Category Archives: Gene therapy

AHWATUKEE, AZ (3TV/CBS 5) -An Ahwatukee family is dealing with a worst-case scenario after two of their three young daughters were diagnosed with an extremely rare brain disease.

Only one of the girls is eligible for gene therapy, but the treatment is in a different country, and they need help with finances to make it possible.

Dave and Kendra Riley have their hands full with three girls: 5-year-old Eva, 2-year-old Olivia, and 5-month-old Keira.

But this past year, they noticed Olivia was starting to have trouble walking and talking.

Then the irises of her eyes started vibrating a little, and were like okay let's get her checked out, said Kendra.

In March, a neurologist diagnosed her with Metachromatic Leukodystrophy, known as MLD, a rare genetic brain disease. Her motor skills have quickly been regressing since.

Most cases of MLD at her age group, without any treatment, she'll maybe have another 2 to 4 years left, said Kendra.

There isn't a day that goes by where I don't cry somewhere at some point in time because it's just, what can we do to take the pain away? said Dave.

They take Olivia every week to a clinical study treatment in Iowa, but because both Kendra and Dave are carriers of genes that formed the mutation, they had to get their other two daughters tested.

Their oldest, Eva, is only a carrier and will live a normal life. But two weeks ago they got the results for baby Keira.

She came back with both gene mutations, so just like Olivia, she has MLD, said Kendra.

Because Keira isn't showing symptoms yet, she's eligible for a rare gene therapy treatment in Italy, where 29 kids in the world have gone through it.

Olivia will never be able to recover, but they may be able to save Keira if they can get to Italy in the next month.

The community has been raising money to help them get there amid the pandemic, and for that, the Riley's are grateful.

The smallest donations of a dollar or five dollars are just as heartwarming to us as the ones that are larger because that could be all they have, said Kendra with tears in her eyes.

Here is a link to the Riley familys GoFundMe if you would like to help donate to their expenses and the girls treatment.

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Ahwatukee family seeks treatment in Italy for daughter diagnosed with rare brain disease - AZFamily

Madison researchers have developed a safer and more efficient way to deliver a promising new method for treating cancer and liver disorders and for vaccination including a COVID-19 vaccine from Moderna Therapeutics that has advanced to clinical trials with humans.

The technology relies on inserting into cells pieces of carefully designed messenger RNA (mRNA), a strip of genetic material that human cells typically transcribe from a persons DNA in order to make useful proteins and go about their business. Problems delivering mRNA safely and intact without running afoul of the immune system have held back mRNA-based therapy, but UWMadison researchers are making tiny balls of minerals that appear to do the trick in mice.

These microparticles have pores on their surface that are on the nanometer scale that allow them to pick up and carry molecules like proteins or messenger RNA, saysWilliam Murphy, a UWMadison professor of biomedical engineering and orthopedics. They mimic something commonly seen in archaeology, when we find intact protein or DNA on a bone sample or an eggshell from thousands of years ago. The mineral components helped to stabilize those molecules for all that time.

Murphy and UWMadison collaborators used the mineral-coated microparticles (MCMs) which are 5 to 10 micrometers in diameter, about the size of a human cell in a series of experiments to deliver mRNA to cells surrounding wounds in diabetic mice. Wounds healed faster in MCM-treated mice, and cells in related experiments showed much more efficient pickup of the mRNA molecules than other delivery methods.

The researchers described their findings today in the journal Science Advances.In a healthy cell, DNA is transcribed into mRNA, and mRNA serves as the instructions the cells machinery uses to make proteins. A strip of mRNA created in a lab can be substituted into the process to tell a cell to make something new. If that something is a certain kind of antigen, a molecule that alerts the immune system to the presence of a potentially harmful virus, the mRNA has done the job of a vaccine.

The UWMadison researchers coded mRNA with instructions directing cell ribosomes to pump out a growth factor, a protein that prompts healing processes that are otherwise slow to unfold or nonexistent in the diabetic mice (and many severely diabetic people).

mRNA is short-lived in the body, though, so to deliver enough to cells typically means administering large and frequent doses in which the mRNA strands are carried by containers made of molecules called cationic polymers.

Oftentimes the cationic component is toxic. The more mRNA you deliver, the more therapeutic effect you get, but the more likely it is that youre going to see toxic effect, too. So, its a trade-off, Murphy says. What we found is when we deliver from the MCMs, we dont see that toxicity. And because MCM delivery protects the mRNA from degrading, you can get more mRNA where you want it while mitigating the toxic effects.

The new study also paired mRNA with an immune-system-inhibiting protein, to make sure the target cells didnt pick the mRNA out as a foreign object and destroy or eject it.

Successful mRNA delivery usually keeps a cell working on new instructions for about 24 hours, and the molecules they produce disperse throughout the body. Thats enough for vaccines and the antigens they produce. To keep lengthy processes like growing replacement tissue to heal skin or organs, the proteins or growth factors produced by the cells need to hang around for much longer.

What weve seen with the MCMs is, once the cells take up the mRNA and start making protein, that protein will bind right back within the MCM particle, Murphy says. Then it gets released over the course of weeks. Were basically taking something that would normally last maybe hours or even a day, and were making it last for a long time.

And because the MCMs are large enough that they dont enter the bloodstream and float away, they stay right where they are needed to keep releasing helpful therapy. In the mice, that therapeutic activity kept going for more than 20 days.

They are made of minerals similar to tooth enamel and bone, but designed to be reabsorbed by the body when theyre not useful anymore, says Murphy, whose work is supported by the Environmental Protection Agency, the National Institutes of Health and the National Science Foundation and a donation from UWMadison alums Michael and Mary Sue Shannon.

We can control their lifespan by adjusting the way theyre made, so they dissolve harmlessly when we want.

The technology behind the microparticles was patented with the help of the Wisconsin Alumni Research Foundation and is licensed to Dianomi Therapeutics, a company Murphy co-founded.

The researchers are now working on growing bone and cartilage and repairing spinal cord injuries with mRNA delivered by MCMs.

Reference: Khalil et al. (2020).Single-dose mRNA therapy via biomaterial-mediated sequestration of overexpressed proteins. Science Advances.DOI: 10.1126/sciadv.aba2422.

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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Safer and More Efficient Method To Deliver Gene Therapy - Technology Networks

Biogen has picked up a new, preclinical gene therapy program from Massachusetts Eye and Ear for inherited retinal degeneration due to mutations in the PRPF31 gene, among the most common causes for autosomal dominant retinitis pigmentosa. Theyre building on the work of Harvards Eric Pierce. The treatment of IRDs with highly effective AAV-based gene therapies is core to Biogens ophthalmology strategy, said Chris Henderson, the research head at Biogen. This agreement underscores our commitment to that strategy and builds off of our acquisition of Nightstar Therapeutics in 2019 and our active clinical trials of gene therapies for different genetic forms of IRD.

Sarepta has inked a collaboration with Hansa to develop their experimental drug imlifidase as a pre-treatment for their gene therapies. The drug is intended for use in patients who have neutralizing antibodies that would prevent gene therapies for Duchenne muscular dystrophy and Limb-girdle muscular dystrophy from working. Hansa gets a $10 million upfront and up to $397.5 million in milestones.

Its been raining money on Wall Street at least when it comes to drug developers. CRISPR Therapeutics $CRSP and Acceleron each raised a whopping $450 million this week after pricing follow-on offerings. CRISPR priced 6,428,572 common shares at a public offering price of $70.00 per share, while Acceleron $XLRN auctioned off 4,864,864 shares of common stock at a price to the public of $92.50 per share.

The transatlantic biotech player Immatics has completed its flip onto Nasdaq through the Arya Sciences Acquisition Corp. The cancer drug biotech will trade as $IMTX after it raised $253 million in the process. The SPAC was set up by Perceptive Advisors.

Seattle-based Neoleukin Therapeutics, meanwhile, raised $76.2 million $NLTX for its work on protein therapeutics.

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Biogen bags an AAV gene therapy program from Massachusetts Eye and Ear; Biotechs raised $1B-plus in latest round of follow-ons - Endpoints News

AVROBIO: On Monday, clinical-stage gene therapy company AVROBIO, headquartered in Massachusetts, announced the appointment of Kim Raineri as chief manufacturing and technology officer.

I am thrilled to join AVROBIO, a leader in lentiviral gene therapy and a true pioneer in driving manufacturing advances that address the gene therapy fields need for faster, more scalable and more automated production, Raineri said. The AVROBIO team has created a state-of-the-art gene therapy platform and is clearly committed to continuous innovation on behalf of the patient communities they strive to serve. I am excited to contribute to that work.

Raineri will be replacing AVROBIO co-founder Kim Warren in the position, who will be retiring at the end of July. Before joining AVROBIO, Raineri served as the vice president of operations for Nikon CeLL Innovation Co.

Scenic Biotech: On Wednesday, Netherlands-based Scenic Biotech announced the appointment of their new chief executive officer. Newly appointed CEO Oscar Izeboud brings more than 20 years of life sciences and finance industry experience.

Prior to joining Scenic, Izeboud served as managing director at NIBC Bank in Amsterdam, where he led its corporate finance and capital markets team with a focus on innovation and growth companies.

Former acting CEO and scientific co-founder Sebastian Nijman takes on the role of chief scientific officer.

Akari Therapeutics: Biopharmaceutical company Akari Therapeutics on Wednesday announced the appointment of Torsten Hombeck as chief financial officer and a member of the company's executive team.

Torsten brings a deep understanding of financial strategy, the capital markets and business development to Akari. We are delighted to have him as a permanent member of Akaris executive leadership team," said Clive Richardson, Chief Executive Officer of Akari Therapeutics. "His appointment comes at a time of significant company opportunity and growth. His business and financial expertise will be instrumental in helping us to further develop the Company."

Hormbeck joins Akari with over 20 years of biopharmaceutical industry experience in financial and strategic planning.

Sarepta Therapeutics: Earlier this week, Cambridge-based Sarepta Therapeutics announced the retirement of Sandy Mahatme, the company's executive vice president, chief financial officer and chief business officer. Mahatme will be leaving the company effective July 10.

The Sarepta from which Sandy retires is a very different one from the organization he joined as our chief financial officer some eight years ago. And the Sarepta of today a financially solid biotechnology organization with perhaps the industrys deepest and most valuable pipeline of genetic medicine candidates with the potential to extend and improve lives would not have been possible without Sandys business acumen and dedication, said Doug Ingram, president and chief executive officer of Sarepta Therapeutics.

Sarepta has launched a search to identify the future chief financial officer.

BioMarin: On June 29, BioMarin, a global biotechnology company, announced a pair of promotions. Brian Mueller was promoted to executive vice president, chief financial officer and Andrea Acosta was promoted to group vice president, chief accounting officer.

Mueller has been with BioMarin since 2002, during which he has taken on roles of increasing responsibility. Acosta has been with BioMarin since 2017 as vice president, corporate controller.

Theravance Biopharma: Dublin-based Theravance Biopharma on Thursday announced the appointment of Deepika Pakianathan to its Board of Directors. Pakianathan serves as a managing member at Delphi Ventures, a venture capital firm focused on biotechnology and medical device investments.

"We are honored to welcome Dr. Pakianathan to our board of directors," said Rick Winningham, chief executive officer of Theravance. "We believe her vast experience in the biotechnology sector, translating breakthrough science and taking important therapies from pipeline to patients, will further enhance our already talented Board of Directors."

Novavax: On Thursday, Maryland-based Novavax announced the appointment of Frank Czworka as senior vice president, global sales. Czworka will be responsible for leading sales planning and distribution for the company. He brings more than 20 years of biopharmaceutical experience to the company, with his most recent experience being as vice president, global customer enngagement at U.S. Pharmacopeia.

Novavax also announced the promotion of Brian Webb to senior vice president, manufacturing. Webb will be responsible for overseeing antigen manufacturing and supply activities in support of the company's vaccine candidates. Webb has been with Novavax since May 2014.

eGenesis: On Wednesday, Massachusetts-based eGenesis announced that it appointed Peter Hanson as chief operating officer. Hanson will be in charge directing eGenesis' day-to-day organizational and operational activities including production and manufacturing.

Peter is a highly experienced biopharmaceutical executive across multiple disciplines, which will be critical to support our next phase of growth as we integrate production and R&D, said Paul Sekhri, President and Chief Executive Officer of eGenesis. Peters operational leadership and veterinary knowledge will help us accelerate our product development as we move closer to IND filing for human clinical studies. We are very grateful for Kenneth Fans many contributions as our founding COO. I am delighted that he will continue to serve as an advisor to the company.

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Movers & Shakers, July 3 | BioSpace - BioSpace

Avantor, a global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries, announced the expansion of the companys Bridgewater, NJ (USA) innovation center. Located close to biotech companies, the site now has nearly double the laboratory and support space.

Bioprocessing efficiency that doesnt compromise quality continues to be a top priority for the biopharma industry. At the same time, its critical to meet increasingly stringent global regulatory standards.

Innovation is about solving customer problems in a rapidly progressing biotechnology environment using contemporary technologies and thinking, said Dr. Ger Brophy, Executive Vice President, Biopharma Production at Avantor. We collaborate with our customers from their initial discovery and development activities through scale-up and commercialization. Our solutions support industry downward cost pressure by emphasizing supply chain efficiency and improving production process performance.

Avantors expanded innovation center enhances the Companys R&D support for customized product and bioprocess development, and custom cell and gene therapy reagent manufacturing to support customers therapeutic products that are developed for advancements in personalized medicine.

The site capabilities support new bioprocessing products designed to help minimize risk of contamination and variability for cell and gene therapy (C>), monoclonal antibodies (mAbs) and vaccines, including high purity products, single-use solutions and small volume cGMP reagents in single use bags. The site developed Avantors recently launched novel J.T.Baker BAKERBOND PROchievA recombinant protein A resin for purifying monoclonal antibodies (mAbs) and other complex molecules.

Monoclonal antibodies (mAbs) are used in a wide variety of clinical indications, including asthma, cancer, Crohns disease, and rheumatoid arthritis, and most recently being tested for use as a treatment for COVID-19 according to the National Institutes for Health (NIH).

The Bridgewater innovation center expansion follows the December 2019 opening of Avantors innovation and customer support center in Shanghai, China.

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Avantor expands Life Sciences Innovation Center to address bioprocessing needs in protein science, monoclonal antibodies (mAbs), cell and gene therapy...

Gene Therapy Market Snapshot

Introduction of new production, availability of reimbursement together with high occurrences of cancer are estimated to propel growth of the global gene therapy market in the years to come. Gene therapy refers to an experimental technique, which utilizes genes for the prevention and treatment of various diseases. It is expected that in the near future, this technology could assist doctors to place a gene into the cells of a patient for the purpose of his treatment. This therapy could be used as an alternative to surgery or drugs. Scientists are examining various approaches to this therapy, which could comprise

Gene therapy has emerged as a promising treatment option for a large number of diseases such as certain viral infections, certain cancers, and inherited disorders. This factor is likely to work in favor of the global gene therapy market in the years to come.

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Progress in Gene-editing and Genomics Tools to Stoke Demand of Gene Therapy

CAR T-cell use has recently garnered considerable attention from the sponsors following the exhibition of its immense promise in the treatment of several diseases. The promising future of CAR T-cell is estimated to amplify the growth opportunities of the global gene therapy market. Sponsors hail CAR T-cell use as a brand new business model of the future.

In the pipeline of pharmaceutical industry, gene therapy account for a considerable share and this trend is likely to continue in the years to come. In addition, significant advancement has been made in the fields of cellular and molecular biology is likely to fuel growth of the global gene therapy market in the years to come. rapid technological progress made in the gene-editing and genomics tools are further estimated to drive the demand for gene therapy.

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Global Gene Therapy Market Snapshot

Expanding at a stellar, double-digits CAGR (Compound Annual Growth Rate) of 40% over the forecast period of 2018 to 2026, the global gene therapy market is a dizzying trajectory, marking out a rosy landscape for players operating in the playfield. As per a Transparency Market Research report, based on extensive primary and secondary research, states that over the period states, the market would accrue a worth of USD 5164.03 million a steep and impressive increase from the USD 17 million worth noted in 2017.

Fixing defective genes via introduction of new and healthy ones to fix severe genetic and chronic disorders is seeing an upward curve in demand for reasons of improved medical outcomes, major leaps in terms of technological advancement and minimal die-effects as compared to alternatives. Some of the most significant alternatives include surgery, and drug use.

Some of the most significant factors that the analysts of the report note include focused efforts towards marketing and commercialization, and a slew of approvals of new products hitting the global gene therapy market. Additionally, there are factors such as growing demand experienced for this treatment by a large pool of patients.

It is pertinent to note here that the global gene therapy market is consolidated and is dependent on clinincal research and development of the highest standards in order to chart growth. And, some of the players that operate the market landscape, and are into significant research projects include Gilead Sciences, Inc., Novartis AG, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., CELGENE CORPORATION, and Orchard Therapeutics Limited.

Yescarta to Dominate Global Gene Therapy Market over the Forecast Period

The global gene therapy market report by TMR is segmented based on type, application, and region. The former includes the only five products that have been approved so far for commercial use. These include Gendicine, Yescarta, Strimvelis, Kymriah, and Luxturna. Riding the first mover benefits, Yescarta helf the dominant position in the market in 2017, and the trend will continue, adding more worth to the sub-segment. This is the product that brought out the initial CAR T therapy in the market for large B-cell lymphoma that relapse.

It is worth noting here that as per the global gene therapy market report, the high incidence of DLBCL and massive commercialization efforts directed towards Yescarta, particularly in Europe, will contribute positively and significantly to the overall growth of the global gene therapy market. The other sub-segment to make a mark over the global gene therapy market landscape will be Luxturna, owing to rising awareness levels and massive efforts towards comercialization.

Europe to be Ahead of the Global Gene Therapy Market Growth Curve over the Forecast Period

The global gene therapy markets regional segmentation includes incisive growth insights into some of the most significant areas that will shape up the overall growth in the market. These include North America, Europe, and Rest of the World. Researchers involved with the preparation of report claim that a massive chunk of about 40% would be accounted for by Europe over the forecast period. Some of the factors backing-up the market dominance of the region include high incidence of non-Hodgkin lymphoma and increase in number of treatment centers into gene therapy.

This growth would be followed by North America region, owing to huge contributions from the United States of America which witnesses about 7500 cases of refractory DLBCL each year. These are ones that qualify for the CAR T therapy.

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Analysis of COVID-19 Gene Therapy Market Cole Reports - Cole of Duty

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Latest research report on Gene Therapy for Age-related Macular Degeneration Market delivers a comprehensive study on current market trends. The outcome also includes revenue forecasts, statistics, market valuations which illustrates its growth trends and competitive landscape as well as the key players in the business.

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Macular degeneration is a condition in which, macula, a part of the retina, gets damaged or deteriorated. This condition usually affects individuals who are aged 50 years and above and therefore, it is called age-related macular degeneration (AMD). AMD is the leading cause of vision loss and is directly related to the advancement of age. But smoking also plays a vital role in causing AMD. AMD is characterized by the presence of a blurred area near the center of vision that leads to distorted vision. There are two different types of AMD, including dry (atrophic) AMD (dAMD) and wet (neovascular/exudative) AMD (wAMD). The dAMD is the most common type of AMD and accounts for almost 80%-90% of the overall AMD cases.It has been observed that age-related macular degeneration (AMD) is one of the major causes for vision loss and is characterized by the formation of a blurred area near the center of vision, a condition that mostly affects the geriatric population. According to the CDC, almost 2 million individuals in the US suffer from AMD and by 2050, this number will reach more than 5 million. This will subsequently demand the need for the development of innovative treatments for AMD, driving the market's growth.The market research analysts have predicted that with the introduction of techniques such as fluorescein angiography, the global age-related macular degeneration market will register a CAGR of more than 7% by 2020. With the unavailability of FDA-approved treatment for dry AMD (dAMD) and the treatment of wet AMD (wAMD) involving the need of intravitreal injections for an indefinite period, gene therapy is emerging as the most-efficient approach for the treatment of age-related macular degeneration (AMD).According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for wet AMD (wAMD). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.This report focuses on the global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China. The key players covered in this study RetroSense Therapeutics REGENXBIO AGTC Market segment by Type, the product can be split into Subretinal Intravitreal Unspecified Market segment by Application, split into Monotherapy Combination Therapy

In this study, the years considered to estimate the market size of Gene Therapy for Age-related Macular Degeneration are as follows: History Year: 2014-2018 Base Year: 2018 Estimated Year: 2019 Forecast Year 2019 to 2025 Market segment by Regions/Countries, this report covers United States Europe China Japan Southeast Asia India Central & South America The study objectives of this report are: To analyze global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. To present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China. To strategically profile the key players and comprehensively analyze their development plan and strategies. To define, describe and forecast the market by product type, market and key regions.For the data information by region, company, type and application, 2018 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 Age-related Macular Degeneration Market Size 2020 By Top Key Players, By Type and Application, Forecast to 2025 - MENAFN.COM

Prophecy Market Insights Personalized Gene Therapy Treatment market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges during COVID-19 pandemic across the globe.

Personalized Gene Therapy Treatment Devices Market reports provide in-depth analysis of Top Players, Geography, End users, Applications, Competitor analysis, Revenue, Financial Analysis, Market Share, COVID-19 Analysis, Trends and Forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.

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An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Personalized Gene Therapy Treatment market

Detailed analysis of the COVID-19 impact will be given in the report, as our analyst and research associates are working hard to understand the impact of COVID-19 disaster on many corporations, sectors and help our clients in taking excellent business decisions. We acknowledge everyone who is doing their part in this financial and healthcare crisis.

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Segmentation Overview:

The Personalized Gene Therapy Treatment research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Personalized Gene Therapy Treatment market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.

Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Personalized Gene Therapy TreatmentMarket Key Players:

Amgen, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., SynerGene Therapeutics, Inc., Cold Genesys, Inc., Bellicum Pharmaceuticals, Inc., Takara Bio, Inc.,Ziopharm Oncology, Inc., , Sevion Therapeutics, Inc., OncoSec Medical, Inc., and Burzynski Clinic.

The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. With the tables and figures, the report provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

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Rapidly increasing demand and COVID-19 Analysis of Personalized Gene Therapy Treatment Market - Cole of Duty

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced that it will host a Key Opinion Leader (KOL) call on July 9, 2020 from 10:00 am to 11:00 am EST. The call will feature presentations by KOLs Nancy Newman, MD (Emory Eye Center) and Sean Donahue, MD, PhD (Vanderbilt Children's Hospital) who will discuss the current treatment landscape and unmet medical needs in Leber Hereditary Optic Neuropathy (LHON). Drs. Newman and Donahue will be available to answer questions at the end of the call.

This KOL call will also be an opportunity for GenSight's management to provide an update on the company and discuss the results from the REVERSE and RESCUE Phase III trials for LUMEVOQ (GS010). RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene. LUMEVOQ (GS010) is expected to be submitted for European approval in September 2020.

The call will be webcast live at https://bit.ly/3dZVQlX. For those not available to attend or listen to the live broadcast, a replay will be archived and available using the same link.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010), is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About LUMEVOQ (GS010)

LUMEVOQ (GS010) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,200 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint will measure the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints will involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis will be evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics will include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials are conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767

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GenSight Biologics to Host LUMEVOQ Key Opinion Leader Call on July 9, 2020 - Business Wire

The report covers the forecast and analysis of the Cell and Gene Therapy Consumables market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the Cell and Gene Therapy Consumables market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the Cell and Gene Therapy Consumables market on a global level.

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In order to give the users of this report a comprehensive view of the Cell and Gene Therapy Consumables market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

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The study provides a decisive view of the Cell and Gene Therapy Consumables market by segmenting the market based on product type, application/therapeutics, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

A rise in the awareness about the gene & cell therapies will propel the market growth during the period from 2019 to 2027. Nevertheless, conducting randomized control tests will inhibit the expansion of the market during the forecast timeline. However, the growing trend for treating neurodegenerative ailments through the use of gene treatment will proliferate the market growth over the forecast period.

The expansion of the market during the forecast timespan is owing to the high frequency of chronic ailments including cancer and heart disorders. Apart from this, inflation in the occurrence of these disorders produces lucrative demand for enhanced therapies and this will culminate in the market demand over the forecast time span.

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Based on the product type, the market is sectored into Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media. Application/ Therapeutics- wise, the market for cell and gene therapy consumables are classified into Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology.

Some of the key players in the market include Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife.

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Cell And Gene Therapy Consumables Market Size, Share And Top Key Players During 2020-2027 Bulletin Line - Bulletin Line