Category Archives: Genetic Therapy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced that new data from ongoing Phase 3 studies of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for -thalassemia gene therapy) show pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent -thalassemia (TDT) achieve and maintain transfusion independence with hemoglobin (Hb) levels that are near-normal (10.5 g/dL). These data are being presented at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

With more than a decade of clinical experience evaluating gene therapy in patients with transfusion dependent -thalassemia across a wide range of ages and genotypes, we have built the most comprehensive understanding of treatment outcomes in the field, said David Davidson, M.D., chief medical officer, bluebird bio. Seeing patients achieve transfusion independence and maintain that positive clinical benefit over time with robust hemoglobin levels reflects our initial vision of the potential of beti-cel. The accumulating long-term data demonstrating improvements in bone marrow histology, iron balance and red cell biology support the potential of beti-cel to correct the underlying pathophysiology of transfusion-dependent -thalassemia.

A total of 60 pediatric, adolescent and adult patients across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies as of March 3, 2020. In studies of beti-cel, transfusion independence is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

TDT is a severe genetic disease caused by mutations in the -globin gene that results in significantly reduced or absent adult hemoglobin (HbA). In order to survive, people with TDT maintain Hb levels through lifelong, chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Patients with transfusion-dependent -thalassemia do not make enough healthy red blood cells and cannot live without chronic transfusions; for patients that means a lifetime of necessary visits to a hospital or clinic and reliance on an often unreliable blood supply, which compounds the challenges of managing this disease, said presenting study author Professor John B. Porter, MA, M.D., FRCP, FRCPath, University College London Hospital, London, UK. These results showing patients free from transfusions and maintaining near-normal hemoglobin levels after treatment with beti-cel is a positive outcome for people living with transfusion-dependent -thalassemia. In addition, we now have more data that provide further evidence that most of these patients have a measurable improvement in markers of healthy red blood cell production.

Beti-cel is a one-time gene therapy designed to address the underlying genetic cause of TDT by adding functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). This means there is no need for donor HSCs from another person, as is required for allogeneic HSC transplantation (allo-HSCT). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived Hb, at levels that eliminate or significantly reduce the need for transfusions.

Northstar-2 (HGB-207) Efficacy

As of March 3, 2020, all 23 patients in HGB-207 were treated and have been followed for a median of 19.4 months. These patients ranged in age from four to 34 years, including eight pediatric (<12 years of age) and 15 adolescent/adult (>12 years of age) patients. Only 19 patients were evaluable for transfusion independence; four additional patients do not yet have sufficient follow-up to be assessed for transfusion independence.

Eighty-nine percent of evaluable patients (17/19) achieved transfusion independence, with median weighted average total Hb levels of 11.9 g/dL (min-max: 9.4 12.9 g/dL) over a median of 19.4 months of follow-up to date (min-max: 12.3 31.4 months). These 17 patients previously required a median of 17.5 transfusions per year (min-max: 11.5 37 transfusions per year).

Improved iron levels, as measured by serum ferritin and hepcidin levels (proteins involved in iron storage and homeostasis), were observed and trends toward improved iron management were seen. Over half of patients stopped chelation therapy, which is needed to reduce excess iron caused by chronic blood transfusions. Seven out of 23 patients began using phlebotomy for iron reduction.

Analysis of Healthy Red Blood Cell Production

In exploratory analyses, biomarkers of ineffective erythropoiesis (red blood cell production) were evaluated in patients who achieved transfusion independence in HGB-207.

The myeloid to erythroid (M:E) ratio in bone marrow from patients who achieved transfusion independence increased from a median of 1:3 (n=17) at baseline to 1:1.2 (n=16) at Month 12. Improvement of the M:E ratio, the ratio of white blood cell and red blood cell precursors in the bone marrow, suggests an improvement in mature red blood cell production. Images illustrating the bone marrow cellularity at baseline, Month 12 and Month 24 are available in the EHA25 presentation (abstract #S296): Improvement in erythropoiesis in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) in the Phase 3 HGB-207 study.

Additionally, biomarkers of erythropoiesis continue to demonstrate a trend toward normalization in patients who achieved transfusion independence, including improved levels over time of erythropoietin, a hormone involved in red blood cell production; reticulocytes, immature red blood cells; and soluble transferrin receptor, a protein measured to help evaluate iron status. The continued normalization of red blood cell production over time among some patients who achieved transfusion independence supports the disease-modifying potential of beti-cel in patients with TDT.

Northstar-3 (HGB-212) Efficacy

As of March 3, 2020, 15 patients (genotypes: 9 0/0, 3 0/ +IVS1-110, 3 homozygous IVS-1-110 mutation) were treated and had a median follow-up of 14.4 months (min-max: 1.124.0 months). Median age at enrollment was 15 (min-max: 4 33 years).

Six of eight evaluable patients achieved transfusion independence, with median weighted average total Hb levels of 11.5 g/dL (min-max: 9.5 13.5 g/dL), and continued to maintain transfusion independence for a median duration of 13.6 months (min-max: 12.2 21.2 months) as of the data cutoff.

Eighty-five percent of patients (11/13) with at least seven months of follow-up had not received a transfusion in more than seven months at time of data cutoff. These 11 patients previously required a median of 18.5 transfusions per year (min-max: 11.0 39.5 transfusions per year). In these patients, gene therapy-derived HbAT87Q supported total Hb levels ranging from 8.814.0 g/dL at last visit.

Betibeglogene autotemcel Safety

Non-serious adverse events (AEs) observed during the HGB-207 and HGB-212 trials that were considered related or possibly related to beti-cel were tachycardia, abdominal pain, pain in extremities, leukopenia, neutropenia and thrombocytopenia. One serious event of thrombocytopenia was considered possibly related to beti-cel.

In HGB-207, serious events post-infusion in two patients included three events of veno-occlusive liver disease and two events of thrombocytopenia. In HGB-212, serious events post-infusion in two patients included two events of pyrexia.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

In both Phase 3 studies, there have been no deaths, no graft failure, no cases of vector-mediated replication competent lentivirus or clonal dominance, no leukemia and no lymphoma.

The presentations are now available on demand on the EHA25 website:

About betibeglogene autotemcel

The European Commission granted conditional marketing authorization (CMA) for betibeglogene autotemcel (beti-cel; formerly LentiGlobin gene therapy for -thalassemia), marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for ZYNTEGLO, supported by data from 32 patients treated with ZYNTEGLO, including three patients with up to five years of follow-up.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Beti-cel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnea, pain in extremity and non-cardiac chest pain. Two serious adverse events (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration (FDA) granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of transfusion-dependent -thalassemia. Beti-cel is not approved in the U.S.

Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207) and NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel or LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

bluebird bio Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that the COVID-19 pandemic and resulting impact on our operations and healthcare systems will affect the execution of our development plans or the conduct of our clinical studies; the risk that the efficacy and safety results observed in the patients treated in our prior and ongoing clinical trials of beti-cel may not persist; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated with additional patients in our ongoing or planned clinical trials or in the commercial context; the risk that the FDA will require additional information regarding beti-cel, resulting in a delay to our anticipated timelines for regulatory submissions, including submission of our BLA. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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Majority of Evaluable Patients Across Genotypes Achieve Transfusion Independence and Maintain It with Near-Normal Hemoglobin Levels in Phase 3 Studies...

bluebird on the Go with Multiple Positive Trial Data

bluebird bio (BLUE) announced data from its different trials, including Phase 1/2 clinical trial for sickle cell disease and from a Phase 3 clinical trial for transfusion-dependent beta-thalassemia patients.

Ongoing Phase 1/2 clinical trial HGB-206 aims to assess the potential of LentiGlobin in treating adolescents and adults with sickle cell disease. As on the data cutoff date, 37 patients had been treated. Group C showed promising results as all the patients included in the cohort stopped regular blood transfusions. These patients also remained transfusion-free for the time period of three months after the treatment.

Sixteen patients who were followed up for minimum six months maintained the median levels of gene therapy-derived anti-sickling hemoglobin. It also contributed to minimum 40 percent of total hemoglobin. 14 patients with previous background of acute chest syndrome (ACS) and vaso-occlusive crisis (VOC) who have been observed for a minimum time period of six months showed a 99.5 percent drop in the annualized rate of ACSs and VOCs.

Its ongoing Phase 3 clinical trials aimed to test the prospects of Zynteglo, formerly known as LentiGlobin, in treating adolescent and adult patients suffering from a range of genotypes of transfusion-dependent beta-thalassemia. Phase 3 of the Northstar-2 study involved 23 patients who were treated and observed for a median time period of 19.4 months. 89.5 percent of evaluable participants numbering 19 showed transfusion independence. Earlier, the 17 needed a median of 17.5 transfusions each year.

The Northstar-3 study involved 15 patients with genotypes of different variations. These patients were treated and then observed for a median time period of 14.4 months. 75.0% (n=6/8) reported to be transfusion independent. 84.6% (n=11/13) with a minimum of seven months' follow-up did not require a transfusion in more than seven months. Earlier, 11 of the patients needed a median of 18.5 transfusions every year.

bluebirds gene therapy involves extracting a patients stem cell and then altering it with new genetic instructions. Such altered stem cell is then infused back into the patients body. David Davidson, M.D., chief medical officer, bluebird bio, said, Vaso-occlusive crises (VOCs) are the painful, life-threatening episodes that are the primary clinical manifestation of sickle cell disease. The nearly complete elimination of VOCs that we saw in this study is impressive and demonstrates the potential of LentiGlobin for SCD as a treatment for this serious disease.

The Northstar-2 study showed three and two serious occurrences of veno-occlusive liver disease and thrombocytopenia respectively. Northstar-3 showed two serious occurrences of pyrexia. However, there was no reporting of any death, graft failure or leukemia. Zynteglo is already approved in the European Union, and the company is looking to start treating patients in Germany in the near future. It also plans to apply for the FDA approval in mid-2021.

For its Phase 1/2 trial in sickle cell anemia, an earlier report had shown significant improvement in VOCs and ACS after the gene therapy. Pursuant to these results, the company consulted with the FDA to alter the primary endpoint of the trial from improvement in hemoglobin measurements to the elimination of VOCs.

BeiGene (BGNE) announced new data from its trials aimed at assessing the potential of BTK inhibitor Brukinsa (zanubrutinib) and PD-1 inhibitor tislelizumab in treating various blood cancers. Brukinsa is a small molecule inhibitor of Brutons tyrosine kinase. It is currently being assessed as a monotherapy and in combination with other therapies of treating a wide range of B-cell malignancies.

For Phase 1/2 study of Brukinsa in patients suffering from B-cell malignancies, the overall response rate was 80 percent, while complete response rate was 15 percent. Further, the partial response rate for the trial was 65 percent. The median time to response was measured at 2.8 months. The progression-free survival rate and overall survival rate at 24 months were 59.4 percent and 83.2 percent respectively. Zanubrutinib was found to be well-tolerated in patients with R/R MZL, however, all patients experienced at least one adverse event.

The company also presented data from a Phase 2 trial of zanubrutinib with rituximab in patients suffering from R/R NHL. The trial had 41 participants, including 20 patients suffering from non-GCB DLBCL who previously received standard anthracycline rituximab-based treatment. For patients with R/R non-GCB DLBCL, the ORR was 35 percent and the CR and PR rates were 5 percent and 30 percent, respectively. For patients with R/R FL, the OR was 56.3 percent, with CR and PR at 19 percent and 38 percent, respectively.

Brukinsa is already approved in the United States for treating adult patients suffering from mantle cell lymphoma who have been given minimum one prior therapy. It was given the approval under accelerated approval pathway on the basis of its overall response rate. The drug is approved in China for treating MCL in adult patients who have received at least one prior therapy and CLL or SLL in adult patients who have received at least one prior therapy. However, the drug is not approved outside of the United States and China.

TG Therapeutics (TGTX) reported positive interim data from its Phase 1 parallel dose-escalation study of TG-1701. The drug candidate is being tested as a monotherapy and in combination with U2 for treating relapsed/refractory B-cell malignancies.

The data pertained to 82 patients suffering from the condition. Sixty-nine patients were given single agent TG-1701, out of which 25 were included in the monotherapy dose escalation cohort of the study and were given the drug candidate in 100mg to 400mg dosage once a day daily. The remaining 44 patients were included in the monotherapy dose expansion arm and were administered 200mg of the drug candidate. The TG-1701 plus U2 dose escalation portion of the study treated an additional 13 patients.

For the monotherapy dose escalation cohort with 25 patients, the drug candidate showed partial response for all the dose levels in CLL, MCL, WM and SLL. For the monotherapy dose expansion cohort, the overall response rate was recorded at 92 percent in CLL patients. MCL patients and WM patients showed 33 percent and 86 percent ORR respectively. The combination of the drug candidate with U2 showed positive clinical activity with a 77 percent ORR for all disease types. It included complete response rate in three patients. The company continues with dose escalation.

TG-1701 showed a positive preliminary safety profile for all dose levels. No patient had to discontinue the treatment, but 3 percent of the patients had to reduce the dose due to the occurrence of treatment-related adverse events. The company also provided data pertaining Phase I/Ib study aiming to assess the potential of ibrutinib in blend with umbralisib for patients with relapsed/refractory CLL or MCL.

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bluebird On The Go, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

The global Genetic Modification Therapies market report provides geographic analysis covering regions, such as North America, Europe, Asia-Pacific, and Rest of the World. The Genetic Modification Therapies market for each region is further segmented for major countries including the U.S., Canada, Germany, the U.K., France, Italy, China, India, Japan, Brazil, South Africa, and others.

The global Genetic Modification Therapies market is expected to exceed more than US$ 3.5 Billion by 2024 at a CAGR of 34% in the given forecast period.

Genetic modification therapies, significantly gene therapy and RNA therapy, have existed for many years, with very little clinical success. However, recent enhancements in these therapies, together with higher delivery systems, additional economical and sturdy gene expression constructs, precise polymer editing tools, have brought this industry to the forefront, and its currently poised for explosive growth within the coming back years.

Browse Full Report: https://www.marketresearchengine.com/genetic-modification-therapies-market

Because of the potentially curative nature of those medicines theres monumental potential in several applications, starting from cancer to neurology to rare diseases. Genetic modification therapies represent consecutive wave of medicines with monumental potential for treating and curing draining and high diseases. As a result of its wide scope, genetic modification therapy can play a vital role within the future world medical economy.

Continuing advances in key technologies like DNA editing, viral design and production, and gene expression, further as a pressing medical want in several serious and enervating disorders, are driving the expansion of the marketplace for genetic modification therapies. Developments in these multidisciplinary fields promise to advance the genetic modification therapies trade and build distinctive market opportunities.

The overall market is anticipated to witness important growth in opportunities for a spread of stakeholders within the returning decade. its necessary to spotlight that many technology suppliers, reaching to develop and / or support the event of gene therapies, with improved effectiveness and safety, have designed and already introduced advanced platforms for the engineering of vectors. Innovation during this domain has additionally semiconductor diode to the invention of novel molecular targets and strong the analysis pipelines of corporations targeted during this house. the potential to focus on numerous therapeutic areas is taken into account to be amongst the foremost outstanding growth drivers of this market.

Market Insights

The global Genetic Modification Therapies market is segregated on the basis of Platform Technology as Gene editing, Gene Therapies, Genetically Modified Cell Therapies, and RNA Therapies. Based on Delivery Technologies the global Genetic Modification Therapies market is segmented in AAV, Adenovirus, Lentivirus, Retrovirus, Other Viral, and Nonviral Based on End-User Industry the global Genetic Modification Therapies market is segmented in Hospitals, Diagnostics and Testing Laboratories, Academic and Research Organizations, and Others.

Based on Disease, the global Genetic Modification Therapies market is segmented in Cardiology, Oncology, Ophthalmology, Hematology, Musculoskeletal, Neurology, Rare Diseases, Other Indications.

Competitive Rivalry

4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore, Jivana Biotechnology, and others are among the major players in the global Genetic Modification Therapies market. The companies are involved in several growth and expansion strategies to gain a competitive advantage. Industry participants also follow value chain integration with business operations in multiple stages of the value chain.

The Genetic Modification Therapies Market has been segmented as below:

The Genetic Modification Therapies Market is segmented on the lines of Genetic Modification Therapies Market, By Platform Technology, Genetic Modification Therapies Market, By Delivery Technologies, Genetic Modification Therapies Market, By End-User Industry, Genetic Modification Therapies Market, By Disease, Genetic Modification Therapies Market, By Region and Genetic Modification Therapies Market, By Company.

Genetic Modification Therapies Market, By Platform Technology this market is segmented on the basis of Gene editing, Gene Therapies, Genetically Modified Cell Therapies and RNA Therapies. Genetic Modification Therapies Market, By Delivery Technologies this market is segmented on the basis of AAV, Adenovirus, Lentivirus, Retrovirus, Other Viral and Nonviral. Genetic Modification Therapies Market, By End-User Industry this market is segmented on the basis of Hospitals, Diagnostics and Testing Laboratories, Academic and Research Organizations and Others. Genetic Modification Therapies Market, By Disease this market is segmented on the basis of Cardiology, Oncology, Ophthalmology, Hematology, Musculoskeletal, Neurology, Rare Diseases and Other Indications. Genetic Modification Therapies Market, By Region this market is segmented on the basis of North America, Europe, Asia-Pacific and Rest of the World. Genetic Modification Therapies Market, By Company this market is segmented on the basis of 4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore and Jivana Biotechnology.

The report covers:

Global Genetic Modification Therapies market sizes from 2015 to 2024, along with CAGR for 2018-2024Market size comparison for 2017 vs 2024, with actual data for 2017, estimates for 2018 and forecast from 2019 to 2024Global Genetic Modification Therapies market trends, covering comprehensive range of consumer trends & manufacturer trendsValue chain analysis covering participants from raw material suppliers to the downstream buyer in the global Genetic Modification Therapies marketMajor market opportunities and challenges in forecast timeframe to be focusedCompetitive landscape with analysis on competition pattern, portfolio comparisons, development trends and strategic managementComprehensive company profiles of the key industry players

Report Scope:

The global Genetic Modification Therapies market report scope includes detailed study covering underlying factors influencing the industry trends.

The report covers analysis on regional and country level market dynamics. The scope also covers competitive overview providing company market shares along with company profiles for major revenue contributing companies.

The report scope includes detailed competitive outlook covering market shares and profiles key participants in the global Genetic Modification Therapies market share. Major industry players with significant revenue share include 4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore, Jivana Biotechnology, and others.

Reasons to Buy this Report:

Gain detailed insights on the Genetic Modification Therapies industry trendsFind complete analysis on the market statusIdentify the Genetic Modification Therapies market opportunities and growth segmentsAnalyse competitive dynamics by evaluating business segments & product portfoliosFacilitate strategy planning and industry dynamics to enhance decision making

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Table of Contents:

IntroductionResearch MethodologyExecutive SummaryMarket Overview4.1 Introduction4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.2.4 Challenges4.2 Porters Five Force AnalysisGenetic Modification Therapies Market, By Platform TechnologyGenetic Modification Therapies Market, By Delivery TechnologiesGenetic Modification Therapies Market, By End-User IndustryGenetic Modification Therapies Market, By DiseaseGenetic Modification Therapies Market, By GeographyCompetitive InsightsCompany Profiles11.1 4D Molecular Therapeutics11.1.1 Company Overview11.1.2 Product/Service Landscape11.1.3 Financial Overview11.1.4 Recent Developments11.2 Abeona Therapeutics11.2.1 Company Overview11.2.2 Product/Service Landscape11.2.3 Financial Overview11.2.4 Recent Developments11.3 Beam Therapeutics,11.3.1 Company Overview11.3.2 Product/Service Landscape11.3.3 Financial Overview11.3.4 Recent Developments11.4 Casebia Therapeutics,11.4.1 Company Overview11.4.2 Product/Service Landscape11.4.3 Financial Overview11.4.4 Recent Developments11.5 Editas Medicine,11.5.1 Company Overview11.5.2 Product/Service Landscape11.5.3 Financial Overview11.5.4 Recent Developments11.6 Fate Therapeutics,11.6.1 Company Overview11.6.2 Product/Service Landscape11.6.3 Financial Overview11.6.4 Recent Developments11.7 GE Healthcare,11.7.1 Company Overview11.7.2 Product/Service Landscape11.7.3 Financial Overview11.7.4 Recent Developments11.8 Hitachi Chemical Advanced Therapeutics,11.8.1 Company Overview11.8.2 Product/Service Landscape11.8.3 Financial Overview11.8.4 Recent Developments11.9 Immunocore,11.9.1 Company Overview11.9.2 Product/Service Landscape11.9.3 Financial Overview11.9.4 Recent Developments11.10 Jivana Biotechnology,11.10.1 Company Overview11.10.2 Product/Service Landscape11.10.3 Financial Overview11.10.4 Recent Developments

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Genetic Modification Therapies Market 2019 | How The Industry Will Witness Substantial Growth In The Upcoming Years | Exclusive Report By MRE - Cole...

(Representative image) by Dr. Indumathi MariappanResearch Scientist, LV Prasad Eye Institute, Hyderabad

Retinal Blindness

Millions of people the world over suffer visual disability as a result of retinal dystrophy that involves the death of retinal cells that are important for the light sensing function of the eye. Enormous progress has been made in other blinding conditions involving the cornea, lens, among others. However, the retinal dystrophies and optic nerve atrophies do not have any proven therapy till date. The major forms of retinal dystrophies such as Age-related macular degeneration (AMD), retinitis pigmentosa (RP), Lebers congenital amaurosis (LCA), Stargardts disease etc. are either inherited disorders or developed with aging. In most cases, the retinal cells are present at birth, but undergo gradual death during the later stages of life. It is typically characterized by initial symptoms of low vision and night blindness during early childhood, which progresses to severe visual impairment and total blindness at different stages of adulthood. Inherited defects in many genes involved in retina-specific functions and vitamin A metabolism are linked to various forms of retinal dystrophies. These genetic defects affect the normal cellular functions of the retina, leading to gradual cell death and ultimately the patient becomes legally blind.

Recent Technologies and Novel Treatment Options

The current modalities for the treatment of such patients mainly include dietary supplements, visual aids and rehabilitation support. However, a radical approach is required either to preserve or to restore visual function in these patients. Some of them include the replacement of either the lost retinal cells or the defective genes within the surviving, but non-functional retinal cells. This has been the principle behind the massive efforts involved in the development of cell and gene-based therapies. They are currently at different stages of product development and clinical trial evaluation. In cell therapy, normal retinal cells are prepared from specialized stem cells and are injected into the eye to replace the lost cells and to restore retinal functions. Clinical safety trials using cell therapy are ongoing in many countries such as USA, Japan, UK and others (Weblinks 1-4). In gene therapy, the prime strategy is to introduce a normal copy of the affected gene into the surviving retinal cells of the patient, to restore normal cellular functions and improvements in vision. This is achieved by engineering safe viral vectors to carry a normal copy of the desired gene as their cargo. When injected into the eye, the viruses can infect the retinal cells once and deliver the normal gene to restore cellular functions (Weblinks 5-7). A step further is an advanced method of DNA microsurgery, wherein, the defective part of the retinal cell DNA is precisely edited to correct the genetic defect and to restore cellular functions. This could be achieved using the latest gene editing tools such as ZFNs, TALENs, CRISPR/Cas systems etc. These are naturally occurring molecular scissors, employed as host defense mechanism and immune memory to combat viral infections in different species of bacteria. These systems are now engineered to enable DNA and RNA editing in almost any living cells. Such tools are now combined with either cell therapy or gene therapy to develop novel drugs for the treatment of various inherited genetic diseases (Weblink 8).

Gene therapy products approved for clinical use:

LUXTURNATM (Weblink 5)

This is the first commercial gene therapy drug approved by the US-FDA and European Commission for the treatment of an early childhood retinal dystrophic condition called the Leber Congenital Amaurosis 2 (LCA2). This disease is caused due to genetic defects in the gene called RPE65. LUXTURNA (AAV2-hRPE65v2 or Voretigene neparovec-rzyl) is an engineered adeno-associated virus 2 (AAV2) vector carrying a normal copy of the human RPE65 gene. This product was developed and marketed by Spark Therapeutics, a US-based startup now owned by Roche, a Swiss pharma company.

This drug has been tested on 20 patients, aged 3 years or older, in a randomized, controlled, open label, phase 3 interventional clinical trial at two sites in the US from June 2015. All treated individuals showed significantly improved functional vision, with no product-related serious adverse events or deleterious immune responses. The treated patient will be followed for further 15 years until March 2030 to assess the long-term retinal gene expression and stable maintenance of functional vision. It is administered as a onetime injection behind the retina of an eye of patients genetically diagnosed to carry mutations in RPE65 gene and also have sufficient viable retinal cells. It is priced at $850,000 for two eyes in the US and UK, which translates to about 6.5 crores in Indian rupees.

Many such gene therapy vectors are currently under clinical trial evaluation for the delivery of other retinal gene such as REP1, PDE6B, RPGR, OAT (Ornithine aminotransferase), MERTK, sFLT1etc.

EDIT101 (Weblink 8)

This is the first gene editing based drug approved by US-FDA, for the treatment of another early childhood retinal dystrophic condition called LCA10, caused by defects in the CEP290 gene. Here, it is important to understand that a gene editing approach is different from a gene therapy. In gene therapy, a normal copy of entire gene is delivered to the retina to complement the defective gene. In CRISPR/Cas9 based gene editing, only the mutated region of the gene is edited/corrected in situ inside the target cells. This is an attractive approach for correcting a variety of gene mutations, especially those in large genes which exceed the cargo capacity of the commonly used AAV-based gene therapy vectors.

EDIT101 (AGN-151587) is an engineered adeno-associated virus 5 (AAV5) vector carrying a CRISPR/Cas9 based DNA editing machinery to locate and remove a specific mutation hotspot within the intron 26 of human CEP290 gene. When injected behind the retina, the virus will infect the surviving photoreceptor cells and deliver the CRISPRs to enable mutation editing. Successful DNA edits in photoreceptor cells would inactivate a spurious splice site created by the mutation and restore normal protein expression and retinal function.

Preclinical testing in mice and monkey eyes has proved significant edit efficiency of up to 28%, which was above the expected 10% threshold required for clinical efficacy in human trials. This drug was developed by the gene editing company, Editas Medicine, Inc. and is being tested in 18 participants in a Phase 1/2 clinical trial sponsored by Allergan, at four sites in the US from March 2019 and the outcomes are awaited.

Similar gene editing strategy is being explored at different centers for mutation correction in other retinal genes such as KCNJ13, RP1, USH2A, MYO7A, RDH12 etc.

Who can benefit?

Both gene therapy and gene editing approaches have opened up newer hopes for the treatment of various genetic condition affecting different cell types of the body. However, only a small subset of patients can benefit from such therapies at the moment. Such treatment considerations require a thorough genetic screening/genotyping to confirm the identity of the gene affected in a specific patient. Further, the patients should retain some viable cells in the retina for the treatment to be clinically effective.

Research efforts in India

Many labs in the country are developing gene therapies and gene editing based therapeutics for the treatment of various diseases affecting the blood, retina, liveretc. Researchers at the CMC, Vellore, CSIR-IGIB, Delhi, CSIR-CCMB, Hyderabad are developing gene therapeutics for the treatment of different forms of blood disorders. Narayana Nethralaya, Bangalore is engaged in developing AAV-based gene therapies for various retinal dystrophies. Our lab at the LV Prasad Eye Institute is collaborating with the research teams at IIT-Kanpur and CSIR-IGIB, Delhi to develop modified gene therapy vectors for retinal gene delivery and cell-based therapies using CRISPR edited stem cells and retinal cells respectively.

The way forward

As of May 2020, the RetNet database lists about 271 genes to be associated with different forms of retinal dystrophies. This requires a larger library of gene delivery vectors to be developed and made available at affordable costs for the treatment of a large number of patients. This mandates the need for developing indigenous and cost-effective therapeutics and ICMR has set up a dedicated task force on gene therapy research, to identify and support promising research ideas in this newly emerging area of biomedical research. A national guideline for gene therapy product development and clinical trials has been jointly formulated and released by the DBT and ICMR in 2019. It is hoped that the streamlined regulatory framework would fast track our basic and translational research efforts into developing novel and cost-effective treatment options in the near future.

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Gene Therapy and Editing : Novel options for inherited retinal blindness - ETHealthworld.com

By John Vandermosten, CFA

TSX:PMN.TO | OTC:ARFXF

Neurodegenerative diseases continue to afflict millions of people worldwide and effective therapies have remained elusive. The best known, most prevalent neurodegenerative pathosis is Alzheimers Disease (AD) but others, such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), are also important diseases that lack an effective therapy. There are no approved disease modifying therapies available for these conditions and only symptomatic treatments are available. However, the latest developments in gene therapy have added another dimension to the fight against neurodegenerative disease with the introduction of intrabodies. These intracellular antibodies have several beneficial characteristics including an ability to bind to misfolded proteins inside the cell and prevent the formation and spread of unwanted aggregates. Combined with output from ProMIS (TSX:PMN.TO) (OTC:ARFXF) computational algorithm, the intrabodies can be very specific, neutralizing only the toxic forms of proteins while sparing normal forms that have important physiological functions.

Antibodies in Neurodegenerative Disease

In all of its forms, neurodegenerative disease is one of the most pressing challenges that modern healthcare faces. It presents detrimental physiological, economic and social effects. Families watch as their loved ones become unable to conduct normal daily tasks and lose the ability to think and control their bodies1. AD, ALS and FTD are all characterized by the accumulation of toxic aggregates that kill neurons. Harnessing the bodys cellular machinery by applying recent advances in viral vectors allows the cell to be used to manufacture the very antibodies that are necessary to stem the spread of neurodegenerative disease.

Antibodies, also known as immunoglobulins, are proteins produced by the body in response to foreign antigens, most commonly bacteria and viruses. Antibodies can be thought of as flags to mark pathogens for sequestering and destruction; the properties of the antibody binding sites determine the antibodys specificity. Antibodies can now be engineered to target specific antigens, a technique currently being exploited across a multitude of indications2. Antigens can include proteins, sugars, or even nucleic acids. The antigens binding site is called the epitope3. Monoclonal antibodies (mAb) can be designed to target the epitope and be recognized by the human immune system.

Shortcomings in Current Approaches

Previous AD-targeted antibody therapies included Roche's gantenerumab, Eli Lilly's solanezumab, and Pfizer's bapineuzumab; these candidates made it to the final stages of clinical trials, but did not progress further. The results showed poor efficacy, highlighting the importance of 1) specificity and 2) identifying the right target.

Of recent interest has been Biogens aducanumab, another amyloid- directed antibody. Aducanumabs clinical trials showed that higher doses aducanumab had statistically significant benefits4. While aducanumab showed that intravenous A-targeted mAb could be effective, modest efficacy, possible dose-limiting edema (ARIA-E) and the necessity of large, frequent dosing indicated there was still more work to be done.

The previous mAb trials have taught invaluable lessons about developing therapies for neurodegenerative diseases, and have elucidated fundamental flaws in intravenously administered mAb. These shortcomings include high cost, frequent dosing, low permeability across the blood-brain barrier, absence of intracellular activity5 and low specificity. A new approach, known as vectorized antibodies, can be used to neutralize misfolded proteins before the leave the cell and dramatically improve the efficacy and safety of neurodegenerative therapies.

Vectorized Intrabodies

Vectorized intrabodies may provide a new treatment pathway that can address the shortcomings of intravenous mAb approaches. To achieve lasting benefit at the minimum dose necessary, viral vectors can be used to deliver the genetic sequence for therapeutic mAb expression in target cells.

A variety of viral vectors have been explored, each with its own advantages. In general, a viral vectors desirability relates to its expression duration, payload capacity and immunogenicity. The most common viral vectors for gene therapy are adenoviruses, adeno-associated viruses (AAV), herpes simplex viruses (HSV), and retroviruses. AAVs and retroviruses can achieve expression in cells that will last for years. The virus with the highest carrying capacity is the HSV at 40kB and the viruses with least immunogenicity are AAVs and retroviruses. Retroviruses integrate their payload into the host genome, causing a permanent modification, but risk mutagenesis. With low immunogenicity and long duration of expression, AAVs have become popular in gene therapy applications6.

Comparison of Viral Vectors for Gene Therapy7

Depending on the target cells, a variety of vector-administration approaches can be used. In the case of the brain, crossing the blood brain barrier is a challenge for viruses and delivery is accomplished either via intracerebral or intraventricular injection. Intracerebral injections allow delivery to a specific part of the brain, while intraventricular exposes the whole brain. Once administered, the AAVs deliver the genetic sequences for mAbs to the target cells and the cells produce the mAb. Intracerebral injection of AAVs has been shown to be safe in humans8.

Apart from the convenience and potential cost savings of lasting mAb expression, by expressing the antibody in the cell (intrabody), targeted proteins can be destroyed before ever leaving the cell. For AD, intrabodies can be used to mark neurotoxic A-oligomers for degradation9. In tauopathy, such as AD and PD, the majority of pathogenic (hyperphosphorylated) tau remains in the cell, and a tau-targeted intrabody can mark hyperphosphorylated tau for degradation, or simply competitively bind tau, minimizing the formation of tau-inclusions10. Externally administered mAb would have limited, if any, efficacy against targets inside cells.

Anti-tau intrabodies have already been studied in mice and the results support neuroprotectivity of tau-targeted immunotherapy11. Gallardo et al. fused ubiquitin, an intracellular degradation signaling molecule, to tau-targeting antibodies, transfected using AAV, and observed substantially decreased intracellular tau levels. While this particular study did not evaluate the mice behaviorally, others have, and tau-directed immunotherapy was shown to elicit behavioral improvements. The therapeutic genre has shown efficacy in mice, and remains to be seen whether it is effective in man.

Specificity and ProMIS Discovery Engine

The importance of specificity has been emphasized by ProMIS in many of its presentations. Many developed antibodies target with a scattershot approach in the hope that enough drug will sufficiently bind to disease causing proteins to have a beneficial effect overall without being diluted by binding non-disease related proteins. ProMIS approach to disease modifying therapy relies not on hope, but rather specificity. Only misfolded toxic species of amyloid-, -synuclein and TDP43 should be bound. Normal forms of these proteins have important physiological functions. Amyloid- aids in synaptic remodeling, -synuclein helps with DNA repair and TDP43 monomer and homodimer play a role in RNA transport. Broadly targeting both toxic and normal forms of proteins will not only miss the intended therapeutic effect but also result in serious side effects that may be as debilitating as the disease itself. To combat the extensive reach of non-specific antibodies, ProMIS employs its proprietary discovery platforms that are able to predict unique epitopes on misfolded toxic forms of proteins implicated in disease. This specificity allows for lower doses of drug to be used and avoids negative outcomes from binding to necessary healthy proteins.

The scientists leading ProMIS have been able to merge chemistry, biology and physics to optimize drug design by employing proprietary algorithms that are able to identify high probability conformations of misfolded proteins. In other words, they have used cutting edge technology to make a better drug.

This type of achievement would not have been possible years ago due to the computational burden required to solve the complex algorithm. However, as technology plays an even larger role in the advancement of medicine and science, ProMIS is in a position to specifically neutralize misfolded proteins in neurodegenerative disease.

Summary

Neurodegenerative diseases persist as a modern healthcare challenge. With the aging population, the number of patients suffering from this affliction will become an increasingly large burden unless we can find an effective solution. There are no disease modifying therapies for the group of neurodegenerative diseases that dominate the space; however, much progress has been made. We now understand that it is the toxic forms of certain proteins that cause many of the neurodegenerative diseases. We also understand that the normal forms of these proteins have beneficial physiological functions that cannot be disrupted without causing negative and damaging side effects. With increased understanding of these pathologies at the protein/cellular level combined with the use of viral vectors to deliver DNA medicines and the ability to identify epitopes specific to the damaging forms of misfolded proteins we have achieved material advancements. This approach provides a new paradigm of therapy that can address neurodegenerative disease even before it leaves the cell.

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PMN.TO: Vectorized Antibodies: Cutting Edge Neurodegenerative Therapy - Zacks Small Cap Research

MILAN--(BUSINESS WIRE)--Epsilen Bio, a biotechnology company developing transformative therapies for patients affected by underserved medical conditions through stable epigenetic silencing of genes involved in pathological processes, announced today the appointments of Julia Berretta, Ph.D, as Chief Executive Officer, and Mathieu Simon, M.D., as Chairman of the Board.

The company also announced it has entered into a strategic collaboration with the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), a world-renowned research center devoted to gene therapy, directed by Professor Luigi Naldini. Within the collaboration Epsilen Bio will further develop the epigenetic silencing technology with the group of Epigenetic Regulation and Targeted Genome Editing led by Dr. Angelo Lombardo, and exclusively in-licensed by Epsilen Bio.

The announcements follow the successful close of a seed financing of 2.3 million from Sofinnova Partners, a leading European life sciences venture capital firm, through the Sofinnova Telethon Fund. The fund is the largest in Italy dedicated to early-stage biotech startups targeting cures for rare and genetic diseases.

Dr. Simon and Dr. Berretta join Board Members Paola Pozzi, Partner at Sofinnova Partners, Luca Guidotti, Deputy Scientific Director of the IRCCS Ospedale San Raffaele, and Francesca Pasinelli, CEO of Fondazione Telethon and Board Observer.

Dr. Berretta is also CEO of Genespire, a gene therapy company that is part of a series of investments recently made by Sofinnova Partners. Additionally, she is an independent Board member of Treefrog Therapeutics, an innovative stem cell company. Previously, Dr. Berretta was part of the Executive Committee of Cellectis S.A., a Nasdaq-listed clinical stage gene editing company developing CAR-T cell therapies for cancer, where she led business development as well as strategic planning.

Dr. Simon is Chairman of the Board of Idorsia and Independent Board Member of VAXIMM and Lysogene. He is also a member of the AFFIMED supervisory board. Previously, Dr. Simon was Chief Operating Officer of Cellectis SA and CEO of Cellectis Therapeutics. He was the former head of global pharma operations at Pierre Fabre and also held EU regional management roles and senior corporate functions at Wyeth Pharmaceuticals.

We are excited to have Dr. Berretta and Dr. Simon joining Epsilen Bio, said Ms. Pozzi. These appointments add both an industrial and an international perspective to the company, and we are delighted to support such a distinguished global team working on highly transformative science.

Epsilen Bios scientific co-founder, Dr. Angelo Lombardo, added, Dr. Berretta and Dr. Simon have a deep knowledge of the pharmaceutical and the biotechnology industry. Their expertise will be crucial in helping to translate the science we developed at SR-Tiget into candidate therapeutic products for patients affected by underserved medical conditions.

I am extremely pleased to join Epsilen Bio as Chairman of the Board, Dr. Simon said The companys unique technology of episilencing, developed at SR-Tiget by Dr. Lombardo and Pr. Naldini, has the potential to be a game changer in the field given its versatility and applicability to a variety of diseases.

About Epsilen Bio

Epsilen Bio is a biotechnology company developing transformative therapies for patients affected by underserved medical conditions, through stable and long-lasting epigenetic silencing of genes involved in pathological processes. Based in Milan, Italy, Epsilen Bio was founded in December 2019 by the renowned gene therapy experts Dr. Angelo Lombardo and Prof. Luigi Naldini, as well as Fondazione Telethon and Ospedale San Raffaele. Epsilen Bio is a spin-off of SR-Tiget, a world-leading cell and gene therapy research institute, and received seed funding from Sofinnova Partners. For more information, please visit: http://www.epsilenbio.com

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management. For more information, please visit:www.sofinnovapartners.com

About SR-Tiget

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

About Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit: http://www.telethon.it

About Ospedale San Raffaele

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctor to grow professionally. For more information, please visit: http://www.hsr.it

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Epsilen Bio appoints Julia Berretta as Chief Executive Officer and Mathieu Simon as Chairman of the Board - Business Wire