Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.
Abeona Therapeutics Inc. and Beacon Therapeutics Ltd. have signed an agreement for evaluation by Beacon of Abeona’s patented AAV204 capsid for the development and commercialization of potential gene therapies for select ophthalmology indications.
Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.
Scientists at the University of Washington have engineered human plasma B cells modified to express long-lasting bispecific antibodies that could be used to treat leukemia without requiring continuous dosing.
Scientists at the University of Washington have engineered human plasma B cells modified to express long-lasting bispecific antibodies that could be used to treat leukemia without requiring continuous dosing.
“We are trying to engineer plasma cells to make as a stable source for biologic drugs. One thing that is really unique about plasma cells is that they can live for a really long time … up to 10 years or even 100 years depending on the type of plasma cell that that you make,” Richard James, senior author of the study, principal investigator at Seattle Children’s Research Institute, and associate professor at the University of Washington, told BioWorld.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria. The advance opens the door to the development of programmable methods for rearranging DNA, using recombinase enzymes guided by RNA. The two different approaches to using insertion sequences (IS) – some of the simplest and most compact mobile genetic elements – are described in two papers published in Nature and Nature Communications.
Fibrobiologics Inc. has announced the development of a novel artificial thymus organoid that can restore immune function in a severe combined immunodeficiency (SCID) mouse model.
A new approach against non-small-cell lung cancer (NSCLC) has combined immunotherapy with molecularly targeted therapy to activate the immune response and inhibit oncogenic pathways, which prevented tumor progression and eliminated cancer cells. Brigham and Women’s Hospital scientists have developed nanoparticles loaded with antibody conjugates that could deliver large amounts of treatment to the tumor tissue. This new strategy could improve the results of conventional immunotherapy in these patients and reduce toxicity of existing treatments.
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