A Landmark Permanent Treatment for Children Born with a Deadly Immune Disorder
In June 2022, the first stem cell gene therapy of Dutch origin was successfully given to a patient by researchers at the Leiden University Medical Centre (LUMC). A baby with severe congenital immune disorder (SCID) received the complex but one-time treatment, marking the first time stem cell gene therapy ...
Treating Cardiovascular Disease by Changing a Single Letter of DNA
A simple spelling change in liver DNA could improve the future of cardiovascular disease treatment. On July 12, biotechnology company Verve Therapeutics announced that the first patient had received a novel gene editing medicine designed to permanently reduce "bad" LDL cholesterol — the fatty molecule that causes arteries to ...
FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients
In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in the pipeline. As oligonucleotide therapeutics continue to develop as an emerging and promising treatment for rare diseases, the US Food ...
A New Conjugated siRNA provides broad delivery in the CNS, Lung, and Eye
Ever since the groundbreaking discovery of RNA interference and its role as a gene-silencing mechanism in mammalian cells, an ongoing process of discovery has been underway to harness its potential in treating disease. A key mechanism that induces gene-silencing in the RNA pathway is small interfering RNA (siRNA). Although ...
Positive Results from Tofersen VALOR Trial and OLE Integrated Data
People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective treatment that could delay the onset of symptoms or extend their lifespan. However, as a result of recent advances in science and medicine, multiple oligonucleotide therapeutics are being developed to provide treatments, one of ...
CRISPR Babies and the Scientist Behind Their Editing
At an international genome-editing summit in Hong Kong, MIT Technology Review reporter Antonio Regalado uncovered the existence of the CRISPR baby project. The now infamous scientist, He Jiankui, had used CRISPR-Cas9, a genetic engineering tool, to edit the CCR5 gene of three embryos with the goal of making them ...
CRISPR Patent Rights and Their Effect on the Industry
A decade ago, Jennifer Doudna of the University of California, Berkley and Emmanuelle Charpentier of the Max Planck Institute for Infection Biology drafted blueprints for a groundbreaking gene editing technique. The two scientists had found a way to cut and make precise changes to DNA — a discovery that ...
Could Artificial Intelligence Provide the Key to Life Saving Medicines?
Drug discovery is time consuming and expensive, requiring trial and error screening. This means that many people have to wait decades, or a lifetime, to find a treatment for their disease. Others, especially those with rare diseases, may never have hope of a treatment. However, AI and deep learning ...
A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD
Duchenne muscular dystrophy (DMD) is a devastating disease that causes the body's muscle tissues to progressively waste away until all mobility is lost. Eventually, the heart muscle becomes fatty and fibrotic, typically leading to heart failure and death by age 30. There is no cure, and the approved drugs ...
Could a Single Oligonucleotide Medication Treat Multiple Different Diseases?
Our relatively new field is swiftly growing, and another exciting novel therapeutic RNA modality is now gaining traction. tRNA is vital in protein synthesis and by engineering tRNA molecules...
A Landmark Permanent Treatment for Children Born with a Deadly Immune Disorder
In June 2022, the first stem cell gene therapy of Dutch origin was successfully given to a patient by researchers at the Leiden University Medical Centre (LUMC). A baby ...
Treating Cardiovascular Disease by Changing a Single Letter of DNA
A simple spelling change in liver DNA could improve the future of cardiovascular disease treatment. On July 12, biotechnology company Verve Therapeutics announced that the first patient had received ...
FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients
In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in ...
A New Conjugated siRNA provides broad delivery in the CNS, Lung, and Eye
Ever since the groundbreaking discovery of RNA interference and its role as a gene-silencing mechanism in mammalian cells, an ongoing process of discovery has been underway to harness its ...
Positive Results from Tofersen VALOR Trial and OLE Integrated Data
People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective treatment that could delay the onset of symptoms or extend their lifespan. However, as ...
CRISPR Babies and the Scientist Behind Their Editing
At an international genome-editing summit in Hong Kong, MIT Technology Review reporter Antonio Regalado uncovered the existence of the CRISPR baby project. The now infamous scientist, He Jiankui, had ...
CRISPR Patent Rights and Their Effect on the Industry
A decade ago, Jennifer Doudna of the University of California, Berkley and Emmanuelle Charpentier of the Max Planck Institute for Infection Biology drafted blueprints for a groundbreaking gene editing ...
Could Artificial Intelligence Provide the Key to Life Saving Medicines?
Drug discovery is time consuming and expensive, requiring trial and error screening. This means that many people have to wait decades, or a lifetime, to find a treatment for ...
A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD
Duchenne muscular dystrophy (DMD) is a devastating disease that causes the body's muscle tissues to progressively waste away until all mobility is lost. Eventually, the heart muscle becomes fatty ...
Could a Single Oligonucleotide Medication Treat Multiple Different Diseases?
Our relatively new field is swiftly growing, and another exciting novel therapeutic RNA modality is now gaining traction. tRNA is vital in protein synthesis and by engineering tRNA molecules...
