Archive for the ‘Gene Therapy Research’ Category

Portland, OR, Aug. 05, 2020 (GLOBE NEWSWIRE) -- According to the report, the global Gene Therapy Marketwas pegged at$393.35 millionin 2018, and is projected to reach$6.21 billionby 2026, registering a CAGR of 34.8% from 2019 to 2026. The report offers an extensive analysis of changing market dynamics, key winning strategies, business performance, major segments, and competitive scenarios.

High investment in R&D activities, increase in prevalence of cancer, and growth in awareness regarding gene therapy have boosted the growth of the global gene therapy market. However, high costs associated with gene therapies and unwanted immune responses hamper the market growth. On the contrary, untapped potential for the emerging market is expected to create lucrative opportunities in the near future.

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Covid-19 scenario:

The viral vector segment held the largest share in 2018, contributing to more than half of the global gene therapy market, owing to easier modifications of many viruses such as Lentivirus, Adeno-Associated Virus (AAV), RetroVirus & Gamma RetroVirus to deliver gene therapy drugs. However, the non-viral vector segment is expected to register the fastest CAGR of 38.8% during the forecast period. This is attributed to the technological advancements in physicochemical approaches such as physical methods and chemical methods of non-viral vectors.

The tumor suppressor segment to portray the fastest CAGR of 52.9% during the forecast period, owing to rise in number of methodology and clinical trials of tumor suppressor for the gene therapy treatment. However, the antigen segment held the largest share in 2018, contributing to more than one-fifth of the global gene therapy market, due to the presence of a wide range of genetic mutations and dysregulated gene expression of tumor cells.

The global gene therapy market acrossNorth Americaheld the largest share in 2018, accounting for nearly half of the market, owing to high prevalence rate of cancer, presence of high disposable income, and increase in funding for R&D activities associated with gene therapy. However, the market across theAsia-Pacificregion is projected to register the fastest CAGR of 45.4% during the forecast period, owing to rise in number of people prone to various chronic diseases and approval & launch of gene therapy products.

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The key players operating in the global gene therapy market include Adaptimmune Therapeutics Plc., Anchiano Therapeutics Ltd., Achieve Life Sciences, Inc., Adverum Biotechnologies, Inc., Abeona Therapeutics Inc., Applied Genetic Technologies Corporation, Arbutus Biopharma Corporation, Audentes Therapeutics, Inc., AveXis, Inc., Bluebird Bio, Inc., Celgene Corporation, CRISPR Therapeutics AG, Editas Medicine, Inc., Editas Medicine, Inc., GlaxoSmithKline Plc., Intellia Therapeutics, Inc., Merck & Co., Inc., Novartis AG, REGENXBIO Inc., Spark Therapeutics, Inc., Sangamo Therapeutics, Inc., Uniqure N. V., Voyager Therapeutics, Inc. Other prominent players in the value chain (companies not profiled in the report) includes Amgen, Epeius Biotechnologies, Sanofi, Juno Therapeutics, and Advantagene.

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Gene Therapy Market to Reach $6.21 Billion by 2026: Allied Market Research - GlobeNewswire

A lone genetic mutation can cause a life-changing disorder with effects on multiple body systems. Lysosomal storage diseases, for example, of which there are dozens, arise due to single mutations that affect production of critical enzymes required to metabolize large molecules in cells. These disorders affect multiple organs including, notably, the brain, causing intellectual disability of varying degrees.Gene therapy holds promise to address these conditions, but the brains own protective mechanism the blood-brain barrier has been a formidable challenge for researchers working to develop one.

In a new study published in the journal Brain, a team led by John H. Wolfe, a researcher with Penns School of Veterinary Medicine and Perelman School of Medicine and the Childrens Hospital of Philadelphia, successfully applied a gene therapy platform to completely correct brain defects in a large animal model of a human genetic disease.

This is the first example of a large-brain mammal with a bona fide human genetic disease that has intellectual disability as part of the human syndrome where weve been able to correct the biochemistry and pathologic lesions in the whole brain, says Wolfe.

Wolfe has worked on models of human genetic diseases that impact the brain for many years. With gene therapy, a delivery vehicle typically a viral vector is used to provide the normal version of a mutated gene to correct a condition. Wolfe and other scientists working in this area have made steady progress to treat neurogenetic diseases in rodents. However, applying the same treatment to the much larger brain of higher mammals has only been able to produce partial corrections.

Theres been a lot of excitement for the last 10 years or so that specific vectors can be injected into the blood and enter the brain, says Wolfe. They do cross the blood-brain barrier. One such treatment with restricted distribution has been effective in treating a disease that primarily affects the spinal cord.

And while scientists have shown these therapies can reverse the pathology throughout the brains of mice, its been hard to judge what effect it would have in patients, as the rodent brains have a much smaller cerebral cortex than larger mammals, like humans.

In the current study, the team used an animal model with a brain more similar to humans, cats, to assess the effectiveness of a gene-correcting therapy for one type of lysosomal storage disease: a condition called alpha-mannosidosis, which naturally occurs in cats and results from a mutated copy of the alpha-mannosidase gene.

Having refined the gene delivery technique during many years of work, the researchers selected a specific vector that they showed, in mice, was capable of crossing the blood-brain barrier to reach sites throughout the brain.

They next delivered the vector, containing a reporter gene, to normal cats. Several weeks later, they were able to find evidence that the corrected gene had distributed to various parts of the brain, including the cerebral cortex, hippocampus, and mid-brain.

Finally the research team assessed the therapy in cats with alpha-mannosidosis, using either a low or high dose of the vector. They injected the therapy into the carotid artery, so that it would go directly to the brain before traveling to other parts of the body. Compared to untreated cats, treated animals had a significant delayed onset of certain neurological symptoms and a longer lifespan; those that received the higher dose of the vector delivered through the carotid artery lived the longest.

Its a big advance, says Wolfe. Nobody has been able to treat the whole brain of a large-brained animal before. Were hopeful that this will translate into clinical use in humans.

Wolfe cautions, however, the findings dont amount to a cure.

These were significant improvements, but they were only just improvements on a serious condition, Wolfe says. The cats werent cured, and we dont know what impact this has on mental ability. However, since the pathology is found throughout the brain, it is thought that complete correction will be necessary.

As alpha-mannosidosis is a childhood-onset disease with no cure, however, any improvements that lessen the severity of symptoms are welcome. The approach the researchers developed may potentially be employed to treat many other diseases that affect the whole central nervous system.

In future work, Wolfe and his collaborators hope to refine their methods to achieve the same outcomes with a lower dose, making an effective treatment safer as well as more affordable. And they will continue to work to understand the details of why their treatment works, including precisely how the vector travels through the brain, a line of investigation that could shed light on additional strategies to address these serious disorders.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Gene Therapy Beats the Blood-Brain Barrier To Cure Cat Disease - Technology Networks

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Global Cell and Gene Therapy Market Report. The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.

New York, Aug. 04, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" - https://www.reportlinker.com/p05827567/?utm_source=GNW 90% during the period 20192025.

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe. The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

The following factors are likely to contribute to the growth of the cell and gene therapy market during the forecast period: Increase in Strategic Acquisition Activities Increased Funding for Cell & Gene Therapy Products Expanding Applications of Cell and Gene Therapies Increased in the Patient Pool

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019?2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market. Cell And Gene Therapy Market Segmentation The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography. In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product Cell Therapy Gene Therapy Segmentation by Disease Dermatology Musculoskeletal Oncology Genetic Disorders Others Segmentation by End-user Hospitality Cancer Care Centers Wound Care Centers Ambulatory Surgical Centers Others

INSIGHTS BY GEOGRAPHY In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 12 years, the number of approved gene therapies is expected to double. The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography North America o US o Canada Europe o UK o Germany o France o Spain o Italy APAC o China o Japan o South Korea o Australia o India Latin America o Brazil o Mexico Middle East & Africa o Saudi Arabia o Turkey o South Africa o UAE

INSIGHTS BY VENDORS The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares. Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors Gilead Sciences Spark Therapeutics Novartis Organogenesis Amgen Osiris Therapeutics Dendreon Vericel

Other Prominent Vendors Anterogen Tego Sciences Japan Tissue Engineering JCR Pharmaceuticals Medipost MolMed AVITA Medical CollPlant Biosolution Stempeutics Research Kolon Tissue Gene Orchard Therapeutics Sibiono GeneTech NuVasive Corestem Pharmicell Shanghai Sunway Biotech RMS Regenerative Medical System Takeda Pharmaceutical Company CHIESI Farmaceutici CO.DON AnGes GC Pharma Human Stem Cells Institute JW CreaGene APAC Biotech Nipro Terumo Orthocell bluebird bio

Key Questions Answered 1. What is the cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors impacting the growth of the cell and gene therapy market share? 3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market? 4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares? 5. Which product type/ end-user type/region is generating the largest revenue in the Asia Pacific region?Read the full report: https://www.reportlinker.com/p05827567/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Yahoo Finance

PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., Aug. 4, 2020 /PRNewswire/ --Kriya Therapeutics announced today that it has secured a 51,350 square foot operational manufacturing facility in Research Triangle Park (RTP), North Carolina to support the scalable production of its pipeline of AAV-based gene therapies for highly prevalent serious diseases. The facility is designed to have its own fully integrated process development lab, quality control and analytical development capability, pilot production suite, and current good manufacturing practice (cGMP) production infrastructure. Kriya will manufacture gene therapies at the facility using its scalable suspension cell culture manufacturing process at up to 2,000-liter bioreactor scale. The facility's pilot production suite and full cGMP manufacturing infrastructure are expected to be online in the first and second half of 2021, respectively.

"Manufacturing continues to be a critical bottleneck to the advancement of gene therapies for prevalent diseases," said Britt Petty, Chief Manufacturing Officer at Kriya Therapeutics. "With the establishment of our cGMP manufacturing facility in North Carolina, we are preparing to have the capacity to support our pipeline of programs addressing large patient populations, from initial INDs through late-phase clinical studies. Moreover, we are investing in process innovation and scalable infrastructure with the goal of reducing the cost of goods of our therapies by orders of magnitude."

"As we develop our platform technologies and advance our pipeline of gene therapies, we are committed to securing the capacity to support the manufacturing of our products at scale," said Shankar Ramaswamy, M.D., Co-Founder, Chairman, and CEO of Kriya Therapeutics. "Our investment in our RTP facility helps establish this capability in a region with tremendous talent in gene therapy manufacturing, while also enabling our team to focus on innovations to bring down the cost of goods of our gene therapies."

About Kriya Therapeutics

Kriya Therapeutics is a next-generation gene therapy company focused on developing transformative treatments for highly prevalent serious diseases. With core operations in California and North Carolina, Kriya's technology-enabled platform is directed to the rational design and clinical translation of gene therapies for large patient populations. For more information, please visit http://www.kriyatx.com.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements pertaining to the usage and capabilities of our manufacturing facility, our costs, and the potential of our platform. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. The forward-looking statements contained in this press release reflect Kriya's current views with respect to future events, and Kriya does not undertake and specifically disclaims any obligation to update any forward-looking statements.

Contact Daniel Chen Chief Financial Officer [emailprotected]

SOURCE Kriya Therapeutics

https://www.kriyatx.com/

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Kriya Therapeutics announces the establishment of its internal manufacturing facility for process development and scalable cGMP production of gene...

NEW YORK--(BUSINESS WIRE)--IVERIC bio, Inc. (Nasdaq: ISEE) today announced financial and operating results for the fiscal quarter ended June 30, 2020 and provided a general business update.

Following the positive results from our GATHER1 Phase 3 clinical trial of Zimura for the treatment of geographic atrophy secondary to age-related macular degeneration, our main priority is to aggressively drive patient recruitment and retention in GATHER2, our second Phase 3 clinical trial for the treatment of GA secondary to AMD, stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. We believe GATHER1 is currently the only Phase 3 clinical trial showing early suppression of GA growth which continued for 18 months with continuous treatment. The enthusiasm, resiliency and dedication of patients, physicians and their staffs fueled by their confidence in the GATHER1 Phase 3 results are exceeding our expectations.

Mr. Sblendorio added, During the second quarter, we strengthened our balance sheet with a public offering and a concurrent private placement. We believe this enables us to further execute on our strategy to develop and deliver retinal treatments through our Zimura, gene therapy and HtrA1 inhibitor programs, with the potential to create long-term shareholder value.

Age-Related Macular Degeneration Programs

Zimura (avacincaptad pegol): Complement C5 Inhibitor

IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

Corporate UpdateIn April 2020, the Company appointed Pravin U. Dugel, MD as Executive Vice President and Chief Strategy and Business Officer. In July 2020, Mark S. Blumenkranz, MD, MMS, joined its board of directors.

Second Quarter 2020 Operational Update and Cash GuidanceAs of June 30, 2020, the Company had $245.7 million in cash and cash equivalents. In June 2020, the Company raised approximately $150 million in net proceeds in an underwritten public offering of common stock, and pre-funded warrants in lieu of common stock, and a concurrent private placement of common stock. The Company now estimates that its year-end 2020 cash and cash equivalents will range between $215 million and $220 million. The Company also estimates that its cash and cash equivalents will be sufficient to fund its currently planned capital expenditure requirements and operating expenses, excluding any potential approval or sales milestones payable to Archemix Corp. or any commercialization expenses for Zimura, through at least mid-2024. These estimates are based on the Companys current business plan, which includes the continuation of the Companys clinical development programs for Zimura, the progression of the Companys IC-100 and IC-200 programs into the clinic, and the advancement of the Companys IC-500 development program. These estimates assume that the Company will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies, commencement of any new sponsored research programs, or any associated develop that the Company may pursue.

2020 Q2 Financial Highlights

Conference Call/Web Cast InformationIVERIC bio will host a conference call/webcast to discuss the Companys financial and operating results and provide a business update. The call is scheduled for August 5, 2020 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-220-8451 (USA) or 323-794-2588 (International), passcode 2738321. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the IVERIC bio website at http://www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA), passcode 2738321.

About IVERIC bioIVERIC bio is a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. The Company is currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases. Vision is Our Mission. For more information on the Company, please visit http://www.ivericbio.com.

Forward-looking StatementsAny statements in this press release about the Companys future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Companys strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words anticipate, believe, estimate, expect, intend, goal, may, might, plan, predict, project, seek, target, potential, will, would, could, should, continue, and similar expressions. In this press release, the Companys forward looking statements include statements about the impact of the COVID-19 pandemic on the Companys research and development programs, operations and financial position, its expectations regarding patient enrollment and patient retention in its second Phase 3 trial (GATHER2) of Zimura in geographic atrophy secondary to AMD and to use the results of its completed clinical trial of Zimura for the treatment of geographic atrophy secondary to AMD (GATHER1) as a Phase 3 trial, expectations regarding patient enrollment and patient retention in its Phase 2b screening trial of Zimura for autosomal recessive Stargardt disease, its development and regulatory strategy for Zimura and its other product candidates, the implementation of its business plan, the projected use of cash and cash balances, the timing, progress and results of clinical trials and other research and development activities and regulatory submissions, the potential utility of its product candidates, and the potential for its business development strategy. Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the progression and duration of the COVID-19 pandemic and responsive measures thereto and related effects on the Companys research and development programs, operations and financial position, the initiation and the progress of research and development programs and clinical trials, availability of data from these programs, reliance on contract development and manufacturing organizations, university collaborators and other third parties, establishment of manufacturing capabilities, expectations for regulatory matters, need for additional financing and negotiation and consummation of business development transactions and other factors discussed in the Risk Factors section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Companys views only as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. While the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so except as required by law.

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IVERIC bio Reports Second Quarter 2020 Operational Highlights and Financial Results - Business Wire

The report gives a complete investigation of the Cancer Gene Therapy Market and key improvements. The exploration record comprises of past and figure showcase data, prerequisite, territories of use, value strategies, and friends portions of the main organizations by topographical district. The Cancer Gene Therapy report separates the market size, by volume and worth, depending upon the kind of utilization and area.

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With everything taken into account, the Cancer Gene Therapy report offers inside and out profile and information data life structures of driving Cancer Gene Therapy organizations.

Top Companies Name: Urigen Pharmaceuticals Inc. (U.S), GenVec.Inc (U.S), Oxford BioMedica (U.K), Vical (U.S), ANI Pharmaceuticals, Inc. (U.S), and Genzyme Corporation (U.S). Novartis AG and Others.

Global Cancer Gene Therapy Market by Geography: Latin America, North America, Asia Pacific, the Middle East and Africa and Europe.

Types:-

Retroviral Therapy

Adenoviral Therapy

Others

Applications:-

Hospitals

Oncology institutes

Biotechnological companies

Others

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The Cancer Gene Therapy report presents a point by point estimation of the market through complete appraisal, fantastic experiences, and bona fide expectations managing the Cancer Gene Therapy market size. It depends on attempted and tried methodologies alongside convictions in the event of the estimate made accessible. In this manner the nitty gritty investigation of Cancer Gene Therapy market fills in as a repository of examination and information for each part of the market, especially concerning nearby markets, innovation, classifications, and use.

The report involves the estimation of the Global Cancer Gene Therapy Market. The accompanying Industry is appeared to advance with a critical ascent in the Compound Annual Growth Rate (CAGR) during the conjecture time frame attributable to different elements driving the market.

The key points of the report:

The extent of the report stretches out from market situations to similar valuing between significant players, cost and benefit of the predetermined market areas. The numerical information is upheld up by factual apparatuses, for example, SWOT investigation, Porters Five Analysis, PESTLE examination, etc.

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Cancer Gene Therapy Market Latest Treatment Methodology 2020 to 2025 - Owned

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 04 August 2020 16:01Hits: 455

ROCKVILLE, MD, USA I August 04, 2020 I REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAVTechnology Platform, today reported positive one year data from patients in Cohorts 4 and 5 of the Phase I/IIa trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). The Company plans to initiate a pivotal program for subretinal delivery of RGX-314 in patients with wet AMD by the end of 2020. In addition, REGENXBIO today announced that a Phase II trial of RGX-314 for the treatment of wet AMD delivered to the suprachoroidal space (AAVIATE) is active and expected to enroll patients in the third quarter of 2020.

"Today's results provide further evidence of the clinical profile of RGX-314 as a promising one-time gene therapy treatment paradigm for patients with wet AMD," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "The data demonstrated stable to improved visual acuity and retinal thickness, as well as a meaningful reduction in anti-VEGF injection burden, in these higher dose levels at one year. Results from the Phase I/IIa trial will inform the design of the pivotal program of RGX-314 in patients with wet AMD, which we look forward to initiating by the end of this year."

Dr. Pakola continued: "I am also pleased to announce that our AAVIATE trial, a Phase II trial of RGX-314 for the treatment of wet AMD utilizing the SCS Microinjector, is active and we expect to dose the first patient this quarter. The targeted, in-office suprachoroidal delivery approach may provide additional options for patients in all settings of patient care. We anticipate providing an interim data update from the first cohort in late 2020."

"Based on the overall results to date from the Phase I/IIa trial, I believe that RGX-314 has the potential to profoundly impact all aspects of clinical management for patients with wet AMD," said Robert Avery, M.D., Founder of California Retina Consultants and Research Foundation and investigator surgeon in the Phase I/IIa RGX-314 trial. "Wet AMD affects a large number of adults, and often results in loss of vision over time due to non-compliance with the current standard of care of frequent anti-VEGF injections. I am encouraged that RGX-314 has the potential to become a one-time gene therapy treatment option for a broad range of patients."

Study Design and Safety Update from Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD using Subretinal Delivery

In the Phase I/IIa trial of RGX-314, 42 patients with long-standing severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3x109 GC/eye to 2.5x1011 GC/eye. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic or supplemental immune suppressive corticosteroid therapy for RGX-314.

Patients in the study are being assessed each month for two years and will receive safety follow-up for five years after RGX-314 administration. Efficacy assessments for the study include number of anti-VEGF intravitreal injections, change in vision as measured by Best Corrected Visual Acuity (BCVA), change in central retinal thickness (CRT) as measured by spectral domain optical coherence tomography (SD-OCT), and RGX-314 protein expression levels as measured from aqueous samples by electrochemiluminescence immunoassay (ECL).

As of July 13, 2020, RGX-314 was generally well-tolerated across all cohorts. Eighteen serious adverse events (SAEs) were reported in 11 patients, including 17 that were not related to RGX-314. One possibly drug-related SAE of significant decrease in vision was reported in Cohort 5 at Month 11 in a patient who had retinal pigmentary changes that involved the macula. The most common nonserious adverse events in the eye were generally assessed as mild (77%). These included post-operative conjunctival hemorrhage (69% of patients), post-operative inflammation (36% of patients), eye irritation (17% of patients), eye pain (17% of patients), and post-operative visual acuity reduction (17% of patients). In 67% of patients across all cohorts, and in 83% of patients in Cohorts 3 through 5, retinal pigmentary changes were observed on imaging, the majority of which were in the peripheral inferior retina. Retinal hemorrhage was observed in 24% of patients and is an anticipated event in patients with severe wet AMD. There have been no reports of clinically determined immune responses, drug-related ocular inflammation, or post-surgical inflammation beyond what is expected following routine vitrectomy.

Summary of Data from Cohorts 4 and 5 of Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD using Subretinal Delivery

Today's update includes data from Cohorts 4 and 5 as of July 13, 2020. Each cohort enrolled 12 patients each at doses of 1.6x1011 GC/eye and 2.5x1011 GC/eye, respectively.

Patients in Cohort 4 and Cohort 5 at one year after administration of RGX-314 demonstrated stable visual acuity with a mean BCVA change of +4 letters and -2 letters from baseline, respectively, as well as decreased retinal thickness, with a mean change in CRT of -61 m and -79 m, respectively.

There was a clinically significant and meaningful reduction in anti-VEGF treatment burden in both Cohorts 4 and 5 compared to the 12 months prior to RGX-314 administration. Patients in Cohort 4 received a mean of 4.1 injections over one year following administration of RGX-314, a 61% reduction in treatment burden. Patients in Cohort 5 received a mean of 1.4 injections over one year following administration of RGX-314, a reduction in treatment burden of 85%.

In Cohort 4, three out of twelve (25%) patients received no anti-VEGF injections over one year, and these patients demonstrated a mean BCVA improvement of +6 letters and a mean reduction in CRT of -62 m at one year. In Cohort 5, eight out of the eleven (73%) patients observed through one year have received no anti-VEGF injections after administration of RGX-314 and these patients demonstrated a stable mean BCVA change of 0 letters and a mean reduction in CRT of -95 m at one year.

Consistent with previous results, intraocular RGX-314 protein expression levels were observed in a dose-dependent manner across each cohort at one year after administration of RGX-314. The mean protein expression levels in Cohort 4 and Cohort 5 were 420.9 ng/ml and 457.5 ng/ml, respectively.

Study Design for Phase II Trial for RGX-314 for Treatment of Wet AMD using Suprachoroidal Delivery (AAVIATE)

REGENXBIO also announced that a Phase II trial, AAVIATE, to evaluate the suprachoroidal delivery of RGX-314 in patients with wet AMD, will begin dosing patients in the third quarter of 2020. AAVIATE is a multi-center, open-label, randomized, active-controlled, dose-escalation study that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 using the SCS Microinjector, a targeted, in-office route of administration.

AAVIATE will enroll 40 patients with severe wet AMD who are responsive to anti-VEGF treatment. Patients will be randomized to one-time RGX-314 SCS delivery versus monthly 0.5 mg ranibizumab intraocular injection at a 3:1 ratio and two dose levels of RGX-314 will be evaluated: 2.5x1011 GC/eye and 5x1011 GC/eye.Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.

The primary endpoint of the study is mean change in vision, as measured by BCVA, at 40 weeks from baseline compared to monthly ranibizumab. Other endpoints include mean change in CRT and number of anti-VEGF intravitreal injections.

The Company expects to report interim data from the first cohort of this trial by the end of 2020.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other additional chronic retinal conditions treated with anti-VEGF. RGX-314 consists of the NAV AAV8 vector encoding an antibody fragment which is designed to inhibit VEGF, modifying the pathway for formation of new leaky blood vessels which lead to retinal fluid accumulation and vision loss.

About the Phase I/IIa Clinical Trial of RGX-314

RGX314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, doseescalation study in adult patients with wet AMD in the United States. The study includes patients previously treated for wet AMD who are responsive to anti-VEGF therapy. The study is designed to evaluate five escalating doses of RGX-314, with six patients in the first three dose cohorts and 12 patients in the fourth and fifth dose cohorts. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the study is safety at 6 months following administration of RGX-314. Secondary endpoints include visual acuity, retinal thickness on SDOCT, ocular RGX-314 protein expression, and the need for additional anti-VEGF therapy. Following completion of the primary study period, patients enter a follow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

AboutREGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

SOURCE: REGENXBIO

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REGENXBIO Announces Additional Positive Interim Phase I/IIa Trial Update and Program Updates for RGX-314 for the Treatment of Wet AMD | DNA RNA and...

CAMBRIDGE, Mass., Aug. 03, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced the termination of its tau and alpha-synuclein vectorized antibody collaborations with AbbVie. Voyager retains full rights to the vectorization technology and certain novel vectorized antibodies developed as part of the collaborations.

Our efforts to harness AAV-based gene therapy to produce antibodies directly in the brain and overcome major limitations with delivery of current biologics across the blood-brain barrier have been highly productive, said Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head of R&D at Voyager. Through the tau and alpha-synuclein collaborations, we believe we have made considerable progress against targets for neurodegenerative diseases with this novel approach, reinforcing our enthusiasm for its potential to deliver therapeutically efficacious levels of biologics to the brain and central nervous system. We believe our continued work on discovery and design of novel AAV capsids with substantially improved blood-brain barrier penetrance will also considerably broaden the potential of AAV-based gene therapy, including vectorized antibodies or other biologics, for the treatment of severe neurological diseases.

The tau and alpha-synuclein research collaborations were formed in 2018 and 2019, respectively. Under the terms of the collaboration agreements, Voyager received upfront payments to perform research and preclinical development of vectorized antibodies directed against tau and alpha-synuclein. With the conclusion of the collaborations, Voyager has regained full clinical development and commercialization rights to certain product candidates developed within the context of the collaboration for the tau program. Voyager is free to pursue vectorized antibody programs for tau and alpha-synuclein alone or in collaboration with another partner.

Voyager does not anticipate any changes to its cash runway guidance due to the termination of the agreements. As of March 31, 2020, the Company had cash, cash equivalents and marketable debt securities of $250.9 million, which, along with amounts expected to be received for reimbursement of development costs from Neurocrine Biosciences, is expected to be sufficient to meet Voyagers projected operating expenses and capital expenditure requirements into mid-2022.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information, please visit http://www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, plan, anticipate, believe, estimate, undoubtedly, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the ability of Voyager to maintain research and development activities currently included within the collaboration agreements with AbbVie; Voyagers ability to advance its AAV-based gene therapies and its ability to continue to develop its gene therapy platform; the scope of the intellectual property rights and other rights that will be available to Voyager following the termination of the AbbVie collaboration agreements; the anticipated effects of the termination of the AbbVie collaboration agreements on Voyagers anticipated financial results, including Voyagers available cash, cash equivalents and marketable debt securities; and Voyagers ability to fund its operating expenses with its current cash, cash equivalents and marketable debt securities through a stated time period are forward looking. All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued cooperation of AbbVie in activities arising from the termination of the AbbVie collaboration agreements, the development of the gene therapy platform; Voyagers scientific approach and general development progress; Voyagers ability to create and protect its intellectual property; and the sufficiency of Voyagers cash resources. These statements are also subject to a number of material risks and uncertainties that are described in Voyagers most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investors:Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media:Sheryl SeapyW2Opure949-903-4750sseapy@purecommunications.com

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Voyager Therapeutics Provides Update on AbbVie Vectorized Antibody Collaborations - GlobeNewswire

By David Fierro and Matthew Brown

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of Greenwich's own Julian Fraser, who lost his life battling a rare form of cancer, raised over $45,000 for cancer research. Over 50 swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, Including Julian mother, Cristy Fraser, kept watch over the group during the benefit swim.

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of

Photo: Matthew Brown / Hearst Connecticut Media

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of Greenwich's own Julian Fraser, who lost his life battling a rare form of cancer, raised over $45,000 for cancer research. Over 50 swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, Including Julian mother, Cristy Fraser, kept watch over the group during the benefit swim.

Meghan Lynch participates in the "Buoy-to-Buoy" swim at Rocky Point Club in Old Greenwich, Connecticut on August 1, 2020. The Swim Across America Event organized by Team Julian, honoring the legacy of

Greenwich swimmers take the plunge to fight cancer

GREENWICH A team of over 50 swimmers and 15 volunteers joined forces to raise more than $45,000 in the fight against cancer and to honor a 2014 Greenwich High graduate and standout swimmer who died of osteosarcoma.

The group known as Team Julian team gathered Saturday at Rocky Point Club in Old Greenwich for the endurance swim on Long Island Sound to benefit Swim Across America Fairfield County.

The event honored the legacy of Julian Fraser, who lost his battle with a rare form of bone cancer in 2017 at the age of 20.

The swimmers from Rocky Point Club and Greenwich High School's Swim and Water Pola teams participated in the 1-mile swim in the Long Island Sound. Several volunteers in kayaks and on paddle boards, including Julian Frasers mother, Cristy Fraser, kept watch over the group during the long benefit swim.

Julian Fraser earned All-America honors as a swimmer and water polo player. He competed on numerous championship teams for GHS coach Terry Lowe, then excelled on the Santa Clara University water polo team.

We are united to raise money for the fight against cancer and to honor Julian, said Lowe, who is one of the organizers of Team Julian.

Proceeds from the event supports the Stamford-based Alliance for Cancer Gene Therapy, the nations only foundation dedicated exclusively to funding cancer gene therapy research.

To learn more about Swim Across America Fairfield County or make a donation, visit swimacrossamerica.org/fc.

dfierro@greenwichtime.com

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Greenwich swimmers take the plunge to fight cancer - Greenwich Time

Novartis has agreed to license Sangamo Therapeutics zinc finger protein transcription factors (ZFP-TFs) to develop gene regulation therapies for neurodevelopmental disorders that include autism spectrum disorder and intellectual disability, the companies said, through a collaboration that could generate more than $795 million for Sangamo.

Through Sangamos ZFP-TFs, the companies plan over three years to develop treatments addressing three target genes associated with neurodevelopmental disorders, by upregulating the expression of key genes that are inadequately expressed in individuals with certain neurodevelopmental disorders.

The goal is to create new gene regulation therapies that act at the genomic level, moving us beyond the symptom focused treatments of today and toward therapies that can address some of the most challenging neurodevelopmental disorders, Jay Bradner, president of the Novartis Institutes for BioMedical Research, said in a statement. This collaboration with Sangamo is part of our commitment to pioneering the next generation of neurodevelopmental treatments.

Sangamos ZFP-TF genome regulation technology, now delivered via adeno-associated viruses (AAVs), is designed to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect, at the DNA level.

While AAVs have many advantages that make them well-suited for gene therapy, Novartis says, they also have one disadvantage: They cant carry large genes. The collaboration is designed to enable the companies to target diseases caused by mutations in one copy of a large gene.

The gene for a ZFP-TF is small enough to fit inside an AAV, Ricardo Dolmetsch, head of neuroscience at the Novartis Institutes for BioMedical Research, explained in a post on the companys blog. We can use it to increase the production of a large gene in someone who still has one intact copy of the gene. This dramatically expands the range of diseases that we can potentially target with gene therapy because many diseases are caused by the loss of a single copy of a gene.

Each of Sangamos ZFP-TFs is engineered to bind to a target region of genomic DNA in a highly specific and selective manner. They can be designed to precisely modulate the expression of targeted genes to varying extents. After identifying its target, the ZFP-TF recruits other proteins that help switch genes on or off.

The zinc finger nuclease technology is remarkable, Macrae told GEN in January. Its one of the commonest transcripts in the body. Its natural and human, and its very, very adaptable. The individual units are modular, so one is always able to come up with a solution for any part of the genome.

For its part, Sangamo reasons that it can engineer zinc finger proteins to address virtually any genomic target.

We are building a broad pipeline of wholly owned and partnered programs with the goal to bring our genomic medicines to patients, stated Sangamo CEO Sandy Macrae. In the case of the central nervous system, there are potentially hundreds of neurological disease gene targets that may be addressable by our zinc finger platform.

For Sangamo, the collaboration with Novartis is the second signed this year that focuses on applying ZFP-TFs toward treating neurological disorders. In February, Sangamo launched a potentially more than $2.7 billion partnership to develop and commercialize Sangamo gene regulation therapies. The collaboration included ST-501 for tauopathies including Alzheimers disease, ST-502 for synucleinopathies including Parkinsons disease, a third treatment targeting an undisclosed neuromuscular disease target, and additional treatments for up to nine additional undisclosed neurological disease targets over five years.

In its latest partnership with Novartis, the Swiss pharma giant will have exclusive rights to ZFP-TFs targeting the genes, which are undisclosed, during the three-year collaboration period.

Novartis also has the option to license Sangamos AAVs. Sangamo has agreed to oversee specified research and associated manufacturing activities, all of which will be funded by Novartiswhile Novartis agreed to oversee additional research activities, investigational new drug-enabling studies, clinical development, related regulatory interactions, manufacturing, and global commercialization.

Novartis agreed to pay Sangamo a $75 million upfront license fee within 30 days, plus up to $720 million tied to achieving development and commercial milestonesconsisting of up to $420 million in development milestones and up to $300 million in commercial milestones.

Sangamo is also eligible to receive from Novartis tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration.

Partnering Sangamos proprietary technology with Novartis deep experience in neuroscience drug development is a powerful combination which expands Sangamos pipeline and allows us to tackle challenging neurodevelopmental conditions, Macrae added.

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Novartis Taps Sangamo Zinc Finger Tech for Gene Regulation Therapies - Genetic Engineering & Biotechnology News

ASCO-Jonathan W. Friedberg has been appointed as the next editor-in-chief of theJournal of Clinical Oncology(JCO), the flagship journal of the American Society of Clinical Oncology (ASCO).JCOpublishes cutting-edge research on the diagnosis and treatment of patients with cancer and is one of the most highly cited oncology journals in the world. A hematologic oncologist for more than 20 years, Friedberg is currently director of the Wilmot Cancer Institute and Samuel Durand Professor of Medicine at the University of Rochester Medical Center in Rochester, New York. Friedbergs five-year term asJCOeditor-in-chief will begin in June 2021. He will succeed Stephen A. Cannistra, who will be completing his 10th year as the journals editor-in-chief.

Apic Bio Cambridge, Mass.-based Apic Bio formed a Scientific Advisory Board (SAB) which includes internationally recognized gene therapy experts. TheSAB will provide clinical and scientific expertise to help the company advance its pipeline and leverage its proprietary silence and replace THRIVE platform. The appointments to Apics Scientific Advisory Board include Robert H. Brown Jr., Leo P. and Theresa M. LaChance Chair in Medical Research, Professor of Neurology, Director of the Program in Neurotherapeutics, University of Massachusetts Medical School; R. Jude Samulski, president and chief scientific officer at AskBio; Barry Byrne, director of the Powell Gene Therapy Center at the University of Florida and Professor of Pediatrics and Molecular Genetics & Microbiology; Rob Kotin, adjunct professor at University of Massachusetts Medical School; Kevin Flanigan, Robert F. and Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research and Director of the Center for Gene Therapy in The Abigail Wexner Research Institute at Nationwide Childrens Hospital, and professor of Pediatrics and Neurology at The Ohio State University College of Medicine; Mark Kay, Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine.

I-Mab Shanghai-based I-Mab named Ivan Yifei Zhuas its chief commercial officer effectiveAug. 10. In this role, Zhu will focus on building and developing I-Mab's commercialization infrastructure and strategies and preparing the company for upcoming product launches. Before joining I-Mab, he served as vice president and General Manager of the sales division of Qilu Pharmaceutical Group where he managed the company's sales and marketing team. Zhu also served as the CCO of BeiGene where he played an instrumental role in the expansion of BeiGene's commercialization team and the implementation of its commercialization strategies.

Caladrius Biosciences Michael H. Davidson was named to the board of directors of Caladrius Biosciences. Davidson is the founder and chief scientific officer of Corvidia Therapeutics, which recently announced its sale to Novo-Nordisk for $2.1 billion. Davidson also serves as clinical professor and director of the Lipid Clinic at the University of Chicago Pritzker School of Medicine.

Cidara Therapeutics-Cidara Therapeutics announced the formation of a scientific advisory board (SAB) and the appointment of four leading experts in viral and fungal infections and hematology. TheSABwill work closely with the Cidara management team to help guide the direction and design of the companys development programs with an emphasis on the continued development and expansion of the companys Cloudbreak Antiviral Conjugate (AVC) program.Cidara TherapeuticsSABincludes Frederick G. Hayden,Stuart S. Richardson Professor Emeritus of Clinical Virology atUniversity of Virginia School of Medicine; Professor Emeritus of Medicine at the University of Virginia; Michael G. Ison, professor, Divisions of Infectious Diseases and Organ Transplantation atNorthwestern University Feinberg School of Medicine; Medical Director, Transplant & Immunocompromised Host Infectious Diseases Service at Northwestern University Comprehensive Transplant Center,Chicago; Johan A Maertens,professor of Internal Medicine and Hematology, Acute Leukemia and Transplantation Unit at University Hospitals Leuven;Department of Microbiology, Immunology and Transplantationat KULeuven, Leuven,Belgium; Kieren Marr, Professor of Medicine and Oncology Director, Transplant and Oncology Infectious Diseases Program and Vice-Chair for Innovation in Healthcare Implementation,Department of MedicineatJohns Hopkins University School of Medicine.

Alydia Health Following a $13.9 million Series C financing round, Alydia Health announced Rob Binney as its new chief executive officer. Binney joins the company from Intersect ENT where he served as chief commercial officer. Prior to Intersect ENT, Binney worked in leadership roles with both startups and large medical device companies, including Boston Scientific and AccessClosure, now part of Cardinal Health. Binney succeeds Anne Morrissey, who recently stepped down as the companys CEO. Colby Holtshouse, previously Alydias interim CEO and head of Marketing, will assume the role of chief operating officer. In association with the financing round, Zina Affas Besse, managing partner at Global Health Funds will join the companys board of directors.

Synlogic, Inc. Antoine Tony Awad was named chief operating officer of Synlogic. As COO, Awad will drive Synlogic's end to end development & manufacturing organization and supply chain as well as have broader corporate operations responsibilities, inclusive of laboratory operations, IT infrastructure, and the quality organization. Awad joined Synlogic in December 2018 as head of Technical Operations. Prior to joining Synlogic, he served as senior vice president of CMC and Operations at Abpro Therapeutics and L.E.A.F. Pharmaceuticals and served in roles of increasing responsibility at Ipsen Biosciences and Merrimack Pharmaceuticals. In addition to Awad, Synlogic also named Andrew March as head of Development Operations. Prior to joining Synlogic March served as Ra Pharmaceuticals' head of Clinical Development.

Engrail Therapeutics San Diego-based Engrail announced two key executive appointments. Kimberly Vanover has been named chief scientific officer, leading the building and advancement of the companys pipeline. Eve Taylor has been named vice president of clinical development, overseeing all facets of Engrails clinical development programs and operations. Stephen Cunningham, previously executive vice president, development and CSO, will assume the role of chief development officer. Vanover most recently served as senior vice president, early-stage clinical development and translational medicine at Intra-Cellular Therapies. Taylor most recently helped lead early-stage clinical development at Intra-Cellular Therapies. Previously, Taylor led clinical development activities at Cytori Therapeutics, Ceregene and ACADIA Pharmaceuticals. The company also expanded its board of directors to five members. Tony Ho was appointed to Engrails board of directors effective immediately. Ho served as CRISPR Therapeutics executive vice president, research and development since August 2017. Prior to joining CRISPR, Tony held a number of roles at AstraZeneca, most recently senior vice president and head of oncology integration and innovation.

CressetU.K.-based Cresset announced its SAB. The Cresset Scientific Advisory Board members are Stevan W Djuric, chairman of Discovery Chemistry and Technology Consulting LLC, USA;Richard Lewis of Novartis Pharma AG; Alexander Hillisch of Bayer; AstraZenecas Andreas Bender; Julien Michel of the University of Edinburgh; Paul Czodrowski of TU Dortmund University in Germany; and Kazuyoshi Ikeda of Keio University in Japan.

NeuBase Therapeutics William Mann was named chief operating officer of NeuBase Therapeutics. Mann will leverage his decades of experience in the biopharma industry to manage NeuBase operations and advance its strategic goals. Mann most recently served as the president and CEO of Helsinn Therapeutics. Before joining Helsinn, he held multiple positions at Sapphire Therapeutics, Inc.

Originally posted here:
Movers & Shakers, July 31 | BioSpace - BioSpace

Global Gene Therapy for Inherited Genetic Disorders market- Report defines the vital growth factors, opportunities and market segment of top players during the forecast period from 2019 to 2025. The report Gene Therapy for Inherited Genetic Disorders offers a complete market outlook and development rate during the past, present, and the forecast period, with concise study, Gene Therapy for Inherited Genetic Disorders market effectively defines the market value, volume, price trend, and development opportunities. The comprehensive, versatile and up-to-date information on Gene Therapy for Inherited Genetic Disorders market is provided in this report.

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The key players covered in this studyBioMarin Pharmaceutical Inc.bluebird bio Inc.Novartis AGOrchard Therapeutics PlcSpark Therapeutics Inc.

Market segment by Type, the product can be split intoEye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthersMarket segment by Application, split intoHospitalClinicResearch InstituteOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

The study objectives of this report are:To analyze global Gene Therapy for Inherited Genetic Disorders status, future forecast, growth opportunity, key market and key players.To present the Gene Therapy for Inherited Genetic Disorders development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by type, market and key regions.

In this study, the years considered to estimate the market size of Gene Therapy for Inherited Genetic Disorders are as follows:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year 2020 to 2026For the data information by region, company, type and application, 2019 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Gene Therapy for Inherited Genetic Disorders Market size in terms of volume and value 2019-2025 - Market Research Correspondent

MALVERN, Pa., July 27, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the third Orphan Drug Designation (ODD) for OCU400 in the treatment of RHO mutation-associated retinal degeneration. The RHO mutation is part of the Retinitis Pigmentosa (RP) group of rare, genetic disorders that involve a breakdown and loss of cells in the retina and can lead to visual impairment and blindness. This is one of the larger mutations within the RP class, representing about 12% of RP patients in the US.

A novel gene therapy product candidate, OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (NHR) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis and potentially offer longer benefit, stabilizing cells and rescuing photoreceptor degeneration and vision loss.

Adding to ODDs for OCU400 for NR2E3 and CEP290 mutation-associated retinal degeneration, RHO gene mutation-associated retinal degeneration further supports Ocugens breakthrough modifier gene therapy platforms potential to treat multiple blindness diseases with a single product. RP is a group of heterogenic inherited retinal diseases associated with over 150 gene mutations, affecting over 1.5 million individuals worldwide. In addition, ~40% of RP patients cannot be genetically diagnosed, confounding the ability to develop personalized RP therapies. Traditional gene therapy or gene editing approaches may require more than 150 products to rescue these patients from vision loss. OCU400, a single product candidate, has potential to address broad-spectrum RP.

OCU400, comprising the nuclear hormone receptor geneNR2E3, has the potential to help modulate numerous biological pathways that function in maintaining the health of the retina. A recent preclinical study published in Nature Gene Therapy demonstrated the potency of NR2E3 to elicit broad-spectrum therapeutic benefits in early and intermediate stages of RP in five unique mouse models, said Dr. Mohamed Genead, acting Chief Medical Officer of Ocugen and Chair of Ocugens Retina Scientific Advisory Board. We believe OCU400 has the potential to address multiple genetic mutations associated with RP and, therefore, help a broader pool of patients, Dr. Genead continued.

Our third ODD for OCU400 from the FDA is an important step towards developing a broad-spectrum treatment for RP and getting a therapy faster to patients who are in desperate need of rescue, said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen. Orphan designation for this indication supports the goal of our Modifier Gene Therapy Platform to treat a variety of inherited retinal diseases with a single gene therapy product. There are currently no approved treatments which slow or stop the progression of multiple forms of RP, which is why were excited to have a platform that can potentially address multiple mutations, including mutations in the Rhodopsin gene, with one therapy.

The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including a seven-year period of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.

About OCU400OCU400 (AAV-hNR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration and vision loss.

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. For more information, please visithttps://ocugen.com/

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact: LaVoieHealthScienceEmmie Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042

Originally posted here:
Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative...

Hemophilia Gene Therapy Market 2020 Research Report provides Emerging Market trends, Market Segmentation, regional outlook and comprehensive analysis on different market segments. It also provides key analysis on the market status of the Hemophilia Gene Therapy manufacturers with best facts and figures, meaning, definition, SWOT analysis, expert opinions and the latest developments across the globe.This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets.

Final Report will add the analysis of the impact of COVID-19 on this industry.

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Short Description About Hemophilia Gene Therapy Market :

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is a monogenic disease (a disease that is caused by a genetic defect in a single gene). There are two types of hemophilia caused by mutations in genes that encode protein factors which help the blood clot and stop bleeding when blood vessels are injured. Individuals with hemophilia experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. The most frequent forms of hemophilia affect males.

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The research covers the current Hemophilia Gene Therapy market size of the market and its growth rates based on 5-year records with company outline ofKey players/manufacturers:

Scope of the Hemophilia Gene Therapy Market Report:

About 80% of them have hemophilia A, which affects the clotting factor VIII. The second most common form, hemophilia B, is due to a deficiency of the clotting factor IX. Several biotechs are racing to launch the first gene therapy for hemophilia. Currently, uniQure in the Netherlands and Spark Therapeutics in the US have the most advanced programs. Spark scored a victory in December when it presented Phase I/II for its candidate SPK-9001. The gene therapy was able to reduce annual bleeding episodes by 97%, as compared to its competitor uniQures candidate, AMT-060. However, uniQure has fought back. The company added a modification in its gene therapy that is known to increase clotting activity by 8- to 9- fold. This improved version has already been cleared to start a Phase III clinical trial in both Europe and the US.

Hemophilia B has traditionally been the main focus of biotechs, since most big pharma efforts were focused on the bigger hemophilia A market. In addition, applying gene therapy to hemophilia A is more challenging; The gene coding for the factor IX protein missing in hemophilia B is simply smaller than that for factor VIII missing in hemophilia A, and therefore easier to fit in the viral vectors used for gene delivery. US-based BioMarin is leading the development of a gene therapy. To overcome the size limit, the company has deleted a region from the factor VIII protein that is not necessary for clotting.

Hemophilia treatment is currently in the pre-clinical stage. And the multiple treatments that are underway might significantly improve the quality of life of patients with hemophilia, by getting rid of frequent infusions and hospital visits, and transitioning patients from severe to mild hemophilia.

The global Hemophilia Gene Therapy market is valued at xx million USD in 2018 and is expected to reach xx million USD by the end of 2024, growing at a CAGR of xx% between 2019 and 2024.

The Asia-Pacific will occupy for more market share in following years, especially in China, also fast growing India and Southeast Asia regions.

North America, especially The United States, will still play an important role which cannot be ignored. Any changes from United States might affect the development trend of Hemophilia Gene Therapy.

Europe also play important roles in global market, with market size of xx million USD in 2019 and will be xx million USD in 2024, with a CAGR of xx%.

This report studies the Hemophilia Gene Therapy market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Hemophilia Gene Therapy market by product type and applications/end industries.

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Report further studies the market development status and future Hemophilia Gene Therapy Market trend across the world. Also, it splits Hemophilia Gene Therapy market Segmentation by Type and by Applications to fully and deeply research and reveal market profile and prospects.

Major Classifications are as follows:

Major Applications are as follows:

Geographically, this report is segmented into several key regions, with sales, revenue, market share and growth Rate of Hemophilia Gene Therapy in these regions, from 2014 to 2024, covering

This Hemophilia Gene Therapy Market Research/Analysis Report Contains Answers to your following Questions

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Major Points from Table of Contents:

1. Market Overview1.1 Hemophilia Gene Therapy Introduction1.2 Market Analysis by Type1.3 Market Analysis by Applications1.4 Market Dynamics1.4.1 Market Opportunities1.4.2 Market Risk1.4.3 Market Driving Force

2.Manufacturers Profiles

2.4.1 Business Overview2.4.2 Hemophilia Gene Therapy Type and Applications2.4.2.1 Product A2.4.2.2 Product B

3.Global Hemophilia Gene Therapy Sales, Revenue, Market Share and Competition By Manufacturer (2019-2020)

3.1 Global Hemophilia Gene Therapy Sales and Market Share by Manufacturer (2019-2020)3.2 Global Hemophilia Gene Therapy Revenue and Market Share by Manufacturer (2019-2020)3.3 Market Concentration Rates3.3.1 Top 3 Hemophilia Gene Therapy Manufacturer Market Share in 20203.3.2 Top 6 Hemophilia Gene Therapy Manufacturer Market Share in 20203.4 Market Competition Trend

4.Global Hemophilia Gene Therapy Market Analysis by Regions

4.1 Global Hemophilia Gene Therapy Sales, Revenue and Market Share by Regions4.1.1 Global Hemophilia Gene Therapy Sales and Market Share by Regions (2014-2019)4.1.2 Global Hemophilia Gene Therapy Revenue and Market Share by Regions (2014-2019)4.2 North America Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.3 Europe Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.4 Asia-Pacific Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.6 South America Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)4.6 Middle East and Africa Hemophilia Gene Therapy Sales and Growth Rate (2014-2019)

5.Hemophilia Gene Therapy Market Forecast (2020-2024)5.1 Global Hemophilia Gene Therapy Sales, Revenue and Growth Rate (2020-2024)5.2 Hemophilia Gene Therapy Market Forecast by Regions (2020-2024)5.3 Hemophilia Gene Therapy Market Forecast by Type (2020-2024)5.3.1 Global Hemophilia Gene Therapy Sales Forecast by Type (2020-2024)5.3.2 Global Hemophilia Gene Therapy Market Share Forecast by Type (2020-2024)5.4 Hemophilia Gene Therapy Market Forecast by Application (2020-2024)5.4.1 Global Hemophilia Gene Therapy Sales Forecast by Application (2020-2024)5.4.2 Global Hemophilia Gene Therapy Market Share Forecast by Application (2020-2024)

6.Sales Channel, Distributors, Traders and Dealers6.1 Sales Channel6.1.1 Direct Marketing6.1.2 Indirect Marketing6.1.3 Marketing Channel Future Trend6.2 Distributors, Traders and Dealers

7.Research Findings and Conclusion

8.Appendix8.1 Methodology8.2 Data Source

Continued..

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Hemophilia Gene Therapy Market 2020 : Industry Outlook with Top Countries Data, Market Share, Growth Analysis, Opportunities and Forecast To 2024 -...

The Viral Vector and Plasmid DNA Manufacturing Market report 2020 covers all the significant developments which are recently being adopted across the global market. The prime objective of the Viral Vector and Plasmid DNA Manufacturing market report is to provides an in-depth analysis of all market dynamics including drivers and restraints, and trends, and opportunities. The Viral Vector and Plasmid DNA Manufacturing market report covers both the demand and supply aspects of the market. The report also highlighted the future trends in the Viral Vector and Plasmid DNA Manufacturing market that will impact the demand during the forecast period.

Scope of the Report:

As per the scope of this report, viral vectors and plasmid DNA are the products of gene therapy, which are used for the treatment of several diseases, such as Alzheimers disease, Parkinsons disease, and rheumatoid arthritis. Gene therapies and other therapies that require genetic modification include the introduction of therapeutic DNA/gene into a patients body or cell. This process is accomplished by the use of vectors. Over the past few decades, various viral and non-viral vectors have been developed and standardized for this purpose. Moreover, viral vectors and plasmid DNA are known to reduce the cost of treatment and help in decreasing repeated administrations of medications

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Market Overview:

Some of the Top Key Players of Viral Vector and Plasmid DNA Manufacturing Market Report Are:

Report Highlights:

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Key Market Trends:

Plasmid DNA Segment is Expected to Register Robust Growth.

A plasmid DNA is a small DNA molecule within a cell that is different from the chromosomal DNA, which can replicate independently. Plasmid DNA is currently gaining increasing importance, due to the clinical research applications in gene therapy and genetic vaccination. The demand and therapeutic applications for plasmid DNA (pDNA) have rapidly grown and expanded. Plasmid DNA manufacturing is used for indirect and direct clinical applications. For the direct gene transfer into the human body, good manufacturing practice (GMP)-grade plasmid DNA is being required. The plasmid DNA manufacturing is invariably outsourced to the specialized manufacturers using the in-house platform processes.

North America Dominates the Market in Terms of Revenue Generated

Currently, North America is witnessing a rapid growth in the market studied, followed by Europe. The United States has been a major destination for all the recent advancements in the field of medicine. Increasing investments in gene therapy products in the United States have led to the growth of the market studied in recent years. In the United States, regulatory encouragement and patient advocacy have pushed rare disease clinical research to the center stage. The significant incentives offer through Orphan Drugs Act (United States) and the Orphan Medicinal Products Regulations (European Union) have encouraged pharmaceutical and biotechnology companies to consider the development of rare disease medicines as a potentially profitable endeavour.

The Report Covers:

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Detailed TOC of Viral Vector and Plasmid DNA Manufacturing Market Report 2020-2024:

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Rising Prevalence of Genetic Disorders, Cancer, and Infectious Diseases4.2.2 Increasing Number of Clinical Studies and Availability of Funding for Gene Therapy Development4.2.3 Potential Applications in Novel Drug Delivery Approaches4.3 Market Restraints4.3.1 High Cost of Gene Therapies4.3.2 Challenges in Viral Vector Manufacturing Capacity4.4 Porters Five Force Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Product Type5.1.1 Plasmid DNA5.1.2 Viral Vector5.1.3 Non-viral Vector5.2 By Application5.2.1 Cancer5.2.2 Genetic Disorder5.2.3 Infectious Disease5.2.4 Other Diseases5.3 Geography5.3.1 North America5.3.1.1 US5.3.1.2 Canada5.3.1.3 Mexico5.3.2 Europe5.3.2.1 Germany5.3.2.2 UK5.3.2.3 France5.3.2.4 Italy5.3.2.5 Spain5.3.2.6 Rest of Europe5.3.3 Asia-Pacific5.3.3.1 China5.3.3.2 Japan5.3.3.3 India5.3.3.4 Australia5.3.3.5 South Korea5.3.3.6 Rest of Asia-Pacific5.3.4 Middle East & Africa5.3.4.1 GCC5.3.4.2 South Africa5.3.4.3 Rest of Middle East & Africa5.3.5 South America5.3.5.1 Brazil5.3.5.2 Argentina5.3.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Brammer Bio6.1.2 Cobra Biologics6.1.3 Cell and Gene Therapy Catapult6.1.4 FinVector Vision Therapies6.1.5 Fujifilm Diosynth Biotechnologies6.1.6 MassBiologics6.1.7 SIRION Biotech6.1.8 Merck KGaA Inc.6.1.9 Thermo Fisher Scientific6.1.10 Uniqure NV

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

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Viral Vector and Plasmid DNA Manufacturing Market Report 2020: Global Industry Size Analysis by Value Chain, SWOT Analysis, Drivers and Opportunities...

The Gene Therapy Market market report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

It incorporates Gene Therapy Market market evolution study, involving the current scenario, growth rate (CAGR), and SWOT analysis. Important the study on Gene Therapy Market market takes a closer look at the top market performers and monitors the strategies that have enabled them to occupy a strong foothold in the market. Apart from this, the research brings to light real-time data about opportunities that will completely transform the trajectory of the business environment in the coming years to 2025. Some of the key players in the global Gene Therapy Market market is Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

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Gene Therapy Market Market: Competitive Landscape:

The competitive landscape further includes details about different players and their position on a global and a local level is also explained in detail in this compiled study. These insights were prepared through mapping business strategies and products that offer high revenue generation capacities. An overview of the Gene Therapy Market market Comprehensive analysis of the market Analyses of recent developments in the market Events in the market scenario of the past few years Emerging market segments and regional Gene Therapy Market markets Segmentations up to the second and/or third level Historical, current, and estimated market size in terms of value and volume Competitive analysis, with company overview, products, revenue, and strategies. An impartial assessment of the market Strategic recommendations to help companies increase their Gene Therapy Market market presence.

The Key Manufacturers covered in this Report:- Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

By Therapy Type:-SomaticGermlineBy Vector Type:-

non-viral Vectorsviral vectorsBy Application:-

Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular Diseases Others

The key regions covered in the Gene Therapy Market market report are:

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

South America (Cuba, Brazil, Argentina, and many others.)

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

Asia (China, India, Russia, and many other Asian nations.)

Pacific region (Indonesia, Japan, and many other Pacific nations.)

Middle East & Africa (Saudi Arabia, South Africa, and many others.)

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Table of Contents

Report Overview: It includes major players of the global Gene Therapy Market Market covered in the research study, research scope, and Market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends: This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the global Gene Therapy Market Market. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the global Gene Therapy Market Market are discussed.

Market Share by Manufacturers: Here, the report provides details about revenue by manufacturers, production and capacity by manufacturers, price by manufacturers, expansion plans, mergers and acquisitions, and products, market entry dates, distribution, and market areas of key manufacturers.

Market Size by Type: This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

Market Size by Application: Besides an overview of the global Gene Therapy Market Market by application, it gives a study on the consumption in the global Gene Therapy Market Market by application.

Production by Region: Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the global Gene Therapy Market Market are profiled in this section. The analysts have provided information about their recent developments in the global Gene Therapy Market Market, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the global Gene Therapy Market Market as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the global Gene Therapy Market Market as well as for key regional markets.

Value Chain and Sales Analysis:It deeply analyzes customers, distributors, sales channels, and value chain of the global Gene Therapy Market Market.

Key Findings: This section gives a quick look at important findings of the research study.

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Global Gene Therapy Market Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2025 - Research Newspaper

Global Cell and Gene Therapy Industry market Report 2020 presents critical information and factual data about the Cell and Gene Therapy Industry market, providing an overall statistical study of this market on the basis of market drivers, market limitations, and its future prospects. The widespread Cell and Gene Therapy Industry market opportunities and trends are also taken into consideration in Cell and Gene Therapy Industry industry. with growth trends, various stakeholders like investors, traders, suppliers, SWOT analysis Opportunities and Threat to the organization and others.

The Cell and Gene Therapy Industry market report comprises of the key trends which influence the industry growth with respect to the regional terrain and competitive arena. The study highlights the opportunities that will support the industry expansion in existing and untapped markets along with the challenges the business sphere will face. Besides this, the report also offers an intricate analysis of case studies including those of COVID-19 pandemic, with the aim to provide a clear picture of this industry vertical to all shareholders.

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Pivotal pointers from COVID-19 impact assessment:

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Analysis of the regional terrain:

Highlights of the Cell and Gene Therapy Industry market report:

Key Coverage of report:

Impact of the latest technological innovations on the Cell and Gene Therapy Industry market

Key growth strategies adopted by the prominent market players to address the challenges and restraints put forward by the COVID-19 pandemic

Historical and current trends likely to affect the overall market dynamics of the Cell and Gene Therapy Industry market

Growth assessment of the various market segments over the forecast timeline

Regional and global presence of major market players in the Cell and Gene Therapy Industry market

Table of Content:

1 Cell and Gene Therapy Industry market Introduction and Market Overview

1.1 Objectives of the Study

1.2 Overview of Cell and Gene Therapy Industry market

1.3 Scope of The Study

1.3.1 Key Market Segments

1.3.2 Players Covered

1.3.3 COVID-19's impact on the Cell and Gene Therapy Industry industry

1.4 Methodology of The Study

1.5 Research Data Source

2 Executive Summary

2.1 Market Overview

2.1.1 Global Cell and Gene Therapy Industry market Size, 2015 - 2020

2.1.2 Global Cell and Gene Therapy Industry market Size by Type, 2015 - 2020

2.1.3 Global Cell and Gene Therapy Industry market Size by Application, 2015 - 2020

2.1.4 Global Cell and Gene Therapy Industry market Size by Region, 2015 - 2025

2.2 Business Environment Analysis

2.2.1 Global COVID-19 Status and Economic Overview

2.2.2 Influence of COVID-19 Outbreak on Cell and Gene Therapy Industry Industry Development

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Cell and Gene Therapy Industry Market with manufacturers, Application, regions and SWOT Analysis 2025 - CueReport

The treatment stops the body's own immune system attacking healthy eye cells.

Experts said the breakthrough signalled a "new era" for patients suffering from dry age-related macular degeneration, a type of blindness previously untreatable.

The research, which featured trials in the UK, Europe and the US, found new drugs injected into the eye can preserve sight by slowing the spread of the disease.

Dry macular degeneration is caused when the immune system becomes faulty and destroys healthy cells.

Researchers found two new injectable drugs which are being used to combat this - Pegcetacoplan and Zimura. Both work by introducing proteins which stop the faulty inflammatory process from causing an attack on eye cell membranes.

The monthly injectable treatment has so far been tested in 286 patients.

It saw disease progression slow by up to 29 percent over a year compared with those who received a placebo, or dummy treatment.

Scientists believe the drugs will continue to stop the spread of the disease when used over a longer period.

It is also hoped the revolutionary therapy will be on the market for widespread use within four years.

Professor Paulo Stanga, a worldleading eye surgeon who works at the London Vision Clinic in Harley Street, has been involved with the research. He said: "These results are exciting and fantastic news.

"At present there is no approved treatment for this previously irreversible AMD and over the years patients have been told nothing could be done. We know increasing numbers of patients will end up with complete loss of central vision if we do not find a solution.

"It is essential to treat dry AMD in the early stages before there is more loss of vision.

"The evidence from this trial suggests the treatment preserves vision over time.

"There is already evidence to be confident we can slow progression of dry AMD, potentially saving tens of thousands of people from sight loss, which is fantastic."

Alongside the injection-based trials there are two other potential drug targets for dry AMD for those who have already lost some vision.

The first is a one-time gene therapy surgically delivered into the eye.

This is currently being studied in an early trial known as the Focus Study. It is designed to dampen down the activity of the faulty genes that cause inflammation.

Another study is looking into low level light therapy. This has been shown to stimulate oxygen and blood flow in the eye, reducing inflammation and increasing vision in a trial of 30 subjects.

This study found patients were able to read a smaller line of letters on an eye chart than before treatment.

Macular degeneration currently affects up to 700,000 people in the UK.

There are two types, dry and wet, with a largely equal split in the numbers of cases.

It is estimated there will be 1.3 million people suffering from late stage AMD by 2050 - almost double the number today.

The scientific work was published in the medical journal Ophthalmology.

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Blindness breakthrough as monthly jabs first to half leading cause of sight loss - Express

Impact Analysis of Covid-19

The complete version of the Report will include the impact of the COVID-19, and anticipated change on the future outlook of the industry, by taking into the account the political, economic, social, and technological parameters.

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The Gene Therapy Market is expected to have a highly positive outlook for the next five years 2020-2027 according to a recently released Gene Therapy Market research report. The report has been added to his large database by Coherent Market Insights. This report is a guide that covers key strategic developments of the market including acquisitions & mergers, new technology launch, agreements, partnerships, collaborations & joint ventures, research & development, technology, and regional expansion of major participants involved in the market on the global and regional basis.

It also going to elaborate the opportunities out there in micro niches for stakeholders to take a position, step by step investigation of the competitive landscape and even commodity professional services of famous players which include GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

The key features of the Gene Therapy Market report 2020-2027 are the organization, extensive amount of analysis, and data from previous and current years as well as forecast data for the next five years. Most of the report is made up of tables, charts, and figures that give our clients a clear picture of the market.

The comprehensive value chain analysis of the Gene Therapy market will assist in attaining better product differentiation, along with a detailed understanding of the core competency of each activity involved. The market attractiveness analysis provided in the report measures the potential value of the Gene Therapy providing business strategists with the latest growth opportunities.

Questions have been answered at Gene Therapy Market report:

Economy Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, along with Strategies) The length of the global Gene Therapy market opportunity? How share promote Gene Therapy their worth from different manufacturing brands? Which will be the niches at which players profiling with thorough plans, financials, and also recent advancements should set a presence? Which will be the anticipated growth rates for your own Gene Therapy economy altogether and also for every segment inside? Which will be the Gene Therapy application and types and forecast accompanied closely by producers?

The research report on the Gene Therapy market evaluates the growth trends of the industry through historical study and estimates future prospects based on comprehensive research. The report extensively provides market share, growth, trends, and forecasts for the period 2020-2027. The market size in terms of revenue (USD MN) is calculated for the study period along with the details of the factors affecting the market growth (drivers and restraints).

Key Topics Covered:

Part 01: Executive SummaryPart 02: Scope Of The ReportPart 03: Research MethodologyPart 04: Market Landscape Market Ecosystem Market Characteristics Market Segmentation Analysis

Part 05: Market Sizing Market Definition Market Sizing 2020 Market Size And Forecast 2020-2027

Part 06: Five Forces Analysis Bargaining Power of Buyers Bargaining Power of Suppliers Threat of New Entrants Threat of Substitutes Threat of Rivalry Market Condition

Part 07: Customer LandscapePart 08: Regional Landscape Geographical Segmentation Regional Comparison Americas Market Size And Forecast 2020-2027 EMEA Market Size And Forecast 2020-2027 APAC Market Size And Forecast 2020-2027

Part 09: Decision FrameworkPart 10: Drivers And Challenges Market Drivers Market Challenges

Part 11: Market TrendsPart 12: Vendor Landscape Overview Landscape Disruption Vendors Covered Vendor Classification Market Positioning Of Vendors

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Gene Therapy Market Professional Industry Overview 2020 Segmentation and Development Analysis BY 2027 - Science Examiner

Horizon Discovery Group plc

Half Year Trading Update

Cambridge, UK, 29 July 2020:Horizon Discovery Group plc (LSE: HZD) ("Horizon", "the Group" or the Company), a cell engineering company focused on commercialising the application of gene editing and gene modulation to accelerate scientific innovation and biopharmaceutical drug development, today provides an unaudited pre-close trading update for the six months ended 30 June 2020. The Company plans to report its half year 2020 results on 17 August 2020.

Financials

Horizon expects to report half year 2020 revenues of approximately 22.4 million (HY 2019: 26.1 million, c. -13.9%)1, approximately 22.0 million (c. -15.5%) on a constant currency basis. The change was largely due to the rapid reduction of academic research work caused by the COVID-19 pandemic impacting the Groups Research Reagents business unit and was broadly in line with Board expectations. The greatest impact was seen in the second quarter of 2020, most notably in April. This was then followed by a period of sustained recovery, which resulted in large parts of the business regaining momentum and returning towards 2019 levels of revenue by the end of June 2020.

Gross margin from continuing operations1 is expected to be approximately 66.0% (HY 2019: 71.2%). The margin decline is primarily due to the impact of COVID-19 on manufacturing costs and additional provisioning for aged inventory. Adjusted EBITDA is expected to be a loss of 4.6 million (HY 2019: 0.0 million1).

The Groups cash position was bolstered by a successful Placing in April 2020, which raised 6.9 million in gross proceeds, and was further strengthened by the implementation of enhanced cash control measures implemented in April. Taking this into account, the Group had cash and cash equivalents of 23.6 million as of 30 June 2020 (HY 2019: 24.8 million; FY 2019: 18.8 million). The Groups strong balance sheet provides financial flexibility to ensure operational delivery and continued strategic investment to fully benefit from market recovery.

1. In December 2019, the Company completed its divestment of its non-core In Vivo business unit to Envigo RMS LLC. Revenues generated in the period in the financial year that the Company owned In Vivo are excluded from the Companys continuing operations

Terry Pizzie, Chief Executive Officer of Horizon Discovery, commented:

Thanks to the fantastic efforts of our staff, we have continued to operate effectively throughout the crisis and have built stronger and deeper relationships with our biopharma customers based on our ability to add value in difficult conditions. Our customers have increasingly adopted outsourcing as a

solution to their own business challenges and we have become recognised as an invaluable long-term partner.

We expect the trend for increased outsourcing to continue, for these relationships to endure and facilitate high level access within our biopharma customer base that will help lay the foundations for commercialising our new high growth areas.

We are encouraged by our H2 2020 prospects and look forward to the remainder of the year with optimism and confidence about the Groups strategy and prospects.

Performance by Business Unit

Research ReagentsRevenue for the Research Reagents business was 16.0 million, down 15.3% against the same period in the prior year, with trading significantly impacted by COVID-19. Academic and government research labs account for approximately 50% of the Research Reagents business units revenues, and the closure of many of these facilities starting from mid-March, led to a rapid reduction in orders for RNAi and CRISPR reagents and cell line products.

In April, revenues for reagents reduced to a low of 56% of the same period in 2019. Trading started to improve in May as researchers began to return to work and, by the end of June, trading had recovered to 2019 levels.

As a result of the repositioning of the Groups Cell Line Engineering (CLE) business, with a new value proposition, pricing and go-to-market strategy introduced at the end of Q1 2020, demand has increased significantly in Q2. This is not yet reflected in the reported revenues as a result of c. 10-week project delivery and revenue recognition timelines. Given the significant increase in demand, the Group has committed to additional investment in lab equipment and automation of approximately 3.5 million over the next 18 months, to further increase capacity to levels 25 times those of January 2019. To conserve near-term cash, this investment will be largely funded through leasing equipment. More detail on this will be provided at the Groups half year 2020 results. In H2 2020, the Group also expects to benefit from demand for CLE from academic and government research, as most researchers return to work following the loosening of lock-down restrictions.

For the first time, the results for this business unit now include Diagnostics due to operational synergies, including e-commerce. Diagnostics revenues were down 8.5% from H1 2019. The Group expects this part of the business to return to growth in H2 2020.

ScreeningOverall revenue of 4.4 million represented a 2.8% increase on the same period in the prior year. In line with typical biopharma spending patterns, the Screening business saw a relatively flat first quarter. However, after the initial slowdown in late March, the Group saw a notable increase in orders from its biopharma customer base, as these companies responded to the challenges of the COVID-19 crisis by prioritising key projects and supplementing their own in-house resources by outsourcing both CRISPR screening and High Throughput Screening (HTS). The Group expects the trend for outsourcing to continue in H2 2020.

BioProductionOverall revenue of 2.0 million for H1 2020 was down 29.7% on the same period in the prior year, with revenues suffering from the impact of COVID-19 restrictions and changes of prioritisation within bioproduction facilities globally, resulting in delays to evaluations of our CHO cell line. Pick-up in activity in May and June indicates a potential road to recovery in H2 2020.

The Group remains excited about its collaboration with Mammoth Biosciences and the development of a suite of next generation engineered CHO lines as these initiatives are progressing well.

Base EditingAfter establishing a new Base Editing business unit in H1 2020, the Group has making good progress working with its early access customers, including a large global pharma company, to assess and shape the development of this technology. The Group believes this technology is well-positioned to benefit both the CAR-T therapy market and the gene therapy market.

Supporting COVID-19 Research

Horizons products and services are being used by our customers in several important areas of COVID-19 research, including in efforts to uncover the virus weaknesses, find targets for potential therapies and help guide vaccine design. The Groups RNAi and CRISPR reagents are being used by high profile organisations such as the Krogan Lab, based at University of California San Francisco. Nevan Krogan, PhD, Professor of Cellular Molecular Pharmacology at UCSF, who leads the facility, has cited Horizons ability to be quick and flexible in meeting the laboratorys needs for CRISPR and siRNA reagents as being instrumental in the rapid progression of its COVID research.

Outlook

The fundamental drivers and demands for Horizons products and services remain strong and the Group is confident of a return to growth in the second half of 2020. Horizon remains focused on executing its strategy, and the management team is excited about the continued expansion of its screening market and the opening-up of new high growth areas that will come from commercialising its investments in BioProduction and Base Editing.

Horizons strong balance sheet position provides financial flexibility to ensure operational delivery and continued R&D investment to fully benefit from market recovery. The Group will continue to prudently manage the business, ensuring operational delivery and focused investment in order to be in a strong position as markets normalise.

Ends

Capital Markets Webcast and conference call today:

Horizon Discovery will host a Capital Markets Webinar for institutional investors and analysts at 15:00pm BST. There will be a simultaneous live conference call. The joining instructions are as follows:

Webinar link: https://www.lsegissuerservices.com/spark/HorizonDiscoveryGroup/events/4ab97991-3d5b-45de-959b-9486da1e2d60

Conference call details for analysts and investors:

The webcast and presentation slides will be available on the Groups website shortly after the event: https://horizondiscoveryplc.com/news-events/presentations-and-recordings/

For further information from Horizon Discovery Group plc, please contact:

Horizon Discovery Group plcTerry Pizzie, Chief Executive OfficerJayesh Pankhania, Chief Financial OfficerJon Davies, Head of Investor RelationsTel: +44 (0) 1223 655 580

Numis Securities Limited (Broker and NOMAD)Freddie Barnfield / Duncan MonteithTel: +44 (0) 207 260 1000

Consilium Strategic Communications (Financial Media and UK Investor Relations)Mary-Jane Elliott / Matthew Neal / Melissa GardinerTel: +44 (0) 20 3709 5700Email:horizon@consilium-comms.com

Zyme Communications (Trade and Regional Media)Lorna CuddonT Tel: +44 (0) 7811 996 942Email:Lorna.cuddon@zymecommunications.com

Westwicke, an ICR Company (US Investor Relations)Stephanie CarringtonTel. +1 646-277-1282Email:horizondiscovery@icrinc.com

About Horizon Discovery Group plc http://www.horizondiscovery.com

Horizon Discovery Group plc (LSE: HZD) ("Horizon") is a cell engineering company focused on commercializing the application of gene editing and gene modulation to accelerate scientific innovation and biopharmaceutical drug development. Horizons portfolio of tools and services is built on decades of experience in altering the expression of genes across mammalian and human cell types to provide cell engineering tools and services to customers in three key areas of the therapeutic ecosystem: basic research, drug discovery and development and therapeutic applications. Horizons offerings support and enable critical elements of the drug development and therapeutic value chain, particularly in the area of precision medicine. Horizons customers include biopharmaceutical and diagnostics companies, contract research and manufacturing organizations and academic researchers across the globe.

Horizon is headquartered in Cambridge, UK with offices in USA and Japan. The Group is listed on the London Stock Exchange's AIM market under the ticker HZD.

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Horizon Discovery Group plc - Horizon Discovery Group plc Half Year Trading Update - GlobeNewswire

Scientists at the University of California-San Diego report that an investigational drug candidate called tipifarnib showed promise in treating head and neck squamous cell carcinoma (HNSCC) tumors with mutations in the HRAS gene. Head and neck cancer is one of the leading causes of cancer-related deaths worldwide, and squamous cell carcinomas account for the majority of these cases.

The study Tipifarnib as a Precision Therapy for HRAS-Mutant Head and Neck Squamous Cell Carcinomas, published in Molecular Cancer Therapeutics, findings shed new light on the HRAS gene, a member of the RAS family of genes that produce proteins that regulate a variety of cellular processes, including growth, movement and differentiation. In 4 to 8 percent of HNSCC tumors, the HRAS gene is mutated.

Tipifarnib is a potent and highly selective inhibitor of farnesyltransferase (FT). FT catalyzes the post-translational attachment of farnesyl groups to signaling proteins that are required for localization to cell membranes. Although all RAS isoforms are FT substrates, only HRAS is exclusively dependent upon farnesylation, raising the possibility that HRAS mutant tumors might be susceptible to tipifarnib-mediated inhibition of FT, write the investigators.

Here, we report the characterization of tipifarnib activity in a wide panel of HRAS mutant and wild type HNSCC xenograft models. Tipifarnib treatment displaced both mutant and wild type HRAS from membranes but only inhibited proliferation, survival and spheroid formation of HRAS mutant cells. In vivo, tipifarnib treatment induced tumor stasis or regression in all six HRAS mutant xenografts tested but displayed no activity in six HRAS wild type PDX models.

Mechanistically, drug treatment resulted in reduction of MAPK pathway signaling, inhibition of proliferation and induction of apoptosis and robust abrogation of neovascularization, apparently via effects on both tumor cells and endothelial cells. Bioinformatics and quantitative image analysis further revealed that FT inhibition induces progressive squamous cell differentiation in tipifarnib-treated HNSCC PDX.

These preclinical findings support that HRAS represents a druggable oncogene in HNSCC through FT inhibition by tipifarnib, thereby identifying a precision therapeutic option for HNSCCs harboring HRAS mutations.

This preclinical research has the potential to extend to the entire HNSCC patient community, whose overall survival rates are limited in recurrent or metastatic disease, and existing therapeutic options that are far from optimal, with response rates of roughly 10 to 20 percent, said senior co-author J. Silvio Gutkind, PhD, Distinguished Professor of Pharmacology and associate director of basic science at UC San Diego Moores Cancer Center.

These preclinical findings support the idea that HRAS represents a druggable oncogene in HNSCC through tipifarnibs inhibition of a key enzyme. It is a precision therapeutic option for HNSCCs harboring HRAS mutations.

In the study, UC San Diego researchers found that cell line- and patient-derived HNSCC models harboring HRAS mutations were highly sensitive to tipifarnib, which the authors said has demonstrated encouraging preliminary clinical activity in patients with relapsed or refractory HRAS-mutant HNSCC to date.

Currently, Kura Oncology, a San Diego-based biopharmaceutical company, is conducting a nationwide clinical trialto assess the safety and efficacy of tipifarnib in head and neck cancer with HRAS mutations. Employees of Kura are co-authors of the new paper. UC San Diego is not part of the trial.

Treatment with tipifarnib, wrote study authors, had a multifaceted effect on the biology of HRAS-mutant HNSCC tumors, reducing oncogenic signaling and proliferation, while increasing apoptosis, blocking angiogenesis, and driving squamous differentiation of tumors.

Head and neck cancer accounts for approximately 650,000 cases and 330,000 deaths annually worldwide. In the United States, approximately 4 percent of all cancers are head and neck, with an estimated 65,630 persons diagnosed each year, two-thirds of them men and 14,500 deaths, according to Cancer.Net.

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Critical Recurring Mutation Identified in Head and Neck Cancers - Genetic Engineering & Biotechnology News

Manufacturing cell therapies is technically and financially demanding; as a result, despite therapy developers gradually strengthening their in-house expertise, they are also becoming increasingly reliant on contract service providers

Roots Analysis is pleased to announce the publication of its recent study, titled, Cell Therapy Manufacturing Market (3rd Edition), 2019 2030.

The report features an extensive study of the current market landscape and future opportunities associated with cell therapy manufacturing. It focuses on both contract manufacturers, as well as developers with in-house manufacturing facilities, offering in-depth analyses of the various business entities that are engaged in this domain, across different global regions. Amongst other elements, the report includes:

Read Detailed Analysis: https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html

A detailed market forecast, featuring analysis of the current and projected future opportunity across key market segments (listed below)

Type of therapy

Source of cells

Scale of operation

Purpose of manufacturing

Key geographical regions

Key companies covered in the report

For more information, please click on the following link:

https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

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Cell Therapy Manufacturing Market is estimated to reach close to USD 11 Billion by 2030, predicts Roots Analysis - Market Research Posts

Cancer Gene Therapy Market Report 2020-26: COVID-19 Impact Analysis, Growth, and Change by Facts and Factors provide the strategists, marketers, and senior management with the critical information they need to assess the Cancer Gene Therapy market.

Leading market research company Facts & Factors (FnF) published the latest research report on "Cancer Gene Therapy Market By Type (Ex-vivo and In-vivo) and By Product (Viral Vectors, Non-viral Vectors, and Others): Global Industry Outlook, Market Size, Business Intelligence, Consumer Preferences, Statistical Surveys, Comprehensive Analysis, Historical Developments, Current Trends, and Forecasts, 20202026" in its database which covering in-depth analysis of size, segmentation market growth, and market share, competitive Landscape, Detailed List of Key Buyers and End-Users.

This report focuses on the Cancer Gene Therapy market which is experiencing strong growth. The report gives a guide to the Cancer Gene Therapy market which will be shaping and changing our lives over the next six years and beyond, including the market's response to the challenge of the global pandemic.

Request an Exclusive Free Sample Report of Cancer Gene Therapy Market: http://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

(The sample of this report is readily available on request).

Benefits of Requesting a FREE Sample Report Before Purchase:

(Note: The sample of this report is updated with COVID-19 analysis before delivery)

Spreadsheet Chapters May include

Market Consumption in US$ by Country by Product/Service by Year. Market, Financial, Competitive, Market Segmentation, Industry, Critical Parameters, Marketing Costs, Markets, Decision Makers, Performance, Product Launch.

Some of Following Top Market Players Profile Included in This Report:

Cancer Gene Therapy Market Analysis by Leading Market Players

Leading top market players in the Cancer Gene Therapy market are analyzed in the report along with their business overview, operations, geographical locations, financial analysis, SWOT profile, and Carbon Black products & services.

COVID-19- Current Scenario, Potential Impact, and Strategic Situation Analysis

Various communities and companies are doing their best to function and perform, and eventually cope with the challenges raised by COVID-19 pandemic. The COVID-19 pandemic had a negative impact on the market size for the year 2020, with small and medium scale companies struggling to sustain their businesses in the near term future. Industry leaders are now focusing to create new business practices to deal with crisis situations like COVID-19 pandemic.

Enquire About COVID-19 Updates for This Report: http://www.fnfresearch.com/inquiry/cancer-gene-therapy-market-by-type-ex-vivo-898

The report covers market characteristics, market size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the markets historic and forecast market growth by geography. It places the market within the context of the wider Cancer Gene Therapy market, and compares it with other markets.

The report contains a deep analysis of the Cancer Gene Therapy market with size in terms of both value and volume. The report provides a clear insight into current and future developments of the Cancer Gene Therapy market. It provides essential insights into Cancer Gene Therapy market revenue for the top 21 regional and national, and forecast until 2026.

Request Customized Copy of Report @ http://www.fnfresearch.com/customization/cancer-gene-therapy-market-by-type-ex-vivo-898

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Free Customization

This report includes Free Customization which generally involves a breakout of particular industries or country data at no additional charge. An analyst call can be arranged as part of this which is included in the free customization offering. The Cancer Gene Therapy Market can be customized to the country level or any other market segment.

The report concludes with the profiles of the key players in the Cancer Gene Therapy market. The key players are evaluated on various parameters such as business overview, development, sales value analysis, and recent development. The emerging players are evaluated on various parameters such as business overview, promising vaccines in the clinical development with phase, platform technology, and recent development.

For More Report Details, Visit: http://www.fnfresearch.com/cancer-gene-therapy-market-by-type-ex-vivo-898

KEY REPORT POINTERS & HIGHLIGHTS:

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Facts & Factors is a leading market research company and offers customized research reports and consulting services. Facts & Factors aims at management consulting, industry chain research, and advanced research to assist our clients by providing planned revenue model for their business. Our report and services are used by prestigious academic institutions, start-ups, and companies globally to understand the international and regional business background. Our wide-ranging database offers statistics and detailed analysis of different industries worldwide that help the clients in achieving sustainable progress. The well-organized reports help clients in developing strategies and making informed business decisions.

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Cancer Gene Therapy Market Size & Trends 2020 Analysis and Growth Forecast till 2026 | Updated Report With COVID-19 Impact Analysis - Northwest...

Roots Analysis has announced the addition of Non-Viral Transfection Reagents and Systems Market, 2020-2030 report to its list of offerings.

Over time, innovative approaches surrounding the development of potential therapies using non-viral transfection systems have prompted several companies to commercialize proprietary technologies to facilitate gene transfer into cells, via a variety of physical, chemical and other non-viral methods. The growing demand for safe and effective genetically engineered ATMPs is likely to further propel the opportunity for non-viral transfection system developers.

To order this 220+ page report, which features 100+ figures and 125+ tables, please visit this link

Key Market Insights

More than 110 companies claim to offer different types of non-viral transfection systems

The majority of players engaged in the development and commercialization of non-viral transfection systems offer reagents (52%), followed by companies offering electroporation-based transfection systems (17%) and other non-viral transfection systems (31%).

13,000+ patents related to non-viral transfection systems filed in the last three years

Over 50% of patents were filed in North America, and were primarily focused on novel transfection methods and affiliated technologies for use in therapy development, cellular reprogramming and gene editing.

300+ registered trials, as of January 2020, claim to involve non-viral transfection systems

Majority of the abovementioned clinical studies are / were focused on the evaluation of gene therapies, most of which are / were being evaluated for the treatment of oncological disorders. Further, it is worth noting that close to 50% of these trials were initiated in the last five years alone.

Over 500 cell / gene therapy developers are likely to enter into partnership with non-viral transfection system providers

It is worth noting that most of the players that are likely to enter into partnership with companies engaged in this domain are based in North America (52%), followed by those headquartered in Asia-Pacific (29%) and Europe (19%).

Revenues from the sales of non-viral transfection systems intended for research purposes, currently represent majority of the market share

By 2030, the contribution (in terms of sales-based revenues) of non-viral transfection systems for clinical applications, is anticipated to increase to 30% of the overall market. Further, the analysis presented in the report demonstrates that, in the foreseen future, around 50% of the markets revenues are likely to come in from the sales of electroporation-based transfection systems.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 1.4 billion (by 2030) financial opportunity within the non-viral transfection reagents and systems market has been analyzed across the following segments:

Type of Transfection System

End-User

Application Area

Key Geographical Regions

The report features inputs from eminent industry stakeholders, according to whom, a significant increase in adoption of non-viral transfection systems is anticipated in clinical applications, in the foreseen future. The report includes detailed transcripts of the discussions held with the following experts:

The research includes detailed profiles of key players engaged in this domain; each profile features an overview of the company, its financial information (if available), recent developments and an informed future outlook.

For additional details, please visit

https://www.rootsanalysis.com/reports/view_document/non-viral-transfection/307.html

or email [emailprotected]

You may also be interested in the following titles:

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Non-Viral Transfection Reagents And Systems Market is projected to be worth USD 1.4 billion by 2030, growing at a CAGR of 9.2%, claims Roots Analysis...

The Protein Expression Market report 2020 covers all the significant developments which are recently being adopted across the global market. The prime objective of the Protein Expression market report is to provides an in-depth analysis of all market dynamics including drivers and restraints, and trends, and opportunities. The Protein Expression market report covers both the demand and supply aspects of the market. The report also highlighted the future trends in the Protein Expression market that will impact the demand during the forecast period.

Scope of the Report:

As per the , protein expression is the way in which proteins are modified, synthesized, and regulated in living organisms. The term is applicable to either the object of study or the laboratory techniques required to manufacture proteins. Proteins are regulated and synthesized depending upon the functional need in the host cell. The translation, transcription, and post-translational modifications are the three processes involved in protein expression.

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Market Overview:

Some of the Top Key Players of Protein Expression Market Report Are:

Report Highlights:

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Key Market Trends:

Cell Free Expression is the Largest Segment Under Expression System that is Expected to Grow During the Forecast Period

The cell-free protein expression is well-established in the market, due to the acceptance of it and this protein expression is performed without the use of living cells. This technique has many advantages over other protein expression techniques and because of its advantages, it is widely used. Due to the advantages of cell-free protein techniques, such as increased speed, ability to express toxic proteins, ease of amino acid type selective labeling, among others, these are making the cell expression market to grow.

Therefore, the unique technology may increase the growth of the cell-free expression market, which in turn, is expected to boost the protein expression market.

North America is the Largest Growing Market that is Expected to Continue the Same Trend Over the Forecast Period

North America is the largest market for protein expression and is expected to dominate the overall protein expression market due to factors like the presence of well-established healthcare infrastructure in these regions combined with the intense R&D activities carried out. The United States protein expression market is driven by factors, such as an increase in the usage of protein technologies, the rapid evolution of the pharmaceutical industry in the region, and the presence of several companies in the region itself.

The Report Covers:

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Detailed TOC of Protein Expression Market Report 2020-2024:

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Rise in the R&D Activity in the Pharma Sector4.2.2 Opportunities in the Field of Proteomics4.2.3 Advancements in the Biologics Market set to Boost the Market Growth4.2.4 Increasing Incidences of Chronic Diseases4.3 Market Restraints4.3.1 High Cost of Protein Expression Reagents and Instruments4.3.2 Increasing Consolidation and High Barriers for New Entrants4.4 Porters Five Forces Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Expression System5.1.1 Cell-Free Expression5.1.2 Bacterial Expression5.1.3 Yeast Expression5.1.4 Algal Expression5.1.5 Insect Expression5.1.6 Mammalian Expression5.1.7 Other Expression Systems5.2 By Product5.2.1 Instrument5.2.2 Reagent5.2.3 Service5.2.4 Other Products5.3 By End User5.3.1 Academia5.3.2 Industry5.3.3 Other End Users5.4 Geography5.4.1 North America5.4.1.1 US5.4.1.2 Canada5.4.1.3 Mexico5.4.2 Europe5.4.2.1 Germany5.4.2.2 UK5.4.2.3 France5.4.2.4 Italy5.4.2.5 Spain5.4.2.6 Rest of Europe5.4.3 Asia-Pacific5.4.3.1 China5.4.3.2 Japan5.4.3.3 India5.4.3.4 Australia5.4.3.5 South Korea5.4.3.6 Rest of Asia-Pacific5.4.4 Middle East & Africa5.4.4.1 GCC5.4.4.2 South Africa5.4.4.3 Rest of Middle East & Africa5.4.5 South America5.4.5.1 Brazil5.4.5.2 Argentina5.4.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Agilent Technologies Inc.6.1.2 Bio-Rad Technologies6.1.3 EMD Millipore6.1.4 Takara Bio Inc.6.1.5 New England Biolabs Inc.6.1.6 Oxford Expression Technologies Ltd6.1.7 Promega Corporation6.1.8 Qiagen NV6.1.9 Sigma-Aldrich Corporation6.1.10 Thermo Fisher Scientific Inc.

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

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