Stem Cell Clinic

Aspen Neuro Bags $6.5M to Test Parkinson’s Disease Stem Cell Therapy – Xconomy

XconomySan Diego

Nearly nine years ago Jeanne Loring and her colleagues at Scripps Research debuted a test that leveraged advances in genomics and data science to determine, without testing in animals, whether human stem cells were pluripotent, or able to become any type of cell in the body.

Being able to prove that has become increasingly important as scientists look to induced pluripotent stem cells (iPSCs)mature, specialized cells that have been reprogrammed as immature cells, regaining the capability of becoming any type of cellas material for new regenerative medicines.

Now Loring and Andres Bratt-Leal, who joined her lab in 2012 as a post-doctoral researcher, have founded a biotech that combines stem cell biology and genomics know-how to advance a potential cell therapy for Parkinsons disease.

The startup announced Thursday it raised a seed round of $6.5 million to support its work. Aspens lead drug candidate, which is in preclinical testing, is intended to replace neurons in the brains of people with the disease, which causes those cells to become damaged or die.

When people with Parkinsons disease lose neurons, they also lose a chemical messenger the cells produce, called dopamine. Without dopamine, communication between nerve cells falters, which leads to the debilitating motor problems that characterize the disease. Existing Parkinsons drugs aim to alter dopamine levels. Aspen, however, wants to fix the upstream problem that leads to those lowered levels by reconstructing patients damaged neural networks.

The cell therapy would involve harvesting patients own living cells through a skin biopsy, reprogramming them to immature cells, or iPSCs, then further engineering them to become predisposed to mature into neurons. Once enough of those cells have been grown in the lab, those neuron precursor cells would be delivered directly to the brain.

Using a patients own cells avoids the dangerous immune system reactions that can occur when donor cells are used in such therapies, and obviates the need for immunosuppression drugs. Two cell therapies that use genetic engineering have been approved by the FDA, both of which take and tweak patients T cells into treatments for cancer. Stem cell transplants have been used to treat some cancers.

Aspen worked to ensure the company could ably manufacture a so-called autologous replacement cell therapy, or one from a patients one cells, by improving the process of differentiating iPSCs into dopamine neurons, Loring says. And the group developed another predictive genomic-based test, similar to the effort Loring spearheaded nearly a decade ago to determine whether cells were pluripotent, that can detect which iPSCs are destined to become neurons.

(Bratt-Leal) put his biological engineering expertise into coming up with a way that was reproducible, that we would get the same cells no matter who we got the original cells from, she says.

The company plans to test the therapy in patients that they determine, through genomic testing, have the most common form of Parkinsons, which is referred to as sporadic and arises without a clear genetic predisposition. It also has a second treatment in the works that it intends to develop for patients with familial forms of the disease, and uses a gene editing toolyet to be selectedto alter their stem cells during the reprogramming process.

Howard Federoff, who was most recently vice chancellor for health affairs and CEO of the UC Irvine Health system, is Aspens CEO. Federoff says he has come to believe that Parkinsons patients need more than just to stabilize their disease They need to turn the clock back.

Many companies are working on drugs to treat Parkinsons, but most are meant to manage symptoms rather than reverse the disease. Levodopa, which supplants missing dopamine, is used widely, but it can cause side effects, including involuntary movement called dyskinesia; and, as the disease progresses, the drug eventually stops working between doses.

Aspen claims it is the only company working toward an autologous neuron replacement. The company, however, will need to raise a Series A round to move its drug candidates through Phase 2 proof-of-concept trials, Loring says.

The company raised its seed round from a group of investors including Domain Associates, Alexandria Venture Investments, Arch Venture Partners, Axon Ventures, OrbiMed, and Section 32. Initially, it was financed through grants from Summit for Stem Cell, a San Diego-based nonprofit.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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Aspen Neuro Bags $6.5M to Test Parkinson's Disease Stem Cell Therapy - Xconomy

LentiGlobin Gene Therapy Continues to Show Promising Results in SCD, Updated Trial Data Shows – Sickle Cell Anemia News

LentiGlobin, Bluebird Bios investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the companys Phase 1/2 HGB-206 clinical trial, according to the latest study data.

The new findings which included data from additional patients treated in the trial, updated data from those previously reported, and exploratory analyses were presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 6-10 in Orlando, Fla.

LentiGlobinisa gene therapy that has been developed to increase the levels of hemoglobin the protein that transports oxygen in the blood in people with SCD.

The therapy works by delivering functional copies of a modified form of the beta-globin gene (A-T87Q-globin gene) into patients red blood cell precursors, known as hematopoietic stem cells, or HSCs. Once these precursors differentiate, their red blood cells start producing a modified version of hemoglobin, called HbAT87Q.

By boosting the production of this anti-sickling form of the protein, LentiGlobin reduces the proportion of defective hemoglobin in patients red blood cells. That, in turn, reduces the sickling and destruction of these red blood cells and other complications associated with SCD.

The safety and efficacy of LentiGlobin is currently being evaluated in three groups identified as A-C of SCD patients participating in Bluebirds ongoing open-label, Phase 1/2 HGB-206 trial (NCT02140554).

Those in group A were treated per the original trial protocol. Meanwhile, those in groups B and C received an enhanced treatment protocol, approved in 2016, that is designed to increase the therapys efficiency. In groups A and B, patients HSCs were extracted from the bone marrow, while in group C, they were extracted from the blood.

As of the data cutoff date of August 26, 2019, seven participants in group A, two in group B, and 17 in group C had been treated with LentiGlobin. According to new data presented at the meeting, only two patients from group A required regular blood transfusions after the treatment.

In addition, the updated findings revealed that the levels of anti-sickling HbAT87Q remained stable in all participants from groups A and B over a post-treatment follow-up period of three years. Similarly, levels of total hemoglobin also were found to have remained stable in both patient groups over a two-year follow-up.

At the trial participants last visit, the median levels of anti-sickling HbAT87Q were 0.9 g/dL among those from group A, and 3.6 g/dL and 7.1 g/dL in the two patients from group B. The median levels of total hemoglobin were 9.0 g/dL among patients from group A, and 11.3 g/dL and 13.0 g/dL among those from group B.

Normal levels of hemoglobin in the blood range from 12.5 to 17.5 g/dL.

Among 12 patients from group C who were followed for at least six months, the median levels of anti-sickling HbAT87Q made up at least 40% of their total hemoglobin. At their last visit, the levels of anti-sickling HbAT87Q ranged from 2.7 to 9.0 g/dL, and the levels of total hemoglobin from 9.3 to 15.2 g/dL.

In groups A and B, LentiGlobin reduced the frequency of painful vaso-occlusive crises (VOCs) and acute chest syndrome (ACS) in the two years following treatment.

Nine patients from group C who were followed for at least six months had experienced four or more VOCs or ACS episodes in the two years prior to receiving LentiGlobin. Treatment with the gene therapy led to a reduction of 99% in the frequency of annual VOCs and ACS. In this group, there were no reports of ACS or severe VOCs for up to 21 months following treatment.

Moreover, among those from group C, LentiGlobin reduced the levels of different markers of red blood cells destruction, including reticulocytes, lactate dehydrogenase (LDH), and bilirubin.

LentiGlobins safety profile was consistent with previous data. No serious adverse events related to treatment were reported during the study. Only one mild, non-serious event of hot flush was found to be related to LentiGlobin. That event was rapidly resolved and did not require treatment.

Exploratory analyses were performed in a sub-group of patients from all three groups. In 12 participants who had been followed for at least six months, more than 70% of the individuals red blood cells were found to contain the anti-sickling HbAT87Q at the last study visit, these analyses showed. Moreover, in four of these patients, nearly all their red blood cells (90%) were positive for HbAT87Q.

In addition, exploratory analyses revealed that participants red blood cells were less prone to sickling following treatment with LentiGlobin.

At ASH, the growing body of data from our clinical studies of LentiGlobin for SCD reflects results from 26 treated patients with up to four years of follow-up, David Davidson, MD, Bluebird Bios chief medical officer, said in a press release.

We continue to observe patients treated in Group C producing high levels of gene-therapy derived anti-sickling hemoglobin, HbAT87Q, accounting for at least 40% of total hemoglobin in those with six or more months of follow-up, and exploratory assays show that HbAT87Q is present in most red blood cells of treated patients, Davidson said.

The robust production of HbAT87Q was associated with substantial reductions of sickle hemoglobin, HbS, as well as improvement in key markers of hemolysis [red blood cells destruction]. Most importantly, patients in Group C have not experienced any episodes of acute chest syndrome or serious vaso-occlusive crises following LentiGlobin for SCD treatment, he added.

The company is recruiting participants with transfusion-dependent -thalassemia (TDT) for a Phase 3 trial (NCT03207009) testing LentiGlobin. Moreover, according to the companys pipeline, there is a Phase 2/3 trial planned in sickle cell disease for this gene therapy.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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LentiGlobin Gene Therapy Continues to Show Promising Results in SCD, Updated Trial Data Shows - Sickle Cell Anemia News

Treatment Characteristics and Outcomes for Patients With Rare Forms of T-Cell Lymphoma – Hematology Advisor

Researchers evaluated treatment characteristics and outcomes of 3 rare subtypes of T-cell lymphoma, and their findings were published in the American Journal of Hematology.

The prospective study assessed treatments and clinical outcomes for patients with enteropathy-associated T-cell lymphoma (EATL), hepatosplenic T-cell lymphoma (HSTCL), and peripheral gamma delta T-cell lymphoma (PGDTCL). Patient cases were identified from the T Cell Project (ClinicalTrials.gov Identifier: NCT01142674) database, and therapies were guided by treating physicians.

Among 1553 eligible patient cases, a total of 65 patients (4.2%) were diagnosed with EATL, 31 (2%) with HSTCL, and 19 (1.2%) with PGDTCL.

Median study follow-up was 77 months for patients with PGDTCL, 64 months for patients with HSTCL, and 32 months for patients with EATL.

The 5-year overall survival (OS) rates were 30% with EATL, 40% with HSTCL, and 51% with PGDTCL. Median progression-free survival (PFS) was shortest for patients with EATL, at 7 months (95% CI, 4-10). For patients with HSTCL, median PFS was 11 months (95% CI, 8-14), and for patients with PGDTCL, median PFS was 14 months (95% CI, 6-21).

Treatment data were evaluable for 93 patients. All evaluable patients with PGDTCL and 97% of those with EATL received anthracycline-based therapies, compared with 60% of evaluable patients with HSCTL. Complete response rates to initial chemotherapy regimens were 30% for EATL, 40% for HSCTL, and 25% for PGDTCL. Autologous stem cell transplantation was used during first remission in a minority of patients with each of the 3 subtypes of T-cell lymphoma.

The study investigators highlighted the value of registries in studies of rare diseases. In the case of this study, the investigators noted a lack of standardized treatment practices for the examined T-cell lymphoma subtypes, and they recommended that patients with rare T-cell lymphoma subtypes be included in studies of novel agents.

Reference

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Treatment Characteristics and Outcomes for Patients With Rare Forms of T-Cell Lymphoma - Hematology Advisor

CHMP Issues Positive Opinion Recommending DARZALEX (Daratumumab) in Combination with Bortezomib, Thalidomide and Dexamethasone in Frontline Multiple…

Company Announcement

Copenhagen, Denmark; December 13, 2019 Genmab A/S (GMAB) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending broadening the existing marketing authorization for DARZALEX (daratumumab) in the European Union. The recommendation is for the use of DARZALEX in in combination with bortezomib, thalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant (ASCT).

We are very pleased with this positive opinion from the CHMP as, if approved, the combination of DARZALEX plus bortezomib, thalidomide and dexamethasone would be the first DARZALEX containing regimen that would be a potential treatment option for newly diagnosed patients with multiple myeloma in Europe who are eligible for ASCT, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The Type II variation application was submitted to the EMA by Janssen Pharmaceutica NV in March 2019 and was based on the Phase III CASSIOPEIA (MMY3006) study sponsored by the French Intergroupe Francophone du Myelome (IFM) in collaboration with the Dutch-Belgian Cooperative Trial Group for Hematology Oncology (HOVON) and Janssen R&D, LLC. Data from this study was published in The Lancet and presented at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting. In August 2012, Genmab granted Janssen Biotech, Inc. an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

About the CASSIOPEIA (MMY3006) studyThis Phase III study is a randomized, open-label, multicenter study, run by the French Intergroupe Francophone du Myelome (IFM) in collaboration with the Dutch-Belgian Cooperative Trial Group for Hematology Oncology (HOVON) and Janssen R&D, LLC, including 1,085 newly diagnosed subjects with previously untreated symptomatic multiple myeloma who are eligible for high dose chemotherapy and stem cell transplant. In the first part of the study, patients were randomized to receive induction and consolidation treatment with daratumumab combined with bortezomib, thalidomide (an immunomodulatory agent) and dexamethasone (a corticosteroid) or treatment with bortezomib, thalidomide and dexamethasone alone. The primary endpoint is the proportion of patients that achieve a stringent Complete Response (sCR). In the second part of the study, patients that achieved a response will undergo a second randomization to either receive maintenance treatment of daratumumab 16 mg/kg every 8 weeks for up to 2 years versus no further treatment (observation). The primary endpoint of this part of the study is progression free survival (PFS).

About multiple myelomaMultiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Approximately 16,830 new patients were expected to be diagnosed with multiple myeloma and approximately 10,480 people were expected to die from the disease in the Western Europe in 2018.2 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.3 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.4

About DARZALEX (daratumumab)DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX intravenous infusion is indicated for the treatment of adult patients in Europe: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy6. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

Story continues

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).5,6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis, NKT-cell lymphoma and B-cell and T-cell ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company has two approved antibodies, DARZALEX (daratumumab) for the treatment of certain multiple myeloma indications, and Arzerra (ofatumumab) for the treatment of certain chronic lymphocytic leukemia indications. Daratumumab is in clinical development for additional multiple myeloma indications, other blood cancers and amyloidosis. A subcutaneous formulation of ofatumumab is in development for relapsing multiple sclerosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with core sites in Utrecht, the Netherlands and Princeton, New Jersey, U.S.

Contact: Marisol Peron, Corporate Vice President, Communications & Investor Relations T: +1 609 524 0065; E: mmp@genmab.com

For Investor Relations: Andrew Carlsen, Senior Director, Investor RelationsT: +45 3377 9558; E: acn@genmab.com

This Company Announcement contains forward looking statements. The words believe, expect, anticipate, intend and plan and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with pre-clinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmabs most recent financial reports, which are available on http://www.genmab.com and the risk factors included in Genmabs final prospectus for our U.S. public offering and listing and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at http://www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab; the Y-shaped Genmab logo; Genmab in combination with the Y-shaped Genmab logo; HuMax; DuoBody; DuoBody in combination with the DuoBody logo; HexaBody; HexaBody in combination with the HexaBody logo; DuoHexaBody; HexElect; and UniBody. Arzerra is a trademark of Novartis AG or its affiliates. DARZALEX is a trademark of Janssen Pharmaceutica NV.

1 American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma.Accessed June 2016.2 Globocan 2018. Western Europe Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/926-western-europe-fact-sheets.pdf Accessed March 20183 Globocan 2018. World Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-sheets.pdf. Accessed December 2018.4 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed June 20165 DARZALEX Prescribing information, September 2019. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761036s024lbl.pdf Last accessed September 20196 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed October 20197De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.8 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.9 Krejcik MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.10Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974.

Company Announcement no. 60CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122

Genmab A/SKalvebod Brygge 431560 Copenhagen VDenmark

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Researchers ID Molecule that Appears to Halt and Reverse Scarring in Fibrotic Diseases – BioSpace

Fibrosis, or scarring, is central to a number of diseases, including cirrhosis of the liver, chronic kidney disease and several lung diseases. Generally, any organ in the body can repair itself after injury. Normally, scarring occurs and then recedes, making room for normal tissue as healing occurs. But sometimes the healing goes awry and the cells that make up scar tissue continue dividing and spreading until the scar tissue itself strangles the organ it was healing. This can cause organ failure.

Researchers at the University of California-Los Angeles Health Sciences have developed a scar-in-a-dish model derived from stem cells that can mimic fibrosis. They then identified a drug that could stop the fibrotic progression and, in further animal models, actually reverse fibrosis. They published their research in the journal Cell Reports.

Millions of people living with fibrosis have very limited treatment options, said Brigitte Gomperts, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. Once scarring gets out of control, we dont have any treatments that can stop it, except for whole-organ transplant.

The scar-in-a-dish model utilized several different types of cells derived from human stem cells. It used induced pluripotent stem cells (iPS).

Fibrosis likely occurs as the result of interactions between multiple different cell types, so we didnt think it made sense to use just one cell type to generate a scarring model, said Preethi Vijayaraj, the reports first author and an assistant adjunct professor of pediatric hematology/oncology at the David Geffen School of Medicine at UCLA and a member of the UCLA Johnsson Comprehensive Cancer Center.

The mixture of cells they grew had many types that are believed to participate in fibrosis, including mesenchymal cells, epithelial cells and immune cells. All of them maintained some plasticity, allowing them to change cells types. This is the first known model to recreate that plasticity, which is associated with progressive fibrosis.

They then placed the cells in a rigid hydrogel that was similar to the stiffness of a scarred organ. The cells behaved the same way they would to injury, producing damage signals and activating transforming growth factor beta (TGF beta), which typically stimulates fibrosis.

The use of the gel, as opposed to tissue, meant it couldnt heal itself. This allowed the researchers to test molecules on the scarring in a way that isolated the drug and scarring tissues. They tested more than 17,000 small molecules. They identified one that stopped progressive scarring and healed the damage. They believe the compound activates the cells innate wound healing processes.

This drug candidate seems to be able to stop and reverse progressive scarring in a dish by actually breaking down the scar tissue, said Gomperts. We tested it in animal models of fibrosis of the lungs and eyes and found that it has promise to treat both of those diseases.

The next steps are to determine how the drug candidate works and also screen more molecules. The drug has not been tested in humans. The therapeutic strategy is covered by a patent application the UCLA Technology Development Group filed on behalf of the Regents of the University of California, with Gomperts and Vijayaraj listed as co-inventors. Gomperts is also a co-founder and stock owner of a biotech company, InSpira, which is focused on developing the molecule and strategy for fibrosis.

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Researchers ID Molecule that Appears to Halt and Reverse Scarring in Fibrotic Diseases - BioSpace

Canine Stem Cell Therapy Market with Future Prospects, Key Player SWOT Analysis and Forecast To 2024 – Tech Estate Today

Canine Stem Cell Therapy market report examines the short-and medium-term economic and profitability outlook for Canine Stem Cell Therapy industry..

The Global Canine Stem Cell Therapy Market is poised to grow strong during the forecast period 2017 to 2027. Canine Stem Cell Therapy market is the definitive study of the global Canine Stem Cell Therapy industry. The report content includes technology, industry drivers, geographic trends, market statistics, market forecasts, producers, and raw material/equipment suppliers.

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The Canine Stem Cell Therapy industry study concludes with a list of leading companies/suppliers operating in this industry at different stages of the value chain.

List of key players profiled in the report:

VETSTEM BIOPHARMA , Cell Therapy Sciences , Regeneus Ltd. , Aratana Therapeutics , Medivet Biologics LLC , Okyanos , Vetbiologics , VetMatrix , Magellan Stem Cells , ANIMAL CELL THERAPIES, INC , stemcellvet.co.uk ,

By Product TypeAllogeneic Stem Cells, Autologous Stem cells ,

By ApplicationArthritis, Dysplasia, Tendonitis, Lameness, Others

By End UserVeterinary Hospitals, Veterinary Clinics, Veterinary Research Institutes

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Canine Stem Cell Therapy Market with Future Prospects, Key Player SWOT Analysis and Forecast To 2024 - Tech Estate Today