Monthly Archives: June 2022

CRISPR Therapeutics to Host Innovation Day on June 21, 2022 – GuruFocus.com

Posted: June 22, 2022 at 2:46 am

ZUG, Switzerland and CAMBRIDGE, Mass., May 31, 2022 (GLOBE NEWSWIRE) -- CRISPR Therapeutics ( CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that it will host an Innovation Day focused on early research and development on Tuesday, June 21, 2022 at 2:00 PM ET.

CRISPR Therapeutics Innovation Day will focus on the Company's differentiated genetic engineering platform technologies, novel approach to cell and gene therapy, and emerging discovery programs.

A live webcast of the event will be available on the "Events & Presentations" page in the Investors section of the Company's website at https://crisprtx.gcs-web.com/events. A replay of the webcast will be archived on the Company's website for 30 days following the presentation. Please contact [emailprotected] for any questions regarding the event.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR THERAPEUTICS word mark and design logo are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

Investor Contact:Susan Kim+1-617-307-7503[emailprotected]

Media Contact:Rachel Eides+1-617-315-4493[emailprotected]

See the rest here:
CRISPR Therapeutics to Host Innovation Day on June 21, 2022 - GuruFocus.com

Posted in Genetic Engineering | Comments Off on CRISPR Therapeutics to Host Innovation Day on June 21, 2022 – GuruFocus.com

Global Therapeutic Proteins Market Research Report 2022: Focus on Insulin; Fusion Protein; Erythropoietin; Interferon; Human Growth Hormone; Follicle…

Posted: June 22, 2022 at 2:46 am

DUBLIN--(BUSINESS WIRE)--The "Therapeutic Proteins Global Market Report 2022: By Product, By Application, By Function" report has been added to ResearchAndMarkets.com's offering.

The global therapeutic proteins market is expected to grow from $100.06 billion in 2021 to $112.17 billion in 2022 at a compound annual growth rate (CAGR) of 12.1%. The market is expected to reach $177.30 billion in 2026 at a CAGR of 12.1%.

The therapeutic proteins market consists of sales of therapeutic proteins. Therapeutic proteins provide important therapies for diseases such as diabetes, cancer, infectious diseases, hemophilia, and anemia.

The main types of products in therapeutic proteins are insulin, fusion protein, erythropoietin, interferon, human growth hormone and follicle stimulating hormone. Insulin is a peptide hormone produced by beta cells in the pancreatic cells that serves as the individual 's primary anabolic hormone. It affects fat, carbohydrate and protein metabolism by boosting glucose uptake from the blood into the fat, liver and skeletal muscle cells.

The different functions include enzymatic and regulatory activity, special targeting activity, vaccines, protein diagnostics and is used in various applications such as metabolic disorders, immunologic disorders, hematological disorders, cancer, hormonal disorders, genetic disorders, others.

Advance technologies for protein-based drug development drives the therapeutic proteins market. Therapeutic proteins cannot be synthesized chemically, they need to be produced by genetic engineering and recombinant DNA technology in living cells or organisms.

Protein-engineering platform technologies such as glycoengineering, pegylation, Fc-fusion, albumin fusion, albumin drug conjugation help to increase the production yield, product purity, circulating half-life, targeting, and functionality of therapeutic protein drugs. Belimumab, ipilimumab, taliglucerase alfa, albiglutide, coagulation factor IX recombinant human are some therapeutic protein drugs developed using protein engineering technologies approved by FDA in the past five years.

Increasing biosimilar drugs in global market decline the growth of the therapeutic proteins market. Patent expiry of therapeutic proteins such as monoclonal antibodies give space for entry of biosimilar. In EU, AbbVie evidenced patent expiration of Humira (adalimumab) in 2018, five biosimilar of Humira from Mylan, Amgen, Sandoz, Samsung Bioepis received drug approvals from European commission to enter the EU market. These cost-effective treatments similar to original biologics decline the revenue and sales of therapeutic proteins.

Monoclonal antibody drug approvals are increasing in the protein therapeutic segment. Chronic diseases such as cancer, immunological disorders are well treated with monoclonal antibodies. Monoclonal antibodies are dominant and well-established product class in the protein therapeutic segment with more safety and immunogenicity than antibodies.

Cell-based expression systems such as Chinese hamster ovary (CHO) mammalian cell expression system with latest technologies increased the productivity of monoclonal antibodies by overcoming the problems associated with earlier antibody drugs. In last five years, FDA approved 213 drugs, among them 44 are monoclonal antibodies. For instance, twelve monoclonal antibodies were approved by FDA for the treatment of cancer and immunological disorders.

In the United States, therapeutic protein drug manufacturers file therapeutic biologics application (BLA) to FDA for the product approvals. The drug approved through BLA should be proved as safe, pure and potent. FDA consolidated review of most therapeutic proteins in Center for Drug Evaluation and Research (CDER). In European Union, biologics are regulated by Committee for Medicinal Products for Human Use (CHMP) for marketing authorization.

In July 2021, Eli Lilly and Company, a US-based pharmaceutical company acquired Protomer Technologies Inc. for $1 billion. Lilly is delighted to add protomer's breakthrough technology to its diabetes pipeline through this acquisition, since the company's glucose-sensing insulin programme, which is based on its proprietary molecular engineering of protein sensors (MEPS) platform, is exhibiting great potential.

Major players in the therapeutic proteins market are

Key Topics Covered:

1. Executive Summary

2. Therapeutic Proteins Market Characteristics

3. Therapeutic Proteins Market Trends And Strategies

4. Impact Of COVID-19 On Therapeutic Proteins

5. Therapeutic Proteins Market Size And Growth

5.1. Global Therapeutic Proteins Historic Market, 2016-2021, $ Billion

5.1.1. Drivers Of The Market

5.1.2. Restraints On The Market

5.2. Global Therapeutic Proteins Forecast Market, 2021-2026F, 2031F, $ Billion

5.2.1. Drivers Of The Market

5.2.2. Restraints On the Market

6. Therapeutic Proteins Market Segmentation

6.1. Global Therapeutic Proteins Market, Segmentation By Product Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

6.2. Global Therapeutic Proteins Market, Segmentation By Application, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

6.3. Global Therapeutic Proteins Market, Segmentation By Function, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

7. Therapeutic Proteins Market Regional And Country Analysis

7.1. Global Therapeutic Proteins Market, Split By Region, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

7.2. Global Therapeutic Proteins Market, Split By Country, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

For more information about this report visit https://www.researchandmarkets.com/r/ktb394

View original post here:
Global Therapeutic Proteins Market Research Report 2022: Focus on Insulin; Fusion Protein; Erythropoietin; Interferon; Human Growth Hormone; Follicle...

Posted in Genetic Engineering | Comments Off on Global Therapeutic Proteins Market Research Report 2022: Focus on Insulin; Fusion Protein; Erythropoietin; Interferon; Human Growth Hormone; Follicle…

An Experimental Treatment Failed in Mice, and Researchers Did the Right Thing: They Published About It – UConn Today – UConn

Posted: June 22, 2022 at 2:45 am

Blocking the mutant protein with an antibody didnt stop the strange, abnormal bone growths in mice. But the knowledge gained could steer scientists toward more promising approaches, report researchers from UConn and Alexion Pharmaceuticals in the 15 June issue of The Journal of Clinical Investigation.

Fewer than 4,000 people worldwide are afflicted with fibrodysplasia ossificans progressiva (FOP), an inherited disease in which small injuries or bruises to skeletal muscle provoke the growth of massive, abnormal bone and cartilage. Gradually much of the bodys soft tissue turns to bone. Now, researchers at UConn and Alexion Pharmaceuticals who were investigating a potential cure instead found a concerning surpriseblocking the protein responsible for the disease with a monoclonal antibody made the abnormal bone growth worse in mice .

Normally, stem cells help repair muscle damaged by injury or disease. But in people with FOP, certain stem cells get the wrong message from a mutant receptor on their surface. Instead of promoting muscle regeneration, the stem cells develop into bone.

UConn Professor of Molecular and Cell Biology David Goldhamer, Alexion Pharmaceuticals researcher Jeffrey Hunter, and colleagues worked for years to discover a potential antibody therapy for FOP using accurate genetic mouse models of the disease developed by the two groups. The idea was that the antibody would block the mutant receptor and prevent the responsible stem cells from making new bone. But the results were exactly the opposite.

The unexpected result: injecting the antibody into FOP mice caused a dramatic increase in inappropriate bone formation, instead of protecting them as wed hoped, Goldhamer says.

Goldhamer, Hunter and their teams worked with an antibody discovered by Alexion that interferes with the specific cell-surface receptor involved in FOP called Activin A receptor type 1, or ACVR1. The researchers thought that if they blocked ACVR1, the abnormal bone growth would stop. But instead, it was exacerbated.

The antibody appears to lower the injury threshold needed to stimulate bone growth within muscle tissue. Mild injuries that normally dont make bone in FOP mice suddenly make lots of bone. Additionally, the antibody increases and prolongs the immune response to the injury, Goldhamer says.

Another team working on antibody-based therapies for FOP ran into the same effect. Regeneron Pharmaceuticals used different antibodies they derived independently, as well as a different strain of FOP mice, but got the same adverse result. Their paper also appears in the JCI this week.

The teams dont yet know precisely why the antibodies dramatically worsen the disease in mice, but their work raises serious safety and efficacy concerns for the clinical application of this approach, which has not yet been tested in humans. Neither team has plans to pursue this clinically.

This work was funded by a grant from the National Institutes of Health (R01AR072052) and a sponsored research agreement between Alexion Pharmaceuticals and the University of Connecticut.

Go here to read the rest:
An Experimental Treatment Failed in Mice, and Researchers Did the Right Thing: They Published About It - UConn Today - UConn

Posted in Connecticut Stem Cells | Comments Off on An Experimental Treatment Failed in Mice, and Researchers Did the Right Thing: They Published About It – UConn Today – UConn

Gamida Cell Submits Biologics License Application for Omidubicel to the FDA – Pharmacy Times

Posted: June 22, 2022 at 2:43 am

The drug is being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant.

Gamida Cell has completed the rolling biologics license application (BLA) submission to the FDA for omidubicel for the treatment of individuals with blood cancer who need an allogenic hematopoietic stem cell transplant, the company said in a statement.1

Omidubicel is an advanced cell therapy candidate that was developed as an allogeneic hematopoietic stem cell transplant for individuals with blood cancer. It is the first stem cell transplant donor source to receive breakthrough therapy designation from the FDA.1

Additionally, it has received orphan drug designation in the European Union and the United States.1

The BLA submission marks an important milestone for both Gamida and the transplant community, as omidubicel has the potential to be the first approved advanced cell therapy product for allogeneic stem cell transplantation, Julian Adams, PhD, CEO of Gamida Cell, said in the statement.1

Completion of this BLA submission is a key inflection point in our mission to deliver a new treatment option for patients with blood cancers. We look forward to working closely with the FDA to bring this potentially important therapy to patients, Adams said.1

The FDA has 60 days to determine whether the BLA for omidubicel is acceptable for filing.1

The BLA is supported by the results of a phase 3 study published in Blood, the journal of the American Society of Hematology. The study met its primary endpoint of the median time to neutrophil engraftment in individuals with hematologic malignancies undergoing allogeneic bone marrow transplant, with patients receiving omidubicel having a median time of 12 days compared with 22 days for the umbilical cord blood graft group.1

In the key secondary endpoints of the study, approximately 55% of individuals receiving omidubicel achieved platelet engraftment by day 42 compared with approximately 35% for the comparator.1

Additionally, the rate of infection was significantly reduced, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for individuals randomized to omidubicel being 37% compared with 57% for the comparator.1

Hospitalization in the first 100 days after transplant was significantly reduced with a median number of days alive and out of hospital for individuals randomized to omidubicel was 61 days compared with 48 days for the comparator.1

Furthermore, 1-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by a significant reduction in infectious complications and reduced non-relapse mortality. There was no significant increase in increases in graft-versus-host-disease rates or the relapse rate.1

The results showed that transplantation after an individual received omidubicel had fewer bacterial and viral infections and less time in the hospital, investigators said.2

Omidubicel was generally well tolerated in the phase 3 study.1

Investigators reported that the incidence of treatment-emergent serious adverse events were similar in both arms, with approximately 40% in the omidubicel group and 41% for the comparator.2

References

1. Omidubicel has orphan drug designation and breakthrough therapy designation. Gamida Cell. News release. June 2, 2022. Accessed June 3, 2022. https://investors.gamida-cell.com/news-events/press-releases/news-release-details/gamida-cell-completes-rolling-biologics-license

2. Horwitz ME, Stiff PJ, Cutler C, Brunstein C, et al; Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021;138(16):1429-1440. doi: 10.1182/blood.2021011719

See original here:
Gamida Cell Submits Biologics License Application for Omidubicel to the FDA - Pharmacy Times

Posted in Stem Cell Therapy | Comments Off on Gamida Cell Submits Biologics License Application for Omidubicel to the FDA – Pharmacy Times

Global Rheumatoid Arthritis Stem Cell Therapy Market 2022 Swot Analysis by Top Key Vendors, Demand And Forecast Research to 2028 Designer Women -…

Posted: June 22, 2022 at 2:43 am

MarketQuest.biz has announced the addition of new research titled Global Rheumatoid Arthritis Stem Cell Therapy Market from 2022 to 2028, which encompasses regional and global market data and is predicted to generate attractive valuation.The Rheumatoid Arthritis Stem Cell Therapy research covers market drivers, opportunities, limiting factors, and barriers. It provides a quantitative market study based on annual reports, product literature, industry announcements, and other sources.

The report explains the market definition, classifications, applications, engagements, and global Rheumatoid Arthritis Stem Cell Therapy industry trends are.It gives a realistic picture of the current market position incorporating original and predicted market estimates.The report gives a thorough analysis of their product portfolios to investigate the products and applications they focus on while working in the worldwide Rheumatoid Arthritis Stem Cell Therapy market. The report offers valuable suggestions to new just as set up players of the market.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketquest.biz/sample-request/121261

In order to improve industrial planning, data points such as flow patterns, openings, drivers, limits, and statistics are acquired from trusted sources. The data and numbers in the research report have been provided comprehensively, using graphical and pictorial representations to understand the market better.Further when datais synthesised, statistical analysis takes place. Several processes, including screening, integration, and data extrapolation, must be performed prior to data validation.

The product types covered in the report include:

The application types covered in the report include:

The countries covered in the market report are:

The key and emerging market players in the global market include:

ACCESS FULL REPORT: https://www.marketquest.biz/report/121261/global-rheumatoid-arthritis-stem-cell-therapy-market-2022-by-company-regions-type-and-application-forecast-to-2028

Significance of The Report gives the idea about thebroad and precise understanding, industry drivers and challenges affecting the industry growth, planning the business strategies and factors leading to the market development, and evaluating the market competition and planning

Customization of the Report:

This report can be customized to meet the clients requirements. Please connect with our sales team (sales@marketquest.biz), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on 1-201-465-4211 to share your research requirements.

Contact UsMark StoneHead of Business DevelopmentPhone: 1-201-465-4211Email: sales@marketquest.biz

Link:
Global Rheumatoid Arthritis Stem Cell Therapy Market 2022 Swot Analysis by Top Key Vendors, Demand And Forecast Research to 2028 Designer Women -...

Posted in Stem Cell Therapy | Comments Off on Global Rheumatoid Arthritis Stem Cell Therapy Market 2022 Swot Analysis by Top Key Vendors, Demand And Forecast Research to 2028 Designer Women -…

AIM ImmunoTech Secures New State-of-the-Art Facility for Product Development and Testing

Posted: June 22, 2022 at 2:43 am

Company enters into a lease agreement with the state-supported New Jersey Bioscience Center at North Brunswick, one of the nation’s leading business incubators dedicated to life sciences and biotechnology Company enters into a lease agreement with the state-supported New Jersey Bioscience Center at North Brunswick, one of the nation’s leading business incubators dedicated to life sciences and biotechnology

Follow this link:
AIM ImmunoTech Secures New State-of-the-Art Facility for Product Development and Testing

Posted in Global News Feed | Comments Off on AIM ImmunoTech Secures New State-of-the-Art Facility for Product Development and Testing

HOOKIPA Announces Executive Leadership Changes

Posted: June 22, 2022 at 2:43 am

NEW YORK and VIENNA, Austria, June 21, 2022 (GLOBE NEWSWIRE) -- HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, today announced several leadership changes as the company expands its clinical development and pipeline programs. All new appointments are effective July 1, 2022.

View post:
HOOKIPA Announces Executive Leadership Changes

Posted in Global News Feed | Comments Off on HOOKIPA Announces Executive Leadership Changes

Yale’s Department of Psychiatry Chair to Join Clearmind Medicine

Posted: June 22, 2022 at 2:43 am

Dr. John H. Krystal, world-leading expert in alcoholism and depression, will serve on scientific advisory board

See original here:
Yale’s Department of Psychiatry Chair to Join Clearmind Medicine

Posted in Global News Feed | Comments Off on Yale’s Department of Psychiatry Chair to Join Clearmind Medicine

LaunchPad Café, LTD, a subsidiary of Ventana Biotech, Inc. (VNTA), Announces its First financing client

Posted: June 22, 2022 at 2:43 am

DENVER, June 21, 2022 (GLOBE NEWSWIRE) -- LaunchPad Café, Ltd, the finance subsidiary of Ventana Biotech, Inc. (OTC: VNTA), is pleased to announce that it has contracted for its first financing client as a public company.

Go here to read the rest:
LaunchPad Café, LTD, a subsidiary of Ventana Biotech, Inc. (VNTA), Announces its First financing client

Posted in Global News Feed | Comments Off on LaunchPad Café, LTD, a subsidiary of Ventana Biotech, Inc. (VNTA), Announces its First financing client

Parnell Pharmaceuticals Gives Notice of Annual General Meeting and Explanatory Memorandum

Posted: June 22, 2022 at 2:43 am

SYDNEY, June 21, 2022 (GLOBE NEWSWIRE) -- Parnell Pharmaceuticals Holdings Ltd (OTC: PARNF), a fully integrated, commercial-stage pharmaceutical company focused on developing, manufacturing and marketing innovative animal health solutions, provides notice of its Annual General Meeting to be held Friday, 15th July 2022 at 8:30 a.m. (Australian Eastern Standard Time).

Read more:
Parnell Pharmaceuticals Gives Notice of Annual General Meeting and Explanatory Memorandum

Posted in Global News Feed | Comments Off on Parnell Pharmaceuticals Gives Notice of Annual General Meeting and Explanatory Memorandum