BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, today announced that hemophilia A gene therapy clinical data and hemoglobinopathies ex vivo gene-edited cell therapy data will be featured in poster presentations at the 61st Annual Meeting of the American Society of Hematology (ASH). The ASH abstracts, which were submitted on August 3, 2019, were released online this morning. The conference will take place in Orlando, FL, from December 7-10, 2019.

Gene Therapy

The SB-525 poster will show updated Alta study data including durability of Factor VIII (FVIII) levels, bleeding rate, factor usage, and safety, for all five patients in the high dose cohort of 3e13 vg/kg, with approximately 4 months to 11 months of follow-up after treatment with SB-525.

As of the abstract submission date, four patients in the 3e13 vg/kg cohort achieved FVIII levels within the normal range with no bleeding events reported up to 24 weeks post-administration. These patients did not require FVIII replacement therapy following the initial prophylactic period of up to approximately 3 weeks post-SB-525 administration. The fifth patient in the 3e13 vg/kg cohort had only recently undergone treatment with SB-525 at the time of the abstract submission. As previously reported, one patient had treatment-related serious adverse events (SAEs) of hypotension and fever, which occurred approximately 6 hours after completion of the vector infusion and resolved with treatment within 24 hours, with no loss of FVIII expression. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer.

The rapid kinetics of Factor VIII expression, durability of response, and the relatively low intra-cohort variability in the context of a complete cessation of bleeding events and elimination of exogenous Factor VIII usage continues to suggest SB-525 is a differentiated hemophilia A gene therapy, said Bettina Cockroft, M.D., M.B.A., Chief Medical Officer of Sangamo, commenting on the published abstract. We are pleased with the progress of the program toward a registrational Phase 3 study led by Pfizer, who announced it has enrolled its first patient in the 6-month Phase 3 lead-in study. We have recently completed the manufacturing technology transfer to Pfizer and initiated the transfer of the IND.

Ex Vivo Gene-Edited Cell Therapy

The ST-400 beta thalassemia poster will show preliminary results from the first three patients enrolled in the Phase 1/2 THALES study. In this study, hematopoietic stem progenitor cells (HSPCs) are apheresed from the patient, edited to knock out the erythroid specific enhancer of the BCL11A gene, and cryopreserved prior to infusion back into the patient following myeloablative conditioning with busulfan. The first three patients all have severe beta thalassemia genotypes: 0/0, homozygous for the severe + IVS-I-5 (G>C) mutation, and 0/+ genotype including the severe IVS-II-654 (C>T) mutation, respectively.

As of the abstract submission date, Patient 1 and Patient 2 had experienced prompt hematopoietic reconstitution. Patient 1 had increasing fetal hemoglobin (HbF) fraction that contributed to a stable total hemoglobin. After being free from packed red blood cell (PRBC) transfusions for 6 weeks, the patient subsequently required intermittent transfusions. Patient 2 had rising HbF levels observed through 90 days post-infusion. For both patients, as of the most recent follow-up reported in the abstract, on-target insertions and deletions (indels) were present in circulating white blood cells. Patient 3 had just completed ST-400 manufacturing at the time of abstract submission. As previously disclosed, Patient 1 experienced an SAE of hypersensitivity during ST-400 infusion considered by the investigator to be related to the product cryoprotectant, DSMO, and which resolved by the end of the infusion. No other SAEs related to ST-400 have been reported and all other AEs have been consistent with myeloablation. No clonal hematopoiesis has been observed. Longer follow-up will be required to assess the clinical significance of these early results. ST-400 is being developed as part of a global collaboration between Sangamo and Sanofi, along with support through a grant from the California Institute for Regenerative Medicine (CIRM).

The first three patients enrolled in the THALES study all have severe beta thalassemia genotypes that result in almost no endogenous beta globin production. The increases in fetal hemoglobin and presence of on-target indels in circulating blood cells suggests successful editing using zinc finger nucleases. The results are preliminary and will require additional patients and longer-term follow-up to assess their clinical significance, said Adrian Woolfson, BM., B.Ch., Ph.D., Head of Research and Development. It is important to note that myeloablative hematopoietic stem cell transplantation reboots the hematopoietic system, and that sufficient time is required for the stem cells to fully repopulate the marrow and for new blood cells to form. In other myeloablative conditioning studies in a similar patient population, full manifestation of the effects of gene modification in the red blood cell compartment has taken as long as 12 months or more to become evident.

Sanofis in vitro sickle cell disease poster details a similar approach to ST-400, using mobilized HSPCs from normal donors and SCD patients and utilizing the same zinc finger nuclease for gene editing, delivered as transient non-viral RNA, and designed to disrupt the erythroid specific enhancer of the BCL11A gene, which represses the expression of the gamma globin genes, thereby switching off HbF synthesis. Results from ex vivo studies demonstrated enriched biallelic editing, increased HbF, and reduced sickling in erythroid cells derived from non-treated sickle cell disease patients. Sanofi has initiated a Phase 1/2 trial evaluating BIVV003, an ex vivo gene-edited cell therapy using ZFN gene editing technology to modify autologous hematopoietic stem cells using fetal hemoglobin to produce functional red blood cells with higher BhF content that are resistant to sickling in patients with severe sickle cell disease. Recruitment is ongoing.

About the Alta study

The Phase 1/2 Alta study is an open-label, dose-ranging clinical trial designed to assess the safety and tolerability of SB-525 gene therapy in patients with severe hemophilia A. SB-525 was administered to 11 patients in 4 cohorts of 2 patients each across 4 ascending doses (9e11 vg/kg, 2e12 vg/kg, 1e13vg/kg and 3e13vg/kg) with expansion of the highest dose cohort by 3 additional patients. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency.

About the THALES study

The Phase 1/2 THALES study is a single-arm, multi-site study to assess the safety, tolerability, and efficacy of ST-400 autologous hematopoietic stem cell transplant in 6 patients with transfusion-dependent beta thalassemia (TDT). ST-400 is manufactured by ex vivo gene editing of a patient's own (autologous) hematopoietic stem cells using non-viral delivery of zinc finger nuclease technology. The THALES study inclusion criteria include all patients with TDT (0/0 or non- 0/0) who have received at least 8 packed red blood cell transfusions per year for the two years before enrollment in the study. The FDA has granted Orphan Drug status to ST-400.

About Sangamo Therapeutics

Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic medicines with the potential to transform patients' lives using gene therapy, ex vivo gene-edited cell therapy, in vivo genome editing, and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Forward-Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding the Company's ability to develop and commercialize product candidates to address genetic diseases with the Company's proprietary technologies, as well as the timing of commencement of clinical programs and the anticipated benefits therefrom. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the outcomes of clinical trials, the uncertain regulatory approval process, uncertainties related to the execution of clinical trials, Sangamo's reliance on partners and other third-parties to meet their clinical and manufacturing obligations, and the ability to maintain strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable product candidates. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission and Sangamo's most recent Quarterly Report on Form 10-Q. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

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Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting - Business...

University of Pennsylvania scientists are piloting the use of special imaging markers to track the movement of one cancer therapy within the body.

Theyre doing so to gauge the success of CAR T, an immunotherapy that involves genetically modifying a patients immune cells and inserting them back into the body to kill cancer. Such therapy has revolutionized care for some cancers, but doctors need help viewing the travel of cells to measure progress. Early results of the experiment were detailed last month in Molecular Therapy.

"Currently, the only way to know whether a gene or cell therapy is still present in the body is to regularly biopsy tumors or draw blood, which offer very crude measurements of the therapy, Mark Sellmyer, MD, PhD, an assistant professor of radiology at Penn Medicine and lead author of the study, said in a statement. With our technology, clinicians would be able to see, quantitatively, the number and location of CAR T cells that have lasted in the body over time, which is an indicator of the therapy's durability and potential efficacy.

Read more:
Researchers use imaging tags, PET/CT scanner to better track cancer therapies - Radiology Business

The Phase 3 clinical trial testing BrainStorm Cell Therapeutics cell therapy candidateNurOwn inamyotrophic lateral sclerosis (ALS) patients is continuing as planned after a second safety assessment by the trials independent Data Safety Monitoring Board (DSMB) found no reasons to stop, the company announced.

The DSMBs recommendation comes after a pre-specified interim analysis of the first 106 ALS patients treated repeatedly with NurOwn in this randomized, placebo-controlled clinical trial.

After reviewing all of the safety data as of September 30th, the DSMB has recommended the study continue without any changes in the protocol. We did not identify any significant safety concerns, Carlayne Jackson, MD, a professor of Neurology and Otolaryngology UT Health San Antonioand the DSMB chairperson, said in a press release.

DSMBs consist of research experts who monitor the progress of a clinical trial and review safety and efficacy data while the study is ongoing. This panel can recommend that a trial be stopped early because of safety concerns or evidence a therapy is not working as intended, or if the trials main goals have already been reached.

NurOwn consists of mesenchymal stem cells (MSCs; stems cells able to generate various cell types) collected from a patients bone marrow. These MSCs are expanded and matured into a specific cell type called MSC-NTF by growing them under conditions that induce them to secrete high levels of neurotrophic factors (NTFs) that support the growth, survival, and maturation of nerve cells.

MSC-NTF cells also deliver immune system regulating cytokines, small proteins important in cell signaling or messaging, to sites of damage, BrainStorm reports. It is thought this will help to slow or stabilize disease progression.

The double-blind Phase 3 trial (NCT03280056),fully enrolledat its six U.S. sites, is investigating use of NurOwn in 200 ALS patients whose symptoms became evident within two years of the studys start. Patients are randomized 1:1 to either NurOwn or placebo, given via intrathecal (spinal canal) injection every two months.

The studysprimary measures of safety and efficacy are being determined using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) in patients after 28 weeks of treatment compared to placebo.

A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory agents, andcytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) after treatment with NurOwn.

BrainStorm is expecting to have topline trial data by the end of 2020, which will potentially support the submission of a Biologics License Application (BLA) to theU.S. Food and Drug Administration requesting approval.

We are very pleased with the DSMB recommendation that the Phase 3 clinical trial continue without any protocol modification. This represents an important clinical trial advancement for BrainStorm and for the development of NurOwn as an innovative cellular therapy approach for ALS patients, added Ralph Kern MD, BrainStorms chief operating officer and chief medical officer.

This clinical trial is being funded by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and other types of investment.

NurOwn was given orphan drug status by both theFDA and the European Medicines Agency (EMA) as apotential ALS treatment.

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queens University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimers disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.

Total Posts: 5

Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

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Phase 3 Trial of ALS Cell Therapy, NurOwn, Gets Thumbs Up in Safety Review by Monitoring Board - ALS News Today

Nonalcoholic steatohepatitis (NASH) is the most severe form of nonalcoholic fatty liver disease (NAFLD) and is characterized by a fatty liver, inflammation, and liver cell damage. If left untreated, NASH can further develop into liver fibrosis, the formation of scar tissue in the liver, and liver cirrhosis. To date, a patients only option at the final stage of liver disease is a liver transplant. There is no alternative available yet.

This year alone has already seen a number of disappointments within the NASH clinical trial landscape. In April, Gilead announced that its anti-fibrosis compound selonsertib failed to reach the endpoint in phase III. In June, Conatus and Novartis NASH cirrhosis treatment emricasan also missed its primary endpoint in a phase IIb trial. And although Intercepts Ocaliva showed an effect on fibrosis in phase III, the results were overshadowed by strong side effects.

The therapy landscape for NASH is currently dominated by small molecules that address defined targets. Many of the currently ongoing trials are focusing on the earlier stages of NASH, covering the fibrotic stages F1 and F2. The later stages of NASH, F3 and F4, on the other hand, are receiving attention at the clinical stage but are difficult to treat.

If you look at the number of therapies in development for cirrhotic NASH F4 and indications further down the disease progression path, you will find a blue ocean, says Henrik Luessen, CBO at Promethera. There is not much competition to be found in that space. NASH is a spectrum disease in which many factors play a role, including inflammation, immune activation, stellate cell activation, lipid metabolism, and fibrosis. Most molecules can only target one of these factors and the action is therefore very limited. In later stages, where many more factors are involved, there is a need for an approach that addresses more than one target. That is the key challenge in NASH.

Promethera has taken a leap into the blue ocean and has developed a cell therapy in which liver-derived cells can address several factors of the disease. These cells have a potent paracrine effect and are able to respond to the cause of the disease by secreting or expressing various molecules, including hepatic growth factor (HGF), indoleamine 2,3-dioxygenase (IDO), and prostaglandin E2 (PGE2), to reduce inflammation, inhibit stellate cell activation, and to further interrupt the fibrotic or cirrhotic process and restore liver function.

Based on the behavior of our cells observed over more than six years of intense clinical research following the inception of Promethera, we decided to focus on end-stage NASH and acute-on-chronic liver failure (ACLF), the end-stages of the disease where patients are suffering from at least one organ failure and have a very low prognosis to survive the next three months, Luessen explains. When we found promising signs of efficacy and safety in ACLF we decided to move further into the NASH field and focus on the late stages of the disease where there is a strong unmet medical need. In both cases, we are positioning our cells as an alternative to liver transplantation.

The data of the completed HEP101 study in ACLF patients will be published at the upcoming American Association for the Study of Liver Diseases (AASLD) meeting in Boston on 10th November 2019, for the first time.

Moreover, in May 2019, Promethera started the PANASH study, which is evaluating the safety of HepaStem in NASH F3 and F4 patients at different dosages. Efficacy markers will also be investigated as a study outcome.

Prometheras pipeline is comprised of two allogeneic cell therapies and one antibody. Designed to target the tumor necrosis factor receptor 1 (TNF-R1), the companys antibody Atrosimab is currently in preclinical development. TNF is known to interact with receptors one (R1) and two (R2). While R1 is responsible for triggering inflammatory responses and apoptosis, R2 is actually a type of regenerative receptor.

Classical TNF inhibitors inhibit all systemic TNF, including all interactions with R2, Luessen says. Atrosimab only inhibits R1, which allows the regenerative properties of R2 to be maintained. It can also be potentially used in combination with our HepaStem technology.

HepaStem, currently in phase II, is a cell therapy that addresses different components of the NASH disease progression. Consisting of liver-derived mesenchymal stem cells (MSC), HepaStem is administered intravenously without the need for immunosuppressants and enters the liver via the bloodstream, where it then targets multiple changed pathways. HepaStem can reduce tissue fibrosis and promote the restoration of liver function by lowering inflammation, deactivating stellate cells, and reducing fibrosis.

Prometheras second cell therapy, H2Stem, is currently at a preclinical stage. H2Stem are liver-derived progenitor cells from the hepatobiliary tract that can express various markers, can differentiate into hepatocyte-like cells in vitro and have been observed to home and repopulate the liver of humanized mice. H2Stem has shown good signs of engraftment in mice, which allows us to assume that it might have strong regenerative or repair properties, but we are still collecting evidence, says Luessen.

Prometheras allogeneic cell line is unique in that it also has the potential for liver homing, meaning the cells have a liver imprint and remain in the liver where they can fight the cause of the disease. Moreover, the cells do not provoke immunogenic responses, which enables the intravenous injection of the therapy without needing immune suppression. Even a second injection, triggers no acute immunogenicity or toxicity.

Our product has undergone the chemistry, manufacturing, and controls (CMC) part of development, Luessen explains. This means that we have a very scalable product. We are now moving to bioreactors, which will allow us to treat thousands of patients with only one liver. We are confident that we can meet global demand with our new in-house process, in particular when we move to our state-of-the-art facilities in Gosselies, Belgium, in 2020. At the moment, the only option for end-stage liver disease patients is a liver transplant, so with one liver we could avoid a very high number of liver transplants, at least exceeding the currently recorded global numbers of transplants. Thats what makes our technology unique and very promising.

Many companies are now becoming increasingly aware that addressing only one target in early-stage NASH does not add many benefits to patients, Luessen says. In fact, patients often have to take several medications continuously, which can come with unpleasant side effects. Furthermore, early-stage NASH patients can greatly benefit from lifestyle changes, including dietary measures and physical exercise.

In the early stages of NASH, during F1 and F2, many patients are not aware of their disease because they do not show any symptoms. Once the disease is felt, patients are often already in the later stages, F3 and F4, and here, the medicinal landscape becomes very poor, Luessen explains. Looking at the future, I think this will be recognized and there will be a greater focus on the fibrotic and cirrhotic stages of NASH. We can already see different pharma companies, who are usually competitors, working together. They are combining their compounds to have a more efficient treatment. This will be the only way to address the disease outside of cell therapy.

Do you want to discover more about how Prometheras cell therapy could revolutionize NASH treatments? Check out Prometheras website for more information or get in touch with their experienced team!

Images via Promethera and Shutterstock.com

Author: Larissa Warneck, Science Journalist, Labiotech.eu

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Can This Cell Therapy Help Fight the NASH... - Labiotech.eu

Across 2019, Vertex has struck deals intended to yield a new generation of breakthrough medicines.

In June, Vertex agreed to pay $245m (220m) upfront to acquire Exonics Therapeutics for its gene editing technology and pipeline of programs targeting diseases including Duchenne muscular dystrophy (DMD). Months later, Vertex put up another $950m to buy Semma Therapeutics and its cell therapy treatment for type I diabetes.

The acquisitions moved Vertex, which started out in small molecules, into new areas, and building out capabilities in those areas will cost money.

In recent years, Vertex has grown its annual operating expenses by 10% to 14%. Talking on a recent quarterly results conference call, Vertex CFO Charles Wagner warned investors to expect costs to rise faster in 2020.

Wagner said, Our current expectation is that the rate of growth will be somewhat higher in 2020 as we invest in research and preclinical manufacturing for selling genetic therapies in support of our programs in type I diabetes, DMD and other diseases.

The move into type I diabetes also takes Vertex into territory that, to some observers, looks different than the areas the company has targeted historically.

Asked by an analyst about the shift in focus, Vertex CEO Jeff Leiden downplayed the differences, noting that type I diabetes is treated in the US in a relatively small number ofcenters that can be targeted by a speciality sales force.

Researchers have achieved positive, long-term outcomes by transplanting cadaveric islets into patients but two barriers have stopped companies from industrialising that approach.

Firstly, there are too few cadaveric islets to treat all type I diabetics. Secondly, immunosuppression is needed to stop patients from rejecting the transplanted cells.

Semma is trying to tackle the problems by differentiating stem cells and using a device to protect them from the immune system. Vertex thinks these technologies are the breakthroughs the field needs to industrialize the concept.

Leiden said, We were watching companies who are addressing those two problems for the last two, three years. And over the last six to eight months, we were convinced that Semma has actually solved both of those problems.

Vertex reached that conclusion on the strength of preclinical data. Now, Vertex is set to invest to find out whether the idea works in the clinic.

Excerpt from:
Vertex invests in gene therapy manufacturing - BioPharma-Reporter.com

NEW YORK and SHANGHAI, Nov. 6, 2019 /PRNewswire/ --Cellular Biomedicine Group Inc.(NASDAQ: CBMG) ("CBMG" or the "Company"), a biopharmaceutical firm engaged in the drug development of immunotherapies for cancer and stem cell therapies for degenerative diseases, today reported its financial results and business highlights for the third quarter of 2019.

"During the third quarter of 2019, we made great strides in both corporate and clinical progress. We started our U.S. expansion for research and clinical development in a new 22,000 square foot facility in Rockville, Maryland in October 2019. This milestone will allow us to foster strategic partnerships, develop new innovations and support continued development of CBMG's cell therapy-based immune-oncology assets that have shown promise in early proof-of-concept trials in China," said Tony (Bizuo) Liu, Chief Executive Officer for the Company.

"We also had continued progress on the clinical side, with the initiation of our Phase II clinical trial in China of AlloJoin therapy for knee osteoarthritis (KOA). Additionally, our autologous stem cell therapy program for KOA, ReJoin, was accepted by the NMPA in China to begin a Phase II clinical trial. We are excited about our regenerative medicine programs as we are currently the only company that has received two clinical trial acceptances for any stem cell program in China."

Mr. Liu continued, "Presentations of our pre-clinical and clinical data at upcoming medical conferences later this year will demonstrate continued focus on our immune-oncology pipeline and we are proud to provide an update of our commitment to cancer immunotherapy."

Third Quarter 2019 and Other Recent Corporate Developments

Upcoming Clinical and Preclinical Presentations:

Financial Results for the Third Quarter of 2019

Conference Call and Webcast InformationThe Company will host a conference call and webcast with the investment community on Wednesday, November 6th at 4:30 p.m. Eastern Time featuring remarks by Tony Liu, Executive Director, CEO and CFO of CBMG.

Live Call:

Toll-Free: 1-855-327-6838

International: 1-604-235-2082

Webcast:

http://public.viavid.com/index.php?id=136796

Replay:

Toll-Free: 1-844-512-2921

International: 1-412-317-6671

Conference ID: 10007976

(Available approximately two hours after the completion of the live call until 11:59 p.m. ET on November 20, 2019)

About Cellular Biomedicine GroupCellular Biomedicine Group, Inc. (NASDAQ: CBMG) develops proprietary cell therapies for the treatment of cancer and degenerative diseases. It conducts immuno-oncology and stem cell clinical trials in China using products from its integrated GMP laboratory. The Company's GMP facilities in China, consisting of twelve independent cell production lines, are designed and managed according to both China and U.S. GMP standards. Its Shanghai facility includes a "Joint Laboratory of Cell Therapy" with GE Healthcare and a "Joint Cell Therapy Technology Innovation and Application Center" with Thermo Fisher Scientific, which partnerships focus on improving manufacturing processes for cell therapies. CBMG currently has ongoing CAR-T Phase I clinical trials in China. The China NMPA (formerly CFDA) accepted the Company's IND application for a Phase II trial for AlloJoin, CBMG's "Off-the-Shelf" allogenic haMPC therapy for the treatment of Knee Osteoarthritis (KOA), and the Company's IND application for a Phase II trial for ReJoin autologous haMPC therapy for the treatment of KOA. CBMG is included in the broad-market Russell 3000 Index and the small-cap Russell 2000 Index, and the Loncar China BioPharma index. To learn more about CBMG, please visit http://www.cellbiomedgroup.com.

Forward-Looking StatementsStatements in this press release relating to plans, strategies, trends, specific activities or investments, and other statements that are not descriptions of historical facts and may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking information is inherently subject to risks and uncertainties, and actual results could differ materially from those currently anticipated due to a number of factors, which include those regarding our ability to implement our plans, strategies and objectives for future operations, including regulatory approval of our IND applications, our plan to configure part of our Shanghai facility with GE Healthcare's FlexFactory platform, our ability to execute on our obligations under the terms of our licensing and collaboration arrangement with Novartis, our ability to execute on proposed new products, services or development thereof, results of our clinical research and development, regulatory infrastructure governing cell therapy and cellular biopharmaceuticals, our ability to enter into agreements with any necessary manufacturing, marketing and/or distribution partners for purposes of commercialization, our ability to seek intellectual property rights for our product candidates, competition in the industry in which we operate, overall market conditions, any statements or assumptions underlying any of the foregoing and other risks detailed from time to time in CBMG's reports filed with the Securities and Exchange Commission, Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. Forward-looking statements may be identified by terms such as "may," "will," "expects," "plans," "intends," "estimates," "potential," or "continue," or similar terms or the negative of these terms. Although CBMG believes the expectations reflected in the forward-looking statements are reasonable, they cannot guarantee that future results, levels of activity, performance or achievements will be obtained. CBMG does not have any obligation to update these forward-looking statements other than as required by law.

For more information, please contact:

Company Contact:Derrick C. LiHead of Strategy and Investor Relations, CBMGPhone: 917-717-0994Email: derrick.li@cellbiomedgroup.com

Investor Contact:Valter Pinto / Allison SossKCSA Strategic CommunicationsPhone: 212-896-1254 / 212-896-1267Email: cellbiomed@kcsa.com

CELLULAR BIOMEDICINE GROUP, INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(UNAUDITED)

September 30,

December 31,

2019

2018

Assets

Cash and cash equivalents

$29,035,677

$52,812,880

Restricted cash

17,000,000

-

Accounts receivable, less allowance for doubtful accounts of nil and $94,868 as of September 30, 2019 and December 31, 2018, respectively

-

787

Other receivables

591,271

101,909

Prepaid expenses

1,589,479

1,692,135

Total current assets

48,216,427

54,607,711

Investments

240,000

240,000

Property, plant and equipment, net

19,856,287

15,193,761

Right of use

14,298,613

15,938,203

Goodwill

7,678,789

7,678,789

Intangibles, net

7,521,523

7,970,692

Long-term prepaid expenses and other assets

7,640,535

5,952,193

Total assets

$105,452,174

$107,581,349

Liabilities and Stockholders' Equity

Liabilities:

Short-term debt

$14,138,419

$-

Accounts payable

5,686,023

422,752

Accrued expenses

1,477,174

1,878,926

Taxes payable

28,625

28,950

Other current liabilities

4,526,594

5,710,578

Total current liabilities

25,856,835

8,041,206

Other non-current liabilities

12,545,245

14,321,751

Total liabilities

38,402,080

22,362,957

Stockholders' equity:

Preferred stock, par value $.001, 50,000,000 shares authorized; none issued and outstanding as of September 30, 2019 and December 31, 2018, respectively

-

-

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Cellular Biomedicine Group Reports Third Quarter of 2019 Financial Results and Business Highlights - P&T Community

Anyone who specializes in neurosciences R&D has to prepare themselves for some frustration along the way. And the team at Arkuda Therapeutics can tell you all about it.

The CEO and co-founder is Gerhard Koenig, who you may recall headed up the team at Quartet Medicines, which worked on neuronal and inflammatory cells, until they folded the shop after running into a blind alley. Before that, he was CSO at Forum, which Deborah Dunsire now CEO at Lundbeck had helmed as it tried to break new ground in Alzheimers and schizophrenia.

It didnt work out either.

But even though Atlas closed the checkbook on Quartet, Bruce Booth never blamed the crew. You want to try something cutting edge here, you pay your money and you take your chances. And sometimes you write off your losses.

Thats biotech.

So now Koenig and some of the execs hes known along the way are back, knocking the door on a new approach to neurodegeneration, another high-risk, high-reward play where they are looking to break new ground. And Booth has been bankrolling the incubator work in hopes of seeing a new venture fly.

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UPDATED: Andy Plump teams with MD Anderson on the race to cell therapy 2.0 and they're already planning a pivotal - Endpoints News

The company will present data at the 61st Annual Meeting of the American Society of Hematology in Orlando, Florida between December 7 and 10.

Sangamo Therapeutics (NASDAQ:SGMO) has announced that data from its hemophilia A gene therapy clinical data and hemoglobinopathies ex vivo gene-edited cell therapy will be featured on a poster at the Annual Meeting of the American Society of Hematology (ASH).

As quoted in the press release:

Gene Therapy

The SB-525 poster will show updated Alta study data including durability of Factor VIII (FVIII) levels, bleeding rate, factor usage, and safety, for all five patients in the high dose cohort of 3e13 vg/kg, with approximately 4 months to 11 months of follow-up after treatment with SB-525.

As of the abstract submission date, four patients in the 3e13 vg/kg cohort achieved FVIII levels within the normal range with no bleeding events reported up to 24 weeks post-administration. These patients did not require FVIII replacement therapy following the initial prophylactic period of up to approximately 3 weeks post-SB-525 administration. The fifth patient in the 3e13 vg/kg cohort had only recently undergone treatment with SB-525 at the time of the abstract submission. As previously reported, one patient had treatment-related serious adverse events (SAEs) of hypotension and fever, which occurred approximately 6 hours after completion of the vector infusion and resolved with treatment within 24 hours, with no loss of FVIII expression. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer.

Click here to read the full press release.

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Sangamo to Present Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data | INN - Investing News Network

No new drug has been approved for Alzheimers since 2003, and researchers endeavoring to change that have been greeted with a graveyard of failed therapies. So when Chinese regulators waved a treatment developed by an obscure biotech through to the market albeit a conditional approval contingent upon confirmatory data it makes sense that scientists who woke up to the news were more in doubt than in awe.

While several prominent Alzheimers experts threw their weight behind the seaweed-derived therapy, oligomannate (GV-971), others contacted by Endpoints News were much more skeptical. As Green Valley Pharma has yet to release full data of the single Phase III trial on which the decision was based, most are taking a wait-and-see stance while the company prepares to launch a second, global trial in early 2020.

Among the deluge of questions surrounding the surprise approval, two seem particularly crucial: Did Green Valley offer enough credible evidence to warrant an OK? And did the drug really work as the biotech claimed?

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Data suggest gene, cell therapy trial initiations in Europe lag behind North America here's why - Endpoints News

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sigilon Therapeutics announced today that it will present data at the American Society of Hematology Annual Meeting demonstrating that its novel Shielded Living TherapeuticsTM platform for rare bleeding disorders remains viable in animal models for at least six months. The data also demonstrate dose-responsive in vivo expression of human coagulation factor VIII (hFVIII) and correction of the bleeding phenotype in immunocompetent hemophilia A mice.

The data we will be sharing at ASH are another important validation of our Shielded Living Therapeutics platform and our ability to deliver sustained production of crucial proteins, such as factor VIII, with a single treatment, said David Moller, M.D., Sigilons Chief Scientific Officer.

The ASH abstract details Sigilons novel approach to developing durable cellular therapies. The process starts with engineering human cell lines to express high levels of hFVIII without the use of viral vectors. These cells are then encapsulated inside dual-compartment spheres. The inner compartment is designed to maximize cell viability and protein production in vivo. The outer layer of the spheres contains proprietary small molecules conjugated to alginate which avoids fibrosis and immune system attack.

In the latest studies, the therapeutic cells remained viable and continued producing stable levels of hFVIII six months after implantation in mice, after which the study was terminated. If these data are replicated in the clinic, the implanted cells could potentially eliminate the need for patients to get regular factor or non-factor injections.

These data are unprecedented in demonstrating sustained expression of therapeutic proteins for the treatment of hemophilia, said Deya Corzo, M.D., Sigilons Chief Medical Officer. The results bolster our confidence in our Shielded Living Therapeutics platform, which enables a modular approach to harnessing the bodys most powerful machine the human cell to express therapeutic proteins for a wide array of indications.

Sigilon recently received Orphan Drug Designation for SIG-001, its investigational therapy for hemophilia A. Clinical trials of SIG-001 are expected to begin in 2020. We are looking forward to moving SIG-001 into the clinic as we advance our mission to replace fear with hope in patients living with chronic disease, Dr. Corzo said.

The ASH abstract 2065 is entitled: Correcting Rare Blood Disorders Using Coagulation Factors Produced in vivo by Shielded Living Therapeutics Products and will be presented on Saturday, Dec. 7 from 5:30 7:30 p.m. ET during the Gene Therapy and Transfer: Poster I Session in Hall B of the Orange County Convention Center.

About Sigilon Therapeutics

Sigilon Therapeutics is developing functional cures for chronic diseases through its Shielded Living Therapeutics platform. Sigilons therapeutics consist of novel human cells engineered to produce the crucial proteins, enzymes or factors needed by patients living with chronic diseases such as hemophilia, diabetes and lysosomal storage disorders. The engineered cells are protected by Sigilons Afibromer biomaterials matrix, which shields them from immune rejection and fibrosis. Sigilon was founded by Flagship Pioneering in conjunction with Daniel Anderson, Ph.D., and Robert Langer, Sc.D., of the Massachusetts Institute of Technology.

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Sigilon Therapeutics to Present Preclinical Data Showing Six Months Durability for Cell Therapies in Rare Blood Disorders - Business Wire