Editor's note: Life can be stressful, and many of us have a hard time administering self-care. The current world situation ripe with conflicts, shortages and a pandemic makes things even harder. Dr. Amit Sood, formerly of Mayo Clinic and now head of the Global Center for Resiliency and Wellbeing, is the author of books including "The Mayo Clinic Handbook for Happiness" and "SMART with Dr. Sood, and creator of the mobile app Zizo: Your Resilience Pal. Now, he is writing a weekly column answering readers' questions on these topics. See the tagline to learn how to send him your questions, and he will answer in future columns.

Dear friend,

Of the hundreds of ice-creams I have eaten so far, the only one I remember is the ice-cream I never ate.

That was in 1993. My wife Richa and I were sitting outside a shop in the sweltering New Delhi. We had just bought an ice cream to cool down. From the corner of my eye, I saw a little boy who could use a few calories. I walked up to him and offered my cone that he gladly took. I have savored the gleam I saw in his eyes at least a dozen times since then.

In a very interesting study, researchers looked at the genetic fingerprint of the two types of happiness hedonistic (self-centric) and eudaimonic (other-centric). People who were hedonistically happy had a higher inflammatory and lower anti-viral gene expression. It was just the opposite for the eudaimonic ones. With many illnesses caused by inflammation, you can see why this is so important for our health.

My take on this research is that our genes and the immune system know what is right for us and society. In the current times when a healthy immune system is extremely important for us, promoting kindness is imperative.

Kindness, research shows, pays three times over. Your kind words and actions enhance your health and wellbeing, help others, and the memory of kind actions by itself enhances your wellbeing. A very simple way to enhance your self-worth and happiness today is to count your previous kind actions.

I suggest take out a pen and paper and write the three most selfless things you have done in your life. If you feel up to it, share your experience with someone. Just counting previous acts of kindness can enhance your self-worth and bring happiness to others (you guessed it right witnessing or hearing about others kind actions also increases happiness).

In kindness,

Amit

Dr. Amit Sood answers your questions about stress, resilience, happiness, relationships, and related topics in his column. Email dearfriend@postbulletin.com.

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Our genes know kindness is the best medicine - Brainerd Dispatch

Culture-adapted AAV2 is a viral vector which is used to deliver gene therapy to the liver. However, clinical trials targeting diseases of the liver have had an 'unexpectedly low success rate' using the vector, according to researchers: who have now found that naturally occurring AAVs may be more effective.

The prototypical AAV2 discovered more than 50 years ago provides the serotype on which the field of AAV vectorology and gene therapy is based. The researchers from Australias Childrens Medical Research Institute (CMRI) say the discovery 'will shake the foundations of the field of AAV-based gene therapeutics and will mark the beginning of a new era not only for biomedical research, but most importantly, for millions of patients affected by genetic disorders'.

One area of interest in gene therapy is using AAVs to target the liver, which is involved in genetic disorders such as haemophilia and various enzyme deficiencies.

AAV2 is a viral vector used to deliver gene therapy to the liver, carrying therapeutic DNA to target cells in the body. It binds to a receptor on the target cell. However, the researchers found that while AAV2 binds to the attachment receptors - heparan sulfate proteoglycans (HSPCs) - it does so too tightly.

This means that the vector can get trapped on other cells in the body and not the target liver cells. This reduces the number of vectors that deliver their therapeutic cargo to the liver, diminishing therapeutic efficacy.

The teams of Dr Leszek Lisowski, Head of the Translational Vectorology Research Unit, and Prof Ian Alexander, Head of the Gene Therapy Research Unit, then turned to naturally occurring vectors isolated from liver samples. They found that these which use an as of yet unknown receptor are much more successful at delivering therapies to the liver.

CMRI researchers are now able to make vectors in the lab that use this better receptor, instead of HSPGs, potentially making the next generation of gene therapy targeting the liver 'vastly more successful'.

Theorizing that manufacturing methods could be playing a role, the researcherscompared traditional AAV vectors grown in culture with naturally occurring vectors that they isolated from liver samples. They observed that the cultured vectors rapidly mutated as they replicated in the lab: with these changes making the vectors bind more tightly to molecules called HSPGs on the surface of liver cells, but also impeding their ability to infect humanized liver cells in mice.

In contrast, the naturally occurring vectors infected liver cells more efficiently and bound less tightly to HSPGs, although these effects disappeared when the scientists grew the natural vectors in culture over time.

This really challenges a basic concept in our field that binding strongly to HSPG was essential for AAVs' entry into human cells and suggests that vectors targeting the other receptor used by natural AAVs, of human liver origin, are likely to be more effective for clinical gene therapy applications, said Dr Lisowski. The prototypical AAV2, discovered over 50yrs ago, is the serotype on which the entire field of AAV vectorology and gene therapy is based.

Our study sheds new light and challenges our previous understanding and corrects misconceptions about how the vector binds to the cells.

Researchers at the CMRI can now start to improve on the use of vectors to help children with liver conditions. A better vector can increase safety and improve efficiency, while the increased therapeutic efficacy will mean lower doses are needed and thus reduce the cost of treatment.

The insights on adeno-associated virus receptor binding can potentially be extended to other tissues beyond the liver, add researchers. This makes this a very impactful study which will change the trajectory of AAV-based gene therapies.

Adeno-associated viruses (AAVs) were discovered in the 1960s. The vectorization of AAV2, a human isolate, in 1984 set in motion the development the use of the viral vector in gene therapy.

The liver is a key target for developing more efficient AAV vector delivery, given its direct involvement in a number of genetic and acquired diseases.

Source: Science Translational Medicine, September 9, 2020.DOI: 10.1126/scitranslmed.aba3312

Title: Restoring the natural tropism of AAV2 vectors for human liver

Authors: M. Cabanes-Creus; C.V. Hallwirth; A. Westhaus; B.H. Ng; S.H.Y. Liao; E. Zhu; R.G. Navarro; G. Baltazar; M. Drouyer; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander; L. Lisowski at University of Sydney in Westmead, NSW, Australia; C.V. Hallwirth; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander at Sydney Children's Hospitals Network in Westmead, NSW, Australia; A. Westhaus; G. Santilli; A.J. Thrasher at University College London in London, UK.

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Researchers explore naturally occurring viral vectors: 'Our study will change the trajectory of AAV-based gene therapies' - BioPharma-Reporter.com

MORRISVILLE, N.C., Sept. 23, 2020 /PRNewswire/ --Metabolon, Inc., the global leader in metabolomics solutions advancing drug development and precision medicine, today announced the close on $72 million in combined debt and equity financing. This round of financing included Perceptive Advisors as a new participant. EW Healthcare Partners and other existing investors also participated in the financing.

"We are thrilled by this strong show of support from Perceptive Advisors and our current investors," said Rohan (Ro) Hastie, President and CEO, Metabolon. "I am also extremely excited that some of the company's earliest investors participated in this round of investment as well. The incremental funding will help accelerate our growth and expand our client base, in addition to helping further our R&D programs in machine learning to enable novel biomarker discovery and expand our precision medicine platform."

Metabolomics, the large-scale study of all small molecules in a biological system, is the only 'omics technology that provides a current-state functional readout of a biological system. By leveraging the world's largest proprietary metabolite database, Metabolon can decipher thousands of discrete chemical signals from genetic and non-genetic factors to reveal biological pathways. Metabolon's technology makes connections where other 'omics cannot and provides the definitive representation of the phenotype.

The company today also announced the appointment of Robert A. Cascella, Chief Business Leader, Precision Diagnostics, and Executive Vice President of Phillips, as one of three independent members of the Metabolon Board of Directors.

"Rob's 30-year career in the healthcare industry comprises an impressive track record of value creation and delivering integrated solutions," said Hastie, "We are honored to have his expertise on the board to help lead Metabolon to our next stage of growth."

Cascella joins Todd Schermerhorn, retired senior vice president and chief financial officer of C. R. Bard, Inc, and Jan Lundberg, formerly President of Lilly Labs and head of R&D, as independent board members.

About Metabolomics Metabolomics is emerging as the next frontier in 'omics innovation and the key to understanding biology, health and disease. All biological systems continually fuel chemical reactions that produce small molecules, called metabolites, which comprise the metabolome. Metabolomics reveals the comprehensive representation of a phenotype of any biological system by examining the influence of genetic, environmental and lifestyle factors. The data generated by metabolomics studies provides real-time, in vivo insights to advance discoveries for biopharma, population health, consumer products and more.

About MetabolonMetabolon, Inc., is the global leader in metabolomics, with a mission to deliver metabolomics data and insights that expand and accelerate the impact of life sciences research in all its applications. Over 20 years, 10,000+ projects, 2,000+ publications, and ISO 9001:2015 and CLIA certifications, Metabolon has developed industry-leading scientific technology and bioinformatics techniques. Our Precision Metabolomics Platform has enabled the development of the world's largest proprietary metabolomics reference library. Our industry-leading data and translational science experts help our clients address some of the most challenging and pressing questions in the life sciences, accelerating research, and development success. The company offers scalable, customizable metabolomics solutions from discovery through clinical trials and product life-cycle management. For more information, please visit http://www.metabolon.com and follow us on LinkedInand Twitter.

About PerceptiveFounded in 1999, Perceptive Advisors focus on supporting progress in the life sciences industry by identifying opportunities and directing financial resources toward the most promising technologies in modern healthcare. The firm manages approximately $7.3 billion across its portfolio.

About EW Healthcare PartnersWith close to $4 billion raised since inception, EW Healthcare Partners is one of the largest and oldest private healthcare investment firms and seeks to make growth equity investments in fast growing commercial-stage healthcare companies in the pharmaceutical, medical device, diagnostics, and technology-enabled services sectors in the United States and in Europe. Since its founding in 1985, EW Healthcare Partners has maintained its singular commitment to the healthcare industry and has been a long-term investor in over 150 healthcare companies, ranging across sectors, stages and geographies. The team is comprised of over 20 senior investment professionals with offices in Palo Alto, Houston, New York, and London. For more information, visit http://www.ewhealthcare.com.

SOURCE Metabolon, Inc.

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Metabolon, Inc. Announces $72 Million in Debt and Equity Financing to Accelerate Advancements in Precision Medicine and Innovative Drug Development...

(UroToday.com) Since 2015, multiple studies together have suggested that approximately a quarter of metastatic castration-resistant prostate cancer (mCRPC) tumors have mutations in DNA damage repair (DDR) genes. These alterations, especially those in genes for homologous recombination repair (HRR), are associated to varying degrees with response to PARP inhibitor therapy. Two PARP inhibitors are now approved for the care of patients with mCRPC and mutations in certain HRR genes: olaparib (based on PROfound trial) and rucaparib (based on TRITON2 trial). Given the significance of alterations in HRR genes in mCRPC, genomic testing for HRR alterations has been included in mCRPC treatment guidelines with the purpose of guiding genetic counseling and therapy selection.

In this poster, Dr. Neal Shore and colleagues describe real-world patterns and predictors of tissue testing for alterations in HRR genes. They focused on the community cancer care setting. Data were collected from the Flatiron Health Electronic Health Record derived database that was collected between 2013 and March 2019, prior to the approval of PARP inhibitor therapy in mCRPC, and focused on testing for alterations in BRCA1, BRCA2, ATM, CDK12, FANCA, and PALB2). The Flatiron database contains information on patient demographics, prostate cancer characteristics, and treatment, as well as genetic testing results.

From the Flatiron database, 5,213 patients with mCRPC were identified, 91% of which were managed at community oncology care centers. Of these patients, 674 (12.9%) underwent testing for the specified HRR gene alterations. Patient characteristics are shown below.

The breakdown of the 674 patients by testing method and number of genes tested is shown below.

BRCA1 and BRCA2 were the most commonly tested genes. A substantial portion of patients underwent only blood/saliva testing (40.5%, capable of detecting germline or circulating tumor DNA) or tumor-tissue testing only (42.1%, capable of detecting germline or tumor tissue DNA alterations). Only 1.8% of patients undergoing blood/saliva testing were tested for all 6 HRR genes, and 69% of tumor-tissue testing covered all 6 genes. Amongst the 286 patients who had negative blood/saliva testing, only 12.6% underwent tumor-tissue profiling, and 5 of these patients (13.9%) had an HRR alteration found.

The most common testing platform utilized was Foundation Medicine, followed by Guardant. Testing rates and the inclusion of more genes increased with time.

The prevalence of HRR alterations found is shown below.

Treatment at an academic medical center or having received multiple prior lines of therapy was associated with a higher likelihood of having HRR mutational profiling. Older age and higher PSA value at diagnosis of mCRPC were significantly associated with a lower likelihood of HRR mutation profiling.

The poster concludes that rates of HRR mutational testing did not increase dramatically with changes to the NCCN guidelines recommending this testing in 2017. Increased awareness of recommendations for testing, especially with the approval of two therapeutic agents contingent upon the presence of HRR alterations, is critical for the care of patients with mCRPC.

Presented by: Neal Shore, MD FACS, Urologist, and Director of the Carolina Urologic Research Center, Myrtle Beach, South Carolina

Written by: Alok Tewari, MD, Ph.D., Medical Oncologist at the Dana-Farber Cancer Institute, at the 2020 European Society for Medical Oncology Virtual Congress (#ESMO20), September 19th-September 21st, 2020

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ESMO Virtual Congress 2020: Real-World Patterns of Genomic Testing in Patients with Metastatic Castration-R... - UroToday

MIAMI, Sept. 22, 2020 /PRNewswire/ -- The Victor Center for the Prevention of Jewish Genetic Diseases, which offers preconception screening and genetic counseling for prospective parents, has recently expanded services to offer genetic screening and virtual clinical consults to couples and individuals in New York, New Jersey and Maryland.

The center, which also serves Florida, Massachusetts and Pennsylvania, helps future parents identify whether they are at risk of passing on genetic diseases, including those common among people of Jewish ancestry.

"Not everyone knows their full ancestral heritage, so we encourage anyone planning to start a family and their partner to undergo screening," said Debbie Wasserman, Outreach Coordinator/Genetic Counselor for the Victor Center.

Jewish genetic diseases are a group of recessive, inherited disorders that occur with significant frequency in the Ashkenazi Jewish community (those of eastern or central European descent). Individuals of Ashkenazi descent have higher carrier rates for diseases such as Tay-Sachs, Canavan, familial dysautonomia, and Gaucher. Many of the diseases are severe, and some are fatal in childhood.

One in two of those of Ashkenazi descent is a carrier for at least one Jewish genetic condition, and Sephardic and Mizrachi Jews are also at increased risk for certain genetic disorders. More than half of participants in the Victor Center screening program are carriers for one or more of the 225 plus conditions on the expanded screening panel, which also includes disorders found in other ethnicities.

The Victor Center offers a convenient screening process during this time of social distancing. Upon request, a genetic counseling session is scheduled and a screening kit is mailed to the home. Recipients provide saliva samples and return the kit to a lab for processing. Once results are available, a video consult is coordinated to convey understanding and address questions.

There is a fee for Victor Center screening services. However, most medical insurance plans cover the service. For more information, please call 786-897-9587 or visit http://www.victorcenter.org

About the Victor Center The Victor Center was founded in 2002 by Lois B. Victor in partnership with Einstein Healthcare Network in Philadelphia. Ms. Victor lost two children to a Jewish genetic disease before a test for the disorder became available. The experience galvanized her commitment to ensuring that no family endures the heartache of a preventable illness by making certain that Jews of childbearing age are screened and get the information they need to have healthy children. Nicklaus Children's Hospital was appointed the National Office for the Victor Center in 2017. The Nicklaus Children's Hospital Victor Center maintains the nation-wide collaborative work of the center in promoting education related to preconception screening while increasing knowledge, awareness, and access to genetic services.

About Nicklaus Children's HospitalFounded in 1950 by Variety Clubs International, Nicklaus Children's Hospital is South Florida's only licensed specialty hospital exclusively for children, with nearly 800 attending physicians and more than 475 pediatric subspecialists. The 309-bed hospital, known as Miami Children's Hospital from 1983 through 2014, is renowned for excellence in all aspects of pediatric medicine with several specialty programs routinely ranked among the best in the nation by U.S. News & World Report since 2008. The hospital is also home to the largest pediatric teaching program in the southeastern United States and has been designated an American Nurses Credentialing Center (ANCC) Magnet facility, the nursing profession's most prestigious institutional honor. For more information, please visit http://www.nicklauschildrens.org.

For more information: Nicklaus Children's Hospital Rachel Bixby, 305-663-8476

SOURCE Nicklaus Childrens Health System

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Its unusual that a virus would be less severe in children than it is in adults. But when it comes to COVID-19, kids make up just a small percentage of severe cases. Yale researchers are working to understand why that is.

Their discoveries can help guide understanding of the virus and possible treatment options.

Ina paper published recently in Proceedings of the National Academy of Sciences(PNAS),Dr. Naftali Kaminski, theBoehringer-Ingelheim Endowed Professor of Internal Medicine and chief of Pulmonary, Critical Care and Sleep Medicine, and colleagues shared findings related tochildrens surprising immunity to the virus. They detailed how factors including allergies, asthma, the common cold, and existing vaccines may be having a protective effect.

Meanwhile,Carrie Lucas, assistant professor of immunobiology at Yale, is looking at blood samples from the small percentage of children who develop the rare condition known as Multi-Inflammatory Syndrome in Children, or MIS-C, in response to COVID-19. Her lab is analyzing blood samples for molecular and genetic clues to figure out why a certain subset of kids are most at risk.

Findings just published in the journal Science Translational Medicine led byKevan Herold, the C.N.H. Long Professor of Immunology and Internal Medicine at Yale, revealed that children diagnosed with COVID-19 express higher levels of two specific immune system molecules, a factor that might be leading to better health outcomes.

Related story:Childrens immune response more effective against COVID-19

Understanding why children appear to be better protected from severe cases than adults could provide important clues on how the novel coronavirus spreads, who is at greatest risk, and how to treat it.

This is different from other viruses that affect kids more seriously, Kaminski said. Its an interesting conundrum and could provide implications for therapeutics.

In the PNAS paper, researchers point to the possibility that allergies and asthma in children has a protective effect. When the body responds to an allergy or asthma trigger, the immune system releases Th2 cells, which in turn increases a type of cell called the eosinophil in the blood and tissues. This allergic inflammation has been shown to dramatically reduce the levels of a key receptor to the COVID-19 molecule, known as ACE2. They added that astudy of 85 older adultswho died of COVID-19 in China showed that they had very low levels of blood eosinophils.

Initially, there was a concern about the impact of COVID-19 on children with asthma, said Kaminski. Some 7.5% of U.S. children under 18, or 5.5 million kids, have asthma, according to the Centers for Disease Control and Prevention. But, in fact, it seems that compared to other chronic lung diseases, people with asthma are infected less, and, when they are infected, asthma is not a risk factor.

Instead, risk factors known to drive worse COVID-19 outcomes include age, obesity, hypertension, and cardiac diseases.

The greater exposure children have to the common cold may also offer protection. Coronaviruses are a large family of viruses so named for their crown-like shape under a microscope, of which the common cold is one. SARS-CoV-2, which causes COVID-19, is another.

It is thought that exposure to colds may cause viral interference, when one virus interferes with the replication of a second virus. Exposure to common colds, and more severe illnesses like croup, more common in children, are associated with decreased expression of the ACE2 COVID-19 receptor. Studies have found that children symptomatic with COVID-19 may have high viral loads in their noses but, because they have lower levels of ACE2, their lungs are less likely to become infected. In other words, they can still easily spread the virus, but are less likely to develop serious symptoms.

Kaminski added that there is even evidence that vaccines can provide protection. Astudyof Department of Defense personnel found that the 2017-2018 seasonal flu vaccine produced a statistically significant number of individuals who tested positive for common cold-related coronaviruses. If future flu vaccines are designed to increase common coronaviruses, he said, this phenomenon may actually provide some protection to SARS-CoV-2 through cross-reactive immunity.

Of course, not all children are protected from the worst effects of COVID-19. Lucas and her team of pediatric immune disease researchers at Yale are looking at the rare cases of children who have been seriously affected by the virus. Specifically, they looked at children who were asymptomatic during SARS-CoV-2 infection, but weeks later developed a high fever, vomiting, abdominal pain, and sometimes shock, a condition known as MIS-C.As of Sept. 17, there were 935 confirmed cases of MIS-C in the U.S., and 19 deaths.

Lucas lab, which has enrolled 16 pediatric MIS-C patients, is analyzing immune cells in their blood at the single-cell level, as well as thousands of blood proteins, to understand what is happening.

Mostly, right now, our data are showing what the syndrome isnot, she said. For instance, we have found no sign of an active viral or bacterial infection during acute MIS-C.

They are also collecting saliva samples from parents to compare to childrens samples, which might reveal information about genetic variants. Were looking for the needle in the haystack that could be causing this rare manifestation, Lucas said. So far, theres no evidence that this is something that runs in families. I dont know of any cases where two children in a family developed MIS-C.

What they do know, she said, is that inflammatory markers are high, and most patients respond well to immunosuppressive therapies such as steroids. Additional findings will be published in the coming weeks on MedRxiv, a preprint server founded by Yale scientists which publishes studies before they have been peer-reviewed.

While children largely seem to be protected from the immediate effects of COVID-19, there are still long-term concerns, Kaminski and the authors caution. The pandemic and social distancing, they note, affect maturation of the immune system, psychological health, education, and childhood obesity.

We know that the health of children is strongly affected by socioeconomic downturns, Kaminski said, and this potential adverse outcome should not be overlooked.

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What we know about COVID-19 and kids - Yale News

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated(Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).

PRIME is a regulatory mechanism that provides early and proactive support to developers of promising medicines, to optimize development plans and speed up evaluations so these medicines can reach patients faster. The goal of PRIME is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need. PRIME designation was granted based on clinical data from CRISPR and Vertexs ongoing Phase 1/2 trial of CTX001 in patients with severe SCD.

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from transfusion-dependent beta thalassemia (TDT) or severe SCD, in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation from the European Commission, for both TDT and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

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

CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of PRIME designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding CTX001s PRIME designation or its development, the potential benefits of CTX001, our plans and expectations for our clinical trials and clinical trial sites, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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Every year around 460,000 people are admitted to hospital for chronic heart failure just in Germany. More than any other disease. The causes of chronic heart failure include conditions such as high blood pressure, other types of coronary heart disease, heart attacks, or myocarditis. On top of that, some forms of myocardial disease also have a genetic predisposition.

The repercussions of this disease are a significant decline in the quality of life and a higher, premature mortality rate. Treatment of the disease still poses problems. Although there are currently effective drugs available to treat heart failure and especially its underlying conditions, the number of hospitalizations and mortality rates are still very high. However, new research data now offers cause for hope. Two drugs from the group of SGLT 2 inhibitors originally developed for diabetes mellitus have proven to be very effective against heart failure: Dapagliflozin and empagliflozin.

SGLT2 inhibitors block glucose reabsorption back into the blood from the so-called primary urine. This leads to a loss of glucose and thus to a lowering of blood sugar levels. Apart from the loss of glucose via the kidneys, these drugs also cause the body to lose sodium. This consequently leads to metabolic changes that could have a beneficial effect on the heart.

In two large studies conducted in Germany under the direction of the German Cardiology Society (DGK) / Heart and Circulation Research, the effect of the two drugs on cardiac failure has now been examined. These are large multi-center studies with a total of more than 8,000 patients who received double-blind and randomized treatment. Patients with heart failure, with or without diabetes, and with limited ventricular function were included. All of whom continued to receive the optimal standard of care for heart failure.

Both studies revealed that the risk of cardiovascular death and heart failure hospital admissions decreased by approximately 25%. The scientists reported that the effects were comparable in both studies regardless of modern complementary treatments. And in patients with and without diabetes mellitus.

What is impressive is the consistent decline in heart failure complications in diabetics and non-diabetics in these studies, says Prof. Dr. Michael Bhm, press spokesperson of the DGK and scientific director of both studies for Germany. This shows that it is possible to turn a diabetes medication into an effective heart failure medication with proven efficacy in non-diabetics. These study outcomes are really good news for all patients with heart failure. To date, no other drug has shown such convincing results, even more so because it also significantly improves kidney function, emphasizes Prof. Andreas Zeiher, president of the German Society of Cardiology.

Bhm now expects that the SGLT2 inhibitors will most likely be included in the European Guidelines for the Diagnosis and Therapy of Heart Failure, which is due to be published in 2021. This new heart failure guideline is currently being prepared by an international panel of experts.

The results of both studies were published in the New England Journal of Medicine.https://www.nejm.org/doi/10.1056/NEJMc1917241https://www.nejm.org/doi/full/10.1056/NEJMoa2022190

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Diabetes medication effective against chronic heart failure - Innovation Origins