Category Archives: Gene therapy

SAN DIEGO, April 28, 2020 /PRNewswire/ --Denovo Biopharma LLC, a pioneer in applying precision medicine to develop innovative therapies, today announced it is acquiring Tocagen's retroviral replicating vector platform (RRV) in its entirety, including its investigational gene therapy and drug regimen for oncology: Toca 511 and Toca FC (now known as DB107), and several early-stage development programs, including programs targeting PD-L1 and other immunooncology targets. This acquisition is pending the close of Tocagen's anticipated merger with Forte Biosciences. These programs greatly expand Denovo's product portfolio into gene therapy and immunooncology, yet remain consistent with Denovo's precision medicine approach.

Denovo Biopharma provides novel, proprietary biomarker approaches to personalized drug development, including re-evaluating drugs that failed in general patient populations. The company has the first platform for de novo genomic biomarker discovery using archived clinical samples. By retrospectively identifying biomarkers correlated with responses to drugs, Denovo enables clinical trials in targeted patient populations while optimizing efficacy, safety and tolerability. http://www.denovobiopharma.com . (PRNewsFoto/Denovo Biomarkers)

Thelead acquired asset, DB107, is an innovative approach utilizing a proprietary gene therapy platform, RRV, combined with a prodrug, to selectively infect and kill cancer cells while stimulating a robust and durable anti-cancer immune response against a tumor with minimal toxicity. DB107 has been tested clinically in solid tumors including recurrent high grade glioma and colorectal cancer, most recently in a randomized 403-patient Phase 3 trial. Although the Phase 3 trial results were negative overall, there were subsets of patients who showed signs of beneficial activity of DB107. After the acquisition, Denovo will use its unique biomarker platform to search for pharmacogenomic predictor for DB107 efficacy.

"With this strategic acquisition, Denovo gains a pioneering gene therapy platform with unlimited potential. Together with Denovo's world leading precision medicine capabilities, the RRV platform along with lead product candidate DB107 presents us with an unprecedented opportunity to effectively target a wide range of oncology indications," said Wen Luo, Ph.D., Denovo's Chief Executive Officer. "DB107's Phase 3 trial aimed to treat recurrent high grade glioma, which primarily consists of glioblastoma (GBM). This asset, together with our DB102 asset to be tested in the front-line treatment of GBM, positions Denovo as a leader in the field of neuro-oncology that continues to have a high unmet need."

"We are pleased to have a company with the capabilities of Denovo Biopharma acquiring our gene therapy platform technology to build upon the work that was done by our employees and investigators, as well as the patients in our clinical trials," said Marty J. Duvall, Tocagen's Chief Executive Officer.

About Denovo BiopharmaDenovo Biopharma is a clinical stage biopharmaceutical company that applies novel biomarker approaches to re-evaluate medicines that have failed in broad patient populations. The company seeks to discover genomic biomarkers correlated with patients' responses to drug candidates retrospectively. Denovo then designs and executes efficient clinical trials in targeted patient populations to optimize the probability of a successful trial. Denovo is enrolling patients in the U.S. and China with diffuse large B-cell lymphoma (DLBCL) in a Phase 3 clinical trial and will start a Phase 2B study in Glioblastoma (GBM) for its lead product candidate, DB102, which was in-licensed from Eli Lilly. The company has five additional late stage programs targeting major unmet needs: DB103 for schizophrenia, DB104 for depression, DB105 for Alzheimer's Disease, DB106 for acute myeloid leukemia (AML) and DB107 for recurrent high grade glioma. For additional information please visit http://www.denovobiopharma.com.

Contact:Michael F. Haller, Chief Business OfficerDenovo Biopharma LLCmhaller@denovobiopharma.com

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SOURCE Denovo Biopharma LLC

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Denovo Biopharma To Acquire Tocagen's Entire Replicating Gene Therapy Platform and Related Assets - Yahoo Finance

MILAN, Italy--(BUSINESS WIRE)--Genespire, a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, announced today the successful close of a 16M Series A financing from Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan. The company also announced the appointment of Julia Berretta, Ph.D., as Chief Executive Officer and member of the Board of Directors. Graziano Seghezzi, Managing Partner at Sofinnova Partners, and Lucia Faccio, Ph.D., Partner at Sofinnova Partners, will also join the Board.

Genespire was founded in March 2020 as a spin-off of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), one of the worlds leading cell and gene therapy research institutes spearheaded by gene therapy pioneer Prof. Luigi Naldini. The Company was co-founded by Fondazione Telethon and the San Raffaele Hospital, along with Prof. Naldini and Dr. Alessio Cantore.

The funds will be used to advance Genespires leading-edge platform technologies towards the development of novel gene therapies in two main areas: primary immunodeficiencies and metabolic genetic diseases.

Our mission has always been to develop breakthrough solutions for genetic diseases, said Prof. Naldini, Genespires co-founder and Director of SR-Tiget. This financing enables the company to translate our innovative science and early stage programs into clinical development. The appointment of Dr. Berretta as CEO is a major reinforcement of our team.

Sofinnova Partners Dr. Faccio added, Genespire is an exciting investment with all the key ingredients for success: Outstanding scientists that developed the first ex-vivo gene therapy to market, experienced executives brought in through Sofinnova Partners network and game changing technologies that have the potential to impact the lives of patients with genetic diseases.

I am thrilled to be joining Genespire and such exceptional scientific founders, said Dr. Berretta. Genespire was born of decades of experience in the gene therapy field, and is optimally positioned to advance transformative therapies for patients affected by severe inherited diseases.

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. She is also an independent Board member of Treefrog Therapeutics, an innovative stem cell company.

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.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: http://www.sofinnovapartners.com

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

About the San Raffaele-Telethon Institute for Gene Therapy (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.

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Genespire Secures 16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies - Business Wire

CDMO Fujifilm Diosynth Biotechnologies (FDB) says partnering with OXGENE could reduce the lead time of its customers gene therapy projects by up to 25%.

The technology, licensed from UK-based OXGENE for an undisclosed fee, consists of Helper, Rep/Cap and Gene of Interest plasmids, used in combination with a clonal suspension a HEK293 cell line.

The AAV system is expected to reduce the length of the supply chain gene therapy customers, according to contract development and manufacturing organization (CDMO) FDB, with the standard lead-time from the start of process development to the first GMP manufacture potentially reduced by three to six months.

Image: iStock/PashaIgnatov

FDB will manufacture and stock a supply of Helper and Rep /Cap plasmids for clients engaged in process development and GMP manufacture of gene therapy programs. Gene of Interest (GOI) plasmid manufacture will be performed in-house using FDBs existing microbial capabilities and facilities at its site in College Station, Texas with cGMP manufacture planned to commence in Q4 2020.

The site is subject to numerous investments by the CDMO, the latest a $35 million expansion adding cell culture and high throughput manufacturing suites.

OXGENEs AAV system is superior to off-the-shelf plasmid systems for AAV titers, said Andy Topping, chief scientific officer at FDB, adding the agreement gives the CDMO plasmid to drug product capability for AAV systems and allows clients to avoid delays associated with GMP production of plasmids.

UK-based OXGENE was founded as a plasmid catalog business eight years ago. Sophie Lutter, scientific marketing and communications manager, told this publication how the firms technology works at the Phacilitate conference in January.

We start with custom plasmid design and engineering we have plasmid sets optimized for AAV and lentiviral production and then we pair that with our GMP-banked clonal suspension HEK293 cell lines and engineered derivatives, she explained. We take them through to process development, where we can support scales of up to 10 L.

With downstream purification as part of its platform, the company offers a full viral-vector package. This leaves the customer not only with the final viral vector, but also [with] the processes and protocols to take that through to GMP manufacture.

More:
Fujifilm licenses AAV tech to speed gene therapies - Bioprocess Insider - BioProcess Insider

Cancer Gene Therapy Marketis expected to reach 5075 million by 2026 from XX million in 2018 at CAGR of XX %.

The report study has analyzed revenue impact of covid-19 pandemic on the sales revenue of market leaders, market followers and disrupters in the report and same is reflected in our analysis.

REQUEST FOR FREE SAMPLE REPORT:https://www.maximizemarketresearch.com/request-sample/520/

Cancer Gene Therapy Research Report is a method of therapeutic delivery of genetic material into a patients cells as a drug to treat disease and compensate for abnormal genes or to make a beneficial protein. Cancer cells modify themselves (called faults or mutations) in several of their genes which make them divide very often and form a tumor. Gene therapy provides various methods by which doctors can cure cancer like:

Inactivation of a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. Replacement of mutated gene that causes disease with a healthy copy of the gene.Cancer caused 9.02 million deaths in 2017, and is expected to reach 10 million by 2026; the increased no of cancer prevalence is because of increased body mass index, low fruit and vegetable intake, lack of physical activity, increased tobacco and alcohol consumption. Also, Factors like increasing ethical acceptance of gene therapy for treatment of diseases and growing popularity of DNA vaccines, High success rate during the preclinical and clinical trial, Increase in funding for R&D in cancer gene therapy, Increase in geriatric population, favorable government regulations will fuel the global Cancer Gene therapy market. However, the high cost of gene therapy treatment and unwanted immune responses will restrain market growth.The highest revenue-generating region is North America in 2017 followed by Europe; reasons behind this increased growth rate are well-established health care facilities, high per capita health care expenditure, and extensive R&D activities for the gene therapy in the region. However, Asia Pacific is projected to expand at a moderate growth rate during the forecast period.

Key Highlights:

Assessment of market definition along with the identification of key players and an analysis of their strategies to determine the competitive outlook of the market, opportunities, drivers, restraints, and challenges for this market during the forecast period Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, growth strategies that can In-depth analysis of the industry on the basis of market segments, market dynamics, market size, competition & companies involved value chain Cancer Gene Therapy market analysis and comprehensive segmentation with respect to the therapy and geography to assist in strategic business planning Cancer Gene Therapy market Research Report analysis and forecast for five major geographies North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key countries Complete quantitative analysis of the industry from 2017 to 2026 to enable the stakeholders to capitalize on the prevailing market opportunities.For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.

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Research Methodology:

An objective of the study is to estimate the size of the Cancer Gene Therapy market for 2017 and projects its demand till 2026 with quantitative and qualitative analysis of Cancer Gene Therapy market. Industry experts have studied various industry journals, directories, have referred information available with various associations to identify, collect information and to put it in articulated format to make useful for all stake holders in the industry. Primary research has been done and various industry experts and suppliers from worlds wide have given their inputs to make the study more accurate.Key Players in the Cancer Gene Therapy Market Are:

Shenzhen Sibiono Genetech Adaptimmune Glaxosmithkline Oncogenex Pharmaceuticals Bluebird Bio, Inc. Synergene Therapeutics Shanghai Sunway Biotech Biocancell Celgene MerckKey Target Audience:

Cancer Gene Therapy Market Investors Cancer Gene Therapy Marketing Players Pharmaceutical and Biotechnology Companies Healthcare Institutions (Individual Surgeons, Medical Schools, Group Practices, Hospitals, and Governing Bodies) Diabetes Drugs Market Research Associations Diabetes drug Manufacturers & DistributorsScope of the Cancer Gene Therapy Market

Research report categorizes the Cancer Gene Therapy market based on Therapy and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Cancer Gene Therapy market with key developments in companies and market trendsCancer Gene Therapy Market, By Therapy:

Oncolytic Virotherapyo Adenoo Lentiviruso Retro Viruso Adeno Associated Viruso Herpes Simplex Viruso Alpha Viruso Vaccinia Viruso Simian Viruso Others Gene Transfero Naked Plasmid Vectoro Electroporationo Sonoportiono Magnetofectiono Gene Gun Gene-Induced Immunotherapyo Delivery of Cytokines Geneo Delivery of Tumor Antigen GeneCancer Gene Therapy Market, By Geography:

North America Europe Asia Pacific Middle East & Africa Latin America

MAJOR TOC OF THE REPORT

Chapter One: Cancer Gene Therapy Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cancer Gene Therapy Market Competition, by Players

Chapter Four: Global Cancer Gene Therapy Market Size by Regions

Chapter Five: North America Cancer Gene Therapy Revenue by Countries

Chapter Six: Europe Cancer Gene Therapy Revenue by Countries

Chapter Seven: Asia-Pacific Cancer Gene Therapy Revenue by Countries

Chapter Eight: South America Cancer Gene Therapy Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cancer Gene Therapy by Countries

Chapter Ten: Global Cancer Gene Therapy Market Segment by Type

Chapter Eleven: Global Cancer Gene Therapy Market Segment by Application

Chapter Twelve: Global Cancer Gene Therapy Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Cancer Gene Therapy Market Report at:https://www.maximizemarketresearch.com/market-report/cancer-gene-therapy-market/520/

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Cancer Gene Therapy Market : Research Report - MR Invasion

BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, announced today that the Company has scheduled the release of its first quarter 2020 financial results after the market closes on Monday, May 11, 2020. The press release will be followed by a conference call at 5:00 p.m. ET, which will be open to the public via telephone and webcast. During the conference call, the Company will review its financial results and provide a business update.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 6043504. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 6043504.

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.

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Sangamo Therapeutics Announces First Quarter 2020 Conference Call and Webcast - Business Wire

After a comprehensive analysis, Mrinsights.bizhas published a new research study titled GlobalHemophilia Gene Therapy Market Growth 2020-2025 that covers the latest and upcoming industry trends and offers a global spectrum of the Hemophilia Gene Therapy market, and future forecast from 2020 to 2025 years. The market is bifurcated into product type, application, key manufacturers and key regions and countries. The research assists users to achieve competitive leverage with acquiring and preserving market position as key aims of the program. The report expands on details pertaining to contributions by key players, demand and supply analysis as well as market share growth of the industry.

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It covers the leading manufacturers profiles involving market entry strategies, production analysis, market share, revenue forecast. In addition, the regional analysis of the industry is offered where the report delivers analytical information on regional segmentation. Top leadingcompaniesof global Hemophilia Gene Therapy market are:Spark Therapeutics, Ultragenyx, Sangamo Therapeutics, Bioverativ, Shire PLC, Freeline Therapeutics, BioMarin, uniQure

Industry Trends And Opportunities:

The report provides an investigation into the global Hemophilia Gene Therapy market status, shares, supply-demand, market drivers, challenges and opportunities, and geological areas. Key trends and development opportunities are covered in this analysis report. The report then serves information on sales and market share estimates by-product as well as a profile of the companys business.

Regional Analysis:

This research report consists of the worlds crucial region Hemophilia Gene Therapy market share, size (volume), trends including the product profit, price, value, production, capacity, capability utilization, supply, and demand and industry growth rate. It helps readers to understand strategies to make sound investments. The regions are extensively analyzed with respect to every parameter of the geographies in question, comprising: Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia, Spain), Middle East & Africa (Egypt, South Africa, Israel, Turkey, GCC Countries).

The Report Addresses The Following Queries Related To The Market:

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Furthermore in this report, external as well as internal factors that are supposed to affect the business positively or negatively have been investigated. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors by region on the global Hemophilia Gene Therapy market is given in the report. A further section of the report discusses expansion plans of companies, key mergers and acquisitions, funding and investment analysis, company establishment dates, revenues of manufacturers, and their areas served and manufacturing bases.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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Global Hemophilia Gene Therapy Market 2020 Top Companies, Industry Demand, Business Review and Regional Analysis by 2025 Cole Reports - Cole of Duty

CAMBRIDGE, Mass., May 04, 2020 (GLOBE NEWSWIRE) -- TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage immunotherapy company developing the next generation of novel T cell therapies for patients suffering from cancer, today announced the appointment of Stephen Webster to its Board of Directors. With nearly 30 years of biotechnology industry experience in raising capital, business development transactions and operations, Mr. Webster has played important roles as the Chief Financial Officer of Spark Therapeutics, Optimer Pharmacuticals and Adolor Corporation. In connection with Mr. Websters arrival, Mitchell Finer, Ph.D., will be stepping down from the Board of Directors effective May 4, 2020 but will continue to serve TCR2 in an advisory capacity.

"We are delighted to welcome in another successful cell and gene therapy executive as Stephen Webster joins our Board of Directors. His distinguished track record of leading companies through periods of growth will prove invaluable at this moment in time as we prepare to present clinical data for our two lead programs, TC-210 and TC-110, and advance a third mono TRuC-T cell therapy towards the clinic," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "His business development transaction expertise will be particularly useful in helping us strike the right partnerships in pursuing our goal of developing innovative T cell therapies for patients suffering from cancer.

Mr. Webster served as the Chief Financial Officer of Spark Therapeutics, a publicly traded gene therapy biotechnology company, from July 2014 until its acquisition by Roche for $4.3 billion in December 2019. He was previously Senior Vice President (SVP) and Chief Financial Officer of Optimer Pharmaceuticals, a publicly traded biotechnology company, from July 2012 until its acquisition by Cubist Pharmaceuticals in October 2013. Prior to joining Optimer, Mr. Webster served as SVP and Chief Financial Officer of Adolor Corporation, a biopharmaceutical company, from 2008 until its acquisition by Cubist Pharmaceuticals in 2011. Mr. Webster also served in leadership positions in the investment banking healthcare groups of Broadpoint Capital and PaineWebber Incorporated.

Mr. Webster has served as a director of Nabriva Therapeutics AG (formerly Nabriva Therapeutics plc), a publicly traded biopharmaceutical company, since August 2016 and Viking Therapeutics, a publicly traded biopharmaceutical company, since May 2014. Mr. Webster received an A.B. in Economics from Dartmouth College and an M.B.A. in Finance from The Wharton School of the University of Pennsylvania.

I am thrilled to become a director of TCR2 Therapeutics, where there is a great opportunity to turn a distinctive TRuC-T cell platform into a series of novel treatments of cancer, said Mr. Webster. I look forward to working with the TCR2 leadership team and Board of Directors to add my business development expertise in helping the Company achieve its goal of bringing transformational therapies to people living with serious solid tumors and hematologic malignancies.

On behalf of TCR2 and the Board of Directors, I would like to thank Mitchell Finer for his many contributions to the rapid growth of our company, added Dr. Menzel. Our strategy to begin with an automated cell therapy manufacturing process benefited from working very closely with Dr. Finer, whose three decades of cell therapy manufacturing leadership provided us a significant competitive advantage in the cell therapy landscape. We look forward to continuing to benefit from his insights as he transitions from a Board member to a consultant.

About TCR2 Therapeutics

TCR2Therapeutics Inc.is a clinical-stage immunotherapy company developing the next generation of novel Tcell therapies for patients suffering from cancer.TCR2sproprietary T cell receptor (TCR) Fusion Construct Tcells (TRuC-T cells) specifically recognize and kill cancer cells by harnessing signaling from the entire TCR, independent ofhuman leukocyte antigens (HLA). In preclinical studies, TRuC-T cells have demonstrated superior anti-tumor activity compared to chimeric antigen receptor T cells (CAR-T cells), while exhibiting lower levels of cytokine release. The Companys lead TRuC-T cell product candidate targeting solid tumors, TC-210, is currently being studied in a Phase 1/2 clinical trial to treat patients with mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma. The Companys lead TRuC-T cell product candidate targeting hematological malignancies, TC-110, is currently being studied in a Phase 1/2 clinical trial to treat patients with CD19-positive adult acute lymphoblastic leukemia (aALL) and with aggressive or indolent non-Hodgkin lymphoma (NHL). For more information about TCR2, please visitwww.tcr2.com.

Forward-looking Statements

This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could", "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavor," "potential," "continue" or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include, but are not limited to, express or implied statements regarding the development of the Companys product candidates, future business plans and the therapeutic potential of its product candidates and platform.

The expressed or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities, including review under accelerated approval processes; orphan drug designation eligibility; regulatory approvals to conduct trials or to market products; TCR2s ability to maintain sufficient manufacturing capabilities to support its research, development and commercialization efforts, whether TCR2's cash resources will be sufficient to fund TCR2's foreseeable and unforeseeable operating expenses and capital expenditure requirements, the impact of the COVID-19 pandemic on TCR2s ongoing operations; and other risks set forth under the caption "Risk Factors" in TCR2s most recent Annual Report on Form 10-K, most recent Quarterly Report on Form 10-Q and its other filings with theSecurities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although TCR2believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur.

Moreover, except as required by law, neither TCR2nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investor and Media Contact:

Carl MauchDirector, Investor Relations and Corporate CommunicationsTCR2 Therapeutics Inc.(617) 949-5667carl.mauch@tcr2.com

Originally posted here:
TCR Therapeutics Announces Veteran Finance Executive Stephen Webster Joins its Board of Directors - GlobeNewswire

I awoke on Monday morning to the sad news that Max Randell had passed away on April 18. He would have been 23 on October 9.

Maxie wasnt expected to live past the age of 8, or even much past toddlerhood, according to some doctors. But gene therapy, and his incredible family, had something to say about that. COVID-19 didnt claim him his body just tired of fighting.

Max Randells legacy is one of hope, to the rare disease community whose family members step up to participate in the clinical trials that lead to treatments. In this time of the pandemic, attention has, understandably, turned somewhat away from the many people who live with medical limitations all the time. Ill explore that story next week.

A Devastating Diagnosis

Max was diagnosed at 4 months of age with Canavan disease, an inherited neuromuscular disease that never touched his mind nor his ability to communicate with his eyes, even though his body increasingly limited what he could do. Fewer than a thousand people in the US have the condition.

Canavan disease is an enzyme deficiency that melts away the myelin that insulates brain neurons. Gene therapy provides working copies of the affected gene, ASPA.

Babies with Canavan disease are limp and listless. Most never speak, walk, or even turn over. Yet their facial expressions and responses indicate an uncanny awareness. A child laughs when his dad makes a fart-like noise; a little girl flutters her fingers as if they are on a keyboard when a friend plays piano. Theyre smart.

Today, with excellent speech, occupational, and physical therapy and earlier diagnosis, people with Canavan disease can live into their teens or twenties. Those with mild mutations live even longer.

Maxs passing is a tragedy, but he taught researchers about gene therapy to the brain. And that may help others.

Gene Therapy for Canavan

Max had his first gene therapy at 11 months of age and a second a few years later, after slight backsliding when clinical trials halted in the wake of the death of Jesse Gelsingerin a gene therapy trial for a different disease.

Ive written about Maxs journey through many editions of my human genetics textbook, in my book ongene therapy, and in several DNA Science posts, listed at the end.

Ive had the honor to attend two of Maxs birthday parties, which celebrate Canavan kids and the organization that his family founded, Canavan Research Illinois. At one party I brought along birthday cards that students whod read my gene therapy book made for him. And his grandma Peggy, who emailed me of his passing this past Monday, showed me how Max communicated with eyeblinks of differing duration and direction.

Heres what his mom Ilyce wrote about one yearly gathering:

This year will be the 20th Annual Canavan Charity Ball. Each year as I plan this event Im faced with the undeniable reality that theres a chance Maxie wont be here by the time the day rolls around. With each passing year this fear grows stronger and it becomes increasingly difficult to put into print that our annual event is in honor of Maxies birthday. Ive been talking to Maxie a lot lately about his life. He feels happy, strong, loved, content, productive, and fulfilled and he is looking forward to his upcoming 21st birthday. Im excited to celebrate this incredible milestone.

Maxs parents and brother Alex have had the unusual experience of time, of being able to watch their loved one as the years unfolded following gene therapy. They were able to see more subtle improvements than can the parents whose children have more recently had gene therapy to treat a brain disease. Parents watch and wait and hope that language will return, or that a child will become more mobile or less hyperactive, depending on the treated condition. The changes may be subtle, or slow, or restricted and thats what Max taught the world.

For him, the viruses that ferried the healing genes into his brain seem to have gathered at his visual system. His parents noticed improvements in the short term, just before his first birthday, as well as long term.

Within two to three weeks, he started tracking with his eyes, and he got glasses. He became more verbal and his motor skills improved. His vision is still so good that his ophthalmologist only sees him once a year, like any other kid with glasses. She calls him Miracle Max, Ilyce told me in 2010.

In 2016 I heard from Ilyce again:

I wanted to give you an update on Maxie. Hes going to be 19 on October 9th. He graduated from high school in June and is beginning a work program on Monday. Its been very exciting to watch him grow into a young man!

Max had an appointment with his ophthalmologist this week and his vision continues to improve. His doctor said that the gene is still active in his brain because his optic nerve shows absolutely no signs of degeneration and looks the same each year. I wish we could have been able to express the gene throughout more of his brain, but I am grateful for the treatments because of the progress hes made.

Even though gene therapy wasnt a cure for Max, the things we are experiencing definitely give me a lot of hope that once the delivery system is perfected, I can see a potential cure for Canavan disease in the future. Just knowing that the gene is still there 15 years later gives me confidence that a one-time gene transfer would actually work!

Maxs gene therapy circa 2002 targeted less than 1% of brain cells, with fewer viral vectors than are used to deliver healing genes in todays clinical trials. But it looks like some of the vectors may have made their way beyond the optic nerves, judging by the interest in math he had in high school and his critical thinking skills.

A Choice of Gene-Based Therapies

When the Randell family decided to pursue gene therapy, it was pretty much the only game in town. Thats changed.

Only two gene therapies have been approvedin the U.S. But a search at clinicaltrials.gov yielded 602 entriesdeploying the technology. The list still rounds up the usual suspects of years past mostly immune deficiencies, eye disorders, or blood conditions, with a few inborn errors of metabolism.

But one clinical trial mentions the gene-editing tool CRISPR, which can replace a mutant gene, not just add working copies as classical gene therapy does. TheCRISPRtrial is an experiment on stem cells removed from patients with Kabuki syndrome, which affects many body systems.

Spinal muscular atrophy now has two FDA-approved treatments, one an antisense therapy (Spinraza) that silences a mutation and the other (Zolgensma) a gene therapy that infuses copies of the functioning gene. Without treatment, the destruction of motor neurons in the spinal cord is usually lethal by age two.

In 2018, FDA approved the first drug based on RNA interference (RNAi), yet another biotechnology. It silences gene expression, which is at the RNA rather than the DNA level of the other approaches. Onpattro treats the tingling, tickling, and burning sensations from the rare condition hereditary transthyretin-mediated amyloidosis.

When I wrote my book on gene therapy in 2012, the technology was pretty much the only choice of research to pursue besides protein-based therapies like enzyme replacement. Now families raising funds for treatments for single-gene diseases can add antisense, RNAi, and CRISPR gene editing to the list of possibilities.

In any battle, a diversity of weapons ups the odds of defeating the enemy.

RIP Max Randell.

DNA Science posts:

Fighting Canavan: Honoring Rare Disease Week

A Brothers Love Fights Genetic Disease

Gene Therapy for Canavan Disease: Maxs Story

Celebrating the Moms of Gene Therapy

To support research:Canavan Research Illinois

More here:
A Tribute to Max Randell, Gene Therapy Pioneer - PLoS Blogs

Dive Brief:

Merck KGaA, like other contract manufacturers such as Lonza, is betting the next big wave of demand will be for complex production of gene therapies and other products such as viral vaccines and immunotherapies. The gene therapy market will grow to about $10 billion by 2026 from $1 billion in 2018, the company said, citing estimates from Biotech Forecasts.

"Viral vector manufacturing has transitioned from a niche industry to the cornerstone of the future of biopharmaceuticals," said Udit Batra, head of Merck KGaA'slife science business, in a statement.

The German company has been on a spending spree in recent years, announcing plans to invest 1 billion euros in its global headquarters in Darmstadt, more than $400 million in two sites in Switzerland,and $70 million in a research and development hub expansion in Billerica, Massachusetts.

Carlsbad is already home to a Merck KGaA facility that has been involved in gene therapy since 1997, about the time that researchers beginning studying the potential for such treatments in people. At present, the site has 16 modular viral bulk manufacturing clean room suites and two fill/finish suites, Merck KGaA said.

With the new facility, the Carlsbad location will have 27 suites used in different parts of the manufacturing process and will support production at the 1000-liter scale using single-use equipment, Merck KGaA said.

The company also has a manufacturing facility in Glasgow that produces intermediates and final products for gene therapy and viral vaccines.

Merck KGaA, established in 1688, is majority owned by descendants of the original founder and had sales of 16.2 billion euros last year. The U.S. pharmaceutical giant Merck was once a subsidiary but is no longer associated with its German namesake.

Read this article:
Merck KGaA to spend $110M on new gene therapy facility in California - BioPharma Dive

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Regenxbio (RGNX) is a pioneer in gene therapies with a wide set of licensing agreements and an internal pipeline. The company focuses on adeno-associated virus (AAV) gene therapies for gene replacement and antibody delivery pursuing markets in retinal, neurodegenerative, and liver diseases. With a market cap of ~$1.3B (enterprise value of ~$1B), ~$400M of cash on the balance sheet, and ~$35M in revenue, Regenxbio is well-positioned to complete its milestones around manufacturing and clinical development into 2020.

The core investment thesis for Regenxbio is described below.

Validated technology platform to develop successful AAV gene therapies:

Strong financial position:

Undervalued internal assets:

From its 52-week high, Regenxbio's stock is down over 40%. The stock reached a low point from COVID-19 development. This is likely due to a lack of near term catalysts for the stock. With additional data for their lead asset in wet AMD coming in the first half of 2020, the initiation of phase II trials for the asset, and the sales ramp up for Zolgensma, the stock has a few potential catalysts coming up. As a result, there is an attractive entry point for investors to become an owner in Regenxbio.

Figure 1: RGNX daily chart (Source: Capital IQ)

The opportunity is that Regenexbio's licensing agreement with AveXis (NVS) on a medicine called Zolgensma to cure spinal muscular atrophy can potentially alone earn Regenxbio $3B-$4B in revenue. Zolgensma is an AAV gene therapy that delivers a transgene of SMN1 to cure the disease. About 20K people in the US have the disorder. With the medicine being priced at a little over $2M per patient, the market potential is well over $40B.

In 2014, Regenxbio licensed their AAV technology to AveXis to cure spinal muscular atrophy. The deal included various milestone payments to Regenxbio and importantly a mid-single to low double-digit royalties on net sales.

This one deal alone beyond the 20 similar deals Regenxbio has and its internal pipeline makes the company an attractive business. A simple DCF analysis with various assumptions, with the most important being including a capital expenditure of $400M for the clinical work with their flagship internal product, supports that Regenxbio is undervalued:

Figure 2: DCF model for RGNX (Source: Internal)

All the modeling done doesn't really help anyone figure out why this opportunity exists? Why is Regenxbio undervalued? Doubts around the Zolgensma scale up? Worries that capital from licensing deals will be wasted on an internal drug pipeline?

It's unprecedented that a drug company's platform is worth a lot more than the internal pipeline of drugs. From Regenxbio's latest corporate presentation, the company's main internal program, RGX-314, is focused on wet AMD:

Figure 3: Overview of Regenxbio's main asset (Source: Corporate Presentation)

RGX-314 is an AAV therapy for wet AMD. The slide describes the problem (leaky blood vessels in the eye) and how large it is (~2M patients) along with the vector (AAV8) and delivery cargo (anti-VEGF Fab). However, the company doesn't mention Eylea or other wet AMD medicines that are already approved. Regenxbio alludes to issues around delivery of drugs like Eylea (REGN), but doesn't go too deep on this slide or the presentation in general about how competitive their RGX-314 program will really be amongst clinicians.

An important point for any gene therapy is delivery whether it's a transgene or a CRISPR protein. A major reason why Regenxbio focused on wet AMD and ophthalmology in general is that delivering something to the eye is a lot easier than delivering something to the brain. RGX-314 is undergoing a phase I/II trial focused on establishing safety; the pivotal trial will come later. For the phase I portion, the company met their primary endpoint and showed safety so far. They have also shown how increasing doses of their gene therapy reduces the number of injections. This is going to be an important experiment and data set to argue for clinicians to switch over from something like Eylea.

The real value in Regenxbio is in its AveXis deal and the various licensing partnerships:

Figure 4: Regenxbio licensing partnerships (Source: Corporate Presentation)

Figure 5: Regenxbio licensing partnerships (Source: Corporate Presentation)

Figure 6: Regenxbio licensing partnerships (Source: Corporate Presentation)

This business model is enabled by Regenxbio's core technology focused on AAV7-10 and natural or close-to-natural variants:

Figure 7: Overview of Regenxbio's platform (Source: Corporate Presentation)

Over the next two years, the key milestones are:

The business can continue to strike up more licensing deals and expand current ones. The margin of safety here is that Regenxbio is undervalued just for its deal with AveXis and its various licensing deals that provide periodic payments based on progress and potentially more royalties if the drugs are approved and commercialized.

This seems to be a case where the market is focusing on the company's internal pipeline. Regenxbio's headline drug is interesting but unlikely to be competitive. Whereas, the business has a wonderful platform and licensing business that is being ignored. Simple valuations show these cash flows are not being fully appreciated. As a result, Regenxbio is going to grow revenue without any additional work and still has the potential to strike again through the 20 or so deals it has.

Figure 8: Key upcoming milestones for Regenxbio (Source: Corporate Presentation)

Regenxbio's lead candidate is focused on wet age related macular degeneration (AMD). The disease is a severe form of macular degeneration, a condition in which layers of macula get progressively thinner. The wet form is caused by abnormal blood vessels grown under the macula and retina where leaky blood vessels cause problems with vision, ultimately leading to blindness. The current standard-of-care is an anti-vascular endothelial growth factor (anti-VEGF) therapy. Patients require monthly injection of anti-VEGF to stop the growth of leaky blood vessels. Wet AMD is not a genetic disease, but it is a large and established market for Regenxbio to capture.

To frame the market opportunity of wet AMD, a few facts are helpful:

196 million people worldwide & 288 million by 2040 have AMD

10% have wet AMD, but is the leading cause of blindness

175,000 new patients annually in US

Growing number of patients due to aging population

Current treatments are regular injections of anti-VEGF

Figure 9: Overview of wet AMD (Source: JMS)

For wet AMD, competition comes from Genentech, Regeneron (REGN), and Adverum (ADVM). Genentech sells Lucentis at a price of $1850 per dose. Regeneron sells Eylea at $1150 per dose. Genentech's Avastin is also used off lab and is becoming more popular due to its cheap price of $60 per dose; the medicine is currently used for metastatic colorectal cancer. For gene therapies in wet AMD, the sole competitor is Adverum Biotechnologies. With 20M people with wet AMD, the total market opportunity for these medicines are in the billions of dollars.

Where Genentech's and Regeneron's medicines require multiple doses over the lifetime of a patient, a gene therapy has the potential to be curative and remove the multiple dosing requirement. For wet AMD, over 50% recurrence rate in the first year after treatment has stopped, and over 25% recurrence rate in the second year after treatment has stopped For drugs like Eylea and Lucentis, monthly intravitreal injection creates large burden for patients and create difficulty in dosing for clinicians. These problems allow Regenxbio to potentially capture the market with a gene therapy:

Figure 10: Wet AMD market (Source: Reportlinker)

Regenxbio's lead asset, RGX-314 is pursuing wet AMD. So far the company has shown:

Dose dependent protein expression levels and drug efficacy

Sustained protein expression for over 1.5 years

Long term efficacy demonstrated for Cohort 3 for rescue-free patients

No serious adverse events (SAE), but mild adverse events (AE) such as inflammation

Significant improvement in visual acuity for rescue free patients

Figure 11: Trial design of Regenxbio's lead asset (Source: Corporate Presentation)

RGNX

Cohort 1

Cohort 2

Cohort 3

Cohort 4

Cohort 5

Dose

3 x 109 gc/eye

1 x 1010 gc/eye

6 x 1010vg/eye

1.6 x 1011 gc/eye

2.5 x 1011 gc/eye

Rescue Injection Free

Not Available

Mean 4.7 rescue inj.

Not Available

Mean 3.8 rescue inj.

3 / 6 Patients

Mean 1.3 rescue inj.

5 / 12 Patients

Mean 2.2 rescue inj.

9 / 12 Patients

Mean 0.8 rescue inj.

Duration

52 Weeks

52 Weeks

78 Weeks

52 weeks (2H 2020)

52 weeks (2H 2020)

Best Corrected Visual Acuity (BCVA)

In ETDRS letters

Mean: -2.0

Range: -8/+10

Mean: +7

Range: -4/+15

Mean: +8

Range: 0/+21

Mean: +2

Mean: +4

Central Subfield Thickness (m)

Mean: -14

Range -81/+92

Mean: +26

Range -7/+62

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Regenxbio Is A Leader In Gene Therapies - A Case Where The Platform Is Worth More Than The Pipeline - Seeking Alpha