StemCell Therapy

The Southern Pines Town Council unanimously approved a $1.6 million deal to acquire the 157-acre Whitehall Tract during a special called meeting Thursday.

Located on Pee Dee Road, the mostly undeveloped land abuts the towns existing Reservoir Park and nearly doubles the size of this popular recreation destination.

An initial down payment of $250,000 is due next week with closing scheduled on Tuesday, Dec. 15. Two additional payments totaling $575,000 will be paid next year, with a final balloon payment of $822,300 due in spring 2022.

Approximately one-third of the property, which is also known as the Drexel Land, is under a conservation easement and there is an existing 2-mile public walking trail on the tract.

Former property owner Barbara Sherman, a retired veterinarian and professor of North Carolina State Universitys College of Veterinary Medicine, initially approached the town several years ago about the potential land deal. Her goal was to see the open space, biodiversity and natural beauty of the property preserved and protected from future development.

Check out next Wednesdays edition of The Pilot to learn more about the history of Whitehall and how its owners, past and present, have worked to protect this treasured property.

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Southern Pines Adds 175-Acre Site to its Parks & Recreation Facilities - Southern Pines Pilot

Compared with White people, those who are Black have higher cardiovascular risk when early-onset type 2 diabetes (T2D) develops, according to the results of a study published in Diabetes Care.

Studies have shown an increase in the incidence of early-onset T2D diagnosis and a greater risk for T2D among Black people when compared with White people, but the evaluation of ethnicity-specific temporal trends of early-onset T2D diagnosis and the impact of comorbidities at the time of diagnosis had not yet been conducted.

To investigate and compare the trends in early-onset T2D diagnosis of Black and White people as well as the trends of various risk factors at the time of T2D diagnosis, data from 606,440 individuals aged 18 to 70 years who were diagnosed with T2D between 2000 and 2018 were analyzed. Atherosclerotic cardiovascular disease (ASCVD) was defined by the presence of a clinical diagnosis of ischemic heart disease.

Over the past 2 decades, the rate of T2D diagnosis among Black patients within the age groups 18 to 39 and 40 to49 years has consistently been higher than that of their White counterparts (P <.01). The proportion of both Black and White patients diagnosed with T2D before 50 years of age increased significantly from 2012 to 2018 (P <.05).

Black patients had significantly higher mean hemoglobin A1c (HbA1c) than White patients across all age groups. Although no difference was seen in other age groups, Black patients 18 to 39 years of age had significantly higher body mass index (BMI) than their White counterparts (P =.02).

MACE-3 refers to 3-point major adverse cardiovascular events heart failure, myocardial infarction, or stroke. Black patients had a significantly higher risk for MACE-3 compared with White patients across all age groups, with the youngest age group having the highest relative risk (hazard ratio [HR], 1.63; 95% CI, 1.42-1.88) and the lowest relative risk observed in the oldest age group (HR, 1.11; 95% CI, 1.06-1.15).

Taken as a whole, the results of this study illustrated the increasing burden of early-onset T2D and the increased risk of MACE-3 for Black patients. Understanding the trends in diagnosis of early-onset T2D and the differences in the prevalence of related comorbidities among people of different ethnicities may improve healthcare practitioners ability to detect and manage this disease.

Limitations of this study include the use of electronic medical records, which may have resulted in errors in data collection due to condition coding, and the fact that the database did not link directly to hospitalized data.

Future investigation into potential explanations for the differences between ethnicities observed in this study are warranted.

Reference

Dibato JE, Montvida O, Zaccardi F, et al. Association of cardiometabolic multimorbidity and depression with cardiovascular events in early-onset adult type 2 diabetes: a multiethnic study in the US. Published online November 11, 2020. Diabetes Care. doi: 10.2337/dc20-2045

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Differences in Comorbidities and Diagnosis of Early-Onset Type 2 Diabetes by Ethnicity - Endocrinology Advisor

This article was originally published here

BMC Cardiovasc Disord. 2020 Dec 9;20(1):514. doi: 10.1186/s12872-020-01805-6.

ABSTRACT

BACKGROUND: This study aimed to reveal the association of diabetes mellitus and dialysis-dependent renal failure with the lesion severity and chronic total occlusion (CTO) in patients undergoing femoropopliteal endovascular therapy for intermittent claudication.

METHODS: This multicenter retrospective study analyzed the data of 2056 consecutive patients with moderate to severe intermittent claudication, who underwent endovascular therapy for de novo lesions in the superficial femoral artery to the proximal popliteal artery between 2010 and 2018 at five cardiovascular centers in Japan. The association of the clinical characteristics with severity of the lesions, as assessed by the Trans-Atlantic Inter-Society Consensus (TASC) II classification, was investigated using the ordinal logistic regression model. Their association with CTO, lesion length, and severity of calcifications was additionally analyzed using the binomial logistic regression model.

RESULTS: The prevalence of diabetes mellitus and dialysis-dependent renal failure was 54.7% and 21.4%, respectively; 12.5% of the patients had lesions corresponding to TASC II class D, and 39.3% of the patients had CTO. Current smoking and severe claudication were associated with more severe lesions assessed according to the TASC II classification; diabetes mellitus and dialysis dependence were inversely associated with disease severity. The adjusted odds ratios of diabetes mellitus and dialysis dependence were 0.82 (95% confidence interval 0.70-0.97; p = 0.018) and 0.76 (0.62-0.94; p = 0.009), respectively. Diabetes mellitus and dialysis dependence were also inversely associated with CTO (both p < 0.05). Furthermore, diabetes mellitus was inversely associated with a long lesion (p < 0.05). Diabetes mellitus and dialysis dependence were positively associated with severe calcification (both p < 0.05).

CONCLUSIONS: Diabetes mellitus and dialysis-dependent renal failure were inversely associated with the lesion severity, as assessed by the TASC II classification, and CTO in patients undergoing femoropopliteal endovascular therapy for intermittent claudication.

PMID:33297956 | DOI:10.1186/s12872-020-01805-6

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Inverse association of diabetes and dialysis with the severity of femoropopliteal lesions and chronic total occlusion: a cross-sectional study of 2056...

Dear Doctors: We keep hearing about Type 2 diabetes, and I'm embarrassed to say, I don't actually know what it is. What does it do, and how do I know if I have it?

Dear Reader: To understand diabetes, we should first talk about glucose. That's the sugar our bodies make from the foods that we eat, and which our cells use as their main source of fuel. Glucose travels throughout the body via the blood, which is why it's also often referred to as blood sugar. However, it's not immediately available to the cells. That's where insulin, a hormone manufactured by the pancreas, comes into play. Insulin helps transport glucose from the blood into the cells, where it can be used as energy.

When someone has diabetes, it means that the insulin part of that energy equation isn't working properly. Either the body isn't manufacturing enough -- or any -- insulin, or it isn't responding properly to the insulin that is present. That leads to blood-glucose levels that are too high.

Over time, high blood levels of glucose are dangerous. Adverse health effects include damage to the circulatory system, vision problems, nerve damage, stomach or intestinal problems, slow healing, kidney disease and an increase in the risk of heart disease and stroke. Extremely high blood sugar levels can lead to coma, and even death.

In Type 1 diabetes, the pancreas makes little or no insulin. It often develops early in life, but can occur at any age. This type of diabetes is managed with diet and exercise, plus the use of medications and insulin.

See more here:
Insulin is at the heart of both types of diabetes - The Times and Democrat

Data presented during the 2020 San Antonio Breast Cancer Symposium found that adhering to a diabetes risk reduction diet improved survival for women with stage 1 to 3 breast cancer compared to women who did not follow this specific diet.

Diabetes may be common in women with breast cancer, especially since 75%, or more than 2.6 million women, are at least 60 years or older, which means breast cancer survivorship must be managed in consideration with aging-related comorbidity such as diabetes, said Tengteng Wang, PhD, a research fellow at Harvard T.H. Chan School of Public Health during the virtual presentation of the study.

Type 2 diabetes, in particular, is a risk factor for breast cancer incidence and may be a predictive factor for breast cancer mortality. In addition, breast cancer increases the likelihood of developing type 2 diabetes.

Identifying modifiable strategies to prevent type 2 diabetes among breast cancer survivors may be very important to improve their survival outcomes, said Wang.

Researchers analyzed data from 8,320 women with stage 1 to 3 breast cancer from 2 large cohort studies: the Nurses Health Study (1980-2014) and the Nurses Health Study II (1991-2015). Validated questionnaires were completed every 2 to 4 years to collect information on diet among other factors.

This study focused on a diabetes risk reduction diet with 9 dietary components including higher intakes of nuts, cereal fiber, coffee, polyunsaturated-saturated fat ratio and whole fruits, in addition to a lower glycemic index of diet and lower intakes of sugar-sweetened beverages/fruit juices, trans fat and red meat.

The [diabetes risk reduction diet] has been associated with 14% lower type 2 diabetes risk in [a] previous publication of the Nurses Health Study, said Wang.

Researchers calculated an average score of adherence to this diabetes risk reduction diet through repeated measures of diet after a diagnosis of breast cancer. Follow-up was conducted for a median of 16 years after cancer diagnosis.

During follow-up, 2,146 deaths occurred, of which 948 were related to breast cancer. Women with higher diet adherence scores after diagnosis had a 33% lower risk for all-cause mortality (HR = 0.67; 95% CI, 0.58-0.78; P for trend < .0001) and a 17% lower risk for mortality related to breast cancer (HR = 0.83; 95% CI, 0.67-1.02; P for trend = .03) compared with women with lower diet adherence scores.

Our results did not differ by breast tumor ER status or stage, said Wang.

During the discussion portion of the presentation, Wang said that she and her colleagues analyzed what may be the potential mechanism for this association. She said, We looked at how [diabetes risk reduction diet][ influenced gene expression in [the] breast tumor for [a] subgroup of our breast cancer patients, and according to our pathway analysis, the [diabetes risk reduction diet] is more associated with the pathway related to immune regulation and also cell proliferation, so this is, I think, an interesting finding.

When further adjusting for neighborhood socioeconomic status, the association between diet adherence and mortality risk was slightly attenuated, with patients with greater adherence having a 31% lower risk for all-cause mortality (HR = 0.69; 95% CI, 0.6-0.8; P for trend < .0001) and a 14% lower risk for mortality from breast cancer (HR = 0.86; 95% CI, 0.7-1.07; P for trend = .06).

Women who improved their adherence to a diabetes risk reduction diet after breast cancer diagnosis had a lower risk for breast cancer mortality compared with those with consistently low adherence to this diet (HR = 0.81; 95% CI, 0.65-1).

In conclusion, we felt that a greater adherence to the [diabetes risk reduction diet] after breast cancer diagnosis was associated with better survival outcomes, which means promoting dietary changes consistent with prevention of type 2 diabetes may be very important for breast cancer survivors, said Wang.

A version of this story appeared on CURE as Dietary Changes to Reduce Diabetes Risk May Also Increase Survival for Breast Cancer.

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Diabetes Risk Reduction Diet Adherence Improved Survival for Patients with Breast Cancer - Cancer Network

TheStandards of Medical Care in Diabetes2021provides the latest in comprehensive, evidence-based recommendations for the diagnosis and treatment of children and adults with type 1, type 2, or gestational diabetes; strategies for the prevention or delay of type 2 diabetes; and therapeutic approaches that can reduce complications, mitigate cardiovascular and renal risk, and improve health outcomes.

This update presents:

Today, theStandards of Careis available online and is published as a supplement to the January 2021 issue ofDiabetes Care.

"The American Diabetes Association is committed to improving the lives of all those affected by diabetes through this publication of the most widely respected guidelines for health professionals," said Dr. Robert Gabbay, Chief Scientific and Medical Officer at the American Diabetes Association.

Updates to the Standards of Care are established and revised by the ADA's Professional Practice Committee(PPC). The committee is a multidisciplinary team of 16 leading U.S. experts in the field of diabetes care and includes physicians, diabetes care and education specialists, registered dietitians, and others with experience in adult and pediatric endocrinology, epidemiology, public health, cardiovascular risk management, microvascular complications, preconception and pregnancy care, weight management and diabetes prevention, and use of technology in diabetes management. Two designated representatives of the American College of Cardiology (ACC) reviewed and provided feedback on the "Cardiovascular Disease and Risk Management" section, and this section received endorsement from ACC. "As a world leader in diabetes care, the ADA is proud to set the standards!" said Boris Draznin, MD, PhD, Chair of the Professional Practice Committee.

The online version of the Standards of Care will continue to be annotated in real-time with necessary updates if new evidence or regulatory changes merit immediate incorporation through the living Standards of Care process. The ADA also publishes the abridged Standards of Careyearly for primary care providers in its journal, Clinical Diabetes, and offers a convenient Standards of Care appas well as a Standards of Care pocket chart. Other Standards of Care resources, including a webcastwith continuing education credit and a full slide deck, can be found on DiabetesPro.

About Diabetes CareDiabetes Care,a monthly journal of the American Diabetes Association (ADA), is the highest-ranked, peer-reviewed journal in the field of diabetes treatment and prevention. Dedicated to increasing knowledge, stimulating research and promoting better health care for people with diabetes, the journal publishes original articles on human studies in clinical care, education and nutrition; epidemiology, health services and psychosocial research; emerging treatments and technologies; and pathophysiology and complications. Diabetes Care also publishes the ADA's recommendations and statements, clinically relevant review articles, editorials and commentaries. Topics covered are of interest to clinically oriented physicians, researchers, epidemiologists, psychologists, diabetes care and education specialists and other health care professionals.

About the American Diabetes AssociationEvery day more than 4,000 people are newly diagnosed with diabetes in America. More than 122 million Americans have diabetes or prediabetes and are striving to manage their lives while living with the disease. The American Diabetes Association (ADA) is the nation's leading voluntary health organization fighting to bend the curve on the diabetes epidemic and help people living with diabetes thrive. For 80 years the ADA has been driving discovery and research to treat, manage and prevent diabetes, while working relentlessly for a cure. We help people with diabetes thrive by fighting for their rights and developing programs, advocacy and education designed to improve their quality of life. Diabetes has brought us together. What we do next will make us Connected for Life. To learn more or to get involved, visit us at diabetes.org or call 1-800-DIABETES (1-800-342-2383). Join the fight with us on Facebook (American Diabetes Association), Twitter (@AmDiabetesAssn) and Instagram (@AmDiabetesAssn).

Contact:Daisy Diaz, 703-253-4807[emailprotected]

SOURCE American Diabetes Association

http://www.diabetes.org

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ADA Releases 2021 Standards of Medical Care in Diabetes Centered on Evolving Evidence, Technology, and Individualized Care - PRNewswire

ANNAPOLIS, MDGovernor Larry Hogan today announced the commitment of more than $94 million in new investments across Maryland to help people with prediabetes and diabetes prevent or manage their disease during the COVID-19 pandemic.

The $94 million of new investments I am announcing today will be used to help Marylanders across the state battle diabetes, said Governor Hogan. Even in the midst of a pandemic, diabetes continues to be one of the most devastating health issues in our state. And having diabetes puts individuals at risk of serious illness from COVID-19, so this comes at an important time.

There are currently more than 2.1 million Marylanders with either diabetes or prediabetes, more than 34% of the total population, and many dont even know it. Diabetes is the 6th leading cause of death in Maryland, and people with Type 2 diabetes have a greater risk of serious illness from COVID-19, according to the Centers for Disease Control and Prevention (CDC).

Taken together, the measures being funded represent the first major community-based initiatives resulting from the Diabetes Action Plan, published last November.Developed with extensive input from community partners and diabetes experts, the Diabetes ActionPlan catalogs the state of this disease among Maryland residents and outlines comprehensive community-based prevention, management and treatment initiatives.

Todays initiatives announced by the governor include:

Regional Catalyst Grant Program

Recently the Health Services Cost Review Commission (HSCRC) announced the award of its new competitive Regional Partnership Catalyst Grant Program, an investment of more than $86.3 million in Maryland hospitals and their community health resource partners to support diabetes prevention, education, and self-management training programs. The HSCRC grant provides an initial five-year funding stream to help hospitals launch interventions that can be sustained at the end of the grant.

CareFirst Investments in Community Health

CareFirst BlueCross BlueShield will invest more than $6.6 million in four Maryland communities as part of a multi-year initiative to combat social and health disparities for people who are at risk for or have been diagnosed with diabetes. Thispledge will focus on steps and strategies that can be taken to intervene in areas where extensive data shows community and social factors contribute to the onset of diabetes. This work will be done in partnership with Local Health Improvement Coalitions (LHICs) and local health departments. LHICs are comprised of local partners, working with the health officers to create meaningful public-private partnerships supporting community wellness.

CHRC Partners with MDH and UMD to Support Local Health Coalitions

Marylands Community Health Resource Commission (CHRC), consistently supportive of the Diabetes Action Plan, recently awarded $1 million to Marylands local health departments to help LHICs expand capacity and build on innovative partnerships, services, and programming in communities at high-risk for diabetes.In an effort to maximize the impact of the grant funding, MDH will provide technical assistance to LHICs,in partnership with the Horowitz Center for Health Literacy in the School of Public Health, University of Maryland.

In addition, last year the CHRC issued 12 awards totaling $2.2 million to support projects in local communities in support of the Maryland Diabetes Action Plan through promoting food security and addressing other social determinants that impact diabetes.Addressing diabetes is a top funding priority of the CHRC, focusing on the Diabetes Action Plan recommendations to employ local action integrated with community approach in the fight against diabetes.

Along with these investments, Marylands Medicaid program added the National Diabetes Prevention Programs (NDPP) as a covered benefit last fall, providing both in-person and virtual access for eligible HealthChoice enrollees who may not have otherwise been able to afford it. The DPP is available through all nine of its Managed Care Organizations statewide.

Marylanders are urged to speak to their physician about their risk for diabetes, and go online to know their risk. An easy 60-second risk test from the American Diabetes Association can help everyone: https://www.diabetes.org/risk-test

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Governor Larry Hogan Announces $94 Million in Funding to Support Diabetes Prevention and Treatment for Marylanders - The Southern Maryland Chronicle

Why Global Citizens Should Care

Noncommunicable diseases (NCDs), such as cancers and diabetes, are now the leading cause of death in the world, according to the World Health Organizations (WHO) 2019 Global Health Estimates report published Wednesday.

The top10 diseases accounted for 55% of the 55.4 million deaths worldwide that were reported in 2019.

It is important to understand which diseases people die from to measure how people live and to ensure that health care systems areprepared to respond to patients needs, according to the WHO.

The WHOdefines NCDs as chronic diseases that are usually the result of genetic, physiological, environmental, and behavioral factors. In comparison, communicable diseases are transmitted between people, such as HIV/AIDS, tuberculosis, malaria, and tropical diseases.

At the global level, 7 of the 10 leading causes of death were NCDs in 2019. This is a dramatic rise from 2000, when only 4 of the top 10 diseases were classified as noncommunicable.

These new estimates are another reminder that we need to rapidly step up prevention, diagnosis,and treatment of noncommunicable diseases, WHO Director-GeneralDr. Tedros AdhanomGhebreyesussaid.

Related Stories Dec. 9, 2020 Thomson Reuters Foundation Poor Countries Are at Risk of Missing Out on COVID-19 Vaccines as Rich Nations Hoard Supplies

While the WHOs list outlines the top causes of death globally, the leading causes of death within eachcountry arevaried. The causes of death across high-,middle-,and low-income countries showthe difference between lifestyle and health care around the world.

In 2019, heart disease remained the number one killer globally and accounted for 16% of total diseases around the world, according to the report.

Heart disease has been the leading cause of death globally for the last 20 years. The number of people who died from the disease in 2019 rose to almost 9 million.

Related Stories Nov. 20, 2020 The WHO Just Introduced a Plan to Eliminate Cervical Cancer Around the World

For the first time, Alzheimers disease and other forms of dementia entered the top 10 causes of global death.The report also showed that women were more at risk of degenerative diseases, as 65% of Alzheimers and other forms ofdementia deaths were women.

Diabetes also entered the top 10 list in 2019. The number of people who died from the disease has increased by 70% since 2000. Men are more at risk of this disease and there was an 80% rise in death among men in the past two decades.

While Alzheimers disease, dementia, and diabetesare all classified as NCDs, none of themwere amongthe top 10 causes of death in low- or middle-income countries.

Related Stories Nov. 30, 2020 Malaria Will Kill More People Than COVID-19 in Sub-Saharan Africa This Year: WHO

Deaths from communicable diseases declined globally from 2000 to 2019, however, they are still a challenge in lower- and middle-income countries. In low-income countries, 6 of the top 10 causes of death were communicable diseases.

HIV/AIDS was nolonger listedamong the top 10 global diseases in 2019. Deaths from HIV/AIDS have decreased by 51% globally since 2000.However, in low-income countries, HIV/AIDS, malaria, and tuberculosis remainedin the top 10.

Dr. Samira Asma, assistant director-general for the division of data, analytics,and delivery for impact at the WHO, explained the importance of collecting data on worldwide deaths in the report.

The WHO Global Health Estimates are a powerful tool to maximize health and economic impact, she said. We call upon governments and stakeholders to urgently invest in data and health information systems to support timely and effective decision-making.

Continued here:
Noncommunicable Diseases Like Cancer and Diabetes Are the Top Causes of Death Globally: WHO - Global Citizen

By: Express Explained | Updated: December 12, 2020 5:18:59 pmA significant finding of their study says that owning a dog with diabetes was associated with a 38 per cent increased risk of diabetes in the owner.(Thinkstock)

A new study published in the British Medical Journal (BMJ) says that diabetes in dogs may indicate an elevated risk of type 2 diabetes in their owners.

The study

The study included more than 175,000 dog owners and nearly 90,000 cat owners along with their dogs and cats. The dog and cat owners included in the study were all middle-aged or older at the start of the study and were followed through for a period of six years (January 1, 2007 December 31, 2012).

Through this period, researchers analysed the incidence of type 2 diabetes in the pet owners and canine and feline diabetes in their pets.

So what does the research say?

Authors of the study, which was conducted at Uppsala University in Sweden in collaboration with three other universities, say that owners of a dog with diabetes are more likely to develop type 2 diabetes than owners of a dog without diabetes. A similar shared risk of diabetes could not be detected for cat owners and their pets, the researchers have said.

What could explain this association?

A significant finding of their study says that owning a dog with diabetes was associated with a 38 per cent increased risk of diabetes in the owner. Beatrice Kennedy, one of the senior authors of the study was quoted as saying in a press release that the association of diabetes between dogs and their owners might be explained by physical activity patterns, possibly also by their shared dietary habits and adiposity. The WHO defines adiposity as having a body mass index (BMI) of over 30 kg per metre square.

Significantly, if the reason for the association of a shared risk of the disease between dogs and their owners is indeed a result of their physical activity patterns, it would also explain why the researchers did not see a shared risk association of the disease between cats and their owners. Follow Express Explained on Telegram

Humans and dogs have lived together for at least 15,000 years, and continue to share their everyday lives for better or worse. In this unique study, we show that there might be common lifestyle and environmental factors that influence the risk of diabetes in the household, both in the dogs and in their owners, Tove Fall, another author of the study was quoted as saying.

What is type 2 diabetes?

Type 2 diabetes is the most common type of diabetes and occurs when blood glucose or blood sugar is too high and is most likely a result of excess body weight and physical inactivity. According to the World Health Organisation (WHO), this type of diabetes was seen only in adults but is now also occurring increasingly frequently in children. Overall, 422 million adults in the world have diabetes, including type 1.

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Explained: What a new study says about the shared risk of diabetes between dog and cat owners and their pets - The Indian Express

THE WOODLANDS, Texas, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the development of LX9211 in diabetic peripheral neuropathic pain.

The FDAs Fast Track designation of LX9211 reflects the serious unmet medical need of people suffering from diabetic peripheral neuropathic pain, said Praveen Tyle, Ph.D., executive vice president of research and development. We look forward to working closely with the FDA throughout the clinical development process to bring this potential new innovative treatment to patients as quickly as possible.

Lexicon is currently enrolling patients with diabetic peripheral neuropathic pain in a Phase 2 proof-of-concept study of LX9211 and is preparing to initiate a second Phase 2 clinical trial of LX9211 in post-herpetic neuralgia.

The FDAs Fast Track designation is designed to facilitate the development and expedite the review of drugs that are being developed to treat serious conditions and fill unmet medical needs. The purpose of the designation is to expedite the timeline for bringing important new drugs to patients. Programs receiving Fast Track designation may benefit from early and frequent interactions with the FDA over the course of drug development. In addition, the Fast Track designation program provides eligibility for accelerated approval and priority review if relevant criteria are met and enables sponsors to submit individual sections of a New Drug Application (NDA) for review on a rolling-submission basis.

About LX9211

LX9211 is a potent, orally delivered, selective small molecule inhibitor of adapter-associated kinase 1 (AAK1). Lexicon identified AAK1 in its target discovery efforts as a promising approach for the treatment of neuropathic pain, and identified LX9211 and another development candidate in a neuroscience drug discovery alliance with Bristol-Myers Squibb from which Lexicon holds exclusive development and commercialization rights. Preclinical studies of LX9211 demonstrated central nervous system penetration and reduction in pain behavior in models of neuropathic pain without affecting opiate pathways.

About Lexicon Pharmaceuticals

Lexicon is a biopharmaceutical company with a mission of pioneering medicines that transform patients lives. Through its Genome5000 program, Lexicon scientists studied the role and function of nearly 5,000 genes and identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Through the precise targeting of these proteins, Lexicon is pioneering the discovery and development of innovative medicines to safely and effectively treat disease. Lexicon advanced one of these medicines to market and has a pipeline of promising drug candidates in discovery and clinical and preclinical development in neuropathic pain, heart failure, diabetes and metabolism and other indications. For additional information, please visit http://www.lexpharma.com.

Safe Harbor Statement

This press release contains forward-looking statements, including statements relating to Lexicons financial position, long-term outlook on its business and the clinical development and therapeutic and commercial potential of its drug candidates. In addition, this press release also contains forward looking statements relating to Lexicons growth and future operating results, discovery and development of products, strategic alliances and intellectual property, as well as other matters that are not historical facts or information. All forward-looking statements are based on managements current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including Lexicons ability to meet its capital requirements, successfully conduct preclinical and clinical development and obtain necessary regulatory approvals of LX9211, sotagliflozin and its other potential drug candidates on its anticipated timelines, achieve its operational objectives, obtain patent protection for its discoveries and establish strategic alliances, as well as additional factors relating to manufacturing, intellectual property rights, and the therapeutic or commercial value of its drug candidates. Any of these risks, uncertainties and other factors may cause Lexicons actual results to be materially different from any future results expressed or implied by such forward-looking statements. Information identifying such important factors is contained under Risk Factors in Lexicons annual report on Form 10-K for the year ended December 31, 2019, as filed with the Securities and Exchange Commission. Lexicon undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.

For Inquiries:

Chas SchultzExecutive Director, Corporate Communications and Investor RelationsLexicon Pharmaceuticals(281) 863-3421cschultz@lexpharma.com

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Lexicon Pharmaceuticals Receives Fast Track Designation From the FDA for LX9211 for Diabetic Peripheral Neuropathic Pain - GlobeNewswire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, today announced final clinical results from its earlier completed Phase 1 clinical trial as well as development updates for its MGTA-145 stem cell mobilization therapy, including commencement of enrollment in a Phase 2 clinical trial in multiple myeloma, and its plans for a Phase 2 clinical trial in allogeneic stem cell transplant for patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndrome (MDS). The company also previously announced a clinical collaboration with bluebird bio to evaluate MGTA-145 for mobilizing and collecting stem cells in adults and adolescents with sickle cell disease (SCD). Additional preclinical results were also presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020, on the Magenta conditioning platform, including MGTA-117 program, which is a targeted antibody-drug conjugate (ADC) to prepare patients for stem cell transplant.

MGTA-145 Advancement to Phase 2 Development in Blood Cancers

The company announced that enrollment has started and is ongoing in a Phase 2 clinical trial of MGTA-145, used in combination with plerixafor, to mobilize and collect stem cells for autologous stem cell transplantation of multiple myeloma patients at Stanford University. Magenta expects that this trial will provide patient-level data on stem cell mobilization and collection, characteristics of the mobilized graft and engraftment in patients with multiple myeloma.

Additionally, through a collaboration with the National Marrow Donor Program/Be The Match, a global leader in facilitating allogeneic hematopoietic stem cell transplantation, Magenta plans to initiate a Phase 2 clinical trial in early 2021 using MGTA-145 to mobilize and collect stem cells from allogeneic donors for transplant in patients with AML, ALL and MDS. Allogeneic stem cell transplant provides a potentially curative therapeutic option for patients with these diseases. This clinical trial will evaluate stem cell mobilization, collection, cell quality, engraftment and the potential for reduced Graft-versus-Host Disease (GvHD), which is of particular importance in the allogeneic transplant setting.

MGTA-145 in Sickle Cell Disease

Magenta Therapeutics recently announced an exclusive clinical collaboration with bluebird bio to evaluate the utility of MGTA-145, in combination with plerixafor, for the mobilization and collection of stem cells in adults and adolescents with SCD.

The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. bluebird bios experience with plerixafor as a mobilization agent in SCD aligns with Magentas combination therapy approach, utilizing MGTA-145 plus plerixafor with potential for safe, rapid and reliable mobilization of sufficient quantities of high-quality stem cells to improve outcomes associated with stem cell transplantation.

MGTA-145 Presentations at ASH

Magenta presented final clinical data from its MGTA-145 stem cell mobilization Phase 1 clinical trial in healthy volunteers at the ASH Annual Meeting. All primary and secondary endpoints were met in the study completed earlier this year.

The results demonstrate that a single dose of MGTA-145, in combination with plerixafor, rapidly and reliably mobilized high numbers of stem cells in a single day without the need for G-CSF for potential use in diseases that can benefit from autologous and/or allogeneic stem cell transplantation. The additional data also offer further confirmation that MGTA-145, in combination with plerixafor, was well tolerated and provides a rapid and reliable method to obtain large numbers of hematopoietic stem cells. Transplant of these cells in preclinical models resulted in enhanced, durable engraftment, in addition to highly immunosuppressive properties, leading to reduced GvHD.

Results from this study provide a robust dataset and proof of concept that MGTA-145, in combination with plerixafor, provides rapid and robust mobilization of stem cells and that these cells have better engraftment potential, are able to be gene modified and engraft and reduce GvHD in preclinical models compared to cells mobilized with other available agents. The data reinforce the availability of compelling opportunities for development in both the autologous and allogeneic transplant settings, said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics.

The data were presented by Steven M. Devine, MD, Chief Medical Officer of the National Marrow Donor Program/Be The Match and Associate Scientific Director of the CIBMTR (Center for International Blood and Marrow Transplant Research).

Conditioning Program (MGTA-117 and CD45-ADC) Presentations at ASH

Magenta also provided updates on its conditioning platform at the ASH Annual Meeting, including MGTA-117 and CD45-ADC programs. Preclinical data from a study of MGTA-117 demonstrate that it is an effective, potent conditioning agent for transplant with anti-leukemic activity, significantly decreasing tumor burdens, leading to delayed tumor growth and increased median survival rates in animal models of AML. Ongoing GLP toxicology and GMP manufacturing progress continue to be supportive of advancing MGTA-117 towards an IND filing in AML and MDS.

Additionally, preclinical data from a study of Magentas CD45-ADC, a CD45-targeted conditioning agent designed to remove the cells that cause autoimmune diseases to enable curative immune reset, demonstrated the ability to achieve successful outcomes as a single agent in the most challenging disease model through fully mismatched allogeneic hematopoietic stem cell transplant, where only radiation or combinations of toxic chemotherapies are available, potentially providing patients the option of a reduced toxicity conditioning regimen. The company continues to evaluate this program preclinically.

About MGTA-145

MGTA-145 is being developed in combination with plerixafor to harness complementary chemokine mechanisms to mobilize hematopoietic stem cells for collection and transplantation. This new combination has the potential to be the preferred mobilization regimen for rapid and reliable mobilization and collection of hematopoietic stem cells to improve outcomes in autologous and allogeneic stem cell transplantation, which can rebuild a healthy immune system for patients with blood cancers, genetic diseases and autoimmune disorders.

MGTA-145 has the potential to replace the current standard of care for patients and allogeneic donors who currently rely on the use of granulocyte-colony stimulating factor (G-CSF) alone or in combination with plerixafor, which can take up to five days or longer to mobilize sufficient numbers of stem cells, often resulting in significant bone pain and other side effects.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with blood cancer, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics Announces Commencement of First Phase 2 Clinical Trial of MGTA-145 for Stem Cell Mobilization, Oral Presentation of MGTA-145...

The combination of daratumumab (Darzalex) with lenalidomide, bortezomib, and dexamethasone (D-RVd) followed by maintenance therapy with daratumumab and lenalidomide (D-R) improved response rates and depth of response rates with statistically significance as treatment of patients with transplant-eligible newly diagnosed multiple myeloma compared with RVd followed by lenalidomide maintenance, according to findings presented at the 2020 American Society of Hematology (ASH) Annual Meeting.

The updated analysis of the GRIFFEN study showcased that D-R maintenance therapy improved the depth of response for patients while maintaining remissions.

As noted, these results support D-RVd as a potential new standard of care for transplant eligible newly diagnosed multiple myeloma patients, Jonathan L. Kaufman, MD, Winship Cancer Institute, Emory University, said in his presentation of the data.

The researchers found in earlier data cuts that the responses from patients to these treatments deepened over time, with response rates and depths greater for patients in the D-RVd arm at all points in time. At the end of consolidation, the stringent complete response (sCR) rate was 42.4% in the D-RVd arm (n = 104) compared to 32% in the RVd arm alone (n = 103; P < .0014).

For the data regarding the 12 months of maintenance cutoff, the sCR rate was 63.6% versus 47,4% for the D-RVd arm and RVd alone arm, respectively.

Researchers also examined minimal residual disease (MRD)-negativity after 12 months of maintenance therapy, finding that, in an intent to treat manner, the total MRD-negative rate in the D-RVd arm was 62.5% compared to 27.2% for the RVd alone arm (P < .0001). Solely examining the MRD evaluable group of patients, the MRD-negativity rate was 78.3% for the D-RVd arm (n = 83) versus 39.4% for the RVd alone arm (n = 71; P < .0001).

More, durability of MRD-negativity at both 6 and 12 months was significantly higher in the D-RVd arm versus the RVd alone arm. For 6 months, the MRD-negative rate was 37.5% in the D-RVd arm, compared to only 7.8% in the RVd alone arm (P < .0001). At 12 months, the rates for the D-RVd and RVd alone arms were 28.8% and 2.9%, respectively (P < .0001). The data showed a significant improvement in the rates of sustained MRD-negativity for the D-RVd arm versus RVd alone.

From an MRD-negativity standpoint, there is not a subgroup of patients that does not benefit from adding DARA to RVd treatment.

D-RVd is now considered a treatment that is appropriate for (the National Comprehensive Cancer Network). I know that we have made this our standard of care at our center, explained Kaufman It is a regimen that is well tolerated, its effective, it doesnt negatively impact the ability to collect stem cells or patients to engraft after stem cell transplant. The goal of our induction therapy is to induce MRD-negatively and what we see here is that this regimen is very good at reducing MRD-negativity.

The primary end point of the study was sCR, with secondary end points including MRD-negativity rates and overall response rates (ORR).

The researchers also explained that there is currently no statistical difference between progression-free or overall survival in both arms, with a median follow-up of 27.4 months.

While there was a noticeable increase in rates of neutropenia and upper respiratory infections for the D-RVd group compared to RVd alone, these increased rates did not lead to higher rates of discontinuation for patients. More, similar rates of any grade and grade3/4 infections were observed for D-RVd versus RVd.

Looking ahead, there is an ongoing phase 3 PERSEUS study that is evaluating subcutaneous D-RVd in the transplant-eligible population of patients with newly diagnosed multiple myeloma.

Reference

Kaufman JL, Laubach J, Sborov DW, et al. Daratumumab (DARA) Plus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Patients with Transplant-Eligible Newly Diagnosed Multiple Myeloma (NDMM): Updated Analysis of Griffin after 12 Months of Maintenance Therapy. Presented at: 2020 ASH Annual Meeting and Exposition; December 5-8, 2020; Virtual. Abstract 549.

Originally posted here:
Daratumumab Regimen Shows Promise in Transplant-Eligible Patients With Newly Diagnosed Myeloma - Targeted Oncology

Allogeneic hematopoietic stem cell transplant (allo-HSCT) may be a curative option for some patients with relapsed/refractory T-cell lymphoma (TCL), according to results of a retrospective study presented at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.

Outcomes from previous registry studies suggest that only about one-third of patients with relapsed TCL are disease-free 3 years after allo-HSCT, including a prior study by this same group of researchers, which found the 2-year PFS and OS to be 48.9% and 61.7%, respectively.

The group conducted the current study to better understand the dynamics of allogeneic transplant in T-cell lymphoma and with longer follow-up, Neha Mehta-Shah, MD, of the Washington University School of Medicine in St. Louis, Missouri, and presenter of the study, said.

The retrospective study included 508 consecutive patients with TCL who underwent allo-HSCT between 2000 and 2019 across 12 academic institutions. The median age of patients in the cohort was 51 years, and at the time of transplant, 54.4% were in complete remission (CR), 37.2% were in partial remission (PR), 5% had stable disease, and 3.2% had progressive disease. There were 15.5% of patients who had undergone prior autologous transplant. The conditioning regimens used prior to transplant were myeloablative in 35.4%, reduced intensity/nonmyeloablative in 63.6%, and unknown in less than 1% of patients.

After allo-HSCT, the 2- and 5-year progression-free survival (PFS) rates were 45.8% and 39.4%, respectively.

The time from relapse to death post allo-HSCT was a median 10.2 months. The 2- and 5-year overall survival (OS) was 59.1% and 50.8%, respectively. Among the 261 deaths that were recorded, 31% were transplant-related, 26.4% were due to progressive disease, and 42.5% were nonrelapse-related and/or unknown.

PFS was similar among patients with the angioimmunoblastic, not otherwise specified, and ALK-positive or -negative anaplastic large cell subtypes. Although PFS outcomes were worse for patients with cutaneous TCL (P =.0008), OS outcomes were not significantly different from other TCL subtypes (44% for cutaneous TCL vs 53.1% for others; P =.4573).

CR at the time of transplant was associated with longer median PFS and OS, with progressive disease associated with the shortest PFS and OS.

Dr Mehta-Shah concluded that in the largest series of allogeneic transplant to date, allogeneic transplant for patients with TCL can lead to durable remissions in patients with otherwise poor outcomes.

Disclosures: Some of the presenters disclosed financial relationships with the pharmaceutical industry and/or the medical device industry. For a full list of disclosures, please refer to the presentation abstract.

Read more of Cancer Therapy Advisors coverage of the ASH 2020 meeting by visiting the conference page.

Reference

Mehta-Shah N, Kommalapati A, Teja S, et al. Successful treatment of mature T-cell lymphoma with allogeneic stem cell transplantation: the largest multicenter retrospective analysis. Presented at: 62nd American Society of Hematology (ASH) Annual Meeting and Exposition; virtual; December 5-9, 2020. Abstract 41.

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HSCT Found Potentially Curative for Some T-Cell Lymphoma Patients - Cancer Therapy Advisor

Researchers Trace the Origin of Blood Cancer to Early Childhood, Decades before Diagnosis

PR Newswire

WASHINGTON, Dec. 8, 2020

Study in ASH Late-Breaking Abstracts session may pinpoint potential cancer origins and present opportunities for earlier detection efforts

WASHINGTON, Dec. 8, 2020 /PRNewswire/ -- Genetic mutations linked with cancer can occur during childhood or even before birth and proliferate in the body for many years before causing cancer symptoms, according to a new study. The study, which traced the genetic origins of a blood cancer in 10 individuals, suggests there may be untapped opportunities to detect cancer warning signs much earlier and potentially intervene to prevent or slow cancer development.

"Our preliminary findings show these cancer driver mutations were often acquired in childhood, many decades before the cancer diagnosis," said senior study author Jyoti Nangalia, MD, of the Wellcome Sanger Institute and University of Cambridge. "Our results finally answer the common question posed by patients, 'How long has this cancer been growing?' as we were able to study how these particular cancers developed over the entire lifetime of individual patients."

The researchers analyzed bone marrow and blood samples from 10 people with Philadelphia-negative myeloproliferative neoplasms, a type of cancer that causes stem cells in the bone marrow to produce too many blood cells. In the majority of patients, this cancer is driven by a genetic mutation called JAK2V617F. By assessing JAK2V617F, other cancer-linked mutations and hundreds of thousands of other mutations that a person naturally acquires throughout life, the researchers were able to trace the ancestry of different blood cells and estimate the time at which each patient acquired JAK2V617F and other important mutations.

They determined that, in these 10 patients, the first cancer-linked mutations emerged as early as a few weeks after the start of life and up to the first decade of childhood despite clinical disease presenting many decades later in life.

Story continues

"We were not expecting this," said Dr. Nangalia. "In fact, in one patient, the JAK2 mutation was acquired more than 50 years before their diagnosis."

While it is often assumed that most cancers are diagnosed within a few years of their emergence, the findings point to a more gradual, lifelong process in which a single cell acquires a cancer-linked mutation early in life and then slowly grows over decades, ultimately leading to cancer.

"Some of these cancer-linked mutations are found in healthy individuals as we get older, suggesting that aging causes them," said Dr. Nangalia. "However, aging per se doesn't drive such growth it simply takes a long time for the clones to grow." Sometimes, the growing clones pick up additional cancer-linked mutations along the way, accelerating their growth, researchers found.

"For these patients, we calculated how many of these cancer clones would have been present in the past, and our results suggest that these clones may have been detectable up to 10 to 40 years before diagnosis," said Dr. Nangalia. "In addition to detecting the mutations, the rate at which the mutated clones grew was also very important in determining whether, and when, cancer develops." The findings suggest that genetic testing could help identify people at risk for cancer much earlier than current methods allow, according to researchers.

The next steps would be to understand the factors that influence the different rates of cancer growth and determine whether there could be ways to intervene and slow the growth of cells with cancer-linked mutations. The researchers say their method for pinpointing the origin of this blood cancer could also be applied to other mutations and other blood cancers. "Understanding the timelines of development of different cancers is critical for efforts aimed at early cancer detection and prevention," said Dr. Nangalia.

Jyoti Nangalia, MBBChir, Wellcome Sanger Institute and University of Cambridge, will present this study during the Late-Breaking Abstracts session on Tuesday, December 8 at 7:00 a.m. Pacific time on the ASH annual meeting virtual platform.

For the complete annual meeting program and abstracts, visit http://www.hematology.org/annual-meeting. Follow ASH and #ASH20 on Twitter, Instagram, LinkedIn, and Facebook for the most up-to-date information about the 2020 ASH Annual Meeting.

The American Society of Hematology (ASH) (www.hematology.org) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open-access journal.

View original content to download multimedia:http://www.prnewswire.com/news-releases/researchers-trace-the-origin-of-blood-cancer-to-early-childhood-decades-before-diagnosis-301188621.html

SOURCE American Society of Hematology

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Researchers Trace the Origin of Blood Cancer to Early Childhood, Decades before Diagnosis - Yahoo Finance

Treatment with an off-the-shelf CAR T-cell therapy that targets B-cell maturation antigen (BCMA), ALLO-715, elicited responses in heavily pretreated patients with relapsed/refractory multiple myeloma in early findings from a first-in-human study presented at the 2020 ASH Meeting.1

The therapy generated responses in 6 of 10 patients (60%), including a very good partial-plus response (VGPR+) in 4 patients (40%), who were treated with ALLO-715 at a dose of 320 x 106 CAR cells plus a lymphodepleting regimen that included ALLO-647, an anti-CD52 monoclonal antibody, during the ongoing phase 1 UNIVERSAL study (NCT04093596).1

The findings mark the first results for an allogeneic CAR therapy directed at BCMA, said lead study author Sham Mailankody, MBBS, a medical oncologist and investigator in the Cellular Therapeutics Center at Memorial Sloan Kettering Cancer Center in New York, New York. BCMA, which is highly expressed on plasma and multiple myeloma cells, has sparked intensive research interest.2

These results demonstrate the feasibility and safety of an off-the-shelf CAR T cell therapy for multiple myeloma. In this first report of an allogeneic BCMA CAR T-cell therapy, we show that nearly 90% of patients were treated within 5 days of enrollment and without needing any bridging therapy, Mailankody said.

Allogeneic CAR therapy offers the potential for scalable manufacturing for on-demand treatment with shorter waiting times, which would overcome some of the logistical challenges posed by autologous CAR therapy, Mailankody said. The T cells needed for ALLO-715 are harvested from healthy donors and genetically engineered to express CARs aimed at specific cancer targets, according to Allogene Therapeutics, the company developing the therapy.3

ALLO-715 includes a human-derived single-chain variable fragment anti-BCMA cell with a 4-1BB costimulatory domain. Mailankody said the 2 key attributes of the construct are a knockout of CD52, which allows for selective lymphodepletion with ALLO-647 to prevent graft rejection without affecting the CAR T cells, and a knockout of the TRAC gene, which also minimizes the risk of graft-versus-host disease (GVHD).1

The UNIVERSAL study, which is being conducted at 11 cancer centers in the United States, is recruiting patients with multiple myeloma who have received 3 or more prior therapies, including an immunomodulatory drug, a proteasome inhibitor, and an anti-CD38 agent, and are refractory to their last treatment. Participants must have an ECOG performance status score of 0 or 1.

The dose escalation portion of the study is testing ALLO-715 as a single infusion across 4 doses: 40, 160, 320, or 480 x 106 CARs. Lymphodepletion regimens consist of fludarabine (F; 30 mg/m2/day) plus cyclophosphamide (C; 300 mg/m2/day) given on 3 days with ALLO-647 (A; 13-30 mg x 3 days; FCA) or cyclophosphamide plus ALLO-647 (CA).

Among the 35 patients enrolled at the time of the presentation, 4 became ineligible because of organ failure due to rapidly progressing disease. Of 31 patients in the safety population, the median age was 65 years (range, 46-76). Nearly half of the patients (48%) have high-risk cytogenetics and 23% had extramedullary disease. The efficacy population at data cutoff on October 30, 2020, comprised 26 patients across the 4 dosing levels, with a median follow-up of 3.2 months.1

The overall response rate (ORR) varied across dosing cohorts and lymphodepleting regimens. No responses were observed among 3 patients each who received CARs at 40 x 106 with FCA or 160 x 106 with CA, both with low-dose ALLO-647. The ORRs were 50% in 4 patients who received CARs at 160 x 106 with lowALLO-647 FCA; 33% in 3 at 480 x 106 with lowALLO-647 FCA; and 67% in 3 at 320 x 106 with lowALLO-647 CA.

The most robust responses were seen among those who received ALLO-647 at 320 x106. For this cohort, the ORR was 60% among 10 patients, including 3 of 6 who received CARs with lowALLO-647 FCA and 3 of 4 who had the therapy with highALLO-647 FCA. Overall, 6 patients had a VGPR+, defined as stringent complete response, complete response, or VGPR. These included 1 at 160, 4 at 320, and 1 at 480 10 x 106 CARs. Of the VGPR+ patients, 5 were negative for measurable residual disease. Additionally, 6 of 9 patients treated at the 320 or 480 x 106 dose levels remain in response.

Mailankody highlighted the experience of 1 participant, a 71-year-old man whose myeloma had progressed after undergoing 9 prior lines of therapy including autologous stem cell transplant and an experimental BCMA-targeted therapy. The patient received a conditioning regimen of FCA with low-dose ALLO-647 and ALLO-715 at 320 x 106. He reached a VGPR on day 14 that deepened to a stringent complete response by day 28 that remains in effect at 6 months, while experiencing grade 1 cytokine release syndrome (CRS).

The patient is clinically doing very well and is back at work, Mailankody said.

Among 31 patients in the safety population, most adverse effects were of grade 1 or 2 severity. These included CRS in 14 patients (45%) and infusion-related reactions to ALLO-647 in 7 patients (23%). The use of drugs to manage CRS also was low, at 19% for tocilizumab and 10% for steroids.

All-grade infections were reported in 13 patients (42%), including grade 3 events in 4 (13%). One patient (3%) died from a presumed fungal pneumonia related to progressive disease and the CA conditioning regimen but unrelated to ALLO-715. There were no instances of neurotoxicity or GVHD.

Notably, the fact that we did not see any GVHD is encouraging for an off-the-shelf allogeneic product, Mailankody said.

In response to a question from a conference attendee, Mailankody said it is too soon to compare efficacy levels seen with this allogeneic CAR therapy with those observed with investigational autologous CARs, which have been under study for several years.

Moving forward, investigators are continuing to evaluate dosing levels for ALLO-715. UNIVERSAL is enrolling patients to the 480 x 106 cohort, Mailankody said, adding that the appropriate dose likely would land between 320 and 480 x 106.

References

1. Mailankody S, Matous JV, Liedtke M, et al. Universal: an allogeneic first-in-human study of the anti-Bcma ALLO-715 and the Anti-CD52 ALLO-647 in relapsed/refractory multiple myeloma. Presented at: 2020 American Society of Hematology Annual Meeting and Exposition. December 5-8, 2020; Virtual. Abstract 129. Accessed December 5, 2020. https://ash.confex.com/ash/2020/webprogram/Paper140641.html

2. Cho SF, Anderson KC, Tai YT. Targeting B cell maturation antigen (BCMA) in multiple myeloma: potential uses of BCMA-based immunotherapy. Front Immunol. 2019;9:1821. doi:10.3389/fimmu.2018.01821

3. AlloCAR T Therapy. Allogene Therapeutics. Accessed December 5, 2020. https://www.allogene.com/allocar-t-therapy

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ALLO-715, Off-the-Shelf CAR T-Cell Therapy, Produces Early Promise in Multiple Myeloma - Cancer Network

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the China National Medical Products Administration (NMPA) has approved BLINCYTO (blinatumomab) for injection for the treatment of adult patients with relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukemia (ALL). The biologics license application (BLA) had been submitted by Amgen and received priority review by the Center for Drug Evaluation (CDE) of the NMPA. Developed by Amgen and licensed to BeiGene in China under a strategic collaboration commenced earlier this year, this is the first approval for BLINCYTO in China and BeiGenes first product licensed from Amgen to be newly approved. With this approval, BLINCYTO has become the first bispecific immunotherapy approved in China.

This approval of BLINCYTO provides us with an opportunity to offer adult patients in China with relapsed or refractory B-cell precursor ALL the first approved immunotherapy treatment for their disease. BLINCYTO is the first immunotherapy to demonstrate superior overall survival versus chemotherapy, more than doubling patients chances for survival, when used in first salvage R/R ALL in studies outside of China, commented Xiaobin Wu, Ph.D., General Manager of China and President of BeiGene. We are working to ensure BLINCYTO is available to patients in China as soon as possible. Our commercial organization of more than 1,500 people in China is excited to add BLINCYTO to our product portfolio, which now includes six approved cancer treatments.

The approval of BLINCYTO was based on results from the Phase 3 trial (NCT03476239) in China evaluating the efficacy and safety of BLINCYTO in adult patients with Philadelphia-negative R/R B-cell precursor ALL. Results of the interim analysis of 67 patients showed that the efficacy results in Chinese subjects were generally consistent with those in the global and Japan studies in subjects with Philadelphia-negative R/R ALL. The complete response/complete response with partial recovery of blood cells (CR/CRh) rate within two cycles of BLINCYTO treatment (the primary endpoint) was 47.8% (32 of 67 subjects; 95% CI: 35.4, 60.3). The median overall survival time was 9.6 months (95% CI: 6.4, not estimable). The safety profile observed for Chinese subjects in this study was consistent with that observed in the global studies evaluating BLINCYTO in R/R ALL. No new safety risks were identified based on these interim analyses of adverse events in Chinese subjects.

Our collaboration with BeiGene is advancing Amgens oncology pipeline for patients with significant unmet medical needs. We are confident the approval of BLINCYTO in China has the potential to make a meaningful difference to adult patients with R/R B-cell precursor acute lymphoblastic leukemia, said My Linh Kha, Vice President & General Manager, Amgen Japan Asia-Pacific (JAPAC). We are deeply committed to continuing to bring therapeutic options to treat debilitating cancers for patients in China, while also actively supporting the Governments focus on healthy aging through innovative products and initiatives designed to prevent chronic diseases, such as cardiovascular disease and fragility fracture.

About Acute Lymphoblastic Leukemia (ALL)

Acute lymphoblastic leukemia (ALL), also known as acute lymphocytic leukemia, is a rapidly progressing cancer of the blood and bone marrow that occurs in both adults and children1. ALL accounts for approximately 20% of all adult leukemia, and in China there were an estimated 82,607 new cases of leukemia in 20182,3. In children, the relapse rate of ALL is nearly 10%, while in adults the relapse rate is closer to 50%4.

About BLINCYTO (blinatumomab)

BLINCYTO is a bispecific CD19-directed CD3 T cell engager (BiTE) immuno-oncology molecule that binds specifically to CD19 expressed on the surface of cells of B-lineage origin and CD3 expressed on the surface of T cells.

BiTE molecules are a type of immuno-oncology therapy being investigated for fighting cancer by helping the body's immune system to detect and target malignant cells. The modified molecules are designed to engage two different targets simultaneously, thereby juxtaposing T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. BiTE immuno-oncology molecules help place the T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins and trigger the cancer cell to die (apoptosis). BiTE immuno-oncology therapies are currently being investigated for their potential to treat a wide variety of cancers.

BLINCYTO was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration and is approved in the U.S. for the treatment of:

In the European Union (EU), BLINCYTO is indicated as monotherapy for the treatment of:

In China, BLINCYTO is indicated for the treatment of adult patients with relapsed or refractory B-cell precursor ALL.

Important U.S. Safety Information

WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGICAL TOXICITIES

Contraindications

BLINCYTO is contraindicated in patients with a known hypersensitivity to blinatumomab or to any component of the product formulation.

Warnings and Precautions

Adverse Reactions

Dosage and Administration Guidelines

Please see full Prescribing Information and medication guide for BLINCYTO at http://www.BLINCYTO.com.

About BeiGene

BeiGene is a global, commercial-stage biotechnology company focused on discovering, developing, manufacturing, and commercializing innovative medicines to improve treatment outcomes and access for patients worldwide. Our 4,700+ employees in China, the United States, Australia, Europe, and elsewhere are committed to expediting the development of a diverse pipeline of novel therapeutics. We currently market two internally discovered oncology products: BTK inhibitor BRUKINSA (zanubrutinib) in the United States and China, and anti-PD-1 antibody tislelizumab in China. We also market or plan to market in China additional oncology products licensed from Amgen Inc., Celgene Logistics Srl, a Bristol Myers Squibb (BMS) company, and EUSA Pharma. To learn more about BeiGene, please visit http://www.beigene.com and follow us on Twitter at @BeiGeneUSA.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the commercialization and potential benefits of BLINCYTO; and BeiGenes plans and expectations for the commercialization of its and Amgens other oncology products and pipeline assets. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on the Companys clinical development, commercial and other operations, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

BLINCYTO and BiTE are registered trademarks of Amgen Inc.

1 Mayo Clinic. Acute lymphocytic leukemia. https://www.mayoclinic.org/diseases-conditions/acute-lymphocytic-leukemia/symptoms-causes/syc-20369077

2 Baljevic M, Jabbour E, O'Brien S, Kantarjian HM (2016). "Acute Lymphoblastic Leukemia".

3 Global Cancer Observatory. https://gco.iarc.fr/today/data/factsheets/populations/160-china-fact-sheets.pdf

4 Leukaemia Care. Relapse in Acute Lymphoblastic Leukaemia (ALL). https://media.leukaemiacare.org.uk/wp-content/uploads/Relapse-in-Acute-Lymphoblastic-Leukaemia-ALL-Web-Version.pdf

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BeiGene Announces the Approval in China of BLINCYTO (Blinatumomab) for Injection for Adult Patients with Relapsed or Refractory B-Cell Precursor Acute...

A FLINTSHIRE superhero is raising hundreds of pounds for a cause close to her heart in a bid to 'turn a negative into something positive'.

In 2019, Lauren Claire Pringle was diagnosed with Non-Hodgkins Lymphoma and began a course of gruelling treatment.

However, since finding out it has not gone completely, Lauren must have more chemotherapy and eventually a stem cell transplant.

Daughter Lily Pringle-Purcell decided to play her part and raise funds for a charity that has helped their family, and others going through similar.

The seven-year-old set out a target to raise 1,000 for Lymphoma Action and after shaving her mother's hair off and completing a five-mile walk, she has since raised over 800 of her target.

But the fundraising will not stop there.

Mum Lauren told the Leader: "She's done really well, I'm so proud of her.

"She's a fabulous little girl, really caring and always thinking about other people."

Anyone wishing to donate can do so online at https://www.justgiving.com/fundraising/laurenandlily.

The fundraising page reads: "My daughter has been raising money for Lymphoma Action. She has raised 720 so far on her Just Giving page. She shaved my head to raise money and also we did sponsored walk today of 5 miles to raise more money. She wanted to do this after my diagnosis last year of lymphoma and ongoing treatment.

"My name is Lily and I am 7 and in September 2019 my mum Lauren was diagnosed with Non Hodgkins Lymphoma. She had to have 6 cycles of chemotherapy and 12 sessions of radiotherapy. Recently we found out it hasnt all gone and she needs to have more chemotherapy and eventually a stem cell transplant. This has been really hard on us as a family and we want to try and turn a negative situation into something positive and raise as much money and awareness as we can to hopefully find better treatments and help other families going through the same thing.

"I want to do a sponsored walk and a sponsored bike ride and I am thinking of other things I can do to try and raise as much money as I can.

"If anyone has any ideas then please let me know! Thankyou all so much! Lots of Love Lily and Lauren."

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Flintshire youngster goes the extra mile to raise funds for Lymphoma Action | The Leader - LeaderLive

NESSZIONA,Israel, Dec. 8, 2020 /PRNewswire/ -- Meat-Tech 3D Ltd. (TASE: MEAT), today announced that it has signed an agreement to acquire 100% of the share capital of Peace of Meat PV, a pioneering Belgian producer of cultured avian products, for EUR 15 million in a combination of cash and Meat-Tech ordinary shares. The Company believes that it will be able to leverage Peace of Meat's technologies, including through novel hybrid food products, to expedite market entry while Meat-Tech develops an industrial process for cultivating and producing real meat using 3D bioprinting technology, without harming animals. The acquisition is expected to close in the coming weeks, subject to customary closing conditions.

Peace of Meat has developed a proprietary, stem-cell-based bioreactor technology for cultivating animal fats from chicken and ducks, without harming animals. It has conducted a number of taste tests, demonstrating the potential that its cultured fat has to enhance the taste of plant-based protein products. The technology's first expected application is in hybrid food products, combining plant-based protein with cultured animal fat, designed to provide meat analogues with qualities of "meatiness" (taste and texture) closer to that of conventional meat products. Meat-Tech estimates that the first hybrid products based on Peace of Meat technology could hit the market as early as 2022.

Pursuant to the acquisition agreement, Meat-Tech will pay half of the consideration immediately, with the payment of the balance subject to Peace of Meat complying with preset technological milestones over a period of two years, that were designed to scale up cultured fat production capabilities in preparation for market entry. To that end, it was agreed that Peace of Meat's management will continue in place to lead the development process.

This acquisition is consistent with Meat-Tech's growth strategy, aiming to streamline development processes and expand the Company's product range to penetrate cultured meat technology markets as quickly as possible. Meat-Tech is working to create synergy and added value for food manufacturers in the advanced production of cultured meat, while sustaining animal welfare and meeting the growing global demand for meat.

Sharon Fima, Meat-Tech's CEO: "Meat-Tech's novel technology for producing meat using 3D printing is gaining increasing international recognition. Boosted by our acquisition strategy, we believe we can turn Meat-Tech into a leading global center and home for innovative and groundbreaking cell-based food solutions that are both healthy and environmentally friendly. The combination of Peace of Meat's human capital and technology make this acquisition a significant step in that direction. I am pleased that both management teams share a common vision and strategy, and can join forces to advance the development of cultured food products with the potential to create real alternatives in the global meat market."

David Brandes and Dirk von Heinrichshorst, Co-Founders of Peace of Meat:"In an industry that is working towards a kinder, more sustainable planet, joining forces makes us stronger together. Peace of Meat has developed a powerful system for upscaled cultured biomass production and together with Meat-Tech we intend to accelerate product development toward commercialization.

"While Peace of Meat's core activity remains focused on the production of tasty, cultured fat as a B2B ingredient for meat alternatives, we see tremendous opportunity in jointly building a leading food-tech enterprise with Meat-Tech, based on a cellular platform.

"As entrepreneurs, we are excited about this acquisition as it poses a novel way of building and growing a company while significantly increasing the prospects of launching our product into the market."

About Peace of Meat:

Peace of Meat was established in Belgium in 2019 and is developing cultured chicken fat directly from animal cells without the need to grow or kill animals. The company believes that its innovative technology has the potential to support an industrial process for the production of cultured chicken fat. Peace Of Meat has entered into a number of scientific and commercial collaborations, in the process of positioning itself as a future B2B provider, with the potential to cover the entire value chain and to accelerate research and production processes in the industry, and has conducted taste tests for hybrid products it has developed.

About Meat-Tech:

Meat-Tech is developing a novel biological printing process designed to create living, edible meat tissue using cellular agriculture. Meat-Tech is developing technologies, processes and machines for cultivating, producing, and printing cultured meat. The company believes that it was the first in the world to use edible biological inks to 3D-print living tissue made up of various cells of bovine origin. The Company has the technology, knowledge and experience in applying tissue engineering practices for producing fat and muscle tissue for food consumption, as well as the ability to print, using a 3D bioprinter, a combination of live animal cells, growth factors and biological materials to produce living tissues that mimic the characteristics of natural tissue.

Forward-Looking Statements:

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, but not limited to, statements regarding the Company's development of the next generation of cultured meat food products by leveraging 3D digital printing technology, Peace of Meat's development of cultured fat products, the expected closing of the Company's acquisition of Peace of Meat and the expected post-closing synergies of the combined companies. These forward-looking statements include information about possible or assumed future results of the Company's business, financial condition, results of operations, liquidity, plans and objectives. In some cases, you can identify forward-looking statements by terminology such as "believe," "may," "estimate," "continue," "anticipate," "intend," "should," "plan," "expect," "predict," "potential," or the negative of these terms or other similar expressions. Forward-looking statements are based on information the Company has when those statements are made or management's current expectation and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. Actual results could differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements after the date of this press release to conform these statements.

COMPANY / INVESTOR CONTACT:Eran Gabay, Partner, Director of Strategy Gelbart-Kahana Investor Relations: [emailprotected]

SOURCE Meat-Tech 3D Ltd.

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Meat-Tech Agrees to Acquire Cultured Fat Pioneer 'Peace of Meat' - PRNewswire

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Stem Cell Manufacturing Market Size, Overview with Detailed Analysis, Competitive landscape, Forecast to 2027 - Cheshire Media

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today presents updated interim data from its Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs at the 62nd American Society of Hematology (ASH) Annual Meeting. The data are highlighted in two oral presentations.

We are highly pleased with the data presented at ASH demonstrating ongoing evidence of efficacy and durability using Process B in both FA and LAD-I as we move towards potential registration, said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. Follow-up data from the Phase 1 and 2 trials for FA continue to support RP-L102 as a potential hematologic treatment option in the absence of cytotoxic conditioning. In five of the seven patients treated as of October 2020, there was evidence of engraftment. In addition, stabilization of peripheral blood counts in two of the three patients with at least 12-month follow-up, which declined substantially in these patients prior to gene therapy, suggests a halt in bone marrow failure progression. We look forward to reporting longer-term follow-up on these patients in the first half of 2021.

Dr. Shah continued, Additionally, we continue to see encouraging evidence of efficacy for RP-L201 for the treatment of LAD-I. Patients have shown sustained CD18 expression of 23% to 40%, far exceeding the 4-10% threshold associated with survival into adulthood. These data, on top of our exciting results from our lentiviral program for PKD, show our steady progress across three of our five gene therapy programs. We are proud of this progress and are committed to advancing our investigational gene therapies through development for patients and families facing these devastating disorders.

Key findings and details for each presentation are highlighted below. To access the presentations at the conclusion of the oral presentation, please visit: https://www.rocketpharma.com/ash-presentations/

Gene Therapy for Fanconi Anemia, Complementation Group A: Updated Results from Ongoing Global Clinical Studies of RP-L102The data presented in the oral presentation are from seven of the nine patients treated as of the cutoff date of October 2020 in both the U.S. Phase 1 and global Phase 2 studies of RP-L102 for FA. Seven patients had follow-up data of at least 2-months, and three of the seven patients had been followed for 12-months or longer. Key highlights from the presentation include:

Presentation Details:Title: Gene Therapy for Fanconi Anemia, Complementation Group A: Updated Results from Ongoing Global Clinical Studies of RP-L102Session Title: Gene Editing, Therapy and Transfer IPresenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineSession Date: Monday, December 7, 2020Session Time: 11:30 a.m. - 1:00 p.m. (Pacific Time)Presentation Time: 12:15 p.m. (Pacific Time)

Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1The data presented in the oral presentation are from three pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. The patients were treated with RP-L201, Rockets ex-vivo lentiviral gene therapy candidate. Patient L201-003-1001 was 9-years of age at enrollment and had been followed for 12-months as of a cutoff date of November 2020. Patient L201-003-1004 was 3-years of age at enrollment and had been followed for over 6-months. Patient L201-003-2006 was 7-months of age at enrollment and was recently treated with RP-L201. Key highlights from the presentation include:

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

Presentation Details:Title: Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1Session Title: Gene Editing, Therapy and Transfer IPresenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date: Monday, December 7, 2020Session Time: 11:30 a.m. - 1:00 p.m. (Pacific Time)Presentation Time: 12:30 p.m. (Pacific Time)

Conference Call DetailsRocket management will host a conference call and webcast today December 7, at 6:00 p.m. EST. To access the call and webcast, please click here. The webcast replay will be available on the Rocket website following the completion of the call.

Investors may listen to the call by dialing (866) 866-1333 from locations in the United States or +1 (404) 260-1421 from outside the United States. Please refer to conference ID number 50038102

About Fanconi AnemiaFanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-ISevere Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, filed November 6, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Presents Positive Clinical Data from its Fanconi Anemia and Leukocyte Adhesion Deficiency-I Programs at the 62nd American...