Tag: Amyloidosis

Cancer is one of the leading causes of disease-associated mortality, with a rising incidence worldwide [1]. Although many therapeutic methods have been used in cancer treatment, such as chemotherapy, radiotherapy, surgery, and targeted therapy, the overall therapeutic outcome remains unsatisfactory. Therefore, developing new therapeutic means is urgently needed. With advances…

These details matter because there are certain cardiovascular conditions linked to genetic variants that are more commonly identified in Black people, and these variants are often linked to ancestry. Take transthyretin amyloidosis, for example. The condition, which can cause a buildup of abnormal protein deposits in the body, is associated…

Introduction The incidence and mortality rates of AF are continuously increasing, leading to serious complications such as heart failure and stroke (Fig. 1) (Sagris et al., 2021). The occurrence and development of AF involve different mechanisms and interactions (Kornej et al., 2020). AF often progresses from paroxysmal to persistent (Nattel…

SHANGHAI, Dec. 17, 2023 /PRNewswire/ — Yoltech Therapeutics today announced that the first patient has been administered a dose of YOLT-201, the company’s first in vivo genome editing candidate being developed as a single dose, Which is a potentially curative therapy for hereditary transthyretin amyloidosis. With cardiomyopathy (ATTR-CM). The study…

SHANGHAI, Dec. 17, 2023 /PRNewswire/ — YolTech Therapeutics today announced that the first patient has been dosed with YOLT-201, the company’s first in vivo genome editing candidate being developed as a single dose, potentially curative therapy for hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The study by YolTech is a single-arm,…

A little more than a year of treatment with eplontersen was found to stabilize or even improve the heart’s structure and function in people with familial amyloid polyneuropathy (FAP) who also were experiencing symptoms of heart disease, known as cardiomyopathy. That’s according to an exploratory analysis of data from the…

In the realm of genetic manipulation, Crispr technology emerges as a double-edged sword. Enabling precise gene editing, it holds promise for eradicating diseases like sickle cell through treatments like Casgevy. Yet, with the power to shape the very fabric of life, ethical concerns loom large. While some foresee a future…

Credit: Pixabay/CC0 Public Domain In 2007, Luciano Marraffini struck out on what was then a scientifically lonely path: to understand CRISPR, which had been discovered in bacteria only about a decade before. Seventeen years later, we all know what CRISPR is: a revolution in medicine; a once-in-a-lifetime scientific breakthrough; the…

In a landmark decision last week, the US Food and Drug Administration (FDA) approved two treatments involving cell-based gene therapies.  An agency within the US Department of Health and Human Services, the FDA assures the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for…

Bone marrow aspirate cytology of multiple myeloma: ©David A Litman – stock.adobe.com Findings from the phase 3 PERSEUS study (NCT03710603) presented at the 2023 ASH Annual Meeting showed that subcutaneous daratumumab (Darzalex) followed by autologous stem cell transplant (ASCT) and daratumumab, bortezomib (Velcade), lenalidomide (Revlimid), and dexamethasone (D-VRd) consolidation and…

Recently, bluebird bio, Inc. BLUE obtained FDA approval for its third gene therapy, lovotibeglogene autotemcel (lovo-cel), for the treatment of sickle cell disease (“SCD”) in patients aged 12 and older who have a history of vaso-occlusive events (“VOEs”). The FDA approved lovo-cel under the brand name Lyfgenia. Concurrently, it also…

Intellia Therapeutics (NASDAQ: NTLA) and CRISPR Therapeutics (NASDAQ: CRSP) have one big thing in common: They both specialize in the hot area of gene editing, or the repairing of faulty genes responsible for disease. But CRISPR Therapeutics is farther along on the path to commercialization, recently scoring its first product…

Introduction Cancer, also called malignancy, is a group of diseases caused by the rapid multiplication and spread of malignant cells.1,2 According to statistics from the National Cancer Center, more than 100 types of cancer have been identified.3 The ability of malignant tumor cells to metastasize via the blood-lymphatic system and…

Huang, X. et al. The landscape of mRNA nanomedicine. Nat. Med. 28, 2273–2287 (2022). Article  CAS  Google Scholar  Rohner, E., Yang, R., Foo, K. S., Goedel, A. & Chien, K. R. Unlocking the promise of mRNA therapeutics. Nat. Biotechnol. 40, 1586–1600 (2022). Article  CAS  Google Scholar  Brown, A., Shao, S.,…

Pictured: A collage of medical notes and anatomy/Nicole Bean for BioSpace With all the recent headlines being made in genetic medicine, a presentation from Turn Biotechnologies announcing the first successful in vivo delivery of messenger RNA to the skin with no off-target effects flew largely under the radar. While Turn…

Just one more article to add to the thousands that have been written about CRISPR-Cas9, but the hype is absolutely worth it. CRISPR-Cas9 has caused what could be described as a revolution in the world of genetic engineering. Domains that were once considered out of reach are now fully within…

New research published in Alzheimer’s and Dementia found that differences in microglial “immunometabolism endophenotypes”, defined by interactions between immune and metabolic reactions in the brain, may contribute to the pathogenesis and disease progression of Alzheimer’s disease (AD) in females.1 Although women comprise a higher proportion of AD cases than men,…

Limitations of siRNA as a proprietary drug Naked siRNA is easily degraded by nucleases in blood, and its relatively high molecular weight, negative charge, and hydrophilicity make it difficult to penetrate cell membranes. siRNA tends to accumulate in the kidney and be excreted in urine or is captured by the…

Today, the United Kingdom became the first country to give regulatory approval to a medical procedure that uses CRISPR gene editing. The Medicines and Healthcare products Regulatory Agency (MHRA) approved Casgevy, a therapy that will be used to treat sickle cell disease and beta thalassemia (also called β -thalassaemia).  [Related:…

MU School of Medicine students presented roughly 250 research projects at the annual 2023 Health Sciences Research Day (HSRD), held Nov. 10, 2023, and featured the work of undergraduates, medical and nursing students, PhD students and postdoctoral trainees. The research posters covered a variety of topics within the health care…

News Home Tuesday, November 14, 2023 10:04 AM | InvestorsObserver Analysts Mentioned in this article The market has been down on Intellia Therapeutics Inc (NTLA) stock recently. NTLA gets a Bearish score from InvestorsObserver Stock Sentiment Indicator. Intellia Therapeutics Inc has a Bearish sentiment reading. Find out what this means…

Drug Pharmacologic Class Proposed Indication Status Cardiovascular Disorders Patisiran (Alnylam) Transthyretin-directed small interfering RNA Treatment of the cardiomyopathy of transthyretin-mediated amyloidosis. Complete Response Letter issued Dermatological Disorders Dupilumab (Regeneron and…

– As previously announced, the primary endpoint (a hierarchical analysis inclusive of all-cause mortality and frequency of cardiovascular-related hospitalization) was met (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001) – The placebo and acoramidis time-to-first event Kaplan-Meier (K-M) curves for a composite of all-cause mortality (ACM) and…

You are reading Labiotech’s weekly roundup of the latest biotech news. Korro Bio and Frequency Therapeutics close merger Last Friday, Korro Bio announced the completion of the previously announced business combination between Frequency Therapeutics, Inc. and the entity formerly known as Korro Bio. The combined company will now operate under…

A single infusion of NTLA-2001, an experimental gene editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). These updated, interim data cover 65 of 72 patients…

In a new post-hoc analysis of the phase 3 HELIOS-A study (NCT03759379) assessing vutrisiran (Amvuttra; Alnylam), an FDA-approved RNA interference therapeutic, the therapy demonstrated benefit in key measures, compared with external placebo, across all baseline polyneuropathy severities among patients with hereditary transthyretin-mediated (hATTR) amyloidosis. These results suggest patients who initiate…

BridgeBio Pharma, Inc. – Presented detailed positive results from the ATTRibute-CM Phase 3 study of acoramidis at the European Society of Cardiology (ESC) Congress, demonstrating that patients survived more and were hospitalized less than has been seen in other interventional studies of transthyretin amyloid cardiomyopathy (ATTR-CM) to the Company’s knowledge,…

Updated data from over 60 patients showed consistent, deep and durable serum TTR reduction achieved with a single dose of NTLA-2001, including in 29 patients who have now reached 12 months or more of follow-up NTLA-2001 was generally well-tolerated across both polyneuropathy and cardiomyopathy arms at all dose levels tested…

By Jonathan D. Grinstein, PhD Fyodor Urnov, PhD, is a pioneer in the field of genome editing and one of the scientists most invested in expanding the availability and utility of CRISPR-based therapies to the broadest possible population. He envisions a world in which genome editing can treat the nearly…

Alnylam Pharmaceuticals, Inc. announced that results from the APOLLO-B Phase 3 study of investigational patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were published online in the New England Journal of Medicine (NEJM). The data reported in the APOLLO-B Phase 3 study publication demonstrate that patisiran, an RNAi therapeutic targeting transthyretin (TTR), preserved…

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that results from the APOLLO-B Phase 3 study of investigational patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were published online in the New England Journal of Medicine (NEJM). The data reported in the APOLLO-B Phase…

– Treatment with an RNAi Therapeutic Preserved Functional Capacity and Health Status and Quality of Life Compared with Placebo at 12 Months – – Patisiran Demonstrated an Encouraging Safety and Tolerability Profile in Patients with the Cardiomyopathy of ATTR Amyloidosis – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics…

– Treatment with an RNAi Therapeutic Preserved Functional Capacity and Health Status and Quality of Life Compared with Placebo at 12 Months – – Patisiran Demonstrated an Encouraging Safety and Tolerability Profile in Patients with the Cardiomyopathy of ATTR Amyloidosis – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics…

Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study, which is expected to start by the end of the year, follows the recent approval…

Intellia Therapeutics, Inc. NTLA is a clinical-stage genome editing company focused on developing CRISPR/Cas9-based therapeutics. The company is evaluating its leading in vivo genome-editing candidates — NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis and NTLA-2002 for the treatment of hereditary angioedema (HAE). Earlier this week, the FDA cleared an…

Intellia Therapeutics, Inc. (NTLA Quick QuoteNTLA – Free Report) is a clinical-stage genome editing company focused on developing CRISPR/Cas9-based therapeutics. The company is evaluating its leading in vivo genome-editing candidates — NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis and NTLA-2002 for the treatment of hereditary angioedema (HAE). Earlier this…

Drug Pharmacologic Class Proposed Indication Status Allergic Disorders Neffy (internal epinephrine; ARS Pharmaceuticals) Non-selective alpha and beta adrenergic agonist Treatment of severe allergic reactions, including anaphylaxis, for adults and children weighing at least 30kg. Complete Response Letter…

The world’s first investigational in vivo CRISPR-based gene editing therapy cleared for late-stage clinical development is expected to enter Phase III in late 2023. The Investigational New Drug (IND) application for Intellia Therapeutics’ in vivo CRISPR-based candidate NTLA-2001, has been cleared as a gene editing therapy for transthyretin (ATTR) amyloidosis…

(RTTNews) – Intellia Therapeutics, Inc. (NTLA) announced the FDA has cleared the company’s Investigational New Drug application for NTLA-2001 for the treatment of transthyretin amyloidosis with cardiomyopathy. The global Phase 3 study of NTLA-2001, an in vivo CRISPR-based gene editing candidate, is anticipated to initiate by year-end 2023. “The FDA…

Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy….

Intellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the company?s Investigational New Drug (IND) application for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy. The global Phase 3 study of NTLA-2001, an in vivo CRISPR-based gene editing candidate, is expected to initiate…

This year has been a tale of two markets for growth stocks. Large-cap growth companies with exposure to either artificial intelligence (AI) or weight-loss products have posted enormous returns for shareholders in 2023. Small- to mid-cap growth stocks, on the other hand, have softened in the face of rising interest…

Cardiac amyloidosis OGphoto Alnylam logo We rate Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) a Buy on the dip after the complete response letter, CRL for Patisiran. The investment thesis is based on the high probability of success for Vutrisiran, the company’s next-generation inverse RNA or RNAi candidate, in treating cardiac amyloidosis in…

The US Food and Drug Administration (FDA) has declined to approve the RNA interference (RNAi) therapeutic agent patisiran (Onpattro, Alnylam Pharmaceuticals) for treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, the company has announced. ATTR amyloidosis is an underdiagnosed, rapidly progressive, debilitating, fatal disease caused by misfolded TTR proteins, which accumulate…

An Alnylam Pharmaceuticals drug developed to treat a rare disease’s potentially fatal effects on the heart has fallen short in its bid for FDA approval, costing the company a chance to immediately challenge a Pfizer product that has become a blockbuster seller in the indication. But the biotech still has…

In September 2023, the FDA granted approval to 3 agents, including motixafortide in combination with filgrastim to aid in hematopoietic stem cell mobilization in patients with multiple myeloma, momelotinib (Ojjaara) for the treatment of patients with myelofibrosis and anemia, and bosutinib for pediatric patients aged 1 year or older with…

Pictured: Illustration of a CRISPR-Cas9 system conducting edits on a DNA strand/iStock, Meletios Verras Regeneron on Tuesday announced it is expanding an existing collaboration agreement with Intellia Therapeutics to develop CRISPR-based gene editing therapies targeting neurological and muscular diseases. The expanded agreement will combine Regeneron’s proprietary delivery systems and antibody-targeted adeno-associated…

Regenereon and Intellia Therapeutics expanded a long-running research pact to develop additional CRISPR-based gene editing therapies focused on neurological and muscular diseases. Terms call for the companies to research two in vivo non-liver targets, according to a company press release issued Tuesday. Intellia will lead the design of the editing methodology…

Intellia Therapeutics NTLA announced that it has expanded its existing collaboration with Regeneron Pharmaceuticals, Inc. REGN to develop additional in vivo CRISPR-based gene-editing therapies focused on neurological and muscular diseases. Under the expanded collaboration, Regeneron will supply its proprietary antibody-targeted adeno-associated virus vectors and delivery systems, while Intellia will bring its proprietary Nme2…

Lo MT, Kauppi K, Fan CC, Sanyal N, Reas ET, Sundar VS, Lee WC, Desikan RS, McEvoy LK, Chen CH, Alzheimer’s Disease Genetics, C. Identification of genetic heterogeneity of Alzheimer’s disease across age. Neurobiol Aging. 2019;84(243):e241-243. doi.org/10.1016/j.neurobiolaging.2019.02.022. Article  Google Scholar  Nacmias B, Bagnoli S, Piaceri I, Sorbi S. Genetic heterogeneity…

More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the protein that accumulates to toxic levels in familial amyloid polyneuropathy (FAP) — eases nerve damage-related disability, and improves quality of life for patients. That’s according to the full results of the…

Ionis Pharmaceuticals has revenue from commercialized medicines, but those products reached the market in the hands of biopharma industry partners. The genetic medicines company does have wholly owned assets, and one of them now has preliminary Phase 3 data that put it on the path for an FDA submission. The…

Google’s DeepMind unit has used artificial intelligence (AI) to catalogue millions of ‘missense’ mutations in human DNA that could be related to the development of genetic diseases. Developed by DeepMind researchers as an extension of its AlphaFold database, the AlphaMissense AI tool has classified the effects of 71 million missense…

Gene therapy and gene editing are on the cutting edge of modern biotechnology. Gene therapies are used to correct genetic abnormalities by introducing genetic material at a cellular level and can often take the form of adding a functioning copy of a gene. Gene editing takes the science to the…

Aken BL, Ayling S, Barrell D, Clarke L, Curwen V, Fairley S et al (2016) The Ensembl gene annotation system. Database (Oxford) baw093 Bagaria J, Nho K, An SSA (2021) Importance of GWAS in finding un-targeted genetic association of sporadic Alzheimer’s disease. Mol Cell Toxicol 17(3):233–244 Article  CAS  Google Scholar …

Introduction Alopecia areata (AA) is one of Taiwan’s most common autoimmune hair diseases and incidence rate of AA is 0.22%.1–3 The main symptoms of AA are rapid, non-scarring hair loss that affects body hair, facial hair, eyelashes, and brows.1,2 In the United States, the prevalence of AA is estimated to…

Li, Y., Glass, Z., Huang, M., Chen, Z. Y. & Xu, Q. Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. Biomaterials 234, 119711 (2020). Article  CAS  PubMed  PubMed Central  Google Scholar  Dong, W. & Kantor, B. Lentiviral vectors for delivery of gene-editing systems based on CRISPR/Cas: current state and…

PRESS RELEASE Published September 15, 2023 DelveInsight’s, “AL Amyloidosis Pipeline Insight 2023,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the AL Amyloidosis pipeline landscape. It covers the AL Amyloidosis pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by…

The global gene-editing market is projected to skyrocket from US$5 billion in 2021 to US$21.4 billion in 2030, growing 17.3% annually. The increasing prevalence of genetic disorders, leading to a range of diseases, is fuelling demand for personalized medicine and is the driving force behind the sharp rise of the…

Grandbrothers The U.S. Food and Drug Administration (FDA) said Monday that patisiran, a RNAi therapeutic developed by Alnylam Pharmaceuticals (NASDAQ:ALNY) for amyloidosis, caused only a “small” clinical benefit in a Phase 3 trial. The FDA’s views came in briefing documents released ahead of an advisory committee meeting on Alnylam’s (ALNY)…

Perceived wisdom goes that the way to outperform the market this year has been by leaning into the Magnificent Seven stocks (AAPL, AMZN, GOOGL, META, MSFT, NVDA, TSLA). These market leaders – all tech giants – are seen as being responsible for the market’s rally, but hold that thought, says…

According to the report published by Allied Market Research, the global nucleic acid therapeutics market was estimated at $4.1 billion in 2021 and is expected to hit $12.2 billion by 2031, registering a CAGR of 11.6% from 2022 to 2031. The study analyzes the important strategies, drivers, competition, market dynamics, size, and…

PALO ALTO, Calif., Aug. 24, 2023 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that detailed Phase 3 results from ATTRibute-CM, its study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM, will be presented at…

BridgeBio Pharma, a leading biopharmaceutical company, is set to unveil groundbreaking findings from its Phase 3 study, ATTRibute-CM, at the highly anticipated European Society of Cardiology (ESC) Congress 2023 in Amsterdam, Netherlands. The event, taking place from August 25 to August 28, 2023, will serve as a platform for BridgeBio…

Zack MM, Kobau R (2017) National and state estimates of the numbers of adults and children with active epilepsy – United States, 2015. MMWR Morb Mortal Wkly Rep 66:821–825. doi.org/10.15585/mmwr.mm6631a1 Article  PubMed  PubMed Central  Google Scholar  Pottoo F et al (2020) Impact of adherence to antiepileptic medications on quality of…

Introduction Since the 20th century, tThe incidence of ulcerative colitis (UC) has been increasing over time, negatively affecting peoples’ health.1,2 UC is a multifactorial induced inflammatory bowel disease (IBD) characterized by chronic recurrent and remitting mucosal inflammation.3,4 It originates in the rectum and then extends to the proximal segments of…

Fortress Biotech Reports Second Quarter 2023 Financial Results and Recent Corporate Highlights Total net revenue was $17.4 million in the second quarter of 2023, a 40% increase from $12.4 million in the first quarter of 2023 Positive topline results from two Phase 3 clinical trials evaluating DFD-29 demonstrated achievement of…

Intellia Therapeutics Inc. (NASDAQ:NTLA) is a leading biotechnology company that has garnered attention for its groundbreaking work in the field of gene editing. Utilizing a revolutionary technology known as CRISPR-Cas9, Intellia is at the forefront of a scientific revolution that has the potential to transform the way we treat…

Ionis Pharmaceuticals, Inc. is engaged in discovering and developing ribonucleic acid (RNA)-targeted therapeutics. The Company is primarily focused on cardiovascular and neurology franchises. The Company’s products include SPINRAZA, TEGSEDI and WAYLIVRA. SPINRAZA is for the treatment of patients with spinal muscular atrophy (SMA), a progressive, debilitating and often fatal genetic…

CARDIO-TTRansform is the largest, most comprehensive ATTR-CM study with more than 1,400 patients enrolled Eplontersen is currently under U.S. FDA review for ATTRv-polyneuropathy, with ATTR-CM representing a second, larger potential patient population CARLSBAD, Calif., July 31, 2023 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the completion of enrollment in…

Part of the job as a pediatric hematologist for Michael P. Triebwasser, MD, PhD, is to take care of patients during their bone marrow transplantation. Just last week, he took care of a patient with a severe combined immunodeficiency (SCID) disorder and another with a bone marrow disorder. Even though…

PRESS RELEASE Published July 24, 2023 Global RNAi Therapeutics Market report provides CAGR values along with its fluctuations for the definite forecast period. The key research methodology used here by DBMR research team is data triangulation which involves data mining, analysis of the impact of data variables on the market,…

Lenalidomide Plus Rituximab as Initial Treatment for Elderly Frail Patients With DLBCL | PracticeUpdate …

In recent years, multiple clinical trials have demonstrated the tremendous potential of RNA interference (RNAi) drugs targeting disease-causing genes to treat diseases that have plagued humans for years. Harnessing the power of RNAi, our company is committed to providing customers with specially designed small interfering RNAs (siRNAs) to knock down the…

Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127:2391–405. Article  CAS  PubMed  Google Scholar  Grimwade D, Walker H, Harrison G, Oliver F, Chatters S, Harrison CJ, et…

Credit: iStock/SvetaP DNA double helix: iStock/Kagenmi BridgeBio’s candidate therapy for the rare disease transthyretin amyloid cardiomyopathy met its primary endpoint, according to results from a 30-month Phase III trial. The findings showed 81% of participants taking the drug candidate acoramidis were alive at 30 months versus 74% of the placebo…

Source/Disclosures Published by: Disclosures: Finkel is president of Amyloidosis Support Groups. Fox is an employee of BridgeBio Cardiorenal. Judge is co-chair of the steering committee for a trial sponsored by BridgeBio Pharma. ADD TOPIC TO EMAIL ALERTS Receive an email when…

Someone’s famous uncle once said, “With great power comes great responsibility.” Genome editing is an incredibly powerful technique with huge promise and potential for medicine, as well as many other fields including agriculture and the environment. But those wielding the power must cut through the hype, evaluate the potential, and…

First time published in a peer-reviewed journal: birtamimab is the only investigational drug that has shown a significant survival benefit in patients with Mayo Stage IV AL amyloidosis in a double-blind placebo-controlled clinical trial A significant improvement in time to all-cause mortality at month 9 was observed with birtamimab versus…

Three elderly men diagnosed with an until-now irreversible heart condition, transthyretin cardiac amyloidosis, experienced an unprecedented spontaneous recovery confirmed by medical scans, leading researchers at UCL and the Royal Free Hospital to identify a unique amyloid-targeting immune response in these patients. This breakthrough raises the potential for new treatments and…

Intellia Therapeutics, Inc. is a clinical-stage genome editing company, which is focused on developing curative therapeutics using Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) technology. CRISPR/Cas9 is a technology for genome editing, the process of altering selected sequences of genomic deoxyribonucleic acid. It is focused on leveraging its…

Olga Seifutdinova/iStock via Getty Images Ionis Pharmaceuticals (NASDAQ:IONS) is a good speculative biotech play to look into. That’s because it has a very solid program in progress, which is using donidalorsen for the prevention of hereditary angioedema [HAE] attacks. Thus far, a phase 2 open-label study has shown this drug…

Text size Other gene editors pursuing sickle cell include Beam Therapeutics. Dreamstime Gene-editing stocks are regaining favor on Wall Street this year as evidence builds for their treatments of deadly diseases such as sickle cell. Shares of Crispr Therapeutics (ticker: CRSP) are up 45% this year, compared with the Nasdaq…

In this article, we discuss 10 DNA stocks that billionaires love. If you want to skip our detailed analysis of the DNA market, head over to 5 DNA Stocks Billionaires Are Loading Up On.  Genomics is the study of a complete set of DNA within an organism. The global genomics…

Department of Dermatology, Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, People’s Republic of China Correspondence: Jianyou Wang, Department of Dermatology, Second Affiliated Hospital, Zhejiang University School of Medicine, 88 Jiefang Road, Hangzhou, 310009, People’s Republic of China, Email [email protected] Abstract: Lipoid proteinosis (LP) is a rare autosomal recessive…

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…