StemCell Therapy

When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that theyd get office space as soon as her living room table was no longer big enough to hold company meetings.

We lasted about a year before my living room couldnt take it anymore, she said.

With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.

Gene therapy has generated so much enthusiasm for patients and families with these devastating disorders, but theres still a lot of science and innovation left on the table, McMinn said.

The CEO said Neurogene will split the Series B funds into four buckets, the first of which is advancing multiple gene therapy programs into the clinic. She anticipates filing the first IND in 2021 for CLN5, a rapidly progressive subtype of Batten disease caused by a variant in the CLN5 gene.

The second so-called bucket for the Series B funds will be expanding the companys portfolio, followed by another bucket for augmenting our resources for our novel technology platform, the CEO said. Then comes manufacturing.

Weve got the ability to make virus in-house, and the money from the financing will allow us to take that vector to the next stage and make GMP quality vector for use in dosing and clinical trials, McMinn said.

Because Neurogene manufactures products in-house, the biotech has gotten around the massive gene therapy manufacturing bottleneck, which has Big Pharma and big biotech spending billions on retrofitted plants and gene therapy factories.

The concept of gene therapy is simple: A viral particle is used to deliver a healthy copy of a gene to a patient with a dysfunctional gene. In the case of Neurogenes CLN5 candidate, viral vectors shuttle a payload into the body designed to make the CLN5 gene.

Over the next year, key milestones will be filing our first IND, completing the refurbishment of our GMP manufacturing facility, (and) advancing our programs towards the clinic, McMinn said. After CLN5, the goal is to file one to two INDs a year, she added.

The CEO previously served as an analyst at Piper Jaffray, Cowen and Bank of America Merrill Lynch, and as chief business and strategy officer at Intercept. During her time as an analyst, McMinn said most people would stay away from investing in neurology companies because drugs inevitably fail.

Theres really been nothing, very little innovation in devastating neurological disorders, for quite a long time, she said, adding that she was inspired to jump into R&D by an older brother who is neurologically impaired.

Neurogene attracted a slate of new and old investors, including EcoR1 Capital which led the round, and Redmile Group, Samsara BioCapital, Cormorant Asset Management, BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, Alexandria Venture Investments, and an undisclosed leading healthcare investment fund.

For me, I really want to make a difference, McMinn said, adding later, Im personally driven by developing something that is life-altering for people that really have no other option.

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After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News

Taysha Gene Therapies went public with a bang earlier this year with a $157 million IPO just five months after its Series A financing. Now, with the help of the city of Durham, North Carolina, Taysha is plotting an expansive development and manufacturing facility to ramp up supplies for the clinic and potentially the commercial market.

The new 187,000-square-foot cGMP plant will create roughly 200 jobs for Taysha Gene Therapies, which formed in late 2019 with the sole focus of developing therapeutics for monogenic diseases using an adeno-associate virus methodology. The announcement continues a rapid ascent for Taysha, which launched Series A financing just in April, and a mere five months later had its own Nasdaq ticker, offering a $157 million IPO and pricing shares at $20 apiece.

The facility is the result of a public-private partnership between Taysha, the city of Durham and the state of North Carolina. Taysha will invest $75 million into the facility, with additional state and local incentives totaling another $9.4 million. All told, the 200 jobs will come to fruition over a two-and-a-half-year period across all functions, including gene therapy development, analytics, manufacturing and quality control testing.

Tayshas facility is expected to be fully operational by 2023 and will add 2,000 liters of capacity supporting all aspects of scalable gene therapy manufacturing. The company said in a news release it will now be able to meet the clinical and commercial demands within the field when combined with its existing collaborations with the University of Texas Southwestern Medical Center and CDMO Catalent.

With our outstanding team of experts leading the charge, we expect this facility will serve as a center of excellence for gene therapy development, from preclinical studies through commercialization, and will further our leadership position in gene therapy as well as support our next phase of growth, RA Session II, Tayshas president, founder and CEO, said in a press release.

The new facility will serve as an integral part of rapid expansion for the Dallas-based biotech, which said it expects to have four open Investigational New Drug applications in 2021. Tayshas most advanced of those is a drug being developed for the treatment of GM2 gangliosidosis, a family of neurodegenerative disorders that includes Tay-Sachs disease and Sandhoff disease.

The other three such programs are looking to treat Rett Syndrome, epilepsy and SURF1 deficiencies.

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Taysha Gene Therapies looks to build on rapid IPO with $85M dev-manufacturing plant in North Carolina - Endpoints News

DUBLIN, Dec. 17, 2020 /PRNewswire/ -- The "Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global gene therapy market market.

Major players in the gene therapy market are Novartis AG, Bluebird Bio, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, Voyager Therapeutics, Applied Genetic Technologies Corporation, UniQure N.V., Celgene Corporation, Cellectis S.A. and Sangamo Therapeutics.

The global gene therapy market is expected to decline from $3.22 billion in 2019 to $3.18 billion in 2020 at a compound annual growth rate (CAGR) of -1.30%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The market is then expected to recover and reach $6.84 billion in 2023 at a CAGR of 29.09%.

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

North America was the largest region in the gene therapy market in 2019.

The gene therapy market covered in this report is segmented by gene type into antigen; cytokine; suicide gene; others. It is also segmented by vector into viral vector; non-viral vector; others, by application into oncological disorders; rare diseases; cardiovascular diseases; neurological disorders; infectious diseases; others, and by end users into hospitals; homecare; specialty clinics; others.

In December 2019, Roche, a Switzerland-based company, completed its acquisition of Spark Therapeutics for $4.3 billion. With this deal, Roche is expected to strengthen its presence in the gene therapy segment, support transformational therapies and increase its product portfolio. Spark Therapeutics is a US-based company involved in gene therapy.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. For instance, since January 2020, GlaxoSmithKline, a pharmaceutical company, has been investing in AI to optimize gene therapy and develop off-the-shelf solutions for patients. It is also expected to reduce turnaround time and also the cost of gene therapies.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Size And Growth 3.1. Global Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Gene Therapy Market Segmentation 4.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Gene Therapy Market Regional And Country Analysis 5.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/fltbmv

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager [emailprotected]

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Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,...

NEW YORK and CLEVELAND, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that abstracts detailing new interim results from its ABO-102 Phase 1/2 Transpher A study for MPS IIIA and ABO-101 Phase 1/2 Transpher B study for MPS IIIB have been accepted for platform oral presentations during the late-breaking abstract session at the 17th Annual WORLDSymposium being held February 8-12, 2021.

Children born with MPS IIIA and MPS IIIB experience progressive neurodevelopmental decline and loss of motor function that is life-threatening, said Michael Amoroso, Chief Operating Officer of Abeona. We are excited to share new analyses from the Transpher A study that will add to the understanding of the potential for ABO-102 to help preserve neurocognitive development in patients with MPS IIIA when they are treated at a young age, and new results from the Transpher B study that will provide insights into ABO-101s biologic effect in patients with MPS IIIB.

Presentation Details

Title: Updated Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)Abstract Number: 390Presenter: Kevin Flanigan, M.D., Center for Gene Therapy at Nationwide Childrens HospitalDate/Time: Friday, February 12, 2021, time to be determined

Title: Updated Results of Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB)Abstract Number: 407Presenter: Maria Jose de Castro, M.D., Hospital Clnico Universitario Santiago de CompostelaDate/Time: Friday, February 12, 2021, time to be determined

About the Annual WORLDSymposium The WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 17th Annual WORLDSymposium, please visit https://worldsymposia.org/.

About the Transpher A Study The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About the Transpher B Study The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-101 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102 ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About ABO-101 ABO-101 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

About Sanfilippo Syndrome Type A (MPS IIIA) Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Sanfilippo syndrome type B (MPS IIIB) Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeonas novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Companys most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

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Abeona Therapeutics Announces Acceptance of Late-Breaker Abstracts Highlighting New Clinical Data for Novel AAV-based Gene Therapies in MPS IIIA and...

First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

For more information:

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

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Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire

Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defectiveSMN1gene1.It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein1.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking.Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa."The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy.The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mre Enfant Soleil, Universit Laval in Qubec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3. Without a functionalSMN1gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90%of cases4.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene1.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular AtrophySMA is the leading cause of genetic infant death2. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities5.This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression6.Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly5. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta7,8.

About Novartis in Gene Therapy and Rare DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for thein vivogene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon forin vivodelivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

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Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire

I have heard about people using genes to treat diseases nowadays, but I am not sure what this gene therapy means.

Gene therapy involves trying to alter or modify the genes inside your bodys cells in order to treat or stop a disease.

Since 2017, the US Food and Drug Administration (FDA) has approved three different types of gene therapy.

Maybe we can start at the beginning: what are genes?

Genes are the basic physical and functional unit of heredity.

Our genes are made out of DNA (deoxyribonucleic acid).

Each person has two copies of each gene one inherited from your mother and the other inherited from your father.

Each human being has around 20,000 to 25,000 genes.

These genes code for the way your body and mind are structured.

Some genes act as instructions (a blueprint) for your body to make various proteins, which in turn form your cells and organs, and the enzymes and hormones that regulate your body.

Other genes do not code for proteins.

Most genes are the same for all human beings, which is why we all look like human beings (and not a kangaroo, fish, bird or an alien)!

However, just under 1% of our genes vary slightly between each person.

That is why we have different races, heights, propensity for different diseases, curly or straight hair, etc.

These small differences also contribute to why we all look different from one another.

Genes that dont work as they should also cause diseases in the human body.

What types of diseases are caused by faulty genes?

These are what we call genetic disorders.

A genetic disease is any type of disease caused by an abnormality in our genetic blueprint.

This abnormality can range from very minor to significantly major.

What we consider minor is, for example, a small mutation in the DNA of a single gene resulting in the change of a single base protein.

What we consider major is a gross chromosomal abnormality, such as the addition of a whole chromosome or the subtraction of one.

Some genetic disorders are inherited from our parents.

Others are caused by mutations due to our environment.

Examples of single gene disorders, which are caused by the alteration of just one gene in our bodies, are:

Examples of multifactorial inheritance, which are caused by a combination of environmental factors and mutations in many of our genes, are:

If we inherited these genes from our parents, then how can we possibly modify or alter them? This sounds terribly like science fiction.

We are rapidly approaching that era where what used to be science fiction could become part of our everyday life.

In gene therapy, scientists can:

How do they do this? Do they have to harvest my cells? Im scared just thinking about it!

Many of the vectors are viruses, especially adenoviruses (not coronaviruses!).

Viruses have a natural ability to deliver genetic material into our cells.

After all, their main purpose is to attach themselves to cells and reproduce themselves.

Sometimes, the vector or virus is injected straight into our bodies, where they will deliver the gene that will modify our cells.

They are injected straight into the part of our body that has those defective cells.

Other times, we have to harvest healthy tissue from our body that needs to be modified.

These are usually tissues containing immune cells or stem cells, e.g. blood or bone marrow.

These tissue samples are then taken to the lab and specific cells are separated out.

The viral vector containing the corrective gene is then introduced to the harvested cells in the lab.

The modified cells are left to multiply, and then injected back into us.

Once inside our bodies, they will continue to multiply and eventually treat the disease or correct the defect within us.

Learn more about gene therapy in the next Tell Me About column on Dec 31 (2020).

Dr YLM graduated as a medical doctor, and has been writing for many years on various subjects such as medicine, health, computers and entertainment. For further information, email starhealth@thestar.com.my. The information contained in this column is for general educational purposes only. Neither The Star nor the author gives any warranty on accuracy, completeness, functionality, usefulness or other assurances as to such information. The Star and the author disclaim all responsibility for any losses, damage to property or personal injury suffered directly or indirectly from reliance on such information.

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What is gene therapy? - The Star Online

University of Minnesota Twin Cities researchers in the Department of Chemistry have created a new polymer to deliver DNA and RNA-based therapies for diseases. For the first time in the industry, the researchers were able to see exactly how polymers interact with human cells when delivering medicines into the body. This discovery opens the door for more widespread use of polymers in applications like gene therapy and vaccine development.

The research is published in the Proceedings of the National Academy of Sciences (PNAS), a peer-reviewed multidisciplinary scientific journal.

Gene therapy involves altering the genes inside the bodys cells to treat or cure diseases. It requires a carrier that packages the DNA to deliver it into the celloftentimes, a virus is used as a carrier. Packaging of nucleic acids is also used in vaccines, such as the recently developed messenger RNA (mRNA) COVID-19 vaccine, which is enclosed in a lipid.

The research team is led by chemistry professor Theresa Reineke and associate professor Renee Frontiera. Reinekes lab synthesizes polymers, which are long-chain molecules that make up plastics, to use for packaging the nucleic acids instead.

Its kind of like ordering something from Amazon, and its shipped in a box, Reineke explained. Things get broken if theyre not delivered in a package. Thats basically what were doing here but on a nano-level. Were taking these really sensitive RNA and DNA cargo that are susceptible to enzymatic degradation, that wont get to their target unless you have something to protect them.

The researchers designed the copolymer using quinine, a naturally occurring substance used in tonic water, and 2-hydroxyethyl acrylate (HEA), which makes the material soluble and is used in a variety of personal care and medical materials. Because quinine is fluorescent, the research team was able to track the DNA package throughout the body and into the cells using Raman spectroscopy, a chemical imaging technique.

Weve discovered a new packaging tool with this natural product thats important for all of these high-flying, important fields like gene therapy and vaccines, said Reineke, who is also a Distinguished McKnight University Professor. And, it works in a variety of cell-types. On top of that, its got all of these cool featuresits fluorescent, we can track it, its Raman active, and that allowed us to understand a lot of fundamentals about these packaging systems that were impossible to probe before we incorporated this natural product.

Polymer-based drug delivery is significantly cheaper than using viruses, especially for gene therapy, which can cost up to $2 million for a single injection. However, the main barrier preventing widespread polymer use was that scientists didnt know a lot about how the polymer package actually interacts with cells in the body.

This research helps clear up that uncertainty. Frontieras lab specializes in chemical imaging. Using Raman spectroscopy, they discovered that a cells own proteins play a key role in unpacking the nucleic acid cargo once the polymer carrier enters the cell.

Its very satisfying to know how this is actually happening, what the process of delivery is, and to actually see that in real-time, Frontiera said. A key point is that these polymers also work very well. For all the beneficial attributes, theyre also incredibly effective at getting the payload into cells, and we were able to tell why, which doesnt always happen in this field.

Reineke and Frontiera have been collaborating since 2013. Reinekes lab has patented the quinine polymers, and the researchers hope that a company might license this technology in the future. The College of Science and Engineering team also collaborated with University of Minnesota Medical School Distinguished McKnight University Professor Jakub Tolar to test the effectiveness of the polymer carriers in relevant cell types.

Other members of the research team include chemistry researchers Craig Van Bruggen (Ph.D. student) and David Punihaole (postdoctoral associate), chemical engineering and materials science student Andrew Schmitz, and genetics Ph.D. student Allison Keith. This research was funded by the National Science Foundation and the National Institutes of Health.

Read the full research paper entitled Quinine copolymer reporters promote efficient intracellular DNA delivery and illuminate a protein-induced unpackaging mechanism on the PNAS website.

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Researchers discover new way to deliver DNA-based therapies for diseases - UMN News

A Boutique Pilates Studio in Verona, NJ. We offer Private and Duet Pilates sessions at the studio as well as remote pilates classes online.

We know that many people feel intimidated walking into a gym or Pilates Studio.At Longevity Pilates of New Jersey, our goal is to offer you a warm, personalized, nurturing, non-threatening approach to fitness.We've been helping people attain their fitness goals for more than 15 years.

With more than 15 years of teaching experience our certified teachers are well equipped to work with people of all ages, sizes and fitness levels.Whether you are training for a marathon, a retired grandmother, looking to improve your golf swing, trying to lose weight or recovering from an injury, our certified Pilates teachers will put together a Private or Duet session specifically designed for your individual needs. Live Long. Live Strong.

COVID-19 Update: At Longevity Pilates, we are doing all we can to keep you safe. This includes masking, disinfecting, and observing social distancing. If you prefer to work from home, we also offer remote Pilates training and group mat Zoom class on Saturdays at 1pm.

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Longevity Pilates of New Jersey

Eating healthy doesnt have to be complicated. Theres not a long list of rules you need to follow, or a complicated diet plan to stick to. Just ask one of the many centenarians living in the Blue Zones, who will tell you a big part of living a long and happy life is enjoying a wholesome, plant-forward diet. And your refrigerator is a good place to start stocking foods for longevity.

Through his research, Dan Buettner, longevity expert and author ofThe Blue Zones Kitchen, has found its mostly about making healthy choices every day that, together, add up to a pretty amazing life. None of the Blue Zones centenarians Ive ever met tried to live to 100. No one said at age 50, You know what, Im going to get on that longevity diet and live another 50 years! They dont count calories, take vitamins, weigh protein grams, or even read labels. They dont restrict their food intakein fact, they allcelebrate with food, says Buettner. As we have applied the wisdom of the worlds Blue Zones diet to transform cities in the United States, Ive begun to believe that we can create the same sort of culture here.

Turning your home into a Blue Zone can be as simple as keeping your refrigerator, freezer, and pantry stocked with healthy staples to promote longevity.

According to Buettner, nut-eaters outlive those who dont eat nuts. Because of that, hes a big proponent of eating a handfulaka two ouncesof mixed nuts a day. Nuts come in a variety of flavors, and theyre full of nutrients and healthy fat that satiate your appetite, says Buettner. Small quantities are best, since the oils in nuts degrade (oxidize). Larger quantities can be stored in the refrigerator or freezer for a couple of months.

Buettner says 95 percent of the food you eat should be plant-based.In the Blue Zones, people eat an impressive variety of garden vegetables when they are in season, and then they pickle or dry the surplus to enjoy during the off-season, he says. The best of the best longevity foods in the Blue Zones diet are leafy greens such as spinach, kale, beet and turnip tops, chard, and collards. The Blue Zones also recommends broccoli, Brussels sprouts, cabbage, cauliflower, and peppersreally, anything you like.

Fresh fruit is another Blue Zones refrigerator staple. Think apples, melons, grapefruit, oranges, and clementines. Fruit that lasts a long time, he says. You can also keep bananas in your fridge. He says doing so when theyre almost ripe will make them last longer. Aside from filling your fridge with fruit, placement is also key. Buettner recommends keeping your fruit (as well as your veggies) on the top shelf of your refrigerator, as doing so puts them at eye level and encourages you to eat more of them.

Animal protein is limited when youre eating like you live in the Blue Zones. Designate two days a week when you eat meat or other animal-derived foodand enjoy it only on those days, Buettner says. Instead, opt for plant-based protein, like beans in your pantry and tofu in your fridge. Find plant-based substitutes for the meat Americans are used to having at the center of a meal, he says. Try lightly sauted tofu, drizzled with olive oil, or tempeh. There are so many different ways you can use tofu, too, all of which are anything but boring.

While meat is limited, Buettner says you can eat up to three ounces of fish daily, as those who ate a plant-based diet that included small portions of fish lived the longest. Favor mid-chain fish like trout, snapper, grouper, sardines, and anchovies, he says. And steer clear of farmed fish, as they are typically raised in overcrowded pens that make it necessary to use antibiotics, pesticides, and coloring.

Dairy should be reduced when youre eating a Blue Zones diet. While Americans have relied on milk for calcium and protein for decades, in the Blue Zones diet people get these nutrients from plant-based sources, Buettner writes. One cup of cooked kale or two-thirds of a cup of tofu, for instance, provides just as much bioavailable calcium as a cup of milk. He also recommends using alt-milks, including unsweetened soy, coconut, or almond milk: Most have as much protein as regular milk and often taste as good or better.

Blue Zones guidelines stipulate that eggs arent necessary for living a long life. Keep your egg consumption to no more than three eggs per week, says Buettner. If you choose to eat eggs, select a variety from chickens that range freely, eat a wide variety of natural foods, and dont receive hormones or antibiotics, Buettner says.

Tracy Lockwood Beckerman, RD, says eggs are part of a healthy diet: Eggs are cheap, satiating, and easy to find, and theyre an excellent source of high quality protein, she says. Your body is able to fully absorb all the protein from the eggs to help lower blood pressure. If you choose to eat eggs regularly, just make sure theyre high-quality to keep them Blue Zones-approved.

For more healthy recipes and cooking ideas from our community, join Well+Goods Cook With Us Facebook group.

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6 Foods You Should Always Have in Your Refrigerator, According to a Longevity Expert - Well+Good

NEW YORK, Dec. 18, 2020 /PRNewswire/ --Capt. Franz Almeida has a practiced eagle's eye, honed over thousands of flight hours as a pilot. Junaid Mian, RPh, understands the challenges inherent to maintaining human health through the lens of work as a pharmacist. Both followed unique paths, which led them to angel investing. The two met at the NY chapter of the Harvard Business School.

Together they identified a yawning gap between space technology and biotechparticularly longevityverticals. They recognized that the world's economy is in the throes of change so radical that future generations will see the 21st century as a clear delineation marking a decidedly Earthbound humanity split from humanity that can freely live in and explore space.

The space economy explosion is happening, and there is no better time to invest than as early on as possible during that explosion. The latest estimates of the global space economy are well over $400 billion, and if growth continues to accelerate, many analyses point to a $1 trillion space economy just around the corner. The human longevity market is nearly as massive: A Merrill Lynch analysis revealed that the sector generates over $110 billion annually now and is growing to over $600 billion by 2025.

Without massive and ongoing investment and progress across both space technology and human longevity verticals, we will never be able to truly unlock the potential that our Solar System, and our galaxy, holds.

"A big part of why progress against aging used to be so slow was that so few experts on aging had an engineer's way of thinking. The convergence of those communities is making all the difference."

Aubrey de Grey, VP of New Technology Discovery at AgeX Therapeutics, Inc and Chief Science Officer of the SENS Research Foundation and Human Longevity advisor at SP8CEVC

And that's where Junaid Mian, RPh, and Capt. Franz Almeida comes in. The pair's varied perspectives have been a decisive match when it comes to examining the potential in merging investments across the space tech and human longevity verticals. Almeida and Mian are launching a new Rolling Fund in partnership with AngelList, with an exacting focus on solving the problem that is the future of humankind.

Under the umbrella of SP8CEVC, LPs will be able to gain exposure to deals to empower growing ventures in space technology and human longevity. Why these two rather specific categories? Simple: Moving to an economy based on the resources of our entire Solar System enables a much more substantial timeline for humanity's existence, and biotechnology work in longevity enables people to live and work in space.

The SP8CEVC partners who initially started with a traditional fund structure have chosen to use AngelList's rolling fund Reg 506(c) model to open up investment possibilities in these most critical verticals. As a series of quarterly pooled investment funds, SP8CEVC will give investors access to their deal flow quarterly on a subscription basis.

This new structure accelerates the pace of investing and, as a benefit, also helps promote innovation in those businesses.

The SP8CEVC team launched yesterday at TechCrunch Sessions: SPACE 2020. Investors and LPs interested in setting the pace for future advancement can have access to SP8CEVC's deal flow before anyone else here.

Media Contact:Franz Almeida917-287-5674 [emailprotected]

SOURCE SP8CEVC

https://www.sp8cevc.com/

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Introducing SP8CEVC: The World's First Space Technology and Human Longevity Focused Rolling Fund in Partnership with AngelList - PRNewswire

MUMBAI: From the start of daily soaps in 2000, Hindi GEC fiction haswitnessed multiple eras: From the now-notorious saas-bahu dramas in the first decade, to shows addressing societal evils and women empowerment,tofamily-based love stories that brought in the younger viewers. With each passing era, characters in the Hindi GEC category havebecome smarter, more fashionable and more progressive in their outlook. Whats interesting, however, is that characters who have managed to retain long-term popularity among the audience have one aspect common to their personalities: they are generally over-indexed on the personality dimension sincerity.

In her brand personality framework, psychologist Jennifer Aaker identifies five dimensions to a personality sincerity, excitement, competence, sophistication and ruggedness. Most brands will have some image associations with these dimensions, but the more enduring brands largely have one emphasised primary dimension, and optionally a secondary one.

While originally designed for brands, the framework works equally well for celebrities and characters. In the Hindi GEC category, the one emphasised dimensionacross most long-standing popular fiction characters is the sincerity dimension. Five of the seven popular Hindi GEC characters between 2016-19 (based on Ormax Characters India Loves)had sincerity as their strongest, or a strong number two, dimension. These five characters span a width of age, profession and content genres. Theres themiddle-aged shop keeper and familyman Jethalalin a sitcom; young dentistIshita engulfed in across-cultural family drama where she falls in love with a divorced father;the quintessential girl-to-bahu journey of Akshara; the young and quirky Pragya who marries a rock star accidentally; and the talentedvillage singing prodigy Kullfi,who moves to the city in search of her father.

The trend continues in 2020. The undisputed success story of this year has been Star PlusJuly launch Anupamaa. The show has achieved record ratingsin a challenging and disruptive Covid2019 period. The lead character of the showraced to number two on Ormax Characters India Loves within a month of the shows launch, a new record in character popularity tracking in the last decade.

Anupamaas rapid growth is a combination of various factors, such as a unique yet relevant concept (it addresses the anti-family topic of extra-marital relationship in a very family-palatable way), strongwriting and direction, and the performance by thelead actor herself. It is no surprise, however, that Anupamaas character too is heavily loaded on the sincerity dimension, even more than the five characters listed above.

So why do sincere characters strikesuch adeep chord with the audience?

Storytelling on Indian televisionis different from films and web-series, because in serials, audience look for ideas that are important to them. They connect with charactersthatare avatars of themselves. They feel their feeling, live their emotions. As a collectivistic society, most Indians value family cohesion and cooperation as a non-negotiable aspect of their identity. They take great pride, and seek comfort, in the strong emotional bonds they share with their family members. Watching stories driven by characters who reinforce family values give these shows a higher purposebeyond entertainment.

In this context, characters like Jethalal and Anupamaa are the quintessential Indianfamily man and Indian family woman respectively, who value their families and relationships above everything else. While the former is in a sitcom and the latter leads a familydrama, it is their fundamentallyearnest personalities thatendear them to the Indian value system. Coupled with good storytelling, suchcharacters build long-term equity, engaging the audience with their journeys that fundamentally revolve around their large families in a manner thats truly Indian at heart, i.e.,sincere!

Story and storytelling will continue to get more progressive with time. But the cherished place characters high on sincerity have in the hearts of Indian TV audience is unlikely toweaken anytime soon.

(The author is Ormax Media partner. Indiantelevision.com may not subscribe to their views.)

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Sincerely yours: Key to longevity in the Hindi GEC category - Indiantelevision.com

The iconic former goalkeeper, who worked alongside the Portuguese in Spain, feels the five-time Ballon dOr winner can continue for a very long time

Cristiano Ronaldo's small rivalry with Lionel Messi has made him a better player, claims Iker Casillas, with the Juventus superstar being backed to remain at the top for a very long time.

The Portugal international missed out on the latest FIFA Best prize to Bayern Munich striker Robert Lewandowski,but he has collected that award on two previous occasions and has five Ballons dOr to his name.

Only Barcelona icon Messi can claim to have bettered that return, with the mercurial Argentine another of those to have sent records tumbling over the course of a remarkable career.

Ronaldo has matched the South American stride for stride, re-writing the history books himself on a regular basis, with two all-time greats using the exploits of one another as added motivationwhen raising their own individual bars.

Real Madrid icon Casillas admits as much, with the World Cup winner telling The Nationalof his former Blancos team-mate Ronaldo and why the 35-year-old has starred for so long: Obviously hes a good professional, he looks after himself, eats well, gives the necessary importance to rest also.

So this is just a result of being what a footballer is. Other than that, hes also a great athlete. The reason is hes always hungry and always wanting to improve and better himself.

Perhaps also his small rivalry with [Lionel] Messi has helped him better himself. But its basically down to his professionalism and ambition; that has taken him to the top of the footballing world.

Ronaldo has been showing no sign of slowing down during his time in Italy, with 79 goals recorded through 101 appearances for Juve.

He has also reached 102 efforts for his country, as he looks to become international footballs all-time leading marksman,and appears to have plenty left in the tank.

Casillas cannot see the Portuguese hanging up his boots any time soon, adding on how much longer a modern-day phenomenon can go on for: I hope he can continue to play for a very long time. But, of course, football and its nature is what it is.

We all know that everything must come to an end and time does go by for everyone. Hopefully we will be able to still enjoy his football for a long time, but its difficult to say.

Ronaldo is tied to a contract in Turin through to the summer of 2022 and has stated in the past that he intends to play on beyond his 40th birthday.

Link:
Ronaldos rivalry with Messi has made him better Longevity of Juventus star doesnt surprise Real Madri... - Goal.com

Doctors report that singing may also help to reduce blood pressure.

A 76-year-old woman who had experienced severe preoperativehypertensionprior to totalknee replacement surgery for osteoarthritis (OA).

While the patient was unresponsive to aggressive pharmacologic interventions, the womans blood pressure dropped dramatically when she sang several religious songs.

This case-report appears in the April issue of Arthritis Care & Research, a journal published by Wiley-Blackwell on behalf of the American College of Rheumatology (ACR).

Several studies suggest that listening to music can be effective in reducing blood pressure by calming or diverting patients prior to surgery, which lessensstress and anxiety, explains lead author Nina Niu, a researcher from Harvard Medical School in Boston.

Our case study expands on medical evidence by showing that producing music or singing also has potential therapeutic effects in the preoperative setting.

Niu commented, Singing is simple, safe, and free. Patients should be encouraged to sing if they wish.

This single case study showed the positive effective of singing in reducing blood pressure and controlling pain.

To be formally considered as an alternative therapy for the OA patient population, larger studies are needed to explore the effects of singing on hypertension and chronic pain relief, said Niu.

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How to live longer: Singing could reduce your dementia risk and boost longevity - Express

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LAKE BUENA VISTA, FLORIDA AUGUST 06: (Photo by Kim Klement-Pool/Getty Images)

The end to the speculation of whether Giannis Antetokounmpo will or wont sign his supermax extension thankfully came to an end Tuesday afternoon.

The Milwaukee Bucks superstar wound up putting pen to paper and signed the richest contract in NBA history at five years and $228 million, which includes an opt-out clause in the summer of 2025. Still, the Bucks have the two-time MVP under contract for the next six seasons and all Bucks fans subsequently drew a sigh of relief around the city of Milwaukee, the state of Wisconsin and elsewhere.

Antetokounmpo signing his extensionis as much a victory for Bucks fans as it is for the 26-year-old and the organization itself, especially after so much anxiety and fear in the years leading up to his decision.

The constant threat and speculation of how a small market like the Bucks have historically struggled to keep past and present superstars loomed over Giannis decision. Stories of Kareem Abdul-Jabbar, LeBron James and Kevin Durant were constant reminders of stars leaving small markets for greener pastures.

For now, Antetokounmpos decision has seen those worries and skepticism scurry away from the discussion as Bucks fans jump for joy to see the Greek superstar commit the prime of his career to the Bucks.

As all Bucks fans continue to bask in a state of pure bliss,the doubts are finally over and we have prevailed. Now to quote King George from Hamilton after losing in the Revolutionary War, What comes next?

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Giannis Antetokounmpo: What his extension means for Bucks longevity - Behind the Buck Pass

Fortunately, heart disease is a preventable condition and diet is a modifiable risk factor.

Many dietary items can help to stave off the risk by stymying the mechanisms that contribute to heart disease.

Acai berries, a Brazilian superfruit native to the Amazon region, has been shown to improve LDL cholesterol levels.

LDL cholesterol is a harmful substance that clogs up inside of your artery walls, cutting off the blood supply to the heart - the primary cause of heart disease.

READ MORE:How to live longer: Sex may reduce heart disease risk - how often you should have it

Animal studies have also suggested that acai could help improve cholesterol levels by decreasing total and LDL cholesterol.

While more research is needed, researchers postulatethat anthocyanins in acai could be responsible for their positive impact on cholesterol levels, since studies have linked this plant compound to improvements in HDL and LDL cholesterol.

HDL cholesterol is branded the "good" cholesterol because it counters the harmful effects of LDL cholesterol.

What's more, acai contains plant sterols, which prevent cholesterol from being absorbed by your body, research shows.

While certain items are essential, the most important dietary approach is to focus on heart-healthy food groups.

According to the NHS, a low-fat, high-fibre diet is recommended, which should include plenty of fresh fruit and vegetables (five portions a day) and whole grains.

"You should limit the amount of salt you eat to no more than six grams (0.2oz) a day as too much salt will increase your blood pressure," warns the health body.

High blood pressure can raise your risk of heart disease by narrowing your blood vessels.

You should avoid food containing saturated fats, because these will increase the levels of bad cholesterol in your blood.

Foods high in saturated fat include:

Harvard Health explains: "Regular exercise also improves factors linked to cardiovascular health, resulting in lower blood pressure, healthier cholesterol levels, and better blood sugar regulation."

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How to live longer: Acai smoothies may reduce your risk of the global killer heart disease - Express

Photo Source: Nathan Arizona

After getting signed by an agent out of high school, Mare Winningham quickly carved a small-screen niche for herself with dozens of TV movies, ultimately earning two Emmys. Her career continued with films St. Elmos Fire and Georgia, the latter notching her Screen Actors Guild and Academy Award nominations. On hiatus from acclaimed Broadway musical Girl From the North Country, she can next be seen in the Tom Hanksled News of the World.

How did you land your first agent?I grew up in the San Fernando Valley and went to public schools. There was something in the water at that time in the 70s in the Valleywe were all very theater-obsessed kids. There were all these festivals and competitions we were involved in, in addition to our productions. I was very single-minded about theater; I did it in my free time and during school. We started getting noticed because of all these festival competitions around Los Angeles, and our school was always winning. I think somebody had a relative who worked at an agency, and they came out to see The Sound of Music, which we were doing when I was in 12th grade. I got invited to see this agent, Meyer Mishkin, who had a boutique agencyone of the few that was high-powered but was just a single-entity agency. He ran it himself, and it was called the Meyer Mishkin Agency. Lee Marvin was a client. I remember running into Richard Dreyfuss in the waiting room. He said that he was going to take me on as a client. This was at the very end of high school, just about when I was wondering, How do I parlay this love of theater into a career? From the beginning, he had this plan. He said, Youre not going to do commercials. Were going to do TV movies. At that stage, it was very divided: Movie actors didnt do television. I think he thought: Youre going to corner that market, kid. I started working right away, mostly small parts on Starsky & Hutch and Family, these TV series of the late 70s. Then I got my first TV movie and started working pretty much in television movies for many years.

What advice would you give your younger self? Open up, dont gossip, and dont listen to gossip. Film sets and rehearsal roomseveryone describes them as instant families. There are good families and there are bad families. I would tell myself to not be fraught and not worry so much and let stuff roll off a bit. And if you are a leader, lead with love.

READ: How Negativity Affects Career Longevity

How did you first get your SAG-AFTRA card? I remember being confused about this Catch-22 about how you cant get a SAG card without having a job, and you cant get a job without having a SAG card. I was grateful that there was a casting director casting this teen idol show that was very popular called James at 15, and in that episode, he was having a Miss 15 pageant. There were about six of us; each of us was a contestant in this Miss 15 pageant. They had backstage scenes where we were all discussing James. We all got our cards. In some ways, I dont know if Ive ever been happier.

Do you have an audition horror story you could share with us? There were so many. I had an interior casting director in me, and in my mind, I would see someone and go, Oh, theyre much better for it than I am, and I would defeatedly go in the room and carry that thought with me. One time, I saw the [character breakdown] sheet, and it actually said, Were looking for a Mare Winningham type, and I didnt get it! I think I was my own worst enemy a lot of times. I probably have a career because I didnt have to audition for those TV movies.

Whats the wildest thing youve ever done to get a role? The best job of my life is the one I currently hold. When Broadway reopens, I have this incredible project that started in London, this Conor McPhersonBob Dylan musical, Girl From the North Country. When I had to audition for it two years ago, I started picking on myself again, and my sweetheart said, No, Im not watching this. Take your dulcimer, go in there, [and] sing the songs. And I said, No, theyre going to have an accompanist there. And he said, Do what you want. Hes going to want to hear your best. This is how you want to sing it? Do it. I fought him and fought him and finally did it his way and got it.

Whats one performance every actor should see and why? When I was a little girl, they used to play The Wizard of Oz before the holidays. I was so frightened of Margaret HamiltonMiss Gulch, the Wicked Witch. She was the first scary person in my life, and I had to leave the room when she was on. Later, as I watched her as an actor, I loved that performance. I find Miss Gulch so awful, and I love the way she built that performance.

Joaquin Phoenix in The Master. He has a close-up in that movie that I couldnt get over. I dont even know if I want to watch it again because it affected me so much. Hes being scrutinized, and its excruciating watching him go through what hes going through. In a more humorous way, no less powerful, would be at the end of Sense and Sensibility, when Emma Thompsons character, its beginning to dawn on her, as shes listening to Hugh Grants character, that their love can be now. Thats a beautiful close-up. Tom Hanks at the end of Captain Phillips. How did he do that scene when hes being examined by the doctor after the ordeal on the ship? Thats how I like to watch acting now: in the quiet dark of a theater or at home. I love to see an actor blow my mind. It makes me want to jump up and shout. Almost everything I watch, I find something that makes me want to jump up.

This story originally appeared in the Dec. 17 issue of Backstage Magazine. Subscribe here.

Looking for remote work? Backstage has got you covered! Click here for auditions you can do from home!

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'News of the World' Interview With Mare Winningham | BACKSTAGE - Backstage

A huge fan of horror movies, Goldberg reveals that she has been lobbying to be involved in a Stephen King adaptation for years, even going back to the 1994 version of this same project.

Ive been looking for a horror movie to do my whole career. What I love more than almost anything is a good scare. Ive been trying to do this since the original miniseries came out, but I was unable to do it, Goldberg says. Then I found out that Ruby Dee was my age now when she did Mother Abigail. So its worked out great. If this had come any sooner, I would have probably just really messed it up.

At the time of the series premiere, the EGOT-winning Goldberg is 65 years old. Dee was 70 years old when filming on the 1994 miniseries began. Both actresses, however, are considerably younger than the wizened Abigail Freemantle of Kings book. This isnt a case of Aunt May getting progressively younger across every Spider-Man franchise but rather an acknowledgement from both miniseries that Abigail has a powerful aura about her that belies her advanced years.

Listen, its all in the lineage, I myself am 108. And my skin looks amazing, Goldberg jokes, before adding. You know, also, shes got a little God dust on her too. You cant look that good without a little God dust. The same goes for (Randall Flagg actor) Alexander (Skarsgrd). He looks great because hes got some devil dust on. Thats how Im looking at it, so it balances out.

In addition to Mother Abigails sprinkling of God dust, what Goldberg finds compelling about the character is her humanity as opposed to her otherworldliness. Given the dearth of Black characters in Kings earlier works, Abigail has sometimes been pointed to as an example of the Magical Negro stereotype, in which a mystical Black character comes to the aid of white characters. Its that representation of the character that Goldberg says she hopes to avoid.

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The Stand: Whoopi Goldberg Reveals the Secret to Mother Abigails Longevity (Its God Dust) - Den of Geek

It is always best to maintain good oral care. You can even go as far as dreaming of having brilliant teeth. After all, it is said to be our faces greatest asset. It is in our smiles that people can see our happiness, awkwardness, shyness, joy, and many other emotions. It is normal to value it as much as we value the rest of our body parts. These are the top dental procedures and teeth whitening that will ensure you have a beautiful smile. Longevity Live Partner Content

There are some things only the human teeth are characteristic of, such as:

So if they are a bit crooked, one too many, or terribly far apart from each other, it only goes to show that youre unique nothing to be ashamed of. Then again, there is nothing wrong with going the extra mile to feel more special. If you feel like they have the potential to be better, then you have all the right to choose whats best for them. Getting your teeth a brand new makeover to help you build confidence in yourself is never a bad thing. Its just one way of showing how much you treasure your own smile!

There are many ways to go about an oral makeover, but most of them require a visit to the local dentist. If youre not quite sure about what dental procedure you should have, we thought the following suggestions might help you out!

If you have nasty cavities plaguing your tooth, you might want to consider getting a dental filling procedure pretty soon. Time is of the essence; cavities wont wait until youve strengthened your resolve. Theyll keep eating away at your tooth until such time that there is nothing left of it. It may sound like nothing to be alarmed about. After all, youre just going to lose a tooth (Whats a single tooth when you have thirty-two or more of them, right?).

However, what you fail to see here is that with the degradation of your tooth comes excruciating, discomforting, and unsettling pain that seems to drain the life out of you. Dont let it get to that point.

Getting a dental filling procedure done should help you avoid having to go through this ordeal and help you save your tooth in the process. Its a simple, almost 100% painless dental procedure that involves cleaning the affected part of the tooth and filling it in with a substitute substance that will become the new surface of your tooth. It can be done in no more than an hour (for a single tooth, that is).

If you find yourself having cavity problems, cure it while early.

Are you tired of dentures? We will all come to a point in our lives when we lose our munching sets no matter how much we take care of them, no matter how careful we are of them. Its a reality we must all face. Other factors, such as aging and wearing down of teeth, may be attributed to this unpreventable teeth loss https://medlineplus.gov/toothdecay.html. However, that doesnt mean that we have to brave life toothless.

There are several substitutes we can use in place of genuine teeth, of course. One of them is having dentures made. These are artificial that can be retracted when the wearer needs or wants to. They fulfill whatever purpose teeth have and are more than capable of doing common tooth tasks such as biting, chewing, and grinding. However, dentures do come with disadvantages.

You have to remove them for cleaning from time to time, and you also risk having them fall off at the most significant times. You can be talking to the CEO of your company when it suddenly decides to jump right out of the basket. Yikes! Total mishap! If you fear having to experience such moments, then maybe dental implants are more suitable for you.

A dental implant is a substitute for real teeth that are permanently attached to the gums. Even though theyre artificial, you can pretty much do the same things as you would with your regular teeth, like brushing and flossing. It can really help you save time and avoid the hassle. The best part of having dental implants is that you get to enjoy all the perks of having healthy teeth, minus the risks of cavities. Make sure to get them checked by your family dentist every year or so, though!

Everyone dreams of having dazzling pearly whites. The kind of brilliance that shines as bright as its wearers smile. Sadly, not everyone is born naturally with it. All too often, one must work very hard to achieve it. Beautifying this part will most probably involve a whitening procedure.

For people that have been meaning to whiten their teeth, there are various options you can try out. Now available in the market are some of the best teeth whitening kits that come at a very affordable price, so you do not necessarily need to do a full bleach at the dentist. If you want something easily accessible, you can try using over-the-counter whitening strips. These are white elastic strips (looks like gauze tape, really) that you wrap around your teeth. Most of these strips have fluoride a chemical long been proved to whiten teeth effectively.

There are risks to using whitening strips, however. This includes blotching and uneven patches. If you find it really bothersome to use whitening strips, you can opt for a more natural method baking soda paste. Baking soda is a familiar name among moms because of its many practical uses around the home. One particular use is stain removal.

If it can remove stains from dirty laundry, wooden floors, and woolen carpets, then it can definitely whiten teeth as well. Just create a paste-like mixture using baking soda powder and water and apply it to the desired area. This is not an overnight remedy to remove yellowness, but it will certainly improve the color in the long run. Of course, if all these other ways dont work for you, having a professional look after your teeth is a terrific choice. Undergoing a whitening procedure will bring you the desired results in less time.

Why dont you ask your family dentist about it today?

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Dental Procedures and Teeth Whitening for A Beautiful Smile - Longevity LIVE

Californias COVID-19 outbreak continues to worsen, with a grim new statistic revealing the 53,000 new confirmed infections diagnosed on Tuesday.

In a press briefing, Los Angeles Countys Public Health Director Barbara Ferrer described Southern California and the Central Valley regions struggle to suppress the surge of COVID-19 hospitalizations, specifically adding that with the current death rate, two people are dying every hour in Los Angeles County.

"We're experiencing an explosive and very deadly surge," Ferrer said.

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The Golden State has been under siege as coronavirus transmission increases across the country during the winter season.

Earlier reports document the state government providing about 5,000 body bags to the embattled Southern California counties, along with 60 refrigerated trailers to serve as additional morgue space.

On the county level, 99.9 percent of all counties in California are experiencing what the state health department classifies as widespread risk levels, bringing the state total of confirmed infections to 1,723,362.

Some 379 new deaths have been recorded across the state as of Dec. 17.

In Los Angeles County specifically, the new case trajectory is increasing,with more than 21,000 new confirmed infections as of Dec. 16.

Theeffect of these statistics on hospitals is substantial, matching statewide trends of rising inpatient admissions and dwindling capacity.

"Hospitals are under siege and our models show no end in sight," Christina Ghaly, Los Angeles Countys health services director said.

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Originally posted here:
'Explosive surge' of COVID-19 in LA sees two people dying every hour | TheHill - The Hill