Tag: Amyloidosis

Share on PinterestA researcher handles a petri dish while observing a CRISPR/Cas9 process through a stereomicroscope. Gregor Fischer/picture alliance via Getty Images Transthyretin cardiac amyloidopathy (ATTR-CM) is a rare but severe and progressive heart condition where sticky, toxic proteins build up in the heart muscle and cause heart failure. Current…

Not many stocks are truly capable of generating a 1,000% return on investment within a reasonable time frame. Even so, a fair number of next-generation technology stocks have hit this lofty milestone over the years. In almost every case, the market woefully underestimated the company’s core value proposition ahead of…

For the first time, a study has shown that cardiac amyloidosis, a deadly heart condition, can be reversed.  Cardiac amyloidosis is a heart disorder caused by deposits of an abnormal protein called amyloid in the heart tissue. The amyloid deposits disrupt the normal functioning of the heart and impede the…

7 June 2023 Three men who had heart failure caused by the build-up of sticky, toxic proteins are now free of symptoms after their condition spontaneously reversed in an unprecedented case described by a team at UCL and the Royal Free Hospital.  The condition, a form of amyloidosis affecting the…

Credit: SERGII IAREMENKO / Getty Images A little over a decade ago, a paper was published in Science1  that made science fiction a reality. Emmanuel Charpentier, Jennifer Doudna, and colleagues reported that they had identified a means of harnessing an element of a bacterial immune system to carry out genome…

Treatment with Onpattro (patisiran) for 1.5 years leads to sustained reductions in disability progression among people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or damage to the heart. That’s according to interim data from the ongoing open-label extension (OLE) portion of the Phase 3 APOLLO-B clinical trial (NCT03997383). That trial’s initial…

Key Points Liver metastasis, which is highly prevalent in advanced malignancies, was associated with poor prognosis. Contemporary therapies in liver metastasis were associated with the lack of metastatic-targeting ability, significant systemic toxicities and incapability of tumor microenvironment (TME) modulations. Lipid nanoparticles (LNP)-based strategies would overcome the deficiencies of conventional approaches…

The long-term extension of the phase 3 APOLLO-B study confirmed the efficacy and safety of patisiran in patients with transthyretin (ATTR) amyloidosis and highlights the importance of early treatment. Functional capacity, health status, and quality of life were preserved throughout the follow-up period. ATTR amyloidosis is a progressive and fatal…

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new results from an interim analysis of exploratory data from the open-label extension (OLE) period of the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy….

– Continued Treatment with Patisiran in Open-Label Extension Period Showed Evidence of Sustained Benefit across Measures of Functional Capacity and Health Status and Quality of Life, as well as Cardiac Stress and Injury, through 18 Months – – Safety Profile Consistent with that Observed in 12-Month Double-Blind Period, with No…

– Continued Treatment with Patisiran in Open-Label Extension Period Showed Evidence of Sustained Benefit across Measures of Functional Capacity and Health Status and Quality of Life, as well as Cardiac Stress and Injury, through 18 Months – – Safety Profile Consistent with that Observed in 12-Month Double-Blind Period, with No…

DUBLIN, May 18, 2023 /PRNewswire/ — The “Global CRISPR and Cas Genes Market, By Product & Service, By Application, By End-use By Region – Industry Trends and Forecast to 2030” report has been added to  ResearchAndMarkets.com’s offering. Research_and_Markets_Logo The CRISPR and Cas Genes market, valued at USD 3.17 billion in 2022,…

Morie A. Gertz, MD, chair, General Internal Medicine, Mayo Clinic Comprehensive Cancer Center, discusses the rationale for the phase 3 AFFIRM-AL trial (NCT04973137) in patients with amyloid light chain (AL) amyloidosis and elaborates on the challenges that may arise when enrolling patients to this trial. Dr Gertz on the Objectives…

News Home Tuesday, May 16, 2023 12:47 PM | InvestorsObserver Analysts Mentioned in this article The market has been high on Intellia Therapeutics Inc (NTLA) stock recently. NTLA gets a Bullish score from InvestorsObserver Stock Sentiment Indicator. Intellia Therapeutics Inc has a Bullish sentiment reading. Find out what this means…

PRESS RELEASE Published May 17, 2023 DelveInsight’s, “CRISPR Therapies Pipeline Insight 2023” report provides comprehensive insights about 25+ companies and 30+ pipeline drugs in CRISPR Therapies pipeline landscape. It covers the CRISPR Therapies pipeline drug profiles, including CRISPR Therapies clinical trials and nonclinical stage products. It also covers the therapeutics…

Bergmann, O. et al. Evidence for cardiomyocyte renewal in humans. Science 324, 98–102 (2009). Article  CAS  PubMed  PubMed Central  Google Scholar  Senyo, S. E. et al. Mammalian heart renewal by pre-existing cardiomyocytes. Nature 493, 433–436 (2013). Article  CAS  PubMed  Google Scholar  Bergmann, O. et al. Dynamics of cell generation and…

Forward-looking Information This Quarterly Report on Form 10-Q contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). These statements may be…

1. Introduction The discovery of prokaryotic adaptive defense systems, which consist of the clustered regularly interspaced short palindromic repeat (CRISPR) and CRISPR-associated (cas) genes [1,2,3], has led to the development of efficient tools for genome editing in different organisms. Type II CRISPR-Cas systems include the Cas9 protein, which forms an…

Amvuttra (vutrisiran) and Onpattro (patisiran) showed similar safety and efficacy for familial amyloid polyneuropathy (FAP) patients, according to new analyses from the Phase 3 HELIOS-A trial that supported Amvuttra’s regulatory approval. Both therapies led to similar reductions in transthyretin, the protein that accumulates in FAP, and they were associated with comparable…

About a month after Ionis Pharmaceuticals announced positive results from its phase 3 NEURO-TTRansform study (NCT04136184) assessing its investigational agent eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), further details of the study were presented at the 2023 American Academy of Neurology (AAN) Annual Meeting, held April 22-27, in…

AstraZeneca have broken out the data they teased last month for transthyretin amyloidosis (ATTR) therapy eplontersen, showing that the drug was effective at slowing down disease progression in patients with polyneuropathy caused by the disease. The results of the NEURO-TTRansform trial were presented at the American Academy of Neurology (AAN)…

Exploring the Promising Potential of siRNA Drugs: From Development History to Future Outlook siRNA drugs are a promising class of therapeutic agents that leverage RNA molecules to silence disease-causing genes. This innovative approach holds potential for treating a wide range of illnesses resulting from overexpressed or dysfunctional genes, including but…

20 Apr 2023 Retroviruses lurking in the human genome aren’t the only potential troublemakers in neurodegenerative diseases (see Part 5 of this series). Herpes simplex virus type 1 (HSV-1) integrates into the genome of trigeminal ganglia nerves, can lie dormant for decades, and flare up as cold sores throughout a person’s…

PRESS RELEASE Published April 12, 2023 DelveInsight’s, “CRISPR Therapies Pipeline Insight 2023” report provides comprehensive insights about 25+ companies and 30+ pipeline drugs in CRISPR Therapies pipeline landscape. It covers the CRISPR Therapies pipeline drug profiles, including CRISPR Therapies clinical trials and nonclinical stage products. It also covers the therapeutics…

Pictured: Illustration of CRISPR-Cas9 editing DNA / iStock, Artur Plawgo Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials. As these programs mature, 2023 could be a pivotal year for companies…

With the aid of CRISPR-Cas9 technology, scientists are shifting the trajectories of the evolution of organisms. A researcher is now proposing that this human control of evolutionary course be called “genetic welding.” The makeup of a basic CRISPR-Cas9 synthetic gene drive element consists of the Cas9 gene and guide RNA…

Pictured: Intellia office building with logo/company courtesy With several recent milestones, Intellia Therapeutics is setting a solid foundation for gene editing in rare diseases like hereditary angioedema (HAE) and transthyretin (ATTR) amyloidosis with an eye toward broader patient populations. Laura Sepp-Lorenzino, Ph.D., EVP and chief scientific officer, joined Intellia in…

Credit: S. Harris/Springer Nature Limited Vertex and CRISPR Therapeutics have submitted their CRISPR-based ex vivo cell therapy exagamglogene autotemcel (exa-cel) for FDA approval, for sickle cell disease (SCD) and beta-thalassemia. A regulatory decision on the gene-editing candidate is expected within 8 to 12 months. The companies have also filed for…

More than one year of treatment with eplontersen safely reduced symptoms of nerve damage and improved quality of life for adults with familial amyloid polyneuropathy (FAP), according to top-line data from the Phase 3 NEURO-TTRansform trial. The experimental therapy from Ionis Pharmaceuticals and AstraZeneca also significantly lowered blood levels of…

The CRISPR & Cas Genes market growth is driven by increasing number of clinical trials associates with CRIPR technology and rising of development of novel technologies. The CRISPR & Cas genes market revenue will reach at USD 8.96 billion by 2030 with a CAGR of around 17.03% from 2022 to…

Image © Tippapatt | iStock Alexander Gebauer, the Chief Executive Officer at Secarna Pharmaceuticals GmbH & Co. KG looks at a new approach to solve unmet medical needs with antisense therapies Despite major therapeutic advances, there is a variety of diseases that are still not adequately addressed by traditional approaches…

An inside view on a platform that could help take RNA therapeutics to the next level of delivery Arthur T Suckow | 03/21/2023 | 5 min read It was during my PhD in Pharmacology at UC San Diego that I found my desire to launch a biotech company. Amazed by…

Keratosis Pilaris Treatment Market Is Expected to rise at A CAGR Of 4.26% during the forecast period 2023 to 2031: Growth Plus Reports Newark, New Castle, USA, March 20, 2023 (GLOBE NEWSWIRE) — The global keratosis pilaris treatment market will register a revenue CAGR of 4.26%, owing to the high…

KEY POINTS Familial Mediterranean fever (FMF) is the most common monogenic autoinflammatory disorder; it is characterized by self-limited episodes of fever, polyserositis and elevated inflammatory markers. While symptoms are nonspecific, FMF should be suspected in patients with recurrent febrile episodes accompanied by peritonitis, pleuritis, pericarditis and elevated C-reactive protein, especially…

Bacteria function strikingly similarly to human cells when it comes to defending against invaders, sharing the same essential machinery needed to turn immune pathways on and off. By examining these bacterial processes, we can learn much about how the human body functions. Jennifer Doudna, a biologist at the University of…

Bredesen DE, Rao RV, Mehlen P. Cell death in the nervous system. Nature. 2006;443:796–802. Article  CAS  PubMed  PubMed Central  Google Scholar  Fricker M, Tolkovsky AM, Borutaite V, Coleman M, Brown GC. Neuronal cell death. Physiol Rev. 2018;98:813–80. Article  CAS  PubMed  PubMed Central  Google Scholar  West MJ, Coleman PD, Flood DG,…

Evgenii Kovalev Background Silence Therapeutics (NASDAQ:SLN) is a UK-based biotechnology company specializing in RNA interference [RNAi] technology. The company is focused on developing and commercializing a pipeline of therapies using its proprietary platform technology to target and silence disease-causing genes. Silence Therapeutics has a diversified pipeline of preclinical and clinical…

Doudna JA, Charpentier E. Genome editing. The new frontier of genome engineering with CRISPR-Cas9. Science. 2014;346(6213):1258096. Article  PubMed  Google Scholar  Doudna JA. The promise and challenge of therapeutic genome editing. Nature. 2020;578(7794):229–36. Article  CAS  PubMed  PubMed Central  Google Scholar  Nambiar TS, Baudrier L, Billon P, Ciccia A. CRISPR-based genome editing…

A life-saving gene editing treatment trial has had life changing results, curing sufferers of a condition that causes unpredictable cases of swelling. About 100 New Zealanders have the condition and seven of them have been at the forefront of the trial. For generations, pain has been passed down through the…

The Food and Drug Administration has cleared Intellia Therapeutics to start human testing in the U.S. of an experimental CRISPR medicine for a rare swelling disorder, a sign the agency could be getting more comfortable with medicines that edit genes inside the body. Intellia on Thursday said the FDA will…

News Home Thursday, March 02, 2023 11:23 AM | InvestorsObserver Analysts Mentioned in this article The market has been high on Intellia Therapeutics Inc (NTLA) stock recently. NTLA gets a Bullish score from InvestorsObserver Stock Sentiment Indicator. Intellia Therapeutics Inc has a Bullish sentiment reading. Find out what this means…

Our management’s discussion and analysis of our financial condition and results of operations are based upon our consolidated financial statements included in this Annual Report on Form 10-K, which have been prepared by us in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and…

21 Feb 2023 Researchers have known since 1993 that Huntington’s disease is caused by a repeat expansion in the huntingtin gene, yet figuring out how to correct this defect has not been easy. One approach, suppressing expression of the mutant protein with antisense oligonucleotides, has so far been unsuccessful in…

[18F]florbetaben PET imaging of patients with suspected cardiac amyloidosis has the potential to become an important tool to simplify and shorten the time to diagnosis, providing earlier and appropriate access to therapy and improved monitoring of such interventions BERLIN, Germany (PRWEB) February 07, 2023 Life Molecular Imaging (LMI) announces…

The tech-heavy Nasdaq represents a collection of more risky stocks than the other major indexes and that is reflected by a poorer performance in bear markets and a better display during bull runs. But the risks associated with the Nasdaq are mere child play compared to edgier funds such as…

Natali_Mis Editas Medicine (NASDAQ:EDIT), Intellia Therapeutics (NASDAQ:NTLA), and Beam Therapeutics (NASDAQ:BEAM) were among clinical-stage biotechs to receive Overweight ratings at Cantor Fitzgerald as the firm launched its coverage on several gene editing companies. The analyst Rick Bienkowski sees multiple catalysts for gene editing companies at a time when the subsector…

In the face of the bear market, biotechnology stocks have been performing better than the broader market, but Morningstar analysts say there are still plenty of undervalued stocks in this dynamic industry. That includes big names such as Moderna MRNA, which was in the spotlight for its coronavirus vaccine, and…

Source: vxhal/ShutterStock.com Gene editing stocks present themselves as one of the more lucrative investments in the healthcare sector. Gene editing, a revolutionary take on the science of genetic engineering, has the potential to countless lives around the globe. By introducing genetic material into cells, scientists can correct genetic abnormalities in…

Ten years ago, a little-known bacterial defense mechanism skyrocketed to fame as a powerful genome editor. In the decade since, CRISPR-Cas9 has spun off multiple variants, expanding into a comprehensive toolbox that can edit the genetic code of life. Far from an ivory tower pursuit, its practical uses in research,…

Lipid nanoparticles (LNPs) have recently risen to prominence as the technology platform that enables the delivery of mRNA, the key component of the Moderna and BioNTech/Pfizer COVID-19 vaccines. We estimate that by the end of 2021, more than two billion people had received a COVID-19 mRNA vaccine or booster that…

Thanks to a breakthrough in biotechnology made by two Nobel Prize-winning researchers in 2020, it will soon be possible to treat genetic diseases and cancer through selective gene sharing. The innovative method CRISPR/Cas9 it has also aroused interest among investors, thanks to which both Nobel laureates were able to create…

Anzalone, A. V., Koblan, L. W. & Liu, D. R. Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. Nat. Biotechnol. 38, 824–844 (2020). Article  CAS  Google Scholar  Clement, K., Hsu, J. Y., Canver, M. C., Joung, J. K. & Pinello, L. Technologies and computational analysis strategies for…

Published a day ago Submitted by CRB Decades of scientific progress in understanding the root cause of many formerly intractable diseases has opened up new treatment options, namely through RNA-based therapies. This second wave of biopharma, in which our understanding of the ways coding portions of our genome relate to…

liulolo/iStock via Getty Images Investment Thesis If you had invested in the CRISPR / Cas9 gene editing pioneer Intellia Therapeutics (NASDAQ:NTLA) five years ago, you would likely be well satisfied with your return to date – a ~285% return on your investment – although you may also feel that it…

Serum TTR reductions were sustained at all doses tested with follow-up now reaching 12 months in the 0.1 and 0.3 mg/kg and six months in the 0.7 and 1.0 mg/kg cohorts Pharmacokinetic modeling and simulation indicated that an 80 mg fixed dose provides similar exposure to the 1.0 mg/kg dose,…

Have you heard of purple tomatoes? Or golden rice? If you haven’t, you might be surprised to know that these have actually been created by scientists via genetic engineering. These are genetically modified foods, with purple tomatoes containing 10 times more antioxidants than a normal red tomato. On the other…

Andy/iStock via Getty Images Intellia Therapeutics (NTLA -3.1%) was upgraded by Brookline Capital Management to Buy from Hold with a $91 price target. Brookline analyst Leah Cann said the patent risk is unchanged, but the valuation has become attractive. In February, the U.S. Patent and Trademark Office backed the Broad…

This story originally appeared on Zacks Editas Medicine, Inc. EDIT has made rapid progress in the development of its lead pipeline candidate, EDIT-101, which employs CRISPR gene editing, to treat Leber congenital amaurosis type 10 (LCA10) — a rare genetic illness that causes blindness. – Zacks The company is evaluating…

Courtesy Intellia Therapeutics Intellia Therapeutics is celebrating another first today as the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for NTLA-5001, its first wholly-owned ex vivo CRISPR genome editing candidate. NTLA-5001 is being developed for the treatment of acute myeloid leukemia (AML). Intellia…

The CRISPR-based biotech startup Mammoth Biosciences is officially a unicorn, the company says. The billion dollar valuation comes on the back of a $150 million series D round led by Redmile Group, with participation from Foresite Capital, Senator Investment Group, Sixth Street, Greenspring Associates, Mayfield, Decheng Capital, Plum Alley and…

Bioengineers reimagine the CRISPR system as a Swiss Army knife packed with many applications. They repurposed it to make a mini version, called CasMINI, that can easily be delivered into human cells for gene therapy and treat various ailments. The new technology, described in the study titled “Engineered Miniature CRISPR-Cas…

Therapeutic applications of genome editing were envisioned at least as early as the mid-1990s, when the first sequence-specific genome editing technologies emerged. Initially, such applications were considered distant prospects, but by 2012, they suddenly seemed near to hand. It was at that time that CRISPR technologies emerged. CRISPR, which stands…

Photo courtesy of Intellia Therapeutics Sports fans know that nothing is better for the chemistry of a team than winning, and on the CRISPR field, Intellia Therapeutics is on a winning streak.    On June 26, Intellia set the biotech world on fire, announcing the first-ever clinical data supporting the safety…

Broadcast Date: September 16, 2021Time: 8 am PT, 11 am ET, 17:00 CET When CRISPR-Cas9 gene-editing technology exploded onto the life science stage almost a decade ago, it was widely touted as a potentially curative therapy for many genetic diseases, using various ex vivo and in vivo delivery methods. That promise is…

Unlike most other CRISPR/Cas-9 therapies that are ex vivo treatments in which cells are modified outside the body, this study was successful with an in vivo treatment Use of CRISPR-Cas9 gene editing technology for therapeutic purposes can be a boon for clinical laboratories. Not only is this application a step…

Shares of Editas Medicine, Inc. EDIT have rallied 78.7% in the past three months compared with the industry’s increase of 7.4%. Zacks Investment Research Image Source: Zacks Investment Research The company has made rapid progress in the development of its lead pipeline candidate, EDIT-101, in this time frame. EDIT-101 employs…