Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I – BioSpace

Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression

Translation of metabolic correction to clinical outcomes in first two patients continues to support potential of hematopoietic stem cell gene therapy in a second neurometabolic disorder

Data support planned initiation of registrational trial in 2021

BOSTON and LONDON, Sept. 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at theSan Raffaele Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today's results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up, saidMaria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. "These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigationalex vivoautologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard

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

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

Availability of Other Information About Orchard

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

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. 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 product candidates, including the product candidates referred to in this release, Orchards expectations regarding the timing of clinical trials for 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, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be successfully developed, approved 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 or that long-term adverse safety findings may be discovered; 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; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

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

Contacts

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

Media Molly Cameron Manager, Corporate Communications +1 978-339-3378 media@orchard-tx.com

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Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - BioSpace

Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and Metagenomi, a gene editing company discovering breakthrough systems for curing genetic disease, today announced that Vor will evaluate the potential use of Metagenomis gene editing technology to develop engineered hematopoietic stem cell-based therapies for the treatment of blood cancers, such as acute myeloid leukemia.

Cancer patients deserve therapies with strong effects on cancer cells and minimal effects on all other cells, said Tirtha Chakraborty, Ph.D., Vors VP and Head of Research. Our new partnership with Metagenomi will help us achieve this goal by engineering hematopoietic stem cells using precise yet flexible gene editing thereby ensuring that targeted therapies can live up to their name."

The collaboration is non-exclusive and applies to pre-clinical research only. Further terms of the agreement are not being disclosed.

This partnership unites two transformative technologies our proprietary gene editing enzymes, and Vors platform for engineering hematopoietic stem cells such that they are inherently treatment-resistant, said Brian C. Thomas, Metagenomis CEO and co-founder. We are excited to be working together to bring both of these cutting-edge approaches into the clinic.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

About Metagenomi

Metagenomi is harnessing the vast information found in life on Earth to develop cures for genetic disease. Using proprietary data collected from around the world, Metagenomi has developed novel gene editing tools that enable next-generation gene and cell therapies.

Metagenomi is based out of Emeryville, California, and was founded by pioneers in the field of metagenomics, Jill Banfield and Brian C. Thomas. Metagenomi generates massive quantities of data from natural environments, producing complete genomes from organisms that are otherwise unknown. Metagenomi then unlocks the information captured in these genomes to develop game-changing in vivo and ex vivo therapeutics.

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Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies - Business Wire

FDA Approves Azacitidine tablets for Continued Treatment of AML – Cancer Network

The FDA has approved 300 mg tablets of azacitidine (Onureg) for the continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy, according to Bristol Myers Squibb, the developer of the agent.

The approval was based on results from the pivotal phase 3 QUAZAR AML-001 study in which treatment with azacitidine tablets resulted in a statistically significant and clinically meaningful improvement in overall survival (OS), the studys primary end point, of almost 10 months compared to placebo. Results from the study were first presented at the American Society of Hematology (ASH) Annual Meeting in December 2019.

Continued treatment with [azacitidine tablets]demonstrated an overall survival benefit in adults with AML who had achieved first complete remission in the QUAZARAML-001 study and, notably, it has the potential to do this in a convenient manner, given its once daily oral formulation, Andrew Wei, MBBS, PhD, QUAZAR AML-001 lead investigator from Alfred Hospital and Monash University in Melbourne, Australia, said in a press release. This approval should help establish continued treatment with [azacitidine tablets] as a standard component of AML therapy for adults who achieved first complete remission following chemotherapy and who cannot proceed to intensive curative therapy, like hematopoietic stem cell transplant.

The phase 3, international, randomized, double-blind study enrolled patients who were 55 years or older, had AML, were within 4 months of achieving first CR or CRi following intensive induction chemotherapy with or without consolidation treatment (per investigator preference prior to study entry), and were not candidates for hematopoietic stem cell transplant (HSCT) at the time of screening. Overall, the study enrolled 472 patients who were randomized 1:1 to receive either 300 mg of azacitidine tablets (n = 238) or placebo (n = 234) orally, once daily, for 14 days of a 28-day cycle, plus best supportive care.

Median OS from time of randomization was greater than 2 years (24.7 months; 95% CI, 18.7-30.5) among patients who received azacitidine tablets compared to 14.8 months (95% CI, 11.7-17.6) among patients receiving placebo (HR, 0.69; 95% CI, 0.55-0.86; P = .0009).Notably, the azacitidine tablets were continued until disease progression or unacceptable toxicity.

Serious adverse events (AEs) occurred in 15% of patients who received azacitidine tablets. Serious AEs observed in at least 2% of patients who received the agent included pneumonia (8%) and febrile neutropenia (7%). Only 1 fatal adverse reaction (sepsis) occurred in a patient who received azacitidine tablets.

The most common adverse events observed with azacitidine tablets versus placebo were nausea (65% vs 24%, respectively), vomiting (60% vs 10%), diarrhea (50% vs 21%), fatigue/asthenia (44% vs 25%), constipation (39% vs 24%), pneumonia (27% vs 17%), abdominal pain (22% vs 13%) arthralgia (14% vs 10%), decreased appetite (13% vs 6%), febrile neutropenia (12% vs 8%), dizziness (11% vs 9%), and pain in extremity (11% vs 5%). Moreover, of those who received azacitidine, permanent discontinuation due to an AE occurred in 8% of patients.

The FDA approval of [azacitidine tablets] is the culmination of over a decade of research and 13 pre-clinical and clinical trials. We are grateful to the patients, families and caregivers who participated in and supported these trials, and who ultimately made todays advancement possible, Giovanni Caforio, MD,chairman and chief executive officer, Bristol Myers Squibb, said in the release. This milestone is representative of our commitment to helping patients with hard-to-treat cancers live longer, and the approval of [azacitidine tablets] as an oral therapy option for patients is more relevant now than ever as the world continues to navigate the COVID-19 pandemic.

Importantly, Bristol Myers Squibb indicated that azacitidine tablets have warnings and precautions for risks of substitution with other azacitidine products, myelosuppression, increased early mortality in patients with myelodysplastic syndromes (MDS) and embryo-fetal toxicity. Due to substantial differences in the pharmacokinetic parameters, the company indicated azacitidine tablets should not be substituted for intravenous or subcutaneous azacitidine as it may result in a fatal adverse reaction.

The new drug application for this indication was previously granted priority review designation by the FDA, and a marketing authorization application for this indication was validated by the European Medicines Agency in May 2020.

Reference:

U.S. Food and Drug Administration Approves Onureg (azacitidine tablets), a New Oral Therapy, as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia [news release]. Princeton, NJ. Published September 1, 2020. Accessed September 1, 2020. https://news.bms.com/press-release/corporatefinancial-news/us-food-and-drug-administration-approves-onureg-azacitidine-ta

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FDA Approves Azacitidine tablets for Continued Treatment of AML - Cancer Network

New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem…

Impact of New Tissue Stem Cell Counting Algorithms

BOSTON (PRWEB) September 01, 2020

Stem cell biotechnology company, Asymmetrex, has been counting tissue stem cells like those used for bone marrow and cord blood transplantation therapies for a few years now. Recently, the company announced the issue of patents for its first-in-kind technology both in the U.S. and the U.K. However, until last Friday, August 28, Asymmetrex had not reported in the peer-reviewed academic literature how it achieves this feat that had been pursued by many distinguished labs for more than six decades.

Now in a report published in a special issue of OBM Transplantation, a peer-review journal for transplantation medicine research, Asymmetrex completes its introduction of the new technology to the fields of stem cell science and stem cell medicine. The report is the second invited article published in a special issue focused on the Isolation and Characterization of Adult Therapeutic Cells.

The new report describes Asymmetrexs discovery of mathematical formulas, call algorithms, that can be used to determine the number of stem cells in complex tissue cell preparations, like experimental samples or patient treatments. The stem cell counting algorithms are specific for different types of tissue stem cells. So, the algorithms defined for blood stem cells are distinct from the algorithms for liver stem cells, or lung stem cells. Once an algorithm is defined by the Asymmetrex technology, it can be used repeatedly as a simple, rapid, and inexpensive test to determine the quantity and dosage of its specific tissue stem cell type.

Asymmetrexs founder and director, James L. Sherley, M.D., Ph.D., anticipated the August publication of the new algorithms in a talk given earlier at the 6th Annual Perinatal Stem Cell Society Congress in March of this year. Then and now, he says that he believes, Now that the tissue stem cell counting algorithms are available, everything will change in stem cell science and medicine.

Prior to Asymmetrexs technology, there was no method for counting tissue stem cells in research, medicine, or for any other of their many uses. So, the impact of the stem cell counting algorithms in research and medicine is far-reaching. Such information is a game changer for accelerating progress in stem cell science and stem cell medicine, including improving treatments like gene therapy whose success depends on targeting tissue stem cells. There will also be tremendous gains in cell biomanufacturing, drug development, and environmental toxicology, all whose capabilities are currently limited by the lack of a facile means to quantify tissue stem cells.

To make the new counting technology readily accessible for evaluation by the greater academic, medical, and industrial stem cell communities, Asymmetrex provides free tissue stem cell counting on its company website.

About Asymmetrex

Asymmetrex, LLC is a Massachusetts life sciences company with a focus on developing technologies to advance stem cell medicine. The companys U.S. and U.K. patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of effective use of human adult tissue stem cells for regenerative medicine and drug development. Asymmetrex markets the first technology for determination of the dose and quality of tissue stem cell preparations (the AlphaSTEM Test) for use in stem cell transplantation therapies and pre-clinical drug evaluations. Asymmetrex is a member company of the Advanced Regenerative Manufacturing Institute BioFabUSA and the Massachusetts Biotechnology Council.

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New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem...

Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respi – PharmiWeb.com

GAITHERSBURG, Md., Sept. 1, 2020 /PRNewswire/ --Cartesian Therapeutics, a fully integrated, clinical-stage biopharmaceutical company developing cell and gene therapies for cancer, autoimmune diseases and respiratory diseases, today announced that it has initiated a Phase 1/2 clinical trial of its lead RNA-engineered mesenchymal stem cell (MSC) therapy, Descartes-30, in patients with moderate-to-severe acute respiratory distress syndrome (ARDS), including that caused by COVID-19. Based upon the company's research and analysis, this program is understood to be the first RNA-engineered cell therapy to enter clinical development for ARDS and COVID-19. It is also the first cell therapy to specifically degrade NETs, webs of extracellular DNA and histones that entrap inflammatory cells, block alveoli and vessels, and drive the pathogenesis of ARDS and COVID-19.

"Patients with ARDS, especially those with COVID-19 ARDS, generate copious amounts of NETs that physically obstruct alveoli and vessels, which leads to respiratory distress, immune-mediated thrombosis and a vicious cycle of inflammation," said Bruce Levy, MD, Chief of Pulmonary and Critical Care Medicine at Brigham and Women's Hospital and Parker B. Francis Professor at Harvard Medical School, and a clinical investigator in the Descartes-30 trial. "We would therefore expect that degrading NETs would improve oxygenation as well as resolve thrombi and quell inflammation in these patients. If successful, Descartes-30 would be a highly differentiated game-changer within our limited toolkit in managing this exceedingly difficult condition."

Descartes-30 is an off-the-shelf (allogeneic) MSC product engineered with Cartesian's RNA ArmorySM cell therapy platform. By expressing a unique combination of DNases that work synergistically, Descartes-30 can eliminate large, macroscopic amounts of NETs within minutes. MSCs are inherently immunomodulatory and naturally travel to the lungs, where they are expected to provide continuous, local delivery of DNases to NET-laden lung tissue.

"We engineered Descartes-30 without genomic modification, and therefore the production of DNases is expected to be time-limited to match the acute nature of ARDS," said Metin Kurtoglu, MD, PhD, Chief Medical Officer at Cartesian. "Given thatDescartes-30will produce DNases locally and transiently, we anticipate that it will have a favorable benefit-to-risk profile. We also anticipate that these properties will enable Descartes-30 to treat a wide array of NET-related autoimmune and cardiovascular diseases."

About the Phase 1/2a Clinical Trial

The "Phase 1/2a Study of Descartes-30 in Acute Respiratory Distress Syndrome" (NCT04524962) is enrolling patients with ARDS at multiple critical care units in the United States. Patients with ARDS due to COVID-19 are given enrollment priority. This first-in-human study aims to determine the safety and preliminary efficacy of Descartes-30 in patients with moderate to severe ARDS. The study, which is estimated to begin treatment in September, aims to enroll approximately 20 patients prior to initiation of a larger study. For more information visit http://www.cartesiantherapeutics.com/Descartes-30-ARDS.

About ARDS and NETs

ARDS is a severe inflammatory lung disease with a mortality of over 40%. Inflammation leads to injury of lung tissue and leakage of blood and plasma into air spaces, resulting in low oxygen levels and often requiring mechanical ventilation. Inflammation in the lung may lead to inflammation elsewhere, causing shock and injury or dysfunction in the kidneys, heart, and muscles. Some causes of ARDS include COVID-19, severe pneumonia (including influenza), sepsis, trauma, and smoke inhalation.

NETs are inflammatory webs of DNA and proteins produced by neutrophils. NETs are commonly found in ARDS and are thought to exacerbate the disease by physically occluding air spaces and vessels, leading to reduced oxygenation and increased risk of immune thrombi. NETs are implicated in a variety of conditions beyond ARDS, including autoimmune and cardiovascular diseases.

About the RNA ArmorySM

The RNA ArmorySM is Cartesian's proprietary RNA-based cell engineering platform that activates and arms cells with carefully selected, mRNA-based therapeutics. Unmodified donor cells enter the RNA ArmorySMin the millions; a battle-ready cell army leaves the RNA ArmorySMin the tens of billions. Each cell is equipped with a combination of therapeutics rationally chosen to have a synergistic effect on the disease. In the body, the cells deliver a precision-targeted treatment regimen directly to the site of disease. The cells express therapeutics with a defined half-life, enhancing their safety profile and making repeat dosing and outpatient administration possible. The platform is agnostic to cell type: we choose the best cell for the job, whether autologous or off-the shelf. For more information visithttps://www.cartesiantherapeutics.com/rna-armory/.

About Cartesian Therapeutics

Founded in 2016,Cartesianis a fully integrated, clinical-stage biopharmaceutical company developing potent yet safer cell and gene therapies designed to benefit the broadest range of patients with cancer, autoimmune and respiratory diseases. Cartesianhas three products in clinical development under four open investigational new drug application (INDs) with the U.S. Food & Drug Administration (FDA). All investigational therapies are manufactured at Cartesian's wholly owned, state-of-the-art cGMP manufacturing facility in Gaithersburg, MD.Cartesian's commanding IP position benefits in part from a broad, exclusive patent license from the National Cancer Institute. For more information visithttps://www.cartesiantherapeutics.com/clinical-trials/.

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Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respi - PharmiWeb.com

COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle – BioPharma-Reporter.com

In mid-August, the US Food and Drug Administrations (FDA) Oncologic Drugs Advisory Committee voiced support for the efficacy of remestemcel-L in children with steroid-resistant graft-versus-host (GvHD) disease. The positive vote moved Mesoblast a step closer to winning approval for the mesenchymal stem cell therapy.

While seeking approval in that long-targeted indication, Mesoblast is also working to show the cell therapy is effective in patients with acute respiratory distress syndrome (ARDS) caused by infection with SARS-CoV-2.

Talking to investors on a fourth quarter results conference call late last week, Mesoblast CEO Silviu Itescu explained how the potential size of the COVID-19 market opportunity creates a substantial challenge.

We ... have to be prepared to substantially scale up manufacturing ... to be in a position next year to make sufficient quantity of product to start to meet some of this unmet need. We are able to implement proprietary xeno-free technologies and we certainly have plans to move into 3D bioreactors to allow us to have sufficient capability to meet this large unmet need, said Itescu.

Like many organizations targeting COVID-19, including groups such as AstraZeneca and Regeneron Pharmaceuticals that have large in-house operations, Mesoblast is planning to partner to gain the scale needed to manufacture the quantities of remestemcel-L it may need.

Mesoblast is currently running a phase 3 trial of remestemcel-L in ARDS. Itescu is assuming that the company will be entering into a strategic partnership for manufacturing commercialization to serve the ARDS indication.

The need for remestemcel-L in ARDS will depend on the progress of COVID-19 vaccines, which could significantly reduce the number of people suffering the complication of SARS-CoV-2 infection, and the strength of the phase 3 data.

Itescu explained the rationale for developing remestemcel-L in COVID-19 on the conference call. Like GvHD, a hyperactive immune response, known as a cytokine storm, is implicated in ARDS. In ARDS the cytokine storm manifests in severe inflammation of the lungs.

Remestemcel-L has shown anti-inflammatory effects during its development in GvHD. In addition, there is evidence the cell therapy migrates to the lungs after intravenous administration, suggesting it will accumulate in the part of the body where it is needed most in ARDS,

US physicians administered remestemcel-L to ventilator-dependent patients under a compassionate use program earlier this year. Nine of the 12 patients were taken off ventilator support, after 10 days in median, and later discharged from the hospital.

The evidence to support the use of remestemcel-L in COVID-19 led Mesoblast to start a 300-subject clinical trial. Mesoblast is assessing the effect of remestemcel-L on mortality after 30 days and is set to hold a series of interim analyses as increasing percentages of participants reach that point. If the data link remestemcel-L to improved survival, Mesoblast will seek expedited regulatory approval.

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COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle - BioPharma-Reporter.com

Prior Use of Novel Agents Does Not Affect alloHCT Outcomes in CLL – Cancer Therapy Advisor

The number of prior novel agents administered or their use immediately prior to allogeneic hemopoietic stem cell transplant (alloHCT) for the treatment of chronic lymphocytic leukemia (CLL) was not associated with outcomes, according to results of a retrospective study published in Blood Advances.1

Novel agents such as BCR, BTK, PI3K, and BCL2 inhibitors have generally improved outcomes among patients with CLL, but a subset of patients experience disease progression, resistance, high-grade transformation, or drug intolerance and require additional curative modalities.

Understanding outcomes for potentially curative modalities including alloHCT following novel agent therapy is critical while devising treatment sequences aimed at long-term disease control, the authors wrote.

This multicenter, retrospective cohort study included 65 patients with CLL who underwent alloHCT. All patients had received at least 1 novel agent prior to their transplant. Patient-related and disease-related characteristics were assessed, as well as prior treatment and transplant characteristics.

At baseline, the median patient age was 50 years at CLL diagnosis and 60 years at alloHCT. The HCT-comorbidity index (CI) was 0 among 38% of patients, 1-2 among 37%, and 3 among 25%. The majority of patients received a reduced-intensity conditioning regimen and a calcineurin inhibitor with methotrexate as graft-versus-host disease (GVHD) prophylaxis. The median number of previous lines of therapy was 3, with a median of 1 prior novel agent.

At 24 months, the progression-free survival (PFS) was 63% and overall survival (OS) was 81%. Nonrelapse mortality was 13% and relapse occurred among 27% of patients.

The number of novel agents used prior to transplant and the timing of their use was not associated with PFS or OS. In univariate analyses, there was no association between PFS and prior use of ibrutinib, venetoclax, PI3K inhibitors, 2 novel agents, or novel agent use immediately before alloHCT. Results were similar for OS.

Poor-risk characteristics, complete versus partial remission, and transplant characteristics were also not associated with PFS after transplant.

Multivariate analysis, however, demonstrated that HCT-CI 1 was significantly associated with PFS (hazard ratio, 3.3; 95% CI, 1.1-9.9; P =.035).

The authors concluded that prior novel agents do not appear to impact the safety of alloHCT, and survival outcomes are similar regardless of number of novel agents received, prior chemoimmunotherapy exposure, or novel agent immediately preceding alloHCT.

Reference

Roeker LE, Dreger P, Brown JR, et al. Allogeneic stem cell transplantation for chronic lymphocytic leukemia in the era of novel agents. Blood Adv. 2020;4:3977-3989. doi:10.1182/bloodadvances.2020001956

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Prior Use of Novel Agents Does Not Affect alloHCT Outcomes in CLL - Cancer Therapy Advisor

MaaT Pharma Announces Positive Data for Its Lead Microbiome Biotherapeutic MaaT013 in Intestinal Acute Graft-versus-Host-Disease at the Virtual 46th…

Details Category: DNA RNA and Cells Published on Tuesday, 01 September 2020 11:37 Hits: 198

-- Clinical data was gathered from eleven patients with gastrointestinal (GI) aGvHD treated with MaaT013, MaaT Pharmas full-ecosystem microbiota biotherapeutic, as part of a compassionate use program --

-- MaaT013 provided positive impact with satisfactory safety profile in all eleven patients --

-- Overall response rate was 82% at day 28 after first dosing, including 5 Complete Responses (CR) and 2 Very Good Partial Responses (VGPR) --

LYON, France I August 31, 2020 I MaaT Pharma announced today clinical data on the compassionate use of its lead full-ecosystem microbiome restoration biotherapeutic, MaaT013. The data included eleven patients that developed gastrointestinal, acute Graft-versus-Host-Disease (GI aGvHD), a severe condition where the transplant donors immune cells attack the patients tissues after receiving an allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) to treat their hematologic malignancies. All patients showed some response to MaaT013, with five patients achieving a complete response and two having a very good partial response 28 days after first dosing. The results were presented by Dr. Florent Malard, Associate Professor of Hematology at Saint-Antoine Hospital and Sorbonne University in an ePoster presentation on August 29, 2020 during the Virtual 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT).

There is a strong rationale suggesting that restoration of the microbiota in the gut could play a role in containing intestinal GvHD by modulating the immune system of the patient and we are starting to see this with the administration of MaaT013 in individuals with no other treatment options, said Dr. Florent Malard. The results from this compassionate use program show a remarkable improvement in the patients who achieved a complete response following treatment.

In the presented evaluation, eleven patients with steroid-dependent (n=3) or steroid-refractory (n=8) intestinal aGVHD following allogeneic hematopoietic stem cell transplantation received at least one and up to three doses of MaaT013 as part of a compassionate program. Most patients (n=10) had developed GI-predominant aGVHD. Treatment response was evaluated seven days after each administration and on day 28 after the first dose. These patients had previously been treated with and failed up to five lines of systemic therapy for aGvHD. Overall response rate was 82% (9/11) at day 28 after first dosing, including 5 Complete Responses (CR), 2 Very Good Partial Responses (VGPR), and 2 Partial Responses (PR). Considering the best GI response that can be achieved, all (11/11) patients experienced at least a PR, with 5 CRs, 4 VGPRs and 2 PRs. Among the 11 treated patients, 6 were still alive at last follow-up (median 214 days; [range, 49-413]). Among the 5 patients with CR, all were still alive at last follow-up and were able to taper or stop steroids and immunosuppressants. Only one patient presented recurrence of GI symptoms at 3 months. Overall, the data demonstrated that reintroduction of a full-ecosystem microbiota provided therapeutic effect in these patients. The safety of the MaaT013 microbiota biotherapeutic was satisfactory in all patients. One patient developed sepsis one day after the third dosing, but no pathogen was identified in blood cultures and the patient recovered after a course of antibiotics.

Data from 8 of these 11 patients had previously been presented at the 61st American Society of Hematology (ASH) 2019 Annual Meeting.

John Weinberg, MD, Chief Medical Officer at MaaT Pharma added, We are encouraged by these positive results from the compassionate use program, which was conducted in parallel to MaaT Pharmas HERACLES Phase II clinical trial to evaluate the safety and efficacy of MaaT013 in gastrointestinal predominant, steroid-refractory aGvHD patients. To date, a total of 65 patients have been treated with MaaT013, including patients under compassionate use, patients in an early access program in France, and patients enrolled in the Phase II clinical trial. We expect a primary readout from the trial in Q1 2021.

The poster can be viewed on the companys website.

About MaaT013

MaaT013 is a full-ecosystem, off-the-shelf, standardized, pooled-donor, high-richness microbiome biotherapeutic, in enema formulation. It is manufactured at MaaT Pharmas centralized European cGMP production facility. The product has a stability of up to 24 months and is characterized by a high diversity and consistent richness of microbial species. MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It is currently being tested in a Phase 2 clinical trial (NCT03359980), and is already being administered in compassionate use.

About MaaT Pharma

MaaT Pharma, a clinical stage company, has established the most complete approach to restoring patient-microbiome symbiosis to improve survival outcomes in life-threatening diseases. Committed to treating cancer and graft-versus-host disease (GvHD), a serious complication of allogeneic stem cell transplantation, MaaT Pharma has already achieved proof of concept in acute myeloid leukemia patients and a Phase 2 clinical trial in acute GvHD is ongoing. Supporting the further expansion of our pipeline into larger indications, we have built a powerful discovery and analysis platform, GutPrint, to evaluate drug candidates, determine novel disease targets and identify biomarkers for microbiome-related conditions. Our therapeutics are produced through a standardized cGMP manufacturing and quality control process to safely deliver the full diversity of the microbiome. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to spearhead microbiome treatment integration into clinical practice.

SOURCE: MaaT Pharma

Excerpt from:
MaaT Pharma Announces Positive Data for Its Lead Microbiome Biotherapeutic MaaT013 in Intestinal Acute Graft-versus-Host-Disease at the Virtual 46th...

CAR T-Cell Optimization Starts in Production, Extends to Therapy – Genetic Engineering & Biotechnology News

Just as heat-seeking missiles race toward the infrared signatures of their targets, chimeric antigen receptor (CAR) T cells home in on cancer-associated or -specific antigens. Once the antigens are engaged, CAR T cells let fly with cytotoxic flak, granules containing perforin and granzymes, while activating supplementary tumor-killing mechanisms such as stromal sensitization and macrophage polarization. It is to be hoped that by the time the cytotoxic smoke clears, the cancer will have been destroyed.

The development of CAR T cells has revolutionized adoptive cellular therapies against cancer. CARs are genetically engineered to combine antigen- or tumor-specific-binding with T-cell activating domains. T cells, obtained from the patient (autologous cells) or from a healthy donor (allogeneic cells), are typically transduced with an engineered vector, expanded, and infused back into the patient for tumor eradication.

In the 10 years since its inception, the CAR T-cell field has progressed rapidly. Two CAR T-cell products have been approved for clinical use, and many more products are undergoing clinical trials or are in development. Although the field initially focused on B-cell malignancies, it is now advancing on solid tumors.

Despite its initial success, the CAR T-cell field must find ways to generate products that are potent, affordable, and available. To achieve enduring success, the CAR T-cell field is undertaking a range of initiatives. These include the engineering of bridging proteins for multiantigen targeting; the creation of nonviral allogeneic off-the-shelf products; the organization of vein-to-vein networks; and the development of precisely tuned therapies, that is, precisely timed and dosed therapies.

Cellular therapy is a living drug, declares Steve Shamah, PhD, senior vice president, Obsidian Therapeutics. As with any drug, damage can occur if the therapy is not carefully regulated. Our company focuses on creating controllable cell therapies by engineering CAR T cells or tumor-infiltrating lymphocytes to produce regulatable cytokines and proteins that can enhance functional activity, especially against solid tumors.

For example, the company is developing a platform that armors CAR T cells with immunomodulatory factors such as interleukin-15 (IL-15) or CD40 ligand. Shamah explains, These factors can enhance functional activity by driving T-cell expansion, conferring resistance to immunosuppression, improving antigen presentation, and inducing antigen spread. However, both factors can also produce systemic toxicity. Our technology modulates the level and timing of their activity in a fully controlled, dose-dependent manner using an FDA-approved small-molecule drug.

The Obsidian platform, cytoDRiVE, adds a drug-responsive domain (DRD) onto a therapeutic protein of interest. DRD tags are misfolded or inherently unstable in the cell. However, they can be reversibly stabilized by the binding of approved small-molecule drugs. When the drug is absent, the DRD-tagged protein is turned off. When the drug is present, the DRD-tagged protein is turned on. When DRD tags are in place, the concentration of the small-molecule drug serves as a biological rheostat for controlling the dosing of the therapeutic protein.

Preclinical in vivo mouse studies assessed anti-CD19 CAR T cells that were engineered to express an IL-15-DRD that responded to the FDA-approved drug acetazolamide. In these studies, tumor regression was demonstrated.

Controlling the precise timing and expression level of these immunomodulatory factors in CAR T cells could significantly enhance safety and therapeutic efficacy, concludes Shamah.

Obsidian is currently focusing on the oncology space, but the company is also exploring other areas such as autoimmunity and even the regulation of transcription factors to enable controllable in vivo CRISPR-Cas9 gene editing.

Despite the remarkable success of CAR T-cell therapies, relapses can occur within six months for up to 50% of patients treated with anti-CD19 CAR T-cell therapy. Failures can occur due to loss of CD19 expression or to continued tumor proliferation. Aleta Biotherapeutics has developed a novel technology to reactivate CAR T cells in relapsed patients.

Our approach utilizes antigen-bridging proteins to coat tumors with CD19, says Paul Rennert, PhD, Aletas president and CSO. [The tumors are then] recognized by the patients anti-CD19 CAR T cells, essentially creating a cytotoxic synapse that results in tumor cell death.

To thwart anti-CD19 CAR T-cell therapy relapses, the company developed a bridging protein using the extracellular domain of CD19 and an anti-CD20 antibody domain. CD20 is an antigen present on the majority of B-cell malignancies. Rennert explains that these injected bridging proteins will coat the patients tumor cells with CD19, creating a target to activate or reactivate a patients anti-CD19 CAR T cells.

To show proof-of-principle, the company performed in vivo studies using a half-life-extended form of the bridging protein injected into mice carrying CD20-positive tumor cells and anti-CD19 CAR T cells. Rennert emphasizes, Our studies demonstrated this strategy can be used to reactivate CD19 CAR T cells to prevent and reverse relapses.

Other programs in development include a bridging protein for injection to improve outcomes in multiple myeloma patients treated with CAR T cells, and bridging protein programs for HER2-positive breast cancer patients with central nervous system metastases. The company is preparing investigational new drug applications for its technology and plans to start Phase I trials in 2021.

Assessing whether engineered CAR T-cell and T-cell receptor (TCR) therapies have successfully attacked and penetrated solid tumors (and not normal cells) can be like finding the proverbial needle in the haystack. Traditional methods using immunohistochemistry are useful for immune profiling, but they cannot differentiate engineered versus endogenous cells, points out Christopher Bunker, PhD, senior director of business development, Advanced Cell Diagnostics, a Bio-Techne brand. We developed a means to easily detect and track engineered therapeutic cells and delineate their pharmacokinetics within the tumor microenvironment of intact tumor biopsies, as well as their on-target/off-tumor activity.

Enter RNAscope, an RNA in situ hybridization technology that can enable single-cell spatial transcriptomics. RNAscope, Bunker asserts, is the only off-the-shelf method that can specifically detect engineered CAR T cells and TCR T cells in solid tumor patient biopsies.

Most cell therapies employ lentivirus transduction. Because CAR or TCR transgenes have unique sequences in the viral untranslated regions, these can be used as tags for identification of engineered cell therapies with RNAscope probes. The technology utilizes pools of paired oligos that can be thought of as a ZZ pair, where the paired 3 ends hybridize to ~50 bases of target mRNA, and where the paired 5 ends hybridize to a signal amplification module, which is built through sequential hybridization steps. The signal amplification of paired oligos results in an assay able to detect individual transcripts that appear as visible and quantifiable dots.

Its a little like planting and lighting Christmas trees, quips Bunker. The ZZ pairs plant trees along the mRNA with branches that are decorated either with fluorophores or chromogens. Although the primary technology currently features four colors, the company has developed a HiPlex (12-plex) assay and foresees even higher-plex assays with different detection methods.

We envision assays based on our core technologies that enable spatial analysis of perhaps a hundred transcripts in combination with tens of proteins, Bunker projects. In the context of cell therapy development, these will enable a more comprehensive understanding of tumor biology and immune cell profiles to determine the most effective treatment strategy for a patient, as well as for monitoring efficacy of solid tumor cellular therapies.

Companies developing CAR T-cell products are also eyeing a future involving GMP production. Thus, a critical early question is how to choose the best T-cell medium for manufacturing processes. To test the suitability of a CAR T-cell growing medium, companies must assess factors such as cell viability, cell expansion, cytokine profiles, and cell purity. A medium suitable for a CAR T-cell manufacturing process also needs to support rapid activation and CAR transduction. Additionally, the selected medium needs to be compatible with a variety of donors.

There are many available choices for T-cell culture media ranging from do-it-yourself recipes to commercially available one-size-fits-all complete formulations. CellGenix has developed a novel T-cell medium that avoids the use of human serum. Sebastian Warth, PhD, a senior scientist at CellGenix, explains, To achieve consistent results, human serum requires extensive testing prior to its use for production of cellular products due to lot-to-lot inconsistencies. Since human serum is a limited resource and might not be available in large quantities, it is unfavorable for commercial-scale manufacturing. Furthermore, the human origin of serum poses a certain risk of containing adventitious agents and is, therefore, a risk to the safety of the T-cell therapy product.

The companys TCM GMP-Prototype medium provides a serum-free and xeno-free product for early-onset T-cell expansion. According to Warth, key advantages include promotion of sustained viability, support for expansion of CD4+ and CD8+ T cells, promotion of a central memory and early differentiated memory T-cell phenotype, and maintenance of a high proportion of cytokine-producing cells including polyfunctional cells. Further, it was optimized for and verified with CAR T cells.

While autologous CAR T-cell therapies have proven highly successful, they also require a long and expensive manufacturing process. The dream of being able to utilize off-the-shelf allogeneic T cells is on the horizon.

Devon J. Shedlock, PhD, senior vice president, research and development, Poseida Therapeutics, reports, With our technology, we are able to genetically modify cells to create a fully allogenic, or off-the-shelf, product that does not require additional immunosuppression treatment like earlier generation approaches. We also have developed technology to allow us to make hundreds of doses from a single manufacturing run from healthy donors, thereby dropping the cost substantially.

According to Shedlock, the technology consists of three key aspects: 1) the piggyback DNA Modification System, 2) the Cas-CLOVER site-specific gene editing system, and 3) the Booster Molecule.

The PiggyBac DNA Modification System is a nonviral technology for stably integrating genes into DNA. One key feature is that piggyBac preferentially inserts into less mature T cells, enabling the production of therapies that have a high proportion of stem cell memory T cells, or Tscm cells.

Viral technologies are virtually excluded from Tscm cells, Shedlock states. However, Tscm cells are the ideal cell type for cell-based therapies because they have the ability to engraft and potentially last a lifetime, can produce wave after wave of more differentiated cells to attack the tumor, and are much more tolerable with low levels of adverse events compared to other CAR T-cell products.

The companys Cas-CLOVER gene editing technology is a hybrid gene editing technology used to edit the T cells to make allogeneic products. Cas-CLOVER works well in resting T cells, which is important in preserving Tscm cells in a fully allogeneic CAR T-cell product, Shedlock elaborates. It also is a very precise and clean system. This is a particularly important safety issue for allogeneic products that may be given to many patients.

The Booster Molecule is added during manufacture and is temporarily expressed on the cell surface to allow cell stimulation. Normally when allogeneic CAR T-cell products are created, the T-cell receptor must be eliminated to avoid the graft-versus-host reaction, which is a major safety issue. Importantly, this booster stimulation occurs while preserving the Tscm phenotype.

Poseida Therapeutics expects to launch a clinical trial for its multiple myeloma allogeneic product late this year or early next year. The company will also begin clinical trials later in 2021 on its pan-solid tumor allogeneic program.

Creation of partnerships can help drive development of CAR T-cell therapeutics from concept through clinical trials. Advanced therapies require advanced supply chain and data management, advises Minh Hong, PhD, head of autologous cell therapy, Lonza Pharma & Biotech. Prior biopharmaceutical models of mass production and distributionand the systems that support themare not effective for personalized therapeutics. As manufacturing demand increases for autologous cell therapies, there is an overarching need to both industrialize and simplify the entire supply chain ecosystem.

Hong says the overall project needs to be considered from a more comprehensive perspective: Due to the criticality of the starting material, everything from cell sourcing, patient coordination and scheduling for collection/infusion, transportation logistics, and manufacturing logistics needs to be coordinated, ensuring the highest standards, regulatory compliance, and safety throughout the process.

To meet these needs, Lonza is building a network of partners to develop a fully integrated vein-to-vein solution, that is, a system that includes all touch points involved in patient scheduling and sample collection, through material shipping logistics, manufacturing, and eventually the infusion of the cell therapy back into the patient. The partner network, Hong indicates, will help participants define smart workflows and execute an integration strategy. Hong sums up the networks therapeutic implications as follows: We believe these partnerships will decrease time to clinical program setup.

Lonza has more than a 20-year history of providing clinical and commercial manufacturing. Hong asserts, Our company brings to the table our process development and manufacturing experience along with proprietary solutions including a manufacturing execution system solution, MODA-ESTM, for electronic batch records and manufacturing traceability. In addition, we have announced partnerships with Vineti for a supply chain orchestration system and Cryoport to aid in shipping and logistics.

Lonza is also looking beyond CAR T-cell therapies. We would not limit our solutions and partnerships to autologous cell therapies, Hong declares. We can envision solutions for our in vivo viral vector manufacturing clients as well as our traditional allogeneic cell therapy clients.

Originally posted here:
CAR T-Cell Optimization Starts in Production, Extends to Therapy - Genetic Engineering & Biotechnology News

Learn global specifications of the Platelet Rich Plasma and Stem Cell Alopecia Treatment Market – StartupNG

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Learn global specifications of the Platelet Rich Plasma and Stem Cell Alopecia Treatment Market - StartupNG