StemCell Therapy

Engineering and construction specialists Merit has been chosen to deliver the third phase expansion of the Cell and Gene Therapy Catapult (CGT Catapult) manufacturing centre in Stevenage, further increasing the capacity of the facility.

The company will use its offsite manufacturing technology and expertise in building controlled environments to deliver seven new quality control laboratories.

Merit completed construction of the expansion phase of the manufacturing centre in October last year using its off-site PAM (pre-assembled module) approach.

For these new laboratories, only 10% of the labour content will be carried out on site. The company will engineer pre-assembled analytical QC laboratory space at its offsite manufacturing centre reducing the build time by 33%, aiming to deliver cost savings to the project while also avoiding disruption to research and manufacturing at the Stevenage facility.

Tony Wells, managing director, Merit, said: We are delighted to have the opportunity to continue to work with the team at CGT Catapult and are proud to have an integral role in the development of such a pioneering off-site initiative. Our proprietary Instant Building technology combines advanced offsite manufacturing with the technical expertise to deliver complex solutions in significantly shorter build schedules. We have a detailed knowledge of the project after successfully completing the expansion phase last year and look forward to working closely with CGT Catapult to once again deliver a high-quality facility.

Backed by over 75 million of funding, including investment from the UK Governments Industrial Strategy Challenge Fund; the department of Business Energy and Industrial Strategy, from Innovate UK, the UKs innovation agency, the European Regional Development Fund and from Hertfordshire Local Enterprise Partnership, the centre is providing the infrastructure and expertise to enable companies to develop their manufacturing capabilities and systems for large scale, commercial cell and gene therapy supply. Companies currently collaborating at the centre are Adaptimmune, Autolus, Freeline Therapeutics and TCRTherapeutics.

Keith Thompson, CEO, Cell and Gene Therapy Catapult, said: It is a pleasure to work with Merit again to expand our laboratory space to provide additional analytical capabilities at the Cell and Gene Therapy Catapult manufacturing centre. Merit provided diligent support and ensured minimal disruption during the building of our expansion phase at the centre, and this is crucial to maintaining the facility operational for us and our collaborating companies manufacturing therapies, in an operational GMP compliant state. We look forward to working with Merit again on what is another significant project for CGT Catapult.

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Engineering specialist selected to deliver expansion of CGT Catapult - EPM Magazine

Global Gene Therapy and Antisense Drugs Market: Snapshot

Since an escalating number of patients are being diagnosed with cancer every day, the global demand for gene therapy and antisense drugs is bound to multiply. The growing incidence of health conditions such as Parkinsons disease and high cholesterol is also boosting the demand for gene therapy and antisense drugs. Several new drugs and therapies have been making their debut in the global gene therapy and antisense drugs market of recent.

For instance, in June 2017, it was announced that a group of scientists at the University of Queensland have developed a new technique that is capable of permanently silencing severe allergies triggered by shellfish, peanuts, and venom. The treatment was found to be successful in animal trials. The technique holds considerable potential for treating asthma completely. During the same month, results of another research study for treating multiple myeloma, a type of blood cancer, were announced at a conference held by the American Society of Clinical Oncology. The new CAR-T therapy consists of filtering the blood of the patients in order to eliminate T cells which are then genetically altered and given back to the patients.

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However, the difficulty pertaining to the delivery of antisense technology to the brain can present key challenges to the expansion of the global market for gene therapy and antisense drugs. Moreover, the unavoidable toxic aftereffects associated with the technology can also inhibit the growth of the gene therapy and antisense drugs market worldwide. However, the present attempts at developing safe and efficient antisense drugs by several market participants including Ionis Pharmaceuticals, which undertook testing of the technology on transgenic mice, can promote the further expansion of the market.

Global Gene Therapy and Antisense Drugs Market: Overview

Antisense gene therapy is emerging as one of the most beneficial therapeutics for various diseases such as tumors, cancer etc. These newer therapies are based on increased knowledge of molecular events that lead to disordered cellular growth. The therapy involves using a gene silencing technique rather than a gene repairing technique for silencing the genes effect.

The research report is a valuable tool for comprehending the progression of the global gene therapy and antisense drugs market between 2017 and 2025.

Global Gene Therapy and Antisense Drugs Market: Treatment Insights

Antisense drugs attach to the mRNA of a target protein, which inhibits the protein production process. Antisense therapeutics can obstruct the expression of oncogenes and other cancer-related genes that express growth factors. Antisense gene therapy involves the utilization of various therapeutic strategies which requires a clear knowledge of the molecular anatomy of cancer related genes. Antisense gene therapy is used to treat various diseases such as hemorrhagic fever, cancer, cystic fibrosis, renal diseases, HIV/AIDS, spinal muscular atrophy, and cardiovascular diseases.

Global Gene Therapy and Antisense Drugs Market: Market Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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Gene Therapy and Antisense Drugs Market Growth Trends, Key Players, Competitive Strategies and Forecasts to 2025 - 3rd Watch News

CAMBRIDGE, Mass., March 18, 2020 (GLOBE NEWSWIRE) -- Anchiano Therapeutics Ltd. (Nasdaq: ANCN) (Anchiano), a biopharmaceutical company focused on discovery and development of novel therapies to treat cancer, today reported financial results forthe year ended December 31, 2019.

Key Developments

Year Ended December 31, 2019 Financial Results:

On December 31, 2019, Anchiano had total cash and cash equivalents of approximately $17.6 million. Financial resources are expected to suffice through the end of 2020.

Research and development expenses for the year ended December 31, 2019 were approximately $13.3 million, compared to approximately $7.5 million for the same period in 2018. This increase was mainly due to an increase in clinical trial expenses, manufacturing expenses and manpower expenses, as well as additional startup and initial ongoing expenses in connection with the Collaboration Agreement with ADT.

General and administrative expenses for the year ended December 31, 2019 were approximately $6.2 million, compared to expenses of approximately $5.5 million for the same period in 2018. The increase was mainly due to increases in professional fees, insurance and manpower expenses, offset by a decrease in share-based payment.

Financing expenses, net, in the year ended December 31, 2019 were approximately $4.2 million, compared to approximately $457 thousand for the same period in 2018. This change was mainly due to a revaluation of investor warrants at fair value during a period where these could not be classified within shareholders equity.

Restructuring expenses in the year ended December 31, 2019 were approximately $3.4 million, and were comprised principally of contract termination costs and employee severance and associated termination costs related to the reduction of workforce resulting from Anchianos decision to discontinue its Phase 2 Codex as described above.

Net loss for the year ended December 31, 2019 was approximately $27.1 million compared to approximately $13.8 million for the same period in 2018.

About Anchiano

Anchiano is a biopharmaceutical company dedicated to the discovery, development, and commercialization of novel targeted therapies to treat cancer in areas of significant clinical need, with its headquarters in Cambridge, MA. Anchiano is developing small-molecule pan-RAS inhibitors and inhibitors of PDE10 and the -catenin pathway. For more information on Anchiano, please visit http://www.anchiano.com.

Forward-Looking Statements

This press release contains forward-looking statements that are subject to risks and uncertainties. Words such as believes, intends, expects, projects, anticipates and future or similar expressions are intended to identify forward-looking statements. These forward-looking statements are subject to the inherent uncertainties in predicting future results and conditions, many of which are beyond the control of Anchiano, including, without limitation, the risk factors and other matters set forth in its filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2019. Anchiano undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Company Contact:Frank Haluska, M.D., Ph.D.President and Chief Executive Officerinfo@anchiano.com

Investor Contact:Ashley R. RobinsonManaging DirectorLifeSci Advisors, LLC617-430-7577arr@lifesciadvisors.com

RESULTS OF OPERATIONS (unaudited)

U.S. dollars in thousands

STATEMENTS OF FINANCIAL POSITION (unaudited)

U.S. dollars in thousands

CASH FLOWS (unaudited)

U.S. dollars in thousands

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Anchiano Therapeutics Reports Year-End 2019 Financial Results - GlobeNewswire

Report Description

A recent market intelligence report that is published by Data Insights Partner onGene Therapy marketmakes an offering of in-depth analysis of segments and sub-segments in the regional and international Gene Therapy market. The research also emphasizes on the impact of restraints, drivers, and macro indicators on the regional and world Gene Therapy market over the short as well as long period of time. A detailed presentation of forecast, trends, and dollar values of international Gene Therapy market is offered. In accordance with the report, the global Gene Therapy market is projected to expand at a CAGR of 30% over the period of forecast.

Market Insight, Drivers, Restraints& Opportunity of the Market:

Gene therapy is a medical procedure which replaces defective genes or introduces new genes n order to prevent or cure genetic disorders. This procedure has become a bench mark in medical industry as there is no requirement of surgery or drugs or other procedure which has side effects on the individuals. Gene therapy was first commercialized in China in 2004 by China based SiBono Gene Tech (product Gendicine).

The global gene therapy market has been expanding due to the rigorous research conducted in the field of genetics. The rising awareness about the capability of cure of several rare genetic diseases by gene therapy is another important driver which leads the global gene therapy market during the forecast period. Gene therapy has capability cure several life threatening diseases such as cancer, cardiac diseases, AIDS, cystic fibrosis, age-related disorders, sickle cell anemia etc. In March 2019, the director of the National Health Institute (NIH), the U.S. announced that the recent clinical trials on the gene therapy for the treatment of sickle cell anemia showed promising result- therefore, increasing prevalence of aforementioned lie threatening diseases would likely to drive the growth of the global gene therapy market during the forecast period.

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On the other hand, treatment cost and stringent regulatory conditions etc. may hamper the growth of the global gene therapy market in the upcoming future. The results of Gendicine clinical trials were published in 2003 and the medicine got approval by the China State Food and Drug Administration in the same year. Although approved in China, Gendicines use is not very promising outside China. There are several concerns among the researchers about the quality of the clinical trials performed and safety and efficacy of the treatment. However, Gendicines equivalent Advexin (company Introgen Therapueitcs) is still waiting for the FDA approval.

Increasing investment to the gene therapy related research (around 10 Bn was invested in 2015 by private and public organizations), new product developments such as (Zolgensma in 2019), strategic alliance among the key players (such as collaboration between Axovant and Yposkesi) would bring the global gene therapy market an opportunity to propel during the forecast period. In May 2019, Avexis (a Novartis company) has got the FDA approval for Zolgensma for treatment of spinal muscular atrophy for the pediatric patients (less than 2 years of age).

Segment Covered:

This market intelligence report on the global gene therapy market encompasses market segments based on product, application, target user and geography. On the basis of product, the sub-markets is segmented into Yescarta, Kymriah, Strimvelis, Gendicine, Zolgensma and others (Advexin). Based on application, the global gene therapy market has been segregated into large B-Cell lymphoma, Car T Cell therapy, ADA-SCID (adenosine deaminase deficiency), muscular atrophy, head and neck squamus cell carcinoma, others (Crigler-Najjar syndrome). By target user, the global gene therapy market is also classified into adult and pediatric. By Geography, the global gene therapy market has been divided into North America (the U.S., Canada), Latin America (Brazil, Mexico, Argentina and other countries), Europe (Germany, France, the U.K., Spain, Italy, Russia, and other countries), Asia Pacific (India, Japan, China, Australia and New Zealand and other countries), Middle East and Africa (GCC, South Africa, Israel and Other countries).

Profiling of Market Players:

This business intelligence report offers profiling of reputed companies that are operating in the market. Companies such as Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others have been profiled into detail so as to offer a glimpse of the market leaders. Moreover, parameters such as gene therapy market related investment & spending and developments by major players of the market are tracked in this global report.

Report Highlights:

In-depth analysis of the micro and macro indicators, market trends, and forecasts of demand is offered by this business intelligence report. Furthermore, the report offers a vivid picture of the factors that are steering and restraining the growth of this market across all geographical segments. In addition to that, IGR-Growth Matrix analysis is also provided in the report so as to share insight of the investment areas that new or existing market players can take into consideration. Various analytical tools such as DRO analysis, Porters five forces analysis has been used in this report to present a clear picture of the market. The study focuses on the present market trends and provides market forecast from the year 2017-2027. Emerging trends that would shape the market demand in the years to come have been highlighted in this report. A competitive analysis in each of the geographical segments gives an insight into market share of the global players.

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Salient Features:

This study offers comprehensive yet detailed analysis of the Gene Therapy market, size of the market (US$ Mn), and Compound Annual Growth Rate (CAGR (%)) for the period of forecast: 2019 2027, taking into account 2017 as the base year

It explains upcoming revenue opportunities across various market segments and attractive matrix of investment proposition for the said market

This market intelligence report also offers pivotal insights about various market opportunities, restraints, drivers, launch of new products, competitive market strategies of leading market players, emerging market trends, and regional outlook

Profiling of key market players in the world Gene Therapy market is done by taking into account various parameters such as company strategies, distribution strategies, product portfolio, financial performance, key developments, geographical presence, and company overview

Leading market players covered this report comprise names such as. Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others

The data of this report would allow management authorities and marketers of companies alike to take informed decision when it comes to launch of products, government initiatives, marketing tactics and expansion, and technical up gradation

The world market for Gene Therapy market caters to the needs of various stakeholders pertaining to this industry, namely suppliers, manufacturers, investors, and distributors for Gene Therapy market. The research also caters to the rising needs of consulting and research firms, financial analysts, and new market entrants

Research methodologies that have been adopted for the purpose of this study have been clearly elaborated so as to facilitate better understanding of the reports

Reports have been made based on the guidelines as mandated by General Data Protection Regulation

Ample number of examples and case studies have been taken into consideration before coming to a conclusion

Reasons to buy:

vIdentify opportunities and plan strategies by having a strong understanding of the investment opportunities in the Gene Therapy market

vIdentification of key factors driving investment opportunities in the Gene Therapy market

vFacilitate decision-making based on strong historic and forecast data

vPosition yourself to gain the maximum advantage of the industrys growth potential

vDevelop strategies based on the latest regulatory events

vIdentify key partners and business development avenues

vRespond to your competitors business structure, strategy and prospects

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Gene Therapy Market size Witness Growth Acceleration during 2027 - Packaging News 24

Global Cancer Gene Therapy Report available at Digits n Markets contains an overview of the Global Cancer Gene Therapy which covers market size, opportunities, trends, growth rate, and competition landscape. The Global Cancer Gene Therapy is segmented Source, Product Type Applications and regions. With forecast to 2027.

Digits n Markets has recently published a comprehensive market research report on the Global Cancer Gene Therapy that includes evaluation of market size and various segments. The competitive environment is analyzed along with study of winning strategies adopted by key players.

The report is a detailed study on the accounting Global Cancer Gene Therapy with details regarding an in-depth evaluation of the industry vertical. The study is performed taking into consideration a twofold aspect of consumption and production. Speaking of the product category, the report provides detailed product remuneration, manufacturing of the product and the gross margins of the firms manufacturing the products. With regards to the consumption, the study reveals the product consumption value and the product consumption volume along with the status of import as well as the export of the products.

The Global Cancer Gene Therapy Market Anticipated to exhibit a CAGR 35.1% during Forecast Period. 2018-2025

Avail a free sample in PDF format along with a quick look at vital report briefs:https://digitsnmarkets.com/sample/6650-global-cancer-gene-therapy-market

Key Questions Answered by the Report:

Numerous micro and macro-economic factors impacting the growth of the market are analyzed and the data is represented in a way to aid the clients to enhance their strategic decision making. Key players operating in the Global Cancer Gene Therapy are:

Table of Content

Chapter 1: IntroductionChapter 2: Executive SummaryChapter 3: Market OverviewChapter 4: Cancer Gene Therapy Market, By TypeChapter 5: Cancer Gene Therapy Market, By ApplicationChapter 6: Cancer Gene Therapy Market, By RegionChapter 7: Competition Landscape

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Global Cancer Gene Therapy Market, Trends, Analysis, Opportunities, Share and Forecast 2018-2025 - Galus Australis

Gene therapy has been used to treat a person with haemophilia for the first time in Ireland, a patient group has announced.

The Irish Haemophilia Society (IHS) confirmed on Thursday morning that the person received gene therapy as part of a clinical trial. IHS chief executive Brian OMahony said the treatment is a momentous occasion for the haemophilia community in Ireland.

The general term haemophilia describes a group of inherited blood disorders in which there is a life-long defect in the clotting mechanism of the blood.

Since the 1970s, haemophilia has been treated by the administration of intravenous infusions of the missing clotting factor. However, work done by companies and academic institutions has given new hope that an effective treatment could be based on gene therapy, the IHS said.

The clinical trial uses a viral vector to deliver gene therapy to the persons liver intravenously. In the past, viruses such as HIV and Hepatitis C decimated the haemophilia population in Ireland through contaminated blood. It is ironic that a virus could now be the delivery system which offers the best hope of a practical cure for severe haemophilia, Mr OMahony said.

It is hoped that the effect of the gene therapy infusion will last for many years and possibly for a lifetime.

The principal investigator on the trial in Ireland is Dr Niamh OConnell of the National Coagulation Centre in St. Jamess Hospital. She said the gene therapy was ground breaking.

The opportunity to participate in clinical trials is part of the commitment of the National Haemophilia Service to personalise treatment and to improve the quality of life and outcomes for people with haemophilia.

The study, which is being run by drug manufacturer UniQure, involves three Irish patients among a total of 60 around the world. There will be an intensive period of monitoring of effectiveness at first, followed by a longer term evaluation over five years. Only one treatment is administered to trial patients.

The particular gene therapy is focused on patients who are missing clotting factor IX, the second most-common type of haemophilia. Earlier results show that the level of clotting factor increased from 1 per cent - generally seen as severe haemophilia - to between 33 and 51 per cent in a small number of individuals treated, levels seen in mild cases or even amongst the non-haemophiliac population.

Professor Martina Hennessy of the Wellcome HRB Clinicial Research facility in St Jamess, where the gene therapy was infused, said that access to high quality research is an integral part of good healthcare because it raises standards and pushes the boundaries of what can be achieved.

Delivering gene therapy requires specialised training and equipment, we have been preparing with Dr OConnell and her team for over a year to undertake this exciting work, in partnership with the Irish Haemophilia Society. Other trials are planned, we hope this expertise leads other Irish patient groups also being able to access potentially life changing treatments in the future, she said.

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Gene therapy used in clinical trial for person with haemophilia - The Irish Times

Listen to the latest episode at this link, or on your favorite app including Alexa, Apple, Google, Spotify and Stitcher. Episodes are available every morning on PennLive. Subscribe/Follow and rate the podcast via your favorite app.

A serial child rapist is going behind bars for life after being sentenced to up to 126 years in jail. Meanwhile, a couple is being charged with arson and insurance fraud after allegedly setting fire to their newspaper business. In Pittsburgh, a UPMC researchers new gene therapy could cure diabetes. Also, with Easter on the horizon, Cadbury is on the hunt for a different kind of bunny -- and one Pennsylvania llama is throwing its hat in the race.

Those are the stories we are covering in the latest episode of Today in Pa, a daily weekday podcast from PennLive.com and hosted by Julia Hatmaker. Today in Pa is dedicated to sharing the most important and interesting stories in the state.

Todays episode refers to the following articles:

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Serial child rapist sentenced to up to 126 years in jail; new gene therapy from UPMC could cure diabetes: Tod - PennLive

Significantly Expands Companys R&D Capabilities to Advance Industrys Largest Portfolio of Rare Disease Gene Therapy Programs

State of the Art 75,000 Sq. Ft. Research Facility Across From University of Pennsylvania Strengthens Collaboration with Wilson Lab

CRANBURY, N.J. and PHILADELPHIA, March 05, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (FOLD) today announced the official opening of the companys Global Research and Gene Therapy Center of Excellence in uCity SquareinPhiladelphia to advance its industry leading portfolio of rare disease gene therapy programs. In 2019, Amicus and the University of Pennsylvania (Penn) announced a major expansion of their Gene Therapy Collaboration which provides Amicus with disease-specific worldwide rights to Penns Next Generation Gene Therapy Technologies from the Wilson Lab for the majority of lysosomal storage disorders, as well as twelve additional more prevalent rare diseases including Rett Syndrome, Angelman Syndrome and select other muscular dystrophies.

John F. Crowley, Chairman and Chief Executive Officer ofAmicus Therapeutics, Inc., stated, This is a remarkable advancement in the history of Amicus and further strengthens our great collaboration with Dr. Jim Wilson and the Gene Therapy Center at Penn. Philadelphia is a magnet for talent in gene therapy and an engine for innovation. This new global research center located in the cradle of liberty will become part of the cradle of cures as we move many gene therapy programs forward toward patients in need. With exclusive global rights to 50 rare diseases in collaboration with Dr. Wilsons team we hope to be able to alleviate an enormous amount of human suffering with the great science work that will be done in this new facility.

The 75,000 sq. ft. Center is located on the top three floors of the new building at 3675 Market Street and consists of office and state-of-the-art laboratories. It will ultimately house approximately 200 researchers and drug developers focused exclusively on gene therapies.

A by invitation only ribbon cutting event takes place today to celebrate the opening with special guests to include Dr. Jim Wilson, government officials and patients living with rare diseases and their families.

About Amicus TherapeuticsAmicus Therapeutics (FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com and follow on Twitter and LinkedIn.

CONTACTS:

Media:Christopher ByrneExecutive Director, Corporate Communicationscbyrne@amicusrx.com(609) 662-2798

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Amicus Opens New Global Research and Gene Therapy Center of Excellence in Philadelphia - Yahoo Finance

Allergan and Editas Medicine have made history after scientists used a CRISPR treatment developed by the partners to edit cells inside the human body.

Until now, researchers have used CRISPR in human trials, but removed patients cells and edited them outside the body before re-injecting them a process known as ex vivo treatment.

Allergan and Editas gene therapy AGN151587 or EDIT-101 is designed to treat a rare, inherited form of blindness known as Leber congenital amaurosis 10 (LCA10).

The condition often first occurs in infancy, and those with the condition have specific mutations in the genes responsible for the development of the retina, the part of the eye that detects light.

The historical CRISPR moment occurred in the phase 1/2 BRILLIANCE study, after the first LCA10 patient was dosed with the CRISPR genome editing medicine inside the body or in vivo treatment.

Allergan and Editas plan to treat a further 18 LCA10 patients with EDIT-101, to evaluate the treatments safety, tolerability and efficacy.

Although there is a gene therapy available for Lebers Sparks Luxturna it doesnt work for the specific gene mutation which causes LCA10.

In comparison to gene therapies, which insert a working copy of the gene which is missing in a rare disorder, CRISPR therapies are designed to modify the gene itself, instead of supplying a working copy.

CRISPR technology does this by breaking a specific place within DNA which triggers a self-repair mechanism.

However, instead of repairing the original sequence, CRISPR serves as a new template that can be used to modify the sequence and correct a faulty gene.

Although Allergan and Editas have claimed the first in vivo instance of CRISPR treatment, a number of pharma companies and biotechs are carrying out research in the area.

That includes Vertex and CRISPR Therapeutics the partners recently revealed initial positive data from the first to patients treated with their investigational CRISPR/Cas9 therapy CTX001 for the treatment of severe haemoglobinopathies.

AstraZeneca is also making a play in the CRISPR field, with a collaboration on a CRISPR-focused research programme with the Wellcome Trust Sanger Institute, the Innovative Genomics Initiative, Thermo Fisher Scientific and the Broad Institute.

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Allergan and Editas claim a CRISPR first in inherited blindness study - PMLiVE

Second highest year for global financings in regenerative medicine, with nearly $10 billion raised globally

Washington, DC, March 05, 2020 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM) today announced the release of its 2019 Annual Report and Sector Year in Review, highlighting the organizations key priorities and initiatives, as well as offering an in-depth look at trends and metrics for the cell therapy, gene therapy and tissue engineering sector.

ARM, which celebrated its 10th anniversary in 2019, is the premier international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector. In its 2019 annual report, the organization provides an update on its work with industry, national and international regulatory agencies, public and private payers, patient organizations and other stakeholders to create a positive environment for the development of and access to these innovative therapies.

Using data sourced from ARMs data partner Informa, the report also provides analysis on industry-specific statistics and trends from nearly 1,000 leading cell therapy, gene therapy, tissue engineering, and other regenerative medicine companies worldwide. Key features of the report include total financings for the sector, partnerships and other deals, clinical trial information, anticipated near-term product approvals and regulatory filings, and expert commentary from industry representatives in the US and Europe.

Key findings from the 2019 annual report include:

Globally, companies active in gene and cell therapies and other regenerative medicines raised nearly $10 billion in 2019, the second highest year on record. Venture financings were particularly strong, making up more than $4 billion in global financings a 33% increase over 2018.

There were 1,066 clinical trials underway worldwide by year-end 2019. 10+ product candidates are poised for approval, and the number of approved gene therapies will likely double in the next one to two years.

Companies headquartered in Europe raised $3 billion, the strongest year on record, and were sponsoring 260 trials by the end of the year.

There is a supportive policy environment for regenerative medicines, with policymakers showing a strong interest in promoting the development of, and patient access to, these innovative therapies.

ARM will continue to update this information through new reports to be released after the close of each quarter, tracking sector performance, key financial information, clinical trial numbers, and clinical data events.

Story continues

The report is available to download onlinehere, with interactive data and downloadable infographics availablehere. Past reports, issued quarterly and annually, are availablehere.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

Kaitlyn Donaldson Dupont803-727-8346kdonaldson@alliancerm.org

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The Alliance for Regenerative Medicine Releases 2019 Annual Report and Sector Year in Review - Yahoo Finance

An emergency room physician, initially unable to diagnose a disoriented patient, finds on the patient a wallet-sized card providing access to his genome, or all his DNA. The physician quickly searches the genome, diagnoses the problem and sends the patient off for a gene-therapy cure. Thats what a Pulitzer prize-winning journalist imagined 2020 would look like when she reported on the Human Genome Project back in 1996.

The Human Genome Project was an international scientific collaboration that successfully mapped, sequenced and made publicly available the genetic content of human chromosomes or all human DNA. Taking place between 1990 and 2003, the project caused many to speculate about the future of medicine.

In 1996, Walter Gilbert, a Nobel laureate, said, The results of the Human Genome Project will produce a tremendous shift in the way we can do medicine and attack problems of human disease. In 2000, Francis Collins, then head of the HGP at the National Institutes of Health, predicted, Perhaps in another 15 or 20 years, you will see a complete transformation in therapeutic medicine. The same year, President Bill Clinton stated the Human Genome Project would revolutionize the diagnosis, prevention and treatment of most, if not all, human diseases.

It is now 2020 and no one carries a genome card. Physicians typically do not examine your DNA to diagnose or treat you. Why not? As I explain in a recent article in the Journal of Neurogenetics, the causes of common debilitating diseases are complex, so they typically are not amenable to simple genetic treatments, despite the hope and hype to the contrary.

The idea that a single gene can cause common diseases has been around for several decades. In the late 1980s and early 1990s, high-profile scientific journals, including Nature and JAMA, announced single-gene causation of bipolar disorder, schizophrenia and alcoholism, among other conditions and behaviors. These articles drew massive attention in the popular media, but were soon retracted or failed attempts at replication. These reevaluations completely undermined the initial conclusions, which often had relied on misguided statistical tests. Biologists were generally aware of these developments, though the follow-up studies received little attention in popular media.

There are indeed individual gene mutations that cause devastating disorders, such as Huntingtons disease. But most common debilitating diseases are not caused by a mutation of a single gene. This is because people who have a debilitating genetic disease, on average, do not survive long enough to have numerous healthy children. In other words, there is strong evolutionary pressure against such mutations. Huntingtons disease is an exception that endures because it typically does not produce symptoms until a patient is beyond their reproductive years. Although new mutations for many other disabling conditions occur by chance, they dont become frequent in the population.

Instead, most common debilitating diseases are caused by combinations of mutations in many genes, each having a very small effect. They interact with one another and with environmental factors, modifying the production of proteins from genes. The many kinds of microbes that live within the human body can play a role, too.

Since common serious diseases are rarely caused by single-gene mutations, they cannot be cured by replacing the mutated gene with a normal copy, the premise for gene therapy. Gene therapy has gradually progressed in research along a very bumpy path, which has included accidentally causing leukemia and at least one death, but doctors recently have been successful treating some rare diseases in which a single-gene mutation has had a large effect. Gene therapy for rare single-gene disorders is likely to succeed, but must be tailored to each individual condition. The enormous cost and the relatively small number of patients who can be helped by such a treatment may create insurmountable financial barriers in these cases. For many diseases, gene therapy may never be useful.

The Human Genome Project has had an enormous impact on almost every field of biological research, by spurring technical advances that facilitate fast, precise and relatively inexpensive sequencing and manipulation of DNA. But these advances in research methods have not led to dramatic improvements in treatment of common debilitating diseases.

Although you cannot bring your genome card to your next doctors appointment, perhaps you can bring a more nuanced understanding of the relationship between genes and disease. A more accurate understanding of disease causation may insulate patients against unrealistic stories and false promises.This article is republished from The Conversation under a Creative Commons license. Read the original article.

Read more here:
Why Sequencing the Human Genome Failed to Produce Big Breakthroughs in Disease - Discover Magazine

A large biotech is partnering with a firm developing cell and gene therapies on treatments for neurological diseases like Alzheimers and Parkinsons.

Cambridge, Massachusetts-based Biogen said Thursday afternoon after markets closed that it had partnered with Brisbane, California-based Sangamo Therapeutics in a deal that could be worth up to $2.7 billion. The partnership will initially focus on two preclinical Sangamo gene therapy candidates ST-501 for tauopathies such as Alzheimers and ST-502 for synucleinopathies like Parkinsons disease, plus an undisclosed neuromuscular target. It also includes exclusive rights for up to nine other undisclosed neurological targets.

Biogen will pay Sangamo $350 million upfront, which includes a license fee and equity investment, while Sangamo will be eligible for up to $2.37 billion in milestone payments, plus royalties.

Shares of Sangamo were up more than 28% on the Nasdaq after markets opened Friday. The company had also announced its fourth quarter and full year 2019 financial results. Biogens shares were down 2.6%.

Sangamo had reached out to multiple companies in a competitive process. While declining to say how many companies the biotech had spoken to, Sangamo head of corporate strategy Stephane Boissel said in a phone interview that it had put together multiple term sheets.

Its a combination of economics, but also the expertise of that partner in that particular field, Boissel said, referring to why the company had chosen Biogen. Biogen, in the pharma world, is probably the best franchise when it comes to neurology.

Adrian Woolfson, Sangamos executive vice president for research and development, said in the same call that it was also because of an appreciation for Biogens enthusiasm and energy.

I think its fair to say we had a very good chemistry with them at a personal level when we went to meet with them in Boston, and we seemed to get along very well, Woolfson said.

Sangamo has existing partnerships with a number of other firms, including Pfizer and Gilead Sciences.

Biogens moves into Alzheimers disease have not been without controversy. The company plans to file for Food and Drug Administration approval of aducanumab, a monoclonal antibody targeting the amyloid beta protein that has long dominated Alzheimers research. The company initially halted the Phase III development program for the drug when it was predicted to fail, but revived it when a post-hoc analysis indicated potential efficacy. Investors have remained skeptical.

Still, that did not come up in the minds of Sangamos executives, Boissel said. While emphasizing that he could not compare the two companies approaches, Woolfson added that gene therapies are potentially better ways to address neurological diseases like Alzheimers because they can switch off genes completely rather than being limited to taking out specific proteins, as monoclonal antibodies are.

ST-501 targets tau, another protein that has been researched as a potential therapeutic target in Alzheimers. ST-501 and ST-502 use adeno-associated viral vectors to deliver zinc finger protein transcription factors (ZFP-TFs), a form of gene therapy that Sangamo said in its quarterly earnings presentation is ideally suited to neurological disorders due to its ability to up- or down-regulate gene expression.

Boissel did not disclose specific timelines for ST-501 and ST-502, but noted that the next steps in their development will be preclinical studies to enable them to enter the clinic.

Photo: John Tlumacki, The Boston Globe, via Getty Images

Continued here:
Biogen teams up with Sangamo in gene therapy deal worth up to $2.7B - MedCity News

Repligen says it will continue to expand capacity to feed demand for its OPUS range of prepacked chromatography columns.

For the full year 2019, revenues at bioprocess vendor Repligen Corporation came in at $270 million (250 million), up from $194 million in 2018. Revenues grew across all the firms divisions, with organic growth of 20%, 30% and 40% for Repligens proteins, filtration and chromatography franchises, respectively.

And within the chromatography franchise, the firms OPUS range of prepacked chromatography columns stood out.

Image: iStock/Oakozhan

Our OPUS business finished up over 30% for the quarter and 50% for the year. The story in the quarter was the continued adoption of our prepacked column technology in CDMOs and gene therapy accounts, which now account for 20% of our OPUS revenue, said CEO Tony Hunt in a conference call discussing results.

We delivered approximately 1,400 columns to our customer base in 2019, up from 700 columns in 2018.

Part of this was the demand driven by a robust biologics market. In the US alone, 10 new mAbs [monoclonal antibodies] and two gene therapy drugs were approved in 2019, said Hunt. With a rich pipeline of over 1,000 biological drug candidates, the expectations are high for strong growth in the years ahead.

And increased capacity allowed Repligen to feed the demand. We were able to improve our lead times significantly by bringing five new production suites online in December, said Hunt.

Last year, the firm announced investment plans to add two OPUS manufacturing suites at its Ravensburg, Germany site and six suites at its Waltham, Massachusetts. Those were expected to come online during the first three to nine months of 2020, so its not clear whether the five suites which began production in December formed part of that investment.

We expect continued momentum for OPUS in 2020 as our customers scale and expand. We expect our OPUS franchise to grow at or above 20% in overall chromatography at 15% in 2020.

Repligen also spoke about the growing demand for its products from the gene therapy sector, something Hunt was vocal about at last months JP Morgan Healthcare conference.

Talking last week, Hunt said Repligen has more than 50 significant customers in the gene therapy space, and the sector now represents around 15% of the firms total business.

We havent seen any slowdown in gene therapy, and were expecting gene therapy will grow about 30% for us in 2020.

Gene therapy was cited as chief among the many tailwinds that underpinned Repligens accelerated growth in 2019 by Jefferies analyst Brandon Couillard.

By our math, this surge in gene therapy demand accounted for two-thirds of Repligens accelerated core growth of +33% in 19 (from +17% in 18), he wrote in a note.

Looking ahead, with the cell/gene therapy market expected to grow 2-3x faster than the broader ~$10 billion bioprocessing market over the next three to five years, the mix shift should continue to be accretive to its overall growth profile over time.

Here is the original post:
Repligen up on OPUS and gene therapy demand - Bioprocess Insider - BioProcess Insider

24 Feb 2020

Another gene therapy for a central nervous system disorder has posted positive results. The Phase 1/2 trial enrolled 18 men with X-linked retinitis pigmentosa (RP), a disease of retinal degeneration that leads to blindness. Robert MacLaren of the University of Oxford led the study, published February 24 in Nature Medicine.

The dose-escalation trialtested six doses; the therapy consisted of an adeno-associated virus 8 expressing a normal copy of the RP GTPase gene. Seven patients saw improvements in their vision that endured for the six-month duration of the trial. Inflammation within the eye cropped up at the higher doses, which may have temporarily blurred therapeutic effects in some patients.

Interest in using gene-based therapy for AD and other neurodegenerative disorders has grown ever since such a therapy was approved for treating spinal muscular atrophy (SMA) in babies and toddlers, and the field is now looking to learn from trials of other nervous system disorders (Nov 2019 news;Dec 2019 news).

X-linked RP is caused by mutations in the RP GTPase regulator (RPGR) gene. The mutations trigger degeneration of photoreceptors starting in childhood. No treatments exist. Recent approval of a gene therapy for another retinal disorderRPE65-related retinal degenerationsuggests retinal gene therapy could work (Russell et al., 2017).

However, the RPGR gene has confounded scientists, as it contains a repetitive, purine-rich stretch that undergoes alternative splicing. Tinkering with the sequence has boosted fidelity and stability of the gene, and conferred therapeutic benefits in animal models of the disease (Fischer et al., 2017).

In this trial, each of the 18 men, who were between 22 and 50 years old, had severe retinal degeneration. They were recruited in six cohorts of three patients each, who received increasing doses of the viral therapy. The participants received an injection of the virus into their subretinal space; they were then monitored for safetythe trials primary outcomeand tested for vision and retinal sensitivity for six months.

Across the cohorts, 55 adverse events occurred, all mild. Seven out of nine patients on the three highest doses experienced mild retinal inflammation, which was corrected by oral corticosteroids. In all, the trial met its primary safety endpoint.

Seven out of 12 patients receiving one of the top four doses had visual gains in the treated eye. This was gauged by retinal microperimetry, a map of the quality of light perceived across the retina. The improvements started at one month and continued at the six-month follow-up. The researchers proposed that inflammation in the top three dose cohorts may have offset visual improvements in some patients.

The trial was not designed to draw conclusions about efficacy. The researchers speculated that the therapys effectiveness will boil down to the stage of retinal degeneration, vector dose, and any interfering effects of inflammation. The second phase of the study will compare two doses to placebo.

Though the eyes are not strictly the brain, retinal therapy can be considered as part of the revitalization of gene-therapy approaches for neurodegenerative diseases. AAV-based expression of survival motor neuron 1 has improved life for those with SMA, but the tricky aspects of RPGE gene expression, and the inflammatory response that cropped up at higher doses, point to potential challenges other gene therapies may have to overcome (Nov 2016 news; Mendell et al., 2017).Jessica Shugart

No Available Further Reading

See the original post here:
Retinal Gene Therapy Trial Posts Positive Result - Alzforum

If you ever end up in an emergency room, the first thing that happens is a doctor or nurse will check your critical vitals: your temperature, blood pressure, respiratory rate, and pulse. If those indicate your life is at risk, your care is prioritized over others who have already been waiting or who have been seen by a doctor but require additional testing (e.g. an x-ray or blood test) or a specialist to review their symptoms. This process, called triaging, is the global standard for allocating resources in emergency care.

Triaging is a marvel of modern healthcare if it is abundantly clear that you are on deaths door. But if theres no textbook description of your condition, it can leave you needlessly suffering while the experts try to figure it out. Thats often the case for those with rare diseases, a group of conditions that are not individually common, but combined, affect an estimated 10% of the global population, some 475 million people. An estimated 80% of the 7,000 identified rare diseases are caused by DNA mutations that occur during pregnancy, meaning most of those with this category of illness are born with it. In many cases, these babies emerge from the womb with life-threatening conditions that doctorsworking the triage systemwill immediately address. However, this also tends to lead doctors to then ignore the underlying rare diseasean unnecessary medical expenditure, in the triage system framework.

I am one of the people living with a rare disease.

I was born with a number of symptoms and signs that put my life at risk a collapsed lung, a premature exit from my mothers body after only six months, and malnutrition from a hole in my amniotic sac. These problems were all treated and resolved over a multiple month stay in the hospital dictated by the triage system. I was allocated hospital resources for my life-threatening conditions until I was deemed stable enough to go home: the point at which I wouldnt die if I left the care of the hospital.

But there was another problem that was overlooked, and which wasnt diagnosed until I was five years old.

Every bone in my body was bent and every muscle atrophied or non-existent. I couldnt move my neck away from shoulder; I couldnt straighten my legs, knees, arms, wrists, ankles, toes, hands, or fingers beyond fixed, fully bent positions.

The triage system worked at saving my life, but never addressed how I would live day to day or even physically move from a single location by myself. It never addressed the underlying issue and root of the problem: a rare orthopedic genetic disease.

The life or death triage standard is one of the primary reasons that it takes, on average, seven years for people with a rare disease to get a diagnosis in the U.S.and

I am one of the lucky ones. Serendipitously, a Because of these surgeries, and additional ones Ive had since, I could feed myself, live free from a wheelchair, go to school through the post-graduate level, and hold a full-time job.

The orthopedic surgeries I neededover 29 of them in less than 30 yearshave cost millions of dollars. And these costs grow each year as I undergo additional exploratory surgery in the absence of any cure. However, these costs are still lower than what I would have incurred had I been left as the triage system deemed stable as an infant. I would have required 24-hour in-home care my entire life. I would never have been able to use the bathroom alone, to get dressed alone or to even leave the house alone. The lifetime value of a working individual according to the US Office of Management and Budget is on average $7 million to $9 million. The cost of a full-time caregiver is on average $40,320 a year; if a rare-disease patient reaches the average US life expectancy of 78 years old, the lifetime cost of full-time care is at least $3 million.

Our healthcare system needs to weigh the long-term costs of leaving behind people with rare disease, and, more specifically, evaluate the economic consequences that follow at a global scale.

Even more so, we need to weigh the costs of creating a pipeline to fill the treatment gap facing people with rare disease., Using genomic sequencing, clinicians can holistically understand the genetic roots of rare disease and even potentially cure rare disease through gene therapy, which modifies and permanently fixes abnormal genes that cause a specific rare disease at birth.

Nonetheless, identifying the root genetic cause of rare disease is the only way to begin to cure a rare disease rather than just treating the symptoms in an ad hoc fashion. While certain pharmaceutical drugs can be developed from the findings in a genomic sequence to help mitigate or lessen symptoms, the science suggests the only way to cure a rare disease is to administer an even newer science called gene therapywhich modifies and permanently fixes genes that are abnormal. Considered to be the most expensive option, a However, this nascent science is costly: gene therapy costs around $2 million for current US Food and Drug Administration (FDA)-approved options.

Insurance plans in the US rarely pay for clinical-grade whole-genomic sequencing (which can carry a price tag of up to $9,000)let alone gene therapies. Yet if the most expensive cost to cure a rare disease is $2 million, thats still far less than $3 million for a lifetime of full-time care (which excludes additional expenses). And new studies, like one the World Economic Forum released in the lead up to this years International Rare Disease Day, show that we can ultimately save money in the long term by funding more treatments and, as an added benefit, potentially develop more cures by learning when treatments work and when treatments dont work.

We cant create clinical pathways for the more than 7,000 rare diseases overnight, but we need a standard of care that goes beyond using death as the primary barometer of focus, over-simplifies the complexity of what it means to be healthy, and only considers short term costs. An economically effective, new model could center on allocating resources with the end goal to allow people to reach a level of health that provides basic mobility or basic independencea level of health allowing economic productivity. We are living in a time of unprecedented medical innovation, and our system of coverage needs to catch up. We can do better than just keeping people alive.

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Contact us at editors@time.com.

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The Bad Economics of the U.S. Health Care System Shows Up Starkly in its Approach to Rare Diseases - TIME

The IPO market is feeling the effects of the coronavirus outbreak with a surge in the VIX volatility index weighing on IPO activity. Since 2015, a week in which VIX volatility has surpassed 35 has been followed by a week averaging two US IPO pricings. Our observational trend continues through 2020, as one biotech entered the public market this past week. Six IPOs and three SPACs submitted initial filings with the SEC.

Passage Bio (PASG), a preclinical biotech developing gene therapies, priced at the high end of the range to raise $216 million at a $840 million market cap. The deal raised 72% more in proceeds than Passage Bio originally filed for. The company is furthering the research from UPenn's Gene Therapy Program, which is headed by co-founder James Wilson. Bolstered by the recent performances of other large early stage biotech IPOs, Passage Bio finished up 23%, another sign that biotechs are pushing back against the effects of the coronavirus.

Chinese medical information platform Zhongchao (NASDAQ:ZCMD) began trading on Monday after raising $12 million in an IPO on Friday 2/21. The company was flat after its first day on the Nasdaq and is currently down 2%.

1 IPO During the Week of February 24th, 2020

IssuerBusiness

DealSize

Market Capat IPO

Price vs.Midpoint

First DayReturn

Returnat 02/28

Passage Bio

$216M

$840M

6%

+23%

+23%

Preclinical biotech developing gene therapies for rare CNS disorders.

Healthcare benefit management platform Accolade (ACCD) filed to raise $100 million, biotechs NLS Pharmaceutics (NLSP) and ORIC Pharmaceutics (ORIC) filed for $40 million and $86 million, respectively. Construction software Procore Technologies (PCOR) filed for an estimated $400 million. Medical device maker Pulmonx (LUNG) filed for $86 million, and marketing data platform ZoomInfo (ZI) filed for $500 million. SPACS, Social Hedosophia II (IPOB.U), Social Hedosophia III (IPOC.U), and GigCaptial3 (GIK.U) filed for $300 million, $600 million, and $200 million.

9 Filings During the Week of February 24th, 2020

IssuerBusiness

DealSize

Sector

LeadUnderwriter

Accolade

$100M

Health Care

Goldman

Provides a platform that helps employees manage their healthcare benefits.

NLS Pharmaceutics

$40M

Health Care

Maxim

Biotech focusing on therapies for CNS and sleep disorders.

ORIC Pharmaceuticals

$86M

Health Care

JP Morgan

Phase 1 biotech developing small molecule therapies for cancer.

Procore Technologies

$400M

Technology

Goldman

Provides cloud-based construction management and collaboration software.

Pulmonx

$86M

Health Care

BofA

Makes minimally invasive medical devices for emphysema.

Social Hedosophia II

$300M

SPAC

Credit Suisse

Second blank check company formed by Social Capital and Hedosophia targeting US tech.

Social Hedosophia III

$600M

SPAC

Credit Suisse

Second blank check company formed by Social Capital and Hedosophia targeting ex-US tech.

ZoomInfo

$500M

Technology

JP Morgan

Provides a data platform for sales, marketing, and recruiting professionals.

GigCapital3

$200M

SPAC

Nomura

Third blank check company led by semiconductor veteran Avi Katz.

The Renaissance IPO Indices are market cap weighted baskets of newly public companies. As of 2/27/20, the Renaissance IPO Index was up 1.4% year-to-date, while the S&P 500 was down 7.8%. Renaissance Capital's IPO ETF (NYSE: IPO) tracks the index, and top ETF holdings include Uber (NYSE:UBER) and Spotify (NYSE:SPOT). The Renaissance International IPO Index was up 2.2% year-to-date, while the ACWX was down 9.3%. Renaissance Capital's International IPO ETF (NYSE: IPOS) tracks the index, and top ETF holdings include Adyen and Meituan-Dianping.

Original Post

Editor's Note: The summary bullets for this article were chosen by Seeking Alpha editors.

Continued here:
IPO Weekly Recap: Yes, The IPO Market Has Caught The Coronavirus - Seeking Alpha

Rare Disease Day is celebrated across the globe to raise awareness amongst the general public and policymakers about rare diseases and how they impact patients lives. The first Rare Disease Day was celebrated in 2008 on February 29 because of its rare date and since then, occurs on the last day in February each year, a month with a rare number of days.

Although rare suggests not many people are affected with a condition, collectively, 300 million people around the world live with a rare disease and they face similar challenges. The barrier to an accurate diagnosis means patients may doctor hop and spend years getting a host of tests done because no one is familiar with the condition and can diagnose it. Theres often frustration due to this lack of understanding from health care professionals, and living in the unknown.

Even with a diagnosis, more than 90% of rare diseases are still without an FDA approved treatment, according to the National Organization for Rare Disorders.

Some rare diseases may cause a multitude of different health problems that keep children from going to school or being able to socialize with others in the same way their peers can. Similarly, rare diseases may affect physical appearance and make children self-conscious or have low self-esteem.

LISTEN UP: Add the newMichigan Medicine News Breakto your Alexa-enabled device, orsubscribe to our daily updates oniTunes,Google PlayandStitcher.

Michigan Medicine researchers are constantly working to better understand the mechanisms behind rare diseases. Education helps health care professionals make accurate diagnoses, create treatment methods and improve the quality of life for those that live with these conditions.

Heres a sampling of their research from the last year.

Systemic Scleroderma Treatments: Where Are We Now?A new and novel outcome measure is being used to determine effectiveness of new scleroderma treatments.

Gene Therapy Treatment Targets Rare Mutation Tied to BlindnessAdvances in gene therapy are yielding new options for treating inherited retinal degenerations, giving specialists new tools and new hope for patients and families.

A Mission to Improve Cystic Fibrosis Treatment Across the GlobeTo reach patients in need, one doctor has developed atraining program to improve testingand care available for those with thegenetic disease, starting intheMiddle East.

Accelerating Childrens Access to New Treatments for High Risk Brain TumorsMichigan Medicine joins an exclusive, global network that helps speed up the process of linking children with incurable brain cancer to promising clinical trials.

A New Clue in the Mystery of ALS, Frontotemporal DementiaMichigan Medicine researchers identify a potential therapeutic target for neurodegenerative conditions using animal models.

Drug Trial Seeking First Ever Treatment for Dangerous Side Effect of Prader-Willi SyndromeA worldwide research effort is underway for finding a treatment option for hyperphagia, the most common genetic cause of life threatening childhood obesity.

Arthritis Treatment Could Provide Relief for Lichen Planus Skin RashIts often difficult to manage patients with this skin inflammation, but new research identifies a target that existing medications may be able to address.

Approach Could Help in Treating Glioblastoma, Other Rare CancersMichigan led research presents a new way of uncovering predictive biomarkers when data from large randomized trials arent available.

Sickle Cell Disease Could Be Treated by Turning Back the ClockReactivating genes normally active before birth could prevent the harmful effects of this blood disorder with few treatment options.

Registry Helps Move Aortic Dissection Care Forward Diagnosis, treatments and outcomes for acute aortic dissection have evolved, with an international registry revealing trends and the power of using data.

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To learn more about Rare Disease Day, visit the National Organization for Rare Disorders website.

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10 Studies That Highlight the Importance of Rare Disease Research - Michigan Medicine

Newswise CLEVELANDResearchers at Case Western Reserve University have used a unique method to safely deliver gene therapy to fight a rare, but irreversible, genetic eye disorder known as Stargardt disease.

By using chemically modified lipidsinstead of the viruses most commonly used as carriersStargardt disease did not return in animal models for up to eight months after treatment, said lead researcher Zheng-Rong Lu, the M. Frank Rudy and MargaretDomiter Rudy Professor ofBiomedical Engineering at the Case School of Engineering, with a dual appointment at the School of Medicine.

Lus research, recently published in the journal Molecular Therapy, was funded in part through a Gund-Harrington Scholar grant, a partnership between Harrington Discovery Institute at University Hospitals and the Foundation Fighting Blindness.

This grant provides innovative scientists like Dr. Lu both funding and drug development expertise to advance research that will defeat diseases that limit millions of people from experiencing the gift of sight, said Jonathan S. Stamler, MD, President, Harrington Discovery Institute and Robert S. and Sylvia K. Reitman Family Foundation Distinguished Chair of Cardiovascular Innovation at University Hospitals and Case Western Reserve University School of Medicine. It is very encouraging to see Dr. Lus work move closer to human trials.

A so-far incurable disorder

The research may give some hope to people with Stargardt disease, an inherited disorder of the retina also known as macular dystrophy or juvenile macular degeneration because it often surfaces during childhood or adolescence.

Although individuals with Stargardt disease rarely go completely blind, they progressively lose vision in both eyes, become very sensitive to light and, in some cases, develop color blindness.

Were really excited because there is a potential to help people with Stargardt, Lu said. But we believe this success could also apply to other disorders as a platform therapy for delivering other genes through the use of the lipids.

Lipids are simple organic compounds, or fatty acids, that are insoluble in water, including various natural oils, waxes and steroids.

Because they are basically unsaturated oil, the likelihood of damage to the eye is low, which isnt always true with the viral gene therapy, Lu said.

Gene therapy offers best hope

There are a lot of researchers trying to figure out how to treat this disease right now with little success, Lu said. The best hope is gene therapy.

Gene therapy is the technology in which genetic material is introduced into cells by an engineered carrier to compensate for abnormal genes or to make a beneficial protein.

The most successful gene therapy carriers so far have been certain viruses (the AAV, or adeno-associated virus, especially) because they can deliver the new gene by infecting the target cell.

But the Stargardt-associated gene, known as the ABCA4 gene, turns out to be too large to fit within that popular virus, Lu said.

Lu said other researchers have attempted to remedy that problem by splitting ABCA4 into pieces and then trying to reassemble it inside the eyewith limited success.

Other researchers have modified a larger virus to carry ABCA4 into the eyes, a technology tested in human trials as far back as 2011, but which still hasnt been fully commercialized.

He said he and his collaborators have also already met with investors to expedite the commercialization of the platform used for Stargardt.

Further, this gene therapy product could be classified as an orphan drug by the U.S. Food and Drug Administration (FDA) because Stargardt is a rare disease, increasing the likelihood of faster FDA approval after clinical trials, Lu said.

The non-viral gene therapy is also much more cost-effective for production than the virus-based therapy and has a potential to significantly reduce the high price of gene therapy in the eye, he said.

We think that within two to three years we could really be helping people after further demonstration of its safety and efficacy, Lu said.

MEDIA CONTACTS:

Michael Scott, Case Western Reserve University

216.368.1004, mike.scott@case.edu

Carly Belsterling, University Hospitals

412.889.8866, carly.belsterling@uhhospitals.org

###

Case Western Reserve University

Case Western Reserve University is one of the country's leading private research institutions. Located in Cleveland, we offer a unique combination of forward-thinking educational opportunities in an inspiring cultural setting. Our leading-edge faculty engage in teaching and research in a collaborative, hands-on environment. Our nationally recognized programs include arts and sciences, dental medicine, engineering, law, management, medicine, nursing and social work. About 5,100 undergraduate and 6,200 graduate students comprise our student body. Visit case.edu to see how Case Western Reserve thinks beyond the possible.

Harrington Discovery Institute

The Harrington Discovery Institute at University Hospitals in Cleveland, OHpart of The Harrington Project for Discovery & Developmentaims to advance medicine and society by enabling our nations most inventive scientists to turn their discoveries into medicines that improve human health.The institute was created in 2012 with a $50 million founding gift from the Harrington family and instantiates the commitment they share with University Hospitals to a Vision for a Better World.

The Harrington Project for Discovery & Development

The Harrington Project for Discovery & Development (The Harrington Project), founded in 2012 by the Harrington Family and University Hospitals of Cleveland, is a $300 million national initiative built to bridge the translational valley of death. It includes the Harrington Discovery Institute and BioMotiv, a for-profit, mission-aligned drug development company that accelerates early discovery into pharma pipelines. For more information, visit:HarringtonDiscovery.org.

Read more:
Scientists successfully test new way to deliver gene therapy - Newswise

A health ministry panel approved Wednesday the production and sale of a gene therapy for treating spinal muscular atrophy, a rare incurable disease that destroys muscle function through a decrease in motor neuron cells.

The health minister is expected to formally approve the drug, Zolgensma, by the end of March, with public health insurance coverage seen starting this summer.

The one-time-only gene therapy, developed by Swiss drug giant Novartis AG, will cover patients below 2 years old with abnormalities in their genes to make proteins necessary for maintaining motor nerves. Novartis estimates 15 to 20 children a year are likely to receive treatment using the drug in Japan.

In the therapy, proteins are created after the genes, carried by adeno-associated viruses, are delivered to motor nerves through intravenous drips. In an overseas clinical trial, all of the 15 severe SMA patients who used the therapy were able to live without an artificial ventilator. If no treatment is offered, about 75 percent of such patients are expected to die or need an artificial ventilator before they reach the age of 13.6 months.

The therapy, which has already been approved in the United States, is expensive, costing the dollar equivalent of 230 million for a one-time infusion.

View original post here:
Japanese government panel OKs expensive gene therapy for rare disease - The Japan Times

For the cell and gene therapy revolution to be fullyrealized, physicians, research scientists, biomanufacturing experts, advocacygroups, regulatory bodies like the Food & Drug Administration (FDA) andother key stakeholders have to Think Different, as Apple famously encouraged.

The vein-to-vein, one batch to one patientformula of personalized medicine is radically and rapidly forcing changes onbiomanufacturing where cell and gene therapy best practices are not yetcalcified and are changing as we speak. Personalized medicine supply chainchallenges are emerging and still being worked through and the high cost ofthese therapies remains a daunting challenge for life science companies andpatients.

Cell and gene therapy challenges across R&D, manufacturing, commercialization, and supply chain as well as bioethical challenges yet to be fully confronted or resolved have been well documented and discussed by experts across the BioHealth Capital Region (BHCR).

One company Jeeva InformaticsSolutions, Inc. (Jeeva) in Herndon, Virginia is developing BigData solutions for a less publicized but equally urgent cell and gene therapychallenge: How to manage a radically new form of clinical trial that could spandecades. Founder and CEO of Jeeva, Harsha K. Rajasimha, is building a companythat leverages Big Data, AI and mobile tech to decentralize clinical trials.Jeevas goal is to make it easier for biotech companies to collect, aggregate,analyze and report required clinical trial data while helping patients stayenrolled and compliant over longer periods of time with little travel from theconvenience of their homes.

The FDA recently passed newindustry guidelines requiring long-term follow up (LTFU) periods ofup to 15 years for gene and cell therapies, representing a sea change for howclinical trials will operate in the future.

The cell and gene therapy space provides significant hope for cures that can reverse genetic mutations. This space is growing. There are more than 900 clinical trials ongoing. Hundreds if not thousands of genetic diseases are likely to have therapies for the first time in our lifetime, stated Rajasimha.

At Jeeva, we are trying to solve a number ofissues but have focused on one particular problem that needs to be addressed ifthese cell and gene therapies are going to be delivered to patients: No oneknows the long term implications of these therapies because they are a one anddone type treatment. If a patient receives therapy today, they are done, and sothe FDA has mandated that recipients need to be monitored for up to 15 years.We are looking to address this issue with digital health technologies and AI,he added.

Jeeva believes that these long-term monitoringchallenges can be managed by making clinical trial participation easier viamobile applications, video conferencing consultations and centralizedscheduling, to name just a few of Jeevas product features. Leveraging digitalhealth tech can reduce the need for travel and eliminate inconveniences thatmight cause a patient to become non-compliant or, worse, drop out of a trialaltogether.

Decentralized clinical trials eliminate the heavy burden of patient travel and makes the process simpler and more efficient. Utilizing a Bring-Your-Own-Device (BYOD) approach and an eVisit consultation model to create decentralized trials can reduce brick and mortar visits by 20% to 80%, according to Jeeva.

Thenew FDA guidelines is the latest attempt to grapple with unchartedsafety protocols for cell and gene therapies. Clinical trial challenges are notnew to the biotechnology industry, however; rather, the approach to clinicaltrials has been inefficient and static for decades, leading to industry-wideproblems with clinical trial enrollment and recruitment that has a dominoeffect that lengthens the commercialization process and increases drugdevelopment costs.

We want to be a catalyst for accelerating thedrug development and delivery process. Patient recruitment is a huge barrierand has made the biopharmaceutical industry unsustainable. The average cost ofbringing a drug to market is $2.5B and takes 10-15 years to get to market. Wefeel that by educating and informing the global community about clinical trialsand enrollment opportunities using AI and digital health tech, we can help allstakeholders in getting people earlier access to treatments and getting thetreatments to markets faster, Rajasimha said.

Jeeva not only can help small to midsize biotechs improve trial recruitment and longer-term safety monitoring, but the company uses AI to improve clinical trial operations. By using AI and high tech tools, biotech companies can leverage historical trial data to shape new trials while empowering real-time adjustments to trials based on real-time monitoring to improve overall success rates.

Rajasimha continued, We have been building AItools to solve our customers specific needs, not just for AIs sake. Wesurveyed our customers last year and they told us Every single clinical trialseems like the first trial ever conducted by mankind. Even companies likePfizer and Novartis, which have been conducting hundreds of trials for decades,feel that when they launch a new trialit is no more efficient than theprevious trial.

So, we have been building an AI assistant that learns from past clinical trials data to make the next trial more efficient, he added. Rajasimha quickly reinforced patient centricity by saying it is not something you fix with an all technology solution, unless the robustly tested technology solution is combined with the human elements and focus on patients perspectives. Having been a global patient advocate in the U.S. over the past six years has given me a unique perspective on how to integrate technology in the lives of patients and caregivers. Moreover, a growing number of trials are recruiting patients from multiple countries and reducing international travel burden on patients over extended durations will be critical to achieve enrollment.

While the concept of virtual clinical trialsmight seem futuristic, Rajasimha and the Jeeva team believe the market is readyfor change.

A number of pilot projects or proof of concept clinical trials, about 20 of them, have been published where patients didnt go to the clinic at all. The feasibility of conducting such remote patients studies has been validated multiple times by the industry now. The tipping point has arrived. One of the key barriers for widespread adoption of decentralized clinical trials was a lack of FDA guidelines. Now the FDA has clarified its expectations about how the industry and stakeholders can share the responsibility to reduce the burden on patients. Enough validation and regulatory guidelines have put us in a position to give our customers what they need, stated Rajasimha.

Rajasimha sees partnering with smaller to midsize biotechs early on in the drug development process meaning well before the start of Phase II or III trials as an inflection point where it can deliver the greatest impact. In addition, we are seeing some initial interest from the Medical Cannabis industry, opioid crisis intervention for chronic pain management, and patient advocacy groups, where patients often live in remote, rural areas, can also benefit from decentralized, hybrid virtual clinical trials. Finally, real-world evidence studies, or longitudinal cohort studies, is also a growing market because companies need to collect and manage patients across longer time horizons, which is Jeevas sweet spot.

Rajasimha and Jeeva are starting to see this growing market interest manifest itself in new funding partnerships. Jeeva recently announced that CIT GAP Funds had invested in the company. Jeeva is currently in an early-stage investment round and the company is in active product development with a validated prototype. Jeeva is seeking new customer pilot projects to add to its ongoing pilots, which include chronic pain, medical cannabis, oncology and cell, and gene therapy products. Later this year, the company plans to complete multiple pilot projects and have validation in Good Clinical Practices (GCP) settings.

Rajasimha and his Jeeva team are certainlyembracing a think different approach to the future of clinical trials. Jeevaand its AI-driven, virtual clinical trial model is poised to help biotechcompanies thrive and meet the unmet medical needs of more patients across theglobe.

You can listen to Rajasimhas interviewwith podcast host Daniel Levine earlier this month on iheart radio here.

Team Jeeva is seeking customer pilot projectsand strategic partners to join the journey and will be exhibiting at the NationalInstitutes of Health Rare Disease Day event on Feb 28, 2020.Rajasimha will also be delivering a keynote speech on AI in rare diseases atthe BIO-IT World West Conference at San Franciscoon March 3rd, 2020.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

Originally posted here:
This Startup is on a Mission to Decentralize Cell and Gene Therapy Clinical Trials - BioBuzz