StemCell Therapy

Gilead CEO Dan ODay has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosens Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. ODay also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. Theres $500 million in potential TIGIT milestones on US regulatory events likely capped by an approval if Gilead partners on it and the stars align on the data. And theres another $150 million opt-in payments for the rest of the Arcus pipeline.

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European regulators accept FibroGen's anemia drug for review; Passage Bio's lead gene therapy gets more love from the FDA - Endpoints News

NewsEnvironmentResearchers at Queen Mary University of London and the Royal Botanic Gardens, Kew, discovered genes that create chemicals harmful to insects

Monday, 25th May 2020, 11:22 pm

A set of genes has been identified that could protect ash trees from a deadly beetle, which attacks by burrowing into their stems.

Named the Emerald Ash Borer (EAB), the killer pest is expected to destroy hundreds of millions of trees worldwide in the years to come without any intervention.

Researchers at Queen Mary University of London and the Royal Botanic Gardens, Kew, discovered genes that create chemicals likely to be harmful to the insects.

They sequenced the genomes of 22 types of ash tree and used this information to analyse how the different species are related to each other.

Help trees fight deadly beetles

Meanwhile, the US Department of Agriculture Forest Service, in Ohio, tested the resistance of more than 20 ash species to EAB by hatching eggs on the bark of trees, and following the fate of the beetle larvae.

Resistant ash trees killed the larvae when they burrowed into their stems, but susceptible ones did not.

The scientists discovered 53 candidate resistance genes, several of which are involved in making chemicals that are likely to be harmful to insects.

The findings suggest that breeding or gene editing could be used to place these resistance genes into ash species currently affected by EAB.

Dr Laura Kelly, an academic visitor at Queen Mary and lead author of the study, published in the journal Nature Ecology & Evolution, said: Knowledge of genes involved in resistance will...help efforts to identify trees that are able to survive the ongoing threat from EAB, and in turn, could facilitate restoration of ash woodlands in areas which have already been invaded."

Originally posted here:
Gene therapy could help millions of ash trees fight deadly beetle Emerald Ash Borer - inews

PENNINGTON, N.J. and SAN DIEGO, May 27, 2020 /PRNewswire/ --OncoSec Medical Inc.(NASDAQ:ONCS) (the "Company" or "OncoSec"), a company developing late-stage intratumoral cancer immunotherapies, today announced Notices of Allowance for three new patent applications covering its interleukin-12 (IL-12) based immunotherapy platform, including its lead product candidate TAVO and next-generation product candidate TAVOPLUS, and electroporation gene delivery system.

"OncoSec continues to prioritize the expansion of its intellectual property portfolio across multiple facets of its technology," said Keir Loiacono, Vice President, Corporate Development at OncoSec Medical Incorporated. "These three new allowances demonstrate OncoSec's strong patent position in three critical areas: device development, new treatment alternatives and novel adjuvant therapies with previously undescribed combinations. As we look to the future, we believe this will not only help to secure our competitive position in the intratumoral oncology space, but potentially create new opportunities for licensing revenue."

About OncoSec Medical IncorporatedOncoSec Medical Incorporated (the "Company," "OncoSec," "we" or "our") is a late-stage biotechnology company focused on developing cytokine-based intratumoral immunotherapies to stimulate the body's immune system to target and attack cancer.OncoSec's lead immunotherapy investigational product candidate TAVO (tavokinogenetelseplasmid) enables the intratumoral delivery of DNA-based interleukin-12 (IL-12), a naturally occurring protein with immune-stimulating functions.The technology, which employs electroporation, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body. OncoSec has built a deep and diverse clinical pipeline utilizing TAVO as a potential treatment for multiple cancer indications either as a monotherapy or in combination with leading checkpoint inhibitors; with the latter potentially enabling OncoSec to address a great unmet medical need in oncology: anti-PD-1 non-responders.Results from recently completed clinical studies of TAVO have demonstrated a local immune response, and subsequently, a systemic effect as either a monotherapy or combination treatment approach along with an acceptable safety profile, warranting further development. In addition to TAVO, OncoSec is identifying and developing new DNA-encoded therapeutic candidates and tumor indications for use with its new Visceral Lesion Applicator (VLA), to target deep visceral lesions, such as liver, lung or pancreatic lesions. For more information, please visitwww.oncosec.com.

TAVO is a trademark of OncoSec Medical Incorporated.

Risk Factors and Forward-Looking StatementsThis release, as well as other information provided from time to time by the Company or its employees, may contain forward-looking statements that involve a number of risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements. Forward-looking statements provide the Company's current beliefs, expectations and intentions regarding future events and involve risks, uncertainties (some of which are beyond the Company's control) and assumptions. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. You can identify forward-looking statements by the fact that they do not relate strictly to historical or current facts. These statements may include words such as "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "should," "will" and "would" and similar expressions (including the negative of these terms). Although we believe that expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements. The Company intends these forward-looking statements to speak only at the time they are published on or as otherwise specified, and does not undertake to update or revise these statements as more information becomes available, except as required under federal securities laws and the rules and regulations of the Securities Exchange Commission ("SEC"). In particular, you should be aware that the success and timing of our clinical trials, including safety and efficacy of our product candidates, patient accrual, unexpected or expected safety events, the impact of COVID-19 on the supply of our candidates or the initiation or completion of clinical trials and the usability of data generated from our trials may differ and may not meet our estimated timelines. Please refer to the risk factors and other cautionary statements provided in the Company's Annual Report on Form 10-K for the fiscal year ended July 31, 2019 and subsequent periodic and current reports filed with the SEC (each of which can be found at the SEC's websitewww.sec.gov), as well as other factors described from time to time in the Company's filings with the SEC.

Company Contact:Gem HopkinsHead of Corporate Communications858-210-7334ghopkins@Oncosec.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/oncosec-strengthens-ip-portfolio-with-three-new-patents-covering-tavo-and-its-electroporation-gene-delivery-system-301066037.html

SOURCE OncoSec Medical Incorporated

Read more from the original source:
OncoSec Strengthens IP Portfolio with Three New Patents Covering TAVO and Its Electroporation Gene Delivery System - BioSpace

R.A. Session II, president, CEO, and founder of Taysha Gene Therapies [Background image: Olena Yepifanova via iStock]

Dallas-based biotech Taysha Gene Therapies has emerged out of stealth with a $30 million seed round and a new strategic partnership with UT Southwestern. The company aims to end severe and life-threatening diseases of the central nervous system caused by variation in a single gene, also known as monogenic CNS diseases.

Hitting the ground running, the company launches with a pipeline of 15 adeno-associated virus gene therapy programs, along with options to four others.

Taysha plans human testing this year and expects to file four Initial New Drug applications in 2021. In addition, the biotech is developing an improved treatment delivery platform that uses machine learning, DNA shuffling, and something it calls directed evolution.

The gene therapy startup closed its round of seed financing in early March just as the Dow dropped a couple of thousand points, according to a report in EndpointsNews.

If you had to ask me, was I worried? Absolutely. I think I wouldnt be human if I wasnt, RA Session II, president, CEO, and founder of Taysha, told the healthcare publication.

Session had a lot of certainty to balance out all the uncertainty of launching a new biotech during a pandemic, noted Endpoints Amber Tong.

The experience of a proven management team, including some who previously led the development and commercialization of the first FDA-approved gene therapy for CNS disease, Zolgensma, is part of the equation.

The startup intends to combine the speed, scale, and expertise of its partners UT Southwestern Gene Therapy Program with the experience of the Taysha team.

Session says the launch reunites former investors and executives from AveXis. Tayshas funding round was co-led by PBM Capital, the first institutional investor in AveXis, and Nolan Capital, the investment fund of former AveXis CEO Sean Nolan.

Tayshas Board of Directors played a key role in the formation of the company, it said. The board is comprised of Sean Nolan, Paul Manning of PBM Capital, Claire Aldridge, Ph.D., of UT Southwestern, and RA Session II, President, CEO, and Founder of Taysha.

Nolan, who serves as Chairman of the Board, sees promise in Tayshas approach for patients suffering from many devastating CNS diseases.

This is an exciting time for gene therapy, he said.

The strategic partnership with UT Southwestern could help Taysha to rapidly and efficiently translate novel AAV gene therapies from bench to bedside, the company said.

UTSW is home to some of the brightest minds in gene therapy, Sessions says. By joining forces with UT Southwestern, Taysha can advance its potential cures with both speed and scale.

The collaboration is ultimately creating an engine for new cures, said Claire Aldridge, Ph.D., a Taysha board member and associate vice president of commercialization and business development at UT Southwestern Medical Center.

Together, Taysha and UTSW will merge cutting-edge translational research, hands-on clinical care, and expertiseboth regulatory and commercial, she said.

Aldridge has already witnessed how quickly and efficiently the partners can leverage their collective expertise and resources. That means getting new gene therapies to the patients who so desperately need them, she said.

The UT Southwestern Gene Therapy Program, led by Steven Gray, Ph.D., Director of the Viral Vector Core and Assistant Professor in the Department of Pediatrics, and Berge Minassian, M.D., Division Chief of Child Neurology, has the capacity to support Tayshas wide range of preclinical and clinical development programs, the company said.

Under the partnership, UT Southwestern, which has developed a state-of-the-art viral vector manufacturing facility, will run discovery and preclinical research, as well as lead studies, provide manufacturing, and execute natural history studies.

For its part, Taysha will lead all clinical development, regulatory strategy, commercial manufacturing, and commercialization activities, according to the company.

A joint steering committee of key leadership members from Taysha and UT Southwestern will govern the collaboration.

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Friday morning's Startup Week activities led off with a women's networking breakfast and a Creative Mornings event. Then, sessions have covered equity in real estate, corporate innovation, fundraising, and the future of philanthropy.

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Dallas Gene Therapy Startup Launches with $30M Seed Round and a UT Southwestern Partnership - dallasinnovates.com

The team that developed Zolgensma is back for round two. A group of former AveXis executives and investors unveiled a new gene therapy company, and theyre wasting no time. With 15 programs, $30 million in seed funding and an unrivaled partnership with UT Southwestern Medical Center, Taysha Gene Therapies plans to be in the clinic by the end of the year.

Its working on adeno-associated vector (AAV) gene therapies for monogenic diseasesthat is, diseases caused by a defect in a single geneof the central nervous system. Tayshas lead program targets GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord, but the companys portfolio includes conditions that are more prevalent.

The company plans to start clinical trials for three more programs by the end of 2021: treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome, Taysha CEO and co-founder R. A. Session II told FierceBiotech.

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RELATED: Passage Bio pulls off $216M IPO, blowing past original goal

And thats not allTaysha has the option to pick up four more prospects from UT Southwestern.

The reason why we can tackle 15 programs with the option to four additional programs is were able to focus on what we do best and were able to allow our collaborators to focus on what they do best, Session said.

Under their partnership, UT Southwestern is working on discovery and preclinical work all the way through IND-enabling studies. It has its own GMP viral manufacturing site to support that work, as well as clinical development, which Taysha will pick up. The company will also take care of regulatory strategy, commercial manufacturing and commercialization. Its a skill set that Tayshas management team honed at AveXis as itdeveloped the gene therapy that eventually became Novartis spinal muscular atrophy treatment Zolgensma.

We essentially flew the plane and built it at the same time when we were developing AveXis We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and were marrying that with a best-in-class academic research institution, Session said.

RELATED: FDA lets Novartis off the hook in Zolgensma data manipulation

Dividing the labor creates this engine for innovation that allows the partners to advance many programs in parallel, Session added. With about 50 people in its gene therapy unit, UT Southwestern can develop a capsidthe protein shell of a virusfor treatment delivery or get a candidate into animal models much more quickly than a biotech going it alone, he said. The same goes for late-stage development and commercialization on Tayshas side.

For its initial programs, Taysha is focusing on AAV gene therapies because the team knows they work.

AAV9 is the best way we have to treat monogenic CNS disease. If its not broke, dont fix it, Session said. We know how to effectively dose AAV9; we know its safe, effective and efficient. And we know its scalable. These are problems we had to solve at our previous company.

But, moving forward, Taysha and UT Southwestern are working on new technologies, including an AAV delivery platform that would allow for the redosing of gene therapies as well as an AAV capsid platform aimed at improving target delivery.

The rest is here:
Taysha Gene Therapies hits the ground running with $30M, 15 programs - FierceBiotech

Biotech executive, Julia Berretta, Ph.D., is named Chief Executive Officer

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

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

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

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

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

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

Dr. Berretta was part of the Executive Committee of Cellectis S.A., a Nasdaq-listed clinical stage gene editing company developing CAR-T cell therapies for cancer, where she led business development as well as strategic planning. She is also an independent Board member of Treefrog Therapeutics, an innovative stem cell company.

About Genespire

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

About Sofinnova Partners

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

For more information, please visit: http://www.sofinnovapartners.com

About Fondazione Telethon

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

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About Ospedale San Raffaele

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

About the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200429005417/en/

Contacts

Julia BerrettaCEO, Genespire S.r.linfo@genespire.com +39 02 83991300

Bommy LeeHead of Communications, Sofinnova Partnersblee@sofinnovapartners.com +33 (0) 6 47 71 38 11

North AmericaRooneyPartners LLCKate Barrettekbarrette@rooneyco.com +1 212 223 0561

FranceStrategiesImage (S&I)Anne Reinanne.rein@strategiesimage.com +33 6 03 35 92 05

Originally posted here:
Genespire Secures 16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies - Yahoo Finance

The Paris-based BrainVectis, a biotech developing gene therapies for Huntingtons disease and Alzheimers disease, has been acquired by the US clinical-stage gene therapy company AskBio.

AskBios gene therapy experience and manufacturing capacity will help to accelerate BrainVectis lead candidate for Huntingtons into phase I testing. In return, AskBio gets to expand its list of target indications.

AskBio recognized the scientific merit of the work at BrainVectis as an opportunity to strengthen our central nervous system clinical pipeline, Robin Fastenau, VP of Communications for AskBio, told me. No financial details about the acquisition were disclosed.

BrainVectis lead candidate focuses on increasing the expression of a protein called CYP46A1. This enzyme is key for turning excess cholesterol into a derivative that can be cleared from the brain into the blood. It is also reduced in Huntingtons and Alzheimers patients, allowing toxic levels of cholesterol to build up in the brain. By increasing the levels of this enzyme, BrainVectis aims to restore normal cholesterol metabolism and improve the clinical outcome.

So far, BrainVectis lead gene therapy candidate has shown proof-of-concept in animal models of Huntingtons. It also received orphan drug designation from the European Commission last year.

According to Nathalie Cartier-Lacave, CEO and Founder of BrainVectis, AskBio offers a strong cell-line manufacturing process. In particular, it can manufacture a range of viral vectors, including BrainVectis vector of choice: the adeno-associated virus.

This powerful adeno-associated virus technology and Askbios expertise in clinical applications will allow us to rapidly go to clinical application in patients, Cartier-Lacave told me.

There are currently no approved treatments able to slow down the progress of Huntingtons and Alzheimers. Combined with aging populations in developed countries, these debilitating diseases are creating a healthcare challenge. Many companies are trying and sometimes failing to develop drugs able to stop the progression of Alzheimers, for example, as such a drug could make a huge impact on society.

According to Cartier-Lacave, Huntingtons disease is the first target for the company going forward. The disease is caused by a mutation in a gene called HTT that is important for the function of nerve cells. Its a very severe disease for which we think the treatment may not only decrease the toxic mutated protein, but also preserve neurons from death, she added.

Gene therapies are becoming ever more popular in the biotech industry for their potential to tackle previously incurable conditions. BrainVectis is one of several companies aiming gene therapies at the brain; another is the French company Lysogene. However, there are major challenges with developing gene therapies for the brain, for example, getting the therapy past the blood-brain barrier and into the brain tissue.

AskBio might be up to the challenge, as it has taken several gene therapies to the clinic. Its proprietary treatment for the neuromuscular indication Pompe disease is currently in phase I/II, and it has licensed its technology to several big pharmaceutical companies. Those currently in clinical development include treatments for Duchenne muscular dystrophy (Pfizer), hemophilia (Takeda), and spinal muscular atrophy (AveXis), which was approved by the FDA last year, and is awaiting marketing approval by the EMA.

Image from Shutterstock

More:
AskBio Takes Over French Gene Therapy Company to Treat Alzheimers and... - Labiotech.eu

Hours after Gilead announced that an NIH trial testing their antiviral drug remdesivir in Covid-19 patients had succeeded, NIAID director Anthony Fauci sat on a couch in the Oval Office and gave the world the top-line readout.

The drug induced a 31% improvement on the primary endpoint of time to recovery: 11 days in the drug arm compared to 15 days in the placebo arm, he said, adding that patients taking the drug appeared less likely to die, with an 8% mortality rate in the drug arm compared to 11% in patients given the placebo.

The mortality data were not yet statistically significant, he cautioned but were trending in the right direction. Fauci, surrounded by President Trump, Vice President Mike Pence and several other advisors, said the news was a very optimistic sign in the hunt for treatments to fight the virus.

Although a 31% improvement doesnt seem like a knockout 100%, it is a very important proof of concept, he said. Because what it has proven, is that a drug has blocked this virus.

Fauci said more details would come and that the study would be submitted to a peer-reviewed journal. Trump, who deferred to Fauci in giving the readout, echoed Faucis commentary.

Its a beginning, that means you build on it, Trump said. But its a very positive event.

Shortly after the briefing, the New York Times reported that the FDA was preparing to issue an emergency use authorization for the drugs use in Covid-19. In an email to Endpoints News, the FDA did not confirm or deny the Times report, but a spokesperson said the agency has been engaged in sustained and ongoing discussions with Gilead Sciences regarding making remdesivir available to patients as quickly as possible, as appropriate.

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In the heart of Texas, the AveXis crew gathers for another go at gene therapy with $30M and a powerhouse academic pact to start - Endpoints News

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

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

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

Image: iStock/PashaIgnatov

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

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

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

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

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

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

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

Global CNS Gene Therapy Market Analysis

Persistence Market Research, in a recently published market study, offers valuable insights related to the overall dynamics of the CNS Gene Therapy market in the current scenario. Further, the report assesses the future prospects of the CNS Gene Therapy by analyzing the various market elements including the current trends, opportunities, restraints, and market drivers. The COVID-19 analysis section within the report offers timely insights regarding the impact of the global pandemic on the market. The presented study also offers data regarding the business and supply chain continuity strategies that are likely to assist stakeholders in the long-run.

As per the report, the CNS Gene Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and exceed a value of ~US$ XX by the end of 2029. Some of the leading factors that are expected to drive the growth of the market include, focus towards research and development, innovations, and evolving consumer preferences among others.

Request Sample Report @ https://www.persistencemarketresearch.co/samples/27514

Regional Outlook

The report scrutinizes the prospects of the CNS Gene Therapy market in different geographical regions. The scope of innovation, consumer behavior, and regulatory framework of each region is thoroughly analyzed in the presented study.

Distribution-Supply Channel Assessment

The report provides a thorough analysis of the different distribution channels adopted by market players in the global CNS Gene Therapy market along with the market attractiveness analysis of each distribution channel. The impact of the COVID-19 pandemic on the different distribution channels is enclosed in the report.

Product Adoption Analysis

key players and product offerings

Request Report Methodology @ https://www.persistencemarketresearch.co/methodology/27514

The report aims to address the following pressing questions related to the CNS Gene Therapy market:

Key Takeaways from the CNS Gene Therapy Market Report

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/27514

Why Companies Trust PMR?

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CNS Gene Therapy Market to Slip Due to Delays in Production Amidst Coronavirus Outbreak Cole Reports - Cole of Duty

BOSTON and LONDON, May 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will host a conference call and live webcast on Thursday, May 7, 2020, at 8:00 a.m. ET to report its first quarter 2020 financial results and other business updates.

A live webcast will be available under "News & Events" in the Investors & Media section of the company's website at orchard-tx.com. The conference call can be accessed by dialing (866) 987-6504 (U.S. domestic) or +1 (602) 563-8620 (international) and referring to conference ID 8348144. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Contacts

Investors

Renee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

Excerpt from:
Orchard Therapeutics to Webcast Conference Call of First Quarter 2020 Financial Results - GlobeNewswire

Documenting the Industry Development of Gene Therapy Market concentrating on the industry that holds a massive market share 2020 both concerning volume and value With top countries data, Manufacturers, Suppliers, In-depth research on market dynamics, export research report and forecast to 2029

As per the report, the Gene Therapy Market is anticipated to gain substantial returns while registering a profitable annual growth rate during the predicted time period.The global gene therapy market research report takes a chapter-wise approach in explaining the dynamics and trends in the gene therapy industry.The report also provides the industry growth with CAGR in the forecast to 2029.

A deep analysis of microeconomic and macroeconomic factors affecting the growth of the market are also discussed in this report. The report includes information related to On-going demand and supply forecast. It gives a wide stage offering numerous open doors for different businesses, firms, associations, and start-ups and also contains authenticate estimations to grow universally by contending among themselves and giving better and agreeable administrations to the clients. In-depth future innovations of gene therapy Market with SWOT analysis on the basis Of type, application, region to understand the Strength, Weaknesses, Opportunities, and threats in front of the businesses.

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***[Note: Our Complimentary Sample Report Accommodate a Brief Introduction To The Synopsis, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology are also Included]

An Evaluation of the Gene Therapy Market:

The report is a detailed competitive outlook including the Gene Therapy Market updates, future growth, business prospects, forthcoming developments and future investments by forecast to 2029. The region-wise analysis of gene therapy market is done in the report that covers revenue, volume, size, value, and such valuable data. The report mentions a brief overview of the manufacturer base of this industry, which is comprised of companies such as- Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc, Advantagene.

Segmentation Overview:

Product Type Segmentation :

Ex vivo, In Vivo

Application Segmentation :

Cancer, Monogenic, Infectious disease, Cardiovascular disease

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Key Highlights of the Gene Therapy Market:

The fundamental details related to Gene Therapy industry like the product definition, product segmentation, price, a variety of statements, demand and supply statistics are covered in this article.

The comprehensive study of gene therapy market based on development opportunities, growth restraining factors and the probability of investment will anticipate the market growth.

The study of emerging Gene Therapy market segments and the existing market segments will help the readers in preparing the marketing strategies.

The study presents major market drivers that will augment the gene therapy market commercialization landscape.

The study performs a complete analysis of these propellers that will impact the profit matrix of this industry positively.

The study exhibits information about the pivotal challenges restraining market expansion

The market review for the global market is done in context to region, share, and size.

The important tactics of top players in the market.

Other points comprised in the Gene Therapy report are driving factors, limiting factors, new upcoming opportunities, encountered challenges, technological advancements, flourishing segments, and major trends of the market.

Check Table of Contents of This Report @https://market.us/report/gene-therapy-market//#toc

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Gene Therapy Market | on(impact of COVID-19) 2020-2029 Analysis on Growth, Future Demand 2020 - Jewish Life News

You can't lump all clinical-stage biotechs together. TakeCara Therapeutics (NASDAQ:CARA) andSangamo Therapeutics (NASDAQ:SGMO), for example. The two biotechs are about as different as night and day. One thing that both Cara and Sangamo have in common, though, are promising pipeline candidates.

Investors have placed a greater value on Sangamo's pipeline based on the company's higher market cap. But which of these biotech stocks is the better pick for long-term investors? Here's how Cara and Sangamo stack up against each other.

Image source: Getty Images.

All of Cara Therapeutics' hopes ride on one drug -- Korsuva. But that one drug has multiple formulations and multiple opportunities.

The most promising of these opportunities is for Korsuva injection in treating chronic kidney disease-associated pruritis (CKD-aP) in patients on hemodialysis. The itching (pruritis) experienced by many patients with CKD is very problematic. So far, there haven't been any approved treatments for CKD-aP.

That could soon change. Last week, Cara and its partner, Vifor Fresenius Medical Care Renal Pharma, announced positive results from a global late-stage study of Korsuva injection in treating CKD-aP in patients on hemodialysis. These results reinforced positive results reported last year from a U.S. study. The two companies plan to file for U.S. regulatory approval for Korsuva injection in the second half of 2020, followed closely by filing for European approval of the drug.

Cara anticipates completing an interim statistical analysis within the next couple of months for a phase 2 study evaluating an oral version of Korsuva in treating pruritis in patients with atopic dermatitis. The biotech is also conducting another phase 2 study of oral Korsuva in treatingpatients with pruritus and hepatic impairment due to primary biliary cholangitis (PBC).

Oral Korsuva should advance to a late-stage study in treating CKD-aP in patients who aren't on hemodialysis later this year. Cara reported results from a phase 2 study in December with the drug meeting its primary endpoint but failing to meet two secondary endpoints.

Korsuva injection could achieve peak annual sales topping $500 million. An oral version of the drug could boost that number considerably. With Cara Therapeutics' market cap currently below $750 million, the stock should have plenty of room to run if Korsuva wins regulatory approvals and reaches its commercial potential.

Unlike Cara Therapeutics, Sangamo isn't hanging its hat on only one candidate. The biotech's pipeline includes five different therapies in clinical testing. However, none of these programs are yet in late-stage testing.

Sangamo specializes in gene editing using a technique known as zinc finger nuclease (ZFN) technology and in developing gene therapies. The company's approach has captured attention from several big drugmakers.

Pfizeris partnering with Sangamo on developing SB-525, a gene therapy targeting hemophilia A. Sanofiteamed up with Sangamo on two gene-edited cell therapies in clinical studies. ST-400 targets treatment of rare blood disease beta-thalassemia, while BIVV-003 targets treatment of sickle cell disease.

Sangamo also has two wholly owned programs in phase 1/2 studies. Gene therapy ST-920 targets Fabry disease. ZFN gene-editing therapy SB-913 targets rare genetic diseasemucopolysaccharidosis type II (MPS II).

In addition to its clinical programs, Sangamo has 11 therapies in preclinical testing. Gilead Sciencesis collaborating with Sangamo on two of these preclinical programs. Pfizer and Japanese drugmaker Takeda have teamed up with the biotech on one preclinical program each.

In February, Sangamo announced a dealwith Biogen to develop gene therapies targeting neurological diseases. Three of Sangamo's preclinical programs are included in this agreement.

Sangamo's market cap stands at a little under $1 billion. While the company has a long way to go before any of its candidates could potentially win approval, it also has multiple shots on goal. Successes for only one or two of its experimental therapies would enable this small biotech to become much larger.

I think that both of these stocks could be big winners over the long run. However, my view is that Cara Therapeutics offers a better risk-reward proposition.

Although it's not a slam dunk that Cara wins FDA and EU approvals for Korvuva injection, I think the odds appear to be pretty good. Assuming those approvals are secured, Cara should be set to begin pulling in some solid revenue by late 2021.

Having said that, it wouldn't surprise me if Sangamo achieves greater success by the end of this decade. I think that investors should keep the biotech on their radar screens.

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Better Buy: Cara Therapeutics vs. Sangamo Therapeutics - Motley Fool

CAMBRIDGE, Mass., May 1, 2020 /PRNewswire/ --Obsidian Therapeutics, a biotechnology company pioneering controllable cell and gene therapies, today announced it will share the architecture and components of its Safe Workplace Function Tool (SWFT) Productivity Solution. The SWFT Solution was designed in response to the COVID19 pandemic to support a safe work environment while maintaining productivity in the lab.

The SWFT Solution is a web-based application built and integrated into Microsoft 365 that allows scientists to view and schedule lab-based activities including by date, time and lab location. SWFT promotes collaboration and coordination between teams by predicting scheduling and occupancy conflicts, which allows team members to adjust their schedules to promote social distancing in the lab and office.

"The SWFT Solution has enabled Obsidian to continue to generate critical data across our cytoDRiVE development programs, while maximizing the safety of our staff," stated Catherine Stehman-Breen, M.D., Chief Research and Development Officer at Obsidian. "We have already shared this technology with large pharmaceutical and small biotechnology companies who are interested in our SWFT Solution to get their labs back up and running. We believe that it is more important than ever before to leverage one another's expertise in order to overcome challenges as we work tirelessly to deliver meaningful outcomes to patients in need."

To create Obsidian's bespoke application, (1) a capacity analysis was conducted, taking into consideration lab space and occupancy levels, and (2) in collaboration with scientists, workflow recommendations were implemented to determine an appropriate shift schedule. These steps facilitated the development of a solution that enabled scientists to plan their experiments with specific lab locations and shifts, as well as be alerted to and prevent any over-capacity issues. Obsidian implemented four three-hour lab blocks, with thirty-minutes of cleaning time between blocks, over a seven-day work week, to ensure that coronavirus-related safety recommendations were met.

Celeste Richardson, Ph.D., Vice President of Cell Therapy of Obsidian, stated, "We have a commitment to our employees to keep the health and safety of our employees top of mind while they work to bring innovative therapies to patients. The development of the SWFT Solution perfectly demonstrates Obsidian's culture of teamwork, determination and innovation."

Obsidian's IT Partner, TRNDigital, is continuing to iterate the tool to ensure it continues to meet scientists' needs and can be made available to others. The solution has been expanded to include density planning in the Obsidian offices. In addition, the SWFT Solution is scalable and flexible to other laboratory setups.

The SWFT solution was developed in-house by Henry Rogalin, Data Scientist, under the leadership of Nic Betts, Head of IT and Facilities, and in collaboration with a safety and facilities capacity team led by Jillian Giguere, Senior Manager of Laboratory Operations, Facilities, and EHS. For more information on SWFT, submit this inquiry form. Informational sessions and training will be hosted as requested.

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact:Maggie BellerRusso Partners, LLCMaggie.beller@russopartnersllc.com646-942-5631

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Obsidian Therapeutics Develops and Shares Safe Workplace Productivity Solution in Response to COVID19 - P&T Community

The research report on Gene Therapy Market provides comprehensive analysis on market status and development pattern, including types, applications, rising technology and region. Gene Therapy Market report covers the present and past market scenarios, market development patterns, and is likely to proceed with a continuing development over the forecast period. The report covers all information on the global and regional markets including historic and future trends for market demand, size, trading, supply, competitors, and prices as well as global predominant vendors information.

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This market research report on the Gene Therapy Market is an all-inclusive study of the business sectors up-to-date outlines, industry enhancement drivers, and manacles. It provides market projections for the coming years. It contains an analysis of late augmentations in innovation, Porters five force model analysis and progressive profiles of hand-picked industry competitors. The report additionally formulates a survey of minor and full-scale factors charging for the new applicants in the market and the ones as of now in the market along with a systematic value chain exploration.

An outline of the manufacturers active within the Gene Therapy Market, consisting of

Sibiono GeneTech,Advantagene,Spark Therapeutics,Shanghai Sunway Biotech Co. LtdBluebird Bio,UniQure NVAvalanche Bio,Celladon,Sangamo,Dimension Therapeutics

The Gene Therapy Market Segmentation by Type:

Viral vectorNon-viral vector

The Gene Therapy Market Segmentation by Application:

Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Market Segment by Regions, regional analysis covers

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The competitive landscape of the Gene Therapy Market is discussed in the report, including the market share and new orders market share by company. The report profiles some of the leading players in the global market for the purpose of an in-depth study of the challenges faced by the industry as well as the growth opportunities in the market. The report also discusses the strategies implemented by the key companies to maintain their hold on the industry. The business overview and financial overview of each of the companies have been analyzed.

This report provide wide-ranging analysis of the impact of these advancements on the markets future growth, wide-ranging analysis of these extensions on the markets future growth. The research report studies the market in a detailed manner by explaining the key facets of the market that are foreseeable to have a countable stimulus on its developing extrapolations over the forecast period.

Key questions answered in this research report:

Table of Contents:

Global Gene Therapy Market Research Report

Chapter 1 Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

.CONTINUED FOR TOC

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Global Gene Therapy Market Size, Share, Growth, Revenue, Global Industry Analysis and Future Demand And Forecast To 2020-2026 Cole Reports - Cole of...

What are exosomes?

Exosomes are a class of cell-derived extracellular vesicles of endosomal origin, and are typically 30-150 nm in diameter the smallest type of extracellular vesicle.1 Enveloped by a lipid bilayer, exosomes are released into the extracellular environment containing a complex cargo of contents derived from the original cell, including proteins, lipids, mRNA, miRNA and DNA.2 Exosomes are defined by how they are formed through the fusion and exocytosis of multivesicular bodies into the extracellular space.

Multivesicular bodies* are unique organelles in the endocytic pathway that function as intermediates between early and late endosomes.3 The main function of multivesicular bodies is to separate components that will be recycled elsewhere from those that will be degraded by lysosomes.4 The vesicles that accumulate within multivesicular bodies are categorized as intraluminal vesicles while inside the cytoplasm and exosomes when released from the cell.

*Confusingly, there is inconsistency in the literature; while some sources differentiate multivesicular bodies from late endosomes, others use the two interchangeably.

Exosomes are of general interest for their role in cell biology, and for their potential therapeutic and diagnostic applications. It was originally thought that exosomes were simply cellular waste products, however their function is now known to extend beyond waste removal. Exosomes represent a novel mode of cell communication and contribute to a spectrum of biological processes in health and disease.2One of the main mechanisms by which exosomes are thought to exert their effects is via the transfer of exosome-associated RNA to recipient cells, where they influence protein machinery. There is growing evidence to support this, such as the identification of intact and functional exosomal RNA in recipient cells and certain RNA-binding proteins have been identified as likely players in the transfer of RNA to target cells.5,6 MicroRNAs and long noncoding RNAs are shuttled by exosomes and alter gene expression while proteins (e.g. heat shock proteins, cytoskeletal proteins, adhesion molecules, membrane transporter and fusion proteins) can directly affect target cells.7,8Exosomes have been described as messengers of both health and disease. While they are essential for normal physiological conditions, they also act to potentiate cellular stress and damage under disease states.2

Multivesicular bodies are a specialized subset of endosomes that contain membrane-bound intraluminal vesicles. Intraluminal vesicles are essentially the precursors of exosomes, and form by budding into the lumen of the multivesicular body. Most intraluminal vesicles fuse with lysosomes for subsequent degradation, while others are released into the extracellular space.9,10 The intraluminal vesicles that are secreted into the extracellular space become exosomes. This release occurs when the multivesicular body fuses with the plasma membrane.

The formation and degradation of exosomes.

This is an active area of research and it is not yet known how exosome release is regulated. However, recent advances in imaging protocols may allow exosome release events to be visualized at high spatiotemporal resolution.11

Exosomes have been implicated in a diverse range of conditions including neurodegenerative diseases, cancer, liver disease and heart failure. Like other microvesicles, the function of exosomes likely depends on the cargo they carry, which is dependent on the cell type in which they were produced.12 Researchers have studied exosomes in disease through a range of approaches, including:

In cancer, exosomes have multiple roles in metastatic spread, drug resistance and angiogenesis. Specifically, exosomes can alter the extracellular matrix to create space for migrating tumor cells.13,14 Several studies also indicate that exosomes can increase the migration, invasion and secretion of cancer cells by influencing genes involved with tumor suppression and extracellular matrix degradation.15,16Through general cell crosstalk, exosomal miRNA and lncRNA affect the progression of lung diseases including chronic obstructive pulmonary disease (COPD), asthma, tuberculosis and interstitial lung diseases. Stressors such as oxidant exposure can influence the secretion and cargo of exosomes, which in turn affect inflammatory reactions.17 Altered exosomal profiles in diseased states also imply a role for exosomes in many other conditions such as in neurodegenerative diseases and mental disorders.18,19Cells exposed to bacteria release exosomes which act like decoys to toxins, suggesting a protective effect during infection.20 In neuronal circuit development, and in many other systems, exosomal signaling is likely to be a sum of overlapping and sometimes opposing signaling networks.21

Exosomes can function as potential biomarkers, as their contents are molecular signatures of their originating cells. Due to the lipid bilayer, exosomal contents are relatively stable and protected against external proteases and other enzymes, making them attractive diagnostic tools. There are increasing reports that profiles of exosomal miRNA and lncRNA differ in patients with certain pathologies, compared with those of healthy people.17 Consequently, exosome-based diagnostic tests are being pursued for the early detection of cancer, diabetes and other diseases.22,23Many exosomal proteins, nucleic acids and lipids are being explored as potential clinically relevant biomarkers.24 Phosphorylation proteins are promising biomarkers that can be separated from exosomal samples even after five years in the freezer25, while exosomal microRNA also appears to be highly stable.26 Exosomes are also highly accessible as they are present in a wide array of biofluids (including blood, urine, saliva, tears, ascites, semen, colostrum, breast milk, amniotic fluid and cerebrospinal fluid), creating many opportunities for liquid biopsies.

Exosomes are being pursued for use in an array of potential therapeutic applications. While externally modified vesicles suffer from toxicity and rapid clearance, membranes of naturally occurring vesicles are better tolerated, offering low immunogenicity and a high resilience in extracellular fluid.27 These naturally-equipped nanovesicles could be therapeutically targeted or engineered as drug delivery systems.

Exosomes bear surface molecules that allow them to be targeted to recipient cells, where they deliver their payload. This could be used to target them to diseased tissues or organs.27 Exosomes may cross the blood-brain barrier, at least under certain conditions28 and could be used to deliver an array of therapies including small molecules, RNA therapies, proteins, viral gene therapy and CRISPR gene-editing.

Different approaches to creating drug-loaded exosomes include27:

Exosomes hold huge potential as a way to complement chimeric antigen receptor T (CAR-T) cells in attacking cancer cells. CAR exosomes, which are released from CAR-T cells, carry CAR on their surface and express a high level of cytotoxic molecules and inhibit tumor growth.29 Cancer cell-derived exosomes carrying associated antigens have also been shown to recruit an antitumor immune response.30

The purification of exosomes is a key challenge in the development of translational tools. Exosomes must be differentiated from other distinct populations of extracellular vesicles, such as microvesicles (which shed from the plasma membrane, also referred to as ectosomes or shedding vesicles) and apoptotic bodies.31 Although ultracentrifugation is regarded as the gold standard for exosome isolation, it has many disadvantages and alternative methods for exosome isolation are currently being sought. Exosome isolation is an active area of research (see Table 1) and many research groups are seeking ways to overcome the disadvantages listed below, while navigating the relevant regulatory hurdles along the way.

Produces a low yield and low purity of the isolated exosomes as other types of extracellular vesicles have similar sedimentation properties.

Low efficiency as it is labor-intensive, time-consuming and requires a large amount of sample. specialized equipment. High centrifugal force can damage exosome integrity

More:
Exosomes: Definition, Function and Use in Therapy - Technology Networks

DetailsCategory: DNA RNA and CellsPublished on Monday, 27 April 2020 17:10Hits: 397

BOSTON, MA, USAandLONDON, UK I April 27, 2020 IOrchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced that the first patient has been dosed in an open-label, proof-of-concept investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). The study is designed to evaluate safety, tolerability and clinical efficacy and is intended to enroll up to five patients between three months and 24 months of age who will be followed for three years. The study also contains a number of key secondary outcome measures such as overall survival, cognition and behavior to help inform future clinical development of HSC gene therapy in this indication.

MPS-IIIA, also known as Sanfilippo syndrome type A, is a rare, inherited neurometabolic disorder caused by genetic mutations that leads to the buildup of sugar molecules called mucopolysaccharides in the body, resulting in progressive intellectual disability and loss of motor function. Children born with MPS-IIIA rarely live past adolescence or early adulthood, and no approved therapies currently exist to treat the disease.

I am very encouraged that we, together with our research and clinical collaborators in Manchester, could achieve this important milestone in our efforts to develop a gene therapy for MPS-IIIA despite the current, challenging global health circumstances, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. It is a testament to the dedication of our collective teams and underscores the truly dire, life-limiting nature of the disease for affected children and their families. This study adds to Orchards clinical pipeline of HSC gene therapies for the treatment of severe neurometabolic disorders and further demonstrates the potential of our platform approach.

About MPS-IIIAMucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease. People with MPS-IIIA are born with a mutation in theN-sulphoglucosamine sulphohydrolase (SGSH)gene, which, when healthy, helps the body break down sugar molecules called mucopolysaccharides. The buildup of mucopolysaccharides in the brain and other tissues leads to intellectual disability and loss of motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

SOURCE: Orchard Therapeutics

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Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome (MPS-IIIA) |...

Kolon Life Science said Monday it has started the administration of an experimental gene therapy KLS-2031 to the first patient to treat neuropathic pain in the phase-1/2a trial in the U.S.

KLS-2031 is a candidate for the first-in-class drug for the treatment of lumbosacral radiculopathy, a pain syndrome caused by compression or irritation of nerve roots in the lower back. The U.S. Food and Drug Administration has granted the investigational drug a fast track designation, which can speed up the development of the drug that may treat the life-threatening disease and address unmet medical needs.

The company said the trial would take place at two clinical institutions in the U.S. and end in 2023. Researchers plan to administer the drug once to 18 patients and observe the progress for 24 months. The company also said it would begin the first dose to the second patient within April.

Existing treatments for neuropathic pain are analgesics that cannot cure the disease fundamentally and do not have a lasting effect. KLS-2031 has combined three therapeutic genes -- GAD65, GDNF, and IL-10. The new gene therapy has a more prolonged analgesic effect than existing treatments for neuropathic pain, and it may treat the disease fundamentally, Kolon Life Science said.

With a successful clinical trial on this pipeline, we will strengthen our position as a cell gene therapy specializing company, CEO Park Moon-hee said. As the drug was granted the fast track designation, we will make it a fundamental treatment and a globally selling new drug,

same@docdocdoc.co.kr

< Korea Biomedical Review, All rights reserved.>

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Kolon begins testing gene therapy for neuropathic pain in US - Korea Biomedical Review

Add a new, 100 million California facility to the growing gene therapy infrastructure.

Merck KGaA announced they are opening a second gene therapy and viral vector factory in Carlsbad, California. The new center will be just one point in the global supply chain Big Pharma is rapidly erecting to keep manufacturing capacity for the new technology at pace with clinical development. Over the past year, Novartis, PTC Therapeutics, Pfizer and Vertex each announced or opened new facilities in Switzerland, North Carolina and New Jersey that will help build gene therapy.

In November,Reutersreported that 11 drugmakers had set aside $2 billion for the manufacturing effort. They were led by Novartis, the giant behind the second FDA-approved gene therapy in Zolgensma which planned to spend $500 million, and Pfizer, which has yet to get a gene therapy approved but will spend $600 million. Catalent and Thermo Fisher, meanwhile, each spent over a billion dollars acquiring companies involved in gene therapy or viral vector manufacturing.

Merck KGaA last shored up its gene therapy manufacturing in 2016 the year before the first gene therapy was approved in the US when they expanded their original Carlsbad facility from 44,000 to 65,000 feet. At 140,000 feet, the new facility will be more than double the size of its older neighbor. Gene therapies and the viral vectors used to deliver them will be cooked up in 1,000 liter bioreactors metal vats roughly the size of 260 gallons of milk. Merck has not said when it is scheduled for completion.

Although Merck KGaA lists no gene therapies in its most recent pipeline, the German drugmaker uses its facilities to help biotechs focused on the emerging modality to manufacture their products. The list of approved gene therapies can still be counted on one hand, but hundreds are now in clinical trials.

Manufacturing emerged as a pressing bottleneck almost as soon as Spark Therapeutics got Luxturna, a treatment for a form of inherited blindness, approved as the US first gene therapy. In an op-ed in STAT last year, Sparks head of technical operations, Diane Blumenthal, described the process of building in Philadelphia one of the first in-house gene therapy manufacturing facilities. With regulators increasingly open to approving the treatments quickly, she encouraged other companies to invest in manufacturing in advance of even knowing if the therapy works, particularly because gene therapy requires customization in ways few other modalities do.

And she warned the problem will only get more pressing.

There isnt a gene therapy manufacturing playbook yet to guide the development of gene therapies, Blumenthal wrote. Manufacturing a gene therapy is only half the battle. The other half is making enough of it, doing that as efficiently as possible, and getting it to the patients who need it. These challenges become even more urgent to tackle as the industry shifts to the next chapter in gene therapy development, from treatments made in small batches for small patient populations to bigger volumes for larger rare-disease populations and commercial scale.

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Merck KGaA adds to gene therapy manufacturing boom with 100M facility in California - Endpoints News

Patients in Cohort 4 of OPTIC will Receive a SingleADVM-022 Dose of 6 x 10 ^11 vg/eye

REDWOOD CITY, Calif., April 27, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc.(Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that the first patient was dosed in Cohort 4 of the ongoing OPTIC Phase 1 clinical trial for ADVM-022 for the treatment of wet age-related macular degeneration (AMD). Patients in Cohort 4 (n=9) are receiving a single intravitreal injection of gene therapy candidate ADVM-022 at a dose of 6 x 10 ^11 vg/eye (same as Cohort 1) and are receiving steroid eye drop prophylaxis for six weeks (same as Cohort 3).

David S. Boyer, M.D., senior partner, Retina-Vitreous Associates Medical Group and adjunct professor of ophthalmology with the University of Southern California/Keck School of Medicine in Los Angeles said, The current standard-of-care requires patients with wet AMD to receive frequent anti-VEGF injections to maintain their vision. A one-time treatment such as ADVM-022, which, similar to standard of care, is administered as an in-office intravitreal injection, could transform the treatment paradigm for wet AMD, particularly at this time when it is more important than ever to reduce the need for frequent injections and clinic visits. The data demonstrated in OPTIC have been positive and underscore the potential of ADVM-022 to be a long-lasting treatment option for patients.

We are pleased to have enrolled our first patient in Cohort 4, furthering our execution of the OPTIC trial, said Aaron Osborne, MBBS, chief medical officer of Adverum. Patients in this Cohort are receiving the higher dose of ADVM-022, which has demonstrated outstanding efficacy and durability in Cohort 1, as has been presented. We believe that utilizing the higher dose of ADVM-022 with the use of steroid eye drop prophylaxis, will further support that our gene therapy candidate, ADVM-022, has the potential to be an important treatment option for patients living with wet AMD. Additionally, Im grateful for the continued support of our clinical trial sites as we all manage through the global pandemic. Due to COVID-19, sites quickly implemented extra safety precautions for patients and their staff, allowing us to proceed with enrollment. Its a pleasure to partner with investigators in OPTIC who share our commitment to develop a novel single-administration approach for treating patients with wet AMD and we look forward to sharing data from all four cohorts later this year.

About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMDThe multi-center, open-label, Phase 1, dose-ranging trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. In Cohort 1, patients (n=6) received ADVM-022 at a higher dose of 6 x 10^11 vg/eye and in Cohort 2, patients (n=6) received ADVM-022 at a lower dose of 2 x 10^11 vg/eye. In Cohort 3, patients (n=9) also received a dose of 2 x 10^11 vg/eye and in Cohort 4, patients (n=9) are receiving a dose of 6 x 10^11 vg/eye. Patients in Cohorts 3 and 4 receive prophylactic steroid eye drops instead of oral steroids which were used in Cohorts 1 and 2. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as the need for anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.

Ten leading retinal centers acrossthe United States(U.S.) are participating in the OPTIC Phase 1 trial for ADVM-022. For more information on the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD, please visithttps://clinicaltrials.gov/ct2/show/NCT03748784.

About ADVM-022 Gene TherapyADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for wet AMD and diabetic retinopathy patients.

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for ADVM-022 for the treatment of this disease.

Adverum is currently evaluating ADVM-022 in the OPTIC study, a Phase 1 clinical trial in patients 50 years and older with wet AMD. Additionally, Adverum plans to initiate a Phase 1/2 clinical trial of ADVM-022 for the treatment of diabetic retinopathy in the second half of 2020.

About Adverum Biotechnologies, Inc.Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration. For more information, please visit http://www.adverum.com.

Forward-looking StatementsStatements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to statements regarding: Adverums plans for advancing ADVM-022; the potential benefits of ADVM-022: the expected timing of submitting an IND for diabetic retinopathy, all of which are based on certain assumptions made by Adverum on current conditions, expected future developments and other factors Adverum believes are appropriate in the circumstances. Adverum may not achieve any of these in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022; obtaining regulatory approval for gene therapy product candidates; enrolling patients in clinical trials; reliance on third parties for conducting the OPTIC trial and vector production; and ability to fund operations through completion of the OPTIC trial and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-K filed with the SEC on March 12, 2020 under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor and Media Inquiries:

Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com1-646-876-5196

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Adverum Biotechnologies Doses First Patient in Cohort 4 of OPTIC Phase 1 Clinical Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD - BioSpace