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Category Archives: Genetic Therapy

Brent Saunders pivots from Allergan sale to a $460M SPAC, making him an overnight player in one of the hottest money-raising gambits in biotech -…

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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Brent Saunders pivots from Allergan sale to a $460M SPAC, making him an overnight player in one of the hottest money-raising gambits in biotech -...

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Covid-19 roundup: NIH spotlights blood thinners in its next big trial; All it took was a pandemic to get JPM out of SF – Endpoints News

After immune modulators and antibodies, blood thinners are the next class of drugs to be put through a Phase III trial organized under the NIHs ACTIV initiative.

Three different anticoagulants have been named for the master protocol: heparin, aspirin and apixaban. Informed by studies suggesting that many patients who died from Covid-19 have formed unusual blood clots throughout their bodies including in their smallest blood vessels the NIH wants to see which therapies are the most effective at preventing or reducing them and thus improving outcomes for patients.

There is currently no standard of care for anticoagulation in hospitalized COVID-19 patients, and there is a desperate need for clinical evidence to guide practice, agency head Francis Collins said in a statement. Conducting trials using multiple existing networks of research sites provides the scale and speed that will get us answers faster.

For the inpatient trial, patients will be given either a low dose or a high dose of heparin.

But hospitalized patients are just one of two groups now being recruited to ACTIV-4, which also includes an outpatient trial broken down into four cohorts given placebo, aspirin, or a low or therapeutic dose of the blood thinner apixaban. The goal there is to reduce life-threatening cardiovascular or pulmonary complications in newly-diagnosed Covid-19 patients who dont need hospital admission.

Gary Gibbons, director of the National Heart, Lung, and Blood Institute, said while heparin has shown promise, physicians really need clinical trial data to determine how much blood thinner, or even anti-platelet medication, to give.

A third trial will start soon involving patients who had moderate or severe disease but have been discharged after hospitalization. It has a slightly different design, with the key metric being whether patients develop thrombotic complications (think heart attack, stroke, blood clots in major veins or arteries and death) within 45 days of being hospitalized.

As is custom in these master protocols, researchers may switch out candidates or add in new therapies as clinical data emerge.

NHLBI is coordinating and overseeing the program, while Operation Warp Speed is providing the funding. Amber Tong

The JP Morgan Healthcare Conference just got a whole lot cheaper.

On Thursday, the bank told clients that the famed conference was going virtual. That means no more packing into ballrooms and conference halls, no more dashing between different hotel rooms for an endless circle of meetings, and crucially no more $2,000 suites and $150 rent-a-tables. Instead, there will only be what there has been for the last 6 months: Endless circles of Zoom meetings, looped in infinite recursion from San Diego to Boston to Cambridge, UK.

The virtual conference means that, after years of chatter, the conference will finally, at least for a year, leave San Francisco. In theory, though, you could still go. A room at the Westin is selling for only $276, breakfast not included. Jason Mast

Longtime FDA officials and drug companies are trying to shore up public confidence in an eventual Covid-19 vaccine, but a new poll suggests that, at least until the election, they could be facing an uphill battle.

The poll, conducted by theKaiser FamilyFoundation,found that most adults, both Republican and Democrat, are wary of taking a vaccine before the election. Six in ten adults, meanwhile, were worried that the Trump Administration would pressure the FDA to rush an approval. The poll was conducted between August 28 and September 3 days after a heavily politicized emergency use authorization for convalescent plasma stoked fears that a vaccine would be authorized under similar conditions.

Fear of political pressure was most acute among Democrats, 51% of whom said they were very worried and 85% of whom were at least somewhat worried. A majority of Republicans said they were not too worried or not at all worried, although 35% expressed some concern.

As to whether they would take a vaccine, though, the splits are reversed. Half of Democrats were willing to take a vaccine, but only 36% of Republicans said they would. These numbers are in linewith long-running differences between Democrats and Republicans on vaccination, though it does suggest that recent events may have dented views. A Gallup pollconducted in late July and early August found 81% of Democrats and 47% of Republicans were willing to take an an FDA-approved vaccine to prevent coronavirus/COVID-19 if it was available right now at no cost. Those questions, though, did not directly reference the election.

Still, most respondents did not think such an eventuality was likely. Eighty-one percent of people, regardless of party, did not expect a vaccine to come before November 3.

The polls come as officials in the US struggle to shore up confidence in any eventually approved vaccine. Earlier this week, 9 major drug companies signed a pledge to only submit vaccine data when it meets safety and efficacy standards and 8 FDA career officialspublishedan open letter in USA Today on their commitment to science. Even before the latest concerns of political interference, public health experts have talked for months about how rising rates of vaccine hesitancy could pose a barrier in pandemic response. Bill Gates has called getting people to actually take a vaccine the final hurdle.

New data highlight how criticalmessaging can be on vaccine uptake. A large study published this morning inThe Lancettracked global vaccine hesitancy from 2015 to 2019 identified misinformation as one of the key factors in places with growing skepticism of vaccines, including South Korea, Malaysia, and Georgia. Anthropologist and director of the Vaccine Confidence Project Heidi Larson, the studys author, said messaging in the US and elsewhere that focused on speed wasnt helping.

Theres a lot of anxiety about the speed of vaccine development (for COVID-19), she told Reuters. But the public is not really keen on speed theyre more keen on thoroughness, effectiveness and safety. Jason Mast

For a look at allEndpoints Newscoronavirus stories, check out ourspecial news channel.

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Covid-19 roundup: NIH spotlights blood thinners in its next big trial; All it took was a pandemic to get JPM out of SF - Endpoints News

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Probiotic skin therapy improves eczema in children – National Institutes of Health

News Release

Wednesday, September 9, 2020

An experimental treatment for eczema that aims to modify the skin microbiome safely reduced disease severity and increased quality of life for children as young as 3 years of age, a National Institutes of Health study has found. These improvements persisted for up to eight months after treatment stopped, researchers report Sept. 9 in Science Translational Medicine.

Atopic dermatitis, commonly called eczema, is a chronic inflammatory skin disease characterized by dry, itchy skin and rashes. The disease is most common in children and is linked to an increased risk of developing asthma, hay fever and food allergy. While available treatments can help manage eczema symptoms, current options can be costly, and many require multiple daily applications.

The experimental therapy contains strains of live Roseomonas mucosaa bacterium naturally present on the skinoriginally isolated from healthy volunteers and grown under carefully controlled laboratory conditions. For four months, clinical trial participants or their caregivers periodically applied this probiotic therapy to areas of skin affected by eczema.

A child suffering from eczema, which can be itchy, painful and distracting for the child, also is very difficult for the entire family, said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID), which led the study. These early-stage findings suggest that R. mucosa therapy may help relieve some children of both the burden of eczema symptoms and the need for daily treatment.

Numerous genetic and environmental factors contribute to eczema, and scientists are learning more about the role that the skins microbiome plays in this condition. In 2016, NIAID researchers reported that R. mucosa strains isolated from healthy human skin improved outcomes in cell culture and mouse models of eczema.

To build on these preclinical findings, NIAID launched a Phase 1/2 clinical trial at the NIH Clinical Center in Bethesda, Maryland, to assess the safety and potential benefit of R. mucosatherapy in people with eczema. Interim results reported in 2018 for 10 adults and five children aged 9 to 14 years indicated that the treatment was safe and associated with reduced eczema severity. Since then, the trial has enrolled an additional 15 children, for a total of 20 children with mild to severe eczema ranging in age from 3 to 16 years.

Twice weekly for three months and every other day for an additional month, children or their caregivers sprayed a solution of sugar water containing liveR. mucosaonto areas of skin with eczema. For the first 15 children enrolled in the study, the dose of live R. mucosa was gradually increased each month. The last five children to enroll received the same dose throughout the four-month treatment period. Regardless of dosing strategy, no serious adverse events were attributed to the therapy.

Most children in the study experienced substantial improvements in their skin and overall wellbeing following R. mucosa therapy. Encouragingly, the therapeutic bacteria stayed on the skin and continued to provide benefit after therapy stopped, said NIAIDs Ian Myles, M.D., principal investigator of the trial. These results support a larger study to further assess the safety and effectiveness of this experimental treatment by comparing it with a placebo.

Seventeen of the 20 children experienced a greater than 50% improvement in eczema severity following treatment. Improvement occurred on all treated skin sites, including the inner elbows, inner knees, hands, trunk and neck. The scientists also observed increases in the skins barrier functionits ability to seal in moisture and keep out allergens. Additionally, most children needed fewer corticosteroids to manage their eczema, experienced less itching, and reported a better quality of life following the therapy. These benefits persisted after treatment ended, and the therapeutic R. mucosa strains remained on the skin for up to eight months.

The NIAID researchers next set out to better understand how R. mucosa therapy improves eczema symptoms. They found that treated skin had increased microbial diversity and reduced levels of Staphylococcus aureusa bacterium known to exacerbate eczema.

In addition to imbalances in the microbiome, the skin of people with eczema is deficient in certain lipids, or oils. By conducting experiments in cell and animal models of eczema, the NIAID scientists found that a specific set of lipids produced by R. mucosa strains isolated from healthy skin can induce skin repair processes and promote turnover of skin tissue. Study participants had increased levels of these lipids on their skin after treatment with R. mucosa.

The researchers emphasize that additional studies are needed to further elucidate the mechanism of R. mucosa therapy and to explore whether genetic or other factors may explain why some participants did not benefit from the experimental treatment.

For more information about the completed Phase 1/2 study Beginning Assessment of Cutaneous Treatment Efficacy forRoseomonasin Atopic Dermatitis (BACTERiAD), see ClinicalTrials.gov using identifier NCT03018275.

NIH has exclusively licensed the R. mucosa therapy to Forte Biosciences to advance this potential treatment through further clinical development,and the company plans to begin enrollment in a Phase 2 placebo-controlled trial later this month. For more information about this study, Evaluation of FB-401 in Children, Adolescents and Adults (2 Years and Older) With Mild to Moderate Atopic Dermatitis, see ClinicalTrials.gov using identifierNCT04504279.

NIAID conducts and supports researchat NIH, throughout the United States, and worldwideto study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

IA Myles et al. Therapeutic responses to Roseomonas mucosa in atopic dermatitis may involve lipid-mediated TNF-related epithelial repair. Science Translational Medicine DOI: 10.1126/scitranslmed.aaz8631 (2020).

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MiNA Therapeutics bags nearly $30M Series A to push activating mRNA tech through the clinic – Endpoints News

Seven years after leaving investment banking to take the helm at MiNA Therapeutics, CEO Robert Habib is on a mission to develop a new class of medicines using small activating mRNA technology. And on Thursday, the London-based biotech announced it landed a modest 23 million (nearly $30 million) Series A to work with.

Habibs father, the prominent Imperial College London professor Nagy Habib, co-founded MiNA in 2008. The companys lead mRNA program is designed to switch genes on, and potentially help treat what the CEO called the undruggable: difficult-to-treat diseases, starting with liver cancer.

Its sort of encouraging the cells own machinery to pay attention and to start producing a bit more of the gene that we are interested in. And that gene obviously will produce more messenger RNA. And that messenger RNA will result in more protein. And if were targeting the right protein, were going to have a therapeutic benefit, Habib said.

In 2017, Sosei put down about $45 million for 25.6% of MiNAs equity. Peter Bains, Sosei CEO at the time, laid out a $534 million plan to acquire MiNA, which was putting its lead candidate in a Phase I/IIa trial for liver cancer. Bains said the deal was another step in expanding Soseis global reach. About a year and a half later, Sosei passed on that option, but kept its stake.

MiNAs lead program goes after CEBPA in the hopes of reducing immune suppression caused by immature myeloid cells to improve the efficacy of cancer therapies. Its candidate, MTL-CEBPA, is being tested as a combination therapy with Bayers sorafenib in advanced liver cancer patients and Mercks pembrolizumab in patients with advanced solid tumors.

The drug doesnt directly affect the cancer just the environment in which it is growing.

That strategy of reducing immune suppression and clearing those immature myeloid cells is believed to be an important strategy in improving the effects of a range of cancer therapies, Habib said.

The Series A funding will be used to conduct a Phase II study of MTL-CEBPA with sorafenib for the liver cancer indication, and complete an ongoing Phase I/Ib study of the pembrolizumab combination. In the Phase II study, MiNA will focus on durability. Habib expects the study to begin early next year, with up to 70 patients in the UK, US, and some Asian and European countries.

When administered on its own, sorafenib typically has an objective response rate of between 0 to 10%, according to Habib. But when given with MTL-CEBPA, were seeing objective responses above that absolute proportion, and were seeing complete responses of liver patients which is an absolutely extraordinary feature, he added.

Liver cancer is one of the cancers which has really not improved by a huge amount over several decades, Habib said. The concept of complete remission is a concept which is very foreign to patients and to physicians in liver cancer.

The company is actively looking to expand its platform to other indications as well.

aMoon, an Israel-based healthtech and life sciences venture fund, led the Series A round and existing investors pitched in. As part of the deal, aMoon managing director Gur Roshwalb is joining MiNAs board of directors.

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Pascal Soriot says 2020 approval still possible for AstraZeneca’s Covid-19 vaccine as the world awaits the safety board’s decision – Endpoints News

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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Pascal Soriot says 2020 approval still possible for AstraZeneca's Covid-19 vaccine as the world awaits the safety board's decision - Endpoints News

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Gene Therapy Market to Eyewitness Massive Growth by 2026: Leading Key Players Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc….

This report focuses on the Global Gene Therapy Market trends, future forecasts, growth opportunities, key end-user industries, and market players. The objectives of the study are to present the key developments of the market across the globe.

The latest research report on Gene Therapy market encompasses a detailed compilation of this industry, and a creditable overview of its segmentation. In short, the study incorporates a generic overview of the Gene Therapy market based on its current status and market size, in terms of volume and returns. The study also comprises a summary of important data considering the geographical terrain of the industry as well as the industry players that seem to have achieved a powerful status across the Gene Therapy market.

Get Sample of this Premium Report @ https://industrystatsreport.com/Request/Sample?ResearchPostId=579&RequestType=Sample

Gene Therapy Market Segmentation

Reports include the following segmentation: By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions OthersBy Type of Vectors Viral vectors Non-viral vectorsBy Type of Cells Somatic cells Germline cellsBy Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

The report has been curated after observing and studying various factors that determine regional growth such as economic, environmental, social, technological, and political status of the particular region. Analysts have studied the data of revenue, production, and manufacturers of each region. This section analyses region-wise revenue and volume for the forecast period of 2015 to 2026. These analyses will help the reader to understand the potential worth of investment in a particular region.

Global Gene Therapy Market: Competitive LandscapeThis section of the report identifies various key manufacturers of the market. It helps the reader understand the strategies and collaborations that players are focusing on combat competition in the market. The comprehensive report provides a significant microscopic look at the market. The reader can identify the footprints of the manufacturers by knowing about the global revenue of manufacturers, the global price of manufacturers, and production by manufacturers during the forecast period of 2015 to 2020.

The major players in the market Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation Others

Global Gene Therapy MarketThis research report providesCOVID-19 Outbreakstudy accumulated to offer Latest insights about acute features of the Gene Therapy Market. The report contains different market predictions related to marketsize, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary andSWOT analysis.It presents the360-degreeoverview of the competitive landscape of the industries. Gene Therapy Market is showing steadygrowthandCAGRis expected to improve during the forecast period.

The main sources are industry experts from the global Gene Therapy industry, including management organizations, processing organizations, and analytical services providers that address the value chain of industry organizations. We interviewed all major sources to collect and certify qualitative and quantitative information and to determine future prospects. The qualities of this study in the industry experts industry, such as CEO, vice president, marketing director, technology and innovation director, founder and key executives of key core companies and institutions in major biomass waste containers around the world in the extensive primary research conducted for this study We interviewed to acquire and verify both sides and quantitative aspects.

Global Gene Therapy Market: Regional AnalysisThe report offers in-depth assessment of the growth and other aspects of the Gene Therapy market in important regions, including the U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, Taiwan, Southeast Asia, Mexico, and Brazil, etc. Key regions covered in the report are North America, Europe, Asia-Pacific and Latin America.

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Complete Analysis of the Gene Therapy Market:

Comprehensive assessable analysis of the industry is provided for the period of 2020-2025 to help investors to capitalize on the essential market opportunities.

The key findings and recommendations highlight vital progressive industry trends in the global Gene Therapy market, thereby allowing players to improve effective long term policies

A complete analysis of the factors that drive market evolution is provided in the report.

To analyze opportunities in the market for stakeholders by categorizing the high-growth segments of the market

The numerous opportunities in the Gene Therapy market are also given.

Report Answers Following Questions:

What are the factors driving the growth of the market?

What factors are inhibiting market growth?

What are the future opportunities in the market?

Which are the most dynamic companies and what are their recent developments within the Gene Therapy Market?

What key developments can be expected in the coming years?

What are the key trends observed in the market?

TABLE OF CONTENT

1 Report Overview

2 Global Growth Trends

3 Market Share by Key Players

4 Breakdown Data by Type and Application

5 United States

6 Europe

7 China

8 Japan

9 Southeast Asia

10 India

11 Central & South America

12 International Players Profiles

13 Market Forecast 2020-2025

14 Analysts Viewpoints/Conclusions

15 Appendix

Read Full Report: https://industrystatsreport.com/Lifesciences-and-Healthcare/Gene-Therapy-Market-Share/Summary

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Gene Therapy Market to Eyewitness Massive Growth by 2026: Leading Key Players Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc....

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