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3 Revolutionary Stocks I’d Buy Right Now Without Hesitation – The Motley Fool

Regeneron Pharmaceuticals (REGN 0.60%), Vertex Pharmaceuticals (VRTX 1.08%) and Biogen (BIIB 1.19%) are developing remarkable therapies that, instead of merely treating disorders, have the potential to cure them or at least make an impact that no other drug has to date.

At the same time, all three companies are profitable, and their successes in the lab have been paying off in the market, with their shares up nearly 10% this year. Let's see why.

Regeneron's shares are up about 10% this year, despite a second quarter that saw its revenue fall 44% year over year. The reason that's not a big cause for alarm is that the company's second-quarter revenue of $2.86 billion would be up 20% over the same period in 2021 if its COVID-19 therapy, REGEN-COV, was excluded from total revenue. The company's sales of the treatment ended late last year when government contracts for the therapy ended. Even without REGEN-COV, the company had earnings per share (EPS) of $7.47 in the quarter.

The biotech company's revolutionary concept is its process of using genetic research to develop specific antibodies that bind to two different antigen proteins. The company has medicines that treat atopic dermatitis, high cholesterol, retinal disease, prostate cancer, Ebola, rheumatoid arthritis, certain skin cancers, and non-small cell lung cancer. That doesn't include the 35 therapies it has in its pipeline.

It just reported positive data for its phase 1 study on ALN-HSD, which it is developing with Alnylam Pharmaceuticals to treat non-alcoholic steatohepatitis (NASH), a form of fatty liver disease which can lead to liver failure, cancer, and death.

Its current blockbuster drug, Dupixent, is approved alone or paired with other drugs to treat atopic dermatitis, asthma, eosinophilic esophagitis (a chronic immune system disorder), and chronic rhinosinusitis with nasal polyps. In the second quarter, it brought in $2.09 billion, up 40% year over year. Overall, the company reported $2.86 billion in revenue and earnings per share of $7.47.

Regeneron is well on its way to developing another blockbuster drug. Eylea (aflibercept), an anti-blindness treatment the company is developing with Bayer, showed effectiveness and safety at high dosages in late-stage trials. The potential label expansion would help a drug that brought in $1.6 million in the second quarter, up 14% over the same period last year.

BIIB data by YCharts.

Vertex Pharmaceuticals' shares are up more than 33% this year. That may be due to several late-stage-trial successes that have investors excited. Most prominent is Exa-cel (CTX001), which the company is developing with CRISPR Therapeuticsand is seen as a revolutionary potential cure for two genetic blood disorders -- transfusion-dependent thalassemia and severe sickle cell disease.

It's also expecting big things this year from VX-880, a stem-cell treatment to cure type 1 diabetes that is in Phase 1/2 trials.

Vertex is already profitable, thanks to cystic fibrosis drug Trikafta. In the second quarter, the company reported product revenue of $2.2 billion, up 22% year over year. The company reported EPS of $3.13 compared to $0.26 in the same period a year ago.

Trikafta alone was responsible for $1.89 billion in sales in the quarter, up 50.7%, year over year. The company raised its full-year revenue guidance to be between $8.6 billion to $8.8 billion, compared to $7.5 billion last year.

Biogen, founded in 1978, is one of the older biotech companies. It focuses on fighting neurological disorders and has a diverse portfolio. The company's stock is up some 36% over the past month, due to a promising Phase 3 trial for Lecanemab, an Alzheimer's drug that Biogen is developing with Japanese pharmaceutical company Eisai. Biogen said that the drug, after 18 months, slowed cognitive decline in Alzheimer's patients by 27%, compared to a placebo, based on the Clinical Dementia Rating Sum of Boxes scale (CDR-SB).

It's the second bite of the apple for Biogen, as another Alzheimer's drug it developed with Eisai, Aduhelm, had mixed clinical trials. Though Aduhelm was approved by the FDA, The Centers for Medicare and Medicaid Services said it would allow Medicare to pay for Aduhelm treatment only for Alzheimer's patients enrolled in qualifying clinical trials. As a consequence, Biogen hasn't been able to profit much from the drug.

Finding therapies that help slow the progression of Alzheimer's has been difficult because many drugs are rendered ineffective by the blood-brain barrier. Unlike Aduhelm and other Alzheimer's therapies, Lecanemab is the first to show a strong connection between reducing amyloid-related plaque in the brain associated with Alzheimer's and slowing the progression of the disease.

Biogen is already on solid financial ground to market Lecanemab. While revenue in the second quarter was a reported $2.589 billion, down 6% year over year, the company had EPS of $7.24, compared to EPS of $2.99 in the same period in 2021. Biogen also released improved guidance based on better top-line performance and cost-cutting measures. It now expects annual revenue of between $9.9 billion to $10.1 billion, up from an earlier range of between $9.7 billion to $10 billion. It also raised non-GAAP (adjusted) EPS from a range of $14.25 to $16.00 up to an estimate $15.25 to $16.75.

Jim Halley has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Alnylam Pharmaceuticals, CRISPR Therapeutics, and Vertex Pharmaceuticals. The Motley Fool recommends Biogen. The Motley Fool has a disclosure policy.

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3 Revolutionary Stocks I'd Buy Right Now Without Hesitation - The Motley Fool

Its a Shame to See Him, How He is Today: Tyson Furys Father Calls Out Mike Tyson Following Deteriorating Health Condition – EssentiallySports

Former world heavyweight champion Mike Tyson was one of the greatest athletes to ever step foot inside the boxing ring. Tysons iconic performances inside the ring and eccentricity outside the ring have only further elevated his status as one of the Titans of boxings very own Tartarus. Meanwhile, Tyson Furys father, John Fury, had a few choice words to say about Tyson. Especially with regard to him undergoing stem cell treatments.

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Fury was in an interview JOE. And, he was talking about his desire to fight Tyson. In conversation, he was initially all praise for the legendary heavyweight. Fury said, Mike Tyson was tricky. He had plenty of movement. Plenty of head movement. He was in and out quick feet. And, he had bombs in each hand and a great fighter.

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Tyson had previously undergone stem cell treatments in preparation for his return to boxing. Talking about the same, Fury stated, You know its a shame to see him, how he is today. You with all you hear about stem cells, blood transfusions to make him younger. Whatever they call them. You know what Im saying? But hes a great fella and I do like him.

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What Good Is Money if Youre Dead?: Fans Go Wild as Ominous Mike Tyson Pic Goes Viral on the Internet

2 days ago

However, Fury added that he likes Tyson and that he would be honored to step inside the ring with the legend. Moreover, it is worth mentioning that Furys statement came at a time when Tysons health and well-being were under scrutiny.

Mike Tyson retired from professional boxing in the year 2005. And he completely moved away from the sport for almost 15 years, even gaining weight in the process.

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Tyson was in conversation with NBA legend Shaquille ONeal. And this was when he opened up about undergoing stem cell treatments. Talking about the same, he said, I feel like a different person, but I cant comprehend why I feel this way. Its really wild what scientists can do.

He mentioned that his body was badly out of shape and that the treatment has been helping him return to the sport. However, it is not known which condition he has been treating with stem cell therapy.

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WATCH THIS STORY Movies and Documentaries About Mike Tyson Every Fan Must Watch

What are your thoughts on Furys statement? Do share in the comments below.

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Its a Shame to See Him, How He is Today: Tyson Furys Father Calls Out Mike Tyson Following Deteriorating Health Condition - EssentiallySports

Is There Pet Insurance With No Waiting Period? – MarketWatch

In this article: Purpose of a Waiting Period | Pet Insurance With No Waiting Period | Typical Waiting Period | Top Companies With Short Waiting Periods | FAQs

When you buy pet insurance, youll need to wait before coverage begins. Accidents, injuries, and symptoms of illnesses or conditions during this waiting period are considered preexisting and arent covered under your policys terms. If you want this waiting period to be as short as possible, these are the best pet insurance companies for you.

Pet insurance is intended to cover health conditions that may happen in the future, not ones that have already occurred. Pet insurance companies would lose money if they insured pets with known expensive health problems, which is why none cover preexisting conditions.

Since most providers dont require your pet to get a health exam before enrolling in coverage, waiting periods are meant to prevent you from noticing symptoms or injuries, quickly signing up for insurance, and then immediately receiving veterinary care paid for by the company. Essentially, the waiting period allows pet owners to enroll in a policy quickly while keeping people from gaming the system.

Nearly all pet insurance plans have a waiting period, though some may have shorter periods for accident-only coverage. A few emergency-only policies dont fit the standard insurance mold and have no waiting periods, but the coverage is often minimal.

The typical waiting period is 14 days, particularly for illness coverage. Some pet insurance providers have a waiting period of only a few days for accident coverage. Hereditary conditions such as hip dysplasia typically have much longer waiting periods up to a year. If your pet develops symptoms of a health problem within that time frame, expenses for that health problem will not be covered whether a vet provides treatment or not.

Here are the pet insurance companies with the shortest waiting periods.

Lemonade offers comprehensive coverage and multiple add-ons at more affordable rates than many providers on this list. It also has a very short waiting period for accident coverage of only 48 hours.

+ Waiting period for accidents is only 48 hours

+ Lower monthly premiums than many competitors

+ Add-on option for preventive care

Requires a recent vet exam to qualify

No coverage for pets older than 14

Lemonade offers one pet insurance plan covering costs related to accidents and illnesses, such as diagnostic tests, hospitalizations, surgery, cancer treatments, broken bones or sprains, prescription medications and more. You can add coverage for wellness exams, vaccines, bloodwork and physical therapy treatments.

To learn more: Lemonade Pet Insurance review

Get your quote: Fill out Lemonades online quote form

A newcomer to the pet insurance industry, Spot offers highly customizable plans, including annual limits from $2,500 to unlimited. Although its 14-day waiting period is average, it applies to knee and ligament conditions, which often have much longer waiting periods.

+ Option for unlimited annual coverage

+ 14-day waiting period for knee and ligament coverage

+ 10% multi-pet discount

Accident coverage doesnt start for 14 days

No customer service on weekends

Spot offers two plans: one for accident-only coverage and one for accidents and illnesses. It also has two wellness add-ons for preventive care.

To learn more: Spot Pet Insurance review

Get your quote: Fill out Spots online quote form

Trupanion offers unlimited coverage caps and a range of deductibles from $0 to $1,000 to customize your plan. Its five-day waiting period for accidents and injuries is shorter than many providers. Its 30-day waiting period for illnesses and other conditions is longer than average, but it applies to joint problems such as cruciate ligament and hip dysplasia.

+ Covers hereditary and congenital conditions

+ Pays veterinarian directly

+ No payout limits

30-day waiting period for illnesses

No option for wellness or preventive care

Trupanion offers one comprehensive accident-and-illness plan. It also covers conditions that can occur outside of an accident or illness, such as congenital and hereditary conditions and other undiagnosed issues. It includes add-on coverage for several unique items, such as alternative treatments, advertising for lost pets, boarding fees and breeding costs.

To learn more: Trupanion review

Get your quote: Fill out Trupanions online quote form

Fetch Pet Insurance, formerly PetPlan, only offers one accident-and-illness plan. However, the plan includes coverage for things many providers dont cover, such as holistic care, stem-cell therapy and treatment related to behavioral problems such as separation anxiety or aggression.

+ Offers coverage for some nonmedical expenses

+ Covers alternative and complementary treatments

+ Healthy Pet Credit discount for 12 years without filing a claim

No multi-pet discount

No add-on for wellness coverage

Fetch offers one accident-and-illness plan. It doesnt have any add-on options.

To learn more: Fetch by The Dodo Pet Insurance review

Get your quote: Fill out Fetchs online quote form

Embrace provides pet parents with multiple opportunities to save, including a 10% discount for each additional pet policy you purchase, a 5% military discount and a 10% discount for employees of companies that offer Embrace pet insurance. It also provides a Healthy Pet Deductible benefit that credits $50 toward your co-payment each year you dont file a claim.

Although Embrace has a six-month waiting period for orthopedic conditions, you can apply for the Orthopedic Exam and Waiver to reduce this time frame. The waiting period for accident coverage is only two days.

+ Option to add coverage for routine care such as vaccinations

+ Diminishing deductible for years without filing claims

+ Coverage for working dogs (racing, guarding, etc.)

Low dollar limit for wellness coverage

No coverage for prescription food or supplements

Embrace offers one accident-and-illness plan plus an add-on for preventive care.

Note: The Wellness Rewards plan is not available in Rhode Island.

To learn more: Embrace Pet Insurance review

Get your quote: Fill out Embraces online quote form

Most companies max out their reimbursement rate at 90%, but Figo offers up to 100% reimbursement for claims. Its one-day waiting period for accidents and injuries is also the lowest in the industry. You can apply for a waiver to get around the six-month waiting period for orthopedic conditions.

+ Offers 100% reimbursement option

+ Shortest waiting period for accidents

+ Extra Care Pack covers end-of-life costs, boarding, theft and more

Coverage for exam fees requires add-on

High deductibles for older pets

Figo offers one accident-and-illness plan with three tiers of coverage and annual limits. It also has a range of add-ons for items such as preventive care and liability coverage for damages or injuries your pet causes.

Get your quote: Fill out Figos online quote form

Pets Best stands out for its coverage of older pets. Unlike some competitors, it has no age limit for coverage, meaning you can enroll your pet anytime. It also offers coverage for items more likely to be required by aging pets, such as prescribed prosthetic devices and wheelchairs.

Pets Best has an average waiting period, but if you add one of the two routine care plans, you can use your pet insurance policy for wellness care immediately.

+ Highly customizable plans

+ Vet Direct Pay reimburses veterinarians directly

+ Covers prosthetic devices and wheelchairs

Exclusions for parasites, behavioral treatment and prescription food

Illness coverage not offered to pets with severe chronic conditions

Pets Best offers an accident-only plan, three levels of accident-and-illness coverage and two routine care add-ons.

Unfortunately, all pet insurance providers have waiting periods. The standard waiting period is 14 to 15 days for accidents and illnesses. Many of the companies listed above offer shorter-than-average waiting periods for accidents or orthopedic conditions. Although the waiting period shouldnt be the only factor you take into account, it may be important to consider while shopping for pet insurance.

Our review of pet insurance companies is based on in-depth industry research that includes reading hundreds of customer reviews, simulating the quote and purchasing process, speaking to representatives on the phone to assess the customer service experience and surveying 1,000 dog and cat owners nationwide to determine the most important elements of pet insurance coverage. We have scored each provider on a 100-point scale based on those elements.

Here are more details about each factor and how theyre weighted:

We use our rating system to compare and contrast each company against key factors to help us determine the best pet insurance companies in the industry. Additionally, we keep our research up to date and revisit our reviews on a regular basis.

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Is There Pet Insurance With No Waiting Period? - MarketWatch

Century Therapeutics Announces Appointment of Daphne Quimi and Timothy Walbert to its Board of Directors – Yahoo Finance

Century Therapeutics, Inc.

PHILADELPHIA, Oct. 03, 2022 (GLOBE NEWSWIRE) -- Century Therapeutics (NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology, today announced the appointment of Daphne Quimi and Timothy Walbert to the Companys Board of Directors. Ms. Quimi is currently Chief Financial Officer of Amicus Therapeutics and brings experience in public accounting and financial reporting to Century. Mr. Walbert is currently Chairman, President, and Chief Executive Officer of Horizon Therapeutics, and brings expertise in product portfolio building and commercialization. In conjunction with these new appointments, Century also announced that Eli Casdin, Chief Investment Officer of Casdin Capital, has resigned from the Board of Directors, effective as of October 1, 2022.

I am thrilled to welcome both Daphne and Tim to our Board. They will each play critical roles as we accelerate our next-generation cell therapy platform, said Lalo Flores, Ph.D., Chief Executive Officer, Century Therapeutics. Daphnes strong financial background and experience at both biotechnology and large pharmaceutical companies will be a tremendous asset as we enter the next transformative years for Century. Tims background, which includes numerous product launches, will be instrumental to our continued evolution, particularly as we progress our pipeline candidates with the ultimate goal of delivering innovative cancer therapies. Additionally, on behalf of the management, Board and all of our employees, we would like to thank Eli for his contributions to Centurys rapid growth and his leadership in our early formative years, where he was a key strategic thought partner.

Before serving as Amicuss Chief Financial officer, Ms. Quimi was Amicuss Senior Vice President, Finance and Corporate Controller. Ms. Quimi is currently a member of the Board of Directors at Amylyx Pharmaceuticals. Prior to Amicus, Ms. Quimi served as Director of Consolidations and External Reporting at Bristol-Myers Squibb. She also held roles of increasing responsibility in the finance department at Johnson & Johnson. Earlier in her career she worked for KPMG. Ms. Quimi received a B.S. in Accountancy from Monmouth University and an M.B.A from the Stern School of Business of New York University.

In addition to his current role of President and Chief Executive Officer of Horizon Therapeutics, Mr. Walbert has served as Chairman of Horizons Board of Directors since 2010. Before joining Horizon, Mr. Walbert served as President, Chief Executive Officer and Director of IDM Pharma Inc., and also held prior senior roles at NeoPharm Inc., Abbott (AbbVie), G.D. Searle & Company, Merck & Co. Inc. and Wyeth. Mr. Walbert received a B.A. in Business from Muhlenberg College.

About Century Therapeutics

Century Therapeutics (NASDAQ: IPSC) is harnessing the power of adult stem cells to develop curative cell therapy products for cancer that we believe will allow us to overcome the limitations of first-generation cell therapies. Our genetically engineered, iPSC-derived iNK and iT cell product candidates are designed to specifically target hematologic and solid tumor cancers. We are leveraging our expertise in cellular reprogramming, genetic engineering, and manufacturing to develop therapies with the potential to overcome many of the challenges inherent to cell therapy and provide a significant advantage over existing cell therapy technologies. We believe our commitment to developing off-the-shelf cell therapies will expand patient access and provide an unparalleled opportunity to advance the course of cancer care. For more information on Century Therapeutics please visit http://www.centurytx.com.

Century Therapeutics Forward-Looking Statement

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, The Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as may, might, will, should, expect, plan, aim, seek, anticipate, could, intend, target, project, contemplate, believe, estimate, predict, forecast, potential or continue or the negative of these terms or other similar expressions. These statements are not guarantees of future performance These risks and uncertainties are described more fully in the Risk Factors section of our most recent filings with the Securities and Exchange Commission and available at http://www.sec.gov. You should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

For More Information:

Company: Elizabeth Krutoholow investor.relations@centurytx.com

Investors: Melissa Forst/Maghan Meyers century@argotpartners.com

Media: Joshua R. Mansbach century@argotpartners.com

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Century Therapeutics Announces Appointment of Daphne Quimi and Timothy Walbert to its Board of Directors - Yahoo Finance

Sysmex : and JCR Pharmaceuticals Establish a Joint Venture in the Field of Regenerative Medicine and Cell Therapy- Aiming for the Research &…

In recent years, the significant potential of regenerative medicine and cell therapy have been established in particular in areas that have traditionally been difficult to address with conventional chemically synthesized low molecular weight drugs1 or biopharmaceuticals,2 such as the restoration of tissues and functions lost as a result of aging, illness, autoimmune diseases, or cancer. In particular, research and development on the therapeutic application of stem cells including hematopoietic stem cells, mesenchymal stem cells, and iPS cells have generated significant attention.

Since its foundation, Sysmex has provided network solutions using IoT (Internet of Things) and automated testing flows to improve the safety of healthcare professionals and for the optimization of operational efficiency in clinical laboratories. In addition, while providing quality control testing for companies which develop regenerative medicine products, Sysmex has conducted research and development related to new pre-transplant compatibility testing examining the patient's immune response against the organ or tissue to be transplanted.

Since its inception, JCR has been engaged in the research, development, manufacturing and sales of pharmaceutical products using regenerative medicine, genetic engineering, and gene therapy technologies to advance therapies in the rare disease field. This is exemplified in the field of regenerative medicine, by the approval of TEMCELL HS Inj.,3 the first allogeneic regenerative medicine in Japan (Non-proprietary name: Human (allogeneic) bone marrow-derived mesenchymal stem cells) in February 2016 for the treatment of acute graft-versus-host disease (acute GVHD),4 a serious complication that develops after hematopoietic stem cell transplantation. In recent years, JCR has further streamlined and integrated its expertise around the establishment of groundbreaking medicines for the advancement of highly innovative medicines that could not be developed without such groundbreaking technologies.

In the joint venture, the two companies aim to realize the social implementation of regenerative medicine and cell therapy by integrating Sysmex's expertise in quality control testing technology and knowledge of workflows efficiency using robotics technology, including IoT, with JCR's expertise in developing, manufacturing and marketing regenerative medicine products. AlliedCel Corporation, which is the corporate name of the joint venture following prior discussions regarding the alliance both companies, was established on October 3, 2022. The joint venture will advance programs of the potential for technology development and commercialization, including the project currently being promoted by both companies using hematopoietic stem cell proliferation technology.

The name AlliedCel stands for the joint venture's aspiration to integrate knowledge and expertise from a broad set of collaborators and stakeholders including business partners, patients and their families, with the united goal of unleashing the power of cells in supporting patients in their need for life-changing therapies. Through the research and development of regenerative medicine products using diverse cells such as stem cells, AlliedCel aims to provide appropriate treatment options to patients and improve their prognosis.

Profile of the Joint Venture

Capital reserve:

100 million JPY

President: Hiroyuki Sonoda

(Vice President, Research and Corporate Strategy, Executive Director of Research Division, JCR Pharmaceuticals Co., Ltd.)

Executive Vice President, Member of the Managing Board: Kenji Tsujimoto

(Executive Vice President of Technology Strategy Division, Sysmex Corporation)

About Sysmex Corporation

About JCR Pharmaceuticals Co., Ltd.

JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is redefining expectations and expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 48-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. Our core values - reliability, confidence, and persistence - benefit all our stakeholders, including employees, partners, and patients. Together we soar.

Disclaimer

Sysmex Corporation published this content on 03 October 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 03 October 2022 06:13:01 UTC.

Publicnow 2022

Technical analysis trends SYSMEX CORPORATION

Income Statement Evolution

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Sysmex : and JCR Pharmaceuticals Establish a Joint Venture in the Field of Regenerative Medicine and Cell Therapy- Aiming for the Research &...

The Importance of Growth Factor Quality in Organoid Cultures – The Scientist

Within a decade from their first demonstration, organoids have become a quintessential tool for fundamental and biomedical research, serving as relevant models for studying human development and disease.1 Animal and classical 2D cell culture models dominated biological research during the late twentieth and early twenty-first centuries. While these models offer unique advantages for understanding cellular signaling pathways, drug action mechanisms, and disease pathologies, one system alone cannot address them all. Therefore, researchers use various systems at different stages of basic and translational research. However, assessing information from different models slows down discovery for human clinical applications.2

Further, several biological phenomena that are specific to humans do not occur in animal models. For example, several human brain cell types are not found in the rodent brain. Also, the human brains neurodevelopmental mechanisms are far more complex compared to those in rodent brains. Moreover, human brain cell physiology differs when grown in 2D cell cultures and cannot reliably predict drug responses at preclinical stages. So far, human 3D organoids are the only system poised to overcome these limitations.

Organoids are small, self-organized 3D tissue cultures derived from adult stem cells, pluripotent stem cells, or primary tissues. They offer a huge advantage over 2D cell cultures by replicating in vivo organ complexity and tissue architecture. Analyzing organoid formation and function provides valuable information about human development, tissue formation, and organ function as well as a direct tool for pharmaceutical drug testing.

Organoid culture overview

Sino Biological

Researchers generate organoid cultures using multipotent or pluripotent stem cells in a 3D matrix, such as Matrigel, under self-organization promoting conditions. The composition of the growth medium is critical for the health and success of organoid cultures, and is typically enhanced with essential growth factors, such asepidermal growth factor (EGF),noggin (NOG), R-spondin (RSPO1),human growth factor (HGF), bone morphogenetic proteins (BMP), andfibroblast growth factor (FGF). These growth factors activate critical signaling pathways in the organoids to drive development or differentiation. Each culture type requires a distinct set of growth factors. For example, human colon organoids require EGF, NOG, and RSPO1 factors for differentiation, whereas human liver organoids need EGF, NOG, FGF10, and BMP7 for differentiation.2

Manufacturers produce these growth factors in eukaryotic expression systems to obtain structurally and functionally viable products. However, this process creates impurities, including secreted proteins and growth factors from the expression cells and their culture medium, affecting organoid survival and growth. Moreover, different cell lines create batch-to-batch variability in growth factor activity, influencing accuracy and reproducibility in organoid cultures.2

Sino Biological developed a host of recombinant growth factors necessary for organoid culture, including human EGF, NOG, RSPO1, and HGF among many others. These pure factors show high bioactivity, minimal endotoxin contamination, and batch-to-batch consistency, enabling optimal and consistent organoid growth.2 For example, using gel electrophoresis and high-performance liquid chromatography, the manufacturer determined the human EGF purity to be more than 95 percent. The activity of the recombinant human EGF was demonstrated by a cell proliferation assay using BALB/c 3T3 mouse embryonic fibroblasts, and the ED50 for this effect was typically 0.02-0.2 ng/mL. In addition, the purified growth factors are proven to promote or inhibit key pathways for proper stem cell development at low concentrations.2

Overall, Sino Biologicals purified growth factors effectively supplement organoid cultures, allowing them to be a trustworthy tool for researchers working to understand human development and disease.

References

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The Importance of Growth Factor Quality in Organoid Cultures - The Scientist

Growth in Cell and Gene Therapy Market – BioPharm International

Biopharma focuses on streamlining biomanufacturing and supply chain issues to drive uptake of cell and gene therapies.

Cell and gene therapies (CGTs) offer significant advances in patient care by helping to treat or potentially cure a range of conditions that have been untouched by small molecule and biologic agents. Over the past two decades, more than 20 CGTs have been approved by FDA in the United States and many of these one-time treatments cost between US$375,00 and US$2 million a shot (1). Given the high financial outlay and patient expectations of these life-saving therapies, it is essential that manufacturers provide integrated services across the whole of the supply chain to ensure efficient biomanufacturing processes and seamless logistics to reduce barriers to uptake.

The following looks at the who, what, when, and why of biomanufacturing and logistics in CGTs in the bio/pharmaceutical industry in more detail.

According to market research, the global gene therapy market will reach US$9.0 billion by 2027 due to favorable reimbursement policies and guidelines, product approvals and fast-track designations, growing demand for chimeric antigen receptor (CAR) T cell-based gene therapies, and improvements in RNA, DNA, and oncolytic viral vectors (1).

In 2020, CGT manufacturers attracted approximately US$2.3 billion in investment funding (1). Key players in the CGT market include Amgen, Bristol-Myers Squibb Company, Dendreon, Gilead Sciences, Novartis, Organogenesis, Roche (Spark Therapeutics), Smith Nephew, and Vericel. In recent years, growth in the CGT market has fueled some high-profile mergers and acquisitions including bluebird bio/BioMarin, Celgene/Juno Therapeutics, Gilead Sciences/Kite, Novartis/AveXis and the CDMO CELLforCURE, Roche/Spark Therapeutics, and Smith & Nephew/Osiris Therapeutics.

Many bio/pharma companies are re-considering their commercialization strategies and have re-invested in R&D to standardize vector productions and purification, implement forward engineering techniques in cell therapies, and improve cryopreservation of cellular samples as well as exploring the development of off-the-shelf allogeneic cell solutions (2).

The successful development of CGTs has highlighted major bottlenecks in the manufacturing facilities, and at times, a shortage of raw materials (3). Pharma companies are now taking a close look at their internal capabilities and either investing in their own manufacturing facilities or outsourcing to contract development and manufacturing organizations (CDMOs) or contract manufacturing organizations (CMOs) to expand their manufacturing abilities (4). Recently, several CDMOsSamsung Biologics, Fujifilm Diosynth, Boehringer Ingelheim, and Lonzahave all expanded their biomanufacturing facilities to meet demand (5).

A major challenge for CGT manufacturers is the seamless delivery of advanced therapies. There is no room for error. If manufacturers cannot deliver the CGT therapy to the patient with ease, the efficacy of the product becomes obsolete. Many of these therapies are not off-the-shelf solutions and therefore require timely delivery and must be maintained at precise temperatures to remain viable. Thus, manufacturers must not only conform to regulations, but they must also put in place logistical processes and contingency plans to optimize tracking, packaging, cold storage, and transportation through the products journey. Time is of the essence, and several manufacturers have failed to meet patient demands, which have significant impacts on the applicability of these agents.

Several CAR T-cell therapies have now been approved; however, research indicates that a fifth of cancer patients who are eligible for CAR-T therapies pass away while waiting for a manufacturing slot (6). Initially, the manufacture of many of these autologous products took around a month, but certain agents can now be produced in fewer than two weeks (7). Companies are exploring new ways to reduce vein-to-vein time (collection and reinfusion) through the development of more advanced gene-transfer tools with CARs (such as transposon, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) among others, and the use of centralized organization with standardized apheresis centers (5). Others are exploring the use of the of allogeneic stem cells including Regen Biopharma, Escape Therapeutics, Lonza, Pluristem Therapeutics, and ViaCord (7).

Several gene therapies have also been approved, mainly in the treatment of rare disease (8). Many companies are evaluating novel gene therapy vectors to increase levels of gene expression/protein productions, reduce immunogenicity and improve durability including Astellas Gene Therapies, Bayer, ArrowHead Pharmaceuticals, Bayer, Bluebird Bio, Intellia Therapeutics, Kystal Biotech, MeiraGTx, Regenxbio, Roche, Rocket Pharmaceuticals, Sangamo Therapeutics, Vertex Pharmaceuticals, Verve Therapeutics, and Voyager Therapeutics (8).

While many biopharma companies have established their own in-house CGT good manufacturing practice (GMP) operation capabilities, others are looking to decentralize manufacturing and improve distribution by relying on external contracts with CDMOs and CMOs such as CELLforCURE, CCRM, Cell Therapies Pty Ltd (CTPL), Cellular Therapeutics Ltd (CTL), Eufets GmbH, Gravitas Biomanufacturing, Hitachi Chemical Advances Therapeutic Solutions, Lonza, MasTHerCell, MEDINET Co., Takara Bio, and XuXi PharmaTech (6, 9, 10).

The top 50 gene therapy start-up companies have attracted more than $11.6 billion in funds in recent years, with the top 10 companies generating US$5.3 billion in series A to D funding rounds (10). US-based Sana Biotechnology leads the field garnering US$700 million to develop scalable manufacturing for genetically engineered cells and its pipeline program, which include CAR-T cell-based therapies in oncology and CNS (Central Nervous System) disorders (11). In second place, Editas Medicine attracted $656.6 million to develop CRISPR nuclease gene editing technologies to develop gene therapies for rare disorders (12).

Overall, CGTs have attracted the pharma industrys attention as they provide an alternative route to target diseases that are poorly served by pharmaceutical and/or medical interventions, such as rare and orphan diseases. Private investors continue to pour money into this sector because a single shot has the potential to bring long-lasting clinical benefits to patients (13). In addition, regulators have approved several products and put in place fast track designation to speed up patient access to these life-saving medicines. Furthermore, healthcare providers have established reimbursement policies and manufacturers have negotiated value- and outcome-based contracts to reduce barriers to access to these premium priced products

On the downside, the manufacture of CGTs is labor intensive and expensive with manufacturing accounting for approximately 25% of operating expenses, plus there is still significant variation in the amount of product produced. On the medical side, many patients may not be suitable candidates for CGTs or not produce durable response due to pre-exposure to the viral vector, poor gene expression, and/or the development of immunogenicity due to pre-exposure to viral vectors. Those that can receive these therapies may suffer infusion site reactions, and unique adverse events such as cytokine release syndrome and neurological problems both of which can be fatal if not treated promptly (14).

Despite the considerable advances that have been made in the CGT field to date, there is still much work needed to enhance the durability of responses, increase biomanufacturing efficiencies and consistency and to implement a seamless supply chain that can ensure these agents are accessible, cost-effective, and a sustainable option to those in need.

Cleo Bern Hartley is a pharma consultant, former pharma analyst, and research scientist.

BioPharm International Vol. 35, No. 10 October 2022 Pages: 4951

When referring to this article, please cite it as C.B. Hartley, "Growth in Cell and Gene Therapy Market," BioPharm International 35 (10) 4951 (2022).

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Organ on a Chip Market – Focus on Products and Technologies – Distribution by Type of Product, Application Area, Purpose, and Key Geographical Regions…

ReportLinker

INTRODUCTION It is a well-known fact that almost 90% of the therapeutic interventions fail in clinical trials, resulting in significant economic losses to the pharmaceutical industry.

New York, Sept. 30, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Organ on a Chip Market - Focus on Products and Technologies - Distribution by Type of Product, Application Area, Purpose, and Key Geographical Regions : Industry Trends and Global Forecasts, 2022-2035" - https://www.reportlinker.com/p06323408/?utm_source=GNW The lack of effective preclinical prediction of drug responses in humans is one of the various reasons for drugs failure to get approved. Animal testing for preclinical evaluation of drugs sometimes fails to identify toxicity signs caused by a drug in humans. Moreover, these studies are quite expensive, time-consuming and are associated with several ethical concerns. In order to reform the drug approval process and use non-animal testing models for preclinical evaluations, the US democrats and republicans introduced the FDA Modernization Act in 2021. The U.S. Environmental Protection Agency (EPA) has also declared the termination of the funds granted for the studies on mammals by 2035. As a result, several stakeholders have opted to modernize their conventional testing methods in order to cope up with the increasing limitations associated with animal models. One such innovative technology, organ-on-chip has the potential to transform the drug discovery process by simulating the human physiological and functional environment on a microfluidic system. The use of such novel testing models in drug discovery and toxicity testing has been steadily increasing. Up till now, several pharmaceutical manufacturers and research institutions have embraced the use of these in vivo like in vitro models; however, a remarkable rise in the adoption rate of these models has been observed since the FDA changed its laws towards putting an end on the animal testing models. , The novel organ-on-chip models have various advantages over the traditional animal-based models, including fine control over microenvironment, lower cost, lesser time, easy to use and portable. , Given the inherent benefits of organ-on-chip technology, a number of players have launched their proprietary products in order to expedite preclinical studies of novel drug interventions across a wide array of disease indications. There are several organ-on-chip models, including lung-on-chip, liver-on-chip, heart-on-chip, brain-on-chip and multiple organ models, which are being offered by various players. Apart from offering efficient user-friendly organ-on-chip models, some developers also offer customization of these models as per the client requests. It is worth mentioning that various developers have made significant efforts in developing organ-on-chip technologies, paving the way for new innovations, primarily integrating artificial intelligence driven technology for early detection of pharmaceuticals and toxicity risks, along with detection of unknown mutations. Driven by promising benefits over animal testing, increasing R&D activity and financial support from investors, the organ-on-chip market is anticipated to grow at a commendable pace in the mid to long term.

SCOPE OF THE REPORT The Organ-on-Chip Market, 2022-2035: Focus on Products and Technologies - Distribution by Type of Product (Organ(s) based Models and Disease(s) based Models), Application Area (Cancer Research, Drug Discovery and Toxicity Testing, Stem Cell Research and Tissue Engineering and Regenerative Medicine), Purpose (Research and Therapeutic Production), and Key Geographical Regions (North America, Europe, Asia-Pacific and Rest of the World): Industry Trends and Global Forecasts report features an extensive study of the current market landscape, offering an informed opinion on the likely adoption of organ-on-chip products and technologies, over the next decade. The report features an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain. In addition to other elements, the study includes: A general introduction of organ-on-chip, including history and development, classification, advantages and limitations and applications and future perspectives of organ-on-chip. A detailed assessment of the current market landscape of organ-on-chips based on a number of relevant parameters, such as type of offering(s) (chip, plate / system, and technology), type of model (organ(s) based and disease(s) based), status of development (commercialized, developed, and under development), type of technology / platform, number of chips in a plate, material used for construction of chip / plate (polymer, glass and silicon), type of polymer (polydimethylsiloxane, cyclic olefin polymer, cyclic olefin copolymer, elastomer, polycarbonate, polypropylene, polystyrene, polyester, tygon, and styrene TEP), compatible tissue / organ, and application area (cancer research, drug discovery and toxicity testing, stem cell research, and tissue engineering and regenerative medicine). In addition, the chapter provides details on the companies engaged in the development of organ-on-chip products and technologies, along with information on their year of establishment, company size and location of headquarters. Elaborate profiles of the key players developing organ-on-chips (which are presently commercialized), which are headquartered in North America, Europe and Asia-Pacific. Each profile features a brief overview of the company, its financial information (if available), organ-on-chip product portfolio, recent developments, and an informed future outlook. An in-depth analysis of various patents that have been filed / granted for organ-on-chip, till 2022, based on various relevant parameters, such as type of patent, publication year, application year, issuing authorities involved, type of organizations, emerging focus area, patent age, CPC symbols, leading patent assignees (in terms of number of patents granted / filed), patent characteristics and geography. It also includes an insightful patent valuation analysis. A detailed brand positioning analysis of the key industry players, highlighting the current perceptions regarding their proprietary products by taking into consideration several relevant aspects, such as experience of the manufacturer, number of products and technologies offered, product diversity, and number of patents published. A study of the various grants that have been awarded to research institutes engaged in projects related to organ-on-chip, between 2017 and 2022, based on parameters, such as year of award, support period, amount awarded, funding institute center, grant type, emerging focus area, type of recipient organization, key regions, and leading recipient organizations. An analysis of the partnerships that have been established since 2017, covering various types of partnerships, such as research and development agreements, clinical trial agreements, product development and commercialization agreements, technology integration agreements, and product development and manufacturing agreements of the companies focused on developing organ-on-chip products and technologies. An analysis of the investments made, including seed financing, venture capital financing, debt financing, grants, capital raised from IPOs and subsequent offerings, at various stages of development in start-ups / small companies (with less than 50 employees) and mid-sized companies (with 51-200 employees) that are focused on developing organ-on-chip products and technologies. A case study on scaffold-free 3D cell culture products, including hanging drop plate, 3D petri dish, and ultra-low attachment plate, featuring a list of more than 60 products that are being used for research and pharmaceutical testing, based on a number of relevant parameters, such as status of development (commercialized and developed, not commercialized), type of system (suspension system, attachment resistant and microfluidic system), type of product (ultra-low attachment plate, plate, hanging drop plate, chips and dish) and material used for fabrication (chemical / polymer based, human based and plant based).

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of organ-on-chip market. Based on multiple parameters, such as overall 3D cell culture market, and share of organ-on-chip, we have provided informed estimates of the evolution of the market for the period 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of type of product (organ(s) based models and disease(s) based models), application area (cancer research, drug discovery and toxicity testing, stem cell research and tissue engineering and regenerative medicine), purpose (research and therapeutic production), key geographical regions (North America, Europe, Asia-Pacific and Rest of the World). In order to account for future uncertainties and to add robustness to our model, we have provided three forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were also influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of discussions held with the following individuals (in alphabetical order of company / organization names): Pierre Gaudriault, (Chief Business Development Officer, Cherry Biotech) Matt Dong-Heon Ha (Chief Executive Officer, EDmicBio) Michael Shuler (President, Hesperos) Jelena Vukasinovic (Chief Executive Officer, Lena Biosciences) Maurizio Aiello (Chief Executive Officer, react4life) Michele Zagnoni (Chief Executive Officer, ScreenIn3D)

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases Press releases from company websites Government policy documents Industry analysts views

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

KEY QUESTIONS ANSWERED Who are the leading players engaged in the development of organ-on-chip products and technologies? What are the different application areas where organ-on-chip can be used? Primarily in which geographical regions, are the organ-on-chip developers located? How has the intellectual property landscape of organ-on-chip, evolved over the years? Which partnership models are commonly adopted by stakeholders in the organ-on-chip domain? What are the investment trends and who are the key investors actively engaged in the research and development of organ-on-chip systems? How is the current and future opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the organ-on-chip market and its likely evolution in the short to mid-term and long term.

Chapter 3 provides a general introduction to organ-on-chip, covering details on the background of organ-on-chips along with their classification. In addition, it also provides information on various advantages and limitations of such products. It also discusses the various application areas and future perspectives of organ-on-chips market.

Chapter 4 provides a detailed analysis of the current market landscape of organ-on-chips based on a number of relevant parameters, such as type of offering(s) (chip, plate / system, and technology), type of model (organ(s) based and disease(s) based), status of development (commercialized, developed, and under development), type of technology / platform, number of chips in a plate, material used for construction (polymer, glass and silicon), type of polymer (polydimethylsiloxane, cyclo olefin polymer, cyclic olefin copolymer, elastomer, polycarbonate, polypropylene, polysterene, polyester, tygon, and styrene TEP), compatible tissue / organ, and application area (cancer research, drug discovery and toxicity testing, stem cell research, and tissue engineering and regenerative medicine). In addition, the chapter provides details on the companies engaged in the development of organ-on-chip products and technologies, along with information on their year of establishment, company size and location of headquarters.

Chapter 5 features elaborate profiles of the key players engaged in development of organ-on-chip (which are presently commercialized), which are headquartered in North America, Europe and Asia-Pacific. Each profile features a brief overview of the company, its financial information (if available), organ-on-chip product portfolio, recent developments, and an informed future outlook.

Chapter 6 features in-depth analysis of various patents that have been filed / granted for organ-on-chip, till July 2022, highlighting key trends associated with these patents, across type of patents, publication year, application year, issuing authorities involved, type of organizations, emerging focus area, patent age, CPC symbols, leading patent assignees (in terms of number of patents granted / filed), patent characteristics and geography. It also includes an insightful patent valuation analysis.

Chapter 7 features brand positioning analysis of the key industry players, highlighting the current perceptions regarding their proprietary products by taking into consideration several relevant aspects, such as experience of the manufacturer, number of products and technologies offered, product diversity, and number of patents published.

Chapter 8 features study of the various grants that have been awarded to research institutes engaged in projects related to organ-on-chip, between 2017 and 2022, highlighting various important parameters, such as year of grant award, amount awarded, funding institute, support period, type of grant application, purpose of grant, activity code, emerging focus area, study section involved, type of recipient organization, key project leaders, key regions, and leading recipient organizations.

Chapter 9 features analysis of the partnerships that have been established since 2017, covering various parameters such as, research and development, clinical trial agreement, product development and commercialization agreement, technology integration agreement, and product development and manufacturing agreement of the companies focused on developing organ-on-chip products and technologies.

Chapter 10 features analysis of the investments made, including seed financing, venture capital financing, debt financing, grants, capital raised from IPOs and subsequent offerings, at various stages of development in start-ups / small-sized companies (with less than 50 employees) that are focused on developing organ-on-chip products and technologies.

Chapter 11 is a case study featuring scaffold-free 3D cell culture products, including hanging drop plate, 3D petri dish, and ultra-low attachment plate, featuring a list of more than 60 products that are being used for research and pharmaceutical testing, based on a number of relevant parameters, such as status of development (commercialized and developed, not commercialized), type of system (suspension system, attachment resistant and microfluidic system), type of product (ultra-low attachment plate, plate, hanging drop plate, chips and dish) and material used for fabrication (chemical / polymer based, human based and plant based).

Chapter 12 features an insightful market forecast analysis, highlighting the future potential of the market till 2035. The current and future opportunity has further been segmented on the basis of type of product (organ(s) based models and disease(s) based models), application area (cancer research, drug discovery and toxicity testing, stem cell research and tissue engineering and regenerative medicine), purpose (research and therapeutic production), key geographical regions (North America, Europe, Asia-Pacific and Rest of the World). It is worth mentioning that we adopted a top-down approach for this analysis, backing our claims with relevant datapoints and credible inputs from primary research.

Chapter 13 is the summary of the overall report, which presents insights on the contemporary market trends and the likely evolution of the organ-on-chip market.

Chapter 14 is a collection of interview transcripts of discussions held with various key stakeholders in this market. The chapter provides a brief overview of the company and details of the interview held with Pierre Gaudriault (Chief Business Development Officer, Cherry Biotech), Matt Dong-Heon Ha (Chief Executive Officer, EdmicBio), Michael Shuler (President, Hesperos), Jelena Vukasinovic (Chief Executive Officer, Lena Biosciences), Maurizio Aiello (Chief Executive Officer, react4life) and Michele Zagnoni (Chief Executive Officer, ScreenIn3D).

Chapter 15 is an appendix, which provides tabulated data and numbers for all the figures provided in the report.

Chapter 16 is an appendix, which contains the list of companies and organizations mentioned in the report. Read the full report: https://www.reportlinker.com/p06323408/?utm_source=GNW

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Curis Announces Date for the 1st Symposium on IRAK4 in Cancer – PR Newswire

Symposium hosted by Guillermo Garcia-Manero, M.D.; Hagop Kantarjian, M.D.; Amit Verma, M.B.B.S.

Academics and industry professionals will gather virtually on October 7, 2022

LEXINGTON, Mass., Oct. 3, 2022 /PRNewswire/ -- Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, today announced the 1st Symposium on IRAK4 in Cancer taking place virtually onOctober 7,9:00am-1:00pm ET.

Hosted by Drs. Guillermo Garcia-Manero, Hagop Kantarjian, and Amit Verma, this symposium will focus on IRAK4, an essential protein in innate immune response and promising target in the treatment of hematologic malignancies and other cancers. Experts across academia and industry will discuss IRAK4 biology, the present understanding of IRAK4 function, and the future of anti-cancer therapeutic development focused on this target.

"Following the success of the VISTA Symposium over the past two years, Curis is proud to be sponsoring the first symposium on IRAK4 in cancer," said James Dentzer, President, and Chief Executive Officer of Curis. "IRAK4 shows great promise as a potential immunotherapy target for the treatment of hematologic cancers, as well as solid tumors, and we look forward to advancing the conversation on how this promise is being translated into meaningful results for patients."

Symposium hosts:

Joining our hosts will be the following speakers and participants:

The virtual event will address topics including:

To learn more about the free to attend symposium and register, please visit https://bit.ly/IRAK4.

AboutCuris, Inc.Curis is a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer. In 2015, Curis entered into a collaboration with Aurigene in the areas of immuno-oncology and precision oncology. As part of this collaboration, Curis has exclusive licenses to oral small molecule antagonists of immune checkpoints including the VISTA/PDL1 antagonist CA-170, and the TIM3/PDL1 antagonist CA-327, as well as the IRAK4 kinase inhibitor, emavusertib (CA-4948). Emavusertib is currently undergoing testing in the Phase 1/2 TakeAim Lymphoma trial, in patients with hematologic malignancies, such as non-Hodgkin's lymphoma and other B cell malignancies, both as a monotherapy and in combination with BTK inhibitor ibrutinib, and the Phase 1/2 TakeAim Leukemia trial in patients with AML and MDS, for which it has received Orphan Drug Designation from the U.S. Food and Drug Administration. The FDA has placed a partial clinical hold on the TakeAim Leukemia trial during which no new patients will be enrolled in the combination phase (Phase 1b) of emavusertib with azacitidine or venetoclax and expansion phase (Phase 2a), and current study participants benefiting from treatment may continue to be treated with emavusertib at doses of 300mg BID or lower. In addition, Curis is engaged in a collaboration with ImmuNext for development of CI-8993, a monoclonal anti-VISTA antibody, which is currently undergoing testing in a Phase 1 trial in patients with solid tumors. Curis is also party to a collaboration with Genentech, a member of the Roche Group, under which Genentech and Roche are commercializing Erivedge for the treatment of advanced basal cell carcinoma. For more information, visit Curis' website atwww.curis.com.

SOURCE Curis, Inc.

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A Clear Vision | The UCSB Current – The UCSB Current

Good vision is essential to everyday living, yet approximately 12 million Americans aged 40 and over live with vision impairment, including 1 million who are legally blind, according to the Centers for Disease Control and Prevention. Although some affected individuals can be treated with surgery or medication, and recent advances in gene and stem cell therapies are showing promise, no effective treatments exist for many people who are blinded by severe degeneration of, or damage to, the retina, the optic nerve or the cortex. In such cases, an electronic visual prosthesis, or bionic eye, may be the only option.

Michael Beyeler, an assistant professor of computer science and psychological and brain sciences at UC Santa Barbara, aims to bring to the mainstream an AI-powered bionic eye that can generate artificial vision, in an effort to increase the quality of life for patients who are blind or visually impaired.

I envision a smart bionic eye that could find misplaced keys on a counter, read out medication labels, inform a user about peoples gestures and facial expressions during social interactions, and warn a user of nearby obstacles and outline safe paths, he said.

For his project, Towards a Smart Bionic Eye: AI-Powered Artificial Vision for the Treatment of Incurable Blindness, Beyeler has been selected for a National Institutes of Health (NIH) Directors New Innovator Award. The five-year, $1.5 million grant was one of 103 awarded this week by the NIH to enable exceptionally creative early-career scientists to push the boundaries of biomedical science and pursue high-impact projects that aim to advance knowledge and enhance health.

I offer my sincerest congratulations to Professor Beyeler for having his innovative research recognized with the prestigious NIH Director's New Innovator Award, saidTresa Pollock,the interim dean of the College of Engineering and Alcoa Distinguished Professor of Materials. His novel approach of using recent advances in computer vision, AI and neuroscience has tremendous potential to uncover new knowledge and provide millions of people with useful vision through a smart bionic eye.

I am tremendously honored and excited to be given this award, said Beyeler, who has previously received the NIH Pathway to Independence Award. As part of the NIHs High-Risk, High-Reward Research program, this award will allow my group to explain the science behind bionic technologies that may one day restore useful vision to millions of people living with incurable blindness.

Bionic eyes, as we know them, transform light, captured by a head-mounted camera, into electrical pulses that are delivered through a microelectrode array implanted in the eye or the visual cortex, which is then interpreted by the brain as visual perceptions, or phosphenes. Although current devices generally offer an improved ability to differentiate light from dark backgrounds and see motion, the vision they provide is blurry, distorted and often hard to interpret.

A major challenge for scientists trying to develop visual prosthetics is thus to predict what implant recipients see when they use their devices. Instead of seeing focal spots of light, current retinal-implant users perceive highly distorted phosphenes that often fail to assemble into more complex objects of perception. Consequently, the vision generated by current prostheses has been widely described as fundamentally different from natural vision, and does not improve over time.

Beyeler takes a different approach. Rather than aiming to make bionic vision as natural as possible, he proposes to focus on how to create practical and useful artificial vision that would be based on artificial intelligence (AI)-based scene understanding and be tailored to specific real-world tasks that affect a blind persons quality of life, such as facial recognition, outdoor navigation and self-care.

His novel, multidisciplinary strategy for this project will investigate the neural code of vision, studying how to translate electrode stimulation into a code that the human brain can understand.

We want to address fundamental questions at the intersection of neuroscience, computer science, and human-computer interaction to enable the development of a Smart Bionic Eye, a visual neuroprosthesis that functions as an AI-powered visual aid for the blind, said Beyeler.

To enable a technology that provides cues to the visually impaired, much as a computer vision system talks to a self-driving car, Beyeler must first understand how visual prostheses interact with the human visual system to shape perception. He said that a common misconception in the field is that each electrode in a devices microelectrode array can be thought of as a pixel in an image, or a minute area of illumination on a display screen, and that to generate a complex visual experience, one simply needs to turn on the right combination of pixels. His research shows, however, that the visual experience provided by current prostheses is highly distorted and unrelated to the number of electrodes.

Current devices do not have sufficient image resolution to convey a complex natural scene. Hence, there is a need for scene simplification, Beyeler said.

One way to simplify the visual scene and create useful artificial vision, according to Beyeler, is through deep-learning-based computer vision, which can be used to highlight nearby obstacles or remove background clutter. Computer vision is a field of AI that enables computers and systems to derive important information from digital images, videos and other visual inputs and take actions or make recommendations based on that information. Computer vision relies on cameras, data, and algorithms, rather than retinas, optic nerves and a visual cortex.

He said that his project will be patient-centric, involving people at all stages of the design process to test out his groups theoretical predictions. The patients will be provided by his collaborators at four universities across the country and in Spain. Beyelers team will design experiments that probe an implants potential to support functional vision for real-world tasks involving object recognition, scene understanding and mobility. This method strays from the typical vision tests performed in clinics that measure acuity, contrast sensitivity and orientation discrimination.

Due to the unique requirements of working with bionic-eye recipients, such as constant assistance, setup time and travel, experimentation remains time-consuming and expensive. Beyeler proposes an interim solution.

A more cost-effective and increasingly popular alternative might be to rely on an immersive virtual reality (VR) prototype based on simulated prosthetic vision (SPV), Beyeler explained.

The classical SPV method relies on sighted subjects wearing a VR head-mounted display (HMD). The subjects are then deprived of natural viewing and allowed to perceive only phosphenes displayed in the HMD. This approach enables sighted participants to see through the eyes of the bionic-eye user as they explore a virtual environment. The visual scene can then be manipulated by researchers according to any desired image-processing or visual-enhancement strategy.

The challenge in the field is less about dreaming up new augmentation strategies and more about finding effective visual representations to support practical, everyday tasks, said Beyeler. This is why, in my project, we utilize a prototyping system that allows us to explore different strategies and find out what works before implanting devices in patients.

In the future, he said, the Smart Bionic Eye could be combined with GPS to give directions, warn users of impending dangers in their immediate surroundings, or even extend the range of visible light with the use of an infrared sensor, providing what he describes as bionic night-time vision. But before any of that can happen, Beyeler said, the fundamental scientific questions must be addressed.

Success of this project would translate to a new potential treatment option for incurable blindness, which affects nearly 40 million people worldwide, said Beyeler, who plans to make all of his groups software, tools and deidentified data available to the scientific community. Overall, this will be a fantastic opportunity for my lab to contribute substantially in the field of sight restoration and make a difference in the world.

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A Clear Vision | The UCSB Current - The UCSB Current