Lung cancer is a leading cause of cancer-related death worldwide, and treatment options are limited for patients with advanced disease. However, a recent study published in the journal Nature has shown promising results for a new approach to treating lung cancer using personalized medicine. The researchers analyzed the genomes of more than 2,000 patients with non-small cell lung cancer and identified several genetic mutations that were associated with a better response to immunotherapy, a type of cancer treatment that helps the immune system fight cancer. The researchers also developed a personalized treatment algorithm that took into account the genetic mutations of each patient’s tumor. They found that patients who received personalized treatment based on their tumor’s genetic profile had a better response to immunotherapy than those who received standard treatment. The lead author of the study, Dr. Trever Bivona, said that the results were “very promising” and showed that personalized ...
Duchenne muscular dystrophy (DMD) is a rare and devastating genetic disorder that causes progressive muscle weakness and eventually leads to death. There is currently no cure for DMD and available treatments only offer modest benefits. However, a recent study published in the journal Nature has shown promising results for a new approach to treating DMD using gene therapy. The researchers used a viral vector to deliver a functional copy of the dystrophin gene, which is mutated in DMD, to muscle cells in mice with the disease. The researchers found that the gene therapy led to an improvement in muscle strength and function in the mice. They also found that the therapy was safe and well-tolerated. The lead author of the study, Dr. Dongsheng Duan, said that the results were “very encouraging” and showed that gene therapy could be a viable option for treating DMD. “This study demonstrates that gene therapy ...
Depression is a common and debilitating mental illness that affects millions of people worldwide. While there are several effective treatments for depression, many patients do not respond to these treatments or experience side effects. A recent study published in the journal Nature Communications has identified a potential new drug target for treating depression. The researchers focused on a protein called L-type calcium channel α1D, which plays a key role in the regulation of mood-related behaviors. Using a mouse model of depression, the researchers found that blocking the activity of this protein led to an improvement in depressive symptoms. They also found that the protein was overactive in the brains of depressed mice and in post-mortem brain tissue from people with depression. The lead author of the study, Dr. Jian Feng, said that the results were “exciting” and showed that targeting this protein could be a promising new approach to treating ...
Aim for participants will be to quickly new antimicrobial therapies to treat chronic respiratory infections The Cystic Fibrosis (CF) Syndicate in Antimicrobial Resistance (AMR) has announced pivotal £3m funding for a Collaborative Discovery Programme. The initiative is designed to encourage drug discovery innovators in the development of new treatments for individuals with CF. Funded by medical research charity LifeArc, the programme will support approximately five collaborative projects – each receiving £500,000 of funding. The aim for participants is to quickly establish new antimicrobial therapies to treat chronic respiratory infections. Researchers within small and medium enterprises (SMEs) and academia across the world are eligible to apply, with projects expected to last up to two years. Applicants are requested to address patients’ priorities for improving treatment set out in the CF AMR Syndicate’s recently-published ‘Target Product Profiles for Antimicrobials’. The programme also delivers a collaborative approach to drug development, with successful applicants ...
Funding is for onasemnogene abeparvovec and concerns babies with spinal muscular atrophy NICE has published final draft guidance regarding the recommendation of routine NHS funding for onasemnogene abeparvovec. The treatment is a potentially curative one-off gene therapy for treating a rare genetic condition, spinal muscular atrophy (SMA). It will be used specifically among babies aged up to 12 months, before they develop symptoms. The drug – also known as Zolgensma – is produced by Novartis Gene Therapies. The recommendation has emerged after NICE reviewed the evidence to determine whether it should be available for routine NHS funding in England and Wales. Onasemnogene abeparvovec is designed to address the genetic cause of SMA and its effects are thought to be life-long. Under NICE’s original guidance, released in 2021, it was recommended for babies before they develop symptoms of SMA as part of a managed access agreement (MAA). The agreement ensured that ...
Qdenga’s approval is supported by the data obtained from 19 clinical trials, and follows similar decisions in other countries. The National Health Surveillance Agency (ANVISA) in Brazil has granted approval for Takeda’s tetravalent dengue virus vaccine, Qdenga. The vaccine, previously known as TAK-003, has received approval for preventing dengue disease in people aged four years to 60 years. Qdenga has been developed based on a live-attenuated dengue serotype 2 virus that offers the genetic backbone for four dengue virus serotypes and is designed to provide protection against any of these. Qdenga is purported to be the only dengue vaccine approved in Brazil for use in people without the need for pre-vaccination testing and regardless of previous exposure. ANVISA’s approval follows similar decisions by the European Commission in December, and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) last month. The company continues to advance regulatory filings in other dengue-endemic countries in Asia and Latin America. In ...
Healthcare startups may turn to traditional large banks and prioritize conserving cash after the collapse of regional lenders Silicon Valley Bank and Signature Bank last week. The fall of SVB created a hole in the life sciences startup sector, locking out funds temporarily for some companies and cutting off a source of short-term cash for healthcare startups. A major bank and lender for healthcare companies, SVB last year had clients in nearly half of U.S. venture-backed technology and life sciences companies. “At this very moment, I think most of the companies are flying to safety, to the big banks,” said Ian Chiang, partner at healthcare investing company Flare Capital, “Perhaps it’s a little bit of a knee-jerk reaction to see what would happen.” However, companies may still deposit with regional banks, depending on size. Healthcare unicorns, meaning companies with valuations exceeding $1 billion, are likely not going to be able to have ...
China National Medical Products Administration has accepted the review of GSK’s drug application for Nucala (mepolizumab) as an add-on maintenance treatment for severe eosinophilic asthma (SEA). If given approval, Nucala will become the first targeted anti-Interleukin-5 (IL-5) treatment in the country for adult and adolescent patients with SEA. A first-in-class monoclonal antibody to target IL-5, Nucala was first approved in 2015 for SEA in the US. It prevents IL-5 from binding to its receptor on the surface of eosinophils, lowering blood eosinophils and maintains them under normal levels. The application to China National Medical Products Administration has been submitted based on positive data from Phase III trial undertaken on patients in China and the global SEA development programme, which included three important clinical trials – DREAM 2, MENSA3 and SIRIUS4. These trials established the efficacy and safety profile of mepolizumab for SEA patients. The Phase III trial for 52 weeks studied the ...
Zavzpret’s approval expands Pfizer’s migraine portfolio that includes an oral therapy for acute and preventive treatment. The US Food and Drug Administration (FDA) has granted approval for Pfizer’s Zavzpret (zavegepant) for the acute treatment of migraine in adult patients with or without aura. Zavzpret is claimed to be the first and only calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray approved to treat migraine. It is a third-generation, selective and structurally unique, small molecule CGRP receptor antagonist and is currently under clinical development with oral and intranasal formulations. The company stated that the regulatory approval expands its migraine portfolio, which includes oral therapy for acute and preventive treatment. Pfizer Global Biopharmaceuticals Business president and chief commercial officer Angela Hwang said: “The FDA approval of Zavzpret marks a significant breakthrough for people with migraine who need freedom from pain and prefer alternative options to oral medications. “Zavzpret underscores Pfizer’s commitment to delivering an additional treatment ...
Novo Nordisk will reduce the list price of four legacy insulin brands, including NovoLog and NovoLog Mix 70/30, by 75%. Novo Nordisk has announced plans to reduce the US wholesale acquisition cost (WAC), also known as the list price, of several pre-filled insulin pens and vials by up to 75% for people living with type 1 and type 2 diabetes. Set to come into effect from 1 January 2024, these changes will be applicable for products including pre-filled pens and vials of basal (long-acting), bolus (short-acting) and pre-mix insulins, Levemir, Novolin, NovoLog and NovoLog Mix 70/30 in specific. Novo Nordisk Market Access & Public Affairs senior vice-president Steve Albers said: “We have been working to develop a sustainable path forward that balances patient affordability, market dynamics, and evolving policy changes. “Novo Nordisk remains committed to ensuring patients living with diabetes can afford our insulins, a responsibility we take seriously.” The company will reduce the ...
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