Stem Cell Clinic

Jasper Therapeutics to Present Updated Data on JSP191 Conditioning in SCID Patients at the 2022 Clinical Immunology Society Annual Meeting – Yahoo…

Jasper Therapeutics

REDWOOD CITY, Calif., March 31, 2022 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced that updated data from the Companys ongoing study of JSP191 as single agent conditioning prior to allogeneic hematopoietic stem cell (HSC) re-transplant in patients with severe combined immunodeficiency (SCID) has been accepted for presentation as a late-breaking poster at the 2022 Clinical Immunology Society (CIS) Annual Meeting, to be held in Charlotte, North Carolina from March 31 to April 3, 2022.

Title: Update: Single-Agent Conditioning with Anti-CD117 Antibody JSP191 Shows Donor Engraftment, Nave Lymphocyte Production, and Clinical Benefit in Patients with Severe Combined Immunodeficiency (SCID) Date and Time: Friday, April 1, 2022, 1:00-2:00 p.m. ET

This updated data indicates that JSP191 at 0.6mg/kg can deplete blood stem cells, leading to long-term donor cell engraftment, immune reconstitution which positively affects the clinical status of SCID patients who suffer from poor T cell and negligible B cell immunity because they failed their first transplant, said Wendy Pang, MD, Ph.D., Senior Vice President of Research and Translational Medicine of Jasper Therapeutics. This population of SCID patients is largely without treatment options and rely on supportive therapies like life long IVIG to provide some level of immune protection. JSP191 based conditioning may provide these patients with the best chance of a safe and successful transplant and reconstituted immune system.

CIS attendees are the primary caregivers for the immune deficient patient population, we are pleased to be able to present this data at the 2022 CIS annual meeting, Ronald Martell, CEO of Jasper. We believe that with our successful clinical efforts, we are one step closer, and uniquely positioned to deliver a targeted non-genotoxic conditioning agent to patients with SCID.

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About JSP191

JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or genetically modified transplanted stem cells to engraft. To date, JSP191 has been evaluated in more than 100 healthy volunteers and patients. Three clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML), severe combined immunodeficiency (SCID) and Fanconi anemia are currently enrolling. The Company plans a new study of JSP191 as a second-line therapeutic in lower risk MDS patients in 2022 as well as to a pivotal study in MDS/AML transplant in early 2023. Enrollment in additional studies are planned in patients with sickle cell disease, chronic granulomatous disease and GATA2 MDS who are undergoing hematopoietic cell transplantation.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical mRNA engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

Forward-Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding the potential long-term benefits of hematopoietic stem cells (HSC) engraftment following targeted single-agent JSP191 conditioning in the treatment of severe combined immunodeficiency (SCID) and Jaspers ability to potentially deliver a targeted non-genotoxic conditioning agent to patients with SCID. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Jaspers product candidates; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that Jaspers product candidates may not be beneficial to patients or successfully commercialized; patients willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jaspers business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jaspers business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jaspers filings with the SEC. If any of these risks materialize or Jaspers assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jaspers assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Contacts: John Mullaly (investors) LifeSci Advisors 617-429-3548 jmullaly@lifesciadvisors.com

Jeet Mahal (investors) Jasper Therapeutics 650-549-1403 jmahal@jaspertherapeutics.com

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Jasper Therapeutics to Present Updated Data on JSP191 Conditioning in SCID Patients at the 2022 Clinical Immunology Society Annual Meeting - Yahoo...

Rheumatoid Arthritis Stem Cell Therapy Market Assessment, With Major Top Companies Analysis, Geographic Analysis, Growing Opportunities Data By…

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Rheumatoid Arthritis Stem Cell Therapy Market Assessment, With Major Top Companies Analysis, Geographic Analysis, Growing Opportunities Data By...

Sana On Track to Remove Barriers for Cell & Gene Therapy – BioSpace

A Sana scientist works in the lab/Photo courtesy of Sana Biotechnology

One of the most important transformations of 21st-centurymedicine will be the ability to reengineer cells as medicines. Before cell and gene therapy can truly become mainstream medicine, the challenges of allogeneic rejection and targeted delivery must be solved. Sana Biotechnology is on the path to do that, with notable strides that may, just possibly, change medicine as we know it.

Our aspirations are to be able to repair or control genes and gene expression of every cell type in the body thats broken, and to transplant or replace whats missing or too far damaged, Steve Harr, M.D., Sanas president and CEO, told BioSpace.

The scope of that mission is huge. The breadth of our aspirations set Sana apart from most biotech companies, he said. And, Sana is approaching the challenge like any other great endeavor: one step at a time. In this case, that means with ex vivo and in vivo cell engineering platforms.

Sanas ex vivo hypoimmune platform (HIP) is designed to prevent allogeneic rejection, in which the immune system recognizes modified cells as foreign and attacks them. This is perhaps the greatest issue that has held cell therapy back from reaching its potential for patients, Harr said.

The hypoimmune platform appears to solve that hurdle, he said. We have shown in preclinical models that we have the potential to prevent allogeneic rejection.

As Sonja Schrepfer,M.D., Ph.D., head of Hypoimmune Platform, elaborated, The human leukocyte antigens (HLA) on a cells surface are like fingerprints for proteins on the cell. They tell the immune system what is inside your cell. We recognized HLA as a major hurdle (in overcoming rejection) and so prevented expression of the HLA. Now the cells fingerprint is missing.

Of course, viruses figured out long ago that removing HLA can hide cells, so the immune system evolved to be smarter, she continued. NK cells and macrophages recognize cells without HLA (missing self) and attack them. To protect those cells from this attack, we overexpress CD47, which basically is a dont attack me molecule. It protects these cells from being recognized by NK cells and macrophages, allowing them to survive.

These cellular modifications can be performed on pluripotent stem cells, which can then be differentiated into essentially any hypoimmune cell, and also on allogeneic donor cells. We intend to derive hypoimmune pancreatic islet cells to treat type 1 diabetes, for example. Our goal is to provide off-the-shelf cells to anyone, anywhere, at any time that can function without requiring the patient to take immunosuppressive medications, Schrepfer said. Sana also is working to develop T cells, glial progenitor cells and cardiomyocytes as allogeneic therapies.

In preclinical models, We have seen that without immunosuppression, the HIP-edited induced pluripotent stem cells (iPSCs) survived for many weeks without activating the immune system or being rejected, Schrepfer said. That was very exciting! The company is planning to file its first IND to test this system in patients as early as this year.

Targeted gene delivery is the second big challenge Sana is addressing. With antiviral vectors (AAVs), you cant control where the vectors go and, because the cellular DNA is not changed, they have less utility in dividing cells. Lipid nanoparticles (LNPs), another popular delivery vehicle, at this time mainly go to the liver and deliver only RNA and proteins, not DNA, Harr said.

Sanas fusogen platform has the potential to overcome those limitations with in vivo cell and gene modification. You can make just about any modification to genes and gene expression in a petri dish, Harr pointed out, but it is very difficult to deliver the gene modification payload in vivo. Fusogens offer a unique delivery capability for gene editing and gene modification machinery. We have a number of programs in preclinical development, including four that modify T cells for multiple oncology indications, one that modifies hepatocytes to address liver-related disorders and one that modifies hematopoietic stem cells to address hemoglobinopathies.

Sanas science has the potential to disrupt the cell and gene therapy sector of the industry, but its effects on the practice of medicine are still at least several years away. As Harr said, Our key competitive advantage, as a smaller company, needs to be decision-making, which is possible because of greater focus and having the right people.

Harr defines the right people as program heads who are global leaders in their fields. Thats really important, Harr explained, because this isnt as easy as normal drug development (which, itself, isnt easy). For example, Richard Mulligan, Ph.D., head of SanaX, is instrumental in Sanas work to deliver genomic material. SanaX is a distinct research arm within Sana focused around long-term, disruptive improvements in cell and gene therapy. Mulligan was among the first to discover how to insert genes into cells in the 1980s. Terry Fry, M.D., SVP and head of T Cell Therapeutics, is a renowned expert in chimeric antigen receptor T cell (CAR T) therapies and was critical in the development of a number of CAR T cell therapies to date, including Yescarta. Chuck Murry, M.D., Ph.D., SVP and head of cardiometabolic cell therapy, pioneered the use of human pluripotent stem cells for heart regeneration.

The executive leaders also bring a wealth of experience. Some, including Harr, joined Sana after senior positions at Juno Therapeutics. Others honed their skills at Genentech, Amazon, Amgen, Sangamo and the U.S. Food and Drug Administration, amongst others.

Sanas staff of approximately 400 is divided among its facilities in South San Francisco, Seattle and Cambridge, Massachusetts.

The company is expanding, so there are opportunities throughout Sana both geographically and in terms of business and technical specialties. Harr said one hiring priority is in the area of process and analytical development for manufacturing. Another area includes hiring experts in analytic genomics and computational biology to enable in-depth interrogation of the genome, both when youre inserting things into the cells and as stem cells divide and differentiate, he added.

Our goal is to make important medicines that matter for patients, and these roles are essential in doing that. That means the Sana team must also be flexible and resilient.

The world is constantly changing, and we live on the tip of the innovation spear, Harr said. We need people who are intellectually curious and who have grit. Being a pioneer is hard. We will have great days, and we also will have setbacks. We need people who are resilient and who can collaborate effectively on teams. The things we are doing are complex enough that none of us have all the answers. It always takes some of us.

Harr said Sana is committed to ensuring we do all we can to increase individuals chances to succeed and rewarding them fairly. Biotech tries to help people from all over the world. To do that, we need an environment where people can thrive.

Harr continued, Inclusion is the soil from which all great cultures grow. To truly feel included, you need diversity. It helps to know that there are people just like me whove succeeded here. Succeeding is easier when you have that example.

With that philosophy, Sanas Inclusion, Diversity and Equity program is robust. The measure of a great culture is whether it helps you succeed or holds you back. Our measure is to help you be your best self, your true self and to accept people for who they are. This is an exciting time to be at Sana, Harr said.

Sana is transforming from a research company to a research and development company. The movement into development is an important next step, Harr said. Its the chance to prove things work.

Sana is working towards developing important cell and gene therapies for patients. As Harr said, This is a dynamic company with complex science. We have chosen to be audacious.

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Sana On Track to Remove Barriers for Cell & Gene Therapy - BioSpace

Are COVID-19-Linked Arrhythmias Caused by Viral Damage to the Heart’s Pacemaker Cells? – Weill Cornell Medicine Newsroom

The SARS-CoV-2 virus can infect specialized pacemaker cells that maintain the hearts rhythmic beat, setting off a self-destruction process within the cells, according to a preclinical study co-led by researchers at Weill Cornell Medicine, NewYork-Presbyterian and NYU Grossman School of Medicine. The findings offer a possible explanation for the heart arrhythmias that are commonly observed in patients with SARS-CoV-2 infection.

In the study, reported March 8 in Circulation Research, the researchers used an animal model as well as human stem cell-derived pacemaker cells to show that SARS-CoV-2 can readily infect pacemaker cells and trigger a process called ferroptosis, in which the cells self-destruct but also produce reactive oxygen molecules that can impact nearby cells.

This is a surprising and apparently unique vulnerability of these cellswe looked at a variety of other human cell types that can be infected by SARS-CoV-2, including even heart muscle cells, but found signs of ferroptosis only in the pacemaker cells, said study co-senior author Dr. Shuibing Chen, the Kilts Family Professor of Surgery and a professor of chemical biology in surgery and of chemical biology in biochemistry at Weill Cornell Medicine.

Arrhythmias including too-quick (tachycardia) and too-slow (bradycardia) heart rhythms have been noted among many COVID-19 patients, and multiple studies have linked these abnormal rhythms to worse COVID-19 outcomes. How SARS-CoV-2 infection could cause such arrhythmias has been unclear, though.

In the new study, the researchers, including co-senior author Dr. Benjamin tenOever of NYU Grossman School of Medicine, examined golden hamstersone of the only lab animals that reliably develops COVID-19-like signs from SARS-CoV-2 infectionand found evidence that following nasal exposure the virus can infect the cells of the natural cardiac pacemaker unit, known as the sinoatrial node.

To study SARS-CoV-2s effects on pacemaker cells in more detail and with human cells, the researchers used advanced stem cell techniques to induce human embryonic stem cells to mature into cells closely resembling sinoatrial node cells. They showed that these induced human pacemaker cells express the receptor ACE2 and other factors SARS-CoV-2 uses to get into cells and are readily infected by SARS-CoV-2. The researchers also observed large increases in inflammatory immune gene activity in the infected cells.

The teams most surprising finding, however, was that the pacemaker cells, in response to the stress of infection, showed clear signs of a cellular self-destruct process called ferroptosis, which involves accumulation of iron and the runaway production of cell-destroying reactive oxygen molecules. The scientists were able to reverse these signs in the cells using compounds that are known to bind iron and inhibit ferroptosis.

This finding suggests that some of the cardiac arrhythmias detected in COVID-19 patients could be caused by ferroptosis damage to the sinoatrial node, said co-senior author Dr. Robert Schwartz, an associate professor of medicine in the Division of Gastroenterology and Hepatology at Weill Cornell Medicine and a hepatologist at NewYork-Presbyterian/Weill Cornell Medical Center.

Although in principle COVID-19 patients could be treated with ferroptosis inhibitors specifically to protect sinoatrial node cells, antiviral drugs that block the effects of SARS-CoV-2 infection in all cell types would be preferable, the researchers said.

The researchers plan to continue to use their cell and animal models to investigate sinoatrial node damage in COVID-19and beyond.

There are other human sinoatrial arrhythmia syndromes we could model with our platform, said co-senior author Dr. Todd Evans, the Peter I. Pressman M.D. Professor of Surgery and associate dean for research at Weill Cornell Medicine. And, although physicians currently can use an artificial electronic pacemaker to replace the function of a damaged sinoatrial node, theres the potential here to use sinoatrial cells such as weve developed as an alternative, cell-based pacemaker therapy.

Many Weill Cornell Medicine physicians and scientists maintain relationships and collaborate with external organizations to foster scientific innovation and provide expert guidance. The institution makes these disclosurespublic to ensure transparency. For this information, see profiles for Dr. Todd Evans, and Dr. Robert Schwartz.

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Are COVID-19-Linked Arrhythmias Caused by Viral Damage to the Heart's Pacemaker Cells? - Weill Cornell Medicine Newsroom

Dr. Dietrich on the Current Treatment Landscape of MCL – OncLive

Martin Dietrich, MD, PhD, discusses the current treatment landscape of mantle cell lymphoma.

Martin Dietrich, MD, PhD, physician, Florida Cancer Specialists and Research Institute, assistant professor of internal medicine, University of Central Florida, discusses the current treatment landscape of mantle cell lymphoma (MCL).

Treatment options have drastically evolved in recent years for MCL, which remains an aggressive and complex subtype of lymphoma, Dietrich says. Disease risk assessment and a patients performance status help inform the optimal first-line treatment, Dietrich adds. First-line treatment options include combination chemotherapy and rituximab (Rituxan) for in clinically fit and higher risk patients, and de-escalation chemotherapy strategies for patients with low-risk disease or a poor performance status, Dietrich explains.

Autologous stem cell transplant (ASCT) has carved out a role in the treatment of patients with high-risk disease. However, it is unclear how impactful ASCT will remain in the future with the emergence of better subsequent therapies, Dietrich continues. For now, ASCT is still recommended for many patients, along with maintenance rituximab for patients after initial induction chemotherapy, Dietrich concludes.

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Dr. Dietrich on the Current Treatment Landscape of MCL - OncLive

Stem Cells Turn Into Bone When Sound Waves Are Near – TechTheLead

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A breakthrough made by Australian researchers might change the way doctors treat a broken or missing bone. Turns out stem cells can turn into bone if certain conditions are met.

Normally, bone can be made only of mesenchymal stem cells (MSCs) which are biologically found in the bone marrow.

Extracting them from there is a difficult and painful procedure while doing so at scale is beyond tricky.

But this could change any moment now after Australian researchers found that stem cells can be converted into bone when a certain type of sound waves are used.

Tests had previously shown that low frequency vibrations were great at inducing cell differentiation but the process took over a week and the results were mixed at best.

Nobody had bothered to look into high frequency sound waves until now. RMIT researchers took a microchip capable of dispersing sound waves in the Mhz range and turned it at MSCs in silicon oil on a culture plate.

The team noticed that after exposing the cells to 10MhZ signals for 10 minutes daily for five days, the markers indicating the bone conversion appeared.

We can use the sound waves to apply just the right amount of pressure in the right places to the stem cells, to trigger the change process, said Leslie Yeo, co-lead researcher on the study. Our device is cheap and simple to use, so could easily be upscaled for treating large numbers of cells simultaneously vital for effective tissue engineering.

This discovery, detailed in the journalSmall, eliminates the need of drugs to make stem cells behave this way. Moreover, the MSCs can be pulled from a variety of places, like fast tissue, not just bone marrow.

By injecting them into the body in case of an injury or disease, they can start working on a new bone faster and more efficient.

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Maryland Cancer Moonshot Initiative Promises $216 Million for Research and Treatment Conduit Street – Conduit Street

Maryland will be committing $216 million to expand and accelerate cancer detection, screening, prevention, treatment, and research through Governor Larry Hogans recently announced Maryland Cancer Moonshot Initiative.

Governor Larry Hogan explained the personal significance of the initiative in a press release:

The reality is that cancer is a disease that has touched nearly every one of us, through family or loved ones, saidGovernor Hogan. On the day I found out I was cancer-free, I pledged that as long as I am governor and long after, I will stand with all those who are fighting this terrible disease. That is why today, I am announcing the Maryland Cancer Moonshot, to dramatically accelerate all of our efforts to detect, prevent, treat, and find a cure for cancer, so that more lives can be saved. This is a watershed moment in the fight against cancer in our state and the region.

The substantial initial investment is a part of Governor Hogans fifth supplemental budget and will include funding for the following:

Greenebaum Cancer Center:$100 million for the expansion of the University of Maryland Medical Systems Greenebaum Comprehensive Cancer Center (UMGCCC) in downtown Baltimore to providestate-of-the-art inpatient and outpatient cancer services. UMGCCC, which is a National Cancer Institute-designated comprehensive cancer center, treats approximately 3,000 new patients annually. This investment completes the states commitment to the project.

Prince Georges Comprehensive Cancer Center:$67 million to fully fund the construction of a new comprehensive cancer center on the campus of the newUniversity of Maryland Capitol Region Medical Centerin Largo. This best-in-class cancer will be a premiere clinical and research center to serve the residents of Prince Georges County and the region. The state funding includes a $27 million commitment by the governor, a $13.5 million commitment by the Maryland Senate and a $26.5 million commitment by the Maryland House of Delegates.

Cancer Research:$25 million for the University of Maryland School of Medicine and Johns Hopkins University to accelerate cancer research projects.

Pediatric Cancer Research:$1 million to support expanding pediatric cancer research at the University of Maryland School of Medicine.

Stem Cell Research Fund:$20.5 million for the Maryland Stem Cell Research Fund (MSCRF) to catalyze investment in regenerative medicine projects to develop novel cures and groundbreaking treatments for prevalent cancers.

Maryland Tech Council:$2.5 million for the BioHub Maryland Initiative to expand the states life sciences and biotechnology research workforce, with a focus on talent development, upskilling opportunities, and outreach to students in underserved communities. Maryland is proud to be home to one of thetop biotech clustersin the United States.

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Maryland Cancer Moonshot Initiative Promises $216 Million for Research and Treatment Conduit Street - Conduit Street

Orchard Therapeutics Extends Runway into 2024, Focusing HSC Gene Therapy Platform Exclusively on Severe Neurometabolic Diseases and Research Platform…

Libmeldy European launch momentum building with multiple MLD patients treated and strong recognition of value proposition; U.S. BLA filing of OTL-200 on track for late 2022 / early 2023

Broad research platform presents opportunities for larger indications and partnerships; preclinical POC data in NOD2 Crohns disease expected by year end and IND filing planned in 2024

Plan to seek strategic alternatives for primary immunodeficiency programs, including OTL-103 in WAS

Refined portfolio and 30% proposed workforce reduction extend cash runway into 2024

BOSTON and LONDON, March 30, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced its intention to focus its hematopoietic stem cell (HSC) gene therapy platform exclusively on severe neurometabolic diseases and early research programs while also reporting its financial results for the quarter and year ended December 31, 2021. These actions are intended to extend the companys cash runway into 2024 and focus operations on the highest value programs in its portfolio.

Moving forward, Orchard will continue its investment in Libmeldy (atidarsagene autotemcel) / OTL-200 for metachromatic leukodystrophy (MLD) to help sustain recent commercial momentum in Europe, as well as to support regulatory and future commercial activities for a potential U.S. approval and launch. The company also will continue to advance clinical development of OTL-203 for mucopolysaccharidosis type I Hurlers syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA). The focus on these neurometabolic programs is expected to allow Orchard to leverage the clinical validation of HSC gene therapy demonstrated with Libmeldy and capture significant commercial synergies, especially given the immense unmet needs in these diseases. Promising early-stage research programs that apply the HSC gene therapy approach in NOD2 Crohns disease, hereditary angioedema (HAE) and progranulin mutated frontotemporal dementia (GRN-FTD) also will remain an important part of the portfolio going forward given their promise in larger indications and as a possible source of future partnerships.

In light of our experiences and knowledge gained in this current and rapidly evolving market environment for gene therapy, our plan is to concentrate resources on programs that have the potential to make a remarkable difference to patients while also providing sustainable value to the business to enable the achievement our long-term vision, said Bobby Gaspar, M.D., Ph.D., chief executive officer. As launch momentum for Libmeldy continues to build in Europe and we prepare for a regulatory filing in the U.S., a focused strategy that utilizes a common infrastructure for future neurometabolic disease launches is critical to our success as a commercial gene therapy company.

Latest Key Milestones

To advance its portfolio of gene therapies for neurometabolic disorders and investigate future applications for the HSC approach, Orchard has provided an updated list of expected milestones:

Libmeldy Recent Highlights

Portfolio and Organizational Updates

To support the companys refined strategic focus and provide runway extension into 2024, Orchard intends to discontinue its investment in and seek alternatives for its programs in rare primary immune deficiencies. These include OTL-103 for the treatment of Wiskott-Aldrich syndrome (WAS), OTL-102 for X-linked chronic granulomatous disease (X-CGD) and Strimvelis, a gammaretroviral vector-based gene therapy approved in Europe for adenosine deaminase severe combined immunodeficiency (ADA-SCID).

Regarding the regulatory status of the OTL-103 program in the U.S., Orchard recently received written feedback from the FDA. The company believes the path to a potential BLA filing may require additional time and further investment.

Gaspar continued, We recognize the significant need that persists for many patients suffering from these rare diseases of the immune system, and we sympathize with the individuals, families and healthcare providers affected by these announcements, as well as our clinical partners and colleagues who worked so hard to advance these programs. These therapies have shown the potential for significant benefit for many patients treated in the clinical studies and we will continue to look for alternative ways to advance them, which could include commercial partnerships.

As a result of these updates, the company has proposed to reduce its current workforce by approximately 30%, which will result in a restructuring charge in 2022. Collectively, the actions announced today are expected to extend the companys existing cash runway into 2024.

Fourth Quarter 2021 Financial Results

Research and development expenses were $23.3 million for the three months ended December 31, 2021, compared to $22.6 million in the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and milestone payments under the companys agreements with third parties, and personnel costs to support these activities. The company expects R&D expenses to decline beginning in the second quarter of 2022 due to the portfolio updates and workforce reduction announced today as well as the completion of activities to support the OTL-200 BLA submission.

Selling, general and administrative expenses were $13.6 million for the three months ended December 31, 2021, compared to $16.2 million in the same period in 2020. The decline from 2020 resulted primarily from lower cash and share-based personnel costs to align with the current filing timelines and commercialization plans. In 2022, the company expects SG&A expenses to decline from 2021 due to the workforce reduction announced today, partially offset by increasing commercialization expenses to support Libmeldy, including preparations for a potential U.S. launch in 2023.

Net loss was $36.4 million for the three months ended December 31, 2021, compared to $ 33.6 million in the same period in 2020. The company had approximately 125.7 million ordinary shares outstanding as of December 31, 2021.

Cash, cash equivalents and investments as of December 31, 2021, were approximately $220.1 million, with $33.0 million of debt outstanding, compared to $191.9 million and $25.0 million of debt outstanding as of December 31, 2020. Following the actions announced today, the company now expects that its existing cash, cash equivalents and investments will fund its anticipated operating and capital expenditure requirements into 2024.

Conference Call & Webcast Information

Orchard will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss the updates to its business strategy. The conference call will be broadcast live in listen-only mode under "News & Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com, and a replay will be archived on the Orchard website following the presentation. To ask a question, please dial (866) 987-6504 (U.S. domestic) or +1 (602) 563-8620 (international) and refer to conference ID 9445456. Please dial in at least 15 minutes in advance to ensure a timely connection to the call.

About Libmeldy / OTL-200

Libmeldy (atidarsagene autotemcel), 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 of Libmeldy, 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 approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the U.S.

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

About Orchards Investigational Primary Immune Deficiency Portfolio

Primary immune deficiencies (PIDs) are a group of rare, genetic disorders in which the immune system does not function properly, leading to frequent infections and other disease manifestations that can be life-threatening. Orchards PID portfolio includes HSC gene therapies in development for the treatment of Wiskott Aldrich syndrome (WAS), X-linked chronic granulomatous disease (X-CGD), and Adenosine deaminase severe combined immunodeficiency (ADA-SCID). More than 100 PID patients have received one of Orchards investigational gene therapy products, with 11 years follow-up in the earliest treated patients. The majority of patients experienced favorable clinical outcomes and there was no evidence of monoclonal expansion, leukoproliferative complications or emergence of replication competent lentivirus.

About Strimvelis

Strimvelis(autologous CD34+enriched cell fraction that contains CD34+cells transduced with retroviral vector that encodes for the human ADA cDNA sequence)is a gammaretroviral vector-based gene therapy approved by the European Medicines Agency (EMA) in 2016. It was the firstex vivoautologous gene therapy approved by the EMA. Strimvelishas not been approved by the U.S. Food and Drug Administration (FDA).

Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)- matched related stem cell donor is available. Strimvelis is intended solely for autologous use and must be given in a specialized hospital by a doctor who is experienced in treating patients with ADA-SCID and in using this type of medicine.

Serious adverse reactions include autoimmunity (e.g., autoimmune hemolytic anemia, autoimmune aplastic anemia, autoimmune hepatitis, autoimmune thrombocytopenia and Guillain-Barr syndrome). The most commonly reported adverse reaction was pyrexia.

For more information about Strimvelis, please see the EU Summary of Product Characteristics available on theEMA website.

About Orchard Therapeutics

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company 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. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard Therapeutics

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.com/orchard_tx and http://www.linkedin.com/company/orchard-therapeutics), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, 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. 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, the therapeutic potential of Orchards products and product candidates, including the products and product candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, the likelihood of approval of such product candidates by the applicable regulatory authorities, the size of the potential markets for Libmeldy and Orchards other product candidates, the expected benefits to Orchards business as a result of the organizational updates referred to in this release, the adequacy of the companys manufacturing capacity and plans for future investment, and the companys financial condition and cash runway into 2024. 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: that the cost of discontinuing or partnering programs may be higher than expected; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings; 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 risk of delays in Orchards ability to commercialize its product candidates, if approved; the risk that the ongoing and evolving COVID-19 pandemic could affect the company's business; and the risk that the market opportunity for Libmeldy and its other product candidates may be lower than estimated. 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 most recent annual or quarterly report filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. 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.

Condensed Consolidated Statements of Operations Data (In thousands, except share and per share data) (Unaudited)

Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited)

Contacts

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

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Extends Runway into 2024, Focusing HSC Gene Therapy Platform Exclusively on Severe Neurometabolic Diseases and Research Platform...