Stem Cell Clinic

Adocia Files Patent on a Hydrogel Scaffold for Cell Therapy in the Treatment of Type 1 Diabetes – Business Wire

By Stem Cell Treatment

LYON, France--(BUSINESS WIRE)--Regulatory News:

Adocia (Paris:ADOC) (Euronext Paris: FR0011184241 ADOC), a clinical stage biopharmaceutical company focused on diabetes treatment and other metabolic diseases with innovative formulations of proteins and peptides, announced today it is developing a hydrogel scaffold that hosts and protects pancreatic cells for replacement of the missing cells of people with type 1 diabetes.

Capitalizing on our expertise in regenerative medicine and diabetes, our multidisciplinary team has developed a scaffold for cells with the aim of being transformed into an organoid controlling glycemia. The collaboration with the team of experts of Professor Pattou has created great momentum and the first results obtained in small animals are really exciting, said Olivier Soula, Deputy CEO and Director of R&D.

Among the 25 million people with type 1 diabetes in the world, and despite intensive and sophisticated insulin treatments, some patients diabetes are uncontrolled and should require pancreatic cell therapy to survive.

Cell therapy consists of the administration of living cells to diabetic patients to restore glycemic control. Since the 1980s, it has been possible to transplant Langerhans islets taken from the pancreas of a deceased donor. However, despite health authorities approval, this technique is restricted to a very limited population due to remaining issues:

To solve these issues, Adocia has designed a new type of hydrogel scaffold able to host transplanted cells allowing them to release insulin while protecting them from immune reaction.

We are on our way to achieving a patient dream, that cell therapy would provide benefit to a large population without requiring immunosuppressant drugs, which is a major drawback today for its use, commented Gerard Soula, President & CEO of Adocia.

The program has been initiated with human Langerhans islet to achieve the proof of concept of a safe and effective implant, that is easily implantable. The next step is to extend the application to stem cells avoiding strong limitation of the limited number of donors.

Academic collaboration has been established with the research team of Professor Franois Pattou (DiabInnov, Inserm Lille, France), a worldwide expert in clinical and non-clinical Langerhans islet transplantation. Preliminary results obtained during this collaboration are promising. A first patent is pending.

We strongly believe that cell therapy has a great future for the treatment of Type 1 if the limitations of cadaveric islets source and the need of immunosuppressant drugs can be addressed. Impressive advances have been made over the past few years in the differentiation of human stem cells toward functional insulin secreting cells. So far, immunoisolation devices implanted with insulin secreting cells, proved to be disappointing in clinical trials. The scaffold invented by Adocia offers promising features to overcome this medical challenge. said Professor Franois Pattou, UMR1190 Translational Research for Diabetes Director, INSERM, University Hospital of Lille in France.

Upcoming events

In January, Adocia Members of management will be presenting at the virtual H.C. Wainwright BioConnect 2021 Conference, then will participate in the 24th ODDO BHF Digital Forum and BIO Partnering at JP Morgan. Please, see details and links on the Calendar below:

January 7 -13: ODDO Forum

January 11-14: H.C. Wainwright Conferences

Gerard Soula Presentation at H.C. Wainwright BioConnect 2021 Conference: Date: Monday, January 11th, 2021 Time: 6:00 am EST Link: https://journey.ct.events/view/05d513ad-9333-4971-9eee-1446015ccf34

The H.C. Wainwright webcast will be available on demand starting Monday, January 11th, 2021 at 6:00 Am EST. Replays of the presentation will be available on the Companys website for 90 days following the event.

January 11-15: BioPartnering at JP Morgan

About Adocia

Adocia is a clinical-stage biotechnology company that specializes in the development of innovative formulations of therapeutic proteins and peptides for the treatment of diabetes and metabolic diseases. In the diabetes field, Adocias portfolio of injectable treatments is among the largest and most differentiated of the industry, featuring five clinical-stage products and three products in preclinical-stage. Adocia aims to expand its portfolio towards the treatment of other metabolic diseases and their comorbidities. The proprietary BioChaperone technological platform is designed to enhance the effectiveness and/or safety of therapeutic proteins while making them easier for patients to use. Adocia customizes BioChaperone to each protein for a given application.

Adocias clinical pipeline includes four novel insulin formulations for prandial treatment of diabetes: two ultra-rapid formulations of insulin analog lispro (BioChaperone Lispro U100 and U200), a combination of basal insulin glargine and rapid-acting insulin lispro (BioChaperone Combo) and one combination of a prandial insulin with amylin analog pramlintide M1Pram. The clinical pipeline also includes an aqueous formulation of human glucagon (BioChaperone Glucagon) for the treatment of hypoglycemia.

Adocia preclinical pipeline includes three bi-hormonal products: two combinations of rapid human insulin analogues and Pramlintide (BioChaperone LisPram andBioChaperone AsPram) and a combination of insulin glargine with Liraglutide (BioChaperone GlaLira) for the treatment of diabetes.

Adocia has now added a fourth program in preclinical with the development of a hydrogel scaffold for cell therapy in the treatment of type 1 diabetes. A first patent has been filed.

Disclaimer

This press release contains certain forward-looking statements concerning Adocia and its business. Such forward-looking statements are based on assumptions that Adocia considers to be reasonable. However, there can be no assurance that the estimates contained in such forward-looking statements will be verified, which estimates are subject to numerous risks including the risks set forth in the Risk Factors section of the Universal Registration Document filed with the French Autorit des marchs financiers on April 22, 2020 (a copy of which is available at http://www.adocia.com) and to the development of economic conditions financial markets and the markets in which Adocia operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Adocia or not currently considered material by Adocia. The occurrence of all or part of such risks could cause actual results, financial conditions, performance, or achievements of Adocia to be materially different from such forward-looking statements. This press release and the information contained herein do not constitute an offer to sell or the solicitation of an offer to buy Adocia shares in any jurisdiction.

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Adocia Files Patent on a Hydrogel Scaffold for Cell Therapy in the Treatment of Type 1 Diabetes - Business Wire

Gene Therapy Market Estimated to Record Highest CAGR by 2024 – KSU | The Sentinel Newspaper

By Stem Cell Treatment

Transparency Market Research (TMR)has published a new report titled,Gene Therapy Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026.According to the report, theglobal gene therapy marketwas valued at US$ 17.0 Mn in 2017 and is projected to expand at a CAGR of 40.0% from 2018 to 2026. New product approvals, promising therapeutic outcomes of gene therapy, and high prevalence of non-Hodgkin Lymphoma are anticipated to drive the global market in the next few years. Europe is projected to dominate the global gene therapy market, followed by U.S., by the end of 2026. Potential unmet needs in the fields of oncology, rare genetic disorders in the U.S. and Europe, new product approvals and commercialization, and high clinical R&D budgets are likely to drive the gene therapy market in these regions during the forecast period. The gene therapy market in Rest of World is projected to expand at a significant CAGR during the forecast period. The high growth rate is attributed to the anticipated approval and commercialization of gene therapy products in developed countries such as Japan, Australia & New Zealand, GCC countries, and China, and high prevalence of non-Hodgkin Lymphoma and head and neck cancers.

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New product approvals & commercializationdrives market

2016, 2017, and 2018 were key milestones in the history of the gene therapy market in the U.S. and Europe, as around four gene therapy products have been approved and commercialized. These products are currently in the infancy stage of commercialization, and have exhibited highly positive therapeutic outcomes. For instance, in May 2016, GlaxoSmithKline (GSK) gene therapy product, Strimvelis, received marketing approval for the treatment of patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency, due to Adenosine Deaminase deficiency). Strimvelis is the first ex-vivo stem cell gene therapy to be approved in Europe for the treatment of ADA-SCID. Furthermore, in August 2017, the USFDA approved Novartis AGs flagship gene therapy product, Kymriah, for the treatment of children and adults up to the age of 25 years affected with B-cell precursor acute lymphoblastic leukemia (ALL). Thus, recent approvals of gene therapy products in the U.S. and Europe for the treatment of various life threatening disorders is projected to fuel the gene therapy market during the forecast period.

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Yescartato be highest revenue generating gene therapy product

The report offers a detailed segmentation of the global gene therapy market based on different gene therapy products approved and commercialized. Based on product, the global gene therapy market has been segmented into Yescarta, Kymriah, Luxturna, Strimvelis, and Gendicine. Yescarta (Axicabtagene Ciloleucel) is a genetically modified autologous Chimeric Antigen Receptor T (CAR T) cell immunotherapy developed by Gilead Sciences, Inc. for the treatment of adult patients with relapsed or refractory large B-cell lymphoma including diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL). It is the first CAR T therapy approved by the US FDA for the treatment of DLBCL. The Yescarta segment is projected to dominate the global gene therapy market by the end of 2026. Anticipated commercialization of Yescarta in Europe and other developed countries and increasing number of treatment centers are key factors that are likely to lead to the dominant share held by Yescarta by the end of 2026.

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Oncologysegment to account for high market share

In terms of application, the global gene therapy market has been segmented into ophthalmology, oncology, and adenosine deaminase ?deficient severe combined immunodeficiency (ADA-SCID). The oncology segment is likely to account for a significant share of the market by the end of 2026. Oncology is a highly studied medical field in the clinical pipeline studies of gene therapy candidates. More than 60% of gene therapy clinical research studies are focused on oncology. The large share held by the oncology segment is attributed to the approval and commercialization of Yescarta and Kymriah, in the last one to two years, for the treatment of certain types of non-Hodgkin lymphoma in the U.S. and Europe. Furthermore, increase in demand for Gendicine in China for the treatment of head and neck cancers is projected to drive the segment during the forecast period.

Europe offers high incremental opportunity

The gene therapy market in Europe is projected to expand at a significant CAGR of 30.6% during the forecast period. Large number of patient population with refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), promising therapeutic outcomes, rising demand for gene therapy treatment, and increasing number of gene therapy treatment centers in Europe are key factors that are likely to fuel the gene therapy market in Europe. Moreover, different pricing models are being evaluated by payers and governments to enable access to high priced gene therapy products. This is likely to drive the demand for gene therapy products in Europe during the forecast period.

Large number of clinical pipeline studies and significant investments in gene therapy to gain the first mover advantage

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The global gene therapy market is highly consolidated, with very few global players accounting for a major share. Currently, only five companies; Gilead life Sciences Inc. Spark Therapeutic Inc., Novartis AG, Sibiono GeneTech Co. Ltd, and Orchard Therapeutics Limited offer gene therapy products in the market. Most biopharmaceutical companies have invested significantly in clinical R&D for the development of gene therapy products for different chronic and genetic disorders. Large number of gene therapy products are under different stages of clinical pipeline studies, and the number of gene therapy candidates is projected to rise consistently during the forecast period. For instance, according to the Journal of Gene Medicine, there were around 2,597 gene therapy candidates under clinical trials, as of 2017. Of the total clinical studies, around 65% of studies were focused on oncology, 11% of studies were focused on monogenetic field, 7% on infectious diseases and cardiovascular disease, each.

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Gene Therapy Market Estimated to Record Highest CAGR by 2024 - KSU | The Sentinel Newspaper

Orchard Therapeutics Announces 2021 Corporate Priorities Supporting the Build-out of its Commercial Business in Hematopoietic Stem Cell (HSC) Gene…

By Stem Cell Clinic

January 11, 2021 07:00 ET | Source: Orchard Therapeutics (Europe) Limited

Preparations on Track for First Half 2021 Commercial Launch of Libmeldy (OTL-200), the First Approved Product for Metachromatic Leukodystrophy (MLD) in the EU

Filing Strategy for OTL-200 Biologics License Application (BLA) in MLD in the U.S. to be Communicated by Mid-2021 Following Additional Regulatory Interactions

Marketing Authorization Application (MAA) Filing for OTL-103 in Wiskott-Aldrich Syndrome (WAS) on Track for Year End 2021 in the EU; Followed by BLA Filing in 2022 in the U.S.

New Clinical Data for OTL-203 (for MPS-I) and OTL-201 (for MPS-IIIA) Accepted for Oral Presentation at February 2021 WORLD Symposium; Preclinical Data from Research Programs in Larger Indications Expected in 2021

$192M in Cash and Investments to Support Strategic Execution into the First Half of 2022

BOSTONandLONDON, Jan. 11, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today outlined the companys 2021 strategic priorities in advance of its attendance at the virtual 39thAnnual J.P. Morgan Healthcare Conference. These priorities support the companys plan of building a successful commercial business in HSC gene therapy and advancing its portfolio of investigational medicines for high-value, high-need indications.

In a year that challenged how we live and work, Im extremely proud of Orchards achievements in 2020, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. Our accomplishments were a direct result of the drive and innovation that fuels our commitment to bring our potentially life-saving HSC therapies to patients, including Libmeldy, which is the first product approved for the treatment of eligible patients with early-onset MLD in the EU. With the HSC approach to gene therapy as our scientific foundation, we are focused on the capabilities that can deliver our therapies on a global commercial scale and support our ability to also treat larger indications over time. It has been a privilege to be a pioneer in changing the way medicine is practiced in these conditions, and we look forward to another year of continued execution and scientific progress.

2021 Corporate Priorities Orchard has outlined the following key corporate objectives and expected milestones for 2021:

In preparation for a European launch, Orchard has put in place the commercial infrastructure to support Libmeldy as well as future product launches. The company is qualifying five treatment centers in the UK, Germany, Italy, France and the Netherlands with specialized expertise in transplant and disease area knowledge. In addition, the company expects to leverage cross-border and treatment abroad reimbursement pathways in both Europe and markets such as the Middle East and Turkey. Activities are also underway to drive timely MLD patient identification and access, including disease awareness, genetic testing and newborn screening studies, which have started or are on track to initiate in five countries in 2021.

The company also provided an update concerning the impact of the COVID-19 pandemic on certain development activities. These include restrictions to laboratory access at Orchard and third-party service providers, which is impacting the timeline to develop a specific functional potency assay for OTL-103 in WAS, as requested by the FDA. As a result, the company now expects to file a BLA for OTL-103 in the U.S. in 2022. Orchard is utilizing the benefits provided under OTL-103s RMAT designation and plans to continue interacting with the FDA in 2021 to confirm the data package for the BLA filing. In addition, with several of the follow-up visits associated with the companys active clinical trials impacted by COVID-19 travel restrictions and other trial site limitations, Orchard is using alternative data collection approaches to capture the necessary data to support future regulatory filings.

Frank Thomas, president and chief operating officer continued, Starting 2021 with a clear set of strategic priorities is crucial to our ability to effectively manage the business while fueling Orchards continued growth. Our launch preparations for Libmeldy not only mark our evolution towards a fully integrated company but establish a common manufacturing, commercial and operational infrastructure to support multiple future potential products. This work is complemented by our exciting proof-of-concept and research pipeline that we look forward to advancing internally or in partnership.

Key 2020 Achievements Orchards key 2020 achievements are highlighted below.

Cash Guidance The company ended 2020 with approximately $192 million of cash and investments. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2020 will enable the funding of its currently anticipated operating expenses and capital expenditure requirements into the first half of 2022. This excludes the $50 million expected to be available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers.

About Libmeldy / OTL-200

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, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene 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.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. 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 more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard

Orchard Therapeuticsis 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. Ourex vivoautologous 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 theSan Raffaele Telethon Institute for Gene Therapy inMilan, 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 TwitterandLinkedIn.

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 (TwitterandLinkedIn), 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.

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. 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, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2022. 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 risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard 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

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

Media Christine Harrison Vice President, Corporate Affairs +1 202-415-0137 media@orchard-tx.com

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Orchard Therapeutics Announces 2021 Corporate Priorities Supporting the Build-out of its Commercial Business in Hematopoietic Stem Cell (HSC) Gene...

Doctors diagnosed a Suffolk teen with a rare condition. Now, his mom is part of the solution – 13newsnow.com WVEC

By Stem Cell Doctors

Data shows disparities between communities in finding a match for blood stem cell transplants.

SUFFOLK, Va. Like any 14-year-old, Jerri McClammy loves to show his brothers whos the best.

In his Suffolk driveway on Pughsville Road, he can often be found playing basketball with any of his younger brothers, but even the simplest of joys come a little harder for the Suffolk teen.

This spring, doctors diagnosed Jerri with aplastic anemia, a rare condition that attacks the bodys ability to produce its own blood.

The last game he played, he couldnt breathe," mother Marie Veal told 13News Now. "Toppled over holding his stomach, just not being Jerri on the court.

Because of Jerri's inability to produce blood all on his own, blood transfusions are a regular part of his life with frequent trips to CHKD with his mom to get either new blood or new platelets.

As of January, doctors have not found a complete donor match for Jerri. According to data from the organization "Be The Match," the odds of Black or African-American communities finding a genetic match for blood stem cell transplants is 23 percent, the lowest of any ethnicity group. White populations have a 77 percent chance of finding a donor match.

On the mental capacity, it's very taxing. Not just Jerri I think of, but the other children too. Its a whole floor of kids," Veal said.

One day I feel good, but the next I'm tired," Jerri described.

The condition can drain Jerri of his energy, however, he and his mother are traveling to Philadelphia for a bone marrow transplant this February. Veal is still only a partial match, and the combined procedure and recovery is expected to take months. The hope is that after the procedure, Jerri can produce enough of his own blood where he can afford to have less frequent blood transfusions.

Beyond just themselves, Veal is sharing their story and her son's journey to bring awareness about disparities for genetic matches.

Its not just a kid thing. It can be adults or kids that need a bone marrow transplant just to save a life," Veal said.

According to Be The Match, Veal has registered more than 25 people on the donor registry alone.

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Doctors diagnosed a Suffolk teen with a rare condition. Now, his mom is part of the solution - 13newsnow.com WVEC

Cassville club assists 2 families as they recover from illness – telegraphherald.com

By Stem Cell Doctors

CASSVILLE, Wis. Community members have rallied around two Cassville families whose lives have been impacted by debilitating diseases.

The Cassville chapter of Royal Neighbors of America, a philanthropic association that participates in difference maker initiatives, recently sponsored fundraisers for David Schauff and Tripp Rouse.

Group members Susan Kremer and Susan Bernhardt spearheaded the efforts, which garnered significant local attention.

We are always looking for something to do that can help out, Bernhardt said. We knew that there was a need. We come from a small town.

Schauff, 41, was diagnosed with POEMS syndrome last year and underwent chemotherapy as well as a stem cell transplant in November.

The syndrome is an incurable blood disorder that causes damage to nerves and organs. David, who still is recuperating, was unavailable for comment, but his wife, Karri, said he is not a quitter.

Simply receiving a diagnosis took the better part of a year, during which he struggled with his balance and experienced neuropathy in his feet and hands.

They do not know what caused it or anything, she said.

The family routinely visited UW Hospital and Clinics in Madison, and the medical bills accumulated.

Karri said the family was floored when they heard that the Royal Neighbors decided to raise money for them. They also received support from residents in Lancaster, where they own childrens clothing store Cruisin Kids.

We couldnt believe that people were thinking of us for starters, Karri said. It was amazing to know that with one call, I would have 20 people there wanting to help.

David hopes to return to his family farm, where he raises beef cattle, corn and beans. His three children, Dayton, Dylan and Kinsey, motivate him to keep pushing on, Karri said.

Elsewhere in Cassville, Tripp Rouse, 5, struggled to maintain his balance and coordination, a circumstance that concerned his mother, Donisha. After doctors ran several tests, they discovered that he had a benign tumor growing in his brain, a type known as pilocytic astrocytoma.

Tripp underwent surgery in July to remove it. The procedure left him temporarily paralyzed on his left side, and he had to undergo therapy to regain his mobility, a process that continues.

But the tumor grew back. A second surgery in November removed about 70% of the mass, but the remainder is too close to his brain stem to risk an additional operation.

He may need surgery again, Donisha said. He may need chemotherapy.

Tripp still is regaining the use of his limbs, but he thinks that he is back to normal and wants to roughhouse and argue with his five brothers, she said.

Tripp also is a huge fan of Spider-Man.

Because he shoots webs, Tripp said. He can climb walls.

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Cassville club assists 2 families as they recover from illness - telegraphherald.com

The 26 billion-dollar startups to watch that are revolutionizing healthcare in 2021 – Business Insider

By Stem Cell Doctors

The financial calamities predicted at the beginning of the coronavirus pandemic didn't materialize for most healthcare startups.

The industry's up-and-coming private companies largely benefited from the one-two punch of financial concerns in other parts of the market and an increased focus on healthcare with all eyes on America's wavering pandemic response.

2020 will likely go down as one of the single most pivotal years for the US healthcare industry in history. Even outside large hospitals and pharmaceutical companies, private startups raised a whopping $17 billion in 2020, a 57% increase over 2019's record, according to a new report from Silicon Valley Bank.

That led to a new herd of healthcare unicorns, private companies valued at or above $1 billion. Some former unicorns, like GoodRx and Amwell, made public market debuts while others continued raising venture capital and private financings while the market was favorable. Some startups raised two separate funding rounds in the last year alone.

Read more: The 8 digital health startups to watch that are changing healthcare in 2021

Business Insider rounded up the 26 healthcare companies currently valued at more than $1 billion, according to Pitchbook and additional reporting.

More here:
The 26 billion-dollar startups to watch that are revolutionizing healthcare in 2021 - Business Insider

Global Precision Medicine Market 2020 Overview By Size, Share, Trends, Growth Factors and Leading Players With Detailed Analysis of Industry Structure…

By Stem Cell Doctors

DBMR has added a new report titled Global Precision Medicine Market with analysis provides the insights which bring marketplace clearly into the focus and thus help organizations make better decisions. This Global Precision Medicine Market research report understands the current and future of the market in both developed and emerging markets. The report assists in realigning the business strategies by highlighting the business priorities. It throws light on the segment expected to dominate the industry and market. It forecast the regions expected to witness the fastest growth. This report is a collection of pragmatic information, quantitative and qualitative estimation by industry experts, the contribution from industry across the value chain. Furthermore, the report also provides the qualitative results of diverse market factors on its geographies and Segments.

Global Precision Medicine Market to grow with a substantial CAGR in the forecast period of 2019-2026. Growing prevalence of cancer worldwide and accelerating demand of novel therapies to prevent of cancer related disorders are the key factors for lucrative growth of market

Global Precision Medicine Market By Application (Diagnostics, Therapeutics and Others), Technologies (Pharmacogenomics, Point-of-Care Testing, Stem Cell Therapy, Pharmacoproteomics and Others), Indication (Oncology, Central Nervous System (CNS) Disorders, Immunology Disorders, Respiratory Disorders, Others), Drugs (Alectinib, Osimertinib, Mepolizumab,Aripiprazole lauroxil and Others), Route of Administration (Oral,Injectable), End- Users (Hospitals, Homecare, Specialty Clinics, Others), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2026

Download Free PDF Sample Copy of Report@ http://databridgemarketresearch.com/request-a-sample/?dbmr=global-precision-medicine-market

Competitive Analysis:

The precision medicine market is highly fragmented and is based on new product launches and clinical results of products. Hence the major players have used various strategies such as new product launches, clinical trials, market initiatives, high expense on research and development, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of mass spectrometry market for global, Europe, North America, Asia Pacific and South America.

Market Definition:

Precision medicines is also known as personalized medicines is an innovative approach to the patient care for disease treatment, diagnosis and prevention base on the persons individual genes. It allows doctors or physicians to select treatment option based on the patients genetic understanding of their disease.

According to the data published in PerMedCoalition, it was estimated that the USFDA has approved 25 novels personalized medicines in the year of 2018. These growing approvals annually by the regulatory authorities and rise in oncology and CNS disorders worldwide are the key factors for market growth.

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Market Drivers

Market Restraints

Key Developments in the Market:

Competitive Analysis:

Global precision medicine market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of global precision medicine market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Players:

Few of the major competitors currently working in the global precision medicine market are Neon Therapeutics, Moderna, Inc, Merck & Co., Inc, Bayer AG, PERSONALIS INC, GENOCEA BIOSCIENCES, INC., F. Hoffmann-La Roche Ltd, CureVac AG, CELLDEX THERAPEUTICS, BIONTECH SE, Advaxis, Inc, GlaxoSmithKline plc, Bioven International Sdn Bhd, Agenus Inc., Immatics Biotechnologies GmbH, Immunovative Therapies, Bristol-Myers Squibb Company, Gritstone Oncology, NantKwest, Inc among others.

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Market Segmentation:

By technology:- big data analytics, bioinformatics, gene sequencing, drug discovery, companion diagnostics, and others.

By application:- oncology, hematology, infectious diseases, cardiology, neurology, endocrinology, pulmonary diseases, ophthalmology, metabolic diseases, pharmagenomics, and others.

On the basis of end-users:- pharmaceuticals, biotechnology, diagnostic companies, laboratories, and healthcare it specialist.

On the basis of geography:- North America & South America, Europe, Asia-Pacific, and Middle East & Africa. U.S., Canada, Germany, France, U.K., Netherlands, Switzerland, Turkey, Russia, China, India, South Korea, Japan, Australia, Singapore, Saudi Arabia, South Africa, and Brazil among others.

In 2017, North America is expected to dominate the market.

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Global Precision Medicine Market 2020 Overview By Size, Share, Trends, Growth Factors and Leading Players With Detailed Analysis of Industry Structure...

More than 250 articles and a book later, the Valley Doctor bids adieu – San Lorenzo Valley Press-Banner

By Stem Cell Doctors

After 25 years in Scotts Valley urgent care, followed by 12 years as a health columnist at the Press Banner, Dr. Terry Hollenbeck is leaving this publication to focus on his health and family.

The Valley Doctor leaves with these words of advice: Take the pandemic seriously. Wearing masks, social distancing, and not gathering will be as important to end the pandemic as the vaccines.

Born in Milwaukee, Hollenbeck moved to California in 1971 after graduating with an MD from the Medical College of Wisconsin. Beginning his medical career as an intern at Santa Clara Valley Medical Center, the doctor said he expected a short stay in San Jose.

I thought, If Im going to be away from Milwaukee for a year, I may as well enjoy California for a bit, he said. I never went back. California has a way of grabbing hold of you.

Hollenbeck was hired by the County of Santa Clara as the chief jail physician. Like many recent graduates, Hollenbeck felt idealistic, well trained, and enjoyed my work.

If I could bring some needed good care to the jail population, I thought Id give a try, he said.

He worked in San Jose for two years, before receiving an opportunity to try emergency care. From 1974-1980, Hollenbeck staffed the Emergency Room at the Santa Clara Valley Medical Center, until he felt it was time for a change.

I have to admit, I got burnt out in the emergency room, with hectic pace and working nights, he said.

He changed his pace greatly for the next two years. He joined the Christian Medical Society, learned Spanish, headed to Honduras, and set up a one-room clinic with the help of Miskito natives.

I grew up with stories about my great uncles career as a medical missionary in Africa. It fascinated me, he said. I really enjoyed that experience. There was no electricity, running water, or cars. Once a week, a plane would bring more supplies and that was it. Going up river in a dugout canoe to see a sick person was magicalBut, after two years I was lonely. There were few english speakers in the area and I missed family and friends.

Hollenbeck returned to California in the beginning of the 80s and joined Doctors on Duty as a medical director for four years.

Urgent care had just become a specialty, and I thought, That sounds good, like emergency care, but less hectic, he said. You can sleep at night.

Soon after, the Santa Cruz Medical Clinic, now a part of the Palo Alto Medical Foundation, began a satellite clinic in Scotts Valley. Hollenbeck found his perfect niche as a member of their urgent care staff for 25 years.

I loved working in Scotts Valley, he said. The people and staff were wonderful.

In 2012 Hollenbeck began his newest career as a Press Banner columnist. Since then, hes written over 250 columns and published a book, House Calls: Guidance on common medical topics from your Doctor-Next-Door.

Ive always tried to write my articles as if I were in the room with patients, hearing their complaints, giving a physical exam, and coming up with treatments Im prejudiced, of course, but I think every household needs this book to run the gamut of medical issues, he said.

In 2013, Hollenbeck and his family received unpleasant news: he was diagnosed with a blood cancer.

For the past seven years, Hollenbeck has received various treatments for his multiple myeloma, including chemotherapy, radiation and a stem cell transplant. He said he and his loved ones look ahead bravely with hope.

Now that Ive gone through the traditional treatments, were not sure what lies ahead, he said. Im hopeful that we can find other treatments and keep going.

In the meantime, hes relaxing at home with family. Hollenbeck sees the silver lining of the pandemic as a chance to spend more quality time with his loved ones.

My wife is the music educator for Scotts Valley Middle School and High School, he said. She has been working from home, so Ive been able to spend a lot of good quality time with her and shes been a fantastic caregiver for me.

Hes also been overjoyed to spend time with his grandson.

I feel so fortunate to live long enough to meet my grandson, he said.

Continued here:
More than 250 articles and a book later, the Valley Doctor bids adieu - San Lorenzo Valley Press-Banner

New Models in Organoids Market Open New Vistas in Stem Cell Research for Cancer, Global Valuation to Reach US$ 12.8 Bn by 2030: TMR – PRNewswire

By Adult Stem Cells

ALBANY, N.Y., Jan. 12, 2021 /PRNewswire/ -- Organoids are stem cell-derived 3D culture systems and are usually derived from induced pluripotent stem cells (iPSCs) and multipotent adult tissue stem cells (ASCs). The technologies in the organoids market have emerged as a novel culture used for human disease modelling. Their amazing capability in recapitulating in vivo anatomy and physiology of organs is utilized to open new paradigms in cell biology areas such as in gene therapy, regenerative medicine, and cancer research. Most prominently, researchers and industry players have harnessed the potential of organoids in regenerative medicine and tissue engineering.

Advent of new methods in generating 3D structures are opening new vistas in human disease modelling, particularly in virology. The utilization of these in drug discovery and personalized medicine will transform medical care in years to come. Europe and North America have emerged as the new hotspots for patient-derived human organoid studies in the global organoids market.

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The revenue of global organoid market is projected to climb from US$ 1.7 Bn in 2019 to touch the mark of US$ 12.8 Bn by 2030.

Key Findings of Organoids Market

In the backdrop of the need for new approaches of studying the pathogenesis of currently emerging Covid-19, organoids market is replete with incredible revenue potential for stakeholders. Researchers are relentlessly working toward new organoids approaches for understanding tissue tropism of SARS-CoV-2. In the last few years, the strides in the organoids market has unarguably expanded the armamentarium of virologists studying infectious diseases. A case in point was Zika virus infection.

Patient-derived human organoids are increasingly being leveraged upon by researchers to open new avenues in tissue engineering and regenerative medicine. These 3D-based cultures have been able to overcome the limitations of 2D cancer-derived cell lines, notably in bladder, colorectal, brain, and liver cancer. There is demand for new patient-derived cell lines for cancer sample biobanking. Integrating biobanking with tumor modelling has undoubtedly expanded the avenue in cancer care. This is also expanding the avenue for precision medicine, the relevance of which is gather traction in patient care.

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Over the years, the organoid market has made some remarkable strides on the back of collaborations between researchers in universities and medical experts in healthcare institutes. Next-gen organoid development for Covid-19 is a case in point where there has been surge in research funding. Giant leaps made by genome editing systems have expanded the avenue in genome engineering of human stem cells. This will test new methods of generating human organoid models. Another researcher directions attracting investments are in development of cerebral organoids for neurological diseases.

Organoids Market: Key Driving Factors and Promising Avenues

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Organoids Market: Competitive Dynamics

Explore Transparency Market Research's award-winning coverage of the global Healthcare Industry:

Gene Therapy Market:https://www.transparencymarketresearch.com/gene-therapy-market.html

Genome Editing Market:https://www.transparencymarketresearch.com/genome-editing-market.html

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New Models in Organoids Market Open New Vistas in Stem Cell Research for Cancer, Global Valuation to Reach US$ 12.8 Bn by 2030: TMR - PRNewswire

Health Canada Approves ONUREG (azacitidine tablets), First Maintenance Therapy for Patients in Remission from Acute Myeloid Leukemia – Canada NewsWire

By Adult Stem Cells

AML is a heterogeneous clonal disorder characterized by immature myeloid cell proliferation and bone marrow failure, and is the most common form of acute leukemia in adults, accounting for approximately 80 per cent of adult cases.2,3,4 An estimated 40-60 per cent of patients aged 60 years and older and 60-80 per cent of patients under 60 years old will obtain complete remission through induction chemotherapy (IC); however, 50 per cent will relapse within a year.5,6 Once a relapse occurs, long-term survival averages at six months.7 In 2015, an estimated 1,235 Canadians were diagnosed with AML and the overall incidence rate in Canada is 3.46/100,000 people.8,9

"While the majority of patients with AML achieve a complete remission with intensive chemotherapy, many remission patients will experience disease relapse, especially if they were not eligible for a stem cell transplant. Until now, there has been no established standard of care for Canadians who are in remission from AML, but are not eligible for a stem cell transplant," noted Dr. Andre Schuh, Princess Margaret Cancer Centre, Toronto. "The approval of ONUREG is significant because it gives transplant ineligible patients with AML in remission a new treatment option that may improve their survival".

ONUREG is a nucleoside metabolic inhibitor that is taken orally and works by preventing cancer cells from growing. ONUREG becomes incorporated into the building blocks of cells (deoxyribonucleic acid (DNA) and ribonucleic acid (RNA)), which interferes with the production of new DNA and RNA. This is thought to kill cancerous cells in leukemia.10

"The approval of ONUREG is an extension of our ongoing commitment to Canadians living with blood cancer," said Al Reba, General Manager, Bristol Myers Squibb Canada. "We are proud that this therapy will help to fill a significant need for Canadians living in remission from AML and hope that it will have a positive impact on their everyday life."

Health Canada's approval of ONUREG is based upon findings from the QUAZAR AML-001 clinical trial. The QUAZAR study, a double-blind, randomized, placebo-controlled, multicenter Phase III study, involved adult patients 55 years or older living with AML. In the study, patients were randomized to Onureg or placebo within four months of achieving first CR/CRi following intensive induction chemotherapy and were not eligible for a stem cell transplant.11In the study, results showed the median overall survival (OS) was significantly longer with ONUREG versus placebo: 24.7 months versus 14.8 months [HR 0.69 (95% CI: 0.55, 0.86); p=0.0009], indicating a 31 per cent reduction in the risk of death in the ONUREG arm. Relapse-free survival (RFS), the key secondary endpoint in the study, supports the OS results. The median RFS was 10.2 months for ONUREG versus 4.8 months for placebo [HR 0.65 (95% CI: 0.52, 0.81); p=0.0001].12

About Bristol Myers Squibb CanadaBristol Myers Squibb Canada Co. is an indirect wholly-owned subsidiary of Bristol Myers Squibb Company, a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb global operations, visitwww.bms.com. Bristol Myers Squibb Canada Co. delivers innovative medicines for serious diseases to Canadian patients in the areas of cardiovascular health, oncology, and immunoscience. Bristol Myers Squibb Canada Co. employs close to 400 people across the country. For more information, please visitwww.bms.com/ca.

About Bristol Myers SquibbBristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us atBMS.comor follow us on LinkedIn, Twitter, YouTube, Facebookand Instagram.

References

_________________________

1 ONUREG Product Monograph, January 2021.

2 Saultz JN, Garzon R. J Clin Med 2016;5:33.

3Leukemia & Lymphoma Society of Canada. Acute Myeloid Leukemia. Available from https://www.llscanada.org/sites/default/files/National/CANADA/Pdf/InfoBooklets/AML%20Fact%20Sheet%2012-2019.pdf. Accessed December 11, 2020.

4De Kouchkovsky I, Abdul-Hay M. Blood Cancer J 2016;e441:DOI:10.1038/bcj.2016.50.

5Dohner et al. Blood. 2017;129(4):42447.

6SEER Cancer Statistics, 2007-2013.

7Xu J, et al. Medicine (Baltimore) 2018;97:e12102.

8Statistics Canada. Population estimates on July 1st, by age and sex. Available from https://www150.statcan.gc.ca/t1/tbl1/en/tv.action?pid=1710000501&pickMembers%5B0%5D=1.1&pickMembers%5B1%5D=2.1&cubeTimeFrame.startYear=2015&cubeTimeFrame.endYear=2016&referencePeriods=20150101%2C20160101. Accessed December 11, 2020.

9Shysh et al. BMS Public Health (2018) 18:94.

10ONUREG Product Monograph, January 2021.

11ONUREG Product Monograph, January 2021.

12 ONUREG Product Monograph, January 2021.

SOURCE Bristol Myers Squibb Canada Co.

For further information: For media requests, please contact: Rachel Yates, Lead, Corporate Affairs, Bristol Myers Squibb Canada, [emailprotected]; Alannah Nugent, Account Executive, Health, Edelman, [emailprotected]

Original post:
Health Canada Approves ONUREG (azacitidine tablets), First Maintenance Therapy for Patients in Remission from Acute Myeloid Leukemia - Canada NewsWire

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