Layoffs at Fla.-based stem cell clinic accused of aggressively marketing patients – ABC Action News

Tampa, Fla. A Tampa-based regenerative clinic accused of using aggressive and unfair marketing tactics to lure the diseased and desperate into spending thousands of dollars on unproven stem cell therapies laid off a small number of non-medical employees earlier this week, a company spokesperson confirmed.

While its unknown exactly how many employees the Lung Health Institute let go of earlier this week, a spokesperson on Wednesday denied the layoffs were the result of a pending lawsuit facing the company and recent media scrutiny questioning its practices. Instead, the spokesperson wrote in a statement the layoffs were the result of a planned transition that aligns with our strategy of moving away from a direct-to-consumer business model and focusing on our goal of ultimately creating industry-leading therapies that are FDA-approved for the treatment of chronic lung disease.

The Lung Health Institute, which is headquartered in Tampa but has locations in four other states, has been criticized by former clients for aggressively pitching stem cell therapies for incurable lung diseases that are not FDA approved or proven to work. In 2018, we spoke with former clients and widows of clients who described how they were pitched therapies for incurable lung diseases.

Hell definitely get some kind of improvement, thats what they told me, said Olga Cassady whose husband died of lung disease about 6 months after, she says, they spent $11,000 for treatment at the institutes Tampa headquarters, formerly known as the Lung Institute.

In 2018, Maureen Rosen of Ocala, also told us how she also bought into the Institutes pitch that its treatment would help slow down her progression with chronic obstructive pulmonary disease (COPD). At the time, Rosen was on an oxygen tank 24/7. But shortly after her treatment at the Lung Institute, Rosen told us, it seems like from the day I had it, I started to get worse, she said last year.

Rosen and Cassady are not the only ones complaining about the treatments offered by the company.

Tampa Attorney Ben Vinson filed the lawsuit on behalf of two former patients and said he has more than 30 others including Rosen and Cassady, who are hoping for a class action lawsuit against the clinic.

In Vinsons original complaint filed in 2017, he accused the Lung Institute of using aggressive and deceptive marketing tactics for sham treatments based on sham science and deception, according to the complaint.

They were desperate when they came to this company. They were all dying slowly, suffocating from various lung illnesses, Vinson said. They were desperate financially, in most cases, to afford the procedure so a lot of them gave up their last dime in order to do so, he said.

Earlier in the day, its spokesperson sent us copies of six testimonials from former patients who said they felt positive results after undergoing treatments at the institute. Several indicated they sought booster treatments after benefits from their original treatments wore off.

According to court filings, before any treatments are done at the institute, patients must sign a patient consent form which warns them that the treatment may not have any impact on their conditions.

A company spokesperson said the company is currently in discussions with the FDA to start clinical trials with investigative new drugs (IND) to help treat patients with lung diseases.

As for this weeks layoffs and its impact on patient treatments at the Institutes facilities, a company spokesperson said, the changes will have no impact on the quality medical care we provide our patients.

Visit link:
Layoffs at Fla.-based stem cell clinic accused of aggressively marketing patients - ABC Action News

Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells – BioSpace

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been reprogrammed or induced to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled Methods of Reprogramming Animal Somatic Cells was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms. For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, Lineages exploration of alternative cell therapy platforms. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005404/en/

Go here to see the original:
Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells - BioSpace

New Mechanism of Bone Maintenance and Repair Discovered – Technology Networks

Led by researchers at Baylor College of Medicine, a study published in the journal Cell Stem Cell reveals a new mechanism that contributes to adult bone maintenance and repair and opens the possibility of developing therapeutic strategies for improving bone healing.

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized, said corresponding author Dr. Dongsu Park, assistant professor of molecular and human genetics and of pathology and immunology at Baylor. Bone stem cells have been found both in the bone marrow inside the bone and also in the periosteum the outer layer of tissue that envelopes the bone. Previous studies have shown that these two populations of stem cells, although they share many characteristics, also have unique functions and specific regulatory mechanisms.

Of the two, periosteum stem cells are the least understood. It is known that they comprise a heterogeneous population of cells that can contribute to bone thickness, shaping and fracture repair, but scientists had not been able to distinguish between different subtypes of bone stem cells to study how their different functions are regulated.

In the current study, Park and his colleagues developed a method to identify different subpopulations of periosteum stem cells, define their contribution to bone fracture repair in live mouse models and identify specific factors that regulate their migration and proliferation under physiological conditions.

Periosteal stem cells are major contributors to bone healing

The researchers discovered specific markers for periosteum stem cells in mouse models. The markers identified a distinct subset of stem cells that contributes to life-long adult bone regeneration.

We also found that periosteum stem cells respond to mechanical injury by engaging in bone healing, Park said. They are important for healing bone fractures in the adult mice and, interestingly, their contribution to bone regeneration is higher than that of bone marrow stem cells.

In addition, the researchers found that periosteal stem cells also respond to inflammatory molecules called chemokines, which are usually produced during bone injury. In particular, they responded to chemokine CCL5.

Periosteal stem cells have receptors molecules on their cell surface that bind to CCL5, which sends a signal to the cells to migrate toward the injured bone and repair it. Deleting the CCL5 gene in mouse models resulted in marked defects in bone repair or delayed healing. When the researchers supplied CCL5 to CCL5-deficient mice, bone healing was accelerated.

The findings suggested potential therapeutic applications. For instance, in individuals with diabetes or osteoporosis in which bone healing is slow and may lead to other complications resulting from limited mobility, accelerating bone healing may reduce hospital stay and improve prognosis.

Our findings contribute to a better understanding of how adult bones heal. We think this is one of the first studies to show that bone stem cells are heterogeneous and that different subtypes have unique properties regulated by specific mechanisms, Park said. We have identified markers that enable us to tell bone stem cell subtypes apart and studied what each subtype contributes to bone health. Understanding how bone stem cell functions are regulated offers the possibility to develop novel therapeutic strategies to treat adult bone injuries.

Reference

Ortinau et al. (2019) Identification of Functionally Distinct Mx1+SMA+ Periosteal Skeletal Stem Cells. Cell Stem Cell. DOI: https://doi.org/10.1016/j.stem.2019.11.003

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Read the original:
New Mechanism of Bone Maintenance and Repair Discovered - Technology Networks

BMS’ oral maintenance therapy boosts survival in AML patients – PMLiVE

Bristol-Myers Squibb has scored significant overall survival results in a phase 3 trial of its oral maintenance therapy CC-486 in acute myeloid leukaemia patients who have limited treatment options.

The QUAZAR AML-001 study investigated CC-486 in newly-diagnosed AML patients who have achieved remission with intensive induction chemotherapy, are at least aged 55 years and are stem-cell transplant ineligible.

The overall survival benefit was significant, with median OS after 41.2 months of follow-up reaching 24.7 months in the CC-486 treatment arm, compared to 14.8 months for placebo an almost ten month improvement.

During a presentation of the results at the annual ASH 2019 conference, Andrew Wei, from the Alfred Hospital and Monash University, Melbourne, Australia, said: Based on the results of the QUAZAR study, we are excited about the clinical development of CC-486 and the potential to establish maintenance therapy as a new treatment paradigm for patients with AML in first remission.

Relapse-free survival also improved in patients treated with CC-486 10.2 months in the treatment arm compared to 4.8 months for placebo.

The study could have a practice-changing impact on the treatment of older AML patients who have a poor prognosis, according to Robert Brodsky, a professor of medicine at Johns Hopkins University in the US.

BMS took on the candidate following its acquisition of Celgene, which had already been developing the drug.

CC-486 is an oral version of Celgenes already-marketed chemotherapy Vidaza (azacitidine), which is administered intravenously it had been a top-selling product for the company, before being hit by generic competition in the US and Europe over recent years.

Vidaza brought in over $600m a year at its peak, as a treatment for AML and myelodysplastic syndrome (MDS), but fell to only $2m last year thanks to being pretty much entirely replaced by generics.

The new formulation could allow BMS to reclaim market share by moving the drug beyond its use as a first-line induction chemotherapy thanks to its oral dosing.

We are extremely encouraged by the results of the QUAZAR AML-001 study as a part of our continuing commitment to both epigenetic research and myeloid diseases, said Samit Hariwat, chief medical officer of BMS.

We now look forward to taking the next steps to bring CC-486 to eligible AML patients in need, he added.

According to a statement issued by BMS, regulatory submissions for CC-486 are planned for the first half of 2020.

The drug is also being tested in MDS, non-Hodgkins lymphoma and other cancers, both alone and in combination with checkpoint inhibitor drugs.

See the article here:
BMS' oral maintenance therapy boosts survival in AML patients - PMLiVE

Autoimmune Technologies Announces New Subsidiary to Develop Novel Antibody and Allogeneic Somatic Stem Cell Therapeutics For the Treatment of Invasive…

NEW ORLEANS, Dec. 9, 2019 /PRNewswire/ --Autoimmune Technologies LLC, a biotechnology company developing diagnostics and therapies for non-infectious and infectious disease, has established a new subsidiary, Stadius Biopharma LLC, to focus on proprietary anti-infective antibody therapeutics for diseases that are inadequately addressed by current standard-of-care medicines.

"Within this standalone entity we'll concentrate on our unique fully human antibodies and our stem cell antibody delivery platform to treat opportunistic viral, bacterial, and fungal infections," said Michael Charbonnet, who will serve as CEO of the newly formed business unit in addition to his continuing responsibilities for the parent company. Current Stadius antibody targets include invasive candidiasis and cytomegalovirus (CMV) infection.

"Ourantibodies bind and disrupt the function of conserved sequences of novel virulence factors associated with pathology of various species of Candida," said CSO Russell Wilson. "Candida aurisis an emerging fungus that presents a serious global health threat, and we'reencouraged by our initial preclinical data that indicates activity against C. auris as well as C. albicans, which is prevalent in high risk patients," he said.

The CDC is monitoring the spread of C. auris colonization and infection in the United States.C. aurisis associated with a high rate of morbidity and mortality and is resistant to current standard of care antifungal treatments. Healthy individualsunknowingly colonized with C. aurisor other Candida species and can spread the fungal cells to surfaces in hospitals, long-term-care facilities, and other healthcare environments, where they pose a threat to people with weakened immune systems.

Also under development are antibody therapeutics to CMV infection. More than 50% of individuals in the U.S. over 40 years of age have been exposed to CMV and carry it without symptoms. It can re-activate in patients with subpar immune systems with the potential for multi-organ involvement, and it can be transmitted through body fluids such as breast milk and saliva. Over time, individuals can become re-infected with different strains of CMV, further complicating prophylaxis and treatment. CMV is the most frequent infectious complication following both solid organ transplantation and bone marrow transplantation.

The company is also developing its proprietary non-viral gene modified allogeneic stem cell platform for delivering antibodies in chronically immune compromised patients. The somatic adipose-derived mesenchymal stem cells, which have unique properties to allow prevention of rejection, are transfected with the genetic information needed to enable the implanted stem cells to produce inside the patient the antibodies needed to prevent or treat infection. Antibody delivery to patients via this platform is intended to provide continuous protection while reducing the requirement for more frequent dosing.

Follow Stadius Biopharma on http://www.stadiusbio.com.

View original content to download multimedia:http://www.prnewswire.com/news-releases/autoimmune-technologies-announces-new-subsidiary-to-develop-novel-antibody-and-allogeneic-somatic-stem-cell-therapeutics-for-the-treatment-of-invasive-candidiasis-and-other-infectious-disease-300971143.html

SOURCE Autoimmune Technologies LLC

More here:
Autoimmune Technologies Announces New Subsidiary to Develop Novel Antibody and Allogeneic Somatic Stem Cell Therapeutics For the Treatment of Invasive...

Microbial Dynamics in Patients Treated With Allogeneic Hematopoietic Stem Cell Transplantation – Oncology Nurse Advisor

Researchers used a computational approach to characterize the gut microbiota of patients who had undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT), and they detected impacts on microbial community composition from dietary changes and antibacterial therapies. The studys results were presented at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, held in Orlando, Florida.

According to the studyinvestigators, the microbiota of patients often experience disruptions withallo-HSCT, and reduced microbial diversity may be associated with consequencessuch as graft-versus-host disease (GVHD).

In this study, theinvestigators performed DNA sequencing of 7930 samples from microbiomes ofpatients (N=1076) who had undergone allo-HSCT. The researchers fed thesequencing data into a clustering algorithm to evaluate patterns of diversityand composition in the microbial communities of these patients. The researchersused this algorithm to monitor changes in microbial communities withenvironmental modifiers that included antibacterial drugs and dietaryalterations.

Piperacillin-tazobactam(pip-tazo) exposure was reported to disrupt a microbial cluster (cluster 1)that had been characterized by high diversity (P <.05). This cluster,however, was not significantly impacted by cefepime and meropenem.

Pip-tazo was alsoassociated with shifts toward a cluster dominated by Streptococcus (P<.001), while shifts toward an Enterococcus-dominated clusterappeared to be encouraged by exposure to cefepime (P <.05) ormeropenem (P <.01).

Using specimens from 46patients who provided dietary details, the investigators found that cluster 1,noted for high diversity, lost stability in the presence of more protein (P<.05), while a diet with a higher fat intake was associated with increasedstability for this cluster (P <.05).

The investigators statedthat a goal of this approach to microbiota assessment, which evaluates entiremicrobial communities, is to help find treatments that enable optimal outcomesfor patients undergoing allo-HSCT.

Disclosure: Multiple authors declaredaffiliations with industry. Please refer to the original abstract for a fulllist of disclosures.

Reference

Nguyen CL, Gomes ALC, Peled JU, et al. Antibiotic exposures and dietary intakes are associated with changes in microbiota compositions in allogeneic hematopoietic stem cell transplant patients. Oral presentation at: 61st ASH Annual Meeting & Exposition; December 7-10, 2019; Orlando, FL. Abstract 597.

Continue reading here:
Microbial Dynamics in Patients Treated With Allogeneic Hematopoietic Stem Cell Transplantation - Oncology Nurse Advisor

CStone’s anti-PD-L1 antibody demonstrates promising antitumor activity with a complete response rate of 33.3% and a good safety profile in patients…

SUZHOU, China, Dec. 9, 2019 /PRNewswire/ -- CStone Pharmaceuticals ("CStone" or the "Company", HKEX: 2616) updated results from the CS1001-201 trial in a poster presentation at the 2019 American Society of Hematology (ASH) Annual Meeting.

CS1001-201 trial is a single-arm, multicenter Phase II clinical study designed to evaluate CS1001 monotherapy in relapsed or refractory extranodal natural killer (NK)/T-cell lymphoma (rr-ENKTL).

ENKTL is a subtype of mature T cell and NK cell lymphoma. It has a higher incidence in Asia than in Europe or North America. ENKTL is characterized by its rapid progression and poor prognosis. Currently, patients with rr-ENKTL lack effective treatment after failing an L-asparaginase-based combination chemotherapy regimen and targeted monotherapy only produces a complete response (CR) rate of below 10%.

"ENKTL accounts for approximately 6% of all lymphoma incidences, and there are vast unmet medical needs in this patient population that has failed the first-line treatment," said Dr. Frank Jiang, Chief Executive Officer and Chairman of CStone. "The CS1001-201 trial is the first clinical study of an anti-PD-L1 antibody in ENKTL worldwide. Our latest data have shown CS1001 to be well-tolerated with preliminary antitumor efficacy and survival benefit in rr-ENKTL patients. We will continue to advance this development program and hope that CS1001 will soon become a new treatment option for rr-ENKTL patients."

"CR is a critical outcome measure for the treatment of ENKTL. Studies have shown that ENKTL patients who achieved CR prior to autologous transplantations have a significantly better prognosis and longer survival than those who achieved partial response," said Dr. Jason Yang, Chief Medical Officer of CStone. "These latest results of CS1001 demonstrated a CR rate of 33.3% with durable response, an objective response rate of 43.3%, and a 1-year overall survival rate of 72.4%. These results represent a major breakthrough compared to current treatment options and support CS1001 as a potential conditioning regimen for hematopoietic stem cell transplantations."

Overview of the CS1001-201 trial

CS1001-201 is a single-arm, multicenter Phase II clinical study designed to evaluate CS1001 monotherapy in relapsed or refractory extranodal natural killer (NK)/T-cell lymphoma (rr-ENKTL).

Results reported in the poster at the 2019 ASH Annual Meeting

As of October 8, 2019, 32 patients were enrolled in the study. Among them, 24 patients (75.0%) had Stage IV ENKTL at screening, 9 patients (28.1%) received 2 lines of prior treatments, and 7 patients (21.9%) received 3 or more lines of prior treatments. All patients received 1200 mg CS1001 intravenously every 3 weeks for up to 2 years, until disease progression, intolerance, etc. The median duration of follow-up was 6.54 months (range, 0.72-15.64).

Preliminary efficacy data

CS1001 demonstrated promising antitumor activity and a favorable CR rate with durable response and survival benefit in rr-ENKTL patients.

Safety data

CS1001 was well tolerated in patients with rr-ENKTL

About CS1001

CS1001 is an investigational monoclonal antibody directed against PD-L1 being developed by CStone. Authorized by the U.S. based Ligand Corporation, CS1001 is developed by the OMT transgenic animal platform, which can generate fully human antibodies in one step. As a fully human, full-length anti-PD-L1 monoclonal antibody, CS1001 is similar to natural G-type immune globulin 4 (IgG4) human antibody, which can reduce the risk of immunogenicity and potential toxicities, potentially representing a unique advantage over similar drugs.

CS1001 has completed a Phase I dose-escalation study in China, in which CS1001 showed good tolerability and produced sustained clinical benefits during the Phase Ia study.

CS1001 is being investigated in a number of ongoing clinical trials, including one Phase I bridging study in the U.S. In China, its clinical program includes one multi-arm Phase Ib study, two pivotal Phase II studies, and three Phase III studies for several tumor types.

About CStone

CStone Pharmaceuticals (HKEX: 2616) is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and molecularly-targeted drugs to address unmet medical needs for cancer patients in China and worldwide. Since the Company's inception in 2015, CStone has assembled a world-class management team that has a full spectrum of complementary skillsets from preclinical research to clinical development and commercialization. With combination therapies as a core strategy, the Company has built a rich oncology pipeline of 15 oncology drug candidates. Currently, five late-stage drug candidates are at or near pivotal trials. With an experienced team, a rich pipeline, a robust clinical development-driven business model, and substantial funding, CStone's vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative and differentiated oncology therapies to cancer patients worldwide.

For more information about CStone Pharmaceuticals, please visit: http://www.cstonepharma.com

Forward-looking Statement

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

View original content:http://www.prnewswire.com/news-releases/cstones-anti-pd-l1-antibody-demonstrates-promising-antitumor-activity-with-a-complete-response-rate-of-33-3-and-a-good-safety-profile-in-patients-with-relapsed-or-refractory-extranodal-natural-killert-cell-lymphoma-300971851.html

SOURCE CStone Pharmaceuticals

Company Codes: HongKong:2616, HongKong:02616

Here is the original post:
CStone's anti-PD-L1 antibody demonstrates promising antitumor activity with a complete response rate of 33.3% and a good safety profile in patients...

Celularity Presents Pre-Clinical and Clinical Data from its Allogeneic, Placental-Derived, Off-the-Shelf Cell Therapy Platform at the 2019 ASH Annual…

WARREN, N.J.--(BUSINESS WIRE)--Celularity, Inc. (Celularity), a clinical-stage cell therapeutics company focused on the development of innovative allogeneic cellular therapies derived from post partum human placentas, announced data introducing an extensive platform in genetically-modified NK- and T cell-based programs, and highlighting long-term safety and efficacy results from the Phase 1 study of PNK-007 in patients with multiple myeloma (MM). These data were announced at the 61st American Society of Hematology (ASH) Annual Meeting in Orlando, F.L.

In pre-clinical studies, Celularitys allogeneic, placental-derived CD19 CAR T cell-based product candidate demonstrated in vivo anti-tumor efficacy in a lymphoma tumor model. Additionally, the Companys CD38 CAR NK cell-based product candidate showed in vitro and in vivo anti-tumor activity against CD38+ lymphoma and multiple myeloma cell lines without any on-target, off-tumor effect. Moreover, the Companys allogeneic, placental CD34+ cell-derived NK product candidate overexpressing a CD16 variant demonstrated its versatile combination potential with monoclonal antibodies to elicit in vitro and in vivo enhanced antibody-dependent cell mediated cytotoxicity (ADCC) function against lymphoma cell lines.

Results from a Phase 1 clinical study evaluating PNK-007 in patients with multiple myeloma demonstrated a single infusion of PNK-007 was well tolerated after autologous stem cell transplant (ASCT) for up to one year. PNK-007 is a first-generation investigational allogeneic off-the-shelf NK cell therapy. No serious adverse events (AEs) were attributable to PNK007, and no dose-limiting toxicity, graft-verse-host disease (GvHD), cytokine release syndrome (CRS), graft failure or graft rejection was observed.

We are excited about the pre-clinical and clinical findings presented at ASH this year which adds to a growing body of evidence about the range and versatility of our allogeneic, off-the-shelf, placental-derived cell therapy platform, and its potential uses against a range of devastating cancers which today have limited treatment options, said Robert Hariri, M.D., Ph.D., Founder, Chairman and CEO at Celularity. At Celularity we believe the next evolution in immunotherapy for cancer demands scalable, high quality and economical solutions which provide fully allogeneic, off-the-shelf products. The Celularity technology platform can provide that solution. We look forward to rapidly advancing multiple placental-derived NK- and T cell-based product candidates with potential as more tolerable, more accessible immunotherapies.

About PNK-007

PNK007 is the only allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. NK cells are a unique class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity. When derived from the placenta, these cells offer intrinsic safety and versatility, allowing potential use across a range of organs and tissues. PNK cells are currently being investigated as a treatment for acute myeloid leukemia (AML) and multiple myeloma (MM).

About Celularity

Celularity, headquartered in Warren, New Jersey, is a clinical-stage cell therapeutics company delivering transformative allogeneic cellular therapies, engineered from the postpartum human placenta. Using proprietary technology in combination with its IMPACT platform, Celularity is the only company harnessing the purity and versatility of placental derived cells to develop and manufacture innovative, highly scalable off-the-shelf treatments for patients with cancer, inflammatory and age-related diseases. To learn more, please visit http://www.celularity.com.

Forward-Looking Statements

This press release contains forward-looking statements. These forward-looking statements are based on expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. The information contained in this press release is believed to be current as of the date of original issue. Celularity expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.

Read the original post:
Celularity Presents Pre-Clinical and Clinical Data from its Allogeneic, Placental-Derived, Off-the-Shelf Cell Therapy Platform at the 2019 ASH Annual...

Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced that new results from its CD117-ADC patient preparation program were presented at the 61st Annual Meeting of the American Society of Hematology (ASH). These results, which were highlighted in an oral presentation at ASH by John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, showed the first-ever successful transplant of gene-modified cells in non-human primates using a targeted, single-agent antibody-drug conjugate (ADC), without the use of chemotherapy or radiation.

Todays conditioning regimens involve high doses of chemotherapy, often paired with radiation, to remove the disease-causing cells. As a result, patients undergoing gene therapy or stem cell transplant are all faced with a difficult choice: whether to endure severe toxicity and risk infertility and cancer for the chance for a cure. Magentas portfolio of targeted ADCs represents an extremely promising new option to prepare patients for gene therapy or transplant with no need for toxic chemotherapy or radiation, said Dr. Tisdale. The results presented today show that a single dose of single agent CD117-ADC achieves the same level of depletion as four doses of busulfan chemotherapy to enable successful engraftment and persistence of stem cells modified with the -globin gene, the gene that causes sickle cell disease and -thalassemia when mutated. Importantly, the animals undergoing preparation with CD117-ADC showed none of the damaging toxicities associated with busulfan conditioning.

Magenta is the only company with the people, platforms and a product engine committed to comprehensively transforming immune and blood system reset, which includes revolutionizing the toxic methods that are used to prepare patients for gene therapy and transplant today. said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. The gene therapy field has learned that higher levels of stem cell depletion, which meant higher doses of busulfan, were needed to ensure long-term engraftment of the gene-modified cells and persistence of gene therapy. Across all the modalities we have tested, we have seen that ADCs are most effective at achieving these high levels of stem cell depletion without chemotherapy to enable engraftment and long-term durability of the transplant. Todays impressive results provide important validation of the ADC approach as well as the CD117 target for patient preparation and underscore Magentas leadership in the field of conditioning.

Results from the CD117-ADC Patient Preparation Program

Title: A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates (Abstract #610)Presenter: John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, Bethesda, Md.

Magentas most advanced patient preparation program, CD117-ADC, targets CD117, a protein expressed on hematopoietic stem cells. CD117-ADC is designed to remove the genetically mutated cells in the bone marrow that cause certain genetic diseases, such as sickle cell disease, enabling curative stem cell transplant or gene therapy.

Results presented by Dr. Tisdale showed:

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Registration Statement on Form S-1, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Read more here:
Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug...

Novartis sets 2020 target for commercial readiness of cell therapy CMO – BioPharma-Reporter.com

In building out its cell therapy manufacturing network, Novartis has established four in-house facilities across the US and Europe.

However, the Swiss drugmaker has also used service providers, for example, by partnering with Germanys Fraunhofer IZI to access chimeric antigen receptor (CAR)-T therapies for use in its first European clinical trials and the early years of commercial sales in the region.

Novartis has taken the outsourcing route in Japan, too, completing the technology transfer for CAR-T therapy Kymriah (tisagenlecleucel) to Kobe-based the Foundation for Biomedical Research and Innovation (FBRI) early this year. FBRI subsequently supported clinical supply in Japan.

However, while the Japanese regulator approved Kymriah in March, it is taking longer to get the cell therapy contract manufacturer ready for commercial supply. At a recent R&D day, Novartis said the contract manufacturer will be commercially ready in the second half of next year.

Work to get FBRI commercially ready is part of a broader effort by Novartis to establish the capacity to support its global cell therapy ambitions. Currently, Novartis lacks that capacity.

Novartis CEO Vas Narasimhan said, We are supply constrained on Kymriah. Its not a demand constraint. Our goal is to work hard to relieve that ... constraint. We continue to be optimistic that Kymriah will reach blockbuster potential within the next five years.

The comment follows a period in which Kymriah, Novartis approved CAR-T, has fallen short of sales expectations. Sales over the first nine months of 2019 totalled around $180m (163m).

Novartis struggles to meet demand for Kymriah reflect the novel challenge of making autologous cell therapies, which are produced by taking a patients own cells, processing them and administering them back into the same patient.

Cell therapies are one of a clutch of emerging modalities and therapeutic concepts that are central to Novartis pipeline plans. Novartis recognises that it needs to match its infrastructure to the changing mix of modalities in its pipeline, as Jay Bradner, president of the Novartis Institutes for BioMedical Research, explained.

Bradner said, Its not enough just to do this in drug discovery, as we have learned. This needs to be a coordinated strategy across the entire enterprise.

That coordinated strategy has led Novartis to add in-house and contract capacity to support plans to introduce more CAR-Ts and CRISPR-edited stem cells in the coming years. Bradner said Novartis is building up scale to accelerate all of these programs[going] forward.

The exact demands Novartis places on that capacity are likely to change in the coming years, with Bradner predicting the future of the field will feature a new way of manufacturing CAR-T cells. One big expected change is the arrival of allogeneic CAR-Ts made from donor cells.

Continued here:
Novartis sets 2020 target for commercial readiness of cell therapy CMO - BioPharma-Reporter.com