Research Efforts Seek to Further Explore the Potential of Uproleselan in AML – OncLive

The addition of the E-selectin antagonist uproleselan (GMI-1271) to chemotherapy has been shown to improve outcomes in patients with relapsed/refractory acute myeloid leukemia (AML), according to Tapan M. Kadia, MD, who added that based on these findings, the investigative agent is now under further exploration in several disease subsets and settings with varying unmet need.

The idea is that [uproleselan] may reduce or subvert chemotherapy resistance. This has been shown in several preclinical studies where mice that had been treated with cytarabine and had leukemic blasts left over after [treatment] showed that they had tight binding to E-selectin within the tumor microenvironment, Kadia explained. When uproleselan, or an antibody blocking E-selectin, was added, those cells then became sensitive to the cytarabine, suggesting that the E-selectin binding was leading to chemotherapy resistance. This [supported the hypothesis that] blocking E-selectin within the microenvironment can be an important mechanism to provide benefit in patients with AML.

Data from a phase 1/2 clinical trial (NCT02306291) showed that when uproleselan was administered at the recommended phase 2 dose of 10 mg/kg twice daily in combination with mitoxantrone, etoposide, and cytarabine (MEC), it produced a remission rate of 41% in those with relapsed/refractory disease (n = 47).1 In a cohort of patients with newly diagnosed disease who were at least 60 years of age (n = 25), the combination of uproleselan plus cytarabine and idarubicin (7+3) resulted in a remission rate of 72%.

Now, a phase 3 trial (NCT03616470) is examining MEC or fludarabine, cytarabine, and idarubicin (FAI) with or without uproleselan in patients with relapsed/refractory AML who are eligible for intensive chemotherapy in the salvage setting.2 Another phase 3 trial (NCT03701308) is exploring 7+3 chemotherapy with or without uproleselan in patients aged 60 years or older who are fit for intensive induction chemotherapy.3 Moreover, a phase 1/2 trial (NCT04848974) is evaluating cladribine and low-dose cytarabine in combination with uproleselan in difficult-to-treat patients with treated secondary AML.4

In an interview with OncLive, Kadia, an associateprofessor in the Department of Leukemia, of the Division of Cancer Medicine, at The University of Texas MD Anderson Cancer Center, discussed what makes uproleselan unique from other agents under investigation in AML and shed light on the many research efforts dedicated to further exploring its use in this disease.

Kadia: E-selectin is a relatively new target, but it is a protein that we have known about for many years. E-selectin is present on activated endothelial cells, [which are] the cells that make up a blood vessel. E-selectin is present, upregulated, and overexpressed in activated endothelial cells at the sites of inflammation and damage.

We [believe] E-selectin is meant to help attract or traffic leukocytes and white blood cells, including monocytes, neutrophils, and natural killer [NK] cells, to the sites of inflammation. Neutrophils, NK cells, and monocytes have E-selectin ligands, which are glycoproteins that are attracted to or attached to E-selectin. E-selectin on the endothelial cells helps to traffic these cells and adhere them to the endothelial cells.

More recently, E-selectin has become recognized as a potentially important marker in malignancy, because they are also expressed in endothelial cells associated with malignancy. For example, in solid tumors, there is a suggestion that it may have a role in metastasis or distant metastasis of solid tumors, such as colon cancer.

In leukemia and hematologic malignancies, the endothelial cells in bone marrow also overexpress E-selectin, particularly in advanced disease. They are expressed at higher levels in patients who have adverse-risk AML, patients who have been previously treated, and they allow the adherence of leukemic blasts of the malignant cells to the endothelial microenvironment within the bone marrow.

As [the endothelial cells do that], we believe that the E-selectin binding to these cells upregulates nuclear factor kappa B [NF-B] within the tumor or the blast, and elicits a type of chemotherapy resistance, or prosurvival pathways, that allow them to survive chemotherapy or treatment. Blocking this [from happening] has been the rationale behind [the development of] uproleselan. Blocking this may help prevent the trafficking of the blast cells to the bone marrow and from adhering to the bone marrow microenvironment, therefore inhibiting activation of the cancer survival pathways, such as NF-B.

Uproleselan is an antagonist of E-selectin that binds to E-selectin and prevents the interaction between E-selectin and E-selectin ligands, which are present on leukemia or AML blasts. It is an intravenous product that is given over 20 minutes twice daily.

[During] an initial study, [investigators] observed no significant toxicities [with uproleselan] as a single agent. The mechanism is that it blocks the interaction between the E-selectin and the E-selectin ligands on the blasts, therefore reducing the trafficking of these leukemic blasts to the bone marrow. It disrupts the adhesion-mediated drug resistance within the bone marrow microenvironment.

In that respect, it also inhibits the activation of potential cancer survival mechanisms, such as upregulation of NF-B, and may reduce chemotherapy-based toxicity that may occur. By reducing [E-selectin adhesion] and adding chemotherapy, you are treating cells that are potentially more sensitive to [chemotherapy].

The [hypothesis] was that blocking E-selectin would thereby sensitize the resistant leukemic blasts to chemotherapy, particularly in the salvage setting. You want to start in patients with relapsed/refractory AML.

This was a phase 1 study that looked at the combination of the E-selectin antagonist, uproleselan, with either MEC chemotherapy in patients with relapsed/refractory AML, or in combination with 7+3 chemotherapy in a small cohort of newly diagnosed patients with AML who were aged 60 years and older. Once patients achieved remission, they could also get uproleselan with their consolidation, whether it be MEC consolidation or intermediate-dose cytarabine-based consolidation.

A total of 66 patients with relapsed/refractory AML were treated, with a median age of 59 years of age. Moreover, 17% of those patients had prior transplant, and one-third of the patients had 2 or more induction regimens; [as such, it was] a heavily pretreated population. Fifty percent of patients had adverse-risk [disease] by European LeukemiaNet risk [classification].

If you look at the adverse [effects (AEs)], and this is 1 of the first striking observations, there may have been potentially lower toxicityparticularly along the gastrointestinal tract starting with mucositis, nausea, and vomitingthan what you would expect with MEC chemotherapy based on historical experience. The most common complications were infections, which are common in patients [with leukemia] who are treated with intensive chemotherapy.

When you look at efficacy among the 66 patients who were treated, the complete response [CR]/CR with incomplete count recovery [CRi] rate was [41%], and the early mortality [rate] was fairly low, at 9% at 60 days, which is reasonable. Patients who had a longer CR1 duration had a higher response rate at 75% vs those who had refractory disease or a short CR1 duration, [with] response rates in the range of 23% and 36%.

[Additionally], 69% of patients had minimal residual disease [MRD] negativity, which is good for a relapsed/refractory cohort setting. The efficacy was there, as seen by the overall response rate [ORR] of 39%, which is in line with what you would expect with salvage chemotherapy in the relapsed/refractory setting. The median overall survival [OS] of the patients is [8.8] months, [which is] promising for a study looking at relapsed/refractory AML.

One of the interesting correlative studies looked at E-selectin ligand expression on the blast cells and survival. Looking at baseline AML, a prior study suggested that patients whose blasts had high expression of E-selectin ligand had a more adverse prognosis then those with low expression. Moreover, E-selectin ligand overexpression [is known to] correlate with relapsed/refractory disease and adverse prognosis disease. As such, high E-selectin ligand is associated with a poor prognosis.

[However, in this correlative study,] patients who had high E-selectin ligand expression and were treated with uproleselan had a more favorable outcome, with a median OS of 12.7 months compared with 5.2 months in those who had low [E-selectin ligand] expression. That suggests that in those patients who typically would have a more adverse prognosis with high E-selectin ligand expression, when you added uproleselan, which blocked that interaction, their prognosis improved. That was an early signal that suggested that targeting that receptor flips the adverse prognosis associated with E-selectin ligand expression.

[The phase 1/2] study also had an arm of newly diagnosed patients, who were treated with 7+3 chemotherapy plus [uproleselan]. These were older patients with newly diagnosed AML; [this cohort was comprised of] 25 patients who had a median age of 67 years. Half of patients had secondary AML, which is commonly seen in that population.

Here, the rates of grade 3 or 4 mucositis were 0%, with about 20% [of patients experiencing] grade 1/2 mucositis, so lower rates in mucositis than we may have expected with intensive chemotherapy. The CR/CRi rate was 72% [with this approach], with 52% [of patients] achieving a complete remission. The early mortality [rate] at 60 days was 12%, [which is] higher than you might expect in older patients, but still reasonable and promising. The MRD negativity [rate] was 56% among the patients who were evaluated for it. As such, this was a pretty good response rate that was in line or higher than what you would expect with intensive chemotherapy.

Based on the promising data from the phase 1 trial, looking at patients with relapsed/refractory AML treated with MEC plus uproleselan, as well as the small cohort of frontline patients treated with uproleselan and 7+3, the sponsor decided to proceed with a couple of phase 3 randomized trials to register uproleselan for patients in these particular settings.

The primary end point for both studies is OS, to evaluate the combination of anti-leukemic activity uproleselan with the respective chemotherapy. Secondary end points also include trying to further study and nail down the incidence of severe oral mucositis. Is it less than what you would expect with the control arm?

The first is a randomized phase 3 study [NCT03616470] for patients between the ages of 18 years and 75 years, with relapsed/refractory AML who are eligible for intensive chemotherapy in the salvage setting. They may have had 1 or fewer allogeneic stem cell transplants [ASCTs] prior to enrollment. Patients are randomized [1:1] to either MEC or FAI chemotherapy, plus or minus uproleselan. If patients achieved remission, they could receive consolidation with high-dose [cytarabine] or intermediate-dose [cytarabine], plus or minus uproleselan. The primary end point of the study is OS. The study is in the early stages [and we] hope to see data in the next couple of years.

The second is an National Cancer Institute study [NCT03701308] that is looking at patients aged 60 years or older who are fit for intensive chemotherapy [in the frontline setting]. Patients who have secondary AML [will be included], but those with FLT3-mutated AML [will not], since there is a standard of care for that [in the form of] FLT3 inhibitors.

Here, patients are randomized [1:1] to 7+3 chemotherapy with or without uproleselan, with consolidation with intermediate-dose cytarabine, with or without uproleselan. The primary end point [is] OS, and there will be an interim analysis looking at event-free survival [EFS]. If there is an inferior EFS at the interim [analysis], then the study would be closed at that point for futility. Otherwise, it would continue to look for OS benefit [with this approach]. Hopefully, we will see some data in the next year or 2 [to shed light on whether] this is a good strategy [for these pateints].

The treatment paradigm in AML has shifted significantly over the past few years with the incorporation of new molecules, such as venetoclax [Venclexta], [plus] IDH1, IDH2, and FLT3 inhibitors. Things are changing rapidly, even as uproleselan is being developed.

Now, instead of saying we have patients who are older and fit for chemotherapy, you must ask [questions about mutations]. Does a patient have a FLT3 mutation? If so, maybe they should be treated with a FLT3 inhibitor combined with chemotherapy. Does a patient have an IDH1 or IDH2 mutation? Recent data from the 2021 ASH Annual Meeting suggested that the combination of ivosidenib [Tibsovo] and azacitidine showed a significant survival benefit in patients who are IDH1 mutated compared with azacitidine alone. As such, there is another option for that specific subset of patients.

We have other medications or intensive chemotherapy for patients who have secondary AML. [For example,] CPX-351 showed a significant survival benefit compared with 7+3 chemotherapy. Where does uproleselan fit in secondary AML? Well, if you start with the relapsed/refractory setting, there is no 1 standard of care. As such, if uproleselan does show significant benefit compared with MEC alone in terms of survival, that is one place to go.

[If patients] have FLT3-, IDH1-, or IDH2-mutated, options such as gilteritinib [Xospata] and ivosidenib are available for those respective subtypes. However, in those patients who do not have those mutations, [uproleselan] could be an option.

A [phase 1/2] pilot study [NCT03214562] that is being done by [investigators at The University of MD Anderson Cancer Center] looked at [the combination of] FLAG [fludarabine, cytarabine, granulocyte colonystimulating factor] plus idarubicin and venetoclax [in patients with relapsed/refractory AML] and showed very high rates of complete remission with MRD negativity. This is a very intensive study, that needs close follow-up and close safety evaluation, but certainly, [we are seeing] high response rates with most of the patients able to proceed to ASCT and good survival in the long term. How does uproleselan fit in that setting?

If [the addition of uproleselan] shows a benefit over MEC as a single agent, it is certainly an option. [Now, we must determine] which patients you would put on that particular study, if they have no targetable mutations or if they cannot tolerate intensive chemotherapy plus venetoclax, whether it be FLAG plus idarubicin/venetoclax, or a regimen we developed, [like CPX-351] plus venetoclax.

In the frontline setting, it gets even more difficult because frontline studies are looking at [combining] a hypomethylating agent [HMA] with venetoclax in older patients. This [approach] is currently approved for patients who are aged 75 and older, or those who are unfit for intensive chemotherapy. However, [this approach] may start to be applied to patients who are slightly younger than that or who are more fit than the most unfit patients. [Investigators] are examining HMA plus venetoclax in that older, fit population. New regimens, such as cladribine, low-dose cytarabine, plus venetoclax, have also demonstrated high response rates in that older, fit population.

A set of studies is evaluating [CTX-351 in secondary AML]. For patients with IDH1 mutations, we now have the option of HMA plus ivosidenib. For FLT3-mutated disease, we are still looking, but HMA/venetoclax has high response rates in that setting. Moreover, triplet combinations are also being investigated, where [agents such as] gilteritinib or quizartinib are being added to the backbone of HMA plus venetoclax.

In the frontline, so many different options [are available] for specific subtypes, so we must define where 7+3 plus uproleselan will fit in, if data are positive. This is still a question that will need to be answered.

We are conducting a trial in a specific subset of patients who do not have great options [available to them] right now: those with treated secondary AML. This is a population of patients who may have had myelodysplastic syndrome [MDS] or chronic myelomonocytic leukemia [CMML] prior to developing AML, which is very common in the population. These patients were treated with the standard of care, which is HMAs and 5-azacytidine or decitabine in the frontline for MDS or CMML.

Eventually, these patients may respond [to treatment], but they may then progress to AML. At the time of their progression, they are considered to have newly diagnosed AML, but they may have received months or years of HMAs. This [scenario] used to be [referred to as] HMA failure, but this is a specific subset of AML that arises from previously treated MDS or CMML. In these patients, the complete remission rates are in the range of 20% to 25% with standard agents, and early mortality is very high. These patients have a median OS in the range of 4 to 5 months at the time of diagnosis AML, so it is a difficult subset of patients [to treat] for whom there really is no therapy [available]. If you look at CPX-351 in that setting, which is treated secondary AML, outcomes are pretty much the same, with high rates of early mortality and poor OS.

We wanted to address this key subset of patients. One of the things that we learned from the preclinical studies with uproleselan is that E-selectin is upregulated and overexpressed in AML blasts that have been previously exposed to HMAs. AML or MDS blasts that have been treated with or exposed to HMAs upregulate E-selectin significantly. The rationale was if these patients who have failed or have been treated extensively with HMAs then develop AML, their blasts may have upregulated E-selectin, and they may be the ideal target for uproleselan in combination with chemotherapy.

We took that specific subset of patients, and we are studying the combination of uproleselan plus cladribine and low-dose cytarabine [as part of a phase 1/2 trial (NCT04848974)]. The cladribine and low-dose cytarabine regimen has been developed at MD Anderson and, for many years now, has been used in frontline AML and treated secondary AML. In that specific subset [of treated secondary AML], we have seen a response rate [ranging from] 35% to 40% in the frontline [setting].

Since it is not [additional treatment with a] HMA, this backbone in combination with uproleselan is being studied in patients with treated secondary AML, with the end point of safety, [as well as] secondary end points of remission rate and OS in this difficult population, where there is a [need] that needs to be critically addressed.

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Research Efforts Seek to Further Explore the Potential of Uproleselan in AML - OncLive

FDA Grants Direct Biologics Regenerative Medicine Advanced Therapy (RMAT) Designation for the use of ExoFlo in COVID-19 Related ARDS USA – English -…

AUSTIN, Texas, April 13, 2022 /PRNewswire/ -- Direct Biologics, an innovative biotechnology company with a groundbreaking extracellular vesicle (EV) platform drug technology, announced that the U.S. Food and Drug Administration (FDA) has awarded their EV drug product ExoFlo with a Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of Acute Respiratory Distress Syndrome (ARDS) associated with COVID-19. The RMAT program is designed to expedite the approval of promising regenerative medical products in the US that demonstrate clinical evidence indicating the ability to address an unmet medical need for a serious life-threatening disease or condition. Under the RMAT designation, the FDA provides intensive guidance on drug development and post-market requirements through early and frequent interactions. Additionally, an RMAT confers eligibility for accelerated approval and priority review of biologics licensing applications (BLA).

"After intensively reviewing our preclinical data, manufacturing processes, and clinical data from our Phase II multicenter, double blinded, placebo controlled randomized clinical trial, the FDA has recognized ExoFlo as a lifesaving treatment for patients suffering from Acute Respiratory Distress Syndrome (ARDS) due to severe or critical COVID-19," said Mark Adams, Chief Executive Officer. "The additional attention, resources, and regulatory benefits provided by an RMAT designation demonstrate that the FDA views ExoFlo as a product that can significantly enhance the standard of care for the thousands still dying from ARDS every week in the US," he said.

"We are very pleased that the FDA has recognized the lifesaving potential of our platform drug technology ExoFlo. The RMAT has provided a pathway to expedite our drug development to achieve a BLA in the shortest possible time," said Joe Schmidt, President. "I am very proud of our team. Everyone has been working around the clock for years in our mission to save human lives taken by a disease that lacks treatment options, both in the US and abroad. We are grateful for the opportunity to accelerate development of ExoFlo under the RMAT designation as it leads us closer to our goal of bringing our life saving drug to patients who desperately need it."

ExoFlo is an acellular human bone marrow mesenchymal stem cell (MSC) derived extracellular vesicle (EV) product. These nanosized EVs deliver thousands of signals in the form of regulatory proteins, microRNA, and messenger RNA to cells in the body, harnessing the anti-inflammatory and regenerative properties of bone marrow MSCs without the cost, complexity and limitations of scalability associated with MSC transplantation. ExoFlo is produced using a proprietary EV platform technology by Direct Biologics, LLC.

Physicians can learn more and may request information on becoming a study site at clinicaltrials.gov. For more information on Direct Biologics and regenerative medicine, visit: https://directbiologics.com.

About Direct BiologicsDirect Biologics, LLC, is headquartered in Austin, Texas, with an R&D facility located at the University of California, and an Operations and Order Fulfillment Center located in San Antonio, Texas. Direct Biologics is a market-leading innovator and cGMP manufacturer of regenerative medical products, including a robust EV platform technology. Direct Biologics' management team holds extensive collective experience in biologics research, development, and commercialization, making the Company a leader in the evolving segment of next generation regenerative biotherapeutics. Direct Biologics has obtained and is pursuing multiple additional clinical indications for ExoFlo through the FDA's investigational new drug (IND) process. For more information visit http://www.directbiologics.com.

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Cell Therapy Market is Expected to Grow by USD 20.2 Billion Progressing at a CAGR of 14.5% By Forecast 2027 – Digital Journal

TheGlobal Cell Therapy Marketwas worth USD7.8 billion in 2021, according to a new analysis released by Maximize Market Research, and is expected to rise to USD 20.2 billion by 2027, with a CAGR of 14.5% percent over the forecast period. From the markets perspective, its ever-changing trends, industrial environment, existing market features, and the current short-term and long-term influence on the market

the research will aid decision-makers in developing the outline and strategies for organisations by region.

The implantation of a human cell to replace or repair damaged tissue or cells is known as cellular therapy. Therapy typically consists of live cells that are safely injected, implanted, or grafted into the patients body to have a therapeutic effect. T-cell and stem cell treatment are two types of cell therapy that are used to combat cancer via cell mediated immunity or to repair damaged tissues. For patients with long-term ailments, cell therapy has become a popular treatment option.

Cell Therapy Market Scope and Dynamics:

The Maximize Market Research report contains a detailed study of factors that will drive and restrain the growth of the Cell Therapy Market Globally. Significant advances in cell therapy, a growing emphasis on regenerative medicine, increased R&D activities in the life sciences sector to develop advanced cellular therapies, and the rising prevalence of cancer, musculoskeletal disorders, cardiovascular diseases, autoimmune disorders, and neurological diseases are all driving the global cell therapy market revenue growth.

Other significant factors driving global market revenue growth include increased awareness and commercialization of stem cell therapies, an increase in the number of clinical trials of new cell therapies, an increase in the use of human cells in cell therapy research and development, and an increase in cellular therapy manufacturing under Good Manufacturing Practices (GMP) supervision. Increased government investments in the healthcare industry, as well as increased collaborations between pharmaceutical and biotechnology behemoths and leading research institutes for the development of advanced cellular therapies for cancer, cardiovascular disease, and other severe chronic diseases, are expected to boost global cell therapy market revenue growth in the coming years.

The Impact of COVID-19 on the Cell Therapy Market:

The COVID-19 pandemic has impacted the majority of biopharmaceutical companies, but several cellular treatment development companies have seen a significant negative impact, which can be related to logistical issues as well as the manufacturing models used in this field. Furthermore, large and reliable funding is required to ensure successful commercial translation of cell-based medicines, a factor that was negatively impacted in 2020, affecting market growth even more.

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Cell Therapy Market Region Insights:

Regional analysis is another highly comprehensive part of the research and analysis study of the global Cell Therapy Market presented in the report. The global cell treatment market is dominated by North America. In 2020, North America held a total market share of 14.5%percent, as new institutions and institutes invest in R&D to propel cell therapy forward. Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Harvard Stem Cell Institute, and Yale Steam Cell Center are among the main universities in the United States that are involved in new research in cell regenerative technologies. During the forecasted period, Asia Pacific is expected to increase at a significant rate of 14.5%percent. China, South Korea, and Japan are spending extensively in regenerative medicine and stem cell therapies. Certain government-funded institutes are devoted to R&D with the goal of pushing the market forward.

What does the report include?

The study on the Global Cell Therapy Market includes qualitative characteristics such as drivers, constraints, and opportunities . The research looks at the current and future rivals in the Global Cell Therapy Market, as well as their product development strategies. The study examines the market in both qualitative and quantitative terms, and it is separated into three segments: component, deployment type, organisation size, and industry. Furthermore, the report provides comparable statistics for the key regions. For each of the above-mentioned segments, actual market sizes and predictions have been presented.

Cell Therapy Market Segmentation:

Global Cell Therapy Market, by Therapy Type:

Autologous Allogeneic

Global Cell Therapy Market, by Cell Type:

T-Cell Stem Cell

Global Cell Therapy Market, by Application:

Malignancies Musculoskeletal Disorder Autoimmune Disorder Dermatology Others

Global Cell Therapy Market, by End User:

Hospitals and clinics Academics and Research Institutes

By Region:

North America Europe Asia Pacific South America Middle East and Africa

Key Players in Cell Therapy Market:

Kolon TissueGene Inc. Anterogen Co. Ltd. JCR Pharmaceuticals Co., Ltd. Castle Creek Biosciences, Inc. The Future of Biotechnology, MEDIPOST Osiris Therapeutics, Inc. PHARMICELL Co., Ltd Tameika Cell Technologies, Inc. Cells for Cells NuVasive, Inc. Vericel Corporation Celgene Corporation Thermo Fisher Scientific Inc. Merck KGaA Danaher Corporation Becton, Dickinson, and Company Lonza Group Sartorius AG Terumo BCT Fresenius Medical Care AG & Co. KGaA

To Get a Detailed Report Summary and Research Scope of Cell Therapy MarketClick here @https://www.maximizemarketresearch.com/market-report/cell-therapy-market/126471/

About Maximize Market Research:

Maximize Market Research, a global market study firm with a dedicated team of specialists and data, has conducted thorough research on the Cell Therapy Market. Maximize Market Research is well-positioned to assess and predict market size while also taking into account the competitive landscape of the various industries. Maximize Market Research has a strong unified team of industry professionals and analysts across sectors to guarantee that the whole industry ecosystem, as well as current developments, new trends, and futuristic the technology effect of uniquely particular industries is taken into consideration.

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Cell Therapy Market is Expected to Grow by USD 20.2 Billion Progressing at a CAGR of 14.5% By Forecast 2027 - Digital Journal

Jasper Therapeutics Announces Management Changes to Strengthen Leadership Team – BioSpace

REDWOOD CITY, Calif., March 21, 2022 (GLOBE NEWSWIRE) --Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced changes to its management team, including the promotions of Jeet Mahal to the newly created position of Chief Operating Officer, and of Wendy Pang, M.D., Ph.D., to Senior Vice President of Research and Translational Medicine. Both promotions are effective as of March 21, 2022. Jasper also announced that a new position of Chief Medical Officer has been created, for which an active search is underway. Judith Shizuru, M.D. PhD, co-founder, and Scientific Advisory Board Chairwoman will lead clinical development activities on an interim basis and Kevin Heller, M.D., EVP of Research and Development, will be transitioning to a consultant role.

Based on the recent progress with JSP191, our anti-CD117 monoclonal antibody, as a targeted non-toxic conditioning agent and our mRNA hematopoietic stem cell program we have decided to advance Jaspers organizational structure with the creation of the roles of Chief Operating Officer and Chief Medical Officer and by elevating our research and translational medicine team to report directly to the CEO, said Ronald Martell, CEO of Jasper Therapeutics. We also are pleased that Dr. Shizuru will lead clinical development activities on an interim basis, a role she served during the companys founding in 2019.

These changes will allow us to advance our upcoming pivotal trial of JSP191 in AML/ MDS and execute on our pipeline opportunities with a best-in-class organization, continued Mr. Martell. We also wish to thank Dr. Heller for his help advancing JSP191 through our initial AML/MDS transplant study.

In the two plus years since we founded Jasper and received our initial funding, the company has been able to advance JSP191 in two clinical studies, develop our mRNA stem cell graft platform and publicly list on NASDAQ, said Dr. Shizuru, co-founder and member of the Board of Directors of Jasper Therapeutics. These changes will strengthen the companys ability to advance the field of hematopoietic stem cell therapies and bring cures to patients with hematologic cancers, autoimmune diseases and debilitating genetic diseases."

Mr. Mahal joined Jasper in 2019 as Chief Finance and Business Officer and has led Finance, Business Development, Marketing and Facilities/ IT since the companys inception. Prior to joining Jasper, he was Vice President, Business Development and Vice President, Strategic Marketing at Portola Pharmaceuticals, where he led the successful execution of multiple business development partnerships for Andexxa, Bevyxxaand cerdulatinib. He also played a key role in the companys equity financings, including its initial public offering and multiple royalty transactions. Earlier in his career, Mr. Mahal was Director, Business and New Product Development, at Johnson & Johnson on the Xareltodevelopment and strategic marketing team. Mr. Mahal holds a BA in Molecular and Cell Biology from U.C. Berkeley, a Masters in Molecular and Cell Biology from the Illinois Institute of Technology, a Masters in Engineering from North Carolina State University and an MBA from Duke University.

Dr. Pang joined Jasper in 2020 and has led early research and development including leading creation of the companys mRNA stem cell graft platform and playing a pivotal role in advancing JSP191 across multiple clinical studies. Previously Dr. Pang was an Instructor in the Division of Blood and Marrow Transplantation at Stanford University and the lead scientist in the preclinical drug development of an anti-CD117 antibody program. She was the lead author on the proof-of-concept studies showing that an anti-CD117 antibody therapy targets disease-initiating human hematopoietic (blood cell-forming) stem cells in myelodysplastic syndrome (MDS). She has authored numerous publications on the characterization of hematopoietic stem and progenitor cell behavior in hematopoieticdiseases, as well as hematopoietic malignancies, including MDS and acute myeloid leukemia (AML), and in hematopoietic stem cell transplantation. Dr. Pang earned her AB and BM in Biology from Harvard University and her MD and PhD in cancer biology from Stanford University.

Dr. Shizuru is a Professor of Medicine (Blood and Marrow Transplantation) and Pediatrics (Stem Cell Transplantation) at StanfordUniversity.She is the clinician-scientist co-founder of Jasper Therapeutics. Dr. Shizuru is an internationally recognized expert on the basic biology of blood stem cell transplantation and the translation of this biology to clinical protocols.Dr Shizuruis a member of the Stanford Blood and Marrow Transplantation (BMT) faculty, the Stanford Immunology Program, and the Institute for Stem Cell Biology and Regenerative Medicine. Shehas been an attending clinicianattendedon the BMT clinical service since 1997.Currently, she oversees a research laboratory focused on understanding the cellular and molecular basis of resistance to engraftment of transplantedallogeneic bone marrow blood stemcells and the way in which bone marrow grafts modify immune responses.Dr. Shizuru earned her BA from Bennington College and her MD and PhD in immunology from Stanford University

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 a hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. Jasper is also advancing JSP191 as a potential therapeutic for patients with lower risk Myelodysplastic Syndrome (MDS). Jasper Therapeutics is also advancing its preclinical mRNA hematopoietic stem cell graft 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,outlookandsimilarexpressionsthat 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 potentialof the Companys JSP191 and mRNA engineered stem cell graft programs. Thesestatementsarebasedonvariousassumptions,whetherornotidentifiedinthispressrelease, and on the current expectations of Jasper and are not predictions of actual performance. These forward-lookingstatementsareprovidedforillustrativepurposesonlyandarenotintendedtoserve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitivestatementoffactorprobability.Actualeventsandcircumstancesaredifficultorimpossible 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 prior study results may not be replicated; 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 beneficialtopatientsorsuccessfullycommercialized;patientswillingnesstotrynewtherapiesand 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 thatJaspers 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 foritsinvestigationalproductsorwillinfringetheintellectualpropertyprotectionofothers;andother risks and uncertainties indicated from time to time in Jaspers filings with the SEC. If any of these risksmaterializeorJaspersassumptionsproveincorrect,actualresultscoulddiffermateriallyfrom the results implied by these forward-looking statements. While Jasper may elect to update these forward-lookingstatementsatsomepointinthefuture,Jasperspecificallydisclaimsanyobligation to do so. These forward-looking statements should not be relied upon as representing Jaspers assessmentsofanydatesubsequenttothedateofthispressrelease.Accordingly,unduereliance should not be placed upon the forward-lookingstatements.

Contacts:

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

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

Originally posted here:
Jasper Therapeutics Announces Management Changes to Strengthen Leadership Team - BioSpace

Stem Cell Assay Market Size And Forecast | Top Key Players Thermo Fisher Scientific, Perkinelmer, Stemcell Technologies, Merck, Bio-Rad Laboratories,…

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Stem Cell Assay Market Size And Forecast | Top Key Players Thermo Fisher Scientific, Perkinelmer, Stemcell Technologies, Merck, Bio-Rad Laboratories,...

Sana Biotechnology Reports Fourth Quarter and Full Year 2021 Financial Results and Business Updates – BioSpace

Plans to present data at multiple scientific conferences in 2022

Expects to file INDs for leading CAR T ex vivo program, SC291, and in vivo program, SG295, in 2022

2021 year-end cash position of $746.9 million

SEATTLE, March 16, 2022 (GLOBE NEWSWIRE) -- Sana Biotechnology Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, today reported financial results and business highlights for the fourth quarter and year ended December 31, 2021.

We are pleased with the progress we are making in our pipeline and in building capabilities to execute our vision of exploiting the potential of engineered cells to treat a number of diseases that dont have effective treatments today, said Steve Harr, Sanas President and Chief Executive Officer. In 2021, we meaningfully strengthened our balance sheet, advanced our pipeline giving us the potential for two investigational new drug applications (INDs) in 2022 and multiple INDs per year going forward, built out our supply chain, including commercial access to gene-editing reagents and pluripotent stem cells, and commenced the build-out of our own manufacturing facility. Most importantly, we successfully attracted talent in key business areas, which, combined with the people already inside of the company, give us the capabilities, insights, focus, and dedication to reach our mission for patients.

Recent Corporate Highlights

Demonstrating forward progress in moving toward clinical trials for Sanas multiple platforms including Sanas ex vivo hypoimmune allogeneic CAR T, in vivo fusogen CAR T, and stem cell-derived programs:

Strengthened balance sheet and Board leadership; signed lease to add internal manufacturing capability

Fourth Quarter 2021 Financial Results

GAAP Results

Non-GAAP Measures

A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under Non-GAAP Financial Measures.

About Sana

Sana Biotechnology, Inc. is focused on creating and delivering engineered cells as medicines for patients. We share a vision of repairing and controlling genes, replacing missing or damaged cells, and making our therapies broadly available to patients. We are more than 380 people working together to create an enduring company that changes how the world treats disease. Sana has operations in Seattle, Cambridge, and South San Francisco.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements about Sana Biotechnology, Inc. (the Company, we, us, or our) within the meaning of the federal securities laws, including those related to the companys vision, progress, and business plans; expectations for its development programs, product candidates and technology platforms, including its pre-clinical, clinical and regulatory development plans and timing expectations; the potential use and utility of licensed technologies for Sanas programs; the potential ability to make hypoimmune-modified iPSCs that survive and evade the immune system without immunosuppression; the potential ability to make hypoimmune allogeneic CAR T cells that evade the immune system; the potential efficacy of CD19-targeted hypoimmune CAR T cells; the potential efficacy of a CD8 targeted fusosome containing a CD20-targeted CAR and of Sanas SG295 program; the potential efficacy of the NIHs CAR construct; the potential benefits of targeting both CD19 and CD22 with an off-the-shelf product, including in combination with Sanas hypoimmune or fusogen platform; the ability to make stem cell-derived pancreatic islet cells and hypoimmune pancreatic islet cells, and the function and efficacy of such cells; and the potential ability to eliminate engraftment arrythmias in hypoimmune-modified pluripotent cell-derived cardiomyocytes. All statements other than statements of historical facts contained in this press release, including, among others, statements regarding the Companys strategy, expectations, cash runway and future financial condition, future operations, and prospects, are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as aim, anticipate, assume, believe, contemplate, continue, could, design, due, estimate, expect, goal, intend, may, objective, plan, positioned, potential, predict, seek, should, target, will, would and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. The Company has based these forward-looking statements largely on its current expectations, estimates, forecasts and projections about future events and financial trends that it believes may affect its financial condition, results of operations, business strategy and financial needs. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. These statements are subject to risks and uncertainties that could cause the actual results to vary materially, including, among others, the risks inherent in drug development such as those associated with the initiation, cost, timing, progress and results of the Companys current and future research and development programs, preclinical and clinical trials, as well as the economic, market and social disruptions due to the ongoing COVID-19 public health crisis. For a detailed discussion of the risk factors that could affect the Companys actual results, please refer to the risk factors identified in the Companys SEC reports, including but not limited to its Annual Report on Form 10-K dated March 16, 2022. Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements for any reason.

Investor Relations & Media: Nicole Keith investor.relations@sana.com media@sana.com

Sana Biotechnology, Inc. Unaudited Selected Consolidated Balance Sheet Data

Sana Biotechnology, Inc. Unaudited Consolidated Statements of Operations

Sana Biotechnology, Inc. Changes in the Estimated Fair Value of Success Payments and Contingent Consideration

(1) Cobalt Biomedicine, Inc. (Cobalt) and the Presidents of Harvard College (Harvard) are entitled to success payments pursuant to the terms of their agreements. The success payments are recorded at fair value and remeasured at each reporting period with changes in the estimated fair value recorded in research and development related success payments and contingent consideration on the statement of operations. (2) Cobalt is entitled to contingent consideration upon the achievement of certain milestones pursuant to the terms of the agreement. Contingent consideration is recorded at fair value and remeasured at each reporting period with changes in the estimated fair value recorded in research and development related success payments and contingent consideration on the statement of operations.

Non-GAAP Financial Measures

To supplement the financial results presented in accordance with generally accepted accounting principles in the United States (GAAP), Sana uses certain non-GAAP financial measures to evaluate its business. Sanas management believes that these non-GAAP financial measures are helpful in understanding Sanas financial performance and potential future results, as well as providing comparability to peer companies and period over period. In particular, Sanas management utilizes non-GAAP operating cash burn, non-GAAP research and development expense and non-GAAP net loss and net loss per share. Sana believes the presentation of these non-GAAP measures provides management and investors greater visibility into the Companys ongoing actual costs to operate its business, including actual research and development costs unaffected by non-cash valuation changes and certain one-time expenses for acquiring technology, as well as facilitating a more meaningful comparison of period-to-period activity. Sana excludes these items because they are highly variable from period to period and, in respect of the non-cash expenses, provides investors with insight into the actual cash investment in the development of its therapeutic programs and platform technologies.

These are not meant to be considered in isolation or as a substitute for comparable GAAP measures and should be read in conjunction with Sanas financial statements prepared in accordance with GAAP. These non-GAAP measures differ from GAAP measures with the same captions, may be different from non-GAAP financial measures with the same or similar captions that are used by other companies, and do not reflect a comprehensive system of accounting. Sanas management uses these supplemental non-GAAP financial measures internally to understand, manage, and evaluate Sanas business and make operating decisions. In addition, Sanas management believes that the presentation of these non-GAAP financial measures is useful to investors because they enhance the ability of investors to compare Sanas results from period to period and allows for greater transparency with respect to key financial metrics Sana uses in making operating decisions. The following are reconciliations of GAAP to non-GAAP financial measures:

Sana Biotechnology, Inc. Unaudited Reconciliation of Change in Cash, Cash Equivalents, and Marketable Securities to Non-GAAP Operating Cash Burn

(1) The non-GAAP adjustment of $52.1 million for the twelve months ended December 31, 2021 consisted of the one-time upfront payment of $50.0 million to Beam to license its genome editing technology and holdback payments of $2.1 million related to the acquisitions of Cytocardia, Inc. in 2019 and Oscine Corp. in 2020. The non-GAAP adjustment of $7.7 million for the twelve months ended December 31, 2020 was the upfront payment related to the acquisition of Oscine Corp. in 2020. (2) The non-GAAP adjustment of $6.0 million for the twelve months ended December 31, 2020 was the payment of a contingent liability due to Harvard in connection with the closing of the Series B convertible preferred stock financing.

Sana Biotechnology, Inc. Unaudited Reconciliation of GAAP to Non-GAAP Research and Development Expense

(1) The non-GAAP adjustment of $8.5 million for the twelve months ended December 31, 2020 was the upfront expense recorded in connection with the acquisition of Oscine Corp. in 2020. (2) The contingent liability was recorded in connection with the Harvard license agreement and paid in June 2020.

Sana Biotechnology, Inc. Unaudited Reconciliation of GAAP to Non-GAAP Net Loss and Net Loss Per Share

(1) The non-GAAP adjustment of $8.5 million for the twelve months ended December 31, 2020 was the upfront expense recorded in connection with the acquisition of Oscine Corp. in 2020. (2) For the three and twelve months ended December 31, 2021, we recorded a gain related to the Cobalt success payment liability of $23.3 million and an expense of $23.6 million, respectively. For the three and twelve months ended December 31, 2020, we recorded expenses related to the Cobalt success payment liability of $27.1 million and $62.3 million, respectively. For the three and twelve months ended December 31, 2021, we recorded a gain related to the Harvard success payment liability of $8.4 million and an expense of $2.4 million, respectively. For the three and twelve months ended December 31, 2020, we recorded expenses related to the Harvard success payment liability of $4.4 million and $9.9 million, respectively. The expense and gain recorded in each period are due to changes in our market capitalization and common stock price during the relative periods. (3) The contingent consideration was recorded in connection with the acquisition of Cobalt. The change in value of the contingent consideration was primarily due to scientific progress toward the achievement of milestones during the relative periods. (4) The contingent liability was recorded in connection with the Harvard license agreement and paid in June 2020.

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Sana Biotechnology Reports Fourth Quarter and Full Year 2021 Financial Results and Business Updates - BioSpace

Current Strategies and the Potential of CAR T-Cell Therapy in Relapsed and Refractory MCL – AJMC.com Managed Markets Network

Mantle cell lymphoma is a difficult cancer type with high relapse rates, but novel targeted approaches such as CAR T-cell therapy hold promise for more successful response rates in the future.

First-line treatment strategies for mantle cell lymphoma (MCL) currently range from intensive chemotherapy and autologous stem cell transplant (ASCT) to combination regimens and novel targeted therapies. As chimeric antigen receptor (CAR) T-cell therapies change the treatment landscape in other hematological cancer types, a recent review sees potential for this novel strategy to improve outcomes for MCL.

MCL is a B-cell malignancy that is rare and challenging to treat, and relapse rates are high. In most cases of MCL, the chromosomal translocation t(11;14) causes overexpression of thecyclin D1 (CCND1) gene, although other mechanisms are also involved. Despite progress in identifying the pathogenesis and risk factors of MCL, there are still no curative treatments for it.

In the first-line setting, the current standard treatment for otherwise healthy younger patients is intensive immunochemotherapy, potentially followed by ASCT to improve response duration. Older patients who cannot tolerate intensive treatment typically undergo treatment with more tolerable combination regimens.

When patients relapse, targeted agents are generally used in lieu of the chemoimmunotherapy seen in first-line treatment. Initially, bortezomib, temsirolimus, and lenalidomide were the only approved targeted second-line treatments, but the current treatment landscape also includes agents such as Bruton tyrosine kinase (BTK) inhibitors, BCL2 inhibitors, lenalidomide, and venetoclax. Three BTK inhibitors ibrutinib, acalabrutinib, and zanubrutinib are currently approved for relapsed or refractory MCL.

Response rates have been promising with targeted therapies, but response durations are often limitedand even on these regimens, many patients relapse. In patients with known risk factors such as TP53aberrations, high Ki-67, or those whose disease progresses on BTK inhibition, treatment is even more challenging and novel approaches must be identified to improve outcomes.

In recent years, CAR T-cell therapy has emerged as a promising treatment option in hematological cancers, including B-cell lymphomas. Four CAR T-cell therapies targeting CD19 are currently approved for B-cell lymphomas: axicabtagene ciloleucel (axi-cel) is approved for diffuse large B-cell lymphoma (DLBCL) in the third-line setting, tisagenlecleucel (tisa-cel) is approved for relapsed and refractory DLBCL, lisocabtagene maraleucel (liso-cel) is approved for DLBCL, and brexucabtagene autoleucel (brexu-cel) is approved for relapsed or refractory MCL.

While research on CAR T-cell therapy is limited in MCL compared with other types of cancer, the review authors highlight 2 trials of brexu-cel and liso-cel in relapsed and refractory MCL.

In the phase 2 ZUMA-2 trial (NCT02601313) of brexu-cel, the first multicenter trial of CAR T-cell therapy in relapsed and refractory MCL, patients who had received 2 or more lines of therapy prior to brexu-cel were given a single infusion. It was highly active in the cohort used for efficacy analysis, with a 93% overall response rate (ORR) and 67% of patients achieving complete response (CR). In the overall cohort of 74 patients, the ORR was 85%, and 59% of patients achieved CR. At 17.5 months of follow-up, 48% of patients remained in response. Hematological toxicity was the most common adverse event (AE), with 94% of patients experiencing grade 3 or higher toxicity.

The TRANSCEND NHL 001 study (NCT02631044) of liso-cell included multiple types of lymphoma. In 32 patients who were infused with liso-cel, the ORR was 84%, and 59% of patients achieved CR. The most common grade 3 or greater AEs were hematologic toxicities, which affected 34% of patients.

In the future, different combinations and novel agents such as second-generation BTK inhibitors that are currently in development may produce more favorable results for patients with MCL. Determining proper sequencing for combination therapies and the best ways to use CAR T-cell therapy are also important factors, the authors noted.

While there has been progress in MCL research and treatment development, it still remains incurable, and the authors point to novel targeted agents and potential combinations with CAR T-cell therapies as likely future routes for progress.

Reference

Tbakhi B, Reagan PM. Chimeric antigen receptor (CAR) T-cell treatment for mantle cell lymphoma (MCL).Ther Adv Hematol. Published online February 26, 2022. doi:10.1177/20406207221080738

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Current Strategies and the Potential of CAR T-Cell Therapy in Relapsed and Refractory MCL - AJMC.com Managed Markets Network

Cortexyme, SQZ, Others Present New Insights into Alzheimer’s and Parkinson’s – BioSpace

The 2022 International Conference on Alzheimers and Parkinsons Disease held virtually and in Spain late last week and this weekend has ended, but it reported on numerous research studies and insights into the diseases. Heres a look at just a few of those stories.

Cortexyme Offered New GAIN Trial Data on Moderate Alzheimers

Cortexymepresented new data from its Phase II/III GAIN trial of COR388 (atuzaginstat) to treat mild to moderate Alzheimers disease. The data described target engagement data showing the drug inhibited lysine gingipains, which demonstrated a 30% to 50% slowing in cognitive decline in participants with high P. gigivalis load. It also described numerical trends in traditional Alzheimers disease biomarkers, including phosphor-tau 181 and total tau.

Our understanding of the impact of lysine gingipain inhibition on neurodegeneration and other Alzheimers disease markers continues to expand, Michael Detke, MD, Ph.D., Cortexymes chief medical officer, said. The evidence demonstrates our target P. gingivalis may play a key upstream role in both of these areas, and correlations between P. gingivalis biomarkers and clinical assessments show that our ability to inhibit this target potentially leads to improved patient outcomes.

SQZ Biotechnologies Receives $2 Million Grant from NIH for Parkinsons

SQZ Biotech was awarded a $2 million SBIR Phase II grant from the National Institute of General Medical Sciences, a National Institutes of Health division. The two-year grant will support the development of cell engineering approaches to reprogram a patients immune cells directly into dopamine-producing neurons. This will be a potential new therapy for Parkinsons disease, marked by the death of dopamine-producing cells in the brain.

Directly creating dopamine-producing neurons by reprogramming a patients own immune cells would be a major breakthrough and could support a new Parkinsons disease treatment paradigm, Jonathan Gilbert, vice president and head of exploratory research at SQZ Biotechnologies, said. Unlike alternative allogeneic cell replacement approaches in development for Parkinsons disease, by using a patients own cells, treatment might not require chronic immunosuppression. Moreover, in altering cell fate with RNA-based cell engineering methods, no changes to the genome are likely to occur that could carry long-term risks.

The companys Cell Squeeze technology may have broader applications than Parkinsons disease because it is used to induce human pluripotent stem cells to create other cells via delivery of an mRNA encoding for a fate-specifying transcription factor.

Memory T Cells a New Target for Parkinsons Therapies?

Researchers at La Jolla Institute for Allergy and Immunologydiscovered that Parkinsons disease patients have a clear genetic signature of the disease in their memory T cells. Parkinsons disease progresses as dopamine-producing neurons in the brain die, although what is causing the death is unknown. However, one clue is the dead cells contain clumps of the alpha-synuclein protein that has been damaged. Their research found that people with Parkinsons disease have T cells that target alpha-synuclein early in the disease. This suggests a previously undiscovered possibility of an autoimmune component of the disease.

Parkinsons disease is not usually seen as an autoimmune disease, said LJI research assistant Professor Cecilia Lindestam Arlehamn. But all of our work points toward T cells having a role in the disease.

One crucial gene observed in these T cells is LRRK2, which is associated with the familial form of Parkinsons. This opens up potential new targets for treatment and prevention.

New Approach Decreases Brain Damage in PSP, Alzheimers and Related Diseases

Investigators at Washington University School of Medicine in St. Louis demonstrated that targeting astrocytes, specific immune cells in the brain, can reduce tau-related brain damage and inflammation at least in laboratory mice. Dysfunctional astrocytes, whose job is to clean debris out of the brain and repair damage, have been associated with Alzheimers and other neurodegenerative diseases. The researchers identified high levels of Alpha2-NKA, a protein that drives astrocyte toxicity, in brain samples from people who died of progressive supranuclear palsy (PSP), Alzheimers and other tau-related neurodegenerative diseases.

Brain inflammation, in general, is believed to be a contributor to Alzheimers disease, and Gilbert Gallardo, PhD, senior author and an assistant professor of neurology at WU said that inflammation is driven by non-neuronal cells in the brain, including astrocytes. Our study highlights that inflamed astrocytes are contributing to tau-associated pathologies and suggests that suppressing their reactivity may be beneficial in reducing brain inflammation and delaying Alzheimers progression.

The researchers also tested the use of a heart drug, digoxin, which interferes with apha2-NKA activity. The drug worked on mice models of tau-opathies, both when they were beginning to develop tau tangles and when the tangles and damage were already established.

The take-home message here is that suppressing the inflamed astrocytic state halts disease progression, said first author Carolyn Mann, who was then a technician in the Gallardos laboratory. This is important because experimental therapeutics for Alzheimers and related tauopathies have focused largely on clearing pathological proteins that have been implicated in neuronal dysfunction and death. But our study gives evidence that targeting inflamed astrocytes and brain inflammation may be the key to successfully treating such conditions.

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Cortexyme, SQZ, Others Present New Insights into Alzheimer's and Parkinson's - BioSpace

SeqLL Announces Formation of Scientific Advisory Board – Yahoo Finance

SeqLL, Inc

BILLERICA, Mass., March 21, 2022 (GLOBE NEWSWIRE) -- SeqLL Inc. (SeqLL or the Company) (NASDAQ: SQL; SQLLW), a technology company providing life sciences instrumentation and research services for collaborative partnerships, today announced the formation of a Scientific Advisory Board (SAB) comprised of distinguished and world-renowned leaders of the scientific community. The SAB will discuss with management potential new development opportunities that leverage the Companys unique True Single Molecule Sequencing (tSMS) technology across the omics fields, as well as advise management with their existing collaborative, scientific, & development partnerships. Each leader has previously utilized the tSMS platform and will leverage their expertise to provide valuable insight to our company.

We are privileged to be working with this distinguished and talented group of scientific advisors as we expand the reach of our tSMS technology, said Daniel Jones, CEO, President, and Co-Founder of SeqLL. These advisors bring valuable scientific insight and industry contacts to our work and will assist us in becoming a world-class leader in creating novel assets with our true single molecule sequencing technology.

Leaders of the SeqLL Scientific Advisory Board include:

Claes Wahlestedt, M.D., Ph.D., Chairman of the SAB. Director of the Center for Therapeutic Innovation, Leonard M. Miller School of Medicine, University of Miami. Dr. Wahlestedt has a long-standing academic as well as big pharma career in drug discovery, genomics and epigenetics, and has pioneered various translational efforts in these fields. From 2005 to 2011, Dr. Wahlestedt was a professor and a director at The Scripps Research Institute. Prior to that he founded and ran the genomics and bioinformatics center at the Karolinska Institute (a joint venture with Pharmacia/Pfizer). Among companies he has co-founded are CuRNA (now part of Opko Health/Camp4), based on his patent for targeting regulatory noncoding RNAs to up-regulate therapeutic proteins, and Epigenetix Inc., focusing on small molecule drugs for a variety of drug targets in cancer and neuroscience. He is the author of over 300 papers in scientific journals, with over 45,000 citations. Dr. Wahlestedt is an expert in genomic medicine pertaining to a number of innovative therapeutic modalities and diagnostic approaches.

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L. Frank Kolakowski, Ph.D., Senior Scientist, Tetracore, Inc. Dr. Kolakowski has a distinguished 30-year scientific research career that includes positions on the faculty of the University of Texas Health Science Center, as an instructor at Harvard Medical School, and as consultant for several U.S. government agencies. As an entrepreneur, Dr. Kolakowski founded and served as CEO of ReceptorBase from 2000-2004, prior to being hired by the prestigious consulting firm Booz Allen Hamilton to initiate and manage their life sciences practice. He received his Doctorate in Chemistry from the University of Pennsylvania and his B.S. in Biology and Chemistry from Towson State University. Tetracore and Dr. Kolakowski are long term partners of SeqLL.

Efrat Shema, Ph.D., Principal Investigator and Assistant Professor at the Weizmann Institute of Science. Recipient of the prestigious Fulbright Scholar, Dr. Shema conducted post-doctoral work under Dr. Bradley Bernstein at Massachusetts General Hospital, Harvard Medical School, and the Broad Institute of MIT and Harvard. During her Ph.D. studies at the Weizmann Institute, Dr. Shema has received multiple prestigious awards, including the Adams Fellowship from the Israel Academy of Sciences and Humanities, the Otto Schwartz Prize for Excellence, and the UNESCO-LOREAL national award for young women in life sciences. Dr. Shema completed her M.Sc. and Ph.D in molecular cell biology at the Weizmann Institute and earned her B.Sc. in life sciences at the Hebrew University in Jerusalem in 2005. Dr. Shemas research has focused on using SeqLLs tSMS platform to decode the cancer epigenome since 2014.

Timothy McCaffrey, Ph.D., Professor of Medicine and Director, Division of Genomic Medicine, George Washington University. Dr. McCaffrey has had a distinguished career in research focused in three major areas: cardiovascular disease, genomics and stem cells. In 2001, he received a prestigious MERIT award from NIH for his work on vascular aging. Dr. McCaffrey is a close collaborator of ours and has used the tSMS platform to identify several panels of RNA transcripts that are highly predictive biomarkers in the fields of cardiovascular disease, infection and inflammation. He co-founded True Bearing Diagnostics to commercialize diagnostics based on those discoveries. He received post-doctoral training at Cornell University Medical College in New York City, his Masters and Doctorate from Purdue University and his B.A. from St. Marys University.

About True Single Molecule Sequencing (tSMS) Technology SeqLLs collaborators are thoroughly committed to using only our tSMS platform in their scientific research due to its unique RNA and DNA sequencing and related services. Our true single molecule sequencing platform is NGS technology offers maximum flexibility and avoids many of the challenges common for standard NGS approaches. It that enables direct sequencing of millions of individual molecules not requiring PCR amplification at any stage of the process and a simple, economical sample prep protocols. Therefore, it captures a precise sample composition, without bias and loss of diversity and rare species. Our tSMS platform is ideally suited for RNA biomarker discovery and diagnostic assay developments, including challenging applications for the standard NGS platform, such as low quantity, difficult or degraded samples of cell-free DNA, FFPE-isolated nucleic acids, ancient DNA and forensic samples.

About SeqLL, Inc. SeqLL Inc. (SeqLL) is a technology company providing life sciences instrumentation and research services in collaborative partnerships aimed at the development of novel scientific assets and intellectual property across multiple omics fields. The Company leverages its expertise with its True Single Molecule Sequencing (tSMS) platform to empower scientists and researchers with improved genetic tools to better understand the molecular mechanisms of disease that is essential to the continued development of new breakthroughs in genomic medicine, and that hopefully address the critical concerns involved with todays precision medicine. In sum, our experienced team works with our collaborators to develop innovative solutions tailored to the needs of each specific project.

Forward Looking Statements This press release contains certain forward-looking statements, including those related to the applicability and viability of the Companys technology to quantifying RNA molecules from blood and other statements that are predictive in nature. Forward-looking statements are based on the Company's current expectations and assumptions. The Private Securities Litigation Reform Act of 1995 provides a safe-harbor for forward-looking statements. These statements may be identified by the use of forward-looking expressions, including, but not limited to, "expect," "anticipate," "intend," "plan," "believe," "estimate," "potential," "predict," "project," "should," "would" and similar expressions and the negatives of those terms. Prospective investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this presentation. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in the Company's filings with the Securities and Exchange Commission, including its registration statement on Form S-1, as amended, under the caption "Risk Factors."

Contacts:

Ashley R. Robinson LifeSci Advisors, LLC Tel: +1 (617) 430-7577 Email: arr@lifesciadvisors.com

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SeqLL Announces Formation of Scientific Advisory Board - Yahoo Finance

Circulating Tumor Cell Diagnostics Market 2022 Examination and Industry Growth till 2028 The Sabre – The Sabre

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Circulating Tumor Cell Diagnostics Market 2022 Examination and Industry Growth till 2028 The Sabre - The Sabre