StemCell Therapy

CHICAGO, Nov. 26, 2020 /PRNewswire/ -- BASE10 Genetics and DNA Link today announced their collaboration on a research project to evaluate the usability of DNA Link's AccuFind COVID-19 IgG antibody test in a healthcare setting.

Study will expand access to antibody testing outside of laboratory settings

Although Covid-19 deaths and new confirmed cases continue to rise across nursing homes in the US, many of those infected do recover. This has led to growing interest in the relationship between antibodies and their protective value whether generated by previous Covid-19 exposure or by a vaccine, once available.

At present, most antibody tests on the market require samples to be sent to a lab for analysis. The extra cost and time associated has constrained population wide studies of SARS-CoV-2 antibodies.

"An antibody test that can be done at the point of care without the need for a lab can more quickly provide information clinicians and administrators need to make decisions for their nursing home patients and staff," says Dr. Michael Fang, BASE10 CEO.

"This research is an important milestone in our efforts to expand access to antibody testing," says Elizabeth Freund, VP of Innovation at BASE10. "DNA Link's AccuFind performs very well in laboratory settings. The next logical step is to test its performance when used by healthcare professionals outside the lab. Many factors have the potential to affect test performance in lesser controlled settings so it's an ambitious goal to be sure. However, if FDA believes the data demonstrates its safety and effectiveness under these conditions, we'll be one step closer to FDA emergency use authorization for point of care use."

The study is set to launch in December.

About BASE10 Genetics, Inc.BASE10 is a precision medicine company based in Chicago. BASE10's proprietary platform identifies precision diagnostics technologies with high potential for improving health outcomes, and creates turnkey, disease management programs that can be deployed at scale for insurance payors.

For more information about BASE10 Genetics and its solutions, contact media@base10genetics.com.

About DNA Link, Inc.DNA Link, Inc. is a leading genomic service corporation based in Korea with a focus on personalized medicine and new drug development. The company combines biotechnology with information & communications technology to support total analysis of genomic and clinical information to accelerate research and development in the field of bio-industry and genomic analysis industry.

For more information about DNA Link and its solutions, contact ikjun.choi@dnalink.com

View original content:http://www.prnewswire.com/news-releases/base10-and-dna-link-partners-on-research-to-support-authorization-of-antibody-test-at-the-point-of-care-301181072.html

SOURCE BASE10 Genetics, Inc.

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BASE10 and DNA Link Partners on Research to Support Authorization of Antibody Test at the Point of Care - BioSpace

The Personalized Medicines Market grew in 2019, as compared to 2018, according to our report, Personalized Medicines Market is likely to have subdued growth in 2020 due to weak demand on account of reduced industry spending post Covid-19 outbreak. Further, Personalized Medicines Market will begin picking up momentum gradually from 2021 onwards and grow at a healthy CAGR between 2021-2025

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Key product type:PM DiagnosticsPM TherapeuticsPersonalized Medical CarePersonalized Nutrition & Wellness

Key applications:Hospitals PharmaciesRetail PharmaciesDietary Care CentersOthers

Key players or companies covered are:3G BiotechQuest DiagnosticsLaboratory Corporation of AmericaAbbottAgendia NVAsuragen IncBecton DickinsonCardioDx Inc.Foundation MedicineQiagenSiemens HealthcareAmgenBristol-Myers SquibbGE HealthcarePfizerSanofi23andMeIllumina

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Contact us:i2iResearch info to intelligenceLocational Office: *India, *United States, *GermanyEmail: [emailprotected]Toll-free: +1-800-419-8865 | Phone: +91 98801 53667

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Personalized Medicines Market Segmentation by Application, Market landscape, Market size and forecast by 2026 - The Market Feed

Latest added DNA Sequencing Market research study by MarketDigits offers detailed product outlook and elaborates market review till 2026. The market Study is segmented by key regions that is accelerating the marketization. At present, the market is sharping its presence and some of the key players in the study are GE Healthcare Life Sciences, Johnson & Johnson, Commonwealth Biotechnologies, ZS Genetics, Agilent Technologies, Siemens AG. The study is a perfect mix of qualitative and quantitative Market data collected and validated majorly through primary data and secondary sources.

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The global DNA sequencing market is projected to achieve an impressive growth rate on the back of its extensive usage in a number of applications and ease of adoption with the emergence of innovative technologies in the field. A DNA sequencing Android app named GelApp 2.0 has been recently introduced with a 56.0% greater band detection accuracy for proteins and 36.0% for DNA than its original version. The interesting, rather most innovative, part of the apps development procedure was the implementation of Monte Carlo Tree Search, a gaming algorithm, for cutting-edge image processing.

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New market players are envisioned to get attracted to the global DNA sequencing market as technological innovations keep taking precedence. Besides this, established players can expect to leverage new opportunities in the market with sophisticated technologies such as chain terminator DNA sequencing and next generation DNA sequencing. Vendors can also find lucrative opportunities in the clinical field as DNA sequencing is highly demanded in numerous clinical applications. Next generation DNA sequencing, particularly, is receiving high attention with the providence of novel platforms from the ongoing quantum leaps in microfluidics, bioinformatics, nanotechnology, and imaging. Such breakthroughs are also predicted to revolutionize a number of life science fields, thus providing an easy path of growth for the global market.

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Among other prominent players in the global DNA sequencing market, Microchip Biotechnologies, Beckman Coulter (Fullerton, CA), Abbott Laboratories, GE Healthcare Life Sciences, Johnson & Johnson, Commonwealth Biotechnologies, ZS Genetics, Agilent Technologies, Siemens AG, Pacific Biosciences, Myriad Genetics, Bayer Corporation, Integrated DNA Technologies, 454 Life Sciences, Hamilton Thorne Biosciences, and LI-COR Biosciences (Lincoln, NE) are expected to make their presence known.

Market segmentation based on type:

Market segmentation based on technology:

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DNA Sequencing Market Reports Table of Contents

1.1. Market Definition and Scope

1.2. Market Segmentation

1.3. Key Research Objectives

1.4. Research Highlights

4.1. Introduction

4.2. Overview

4.3. Market Dynamics

4.4. Porters Five Force Analysis

5.1. Technological Advancements

5.2. Pricing Analysis

5.3. Recent Developments

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About Market Digits :

MarketDigits is one of the leading business research and consulting companies that helps clients to tap new and emerging opportunities and revenue areas, thereby assisting them in operational and strategic decision-making. We at MarketDigits believe that market is a small place and an interface between the supplier and the consumer, thus our focus remains mainly on business research that includes the entire value chain and not only the markets.

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DNA Sequencing Market : Overview Report by 2020, Covid-19 Analysis, Future Plans and Industry Growth with High CAGR by Forecast 2026 - The Courier

Janssen is tapping little-known and privately held Hemera Biosciences for a new gene therapy aimed at reversing a severe disease.

The deal, financials of which are not being shared, is for the investigational phase 1 gene therapy HMR59, which is given as a one-and-done shot into the eye with the aim of helping preserve vision in patients with geographic atrophy, a late-stage and severe form of age-related macular degeneration (AMD).

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

Patients with AMD often have low levels of CD59, a protein that protects the retina from damage caused by an essential part of the body's natural immune response called complement. In geographic atrophy, which affects around 5 million people globally, an overactivity of complement destroys cells in the macula, the central part of the retina.

HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, with the theory being it can help stop further damage and retain a patients vision. It has already passed a phase 1 test, with a second in patients with wet AMD currently conducting follow-up visits to evaluate long-term safety, according to a statement.

There are currently no medical treatments that can regenerate retinal cells that have atrophied, though gene therapies and stem cell therapies are the leading R&D hopes in this field.

Geographic atrophy is a devastating form of AMD that impacts the ability to accomplish everyday tasks, such as reading, driving, cooking, or even seeing faces, said James List, M.D., Ph.D., global therapeutic area head, cardiovascular and metabolism, Janssen R&D.

Our aim with this novel, single-administration gene therapy is to use our development expertise and deep heritage in vision care to help improve patient outcomes by intervening early, halting the progression to blindness, and preserving more years of sight.

RELATED: Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease

Janssen will hope to have the success seen from gene therapy Luxturna, a drug from Novartis and Spark Therapeutics for retinal degeneration caused by mutations in the gene RPE65, after the product was approved in 2017, and helped Spark into a Roche buyout in 2019.

Other companies are also on the hunt, such as the recent tie-up between Biogen and Harvard University's Massachusetts Eye and Ear thats aimed at developing a gene therapy to help some patients with these blinding diseases.

The gene at the center of the new agreement, PRPF31, has been linked to autosomal dominant retinitis pigmentosa. Several other gene therapies are being developed to treat retinitis pigmentosa. They include Allergans RST-001, which the company picked up when it acquired RetroSense Therapeutics for $60 million in 2016.

Iveric Bio is also working on a geographic atrophy therapy, known as Zimura (avacincaptad pegol), with it posting clinically meaningful late-stage follow-up data this summer for the complement C5 inhibitor.

Read more from the original source:
Janssen buys up eye disorder gene therapy asset from Hemera Biosciences - FierceBiotech

Bayer has created a cell and gene therapy platform to support its growing pipeline of advanced therapy medicinal products. The platform is intended to enable Bayer to make its expertise and resources available to its partners while preserving their autonomy and culture.

Germanys Bayer has moved into cell and gene therapies on multiple fronts in recent years, buying up induced pluripotent stem cell specialist BlueRock Therapeutics and adeno-associated virus (AAV) gene therapy player Asklepios BioPharmaceutical while investing in a clutch of other biotechs. The deals have given Bayer a pipeline of five advanced assets and more than 15 preclinical prospects.

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

Rather than subsume BlueRock and AskBio, Bayer opted to allow the businesses to operate as independent companies in an attempt to preserve their cultures. Yet, Bayer also wants to enable the companies to realize the benefits that can come from being part of a larger organization.

The cell and gene therapy platform is the result of that effort to get the best of both worlds. Bayer will use the platform to provide support to its cell and gene therapy businesses and orchestrate its operations in the area across the product life cycle. Specific areas of support offered by the platform span preclinical through to commercial, strategy implementation and project management.

Bayer is investing in its internal capabilities to strengthen the platform as well as looking to enter into strategic collaborations, acquire technologies and strike licensing deals. The deals entered into so far have given Bayer infrastructure as well as product candidates.

Notably, AskBio has a CDMO unit, Viralgen, specializing in AAV gene therapy production. As limited access to manufacturing capacity has been a barrier to speedy gene therapy development, buying the CDMO could help Bayer remove a constraint on the progress of its programs and become a more attractive partner for startups. Bayer thinks allowing acquired startups autonomy makes it attractive, too.

Wolfram Carius, who joined Bayer from Sanofi in 2016, is heading up the new cell and gene therapy platform. Carius said the platform is vital to accelerate innovation at its source, and to ensure its translation into tangible therapies for patients who have no time to wait in a statement.

Bayers platform is a twist on strategies being pursued by many of its peers, which have identified cell and gene therapies as growth areas and bought in assets but sought to avoid smothering the startups. Kite, for example, operates as its own business unit within Gilead Sciences, and Spark Therapeutics is an independent company within the Roche group.

More:
Bayer creates cell and gene therapy platform to support partners - FierceBiotech

Gene therapy pioneer Jim Wilson and the University of Pennsylvania are teaming up with Regeneron to help deliver its COVID-19 antibody cocktail using adeno-associated virus (AAV) tech in the hope of curbing infection via a nasal spray.

The antibody cocktail, made up of casirivimab and imdevimab, was given a speedy authorization by the FDA less than two weeks ago as a treatment for certain COVID-19 patients. But, keeping up with the fast pace of SARS-CoV-02 R&D, Regeneron is not resting on its laurels and now wants to find a quicker way of delivering its therapy while also working on it as a prophylactic.

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

RELATED: Regeneron, following in Lilly's footsteps, wins FDA emergency nod for COVID-19 antibody cocktail

These antibodies are currently injected into patients, but Regeneron and Penn will use Wilsons gene therapy know-how to attempt a nasal spray formulation using AAV vectors. The belief is that this could prevent infection with the virus using a technology typically used in high-tech gene therapies.

The group plans to study the safety and effectiveness of using AAV vectors to introduce the sequence of the cocktails virus-neutralizing antibodies directly to nasal epithelial cells and see whether it can help protect against the disease.

The first step is to finish preclinical trials; if successful, an IND will be sent off to the FDA for human trials.

Wilsons team said it was hopeful that introducing the therapy via single dose of AAV will be able to produce similar protection Regeneron has seen for its cocktail, but for potentially a longer duration.

Regeneron scientists specifically selected casirivimab and imdevimab to block infectivity of SARS-CoV-2, the virus that causes COVID-19, and we have been encouraged by the promising clinical data thus far, said Christos Kyratsous, Ph.D., vice president of research, infectious diseases and viral vector technologies at Regeneron.

In the quest to use cutting-edge science to help end this disruptive and often very devastating disease, we are excited to explore alternate delivery mechanisms such as AAV that may extend the potential benefits of this investigational therapy to even more people around the world.

Read this article:
Wilson, Penn ink Regeneron pact to use gene therapy tech to deliver COVID-19 antibodies - FierceBiotech

Nadofaragene firadenovec, a novel intravesical therapy for nonmuscle-invasive bladder cancer (NMIBC) that does not respond to bacillus Calmette- Gurin (BCG) therapy shows efficacy across important patient subgroups, investigators reported at SUO 2020, the virtual annual meeting of the Society of Urologic Oncology.

In the original phase 3 trial of 157 patients with high-grade BCG-unresponsive NMIBC, clinicians delivered nadofaragene firadenovec (also known as rAd-IFNa/Syn3), a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, by catheter into the bladder epithelium once every 3 months. The 3-month complete response rate was 59.6% overall and 53.4% in patients with carcinoma in situ (CIS) with or without Ta or T1 tumors. In addition, 72.9% of patients with high-grade Ta or T1 tumors and no CIS had freedom from high-grade recurrence. At 12 months, 30.5% overall, 24.3% of patients with CIS (with or without Ta or T1 tumors), and 43.8% of patients with high-grade Ta or T1 tumors (and no CIS) were free from high-grade recurrence.

For both the CIS and high-grade Ta/T1 only cohorts, there were no significant differences in response rates at 3 and 15 months between male and female patients, patients younger and older than 70 years, BCG-refractory vs BCG-relapsed disease, patients with more or less than 3 prior lines of therapy, number of prior non-BCG regimens, and patients with more or less than 3 prior courses of BCG, Vikram Narayan, MD, of Emory University in Atlanta, Georgia, reported.

Duration of response also did not differ significantly among groups, Dr Narayan reported. Only in the CIS cohort, patients who received 3 or fewer prior courses of BCG had significantly longer duration of response compared with patients who received more than 3 courses: 12.68 vs 4.96 months. A multivariable analysis confirmed that none of these baseline characteristics or prior therapy significantly influenced response rates at 3 and 15 months or duration of response.

These results demonstrate the efficacy of nadofaragene firadenovec regardless of patient characteristics or prior treatment history, Dr Narayan stated. Nadofaragene firadenovec represents a potential novel treatment option for patients with high-grade BCG-unresponsive NMIBC that advances the current treatment paradigm.

The FDA is currently reviewing a biologics license application (BLA) for nadofaragene firadenovec.

Disclosure: This clinical trial was supported by FKD Therapies in Oy, Finland. Please see the original reference for a full list of authors disclosures.

References

Narayan V, Boorjian S, Alemozaer M, et al. Subgroup analyses of the phase 3 study of intravesical nadofaragene firadenovec in patients with high-grade, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). Presented at: SUO 2020, December 3-5, 2020. Poster 23.

Boorjian SA, Alemozaffar M, Bad Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Published online November 27, 2020. Lancet Oncol. doi:10.1016/S1470-2045(20)30540-4

Kulkarni GS. Nadofaragene firadenovec: a new gold standard for BCG-unresponsive bladder cancer? Published online November 27, 2020. Lancet Oncol. doi:10.1016/S1470-2045(20)30586-6

TechBear. FerGene announces Landmark Phase 3 study published in Lancet Oncology [news release]. FerGene; November 30, 2020.

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Gene Therapy for NMIBC Effective Regardless of Patient Characteristics - Renal and Urology News

Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act.

But a key analyst covering biopharma believes that theres a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, hes pointing to the 2 leaders in the field as the most likely players to come out ahead way, way ahead.

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UPDATED: Bayer continues its cell and gene therapy push, enveloping different projects under one strategic roof while hunting new deals - Endpoints...

"The need for reproducible, scalable, and economical production of cell and gene therapies is creating a demand for digital bioprocessing technologies," said Nitin Naik, Global Life Sciences Vice President at Frost & Sullivan. "These technologies are critical to realize the true commercial potential of cell and gene therapies in the next two to three years and serve as a conduit to improve market access and control the total cost of therapy."

Naik added: "From a market segment perspective, while the stem cell market is lucrative, the highest growth is expected to be in gene-modified cell therapies, with a pipeline of 269 products,* followed by gene therapies, which account for 182 assets in the pipeline.* Further, although allogeneic stem cell therapies dominate the marketed product catalogs, interest in disease-modifying CAR-T therapies, which are largely autologous, is driving demand for the evolution of manufacturing technologies, models, and capacity expansion investment by CDMOs." (*as of August 2020)

To tap into the growth prospects exposed by the CGT market, companies must focus on:

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026 is the latest addition to Frost & Sullivan's Healthcare research and analyses available through the Frost & Sullivan Leadership Council, which helps organizations identify a continuous flow of growth opportunities to succeed in an unpredictable future.

About Frost & Sullivan

For six decades, Frost & Sullivan has been world-renowned for its role in helping investors, corporate leaders and governments navigate economic changes and identify disruptive technologies, Mega Trends, new business models and companies to action, resulting in a continuous flow of growth opportunities to drive future success.Contact us: Start the discussion.

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026

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Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan - PRNewswire

Bayer has launched a Cell and Gene Therapy (C>) Platform, aiming to accelerate its presence in this area.

Bayer is planning to strengthen its capabilities for its internal C> activities, while also pursuing external strategic collaborations, technology acquisitions and licensing.

The overall aim is to establish robust platforms with broad application against a range of therapeutic areas.

The G> Platform will combine multiple functions to provide support across the entire value chain for the research and development of cell and gene therapies.

Bayer has made a series of acquisitions over the last few years to bolster its presence in C>, including its recent acquisition of Asklepios Biopharmaceutical which closed on 1 December, and its acquisition of BlueRock Therapeutics in 2019.

These two companies will be the first to be integrated into the new C> Platform, Bayer has announced in a statement.

The German pharma company added that it has also established a C> pipeline consisting of five advanced assets and over 15 preclinical candidates.

This is a defining moment for Bayer. Cell and gene therapies are leading innovation in healthcare, and it is our goal to be at the forefront of this revolution in science, said Stefan Oelrich, member of the board of management, Bayer AG and president of its pharmaceuticals division.

The C> field is growing at an unprecedented pace. With the establishment of Bayers own C> Platform our company will propel its presence in this area. This will complement our existing C> pipeline which already includes five advanced assets with at least three investigational new drugs annually for the next years, he added.

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Bayer establishes new cell and gene therapy platform - PharmaTimes

SALT LAKE CITY (Ivanhoe Newswire)

Gene replacement therapy: Its a game-changer when it comes to treating life-threatening illnesses. It can replace disease-causing genes with healthy genes, knock out a gene thats not working right, or add a new gene to the body to help fight disease. To date, the FDA has approved four types of gene therapy including one that was given the OK just in time to save one little boys life.

No doubt about it, Cinch Wight is going to be a cowboy just like his dad.

He loves the dog and the horses and the cows, shared Cinchs dad, Alex Wight.

But it has been a wild ride for this young bronco. A mandatory newborn screening test at birth revealed Cinch had spinal muscular atrophy or SMA.

Cinchs mom, Amber Wight recalled, That was the first time Id ever even heard the term and what it was. And so, it was very scary.

A neuromuscular disorder that can paralyze a baby in the first few weeks of life.

My first thought was, hes never going to be able to ride broncs or anything like that, expressed Alex.

But just one day after Cinch was born, the FDA approved a new gene therapy.

We were pretty excited to get a phone call from the department of health, you know, and have this baby here who we can use this treatment on after its approval, explained Russell Butterfield, MD,pediatric neurologist at University of Utah Health and Intermountain Primary Childrens Hospital.

A critical gene in little Cinch was missing. Pediatric neurologist Russell Butterfield used an infusion to deliver a virus carrying a new copy of the gene into Cinchs nerve cells.

Its like a delivery truck to deliver genes to where you want them to go. What that does do, is it stops the disease right where it is, elaborated Dr. Butterfield.

Just a few years ago, most children born with SMA didnt make it to their second birthday. Now?

The hardest is holding a baby in one hand and holding that drug in the other and really feeling the weight of that. And understanding that how different this childs life will be with his new medicine, expressed Dr. Butterfield.

It took a lot of courage for this family to get this far. Thats why Alex wrote a book for his son. A true story about how real cowboys never give up.

I wanted to let him know that no matter how hard it gets, as long as he keeps going, hell be all right, shared Alex.

Doctors dont know if the one-time infusion will last a lifetime or will have to be repeated and there could be a possible risk of inflammation to the liver that doctors will closely monitor. The gene replacement therapy costs 2.1 million dollars. Insurance paid for most of it, but Alex hopes sales from his childrens book will help pay the rest. You can find the book, A Cowboy and His Horse, at https://www.amazon.com/COWBOY-HIS-HORSE-ALEX-WIGHT/dp/B08CWG46ZX.

Contributors to this news report include Cyndy McGrath, Executive Producer; Marsha Lewis, Field Producer; Rusty Reed, Videographer; Roque Correa, Editor.

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A little cowboy saved by groundbreaking gene replacement therapy - Wink News

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 02, 2020 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient has been dosed in the high-dose cohort of the Phase 1/2 (Stage 1) study for Type I (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.

AXO-AAV-GM1 is the only gene therapy in the clinic targeting patients with Type I and Type II GM1 gangliosidosis, a devastating and fatal pediatric disease, said Gavin Corcoran, M.D., Chief R&D Officer of Sio. The initiation of the high-dose cohort builds on evidence of extension of survival in naturally-occurring GM1 disease animal models and encouraging clinical data from an expanded access study conducted by a National Human Genome Research Institute (NHGRI) team led by our principal investigator, Dr. Cynthia Tifft at the National Institutes of Healths (NIH) Clinical Center. Our team and academic partners are dedicated to improving the lives of children affected by this devastating disease, and we look forward to reporting topline data from the low-dose cohort before year end.

The Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 delivered intravenously in patients with Type I and Type II GM1 gangliosidosis.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation and is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.

GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the -galactosidase enzyme. Currently, there are no approved treatment options for GM1 gangliosidosis. In 2019, Sio reported clinically meaningful improvements from baseline to six-month follow-up for the first GM1 Type II child dosed with low-dose AXO-AAV-GM1 gene therapy under an expanded access protocol.

About AXO-AAV-GM1

AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as will, expect, believe, estimate, and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

David NassifSio Gene Therapies Inc.Chief Financial Officer and General Counsel(646) 677-6770investors@siogtx.com

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Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis - BioSpace

When I first started at CHEO as a neuromuscular expert in 2010, there were absolutely no treatments available, he said, noting that children diagnosed then with SMA were given the necessary nutritional and respiratory supports, and assisted devices such as wheelchairs, to help them live the best life possible.

But research at CHEO, led by Dr. Alex MacKenzie, who discovered a gene NAIP which may contribute to the severity of the disease, helped develop treatment therapies.

One of them, which Aidan was first given when he was about three weeks old, is known as Spinraza. Approved in Canada, it corrects how the faulty gene produces certain proteins, but requires repeated treatments via spinal taps throughout the persons life.

A second treatment, a gene-replacement therapy called Zolgensma, only needs to be administered once, but is not yet approved in Canada and is prohibitively expensive: more than $2 million in the U.S.

Our immediate families volunteered to remortgage their houses and help fundraise, recalls Adam, while the couple planned to sell their house and move in with family.

Fortunately, they didnt have to. Through the manufacturers managed access program, the drug was made available on a compassionate basis to Aidan, who received it two weeks after his first treatment.

Theres no definitive test to say how well Aidans treatment has worked, but, according to Dr. McMillan, many of the newborns who initially received Zolgensma are now five and six years old, and they continue to show robust strong effects to the gene-replacement therapy, and theyve had no symptoms of clinical deterioration and no signs of any wearing-off effects.

For Sully and Deschamps, the proof so far is in seeing their son approach his first birthday with no signs of the disease. We see him as a physically active child who climbs two sets of stairs with ease and cruises around the house like he owns it. He dances to music, he throws and chases balls, and he chases our cats. We are so thankful.

bdeachman@postmedia.com

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Ottawa baby a pioneer of gene therapy for rare disease - Windsor Star

Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.

With 61 codons specifying 20 amino acids, some can be encoded by more than one codon and it is often presumed that it does not matter which one a gene uses. When I first studied genetics, some books I read taught that mutations between such alternative codons (eg, GGA->GGC, both giving glycine) were called synonymous mutations, while others referred to them as silent mutations. However, are synonymous mutations really silent meaning they are identical in terms of fitness and function? Although they may specify the same amino acid, does that mean they are all the same?

Figure 1: Intronless GFP transgene expression is higher for variants of GFP with higher GC content at synonymous sites5

Perhaps one of the biggest surprises over recent years has been the discovery that versions of the same gene, differing only at synonymous sites, can not only have different properties, but effects that are not modest.1-5 For example, two versions of green fluorescent protein (GFP) differing only at synonymous sites can have orders of magnitude differences in their expression level.4 We similarly recently discovered that for an intronless transgene to express in human cell lines it needs to be GC rich, which can be achieved by altering the synonymous sites,5 as seen in Figure 1. It is no accident, we suggest, that the well-expressed endogenous intronless genes in humans (such as histones) are all GC rich and that our functional retrogenes tend to be richer in GC content than their parental genes.

The realisation that synonymous sites matter has clear relevance to the design of transgenes or other artificial genes, be these for experiments, gene therapy, protein production (eg, in bacteria) or for vaccine design. In the case of vaccines, we might wish to modulate a viral protein to be effectively expressed in human cells to illicit a strong and robust immune response.6 Conversely to the design of attenuated vaccines, we seek to produce a tuned down version of the virus that can function but is weak.7

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered. One approach is mass randomisation try many alternatives and see what works.4,8,9 In principle this is fine, but this approach requires many randomisations, which is still technically difficult for long attenuated viruses. An alternative strategy that we have been exploring is to let nature tell us; we can apply tools and ideas from population genetics to better understand what natural selection favours and disfavours and in turn to estimate the strength of selection.

it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus

Estimation of the strength of selection is possible from knowledge of the site frequency spectrum, (ie, how common variants are) from which we can infer the distribution of fitness effects (DFE). If a site is under strong purifying selection, then mutations may occur in the population but these are rapidly eliminated, so variants are always rare. By contrast, if they are selectively neutral, we expect some variants to be quite common. We recently applied this methodology to show that synonymous mutations in human genes that disrupt exonic splice enhancer motifs are often under strong selection and affect many synonymous sites in our genes.10 This has implications for both diagnostics and for transgene design for gene therapy, as we often remove introns in heterologous genes, so freeing up these residues from their role in specifying exons ceases.11

The same DFE methodology cannot easily be applied to viruses, as the methods assume free recombination (ie, we assume one mutation does not impact the fate of others in the same genome). However, other population genetical tools can still be applied. Recently, we examined SARS-CoV-2 and identified the profile of mutations that we see at four-fold degenerate sites.12 From this profile we could estimate what the synonymous site composition would be, assuming that the only forces are mutational biases and neutral evolution (ie, no selection). We observed that in this genome there is a strikingly strong C->U mutation bias and a G->U one. In the raw data this is not so obvious as G and C are quite rare. However, the mutability of the sites per occurrence of the site reveals the underlying patterns.

Figure 2: The rate of mutational flux from one dinucleotide to another in the coding sequence of SARS-CoV-2. The direction of flux is indicated by the indentation of the connecting links: the inner layer represents flux out while the outermost layer represents flux into the node. The frequency of the flux exchange is represented by the width of any given link where it meets the outer axis. Dinucleotide nodes are coloured according to their GC-content. Hence, it is evident that there is high flux away from GC-rich dinucleotides whereas AU-rich dinucleotides are largely conserved.12

With knowledge of the mutational bias we then asked what the equilibrium frequency of the four nucleotides would be using four simultaneous equations. This is the nucleotide content at which for every mutation changing a particular base there is an equal and opposite one creating the same base somewhere else in the genome, ensuring overall unchanged nucleotide content. Given the strong C->U and G->U mutational biases, it is no surprise that the equilibrium content is very U rich (we estimate equilibrium U content should be about 65 percent). However, while the four-fold sites are indeed U rich, they are not that U rich, being closer to 50 percent. A clue as to why the mutation bias is so skewed to generating U comes from analysis of equilibrium UU content: UU residues are predicted to be very common, with CU residues being particularly mutable generating UU (Figure 2) this is expected due to human APOBEC proteins attacking and mutating/editing the virus.13

One probable explanation for this difference between predicted and observed nucleotide content is selection against U content. There may be many U residues appearing in the population, but many are pushed out of the population owing to purification selection, ie, because of the deleterious effects of the mutations. That such selection is happening in the SARS-CoV-2 genome is also clear from the sequence data. We estimate that for every 10 mutations that appear in the sequence databases, another six are lost because of selection prior to genome sequencing. Indeed, UU content is about a quarter of that predicted (Figure 3).

Figure 3: The predicted (under neutral mutational equilibrium) and observed dinucleotide content of SARS-CoV-2. Note the very high predicted levels of UU given the strong mutational flux to UU residues (see Figure 2) and the net underrepresentation in actual sequence.9

This leaves two problems: why is selection operating on SARS-CoV-2 and what can we do with this information? In some cases, we have a good idea as to why: many mutations to U at codon sites generate stop codons. However, we have observed that U destabilises the transcripts and is associated with lower-reported transcript levels;12 a full explanation of the causes of selection on nucleotide content therefore requires manipulation of the sequences.

The second question, what to do with this information, is perhaps more urgent. It has previously been noted that nucleotide content manipulation is a viable means to attenuate viruses.7 Currently there are three groups investigating this route to make a vaccine for SARS-CoV-2: Indian Immunologicals Ltd/Griffith University, Codagenix/Serum Institute of India and Acbadem Labmed Health Services/Mehmet Ali Aydinlar University. In prior attempts, attention has been paid to CpG levels and UpA levels (which we find to be correlated between SARS genes and between different viruses).12 CpGs attract the attention of zinc antiviral protein (ZAP) and UpA attracts an RNAase L. Not surprisingly, some viruses, including SARS-CoV-2, therefore have low levels of both dinucleotide pairs given the levels of the underlying nucleotides.

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered

In the past, attenuation strategies have focused on modulating synonymous sites to increase CpG and UpA, making the virus more visible to antiviral proteins.14 We in turn suggest a general strategy to utilise this method and to increase U content as well.12 Given the evidence that selection on the virus is to reduce U content, while our antiviral proteins are mutating it to increase U content, it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus. This is an unusual circumstance in which we predict that we should build in more of the already most common synonymous site nucleotides (U in this case) to degrade the virus. More generally, it is assumed that the most used codons are those that tend to increase the fitness of the organism. In the face of such a severe mutation bias, however, this simpler logic no longer holds.

Laurence D Hurst is Professor of Evolutionary Genetics and Director of the Milner Centre for Evolution at the University of Bath. He is currently also the President of the Genetics Society. He completed his D.Phil in Oxford, after which he won a research fellowship and then moved to Cambridge University as a Royal Society Research Fellow. While on the fellowship he assumed his current Chair at Bath University. In 2015 he was elected a Fellow of the Academy of Medical Sciences and a Fellow of the Royal Society. He is a recipient of the Genetics Society Medal and the Scientific Medal of the Zoological Society of London.

Related topicsDisease research, DNA, Gene Therapy, Genetic analysis, Genomics, Protein, Proteogenomics, Proteomics, Research & Development, RNAs, Vaccine

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Tweaking synonymous sites for gene therapy and vaccines - Drug Target Review

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTX), a next-generation gene therapy company with an industry-leading RNA targeting technology platform, announces today the unveiling of the AAVidTM capsid discovery platform and results from its first AAV5 variant library in a non-human primate selection campaign.

The AAVidTM capsid discovery platform uses non-random mutational fitness to create massive capsid libraries of billions of unique AAV variants for direct-to-NHP in vivo biological selection. By combining cutting-edge DNA synthesis, advanced synthetic biology, next-generation sequence barcoding and machine learning algorithms, ShapeTX generates industry-leading library size and diversity to enable the development of best-in-class human therapeutics.

Wildtype first-generation AAVs are enabling the recent advances in gene therapy, but they have been plagued by toxicities in the clinic due in part to a lack of tissue specificity, resulting in the need for high doses. Our AAVidTM platform solves the issue by creating novel capsid variants with specific tissue-tropism, said Francois Vigneault Ph.D., President and CEO at ShapeTX. Weve stayed quiet for the past three years while developing a superior AAV platform technology and are excited to announce that we have best-in-class AAV variants in hand. Today, we are announcing our novel liver-tropic AAV5 variants stay tuned for more to come.

David J. Huss, Ph.D., Vice President and Head of Research added, The vast structural space for exploration at the AAV capsid/target cell interface necessitates enormous library size and diversity, which until now, has only been probed with capsid library sizes in the tens of thousands to millions. At ShapeTX, we set out to create a superior AAV capsid discovery platform with library sizes in the billions of unique variants, thereby maximizing the opportunity for novel virus/target cell interactions. Dr. Huss presented the details of the platform at the 2nd RNA Editing Summit on Dec. 2, 2020.

About Shape Therapeutics, Inc

Shape Therapeutics is a biotechnology company developing next-generation RNA-targeted therapies to treat the worlds most challenging diseases. The ShapeTX technology platform includes RNAskip, a proprietary suppressor tRNA technology that enables premature stop codon readthrough; RNAfixTM, a precision RNA editing technology using endogenous Adenosine Deaminase Acting on RNA (ADAR); and AAVidTM, a next-generation engineered adeno-associated virus (AAV) platform producing highly specific, tissue-tropic AAVs. The power of the ShapeTX platforms resides in redirecting the cellular machinery already present in our cells, thereby bypassing the risks of immunogenicity and DNA damage seen with other contemporary editing technologies. ShapeTX is committed to data-driven scientific advancement, passionate people and a mission of providing life-long cures to patients. Shape Life!

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Shape Therapeutics Unveils AAVid Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--FerGene Inc., today announced that The Lancet Oncology published the Phase 3 data from the landmark U.S. clinical trial evaluating an investigational gene therapy, nadofaragene firadenovec (rAd-IFN/Syn3), for the treatment of patients with high-grade, Bacillus Calmette-Gurin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). In the study, patients received nadofaragene firadenovec, an intravesical therapy given once every three months that is believed to target the patients own bladder wall cells to enhance the bodys natural defenses to fight cancer.1

The Phase 3 study of 157 patients from 33 U.S. sites met its primary endpoint with more than half (53.4%) of CIS Ta/T1 patients (carcinoma in situ; with or without concomitant high-grade Ta or T1 disease) achieving a complete response (CR), all by three months. Of the patients who achieved a CR, 45.5% continued to remain free of high-grade recurrence at 12 months. In the study, nadofaragene firadenovec was administered directly into the patients bladder once every three months by a healthcare professional. The long-term follow-up phase of the four-year study is ongoing, and patients are continuing to be monitored.1

The most common adverse events (AEs) observed in the study that occurred in patients in order of decreasing frequency were: instillation site discharge, fatigue, bladder spasm, micturition urgency, and hematuria. The discontinuation rate due to AEs was 1.9%.1

Once patients with high-grade, non-muscle invasive bladder cancer no longer benefit from their initial BCG treatments, patients often make an informed decision to decline cystectomy a highly complex and life-altering bladder removal surgery or are often medically ineligible for this complex operation, leaving them with limited options, said Colin P. N. Dinney, MD, Chairman, Department of Urology, Division of Surgery, University of Texas MD Anderson Cancer Center and senior author of the publication. These data published in The Lancet Oncology show that nadofaragene firadenovec, a first-of-its-kind therapy, may be an effective treatment option for BCG-unresponsive non-muscle invasive bladder cancer patients.

As a practicing urologist, Im encouraged by these efficacy and safety data which demonstrate the potential for a novel treatment option that fits within the urology practice and gives patients the choice of receiving treatment once every three months which may be a particularly important consideration in this evolving healthcare environment, said Gennady Bratslavsky, MD, President of the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). Our organization is proud to have played a key role in the mid- and late-stage clinical studies for nadofaragene firadenovec.

Bladder cancer is the sixth most common cancer in the U.S., with NMIBC representing approximately 75% of all new bladder cancer cases.2,3 BCG remains the first-line standard of care for people living with high-grade NMIBC, however up to 50% of high-grade patients who receive initial treatment with BCG will experience recurrence and disease progression within one year becoming BCG-unresponsive.4,5

We believe the important clinical findings highlighted in The Lancet Oncology with this novel gene therapy may fulfill a significant unmet need for patients and have the potential to be practice-changing, said Vijay Kasturi, MD, Vice President of Medical Affairs at FerGene Inc. We are extremely grateful to the investigators, the patients who participated in the study, FKD Therapies and the SUO-CTC for the important findings highlighted in this publication.

A Biologics License Application (BLA) for nadofaragene firadenovec is currently with the U.S. Food and Drug Administration (FDA).

About Nadofaragene Firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational gene therapy being developed as a treatment for patients with high-grade, BCG-unresponsive NMIBC. It is a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene to do its work. The internal gene/DNA machinery of the cells picks up the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patients own bladder wall cells into interferon microfactories, enhancing the bodys natural defenses against the cancer. Nadofaragene firadenovec has been studied in a clinical trial program that includes 221 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.4 It is estimated that there will be approximately 81,000 new cases of bladder cancer in the U.S. in 20206; 75% of these cases present as NMIBC.3 In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, up to 50% of high-grade patients will experience disease recurrence within one year.4,5 The outcome for BCG-unresponsive patients is poor, with chemotherapy and radiation or total cystectomy (complete removal of the bladder) often being the recommended next treatment options.7

About the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC)

Created, owned and operated by its members, the SUO-CTC is a clinical research investigator network of over 500 members from more than 200 clinical sites in the U.S. and Canada. This national alliance of leading academic and community based uro-oncologists is committed to furthering urology research. The SUO-CTC is a registered 501c3 not-for-profit corporation and has a cooperative relationship with the Society of Urologic Oncology (SUO). The SUO-CTC pursues clinical trials, in concert with sponsors, to investigate therapeutic interventions which address urological cancers including, but not restricted to: Bladder Cancer, Prostate Cancer and Renal Cancer. Together with industry, the SUO-CTC offers enhanced research options for ultimately delivering better quality of life to our patients.

About FerGene Inc.

FerGene Inc. is a gene therapy company committed to revolutionizing the treatment of bladder cancer through its innovative science and unparalleled commitment to patient care. Founded in 2019, as a result of a collaboration between Blackstone Life Sciences and Ferring Pharmaceuticals, FerGene Inc. is singularly focused on evolving the bladder cancer treatment landscape through its novel approach to gene therapy. A trusted partner to medical and advocacy communities, FerGene Inc. is dedicated to bringing new hope to a patient population which has seen little improvement in their standard of care over the past twenty years. For more information, please visit http://www.fergene.com or engage with us on Twitter at @FerGeneBio or on LinkedIn.

2020 FerGene Inc. 20/09 US- ADST-2000114

1 Boorjian, S., Alemozaffar, M., Konety, B., Shore, N., Gomella, L., Kamat, A. et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol. 2020;2045(20)30540-4. doi:10.1016/S1470.2 National Cancer Institute SEER Program. Cancer Stat Facts: Common Cancer Sites. https://seer.cancer.gov/statfacts/html/common.html. Accessed March 5, 2020.3 Burger M, Catto JW, Dalbagni G, et al. Epidemiology and risk factors of urothelial bladder cancer. Eur Urol. 2013;63(2):234-41. doi:10.1016/j.eururo.2012.07.033.4 Sanli, O., Dobruch, J., Knowles, M. et al. Bladder cancer. Nat Rev Dis Primers. 3,17022 (2017) doi:10.1038/nrdp.2017.22.5 Kamat AM, Li R, ODonnell MA, et al. Predicting response to intravesical Bacillus Calmette-Gurin immunotherapy: Are we there yet? A systematic review. Eur Urol. 2018;73(5):738-748. doi:10.1016/j.eururo.2017.10.0036 American Cancer Society. Key Statistics for Bladder Cancer. https://www.cancer.org/cancer/bladder-cancer/about/key-statistics.html. Updated 2020. Accessed March 5, 2020.7 Marqueen K, et al. Identifying high surgical risk in muscle-invasive bladder cancer (MIBC) patients undergoing radical cystectomy (RC). JNCI Cancer Spectr. 2018 Oct; 2(4): pky075.

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Landmark Study in Non-Muscle Invasive Bladder Cancer Evaluating Breakthrough Investigational Gene Therapy Nadofaragene Firadenovec Published in The...

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that the Company will present at the Benzinga Global Small Cap Conference taking place virtually December 8-9. Genprexs President and Chief Executive Officer, Rodney Varner, will virtually deliver a company overview, including recent progress made on its upcoming Acclaim-1 clinical trial, to participating investors.

Event: Benzinga Global Small Cap ConferencePresentation Date: Tuesday, December 8Presentation Time: 2:30 p.m. ESTRegistration Link: https://bit.ly/2Jf5TcN

The Benzinga Global Small Cap Conference will provide investors with direct access to small caps for insights on their leadership, business fundamentals and plans for expansion. Attending investors will also have the opportunity to meet with management of presenting companies on a one-on-one basis.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, REQORSA (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for REQORSA for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone.

For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of REQORSA immunogene therapy drug, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining FDA approval of REQORSA and our other potential product candidates including whether we receive or benefit from fast track or similar regulatory designations; costs associated with developing our product candidates, whether we identify and succeed in acquiring other technologies and whether patents will ever be issued under patent applications that are the subject of our license agreements or otherwise. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex to Present at the Benzinga Global Small Cap Conference on December 8 - Business Wire

A little more than 2 years after Mercks Roger Perlmutter signed off on a deal that would pay Dragonfly up to $695 million for each drug program it picked off for solid tumors, the pharma giant is stepping up with their first opt-in.

We dont know exactly how much this deal costs Merck in the upfront, or which immunotherapy theyre getting, but its a major step forward for Bill Haneys Waltham, MA-based biotech, which built its TriNKET technology platform with the help of Tyler Jacks, an MIT professor, HHMI investigator and director of the David H. Koch Institute for Integrative Cancer Research as well as Berkeleys David Raulet, whose background as an expert in NK cells and tumor immunology helped spotlight some of the big ideas Dragonfly is pursuing.

This latest pact marks the latest in a flurry of BD deals for the pharma giant, just one last step before Perlmutter hangs it up as head of R&D and passes the reins to Dean Li. John Carroll

A little over a year since announcing its Series A, gene therapy biotech ViGeneron has entered into a new deal.

The German company is partnering with WuXi Advanced Therapies, a contract testing, development and manufacturing organization under WuXi AppTec based out of Philadelphia, to ramp up production of ViGenerons lead candidate VG901 for ophthalmic disorders. VG901s current target is for retinitis pigmentosa, also known as rod cone dystrophy, a degenerative eye disease that causes severe vision impairment as early as childhood.

Manufacturing for the candidate should begin before the year is out, ViGeneron said in a statement. The company added that the program came out of its proprietary vgAAV vector platform, which allows for better transduction of retinal cells as well as a less invasive treatment administration.

There is currently no cure for the disease, though there are some methods that can help manage symptoms like the use of low vision aids and portable lighting. Patients often experience worsening peripheral vision and trouble seeing at night. Max Gelman

Lentiviral vector manufacturer iVexSol has raised $13 million in Series A financing, bringing the total haul to $15.2 million from Casdin Capital and BioLife Solutions and a third undisclosed lead investor.

Founded on the promise to change the way this critical raw material is made using next-generation manufacturing tech, the company said it can produce LVVs at significantly greater quantities than traditional transient transfection processes. The companys name is short for intelligent vector solutions.

Much like adeno-associated viral vectors, or AAV, these delivery vehicles are crucial for cell and gene therapies such as CAR-T, iVexSol added, and their shortage means developers often have to wait 12 to 18 months for production slots.

Details on exactly how it plans to revolutionize the space are scant, but CEO Rod Rietze and CSO Mike Greene both bring technical experience from shops like Novartis and Pfizer.

Its new funding will help establish a facility in Lexington, MA housing stable LVV producer cell line master banks and commercial-grade LVV. Amber Tong

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News briefing: Merck's Roger Perlmutter buys his first solid tumor TriNKET from Dragonfly; ViGeneron to expand production of eye gene therapy -...

UCLA has received a $7.3 million grant from the National Institutes of Health to build a state-of-the-art facility in which to produce gene and cell therapies aimed at treating a host of illnesses and conditions.

The new 13,000-square-foot facility, to be constructed in UCLAs Center for the Health Sciences, will provide a highly regulated environment with features such as systems to manage air flow and filtering, laboratory spaces and bioreactors. The new facility is expected to be ready for use in 2023.

This grant provides critical funds to build a facility that will enable the development of a new generation of cellular therapies for cancer and other deadly diseases, said Dr. AntoniRibas, a UCLA professor of medicine and director of the Parker Institute for Cancer Immunotherapy Center at UCLA.

The new facility will be built according to U.S. Food and Drug Administrationgood manufacturing practices, a set of guidelines intended to ensure that facilities producing products for human use are built to maximize safety and effectiveness, and to reduce the risk for contamination.

It will replace a facility in UCLAs Factor Building that UCLA scientists currently use for similar research. But that space, which was put together by combining existing research laboratories, lacks the capacity to process certain cells and handle other bioengineered products, and it cannot accommodate the growing number of UCLA scientists pursuing research on gene and cell therapies, said Dr. Stephen Smale, vice dean for research at the David Geffen School of Medicine at UCLA and principal investigator of the NIH grant.

The new facility will be larger, so it will be able to support more projects simultaneously, and its design will allow a smooth flow of products into and out of the facility, Smale said. The larger number of rooms is really important because even when a single therapy is being tested, cells from each patient need to be processed in their own room.

Dr. Eric Esrailian, chief of theUCLA Vatche and Tamar Manoukian Division of Digestive Diseases, is helping to lead the expansion of UCLAs immunology and immunotherapy efforts. It will be a cornerstone for UCLAs commitments to building on existing strengths in the areas of immunology and immunotherapy and expanding toward the creation of a transformational institute in these fields, he said.

Despite the shortcomings of the current space, UCLA researchers have still produced groundbreaking work in it. These include tumor-targeting therapies developed by Ribas, Dr. Donald Kohn, Dr. Linda Liau, and other UCLA researchers.

Ribas, Kohn and Liau are also members of theUCLA Jonsson Comprehensive Cancer Centerand theUCLA Broad Stem Cell Research Center. Kohn is a distinguished professor of microbiology, immunology and molecular genetics and Liau is chair of UCLAs department of neurosurgery.

Kohn, who alsodeveloped a cure for bubble baby syndrome,said he will welcome the new facility because of its increased capacity for researchers to pursue treatments and cures that could significantly improve the health and quality of life of so many people. For instance, it will have the capacity to produce large batches of viral vectors microbes that make it possible to introduce potentially curative genes into cells for gene therapy studies.

This new facility will allow the innovative cell and gene therapies pioneered at UCLA to be available to a wider number of patients and accelerate the development of novel cures, said Kohn, whose work has also led to an experimental stem cell gene therapy for sickle cell disease that is showing promising early results in clinical trials.

Liau, a neuro-oncologist, said the new facility will enable researchers to create personalized vaccines and cell therapies for a much larger number of patients.

In the current facility, we are only able to enroll one patient at a time in our cell therapy trials, so many eligible patients have had to be turned away, Liau said.With greater capacity to manufacture gene and cell therapy products that meet FDA good manufacturing practice standards, this new UCLA facility will really allow us to further innovate and accelerate our translational research toward a cure for brain cancer.

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UCLA receives $7.3 million grant to build state-of-the-art facility for developing gene, cell therapies - UCLA Newsroom

UNM researchers discover certain materials combined with UV light can kill coronavirus and other viruses

As the deadly COVID-19 pandemic continues to wreak havoc around the world with no end in sight, new ways in which to stop the spread or mitigate the effects of the disease are few.

Although most experts agree that a vaccine would significantly slow or eventually stop the spread, the work to develop, approve and distribute such a vaccine are likely months away. That leaves us with only prevention efforts such as masks, social distancing and disinfecting, which partially due to human inconsistencies in behavior, have proven to be variable in effectiveness.

Despite these grim realities about the novel coronavirus that has taken 2020 by storm, disrupting the work, school and personal lives of nearly everyone on the globe, some University of New Mexico researchers have found a possible breakthrough in how to manage this virus, as well as future ones.

A team led by the Center for Biomedical Engineering faculty David Whitten, Distinguished Professor in the Department of Chemical and Biological Engineering, along with Eva Chi and Linnea Ista, faculty members in the same department, have found some light at the end of the tunnel, so to speak.

The main finding of their research, highlighted in the paper, Highly Effective Inactivation of SARS-CoV-2 by Conjugated Polymers and Oligomers, published this week in the journal ACS Applied Materials & Interfaces, involves the ability of the combination of certain polymers and oligomers, when combined with UV light, to almost completely kill the coronavirus.

UNM co-authors on the paper were Florencia A. Monge, of UNMs Center for Biomedical Engineering and the biomedical engineering graduate program; Virginie Bondu of the Department of Molecular Genetics and Microbiology at the UNM School of Medicine; Alison M. Kell, Department of Molecular Genetics and Microbiology at the UNM School of Medicine; and Patrick L. Donabedian of the nanoscience and microsystems engineering graduate program at UNM. Also on the team are Kirk S. Schanze and Pradeepkumar Jagadesan, both of the Department of Chemistry at the University of Texas at San Antonio.

Although disinfectants such as bleach or alcohol are effective against the virus, they are volatile and corrosive, which limit lasting sterilization of surfaces treated by these products, Whitten said.

What is different about these polymer and oligomer materials is that when activated with UV light, they provide a coating that is shown to be fast acting and highly effective, reducing the concentration of the virus by five orders of magnitude, Chi said.

These materials have shown to have broad-spectrum antiviral properties, she said.

Whitten points out that in order for the material to be active against the virus, it must be exposed to light. Light activates the docking process that is important and necessary for placing the oligomer or polymer at the surface of the virus particle, allowing the absorption of light that generates the reactive oxygen intermediate at the surface of the virus particle.

As far as we know so far, materials such as ours are not active against SARS-CoV-2 in the dark and require activation by irradiation with ultraviolet or visible light, depending on where the specific antimicrobial absorbs light, he said. In the dark, our antimicrobial materials dock with the virus, and then on irradiation, they activate oxygen. It is this active, excited state of oxygen that starts the chain of reactions that inactivate the virus.

And this science can easily be applied into consumer, commercial and healthcare products, such as wipes, sprays, clothing, paint, personal protective equipment (PPE) for healthcare workers, and really almost any surface.

When incorporated into N95 masks, this material works well against the virus, Chi said. In addition to trapping the virus in a mask, this would make for better PPE and prolong its life.

Another unique advantage of this material is that unlike traditional disinfectant products, it is shown to not wash away with water and leaves no toxic residue as a result of the photodegradation process, Chi said.

Studying the potential of conjugated polymers and oligomers is nothing new for UNM researchers. In fact, Whitten and another of the authors on the study, Kirk Schanze, have been researching this area for a couple of decades.

Whitten and Chi said colleagues such as Schanze and others have collected a lot of data on polymer and oligomers, so when the pandemic hit in the spring, Whitten almost immediately started wondering how his area of study could help.

It was the right timing for all of us, Chi said.

Acquiring live coronavirus for research is not an easy feat, but thanks to the efforts a couple of team members, they were able to make it happen.

Linnea Ista is a member of the Biosafety Committee at UNM, and when the pandemic broke out and she was aware of the research that Whitten and Chi were conducting, she realized that she may have a connection on how to make the research happen, due to the fact that representatives from UNMs School of Medicine also sit on the committee.

Alison Kell, a faculty member in the School of Medicine, was the one who was able to acquire the live coronavirus for testing the effectiveness of these materials. She has been working with the SARS-CoV-2 virus in her research and was able to develop a protocol for analyzing samples the team prepared and exposing them to near UV or visible light.

Due to the sensitive nature of working with a virus such as coronavirus, it was crucial for Kell to be part of the team, since the work had to be done in cooperation with the UNM School of Medicine, which has BSL-3 lab facilities that are essential to doing study on the highly-contagious active virus, Ista said.

Whitten said he is hopeful that this discovery can quickly be put into use. He has a company called BioSafe Defenses that he said has hired a former Environmental Protection Agency official to help expedite the regulatory process in taking this discovery to market. He anticipates that once a material is approved, it will be only a matter of months before wipes, masks and other products are in the marketplace.

He said their research has found that adding the material into wipes would add only pennies per wipe. Additionally, the material could be added into masks and other personal protective equipment, changing the game for businesses such as gyms, airlines, cruise ships, groceries, health care facilities, schools and many more industries. In addition to coronavirus, these products could also help eliminate infections by the common cold, seasonal flu and other viral and bacterial infections that plague millions of people annually, causing loss of work and school time.

There is a limitless market for this, he said.

He added that the current pandemic is likely not the last such public health crisis we will see, so even after a vaccine for coronavirus is available, such products could be useful in combatting a wide variety of viruses and bacteria, including the flu or common cold.

Were not just thinking about COVID but other pathogens and any viral agents, Whitten said. We want to be ready for the next pandemic.

This research was funded by a grant from the National Institutes of Health.

Continued here:
Let there be light: UNM Newsroom - UNM Newsroom