In 2024, we will see a lot of scientific developments in biotech. Biotech is a rapidly growing field, and many of our projects are related to current biotech trends.
Alzheimer’s disease is increasing at an alarming pace as the population of the world age. In 2023, there were 187 trials assessing 141 unique treatments for Alzheimer’s disease. Inflammation, amyloid, neurotransmitter receptors, synaptic plasticity, tau biology, oxidation, and proteostasis/proteinopathy represent the major targets for drug development. The Alzheimer’s disease drug development pipeline is leading to new therapies. After a 17-year hiatus in drug approvals, two agents—aducanumab and lecanemab — have entered the market since 2021; brexpiprazole ameliorated agitation in a Phase 3 trial; and suvorexant reduced insomnia in Alzheimer’s disease in a Phase 3 trial. Aducanumab and lecanemab are the first disease-modifying therapies for Alzheimer’s disease and among the first disease-modifying therapies for any neurodegenerative disease.
Amyloid-beta precursor protein is an integral membrane protein concentrated in the synapses of neurons.
Last year marked the 10th anniversary of the development of CRISPR as a genome-editing tool, an achievement that won Jennifer Dounda and Emmanuelle Charpentier the 2020 Nobel Prize in Chemistry. 10 years is not a long time, but CRISPR-based therapies progressed remarkably. At the 2022 Annual Meeting of the American Society of Hematology, some of the most advanced CRISPR-based treatments in development for the treatment of blood disorders were presented. Of these, the gene therapy made by CRISPR Therapeutics and Vertex Pharmaceuticals, exagamglogene autotemcel – also known as exa-cel, is the closest to potential approval.
Cas9 acts as “molecular scissors” to cut DNA at a location specified by a guide RNA.
Since 2017, six CAR T-cell therapies have been approved by the Food and Drug Administration, all for the treatment of blood cancers. Chimeric antigen receptor T cell therapy is a personalized treatment for cancer that involves isolating a patient’s T cells and reprogramming those T cells to produce a specific, targeted receptor on each T cell’s surface. Scientific progress within the field of cancer immunotherapy has led to seven CAR-T cell therapy approvals, including Kymriah, Yescarta, Tecartus, Breyanzi, Abecma, Relma-cel, Carvykti. According to GlobalData, there are 230+ companies engaged in the development and application of CAR-T cell-based compositions.
CAR-T cell has four domains: an antigen-binding domain, a hinge region, a transmembrane domain, and an intracellular domain.
The U.S. FDA approved Novo Nordisk’s diabetes drug semaglutide for increasing weight loss in obesity in 2021. In 2022, Lilly’s drug tirzepatide (Mounjaro) was approved for the treatment of type 2 diabetes. The drug also shows big potential in boosting weight loss in overweight patients, with a phase 3 trial readout due later in 2023. Meanwhile, an oral drug from Pfizer causes a similar amount of weight loss as Novo’s semaglutide, according to results from a Phase 2 clinical trial. The study results also suggest the Pfizer medicine may be as effective for weight loss as Novo’s, although there are stark differences in dosage levels.
Fat mass gain increases adipocyte number and size.
Late 2022 birthed the “tripledemic,” a term that describes the havoc wreaked by a simultaneous surge in Covid-19, the flu, and the respiratory syncytial virus. Pfizer and GSK recently announced impressive data from Phase 3 trials of their respective RSV vaccines. Others, including Moderna and Johnson & Johnson, are working on their own RSV jabs, though Pfizer and GSK are expected to get to market first. Following the success of mRNA-based Covid-19 vaccines, mRNA-based flu vaccines have been all the rage in Big Pharma. Today, mRNA-based flu vaccines are under development by Moderna, Pfizer, and Sanofi. The eventual goal is to develop combination vaccines that could protect against multiple viruses, including the flu, Covid-19, and RSV.
Recombinant Extracellular Transport Vesicles are the vaccines that carry payloads of biochemicals such as nucleic acids, toxins, peptides, etc. to modify a target cell’s activity.
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