundation...
SAN DIEGO – n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, today announced a new partnership with ChemGenes Corporation, a biotechnology company providing unparalleled expertise in the field of DNA/RNA synthesis for more than 40 years. n-Lorem Foundation is the...
North Carolina’s health care system has been unkind to Kay Swan and her family. Her husband suffers from kidney disease and her son was diagnosed with Wolfram syndrome, a rare genetic disorder that can cause diabetes, loss of vision and deafness. When Swan lost her job of 18 years, her...
FORT WORTH, Texas — Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to NPI-001 (N-acetylcysteine amide) tablets, Nacuity’s proprietary, investigational therapy for...