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Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.
Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. sickle cell disease community and the millions around the globe who suffer from this genetic illness. sickle cell disease community and the millions around the globe who suffer from this genetic illness.
The two partners will take an in vivo approach to the problem of so-called sensorineural hearing loss (SNHL) looking at ways to replace sound-detecting hair cells in the inner ear (cochlea) that become damaged in this type of deafness. An estimated 1.57 Astellas is already working on other approaches to treating hearing loss.
The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.
Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences.
Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes. The big biotech meanwhile has an antisense drug in trials for a subtype of inherited ALS, licensed from Ionis Pharma, in phase 3 clinical trials. Benjamin Oakes.
Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.
Sanofi has licensed a potential inflammatory diseases drug from Manchester University spin-off C4X Discovery Holdings in a deal worth up to €414 million ($492 million). C4XD said it has developed a molecule small enough to be formulated into a pill, which has also been able to block IL-17 in vivo.
Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Rare diseases can often be progressive, chronic and fatal. Sadly, one-third of children with rare diseases die before their first birthday.
The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.
Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We
Based on these studies, one of those drugs was licensed to Cantex Pharmaceuticals for the treatment of COVID-19 and other inflammatory lung diseases. Every breath stretches the lungs’ tissues with each inhale and relaxes them with each exhale. a Wyss Technology Development Fellow at the Institute. During his Ph.D.
Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Nielsen, the primary inventor of peptide nucleic acid (PNA) technology, brings extensive experience in genetic medicine to the company.
Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. which will remain a key shareholder. in a statement. . Libra Therapeutics.
Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma. Amgen (NASDAQ:AMGN) announced that the U.S. Tezepelumab is being developed by Amgen in collaboration with AstraZeneca. Reese, M.D., About Severe Asthma.
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