ArmaGen closes $17M Series A financing
ArmaGen Technologies, Inc. today announced the closing of a $17 million Series A financing to support the development of brain-penetrating recombinant protein therapeutics.
View ArticleResearchers to develop new diagnostic tools and treatments for people with...
A multi-million Euro initiative is bringing together researchers from across the world to develop new diagnostic tools and new treatments for people with rare diseases and to connect research data in...
View ArticleSynageva BioPharma reports study results of sebelipase alfa in adults with...
Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, today reported 12-month results from an ongoing extension study with sebelipase alfa in adults...
View ArticleRenaissance in drug development for rare diseases
Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug...
View ArticleFDA designates Synageva's sebelipase alfa as Breakthrough Therapy for early...
Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, today announced that the U.S. Food and Drug Administration granted Breakthrough Therapy...
View ArticleSynageva BioPharma presents data on cholesteryl ester storage disease at NLA...
Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare diseases, announced three poster presentations at the National Lipid Association annual meeting held in...
View ArticleGene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal...
A single session of a gene therapy developed by the Universitat Autònoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects...
View ArticleFDA grants orphan drug designation to ARMAGEN's AGT-182 for treatment of...
ARMAGEN announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as...
View ArticleResearchers to use state-of-the-art brain imaging to study mucopolysaccharidosis
A team of researchers will use state-of-the-art brain imaging to unlock the secrets of a genetic disease, mucopolysaccharidosis, in a landmark study the team hope will lead to new treatments for this...
View ArticleMedical researchers discover structure of potential drug target for rare...
Medical researchers at the University of Alberta have discovered the structure of a potential drug target for a rare genetic disease, paving the way for an alternative treatment for the condition.
View ArticleEuropean scientists set up new therapeutic approaches to tackle gene defects
On 15th April is the 1st International Pompe Disease Day, a campaign to raise awareness of this rare but severe gene defect. Pompe Disease is only one of more than 40 metabolic disorders that mainly...
View ArticleShire, ArmaGen partner to develop AGT-182 drug for treatment of Hunter syndrome
Shire plc, the global specialty biopharmaceutical company, and ArmaGen, a US privately held biotechnology company, today announced a worldwide licensing and collaboration agreement for AGT-182, an...
View ArticleResearch finding opens door to potential treatment for MPS IIIB
MPS IIIB is a devastating and currently untreatable disease that causes progressive damage to the brain, leading to profound intellectual disability, dementia and death -- often before reaching adulthood.
View ArticleFDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome
ArmaGen, Inc., a privately held biotechnology company focused on developing novel therapies to treat severe neurological disorders, announced today that the Investigational New Drug (IND) application...
View ArticleGene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists...
Gene therapy is the delivery of DNA into a patient's cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off...
View ArticleFDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I
Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration has cleared the Company's Investigational New Drug (IND) application for...
View ArticleResearchers agree to test new stem cell gene therapy for Sanfilippo disease...
Scientists in Manchester, who have developed a stem cell gene therapy to reverse a fatal childhood illness, have agreed to work with a new therapeutics company to test it in a human trial.
View ArticleFDA grants permission to market new Seeker System for screening rare...
The U.S. Food and Drug Administration today permitted marketing of the Seeker System for the screening of four, rare Lysosomal Storage Disorders (LSDs) in newborns.
View ArticleResearchers to use state-of-the-art brain imaging to study mucopolysaccharidosis
A team of researchers will use state-of-the-art brain imaging to unlock the secrets of a genetic disease, mucopolysaccharidosis, in a landmark study the team hope will lead to new treatments for this...
View ArticleMedical researchers discover structure of potential drug target for rare...
Medical researchers at the University of Alberta have discovered the structure of a potential drug target for a rare genetic disease, paving the way for an alternative treatment for the condition.
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