Archive for October, 2020

Heres your wrap of the latest technology, innovation, and finance news.

Alternative Foods

How far are cell-based meats from commercialisation? Verdict talked with Memphis Meats about where they are on commercialisation journey.

We need to find clever process solutions and cell feed formulations to ensure that were translating our raw materials into meat in the most cost-effective ways.

For the vegans who dont like soy milk, Impossible Foods is developing a plant-based alternative to cows milk.

As with Impossible Foods meat products, the goal of the milk alternative is to preserve the experience of consuming dairy products, including the texture, mouth feel, and flavour, while reducing the demand for raising animals like cattle.

Gaming

Alexandria Ocasio-Cortez played Among Us on Twitch and 430,000 people tuned in, making her the third most popular streamer on the site ever.

More important, the event offered a glimpse at the future of political campaigning. In the waning weeks of 2020s volatile American election campaign, Democrats are increasingly turning to Twitch streams of popular video games to reach out to young voters and urge every last one of them to show up and vote.

Video games are helping veterans with PTSD.

Amazons cloud-gaming service, Luna, is now available to select gamers in the United States.

Cyberpunk 2077 is using procedurally-generated facial animations to provide full lip-sync for every character in all 10 dubbed languages. See Judy speak English, French, German, and Japanese at the 17:05 point.

Artificial Intelligence

Facebook has developed a new AI thats capable of directly translating between any pair of 100 languages without first translating to English, as many systems do, and its performing pretty well.

The AI outperforms such systems by 10 points on a 100-point scale used by academics to automatically evaluate the quality of machine translations. Translations produced by the model were also assessed by humans, who scored it as around 90 per cent accurate.

A new paper outlines less than one-shot learning, a new technique designed to let AI learn with practically no data.

This is what most interests Tongzhou Wang, an MIT PhD student who led the earlier research on data distillation. The paper builds upon a really novel and important goal: learning powerful models from small data sets, he says of Sucholutskys contribution.

Landing AI is a company founded by Andrew Ng, one of the cofounders of Google Brain and the former chief scientist at Baidu. Theyve recently launched LandingLens, a computer vision platform that enables manufacturers to train AI models.

I feel like this is where the field of AI needs to go. Rather than highly skilled engineers at Landing AI or Google or wherever doing all the machine learning work to build verticalized platforms, someone in a platform [who] really understands what is a dent versus what is a sensible-minded blemish can do the customization. I think this is important for machine learning to reach its full potential, Ng said.

Biology

For the first time, scientists have improved the resolution of cryo-electron microscopes to the point where they can see individual atoms (see paper 1, paper 2).

Now, with the help of improvements in electron beam technology, detectors, and software, two groups of researchersfrom the United Kingdom and Germanyhave narrowed that to 1.25 angstroms or better,sharp enough to work out the position of individual atoms, they report today inNature.

Space

Microsoft is taking Azure cloud computing to outer space.

The goal is to serve as a bridge between satellites and new services from both the public and private sectors, spanning military, telecommunications, agriculture, energy, and more.

NASA has awarded Nokia a $14.1 million contract to build a 4G network on the moon.

Blue Origin has found a business niche with NASA and private science experiments.

Tucked under the collar at the top of the booster on Tuesdays launch were prototypes of sensors that could help NASA astronauts safely reach the lunar surface in a few years. It is part of NASAs Tipping Point program, which seeks to push innovative technologies.

Head of NASAs Pluto mission, Alan Stern, is going to space with Virgin Galactic.

Stern will oversee two different experiments while on board the flight, each meant to take advantage of the brief stay in the space environment.

OSIRIS-REx, a NASA spacecraft, has sampled material from the asteroid Bennu.

Surveillance

Activists around the world are building facial recognition tools to identify police officers that arent wearing identification.

I am involved with developing facial recognition to in fact use on Portland police officers, since they are not identifying themselves to the public, Mr. Howell said. Over the summer, with the city seized by demonstrations against police violence, leaders of the department had told uniformed officers that they couldtape over their name. Mr. Howell wanted to know: Would his use of facial recognition technology become illegal?

Mobility

Boom Supersonic unveiled XB-1, a subscale prototype of their planned supersonic passenger jet.

Dubbed XB-1, the 71-foot-long, single-seat test vehicle was built to validate the design and technology of the companys planned eventual final product, a $200 million airliner called Overture that will be three times XB-1s size and carry 55 passengers to Mach 2.2.

Health

Can gene therapy be used to treat some forms of autism?

Gene therapies are now moving into the autism space, and the Angelman trial is a sign of things to come. A success in this space will completely change the way that we think about genetic testing in autism, saysTimothy Yu, a neurologist and geneticist atBoston Childrens Hospital in Massachusetts. It will hold out the idea that if you can name the disease, you can actually do something to improve the quality of life for that child.

Researchers are using lab-grown tissue grafts for personalised joint replacements.

A team at the University of Leeds have developed a robotic arm to perform colonoscopies.

The researchers successfully tested this method in an artificial colon as well as in two pigs. They believe the magnetically controlled procedure may be lesspainfulthan conventional colonoscopies and could be used on patients without sedation.

Ecommerce

Alibaba is spending $3.6 billion to double its stake in Sun Art Retail Group, Chinas largest big-box retailer with more than 480 large supermarket-department stores.

Gavin Baker argues that leading brick-and-mortar retailers are likely to be the biggest long-term Covid beneficiaries.

Finance

China is experiencing a boom in share sales.

So far this year, exchanges in Shanghai and Shenzhen have hosted more than $47.5 billion of IPOs and listings for firms that have shares already trading elsewhere, Refinitiv data shows.

That is already the highest annual tally compared with any full year since 2010 and an unprecedented 27% of the global total, the data shows. If deals in Hong Kong by Chinese companies are added, the proportion rises to 43%.

Other Snippets

MIT Technology Review looked at Singapores huge bet on vertical farming.

Since then, food security has raced up the agenda. Now the governments stated policy is that it wants to produce enough food to supply 30% of its own nutritional needs by 2030, up from just 10% now. To get there, it says, Singapore will need to grow 50% of all fruits and vegetables consumed domestically, 25% of all proteins, and 25% of all staples, such as brown rice. The commitment effectively aims to triple production by volume in the next 10 years. And since the country is short of land, it has pinned its hopes on technology.

Zoom has begun rolling out end-to-end encryption.

All Zoom users free or paid can now host a meeting with up to 200 participants with end-to-end encryption on the platform, meaning the company cannot access any of the data it is hosting.

The New York Times discussed the problem of free speech in an age of disinformation, and how democracies in Europe and Canada balance free speech with other democratic values.

A Japanese politician is battling to vanquish the ink stamp, the printer, and the fax machine.

Why do we need to print out paper? Mr. Kono asked rhetorically at a news conference soon after taking charge of the issue in September. In many cases, it is simply because the hanko is required. So if we can put a stop to that culture, then it will naturally eliminate the need for printouts and faxes.

Fast Company outlined 25 moments in tech that defined the past 25 years.

Read more:
Innovation Wrap: Cell-Based Meat, AI Less-Than-One-Shot Learning, Gene Therapy & Autism - ShareCafe

NEW DELHI: Convalescent plasma therapy, which uses the blood of recovered Covid-19 patients as a potential treatment, has shown limited effect in reducing the progression to severe disease or death in a trial conducted in India, scientists say. The study, published in the British Medical Journal (BMJ) involved 464 adults with moderate Covid-19 who were admitted to hospitals in India between April and July.

As many as 239 adult patients received two transfusions of convalescent plasma, 24 hours apart, alongside standard care, while the control group comprising of 229 patients received standard care only.

One month later, 44 patients or 19 per cent of those who received the plasma had progressed to severe disease or had died of any cause, compared with 41 patients or 18 per cent in the control group.

"Convalescent plasma was not associated with a reduction in progression to severe Covid-19 or all cause mortality," the researchers wrote in the journal.

"This trial has high generalisability and approximates convalescent plasma use in real life settings with limited laboratory capacity," they said.

The researchers noted that a prior measurement of neutralising antibody titres in donors and participants might further clarify the role of convalescent plasma in the management of Covid-19.

Patients in the study were aged at least 18 years who had confirmed Covid-19 based on a RT-PCR result for SARS-CoV-2, the virus that causes the disease.

Participants in the intervention arm received two doses of 200 millilitre (mL) of convalescent plasma, transfused 24 hours apart, in addition to the best standard of care.

Although the observational studies conducted previously suggested clinical benefits in recipients of convalescent plasma, the trials were stopped early and failed to ascertain any mortality benefit from plasma treatment in patients with Covid-19, the researchers said.

Although plasma treatment was associated with earlier resolution of shortness of breath and fatigue and higher negative conversion of SARS-CoV-2 RNA on day 7 of enrolment, as a potential treatment for patients with moderate Covid-19 it showed limited effectiveness.

View original post here:
Covid-19 plasma therapy has shown little benefit in patients in India, study finds - ETHealthworld.com

The rise in the popularity of cell and Gene Therapy has been widely talked about in recent years. The rise in this popularity has come with its own challenges, a number of which were identified in a recent report by Root Analysis. The challenges vary from the high cost of manufacturing to low capacity. In fact, the Roots Analysis team interviewed several stakeholders to understand their perspective about the key challenges in the vector manufacturing market. The key points from the interviews have been highlighted in this article. In the figure, I have added some of these challenges:

For More Insights Click Here

Capacity constraints remain a key challenge and have been talked about quite actively. In fact, we have seen several players invest heavily in enhancing their vector manufacturing capacity. However, one other area that has silently emerged as a potentially hot topic is the novel vectors (beyond traditional AAV vectors).

Currently, the vector manufacturing market is dominated by viral vectors, such as those based on AAV, adenovirus, lentivirus, and retrovirus. However, certain non-viral vectors, such as plasmid DNA, also hold a considerable share. Roots Analysis, in their report, talked about the rise of these non-viral vectors and identified the key emerging vectors types. Some of the vectors highlighted in the report are alphavirus, Anc80 vector, B. longum, Listeria monocytogenes, minicircle DNA, myxoma virus, Sendai virus, self-complementary vectors (which are essentially improved versions of AAV vectors) Sleeping Beauty transposon-based non-viral vectors, and Vaccinia virus-based vectors.

In fact, several players in the biopharmaceutical industry have already begun using some of the novel vector types mentioned above for the development of their respective pipeline therapy candidates. A few of the players involved in the novel vectors space are highlighted below:

The stakeholders interviewed by Roots Analysis agreed to the rising demand for novel vector types. Here are the excerpts from some of the interviews:

We believe that a few novel vectors, having low immunogenicities and targeting different cell types, are likely to soon be introduced into the market. I am also aware of companies that are researching different (better) versions of adeno-associated viral vectors. Executive & Scientific Officer, A small-sized company based in Belgium

We have worked with a couple of transposons and the Vaccinia viruses as vectors, the latter cannot exactly be regarded as a novel approach. I believe, transposons and Sendai virus are the only novel vector systems that are likely to soon become popular. Managing Director, A management consulting firm for regenerative medicines based in Japan

To overcome the challenges associated with the production of contaminant free final product using conventional plasmids, we are developing a minicircle DNA vector, which are devoid of antibiotic resistance genes and prokaryotic plasmid components, which are crucial for the replication of constructs in bacteria. Project Manager and Marketing Manager, a small-sized company based in Germany

For further information, check out the report here

Read more insights at

Roots Analysis Leaders in Pharmaceutical & Biotechnology Market Research

You may also be interested in the following titles:

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

Read more from the original source:
Cell and Gene Therapy Manufacturing: Rising Demand Forces Companies to Look Beyond Viral Vectors | Roots Analysis - Eurowire

Shortly after Sirnaomics brought in a $47 million Series C for its small interfering RNA pipeline last year, Patrick Lu the founder, president and CEO was asked to outline the scientific advances that will be necessary to make better drugs out of RNA tech.

The next step in the evolution of RNAi as a leading therapeutic will be the ability to safely target organs outside the liver such as lung, brain, etc, he had offered. This will revolutionize disease treatments if the industry can demonstrate similar data sets for non-liver targets as we have seen in liver-based diseases.

Then in April, the trans-Pacific biotech did just that. In a Phase II open-label dose escalation study, Sirnaomics reported interim results suggesting that its lead drug, STP705, helped certain cancer patients clear their squamous cell carcinoma.

Investors now say its time for a Series D, pumping $105 million into the STP705 program as well as another lead drug named STP707. The clinical focus, Sirnaomics added, will be evaluating these dual-targeted siRNA inhibitors, which hit TGF-1 and COX-2 either locally or systemically, together with checkpoint inhibitors. But with almost 10 other programs in the pipeline, the company remains on track to explore not just RNAis application in cancer but also in fibrosis diseases, metabolic diseases and viral infections.

Rotating Boulder Fund, an existing investor, led the round alongside new backers Walvax Biotechnology and Sunshine Riverhead Capital. Others on the syndicate include Sangel Capital, Longmen Capital, HongTao Capital and Alpha Win Capital.

In addition to a potential collaboration with Walvax on technical transfer and commercialization, Lu is open about preparing for an IPO in near future.

The company is the only biopharma venture conducting innovative R&D and clinical development in the field of RNAi therapeutics in both the US and China, the two largest markets for cancer and fibrosis disease treatments, Donald (Xiaochang) Dai, managing partner of Rotating Boulder Fund, noted in a statement.

With offices in Gaithersburg, MD and Suzhou BioBay just west of Shanghai, Sirnaomics recognizes that it is traveling down a path blazed by the likes of Alnylam and Arrowhead. But it boasts of a platform comprising a new polypeptide nanoparticle delivery system and a way to hit two targets at once promising to push RNAi beyond rare diseases or even cardiovascular conditions.

At Sirnaomics specifically, we are forging a path to bring RNAi therapeutics to the mainstream as therapeutic modalities for treatment of many diseases, such as non-melanoma skin cancer, liver cancer, liver fibrosis and NASH, Lu said in his 2019 interview.

Read this article:
Chinese investors wager $105M on an IPO-bound biotech looking to push RNAi as mainstream cancer therapy - Endpoints News

PRINCETON, N.J. & ALAMEDA, Calif.--(BUSINESS WIRE)--Oct 19, 2020--

Bristol Myers Squibb (NYSE: BMY) and Exelixis, Inc. (NASDAQ: EXEL) today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) and supplemental New Drug Application (sNDA), respectively, for OPDIVO (nivolumab) in combination with CABOMETYX (cabozantinib) for patients with advanced renal cell carcinoma (RCC). The FDA granted Priority Review to both applications and assigned a Prescription Drug User Fee Act (PDUFA) goal date, or target action date, of February 20, 2021.

These filings were based on results from the Phase 3 CheckMate -9ER trial, which evaluated OPDIVO in combination with CABOMETYX in patients with previously untreated advanced RCC versus sunitinib. In CheckMate -9ER, OPDIVO in combination with CABOMETYX demonstrated significant improvements across all efficacy endpoints, including overall survival (OS), progression-free survival (PFS) and objective response rate (ORR), versus the comparator, sunitinib.

We have witnessed practice-changing advancements in the treatment of renal cell carcinoma in recent years, but we recognize the importance of providing patients and physicians with additional options that can help them take control of the disease, said Mark Rutstein, vice president, development program lead, OPDIVO, Bristol Myers Squibb. In the CheckMate -9ER trial, combining OPDIVO and CABOMETYX, two proven agents with strong clinical legacies in advanced renal cell carcinoma, led to superior efficacy across all endpoints. We look forward to working with the FDA to bring this potential treatment option to physicians and their patients who choose an immunotherapy plus tyrosine kinase inhibitor regimen.

With their complementary mechanisms of action and evidence that CABOMETYX may promote a more immune-permissive environment, we believe there is opportunity for additive or synergistic effects with this potential combination regimen, said Gisela Schwab, M.D., president, product development and medical affairs and chief medical officer, Exelixis. Based on strong supporting data from CheckMate -9ER, the acceptance of our application is important progress in our efforts to make CABOMETYX in combination with OPDIVO available to patients with advanced kidney cancer who need additional treatment options. We look forward to working with the FDA throughout the ongoing review process.

The combination of OPDIVO plus CABOMETYX was well tolerated, with a low rate of treatment-related discontinuations, and reflected the known safety profiles of the immunotherapy and tyrosine kinase inhibitor components in patients with previously untreated advanced RCC. In addition, patient-reported outcomes data from CheckMate -9ER showed that OPDIVO in combination with CABOMETYX was associated with statistically significant improvements in health-related quality of life at most time points versus sunitinib. On September 19, 2020, results from the trial were presented as a Proffered Paper during a Presidential Symposium at the European Society for Medical Oncology (ESMO) Virtual Congress 2020.

Bristol Myers Squibb and Exelixis thank the patients and investigators who were involved in the CheckMate -9ER clinical trial.

About CheckMate -9ER

CheckMate -9ER is an open-label, randomized, multi-national Phase 3 trial evaluating patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). A total of 651 patients (23% favorable risk, 58% intermediate risk, 20% poor risk; 25% PD-L11%) were randomized to receive OPDIVO plus CABOMETYX (n=323) vs. sunitinib (n=328). The primary endpoint is progression-free survival (PFS). Secondary endpoints include overall survival (OS) and objective response rate (ORR). The primary efficacy analysis is comparing the doublet combination vs. sunitinib in all randomized patients. The trial is sponsored by Bristol Myers Squibb and Ono Pharmaceutical Co and co-funded by Exelixis, Ipsen and Takeda Pharmaceutical Company Limited.

About Renal Cell Carcinoma

Renal cell carcinoma (RCC) is the most common type of kidney cancer in adults, accounting for more than 140,000 deaths worldwide each year. RCC is approximately twice as common in men as in women, with the highest rates of the disease in North America and Europe. The five-year survival rate for those diagnosed with metastatic, or advanced, kidney cancer is 12.1%.

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early- to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Opdivo

Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the bodys own immune system to help restore anti-tumor immune response. By harnessing the bodys own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Opdivo s leading global development program is based on Bristol Myers Squibbs scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.

In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Companys Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.

About CABOMETYX

In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC and for the treatment of patients with HCC who have been previously treated with sorafenib. CABOMETYX tablets have also received regulatory approvals in the European Union, Japan and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the United States.

OPDIVO INDICATIONS

OPDIVO (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.

OPDIVO (nivolumab) is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with progression after platinum-based chemotherapy and at least one other line of therapy. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).

OPDIVO (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC).

OPDIVO (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.

OPDIVO (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

OPDIVO (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.

OPDIVO (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.

OPDIVO IMPORTANT SAFETY INFORMATION

Severe and Fatal Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions listed herein may not be inclusive of all possible severe and fatal immune-mediated adverse reactions.

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. While immune-mediated adverse reactions usually manifest during treatment, they can also occur at any time after starting or discontinuing YERVOY. Early identification and management are essential to ensure safe use of YERVOY. Monitor for signs and symptoms that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate clinical chemistries including liver enzymes, creatinine, adrenocorticotropic hormone (ACTH) level, and thyroid function at baseline and before each dose. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue YERVOY depending on severity. In general, if YERVOY requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less followed by corticosteroid taper for at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reaction is not controlled with corticosteroid therapy. Institute hormone replacement therapy for endocrinopathies as warranted.

Immune-Mediated Pneumonitis

OPDIVO can cause immune-mediated pneumonitis. Fatal cases have been reported. Monitor patients for signs with radiographic imaging and for symptoms of pneumonitis. Administer corticosteroids for Grade 2 or more severe pneumonitis. Permanently discontinue for Grade 3 or 4 and withhold until resolution for Grade 2. In patients receiving OPDIVO monotherapy, fatal cases of immune-mediated pneumonitis have occurred. Immune-mediated pneumonitis occurred in 3.1% (61/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated pneumonitis occurred in 6% (25/407) of patients. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated pneumonitis occurred in 10% (5/49) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated pneumonitis occurred in 4.4% (24/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated pneumonitis occurred in 1.7% (2/119) of patients. In NSCLC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated pneumonitis occurred in 9% (50/576) of patients, including Grade 4 (0.5%), Grade 3 (3.5%), and Grade 2 (4.0%) immune-mediated pneumonitis. Four patients (0.7%) died due to pneumonitis. The incidence and severity of immune-mediated pneumonitis in patients with NSCLC treated with OPDIVO 360 mg every 3 weeks in combination with YERVOY 1 mg/kg every 6 weeks and 2 cycles of platinum-doublet chemotherapy were comparable to treatment with OPDIVO in combination with YERVOY only. The incidence and severity of immune-mediated pneumonitis in patients with malignant pleural mesothelioma treated with OPDIVO 3 mg/kg every 2 weeks with YERVOY 1 mg/kg every 6 weeks were similar to those occurring in NSCLC.

In Checkmate 205 and 039, pneumonitis, including interstitial lung disease, occurred in 6.0% (16/266) of patients receiving OPDIVO. Immune-mediated pneumonitis occurred in 4.9% (13/266) of patients receiving OPDIVO: Grade 3 (n=1) and Grade 2 (n=12).

Immune-Mediated Colitis

OPDIVO can cause immune-mediated colitis. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 (of more than 5 days duration), 3, or 4 colitis. Withhold OPDIVO monotherapy for Grade 2 or 3 and permanently discontinue for Grade 4 or recurrent colitis upon re-initiation of OPDIVO. When administered with YERVOY, withhold OPDIVO and YERVOY for Grade 2 and permanently discontinue for Grade 3 or 4 or recurrent colitis. In patients receiving OPDIVO monotherapy, immune-mediated colitis occurred in 2.9% (58/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated colitis occurred in 26% (107/407) of patients including three fatal cases. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated colitis occurred in 10% (5/49) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated colitis occurred in 10% (52/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated colitis occurred in 7% (8/119) of patients.

In a separate Phase 3 trial of YERVOY 3 mg/kg, immune-mediated diarrhea/colitis occurred in 12% (62/511) of patients, including Grade 3-5 (7%).

Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Addition of an alternative immunosuppressive agent to the corticosteroid therapy, or replacement of the corticosteroid therapy, should be considered in corticosteroid-refractory immune-mediated colitis if other causes are excluded.

Immune-Mediated Hepatitis

OPDIVO can cause immune-mediated hepatitis. Monitor patients for abnormal liver tests prior to and periodically during treatment. Administer corticosteroids for Grade 2 or greater transaminase elevations. For patients without HCC, withhold OPDIVO for Grade 2 and permanently discontinue OPDIVO for Grade 3 or 4. For patients with HCC, withhold OPDIVO and administer corticosteroids if AST/ALT is within normal limits at baseline and increases to >3 and up to 5 times the upper limit of normal (ULN), if AST/ALT is >1 and up to 3 times ULN at baseline and increases to >5 and up to 10 times the ULN, and if AST/ALT is >3 and up to 5 times ULN at baseline and increases to >8 and up to 10 times the ULN. Permanently discontinue OPDIVO and administer corticosteroids if AST or ALT increases to >10 times the ULN or total bilirubin increases >3 times the ULN. In patients receiving OPDIVO monotherapy, immune-mediated hepatitis occurred in 1.8% (35/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated hepatitis occurred in 13% (51/407) of patients. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated hepatitis occurred in 20% (10/49) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated hepatitis occurred in 7% (38/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated hepatitis occurred in 8% (10/119) of patients.

In Checkmate 040, immune-mediated hepatitis requiring systemic corticosteroids occurred in 5% (8/154) of patients receiving OPDIVO.

In a separate Phase 3 trial of YERVOY 3 mg/kg, immune-mediated hepatitis occurred in 4.1% (21/511) of patients, including Grade 3-5 (1.6%).

Immune-Mediated Endocrinopathies

OPDIVO can cause immune-mediated hypophysitis, immune-mediated adrenal insufficiency, autoimmune thyroid disorders, and Type 1 diabetes mellitus. Monitor patients for signs and symptoms of hypophysitis, signs and symptoms of adrenal insufficiency, thyroid function prior to and periodically during treatment, and hyperglycemia. Withhold for Grades 2, 3, or 4 endocrinopathies if not clinically stable. Administer hormone replacement as clinically indicated and corticosteroids for Grade 2 or greater hypophysitis. Withhold for Grade 2 or 3 and permanently discontinue for Grade 4 hypophysitis. Administer corticosteroids for Grade 3 or 4 adrenal insufficiency. Withhold for Grade 2 and permanently discontinue for Grade 3 or 4 adrenal insufficiency. Administer hormone-replacement therapy for hypothyroidism. Initiate medical management for control of hyperthyroidism. Withhold OPDIVO for Grade 3 and permanently discontinue for Grade 4 hyperglycemia.

In patients receiving OPDIVO monotherapy, hypophysitis occurred in 0.6% (12/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, hypophysitis occurred in 9% (36/407) of patients. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, hypophysitis occurred in 4% (2/49) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, hypophysitis occurred in 4.6% (25/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated hypophysitis occurred in 3.4% (4/119) of patients. In patients receiving OPDIVO monotherapy, adrenal insufficiency occurred in 1% (20/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, adrenal insufficiency occurred in 5% (21/407) of patients. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, adrenal insufficiency occurred in 18% (9/49) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, adrenal insufficiency occurred in 7% (41/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, adrenal insufficiency occurred in 5.9% (7/119) of patients. In patients receiving OPDIVO monotherapy, hypothyroidism or thyroiditis resulting in hypothyroidism occurred in 9% (171/1994) of patients. Hyperthyroidism occurred in 2.7% (54/1994) of patients receiving OPDIVO monotherapy. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, hypothyroidism or thyroiditis resulting in hypothyroidism occurred in 22% (89/407) of patients. Hyperthyroidism occurred in 8% (34/407) of patients receiving this dose of OPDIVO with YERVOY. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, hypothyroidism or thyroiditis resulting in hypothyroidism occurred in 22% (11/49) of patients. Hyperthyroidism occurred in 10% (5/49) of patients receiving this dose of OPDIVO with YERVOY. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, hypothyroidism or thyroiditis resulting in hypothyroidism occurred in 22% (119/547) of patients. Hyperthyroidism occurred in 12% (66/547) of patients receiving this dose of OPDIVO with YERVOY. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, hypothyroidism or thyroiditis resulting in hypothyroidism occurred in 15% (18/119) of patients. Hyperthyroidism occurred in 12% (14/119) of patients. In patients receiving OPDIVO monotherapy, diabetes occurred in 0.9% (17/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, diabetes occurred in 1.5% (6/407) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, diabetes occurred in 2.7% (15/547) of patients.

In a separate Phase 3 trial of YERVOY 3 mg/kg, severe to life-threatening endocrinopathies occurred in 9 (1.8%) patients. All 9patients had hypopituitarism, and some had additional concomitant endocrinopathies such as adrenal insufficiency, hypogonadism, and hypothyroidism. Six of the 9 patients were hospitalized for severe endocrinopathies.

Immune-Mediated Nephritis and Renal Dysfunction

OPDIVO can cause immune-mediated nephritis. Monitor patients for elevated serum creatinine prior to and periodically during treatment. Administer corticosteroids for Grades 2-4 increased serum creatinine. Withhold OPDIVO for Grade 2 or 3 and permanently discontinue for Grade 4 increased serum creatinine. In patients receiving OPDIVO monotherapy, immune-mediated nephritis and renal dysfunction occurred in 1.2% (23/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated nephritis and renal dysfunction occurred in 2.2% (9/407) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated nephritis and renal dysfunction occurred in 4.6% (25/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated nephritis and renal dysfunction occurred in 1.7% (2/119) of patients.

Immune-Mediated Skin and Dermatologic Adverse Reactions

OPDIVO can cause immune-mediated rash, including Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN), some cases with fatal outcome. Administer corticosteroids for Grade 3 or 4 rash. Withhold for Grade 3 and permanently discontinue for Grade 4 rash. For symptoms or signs of SJS or TEN, withhold OPDIVO and refer the patient for specialized care for assessment and treatment; if confirmed, permanently discontinue. In patients receiving OPDIVO monotherapy, immune-mediated rash occurred in 9% (171/1994) of patients. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated rash occurred in 22.6% (92/407) of patients. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, immune-mediated rash occurred in 35% (17/49) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated rash occurred in 16% (90/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, immune-mediated rash occurred in 14% (17/119) of patients.

YERVOY can cause immune-mediated rash or dermatitis, including bullous and exfoliative dermatitis, Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN). Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-bullous exfoliative rashes. Withhold YERVOY until specialist assessment for Grade 2 and permanently discontinue for Grade 3 or 4 exfoliative or bullous dermatologic conditions.

In a separate Phase 3 trial of YERVOY 3 mg/kg, immune-mediated rash occurred in 15% (76/511) of patients, including Grade 3-5 (2.5%).

Immune-Mediated Encephalitis

OPDIVO can cause immune-mediated encephalitis. Fatal cases have been reported. Evaluation of patients with neurologic symptoms may include, but not be limited to, consultation with a neurologist, brain MRI, and lumbar puncture. Withhold OPDIVO in patients with new-onset moderate to severe neurologic signs or symptoms and evaluate to rule out other causes. If other etiologies are ruled out, administer corticosteroids and permanently discontinue OPDIVO for immune-mediated encephalitis. In patients receiving OPDIVO monotherapy, encephalitis occurred in 0.2% (3/1994) of patients. Fatal limbic encephalitis occurred in one patient after 7.2 months of exposure despite discontinuation of OPDIVO and administration of corticosteroids. Encephalitis occurred in one melanoma patient receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg (0.2%) after 1.7 months of exposure. Encephalitis occurred in one RCC patient receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg (0.2%) after approximately 4 months of exposure. Encephalitis occurred in one MSI-H/dMMR mCRC patient (0.8%) receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg after 15 days of exposure.

Other Immune-Mediated Adverse Reactions

Based on the severity of the adverse reaction, permanently discontinue or withhold OPDIVO, administer high-dose corticosteroids, and, if appropriate, initiate hormone-replacement therapy. Dose modifications for YERVOY for adverse reactions that require management different from these general guidelines are summarized as follows. Withhold for Grade 2 and permanently discontinue YERVOY for Grade 3 or 4 neurological toxicities. Withhold for Grade 2 and permanently discontinue YERVOY for Grade 3 or 4 myocarditis. Permanently discontinue YERVOY for Grade 2, 3, or 4 ophthalmologic adverse reactions that do not improve to Grade 1 within 2 weeks while receiving topical therapy OR that require systemic therapy. Across clinical trials of OPDIVO monotherapy or in combination with YERVOY , the following clinically significant immune-mediated adverse reactions, some with fatal outcome, occurred in <1.0% of patients receiving OPDIVO: myocarditis, rhabdomyolysis, myositis, uveitis, iritis, pancreatitis, facial and abducens nerve paresis, demyelination, polymyalgia rheumatica, autoimmune neuropathy, Guillain-Barr syndrome, hypopituitarism, systemic inflammatory response syndrome, gastritis, duodenitis, sarcoidosis, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), motor dysfunction, vasculitis, aplastic anemia, pericarditis, myasthenic syndrome, hemophagocytic lymphohistiocytosis (HLH), and autoimmune hemolytic anemia. In addition to the immune-mediated adverse reactions listed above, across clinical trials of YERVOY monotherapy or in combination with OPDIVO, the following clinically significant immune-mediated adverse reactions, some with fatal outcome, occurred in <1% of patients unless otherwise specified: autoimmune neuropathy (2%), meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis, nerve paresis, angiopathy, temporal arteritis, pancreatitis (1.3%), arthritis, polymyositis, conjunctivitis, cytopenias (2.5%), eosinophilia (2.1%), erythema multiforme, hypersensitivity vasculitis, neurosensory hypoacusis, psoriasis, blepharitis, episcleritis, orbital myositis, scleritis, and solid organ transplant rejection. Some cases of ocular IMARs have been associated with retinal detachment.

If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, which has been observed in patients receiving OPDIVO and YERVOY and may require treatment with systemic steroids to reduce the risk of permanent vision loss.

Infusion-Related Reactions

OPDIVO can cause severe infusion-related reactions, which have been reported in <1.0% of patients in clinical trials. Discontinue OPDIVO in patients with Grade 3 or 4 infusion-related reactions. Interrupt or slow the rate of infusion in patients with Grade 1 or 2. Severe infusion-related reactions can also occur with YERVOY. Discontinue YERVOY in patients with severe or life-threatening infusion reactions and interrupt or slow the rate of infusion in patients with mild or moderate infusion reactions. In patients receiving OPDIVO monotherapy as a 60-minute infusion,infusion-relatedreactions occurred in 6.4%(127/1994) of patients. In a separate trial in which patients received OPDIVO monotherapy as a 60-minute infusion or a 30-minute infusion, infusion-related reactions occurred in 2.2% (8/368) and 2.7% (10/369) of patients, respectively. Additionally, 0.5% (2/368) and 1.4% (5/369) of patients, respectively, experienced adverse reactions within 48 hours of infusion that led to dose delay, permanent discontinuation or withholding of OPDIVO. In melanoma patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, infusion-related reactions occurred in 2.5% (10/407) of patients. In HCC patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg, infusion-related reactions occurred in 8% (4/49) of patients. In RCC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, infusion-related reactions occurred in 5.1% (28/547) of patients. In MSI-H/dMMR mCRC patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg, infusion-related reactions occurred in 4.2% (5/119) of patients. In MPM patients receiving OPDIVO 3 mg/kg every 2 weeks with YERVOY 1 mg/kg every 6 weeks, infusion-related reactions occurred in 12% (37/300) of patients.

In separate Phase 3 trials of YERVOY 3 mg/kg and 10 mg/kg, infusion-related reactions occurred in 2.9% (28/982).

Complications of Allogeneic Hematopoietic Stem Cell Transplantation

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1 receptor blocking antibody or YERVOY. Transplant-related complications include hyperacute graft-versus-host-disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease (VOD) after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1 or CTLA-4 receptor blockade and allogeneic HSCT.

Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with a PD-1 receptor blocking antibody or YERVOY prior to or after an allogeneic HSCT.

Embryo-Fetal Toxicity

Based on mechanism of action, OPDIVO and YERVOY can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with OPDIVO or YERVOY and for at least 5 months after the last dose.

Increased Mortality in Patients with Multiple Myeloma when OPDIVO is Added to a Thalidomide Analogue and Dexamethasone

In clinical trials in patients with multiple myeloma, the addition of OPDIVO to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with multiple myeloma with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.

Lactation

It is not known whether OPDIVO or YERVOY is present in human milk. Because many drugs, including antibodies, are excreted in human milk and because of the potential for serious adverse reactions in nursing infants from OPDIVO or YERVOY, advise women not to breastfeed during treatment and for at least 5 months after the last dose.

Serious Adverse Reactions

In Checkmate 037, serious adverse reactions occurred in 41% of patients receiving OPDIVO (n=268). Grade 3 and 4 adverse reactions occurred in 42% of patients receiving OPDIVO. The most frequent Grade 3 and 4 adverse drug reactions reported in 2% to <5% of patients receiving OPDIVO were abdominal pain, hyponatremia, increased aspartate aminotransferase, and increased lipase. In Checkmate 066, serious adverse reactions occurred in 36% of patients receiving OPDIVO (n=206). Grade 3 and 4 adverse reactions occurred in 41% of patients receiving OPDIVO. The most frequent Grade 3 and 4 adverse reactions reported in 2% of patients receiving OPDIVO were gamma-glutamyltransferase increase (3.9%) and diarrhea (3.4%). In Checkmate 067, serious adverse reactions (74% and 44%), adverse reactions leading to permanent discontinuation (47% and 18%) or to dosing delays (58% and 36%), and Grade 3 or 4 adverse reactions (72% and 51%) all occurred more frequently in the OPDIVO plus YERVOY arm (n=313) relative to the OPDIVO arm (n=313). The most frequent (10%) serious adverse reactions in the OPDIVO plus YERVOY arm and the OPDIVO arm, respectively, were diarrhea (13% and 2.2%), colitis (10% and 1.9%), and pyrexia (10% and 1.0%). In Checkmate 227, serious adverse reactions occurred in 58% of patients (n=576). The most frequent (2%) serious adverse reactions were pneumonia, diarrhea/colitis, pneumonitis, hepatitis, pulmonary embolism, adrenal insufficiency, and hypophysitis. Fatal adverse reactions occurred in 1.7% of patients; these included events of pneumonitis (4 patients), myocarditis, acute kidney injury, shock, hyperglycemia, multi-system organ failure, and renal failure. In Checkmate 9LA, serious adverse reactions occurred in 57% of patients (n=358). The most frequent (>2%) serious adverse reactions were pneumonia, diarrhea, febrile neutropenia, anemia, acute kidney injury, musculoskeletal pain, dyspnea, pneumonitis, and respiratory failure. Fatal adverse reactions occurred in 7 (2%) patients, and included hepatic toxicity, acute renal failure, sepsis, pneumonitis, diarrhea with hypokalemia, and massive hemoptysis in the setting of thrombocytopenia. In Checkmate 017 and 057, serious adverse reactions occurred in 46% of patients receiving OPDIVO (n=418). The most frequent serious adverse reactions reported in 2% of patients receiving OPDIVO were pneumonia, pulmonary embolism, dyspnea, pyrexia, pleural effusion, pneumonitis, and respiratory failure. In Checkmate 032, serious adverse reactions occurred in 45% of patients receiving OPDIVO (n=245). The most frequent serious adverse reactions reported in 2% of patients receiving OPDIVO were pneumonia, dyspnea, pneumonitis, pleural effusion, and dehydration. In Checkmate 743, serious adverse reactions occurred in 54% of patients receiving OPDIVO plus YERVOY. The most frequent serious adverse reactions reported in 2% of patients were pneumonia, pyrexia, diarrhea, pneumonitis, pleural effusion, dyspnea, acute kidney injury, infusion-related reaction, musculoskeletal pain, and pulmonary embolism. Fatal adverse reactions occurred in 4 (1.3%) patients and included pneumonitis, acute heart failure, sepsis, and encephalitis. In Checkmate 025, serious adverse reactions occurred in 47% of patients receiving OPDIVO (n=406). The most frequent serious adverse reactions reported in 2% of patients were acute kidney injury, pleural effusion, pneumonia, diarrhea, and hypercalcemia. In Checkmate 214, serious adverse reactions occurred in 59% of patients receiving OPDIVO plus YERVOY. The most frequent serious adverse reactions reported in 2% of patients were diarrhea, pyrexia, pneumonia, pneumonitis, hypophysitis, acute kidney injury, dyspnea, adrenal insufficiency, and colitis. In Checkmate 205 and 039, adverse reactions leading to discontinuation occurred in 7% and dose delays due to adverse reactions occurred in 34% of patients (n=266). Serious adverse reactions occurred in 26% of patients. The most frequent serious adverse reactions reported in 1% of patients were pneumonia, infusion-related reaction, pyrexia, colitis or diarrhea, pleural effusion, pneumonitis, and rash. Eleven patients died from causes other than disease progression: 3 from adverse reactions within 30 days of the last OPDIVO dose, 2 from infection 8 to 9 months after completing OPDIVO, and 6 from complications of allogeneic HSCT. In Checkmate 141, serious adverse reactions occurred in 49% of patients receiving OPDIVO (n=236). The most frequent serious adverse reactions reported in 2% of patients receiving OPDIVO were pneumonia, dyspnea, respiratory failure, respiratory tract infection, and sepsis. In Checkmate 275, serious adverse reactions occurred in 54% of patients receiving OPDIVO (n=270). The most frequent serious adverse reactions reported in 2% of patients receiving OPDIVO were urinary tract infection, sepsis, diarrhea, small intestine obstruction, and general physical health deterioration. In Checkmate 142 in MSI-H/dMMR mCRC patients receiving OPDIVO with YERVOY, serious adverse reactions occurred in 47% of patients. The most frequent serious adverse reactions reported in 2% of patients were colitis/diarrhea, hepatic events, abdominal pain, acute kidney injury, pyrexia, and dehydration. In Checkmate 040, serious adverse reactions occurred in 49% of patients receiving OPDIVO (n=154). The most frequent serious adverse reactions reported in 2% of patients were pyrexia, ascites, back pain, general physical health deterioration, abdominal pain, pneumonia, and anemia. In Checkmate 040, serious adverse reactions occurred in 59% of patients receiving OPDIVO with YERVOY (n=49). Serious adverse reactions reported in 4% of patients were pyrexia, diarrhea, anemia, increased AST, adrenal insufficiency, ascites, esophageal varices hemorrhage, hyponatremia, increased blood bilirubin, and pneumonitis. In Checkmate 238, Grade 3 or 4 adverse reactions occurred in 25% of OPDIVO-treated patients (n=452). The most frequent Grade 3 and 4 adverse reactions reported in 2% of OPDIVO-treated patients were diarrhea and increased lipase and amylase. Serious adverse reactions occurred in 18% of OPDIVO-treated patients. In Attraction-3, serious adverse reactions occurred in 38% of patients receiving OPDIVO (n=209). Serious adverse reactions reported in 2% of patients who received OPDIVO were pneumonia, esophageal fistula, interstitial lung disease and pyrexia. The following fatal adverse reactions occurred in patients who received OPDIVO: interstitial lung disease or pneumonitis (1.4%), pneumonia (1.0%), septic shock (0.5%), esophageal fistula (0.5%), gastrointestinal hemorrhage (0.5%), pulmonary embolism (0.5%), and sudden death (0.5%).

Common Adverse Reactions

In Checkmate 037, the most common adverse reaction (20%) reported with OPDIVO (n=268) was rash (21%). In Checkmate 066, the most common adverse reactions (20%) reported with OPDIVO (n=206) vs dacarbazine (n=205) were fatigue (49% vs 39%), musculoskeletal pain (32% vs 25%), rash (28% vs 12%), and pruritus (23% vs 12%). In Checkmate 067, the most common (20%) adverse reactions in the OPDIVO plus YERVOY arm (n=313) were fatigue (62%), diarrhea (54%), rash (53%), nausea (44%), pyrexia (40%), pruritus (39%), musculoskeletal pain (32%), vomiting (31%), decreased appetite (29%), cough (27%), headache (26%), dyspnea (24%), upper respiratory tract infection (23%), arthralgia (21%), and increased transaminases (25%). In Checkmate 067, the most common (20%) adverse reactions in the OPDIVO arm (n=313) were fatigue (59%), rash (40%), musculoskeletal pain (42%), diarrhea (36%), nausea (30%), cough (28%), pruritus (27%), upper respiratory tract infection (22%), decreased appetite (22%), headache (22%), constipation (21%), arthralgia (21%), and vomiting (20%). In Checkmate 227, the most common (20%) adverse reactions were fatigue (44%), rash (34%), decreased appetite (31%), musculoskeletal pain (27%), diarrhea/colitis (26%), dyspnea (26%), cough (23%), hepatitis (21%), nausea (21%), and pruritus (21%). In Checkmate 9LA, the most common (>20%) adverse reactions were fatigue (49%), musculoskeletal pain (39%), nausea (32%), diarrhea (31%), rash (30%), decreased appetite (28%), constipation (21%), and pruritus (21%). In Checkmate 017 and 057, the most common adverse reactions (20%) in patients receiving OPDIVO (n=418) were fatigue, musculoskeletal pain, cough, dyspnea, and decreased appetite. In Checkmate 032, the most common adverse reactions (20%) in patients receiving OPDIVO (n=245) were fatigue (45%), decreased appetite (27%), musculoskeletal pain (25%), dyspnea (22%), nausea (22%), diarrhea (21%), constipation (20%), and cough (20%). In Checkmate 743, the most common adverse reactions (20%) in patients receiving OPDIVO and YERVOY were fatigue (43%), musculoskeletal pain (38%), rash (34%), diarrhea (32%), dyspnea (27%), nausea (24%), decreased appetite (24%), cough (23%), and pruritus (21%). In Checkmate 025, the most common adverse reactions (20%) reported in patients receiving OPDIVO (n=406) vs everolimus (n=397) were fatigue (56% vs 57%), cough (34% vs 38%), nausea (28% vs 29%), rash (28% vs 36%), dyspnea (27% vs 31%), diarrhea (25% vs 32%), constipation (23% vs 18%), decreased appetite (23% vs 30%), back pain (21% vs 16%), and arthralgia (20% vs 14%). In Checkmate 214, the most common adverse reactions (20%) reported in patients treated with OPDIVO plus YERVOY (n=547) were fatigue (58%), rash (39%), diarrhea (38%), musculoskeletal pain (37%), pruritus (33%), nausea (30%), cough (28%), pyrexia (25%), arthralgia (23%), decreased appetite (21%), dyspnea (20%), and vomiting (20%). In Checkmate 205 and 039, the most common adverse reactions (20%) reported in patients receiving OPDIVO (n=266) were upper respiratory tract infection (44%), fatigue (39%), cough (36%), diarrhea (33%), pyrexia (29%), musculoskeletal pain (26%), rash (24%), nausea (20%) and pruritus (20%). In Checkmate 141, the most common adverse reactions (10%) in patients receiving OPDIVO (n=236) were cough and dyspnea at a higher incidence than investigators choice. In Checkmate 275, the most common adverse reactions (20%) reported in patients receiving OPDIVO (n=270) were fatigue (46%), musculoskeletal pain (30%), nausea (22%), and decreased appetite (22%). In Checkmate 142 in MSI-H/dMMR mCRC patients receiving OPDIVO as a single agent, the most common adverse reactions (20%) were fatigue (54%), diarrhea (43%), abdominal pain (34%), nausea (34%), vomiting (28%), musculoskeletal pain (28%), cough (26%), pyrexia (24%), rash (23%), constipation (20%), and upper respiratory tract infection (20%). In Checkmate 142 in MSI-H/dMMR mCRC patients receiving OPDIVO with YERVOY, the most common adverse reactions (20%) were fatigue (49%), diarrhea (45%), pyrexia (36%), musculoskeletal pain (36%), abdominal pain (30%), pruritus (28%), nausea (26%), rash (25%), decreased appetite (20%), and vomiting (20%). In Checkmate 040, the most common adverse reactions (20%) in patients receiving OPDIVO (n=154) were fatigue (38%), musculoskeletal pain (36%), abdominal pain (34%), pruritus (27%), diarrhea (27%), rash (26%), cough (23%), and decreased appetite (22%). In Checkmate 040, the most common adverse reactions (20%) in patients receiving OPDIVO with YERVOY (n=49), were rash (53%), pruritus (53%), musculoskeletal pain (41%), diarrhea (39%), cough (37%), decreased appetite (35%), fatigue (27%), pyrexia (27%), abdominal pain (22%), headache (22%), nausea (20%), dizziness (20%), hypothyroidism (20%), and weight decreased (20%). In Checkmate 238, the most common adverse reactions (20%) reported in OPDIVO-treated patients (n=452) vs ipilimumab-treated patients (n=453) were fatigue (57% vs 55%), diarrhea (37% vs 55%), rash (35% vs 47%), musculoskeletal pain (32% vs 27%), pruritus (28% vs 37%), headache (23% vs 31%), nausea (23% vs 28%), upper respiratory infection (22% vs 15%), and abdominal pain (21% vs 23%). The most common immune-mediated adverse reactions were rash (16%), diarrhea/colitis (6%), and hepatitis (3%). In Attraction-3, the most common adverse reactions occurring in 20% of OPDIVO-treated patients (n=209) were rash (22%) and decreased appetite (21%).

In a separate Phase 3 trial of YERVOY 3 mg/kg, the most common adverse reactions (5%) in patients who received YERVOY at 3 mg/kg were fatigue (41%), diarrhea (32%), pruritus (31%), rash (29%), and colitis (8%).

Please see U.S. Full Prescribing Information for OPDIVO and YERVOY.

Checkmate Trials and Patient Populations

Checkmate 037previously treated metastatic melanoma; Checkmate 066previously untreated metastatic melanoma; Checkmate 067previously untreated metastatic melanoma, as a single agent or in combination with YERVOY; Checkmate 227previously untreated metastatic non-small cell lung cancer, in combination with YERVOY; Checkmate 9LApreviously untreated recurrent or metastatic non-small cell lung cancer in combination with YERVOY and 2 cycles of platinum-doublet chemotherapy by histology; Checkmate 017second-line treatment of metastatic squamous non-small cell lung cancer; Checkmate 057second-line treatment of metastatic non-squamous non-small cell lung cancer; Checkmate 032small cell lung cancer; Checkmate 743 previously untreated unresectable malignant pleural mesothelioma, in combination with YERVOY; Checkmate 025previously treated renal cell carcinoma; Checkmate 214previously untreated renal cell carcinoma, in combination with YERVOY; Checkmate 205/039classical Hodgkin lymphoma; Checkmate 141recurrent or metastatic squamous cell carcinoma of the head and neck; Checkmate 275urothelial carcinoma; Checkmate 142MSI-H or dMMR metastatic colorectal cancer, as a single agent or in combination with YERVOY; Checkmate 040hepatocellular carcinoma, as a single agent or in combination with YERVOY; Checkmate 238adjuvant treatment of melanoma; Attraction-3esophageal squamous cell carcinoma

CABOMETYX Important Safety Information

Warnings and Precautions

Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients in RCC and HCC studies. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.

Perforations and Fistulas: Gastrointestinal (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of perforations and fistulas, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a Grade 4 fistula or a GI perforation.

Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic event requiring medical intervention.

Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension occurred in 36% (17% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.

Diarrhea: Diarrhea occurred in 63% of CABOMETYX patients. Grade 3 diarrhea occurred in 11% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 diarrhea, Grade 3 diarrhea that cannot be managed with standard antidiarrheal treatments, or Grade 4 diarrhea.

Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 44% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.

Proteinuria: Proteinuria occurred in 7% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. Discontinue CABOMETYX in patients who develop nephrotic syndrome.

Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 3 weeks prior to scheduled dental surgery or invasive dental procedures, if possible. Withhold CABOMETYX for development of ONJ until complete resolution.

Original post:
U.S. Food and Drug Administration Accepts for Priority Review Applications for OPDIVO (nivolumab) in Combination with CABOMETYX (cabozantinib) in...

The Office of Research and the School of Medicine had planned to introduce their Oct. 30 speaking guest as a professor and the founder and director of the Innovative Genomics Institute at UC Berkeley, and a CRISPR pioneer.

Since being booked for the Distinguished Speaker Series in Research and Innovation, however, Jennifer Doudna has added a new title: Nobel laureate.

She and Emmanuelle Charpentier, director of the Max Planck Institute for Infection Biology, won the Nobel Prize in chemistry Oct. 7 for their co-development of CRISPR-Cas9, a genome editing tool that has revolutionized biomedicine and agriculture.

Whats CRISPR? Jennifer Doudna explains in a Radiolab podcast.

Doudna became the first woman on the UC Berkeley faculty to win a Nobel, and she and Charpentier are the first women to share a Nobel in the sciences.

Doudnas topic for her UC Davis talk is CRISPR and Coronavirus.

UC Davis Healths Ralph Green, distinguished professor in the Department of Pathology and Laboratory Medicine, and medical director of UC Davis Diagnostics, recently collaborated with Doudna and others on a project to set up COVID-19 testing for UC Berkeley and the surrounding community and Green is helping with a similar project at UC Davis.

I had the good fortune to get to know Jennifer Doudna through my interaction with her group when they turned their skills and knowledge to setting up, at remarkable speed, a pop-up, PCR-based test for SARS-CoV-2 during the early days of the COVID-19 pandemic when the country was scrambling to meet the need for more testing, Green said.

I have to say that it has been a singular pleasure and privilege for me to interact with Jennifer Doudna and her colleagues.

CRISPR-Cas9 genetic engineering technology enables scientists to change or remove genes quickly and with great precision. Labs worldwide have redirected their research programs to incorporate this new tool, creating a CRISPR revolution with huge implications across biology and medicine.

Doudna is a leader in public discussion of the ethical implications of genome editing for human biology and societies. She advocates for thoughtful approaches to the development of policies around the use of CRISPR-Cas9.

Follow Dateline UC Davis on Twitter.

See the article here:
'CRISPR and Coronavirus': Hear From Nobel Winner Jennifer Doudna - UC Davis

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerinks Geoff Porges has been running the numbers on Pfizers vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

Unlock this story instantly and join 92,200+ biopharma pros reading Endpoints daily and it's free.

SUBSCRIBE SIGN IN

Read the rest here:
UPDATED: CRISPR Therapeutics gets a snapshot of off-the-shelf CAR-T success in B-cell malignancies marred by the death of a patient - Endpoints News

Its been a good year for Intellia: One of its founders, Jennifer Doudna, Ph.D., nabbed the Nobel Prize in Chemistry for her CRISPR research.

Now, the biotech she helped build is putting that to work, saying it now plans the worlds first clinical trial for asingle-course therapy that potentially halts and reverses a condition known as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

This genetic disorder occurs when a person is born with a specific DNA mutation in the TTR gene, which causes the liver to produce a protein called transthyretin (TTR) in a misfolded form and build up in the body.

How to Streamline Your Clinical Research Organization's Processes End to End

Learn how implementing one platform leads to data consistency and ultimately facilitate faster clinical trials while reducing overall trial costs, leave behind spreadsheets and home-grown tools for a predictable trial and the ability to forecast unit delivery resulting in the optics you need to ensure a successful trial, and hear experts share industry trends of what is affecting the Clinical Research Organization industry today.

hATTR can manifest as polyneuropathy (hATTR-PN), which can lead to nerve damage, or cardiomyopathy (hATTR-CM), which involves heart muscle disease that can lead to heart failure.

This disorder has seen a lot of interest in recent years, with an RNAi approach from Alnylam seeing an approval for Onpattro a few years back, specifically for hATTR in adults with damage to peripheral nerves.

Ionis Pharmaceuticals and its rival RNAi drug Tegsedi also saw an approval in 2018 for a similar indication.

They both battle with Pfizers older med tafamidis, which has been approved in Europe for years in polyneuropathy, and the fight could spread to the U.S. soon.

The drug, now marketed as Vyndaqel and Vyndamax, snatched up an FDA nod last May to treat both hereditary and wild-type ATTR patients with a heart condition called cardiomyopathy.

While coming into an increasingly crowed R&D area, Intellia is looking for a next-gen approach, and has been given the go-ahead by regulators ion the U.K, to start a phase 1 this year.

The idea is for Intellias candidate NTLA-2001, which is also partnered with Regeneron, to go beyond its rivals and be the first curative treatment for ATTR.

By applying the companys in vivo liver knockout technology, NTLA-2001 allows for the possibility of lifelong transthyretin (TTR) protein reduction after a single course of treatment. If this works, this could in essence cure patients of the their disease.

The 38-patient is set to start by years end.

Starting our global NTLA-2001 Phase 1 trial for ATTR patients is a major milestone in Intellias mission to develop medicines to cure severe and life-threatening diseases, said Intellias president and chief John Leonard, M.D.

Our trial is the first step toward demonstrating that our therapeutic approach could have a permanent effect, potentially halting and reversing all forms of ATTR. Once we have established safety and the optimal dose, our goal is to expand this study and rapidly move to pivotal studies, in which we aim to enroll both polyneuropathy and cardiomyopathy patients."

Continue reading here:
Intellia to kick-start first single-course 'curative' CRISPR shot, as it hopes to beat rivals Alnylam, Ionis and Pfizer - FierceBiotech

In a study published [September 21] in theNature Biomedical Engineeringjournal, the researchers said they usedCRISPRCas9 and a combination of other genetic technologies to inactivate porcine endogenous retroviruses (PERVs), a group of viruses that could be dangerous to humans, while also enhancing the pigs immunological and blood-coagulation compatibility with humans, which could reduce the risk of rejection by organ recipients.

The engineered pigs exhibited normal physiology, fertility and transmission of the edited genes to their offspring, according to the paper.

Transplants from pigs have long been investigated as a solution to the global shortage of human organs for patients with organ failure, for reasons such as the size of their organs similar enough to those of humans and their relatively short maturity period of about six months.

The risks of organ rejection due to the biological incompatibility of pig organs with human bodies and of transmitting PERVs have limited the clinical applicability of such transplants, but advancements in gene-editing technology have given researchers new hope.

George Church, one of the authors from Harvard Medical School and a co-founder of Hangzhou-based Qihan Bio, was quoted by Chinese news agency Xinhua as saying that if the technology used can be further verified in future research, it could help alleviate the global shortage of human organs to a large extent.

Read the original post

Read more here:
CRISPR has brought pig-to-human organ transplants to the cusp of reality - Genetic Literacy Project

The global CRISPR Cas9 Industry Market is carefully researched in the report while largely concentrating on top players and their business tactics, geographical expansion, market segments, competitive landscape, manufacturing, and pricing and cost structures. Each section of the research study is specially prepared to explore key aspects of the global CRISPR Cas9 Industry market. For instance, the market dynamics section digs deep into the drivers, restraints, trends, and opportunities of the global CRISPR Cas9 Industry market. With qualitative and quantitative analysis, we help you with thorough and comprehensive research on the global CRISPR Cas9 Industry market. We have also focused on SWOT, PESTLE, and Porters Five Forces analyses of the global CRISPR Cas9 Industry market.

The research report on CRISPR Cas9 Industry market elaborates on the major trends defining the industry growth with regards to the regional terrain and competitive scenario. The document also lists out the limitations & challenges faced by industry participants alongside information such as growth opportunities. Apart from this, the report contains information regarding the impact of COVID-19 pandemic on the overall market outlook.

Key insights from COVID-19 impact analysis:

Request Sample Copy of this Report @ https://www.express-journal.com/request-sample/228075

An overview of regional landscape:

Additional information from the CRISPR Cas9 Industry market report:

Table of Contents

Request Customization on This Report @ https://www.express-journal.com/request-for-customization/228075

Visit link:
CRISPR Cas9 Industry Market Overview, Environmental Analysis and Forecast to 2025 - Express Journal

A researcher observes the process of genetic scissors in a laboratory in Berlin (Germany). (GREGOR FISCHER / DPA)

The genetic scissors, called Crispr-Case 9 discovered almost ten years ago, are actually genetic code scissors. They make it possible to separate two strands of DNA and replace or delete a gene. When American and French scientists Jennifer Doudna and Emmanuelle Charpentier made this discovery, it paved the way for much research. Today, American, European and Australian scientists are meeting in videoconference to take stock of their progress.

For example, Crispr is making great progress in research into gene therapy. Today, this technique makes it possible to treat people with Duchenne muscular dystrophy, one of the genetic diseases often mentioned during the Telethon. Inserm was also able to reactivate a gene to fight against sickle cell anemia: a blood disease linked again to a genetic problem. Moreover, the company eGenesis works on pigs free of viruses dangerous to humans. The animals would then become perfect organ donors for patients awaiting transplants of the heart, pancreas, etc.

The Crispr technique has developed in many laboratories around the world and two years ago a Chinese researcher, He Jiankui, caused a scandal after announcing that he had used it to create, through in vitro fertilization, two GMO babies resistant to HIV whose father was a carrier. According to a survey, in the MIT Technology Review, the researcher forced the hand of parents who saw in this genetic manipulation the only way to have children without risk. In addition, today it is not known how babies are doing and what other genetic consequences the use of Crispr has had on them. The researcher is still under house arrest in China.

Emmanuelle Charpentier in an interview on point obviously criticizes the failure to respect ethical criteria for the use of its scissors applied to research for humans. On the other hand, she does not share the doubts of peasant and environmental associations on plants modified by Crispr-Cas 9 and considered as GMOs in Europe. For her, her innovation makes it possible to boost a plant gene, to reproduce in an accelerated manner what can happen in nature. Its not like creating mutant plants with a gene that comes from other species.

Applied to theAgriculture, Crispr-Cas 9 makes it possible to create allergen-free peanuts, gluten-free wheat, more drought-resistant rice, but also to remove the horns of cows or to herd only males. Crispr-Cas 9 therefore asks even more to meditate on Rabelais sentence: Science without consciousness is nothing but the ruin of the soul.

See the original post:
Advances and concerns raised by Crispr-Cas 9, the genetic scissors - Pledge Times

Chemistry Nobel Prize award recipients Jennifer A. Doudna and Emmanuelle Charpentier have joined the ranks of Marie Curie, Frances Arnold, Ada E. Yonath and Dorothy Crowfoot Hodgkin.J.L. Cereijido/EPA

THE Royal Swedish Academy of Sciences yesterday awarded the 2020 Nobel Prize in Chemistry to Emmanuelle Charpentier and Jennifer Doudna for their work on CRISPR, a method of genome editing.

A genome is the full set of genetic instructions that determine how an organism will develop. Using CRISPR, researchers can cut up DNA in an organisms genome and edit its sequence.

CRISPR technology is a powerhouse for basic research and is also changing the world we live in. There are thousands of research papers published every year on its various applications.

These include accelerating research into cancers, mental illness, potential animal to human organ transplants, better food production, eliminating malaria-carrying mosquitoes and saving animals from disease.

Charpentier is the director at the Max Planck Institute for Infection Biology in Berlin, Germany and Doudna is a professor at the University of California, Berkeley. Both played a crucial role in demonstrating how CRISPR could be used to target DNA sequences of interest.

Read more:Why more women dont win science Nobels

CRISPR technology is adapted from a system that is naturally present in bacteria and other unicellular organisms known as archaea.

This natural system gives bacteria a form of acquired immunity. It protects them from foreign genetic elements (such as invading viruses) and lets them remember these in case they reappear.

Like most advances in modern science, the discovery of CRISPR and its emergence as a key genome editing method involved efforts by many researchers, over several decades.

In 1987, Japanese molecular biologist Yoshizumi Ishino and his colleagues were the first to notice, in E. coli bacteria, unusual clusters of repeated DNA sequences interrupted by short sequences.

Spanish molecular biologist Francisco Mojica and colleagues later showed similar structures were present in other organisms and proposed to call them CRISPR: Clustered Regularly Interspaced Short Palindromic Repeats.

In 2005, Mojica and other groups reported the short sequences (or spacers) interrupting the repeats were derived from other DNA belonging to viruses.

Evolutionary biologists Kira Makarova, Eugene Koonin and colleagues eventually proposed CRISPR and the associated Cas9 genes were acting as the immune mechanism. This was experimentally confirmed in 2007 by Rodolphe Barrangou and colleagues.

The CRISPR-associated genes, Cas9, encode a protein that cuts DNA. This is the active part of the defence against viruses, as it destroys the invading DNA.

In 2012, Charpentier and Doudna showed the spacers acted as markers that guided where Cas9 would make a cut in the DNA. They also showed an artificial Cas9 system could be programmed to target any DNA sequence in a lab setting.

This was a groundbreaking discovery which opened the door for CRISPRs wider applications in research.

In 2013, for the first time, groups led by American biochemist Feng Zhang and geneticist George Church reported genome editing in human cell cultures using CRISPR-Cas9. It has since been used in countless organisms from yeast to cows, plants and corals.

Today, CRISPR is the preferred gene-editing tool for thousands of researchers.

Humans have altered the genomes of species for thousands of years. Initially, this was through approaches such as selective breeding.

However, genetic engineering the direct manipulation of DNA by humans outside of breeding and mutations has only existed since the 1970s.

CRISPR-based systems fundamentally changed this field, as they allow for genomes to be edited in living organisms cheaply, with ease and with extreme precision.

CRISPR is currently making a huge impact in health. There are clinical trials on its use for blood disorders such as sickle cell disease or beta-thalassemia, for the treatment of the most common cause of inherited childhood blindness (Leber congenital amaurosis) and for cancer immunotherapy.

CRISPR also has great potential in food production. It can be used to improve crop quality, yield, disease resistance and herbicide resistance.

Used on livestock, it can lead to better disease resistance, increased animal welfare and improved productive traits that is, animals producing more meat, milk or high-quality wool.

A number of challenges to the technology remain, however. Some are technical, such as the risk of off-target modifications (which happen when Cas9 cuts at unintended locations in the genome).

Other problems are societal. CRISPR was famously used in one of the most controversial experiments of recent years.

Read more:Why we need a global citizens assembly on gene editing

Chinese biophysicist He Jiankui unsuccessfully attempted to use the technology to modify human embryos and make them resistant to HIV (human immunodeficiency virus). This led to the birth of twins Lulu and Nana.

We need a broad and inclusive discussion on the regulation of such technologies especially given their vast applications and potential.

To quote CRISPR researcher Fyodor Urnov, Charpentier and Doudnas work really has changed everything.

Dimitri Perrin, Senior Lecturer, Queensland University of Technology

This article is republished from The Conversation under a Creative Commons license. Read the original article.

See the original post:
What is CRISPR, the gene editing technology that won the Chemistry Nobel prize? - Grain Central

As we know, COVID-19 is causing large scale loss of life and severe human suffering. With the pandemic spreading across the globe, researchers are racing against the clock to develop diagnostic tools, vaccines and treatments. Recently, WHO has launched a Solidarity clinical trial to assess relative effectiveness of four potential drugs against COVID-19. Further, there are close to 40 clinical trials of vaccines are ongoing, however, as per experts, it may take more than a year to develop a vaccine.

In order to enhance COVID-19 drug discovery and develop rapid testing kits, various academic institutes, non-profit institutes, scientific pioneers and biopharmaceutical companies have also been leveraging benefits of CRISPR technology.

For more Insights Click under

CRISPR Can be a Solution to Address the COVID-19 Pandemic

The CRISPR / Cas9 system has revolutionized the field of genetic engineering. It enables researchers to alter the genomes of a range of organisms with relative ease. Currently, it has emerged as a promising tool that is used extensively for editing genomes and for the development of novel treatment options. CRISPR is popularly known as search engine for biology, as it has emerged as a location finder, rather than site specific cleavage tool. The figure below highlights the key potential areas and benefits of CRISPR in order to fight against novel coronavirus.

Rapid and Economical Diagnostic Tests

Presently, COVID-19 testing capacity is limited by a number of factors, such as requirements for complex procedures, need for laboratory instrumentation, and dependence on limited supplies. Therefore, there is an urgent need for rapid detection kits. CRISPR has been explored by scientists for diagnosis of infectious diseases. The underlying mechanism involves binding of guide RNA with a protein of Cas family which cuts the target and shreds the nearby RNA or DNA. When CRISPR hits a target, the reporter molecule releases a fluorescent signal. This is further analysed by paper tests dipped into a patient sample, such as blood, urine, or saliva, which further shows up as a line on the testing strip. Researchers have been utilizing CRISPR-based tools and technologies to detect RNA of virus in patient samples. Sherlock Biosciences has already made history, as it received Emergency Use Authorization (EUA) from the US Food and Drug Administration (FDA) for its Sherlock CRISPR SARS-CoV-2 kit for the detection of the virus that causes COVID-19. The kit is designed for use in laboratories and can provide results within an hour. The company claims that more than 1 million tests can be performed within a week.

Enhancing Drug Discovery

CRISPR technology aids in the study of interaction of virus with human cells. This enables the generation of appropriate cell models for faster discovery of new potential treatment options, or identification of an existing drug combination that may provide a treatment solution. For instance, researchers are exploring molecular mechanisms of the novel virus by utilizing CRISPR technology, which can ultimately assist in identifying potential drug combinations.

CRISPR-based COVID-19 Therapy

Researchers at Stanford University have been working on the development of a gene targeting anti-viral agent against COVID-19, using PAC-MAN technology. The technology has been modified to be used against the deadly virus. It consists of a virus-killing enzyme, such as Cas13 and a guide RNA, which commands Cas13 to destroy specific nucleotide sequences in the coronaviruss genome. Based on several studies, it has been revealed that PAC-MAN has the ability to neutralize the coronavirus and stop it from replicating inside cells. Based on information available, work is currently ongoing, and researchers are finding a solution to deliver this technology to lung cells. Multiple delivery methods are currently under evaluation.

A lot of companies are currently active in providing CRISPR-based genome engineering services. To get a detailed information on the key players, recent developments, and the likely market evolution.

For more Insights Click under

CRISPR Can be a Solution to Address the COVID-19 Pandemic

You may also be interested in the following titles:

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

See the rest here:
CRISPR Can be a Solution to Address the COVID-19 Pandemic | Roots Analysis - Eurowire

In a report released today, Raju Prasad from William Blair maintained a Buy rating on Crispr Therapeutics AG (CRSP). The companys shares closed last Wednesday at $94.30.

According to TipRanks.com, Prasad is a 5-star analyst with an average return of 13.4% and a 53.9% success rate. Prasad covers the Healthcare sector, focusing on stocks such as Global Blood Therapeutics, Alexion Pharmaceuticals, and Rocket Pharmaceuticals.

Currently, the analyst consensus on Crispr Therapeutics AG is a Strong Buy with an average price target of $95.88, implying a 5.1% upside from current levels. In a report released today, Needham also assigned a Buy rating to the stock with a $105.00 price target.

See todays analyst top recommended stocks >>

The company has a one-year high of $111.90 and a one-year low of $32.30. Currently, Crispr Therapeutics AG has an average volume of 870.1K.

Based on the recent corporate insider activity of 41 insiders, corporate insider sentiment is neutral on the stock.

TipRanks has tracked 36,000 company insiders and found that a few of them are better than others when it comes to timing their transactions. See which 3 stocks are most likely to make moves following their insider activities.

CRISPR Therapeutics AG engages in the development and commercialization of therapies derived from genome-editing technology. Its proprietary platform CRISPR/Cas9-based therapeutics allows for precise and directed changes to genomic DNA. The company was founded by Rodger Novak, Emmanuelle Charpentier, Shaun Patrick Foy, Matthew Porteus, Daniel Anderson, Chad Cowan and Craig Mellow in 2014 and is headquartered in Zug, Switzerland.

View post:
William Blair Sticks to Its Buy Rating for Crispr Therapeutics AG (CRSP) - Smarter Analyst

CRISPR And CRISPR-Associated (Cas) Genes Market report would come handy to understand the competitors in the market and give an insight into sales, volumes, revenues in the CRISPR And CRISPR-Associated (Cas) Genes Industry & will also assists in making strategic decisions. The report also helps to decide corporate product & marketing strategies. It reduces the risks involved in making decisions as well as strategies for companies and individuals interested in the CRISPR And CRISPR-Associated (Cas) Genes industry. Both established and new players in CRISPR And CRISPR-Associated (Cas) Genes industries can use the report to understand the CRISPR And CRISPR-Associated (Cas) Genes market.

In Global Market, the Following Companies Are Covered:

Get a Sample Copy of the Report @ https://www.360marketupdates.com/enquiry/request-sample/14841056

Analysis of the Market:

Clustered regularly interspaced short palindromic repeats (CRISPR) are segments of prokaryotic DNA containing short repetitions of base sequences. Each repetition is followed by short segments of spacer DNA from previous exposures to a bacteriophage virus or plasmid.

The CRISPR/Cas system is a prokaryotic immune system that confers resistance to foreign genetic elements such as those present within plasmids and phages, and provides a form of acquired immunity. CRISPR associated proteins (Cas) use the CRISPR spacers to recognize and cut these exogenous genetic elements in a manner analogous to RNA interference in eukaryotic organisms. CRISPRs are found in approximately 40% of sequenced bacterial genomes and 90% of sequenced archaea.

CRISPR And CRISPR-Associated (Cas) Genes industry is relatively concentrated, manufacturers are mostly in the Europe and North America. Among them, North America region accounted for more than 45.70% of the total market of global CRISPR And CRISPR-Associated (Cas) Genes.

The global CRISPR And CRISPR-Associated (Cas) Genes market is valued at 713.8 million USD in 2020 is expected to reach 7696.7 million USD by the end of 2026, growing at a CAGR of 40.0% during 2021-2026.

This report focuses on CRISPR And CRISPR-Associated (Cas) Genes volume and value at the global level, regional level and company level. From a global perspective, this report represents overall CRISPR And CRISPR-Associated (Cas) Genes market size by analysing historical data and future prospect. Regionally, this report focuses on several key regions: North America, Europe, China and Japan et

CRISPR And CRISPR-Associated (Cas) Genes Market Breakdown by Types:

CRISPR And CRISPR-Associated (Cas) Genes Market Breakdown by Application:

Critical highlights covered in the Global CRISPR And CRISPR-Associated (Cas) Genes market include:

The information available in the CRISPR And CRISPR-Associated (Cas) Genes Market report is segmented for proper understanding. The Table of contents contains Market outline, Market characteristics, Market segmentation analysis, Market sizing, customer landscape & Regional landscape. For further improving the understand ability various exhibits (Tabular Data & Pie Charts) has also been used in the CRISPR And CRISPR-Associated (Cas) Genes Market report.

Get a Sample Copy of the Report @ https://www.360marketupdates.com/enquiry/request-sample/14841056

Reasons for Buy CRISPR And CRISPR-Associated (Cas) Genes Market Report:

In the end, CRISPR And CRISPR-Associated (Cas) Genes Industry report provides the main region, market conditions with the product price,profit, capacity, production, supply, demand and market growth rateand forecast etc. This report also Present newproject SWOT analysis,investment feasibility analysis, andinvestment return analysis.

Contact Us:

Name: Mr. Ajay More

Email: [emailprotected]

Organization: 360 Market Updates

Phone: +14242530807 / + 44 20 3239 8187

Global Shortening Market Size in 2020 (New Report): Manufacturers Data, Opportunity, Import Export Scenario, Application, Showing Impressive Growth by 2026

Swimwear and Beachwear Market Size 2020-2026 Research Report by Key Companies, Future Trend, Pipeline Projects, Product, Application, Growth and Regional Forecasts

Trunking System Market Size 2020 : Business Opportunity, SWOT Analysis, Applications, Trends and Forecast to 2026 Research Reportby360marketupdates

Global Acraldehyde Market 2020 Industry Analysis, Size, Share, Trends, Market Demand, Growth, Opportunities and Showing Impressive Growth by 2026

Medical Robotic Systems Market Size 2020-2026 Industry Trends, Size, Segments, Competitors Classification, Growth, Up and Down Stream Industry Analysis and Forecast

Solid of Sodium Methylate Market Size, Growth Opportunities, Emerging Technologies, Trends,Growth, Segments, Landscape and Demandby usingForecast to 2026 Research Reportvia360marketupdates

Go here to read the rest:
CRISPR And CRISPR-Associated (Cas) Genes Market 2020 to Flourish with an Impressive CAGR of XX% in the year 2026, Market Size & Growth with...

Report is a detailed study of the CRISPR and CAS Gene market, which covers all the essential information required by a new market entrant as well as the existing players to gain a deeper understanding of the market. The primary objective of this research report named CRISPR and CAS Gene market is to help making reliable strategic decisions regarding the opportunities in CRISPR and CAS Gene market.

Major Market Players with an in-depth analysis:

Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Request Free Sample Copy of CRISPR and CAS Gene Market Research [emailprotected]:https://www.coherentmarketinsights.com/insight/request-sample/2598

The Global CRISPR and CAS Gene Market segments and Market Data Break Down are illuminated below:

By Product Type:Vector-based CasDNA-free CasGlobal CRISPR and CAS Gene Market, By Application:Genome EngineeringDisease modelsFunctional GenomicsKnockdown/activationOther Applications

The CRISPR and CAS Gene market report offers the current state of the market around the world. The report starts with the market outline and key of the CRISPR and CAS Gene market which assumes a significant job for clients to settle on the business choice. It additionally offers the key focuses to upgrade the development in the CRISPR and CAS Gene market. Some fundamental ideas are likewise secured by reports, for example, item definition, its application, industry esteem chain structure and division which help the client to break down the market without any problem. Also, the report covers different factors, for example, arrangements, efficient and innovative which are affecting the CRISPR and CAS Gene business and market elements.

Download Sample Report of CRISPR and CAS Gene Market Report 2020 (Coronavirus Impact Analysis on CRISPR and CAS Gene Market)

Competitive Analysis has been done to understand overall market which will be helpful to take decisions. Major players involved in the manufacture of CRISPR and CAS Gene product has been completely profiled along with their SWOT. Some of the key players include Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc. It helps in understanding their strategy and activities. Business strategy described for every company helps to get idea about the current trends of company. The industry intelligence study of the CRISPR and CAS Gene market covers the estimation size of the market each in phrases of value (Mn/Bn USD) and volume (tons). Report involves detailed chapter on COVID 19 and its impact on this market. Additionally, it involves changing consumer behavior due to outbreak of COVID 19.

Further, report consists of Porters Five Forces and BCG matrix as well as product life cycle to help you in taking wise decisions. Additionally, this report covers the inside and out factual examination and the market elements and requests which give an entire situation of the business.

Regional Analysis for CRISPR and CAS Gene

Any query? Enquire Here For Discount (COVID-19 Impact Analysis Updated Sample):https://www.coherentmarketinsights.com/insight/request-discount/2598

Chapters Define in TOC (Table of Content) of the Report:

Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation

Overview

Chapter 2: COVID Impact

Chapter 3: Market Competition by Manufacturers

Chapter 4: Production by Regions

Chapter 5: Consumption by Regions

Chapter 6: Production, By Types, Revenue and Market share by Types

Chapter 7: Consumption, By Applications, Market share (%) and Growth Rate by

Applications

Chapter 8: Complete profiling and analysis of Manufacturers

Chapter 9: Manufacturing cost analysis, Raw materials analysis, Region-wise

Manufacturing expenses

Chapter 10: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 11: Marketing Strategy Analysis, Distributors/Traders

Chapter 12: Market Effect Factors Analysis

Chapter 13: Market Forecast

Chapter 14: CRISPR and CAS Gene Research Findings and Conclusion, Appendix, methodology and data source

DOWNLOAD FREE SAMPLE [emailprotected]:https://www.coherentmarketinsights.com/insight/request-sample/2598

The qualitative contents for geographical analysis will cover market trends in each region and country which includes highlights of the key players operating in the respective region/country, PEST analysis of each region which includes political, economic, social and technological factors influencing the growth of the market. The research report includes specific segments by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2020 to 2027.

About Us:

Coherent Market Insightsis a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

Contacts Us: +1-206-701-6702Email: [emailprotected]Web: https://www.coherentmarketinsights.com/

**********Download the Entire Report*************************************************

https://www.coherentmarketinsights.com/market-insight/crispr-and-cas-gene-market-2598

Go here to read the rest:
Covid-19 Impact On CRISPR and CAS Gene Market Study: An Emerging Hint of Opportunity Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC,...

Share this Article

You are free to share this article under the Attribution 4.0 International license.

New generation CRISPR technology could pave the way for therapeutics to treat inherited retina diseases, researchers report.

In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function, says Krzysztof Palczewski, chair and a professor in the Gavin Herbert Eye Institutes ophthalmology department at the University of California, Irvine School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, says first author Susie Suh, assistant specialist in the ophthalmology department.

We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, says co-first author Elliot Choi, also an assistant specialist in the ophthalmology department.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the researchers successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases.

Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast, and spatial and temporal frequency, says Palczewski.

These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases.

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status, and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy.

Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

The new paper appears in Nature Biomedical Engineering.

Support for the research came from the National Institutes of Health; the Research to Prevent Blindness Stein Innovation Award; Fight for Sight; the Eye and Tissue Bank Foundation (Finland); the Finnish Cultural Foundation; the Orion Research Foundation; the Helen Hay Whitney Foundation; US Department of Veterans Affairs; and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

Source: UC Irvine

The rest is here:
Therapy restores vision in mice with retina disease - Futurity: Research News

Here are some events that caught our eye, either because of their nod to current events or their unique nature. But really, you'll want to peruse the schedule yourself as the options are seemingly endless and you can customize your search to your interests and age range. Also, you must register (it's free!) to see full details of each event.

Events That Take You Outside

Interactive Bird Scavenger Hunt

If you've ever wanted to get into birding but find the Sibley guides overwhelming, this session is for you. Download the scavenger hunt card and join other aspiring ornithologists in observing local birds and their behavior.

Share the Night Sky

What do you get when you mix a radio show and a planetary show? This show features San Franciscos Urban Astronomer Paul Salazar and KPOO's DJ Marilynn. Tune in for a guided tour of the night sky in real time.

Events for Big Thinkers

Astronomy Talks: Quantum Mechanics vs General Relativity: Clash of the Titans

Join a discussion about unifying the two theories and creating "a single theory that describes the entire Universe." Doesn't get much bigger picture than that, now, does it?

CRISPR, Sickle Cell Disease, and Society: A VR Explainer and Ethics Discussion

Explore how CRISPR genome editing may be used to repair the sickle cell mutation. See a virtual reality tour of the human body, and a talk on health disparities in the U.S. and the ethics of CRISPR.

N ~ 1: Alone in the Milky Way

Dr. Pascal Lee, a planetary scientist who works at the Mars Institute, the SETI Institute and the Haughton-Mars Project at the NASA Ames Research Center, makes the case that "we might be it, in the vastness of our galaxy."

Events for the Foodies (or the Science-Reluctant)

The Science of Sundaes

You had me at ice cream scientist. Dr. Maya Warren will teach families how to make their own sweet treats at home and discuss her career as a "real-life ice cream scientist"!

The Science Behind Hand-Pulled Noodles

Members of the Stanford Polymer Collective will make a basic dough and explain how additives like oil, water and salt change the dough's mechanical properties. Participants are encouraged to follow along at home and decide which dough makes the best hand-pulled noodles.

Events for Climate Activists

Bay Area Youth Climate Activism Panel

Get inspired and learn from the young people in the Bay Area who are trying to save our ecosystems.

Wetland Protectors: Our First Line of Defense

Save the Bay will demonstrate why tidal marshlands and native plants are key to flood protection for bayside communities threatened by climate change.

Events at the Frontline of the Pandemic

Behind the Scenes at a COVID-19 Diagnostic Testing Center

You ever wonder what happens after the nose and throat swabs? We'll go inside a lab that's working with UCSF on COVID-19 testing. "Follow a sample from its arrival at the lab, through RNA extraction and amplification, to the end result to reveal if it is positive or negative for the virus."

Exploring Connections Between Cancer and COVID-19

Talk to researchers from the UCSF Cancer Cell Map Initiative (CCMI) and Quantitative Biology Institute Coronavirus Research Group (QBI-QCRG) about the "connections between the mechanisms of cancer and COVID-19."

Events at the Intersection of Race and Science

Cultural Tax: The Cost of Being the Only or the Few

Tyrone Poster, a principal investigator at Boston University, shares insights on the "silent burden" that often accompanies being a Black STEM student and professional.

A Conversation with the Creators of The Nocturnists

The Nocturnists is a medical storytelling community founded by two UCSF physicians. Their audio documentary series "Stories from a Pandemic" and Black Voices in Healthcare" provide unique first-person accounts from health care workers.

Events for the Creature-Curious

Catch that Critter!

Ever wonder who or what is going bump in the night in your backyard? Here's your chance! Learn how to set up a "wildlife monitoring system and hear about the critters caught on camera at the Cal State East Bay Concord Campus and cameras set up in the Diablo hills. This first event will be a how-two and the second event will give participants the chance to share what they discovered.

A Very Spine-Tingling Spider Screening with KQEDs Deep Look

Yes, this one is a shameless plug! The team behind KQED's own Deep Look video series will present three of their popular spider episodes. Deep Look producers and cinematographers will reveal how they captured the spiders behavior on camera and spider experts will be on hand to answer all your arachnid-related questions.

And there are hundreds more events on all variety of subjects: storytelling and COVID, composting, wildfires. Check out the BASF website to see them all.

Editor's Note: KQED is a media sponsor of the Bay Area Science Festival.

Follow this link:
Bay Area Science Festival 2020: From Black Holes to Sourdough, It's All Virtual - KQED

Indias first indigenously developed test for coronavirus disease (Covid-19) based on the genetic editing tool CRISPR/Cas-9 is likely to be available in the market by the end of October. Heres everything that you need to know about the test:

Why is the test named Feluda?

Feluda is the acronym for FNCAS9 Editor Linked Uniform Detection Assay but the test is named after a fictional private detective from West Bengal created by the renowned writer and filmmaker Satyajit Ray.

Who has developed the test?

The test was developed by a research team led by Debojyoti Chakraborty and Souvik Maiti of the Council of Scientific and Industrial Research (CSIR) and researchers from the Institute of Genomics and Integrative Biology. It will be marketed by Tata Sons and has been approved by the Drug Controller General of India last month.

What is the effectiveness of the test?

It has a sensitivity of 96 per cent and specificity of 98 per cent which means that the test can detect positive and negative cases both up to 96 or 98 per cent of the time. The test matches the accuracy levels of RT- PCR tests which are widely being used for Covid- 19 diagnosis.

How does the test work?

With quicker result time and less expensive equipment, the test uses indigenously developed CRISPR gene-editing technology. It is the worlds first diagnostic test that uses specially adapted Cas9 protein to successfully detect the virus. It requires a nasal swab to be collected and sent to a lab. The Cas9 protein is barcoded to interact with the SARS-CoV2 sequence in the patients genetic material. The Cas9-SARS-CoV2 complex is then put on the paper strip, where using two lines (one control, one test), the test determines whether the sample was infected with Covid-19.

What is the CRISPR technology?

CRISPR, short form for Clustered Regularly Interspaced Short Palindromic Repeats, is a genome editing technology used in correcting genetic defects and treating and preventing the spread of diseases. The CRISPR technology that won the Nobel Prize for chemistry this year can detect specific sequences of DNA within a gene using an enzyme functioning as molecular scissors to snip it. It also allows researchers to easily alter DNA sequences and modify gene function.

What is the cost of the test?

The Feluda test is likely to cost about Rs 500 but the final cost will only be known once the test is commercially available. In contrast, the RT-PCR test now costs anywhere between Rs 1,600 to Rs 2,000.

Read the rest here:
What is the Feluda test that will be commercially available by October 31? - Hindustan Times

This report also researches and evaluates the impact of Covid-19 outbreak on the CRISPR & Cas Genes industry, involving potential opportunity and challenges, drivers and risks. We present the impact assessment of Covid-19 effects on CRISPR & Cas Genes and market growth forecast based on different scenario (optimistic, pessimistic, very optimistic, most likely etc.).

Global CRISPR & Cas Genes Market Overview:

The research report, titled [Global CRISPR & Cas Genes Market 2020 by Company, Regions, Type and Application, Forecast to 2025], presents a detailed analysis of the drivers and restraints impacting the overall market. Analysts have studied the key trends defining the trajectory of the market. The research report also includes an assessment of the achievements made by the players in the global CRISPR & Cas Genes market so far. It also notes the key trends in the market that are likely to be lucrative. The research report aims to provide an unbiased and a comprehensive outlook of the global CRISPR & Cas Genes market to the readers.

Get PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) @ https://www.marketresearchhub.com/enquiry.php?type=S&repid=2696251&source=atm

Global CRISPR & Cas Genes Market: Segmentation

For clearer understanding of the global CRISPR & Cas Genes market, analysts have segmented the market. The segmentation has been done on the basis of application, technology, and users. Each segment has been further explained with the help of graphs figures. This breakdown of the market gives the readers an objective view of the global CRISPR & Cas Genes market, which is essential to make sound investments.

The major players profiled in this report include:CRISPR TherapeuticsAstraZenecaAddgeneCaribou Biosciences, Inc.CellectisEditas Medicine, Inc.EgenesisF. Hoffmann-La Roche Ltd.Horizon Discovery Group PlcGenscripDanaher CorporationIntellia Therapeutics, Inc.LonzaMerck KGaANew England BioLabsTakara Bio, Inc.

To understand the changing political scenario, analysts have regionally segmented the market. This gives an overview of the political and socio-economic status of the regions that is expected to impact the market dynamic.

Global CRISPR & Cas Genes Market: Research Methodology

To begin with, the analysis has been put together using primary and secondary research methodologies. The information has been authenticated by market expert through valuable commentary. Research analysts have also conducted exhaustive interviews with market-relevant questions to collate this research report.

Do You Have Any Query Or Specific Requirement? Ask to Our Industry [emailprotected] https://www.marketresearchhub.com/enquiry.php?type=E&repid=2696251&source=atm

Global CRISPR & Cas Genes Market: Competitive Rivalry

The research report also studied the key players operating in the global CRISPR & Cas Genes market. It has evaluated and elucidated the research and development statuses of these companies, their financial outlooks, and their expansion plans for the forecast period. In addition, the research report also includes the list of strategic initiatives that clearly explain the achievements of the companies in the recent past.

The end users/applications and product categories analysis:On the basis of product, this report displays the sales volume, revenue (Million USD), product price, market share and growth rate of each type, primarily split into-General Type

On the basis on the end users/applications, this report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate of CRISPR & Cas Genes for each application, including-Biomedical

You can Buy This Report from Here @ https://www.marketresearchhub.com/checkout?rep_id=2696251&licType=S&source=atm

Strategic Points Covered in TOC:

Chapter 1: Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global CRISPR & Cas Genes market

Chapter 2: Evaluating the leading manufacturers of the global CRISPR & Cas Genes market which consists of its revenue, sales, and price of the products

Chapter 3: Displaying the competitive nature among key manufacturers, with market share, revenue, and sales

Chapter 4: Presenting global CRISPR & Cas Genes market by regions, market share and with revenue and sales for the projected period

Chapter 5, 6, 7, 8 and 9: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions

Contact Us:

marketresearchhub

Tel: +1-518-621-2074

USA-Canada Toll Free: 866-997-4948

Email: [emailprotected]

About marketresearchhub

marketresearchhub is the one stop online destination to find and buy market research reports & Industry Analysis. We fulfil all your research needs spanning across industry verticals with our huge collection of market research reports. We provide our services to all sizes of organisations and across all industry verticals and markets. Our Research Coordinators have in-depth knowledge of reports as well as publishers and will assist you in making an informed decision by giving you unbiased and deep insights on which reports will satisfy your needs at the best price.

View post:
CRISPR & Cas Genes Market: Analysis and In-depth study on market Size Trends, Emerging Growth Factors and Regional Forecast to 2025 - Eurowire

MarketsandResearch.biz has released a recent industry research report titled Global CRISPR and Cas Genes Market 2020 by Company, Regions, Type and Application, Forecast to 2025 that spotlights on the study of past, present, and future look of the industry. The study conducted is inclusive of the industry trends and competitive and regional analysis covering the period 2020-2025. The report provides an actual industry viewpoint on market trends, dynamics for market growth rate, trading, growth rate, and revenue, in terms of demand and supply, cost structure, barriers, and challenges, product type, key market players, regions, and applications. Studying the market in terms of growth and expansion, the report covers the crucial factors influencing the global CRISPR and Cas Genes market.

Competitive Landscape:

The report analysts have identified direct or indirect market competitors, as well as comprehend their mission, vision, values, niche market, strengths, and weaknesses. The report provides Porters five forces including the threat of substitute products or services, established rivals, new entrants, and two others such as the bargaining power of suppliers and customers. Prominent players joined with their market share are highlighted in the report. The well-established players in the global CRISPR and Cas Genes market are: Synthego, OriGene Technologies, Inc., Addgene, Thermo Fisher Scientific, Inc., Transposagen Biopharmaceuticals, Inc., GenScript, Horizon Discovery Group Co., Integrated DNA Technologies, Inc., Merck, New England Biolabs, Cellecta, Inc., Agilent Technologies, Applied StemCell, Inc.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketsandresearch.biz/sample-request/107687

NOTE: Our report highlights the major issues and hazards that companies might come across due to the unprecedented outbreak of COVID-19.

Geographical provincial information will help you in focusing on all the best-performing locales. The regions are extensively analyzed with respect to every parameter of the geographies in question, comprising, North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

In market segmentation by types, the report covers: Vector-based Cas, DNA-free Cas

In market segmentation by applications, the report covers the following uses: Genome Engineering, Disease Models, Others

The report evaluates production, consumption, and product segmentation highlights the current trend in the global market, and projects the revenue and potential developments of key players. Further, the report has analyzed the market concerning the regional landscape which incorporates extensive details about the types and application spectrums of this business. The research report tracks competitive growths such as joint ventures, tactical alliances, mergers and achievements, new product developments, and research and developments in the global CRISPR and Cas Genes market.

ACCESS FULL REPORT: https://www.marketsandresearch.biz/report/107687/global-crispr-and-cas-genes-market-2020-by-company-regions-type-and-application-forecast-to-2025

Consumer Behavior Analysis:

Furthermore, the report analyzes the behavior of the CRISPR and Cas Genes consumers in the marketplace and looks at motives for those behavioral trends. Later, personal, and social consumer behavior is studied through focus groups, surveys, and tracking sales history. Our consumer behavior study helps businesses to understand consumers value. Not all consumers value the same benefits, so its important for businesses to segment their consumer base. Using the latest technology and analysis on the demand-side, key players are getting into consumer behavior and their changing preferences.

Customization of the Report:

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

Contact UsMark StoneHead of Business DevelopmentPhone: +1-201-465-4211Email: sales@marketsandresearch.bizWeb: http://www.marketsandresearch.biz

Read more:
Global CRISPR and Cas Genes Market 2020 Trending Technologies, Developments, Key Players and End-use Industry to 2025 - Illadel Graff Supply

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues andtumors, wherein their toxicity, impurity, and other aspects are studied.

Get Exclusive PDF Sample Copy Of This Report:https://www.tmrresearch.com/sample/sample?flag=B&rep_id=40

With the growing number of successfulstem cell therapytreatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

Buy This Report @https://www.tmrresearch.com/checkout?rep_id=40<ype=S

Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

To know more about the table of contents, you can click @https://www.tmrresearch.com/sample/sample?flag=T&rep_id=40

About Us:

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

Link:
Stem Cell Assay Market In-Depth Analysis & Forecast 2017-2025 - The Think Curiouser

Five Indian American researchers and one Bangladeshi-American have been named among the 2020 Directors New Innovator Award recipients by the National Institutes of Health.

Among the recipients are Anindita Basu, Subhamoy Dasgupta, Deeptankar DeMazumder, Siddhartha Jaiswal, Shruti Naik, and Mekhail Anwar, according to the NIH website.

Basu, of the University of Chicago, was selected for the project, Profiling Transcriptional Heterogeneity in Microbial Cells at Single Cell Resolution and High-Throughput Using Droplet Microfluidics.

The Indian American is an assistant professor in genetic medicine at the University of Chicago and leads a multi-disciplinary research group that uses genomics, microfluidics, imaging and nano/bio-materials to develop new tools to aid in diagnosis and treatment of disease.

Basu obtained a B.S. in physics and computer engineering at the University of Arkansas, Ph.D. in soft matter physics at University of Pennsylvania, followed by post-doctoral studies in applied physics, molecular biology and bioinformatics at Harvard University and Broad Institute.

Her lab applies high-throughput single-cell and single-nucleus RNA-seq to map cell types and their function in different organs and organisms, using Drop-seq and DroNc-seq that Basu co-invented during her post-doctoral work.

Dasgupta is with the Roswell Park Comprehensive Cancer Center and was named for his project, Decoding the Nuclear Metabolic Processes Regulating Gene Transcription.

Dasgupta is an assistant professor in the Department of Cell Stress Biology at Roswell Park Comprehensive Cancer Center. He earned his B.S. from Bangalore University and M.S. in biochemistry from Banaras Hindu University, India before receiving his Ph.D. in biomedical sciences from University of North Texas Health Science Center at Fort Worth, where, as a Department of Defense predoctoral fellow, he characterized the functions of a novel gene MIEN1 in tumor progression and metastasis.

He then joined the laboratory of Bert W. O'Malley, M.D. at Baylor College of Medicine, where he studied the functions of transcriptional coregulators in tumor cell adaptation and survival, as a Susan G. Komen postdoctoral fellow.

DeMazumder, of the University of Cincinnati College of Medicine, was chosen for the project, Eavesdropping on Heart-Brain Conversations During Sleep for Early Detection and Prevention of Fatal Cardiovascular Disease.

DeMazumder joined the University of Cincinnati in 2017 as assistant professor of medicine, director of the Artificial Intelligence Center of Excellence and a Clinical Cardiac Electrophysiologist after completing his doctorate at SUNY Stony Brook in Synaptic Electrophysiology, a medical degree at Medical College of Virginia-Virginia Commonwealth University, internship at Mount Sinai and residency at University of Virginia in Internal Medicine, and clinical and research fellowships at Johns Hopkins University.

His longstanding goals are to transform clinical observations into testable research hypotheses, translate basic research findings into medical advances, and evaluate personalized treatment protocols in rigorous clinical trials, while caring for patients with heart rhythm disorders and improving their quality of life.

Jaiswal, of Stanford University, was named for his project, Clonal Hematopoiesis in Human Aging and Disease.

Jaiswal is an investigator at Stanford University in the Department of Pathology, where his lab focuses on understanding the biology of the aging hematopoietic system.

As a post-doctoral fellow, he identified a common, pre-malignant state for blood cancers by reanalysis of large sequencing datasets.

This condition, termed "clonal hematopoiesis, is characterized by the presence of stem cell clones harboring certain somatic mutations, primarily in genes involved in epigenetic regulation of hematopoiesis.

Clonal hematopoiesis is prevalent in the aging population and increases the risk of not only blood cancer, but also cardiovascular disease and overall mortality. Understanding the biology of these mutations and how they contribute to the development of cancer and other age-related diseases is the current focus of work in the lab.

Naik, of New York University School of Medicine, was named for her project, Decoding Microbe-Epithelial Stem Cell Interactions in Health and Disease.

Naik is an assistant professor at New York University School of Medicine. She received her doctorate in Immunology from the University of Pennsylvania-National Institutes of Health Graduate Partnership Program.

There she discovered that normal bacteria living on our skin, known as the commensal microbiota, educate the immune system and help protect us from harmful pathogens.

As a Damon Runyon Fellow at the Rockefeller University, Naik found that epithelial stem cells can harbor a memory of inflammation which boosts their regenerative abilities and established a new paradigm in inflammatory memory, her bio states.

The Naik lab studies the dynamic interactions between immune cells, epithelial stem cells, and microbes with a focus on 3 major areas of research: Tissue regeneration and cancer, host-microbe interactions, and early in life immunity.

Anwar, of U.C. San Francisco, was named for his project, Implantable Nanophotonic Sensors forIn VivoImmunoresponse.

Anwar, whose father is from Bangladesh, is a physician-scientist at UCSF, where he is an associate professor in the Department of Radiation Oncology. Driven by the challenges his patients face when fighting cancer specifically addressing the vast heterogeneity in treatment response by identifying the optimal treatment to pair with each patients unique biology he leads a laboratory focused on developing integrated circuits (or computer chips) forin vivocancer sensing.

After completing his bachelors in physics at U.C. Berkeley, where he was awarded the University Medal, he received his medical degree at UCSF, and doctorate in electrical engineering and computer science from the Massachusetts Institute of Technology where his research focused on using micro-fabricated devices for biological detection.

Read more from the original source:
Five Indian American Researchers Named Among NIH 2020 New Innovator Awardees - India West

An absolute insights and know-how of the greatest market opportunities into the relevant markets or industry required for successful business growth can be accomplished only with the best market research report. The Exosome Therapeutic Market business report provides market potential for each geographical region based on the growth rate, macroeconomic parameters, consumer buying patterns, their preferences for particular product and market demand & supply scenarios. All the studies performed to generate this industry report are based on large group sizes and also at global level. This Exosome Therapeutic Market research report provides clients with the supreme level of market data and information which is specific to their niche and their business requirements.

Get Sample PDF (including COVID19 Impact Analysis) of Market Report @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-exosome-therapeutic-market&rp

Market Analysis and Insights:Global Exosome Therapeutic Market

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

The major players covered in theExosome Therapeutic Marketreport areevox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global.Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Get Full TOC, Tables and Figures of Market Report @https://www.databridgemarketresearch.com/toc/?dbmr=global-exosome-therapeutic-market&rp

Exosomes are used to transfer RNA, DNA, and proteins to other cells in the body by making alteration in the function of the target cells. Increasing research activities in exosome therapeutic is augmenting the market growth as demand for exosome therapeutic has increased among healthcare professionals.

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for anAnalyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

Global Exosome Therapeutic Market Scope and Market Size

Global exosome therapeutic market is segmented of the basis of type, source, therapy, transporting capacity, application, route of administration and end user. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

Based on type, the market is segmented into natural exosomes and hybrid exosomes. Natural exosomes are dominating in the market because natural exosomes are used in various biological and pathological processes as well as natural exosomes has many advantages such as good biocompatibility and reduced clearance rate compare than hybrid exosomes.

Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process. The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

Rising demand of exosome therapeutic across the globe as exosome therapeutic is expected to be one of the most prominent therapies for autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases treatment, according to clinical researches exosomes help to processes regulation within the body during treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases. This factor has increased the research activities in exosome therapeutic development around the world for exosome therapeutic. Hence, this factor is leading the clinician and researches to shift towards exosome therapeutic. In the current scenario the exosome therapeutic are highly used in treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases and as anti-aging therapy as it Exosomes has proliferation of fibroblast cells which is significant in maintenance of skin elasticity and strength.

Based on source, the market is segmented into dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, urine and others. Mesenchymal stem cells are dominating in the market because mesenchymal stem cells (MSCs) are self-renewable, multipotent, easily manageable and customarily stretchy in vitro with exceptional genomic stability. Mesenchymal stem cells have a high capacity for genetic manipulation in vitro and also have good potential to produce. It is widely used in treatment of inflammatory and degenerative disease offspring cells encompassing the transgene after transplantation.

Based on therapy, the market is segmented into immunotherapy, gene therapy and chemotherapy. Chemotherapy is dominating in the market because chemotherapy is basically used in treatment of cancer which is major public health issues. The multidrug resistance (MDR) proteins and various tumors associated exosomes such as miRNA and IncRNA are include in in chemotherapy associated resistance.

Based on transporting capacity, the market is segmented into bio macromolecules and small molecules. Bio macromolecules are dominating in the market because bio macromolecules transmit particular biomolecular information and are basically investigated for their delicate properties such as biomarker source and delivery system.

Based on application, the market is segmented into oncology, neurology, metabolic disorders, cardiac disorders, blood disorders, inflammatory disorders, gynecology disorders, organ transplantation and others. Oncology segment is dominating in the market due to rising incidence of various cancers such as lung cancer, breast cancer, leukemia, skin cancer, lymphoma. As per the National Cancer Institute, in 2018 around 1,735,350 new cases of cancer was diagnosed in the U.S. As per the American Cancer Society Inc in 2019 approximately 268,600 new cases of breast cancer diagnosed in the U.S.

Based on route of administration, the market is segmented into oral and parenteral. Parenteral route is dominating in the market because it provides low drug concentration, free from first fast metabolism, low toxicity as compared to oral route as well as it is suitable in unconscious patients, complicated to swallow drug etc.

The exosome therapeutic market, by end user, is segmented into hospitals, diagnostic centers and research & academic institutes. Hospitals are dominating in the market because hospitals provide better treatment facilities and skilled staff as well as treatment available at affordable cost in government hospitals.

Exosome therapeutic Market Country Level Analysis

The global exosome therapeutic market is analysed and market size information is provided by country by type, source, therapy, transporting capacity, application, route of administration and end user as referenced above.

The countries covered in the exosome therapeutic market report are U.S. and Mexico in North America, Turkey in Europe, South Korea, Australia, Hong Kong in the Asia-Pacific, Argentina, Colombia, Peru, Chile, Ecuador, Venezuela, Panama, Dominican Republic, El Salvador, Paraguay, Costa Rica, Puerto Rico, Nicaragua, Uruguay as part of Latin America.

Country Level Analysis, By Type

North America dominates the exosome therapeutic market as the U.S. is leader in exosome therapeutic manufacturing as well as research activities required for exosome therapeutics. At present time Stem Cells Group holding shares around 60.00%. In addition global exosomes therapeutics manufacturers like EXOCOBIO, evox THERAPEUTICS and others are intensifying their efforts in China. The Europe region is expected to grow with the highest growth rate in the forecast period of 2019 to 2026 because of increasing research activities in exosome therapeutic by population.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Huge Investment by Automakers for Exosome Therapeutics and New Technology Penetration

Global exosome therapeutic market also provides you with detailed market analysis for every country growth in pharma industry with exosome therapeutic sales, impact of technological development in exosome therapeutic and changes in regulatory scenarios with their support for the exosome therapeutic market. The data is available for historic period 2010 to 2017.

Competitive Landscape and Exosome Therapeutic Market Share Analysis

Global exosome therapeutic market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, concept cars, product approvals, patents, product width and breadth, application dominance, technology lifeline curve. The above data points provided are only related to the companys focus related to global exosome therapeutic market.

Many joint ventures and developments are also initiated by the companies worldwide which are also accelerating the global exosome therapeutic market.

For instance,

Partnership, joint ventures and other strategies enhances the company market share with increased coverage and presence. It also provides the benefit for organisation to improve their offering for exosome therapeutics through expanded model range.

Customization Available:Global Exosome Therapeutic Market

Data Bridge Market Researchis a leader in advanced formative research. We take pride in servicing our existing and new customers with data and analysis that match and suits their goal. The report can be customised to include price trend analysis of target brands understanding the market for additional countries (ask for the list of countries), clinical trial results data, literature review, refurbished market and product base analysis. Market analysis of target competitors can be analysed from technology-based analysis to market portfolio strategies. We can add as many competitors that you require data about in the format and data style you are looking for. Our team of analysts can also provide you data in crude raw excel files pivot tables (Factbook) or can assist you in creating presentations from the data sets available in the report.

Do You Have Any Query Or Specific Requirement? Ask to Our Industry Expert @https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-exosome-therapeutic-market&rp

About Data Bridge Market Research :

Data Bridge Market Researchis a versatile market research and consulting firm with over 500 analysts working in different industries. We have catered more than 40% of the fortune 500 companies globally and have a network of more than 5000+ clientele around the globe. Our coverage of industries include Medical Devices, Pharmaceuticals, Biotechnology, Semiconductors, Machinery, Information and Communication Technology, Automobiles and Automotive, Chemical and Material, Packaging, Food and Beverages, Cosmetics, Specialty Chemicals, Fast Moving Consumer Goods, Robotics, among many others.

Data Bridge adepts in creating satisfied clients who reckon upon our services and rely on our hard work with certitude.We are content with our glorious 99.9 % client satisfying rate.

Contact Us :

Data Bridge Market Research

US: +1 888 387 2818

UK: +44 208 089 1725

Hong Kong: +852 8192 7475

Mail:[emailprotected]

Go here to see the original:
Exosome Therapeutic Market 2020-2026 to Witness Excellent Growth || Major Gaints Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH,...

Governor Gavin Newsom of California vetoed a bill that would have created new limitations on data sharing for direct-to-consumer genetic testing companies.

The Genetic Information Privacy Act (GIPA) asked testing companies to get informed consent from customers before disclosing their data to third parties. GIPA was aimed as a stop-gap to cover data sharing that is not already regulated by the California Consumer Privacy Act (CCPA) and the federal Health Insurance Portability and Accountability Act (HIPAA).

The final bill gained significant traction in the legislature. It passed both houses without a single vote against it.

In his veto message, the Governor expressed concern about the unintended consequences of the bill. Governor Newsom fears the bill would constrain mandatory reporting of COVID-19 test results to local public health departments and the California Department of Public Health.

California has been exceedingly active in the past few years putting forward innovative regulation of personal information. This veto is an example of the state taking a measured approach to the regulation of health data in the midst of the COVID-19 pandemic.

The Governor signaled that he supports the overall goals of the bill. He directed California state agencies to work with the Legislature on a revised version that takes into account the need to share COVID-19 testing data with authorities.

Putting it Into Practice. This version of the GIPA is put to rest. However, California has signaled it plans to pass a law regulating data sharing for genetic testing companies in the near future. Companies innovating in this area can begin reviewing their existing disclosures and consents with an eye toward getting opt-in consent in the future.

Read the rest here:
California Governor Pulls the Plug on Genetic Information Privacy Act - JD Supra