StemCell Therapy

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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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

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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,

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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

The UK company MicrofluidX has closed a 1.6M (1.4 M) seed funding round to develop a microfluidic platform that could produce cell and gene therapies more cheaply than conventional cell cultures.

The funding round was led by UKI2S, a national seed investment fund targeting early-stage companies, as well as Longwall Ventures and Cambridge Angels.

MicrofluidX will use the funding to establish a prototype of its technology. With this prototype, the company then aims to compare the performance of its microfluidics approach to current cell culture techniques used to produce gene and cell therapies.

In the dynamic cell therapy space, one of the major bottlenecks facing the field is the manufacture and scaling process, as manual cell culturing techniques are often required. Applying microfluidic technology to the manufacturing process may be an answer to this issue.

Although it is less developed at the manufacturing level, microfluidics technology has long been part of research in cell biology. It has several advantages over conventional cell cultures. For example, it allows cell cultures to be controlled more precisely on chips, increases automation, and can reduce the consumption of expensive ingredients in the cell culture process by a factor of twenty.

According to MicrofluidX, its platform could scale up microfluidics far beyond just biology research. The aim is to run dozens of cell cultures in parallel, with the capacity to produce cells more cheaply and with a higher yield than with current manufacturing techniques.

The result is that we can leverage all the inherent advantages of microfluidic cell culture at a scale never seen before, MicrofluidXs founder and CEO, Antoine Espinet, told me. This leads to much lower bioprocessing costs, better control over the final product, and faster translation from research to commercialization.

In particular, the company is investigating its technologys capacity to produce immune T-cells a common type of cell used in immunotherapies such as CAR T-cell therapies and other cell types.

Whilst the regulatory agencies are now warming up to cell and gene therapies, there are still growing pains, especially around manufacturing, Pablo Lubroth, an investor with UKI2S, told me.

It is essential to not only support companies that produce the therapies themselves, but also companies that are developing enabling technologies to ensure these therapies can be effectively commercialized and therefore have a tangible benefit to the patient.

As well as manufacturing, microfluidics is gaining traction in diagnostics and screening. For example, another UK startup, Lightcast Discovery, was founded last year to screen cells using microfluidics and beams of light. Additionally, the Belgian nanofluidics company miDiagnostics last month raised 14M to commercialize its silicon chip diagnostics in collaboration with Johns Hopkins University in the US.

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DetailsCategory: More NewsPublished on Monday, 27 April 2020 15:42Hits: 258

Affinia Therapeutics proprietary AAV vector technology to be used in Vertexs genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis

BOSTON, MA & WALTHAM, MA, USA I April 27, 2020 I Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and Affinia Therapeutics announced today that the two companies have entered into a strategic research collaboration to engineer novel adeno-associated virus (AAV) capsids to deliver transformative genetic therapies to people with serious diseases. Affinia Therapeutics proprietary AAVSmartLibrary and associated technology provides capsids for improved tissue tropism, manufacturability and pre-existing immunity. The collaboration will leverage Affinia Therapeutics capsid engineering expertise and Vertexs scientific, clinical and regulatory capabilities to accelerate the development of genetic therapies for people affected by Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and cystic fibrosis (CF).

This collaboration with Affinia Therapeutics will enhance our existing capabilities in discovering and developing transformative therapies for people with serious diseases, said Bastiano Sanna, Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. Affinia Therapeutics innovative approach to the discovery and design of AAV capsids brings yet another tool to our Vertex Cell and Genetic Therapies toolkit, and were excited to partner with them to bring together their technology platform with our research and development expertise.

At Affinia Therapeutics, were setting a new standard in genetic therapy by leveraging our platform to methodically engineer novel AAV vectors that have unique therapeutic properties, said Rick Modi, Chief Executive Officer. Vertex is an established leader in developing transformative medicines for genetic diseases and renowned for its scientific rigor. We are thankful for the scientific validation this partnership brings and look forward to working closely with them to advance life-changing, differentiated genetic therapies and make a meaningful difference to those affected by these diseases.

About the Collaboration

Under the terms of the agreement, Affinia Therapeutics will apply its vector design and engineering technologies to develop novel capsids with improved properties. The agreement provides Vertex an exclusive license under Affinia Therapeutics proprietary technology and intellectual property (IP) in DMD and DM1 with an exclusive option to license rights for CF and an additional undisclosed disease. The scope of the agreement covers all genetic therapy modalities in these diseases. Affinia Therapeutics will be eligible to receive over $1.6 billion in upfront and development, regulatory and commercial milestones, including $80 million in upfront payments and research milestones that will be paid during the research term, plus tiered royalties on future net global sales on any products that result from the collaboration. Affinia Therapeutics will be responsible for the discovery of capsids that meet certain pre-determined criteria. Vertex will be responsible for and will fund the design and manufacturing of genetic therapies incorporating the selected capsids, preclinical and clinical development efforts, and commercialization of any approved products in the licensed diseases.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need. http://www.affiniatx.com.

About Vertex Pharmaceuticals

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

SOURCE: Vertex Pharmaceuticals

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Vertex Pharmaceuticals and Affinia Therapeutics Establish Multi-Year Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies |...

DGAP-News: CEVEC Pharmaceuticals GmbH / Key word(s): Product LaunchCEVEC Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors

28.04.2020 / 08:30 The issuer is solely responsible for the content of this announcement.

CEVEC Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors

Cologne, Germany, April 28th, 2020

CEVEC Pharmaceuticals GmbH (CEVEC), the leading provider of high-performance cell technology for the manufacturing of advanced biotherapeutics from research to production scale, today announced the launch of the ELEVECTA(R) platform, a unique technology to manufacture AAV (Adeno-associated viral) vectors at large scale. With the launch, CEVEC is the first and only provider on the market to offer AAV gene therapy vector manufacturing technology on the basis of fully stable producer cell lines.

The patent-protected technology has been proven by CEVEC in pilot projects with several partners and is now commercially available for Pharma and Biotech companies running gene therapy programs from late research to all clinical phases.

ELEVECTA(R) takes AAV vector manufacturing to the next level

With the ELEVECTA(R) technology, CEVEC has taken a unique approach based on producer cell lines, which have all necessary elements for AAV production stably integrated in one cell. Up to now, manufacturing of AAV - the most widely used vector for in vivo gene therapy - has required expensive transfection reagents and cGMP-grade plasmids. ELEVECTA(R) now overcomes these limitations and enables efficient, high-performance AAV production from one cell line in consistent quality. The technology can easily be implemented in bioprocess development and large-scale GMP manufacturing facilities running standard suspension bioreactor equipment widely used in industry for monoclonal antibody and recombinant protein production.

"The launch of ELEVECTA(R) forms a milestone on the way into a new era of viral vector manufacturing as it provides a solution for one of the major challenges in gene therapy development. By closing the production gap and providing the necessary quantities of viral vectors, it paves the way to address more common indications such as Alzheimer's, Parkinson's, or Rheumatoid Arthritis as well as therapies that require larger doses," said Nicole Faust, CEO of CEVEC Pharmaceuticals. "With this launch, we provide pharmaceutical and biotechnology companies with the opportunity to benefit right from the beginning from what we believe is the future for viral vector production. The superior features in terms of scalability, production efficiency and robustness of our stable producer cell lines position CEVEC at the leading edge in the rapidly growing field of gene therapy vector manufacturing."

ELEVECTA(R) closes the production gap in gene therapy viral vector production

Gene therapy is considered to be the most effective and often only treatment option for many severe and life-threatening diseases. Moreover, in a growing number of indications, gene therapies for the first time offer the opportunity to cure a disease. In addition, as gene therapy is moving from rare and ultra-rare to more common indications with larger patient numbers and systemic treatments, larger amounts of vector material are required. Consequently, production processes are needed that are fit for this purpose, enabling the manufacture of the necessary quantities of viral vectors with high yield and consistent quality.

ELEVECTA(R) - A concept to match the customers' needs

CEVEC's newly launched ELEVECTA(R) technology overcomes the limitations of existing manufacturing methods such as restricted scalability, time-consuming and cost-intensive material sourcing and complex production processes. ELEVECTA(R) technology is based on a patent-protected, fully documented human suspension cell line developed and optimized by CEVEC.

The ELEVECTA(R) technology works for any combination of serotype-specific capsid and therapeutic gene of interest. The stable integration of these components into the cell results in cell lines designed to produce highly functional AAV vectors. Custom-made ELEVECTA(R) Producer Cell Lines are available as research cell banks or as fully tested cGMP Master Cell Banks for manufacturing of clinical and commercial material.

CEVEC launches the technology in various formats matching the needs of customers in different development phases, with focus on gene therapy programs from late research to any clinical phases. CEVEC offers cell line development services to match the specific needs of gene therapy programs and various license packages from research and development to commercial manufacturing.

Further information:

To get a deeper insight into the ELEVECTA(R) technology register for a webinar on May 5th, at 5:00 p.m. CEST (11:00 a.m. EST) featuring CEVEC's CSO, Dr. Silke Wissing, talking about "Stable AAV producer cell lines: elevating vector manufacturing" and/or visit the ELEVECTA(R) web page.

In addition, you can meet CEVEC's management at the 23rd ASGCT virtual Annual Meeting taking place from May 12 - 15, 2020. During this event, interested parties can listen to the ELEVECTA(R) poster presentations and visit the CEVEC virtual booth. Please request a virtual meeting through the ASGCT webpage or directly contact CEVEC under bizdev@cevec.com.

About CEVEC:

CEVEC is a leading provider of high-performance cell technology for the manufacturing of advanced bio-therapeutics from R&D to manufacturing scale. The company's product portfolio comprises platform technologies for gene therapy viral vectors (AAV, Adenoviral vectors, Lentiviral vectors,), vaccines and complex recombinant proteins. With the ELEVECTA(R) Technology, CEVEC offers a unique solution for large-scale production of AAV vectors using producer cell lines with all necessary components stably integrated into the cell. The technology is based on suspension cells and does not require any expensive transfection reagents and cGMP plasmids. CEVEC's CAP(R) Technology based on human suspension cells is the ideal production platform for RCA-free Adenoviral vectors, Lentiviral vectors, Oncolytic viruses, Viral vaccines and exosomes. With the CAP-Go(R) Technology CEVEC provides a solution to the increasing need for recombinant production of complex and highly glycosylated protein molecules, including laminins, coagulation factors and plasma proteins.

For more information, please visit the Company's website.

Follow CEVEC on LinkedIn and Twitter.

Contact:

28.04.2020 Dissemination of a Corporate News, transmitted by DGAP - a service of EQS Group AG.The issuer is solely responsible for the content of this announcement.

The DGAP Distribution Services include Regulatory Announcements, Financial/Corporate News and Press Releases. Archive at http://www.dgap.de

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CEVEC Pharmaceuticals GmbH : Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors...

MicrofluidX will use fundraising money to advance its cell bioprocessing technology, which uses microfluidics to tackle challenges associated with bioprocessing advanced therapies.

The London, UK-based firm has raised 1.4 million ($1.7 million) in seed funding to advance its microfluidics-based technology, which aims to combat challenges associated with the production of cell and gene therapies (CGTs) such as cost of goods, batch variability, and scalability.

Cell and gene therapies have demonstrated significant therapeutic potential. However, these treatments are now at a critical juncture, Antoine Espinet, CEO of MicrofluidX, told Bioprocess Insider. Complex manufacturing challenges remain and impact the development and accessibility of therapies to patients.

Image: iStock/barbol88

According to Espinet, current technologies lead to high treatment cost of manufacturing, driven by high reagent volumes, logistics-heavy centralized manufacturing in large specialized facilities, and low automation; product variability, both batch-to-batch and within each batch due to low control over all the parameters of the bioprocess; and long process re-engineering to scale from experimental batches to manufacture of therapeutics doses.

MicrofluidXs technology is based on microfluidics cell culture, which Espinet said holds several advantages over conventional single-use technologies. These include a 10-20 times lower reactant consumption, increased process efficiency, up to 50 times more cells per unit volume/surface area, and a high level of precision and control over all process parameters.

MicrofluidX leverages the power of microfluidics thanks to its highly parallelizable microfluidic bioprocessing system that combines multiple cell culture capabilities in one closed system and is fully scalable from thousands to several hundred million cells without the need to re-engineer your process: same yield, same quality, same results.

The financing will be used to build a working prototype that can be used to generate comparative biological data between this platform and conventional single-use technologies.

MicrofluidX is working to bring its products to the market quickly, Espinet said, and is looking to partner with academics, biotechs, pharmas and Contract Manufacturing Organizations (CMO) to develop and test its products on various cell and gene therapy processes.

In particular, an important piece of work is for those partners to run side-by-side comparisons with existing SUTs on their cells and processes. With these comparisons expected to show break-through improvements, MicrofluidX will enter into its industrialization and marketing phase, to make its technology available to the bioprocessing world.

Link:
MicrofluidX to take on single-use with microfluidics - Bioprocess Insider - BioProcess Insider

Hackers leak data related to WHO, Wuhan lab and Gates Foundation

A common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients, say researchers.

Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight.

Current treatments include either eye drops, laser or surgery, all of which have limitations and disadvantages. "At present, there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early," said study researcher Dr Colin Chu from the University of Bristol in the UK.

For the findings, published in the journal Molecular Therapy, the research team tested a new approach that could provide additional treatment options and benefits. The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.

The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye. Using the latest gene-editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure.

"We hope to advance towards clinical trials for this new treatment in the near future. If it's successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money," Chu said. The researchers are currently in discussion with industry partners to support further laboratory work and rapidly progress this new treatment option towards clinical trials.

Read this article:
Glaucoma can be successfully treated with gene therapy - International Business Times, Singapore Edition

Worldwide Gene Therapy Market has been thoroughly elaborated in a research report put on sale by Marketresearch.biz, bearing the title Gene Therapy MarketGlobal Industry Analysis, Size, Share, Growth, Trends, and Forecast 20202029. According to the report, the market is expected to be driven by a wide range of macroeconomic and industry-related factors. The key players in this market are focusing on product strategies and developments to increase their customer base and maintain their position.

The Gene Therapy Market is an intrinsic study of the current status of this business vertical and encompasses a brief synopsis about its segmentation. This industry report is inclusive of a nearly accurate prediction of the market scenario over the forecast period 20202029 market size with respect to valuation as sales volume. The study lends focus to the top magnates comprising the competitive landscape of Gene Therapy market, as well as the geographical areas where the industry extends its horizons, in magnanimous detail.

|| Access insightful study with over 100+ pages, list of tables & figures, profiling 10+ companies. Ask for Free Sample Copy(PDF) @https://marketresearch.biz/report/gene-therapy-market/request-sample

Operational and Emerging Players: Gene Therapy Market- Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

According to the current market situation, this report continuously observing promising growth of the global Gene Therapy market. The report further suggests market appears to progress at an accelerating rate over the forecast period. Also, the major players are elaborated on the basis of the proprietary technologies, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, report also examines R&D developments, legal policies, SWOT Analysis and strategies of Gene Therapy market players.

This Gene Therapy market research report surrounds importance on:

Manufacturing process and technology used in Gene Therapy market, key developments and trends changing in the development

Complete examination, including an evaluation of the parent market

Detailed account of market, volume and forecast, by leading key players, product type and end users applications

Environmental spread, development designs, pieces of the overall industry, key methodologies, and different financials systems of Gene Therapy market

Industrial analysis by upstream raw materials, downstream industry, current market dynamics and ensuing consumers analysis

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Gene Therapy Market Segmentation Outlook:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Gene Therapy Market Section by Region:

ASIA-PACIFIC MARKET: China, Southeast Asia, India, Japan, Korea, Western Asia

THE MIDDLE EAST & AFRICA MARKET: GCC, North Africa, South Africa

NORTH AMERICA MARKET: United States, Canada, Mexico

EUROPE MARKET: Germany, Netherlands, UK, France, Russia, Spain, Italy, Turkey, Switzerland

SOUTH AMERICA MARKET: Brazil, Argentina, Columbia, Chile, Peru

Our Research Methodology is based on the following main points:

i. Data Collections and Interpretation

ii. Analysis

iii. Data Validation

iv. Final Projections and Conclusion

The latest research on the Gene Therapy Market fundamentally delivers insights that can empower stakeholders, business owners, and field marketing executives to make effective investment decisions driven by facts and extremely thorough research. The study aims to provide an evaluation and deliver essential information on the competitive landscape to meet the unique requirements of the companies and individuals operating in the Gene Therapy Market for the forecast period, 20202029. To help firms comprehend the Gene Therapy industry in multiple ways, the report exhaustively assesses the share, size, and growth rate of the business worldwide.

There are 13 Sections to show the global Gene Therapy market:

Chapter 1: Market Overview, Drivers, Segmentation overview, Restraints and Opportunities

Chapter 2: Market competition by key Manufacturers

Chapter 3: Production by Regions

Chapter 4: Consumption by Regions

Chapter 5: Production By Types, Revenue and Market share by Types

Chapter 6: Market share (%) and Growth Rate by Applications, Consumption By Applications

Chapter 7: Complete profiling and analysis of leading Manufacturers

Chapter 8: Region-wise manufacturing expenses, Manufacturing cost analysis, Raw materials analysis

Chapter 9: Sourcing Strategy, Industrial Chain and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Market Effect Factors Analysis, Impact Analysis

Chapter 12: Market Forecast 2020-2029

Chapter 13: Gene Therapy Research Findings and Conclusion, methodology and data source, Appendix

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Conclusively, this report will provide you a clean view of each and every truth of the market without a need to consult another research report or a statistics supply. Our report will offer you with all of the facts about the past, present, and future of the Market.

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Inclusive Comprehension 2020: Gene Therapy Market (Trending PDF) Addressing Structure, Scope, Potential, and Growth Prospects Till 2029 - Jewish Life...

Lysogene S.A.s (EPA:LYS): Lysogene S.A. engages in the research and clinical development of gene therapy for neurodegenerative disorders. The 64m market-cap company announced a latest loss of -4.3m on 31 December 2019 for its most recent financial year result. Many investors are wondering the rate at which LYS will turn a profit, with the big question being when will the company breakeven? Below I will provide a high-level summary of the industry analysts expectations for LYS.

Check out our latest analysis for Lysogene

According to the industry analysts covering LYS, breakeven is near. They expect the company to post a final loss in 2021, before turning a profit of 69m in 2022. LYS is therefore projected to breakeven around 2 years from now. In order to meet this breakeven date, I calculated the rate at which LYS must grow year-on-year. It turns out an average annual growth rate of 50% is expected, which is extremely buoyant. If this rate turns out to be too aggressive, LYS may become profitable much later than analysts predict.

Im not going to go through company-specific developments for LYS given that this is a high-level summary, however, keep in mind that by and large a biotech has lumpy cash flows which are contingent on the product type and stage of development the company is in. This means that a high growth rate is not unusual, especially if the company is currently in an investment period.

One thing Id like to point out is that LYS has managed its capital judiciously, with debt making up 13% of equity. This means that LYS has predominantly funded its operations from equity capital,and its low debt obligation reduces the risk around investing in the loss-making company.

There are too many aspects of LYS to cover in one brief article, but the key fundamentals for the company can all be found in one place LYSs company page on Simply Wall St. Ive also put together a list of relevant aspects you should further research:

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

Read more:
Lysogene S.A. (EPA:LYS): When Will It Breakeven? - Simply Wall St

April 28 (Reuters) - Novartis AG:

* NOVARTIS CEO SAYS CORONAVIRUS IMPACTS ARE GREATEST ON NEW CLINICAL STUDY STARTS, SAYS MANAGING SITUATION VIA DIGITAL TECHNOLOGY

* NOVARTIS CEO SAYS INCREASING REMOTE MONITORING OF PATIENTS IN CLINICAL STUDIES

* NOVARTIS CEO SAYS REGULATORS HAVE COMMITTED TO MAINTAINING TIMELINES FOR NEW MEDICINES

* NOVARTIS CEO SAYS BEOVU WAS OFF TO OUTSTANDING LAUNCH, HAS IDENTIFIED RARE SAFETY SIGNAL FOR RETINAL VASCULITIS, STUDYING THE REASON FOR SAFETY SIGNAL, CONTINUES TO BELIEVE MEDICINE WILL EVENTUALLY EXCEED $1 BILLION IN SALES

* NOVARTIS CEO SAYS GUIDANCE ASSUMES HEALTH CARE SYSTEMS WILL RESUME NORMAL OPERATIONS IN Q2

* NOVARTIS CEO SAYS HAS PAUSED ENROLLMENT IN UK INCLISIRAN TRIAL DUE TO CORONAVIRUS PANDEMIC, HOPEFUL TO RESTART IN COMING QUARTERS

* NOVARTIS CEO SAYS SEES SOME SLOWDOWN IN HOSPITAL VISITS IMPACTING LUTATHERA, KYMRIAH, ZOLGENSMA, SAYS DOING BEST TO REACH OUT TO PATIENTS WHO NEED GENE THERAPY

* NOVARTIS CEO SAYS DIFFICULT TO PREDICT HOW PANDEMIC WILL UNFOLD IN COMING YEAR, AS THINGS EVOLVE WILL UPDATE GUIDANCE AS NEEDED

* NOVARTIS CEO SAYS FEW OF STUDIES SO FAR ON THERAPIES FOR CORONAVIRUS HAVE MET GOLD STANDARD OF RANDOMIZED, BLINDED CLINICAL STUDIES, STILL NEED PROPERLY POWERED BLINDED STUDIES TO REALLY DETERMINE MEDICINES HAVE BENEFIT Further company coverage: (Reporting by John Miller)

Go here to see the original:
BRIEF-Novartis CEO Says Still Need Proper Blinded Studies To Determine Which Drugs Have Benefit for COVID-19 - Reuters UK

The research study on Global Personalized Gene Therapy Treatment market 2019 presents an extensive analysis of current Personalized Gene Therapy Treatment market size, drivers, trends, opportunities, challenges, as well as key Personalized Gene Therapy Treatment market segments. Further, it explains various definitions and classification of the Personalized Gene Therapy Treatment industry, applications, and chain structure.In continuation of this data, the Personalized Gene Therapy Treatment report covers various marketing strategies followed by key players and distributors. Also explains Personalized Gene Therapy Treatment marketing channels, potential buyers and development history. The intent of global Personalized Gene Therapy Treatment research report is to depict the information to the user regarding Personalized Gene Therapy Treatment market forecast and dynamics for the upcoming years. The Personalized Gene Therapy Treatment study lists the essential elements which influence the growth of Personalized Gene Therapy Treatment industry. Long-term evaluation of the worldwide Personalized Gene Therapy Treatment market share from diverse countries and regions is roofed within the Personalized Gene Therapy Treatment report. Additionally, includes Personalized Gene Therapy Treatment type wise and application wise consumption figures.

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Highlights of Global Personalized Gene Therapy Treatment Market Report:* This report provides in detail analysis of the Personalized Gene Therapy Treatment and provides market size (US$ Million) and Cumulative Annual Growth Rate (CAGR (%)) for the forecast period: 2019 ; 2029. * It also elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for world Personalized Gene Therapy Treatment market. * This study also provides key insights about Personalized Gene Therapy Treatment market drivers, restraints, opportunities, new product launches, approvals, regional outlook, and competitive strategies adopted by the leading Personalized Gene Therapy Treatment players. * It profiles leading players in the worldwide Personalized Gene Therapy Treatment market based on the following parameters ; company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies and future plans. * Insights from Personalized Gene Therapy Treatment report would allow marketers and management authorities of companies to make an informed decision with respect to their future product launches, market expansion, and Personalized Gene Therapy Treatment marketing tactics. * The world Personalized Gene Therapy Treatment industry report caters to various stakeholders in Personalized Gene Therapy Treatment market. That includes investors, device manufacturers, distributors and suppliers for Personalized Gene Therapy Treatment equipment. Especially incorporates government organizations, Personalized Gene Therapy Treatment research and consulting firms, new entrants, and financial analysts. *Various strategy matrices used in analyzing the Personalized Gene Therapy Treatment market would provide stakeholders vital inputs to make strategic decisions accordingly.

Global Personalized Gene Therapy Treatment Market Report Provides Comprehensive Analysis of Following: ; Personalized Gene Therapy Treatment Market segments and sub-segments ; Industry size ; Personalized Gene Therapy Treatment shares ; Personalized Gene Therapy Treatment Market trends and dynamics ; Market Drivers and Personalized Gene Therapy Treatment Opportunities ; Supply and demand of world Personalized Gene Therapy Treatment industry ; Technological inventions in Personalized Gene Therapy Treatment trade ; Personalized Gene Therapy Treatment Marketing Channel Development Trend ; Global Personalized Gene Therapy Treatment Industry Positioning ; Pricing and Brand Strategy ; Distributors/Traders List enclosed in Positioning Personalized Gene Therapy Treatment Market.

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Personalized Gene Therapy Treatment Market Analysis By Industry Size, Share, Revenue Growth, Development And Demand Forecast To 2029 - Germany English...

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Global Gene Therapy For CNS Disorders Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

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Gene Therapy For CNS Disorders Market Region Coverage (Regional Production, Demand & Forecast by Countries etc.):

North America (U.S., Canada, Mexico)

Europe (Germany, U.K., France, Italy, Russia, Spain etc.)

Asia-Pacific (China, India, Japan, Southeast Asia etc.)

South America (Brazil, Argentina etc.)

Middle East & Africa (Saudi Arabia, South Africa etc.)

Some Notable Report Offerings:

-> We will give you an assessment of the extent to which the market acquire commercial characteristics along with examples or instances of information that helps your assessment.

-> We will also support to identify standard/customary terms and conditions such as discounts, warranties, inspection, buyer financing, and acceptance for the Gene Therapy For CNS Disorders industry.

-> We will further help you in finding any price ranges, pricing issues, and determination of price fluctuation of products in Gene Therapy For CNS Disorders industry.

-> Furthermore, we will help you to identify any crucial trends to predict Gene Therapy For CNS Disorders market growth rate up to 2026.

-> Lastly, the analyzed report will predict the general tendency for supply and demand in the Gene Therapy For CNS Disorders market.

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Table of Contents:

Study Coverage: It includes study objectives, years considered for the research study, growth rate and Gene Therapy For CNS Disorders market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Gene Therapy For CNS Disorders market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Gene Therapy For CNS Disorders Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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Tags: Gene Therapy For CNS Disorders Market Size, Gene Therapy For CNS Disorders Market Growth, Gene Therapy For CNS Disorders Market Forecast, Gene Therapy For CNS Disorders Market Analysis

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Gene Therapy For CNS Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald

Analysis Report on Gene Therapy Market

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Analytical Tools: The Global Gene Therapy Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, feasibility study, and many other market research tools have been used to analyze the growth of the key players operating in the market.

COVID-19 Impact on Gene Therapy Market

Adapting to the recent novel COVID-19 pandemic, the impact of the COVID-19 pandemic on the global Gene Therapy market is included in the present report. The influence of the novel coronavirus pandemic on the growth of the Gene Therapy market is analyzed and depicted in the report.

The global Gene Therapy market segment by manufacturers include

segmented as follows:

Global Gene Therapy Market, by Product

Global Gene Therapy Market, by Application

Global Gene Therapy Market, by Region

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Moreover, the report highlighted revenue, sales, manufacturing cost, and product and the States that are most competitive in the lucrative market share idea. There is a discussion on the background and financial trouble in the global Gene Therapy economic market. This included the CAGR value during the outlook period leading to 2025.

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Conclusively, this report will provide you a clear view of each and every fact of the market without a need to refer to any other research report or a data source. Our report will provide you with all the facts about the past, present, and future of the concerned Market.

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COVID-19 impact: Gene Therapy Market 2019 Business Insights,Top Companies,Growth, Market Size Global Market Share, Trends, Outlook, Opportunity and...

Plasmid DNA Manufacturing Market research report is the professional study with the premium insights which includes the size of the business, the ongoing patterns, drivers, dangers, conceivable outcomes and primary segments. The report encompasses the competition landscape entailing share analysis of the key players in the Plasmid DNA Manufacturing market based on their revenues and other significant factors. Report analyzes changing trends and competitive analysis which becomes essential to monitor performance and make critical decisions for growth and development. It also provides market information in terms of development and its capacities.

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Leading companies Influencing in this Market are:

Cobra Bio, Richter-Helm, Eurogentec, Cell and Gene Therapy Catapult, VGXI, PlasmidFactory, Kaneka Corporation, Nature Technology Corporation, Waisman Biomanufacturing, FUJIFILM Diosynth Biotechnologies, LakePharma.

In this Plasmid DNA Manufacturing Market research report, the prominent factors driving the advancement of this market were recorded and the business accomplices and end administrators were indulgent. The setup of the business division, examples, and challenges monitoring the market comprehensively are in like manner a bit of this wide examination. Different meetings and social events were driven by the distinguishable pioneers of this industry to get persisting and revived encounters concerned to the market.

The report gathers the essential information including the new strategies for growth of the industry and the potential players of the global Plasmid DNA Manufacturing Market. It enlists the topmost industry player dominating the market along with their contribution to the global market. The report also demonstrates the data in the form of graphs, tables, and figures along with the contacts details and sales of key market players in the global Plasmid DNA Manufacturing Market.

Global Plasmid DNA Manufacturing Market Segmentation:

Segmentation by Type:

HQ Grade Plasmid DNAGMP Grade Plasmid DNANon-GMP Grade Plasmid DNA

Segmentation by Application:

CancersInherited DisordersViral InfectionsOthers

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Key questions answered in the report include:

Table of Contents

Global Plasmid DNA Manufacturing Market Research Report 2020 2026

Chapter 1 Plasmid DNA Manufacturing 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

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Plasmid DNA Manufacturing Market Forecast

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Future Prospects of Plasmid DNA Manufacturing Market by 2026 with Top Key Players Cobra Bio, Richter-Helm, Eurogentec, Cell and Gene Therapy...

Spark Therapeutics Inc.

Global Gene Therapy For Inherited Genetic Disorders Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

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Gene Therapy For Inherited Genetic Disorders Market Region Coverage (Regional Production, Demand & Forecast by Countries etc.):

North America (U.S., Canada, Mexico)

Europe (Germany, U.K., France, Italy, Russia, Spain etc.)

Asia-Pacific (China, India, Japan, Southeast Asia etc.)

South America (Brazil, Argentina etc.)

Middle East & Africa (Saudi Arabia, South Africa etc.)

Some Notable Report Offerings:

-> We will give you an assessment of the extent to which the market acquire commercial characteristics along with examples or instances of information that helps your assessment.

-> We will also support to identify standard/customary terms and conditions such as discounts, warranties, inspection, buyer financing, and acceptance for the Gene Therapy For Inherited Genetic Disorders industry.

-> We will further help you in finding any price ranges, pricing issues, and determination of price fluctuation of products in Gene Therapy For Inherited Genetic Disorders industry.

-> Furthermore, we will help you to identify any crucial trends to predict Gene Therapy For Inherited Genetic Disorders market growth rate up to 2026.

-> Lastly, the analyzed report will predict the general tendency for supply and demand in the Gene Therapy For Inherited Genetic Disorders market.

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Table of Contents:

Study Coverage: It includes study objectives, years considered for the research study, growth rate and Gene Therapy For Inherited Genetic Disorders market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Gene Therapy For Inherited Genetic Disorders market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Gene Therapy For Inherited Genetic Disorders Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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Tags: Gene Therapy For Inherited Genetic Disorders Market Size, Gene Therapy For Inherited Genetic Disorders Market Growth, Gene Therapy For Inherited Genetic Disorders Market Forecast, Gene Therapy For Inherited Genetic Disorders Market Analysis

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Gene Therapy For Inherited Genetic Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald

From Our Archives Medical Author: Frederick Hecht, MD, FAAP, FACMG

Gene therapy is the treatment of disease by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is responsible for the disease. Gene therapy may use the genetic material, DNA, itself as the means of treatment.

DNA or deoxyribonucleic acid is the very long molecule that encodes the genetic information. A gene is a stretch of DNA required to make a functional product such as part or all of a protein. People have about 100,000 to 150,000 genes. During gene therapy, DNA that codes for specific genes is delivered to individual cells in the body.

Most, if not all, diseases have a genetic factor. The genetic factor can be wholly or partially responsible for the disease. For example, in disorders such as cystic fibrosis, hemophilia, and muscular dystrophy, changes in a gene directly result in the condition. In other conditions such as high cholesterol and high blood pressure, genetic and environmental factors interact to cause disease. Disorders associated with aging often involve the loss of gene activity in specific types of cells. Even infections can be related to genes. In fact, they have two sets of genetic determinants: the genes of the infective agent and the genes of the person with the infection.

Uses of gene therapy

Gene therapy is being used in many ways. For example,to:

A large variety of genes are now being tested for use in gene therapy. Examples include: a gene for the treatment of cystic fibrosis (a gene called CFTR that regulates chloride); genes for factors VIII and IX, deficiency of which is responsible for classic hemophilia (hemophilia A) and another form of hemophilia (hemophilia B), respectively; genes called E1A and P53 that cause cancer cells to undergo cell death or revert to normal; AC6 gene which increases the ability of the heart to contract and may help in heart failure; and VEGF, a gene that induces the growth of new blood vessels (angiogenesis) of use in blood vessel disease.

A short synthetic piece of DNA (called an oligonucleotide) is being used by researchers to "pre-treat" veins used as grafts for heart bypass surgery. The piece of DNA seems to switch off certain genes in the grafted veins to prevent their cells from dividing and thereby prevent atherosclerosis.

Delivery of genes into cells

Gene delivery can be used in cells that have been removed from the body (ex vivo gene therapy) or in cells that are still in the body (in vivo gene therapy). Genes can be delivered into cells in different ways. The selection of a gene delivery system depends on the target cell, the duration of gene expression required for therapeutic effect, and the size of the piece of DNA to be used in the gene therapy.

Genes can be carried into cells by viruses. Viral vectors or carriers take advantage of the natural ability of a virus to enter a cell and deliver genetic material to the nucleus of the cell that contains its DNA. In developing virus carriers, the DNA coding for some or all of the normal genes of the virus to be used as a carrier are removed and replaced with a treatment gene. Most of these virus carriers are engineered so that they are able to enter cells, but they cannot reproduce themselves and so are innocuous.

Genes can also be delivered within tiny synthetic "envelopes" of fat molecules. Cell membranes contain a very high concentration of fat molecules. The fat molecule "envelope" can carry the therapeutic gene into the cell by being admitted through the cell membrane as if it were one of its own molecules.

Genes can also gain entrance into cells when an electrical charge is applied to the cell to create tiny openings in the membrane that surrounds a cells. This technique is called electroporation.

A "bionic chip"

A new "bionic chip" has been developed to help gene therapists using electroporation to slip fragments of DNA into cells. Electroporation was originally a hit-or-miss technique because there was no way to determine how much of an electrical jolt it took to open the cell membrane.

The "bionic chip" solves this problem. It contains a single living cell embedded in a tiny silicon circuit. The cell acts as a diode, or electrical gate. When it is hit with just the right charge, the cell membrane opens, allowing the electricity to pass from the top to the bottom of the bionic chip. By recording what voltage caused this phenomenon to occur, it is now posssible to determine precisely how much electricity it takes to pry open different types of cells.

Route of administration of gene therapy

The choice of route for gene therapy depends on the tissue to be treated and the mechanism by which the therapeutic gene exerts its effect. Gene therapy for cystic fibrosis, a disease which effects cells within the lung and airway, may be inhaled. Most genes designed to treat cancer are injected directly into the tumor. Proteins such as factor VIII or IX for hemophilia are also being introduced directly into target tissue (the liver).

The potential power of gene therapy

Most gene therapy for diseases such as cystic fibrosis and hemophilia has been designed only to ease, not to cure, the disease. However, the delivery of functional copies of genes provides a potential method to correct a disease at its most basic level.

Gene therapy also holds the potential to provide "patient-friendly" treatment regimens for a variety of diseases. Today, many patients with hemophilia and diabetes must have repeated injections in order to manage their disease because proteins exist in the blood stream for a limited period of time before they are degraded or eliminated. Since DNA is more stable and functions inside the cell, the delivery of genes may result in longer-term expression of the necessary proteins. SLIDESHOW Heart Disease: Causes of a Heart Attack See Slideshow

Because of its accuracy, gene therapy has the potential to eliminate cancer cells without damaging normal, healthy tissue. Furthermore, cancer gene therapies may provide alternatives when a disease does not respond to other older treatments.

The potential of gene therapy is great but, compared to its promise, the results to date are still quite limited. However, the benefits of gene therapy are believed to be on the near horizon. Gene therapy is one of the hottest areas of medical research today. (And gene therapy companies have been among the hottest in the stock market.)

The remarkable advances in genetics, including the human genome project, have opened new doors for the exploration of gene therapy. New technologies are needed to speed the progress of gene therapy. As these new technologies such as the "bionic chip" arrive, we believe that, without a doubt, gene therapy will play an increasingly important and prominent part in medicine in the decades to come.

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Gene Therapy - The Future Is Here! - MedicineNet