Archive for the ‘Gene Therapy Research’ Category

More than a dozen House Democratshavecalled for the Trump administration to lift restrictions on federally-funded research using fetal tissue from abortions topermit studies for possible treatment for COVID-19.

The lawmakers argued in a letter to Health an Human Services Secretary Alex Azar on Monday that the restrictions on government research with fetal tissue procured through elective abortion, "are preventing federally-funded scientists from advancing important studies that could potentially prevent, treat, or cure the 2019 novel coronavirus disease (COVID-19)."

"You have repeatedly stated that your agency is doing everything you can to develop vaccines and treatments for the novel coronavirus," the House Dems stated in the letter. They then criticized the HHS's 2019 issuance of new restrictions prohibiting federal scientists at the National Institutes of Health (NIH) from obtaining new tissue samples from elective abortions for ongoing NIH research.

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Under those restrictions existing projects funded by NIH grants at outside entities such as universities could continue until the funding expired, but they would then have to go through an ethics advisory boardreview for further approval. Privately-funded research was not affected.

The Democratic lawmakers inferred that the HHS restrictions could put American lives at risk during the coronavirus pandemic and that they were holding U.S. scientists back in researching possible treatment for the virus.

"Because of your restrictions, NIH is unable to utilize human fetal tissue to develop animal models of COVID-19 that can test potential vaccines and treatments to decelerate or even end this global health crisis," they said. "This inaction may ultimately put Americans further at risk of disease or death from COVID-19. Instead of exploring every possible option for a COVID-19 therapeutic, U.S. scientists are now urging their international counterparts to rush to conduct this research while their hands remain tied."

Public health officials have said that a coronavirus vaccine will not be available for another 12 to 18 months, according to The Hill.

A scientist at theNIH had been "thwarted" intrying to restart the government-funded research, Fox News reports, citinga report from the Washington Post that also said the fetal tissue comes from women who have elective abortion, "and critics say that it is unethical to use the material and that taxpayer money should not be used for research that relies on abortion."

Former Deputy Assistant Attorney General John Yoo said on the Fox News at Night program that he did not see a great deal of scientific evidence or research reports from other countries that indicates fetal tissue is key to finding a cure for the coronavirus.

"This is also just about taxpayer-funded research," Yoo said. "If this is so important, then this doctor or other researchers can go to another country or they could go to private sources of funding to get this research done."

"We've got to balance that against the nation's desire not to have this kind of fetal tissue used for medical research," said Yoo. "People are worried about a slippery slope; If we start using fetal tissue here, is the next step, are we going to start having abortions deliberately to get research material, and then are we going to start cloning fetuses to get fetal tissue material?"

Tweet This: "We've got to balance that against the nation's desire not to have this kind of fetal tissue used for medical research"

"You can see where people, and the administration said we don't even want to trigger a train of events where we might go down that slope," he said, "so better just to have a clear rule where we won't do it at all."

The Fox news report further cited scholars from the Charlotte Lozier Institute, who stated that,"None of the vaccines, treatments, or FDA-approved cellular and gene therapy products on the market use human fetal tissue from elective abortions that rely on ongoing abortions."

The move by HHS last year to restrict fetal tissue research through government funds was viewed as a pro-life victory and considered a first step in a complete ban on federally-funded research using fetal tissue.

"Promoting the dignity of human life from conception to natural death is one of the very top priorities of President Trumps administration," the HHS statement had said at the time.

In their letter this week lobbying for lifting restrictions on government-funded research with fetal tissue derived from abortions, the House Democrats wrote, "We urge you to prioritize science during an unprecedented global health emergency and remove all barriers to lifesaving research."

The group behind the letter wasled by Reps. Jared Huffman (Calif.), Jan Schakowsky (Ill.) and Diana DeGette (Colo.).

Also signing on were Reps. Ted Deutch (Fla.); Lois Frankel (Fla.); Jess G. Chuy Garcia (Ill.); Eleanor Holmes Norton (D.C.); Pramilla Jayapal (Wash.); Ro Khanna (Calif.); Seth Moulton (Mass.); Mark Pocan (Wis.); Jamie Raskin (Md.); andJackie Speier (Calif.).

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House Democrats demand Trump administration lift restrictions on government-funded fetal tissue research for coronavirus - Pregnancy Help News

Global Cancer Gene Therapy Market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

The report titled Global Cancer Gene Therapy Market is one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Cancer Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Cancer Gene Therapy market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Cancer Gene Therapy market is carefully analyzed and researched about by the market analysts.

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The Essential Content Covered in the Global Cancer Gene Therapy Market Report:

In terms of region, this research report covers almost all the major regions across the globe such as North America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. While Cancer Gene Therapy Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets. Cancer Gene Therapy Market in South, America region is also expected to grow in near future.

The key players covered in this studyAdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene

Market segment by Type, the product can be split intoOncolytic VirotherapyGene TransferGene-Induced Immunotherapy

Market segment by Application, split intoHospitalsDiagnostics CentersResearch Institutes

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

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Key questions answered in the report

*What will be the market size in terms of value and volume in the next five years?

*Which segment is currently leading the market?

*In which region will the market find its highest growth?

*Which players will take the lead in the market?

*What are the key drivers and restraints of the markets growth?

We provide detailed product mapping and analysis of various market scenarios. Our analysts are experts in providing in-depth analysis and breakdown of the business of key market leaders. We keep a close eye on recent developments and follow latest company news related to different players operating in the global Cancer Gene Therapy market. This helps us to deeply analyze companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study that will help you to stay on top of the competition.

Table of Contents

1 Cancer Gene Therapy Market Overview

1.1 Product Overview and Scope of Cancer Gene Therapy

1.2 Cancer Gene Therapy Segment by Type

1.2.1 Global Cancer Gene Therapy Production Growth Rate Comparison by Type 2020 VS 2026

1.3 Cancer Gene Therapy Segment by Application

1.3.1 Cancer Gene Therapy Consumption Comparison by Application: 2020 VS 2026

1.4 Global Cancer Gene Therapy Market by Region

1.4.1 Global Cancer Gene Therapy Market Size Estimates and Forecasts by Region: 2020 VS 2026

1.4.2 North America Estimates and Forecasts (2015-2026)

1.4.3 Europe Estimates and Forecasts (2015-2026)

1.4.4 China Estimates and Forecasts (2015-2026)

1.4.5 Japan Estimates and Forecasts (2015-2026)

1.5 Global Cancer Gene Therapy Growth Prospects

1.5.1 Global Cancer Gene Therapy Revenue Estimates and Forecasts (2015-2026)

1.5.2 Global Cancer Gene Therapy Production Capacity Estimates and Forecasts (2015-2026)

1.5.3 Global Cancer Gene Therapy Production Estimates and Forecasts (2015-2026)

2 Market Competition by Manufacturers

2.1 Global Cancer Gene Therapy Production Capacity Market Share by Manufacturers (2015-2020)

2.2 Global Cancer Gene Therapy Revenue Share by Manufacturers (2015-2020)

2.3 Market Share by Company Type (Tier 1, Tier 2 and Tier 3)

2.4 Global Cancer Gene Therapy Average Price by Manufacturers (2015-2020)

2.5 Manufacturers Cancer Gene Therapy Production Sites, Area Served, Product Types

2.6 Cancer Gene Therapy Market Competitive Situation and Trends

2.6.1 Cancer Gene Therapy Market Concentration Rate

2.6.2 Global Top 3 and Top 5 Players Market Share by Revenue

2.6.3 Mergers & Acquisitions, Expansion

3 Production Capacity by Region

4 Global Cancer Gene Therapy Consumption by Regions

5 Production, Revenue, Price Trend by Type

5.1 Global Cancer Gene Therapy Production Market Share by Type (2015-2020)

5.2 Global Cancer Gene Therapy Revenue Market Share by Type (2015-2020)

5.3 Global Cancer Gene Therapy Price by Type (2015-2020)

5.4 Global Cancer Gene Therapy Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End

6 Global Cancer Gene Therapy Market Analysis by Application

6.1 Global Cancer Gene Therapy Consumption Market Share by Application (2015-2020)

6.2 Global Cancer Gene Therapy Consumption Growth Rate by Application (2015-2020)

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Cancer Gene Therapy Market Scope Analysis 2019-2025 - Science In Me

SILVER SPRING, Md., April 10, 2020 /PRNewswire/ --Today, the U.S. Food and Drug Administration approved Koselugo (selumetinib) for the treatment of pediatric patients, 2 years of age and older, with neurofibromatosis type 1 (NF1), a genetic disorder of the nervous system causing tumors to grow on nerves. Koselugo is the first drug approved by the FDA to treat this debilitating, progressive and often disfiguring rare disease that typically begins early in life.

"Everyone's daily lives have been disrupted during the COVID-19 pandemic, and in this critical time we want patients to know that the FDA remains committed to making patients with rare tumors and life threatening diseases, and their unique needs, a top priority. We continue to expedite product development for these patients," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research.

Koselugo is approved specifically for patients who have symptomatic, inoperable plexiform neurofibromas (PN), which are tumors involving the nerve sheaths (coating around nerve fibers) and can grow anywhere in the body, including the face, extremities, areas around the spine and deep in the body where they may affect organs. Koselugo is a kinase inhibitor, meaning it functions by blocking a key enzyme, which results in helping to stop the tumor cells from growing.

NF1 is a rare, progressive condition caused by a mutation or flaw in a particular gene. NF1 is usually diagnosed in early childhood and appears in an estimated 1 out of every 3,000 infants. It is characterized by changes in skin coloring (pigmentation), neurologic and skeletal impairments and risk for development of benign and malignant tumors throughout life. Between 30% and 50% of patients born with NF1 develop one or more PNs.

"We are committed to regulatory flexibility and providing extensive guidance to industry in an effort to bring drugs forward that fulfill unmet medical needs. Koselugo represents this commitment," noted Pazdur. "For the first time, pediatric patients now have an FDA-approved drug to treat plexiform neurofibroma, a rare tumor associated with NF1."

The FDA approved Koselugo based on a clinical trial conducted by the National Cancer Institute of pediatric patients who had NF1 and inoperable PN (defined as a PN that could not be completely removed without risk for substantial morbidity to the patient). The efficacy results were from 50 of the patients who received the recommended dose and had routine evaluations of changes in tumor size and tumor-related morbidities during the trial. Patients received Koselugo 25 mg/m2 orally twice a day until disease progression or until they experienced unacceptable adverse reactions. The clinical trial measured the overall response rate (ORR), defined as the percentage of patients with a complete response and those who experienced more than a 20% reduction in PN volume on MRI that was confirmed on a subsequent MRI within 3-6 months. The ORR was 66% and all patients had a partial response, meaning that no patients had complete disappearance of the tumor. Of these patients, 82% had a response lasting 12 months or longer.

Other clinical outcomes for patients during Koselugo treatment including changes in PN-related disfigurement, symptoms and functional impairments. Although the sample sizes of patients assessed for each PN-related morbidity (such as disfigurement, pain, strength and mobility problems, airway compression, visual impairment and bladder or bowel dysfunction) were small, there appeared to be a trend of improvement in PN-related symptoms or functional deficits during treatment.

Common side effects for patients taking Koselugo were vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain (pain in the body affecting bones, muscles, ligaments, tendons and nerves), fever, acneiform rash (acne), stomatitis (inflammation of the mouth and lips), headache, paronychia (infection in the skin that surrounds a toenail or fingernail) and pruritus (itching).

Koselugo can also cause serious side effects including heart failure (manifested as ejection fraction decrease, or when the muscle of the left ventricle of the heart is not pumping as well as normal) and ocular toxicity (acute and chronic damage to the eye) including retinal vein occlusion, retinal pigment epithelial detachment and impaired vision. Patients should have cardiac and ophthalmic assessments performed prior to initiating Koselugo and at regular intervals during treatment. Koselugo can also cause increased creatinine phosphokinase (CPK). CPK is an enzyme found in the heart, brain and skeletal muscles. When muscle tissue is damaged, CPK leaks into a person's blood. CPK elevation in a patient receiving Koselugo should prompt an evaluation for rhabdomyolysis (breakdown of skeletal muscle due to direct or indirect muscle injury). Koselugo should be withheld, dosage reduced or dosage permanently discontinued based on the severity of adverse reactions. Further, Koselugo contains Vitamin E, and patients are at an increased risk of bleeding if their daily intake of Vitamin E exceeds the recommended or safe limits.

Based on findings from animal studies, Koselugo may cause harm to a newborn baby when administered to a pregnant woman. The FDA advises health care professionals to tell females of reproductive age, and males with female partners of reproductive potential, to use effective contraception during treatment with Koselugo, and for one week after the last dose.

The FDA granted this application Priority Reviewand Breakthrough Therapydesignation.Koselugo also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases, and Rare Pediatric Disease Designation for the treatment of pediatric NF1. The application is awarded a Rare Pediatric Disease Priority Review Voucher.

The FDA granted approval of Koselugo to AstraZeneca Pharmaceuticals LP.

Additional Resources:

Media Contact:Nathan Arnold, (301) 796-6248Consumer Inquiries: Emailor 888-INFO-FDA

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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SOURCE U.S. Food and Drug Administration

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FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease - BioSpace

Global Cell Harvesting Market,By Type (Manual Cell Harvesters and Automated Cell Harvesters), By Application (Biopharmaceutical Application, Stem Cell Research and other Applications), By End Users (Hospitals, Ambulatory Centers, Clinics, Community Healthcare, Others), By Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2025

Market Analysis:Global Cell Harvesting Market

The Global Cell Harvesting Market is expected to reach USD 387.9 Million by 2025, from USD 196.9 Million in 2017 growing at a CAGR of 8.9% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2017, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

Complete study compiled with over 100+ pages, list of tables & figures, profiling 10+ companies. Ask for Sample @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-cell-harvesting-market&skp

Market Definition:Global Cell Harvesting Market

This market report defines the market trends and forecast the upcoming opportunities and threats of the cell harvesting market in the next 8 years. Cell harvesting is extracting the cells either from bone marrow and peripheral blood cells and culturing the cells in the culture dish containing nutrient media. Cell harvesting is used in the cell therapy as well as in gene therapy. University of California developed a cure for bubble baby disease for new born babies by using the cell harvesting in stem cells and gene therapy. Moreover, Asterias developed the stem cell therapy to regain the upper body motor function. University of California, Irvine developed the stem cell therapy to destroy the breast cancer cells.. Now a days cell harvesting is also used in the animal research and development. Cell Harvesting is also used in may research labs for in-Vitro testing. In September 2016, Terumo BCT collaborated with Cognate Bioservices for developing the immunotherapies and other related products like cell therapy products. These innovations in the cell harvesting market is notably attributing towards its increasing demand at the global pace. Further, its demand is likely to gain momentum over the forecast period.

Major Market Drivers and Restraints:

Market Segmentation:Global Cell Harvesting Market

Competitive Analysis:Global Cell Harvesting Market

The global cell harvesting market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cell harvesting market for global, Europe, North America, Asia Pacific and South America.

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Major Market competitors/players:Global Cell Harvesting Market

Some of the major players operating in the global cell harvesting market are PerkinElmer Inc, Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical, Argos Technologies, SP Industries, Teleflex Incorporated, Arthrex, Inc, Thomas Scientific, Brand GMBH, Brandel, Cox Scientific, Connectorate, Scinomix, Adstec.

Research Methodology:Global Cell Harvesting Market

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analyzed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more pleaseRequest an Analyst Callor can drop down your inquiry.

Demand Side Primary Contributors: Doctors, Surgeons, Medical Consultants, Nurses, Hospital Buyers, Group Purchasing Organizations, Associations, Insurers, Medical Payers, Healthcare Authorities, Universities, Technological Writers, Scientists, Promoters, and Investors among others.

Supply Side Primary Contributors: Product Managers, Marketing Managers, C-Level Executives, Distributors, Market Intelligence, Regulatory Affairs Managers among others

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Cell Harvesting Market Industry Trends and Forecast to 2025 | PerkinElmer Inc, Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical - Bandera County...

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Bispecific Antibody Therapeutic Market which would mention How the Covid-19 is Affecting the Bispecific Antibody Therapeutic Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Bispecific Antibody Therapeutic Players to Combat Covid-19 Impact.

The report titled Global Bispecific Antibody Therapeutic Market is one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Bispecific Antibody Therapeutic market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Bispecific Antibody Therapeutic market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Bispecific Antibody Therapeutic market is carefully analyzed and researched about by the market analysts.

Key companies operating in the global Bispecific Antibody Therapeuticmarket include_Creative-biolabs, PharmAbcine, Sorrento Therapeutics, Patheon,

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Segmental Analysis :

The report has classified the global Bispecific Antibody Therapeutic industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the Bispecific Antibody Therapeutic manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall Bispecific Antibody Therapeutic industry.

Global Bispecific Antibody Therapeutic Market Segment By Type:

Bearing An Fc Region, Lacking An Fc Region

Global Bispecific Antibody Therapeutic Market Segment By Applications:

Osteology, Pulmonary, Respiratory Diseases, Oncology, Gene Therapy

Critical questions addressed by the Bispecific Antibody Therapeutic Market report

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Table Of Content

Table of Contents 1 Bispecific Antibody Therapeutic Market Overview1.1 Product Overview and Scope of Bispecific Antibody Therapeutic1.2 Bispecific Antibody Therapeutic Segment by Type1.2.1 Global Bispecific Antibody Therapeutic Production Growth Rate Comparison by Type 2020 VS 20261.2.2 Bearing An Fc Region1.2.3 Lacking An Fc Region1.3 Bispecific Antibody Therapeutic Segment by Application1.3.1 Bispecific Antibody Therapeutic Consumption Comparison by Application: 2020 VS 20261.3.2 Osteology1.3.3 Pulmonary1.3.4 Respiratory Diseases1.3.5 Oncology1.3.6 Gene Therapy1.4 Global Bispecific Antibody Therapeutic Market by Region1.4.1 Global Bispecific Antibody Therapeutic Market Size Estimates and Forecasts by Region: 2020 VS 20261.4.2 North America Estimates and Forecasts (2015-2026)1.4.3 Europe Estimates and Forecasts (2015-2026)1.4.4 China Estimates and Forecasts (2015-2026)1.4.5 Japan Estimates and Forecasts (2015-2026)1.5 Global Bispecific Antibody Therapeutic Growth Prospects1.5.1 Global Bispecific Antibody Therapeutic Revenue Estimates and Forecasts (2015-2026)1.5.2 Global Bispecific Antibody Therapeutic Production Capacity Estimates and Forecasts (2015-2026)1.5.3 Global Bispecific Antibody Therapeutic Production Estimates and Forecasts (2015-2026) 2 Market Competition by Manufacturers2.1 Global Bispecific Antibody Therapeutic Production Capacity Market Share by Manufacturers (2015-2020)2.2 Global Bispecific Antibody Therapeutic Revenue Share by Manufacturers (2015-2020)2.3 Market Share by Company Type (Tier 1, Tier 2 and Tier 3)2.4 Global Bispecific Antibody Therapeutic Average Price by Manufacturers (2015-2020)2.5 Manufacturers Bispecific Antibody Therapeutic Production Sites, Area Served, Product Types2.6 Bispecific Antibody Therapeutic Market Competitive Situation and Trends2.6.1 Bispecific Antibody Therapeutic Market Concentration Rate2.6.2 Global Top 3 and Top 5 Players Market Share by Revenue2.6.3 Mergers & Acquisitions, Expansion 3 Production Capacity by Region3.1 Global Production Capacity of Bispecific Antibody Therapeutic Market Share by Regions (2015-2020)3.2 Global Bispecific Antibody Therapeutic Revenue Market Share by Regions (2015-2020)3.3 Global Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)3.4 North America Bispecific Antibody Therapeutic Production3.4.1 North America Bispecific Antibody Therapeutic Production Growth Rate (2015-2020)3.4.2 North America Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)3.5 Europe Bispecific Antibody Therapeutic Production3.5.1 Europe Bispecific Antibody Therapeutic Production Growth Rate (2015-2020)3.5.2 Europe Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)3.6 China Bispecific Antibody Therapeutic Production3.6.1 China Bispecific Antibody Therapeutic Production Growth Rate (2015-2020)3.6.2 China Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)3.7 Japan Bispecific Antibody Therapeutic Production3.7.1 Japan Bispecific Antibody Therapeutic Production Growth Rate (2015-2020)3.7.2 Japan Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020) 4 Global Bispecific Antibody Therapeutic Consumption by Regions4.1 Global Bispecific Antibody Therapeutic Consumption by Regions4.1.1 Global Bispecific Antibody Therapeutic Consumption by Region4.1.2 Global Bispecific Antibody Therapeutic Consumption Market Share by Region4.2 North America4.2.1 North America Bispecific Antibody Therapeutic Consumption by Countries 4.2.2 U.S. 4.2.3 Canada4.3 Europe4.3.1 Europe Bispecific Antibody Therapeutic Consumption by Countries 4.3.2 Germany 4.3.3 France 4.3.4 U.K. 4.3.5 Italy 4.3.6 Russia4.4 Asia Pacific4.4.1 Asia Pacific Bispecific Antibody Therapeutic Consumption by Region 4.4.2 China 4.4.3 Japan 4.4.4 South Korea 4.4.5 Taiwan 4.4.6 Southeast Asia 4.4.7 India 4.4.8 Australia4.5 Latin America4.5.1 Latin America Bispecific Antibody Therapeutic Consumption by Countries 4.5.2 Mexico 4.5.3 Brazil 5 Production, Revenue, Price Trend by Type5.1 Global Bispecific Antibody Therapeutic Production Market Share by Type (2015-2020)5.2 Global Bispecific Antibody Therapeutic Revenue Market Share by Type (2015-2020)5.3 Global Bispecific Antibody Therapeutic Price by Type (2015-2020)5.4 Global Bispecific Antibody Therapeutic Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End 6 Global Bispecific Antibody Therapeutic Market Analysis by Application6.1 Global Bispecific Antibody Therapeutic Consumption Market Share by Application (2015-2020)6.2 Global Bispecific Antibody Therapeutic Consumption Growth Rate by Application (2015-2020) 7 Company Profiles and Key Figures in Bispecific Antibody Therapeutic Business7.1 Creative-biolabs7.1.1 Creative-biolabs Bispecific Antibody Therapeutic Production Sites and Area Served7.1.2 Bispecific Antibody Therapeutic Product Introduction, Application and Specification7.1.3 Creative-biolabs Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)7.1.4 Main Business and Markets Served7.2 PharmAbcine7.2.1 PharmAbcine Bispecific Antibody Therapeutic Production Sites and Area Served7.2.2 Bispecific Antibody Therapeutic Product Introduction, Application and Specification7.2.3 PharmAbcine Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)7.2.4 Main Business and Markets Served7.3 Sorrento Therapeutics7.3.1 Sorrento Therapeutics Bispecific Antibody Therapeutic Production Sites and Area Served7.3.2 Bispecific Antibody Therapeutic Product Introduction, Application and Specification7.3.3 Sorrento Therapeutics Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)7.3.4 Main Business and Markets Served7.4 Patheon7.4.1 Patheon Bispecific Antibody Therapeutic Production Sites and Area Served7.4.2 Bispecific Antibody Therapeutic Product Introduction, Application and Specification7.4.3 Patheon Bispecific Antibody Therapeutic Production Capacity, Revenue, Price and Gross Margin (2015-2020)7.4.4 Main Business and Markets Served 8 Bispecific Antibody Therapeutic Manufacturing Cost Analysis8.1 Bispecific Antibody Therapeutic Key Raw Materials Analysis8.1.1 Key Raw Materials8.1.2 Key Raw Materials Price Trend8.1.3 Key Suppliers of Raw Materials8.2 Proportion of Manufacturing Cost Structure8.3 Manufacturing Process Analysis of Bispecific Antibody Therapeutic8.4 Bispecific Antibody Therapeutic Industrial Chain Analysis 9 Marketing Channel, Distributors and Customers9.1 Marketing Channel9.2 Bispecific Antibody Therapeutic Distributors List9.3 Bispecific Antibody Therapeutic Customers 10 Market Dynamics 10.1 Market Trends 10.2 Opportunities and Drivers 10.3 Challenges 10.4 Porters Five Forces Analysis 11 Production and Supply Forecast11.1 Global Forecasted Production of Bispecific Antibody Therapeutic (2021-2026)11.2 Global Forecasted Revenue of Bispecific Antibody Therapeutic (2021-2026)11.3 Global Forecasted Price of Bispecific Antibody Therapeutic (2021-2026)11.4 Global Bispecific Antibody Therapeutic Production Forecast by Regions (2021-2026)11.4.1 North America Bispecific Antibody Therapeutic Production, Revenue Forecast (2021-2026)11.4.2 Europe Bispecific Antibody Therapeutic Production, Revenue Forecast (2021-2026)11.4.3 China Bispecific Antibody Therapeutic Production, Revenue Forecast (2021-2026)11.4.4 Japan Bispecific Antibody Therapeutic Production, Revenue Forecast (2021-2026) 12 Consumption and Demand Fprecast12.1 Global Forecasted and Consumption Demand Analysis of Bispecific Antibody Therapeutic12.2 North America Forecasted Consumption of Bispecific Antibody Therapeutic by Country12.3 Europe Market Forecasted Consumption of Bispecific Antibody Therapeutic by Country12.4 Asia Pacific Market Forecasted Consumption of Bispecific Antibody Therapeutic by Regions12.5 Latin America Forecasted Consumption of Bispecific Antibody Therapeutic 13 Forecast by Type and by Application (2021-2026)13.1 Global Production, Revenue and Price Forecast by Type (2021-2026)13.1.1 Global Forecasted Production of Bispecific Antibody Therapeutic by Type (2021-2026)13.1.2 Global Forecasted Revenue of Bispecific Antibody Therapeutic by Type (2021-2026)13.1.2 Global Forecasted Price of Bispecific Antibody Therapeutic by Type (2021-2026)13.2 Global Forecasted Consumption of Bispecific Antibody Therapeutic by Application (2021-2026) 14 Reseach Finding and Conclusion 15 Methodology and Data Source 15.1 Methodology/Research Approach 15.1.1 Research Programs/Design 15.1.2 Market Size Estimation 15.1.3 Market Breakdown and Data Triangulation 15.2 Data Source 15.2.1 Secondary Sources 15.2.2 Primary Sources 15.3 Author List 15.4 Disclaimer

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COVID-19 Impact on Bispecific Antibody Therapeutic Market Identify Which Types of Companies Could Potentially Benefit or Loose out From the Impact of...

Metabolism as cancer progresses

Numerous cancer-specific alterations in metabolism have been identified but have not yet resulted in an effective anti cancer therapeutic. In a Review, Faubert et al. discuss how metabolism changes as cancer develops from a small, premalignant lesion to an aggressive primary tumor and then metastasizes. Metabolic vulnerabilities likely change with cancer progression, making the identification of general cancer-associated metabolic features difficult. The authors propose that a more targeted approach to tissues and vulnerabilities identified in patients may be more effective.

Science, this issue p. eaaw5473

Metabolic reprogramming is a hallmark of malignancy first recognized a century ago. In some cases, reprogrammed metabolic activities can be exploited to diagnose, monitor, and treat cancer. Stereotyped metabolic activities in cultured cancer cellsnotably, aerobic glycolysis, glutamine catabolism, macromolecular synthesis, and redox homeostasissupport the requirements of exponential growth and proliferation. These pathways are under cell-autonomous control by oncogenic signaling and transcriptional networks. This has produced the widespread perception that a core set of fixed metabolic dependencies will prove to be excellent therapeutic targets across diverse cancer types. Several metabolic inhibitors designed to target these pathways have advanced into clinical trials.

The past decade has brought numerous advances in our understanding of why tumors develop metabolic phenotypes that differ from adjacent, nonmalignant tissues and when these phenotypes represent actionable therapeutic vulnerabilities. Mechanistic insights into how the oncogenotype dictates metabolic patterns have exploded, aided by the ever-increasing use of advanced analytical techniques to characterize tumor metabolism in detail. This has led to the remarkable discovery of a few metabolic properties that can directly promote tumor initiation, including the accumulation of D-2-hydroxyglutarate in tumors with mutations in isocitrate dehydrogenase-1 and -2. Other advances have demonstrated the extraordinary amount of metabolic heterogeneity among human tumors and, in some cases, even within distinct regions of the same tumor. This heterogeneity results from a complex set of factors, including processes intrinsic and extrinsic to the cancer cell. Many of these studies have identified promising subtype-selective metabolic vulnerabilities in experimental models. However, they have cast doubt on the classical paradigm of convergent, oncogene-driven liabilities among histologically and genetically diverse tumors. Even more fundamentally, it has become increasingly clear that metabolic phenotypes and vulnerabilities evolve as tumors progress from premalignant lesions to locally invasive tumors to metastatic cancer. Microenvironmental and genetic factors appear to induce selective pressures that drive clonal evolution within tumors, and this can create or eliminate metabolic liabilities while facilitating cancer progression. During metastasis, for example, several studies demonstrate that cancer cells need to activate mechanisms to resist oxidative stress, or else these cells are culled by the oxidizing environment of the bloodstream. A major theme arising from recent research is that pathways that stimulate the growth of localized, treatment-nave tumors are distinct from and in some cases irrelevant to the activities that drive mortality by supporting metastasis and therapy resistance.

The emerging view of cancer metabolism is that it is flexible and context-specific, with few fixed, broadly applicable liabilities. Understanding how reprogrammed metabolism supports tumor growthand identifying which reprogrammed activities are most relevant to therapeutic liabilitiesrequires a more sophisticated view of how metabolic phenotypes evolve as cancer progresses. Advanced animal models that recapitulate the landmark events in human cancer progression will be instrumental in discovering the most important metabolic vulnerabilities. These animal studies will need to be complemented by increasing efforts to assess metabolism directly in human tumors through metabolomics, metabolic isotope tracers, and advanced techniques in metabolic imaging. Crucially, cooperative, multidisciplinary research is needed to translate findings from animal models into patients and from human cancer into mouse models for mechanistic studies and hypothesis testing. Ideally, work along these lines will generate efficient ways to detect predictive aspects of metabolic behavior in human tumors to aid in clinical trial design and to stratify patients to receive the most effective therapies. These efforts over the next decade should produce a more nuanced but ultimately more relevant and therapeutically actionable view of cancer metabolism.

Metabolic needs and vulnerabilities evolve throughout cancer progression. Early stages of tumor growth require nutrient uptake and biosynthesis, with additional subtype-selective metabolic needs emerging in locally invasive cancers. Tumors acquire dependence on new pathways during later stages of cancer progression, particularly metastasis and therapy resistance. These include potentially targetable liabilities such as dependence on mechanisms to resist oxidative stress and increased reliance on oxidative phosphorylation.

Metabolic reprogramming is a hallmark of malignancy. As our understanding of the complexity of tumor biology increases, so does our appreciation of the complexity of tumor metabolism. Metabolic heterogeneity among human tumors poses a challenge to developing therapies that exploit metabolic vulnerabilities. Recent work also demonstrates that the metabolic properties and preferences of a tumor change during cancer progression. This produces distinct sets of vulnerabilities between primary tumors and metastatic cancer, even in the same patient or experimental model. We review emerging concepts about metabolic reprogramming in cancer, with particular attention on why metabolic properties evolve during cancer progression and how this information might be used to develop better therapeutic strategies.

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Metabolic reprogramming and cancer progression - Science Magazine

Niche players in the global gene therapy market must leverage greater government expenditure and upgrade their existing infrastructure along with expanding their gene therapy centers for sustaining their market hegemony.

ROCKVILLE, MD / ACCESSWIRE / April 8, 2020 / Global gene therapy market is poised for robust growth with net revenue pool set to exceed approximately US$ 5 Bn by 2026 end. The market is receiving tailwinds from advancements in synthetic biology. On that premise, the gene therapy market will expand 3X through over the forecast period, projects Fact.MR (2020-2026).

"Certain types of cancer such as Diffuse Large B-cell Lymphoma (DLBCL) and lymphoblastic leukemia are contributing to high mortality rates across the world. Gene therapy is gaining increasing recognition in its immense potential for treating rare diseases. Continued research and development in the area of gene therapy is supporting market growth as well," states Fact.MR.

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Gene Therapy Market - Key Findings

Gene Therapy Market - Key Driving Factors

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Gene Therapy Market - Key Restraints

Impact of COVID-19 on Gene Therapy Market

In view of ongoing onslaught of COVID-19 pandemic, the focus of major healthcare authorities of the world has gravitated towards developing vaccines for the deadly respiratory disease. Gene therapy is one such area of research which could help boost antibodies required to treat patients infected with Coronavirus. For instance, Generation Bio is exploring the potential role of gene therapy in treating COVID-19 patients. Hence, the global gene therapy market will benefit from the outbreak in that market players are rushing to develop multiple therapeutic approaches for SARS-CoV-2. Growing fears of similar Coronavirus outbreaks in the future will continue accelerating the development of gene therapy as well.

Competitive Landscape

Prominent players profiled in this Fact.MR study include, but are not limited to, Orchard Therapeutics Limited, CELGENE CORPORATION, Spark Therapeutics, Inc., Sibiono GeneTech Co. Ltd., Spark Therapeutics Inc., Gilead Sciences Inc., and Novartis AG. Developed regions remain the key focus area of major stakeholders in the global gene therapy market. Existing gene therapy centers are being prioritized by market players in order to utilize the full extent of their resources. Moreover, they are benefitting from success rates associated with gene therapy and faster drug approvals. Gilead Sciences Inc. expanded their gene therapy centers to a total of 90 recently.

About the Report

This 170-page study offers in-depth commentary on the gene therapy market. The study provides compelling insights on the gene therapy market on the basis of product (yescarta, kymriah, luxturna, strimvelis, and gendicine), application (ophthalmology, oncology, Adenosine Deaminase Deficiency- Severe Combined Immunodeficiency) across three regions (The United States, Europe, and Rest of the World).

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Gene Therapy Market Set for 3X Expansion Between 2020 and 2026; COVID-19 Stimulating Development of Multiple Therapeutic Approaches: Fact.MR - Yahoo...

A group of Korean researchers said they have confirmed a gene responsible for inherited retinal disorders (IRD) among Koreans.

IRD is a combination of several rare diseases that usually develops at a young age and progresses slowly over the lifetime. The patients gradually lose their sight, and most of them eventually lose their vision entirely due to continuous retinal cell degeneration.

The Seoul National University Bundang Hospital (SNUBH) Department of Ophthalmology and Seoul National University Hospital (SNUH) Department of Laboratory Medicine jointly conducted the study.

Currently, antioxidant therapy, artificial retinal transplantation, and stem cell therapy are being used to treat the disorder regardless of mutations, but the only viable treatment is gene therapy. Even when gene therapy is possible, only less than 1 percent of all IRD patients can be treated with it.

In the West, genetic abnormalities of these retinal diseases have been studied and known well. However, researches on Korean cases are still lacking, and the joint research team tackled the subject to find the causative gene for IRDs with 86 domestic patients, the team said in a news release on Wednesday.

The team studied and identified the gene responsible for the disorders by using the latest technique of gene analysis with the most number of patients who have been reported so far.

The study revealed that only 44 percent of the patients, 38 out of 86, possessed the causal gene for IRDs. Even among the patients with retinitis pigmentosa, the most common disorder among the IRDs, only 41 percent had the causative gene.

The causative genes could be quite diverse even in the same disorder. The patients can find a responsible gene only when they receive genetic counseling very actively and can receive gene counseling, too, the research team explained.

Differences were found in the type and frequency of causal gene mutations between Korean and Western cases. However, there were similarities between those of Korean and other Asian nations, including Japan.

The research and diagnosis environment for IRDs has been very poor until now, and our study has significance as a basic data for diagnosis and treatment for Korean patients with IRDs, SNUH Department of Ophthalmology Professor Woo Se-joon said.

Patients need to receive causal gene tests actively to provide the domestic medical communities with sufficient data, and a list of patients who can be treated. By doing so, clinical trials and new drug development in gene therapy will progress smoothly, he added.

Previously, only a few hospitals could diagnose the causative gene for IRDs and afford to test and treat IRD patients due to the high cost of genetic testing. Recently, however, the chance of diagnosis has increased as more hospitals are conducting genetic tests amid the lowered cost thanks to insurance benefits.

Also, the therapeutic opportunity for IRD patients is likely to get broadened, as the retinal pigment epithelium 65 gene (RPE65) therapy won approval from the U.S. Food and Drug Administration for the first time in the world.

Although we do not have a clear way to prevent IRDs at the moment, the prediction of risk and their early detection are developing through the discovery of family history and causative genes, Professor Woo said. Early diagnosis can prevent impaired vision by gene therapy and vision correction, and the patients will be able to choose appropriate jobs with social activities.

Also taking part in the research team were Professors Joo Kwang-sic and Park Kyu-hyung of SNUBH and Professors Seong Moon-woo and Park Sung-sup of SNUH.

The results of this study were published in the Journal of Korean Medical Science.

shim531@docdocdoc.co.kr

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SNUH team finds causal gene of inherited retinal disorder - Korea Biomedical Review

"The rise in Market Size is due to an increasing incident population of Multiple Myeloma patients in the 6MM, along with expected entry of premium price asset which will have an impact on market size"

LAS VEGAS, April 8, 2020 /PRNewswire/ -- DelveInsight has announced an addition of a new market research report on "CAR T-Cell Therapy for Multiple Myeloma Market Insights and Market Forecast-2030" to its offerings.

The report offers in-depth CAR T-Cell Therapy for Multiple Myeloma Market Analysis, 11 years Market Size Forecast, detailed Epidemiology analysis and 11-year Forecast, Emerging Drugs Market Uptake and information related to Leading Companies along with the Competitive Analysis edge.

Some key highlights from the report

:

Click here to getCAR T-Cell Therapy for Multiple Myelomamarket sample pages: https://delveinsight.com/sample-request/car-t-cell-therapy-for-multiple-myeloma-market

Chimeric Antigen Receptor (CAR) T-cell therapy is a technique that involves genetic modification of patient's autologous T-cells for CAR expression, specific for a tumor antigen, followed by ex-vivo cell expansion and re-infusion back to the patient.

Chimeric Antigen Receptor T-cells are the fusion proteins of a selected single-chain fragment that are the variable from a specific monoclonal antibody and one or more T-cell receptor intracellular signaling domains. T-cell genetic modification may occur either through viral-based gene transfer methods or non-viral methods like DNA-based transposons, CRISPR/Cas9 technology, zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), or direct transfer of in vitro transcribed-mRNA by electroporation.

CAR T-Cell Therapy for Multiple Myeloma Treatment Market

According to WHO, in the year 2018, 159,985 incident cases of multiple myeloma were reported in the world. Multiple myeloma is a rare type of cancer that develops in plasma cells. The patients diagnosed with Multiple Myeloma have cancer cells that eventually supersede the healthy plasma cells. This process exhausts the much-needed white blood cells in the body.

A cure for Multiple Myeloma currently is not available. However, significant research is making progressive leaps toward a Multiple Myeloma treatment that eliminates the disease entirely. Some of them are enumerated below:

Targeted therapy: Targeted drug treatment targets on specific abnormalities within cancer cells that enables them to survive. The drugs like Bortezomib (Velcade), carfilzomib (Kyprolis) and ixazomib (Ninlaro), which may be administered through a vein in the arm or in pill form, block the action of a substance in myeloma cells that breaks down proteins. Eventually, it leads to the myeloma cells to die.

Biological therapy: Biological therapy drugs use body's immune system to kill myeloma cells. The drugs like thalidomide (Thalomid), lenalidomide (Revlimid) and pomalidomide (Pomalyst) improve the immune system cells to identify and fight cancer cells. These medications are usually taken in pill form.

Chemotherapy: Chemotherapy drugs kill fast-growing cells like myeloma cells. Chemotherapy drugs can be administered through a vein in the arm or taken in pill form.

Corticosteroids: Prednisone and dexamethasone regulate the immune system to control inflammation in the body. They are also active against myeloma cells. Corticosteroids can be taken in pill form or administered through a vein in patient's arm.

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Radiation therapy: This treatment uses beams of energy, such as X-rays and protons, to kill myeloma cells and prohibit their growth. Radiation therapy may be used to swiftly shrink myeloma cells in a specific area.

Several biotechnological companies that are working towards the development of therapies and will impact CAR T-Cell Therapy for Multiple Myeloma treatment market scenario in the upcoming years.

Drugs covered in the report are:-

And many others

TheCAR T-Cell Therapy for Multiple Myeloma Market Key Players are:-

And many others

Scope of the report:

Click here to getCAR T-Cell Therapy for Multiple Myelomamarket sample pages: https://delveinsight.com/sample-request/car-t-cell-therapy-for-multiple-myeloma-market

Insight covered regarding Reimbursement Scenario in Multiple Myeloma CAR T-Cell Therapy

Approaching reimbursement can have a positive influence on both during the late stages of product development and after product launch. In report, reimbursement is taken into consideration to identify economically attractive indications and market opportunities. When working with limited resources, the ability to choose the markets with the fewest reimbursement barriers can be a critical business & price strategy.

KOL- Views

To keep up with current market trends, KOLs and SME's opinions, working in CAR T-Cell Therapy for Multiple Myeloma domain are taken into account through primary research to fill the data gaps and validate the secondary research. Their opinion assist to comprehend and validate current and emerging therapies treatment patterns and Multiple Myeloma market trend. This will benefit the clients in potential upcoming novel CAR T-Cell Therapy for Multiple Myeloma treatment by recognizing the overall scenario of the market and the unmet needs.

Click here to getCAR T-Cell Therapy for Multiple Myelomamarket trends sample pages: https://delveinsight.com/sample-request/car-t-cell-therapy-for-multiple-myeloma-market

Reasons to buy

Key Topics Covered:

1

Key Insights

2

Executive Summary

3

CAR T-Cell Therapy Market Overview at a Glance

4

CAR T-Cell Therapy Background and Overview

5

CAR T-Cell Therapy for Multiple Myeloma (MM): 6 Major Market Analysis

6

CAR T-Cell Therapy Market Outlook

7

CAR T-Cell Therapy Emerging Drug Profiles for Multiple Myeloma

7.1

bb2121: Celgene Corporation

7.2

JNJ-68284528 (LCAR-B38M): Janssen Research & Development

7.3

P-BCMA-101: Poseida Therapeutics

7.4

CAR-CD44v6: MolMed S.p.A.

7.5

JCARH125 (Orvacabtagene autoleucel): Celgene Corporation

7.6

Descartes-08: Cartesian Therapeutics

7.7

CT053 : CARsgen Therapeutics)

7.8

PBCAR269A: Precision BioSciences

8

United States Market Size

9

EU-5 Market Size

9.1

Germany

9.2

France

9.3

Italy

9.4

Spain

9.5

United Kingdom

10

CAR T-Cell Therapy Clinical Trials in 6MM

11

KOL Views CAR T-Cell Therapy

12

CAR T-Cell Therapy for Multiple Myeloma Market Drivers

13

CAR T-Cell Therapy for Multiple Myeloma Market Barriers

14

Appendix

15

DelveInsight Capabilities

16

Disclaimer

17

About DelveInsight

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About DelveInsight

DelveInsight is a leading Business Consultant, and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end to end comprehensive solutions to improve their performance.

Related Reports:-

DelveInsight's "CAR T-Cell Therapy for Acute Lymphoblastic Leukemia (ALL) Market Insights and Market Forecast- 2030" report delivers an in-depth understanding of the CAR T-Cell Therapy use for Acute Lymphoblastic Leukemia as well as the CAR T-Cell Therapy market trends for Acute Lymphoblastic Leukemia in the 6MM i.e., United States and EU5 (Germany, Spain, Italy, France and the United Kingdom).

DelveInsight's 'CAR T-Cell Therapy for Non-Hodgkin's lymphoma Market Insights and Market Forecast-2030' report delivers an in-depth understanding of the CAR T-Cell Therapy for Non-Hodgkin's lymphoma and market trends in the 6MM, i.e., United States and EU5 (Germany, Spain, Italy, France and United Kingdom).

Contact us:Shruti Thakurinfo@delveinsight.com+91-9650213330DelveInsight

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Market Size of CAR T-Cell Therapy for Multiple Myeloma in the 6MM is Expected to Be USD 3,766.4 Million in 2030 | Some of the Major Players Involved...

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A genetic variant in a gene called MET is responsible for more aggressive growth of head and neck cancer, and lung cancer, according to a new study.

A further probe into the finding reveals therapeutic strategies that could potentially target this genetic alteration and pave the way for better and more effective treatments.

The MET gene encodes for a cancer promoting protein that relays growth, survival, and transmission of signals in cancer cells, researchers say.

As reported in Nature Communications, researchers also identified a form of MET protein which showed ethnic preference with higher incidence among Asians, and associated with poorer prognosis in patients diagnosed with head and neck squamous cell carcinoma or lung squamous cell carcinoma.

Even though the MET variant does not seem to predispose someone to head and neck cancer or lung cancer, it leads to more aggressive growth of cancers that have already developed.

Unlike other MET mutants, existing MET-blocking drugs do not seem to inhibit this genetic variant, prompting researchers to conduct further investigation on the mechanism behind the genetic alteration.

The team found that the single amino-acid change in the MET receptor from the genetic alteration leads to preferential strong binding to another cancer promoting protein, HER2. Both proteins then work together to drive cancer aggression and allow the cancer cells to survive therapies that involve MET-blocking drugs.

The mechanism of this MET variant is novel and unreported. This finding contributes to the growing evidence of the role of genetic variants in affecting clinical outcome, and underscores the importance of diving deep into our genetic inheritance in cancer research, says Kong Li Ren of the Cancer Science Institute (CSI) Singapore at NUS, who initiated the study.

Knowledge of this unique mechanism also allowed researchers to identify several HER2 inhibitors capable of blocking cancer progression the genetic alteration caused.

Our study represents a conceptual advancement to cancer research, as we have shown that it is possible to block the activity of a cancer-driving gene by administrating a targeted therapy directed not against the mutant protein in question, but rather, a corresponding protein with which it binds to, says Goh Boon Cher, deputy director and senior principal investigator at CSI Singapore.

The remarkable anti-tumor responses observed in our experimental models, coupled with the availability of FDA-approved HER2 inhibitors, also presents a huge opportunity for clinicians to improve disease outcome of this genetic alteration via precision medicine.

The research team is now translating the findings to a clinical trial where patients tested positive for this MET variant gene are treated with suitable medications that have shown effectiveness in the laboratory.

Additional coauthors are from the National University Cancer Institute, the National Cancer Centre Singapore, and the Bioinformatics Institute at the Agency for Science, Technology and Research, Singapore.

Source: National University of Singapore

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Gene variant makes head and neck cancer more aggressive - Futurity: Research News

The two firms plan to leverage Orgenesiss autologous Cell and Gene Therapy Biotech Platform to produce muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes

Inc () is teaming up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells for therapeutic use, it said Wednesday.

The two firms plan to leverage Orgenesiss autologous Cell and Gene Therapy (CGT) Biotech Platform to develop novel therapies and advance clinical trials.

Under the agreement, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and various other cellular products.

Orgenesis and RevaTis are hoping to build on RevaTiss early success in animals to develop therapies and advance human trials using Orgenesiss expertise and Point-of-Care platform, which include automated systems, 3D printing and bioreactor technologies.

RevaTiss technologies are highly differentiated and ideally suited for Orgenesiss CGT Biotech Platform, Orgenesis CEO Vered Caplan told shareholders.

We believe that this exclusive partnership with RevaTis further validates the significant value proposition of the Orgenesis vertically integrated business model, Caplan said in a statement Wednesday.

This model allows us to streamline the entire process of therapeutic development and delivery of cell therapies within the patient care setting through our Cell & Gene Biotech Platform.

RevaTis also has existing partnerships with research institutions in the US, the Middle East and India that will be highly complementary to Orgenesiss POCare Network, according to Caplan. We look forward to utilizing the Orgenesis Cell & Gene Biotech Platform with a goal to lower the costs and accelerate the timeline of bringing these innovative therapies through the clinic and potentially into commercialization.

RevaTis CEO Didier Serteyn said the firm was delighted to partner with Orgenesis on the new venture.

We selected Orgenesis as a result of their extensive experience in the field of autologous cell therapies, including technical, clinical and regulatory expertise, Serteyn said in a statement. We expect that this will be valuable as we aim to advance our platform through commercialization.

Germantown, Maryland-based Orgenesis is a vertically integrated biopharmaceutical company with expertise in developing advanced cell therapies and manufacturing.

Shares of Orgenesis jumped 5.8% on Wednesday to reach $4.40 by midday.

Contact Angela at [emailprotected]

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Orgenesis partners with Belgium"s RevaTis to develop stem cells for therapeutic use - Proactive Investors USA & Canada

MARTINSRIED, Germany--(BUSINESS WIRE)--SIRION Biotech GmbH announced today that Beam Therapeutics licensed rights to use SIRION Biotechs LentiBOOST for use in their CAR-T cell products.

CAR-T cell therapy represents a promising and future-defining shift in cancer treatment. Beam Therapeutics is developing a new generation of CAR-T product candidates using its proprietary base editing technology.

Under the terms of this agreement, SIRION agreed to provide Beam with non-exclusive access to its proprietary lentiviral transduction enhancer LentiBOOST for clinical development and commercialization of Beams portfolio of CAR-T programs. SIRION will be entitled to undisclosed upfront and milestone payments and is eligible to receive royalties on future product net sales plus license fees tied to commercial success.

Dr. Christian Thirion, CEO and founder of SIRION Biotech GmbH explains: LentiBOOST was engineered to improve lentiviral transduction of difficult cell types like T-cells and hematopoietic stem cells. This technology enables robust upscaling of the T-cell production process, and helps to reduce manufacturing costs by lowering the amount of lentiviral vectors needed for production of the cell product while at the same time improving clinical efficacy by increasing vector copy numbers (VCN) per cell. We are delighted that the LentiBOOST technology may help Beam further enhance the clinical success of its CAR-T pipeline.

LentiBOOST is used in an increasing number of clinical trials in the US and in Europe and the technology is more and more considered as a gold standard in manufacturing of cell products. Our non-exclusive licensing strategy makes our technology available to a wide range of companies and research hospitals to boost the efficiency of their various clinical programs, says SVP of Business Development & Licensing, Dr. Sabine Ott.

About SIRION Biotech GmbH

SIRION Biotech was founded in 2005 to lead the next generation of viral vector technologies for gene and cell therapy as well as vaccine development. Now SIRION offers one of the worlds most comprehensive viral vector technology platforms based on lenti-, adeno-, and adeno-associated viruses which expedites gene therapy research and advances drug development. SIRION is becoming a partner of choice in this growing sector. LentiBOOST has been used in a number of clinical trials from early stage clinical Phase 1/2 through late stage clinical Phase 3 trials and demonstrated clinical success in improving transduction of the therapeutic vector.

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Beam Therapeutics Licenses SIRION Biotech's LentiBOOST Technology for its CAR-T pipeline - Business Wire

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After more than 20 years of research, we are now witnessing a breakthrough of small interfering RNA (siRNA)-based therapies. In 2018, the first-ever siRNA drug, Onpattro, reached the market, followed by the approval of Givlaari in 2019, and many other clinical trials are in progress.

Holding the potential to treat a wide range of diseases from cancer to immunological disorders, siRNA therapeutics have received plenty of attention. With the support of a suitable delivery system, they can be directed to downregulate a specific target gene. Both approved siRNA drugs Onpattro and Givlaari are only able to reach the liver, however. Other organs that can be treated by loco-regional administration, such as the lung, are, in principle, good targets for siRNA therapies as well.

In this view, siRNA-baseddrugs could not only act as an ally in the battle against the current COVID-19pandemic but also against other severe lung diseases such as asthma. Despitethe great advances in asthma treatment, this disease still represents an unmetmedical need in about 510% of patients.Moreover, most of the available drugs work symptomatically rather than causally.

In a recent article published in WIREs Nanomedicine and Nanobiotechnology, Domizia Baldassi and Tobias Keil, graduate students in Prof. Olivia Merkels research group at the University of Munich, discuss the groups advances towards developing a nanocarrier that can deliver siRNA into T cells in the lung.

The aim of T-cell delivery is downregulation of GATA-3, the transcription factor of T helper 2 (TH2) cells overexpressed in asthmatic patients, which is recognized as a key factor in the asthmatic inflammatory cascade. Based on their observation that transferrin receptor is overexpressed in activated T cells, the researchers sought to find a virus-like tool to target activated TH2 cells specifically and efficiently in a receptor-mediated manner.

They accomplishedthis goal by creating a conjugate formed by transferrin and low-molecular-weightpolyethylenimine (Tf-PEI). On the one hand, they used a well-known cationic polymerto electrostatically interact with the negatively charged siRNA and protect itfrom degradation during the journey through the airways. And on the other hand,transferrin served as a targeting moiety to mediate a specific, targeteddelivery of siRNA only to activated T cells.

Since theendosomal escape is considered the rate-limiting step in cytoplasmaticdelivery of nanoparticle-based therapies, improving this aspect of theformulation was the focus, and Tf-PEI was blended with a second conjugatecomposed of melittin and PEI (Mel-PEI). Melittin is a well-known membranolyticagent from bee venom that was chemically modified to react in a pH-dependentmanner.

The researchersexploited the intrinsic lytic characteristic of the peptide to improve therelease of siRNA into the cytosol, reaching knockdown levels as high as 70% exvivo. But further steps such as the validation of these results in vivo on anasthma mouse model are needed, as well as possible alternative polymericmaterials.

In the process of developing a new pharmaceutical product, it is crucial to keep the administration route in mind. Spray drying is the most straightforward technique to produce inhalable particles for pulmonary delivery, according to the researchers. In a proof-of-concept study, they obtained nano-in-microparticles by spray drying PEI-pDNA polyplexes together with a cryoprotectant agent. After seeing promising results, their studies to obtain a dry powder formulation of siRNA-based polyplexes are ongoing.

Ultimately, both research fields will be combined and hopefully result in a new therapy for the treatment of severe, uncontrolled asthma and many other lung diseases, concluded Baldassi, Keil, and Merkel.

Reference: Tobias W. M. Keil et al. T-cell targeted pulmonary siRNA delivery for the treatment of asthma. WIREs Nanomedicine and Nanobiotechnology (2020). DOI: 10.1002/10.1002/wnan.1634

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Biotechnology A new, inhaled siRNA therapeutic option for asthma - Advanced Science News

The Gene Therapy market demand is anticipated to flourish during the forecast period 2020-2027. The report offers information related to import and export, along with the current business chain in the market at the global level. This report provides an in-depth overview of the Gene Therapy market. This includes market characteristics, consisting of segmentation, market share, trends and strategies for this market. The Market Size section provides historical forecasts of market growth and future. An in-depth analysis of the major companies operating in the market is also mentioned in this research report.

The global Gene Therapy market is segmented on the basis of type and application. It also provides market size and forecast estimates from the year 2020 to 2027 with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East & Africa (MEA), and South America (SAM). The Gene Therapy market by each region is later sub-segmented by respective countries and segments. The report covers the analysis and forecast of top countries globally along with the current trend and opportunities prevailing in the region.

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A thorough examination of the Gene Therapy market includes each and every aspect, which begins with knowing the market, speaking with clients, and evaluating the complete data of the global market. For more clarification, the global market is segmented on the basis of the manufacture of the kind of products, and their applications. The report also delivers information as per the regions based on the geographical classification of the global Gene Therapy market. The dynamic foundation of the global market is based on the calculation of product supply in different markets, their revenues, capability, and a chain of production.

Qualitative information will discuss the key factors driving the restraining the growth of the market, and the possible growth opportunities of the market, regulatory scenario, value chain & supply chain analysis, export & import analysis, attractive investment proposition, and Porters 5 Forces analysis among others will be a part of qualitative information.

Furthermore, this study will help our clients solve the following issues:

All in all, the Gene Therapy market research study elucidates a detailed evaluation of this business and projects this industry to register a commendable growth rate in the forthcoming years. The Gene Therapy market analysis report also delivers important insights with respect to aspects such as the volume of sales, valuation forecast, market size, and the market competition trends as well as the market concentration rate.

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Finally, this business guide will be helpful for both established and new players to sustain in the competitive Gene Therapy market world as it mentions the key geographies, market landscapes alongside the product price, revenue, volume, production, supply, demand, and market growth rate & gives the maximum possible profit for your company.

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Gene Therapy Market 2020 Overview | Identify Opportunities and Challenges | Size, Share and Industry Analysis by coherentmarketinsights.com -...

The partnership will help accelerate the time-to-market of the Jeeva decentralized clinical trial solution.

WASHINGTON (PRWEB) April 08, 2020

KiwiTech, LLC, an innovation platform that helps startups build viable products, drive traction, and raise capital, today announced a strategic partnership with Jeeva Informatics Solutions Inc, an AI-driven startup that builds patient-focused big data and digital health solutions for accelerating clinical research.

"We are excited to support Jeeva in their mission to decentralize clinical research, reduce patients' travel burden and optimize operations," said Rakesh Gupta, Co-Founder, and CEO of KiwiTech. "Though Jeeva set out on their vision much before the current pandemic, it has become particularly relevant in the context of COVID-19. We look forward to helping Jeeva further its mission and scale through our unique startup ecosystem."

While Biopharmaceutical companies have been conducting randomized controlled clinical trials for over 70 years, the traditional process has become unsustainable. According to a 2018 report by PhRMA, the average cost of bringing one successful therapy to market through FDA approval exceeded $2.5B and took over 12 years.

Jeeva Informatics was selected by the National Science Foundation (NSF) in Spring 2019 into the Innovation Corps grant program at their Massachusetts Institute of Technology (MIT) node, Boston, MA to understand first-hand, the various stakeholders' perspectives on the pain-points and bottlenecks. Having interviewed over 300 customer stakeholders of clinical trials in the U.S., the major pain points recorded consistently were patient recruitment, retention, and protocol adherence.

"Patients' travel burden of visiting brick-and-mortar sites is a single problem that, when solved right, can help address all of those customer pain-points. Jeeva is focused on solving this problem by developing a real-time decentralized solution for collecting patients' outcomes data which replaces in-person interactions with videoconferencing, digital engagement, and remote safety monitoring. Such a robust solution can enable our customers to better manage unforeseen complexities, such as those arising out of a global pandemic." said Harsha Rajasimha, founder and CEO of Jeeva.

Earlier this year in January, Jeeva had announced a round of investment led by CITGapFunds and plans to launch its first product in late 2020 with several clients. Jeeva's initial focus will include long-term follow-ups after the administration of cell and gene therapies for rare diseases and Cancer. Jeeva is seeking additional early adopters and strategic partners with access to real-world clinical settings.

"Our selection process is highly competitive. In 2019, we received 40,734 startup applications, interviewed 2,501 of them, and invested in only 109 of them. Jeeva expects to achieve its minimum viable product in 2020 for its initial set of customers and strategic partners. The partnership with KiwiTech allows Jeeva to leverage our deep technical expertise and insight to realize the promise of decentralized or hybrid clinical trials in the U.S. and eventually globally," said Gupta.

About KiwiTech

Over the last decade, KiwiTech has led innovation across multiple verticals scaling businesses in the tech space. KiwiTech intimately understands the challenges faced by new founders, aspiring unicorns and established leaders and works closely with them to realize their passion and purpose. Through its extensive relationships and expertise, KiwiTech brings a perspective that helps strategically connect technology creators with technology consumers. KiwiTech is an ecosystem of proactive investors and advisors who are passionate about building technology, supporting entrepreneurship, and helping companies realize their maximum potential.

About Jeeva Informatics

Jeeva is a seed-stage venture-backed startup on a mission to develop AI-driven decentralized clinical trials and optimize patient-focused clinical research. The Jeeva Trials solution reduces patient travel burden by replacing in-person trial site visits with videoconferencing, remote monitoring and digital engagement. Jeeva is particularly beneficial for both rare disorders and clinical research studies involving long-term follow-ups and safety monitoring. Jeeva is a member of several life science membership organizations including Virginia BIO, BioHealth Capital Region, and the Global Genes Corporate Alliance.

For the original version on PRWeb visit: https://www.prweb.com/releases/kiwitech_and_jeeva_informatics_solutions_announce_strategic_partnership/prweb17039405.htm

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KiwiTech and Jeeva Informatics Solutions Announce Strategic Partnership - Benzinga

GLASGOW, Scotland, April 8, 2020 /PRNewswire/ -- Lamellar Biomedical Limited (Lamellar), an innovative biotechnology company pioneering new approaches to address respiratory diseases and complex lung disorders with its LAMELLASOME platform, announces the launch of a program to address the potentially fatal consequences of COVID-19 on respiratory function.

A LAMELLASOME nebulised treatment used at the early stages of COVID-19, in hospital or the community, could reduce the damaging and often fatal inflammatory response known as Acute Respiratory Distress Syndrome (ARDS) seen in patients. This early intervention has the potential to inhibit fibroproliferative changes during ARDS, markedly reducing severity, mortality and lowering the significant burden placed on the healthcare system.

The time between onset of symptoms of SARS-CoV2 infection and progression to more severe clinical manifestations of COVID-19 such as viral pneumonia and ARDS, is thought to be between 5 8 days. Inhaled LAMELLASOME treatment administered during this period has the potential to halt or reduce the severity of the disease progression of COVID-19 patients to requiring scarce critical care resource.

Lamellar has strong pre-clinical evidence of inhaled LAMELLASOME's protective nature. In a large animal invivo model of radiation-induced lung injury, designed to replicate the pathology found in the injured lung, which is representative of COVID-19 patient pathology, inhaled LAMELLASOME treatment protected lung cells and tissues from injury, pneumonitis and fibroproliferation/fibrosis at the alveolar/capillary membrane.

Dr Duncan Moore, Chairman of Lamellar, said: "In the past few weeks we have seen the terrible consequences COVID-19 can have on the respiratory function, particularly on vulnerable patient populations. We believe that that the inherent attributes of LAMELLASOME are extremely well suited to be a potential approach to preventing the onset of the serious respiratory symptoms seen in COVID-19 patients. Lamellar is focused on treating complex lung disorders and we believe that our Lamellasome formulations could make an important difference to patients and healthcare providers globally."

Dr Nik Hirani, Reader in Respiratory Medicine and Associate Medical Director in Lothian and Former chair of NICE Thoracic Interstitial Lung Disease guideline committee, said: "Lamellar has very convincing data demonstrating strong in-vivo efficacy for Lamellasome formulations in a model relevant to pneumonitis, lung injury and ARDS. I have been following the development of LAMELLASOME technology and its development as a respiratory therapeutic over the past few years, and I believe it has real potential to manage the inflammatory respiratory symptoms seen in patients infected with SARS-CoV2."

About Lamellar Biomedical

Lamellar Biomedical Limited (Lamellar), is an innovative biotechnology company, developing our proprietary LAMELLASOME based therapies to transform the treatment of complex and rare lung disorders.

Lamellar's lead products are:

In addition, LAMELLASOME technology can be designed to deliver active payloads such as gene therapies, or by utilising intrinsic anti-infective properties which can enhance antibiotics.

Founded in 2007, Lamellar is backed by both institutional and private investors, including Invesco, Scottish Enterprise, Barwell Plc, TRI Capital and has multiple research collaborations with world renowned institutions and universities.

About LAMELLASOME Technology

LAMELLASOME vesicles are synthetic lipidic mimetics of native human lamellar bodies found in lung tissue. These properties provide low immunogenicity and excellent safety, LAMELLASOME vesicles have been shown to have inherent pulmonary protective effects. LAMELLASOME therapies are based on LAMELLASOME's abilities to:

LAMELLASOME formulations have excellent preclinical safety, a very high NOAEL (taken from inhalation toxicology) and excellent clinical safety and tolerability profile.

LAMELLASOME therapeutics are synthetically formulated. As such, they do not require harvesting or human extraction, and they can be manufactured using standard, scalable industry processes and components that facilitate commercially competitive COGS. They can also be optimised to deliver active payloads such as gene therapies and anti-infectives.

Story continues

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Lamellar Biomedical Launches Program Designed to Prevent the Severe Respiratory Effects of COVID-19, Using its Unique LAMELLASOME Technology - Yahoo...

SAN DIEGO Apr 8, 2020 (Thomson StreetEvents) -- Edited Transcript of Organovo Holdings Inc earnings conference call or presentation Thursday, November 9, 2017 at 10:00:00pm GMT

Organovo Holdings, Inc. - CFO

* Steve E. Kunszabo

Organovo Holdings, Inc. - VP of IR & Corporate Communications

* Taylor J. Crouch

Organovo Holdings, Inc. - CEO, President & Director

* George B. Zavoico

B. Riley FBR, Inc., Research Division - Analyst

Good afternoon and welcome to the Organovo Holdings fiscal second quarter 2018 earnings conference call. (Operator Instructions.] Please note this event is being recorded. I would now like to turn the conference over to Steve Kunszabo, Head of Investor Relations. Please go ahead.

Steve E. Kunszabo, Organovo Holdings, Inc. - VP of IR & Corporate Communications [2]

Good afternoon and thanks for joining us. I'd like to welcome you to our fiscal second quarter 2018 earnings call. Joining me on the call this afternoon: our CEO, Taylor Crouch; our CFO, Craig Kussman; and our General Manager, Paul Gallant. Today's call will begin with a discussion of the 2018 fiscal second quarter results, followed by Q&A.

Before I turn things over to Taylor, I'd like to caution all participants that our call this afternoon may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts and include statements about our future expectations, plans and prospects. Such forward-looking statements are based upon our current beliefs and expectations and are subject to risks which could cause the actual results to differ from these forward-looking statements. Such risks are more fully disclosed in our filings with the Securities and Exchange Commission. Our remarks today should be considered in light of such risks. Any forward-looking statements represent our views only as of today. And while we may elect to update forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our expectations or views change.

During the call we'll also be referring to certain non-GAAP financial measures. These non-GAAP financial measures are not prepared in accordance with generally accepted accounting principles. Please refer to today's earnings release for a definition of these non-GAAP financial measures.

With that, let me turn things over to Taylor.

Taylor J. Crouch, Organovo Holdings, Inc. - CEO, President & Director [3]

Thanks, Steve, and good afternoon, everyone. I'll get us started by highlighting that our top line results of $1.4 million for the quarter represented our second-highest quarterly revenue to date and our third consecutive quarter of sequential revenue growth. This is good progress as we streamlined the business around maximizing the uptake of our liver and kidney tissue systems. But we still have more work to do.

We updated our full-year revenue and negative adjusted EBITDA guidance today, which reflects the latest assessment of our growth trajectory. We are prioritizing disease modeling capabilities as we head into the second half of our fiscal year and de-emphasizing our work in the routine toxicology research area. Our guidance also shows the benefit of cash burn from our organizational restructuring. Craig will provide more detail on our financials and the revised elements of our outlook in his remarks.

As I consider the steps we've taken in the last few months to recalibrate our direction and evaluate the best path forward, our strategic identity has come into clearer focus. Our highly customizable 3D tissue platform affords us the ability to monetize our capabilities in many ways. We provision and curate primary human cells for use in research applications through our Samsara business. We've created dynamic disease models that can facilitate drug discovery and profiling in ways never thought possible. We've struck licensing agreements that capitalize on our proprietary technology and intellectual property, and we continue to develop our own novel therapeutic tissue products to address critical unmet disease areas. We are working across a broad value chain that spans the drug discovery and development ecosystem with what we believe are the industry's leading liver and kidney tissue systems.

Moving now to our commercial tissue business where our bioprinted liver remains the primary engine of growth, I'd like to share a few leading indicators underscoring the strength of new business, the health of our revenue mix, the growing value of our services and our significantly reduced cycle times.

First, we added 8 customer accounts in the first half of fiscal 2018 including 3 clients projects with products already in clinical trials and we continue to see momentum in bring new business aboard.

Second, we continued to see a healthy revenue mix during the first 6 months of our fiscal year, with a 60% to 40% split between repeat business and new orders. This is consistent with our breakdown in fiscal 2017 and supports the ongoing move by our clients up the adoption curve.

Third, we've driven our average revenue per order higher over the last 2 quarters, largely as the result of shifting our work to compound screening and disease models. In the first half of fiscal 2018, approximately 65% of our orders represented disease modeling projects. This is good news because our competitive differentiation is particularly strong in these applications including target identification, marker validation and lead optimization.

Lastly, our cycle time from sending out a customer proposal to closing an order has declined sharply. In late 2016 we were averaging over 7 months for this important business development metric. In the first half of fiscal 2018, we've brought the span down to under 2 months, in part owing to the master services agreements and repeat business we have with our existing customers as well as to better understanding our client needs relating to projects outlined and study design. In addition, as a result of our business development, marketing and scientific outreach, many new and existing clients are seeking out our services with a sense of urgency to interrogate their drugs on our platform.

Our progress in studying liver disease has also been bolstered by the $1.7 million grant award we received from the NIH to collaborate with UC San Diego to evaluate NASH in our 3D bioprinted liver model. I also note our collaboration with Viscient Biosciences to develop a custom research platform that targets early discovery work for liver disease. We're also pleased with the distinction and reception our posters received at the recent American Association for the Study of Liver Disease annual meeting, which is one of the most influential conferences in the liver disease space.

Overall, our shift to disease modeling services recognizes the important role that liver disease plays in pharmaceutical R&D while also representing the highest-value opportunity for our commercial business. We're seeing deeper engagement from our clients in this space as we discuss annual budget allocations and framework agreements for our services. Ultimately, these are important steps as our customers move from single-project commitments to dedicated research plans and meaningful annual revenue commitments.

Let's turn now to a quick progress update on our therapeutic tissues business. As many of you saw in our recent announcement, we achieved major scientific milestones for the extended survival and functionality of our liver therapeutic tissue and we remain on track to submit our first investigational new drug application to the FDA during calendar year 2020. Our bioprinted liver tissues showed significant engraftment, retention and functionality through 125 days post-implementation in well-established animal models for one of the inborn areas of metabolism, namely alpha-one-antitrypsin deficiency. Importantly, thorough evaluation of the treated animals suggests an approximately 75% reduction in the pathologic hallmarks of disease in treated areas. In short, our liver patch meaningfully cleared the disease in the immediate area of the implant.

The data show that the approach of delivering a 3D bioprinted tissue patch directly to the liver offers great promise in solving the retention and integration issues that have held back many cell and gene therapy attempts at treating these debilitating pediatric liver diseases. These are also major achievements because we believe they align with the historical expectations of regulators for these types of preclinical studies and allow us to confidently move on to the next phases of our development work now that these elements have been completed.

Regarding next steps, we'll continue to move forward with our application to seek orphan drug designation in the next several months, and we've now begun new animal model studies in a second therapeutic indication within the category of inborn areas of metabolism.

In closing, we've taken concrete steps in the first half of the fiscal year to sharpen our focus on liver and kidney tissue systems, we've shifted our commercial priorities to be centered on higher-demand and higher-value disease modeling projects and we've restructured the organization to support these objectives. In doing so, we've improved our future revenue growth trajectory while also reducing our operating costs and materially lowering our cash burn rate. My emphasis will continue to be on meeting with customers, driving adoption of our platform commercialization, finalizing key proof-of-concept work for our liver therapeutic tissue program and translating all of this progress into value inflection points. And we will continue to be good stewards of our balance sheet. I look forward to communicating with you again soon as we execute against these important commercial and research milestones.

With that, I'll turn it over to Craig for a more complete financial review.

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Craig Kussman, Organovo Holdings, Inc. - CFO [4]

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Thanks, Taylor, and good afternoon, everyone. I'll begin by recapping our key financial metrics for the fiscal second quarter and then summarize the fiscal 2018 guidance we updated today. I'll wrap up my thoughts by briefly reviewing our balance sheet and liquidity profile.

Organovo generated fiscal second quarter total revenue of $1.4 million, which was down 2% from the prior-year period and up 37% sequentially. Total revenue results were driven by lower collaborations revenue, as key collaboration agreements were completed in the prior fiscal year, partially offset by grant payments related to our recently awarded NIH grant. Product and service revenue was $0.9 million, down 8% from the prior-year period. As Taylor noted, we're recalibrating our business development effort to focus on higher-demand and higher-value disease modeling services. Although we saw a significant increase in disease modeling orders, which were approximately 65% of total orders in the second quarter, these gains were not enough to offset the toxicology research services revenue we recorded in the year-ago quarter. Despite the short-term impact as we now deploy our resources against this new market opportunity, we see robust uptake of compound screening and disease models and expect it to be a major revenue growth component going forward.

I'll focus next on operating expenses. We reported $0.3 million in cost of revenues for the fiscal second quarter, a 35% decline from the prior-year period. The drop in cost of revenues was largely due to a greater contribution from higher-margin primary human cell and tissue products. Research and development expenses were $4.9 million, a 9% year-over-year increase, primarily resulting from higher facilities and employee-related costs. We recorded $5.7 million in selling, general and administrative expenses during the fiscal second quarter, a 3% year-over-year decrease, largely due to lower external professional services fees and facilities costs. SG&A also included approximately $0.4 million of one-time CEO transition and employee severance costs. As we look ahead to the fiscal third quarter, it's worth emphasizing that we'll record an approximately $0.9 nonrecurring charge related to our recently announced organizational restructuring.

And finally, a brief review of the full year fiscal 2018 outlook we updated today and a few quick notes on our balance sheet and liquidity profile. We now forecast total revenue between $4.5 million and $6.5 million for fiscal year 2018, with the primary contributions coming from a few key components: continued strong growth of our disease modeling services, recording the balance of our deferred revenues from the previous year, accelerating growth from our primary human cell and tissue products and accelerating progress on our NIH grant, of which $200,000 to $600,000 is reflected in our fiscal 2018 guidance range.

While we've revised our fiscal 2018 outlook and now expect a delay in ramping to a double-digit annual revenue rate, we remain confident that one or more of our customers will move from single-project commitments to annual revenue commitments over the course of the next few quarters. Achieving this goal with our customers should position us to meet this important revenue metric in 2019.

On the same basis for the full year fiscal 2018, we now expect negative adjusted EBITDA between $26 million and $28 million. This is an improvement in our cash burn from our previous estimate and is largely driven by the reduced operating costs we'll benefit from as the result of our organizational restructuring and a streamlined focus on our existing commercial opportunities and therapeutic tissue program. By comparison, we recorded $29.8 million of negative adjusted EBITDA for fiscal 2017.

Before I move on to our balance sheet, a few quick words on the evolution of our Samsara subsidiary and its growing contribution to our business. First, it provides us with great expertise in the sophisticated isolation and curation of our target liver cells. Second, we're able to further explore how disease origin cells perform in our tissue models across various disease states. Finally, it benefits us from a business development perspective, as many clients who worked with Samsara first moved to also engage in more complex liver and kidney disease services. All in all, great synergies between the 2 operations as Samsara becomes a more meaningful portion of our total revenue.

Now for our balance sheet. At the end of the fiscal second quarter, we had a cash and cash equivalents balance of $50.7 million and we have approximately $17 million of funds available under our ATM facility. In combination, this gives us approximately $68 million in available liquidity to carry out our business plan and invest in our key growth initiatives. As circumstances and market dynamics permit, we'll continue to use our ATM facility opportunistically to extend the cash runway for the business, allowing us to scale our liver and kidney tissue research services and moving our promising liver therapeutic tissue closer to human clinical trials. The ATM facility is a flexible tool that lets us strengthen our balance sheet in a disciplined way while moving us forward to key value inflection points as we consider our long-term capital plan. If we're able to successfully execute against our planned ATM strategy, we do not currently envision a traditional equity raise such as a follow-on equity offering for the remainder of fiscal 2018.

I'll wrap up by noting that we believe there's a growing value for Organovo as we continue to advance our liver therapeutic issue and achieve major scientific milestones. As for our commercial business, we continue to streamline our operations and redeploy our effort against the best opportunities to grow our revenue in the liver and kidney space. Disease modeling represents a big part of this future path. We look forward to updating you on our progress in February.

With that, I'll turn things back over to the operator for the Q&A portion of this afternoon's call.

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Questions and Answers

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Operator [1]

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(Operator Instructions) And our first question comes from Brandon Couillard with Jefferies.

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Christian Peter Trigani, Jefferies LLC, Research Division - Former Equity Associate [2]

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This is Christian on for Brandon. First off, I understand you're taking a broader shift into disease modeling, but it would be helpful if you could provide additional commentary in terms of the key variables that drove the materially lower full year revenue outlook. I'm just trying to understand, given the magnitude of the downward revision, why you didn't ultimately decide to prerelease the development or update the full year view concurrent with the restructuring plan announced in early October.

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Taylor J. Crouch, Organovo Holdings, Inc. - CEO, President & Director [3]

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This is Taylor. And one of the challenges that we face, and that's why we've given a broad revenue guidance, even for this remaining several months of our fiscal year, is that we have a number of binary events that we hope and plan and are working toward breaking our way, which could result in significantly more revenues. But as each month closes, we have to recognize the risk that some of these binary events don't go our way in this fiscal year but move into the final -- into the next fiscal year.

As an example, we've mentioned that our deferred revenues for clients that we have hoped, and certainly still plan to try and achieve this year, may or may not fall into this fiscal year. And similarly, you see with our NIH grant revenue a significant range tied to certain operational risk variables and NIH grant approval processes that affect this range. So it's more an issue of being more cautious about the outcomes as we see -- as we evaluate these binary events, as well as reflecting that we truly had hoped to see some more traditional routine tox business from our earliest clients. But now we realize that the investments we would have to make to convince them to move over their thresholds to incorporating us into what ultimately is a lower-margin but higher-volume business, those thresholds don't provide us the same return on investment as we're seeing by investing now heavily in our disease modeling platforms, which are being driven by significant and enthusiastic demand from those clients. So it's a number of forces that are coming together, and our visibility on these forces clarifies as we approach the end of the year. And we just thought it was more prudent to wait until this call to clarify that situation.

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Christian Peter Trigani, Jefferies LLC, Research Division - Former Equity Associate [4]

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Okay, understood. That's very helpful. And then I guess, also probably for Taylor, maybe a 2-part question. I would be very interested to hear an update on how the conversations are progressing in terms of moving the target one or more clients for a larger multiyear routine use engagement. To be clear, do you still expect that to occur within the fiscal year '18? And maybe help us quantify how many customers might be in that Phase 2 bucket currently that could over time move to steady-state use over maybe the next 12 to 18 months.

And then secondly, do you think you have enough current capacity to perhaps inboard those customers over time? And if so, maybe give us a rough percentage of what capacity you think you would need to commit to such customers to fulfill a larger engagement.

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Taylor J. Crouch, Organovo Holdings, Inc. - CEO, President & Director [5]

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Okay, thanks. Both great questions. With regard to our clients moving along the adoption curve, and so the Phase 2 that you referred to, for the others on the call, represents the final validation of our custom research platforms, a process that we're undergoing for each of our clients. And in some cases, by the way, we're validating more than one platform. And the final step of that phase is to begin running standard reference compounds through our platforms to show how they modulate the very diseases that we've created in our tissues in these unique platforms. And I am pleased to say that we now have a handful of clients that are in this final step of the -- of testing the modulation strategies with either reference or, in some cases, with proprietary compounds. As son as that's completed, we believe the very next step is to move to more routine use for proprietary screening and profiling larger numbers of compounds. And we do indeed have conversations underway at more than one client along those lines, and that continues to give us confidence that we will exit this fiscal year tracking toward that routine use, more annual perspective kind of collaboration agreement.

Your second question as regards to capacity, we actually have significant capacity here to expand, certainly to meet the needs both for our commercial tissue business as well as our therapeutic tissue research program to handle various growth scenarios over the next couple of years. So we don't worry about capacity, and we have scalable, modular capacity built around our printing platforms that can easily be scaled up when and if necessary.

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Christian Peter Trigani, Jefferies LLC, Research Division - Former Equity Associate [6]

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Great. And then maybe if I can sneak one more in. Recently we noticed another 3D tissue player in the landscape signed a deal with the CRO, Charles River. Now I understand this player has a somewhat different focus and value proposition, but would be curious to know at a high level if you see an opportunity over time to potentially work more closely with other CROs out there, and if so, possibly over what time frame that could occur. Thank you.

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Taylor J. Crouch, Organovo Holdings, Inc. - CEO, President & Director [7]

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Actually, I'll grab that question as well. As you may know, my background, a big part of my career has been spent in the CRO industry and the biotech positions I've held in those positions, I've also been instrumental in putting strategic CRO collaborations in place with all of the leading CROs. And certainly, we see an opportunity as we scale and as we establish more repetitive use applications on our platform to work with CROs, certainly if we approach a capacity constraint. And so I think it would be very reasonable to expect us to be working, collaborating and announcing appropriate agreements in the fiscal year to come along those lines.

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Operator [8]

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Our next question is from George Zavoico with B. Riley-FBR.

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George B. Zavoico, B. Riley FBR, Inc., Research Division - Analyst [9]

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I have a question about your therapeutic tissues, your liver tissues. Your shift in focus -- part of it is perhaps a disappointing potential revenue stream from the toxicology research services, but it must be balanced by some sort of marketing surveys that you may have done with the therapeutic tissues despite the fact that you're waiting for an IND until 2020. So the question is what kind of supportive marketing information do you have that really speaks to the potential demand and the potential revenue stream from this, and are you looking for partnering opportunities as well in this regard, in this sector?

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Taylor J. Crouch, Organovo Holdings, Inc. - CEO, President & Director [10]

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So first, in terms of demand, we mentioned some of our cycle time statistics. And a year ago, the process of getting a meeting with a classic toxicology decision maker to discuss how our experimental but promising platform might be useful, there was interest, but I wouldn't call it overly enthusiastic, and often it took months and months to close a first revenue opportunity with those decision-makers.

On the disease modeling side, our partners are therapeutic teams, clinicians that actually have real problems they need to solve with a high degree of urgency, and/or people in discovery who so far have had no other in vitro solutions to explore how their drugs will perform in human-like settings in these disease models. So we actually have a significant amount of people -- clients -- reaching out to us. We have clients that we've talked to in the past about toxicology coming much more enthusiastically to discuss our disease models, and I'd say we have a veritable Who's Who of people working in the NAFLD, NSH and fibrosis space across U.S., Europe and Japan beginning to move forward with us along this adoption phase. So it's really a demand-driven opportunity. We see the market opportunity is quite significant because the later you get in discovery, as you know, with research services, the more the value of the drugs you're touching has. And so as you move forward to lead optimization, preclinical candidates and certainly drugs that are always in the clinic, we are creating and enabling a much higher-value outcome for our clients in their decision-making. And we're seeing an uptick in the revenues and the sense of urgency that they have to work with us. And all of these things have built toward the decision to move in this space, as well as recognizing that we, as liver experts, already are developing our own therapeutic solutions with our tissues in and around the liver space. And this gives us tremendous credibility with sophisticated clients focusing liver disease or better understanding their drug in the liver and kidney environment.

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George B. Zavoico, B. Riley FBR, Inc., Research Division - Analyst [11]

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So the disease modeling aspect, which is the services part of your therapeutic tissue sector, that's the part you see, say, perhaps revenue per unit or revenue per client is substantially greater than the toxicology services, and that could drive your own research into developing your own therapeutic tissue for which you expect an INDD in 202? So that's sort of the balance? Is that -- am I understanding that correctly?

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Taylor J. Crouch, Organovo Holdings, Inc. - CEO, President & Director [12]

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I think that's a good way to look at it. And clearly, we're still in an early revenue-generation mode. I see a logical and attractive opportunity to build our revenues along the lines that you just described. We also have mentioned in our comments many of the other ways that complement our platform in terms of how we can monetize our capabilities, starting with our very sophisticated ability to isolate and curate cells, which we do for ourselves, but is also a growing demand out in the marketplace, all the way through to licensing of our technologies for people who would like to bring some of these capabilities in-house. But certainly in the field of NASH, fibrosis and other liver disease areas that we're being asked to begin looking at, we see an overlapping layer by layer of model -- of platform development clients, revenues associated with that, and then the product screening and profiling revenues that come with each of those overlapping platforms that we develop. So it's a nice, healthy business model.

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George B. Zavoico, B. Riley FBR, Inc., Research Division - Analyst [13]

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Continued here:
Edited Transcript of ONVO earnings conference call or presentation 9-Nov-17 10:00pm GMT - Yahoo Finance

Located on the Tufts University Medford, MA campus, this new donor center will enable delivery of fresh blood, leukopaks and buffy coats for COVID-19, cell and gene therapy research within hours of collection

WESTBURY, NY - Apr 6, 2020 - BioIVT, a leading provider of research models and services for drug and diagnostic development, today announced the opening of its new blood donor center on the Tufts University campus in Medford, MA to support academic and pharmaceutical researchers involved in COVID-19, cell and gene therapy research.

BioIVT wants to play a leading role in supporting COVID-19 research efforts and blood donations are a vital resource for the research and development of new therapies, vaccines, and diagnostics. We have many years experience developing blood products, including blood-derived immune cells for cell and gene therapy research, and we want to make that expertise count, said BioIVT CEO Jeff Gatz. Researchers recognize and appreciate BioIVTs rapid response and commitment to high quality, fresh blood products and this new donor center will allow us to offer those attributes and services to additional US clients.

BioIVTs new Boston blood donor center is its seventh. The company has similar facilities located in California, Tennessee and Pennsylvania to serve US clients and in London, UK for EU-based clients.

While the initial focus at our Boston donor center will be on delivering fresh blood, leukopaks and buffy coats within hours of collection, we plan to add more capabilities and donors over time, said Jeff Widdoss, Vice President of Donor Center Operations at BioIVT.

Leukopaks, which contain concentrated white blood cells, are used to help identify promising new drug candidates, assess toxicity levels, and conduct stem cell and gene therapy research. They are particularly useful for researchers who need to obtain large numbers of leukocytes from a single donor.

BioIVT blood products can be supplied with specific clinical data, such as the donor age, ethnicity, gender, BMI and smoking status. Its leukopaks are also human leukocyte antigen (HLA), FC receptor and cytomegalovirus typed. HLA typing is used to match patients and donors for bone marrow or cord blood transplants. FC receptors play an important role in antibody-dependent immune responses.

COVID-19-related Precautions

Blood donor centers are considered essential businesses and will remain open during the COVID-19 quarantine. BioIVT is taking additional safety measures to protect both blood donors and its staff during this difficult time. It has instituted several social distancing measures, including increasing the space between chairs in the waiting room and between donor beds, and limiting the entrance of non-essential personnel. The screening rooms are disinfected between donors and all areas of the center continue to be cleaned at regular intervals.

As soon as each blood donor signs their informed consent form, their temperature is taken. If they have a fever, their appointment is postponed, and they are referred to their physician. Any donor who develops COVID-19 symptoms after donating blood is required to inform the center immediately.

All BioIVT blood collections are conducted under institutional review board (IRB) oversight and according to US Food and Drug Administration (FDA) regulations and American Association of Blood Banks (AABB) guidelines.

Those who would like to donate blood at BioIVTs new Boston-area donor center should call 1-833-GO-4-CURE or visit http://www.biospecialty.com to make an appointment.

Further information about the products available from BioIVTs new donor center can be found at https://info.bioivt.com/ma-donor-ctr-req.

About BioIVT

BioIVT is a leading global provider of research models and value-added research services for drug discovery and development. We specialize in control and disease-state biospecimens including human and animal tissues, cell products, blood and other biofluids. Our unmatched portfolio of clinical specimens directly supports precision medicine research and the effort to improve patient outcomes by coupling comprehensive clinical data with donor samples. Our PHASEZERO Research Services team works collaboratively with clients to provide target and biomarker validation, phenotypic assays to characterize novel therapeutics, clinical assay development and in vitro hepatic modeling solutions. And as the premier supplier of hepatic products, including hepatocytes and subcellular fractions, BioIVT enables scientists to better understand the pharmacokinetics and drug metabolism of newly-discovered compounds and their effects on disease processes. By combining our technical expertise, exceptional customer service, and unparalleled access to biological specimens, BioIVT serves the research community as a trusted partner in elevating science. For more information, please visit http://www.bioivt.com or follow the company on Twitter @BioIVT.

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BioIVT Opens New Blood Donor Center to Support Boston-area Research into COVID-19 Therapies, Vaccines and Diagnostics - Bio-IT World

Evotec has allied with Takeda to expand into gene therapy research. The move sees Evotec establish a 20-person team in Austria, the focal point of Takedas gene therapy operation, and sign up to work on programs for its Japanese partner.

Takeda acquired a gene therapy center in Orth an der Donau, Austria, through its acquisition of Shire, which picked up the site two years earlier in its takeover of Baxalta. Throughout the series of changes in ownership, which began when Baxter spun out Baxalta in 2014, a team at the site has worked on gene therapies.

Now, Evotec is set to start playing a role in those efforts. The German drug discovery shop has set up a gene therapy unit, Evotec GT, staffed by a team of more than 20 scientists in Orth an der Donau who have worked together for many years.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

Evotec disclosed news of the move into gene therapies alongside details of a multiyear partnership with Takeda. The deal, which features an undisclosed upfront fee and other payments over time, tasks Evotec with applying its new gene therapy capabilities and broader drug discovery platform to Takedas cancer, rare disease, neuroscience and gastroenterology programs.

Neither Evotec nor Takeda referred directly to a transfer of employees in the statement to disclose the deal. However, Evotec did reveal that Friedrich Scheiflinger is leading its gene therapy unit. Until recently, Scheiflinger headed up drug discovery for Takeda in Austria, with a particular focus on gene therapies. In light of Evotecs comments about its new gene therapy team having worked together for years, it is likely that other gene therapy researchers made the move from Takeda with Scheiflinger.

The agreement gives Evotec a beachhead in the fast-growing gene therapy sector. In explaining(PDF) the rationale for moving into the space, Evotec expressed a desire to be "modality agnostic" and develop wholly and co-owned candidates.

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Evotec allies with Takeda to move into gene therapy R&D - FierceBiotech

DetailsCategory: More NewsPublished on Tuesday, 07 April 2020 18:36Hits: 263

MARTINSRIED, Germany I April 07, 2020 ISIRION Biotech GmbH announced today that Beam Therapeutics licensed rights to use SIRION Biotechs LentiBOOST for use in their CAR-T cell products.

CAR-T cell therapy represents a promising and future-defining shift in cancer treatment. Beam Therapeutics is developing a new generation of CAR-T product candidates using its proprietary base editing technology.

Under the terms of this agreement, SIRION agreed to provide Beam with non-exclusive access to its proprietary lentiviral transduction enhancer LentiBOOST for clinical development and commercialization of Beams portfolio of CAR-T programs. SIRION will be entitled to undisclosed upfront and milestone payments and is eligible to receive royalties on future product net sales plus license fees tied to commercial success.

Dr. Christian Thirion, CEO and founder of SIRION Biotech GmbH explains: LentiBOOST was engineered to improve lentiviral transduction of difficult cell types like T-cells and hematopoietic stem cells. This technology enables robust upscaling of the T-cell production process, and helps to reduce manufacturing costs by lowering the amount of lentiviral vectors needed for production of the cell product while at the same time improving clinical efficacy by increasing vector copy numbers (VCN) per cell. We are delighted that the LentiBOOST technology may help Beam further enhance the clinical success of its CAR-T pipeline.

LentiBOOST is used in an increasing number of clinical trials in the US and in Europe and the technology is more and more considered as a gold standard in manufacturing of cell products. Our non-exclusive licensing strategy makes our technology available to a wide range of companies and research hospitals to boost the efficiency of their various clinical programs, says SVP of Business Development & Licensing, Dr. Sabine Ott.

About SIRION Biotech GmbH

SIRION Biotech was founded in 2005 to lead the next generation of viral vector technologies for gene and cell therapy as well as vaccine development. Now SIRION offers one of the worlds most comprehensive viral vector technology platforms based on lenti-, adeno-, and adeno-associated viruses which expedites gene therapy research and advances drug development. SIRION is becoming a partner of choice in this growing sector. LentiBOOST has been used in a number of clinical trials from early stage clinical Phase 1/2 through late stage clinical Phase 3 trials and demonstrated clinical success in improving transduction of the therapeutic vector.

SOURCE: SIRION Biotech

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Beam Therapeutics Licenses SIRION Biotech's LentiBOOST Technology for its CAR-T pipeline | More News | News Channels - PipelineReview.com

Genprex Inc. () is a clinical-stage gene therapy company developing a new approach to treating cancer, based upon a novel proprietary technology platform, including initial product candidate, Oncoprex immunogene therapy for non-small cell lung cancer.

The Austin, Texas-based firms platform technologies are designed to administer cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities.

Oncoprex works partly by interrupting the cell signaling pathways that prompt cancer cells to multiply as well as controlling the immune response against cancer cells.

It is basically the tumor suppressor candidate 2 (TUSC2) gene wrapped in a cholesterol nanoparticle that is engineered to target cancer cells.

The nanoparticles are attracted by the opposite electrical charge of tumors, and they pass more easily through immature blood vessels that grow quickly around tumors.

Thats a big advantage because the nanotechnology can be injected intravenously, so it goes through the whole body, as opposed to many traditional gene therapies using viral delivery systems that have been injected directly into tumors.

Thus, Oncoprex can attack metastatic tumors in hard to reach places or are too small to detect. The Nanoparticles are absorbed by tumor cells at rate 10 times to 25 times higher than normal cells, but have little or no effect on normal cells, so toxicity to patients is low relative to other lung cancer drugs.

Genprex went public in April 2018, listing on the Nasdaq under the ticker GNPX and raising US$6.4 million. A private investment in public equity, or PIPE, in May that year raised an additional US$10 million.

Genprex had a great start to 2020, announcing on January 21 that the US Food and Drug Administration had granted Fast Track Designation for its Oncoprex immunogene therapy to treat lung cancer.

The FDA gave its approval to the therapy combination with EGFR inhibitor osimertinib - PLCs (NYSE:AZN) Tagrisso. That drug had worldwide sales in 2018 of $1.86 billion, $2.31 billion in the first nine months of 2019 and is currently s highest grossing product for the treatment of non-small cell lung cancer (NSCLC) patients with EFGR mutations that progressed after treatment with osimertinib alone.

FDA may award Fast Track Designation if it determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This provision is intended to facilitate development and expedite the review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.

Genprex said a few weeks later on February 5 that it plannedto initiate a Phase I/II clinical trial of Oncoprex combined with osimertinib in mid-2020 at multiple cancer centers across the US.

It added that it did not at that time intend to reopen enrollment in its current Phase I/II trial using a combination of Oncoprex and EGFR inhibitor erlotinib (marketed by Genentech in the US and elsewhere by Roche as Tarceva) against NSCLC.

However, the group noted that tumor shrinkage in patients resistant to erlotinib enrolled in that trial showed that Oncoprex can overcome resistance to TKIs and provided support for the Fast Track Designation for the combination with osimertinib.

Genprex also said it planned to file an amendment to its Investigational New Drug (IND) application with the FDA for the Oncoprex and osimertinib combination therapy trial within the first quarter of 2020.

The group said it would also proceed with a plan to file an Investigational New Drug (IND) application for the additional combination therapy of Oncoprex combined with the immunotherapy drug pembrolizumab -- marketed as Keytruda by Merck & Co Inc () in the US for NSCLC.

The FDA fast track designation for NSCLC came after a good run of data for Oncoprex use against other cancers.

In September last year, a study by an independent researcher reported that the active ingredient in Oncoprex had been shown to prevent tumor growth in triple-negative breast cancer (TNBC).

The study,published in the medical journal 'Nature', showed that researchers found TUSC2, the lead component of the drug, acts as a tumor-preventing gene.

Then in November, Genprex revealed positive preclinical data for the treatment of some of the most resistant metastatic lung cancers using its immunogene therapy.

Its collaborators from The University of Texas MD Anderson Cancer Center presented the data at the American Association of Cancer Research Tumor Immunology and Immunotherapy 2019 Meeting.

And at the end of January, Genprex noted that independent researchers had reported in a study that TUSC2, a tumor suppressor gene and the active agent in Oncoprex, is a potential target and biomarker for thyroid carcinoma.

Published in the International Journal of Molecular Sciences, the study found that TUSC2 overexpression decreased thyroid cancer proliferation, migration and invasion. Cell proliferation, migration and invasion ability are essential steps in tumor metastasis

Away from cancer, in February, Genprex also revealed that it has signed a licensing agreement with the University of Pittsburgh for a diabetes gene therapy that could have the potential to cure Type 1 and Type 2 diabetes.

The gene therapy, developed at the Rangos Research Center at UPMC Childrens Hospital of Pittsburgh, works by reprogramming beta cells in the pancreas to restore their function, allowing them to replenish insulin levels.

Genprex has also added to its cash coffers. At the end of February, the company closed a $17.5 million offering of 5 million shares, at a price of $3.50 per share, and said it planned to use the proceeds to advance its drug development programs, for working capital and for general corporate purposes.

That followed an at-the-market stock offering at the end of January that raised $8 million through the sale of 7.6 million shares at a price of $1.05 per share.

On the management front, near the end of March, Genprex appointed two experienced life sciences executives, with Catherine MVaczy becoming executive vice president and chief strategy officer, while Michael TRedman was appointed executive vice president and chief operating officer.

Vaczy has over 20 years' experience as a founder and senior executive of life science companies. Her CV includes experiences with NeoStem Inc (now Caladrius Biosciences), a Nasdaq-listed clinical-stage biotechnology company and ImClone Systems Incorporated (sold to Eli Lily and Company).

Meanwhile, Redman brings more than 30 years of experience in the life sciences industry, having been president, CEO and director of OncolixInc, a publicly-traded clinical-stage biopharma focused on developing therapies for womens and childrens cancers.

In 2001, he co-founded Opexa Pharmaceuticals, a company developing immunotherapies for a variety of diseases, and served as its president and CEO until 2005. He has worked at Zonagen (now , which is a part of ), Aronex Pharmaceuticals, Biovail Corporation and American Home Products (acquired by ).

A few days later, the group bolstered its board further with the appointment of three new directors. The trio were Brent Longnecker, CEO of Longnecker & Associates; Jose A. Moreno Toscano, CEO at LFB USA Inc; and William R. Wilson, Jr, chairman, president and CEO of Wilson Land & Cattle Co.

In a statement with the FDA Fast Track news in January, CEO Rodney Varner said: Genprex is excited to receive this important FDA designation.

In addition to potentially facilitating and expediting our pathway to approval, we believe that this FDA designation validates our plan to commercialize Oncoprex immunogene therapy in combination with EGFR inhibitors for the treatment of lung cancer. We hope that Fast Track Designation helps us bring our gene therapy to patients more rapidly and that our unique gene therapy platform is more widely recognized for its potential in cancer treatment, he added.

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Genprex gene therapy candidate Oncoprex fast-tracking for non-small cell lung cancer treatment - Proactive Investors Australia

The Report Provides detailed knowledge of upcoming market trends and current conditions in the global market. This report covers the past, present and forecast period for the long-term and collective examination.

ReportsnReports added a new report on The Gene Therapy in CVMD Marketreport delivers the clean elaborated structure of the Report comprising each and every business related information of the market at a global level. The complete range of information related to the Gene Therapy in CVMD Market is obtained through various sources and this obtained the bulk of the information is arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis to generate a whole set of trade based study regarding the Gene Therapy in CVMD Market.

Report at: http://www.reportsnreports.com/contactme=1591140

Gene therapies have been a point of discussion during the last several years as a potential curative option for a variety of disease indications. While mainly still in preclinical stages, gene therapy aims to treat or alleviate a disease by genetically modifying the cells of a patient.

This report focuses on gene therapies in development across the 8MM for cardiovascular and metabolic disorders, including coronary artery disease, critical limb ischemia, diabetic foot ulcers, and Pompe Disease. In addition, this report provides an assessment of the pipeline, clinical, and commercial landscape of gene therapies in CVMD supplemented with a variety of KOL and payer perspectives.

Reason to access this Report:

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline, Develop business strategies by understanding the trends shaping and driving the global CVMD gene therapy market, Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global CVMD gene therapy market in the future, Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors, Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage, Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Report:

http://www.reportsnreports.com/.aspx?name=1591140

Scope of Gene Therapy in CVMD Market Report:

Overview of CVMD and Gene Therapies: epidemiology and regulatory oversight, Pipeline Assessment: regional breakdown, promising late-stage products, early-stage pipeline by molecule type, Clinical Trials Assessment: trial breakdown by phase, leading industry and non-industry sponsors, Market Access: considerations for reimbursement, pricing, and unmet needs, Market Outlook: competitive assessment and key market events (2018-2025).

Table of contents for Gene Therapy in CVMD Market:

1.1 Related Reports 4

1.2 Upcoming Related Reports 5

1.3 Abbreviations 6

2.1 Key Findings 10

2.2 KOL and Payer Insight on CVMD Gene Therapy Competitive Landscape 11

3.1 What is Gene Therapy? 14

3.2 Gene Transfer Methods and Vectors Used for Gene Therapy 18

3.3 Viral Vectors vs. Non Viral Vectors 20

3.4 Therapeutic Gene Therapy Strategies Employed in CVMD 24

3.5 Gene Therapy in the 8MM 25

4.1 Coronary Artery Disease 33

4.2 Peripheral Artery Disease 34

4.3 Peripheral Artery Disease with Critical Limb Ischemia 35

4.4 Systolic Heart Failure 36

4.5 Diabetic Foot Ulcers 37

4.6 Diabetic Neuropathy 38

4.7 Pompe Disease 39

5.1 CVMD Gene Therapy Pipeline in the 8MM 42

5.2 Pipeline Products - Phase III 43

5.3 AnGes MGs Collategene 44

5.4 Angionetics Generx 45

5.5 ViroMeds Donaperminogene Seltoplasmid 46

5.6 Renovas RT-100 49

5.7 Pipeline Products - Phase II 50

5.8 ID Pharmas DVC-10101 51

5.9 Juventas JVS-100 52

5.10 UFs Gene Therapy to Activate Acid Alpha Glucosidase for Pompe Disease 54

6.1 Clinical Trial Mapping 57

6.2 Clinical Trial Design 59

7.1 Current CVMD Space 62

7.2 Challenges Associated with Reimbursement of Novel CVMD Therapies 63

7.3 Prospective Payer Strategies for CVMD Gene Therapies 64

8.1 Phase III CVMD Gene Therapy Pipeline 67

8.2 Key Launch Dates for Phase II and III CVMD Gene Therapy Pipeline 71

9.1 Sources 73

9.1 Methodology 74

9.2 Primary Research 75

9.3 About the Authors 76

And more...

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Gene Therapy in CVMD Market report made possible by top research firm - WhaTech Technology and Markets News

Investigators have identified genomic alterations that appear to be associated with prognosis in men with metastatic castration-sensitive prostate cancer (mCSPC).

Astudy of 424 patients with mCSPC treated at a tertiary care center revealedthat alterations in the androgen receptor (AR), TP53, cell-cycle, MYC oncogenicsignaling pathways occur more commonly in tumors with worse overall survivaland decreased time to castration-resistant disease, whereas alterations in theSPOP and MNT pathways occur more frequently in tumors with a better prognosis,according to findings published in ClinicalCancer Research.

Thegenomics of metastatic castration-sensitive prostate cancer have not been wellcharacterized in the literature, but it is now clear that upfront treatmentintensification with taxanes or next-generation AR-directed therapies offerbenefit in the overall patient population, said the studys co-senior authorWassim Abida, MD, a medical oncologist at Memorial Sloan Kettering CancerCenter in New York. The question remains whether treatment selection ortargeted therapies can be employed based on genomic characteristics.

Theassociation between alterations in cell-cycle genes and TP53 and MYC pathwaygenes and worse outcomes may pave the way for targeted therapy in thesehigher-risk groups, Dr Abida said.

Thestudy compared genomic alterations according to clinical phenotypes: high- vslow-volume disease and de novo vsmetastatic recurrence. Of the 424 patients in the study, 213 men (50%) hadhigh-volume disease (4 or more bone metastases or visceral metastases) and 211(50%) had low-volume disease; 65% had de novometastases and 35% had metastatic recurrence. At the time of sample collection, patients had a medianage of 66 years. The investigators conducted gene sequencing from May 2015 to September2018.

High- vs low-volume disease

Inadjusted analyses, men with higher-volume disease had significant 1.8- and3.7-fold increased risks of castration-resistant disease and death,respectively, compared with men who had low-volume disease. Tumor specimensfrom men with high-volume disease had more copy number alterations.

Amongmen with high-volume disease, the highest-ranking pathways were the NOTCH,cell-cycle, and epigenetic modifiers pathways.

Althoughthe prevalence of CDK12 alterations differed between patients with de novo metastatic and those with metastaticrecurrences, the groups had similar prognoses. I was actually surprised therewere not more dramatic genomic differences between de novo and relapsed disease, said study co-senior author PhilipKantoff, MD, a medical oncologist and Chair of the Department of Medicine atMemorial Sloan Kettering Cancer Center in New York.

Afteradjusting for disease volume and other genomic pathways, the researchers foundthat the rates of castration resistance differed by 1.5-fold and up to 5-fold accordingto alterations in the AR, SPOP (inverse), TP53, cell-cycle, WNT (inverse), andMYC pathways. Overall survival (OS) rates varied from 2- to 4-fold according toalterations in the AR, SPOP (inverse), WNT (inverse), and cell-cycle pathways.PI3K pathway alterations were not associated with prognosis.

Docetaxeland next-generation AR axis-directed therapies have been shown to prolong OS, butit remains uncertain which patients benefit the most from intensifiedtherapies. We did not find any obvious genomic reason to explain thedifferences in docetaxel sensitivity between high- and low-volume disease, DrKantoff said.

Theauthors pointed out that genomic landscape studies of tumor DNA profiling inprostate cancer in general have excluded metastatic castration-sensitive tumors.Instead, most studies have focus on localized disease or metastaticcastration-resistant disease.

DrAbida and his colleagues acknowledged that their study has inherent biases becauseit was hospital based and enrolled patients at an academic referral center.

Moleculardeterminants of castration resistance or survival in patients with mHSPC have beenunclear, but the new study sheds new light on molecular alterations associatedwith poor outcomes in men with mHSPC, particularly alterations in the AR, cellcycle genes, MYC, and TP53 genes, said Joshi Alumkal, MD, the Leader of theProstate/Genitourinary Medical Oncology Section and Associate Division Chieffor Basic Research in the Hematology-Oncology Division at the University ofMichigan School of Medicine.

Severalrandomized phase 3 clinical trials now show a benefit of escalating treatmentin men with mHSPC by adding novel AR-targeting agents or chemotherapy plusmedical castration versus medical castration alone, Dr Alumkal said. Whetherthe addition of any of these specific agents to medical castration isassociated with improved outcomes in patients with poor-risk molecularalterations identified by the new study is a critical next question, he said.

Urologiconcologist James Mohler, MD, Senior Vice President for Translational Researchat Roswell Park Comprehensive Cancer Center in Buffalo, New York, said the new study found relativelysmall differences among the clinical phenotypes, but that is not surprisingbecause the temporal differences in the evolution of tumor biology occur overlong periods of time, much of which precedes clinical presentation. The hazardratios for association between mutational analysis and oncologic outcome insome cases were statistically significant, but are so small as to not beclinically significant. Part of the reason for this may be that prostatecancer, once metastatic, is so complex that no single mutation or single genepathway is driving growth and hence targetable at a high rate beyond the long provenbenefit from androgen deprivation therapy, Dr Mohler said.

The results reported by these authors may be disappointing to many clinicians, but are important because they represent a comprehensive analysis of mCSPC. The authors appropriately acknowledge that better tumor sampling and more comprehensive genetic analysis and larger numbers of patients may be required to find any benefit to genomic or somatic sequencing, Dr Mohler said. I am afraid that these limitations are not just of their work but a biological limitation of aggressive prostate cancer, which makes improving treatment of advanced prostate cancer in an individual patient extremely challenging.

Reference

Stopsack KH, Nandakumar S, Wibmer AG, et al. Oncogenic genomic alterations, clinical phenotypes, and outcomes in metastatic castration-sensitive prostate cancer [published online March 27, 2020]. Clin Cancer Res. doi: 10.1158/1078-0432.CCR-20-0168

This article originally appeared on Renal and Urology News

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Genomic Alterations Linked to Outcomes in mCSPC - Cancer Therapy Advisor

New knowledge derived from the study by NUS researchers opens up opportunities for personalised treatment against aggressive head and neck, and lung cancers in Asian population

Researchers from the Cancer Science Institute of Singapore (CSI Singapore) at the National University of Singapore (NUS) have uncovered a genetic variant in a gene called MET that is responsible for more aggressive growth of head and neck, and lung cancers. A further probe into the finding revealed therapeutic strategies that could potentially target this genetic alteration, thereby paving the way for clinicians to develop better and more effective treatments for cancer patients of such profile.

The study, published in the prestigious scientific journalNature Communicationson 25 March 2020, was conducted in close collaboration with clinicians from the National University Cancer Institute, as well as researchers from the National Cancer Centre Singapore and the Bioinformatics Institute at the Agency for Science, Technology and Research, Singapore.

The MET gene encodes for a cancer-promoting protein that relays growth, survival and transmission of signals in cancer cells. In the study led by Professor Goh Boon Cher and Dr Kong Li Ren from CSI Singapore, the team of researchers identified a form of MET protein, which showed ethnic preference with a higher incidence among Asians, and is associated with poorer prognosis in patients diagnosed with head and neck squamous cell carcinoma or lung squamous cell carcinoma. Even though the MET variant does not seem to predispose an individual to cancer, it leads to the more aggressive growthof cancers that have already developed.

Unlike other MET mutants, this genetic variant also does not appear to be inhibited by existing MET-blocking drugs that have been developed and approved in the clinical setting, prompting the researchers to conduct further investigation on the mechanism behind the genetic alteration.

Leveraging the research teams multi-disciplinary expertise and state-of-the-art molecular modelling, the team found that the single amino-acid change in the MET receptor from the genetic alternation leads to preferential strong binding to another cancer-promoting protein, HER2. Both proteins then work cooperatively to drive cancer aggression and enable cancer cells to survive therapies involving MET-blocking drugs.

The mechanism of this MET variant is novel and unreported. This finding contributes to the growing evidence of the role of genetic variants in affecting clinical outcome, and underscores the importance of diving deep into our genetic inheritance in cancer research, said Dr Kong, Research Fellow at CSI Singapore who initiated the study.

Knowledge of this unique mechanism also facilitated the team in identifying several HER2 inhibitors capable of blocking cancer progression caused by this genetic alteration using laboratory models.

Prof Goh, Deputy Director and Senior Principal Investigator at CSI Singapore, said, Our study represents a conceptual advancement to cancer research, as we have shown that it is possible to block the activity of a cancer-driving gene by administrating a targeted therapy directed not against the mutant protein in question, but rather, a corresponding protein with which it binds to. The remarkable anti-tumour responses observed in our experimental models, coupled with the availability of FDA-approved HER2 inhibitors also presents a huge opportunity for clinicians to improve the disease outcome of this genetic alteration via precision medicine.

The research team is now translating the findings to a clinical trial where patients tested positive for this MET variant gene are treated with suitable medications that have shown effectiveness in the laboratory

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Singapore uncovers cancer-causing hereditary mutation in Asians - BSA bureau

The next frontier for university technology transfer will likely be in the transformation of data-rich sectors using artificial intelligence (AI) and machine learning technologies. One area largely accumulating data is the healthcare sector. Medical knowledge is doubling every 73 days, yet we are barely scratching the surface of utilizing this data.

Over $71 billion USD was spent in federally sponsored research at universities in 2018 in the US alone. Approximately $2.94 billion in licensing revenue was generated in 2018 directly from the process of taking academic inventions to market, otherwise known as technology transfer (TT). Including federal laboratories, the US invests more than $100 billion each year in federal research funding, with a cumulative spending of more than a trillion dollars over the last 15 years. In this article, we report trends in academic TT from surveys and literature and provide perspective on the future directions of the field.

Figure 1. This dashboard shows the metrics reported by AUTM for 2018, exhibiting the impact of technology transfer in the US.

There have been several high-profile hot takes on academic TT in the past several years, claiming that by-the-numbers TT is a failing endeavor. Leaders of TT argue, with merit, that this criticism misses the point and overlooks the fact that TT is able to improve the public good through commercializing federally funded inventions. We agree with the latter viewpoint and examine TT numbers to shed more light into this field. University administrations and other stakeholders evaluate technology transfer offices (TTO) via 1) revenue generated, 2) licenses executed, 3) startups created, 4) invention disclosure forms (IDF) received, and 5) patents issued. In this piece we examine AUTMs STATT survey data that measures these indicators for all years in which most data is available (20082018) and provide context and graphics of interest.

While revenue is a cornerstone performance indicator in business, its use as an indicator of performance is a touchy subject for TTOs. Only a small proportion of the total sales from the licenses goes back to the university TTO. For example, consider a university TTO with a $10 million annual budget: according to a survey conducted by AUTM, the median royalty received by universities is approximately 2% of product sales. The TTO typically receives around 25% of those royalty revenues that is received by a university. Therefore, for the TTO to break even the university needs to receive $40 million in royalties, which requires $2 billion in cumulative sales for the companies licensing university IP! That is a lofty goal and most universities fall short. The exceptions are unicorns, where single licenses earn the university hundreds of millions (e.g., Lyrica from Northwestern brought $1.4 billion). This is why revenues do not tell the whole story of academic technology commercialization; most revenues are generated by a miniscule subset of licenses. Yet, STATT reports $2.94 billion in total revenues in 2018. Assuming the median 2% royalty rate, that implies about $147 billion in sales of products derived from academic technology. Not bad in terms of ROI from $71 billion. In line with the spirit of the Bayh-Dole Act of 1980, another measure of efficiency for the TTOs are the number of licenses.

Figure 2. The top 20 individual universities with the most gross licensing revenue compared with the number of executed licenses from 20082018 according to STATT data. Three university systems had high revenue number but are excluded because they dont report individual statistics: The University of California System with $1,513,052,284 in revenue and 2448 licenses, The University of Texas System with $694,903,592 in revenue and 1601 licenses, and The University of Massachusetts System with $387,504,344 in revenue and 280 licenses. Missing data: City of Hope NMC (201415), Princeton University (200810).

It is no surprise that there is a positive correlation between patents issued and licenses executed (and startup creation and revenues earned). Despite this, the federal government disallows any of the $71 billion in research funding to be spent on filing patents to protect inventions. Yet, in 2018, universities spent over $425 million in patent-related legal expenses. Anyone who has worked at a TTO can attest to the challenge of managing a portfolio of patents on a shoestring budget. Patents are expensive and there are never enough funds to file for patents on all of the inventions that warrant protection. As a result, more than 95% of foreign rights are never protected, and less than 30% of patents are ever converted to a full patent from the provisional filing. This problem is compounded by other challenges including the inability to protect some gene-based and software inventions through patenting, which has an outsized effect on academic research institutions.

Figure 3. The top 20 individual universities with the most patents issued from 20082018 compared with legal expenditures according to STATT data. Two university systems had high numbers of patents issued but are excluded because they dont report individual university statistics: University of California with 4335 patents issued and $ $426,655,283 in legal fees and The University of Texas System with 1883 patents issued and $99,084,203 in legal fees. Missing data: University of South Florida (2009).

In recent years there has been a paradigmatic shift towards commercializing technology through startups. There is a universal understanding that university inventions are in early technology readiness level and need substantial development to be ready to go to market. Many universities have taken it upon themselves to fund some of the startups, sometimes co-funding alongside venture funds. The trend of the top-20 universities for initiated startups (20082018) provides several trends of interest. As expected, the Silicon Valley, Cambridge and other mature startup ecosystems create a huge number of startups. Yet other noteworthy regions have built strong reputations for startup creation as well. Factors that influence the rankings include 1) access to funding, 2) access to entrepreneurs, and 3) an ecosystem of other startups and similar companies. Critical factors that enhance startup success is the stability of the parent TTO (e.g., Columbia University), leadership and support from the university (e.g., Purdue University), support from the alumni (e.g., University of Florida and University of Michigan), and size of available research funding (e.g., University of Washington and University of Pennsylvania).

Figure 4. The top 20 universities with the most startups initiated from 20082018 according to AUTM STATTs database. Four university systems had high numbers of startups issued but are excluded because they dont report individual university statistics: University of California System with 785 startups, University of Texas System with 277 startups, University System of Maryland with 121 startups, and the Research Foundation of SUNY with 108 new startups. Missing data: University of Colorado (2014), University of Utah (2016).

Although the total number of startups from the academic world is very small compared to the overall national numbers, the impact of these spinouts on the economy is substantial. Stanford University alone originated giants including Google, Cisco, and HP. Survival rates, funds raised, and successful exits are all higher among university/alumni-based startups that are based on university-licensed technology. Furthermore, in addition to high technology, startups from universities include a large number of impactful biotechnology and gene therapy companies, such as Juno Therapeutics (Fred Hutchison Cancer Research Center, Memorial Sloan-Kettering Center, and Seattle Childrens Research Institute). When Juno sold to Celgene for $9 billion, the primary investor in the company (ARCH Ventures) received 23x return on their 15% stake. Endocyte Inc. from Purdue University (acquired by Novartis AG for $2.1 billion) and AveXis from Nationwide Childrens Hospital and Ohio State University (acquired by Novartis AG for $8.7 billion) are other notable examples. While the dollar figures might be astonishing for some of these exits, the impact of these life science companies over the last four decades has also been enormous. A study by Dr. Ashley Stevens et. al. of Boston University found 153 new drugs and vaccines were developed by public research institutions between 1981 and 2011.

Figure 5. The number of startups more than tripled in 20 years, from 306 in 1998 to almost 1098 in 2018. This can be attributed to a sustained effort by universities to incubate early-stage technologies through startups.

If patents are the finished goods of university technology commercialization, then invention disclosures are the raw materials that must first be processed and refined. We examined the cumulative number of invention disclosures between 20062017 at these universities and found, as would be expected, that the universities with the highest numbers of IDFs also have the highest number of patents, with several notable exceptions. University of South Florida, Northwestern University, NYU and USC have high patent numbers although they are not in the top 20 for IDFs. On the other hand, University of Minnesota, University of Pittsburgh, and Ohio State University, do not appear on the top 20 for patents although they are in the top 20 for IDFs. We analyzed the IDFs from 2006-2017 because we wanted to comparethose numbers to the total number of publications from those institutions using the Leiden CWTS publication data which displays total number of publications from 20062017. The most startling observation was that the number of IDFs were largely predicted by the number of publications in a 1:10 ratio. The outliers were Harvard University and University of Michigan.The smallest ratios were from engineering-focused California Institute of Technology, Massachusetts Institute of Technology, Georgia Institute of Technology, and Purdue University.

A challenge faced by all university TTOs is the huge number of research publications describing inventions that are not reported as IDFs. As we state above, most TTOs do not have the budget to file patent applications on every promising IDF. This is discouraging for faculty who in turn do not report subsequent inventions. There are also faculty members who do not believe in filing patents and want their research to remain in the public domain. These factors are perhaps the biggest opportunity in the field of technology commercialization; if the research is not disclosed, then less intellectual capital reaches the market.

Figure 6. The top 20 universities with the most invention disclosure forms (IDFs) (green) and number of publications (orange) for 20062017. Note that hospital publications are not tracked by Leiden and they were excluded, as were the university systems University of California with 18636 IDFs, University of Texas with 7293 IDFs, Research Foundation of New York with 3409, and University System of Maryland with 2742 IDFs. Publication numbers were not available for the Mayo Foundation of Medical Education and Research and the Massachusetts General Hospital.

In the early days of TT, universities could not have predicted how this profession would change the face of the US economy and the impact it would have overall. Consider the following revolutionary changes that have taken place at universities and TTOs:

Dr. Dipanjan DJ Nag is the Chief Investment Officer at Ventech Solutions, a healthcare technology company that manages quality data for the Center for Medicare and Medicaid Services (CMS). He has successfully led Ohio State University, Rutgers University and University of Nebraskas technology transfer operations that included licensing, startup and investments. As an entrepreneur he led a number of start-ups in the intellectual property strategy, artificial intelligence, and medical device space. As a consultant in patent monetization and intellectual property strategy he has worked with many Fortune 500 companies, universities, and national governments. He was a Director of Ocean Tomo and a Vice President at ICAP Ocean Tomo leading patent transaction markets. He was recognized as one of the top IP strategists by IAM300 in 2019. DJ was on the Board of AUTM from 2012-14 focused on educating the members around world the importance of technology transfer and intellectual property. He is widely recognized as a global intellectual property strategist working with government and universities in Poland, Japan, India, Turkey, Brazil, South Korea, Ukraine and many other countries. Currently he teaches intellectual property strategy and negotiations as a Professor of Practice at Rutgers University and a Visiting Professor at Shizuoka University. He volunteers as the first Executive-in-Residence at the Dublin City Schools leading a startup academy for high school students and serves on the foundation board at the Dublin Methodist Hospital.

Antara Gupta graduated from Case Western Reserve University in 2017 with a bachelors degree in Finance with a minor in Economics. She acquired her Series 7 license in 2017 before she began a financial analyst job at Sycamore Growth Group. She was a financial analyst calculating R&D tax credits, job creation tax credits, and training grants for manufacturers. She worked alongside the president of the company communicating with current and potential clients. Antara then went on to Ventech Solutions, Inc as the Strategic Venture Analyst in March of 2019. She provided due diligence, market analysis and IP landscape reports for the leadership team. In the beginning of 2019, Antara began a volunteer analyst position at the Dublin City Schools helping prepare the financial projections and structure for the Entrepreneurship Program for High School students. Antaras main passions are in the field of finance, entrepreneurship, education, economic development, and intellectual property analysis.

Alex Turo is the Intellectual Property Fellow at Ventech Solutions, a healthcare technology company that manages quality data for the Center for Medicare and Medicaid Services (CMS). He has worked in technology commercialization and business development at The Ohio State University where he is also a graduate research fellow in life sciences. He is a subject matter expert in biotechnology and bioinformatics with a masters degree from Binghamton University (SUNY) and 7 years of experience in the analysis of next-generation sequencing data.

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The Evolution of University Technology Transfer: By the Numbers - IPWatchdog.com