Veterans Memorial Hospital’s Family Wellness Fair to be held … – Waukon Standard

The annual Family Wellness Fair will be held this Wednesday, May 10 from 3:30-6 p.m. at Veterans Memorial Hospital and Clinics. The Family Wellness Fair is a community wide event offering families of all ages a night of entertainment and education about the local resources available to them, plus many free health testing opportunities. This fun-filled family event is free and open to all members of the community of any age.

New this year, visitors to the fair can participate in the Be the Match stem cell sampling which will be provided by the hospitals Laboratory. This is the same program that Robyn Roberts from Good Morning America endorses following her own successful bone marrow transplant match 10 years ago. A blood stem cell donation can be a cure for blood cancer, sickle cell and other deadly diseases. Many patients and their families are counting on drives like this to find their match. The process is simple and painless and will be available for all visitors to the wellness fair.

Gundersen Medical Clinic will also be hosting a Teddy Bear Clinic that evening and giving tours of its remodeled facility.

Entertainment will be provided, plus over 40 exhibitors will have tables on display providing demonstrations, games and crafts for the entire family. Veterans Memorial Hospital staff will be on hand to perform a number of free health tests. Plus, over 100 door prizes will be given away throughout the evening. All visitors to the Family Wellness Fair are eligible to win just by signing in at the registration table as they enter the fair.

All visitors to the Family Wellness Fair are asked to enter the fair through the Medical Clinic main entrance near where the fire truck will be parked. Parking will be available at both the clinic and hospital lots as well as all street parking.

Everyone in all the area communities is welcome to attend this free event, sponsored by Veterans Memorial Hospital.

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Veterans Memorial Hospital's Family Wellness Fair to be held ... - Waukon Standard

CAR Technology in Cancer Therapy: From CAR-T to CAR-NK – The Scientist

One-Stop Research Support for CAR-NK Therapy

Sino Biological

Cell-based immunotherapy is a targeted approach to cancer treatment that continues to evolve beyond its early successes treating leukemia. Researchers employ new technologies, such as chimeric antigen receptors (CARs) expressed on the immune cell surface, to effectively target hematological malignancies and solid tumors with cell therapy.1,2

To develop CARs, scientists engineer hybrid molecules made of T cell receptors and antibody fragments. These receptors direct immune cells to recognize and destroy cancer cells that express a specific antigen targeted by the CAR antibody domain. In recent years, scientists fine tuned CAR therapies for improved safety and efficacy. Optimized CAR molecules have high target antigen specificity, enable efficient immune cell activation, and cause minimal off-target effects.3,4

Once an optimal CAR is developed, researchers use reliable lentiviral packaging methods to transfer it to immune cells for CAR expression and quality control experiments such as immunoassays and cell viability assays. CAR-T therapies, T cells equipped with CAR expression, continue to lead the way in cell-based therapeutics. Therefore, researchers must design CAR molecules in ways that ensure the durability and persistence of CAR-expressing immune cells in the body.2-6

For CAR-T cell therapy, researchers genetically engineer T cells derived from either a patient or a donor, modify them to express CARs, expand the cells ex vivo, and infuse them into a patients bloodstream. Since 2017, over 700 CAR-T therapy clinical trials have been registered and the FDA has approved six CAR-T therapies for the treating hematological malignancies, including leukemia, lymphoma, and multiple myeloma.1 However, despite their clinical significance, there are limitations to producing and employing CAR-T therapies. Collecting T cells from a patient or donor is costly and time-consuming, especially for patients with compromised immune systems. Additionally, CAR-T cell therapy may cause severe side effects such as cytokine release syndrome (CRS) or neurotoxicity. Finally, CAR-T cells struggle to penetrate the tumor microenvironment (TME), which can hinder their therapeutic function.3,4

To overcome conventional CAR-T limitations, researchers turn to promising new CAR strategies. In multi-target CAR-T, scientists design CARs that recognize two or more antigens per receptor, resulting in broader immune coverage. Another new avenue involves alternative immune cell types such as natural killer (NK)-based cell therapies. Part of the innate immune response, NK cells can eliminate abnormal cells, including tumor cells and virally-infected cells. Researchers produce CAR-NK cell therapies with the same receptor-antigen hybrid technology as CAR-T, but express the CAR in NK cells. Scientists can derive NK cells from a variety of sources, including umbilical cord blood (UCB), peripheral blood (PB), human-induced pluripotent stem cells, human embryonic stem cells, hematopoietic stem cells, and NK cell lines. In comparison to CAR-T therapy, CAR-NK cell therapy is safer with a lower chance of negative immune side effects, more efficient antitumor activity, and higher efficiency for off-the-shelf production. CAR-NK cell therapies for solid tumors and hematological malignancies have shown promising results in clinical trials.1,3,6,7

Comprehensive Solutions for CAR-T Therapy

Sino Biological

Before cell therapies reach the clinic, scientists develop them through a multistep process. Whether the therapy is CAR-T or CAR-NK, researchers must collect immune cells, employ genetic engineering to create a targeted CAR, transduce immune cells for CAR expression, culture and expand the CAR-expressing cells, and test for quality control.

As a global leading supplier of bioreagents and CRO services for the biopharmaceutical field, Sino Biological provides comprehensive solutions for scientists developing CAR-T and CAR-NK cell therapies.5,6 These solutions cover each step from CAR development to CAR-T and CAR-NK cell preparation and cell quality control. Sino Biologicals reagents and services can support researchers during each stage of cell therapy development from early target discovery to preclinical phase.

References

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CAR Technology in Cancer Therapy: From CAR-T to CAR-NK - The Scientist

Editas Medicine to Present Clinical Data from the RUBY Trial of EDIT-301 for the Treatment of Severe Sickle Cell Disease at the EHA 2023 Congress -…

Editas Medicine, Inc.

Company-sponsored webinar to be announced

CAMBRIDGE,Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced that a scientific abstract detailing safety and efficacy clinical data from the Phase 1/2 RUBY trial of EDIT-301 in patients with severe sickle cell disease has been accepted for an oral presentation at the European Hematology Association (EHA) Hybrid Congress being held June 8-11, 2023, in Frankfurt, Germany, and via live stream.

Key data from multiple patients will be shared in the oral presentation at EHA, confirming initial clinical data, include:

Efficacy data, including total hemoglobin, fetal hemoglobin, percentage of F-cells, mean corpuscular fetal hemoglobin, and vaso-occlusive events (VOEs) post-infusion with EDIT-301.

Safety data, including neutrophil and platelet engraftment.

We are making significant progress with EDIT-301, and we look forward to sharing clinical data, including additional data that has been collected since the submission of the abstract, from the RUBY trial next month at the European Hematology Association Congress and in a Company-sponsored webinar. This data further supports our belief that EDIT-301 can be a potentially clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients, Baisong Mei, M.D., Ph.D., Senior Vice President and Chief Medical Officer, Editas Medicine. I would like to thank the participants, their families, clinicians, and colleagues at collaborating institutions that contribute to the RUBY trial.

The abstract can be accessed on theEHA website.

Oral Presentation Details:

Title: EDIT-301 Shows Promising Preliminary Safety and Efficacy Results in the Phase I/II Clinical Trial (RUBY) of Patients with Severe Sickle Cell Disease Using Highly Specific and Efficient AsCas12a EnzymePresenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Childrens, Cleveland, OH, United StatesDate/Time: Saturday, June 10, 2023, 4:30 5:45 p.m. CEST/ 10:30 11:45 a.m. EDTLocation: Harmonie 1, Messe FrankfurtSession: s437 Gene therapy and cellular immunotherapy Clinical

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EDIT-301 is currently being investigated in a clinical study in patients with severe sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta thalassemia (EDITHAL trial, NCT05444894). In addition to the clinical data update from the RUBY trial at EHA and in a Company-sponsored webinar next month, the Company will present a further clinical update from the RUBY trial by year-end. Additionally, the Company is on-track to dose 20 patients in the RUBY trial by year-end.

About Sickle Cell DiseaseSickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin (HbS). In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape. The abnormal shape causes the red blood cells to have shortened lifespan and to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing the manifestation of sickling.

About EDIT-301EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.

About RUBYThe RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available onwww.clinicaltrials.gov(NCT04853576).

AboutEditas MedicineAs a clinical stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard Universitys Cas9 patent estates and Broad Institutes Cas12a patent estate for human medicines. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companys expectation to provide a further clinical data update for the RUBY trial by year-end and to dose 20 total patients by year-end. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials, including the RUBY trial, and clinical development of the Companys product candidates, including EDIT-301; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission,and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

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Editas Medicine to Present Clinical Data from the RUBY Trial of EDIT-301 for the Treatment of Severe Sickle Cell Disease at the EHA 2023 Congress -...

Researchers solve mystery of how statins improve blood vessel health – Stanford Medical Center Report

ATAC-seq revealed that simvastatin-treated cells had closed chromatin structures that reduced the expression of genes that cause the endothelial-to-mesenchymal transition. Working backward, the researchers found that simvastatin prevents a protein known as YAP from entering the nucleus and opening chromatin.

The YAP protein is known to play important roles in development, such as regulating the size of our organs, but also has been implicated in the abnormal cell growth seen in cancer.

To see the drug in context, the researchers tested simvastatin on diabetic mice. Diabetes causes subtle changes to blood vessels that mimic the damage commonly seen in people who are prescribed statins older patients who do not have a cardiovascular condition, Liu said.

They found that after eight weeks on simvastatin, the diabetic mice had significantly improved vascular function, with arteries that more easily relaxed and contracted.

If we can understand the mechanism, we can fine-tune this drug to be more specific to rescuing vascular function, Liu said.

The findings also provide a more detailed picture of the vascular disease process, which could help doctors identify and treat early signs of vascular damage.

Ive been taking statins for the past 10 years to keep my cholesterol down. I also knew it has good vascular effects. I just didnt know how it does it, said Wu, the Simon H. Stertzer, MD, Professor who is also the director of the Stanford Cardiovascular Institute. This study explains how.

Researchers from the University of North Texas and the Ohio State University College of Medicine contributed to this study.

The study was supported by funding from the National Institutes of Health (grants R01 HL130020, R01 HL150693, R01 HL163680, R01 HL145676, P01 HL141084, R01 HL141371, R01 HL126527, R01 HL15864, R01 HL161002, R01 HL155282 and 18CDA34110293), an American Heart Association SFRN grant, an AHA Career Development Award and the Tobacco-Related Disease Research Program.

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Researchers solve mystery of how statins improve blood vessel health - Stanford Medical Center Report

What’s Exosome Therapy And How Can It Help Your Skin & Hair? – Glam

As you age, your skin's collagen production isn't the only thing that slows down. Your hair follicles do, too, with some growing out considerably thinner hair and others ending the hair growth cycle altogether. In addition, sebum production also reduces as you age, leading to duller, lustreless hair.The drop in hair growth and thickness eventually leads to permanent hair loss. But of course, there are other causative factors of hair loss, such as genetics, medications, and stress.

When fed into the follicles and scalp, exosomes reawaken the follicular cells, jumpstarting them and fostering hair growth. The therapy also helps prolong the anagen phase of the hair growth cycle and decrease or delay the catagen phase. Anagen is the growth phase, while catagen is the phase where hair growth stops. By prolonging the anagen phase, the follicles have more time to produce hair and combat hair loss. With their wound-healing and anti-inflammatory properties, exosomes can also help repair damaged tissues and follicles in the scalp. And everyone knows that healthy follicles equal healthy strands.

You can have more than one exosome session to reap the full benefits. However, waiting 3 to 6 months between sessions is preferred to evaluate if more sessions are necessary.Now that you know the benefits of exosome therapy for your skin and hair, we're sure you're wondering about the procedure. Well, here's what you need to know pre- and post-procedure.

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What's Exosome Therapy And How Can It Help Your Skin & Hair? - Glam

Thalassemia gene therapy approval marks a new era of treatment – Labiotech.eu

A newly-approved gene therapy is transforming the lives of patients with beta thalassemia an inherited blood disorder by helping to stop or significantly reduce their reliance on blood transfusions, says an expert from a leading global health system, Cleveland Clinic, on International Thalassemia Day on May 8.

A gene therapy known as betibeglogene autotemcel, or beti-cel, was approved in August last year by the US Food and Drug Administration (FDA) as safe and efficacious for adult and pediatric patients with transfusion-dependent beta thalassemia.

Rabi Hanna, a pediatric hematologist-oncologist at Cleveland Clinic Childrens which is in the process of becoming one of the few centers in the world certified to offer the gene therapy said: This is a milestone in thalassemia treatment as gene therapy uses patients own hematopoietic stem cells to produce healthier red blood cells and cure their blood disorder or at the very least, significantly reduce the number of blood transfusions needed to manage their condition. Thanks to gene therapy, we can remove the challenges that thalassemia patients face and give them the potential to pursue their goals and dreams without the restriction of blood transfusion or complications of iron overload.

Thalassemia, which has two types alpha and beta is among the most common genetic disorders and occurs most frequently in people from the Middle East, Mediterranean countries, North Africa, India, Central Asia, and Southeast Asia.

Beta thalassemia affects the bodys ability to produce hemoglobin, a protein in red blood cells that allows them to transport oxygen throughout the body. Individuals diagnosed with beta thalassemia major develop serious symptoms at a young age, which can be treated with frequent blood transfusions, but the transfusions themselves create the risk of iron overload with the potential to damage patients liver, heart, and endocrine system.

The new gene therapy, which resolves this problem, is ideal for teenagers or slightly older patients who have already started to experience complications from blood transfusions, Hanna said.

Hanna said: The gene therapy includes a short period (four days) of a chemotherapy called busulfan that may impact on male and female patients fertility, so it is better to wait until patients reach their teenage years so that they can undergo fertility-preservation treatment, although we do have methods to preserve fertility even in pre-pubescent patients. On the other hand, it is best not to leave it too long as after years of infusions, patients hearts and livers may have already been severely impaired from iron overload. Generally, the healthier the patients are at the start of the gene therapy process, the fewer side effects they will have.

The minimally invasive beti-cel treatment is delivered in three phases over several months, Hanna said. In the first phase, two medications (G-CSF and plerixafor) are used to stimulate the patients bone marrow to produce more stem cells. In the second, blood is drawn for stem cell collection from peripheral blood, and these stem cells are sent to the treatment company to perform the gene modification, that is, inserting a gene that enables them to produce non-affected hemoglobin. The modification process takes around two months.

The final phase takes place in a hospital so patients can be closely monitored. They receive a single chemotherapy medication once a day for four days, followed by a few days of rest, to prepare the body for the modified stem cells to be reintroduced through infusion into a vein, similar to an IV drip.

Within about a month after the infusion of the modified cells, Hanna noted, the vast majority of patients will engraft with the new stem cells and will not need any more blood transfusions or other treatments as they will have cells established in their bone marrow capable of producing hemoglobin normally. Even in the few exceptional cases where gene therapy is not curative, patients need for blood transfusions will have been reduced substantially.

Aside from the new gene therapy treatment, other advances in recent years have greatly improved the quality of life for thalassemia patients, Hanna said.

A medication with the generic name luspatercept-aamt was approved by the FDA in 2019 to reduce patients reliance on blood transfusions. It is given in the form of an injection every three weeks and works by enhancing erythroid (red blood cell) maturation and reducing the need for blood transfusions by up to 50% in some patients.

Luspatercept-aamt can also be used to enhance outcomes in curative bone marrow transplantations, which have become more widely accessible in recent years thanks to a new transplant approach. Hanna was among the pioneers of the haplo-identical bone marrow transplant, which allows for non-identical-HLA (human leukocyte antigen) donors.

Hanna said the reduction in blood transfusions enabled by luspatercept-aamt means the patient has better health prior to bone marrow transplants, and fewer antibodies that could potentially attack transplanted bone marrow.

With the wider adoption of gene therapy and the use of luspatercept-aamt to improve bone-marrow transplant outcomes, almost any patient can be cured, Hanna said.

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Thalassemia gene therapy approval marks a new era of treatment - Labiotech.eu

CRISPR Therapeutics Provides Business Update and Reports First Quarter 2023 Financial Results – Yahoo Finance

CRISPR Therapeutics AG

-Regulatory submissions complete for exagamglogene autotemcel (exa-cel), formerly known as CTX001, in the U.S. for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD)-

-EU and U.K. submissions validated by European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA); exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA-

-Enrollment and dosing ongoing for CTX110, targeting CD19+ B-cell malignancies, and CTX130, targeting CD70 for the treatment of T cell lymphomas-

-Initiated clinical trials for next generation CAR T candidates, CTX112 targeting CD19+ B-cell malignancies and CTX131, targeting CD70+ solid tumors-

-Enrollment and dosing ongoing in a Phase 1/2 clinical trial of VCTX211 for the treatment of Type 1 Diabetes (T1D)-

-Continues to advance its lead in vivo program, CTX310, targeting angiopoietin-related protein 3 (ANGPTL3) into clinical trials this year-

ZUG, Switzerland and BOSTON, May 08, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2023.

In the first quarter of 2023, we continued strong momentum across our portfolio. We and our partner Vertex have now completed regulatory submissions for exa-cel in the United States, European Union and United Kingdom, positioning exa-cel to potentially become the first approved CRISPR-based therapy in the world, a remarkable pace of progress considering the discovery of the CRISPR platform took place a little more than a decade ago, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. In parallel, we continue to drive our portfolio programs, including the initiation of clinical trials for our next-generation CAR T candidates, CTX112 and CTX131. In addition, we expect to advance CTX310, our lead in vivo program targeting ANGPTL3, into clinical trials later this year. We are well-positioned to drive towards our mission of bringing transformative and potentially curative therapies to patients in need.

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Recent Highlights and Outlook

FirstQuarter 2023 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1,889.5 million as of March 31, 2023, compared to $1,868.4 million as of December 31, 2022. The increase in cash of $21.1 million was primarily driven by the upfront payment received from Vertex in connection with a non-exclusive license agreement and a benefit from changes in net-working capital, offset by operating expenses.

Revenue: Total collaboration revenue was $100.0 million for the quarter ended March 31, 2023. Collaboration revenue for the first quarter of 2022 was not material. Collaboration revenue recognized in the first quarter of 2023 was primarily attributable to revenue recognized in connection with the upfront payment from Vertex.

R&D Expenses: R&D expenses were $99.9 million for the first quarter of 2023, compared to $118.2 million for the first quarter of 2022. The decrease in R&D expense was primarily driven by reduced variable external research and manufacturing costs.

G&A Expenses: General and administrative expenses were $22.4 million for the first quarter of 2023, compared to $28.0 million for the first quarter of 2022. The decrease in G&A expense was primarily driven by a decrease in external professional costs.

Collaboration Expense: Collaboration expense, net, was $42.2 million for the first quarter of 2023, compared to $30.6 million for the first quarter of 2022. The increase in collaboration expense, net, was primarily driven by an increase in manufacturing and pre-commercial costs associated with the exa-cel program.

Net Loss: Net loss was $53.1 million for the first quarter of 2023, compared to a net loss of $179.2 million for the first quarter of 2022.

About exagamglogene autotemcel (exa-cel)Exa-cel, formerly known as CTX001, is an investigational, autologous,ex vivoCRISPR/Cas9 gene-edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent vaso-occlusive crises (VOCs), in which a patients own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exa-cel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published inThe New England Journal of Medicinein January of 2021.

Based on progress in this program to date, exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from theFDA for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from theEuropean Commission, as well as Priority Medicines (PRIME) designation from theEuropean Medicines Agency(EMA), for both TDT and SCD. In the U.K., exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.

About CLIMB-111 and CLIMB-121The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exa-cel infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up trial.

About CLIMB-131This is a long-term, open-label trial to evaluate the safety and efficacy of exa-cel in patients who received exa-cel in CLIMB-111, CLIMB-121, CLIMB-141 or CLIMB-151. The trial is designed to follow participants for up to 15 years after exa-cel infusion.

About CLIMB-141 and CLIMB-151The ongoing Phase 3 open-label trials, CLIMB-141 and CLIMB-151, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to patients 2 to less than 5 years of age at a later date. Each trial will enroll approximately 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up- trial.

About the CRISPR-Vertex CollaborationCRISPR Therapeuticsand Vertex Pharmaceuticals 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. Exa-cel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exa-cel and splits program costs and profits worldwide 60/40 withCRISPR Therapeutics.

About CTX110 and CTX112CTX110, a wholly owned program ofCRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON clinical trial, which is designed to assess the safety and efficacy of CTX110 in adult patients with relapsed or refractory CD19-positive B-cell malignancies who have received at least two prior lines of therapy. CTX110 has been granted RMAT designation by the FDA. In addition, CTX112, a next-generation allogeneic CAR T cell therapy targeting CD19, is being investigated in a clinical trial. CTX112 includes two additional edits beyond CTX110 that are designed to enhance the potency of the CAR T cells.

About CTX130 and CTX131CTX130, a wholly owned program ofCRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being investigated for the treatment of relapsed or refractory T-cell hematologic malignancies in the COBALT-LYM trial and for renal cell carcinoma in the COBALT-RCC trial. CTX130 has been granted Orphan Drug designation for the treatment of T cell lymphoma by the FDA and RMAT designation for the treatment of relapsed or refractory Mycosis Fungoides and Szary Syndrome (MF/SS), types of cutaneous T cell lymphoma (CTCL). In addition, CTX131, a next-generation allogeneic CAR T cell therapy targeting CD70, is being assessed for safety and efficacy in a clinical trial investigating a basket of select solid tumors. CTX131 includes two additional edits beyond CTX130 that are designed to enhance the potency of the CAR T cells.

About VCTX210 and VCTX211VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived investigational therapy for the treatment of T1D. VCTX210 is being developed under a co-development and co-commercialization agreement betweenCRISPR TherapeuticsandViaCyte, Inc. VCTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of T1D, which incorporates additional gene edits that aim to further enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose.

AboutCRISPR TherapeuticsCRISPR Therapeuticsis 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 Therapeuticshas 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 Therapeuticshas established strategic partnerships with leading companies includingBayer, Vertex PharmaceuticalsandViaCyte, Inc.CRISPR Therapeutics AGis headquartered inZug, Switzerland, with its wholly-ownedU.S.subsidiary,CRISPR Therapeutics, Inc., and R&D operations based inBoston, MassachusettsandSan Francisco, California, and business offices inLondon, United Kingdom. For more information, please visitwww.crisprtx.com.

CRISPR THERAPEUTICS standard character mark and design logo,COBALT, CTX001, CTX110, CTX112, CTX130, CTX131, CTX310, VCTX210, VCTX211 are trademarks and registered trademarks ofCRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made byDr. Kulkarniin this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) its preclinical studies, clinical trials and pipeline products and programs, including, without limitation, the status of such studies and trials, regulatory filings for exa-cel and timing of data releases and regulatory submissions; (ii) potential benefits of exa-cel and the FDAs review of the BLAs and impact of Priority Review on such timing; (iii) benefits of Dr. Prasads employment; (iv) the sufficiency of its cash resources; (v) benefits of its collaborations, including potential milestone payments and royalties on future products under the non-exclusive license agreement; and (vi) 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. AlthoughCRISPR Therapeuticsbelieves 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: the potential for preliminary data from any clinical trial not to be indicative of final trial results; the potential that clinical trial results may not be favorable; that one or more of its internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for its product candidates; uncertainties inherent in the initiation and completion of preclinical studies for its product candidates and whether results from such studies will be predictive of future results of future studies or clinical trials; uncertainties about regulatory approvals to conduct trials or to market products; it may not realize the potential benefits of its collaborations; uncertainties regarding the intellectual property protection for its 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 byCRISPR Therapeuticswith theU.S. Securities and Exchange Commission, which are available on theSEC'swebsite atwww.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 Therapeuticsdisclaims 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.

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

Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

CRISPR Therapeutics AGCondensed Consolidated Statements of Operations(Unaudited, In thousands except share data and per share data)

Three Months Ended March 31,

2023

2022

Revenue:

Collaboration revenue

$

100,000

$

178

Grant revenue

762

Total revenue

$

100,000

$

940

Operating expenses:

Research and development

99,935

118,245

General and administrative

22,360

See the rest here:
CRISPR Therapeutics Provides Business Update and Reports First Quarter 2023 Financial Results - Yahoo Finance

Board of Regents medals awarded for teaching excellence … – University of Hawaii

The Regents Medal for Excellence in Teaching is awarded by the Board of Regents as tribute to faculty members who exhibit an extraordinary level of subject mastery and scholarship, teaching effectiveness and creativity and personal values that benefit students.

Rosanna Alegado is an associate professor of oceanography in the University of Hawaii at Mnoa School of Ocean and Earth Science and Technology (SOEST). Her work involves meaningful academic collaborations and partnerships with Indigenous communities.

She led SOESTs significant curriculum revision toward a required immersive course to ground all incoming graduate students in an understanding of working as marine biologists within Hawaiian culture. Its success has been recognized by the National Science Foundation with multi-year funding to foreground Indigenous knowledge, practices and values, and to transform and Indigenize higher education in STEM.

Alegado is regarded as an influential educator for other teaching faculty, as well as her students. She said, By challenging my students to integrate multiple didactic frameworks, one can achieve the most comprehensive understanding of a subject.

Her colleagues say that Rosie is not popular by being easy, and that her efforts are the epitome of teaching exceptionalism.

Tammy Hailipua Baker is an associate professor of theatre and dance in the University of Hawaii at Mnoa College of Arts, Languages & Letters. As a steward of Indigenous knowledge, she fulfilled that kuleana (responsibility) by building the Hawaiian Theatre Program, the only one of its kind focused primarily on performance.

A colleague, who was also her student in Hawaiian language, views the experience of acting in her productions as a master class in pedagogy. Professor Baker is continually supporting students and others in the production of 40 performers in speaking and singing lines individually and collectively. [She was] clearly the director throughout, nevertheless each actor (students) and production staff (teachers) were all made to feel their work was necessary and appreciated.

Baker is internationally recognized, the first from Hawaii to receive the Kennedy Centers Medallion of Excellence. A reviewer of her plays describes them as guides to restoring language and reclaiming the stories of generations of Indigenous populations; gifts to a culture whose language and history have been suppressed. Her transformative work shines through the passion, voice and aloha spirit of her students.

Richard C. Chen is an associate professor at the William S. Richardson School of Law at the University of Hawaii at Mnoa. He brings patience and empathy to all his interactions with students, never assuming the problem is with the students. This tenet is a teaching practice that extends into a way of modeling for the students as they enter the profession of law, as lawyers who seek to listen, learn and improve throughout their careers.

A cohort of 19 evening students for four straight semesters signed an enthusiastic letter of support for Chen, stating they collectively hope that our endorsement can begin to illuminate our appreciation of his talents as an educator and the positive impact he made during the formative stages of our legal education.

A colleague stated, Who wouldnt want to be in his classes? He is a professor whose empathy, kindness, brilliance and skill shine through in everything he does and it is elevating, inspirational and contagious.

Lincoln A. Gotshalk is a professor of kinesiology and exercise science in the University of Hawaii at Hilos College of Natural and Health Sciences. He is a musculoskeletal physiologist, anatomist and exercise physiologist with a strong background in muscular strength and power training and total body systemic response to exercise and stress.

He advises students, and teaches anatomy and physiology, research methods, nutrition and the science of diet and weight control, basic and advanced kinesiology courses, physiology of exercise and the science behind athletic training programs. Gotshalk is the director of the Laboratory for Exercise Sciences, which manages concurrent research projects.

Dr. Gotshalk most definitely has the ability to make every student feel appreciated and an important part of both the classroom and the lab group, noted a nominator. The experiences I have gained are ones I will never forget and I am thankful for all he has done to help me find my place in the UH Hilo community.

Karadeen Kam-Kalani is a professor of speech at Honolulu Community College. Her teaching philosophy recognizes that positive encouragement goes a long way in helping students gain the confidence they need to become better public speakers.

She is an inspiring and motivating instructor who strives to provide an environment for her students to foster self-discovery, steady improvement and growing confidence.

One student, self-conscious about his stutter, was nervous about taking a speech class. In Kam-Kalanis course, however, he learned to take a breath between sentences, use hand gestures to complement his talking points, and engage his audience with thoughtful questions. Her positive feedback helped him to improve his speaking capabilities.

The highlight of his learning journey came when one of his speeches was chosen as an example for other students to emulate. When he was asked how this recognition made him feel, his face lit up and he said, I felt awesome!

Tiffany-Joy Kawaguchi, serves as the program director and interim academic fieldwork coordinator in the occupational therapy assistant (OTA) program at Kapiolani Community College. Kawaguchi is an occupational therapist (OT) with more than 22 years of experience.

In 2015, Kawaguchi started a federally funded pro-bono clinic for the OTA program based on her belief that through doing, students become what they have the capacity to be. She utilizes meaningful experiences and intentional practice opportunities to help students access and then apply critical pieces of information to the OT process.

Dr. Tiff is undeniably dedicated to enabling her students to succeed, said an OTA program student. She accommodates numerous learning styles, grades fairly and offers detailed feedback so we know how to improve. Despite the endless list of things she has to do, she makes each one of us feel valued.

In 2016, Kawaguchi received the Laura N. Dowsett OT of the Year Award from the OT Association of Hawaii and was selected to represent Kapiolani CC in the inaugural Hawaii Association for Career and Technical Education Emerging CTE Leader Program in 2018. In 2021, Kawaguchi was awarded the Francis Davis Award for Excellence in Undergraduate Teaching.

Kamuela Kimokeo is the director of the Hawaii Music Institute and head of the music program at Windward Community College, where he teaches ukulele and slack key guitar. He created the groundbreaking Kaohekani Hawaiian music certificatea series of 8-week online classes taught by some of Hawaiis legendary artists.

Kimokeo shares his passion for music and instills in his students the joy of learning.

Ive come away from his courses a better musician and have a much better understanding and appreciation for the music of Hawaii, said one student. I am very proud to say that I have composed my own song.

The American Educational Research Association recently recognized Kimokeo for his research on song composition and performance as educational tools of personal empowerment. He earned his PhD in curriculum and instruction with a music emphasis, and his MEdT from UH Mnoa.

Kimokeo performs with Jerry Santos and his own N Hk Hanohano award-winning group Hiikua.

Monica LaBriola is an assistant professor of history in the University of Hawaii at Mnoa College of Arts, Languages & Letters. Her work focuses on engaging, yet challenging approaches to the area of Pacific studies, at the forefront of instructional excellence at UH Mnoa, while touching lives beyond the academic community.

At public forums and conferences, LaBriola draws diverse cultural workers passionate about the Pacific region as well as academics. Her guidance and vision on the development of resources in this area is praised by a colleague, who said that LaBriolas editorship of Teaching Oceania has impacted education across Hawaii, the Pacific, nationally and internationally.

She initiated and led two cohorts of Women in Pacific Studies, and is lauded by colleagues and students for successfully supporting the education of the student community experiencing the least educational equity at UH Mnoa and across the UH System.

A cohort member wrote, Professor LaBriola acknowledges the complexity of the university and encourages us to continue in academia while also dreaming of alternatives to knowledge production and dissemination.

Donald K. Maruyama is a culinary arts professor at Leeward Community College. Prior to joining Leeward CC as a chef instructor in 2007, he spent more than 20 years in the food and beverage industry.

He served as the culinary arts program coordinator from 2016 to 2020. For the past three years, Maruyama has been the professional arts and technology division chair, overseeing the automotive technology, culinary arts and digital media programs.

Dons strength as an instructor is his enthusiasm to share his personal experiences to his students about how true and real it is working in the industry as he does not sugarcoat, said Ron Umehira, dean of career and technical education. His strengths as a program and division colleague are his patience to listen, gather the facts, analyze and then support the best course of action.

Maruyama attended Kapiolani Community College, the University of Hawaii at Mnoa and Grinnell College. He currently serves as a Hawaii Culinary Education Foundation advisory board member, Hawaii Food & Wine Festival committee member, and on the board of directors for Hawaii Restaurant Association Education Foundation.

Summer Maunakea is an assistant professor in curriculum studies in the University of Hawaii at Mnoa College of Education. She grounds her teaching practices in academic rigor, agency and aloha. A colleague described observing her as expertly weaving place-based teaching and learning, ina (land)-based education and stewardship and Indigenous epistemology and practice.

She holds herself to high expectations as a teacher, knowing her instruction must have a positive intergenerational impact for students to grow holistically into healthy individuals capable of making pono (righteous) decisions and contributing to their communities.

For me, this is what love looks like in education, said a graduate student. The love and community that Professor Maunakea cultivates in the classroom supports immense intellectual experimentation and risk taking. I am immensely grateful for her teaching.

To a senior colleague, her teaching, research and service are considered to be visionary, meaningfully advocating for Indigenous education, sustainability, eco-justice, inclusive outdoor education and school-community partnerships.

Alexander Stokes is an assistant professor of cell and molecular biology at the John A. Burns School of Medicine at the University of Hawaii at Mnoa. They developed practices to create inclusive, rigorous classroom settings with each student fully engaged. One method, Problem-Based Learning, values students directing their own learning, developing team-learning skills and assuming very active roles in their education.

Stokes developed a tool kit for inclusive pedagogy reflecting under-represented, predominantly female, low-income, first-generation students in undergraduate classes. A student said, Professor Stokes utilizes a cutting-edge hybrid teaching style that unlocks students intellectual potential by acting as a conductor of a symphony in a collaborative learning orchestra. I was imbued with a passion and was inspired to further academic pursuits.

A colleague said, Alex is that professor, the one who transports students to a new view of themselves. Stokes is a leader in pedagogical innovation at the interface between biology, biomedicine and data science education in Hawaii.

Shawn Sumiki is an instructor of culinary arts at Hawaii Community College. Known for his outstanding work ethic, calm demeanor and generosity, Sumiki has taught at Hawaii CC since 2008 and is an alumnus of the program he now leads.

Culinary Arts students value his talent and experience, and appreciate the positive environment he creates in the program.

Chef Shawn is an incredible teacher, and I am so grateful to have him as my culinary instructor, one student wrote in support of his nomination. He creates a friendly environment around him that encourages learning and growth.

Sumiki is very supportive of campus events and collaborates frequently with community partners on opportunities that provide students with real-world experience and networking in the food and hospitality industry. He has donated his time and talent preparing meals to support disaster relief efforts on Hawaii Island. In 2019, Sumiki was honored with the Hawaii Community College Outstanding Service Award.

Maureen Mo Tabura is an assistant professor in the nursing program at Kauai Community College and has been teaching for more than 17 years. She has been the nursing program coordinator since 2016. She and Division Chair Tammie Napoleon are the face of the Kauai CC nursing program.

Professor Maureen Mo Tabura is one of a kind. Her commitment to teaching is immeasurable, said nursing student Ma Suerte Rebucal. She is not only excellent in imparting her knowledge through her life-changing lectures, but she brings out the best in us. I have encountered great lecturers as well as teachers who bring out the best in their students, but I have never seen someone who does both except for Professor Mo.

Tabura earned her BS of Nursing at the College of New Jersey, and her Masters in Nursing Education from UH Mnoa. She was a UH Community Colleges Leadership Champion from 2011 to 2012. She has been a board member of Kauai United Way since 1996, receiving the Founders Award in 2008. Tabura also served on the Kauai County Board of Ethics from 2014 to 2018.

Eli Tsukayama is an associate professor of marketing at UH West Oahu. His research focuses on understanding individual differences (e.g., personality traits) that can be used to segment and understand target markets. He has an extensive background in statistics as well as seven years of experience working in the corporate world as an Information Technology consultant.

One of Tsukayamas students said, Although I was nervous in the beginning with his reminder of how hard the course was, I decided to stick it out and Im glad I did because I learned a lot of life lessons from himto take criticism as a lesson, or how to properly ask qualitative and quantitative questions.

Tsukayama was among the authors of A megastudy of text-based nudges encouraging patients to get vaccinated at an upcoming doctors appointment. The paper was published on April 29 in the Proceedings of the National Academy of Sciences, the official journal of the National Academy of Sciences and one of the worlds most-cited and comprehensive multidisciplinary scientific journals.

Rosemary Rosie Vierra is a professor and the coordinator for UH Maui Colleges dental hygiene program. She has taught in the program since 2008. Many students attest to Vierras dedication and passion for the success of her students as evident in the hours she spends teaching, coordinating and striving to elevate the program.

One student described Vierras authentic concern for student well-being and success: She always puts the students first and makes us feel like our voices matter. She not only cares about our success but also our personal well-being. She is a big advocate for mental health, which I appreciate very much.

Vierras energy and connection with the community enable her to create enriching learning opportunities such as service learning, outreach to public high schools and partnerships with businesses and organizations that provide students valuable experiences in the field.

One student said, Since the beginning of our cohort in the fall of 2021, she has gone above and beyond for us students to succeed. Her priority is always to help us succeed, whether its volunteering to help us meet our clinical requirements to finding us patients.

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Board of Regents medals awarded for teaching excellence ... - University of Hawaii