Alnylam Pharmaceuticals’ investigational RNAi therapeutic has shown promise in patients with early-onset Alzheimer’s disease, according to phase 1 results presented by the company at this year’s Alzheimer’s Association International Conference (AAIC). Interim results from the early-stage study showed that a single injection of ALN-APP, which is designed to switch off the production of amyloid precursor protein (APP) in the central nervous system, was able to rapidly reduce levels of the protein, with clinical effect sustained over six months. ALN-APP is being developed in collaboration with Regeneron Pharmaceuticals for both Alzheimer’s disease and the related disorder cerebral amyloid angiopathy (CAA) and is now the first investigational RNAi therapeutic to demonstrate gene silencing in the human brain. “We’ve known for decades that mutations that increase APP production, or alter its proteolysis, cause early-onset Alzheimer’s disease, early-onset CAA or both,” said Dr Sharon Cohen, neurologist and medical director, Toronto ...
Sanofi and Scribe Therapeutics have announced an expanded collaboration agreement worth over $1.2bn to advance in vivo genetic medicines for sickle cell and other genomic diseases. The agreement follows the launch of the companies’ existing partnership focused on developing ex vivo editing of natural killer cell therapies for cancer. Under the latest deal, Sanofi will gain an exclusive licence to use Scribe’s CRISPR X-Editing genome editing technologies to develop in vivo therapies. In exchange, Scribe will receive a $40m upfront payment and will also be eligible for more than $1.2bn based on the successful completion of certain development and sales milestones. Sanofi will largely be responsible for any resulting candidates, but Scribe will have the opportunity to opt into sharing the development costs and profits on one future programme in the US. Christian Mueller, global head of Genomic Medicine Unit at Sanofi, said: “We’re ...
Pfizer is looking to enrich its pipeline with up to 10 new innovative medicines by partnering with startup creator Flagship Pioneering, the companies announced Tuesday. Under the terms of the agreement, Pfizer and Flagship will each contribute an upfront investment of $50 million. The collaboration will leverage Flagship’s rich ecosystem of biotechnology platforms and more than 40 human health companies. Pfizer and Flagship will explore up to 10 single-asset programs, which Pfizer will fund and have the option to acquire. Flagship and its companies will be eligible to receive up to $700 million in milestones and royalties for each program that hits the market, giving Tuesday’s deal a maximum potential value of $7 billion. This partnership will bring together expertise from Pfizer and Flagship “to maximize discovery and development potential from inception to impact,” Paul Biondi, executive partner at Flagship and president of Pioneering Medicines, said in a statement. ...
Pictured: A silhouette of a woman sitting on the floor with her head in her hands/iStock, simpson33 Neumora Therapeutics is making big moves this week. On Tuesday the young biotech announced the initiation of a Phase III program for its potential depression treatment along with a new CEO to lead the way. The nearly two-year-old startup posted statistically significant results for treating moderate-to-severe major depressive disorder (MDD) in its Phase II trial of navacaprant, a kappa opioid receptor (KOR) antagonist. Initiated by BlackThorn Therapeutics prior to its acquisition by Neumora, the trial was amended to include those more serious MDD patients, the population in which it appears to be most effective. In moderate-to-severe patients, navacaprant had statistically significant results in lowering patients’ 17-item Hamilton Rating Scale for Depression score by 3 points at week 4 and 2.8 points by week 8. Across all patients, which included mildly depressed patients, navacaprant did not achieve ...
Performance of CoDoC in breast cancer prediction compared to that of a standalone predictive AI system and clinical readers. Credit: Nature Medicine (2023). DOI: 10.1038/s41591-023-02437-x A team of AI and medical specialists working with or for Google Research and Google DeepMind, has developed an AI based system designed to judge the confidence level of existing AI systems used for analyzing medical scans as a means of improving analysis of diagnostic tools, such as mammograms or chest X-rays. In their paper, published in the journal Nature Medicine, the group describes how they built the system and how well it worked when tested. Fiona Gilbert, with the University of Cambridge’s Clinical School of Medicine, has published a News & Views piece in the same journal issue outlining the work done by the team on this new effort. Over the past several years, as AI applications have become more refined, the medical establishment has embraced the ...
Pfizer and Flagship Pioneering are partnering in a drug research alliance leveraging the capabilities of the more than 40 biotech startups in the venture capital firm’s portfolio. The new partners aim to develop drugs for unmet needs, including those in broad patient populations. Pharmaceutical companies frequently strike deals with smaller biotech companies as a way to build up their drug pipelines, and Pfizer is no exception. But in a twist to this strategy, the pharma giant is now turning to Flagship Pioneering, the venture capital firm whose highest profile creation might be the messenger RNA company Moderna. The alliance announced Tuesday calls for Flagship and Pfizer to each invest $50 million up front. The cash will go toward the R&D of 10 single-asset programs. Flagship does not create single-asset companies. The startups that spring from its labs are all formed around platform technologies, each with the capability of yielding ...
Recently, Zhejiang Pukang Biotechnology Co., Ltd. has completed the Phase IV clinical trial titled “Single-Arm, Multi-Center Evaluation of the Safety and Immunogenicity of Freeze-Dried Live Attenuated Hepatitis A Vaccine in Chinese Children Aged 18-24 Months” and received the final report. This Phase IV clinical study, involving a large sample size of 10,000 cases across multiple centers, aimed to observe the safety and immunogenicity of the freeze-dried live attenuated Hepatitis A vaccine in the real world. The study results show that after a single dose of the vaccine, the AEFI incidence rate in the 18-24 month-old children was 0.34%, with no serious AEFI reported, and an antibody seroconversion rate of 98.91%. This confirms the vaccine’s good safety and immunogenicity profile, providing richer data support for its widespread application. The freeze-dried live attenuated Hepatitis A vaccine (H2 strain), developed and produced by Zhejiang Pukang Biotechnology Co., Ltd., simulates ...
Black patients with implantable cardioverter defibrillators (ICDs) have a significantly higher burden of disease than white patients with the same device, according to a new study from University of Rochester Medical Center (URMC) cardiology researchers. Analyzing data from clinical trials conducted over a 20-year period by the Clinical Cardiovascular Research Center (CCRC) at URMC, investigators concluded that not only did Black patients with ICDs tend to be significantly younger than white patients, but they also had a higher rate of post-implant cardiac events and risk of death. When a patient is at risk for cardiac arrest, an ICD helps to monitor their heart rhythm, and if an abnormality is detected, the device delivers a shock of electricity to reset the heartbeat to a normal rhythm. The study, published in Circulation, examines the rate of events that a patient experiences after their ICD is implanted. After three years of ...
Bioengineers have found a way to program the size and shape of virus particles by combining viral protein building blocks and templates made from DNA. The resulting nanostructures could have applications in vaccine development and transporting drugs inside the body. Virus capsid proteins-;the proteins that shield the genome of a virus-;can be used to build precisely structured protein assemblies. Their shapes and geometry, however, depend largely on the virus strain. Reprogramming these assemblies, no matter the original viral blueprint, is an intriguing possibility for drug delivery and vaccine development. Scientists tackled the challenge by generating a “structured genome” template on which capsid proteins can assemble. To avoid deforming the flexible genome and creating unintended shapes, they used rigid DNA origami structures. These structures are only tens to hundreds of nanometres in length, but entirely made of DNA, which is folded accurately into the desired template shape. ...
Apellis Pharmaceuticals’ Syfovre, after achieving the first FDA approval for advanced eye disease geographic atrophy, has been associated with rare but severe side effects, according to the American Society of Retinal Specialists (ASRS).The group issued a letter to doctors Saturday flagging cases of eye inflammation and six reports of occlusive retinal vasculitis in patients who took the drug, BioPharma Dive reported. The condition is a type of inflammation that blocks blood flow to the retina and could potentially cause blindness.ASRS didn’t tie the safety issues to a specific batch of product but noted that the side effects began one to two weeks after a patient’s first Syfovre injection. The organization urged vigilance and close follow-up after administration, according to the news outlet. The news caused Apellis’ stock price to sink nearly 32% to $52.62 by Monday afternoon. The company puts the Syfovre-associated retinal vasculitis rates at 0.01% per ...
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