Category Archives: Gene therapy

Brussels, Belgium Ovizio Imaging Systems, a life science company providing an innovative image-based cell analyzer for process automation and process control to the bioprocessing market, today enters into a commercial partnership with the Life Sciences Business Sector of Merck KGaA, Darmstadt, Germany, for its iLine F PRO solution.

Merck will ensure commercial promotion of the iLine F PRO solution, consisting of a cell analyzer and single-use disposable for companies working in the cell & gene therapy space in Europe and North America.

The partnership strategically aligns both companies decisive goals to simplify cell therapy manufacturing by combining the strength of the ekko Acoustic Cell Processing System, a multi-use platform for cell concentration and wash combined with aggregate processing, with Ovizios iLine F PRO cell analyzer, a Process Analytical Technology (PAT) providing real-time monitoring of cell expansion in a bioreactor.

Emilie Viey, PhD, Chief Executive Officer of Ovizio Imaging Systems:For biologics, the link between the product and process is typically well defined and Cell Quality Attributes (CQAs) can be suitably characterized. In contrast, cell therapy programs autologous cell therapy programs in particular begin with highly complex and variable patient material, which necessitates more complex and diverse processes to generate a high-quality product. Offering automated and modular solutions to decrease variability and gain more understanding of the cell-based process is our driver. We couldnt have wished for a better commercial partner to offer a solution to the major players in cell & gene therapy that covers delicate and key steps in their process.

About Ovizio

Ovizio Imaging Systems develops and commercializes label-free cell analyzers designed for the bioprocessing market, with a focus on cell & gene therapy and vaccine production. We offer in-process analytical solutions that enable deep process understanding during development, and increased process control and consistency during manufacturing.

About the iLine F PRO

The iLine F PRO analyzer uses Ovizios proprietary label-free technology, a unique hologram-based method (patented Double Differential Digital Holographic Microscopy (D3HM)), for on-line, non-invasive classification and counting of suspension cells. Upon connection with a bioreactor, it continuously tracks objects in real-time at a single cell level with high accuracy, and enables automation of current manual processes. The iLine F PRO is cGMPcompliant and is compatible with standard communication protocols (OPC UA) and its applications, such as Scada and DeltaV, enabling further process automation and control when used in conjunction with other devices.

The BioConnect is a single-use disposable that has been designed for closed loop fluidics. It allows the cells to circulate between the analyzer, where cells are imaged, and the bioreactor, where cells are grown. The risk of contamination is, therefore, drastically reduced and no biological material is wasted in the analysis process.

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Ovizio Imaging Systems Is Entering Into A Commercial Partnership With Merck To Promote Its iLine F PRO Solution For The Cell Gene Therapy Market -...

Humans are afflicted by thousands of inherited genetic diseases, and most of these affect children. The vast majority of genetic diseases do not have safe and effective treatments available. Hundreds of thousands of people, mainly children, suffer greatly; their disease symptoms are determined by which of the bodys ~25,000 genes is defective. Some children lose their sight, others bleed frequently, and others lose the ability to simply walk or breath normally. Some of these patients tragically die at a young age. In a few diseases, children can be injected frequently (every week or two) with replacement proteins that can partially stem the tide; nevertheless, the disease progresses steadily and the childs quality of life is progressively degraded.

We need effective therapies for these kids. We need cures for these kids. This is what 4DMT fights for every day.

WHAT ARE GENES, AND WHY DOES IT MATTER WHEN THEYARE DEFECTIVE IN A GENETIC DISEASE?

Genes are blueprints for the manufacturing of proteins by cells in the body. Humans have about 25,000 genes for the same number of proteins. Each gene is important for a normal life. While every cell in the body has all of these genes, each cell only expresses (ie, turns on and makes the encoded protein) a small subset required for its specialized function in the body.

These genes are the cells software, coding for how each cell should function through the action of proteins. The proteins that genes encode for are the hardware that carry out almost every function in the body, from structures (eg, bone, cartilage) to enzymes (eg, that digest food) to messengers (eg, hormones).

When a gene is defective, either missing or mutated, the result is that the corresponding normal functional protein will not be present in the cells where it should be. Since a normal functional protein is missing, or at an insufficient level, the affected person suffers. If the gene for vision is defective, the person lacks a normal gene in their retina (tissue at the back of the eye) and they will develop progressive blindness (eg, choroideremia, retinitis pigmentosa and others). If the gene for a muscle factor is defective, the person lacks a normal muscle strength protein and they will have problems with muscle wasting and even with normal activities (eg, muscular dystrophy or lysosomal storage diseases). If the gene for a specific lung factor is defective, the person lacks a normal lung maintenance protein and they will have problems with breathing and lung infections (eg, cystic fibrosis).

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GENE THERAPY - 4D Molecular Therapeutics

An Air Force medical technician draws a dose of the COVID-19 vaccine to inoculate Air Force reservists at Joint Base Lewis McChord, Washington, Sept. 12, 2021. (U.S. Air Force photo by Staff Sgt. Paolo Felicitas)

Many skeptics have contended that the mRNA-based Pfizer and Moderna COVID-19 shots are not "vaccines" but rather a form of gene therapy that poses untold risks by altering a recipient's DNA.

The federal government and health-care experts have denied that claim. But the president of Bayer's Pharmaceuticals Division is on record describing the mRNA shots as "cell and gene therapy" and acknowledging public wariness of the technology.

Bayer executive Stefan Oelrich, LifeSiteNews reported, made the statement at the World Health Summit, which took place in Berlin Oct. 24-26, drawing 6,000 people from 120 countries.

Oelrich said his company is "really taking that leap" to drive innovation "in cell and gene therapies."

"Ultimately, the mRNA vaccines are an example for that cell and gene therapy," he said.

"I always like to say: If we had surveyed two years ago in the public 'would you be willing to take a gene or cell therapy and inject it into your body?' we probably would have had a 95% refusal rate," Oelrich said.

In August, Reuters ran a "fact check" citing experts who contend that the technology in the Pfizer/BioNTech and Moderna shots are not gene therapy.

Both shots usea piece of genetic code from SARS-CoV-2 to prompt an immune response in recipients. But Dr. Adam Taylor, a virologist and researcher at Griffith University in Australia, insisted that while it's "a genetic-based therapy," it doesn't alter a person's genes.

Gene therapy, in the classical sense, involves making deliberate changes to a patients DNA in order to treat or cure them," he said. "mRNA vaccines will not enter a cells nucleus that houses your DNA genome. There is zero risk of these vaccines integrating into our own genome or altering our genetic makeup."

At the Berlin summit, the Bayer executive said that his company's "successes" over the 18 months of the pandemic "should embolden us to fully focus much more closely on access, innovation and collaboration to unleash health for all, especially as we enter, on top of everything else that is happening, a new era of science a lot of people talk about the Bio Revolution in this context."

LifeSiteNews noted that, according to the McKinsey Global Institute, the "Bio Revolution" is "a confluence of advances in biological science and accelerating development of computing, automation, and artificial intelligence [that] is fueling a new wave of innovation."

"This Bio Revolution could have significant impact on economies and our lives, from health and agriculture to consumer goods, and energy and materials."

Oelrich said Bayer also is working at reducing the populations of Third World countries, investing $400 million in "long-acting contraceptives" and partnering with the Bill and Melinda Gates Foundation on "family planning initiatives."

EDITOR'S NOTE: Last year, America's doctors, nurses and paramedics were celebrated as frontline heroes battling a fearsome new pandemic. Today, under Joe Biden, tens of thousands of these same heroes are denounced as rebels, conspiracy theorists, extremists and potential terrorists. Along with massive numbers of police, firemen, Border Patrol agents, Navy SEALs, pilots, air-traffic controllers, and countless other truly essential Americans, they're all considered so dangerous as to merit termination, their professional and personal lives turned upside down due to their decision not to be injected with the experimental COVID vaccines. Bidens tyrannical mandate threatens to cripple American society from law enforcement to airlines to commercial supply chains to hospitals. It's already happening. But the good news is that huge numbers of "yesterdays heroes" are now fighting back bravely and boldly. The whole epic showdown is laid out as never before in the sensational October issue of WND's monthly Whistleblower magazine, titled "THE GREAT AMERICAN REBELLION: 'We will not comply!' COVID-19 power grab ignites bold new era of national defiance."

Content created by the WND News Center is available for re-publication without charge to any eligible news publisher that can provide a large audience. For licensing opportunities of our original content, please contact licensing@wndnewscenter.org.

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Document: Big Pharma exec: COVID shots are 'gene therapy' - CQ

For Immediate Release: August 17, 2022

Today, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Todays approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. Given the potential health complications associated with this serious disease, this action highlights the FDAs continued commitment to supporting development of innovative therapies for patients who have limited treatment options.

Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood, through mutations in the beta-globin subunit, leading to insufficient delivery of oxygen in the body. The reduced levels of red blood cells can lead to a number of health issues including dizziness, weakness, fatigue, bone abnormalities and more serious complications. Transfusion-dependent beta-thalassemia, the most severe form of the condition, generally requires life-long red blood cell transfusions as the standard course of treatment. These regular transfusions can be associated with multiple health complications of their own, including problems in the heart, liver and other organs due to an excessive build-up of iron in the body.

Zynteglo is a one-time gene therapy product administered as a single dose. Each dose of Zynteglo is a customized treatment created using the patients own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin (a hemoglobin component).

The safety and effectiveness of Zynteglo were established in two multicenter clinical studies that included adult and pediatric patients with beta-thalassemia requiring regular transfusions. Effectiveness was established based on achievement of transfusion independence, which is attained when the patient maintains a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. Of 41 patients receiving Zynteglo, 89% achieved transfusion independence.

The most common adverse reactions associated with Zynteglo included reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nosebleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus (itch).

There is a potential risk of blood cancer associated with this treatment; however, no cases have been seen in studies of Zynteglo. Patients who receive Zynteglo should have their blood monitored for at least 15 years for any evidence of cancer. Patients should also be monitored for hypersensitivity reactions during Zynteglo administration and should be monitored for thrombocytopenia and bleeding.

This application was granted a rare pediatric disease voucher, in addition to receiving Priority Review, Fast Track, Breakthrough Therapy, and Orphan designations.

The FDA granted approval of Zynteglo to bluebird bio, Inc.

###

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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

08/17/2022

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FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions -...

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For approximately 90% of all cases of amyotrophic lateral sclerosis (ALS), there is no known family history of the disease or presence of a genetic mutation linked to the disease. Although, for the remaining 5% to 10%, there is a known family history of the disease that has opened the door for gene therapy, a targeted approach that can potentially fix or block the negative effects of coding errors. Some of the most promising gene-therapybased approaches for ALS to date include antisense oligonucleotides, RNA interference, or gene editing technology such as CRISPR.

With close to 40 different genes discovered that are linked to the disease, the main issue is now validating the genes. One of these, SARM1, a variant thought to promote neuron death, is also being evaluated by several researchers from Kings College London. The project will use stem cells converted to neurons in a laboratory to firmly establish a causative role for the variant, as well as manipulate the cell culture environment and other genes to identify risk factors interacting with it.

There are several reasons why the research has not translated to therapeutic success, says Anna Underhill, BS, an investigator of the project. Underhill, a postdoctoral researcher at Kings College London, sat down with NeurologyLive to discuss the complexities of gene therapy, where the lag stems from, and the future directions of her study. She also provided detail on the uniqueness of SARM1 mutation, including a possible compensatory mechanism in the cell.

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Overcoming the Complexities With Gene Therapy in ALS: Anna Underhill, BS - Neurology Live

Dublin, Aug. 19, 2022 (GLOBE NEWSWIRE) -- The "Gene Therapy - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

Global Gene Therapy Market to Reach $2.7 Billion by 2026

Amid the COVID-19 crisis, the global market for Gene Therapy estimated at US$1.2 Billion in the year 2022, is projected to reach a revised size of US$2.7 Billion by 2026, growing at a CAGR of 19.5% over the analysis period.

Gene therapy is growing at an impressive pace, having emerged as a prospective segment in the field of biotechnology, and also presenting a bright outlook, given the increasing investments in R&D as well as mounting incidence of genetic disorders.

The rapid growth in global population, along with growing incidence of cancer and chronic disorders, introduction of novel gene therapy-based products, strong clinical evidence for therapeutic efficacy and safety of gene therapy products, favorable reimbursement policies and guidelines, and rapidly growing demand for chimeric antigen receptor (CAR) T-cell-based gene therapies are some of the key factors driving growth in the market.

Moreover, rising awareness regarding the potential of gene therapy in treating various chronic diseases, ethical acceptability of gene therapy for treating cancer, and increased government support for promoting research in gene therapy are expected to bolster the market growth in the coming years

Viral, one of the segments analyzed in the report, is projected to record 19.7% CAGR and reach US$3 Billion by the end of the analysis period. After a thorough analysis of the business implications of the pandemic and its induced economic crisis, growth in the Non-Viral segment is readjusted to a revised 17.6% CAGR for the next 7-year period. Viral segment accounts for a major share of the market.

Deployment of viral vectors in delivery of gene therapy drugs continue to exhibit progressive tide, driven by wider availability of advanced mechanisms for quicker and effective modifications of viruses such as Adeno-Associated Virus (AAV), Lentivirus, Retrovirus & Gamma-Retrovirus, Herpes Simplex Virus, Poxvirus, and Vaccinia Virus, among others.

Robust improvement in RNA, DNA, and oncolytic viral vectors is encouraging the prospects of viral vectors. Non-viral vectors are perceived as safer substitute to viral vectors, and playing an important role in redirecting pharmaceutical industries and clinicians towards gene therapy. The demand for non-viral vectors is also attributed to factors like easy characterization, mass production, enhanced reproducibility, superior transgenic capacity, and few bio-security issues.

The U.S. Market is Estimated at $628.1 Million in 2022, While China is Forecast to Reach $82.3 Million by 2026

The Gene Therapy market in the U.S. is estimated at US$628.1 Million in the year 2022. China, the world's second largest economy, is forecast to reach a projected market size of US$82.3 Million by the year 2026 trailing a CAGR of 25.1% over the analysis period. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 17.3% and 18.9% respectively over the analysis period.

Within Europe, Germany is forecast to grow at approximately 18.6% CAGR. The presence of large market players, availability of favorable reimbursement policies, prevalence of advanced healthcare infrastructure, high healthcare spending, the availability of high disposable income among the consumer base, and rising incidence of chronic diseases are some of the key factors that are expected to continue driving growth in the US market.

In Europe, rising funding for cell and gene therapy programs in countries such as the UK is expected to steer the gene therapy market in the region. European companies with commercial gene therapy portfolios are actively seeking roll out of their treatments in other regional markets by winning approvals from concerned regulatory bodies.

Growth in the Asia-Pacific region is attributed to the rising prevalence of cancer, growing government initiatives for improving healthcare infrastructure, and increasing healthcare expenditure by various countries across the region.

What`s New for 2022?

Key Topics Covered:

I. METHODOLOGY

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW

2. FOCUS ON SELECT PLAYERS(Total 154 Featured):

3. MARKET TRENDS & DRIVERS

4. GLOBAL MARKET PERSPECTIVE

III. REGIONAL MARKET ANALYSIS

IV. COMPETITION

For more information about this report visit https://www.researchandmarkets.com/r/qge6hb

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Global Gene Therapy Market Report 2022: Amid the COVID-19 Crisis, the Market is Projected to Reach a Revised Size of $2.7 Billion by 2026 -...

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The family is cautiously optimistic about the improvements they are seeing in Michael, 4, since the gene therapy three months ago

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Doctors and researchers at The Hospital for Sick Children in Toronto have conducted one of the first individualized gene therapies as part of a single-patient clinical trial.

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There is hope that this success will begin to carve a path for precision child health care and more patients will have the opportunity to receive individualized care and treatments for a wide range of conditions.

David Malkin, lead of the Precision Child Health initiative, director of the Cancer Genetics Program, and the CIBC Childrens Foundation Chair in Child Health Research at SickKids, said the idea behind precision medicine is to use the unique features of an individual to make diagnosis more precise and to predict approaches and outcomes to treatments.

The concept is that we take all information, from the postal code to the genetic code, so it is more than medicine, its overall health and everything that encompasses, said Malkin.

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This groundbreaking individualized gene therapy procedure was years in the making.

After fundraising over $3.5 million to develop the therapy, successfully testing it in mice, and finally getting Health Canada approval, Toronto-born four-year-old Michael Pirovolakis received the procedure in March to hopefully slow the progression of his ultra-rare genetic condition, SPG50.

In April 2019, Michael was diagnosed with the progressive neurodegenerative disorder spastic paraplegia type 50 or SPG50. This condition, which is caused by variants in a gene called AP4M1, causes developmental delays, speech impairment, seizures, and progressive paralysis of the arms and legs. Over the course of a few years, children lose the ability to walk and use their hands, and eventually lose their mental capacity. It is also likely to be fatal.

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Michael is currently the only known patient with the condition in Canada. It is estimated that there are around 80 other children with SPG50 around the world, making it an ultra-rare disease.

Having a child with an ultra-rare disease is difficult, said Terry Pirovolakis, Michaels father. Someone has to be watching him at all times because he doesnt understand that climbing up on the couch or opening the fridge door could be unsafe.

We love him more than anything, you know, but it is difficult, he said.

Upon diagnosis, the treatment options for SPG50 were extremely limited. So, Terry and his wife Georgia started the charity CureSPG50 to raise money to develop a gene therapy that would help their son and others with SPG50.

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Gene therapies are usually used to treat conditions caused by genetic mutations, said Jim Dowling, staff physician in the Division of Neurology and senior scientist in the Genetics & Genome Biology program at SickKids.

The idea is that through some delivery mechanism, a gene is added back to an individual, he said.

Currently, the standard way of delivery is to replace the DNA of a virus, most commonly an adeno-associated virus (AAV), with the healthy DNA of the mutated gene. An AAV is used because people do not get sick when they are exposed to it, said Dowling.

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The virus is then given to the patient in a way that is specific to their condition. Sometimes this is through an IV, sometimes into the muscle, or even into the eyeball. For SPG50, gene therapy is given into the spinal fluid so it can easily access the brain.

There are risks associated with the gene therapy procedure, specifically if the patient may develop cancer.

It was exciting that we can give Michael a better life, but scary at the same time because the last thing I ever want to do is hurt my child, said Terry

What was especially unique about Michaels gene therapy was that it was designed specifically for him and his condition, said Dowling, who led Michaels clinical trial.

This type of individualized treatment is what the doctors behind the Precision Child Health initiative at SickKids have been working towards.

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Currently, the precision child health team is gathering case studies that show the use of individualized medicine, like Michaels gene therapy, to create a plan on how to go from the discovery of disease to medical intervention.

They hope that they will soon be able to give the same specialized treatment that Michael received to many more children with many different types of conditions.

Terry said that he and his family are cautiously optimistic about the improvements they are seeing in Michael since the gene therapy three months ago. He is doing well and there are small signs that his symptoms may be improving.

We wouldnt have gotten here without the amazing people helping us along the way, said Terry. I want to thank everybody for just truly being there for us.

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Michael will hopefully not be the only child that receives gene therapy to treat SPG50.

Currently, another batch of treatment is being made with the hope of having 10 doses to give to other kids, said Terry. If all goes to plan and the U.S. Food and Drug Administration (FDA) approves the treatment, in October there will be another clinical trial in Texas.

Terry said this plan relies on CureSPG50 raising another quarter of a million dollars per child. The money is needed to cover a five-year study and hospital costs.

Our goal is to save as many kids as we humanly can, said Terry. I hope we can raise enough money to eradicate this disease.

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Canadian family raised $3.5M to develop individualized gene therapy for son's rare condition - National Post

Prescient has joined forces with Thermo Fisher Scientific for a research program aiming to advance development of its next-generation cellular therapies.

Clinical stage oncology companyPrescient Therapeutics (ASX:PTX) has signed an agreement with Thermo Fisher Scientific to accelerate development and commercialisation of a highly scalable version of its game-changing OmniCAR cell therapy platform.

Thermo Fisher is a world leader in cell and gene technologies. The agreements development plan will evaluate the potential of using automated, closed cell therapy solutions to develop a novel process of manufacturing cell therapies on Prescients OmniCAR platform using non-viral methods.

Prescient is working to develop personalised therapies to treat cancer, with the deal leveraging Thermo Fishers expertise in cell and gene therapy to create a scalable cell therapy platform. The work program aims to develop the next generation OmniCAR cells that can be produced with greater efficiency, lower costs and unmatched reproducibility.

Its been a week of positive news for Prescient, which also announced it had entered a manufacturing servicesdeal with specialist cell therapy manufacturer, Q-Gen Cell Therapeutics (Q-Gen), to produce itsOmniCAR cell lines for upcoming clinical trials.

All CAR-T therapies approved by the US Food and Drug Administration (FDA) so far, and most CAR-T therapies in development, employ viral vectors to insert genetic material into immune cells to create chimeric antigen receptors (CARs)-expressing immune cells.

Viral vectors are expensive, relatively inefficient, and time consuming to develop, often representing a key bottleneck and major cost contributor to CAR-T manufacturing.

Viral transduction processes are highly complex manual processes which are challenging for tech transfer, labour intensive and prone to operator variations, therefore producing highly variable and unpredictable results.

The work plan under this agreement will focus on creating OmniCAR cells with Thermo Fishers portfolio of proprietary equipment and specialised cell and gene therapy manufacturing expertise.

A key Prescient objective is to create a second generation of the OmniCAR platform by being able to manufacture OmniCAR cells with greater efficiency and lower costs that are suitable for tech transfer to GMP-licensed contract development and manufacturing organisations globally.

This in turn feeds into Prescients vision of decentralised manufacturing, which is best suited for multi-centre treatments, both during development and eventually for commercial roll-out.

The substantial research is forecast to take around 12 months to complete, with Prescient receiving full ownership of outcomes from the collaboration. Prescient is not required to make any cash contribution to the project.

Prescient believes that the agreement provides an opportunity for future development of other gene edits for incorporation into further-enhanced OmniCAR cell therapies to address exhaustion and immune suppression.

Prescient Managing Director and CEO Steven Yatomi-Clarke said the company elected to develop its first internal OmniCAR programs using the validated approach of lentiviral transduction.

However, he said it had always had an eye on the future for any emerging advantages that can be incorporated into its programs whilst mitigating development risk.

We are optimistic that this agreement with Thermo Fisher will accelerate the development of the OmniCAR platform to the point where one or more of Prescients internal OmniCAR programs can incorporate the advancements this agreement produces, he said.

Importantly, the agreement should result in an improved product and process that can be decentralised and therefore scaled with high efficiency and reproducibility.

Such innovation is crucial in the clinical and eventual commercial rollout of OmniCAR products and will facilitate third-party development of OmniCAR and the companys ultimate vision of a patient-centric treatment ecosystem.

Join Prescient Therapeutics Managing Director and CEO Steven Yatomi-Clarke for an investor briefing on Tuesday 23rd August at 12pm (AEST). Register for the session here.

This article was developed in collaboration with Prescient Therapeutics, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.

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Prescient joins world-leader in cell and gene therapy to accelerate OmniCAR platform - Stockhead

New DME treatments aim to extend time between intravitreal injections.

Therapies that block VEGF have become the mainstay of interventional treatment for retinal vascular disorders including neovascular age-related macular degeneration (nvAMD), diabetic macular edema (DME), and retinal venous occlusive disease.1

Over the past few years, intravitreal anti-VEGF therapy (AVT) has been shown to be very effective for patients with diabetic retinopathy (DR), reducing its severity and subsequent vision-threatening complications such as proliferative diabetic retinopathy (PDR) and anterior segment neovascularization in patients with nonproliferative DR,2 as well as center-involved diabetic macular edema (CI-DME).3

In January, the FDA approved faricimab (Vabysmo, Genentech) to treat DME and nvAMD.4 It joins ranibizumab (Lucentis, Genentech) and aflibercept (Eylea, Regeneron) for this indication, although it is not yet approved for treating DR in isolation.

Faricimab is a bispecific monoclonal antibody targeted against both VEGF-A and angiopoietin-2 receptors. In the RHINE (NCT03622593) and YOSEMITE (NCT03622580) trials, faricimab demonstrated noninferiority compared with aflibercept against CI-DME in terms of vision gained and reduction in central retinal thickness.

Trial participants randomly assigned to take faricimab gained 1 ETDRS letter and 20 to 30 m additional reduction in optical coherence tomography (OCT), central subfield thickness (CST) at 1 year, and these gains were sustained at 2 years.4

Moreover, 70% of participants taking faricimab were able to extend the time between injections to 12 or more weeks (50% were able to extend to 16 weeks), compared with 30% of participants taking aflibercept, thus reducing the burden of treatment.

In year 2, 80% of eyes were able to extend treatment intervals to 12 or more weeks with faricimab.5 No new safety signals were seen in roughly 900 participants in each arm, but it is worth noting that 5 eyes in the faricimab group versus 1 eye in the aflibercept group experienced a retinal artery occlusion, vein occlusion, or other retinal embolic eventthough none of these were associated with inflammation/vasculitis.

Brolucizumab (Beovu, Novartis) is another AVT monoclonal antibody previously approved for nvAMD. Unfortunately, postapproval analysis showed a small but definite increased risk of intraocular inflammation (IOI) and/or retinal vascular occlusion (RO), with an overall incidence of 2.4%. Risk was substantially higher in patients who had IOI/RO within 12 months of drug initiation.6

Novartis is currently seeking approval for brolucizumab in patients with DME based on results from the KESTREL (NCT03481634) and KITE (NCT03481660) studies showing noninferiority compared with aflibercept.

More than 50% of participants achieved every 12-week dosing. Further, the incidence of IOI/RO was lower than what was seen in patients with nvAMD, but was still higher than those with aflibercept (4.1% for brolucizumab versus 1% aflibercept).7

One goal of these newer therapies is extending the time between intravitreal injections required to maintain both absence of disease activity and vision gains, given the relatively short half-life of current AVT. Gene therapy introduces genetic material into patients cells to compensate for faulty genes or deliver therapeutic transgenes capable of producing therapeutic molecules.8

In diabetic retinal disease, adenovirus-associated vectors (AAVs) modified to produce anti-VEGF and other antiangiogenic or neurovascular-protective molecules are implanted within the vitreous, sub-retinal pigment epithelium (RPE), or suprachoroidal space.

Animal trials have shown success, and results from a 1 phase II human trial showed improvement in DR severity sans DME at 6 months,9 whereas another trial in participants with CI-DME was terminated early due to safety concerns that included hypotony, inflammation, and vision loss.10 Retina specialists are optimistic that AAVs hold great promise for both DR/DME and AMD.

Another pathway distinct from VEGF, but directly implicated in the pathogenesis of DME, is plasma kallikrein (PKal), a protein synthesized in the liver that mediates vascular leakage and inflammation, levels of which are elevated in the vitreous of patients with diabetic retinopathy.11

Results from KALAHARI (NCT04527107), a phase II study of PKal inhibitor THR-149 (Oxurion), showed 6.1 letters of visual acuity improvement and CST reduction of 100 m after 3 monthly injections in participants with DME suboptimally responsive to a minimum of 5 prior anti-VEGF injections (baseline BCVA ranging from 20/40 to 20/160, with baseline CST averaging 421 m). At 6 months, 50% of participants had a 2-line improvement in BCVA with no additional rescue therapy.

The study was small20 total participantsand only the highest dose affected acuity gains, but these findings offer additional hope of improved vision for patients who dont respond adequately to AVT.12

Integrins are transmembrane receptors that allow cell-to-cell and cell-to-extracellular matrix adhesion and biochemical signal transduction. They have been implicated in DR and DME by activating growth factor receptors both upstream and downstream from VEGF.13

The integrin antagonist THR-687 (Oxurion) was assessed in a phase 1 trial (NCT03666923) at 3 doses in 12 participants with DME previously treated with AVT, mean visual acuity of 20/80 and mean CST of 542 m.

Mean improvement of 7.2 letters was seen at 1 week and 9.2 letters at 1 month, as well as a 106-m decrease in CST at 2 weeks that waned to a 37-m reduction by month 3.

A phase 2 trial (NCT05063734) of THR-687 in treatment-nave participants with DME is due for completion in August 2023. Several other anti-integrin therapies are in clinical trials, including at least 1 self-administered eye drop that reaches the posterior segment.14,15

Another possible treatment for DME is photobiomodulation (PBM), an LED or LASER application of visible light (typically 670 nm) that activates cytochrome-C oxidase within retinal mitochondria, enhancing cellular metabolism (ATP production) and reducing reactive oxygen species that play a critical role in diabetic eye disease.

Results of a study of patients with CI-DME, CST greater than 300 m, and best-corrected vision ranging from 20/30 to 20/200 showed a significant 59-m reduction at 2 months (p = 0.03), though acuity results were not reported.16

Investigators in a recent clinical trial from the DRCR Retina Network (Protocol AE) compared placebolow energy, broad spectrum white lightwith PBM for 90 seconds, twice daily for 4 months in 135 participants with center-involved DME and good vision (> 20/25).15 Unfortunately, both mean CST and vision in the treated group worsened only 2 m/0.4 letters less in the PBM group versus placebo (both insignificant).17 This suggests PBM may be most effective for those with more substantial DME.

Of note, top-line data from the LightSite III trial (NCT04065490) showed that using PBM at 3 wavelengthsyellow, red, and near infraredfor intermediate dry AMD yielded a 5.5-letter improvement in the treatment (n = 91 eyes) versus sham treatment (n = 54 eyes) arms at 13 months.18 The investigation of PBM for both AMD and DME continues.

There now are multiple therapies for treating both DME and DR, with more on the horizon. This is particularly good news for those patients who dont respond well to traditional therapies and may reduce the treatment and vision burden of these all-too-common disorders.

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It's not your grandparents' anti-VEGF therapy - Optometry Times

Alzheimer's has been reversed in mice after scientists boosted the formation of new brain cells. A gene therapy fuelled neurons in the hippocampus - a region vital for learning and remembering.

The breakthrough could lead to new treatments. The number of dementia cases worldwide will triple to 150 million by 2050. There is no cure. Lead author Professor Orly Lazarov, of the University of Illinois, Chicago, said: "Taken together, our results suggest augmenting neurogenesis may be of therapeutic value."

Experiments have shown the process is impaired in patients and mice with mutations linked to Alzheimer's - particularly in the hippocampus. The US team found increasing production of neurons rescued the lab rodent's defects.

They were incorporated into memory circuits - restoring normal function. The study offers hope of a viable strategy. Current drugs target just the symptoms - and not the cause.

Brain cells send electric signals. We keep making them throughout our lives. They are produced by neural stem cells. But numbers tail off as we age - and fall dramatically in Alzheimer's. Evidence is improving neurogenesis holds the key to curing dementia.

The hippocampus is the region of grey matter you need, for instance, to remember where you parked your car. Prof Lazarov said: "However, the role of newly formed neurons in memory formation, and whether defects in neurogenesis contribute to the cognitive impairments associated with Alzheimer's, is unclear."

In the study, stem cell survival was enhanced by deleting a gene called Bax - leading to the maturation of more neurons. Afterwards, the animals regained the animals' regained their spatial recognition and contextual memory skills. tests included finding their way around a maze.

Scans of healthy mice showed the circuits involved in storing memories include many newly formed neurons alongside older ones. Neurons were fluorescently labelled - lighting up as they were activated during acquisition and retrieval.

The memory-stowing loops of mice with Alzheimer's contained fewer. But integration of newly formed brain cells was restored when neurogenesis was increased. Further analyses revealed there was also a rise in the number of tiny protrusions called dendritic spines. They connect neurons - and are critical for memory formation.

When the researchers specifically inactivated the new neurons, mice with dementia lost any improvement in memory - confirming the results. Prof Lazarov added: "Our study is the first to show impairments in hippocampal neurogenesis play a role in the memory deficits associated with Alzheimer's by decreasing the availability of immature neurons for memory formation."

Alzheimer's and other forms of dementia affect more than 920,000 people in the UK - a figure that will reach teo million within three decades. The study was published in the Journal of Experimental Medicine (JEM).

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Alzheimer's breakthrough: Scientists reverse disease with gene therapy - Nottinghamshire Live