Archive for the ‘Gene Therapy Research’ Category

Family members noticed initial signs that their children could recognize sounds two weeks after application of the therapy.

(CN) Five children born deaf can now hear in both ears after a trial of a novel gene therapy focused on repairing a hereditary dysfunction that prevents auditory signals from reaching the brain, according to authors of a study published in the journal Nature Medicine Wednesday.

The success of the therapy in both ears demonstrates that the treatment can restore a full range of hearing functions including identifying the locations of sounds, recognizing specific sounds in noisy environments and the ability to perceive and develop speech.

The researchers used a minimally invasive surgical procedure to inject vector viruses into the inner ear. The viruses transfer genes into specialized cells that convert physical sound waves into the electrical signals that can be transmitted by nerve cells.

The new genetic material allows the cells to correctly replicate a protein that is necessary to send the converted electrical signals from the inner ear to the brain.

Family members noticed initial signs that their children could recognize sounds two weeks after application of the therapy. All the children were born deaf, four of them were between one and three years of age and the fifth participant was an 11-year-old.

At four to six weeks the children would turn to face the sound when a parent called to them from outside of their plane of view. At 13 to 15 weeks children could dance to music and form simple syllables, and at 26 weeks the children used complete words and showed more advanced communication.

Its very emotional for the whole family and even for my team, some of my students were so touched that they began to cry, said lead study author Dr. Yilai Shu, director of the Diagnoses and Treatment Center of Genetic Hearing Loss affiliated with the Eye and ENT Hospital of Fudan University in Shanghai.

The successful trials grew out of a collaboration between Shu and the studys co-senior author Zheng-Yi Chen, associate scientist in the Eaton-Peabody Laboratories at Mass Eye and Ear in Boston. Shu was a student of Chens and they worked together during Shus postdoctoral fellowship at Mass Eye and Ear. Shu returned to China over a decade ago, but the two scientists continued to collaborate.

The research team conducted the trials at the Eye and ENT Hospital of Fudan in Shanghi, China.

The five children that participated have a specific genetic disorder caused by the mutation of a single gene that prevents the cells in the inner from replicating the protein otoferlin that is crucial for sound signal transmission via the nervous system.

Globally, 430 million people have some form of disabling hearing loss, according to the World Health Organization. About 26 million people were born with hearing loss and 60% of those cases are due to genetic factors, the study's authors say.

This really opens a new era in treating hearing loss because there are over 150 genes that can cause genetic deafness, Shu said.

In animal trials, the research team has shown that gene therapies are effective in treating several of these forms of genetic deafness, said Chen. The teams successful treatment of the otoferlin genetic deficiency in humans means that the other models with proven success in animals can now move into human trials.

Eventually, the researchers hope to develop gene therapies for noise and age-related deafness as well.

One of the challenges of using the viral vector gene therapy with the otoferlin gene is that the gene itself is too large for the virus to carry, said Shu. To overcome this the researchers used a new technique that involves splitting the gene in half and allowing viruses to carry each half into the cells where the gene can recombine.

This is actually a technology breakthrough, said Chen, So many genes for human diseases are also very big so this approach can be broadly applied in gene therapies to treat other diseases, not only deafness.

In January the research team published the results of a trial conducted in 2022 which successfully tested the therapy in one ear. The bilateral trial involving two ears, which has allowed for participants to recover a full range of hearing functions including ability to detect the location of sound, requires a procedure for each ear resulting in an increased dosage of the viral vector.

The researchers choose to test the therapy in one ear first because the additional dosage of the viral vector is more likely to trigger an immune response which can lead to adverse affects. All five children involved in the bilateral study recovered their hearing and the adverse affects that researchers detected such as fever or elevated white blood count were well below the safety threshold.

Chen, who is also an associate professor of OtolaryngologyHead and Neck Surgery at Harvard Medical School said that international cooperation has been important to developing the treatment and Shu agrees.

This type of research is very difficult and relies on decades of international study, Shu said.

We want to use this as an example that there is a future for this kind of collaboration, Chen said. Its not a political issue its really just for human health and a common benefit for all of us.

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Children born deaf react to sound after successful gene therapy trial - Courthouse News Service

All five children taking part in a Shanghai-led clinical trial of a gene therapy to treat congenital hearing loss showed significant restoration of hearing in both ears, researchers said on Wednesday.

Their speech function and ability to locate the sources of sound were also greatly improved, researchers from the Eye and ENT Hospital of Fudan University in Shanghai said.

It was the world's first clinical trial of a gene therapy for both ears, they said.

The children, aged between 1 and 11, all have autosomal recessive deafness 9, mainly caused by mutation of a particular gene, OTOF. The research team injected gene therapy medicine developed in its previous studies into both ears of the patients in the same session, using minimally invasive and microscopic injection methods.

"Good safety of the therapy was demonstrated during their follow-up observations, proving that the binaural gene therapy with the treatment known as AAV1-hOTOF was safe and effective," said Shu Yilai, one of the leading researchers and director of the inherited deafness diagnosis and treatment center affiliated with the Shanghai hospital.

A paper about their research, a joint effort with Harvard Medical School associate professor Zheng-Yi Chen, was published recently in the journal Nature Medicine.

There are an estimated 26 million people with congenital hearing loss worldwide, and approximately 30,000 infants are born with the condition in China each year. Experts said that 60 percent of them are related to genetic factors, which severely damage their speech, cognition and intellectual development. There is, as yet, no effective clinical therapy.

People with the OTOF mutation usually suffer from severe or even complete hearing loss and speech impairment. In China, among the infants and young children diagnosed with auditory neuropathy spectrum disorder, up to 41 percent have a mutation in the OTOF gene.

Gene therapy is widely considered by experts to be one of the most promising strategies for curing hereditary deafness. It can deliver genes with normal function directly to the inner ear through a delivery vehicle, fundamentally restoring or improving hearing for such patients, the research team said.

It began recruiting participants from China for the clinical trial to receive treatment in one ear in October 2022, and completed the treatment of the first patient outside China in December that year.

Six patients were included in the trial to receive gene therapy in one ear, and the longest follow-up time for a participant has now reached 17 months, with the young patient later able to hold daily conversations.

Shu shared the clinical trial data at the annual conference of the European Society of Gene and Cell Therapy, one of the world's most authoritative international academic conferences in the field of gene and cell therapy, in Belgium in October.

The results of the clinical research that used gene therapy in one ear were published in The Lancet in January. International peers said it could open a new era for treating hearing impairment and even more diseases through gene therapy.

The clinical trial of the gene therapy in both ears began recruiting participants in July last year.

"We took a step further to try to restore the natural ability of human hearing in both ears for the patients," Shu said. "It'll also help to restore their ability to hear three-dimensional sound, locate sound sources, and distinguish speech in the context of noise."

He shared the latest progress in the trial with experts and scholars from around the world during the annual conference of the American Society of Gene& Cell Therapy held last month in Baltimore in the United States.

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Gene therapy improves deaf children's hearing - ecns

Visiongain has published a new report entitled Gene Therapy R&D Market Report 2024-2034: Forecasts by Disease (Cancer, Rare Diseases (Oncologic, Non-oncologic), Cardiovascular Diseases, Ophthalmic Diseases, Haematology, Neurological, Diabetes Mellitus, Other)), by Vector (Viral (Retrovirus, Adenovirus, AAV, Lentivirus, Others), Non-viral (Naked DNA, Gene Gun, Electroporation, Lipofection)), by Techniques (Gene Augmentation Therapy, Gene Replacement Therapy), by Participants (Small/Medium Pharma & Biotech, Universities & Research Institutes, Hospitals, Government & Public Bodies, Big Pharma) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies AND COVID-19 Impact and Recovery Pattern Analysis.

The gene therapy R&D market is estimated at US$3,641.7 million in 2024 and is projected to grow at a CAGR of 29.8% during the forecast period 2024-2034.

Gene Therapy: Shaping Tomorrow's Healthcare Landscape Through Innovation and Investment

Over the past five years, gene therapy has seen a remarkable resurgence fuelled by significant strides in virology, vector and capsid architecture, and manufacturing technologies. These advancements have sparked a surge in promising outcomes, attracting the attention of biotech investors and reigniting interest in the field. This renewed enthusiasm has spurred a flurry of strategic manoeuvres among biopharma firms vying for an edge in this burgeoning market.

Recent advancements in gene therapy are grounded in a deeper comprehension of viral vectors, pivotal for delivering therapeutic genes to target cells. Refinements in vector design and capsid engineering have bolstered the efficiency, specificity, and safety of gene delivery. Concurrently, progress in manufacturing processes has bolstered the scalability and consistency of gene therapy products, enabling the production of treatments for larger patient cohorts.

The market for gene and cell therapies (CGTs) is expanding rapidly, with over 1,400 companies globally focusing on CGTs and more than 3,500 therapies in preclinical and clinical development. Presently, there are over 2,000 active clinical trials, with North America leading with 964 trials, trailed by the Asia Pacific with 848, Europe with 244, and other regions with 139 active trials. Oncology and rare diseases emerge as the primary therapeutic domains targeted by CGTs, underlining the potential to address unmet medical needs and furnish innovative treatment avenues.

Investment in the CGT sector remains robust, with venture capital financing being the primary growth catalyst. The persistent inflow of capital underscores a strong belief in scientific breakthroughs and the potential to revolutionize the treatment landscape for a spectrum of diseases, encompassing both rare and common ailments.

Despite significant progress and optimism surrounding gene therapy, the field confronts several challenges, including high treatment costs, intricate regulatory requisites, and the imperative for long-term safety and efficacy data. Tackling these obstacles demands sustained collaboration among industry stakeholders, regulatory bodies, and the scientific community.

Looking ahead, the future of gene therapy holds promise. Ongoing technological advancements, coupled with substantial investment and supportive regulatory frameworks, are poised to propel further innovations and broaden the therapeutic applications of gene therapy. As the industry matures, gene therapy stands to revolutionize the treatment paradigm for a diverse array of diseases, offering newfound hope to patients who previously faced limited or no treatment options.

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How has COVID-19 had a Significant Impact on the Gene Therapy R&D Market?

The COVID-19 pandemic fundamentally transformed the Gene Therapy Research and Development (R&D) market, presenting a combination of challenges and opportunities that both accelerated and impeded progress. On the positive side, the urgent demand for COVID-19 treatments and vaccines spurred technological advancements, particularly in mRNA and viral vector technologies crucial to gene therapy. This urgency drove unprecedented levels of funding, collaboration, and resource allocation, fostering innovation and expediting the translation of gene therapy concepts into clinical applications. Regulatory bodies responded by streamlining approval processes for urgent therapies, potentially benefiting future gene therapy products.

The pandemic underscored the importance of robust manufacturing and distribution networks, prompting significant investments in scaling up production capabilities for gene therapies. Conversely, the pandemic caused substantial disruptions in clinical trials, with many studies delayed or halted due to lockdowns, travel restrictions, and the prioritization of COVID-19 related research, leading to setbacks in project timelines. Supply chain interruptions also impacted the availability of critical materials and reagents, complicating R&D efforts. Despite these challenges, the pandemic highlighted gene therapy's potential to address genetic disorders and emergent viral threats, reinforcing its strategic importance. The industry's resilience and adaptability were evident in its response, suggesting that the lessons learned will strengthen future gene therapy R&D efforts.

Furthermore, the pandemic emphasized the importance of global collaboration, with international partnerships playing a crucial role in accelerating the development and distribution of new technologies. Consequently, the gene therapy market is poised for sustained growth, driven by technological advances made during the pandemic and increased recognition of the value of rapid, adaptable therapeutic development platforms. Overall, while the COVID-19 pandemic presented significant obstacles, it also served as a catalyst for innovation and structural improvements within the gene therapy R&D landscape, positioning the industry for a stronger and more agile future.

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Visiongains 340-page report provides 156 tables and 192 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the gene therapy R&D market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Gene Therapy R&D. Get financial analysis of the overall market and different segments including disease, vector, techniques, participants, and capture higher market share. We believe that there are strong opportunities in this fast-growing gene therapy R&D market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

Robust Gene Therapy Pipeline Anticipated to Boost Growth

Cell and gene therapies (CGTs) are transitioning from research labs to clinical settings, offering targeted treatments for specific diseases, unlike the broader targets of small-molecule therapies. These therapies aim to introduce healthy cells or correct genetic defects in patients, with cancer emerging as a primary focus, representing nearly half of all CGTs in clinical trials.

One promising approach is CAR-T therapy (Chimeric Antigen Receptors), which combines cell and gene therapy to combat cancer. By modifying a patient's immune cells' DNA, CAR-T therapy enhances their ability to recognize and attack cancer cells. FDA approval for two CAR-T therapies signifies their efficacy, particularly for cancer patients with limited treatment options. Continued research endeavours hold the potential to expand the availability and efficacy of these therapies across a broader spectrum of cancers.

Recent data show a notable increase in gene therapies entering Phase III trials, signalling progress and momentum in this field.

Technological Advancements Projected to Fuel Market Growth

Gene therapy, both as a cutting-edge medical technique and a burgeoning biomedical business, holds a promising future propelled by technological advancements and industry promotion. While genome editing technologies enable precise manipulation of specific genes within DNA sequences, concerns regarding off-target effects, which may inadvertently edit genes with similar sequences, pose challenges such as tumorigenicity. Despite these challenges, genome editing offers the potential for lasting genomic changes, albeit with careful consideration of safety implications.

Moreover, the market for digital bioprocessing technology is driven by the demand for reproducible, efficient, and cost-effective development of cell and gene therapies. These advancements are pivotal for realizing the commercial potential of these therapies in the coming years, enhancing market penetration, and reducing overall therapy costs. Notably, breakthroughs like the use of gold nanoparticles for gene therapy delivery by researchers at the Fred Hutchinson Cancer Research Center signify promising alternatives to conventional delivery methods, potentially revolutionizing scalability and accessibility.

CRISPR-Cas9, a transformative genome editing tool, is revolutionizing scientific research with its versatility, speed, cost-effectiveness, and accuracy compared to previous methods. Its applications span across animal research, human gene therapy, medical research, and plant science, facilitating precise gene targeting, alterations, insertions, deletions, and single base pair conversions. As breakthroughs in this field continue to unfold, the gene therapy R&D market is poised for significant growth in the forecast period.

Get Detailed ToC https://www.visiongain.com/report/gene-therapy-market-2024/

Where are the Market Opportunities?

Artificial Intelligence Can Bring Value to Gene Therapy R&D

While fully AI-generated drug discovery hasn't yet yielded approved drugs, the number of drugs in development with AI-associated platforms is steadily increasing. In 2023 alone, at least 19 drugs attributed to AI were in clinical development, with potential advancement in the clinical pipeline anticipated for 2024.

AI holds significant potential in various stages of cell and gene therapy R&D, with McKinsey & Company identifying its greatest impact in target identification, payload design optimization, and translational and clinical development. For instance, AI and machine learning (ML) enhance target selection to optimize therapeutic success.

In viral therapeutics aiming for genome editing, algorithms predicting CRISPR target sites aid in identifying genomic locations conducive to efficient editing with minimal off-target activity. For mRNA-based vaccines, AI predicts tumour epitopes for therapeutic molecule binding. Similarly, in CAR T-cell therapies, AI facilitates antigen and binding site identification, enhancing CAR design for improved activity and reduced cytotoxicity.

AI and ML models rapidly screen numerous candidates and select designs meeting desired criteria, especially when integrated into an AI-enabled closed-loop research system. This system automatically feeds initial screening results into an ML pipeline, which learns from computational features to suggest optimized payload candidates for experimentation. Experimental data then feed back into the system, perpetuating learning and closing the research loop.

In translational and clinical development, AI and ML mitigate safety risks in clinical trials by identifying translational biomarkers indicative of success, selecting appropriate patients, estimating optimal dosing, and predicting severe adverse events based on patient profiles and real-world data from similar treatments.

Facility Expansion Anticipated to Offer Lucrative Growth Prospects

The surge in clinical-stage start-ups has led to a scarcity of viral vector manufacturing capacity among contract manufacturers, upon which new gene and cell therapy companies heavily rely for early-stage development. As these firms mature into commercial entities, they often opt for in-house manufacturing to circumvent outsourcing complexities. Consequently, biotech companies are formulating growth strategies, establishing internal teams, and seeking site consultant assistance. These experts aid in the rigorous search for suitable lab and development facilities, or, increasingly, in securing new construction sites in tight real estate markets.

These internal capabilities empower gene and cell therapy companies to swiftly scale up production from clinical batches to commercial scale, even during the research and development phase. Moreover, co-locating with drug research and development operations facilitates seamless technology transfer and minimizes disruption, especially during clinical trials.

The imperative for expedited "time-to-market" underscores the focus on existing buildings, which are increasingly scarce in established biotech hubs due to market demand. These hubs offer advantages such as tailored university programs and engagement with other gene and cell therapy companies, both as competitors and potential collaborators, creating a fertile environment for talent acquisition. While cost sensitivity is paramount for all ventures, venture-funded enterprises are particularly keen on cost reduction, necessitating efforts to minimize both upfront and ongoing cash outlays.

Competitive Landscape

The major players operating in the gene therapy R&D market are Astellas Pharma Inc., American Gene Technologies, Applied Genetic, Bayer, Benitec BioPharma, Biogen, Bluebird Bio, Bristol Myers Squibb, Calimmune, Inc. (CSL Behiring), Cellectis, GenSight Biologics, Gilead Lifesciences, Inc., Novartis AG, Orchard Therapeutics, Oxford Biomedica, Pfizer, Inc., REGENXBIO Inc., Sangamo Therapeutics, Inc., Sanofi, Spark Therapeutics (Subsidiary of Roche), Takeda Pharmaceuticals, Transgene, UniQure N. V., Voyager Therapeutics, ViGeneron, GQ Bio Therapeutics GmbH, OCUGEN, INC., Taysha GTx, and Sarepta Therapeutics, Inc.. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.

Recent Developments

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Gene Therapy R&D market is projected to grow at a CAGR of 29.8% by 2034: Visiongain - GlobeNewswire

Five children with congenital deafness have had their hearing significantly improved after receiving gene therapy, the Eye, Ear, Nose and Throat Hospital of Fudan University said on Wednesday.

The children, between the ages of 1 and 11 all suffered hereditary deafness due to a genetic mutation.

Around 30,000 children are born deaf in China each year with 60 percent of cases related to genetic defects, seriously impacting their language, cognition and intelligence development. There is no effective medication so far.

Gene therapy is a promising treatment for hereditary deafness, taking effect by injecting properly functioning genes directly into the inner ear.

Dr Shu Yilai led his team at the hospital to develop a genetic medicine targeting deafness due to the mutation with a precise and minimally invasive drug delivery route and equipment.

Since December 2022, six children have received an injection to one ear. The first, who has been followed for a year and five months, has been able to develop proper daily communication.

To achieve a bigger benefit to patients and help them regain their hearing and build 3D and accurate auditory localization, doctors started gene therapy on patients' both ears in July 2023.

The world's first gene therapy on deafness genes in both ears has testified to the safety and effects of gene therapy in congenitally deaf people and greatly boosts its development, showing a strong potential of gene therapy in congenital deafness treatment, experts said.

The research was published by world-leading journal Nature Medicine.

The research is published in world-leading journal Nature Medicine.

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Gene therapy in both ears proves a success for deaf children - SHINE News

From Philly and the Pa. suburbs to South Jersey and Delaware, what would you like WHYY News to cover? Let us know!

Within the last decade, cell and gene therapy research and manufacturing have produced new options and hope for people with cancer, inherited or rare disorders, and chronic illnesses that may otherwise be fatal or severely debilitating.

The field is still in its infancy, but scientists are optimistic that these new therapeutics will lead to major advancements in how health care providers can treat, prevent or cure disease.

And a growing number of cell and gene therapies companies and experts doing this kind of work are calling Philadelphia home, according to a new report by the Chamber of Commerce for Greater Philadelphia.

Claire Greenwood, chamber senior vice president of economic competitiveness, said over time, this could make the region a top hub for innovative research and breakthroughs, and make it a major competitor with markets in Boston and San Francisco.

I think what weve seen and what well continue to see is when our institutions invest in really cutting-edge science, if we bring all the other pieces together, were going to see advantages around how that allows us to compete, Greenwood said.

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Philadelphia cell and gene therapy industry sees rapid growth - WHYY

The PlasmidFactory GmbH was founded in 2000 in Bielefeld/Germany, with four employees. In the meantime, under the leadership of the Founder and Managing Director , Dr Martin Schleef, the company has become a well-known contract manufacturer (CDMO) for plasmid and minicircle DNA. Today, PlasmidFactory has 50 highly qualified employees. For more than four years now, the company has been working at full speed and on an extraordinary scale - in addition to scientific research and optimisation of production especially for production of mRNA vaccines. Dr Schleef shares his insights about PlasmidFactory and his rich experience in developing life-saving products.

You have been working in plasmid research and production for more than 30 years and have played a crucial part in contributing to the development of the COVID-19 mRNA vaccine. How did this come about?

PlasmidFactory specialises in producing DNA of extraordinarily high purity for gene therapy and genetic vaccine research. We are an established supplier to biotechnology and pharmaceutical companies as well as to universities and research institutes worldwide. We are well-known within the industry. All manufacturing, research and development is concentrated at the Bielefeld site in Germany. Our manufacturing processes for plasmid DNA are ideally suited for producing mRNA vaccines based on it.

Was the rapid growth in demand for mRNA for vaccines surprising?

Not really. Plasmids have been used as starting materials for manufacturing viral vectors (AAV, Lenti, etc.), and for producing RNA for quite some time. In particular, the production of plasmid DNA as a starting material to produce RNA cancer therapeutics and vaccines has become very important, especially against the background of the COVID-19. RNA is considered a promising vaccine candidate for the prevention of certain viral infections. It has the advantage of neither integrating into the genome of the cell nor remaining in the long term as a potentially effective active molecule in a patient's body. Besides protection against COVID-19, recently developed mRNA-based vaccines have been tested for their protection against influenza virus, Zika virus, cytomegalovirus, and many others. In addition, various mRNA vaccines are currently being tested for their tolerability when used as combination vaccination. So, as you can see, the current applications for mRNA are not yet exhaustive, and we look forward to continuing to play an essential role in the value chain.

Large-scale manufacturing and production in the highest purity grades of plasmid and minicircle DNA as a starting material for RNA vaccines has gained importance. How did you ensure that PlasmidFactory can handle both parameters simultaneously?

We have been working in this area for several years and are constantly optimising it. Even before COVID-19, we significantly expanded our capacity in 2020 to meet the orders of our national and international customers. In the summer of 2020, the concept for the laboratory expansion was finalised. We received funding from the NRW state government and have been able to produce on a multi-gram scale in High Quality Grade to support the COVID-19 vaccine industry.

Moreover, investments have been made to implement the manufacturing of DNA in full GMP Grade. The newly built GMP facility is also located in Bielefeld and will be up and running by the end of summer 2024. This will enable us to provide our global customers not only with High Quality Grade but also with GMP Grade plasmid and minicircle DNA on a large scale.

You mentioned the term "High Quality Grade". Is it your creation, and what exactly does it mean? How does it differ from GMP Grade?

Yes, initially, it is, but it denotes "high quality", so the term is now also used by other manufacturers.

Our "High Quality Grade" plasmid DNA was established over 15 years ago based on the EMA guidelines CHMP/BWP/2458/03, CPMP/BWP/3088/99, and, since 2021, on EMA/246400/2021 for highest quality requirements. For product safety reasons, the manufacturing process avoids using substances of animal origin throughout the entire production chain. It guarantees the highest possible product purity through reliable separation of impurities, e.g., bacterial chromosomal DNA or damaged plasmids. To prevent further contamination, only one plasmid is produced at a time in the facility used exclusively for High Quality (HQ) Grade plasmids; no parallel plasmid productions occur in the same facility.

The HQ fermentation is physically separated from the purification (chromatography) to ensure that downstream processing of the sensitive DNA is not affected by live contaminants.

Actually, HQ Grade products are already being used in clinical trials studies. However, GMP Grade goes one step further by exclusively using single-use equipment and complying with applicable GMP guidelines, and it is, of course, GMP certified. This is a stand-alone trait and signifies another important step in PlasmidFactorys goal to further extend the lead over the competition.

What unique expertise do you have in this field?

The proprietary, unique purification process is one of our unrivalled advantages. It results in a high grade of pure, supercoiled (ccc) plasmid monomers that meet regulatory requirements to form a defined, homogeneous product, which undergoes a series of cell bank and plasmid DNA product quality control checks before release.

High-quality Grade Plasmid DNA is produced based on a cell bank (RCB) created at PlasmidFactory and the uniquely effective proprietary ccc Grade DNA technology. For both the cell bank and the plasmid DNA product, PlasmidFactory offers a wide range of quality controls, so a product is ultimately created that is tailor-made for the respective application or as per the corresponding regulatory requirements.

For example, our High Quality Grade Plasmid DNA is used in the GMP-compliant production of recombinant viruses, antibodies and RNA for clinical trials.

Our products and processes are continuously and precisely optimised and, if necessary, further developed because we want to be uncompromising in quality and competence.

HQ and GMP Grade DNA are required not only for vaccine production but also in the field of cancer research. Could you elaborate?

That's right, with our national and international customer base, we at PlasmidFactory are also well positioned in other research areas.

Exciting and no less important is the so-called CAR-T cell development. Ongoing research and advancements in CAR-T-cell technology currently unlock new possibilities for personalised and targeted cancer treatments. This groundbreaking approach has demonstrated remarkable success.

PlasmidFactory has developed and patented a method for producing CAR-T cells. In contrast to conventional methods, no viral vectors are used here, but PlasmidFactory's proprietary minicircle technology. Corresponding products are currently undergoing clinical trials.

Has the proprietary minicircle (MC) technology contributed to the success of CAR-T cell therapy?

Yes, without "MC" it does not work: Minicircle DNA contains practically only the "Gene of Interest" (GOI). Unnecessary sequences used only for the plasmid production process are completely removed. A safe and highly effective vector system is the result. It already meets the future regulatory requirements for gene therapy and vaccination.

We also produce customised minicircles using our unique, patented method: the plasmid containing GOI is the starting material. This is inserted into the so-called "parental plasmid". From this, the minicircle DNA molecule is produced by recombination, which consists almost exclusively of GOI.

The minicircle DNA, produced with our proprietary technology, is patented for use in CAR-T cells worldwide and is exclusively available at PlasmidFactory.

What kind of R&D activities do you have at PlasmidFactory?

The R&D activities of the PlasmidFactory are carried out in our laboratories, as well as in close cooperation with national and international partners.

For example, in the fields of:

- Optimisation of vectors to produce viral vectors (AAV or LV) or for efficient antibody or RNA production

- Development of resistance gene-free vector systems (e.g., minicircles)

- Investigation of the influence of various factors on the long-term stability of plasmid DNA (e.g., plasmid size, DNA concentration, storage medium, freezing and thawing conditions)

- DNA vaccines

- High cell density cultivation

- Single use technology in process technology

- Linear DNA vectors with loops at their ends (MIDGE)

- Vector development and gene transfer

Besides these scientific collaborations, we are also implementing strategic partnerships. In 2022, we partnered with ARCHIMED, a leading investment firm focused exclusively on healthcare industries. With ARCHIMED at our side, we are strengthened in expanding our business globally.

In addition, we have also found a more specific partner to support us in entering the Indian market.

Does this mean you have a new strategy to meet the increasing demand for highest quality DNA in the Indian market?

Yes, we do. After the success in handling COVID-19 in India, a large number of biotechnology and pharmaceutical firms are investing heavily in mRNA R&D. We are pleased to introduce Dr Nagaraj Rao, RRR Labs, Navi Mumbai, as our new partner in India. Dr Rao is well-known in the Indian pharma industry and among Indian biopharmaceutical and vaccine manufacturers for providing customers with state-of-the-art media and feeds for mammalian cell culture in recent years. Having lived in Germany for a decade earlier, his strong support and his role in bridging communication gaps between Indian and German companies play a crucial role in the success of such technology-driven businesses.

Being a biologist, how are you able to balance the business and research activities?

I am a biologist and researcher who manages the PlasmidFactory family. PlasmidFactory's products are manufactured by an energetic team of motivated colleagues.

Our work here is in the service of science, but of course, science also meets entrepreneurship in our company. We remain researchers for researchers!

With new convincing ideas and unique techniques, we want to advance biotechnology together - a simple part of my DNA.

Continued here:
We specialise in producing DNA of extraordinarily high purity for gene therapy and genetic vaccine research - BSI bureau

MALVERN, PA Ocugen, Inc. (NASDAQ: OCGN) announced a positive outcome from the Data and Safety Monitoring Board (DSMB) review of its Phase 1/2 ArMaDa clinical trial for OCU410, a gene therapy candidate aimed at treating geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dAMD). GA affects approximately 2-3 million people in the U.S. and Europe.

To date, six patients with GA have participated in the trial. Three received a low dose of the therapy, while three others received a medium dose. The next phase will involve dosing an additional three patients with a high dose of OCU410.

The DSMB has recommended to proceed with dosing subsequent GA subjects with the high dose of OCU410 in the dose-expansion phase of the study and concurrently initiate Phase 2 dosing, said Dr. Peter Chang, Chair of the DSMB for the OCU410 clinical trial. No serious adverse events related to OCU410 have been reported to date in both low- and medium-dose cohorts. I believe that this marks a critical next step towards determining the maximum tolerated dose for OCU410 and is an important milestone for its clinical development.

Huma Qamar, Chief Medical Officer of Ocugen, expressed optimism about the therapys potential. We are delighted to report a second positive DSMB recommendation for the treatment of GA, which significantly builds on the favorable safety and tolerability profile exhibited by OCU410, she said. We are very enthusiastic about the potential of OCU410 as a potential one-time treatment for GA with a single sub-retinal injection.

The ArMaDa trial aims to assess the safety and efficacy of OCU410 administered subretinally. This trial is structured in two phases:

Phase 1: This is a multicenter, open-label, dose-ranging study. It includes three dose levels: low (2.510 vg/mL), medium (510 vg/mL), and high (1.510 vg/mL).

Phase 2: This phase will be a randomized, outcome assessor-blinded, dose-expansion study. Participants will be randomly assigned to one of two OCU410 treatment groups or an untreated control group in a 1:1:1 ratio.

The DSMBs positive review is significant for several reasons.

Safety and Tolerability: Initial results indicate that OCU410 is safe and well-tolerated at both low and medium doses. This builds confidence in the therapys potential for broader application.

Innovative Treatment Approach: Current treatments for GA target only the complement pathway and require multiple annual injections. OCU410 offers a different approach by addressing several pathways, including complement, lipid metabolism, inflammation, and oxidative stress. This could provide a more comprehensive and long-term benefit to patients.

Reduced Treatment Burden: If successful, OCU410 could reduce the number of required treatments to a single sub-retinal injection. This would significantly ease the treatment burden on patients compared to the frequent injections needed with existing therapies.

Gene therapy represents a frontier in medical research, offering hope for conditions that currently have limited treatment options. The success of OCU410 could pave the way for similar therapies targeting other complex diseases. As the population ages, the incidence of age-related conditions like GA is expected to rise, making innovative treatments even more crucial.

Ocugens progress with OCU410 thus far signals a promising development in the fight against dAMD. If the therapy continues to show positive results, it could transform the standard of care for millions of patients affected by this debilitating condition.

For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and Microsoft Start.

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Ocugen's Gene Therapy Shows Promise in Early Trial for Age-Related Macular Degeneration - MyChesCo

The Bioethics Observatory of the Catholic University of Valencia (UCV) invites an in-depth analysis of genetic research at the conference Genetic research: possibilities and risks. An approach from bioethics. This event, which will take place on July 4, 2024, at the UCV San Juan y San Vicente headquarters (18 Jorge Juan Street), will bring together experts from various fields to explore the ethical implications of scientific advances in this area.

In-person attendance at the congress requires prior registration, but the possibility of following it online will also be offered through the following link: https://youtube.com/live/.

The advancement of genetic research constitutes one of the spearheads of biomedical sciences and opens up enormous application possibilities in the fields of bioengineering, editing, and gene therapies. In parallel, with the development of these new tools, new bioethical dilemmas arise related to their fields of application, their safety and effectiveness, and regulation and control needs that urgently need to be addressed.

In our congress we propose a scientific approach to the current state of genetic research, analyzing the most recent evidence, such as that related to epigenetic processes, the therapeutic applications of the editing processes and obtaining mini human organs through bioengineering procedures, the aspects ethics of the heritability of potential changes and the need for ethical and legal regulation of related practices.

A prestigious team of expert researchers in each of these areas will provide us with updated access to this evidence that allows its bioethical assessment based on scientific rigor.

It is aimed at researchers, teachers, students and anyone with an interest in the field of Bioethics, and especially in genetics.

REGISTRATION HERE

PROGRAM

10:00. Institutional inauguration

10:15. Round Table: Epigenetics and genome editing: A scientific update

Ethics and epigenetics.

Luis Franco. Full member of the Royal Academy of Sciences of Spain and the Royal Academy of Medicine of the Valencian Community. University of Valencia.

10:45. Genome editing. Therapeutic advances and bioethical uncertainties.

Nicolas Jouve. Emeritus Professor of Genetics, former member of the Bioethics Committee of Spain.

11:15. Colloquium

Moderator:Luca Gmez Tatay. Professor of cell biology, biochemistry and bioethics. Catholic University of Valencia.

11:30. Coffee Break

12:00. Round Table: Bioengineering and gene therapy

Deciphering the potential of human mini-organs in the laboratory through ethics and bioengineering.

Nria Montserrat. ICREA research professor and principal researcher at the Institute of Bioengineering of Catalonia (IBEC).

12:30. Advances in the therapeutic application of gene editing systems based on CRISPR. Juan Roberto Rodrguez-Madoz. Researcher of the Hemato-Oncology Program. TOP. University of Navarra.

13:00. Colloquium.

Moderator:Jos Miguel Hernndez Andreu. Professor and researcher of biochemistry and molecular biology. Catholic University of Valencia.

16:15. Round Table: Ethical limits in genetic manipulation

Heritable gene editing in humans and future generations.

Vicente Bellver. Professor of Philosophy of Law at the University of Valencia. President of the Bioethics Committee of the Valencian Community.

16:45. Regulating gene editing: principles versus rules.

Federico de Montalvo. Vice Chancellor of Institutional Relations and Secretary General of the Universidad Pontificia Comillas.

17:15. Gene editing: what should really scare us?

igo De Miguel. Research Group of the Chair of Law and Human Genome of the Department of Public Law. University of the Basque Country Euskal Herriko Unibertsitatea.

17:45. Colloquium

Moderator:Mara Jos Salar. Coordinator of the Philosophy Degree. Professor at the Faculty of Economic and Social Legal Sciences of the Catholic University of Valencia.

18:00. Closure. Mr. Julio Tudela. Director of the Bioethics Observatory of the Catholic University of Valencia.

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Genetic research: possibilities and risks Exaudi - Exaudi

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DALLAS May 23, 2024 A new study focused on the gene tied to a rare form of autism spectrum disorder (ASD) called FOXP1 syndrome offers hope that gene therapy might be able to help patients with this condition.

Genevieve Konopka, Ph.D., is Professor of Neuroscience and an Investigator in the Peter O'Donnell Jr. Brain Institute at UTSouthwestern. Dr. Konopka is a Jon Heighten Scholar in Autism Research and holds the Townsend Distinguished Chair in Research on Autism Spectrum Disorders.

In a study published in Science Advances, researchers from UT Southwestern Medical Center found that using gene therapy to restore the Foxp1 gene to adult mice from which it had been deleted before birth restored the activity of other genes whose levels are controlled by Foxp1. This intervention also corrected some abnormal behaviors characteristic of mice that lack Foxp1. The findings could shed light on other forms of ASD as well.

The ability to partially remedy brain pathway changes at later developmental stages suggests that gene therapy may be effectively applied in FOXP1 syndrome and actually normalize symptoms, said Genevieve Konopka, Ph.D., who co-led the study with Jay Gibson, Ph.D. Both are Professors of Neuroscience and Investigators in the Peter ODonnell Jr. Brain Institute at UT Southwestern.

Jay Gibson, Ph.D., is Professor of Neuroscience and an Investigator in the Peter O'Donnell Jr. Brain Institute at UTSouthwestern.

About 200 individuals worldwide have FOXP1 syndrome, a genetic condition caused by mutations in the FOXP1 gene that render it nonfunctional. Along with intellectual deficits, developmental delays, and other symptoms, people with this disease also tend to have ASD or exhibit autistic behaviors. But how the loss of FOXP1 contributes to these symptoms has been unclear, Dr. Gibson explained.

A key circuit thats disrupted in FOXP1 syndrome connects regions of the brain called the cortex, thalamus, and striatum. To better understand FOXP1s involvement in this circuit, Drs. Konopkaand Gibson and their colleagues used a technique to delete this gene in mice in two populations of neurons in the striatum, which receives inputs from both the cortex and thalamus through a chemical called glutamate. Glutamate causes these neurons to fire when its taken up at structures called synapses that connect neurons.

In one population of neurons, the deletion altered the functions of thousands of other genes and caused changes in neuronal responses as well as significant differences in behavior; the animals had problems building nests and spent more time on the edges of their enclosures. When the researchers used a genetic technique to reinstate Foxp1, this intervention normalized how neurons responded to glutamate and restored activity in 78 genes, most known to function in neural synapses. It also normalized some behaviors, such as nesting and time spent in enclosures.

Further study of this gene and the thousands of other genes it regulates couldidentify new targets for pharmaceuticals to treat this condition. Because some of these genes have also been implicated in other forms of ASD, continuing to study FOXP1 could lead to a better understanding and potential treatments for ASD in general, Dr. Konopka noted.

Other UTSW researchers who contributed to this study are first author Nitin Khandelwal, Ph.D., Instructor of Neuroscience; Ashwinikumar Kulkarni, Ph.D., Assistant Professor of Neuroscience; and Matthew Harper, M.S., Research Associate.

Dr. Konopka is a Jon Heighten Scholar in Autism Research and holds the Townsend Distinguished Chair in Research on Autism Spectrum Disorders.

This study was funded by grants from the National Institute of Mental Health (MH126481 and MH102603), the National Institute of Neurological Disorders and Stroke (NS126143 and NS115821), the James S. McDonnell Foundation 21st Century Science Initiative in Understanding Human Cognition Scholar Award (220020467), and the Simons Foundation (573689).

About UTSouthwestern Medical Center

UTSouthwestern, one of the nations premier academic medical centers, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty members have received six Nobel Prizes and include 25 members of the National Academy of Sciences, 21 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 3,100 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UTSouthwestern physicians provide care in more than 80 specialties to more than 120,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 5 million outpatient visits a year.

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UTSW study sheds light on rare form of autism - UT Southwestern

In May 2023, former health minister Lord James OShaughnessy acknowledged in a report that there were many issues with the UKs clinical trial industry. The report showed that the country fell from fourth to tenth globally for trial initiation, with a big drop in the number of Phase III trials initiated.

At the same time, the UKs National Health Service (NHS) has been under extreme pressure with staffing woes, including the recent junior doctor strikes and financial difficulties impeding clinicians ability to participate in research, especially in more demanding cell and gene therapy trials.

With a snap general election now looming on 4 July and the NHS being one of the top concerns for voters, it is more important than ever for political parties to set out their manifesto including how they are going to facilitate research and support the NHS.

The UK Conservative government has tried to improve the countrys ability to develop cell and gene therapies, with a $10m grant in March 2023 for the NHS Blood and Transplant (NHSBT) to open a facility to develop and manufacture new gene and cell therapies called the Clinical Biotechnology Centre (CBC).

Funds have also been provided to several companies through the Life Sciences Innovative Manufacturing Fund (LSIMF) grants, including 151m for Pharmaron and 14m for Touchlight for cell and gene therapy development and manufacturing.

On top of all this, the UKs National Institute for Health and Care Research (NIHR) has announced a 17.9m investment in the Advanced Therapy Treatment Centre Network (ATTC Network).

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A year on from Lord OShaughnessys report and despite the governments investment in the industry, the UK is still struggling to contribute to cell and gene therapy trials says haematologist and chief medical officer of stem cell charity Anthony Nolan Dr Robert Danby. Danby speaks exclusively to the Clinical Trials Arena about the difficulties facing the NHS in contributing to cell and gene therapy research.

Robert Danby (RD): The UK Government has committed to implementing all of Lord OShaughnessys recommendations, and we have seen some progress in the year since. Average trial approval and set-up times have been reduced. The number of people taking part in industry clinical studies is rising. According to the Department of Health and Social Care, 82% of commercial studies are on track.

Despite this, there is still more work to do. A thriving clinical research environment depends on an NHS that has the resources and workforce to support it with trials relying on the UKs network of clinicians, nurses, allied health professionals, statisticians, data managers, and more.

But as organisations such as Cancer Research UK have pointed out, were still a long way from NHS staff having the capacity to prioritise research. A survey of health care professionals revealed nearly four in five clinical researchers described lack of capacity as a substantial or extreme barrier to their work. It means clinical research is at risk of being seen as a nice-to-have, rather than an essential mechanism for bringing new and potentially life-saving therapies to the UK.

The OShaughnessy review said less about tackling the barriers patients face to getting onto a clinical trial. We need to think more ambitiously about the diversity of trial participants, to ensure no groups miss out on the potential improvements to care they offer. We also need to see investment in the real-world data landscape that can play such a pivotal role in advanced cell and gene therapies research.

RD: While we have seen commercial trial capacity increase in the last year, cell and gene therapies present additional research challenges that require a concerted effort to overcome. As a haematologist and in my work at Anthony Nolan, I see this close up.

Blood cancers and other haematological disorders are at the forefront of the oncoming wave of new cell and gene therapies. With over 50 years experience in cell therapies, both allogeneic and autologous haematopoietic cell transplantation, haematology is one of the few medical fields with the necessary skills and expertise to deliver clinical research into these potentially transformative treatments.

But the UKs haematology departments are not yet equipped, or resourced, to deliver this next wave. For example, cell and gene therapies are reliant on apheresis technology to collect the cells which form the basis of new engineered treatments. But current infrastructure is struggling to cope with basic clinical needs for standard indications, never mind additional requirements for therapy development.

Challenges to recruit to cell and gene therapy trials and rigorous regulatory pathways also contribute to the strain faced by NHS teams expected to deliver both basic care and clinical research. And as cell and gene therapy trials move into other areas of medicine like solid cancers or autoimmune diseases, there will need to be a massive increase in resources, training, and education across the breadth of the NHS.

In order to sustain the development of future cell and gene therapies, long-term investment into NHS capacity, education and training is essential. A more streamlined journey from research to regulatory approval and implementation, that is appropriate for the specific requirements of cell and gene therapies, is also needed.

RD: Brexit has compounded many of the issues affecting the UKs position in the global clinical research market. In my field of haematology, the well-recorded loss of NHS and academic staff due to Brexit has had major implications on our ability to carry out not only day-to-day care but also clinical research.

Weve also heard that differences in the regulatory framework and bodies between the UK and Europe post-Brexit have made the industry reluctant to start trials in the UK because implementation into routine care use looks too time-consuming and challenging.

RD: Accelerating Clinical Trials (ACT) is an innovative self-sustaining model to facilitate the delivery of clinical trials for blood cancers and blood disorders.

The ACT operational hub provides services to industry-sponsored commercial trials including access to a national network of recruitment sites, clinical research expertise and operational support and reinvests the income into non-profit academic investigator-led trials.

The OShaughnessy review highlighted that in its first 12 months, ACT attracted investment from two international pharmaceutical companies to deliver practice-informing blood cancer trials. With the income, ACT works with two major national trial acceleration networks which have recruited more than 2,500 patients in recent years, to the benefit of both patients and the UK life sciences sector.

Earlier this year, Anthony Nolan announced its 1m investment into the programme.

RD: We recognise we are seeing a revolution in cell and gene therapies, and the opportunity in the UK is huge. Our nationalised health care system, with strong links to academia, and healthcare data mean there is enormous potential for UK patients to be some of the first to benefit from these innovative and potentially curative therapies. But only if we act now to address issues in NHS capacity, invest in world-leading data systems and simplify the pathway to therapy implementation.

Editorial content is independently produced and follows thehighest standardsof journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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NHS investment critical to drive cell and gene therapy research - Clinical Trials Arena

Cell and gene therapy research is crucial for biopharma development, with HiFi long-read sequencing significantly enhancing many sequencing applications throughout the process. Even though AAV sequencing is one of the newest applications of genomics, it is also one of the most promising in disease research today. Adeno-associated virus (AAV) ranks among the most actively experimented upon vehicles for gene therapy.1 Gene therapies using AAV and cell therapies like CAR-T hold the potential to cure previously incurable diseases. CAR-T cell cancer treatments, in particular, are showing great promise in combating this devastating illness. In 2023, the FDA approved several new AAV-based therapies and cell-based gene therapies for treating Duchenne muscular dystrophy, severe hemophilia A, and sickle cell disease.2,3,4

The design of AAV vectors has consequences on gene therapy research, which could be the last stand in the fight against these diseases, and maybe many more. Highly accurate long-read sequencing supports the investigation into design, validation, and optimization of potential gene therapies using such viral vectors.

This is the final episode in our six-part myth-busting series. Today, were debunking common misconceptions about PacBio HiFi sequencing in cell and gene therapy research.

PacBio HiFi sequencing is too expensive to use when developing and assessing or optimizing gene therapy product design, efficacy and potential safety.

This statement is misleading.

Vector design plays a crucial role in gene therapy development success. In cell and gene therapy, where safety is paramount and R&D takes notoriously long, unexpected errors can quickly derail years of work, potentially delaying lifesaving therapies for those who need it. Fullyand accuratelycharacterizing your AAV product can mean reducing the risk of extremely costly failures during clinical trials.

Understanding the full extent of on- and off-target editing, vector or construct integration, and insertional mutagenesis are key components of validating AAV product design and ensuring its manufacturability. Using the exceptional accuracy and lengths of HiFi reads means that you can be confident in your product designs and avoid surprises down the line that require you to go back to the drawing board.

For more, read our best practices for gene therapy product characterization using HiFi sequencing with Dr. Claire Aldridge at Form Bio.

PacBio long reads are only good for de novo genome assemblies.

This statement is incomplete.

HiFi reads are good for assembling genomes, its true, but they can do so much more.

Count cell and gene therapy research are among the many applications you can do with HiFi sequencing. Whether its full-length AAV sequencing, gene editing assessments, plasmid or amplicon library screening, or vector integration, the winning combination of >20 kb reads and 99.9% accuracy with HiFi allows you to detect variants or events that short-read sequencing would miss.

You can use HiFi sequencing for every research stage of AAV gene therapy development:

Discover AAV vectors: discover novel capsids with targeted sequencing Optimize AAV vector design: improve designs by observing the frequency of truncations, fragmentation, and other non-full-length anomalies Confirm mRNA transcripts: quantify isoforms with full-length isoform sequencing Study host integration: understand the frequency of these events, to ensure the potential safety and efficacy of your product Ensure quality in AAV production: compare vectors and unresolved genomes and assess vector preps produced by different platforms

Read more about what you can do with highly accurate HiFi reads for AAV sequencing.

And what about gene editing? HiFi sequencing can power your gene editing research by enabling you to:

Sequence beyond your target to fully understand the extent of CRISPR-Cas9 editing Assess indels and other mutations that result from gene insertion at a CRISPR target locus Detect rare off-target mutations Understand the effects of haplotype and SNVs on gene editing in cases of allele-specific Cas9 cleavage Avoid PCR biases and limitations by using amplification-free approaches

Read more about what you can do with highly accurate HiFi reads for gene editing research.

Accuracy isnt that important for characterizing AAV impurities, such as partial genomes.

This statement is false.

Accuracy is imperative in all areas of science, and the stakes are arguably even higher in cell and gene therapy research, where clinical trials, patient outcomes, and life-changing therapeutics are on the line.

With highly accurate HiFi long reads, you can assess your AAV for impurities, like partial genomes, while at the same time getting critical information about sequence identity. PacBio HiFi sequencing unites the advantages of long reads with Sanger-level accuracy. This means you can monitor and improve AAV discovery and manufacturing with a single technology, reducing the risk of costly failures, which can set development timelines back by months or even years.

Missing crucial information about the quality of your vector can impact the effectiveness of your design. Hear how biopharmaceutical researchers at Homology Medicines are using highly accurate HiFi reads to discover novel vectors and improve their vector designs in this on-demand webinar.

Pairing nanopore with short-read sequencing is the easiest way to characterize AAVs from inverted tandem repeat (ITR) to ITR.

This statement is short-sighted.

Neither ONT nor short-reads can fully resolve ITRs and additional bioinformatics alignment work is necessary when using short reads. Why run multiple assays when you can do it better in one? Highly accurate long-read sequencing combines the accuracy and read length that you need in one experiment. Never again throw away reads because accuracy is too low or because they are too short to align. In addition, long-read sequencing with HiFi reads support a broad menu of applications that are critical for cell and gene therapy product design and development, including:

AAV sequencing Amplicon-based construct screening Gene editing and on- and off-target assessment Full-length plasmid sequencing Vector integration RNA sequencing Whole-genome sequencing

AAV is complex and difficult to sequence using PacBio HiFi sequencing.

This statement is incorrect.

PacBio and our partners at Form Bio offer an end-to-end workflow for AAV sequencing and data analysis, in an all-in-one solution to optimize your AAV vector designs. This workflow accommodates both ssAAV and scAAV sequencing using the same protocol. Form Bio workflows are certified PacBio compatible and provide analysis software to help you analyze and visualize your AAV data, so you can save time and resources.

With this protocol, you can use HiFi reads to:

Sequence tissues for novel AAV vector discovery Improve vector design Identify impurities, truncation events, and host integration events

See how HiFi sequencing makes it easy to sequence AAV genome populations to identify truncation, mutation, and host integration events.

As weve shown here and throughout our myth-busting series PacBio HiFi sequencing can benefit almost any genomics application by virtue of its long read lengths and exceptional accuracy. Leave those dated misconceptions in the past and start using the power of HiFi sequencing to fuel tomorrows groundbreaking discoveries.

Did you miss the other installments in our myth-busting series? Dont worry, you can catch up here:

Part 1 HiFi sequencing Part 2 human genomics Part 3 cancer genomics Part 4 plant and animal genomics Part 5 microbiology

Are there any other myths about long-read sequencing that you want busted? Let us know! Speak with a PacBio scientist to find out what you can do with HiFi sequencing.

Learn more

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Long-read sequencing myths: debunked. Part 6 cell & gene therapy - Pacific Biosciences

This years prize carrying a gold medal and a $1.2 million award is shared by Stuart Orkin, MD, a researcher at the Dana-Farber/Boston Childrens Cancer and Blood Disorders Center, and Swee Lay Thein, PhD, senior investigator and chief of the sickle cell branch of the National Heart, Lung, and Blood Institute at the National Institutes of Health.

Their work unveiled the genetic and molecular mechanisms underlying the transition in the production of fetal to adult hemoglobin. These discoveries led to work that culminated in the development and approval of Casgevy (exagamglogene autotemcel), the first gene-editing therapy based on CRISPR/Cas9 to be approved for SCD and transfusion-dependent beta thalassemia, a related blood disorder.

Receiving The Shaw Prize is an honor and a testament to the dedication of countless researchers who have contributed to our understanding of hemoglobin regulation over the years, Orkin, who is also a professor of pediatrics at Harvard Medical School, said in apress release from Boston Childrens.

This recognition underscores the potential of our findings to revolutionize the treatment landscape for sickle cell anemia and [beta] thalassemia, offering new hope to patients worldwide, Orkin added.

Often referred to as the Nobel of the East, the prize recognizes scientists who have made striking contributions to research. It was established in 2002 by Run Run Shaw, a philanthropist. To date, 41 individuals have been named Shaw Prize Laureates in Life Sciences and Medicine. There also is a Shaw Prize in Astronomy, and one in Mathematical Sciences.

This years science winners were recognized for their transformative research (Thein) and elegant work (Orkin), according to a Shaw Prize webpage highlighting their careers.

Over the course of their distinguished careers, Swee Lay Thein and Stuart Orkin each made wide-ranging, independent contributions to the analysis of blood cell disorders. Their work intersected when they made complementary and reinforcing discoveries that led to the development of a therapy to treat sickle cell disease and [beta] thalassemia, the webpage states.

Several versions of hemoglobin, the protein that carries oxygen in red blood cells, can be found in the human body. As its name suggests, fetal hemoglobin is produced while a baby develops in the womb. This version of hemoglobin is replaced after birth by an adult form of the protein that is less effective at transporting oxygen throughout the body.

The production of these different versions of hemoglobin is controlled by different genes. The HBB gene, which contains instructions for making a subunit of adult hemoglobin, is mutated in SCD, resulting in the production of a faulty version of the adult form of the protein. Conversely, the HBG gene, which provides instructions for making a component of fetal hemoglobin, is not affected by SCD-causing mutations.

One of the therapeutic strategies that can be adopted to ease the severity of SCD and beta thalassemia is to reactivate the production of fetal hemoglobin an approach that came on the heels of both Orkins and Theins discoveries.

Theins research led to the identification ofthe BCL11A gene as a regulator of fetal hemoglobin production. Follow-up work by Orkin confirmed the BCL11A protein was involved in the process of shutting off the production of fetal hemoglobin after birth a discovery that has rendered him the recipient of several other awards.

These findings led scientists to postulate that reducing BCL11A levels could boost the production of fetal hemoglobin, which in turn would compensate for the faulty or deficient version of the adult form of the protein in SCD and related blood disorders. Such a mechanism is the rationale behind the gene-editing therapy Casgevy, approved in the U.S. earlier this year.

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Shaw Prize awarded to 2 scientists for work in SCD gene therapies - Sickle Cell Disease News

The traditional randomised controlled trial (RCT) model has been used for decades in drug development. Although it is considered the gold standard of trial designs, sponsors are increasingly using alternative trial designs, especially those for developing cell and gene therapies.

The unique nature of cell and gene therapies, how they are administered, the complex dosing schedule, and the specialised patient population can make it difficult to use a traditional RCT model.

Innovative trial designs, like single group assignment, adaptive, basket, umbrella and platform trials, allow flexibility to be built into a trial, which experts describe as crucial in running cell and gene therapy trials.

Chief medical and scientific officer at UK stem cell charity Anthony Nolan, Dr. Robert Danby, said: Prospective RCTs have traditionally been the gold standard to evaluate the efficacy and safety of new therapies. For emerging cell and gene therapies, modern trial techniques like adaptive trials offer a promising alternative. These modern trial designs could offer patients new and better treatments sooner and do this without compromising on the quality of data required for new therapies to be approved.

Despite data showing cell and gene therapy trials continue to be designed as RCTs, there is agreement amongst the industry professionals that RCTs may not be the best model, says Dr. Odelia Chorin, rare disease paediatrician and clinical geneticist at the Safra Childrens Hospital, Sheba Medical Center.

GlobalDatas Pharmaceutical Intelligence Centre shows that the single-group assignment trial design is by far the most used approach for cell and gene therapies.

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The main reason for using alternative trial designs is to ensure trials are providing robust and interpretable data, says Amy Raymond, PhD, Executive Director Therapeutic Strategy Lead, Cellular and Genetic Medicines, at contract research organisation (CRO) Worldwide Clinical Trials.

While single assignment trials have been the leading design in cell and gene therapy trials in the past decade, Erin Griner PhD, associate director in clinical research methodology at Worldwide Clinical Trials says she is mostly seeing adaptive designs being used by sponsors.

The thing that everybody in the process wants is optimal, efficient data, but the question is how do we get there? Raymond asks.

Ultimately, the design is heavily influenced by the specific characteristics and requirements of both the therapy and the patient population rather than the indication itself, explains Neta Shanwetter Levit, Clinical Operations Lead, PhaseV, a company that develops machine learning (ML) technology to optimise clinical trial design and analysis.

We are seeing a gradual increase in the number of adaptive trials and basket trials. Platform trials are not as frequent in this area yet, but are starting to increase, Shanwetter added.

Dr. Beatrice De Vos, chief medical officer for EXO Biologics, a biotech developing exosome-based cell therapies, said that the flexibility to change protocols is the most helpful element of alternative trial designs, especially at early stages.

The data collected in an adaptive trial is impacting the development immediately, impacting your next steps in clinical trial phases. That is different to the classic model, so I am very much in favour of the adaptive design, De Vos added.

There is some differentiation between the choice of trial designs based on the indication. GlobalDatas Pharmaceutical Intelligence Centre shows following single assignment and RCTs are more common in oncology studies than in other indications while adaptive trials are mostly used to study treatments for genetic disorders.

The challenges in rare disease studies are the limited patient population and the scarce data, Shanwetter said. Therefore, adaptive trials are particularly useful in rare diseases due to their flexibility. Basket trials are frequently used in oncology to test therapies targeting specific genetic mutations across different types of cancers.

Griner agrees saying the basket trial design is used more often in oncology trials.

Despite it being difficult to randomise a gene therapy trial, in ophthalmology, sham controls are commonly used, Raymond says. But synthetic controls are also more commonly used in oncology while rare diseases are often studied in open-label trials and biomarkers are used to measure endpoints, she adds. Chorin agrees that given the lack of comparator, the industry needs to use biomarkers to measure clinical change.

Given how many different types cell and gene therapies, the design of a trial should be tailored on a drug-by-drug basis and there isnt a one-size-fits-all solution, says Shanwetter.

Another benefit of alternative designs is they allow sponsors to compare the treatment arm with real-world or natural history data using synthetic arms.

Natural history data is collected from patients who received no intervention and allows investigators to have a baseline to measure the drugs efficacy. Such approaches have been used in notable gene therapy approvals, Raymond said.

Zolgensma [pivotal trials] used natural history data as a control, which saw the PIs coming together and doing that research. That in my opinion is what really enabled this gene therapy trial to succeed was using that baseline comparison, Raymond explained.

Novartis gene therapy Zolgensma (onasemnogene abeparvovec) was approved for treating spinal muscular atrophy (SMA) in May 2019.

The ability to use existing data as a control is particularly helpful in rare disease trials where there are few patients, which often leads to trials being terminated due to low recruitment. Additionally, patients also might be more willing to participate if they know they will receive the study drug, Griner adds.

For some rare diseases, we are seeing 80% to 90% of patients being willing to try a gene therapy that is as yet unproven because it gives them some hope. Having a trial design that is an open-label study really increases interest and as a result recruitment, Griner adds.

De Vos says it is possible to match historical data to a degree that is similar to classical placebo, but acknowledges in statistical terms, it is not a head-to-head comparison. De Vos says EXO Biologics is running a Phase I/II trial (NCT06279741) of using a single group assignment design. The Phase I portion of the trial, which is evaluating EXOB-001, an exosome-based cell therapy for premature babies with bronchopulmonary dysplasia, is using natural history data as a comparator while the Phase II stage will be a standard RCT model.

The only challenge with using natural history data as a control, Griner explains, is that in some diseases there is very little available.

Overall, Griner says that agencies have been supportive of studies with alternative designs, adding that the US Food and Drug Administration (FDA) has also released guidance on confirmatory evidence.

De Vos said that there is also guidance available in Europe but that it should be read very carefully. [However] having gone through all these guidelines, companies should not refrain from coming up with new designs, rather than sticking to the old fashioned ones to extract the most out of their observation in terms of new product development, De Vos concluded.

Raymond says the FDA and EMA are often aligned, but not always, recalling instances where a trial has required region-specific protocols, and in some cases region-specific primary endpoints.

One thing the regulators need to be looking at is having a more unified framework with alternative trial designs. Its not a small ask but it should be a real priority as it would be helpful to the industry as a whole, Raymond concluded.

Editorial content is independently produced and follows thehighest standardsof journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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Flexibility of alternative trial designs crucial for cell and gene therapy research - Clinical Trials Arena

Scientists at the Broad Institute of MIT and Harvard have engineered a harmless adeno-associated virus (AAV) that can efficiently reach the brain, potentially improving the efficacy of brain-targeted gene therapies for neurological conditions such as Huntingtons disease.

Current AAVs that deliver gene therapies to cells in the body via injection into the bloodstream cannot efficiently cross the blood-brain barrier (BBB), the highly selective membrane that regulates what substances from the bloodstream can access the central nervous system (CNS), which consists of the brain and spinal cord.

In a mouse model modified to produce the human version of an important BBB protein, the newly engineered AAV was more widely distributed across the brain and more effective at delivering a gene therapy to brain cells than AAV serotype 9, which is used in an approved CNS-targeted gene therapy.

The study, An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery, was published in Science.

Since we came to the Broad weve been focused on the mission of enabling gene therapies for the central nervous system, Ben Deverman, PhD, the studys senior author and director of vector engineering at the Broads Stanley Center for Psychiatric Research, said in an institute press release. If this AAV does what we think it will in humans based on our mouse studies, it will be so much more effective than current options.

Ken Chan, PhD, one of the studys first co-authors and group leader in Devermans group, said the AAVs have the potential to change a lot of patients lives.

In Huntingtons, defects in the HTT gene lead to toxic clumps of the huntingtin protein, which are thought to damage various parts of the brain, triggering the onset of symptoms.

Gene therapy, which works by delivering to cells genetic material meant to counterbalance a genetic defect, can potentially treat disorders caused by mutations in a single gene, such as Huntingtons.

Most of these therapies use modified, harmless AAVs to deliver the therapeutic cargo to cells. However, current AAVs cannot efficiently cross the BBB, which protects the brain from harmful substances in the blood while allowing essential nutrients and certain molecules to pass through.

To address these limitations, the researchers engineered an AAV that binds to the transferrin receptor (TfR1), a cell surface protein that is highly present in the human BBB and is an established target of antibody-based therapies designed to reach the brain.

To find such a virus, they first screened large AAV libraries in test tubes. Top candidates were then tested in cells and mice (in vivo) modified to produce human TfR1. Screening against a human protein in mice was done to improve the chances that gene therapies using these AAVs would work in human patients.

Weve learned a lot from in vivo screens but it has been tough finding AAVs that worked this well across species, said research scientist Qin Huang, PhD, the other co-first author of the study. Finding one that works using a human receptor is a big step forward.

Injecting the top AAV candidate, called BI-hTFR1, into the bloodstream of TfR1-modified adult mice dramatically increased levels of the AAV in the CNS compared with unmodified mice. This demonstrated that the AAV was indeed crossing the BBB by binding to human TfR1.

In different brain regions, BI-hTFR1 reached up to 71% of nerve cells and 92% of astrocytes, specialized cells in the brain that provide support to nerve cells.

The team then compared BI-hTFR1 with AAV9, an AAV used as a delivery vehicle for Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy approved for the neuromuscular disorder spinal muscular atrophy.

They found that BI-hTFR1 levels in brain tissue were up to 50 times higher than those of AAV9.

As a model for gene therapy, researchers used the new AAV to deliver a healthy copy of the human GBA1 gene, which encodes an enzyme called beta-glucocerebrosidase, to the modified mice. Mutations in this gene cause Gaucher disease and are linked to Parkinsons disease.Both of those are neurological conditions.

Compared with AAV9, BI-hTFR1 delivered 30 times more copies of the GBA1 gene to brain cells and substantially increased the beta-glucocerebrosidase activity in the brain and cerebrospinal fluid, which surrounds the brain and spinal cord.

When we think about gene therapy for a whole-brain disease you need really systemic delivery and broad biodistribution in order to achieve anything, said Eric Minikel, PhD., senior group leader at the Broad. Naturally occurring AAVs just arent going to get you anywhere, Minikel said. This engineered [AAV] opens up a world of possibilities.

In addition to targeting a human protein, the fact that BI-hTFR1 has a similar production yield to AAV9 using scalable manufacturing methods makes it ideal for CNS-targeting gene therapy, the researchers noted.

Researchers at Apertura Gene Therapy, a biotech company co-founded by Deverman, are already developing new CNS-targeting gene therapies using the new AAV.

The scientists believe that further research can help improve the AAVs gene-delivery efficiency to the CNS, the institute said. It may also help reduce AAV accumulation in the liver, and prevent their inactivation by antibodies in some patients two known complications of current viral-based gene therapies.

Read more:
New viral carrier shows promise for brain-targeted gene therapies - Huntington's Disease News

According to latest study, the global cell & gene therapy bioanalytical testing services market size was estimated at USD 585.19 million in 2023 and is projected to hit around USD 1,194.92 million by 2033, growing at a CAGR of 7.4 % during the forecast period from 2024 to 2033.

The emergence and proven efficacy of novel modalities such as cell and gene therapies have significant growth in the pharmaceutical industry. This growth is derived by substantial investments from drug developers, particularly in areas like rare diseases and immune oncology, where these therapies have demonstrated notable success.

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Cell & Gene Therapy Bioanalytical Testing Services Market Overview

The rapid growth of the cell and gene therapy bioanalytical testing services market is driven by the potential of these therapies to revolutionize treatment across a spectrum of diseases, from cancer to genetic deficiencies. This innovation necessitates new considerations for clinical testing, prompting biopharmaceutical developers to seek quality laboratory testing partners or subject matter experts (SMEs) to navigate the unique challenges of bioanalytical testing in this domain. Such partnerships are essential for driving innovation and ensuring successful development programs as they progress towards regulatory approval.

Gene and cell therapies, utilizing viral or non-viral vectors, present distinct challenges, with gene therapies classified into gene correction, gene editing, and oncolytic virus therapies, while cell therapies encompass adoptive cell and stem cell therapies. Consequently, specialized expertise in CGT solutions and services is crucial, particularly in areas like pharmacokinetics (PK), immunogenicity, and pharmacodynamics (PD) biomarker analysis, where advanced techniques and meticulous care are paramount.

Cell & Gene Therapy Bioanalytical Testing Services Market Key Takeaways

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Cell & Gene Therapy Bioanalytical Testing Services Market Dynamics

Driver

Increasing research and development

The increasing growth of gene and cell therapies in treating previously incurable diseases is the beginning of a transformative era in healthcare. With pharmaceutical companies heavily investing in research and development to unlock the full potential of this field, a surge of new gene and cell therapy products is entering early development stages. Rising trends in research, product releases, and patent applications becomes crucial for identifying global innovation patterns and seizing commercial opportunities. As companies strive to make informed decisions about resource allocation, the demand for specialized cell and gene therapy bioanalytical testing services is escalating. These services play a vital role in ensuring the safety, efficacy, and regulatory compliance of innovative therapies, thus driving the growth of this dynamic market segment.

Restraint

Scalability Challenges

The scalability of cell and gene therapy manufacturing emerges as a pivotal challenge hindering market growth. Manual labor-intensive processes contribute to time and cost inefficiencies, making manufacturing both laborious and expensive. Intricate nature of these therapies, their production remains notoriously costly, thereby limiting accessibility for patients and impeding scalability. The cell and gene therapy bioanalytical testing services market experience constraints as the industry grapples with overcoming these scalability hurdles.

Opportunity

Evolution of Cell and Gene Therapy

The gene editing techniques and transgene delivery systems is revolutionizing patient care, with promising outcomes across various disease domains, including rare diseases and challenging cancers. As the gene and cell therapy field continues to evolve, scientists recognize the need for personalised quantitative measurements specific to their drug programs. This demand for advanced analytics extends beyond traditional methods like chromatography and ligand binding assays. An opportunity arises for the cell and gene therapy bioanalytical testing services market to meet the evolving needs of drug developers by offering innovative solutions and cutting-edge technologies, thereby facilitating further advancements in this burgeoning field.

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By Test Type

The bioavailability & bioequivalence studies dominated the market with a share of 35.22% in 2023, holding a significant share and poised for rapid growth in the forecast period. These studies play a critical role in assessing the in vivo biological equivalence of different proprietary preparations of a drug, ensuring consistency and effectiveness. Bioequivalence studies ascertain whether two pharmaceutical products are essentially interchangeable, while bioavailability studies measure the concentration of a drug in the plasma or blood over time following a systematic protocol. As pharmaceutical companies strive for quality assurance and regulatory compliance, the demand for these essential tests continues to escalate, driving growth in the market segment.

The pharmacokinetic segment is anticipated to experience significant growth throughout the forecast period. Pharmacokinetics, a pivotal field within pharmaceuticals, focuses on understanding how drugs move within the body and how the body interacts with these drugs. Through the application of terms, theories, and equations, practitioners can accurately estimate drug concentrations in various bodily regions. As the importance of pharmacokinetic testing continues to be recognized in drug development and clinical practice, the demand for these services is expected to rise steadily in the coming years.

By Stage of Development Product Type Insights

The non-clinical segment dominated the market with a market share of 66.12% in 2023 and is also expected to witness the fastest growth at a CAGR of 7.5% during the forecast period and poised for rapid growth in the forecast period within the Stage of Development Product Type category. These studies encompass a range of protocols, including animal studies conducted in accordance with Good Laboratory Practice (GLP) regulations. As pharmaceutical companies prioritize safety and efficacy in drug development, the demand for robust non-clinical studies is expected to continue its upward trajectory, driving growth in this market segment.

The clinical segment is poised for growth in the forecast period within the Stage of Development Product Type category. Clinical, comprising three key phases, serve as pivotal stages in the development of new medicines, ensuring their safety, efficacy, and optimal dosage forms before they can be approved for patient use. These are designed to determine the optimal dosage, formulation, safety profile, absorption characteristics, and therapeutic effectiveness of a drug. As pharmaceutical companies strive to bring innovative treatments to market while adhering to rigorous regulatory standards, the demand for clinical trial services is expected to increase, driving growth in this segment.

By Indication Insights

The oncology segment dominated the market and accounted for the largest revenue share of 49.85% in 2023, capturing the largest revenue share within the indication category. Cell and gene therapies represent a revolutionary approach in cancer treatment. These therapies operate by modifying the DNA of a patient's existing cells, providing them with new instructions to detect and combat cancer. By binding the inherent capabilities of the immune system, cell and gene therapies offer a promising avenue for more effective and personalized cancer treatment strategies. As research in this field continues to advance and more therapies gain regulatory approval, the oncology segment is expected to maintain its stronghold in the market.

The rare diseases segment is poised for rapid growth during the forecast period within the indication category. Cell and gene therapies have demonstrated notable efficacy, in addressing clinical indications associated with rare diseases. These therapies hold promise for patients with serious or life-threatening rare diseases by targeting the underlying cause of the condition, rather than merely alleviating symptoms. As research and development efforts continue to expand in this area and more therapies advance through clinical trials, the rare diseases segment is expected to experience significant growth, providing new hope for patients and caregivers alike.

By Product Type Insights

The cell therapy segment dominated the product segment market with a market share of 42.19% in 2023, capturing a significant market share. The potential applications of cell therapies are vast and encompass a wide range of medical conditions, including cancers, autoimmune diseases, urinary problems, infectious diseases, joint cartilage damage, spinal cord injuries, immune system deficiencies, and neurological disorders. Cell therapy encompasses a diverse array of approaches, including both stem cell- and non-stem cell-based therapies, as well as unicellular and multicellular therapies. As research and development in cell therapy continue to advance, fueled by promising clinical outcomes, the market for these innovative treatments is expected to witness sustained growth.

The gene-modified cell therapy segment is poised for the fastest growth during the forecast period within the product category. These therapies, often referred to as Ex Vivo gene therapies, involve modifying cells outside the patient's body and then reintroducing them to combat the disease, as seen in Chimeric antigen receptor T-cell (CAR T-cell) therapy for cancers. Gene-modified cell therapy entails precisely modifying cells ex vivo to enhance the patient's ability to fight the disease, representing a promising frontier in personalized medicine. As advancements in genetic engineering continue to drive innovation in this field, the gene-modified cell therapy market is expected to experience significant growth.

Regional Insights

North America dominated the cell & gene therapy bioanalytical testing services market and accounted for the largest revenue share of 40.56% in 2023, capturing the largest revenue share. Despite the FDA's approval of only a handful of gene therapy treatments, numerous ongoing studies indicate a burgeoning interest in this field. Gene therapy is currently under investigation for a wide array of diseases, including cancer, heart disease, cystic fibrosis, sickle cell disease, and haemophilia A. CAR-T cell therapies stand out as the primary technology utilized in the pipeline of genetically modified cell therapies, representing 52%, followed by a diverse range of technologies grouped under the "other" category at 27%. Notably, 97% of CAR-T cell therapies are focused on cancer indications, with a minority targeting non-oncology areas such as scleroderma, HIV/AIDS, and autoimmune disease. The robust pipeline of cell and gene therapies in the US underscores significant scientific advancements, raising questions about the extent to which these innovations will translate into tangible progress within the US healthcare system.

The Asia Pacific region is poised to experience the fastest CAGR of 7.8% growth in the cell and gene therapy sector during the forecast period. Recent developments in this space across countries like Singapore, China, South Korea, and Japan have been remarkable, with these nations emerging as dynamic hubs for pioneering research. Significant investments from governments and institutions have facilitated the establishment of state-of-the-art facilities and the adoption of cutting-edge technologies. As Asia Pacific continues to make strides in this field, it presents lucrative opportunities for collaboration and innovation in the global healthcare landscape.

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Cell & Gene Therapy Bioanalytical Testing Services Market Recent Developments

Cell & Gene Therapy Bioanalytical Testing Services Market Top Key Companies:

Cell & Gene Therapy Bioanalytical Testing Services Market Report Segmentation

This report forecasts revenue growth at country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the Cell & Gene Therapy Bioanalytical Testing Services market.

By Test Type

By Product Type

By Stage of Development by Product Type

By Indication

By Region

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Cell & Gene Therapy Bioanalytical Testing Services Market Size to Hit USD 1.19 Bn by 2033 - BioSpace

PHILADELPHIA PA Greater Philadelphia has emerged as a powerhouse in the cell and gene therapy industry, housing almost 10 percent of the worlds companies in this rapidly expanding sector. A report commissioned by the Chamber of Commerce for Greater Philadelphia has revealed that this industry now provides employment to more than 7,000 people in the region.

Leading the Way: Cell and Gene Therapy in Greater Philadelphia, hailed as the most comprehensive regional snapshot of the sector, declares cell and gene therapy as a paramount economic catalyst for the region in the last decade. The area displays a rich blend of established and emerging businesses, leading research institutions, and an exceptional pool of industry talent that has collectively sparked a wave of economic prosperity and groundbreaking treatments for myriad diseases.

Chamber President and CEO, Chellie Cameron, is effusive about the regions role, noting, Greater Philadelphia is the top destination for those who wake with a mission to improve lives through cell and gene therapy treatments. The region offers everything that research companies, researchers, and their support teams would want, which is why we continue to make such great strides.

The report, compiled by economic research firm Ninigret Partners LLC and supported by a grant from the Knight Foundation, will serve as a beacon for community members, talent, policymakers, and practitioners seeking to advance parallel sector-specific growth strategies both locally and internationally.

John Churchill, Director, Philadelphia, Knight Foundation acknowledged the regions pioneering role in his statement: Greater Philadelphia is already a global leader in cell and gene advanced therapies and is poised to become one of the top innovation hubs in this field. This study aims to pinpoint the key factors and resources driving success.

Tracking back to the origins of cell and gene therapy discovery 25 years ago in Philadelphia, Claire Greenwood, Executive Director and Senior Vice President of Economic Competitiveness for the Chamber, observes how this mature ecosystem now includes a broad range of economic drivers. The report reveals Greater Philadelphia to be home to 60 of the estimated 500 cell and gene therapy companies worldwide. In addition, the region claims a top-ranking position for NIH funding in gene therapy and is a leader in translational science.

Furthermore, the region has seen a substantial influx of capital into life sciences companies. Since 2018, 547 life sciences companies in Greater Philadelphia have accumulated $18.7 billion through various financial avenues, with approximately $8 billion specifically related to cell and gene therapy.

In addition to the regions robust lineup of cell and gene therapy companies, the Greater Philadelphia ecosystem also showcases a well-developed supportive infrastructure. Recent additions include a Charles River CRADL facility, the entry of Mispro biotech services, the creation of the Cencora CGT Integration Hub, and the IBX Advanced Therapeutics Network.

The Chambers CEO Council for Growth and 11 partner companies, institutions, and universities launched the Cell & Gene Therapy and Connected Health Initiative in January 2019. The main objectives of this Initiative include shared storytelling, critical infrastructure development, talent assessment and attraction, and ecosystem scalability. Greenwood highlights that this new study will further enhance the innovative and collaborative approaches emanating from the Initiative.

As the Greater Philadelphia region continues to establish its dominance in the cell and gene therapy landscape, its clear that its synergistic blend of economic resources, research capabilities, and industry talent is driving both an economic boom and life-altering therapeutic developments. The implications of this include not only a thriving job market but also the promise of new treatments that could revolutionize healthcare for countless individuals worldwide.

For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and Microsoft Start.

Originally posted here:
Greater Philadelphia Dominates Cell and Gene Therapy Landscape - A Beacon of Innovation and Employment - MyChesCo

EBT-101, a CRISPR-based gene-editing therapy from Excision BioTherapeutics, was safe and well tolerated in a Phase I study, but it did not prevent viral rebound in the first three participants who stopped antiretroviral treatment, according to a presentation last week at the American Society of Gene & Cell Therapy (ASGCT) annual meeting.

Excision put a positive spin on the findings, noting that favorable safety data is a necessary step on the path to developing therapies for latent viral infections. The company also touted promising early results from studies of CRISPR-based therapies for herpes simplex virus and hepatitis B. But the HIV rebound news is disappointing, and it underscores the importance of remaining wary of exaggerated claims from industry and the mainstream press about the state of HIV cure research.

Initial data from the EBT-101-001 trial provides important clinical evidence thatagene editingtreatment modality can be safely delivered fortargetingthe HIV DNA reservoirs in human cells, study investigator Rachel Presti, MD, PhD, of Washington University St. Louis School of Medicine, said in a news release. This study provides researchers with invaluable insights for how CRISPR technology can be applied for addressing infectious disease and was an important first step towards additional programs designed to optimize this treatment modality for treating the millions of individuals who are impacted by HIV and other infectious disease.

Antiretroviral therapy can keep HIV replication suppressed indefinitely, but the virus inserts its genetic blueprints into the DNA of human cells and establishes a long-lasting reservoir that the drugs cant reach. This integrated HIV DNA lies dormant in resting T cells during treatment, but it can start churning out new virus when antiretrovirals are stopped, making a cure nearly impossible. The only way to tell whether an intervention leads to long-term remission is to discontinue antiretroviral therapy with careful monitoring, known as an analytic treatment interruption.

Kamel Khalili, PhD, of Temple University, and colleagues have been studying gene therapy to cure HIV for more than a decade. In 2014, they reported that a CRISPR-Cas9 tool could cut out a segment of integrated HIV DNA necessary for viral replication. A study published in 2019 showed that this approach could remove integrated HIV genes and clear latent viral reservoirs in mice.

This led to the development of EBT-101, a CRISPR-based therapy delivered by an adeno-associated virus that uses dual guide RNAs to target three sites on the integrated HIV genome. Making cuts at these locations prevents the production of intact virus. Last August, researchers reported that a single dose of a simian version of the therapy safely and effectively removed an HIV-like virus from viral reservoirs in monkeys on antiretroviral therapy, but this study did not include a treatment interruption.

The first human clinical trial of EBT-101 (NCT05144386) started in 2022, enrolling people on antiretroviral therapy with a stable undetectable viral load. The study protocol called for participants who maintained viral suppression at 12 weeks after receiving the gene therapy to undergo an analytic treatment interruption.

At the European Society for Gene & Cell Therapy annual meeting last October, Presti reported that EBT-101 was detectable in the blood of the first three treated participants after a single IV infusion at the initial dose level. EBT-101 was well tolerated with only mild temporary side effects. She did not present treatment interruption outcomes, but that didnt stop the Daily Mail from proclaiming that a cure for HIV could be months away.

Presti gave an update last week, and the news generally wasnt good. Of the five participants who have so far received the initial dose of EBT-101, three stopped antiretroviral therapy. Unfortunately, all three experienced viral rebound and had to restart their antiretrovirals. This likely occurred because the gene therapy did not reach all cells harboring latent HIV, and even a very small number of cells containing residual HIV DNA is enough to reignite viral replication.

But the news was not all bad. One EBT-101 recipient was able to maintain viral suppression for four months after treatment discontinuationconsiderably longer than it typically takes for the virus to rebound after stopping antiretrovirals. This suggests that EBT-101 or similar CRISPR therapies might play a role in a combination functional cure strategy.

We know that many people were hopeful that a first trial could provide evidence of a possible cure for HIV because the field has been waiting over 20 years for a cure, Excision senior vice president William Kennedy, MD, said in a news release. However, it was essential that this clinical trial establish safety for EBT-101 as a gene therapy product as well as safety related to the use of CRISPR for the field.

Excision is now testing a higher dose of EBT-101 in a second cohort and is exploring new CRISPR delivery methods that might be more efficient than the adeno-associated virus vector. One possibility is lipid nanoparticles like the ones used to deliver messenger RNA (mRNA) in COVID-19 vaccines.

The company is also exploring CRISPR-based approaches for other latent viral infections. Herpes simplex virus (HSV) persists in nerve cells, and it can reactivate to cause cold sores, genital herpes or eye inflammation (keratitis). Hepatitis B virus (HBV) establishes chronic infection in the liver, where it can potentially lead to cirrhosis and liver cancer. Unlike HIV and other retroviruses, however, HSV and HBV do not integrate their genetic blueprints into the chromosomes of human cells, so they may be easier to remove.

In other presentations at the ASGCT meeting, researchers reported preclinical results for another experimental CRISPR therapy dubbed EBT-104, showing that a single dose reduced HSV DNA by more than 99% in laboratory cell cultures. Whats more, it eliminated viral shedding in 11 of 12 rabbits with herpes keratitis, according to the news release.

In other preclinical research, a single dose of EBT-107a CRISPR compound delivered by lipid nanoparticlesreduced HBV DNA, hepatitis B surface antigen and hepatitis B e antigen by 98%, 97% and 92%, respectively, in a mouse model of hepatitis B. Unlike CRISPR delivered by viral vectors, EBT-107 in nanoparticles and could potentially be given as multiple doses to reach more latent virus.

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CRISPR HIV Gene Therapy Disappoints in Early Study - POZ

Pictured: Panelists on stage at ASGCT (L to R: NHLBIs Gary Gibbons, NCATS Joni Rutter, Amy Jenkins of Advanced Research Projects Agency for Health, NINDS Amir Tamiz and ASGCT President Jeffrey Chamberlain)/Photo by Greg Slabodkin

Advancing translational research of novel therapies is a priority for both the scientific community and the National Institutes of Health, according to stakeholders at the 2024 American Society of Gene & Cell Therapy (ASGCT) annual meeting.

In a Friday fireside chat with officials from the National Institute of Health (NIH) at ASGCT 2024, ASGCT President Jeffrey Chamberlain described translational research as the critical nexus between basic science and clinical investigation.

This is an area where were trying to bridge the gap between fundamental new discoveries and their applications to improve human health, Chamberlain said.

The sessions agenda highlighted some of the pivotal basic science and translational studies, as well as the researchers who are making breakthroughs when it comes to transformative genetic therapies.

Gary Gibbons, director of the National Heart, Lung, and Blood Institute (NHLBI) at NIH, touted his organizations launch of the Cure Sickle Cell Initiative in 2018 as a collaborative research effort to accelerate the development of gene therapies to cure sickle cell disease (SCD).

Gibbons said that new FDA-approved curative gene therapies for SCD Vertex Pharmaceuticals CRISPR-based Casgevy and bluebird bios gene therapy Lyfgeniaare a case study in the value of translational research for creating transformative treatments for patients.

We had folks from Vertex and bluebird bio at the table from the beginning, as well as our colleagues at FDA, Gibbons commented. Because we knew at that stage we were defining a regulatory pathway toward that end.

Chamberlain called Casgevy and Lyfgenia possibly the most exciting development ever in the field of gene therapies.

Joni Rutter, director of the National Center for Advancing Translational Sciences (NCATS) at NIH, said that one of her organizations primary missions is to re-engineer the translational research process so new treatments and cures for diseases can reach patients faster. Our mission is essentially to understand how to traverse that pipeline better, faster, more effectively and efficiently, Rutter said.

NCATS has taken credit for enabling a path to market for ReveraGen BioPharmas Duchenne muscular dystrophy therapy, which was approved by the FDA in late 2023. Another NCATS-led program Rutter pointed to is the Platform Vector Gene Therapy (PaVe-GT) pilot project. Launched in 2019, the PaVe-GT pilot aims to test the hypothesis of whether a platform vector approach will increase efficiency in preclinical testing and clinical trial start-up.

Rutter also pointed to the NCATS-led Bespoke Gene Therapy Consortium (BGTC), a public-private collaboration involving NIH, FDA, industry and patient groups intended to help accelerate the delivery of AAV-based gene therapies for rare diseases. With BGTC, were establishing how to optimize the biology and the studies behind developing the AAV vectors and then were also optimizing the manufacturing, Rutter said.

Greg Slabodkin is the news editor at BioSpace. You can reach him atgreg.slabodkin@biospace.com. Follow him onLinkedIn.

Excerpt from:
ASGCT24: Translational Research Powering Potentially Transformative Therapies - BioSpace

Pfizer has reported the death of a patient in the Phase II DAYLIGHT study of experimental gene therapy, fordadistrogene movaparvovec, to treat Duchenne muscular dystrophy (DMD).

The trial is designed to assess the safety and dystrophin expression in young boys with DMD.

In a community letter issued by the company, Pfizer said: We do not yet have complete information and are actively working with the trial site investigator to understand what happened.

The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023.

The company has also paused participant dosing associated with the crossover portion of its Phase III CIFFREO trial, while continuing other trial activities as scheduled.

Pfizer concluded the initial dosing of subjects in placebo-controlled, randomised crossover CIFFREO trial last year.

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Both DAYLIGHT and CIFFREO trials are part of Pfizers research into fordadistrogene movaparvovec for DMD treatment, targeting different age groups.

DAYLIGHT enrols boys aged two to less than four years, whereas CIFFREO involves boys aged four to less than eight years.

Despite the dosing pause in CIFFREO, the company clarified that this action does not extend to other ongoing trials within the fordadistrogene movaparvovec programme, as dosing in those studies has already been completed.

Pfizer is also collaborating with regulatory authorities and an independent external Data Monitoring Committee to investigate the cause of the patients death.

The company added: The safety and well-being of the patients in our clinical trials remains our top priority, and we are committed to sharing more information with the medical and patient community as soon as we can.

We are also aware that many in the patient community are hopeful about the potential benefit of fordadistrogene movaparvovec for the treatment of DMD, and we will continue to collect data from our trials to evaluate its ability to address this disease.

Earlier this month, the company reported net income of $3.11bn in the first quarter (Q1) of 2024, marking a decrease of 44% from $5.54bn posted in Q1 2023.

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Pfizer reports fatality in Phase II DMD gene therapy study - Clinical Trials Arena

In posters presented at theISPORThe Professional Society for Health Economics and Outcomes Researchmeeting held in Atlanta, Georgia, researchers explored Duchenne muscular dystrophy (DMD) caregiver experiences through cross-sectional surveys,1,2 and an economic analysis examined gene therapys impact on work opportunities for caregivers.3

DMD is an inherited neuromuscular disorder that leads to progressive muscle degeneration and weakness.4 It primarily affects boys, and symptom onset is typically in early childhood. Life expectancy for those with DMD has increased in recent years, and survival into the early 30s is becoming more common.

As DMD progresses, affected individuals rely more heavily on their caregivers, the authors of one poster wrote.1 Their cross-sectional survey aimed to characterize the experiences of caregivers in the US, as contemporary data are lacking.

The analysis included 106 US-based caregivers who completed an online survey between March and May 2023. The survey included questions capturing the use of formal (paid) and informal (unpaid) care; informal burden on caregivers rated on a scale from 0 to 10, 10 being the most challenging; and how accessibility barriers impact family plans. The survey results were stratified by patient ambulatory status.

The mean caregiver age was 46 years, and the mean care recipient age was 14.5 years. Eighty-one percent of the caregivers were mothers, and 11% were caregivers for 2 individuals with DMD. Out of 118 total patients with DMD being cared for, 47% (n = 55) were nonambulatory. Caregivers of ambulatory patients reported spending a median of 4.5 hours per day caregiving, while caregivers of nonambulatory patients reported a median of 8 hours per day. The median amount of time spent giving care per day in the overall cohort was 6 hours per day.

Caregivers reported a median burden of 5 overall, with those caring for nonambulatory patients reporting a burden of 6 and caregivers of ambulatory recipients reporting a burden rating of 4. Overall, 75% of caregivers reported utilizing additional informal caregiving, and 26% used additional formal caregiving. Of those using formal caregiving, 13% were caregivers for ambulatory patients and 42% were caregivers for nonambulatory patients. Accessibility barriers led to frequent changes in everyday plans for 74% of families.

Overall, the findings show the significant amounts of time and energy required of caregivers for individuals with DMD, as well as the increasing demands associated with disease progression.

Muscular dystrophy | Image credit: Lemau Studio - stockadobe.com

Another poster highlighted the impacts of caregiving on paid work, including the extent of DMD caregivers paid work accommodations and lost productivity based on survey results.2 The investigators recruited survey participants through a US-based DMD advocacy group and included questions from the Work Productivity and Activity Impairment (WPAI) questionnaire for DMD caregivers, with higher scores indicating greater impairment.

Among 106 caregivers, 81% were mothers, and the mean age was 46, and 89% of caregivers reported caring for 1 individual with DMD. The annual household income was greater than $100,000 annually for 49% of households in the study.

Overall, 68% of respondents (n = 72) were employed, with 55% (n = 52) employed full-time. Based on WPAI scores, 40.7% of respondents had experienced overall activity impairment in the past week, and absenteeism among those employed was 8.4%. Presenteeism, or impairment at work, was reported by 30.5% of caregivers, and absenteeism plus presenteeism (work productivity loss) by 34.8%. Among the 11% of caregivers caring for 2 individuals with DMD, 67% (n = 8) were employed, and 62.5% (n = 5) reported work productivity was impacted by DMD.

Furthermore, 77% (n = 82) of caregivers reported work-related changes due to caregiving responsibilities. Twenty-five percent quit, 26% took lower paying jobs, 34% changed their job role or responsibilities, 29% reduced working hours, and 34% took time off from work.

The results demonstrate the considerable paid work impact associated with caregiving for one or two individuals with DMD, the authors concluded, noting that the findings are limited due to the low number of caregivers of 2 individuals included in the study.

In an economic analysis, investigators explored how delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics), a gene therapy approved for the treatment of ambulatory patients aged 4 to 5 years with DMD, could impact the work opportunity of those caring for patients with DMD.3 They developed a partitioned survival model including 5 statesearly ambulatory, late ambulatory, early nonambulatory, late nonambulatory, and deathand estimated outcomes and caregiver work opportunity with standard-of-care (SOC) treatment vs SOC plus gene therapy for a cohort of 4-year-old early ambulatory patients with DMD. The researchers applied 2.8% salary growth and 3% discount rates annually.

With SOC treatment alone, caregivers were estimated to lose 7194 (15.5%) hours and experience a lifetime income loss of $249,697 (16.1%)/undiscounted $431,911 (16.2%) compared with the general US population. Caregivers of patients treated with SOC plus gene therapy were estimated to lose 4564 (9.8%) hours, equating a lifetime income loss of $153,396 (9.9%)/undiscounted $318,375 (11.9%) compared with the general population.

DMD substantially impacts caregiver work opportunity and income, the authors concluded. Based on this model, delandistrogene moxeparvovec could potentially increase caregiver work opportunity.

Reference

1. Audhya IF, Dunne JS, Patel S, et al. Characterizing the experience of caregiving for those with Duchenne muscular dystrophy (DMD): results from a cross-sectional survey. Presented at: International Society for Pharmacoeconomics and Outcomes Research 2024; May 5-8, 2024; Atlanta, GA. Poster 6044.

2. Patel S, Dunne JS, Audhya IF, et al. The balancing act of paid work and caregiving in Duchenne muscular dystrophy (DMD): results from a cross-sectional survey. Presented at: International Society for Pharmacoeconomics and Outcomes Research 2024; May 5-8, 2024; Atlanta, GA. Poster 6018.

3. Innis B, Henry A, Nelson L, et al. Economic analysis of the impact of delandistrogene moxeparvovec gene therapy on work opportunity in caregivers of individuals with Duchenne muscular dystrophy. Presented at: International Society for Pharmacoeconomics and Outcomes Research 2024; May 5-8, 2024; Atlanta, GA. Poster 2050.

4. Duchenne muscular dystrophy (DMD). Muscular Dystrophy Association. Accessed May 9, 2024.https://www.mda.org/disease/duchenne-muscular-dystrophy

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Posters Characterize DMD Caregiver Experiences, Impact of Gene Therapy on Caregiving Demands - AJMC.com Managed Markets Network

Published: 09 May 2024

A girl born deaf can now hear unaided after participating in a world-first trial.

TheCHORD study is for children born with hearing loss due to a genetic condition called auditory neuropathy.Auditory neuropathy is a condition where the ear can detect sound normally, but has a problem sending signals to the brain.

This can be caused by a fault in the OTOF gene. The gene makes a protein called otoferlin that allows cells in the ear to communicate with the hearing nerve. Changes in this gene can reduce sound transmission from the inner hair cells to the hearing nerves.

Gene therapy aims to deliver a working copy of the faulty OTOF gene. The treatment is given using a neutralised virus, injected into the inner ear under general anaesthetic.

The study started in 2023 using a treatment made by Regeneronand is supported by NIHR Cambridge Clinical Research FacilityandNIHR Cambridge Biomedical Research Centre.

Opal Sandy is the first patient treated in the global gene therapy trial. Opal was born completely deaf because of neuropathy. She was treated shortly before her first birthday.

During surgery, while Opal was given the gene therapy in right ear, a cochlear implant was fitted in her left ear.

Opals mother, Jo Sandy, said: When Opal could first hear us clapping unaided it was mind-blowing - we were so happy when the clinical team confirmed at 24 weeks that her hearing was also picking up softer sounds and speech. The phrase near normal hearing was used, and everyone was so excited that such amazing results had been achieved.

Professor Manohar Bance is the chief investigator for the trial. He is also an ear surgeon at Cambridge University Hospitals NHS Foundation Trust. He said: These results are spectacular and better than I expected. Gene therapy has been the future of otology and audiology for many years and Im so excited that it is now finally here. This is hopefully the start of a new era for gene therapies for the inner ear and many types of hearing loss.

Dr Richard Brown, Consultant Paediatrician at CUH, who is an investigator on the CHORD trial, said: The development of genomic medicine and alternative treatments is vital for patients worldwide, and increasingly offers hope to children with previously incurable disorders. It is likely that in the long run such treatments require less follow up so may prove to be an attractive option, including within the developing world. Follow up appointments have shown effective results so far with no adverse reactions and it is exciting to see the results to date.

Doctors in other countries, including China, are exploring very similar treatments for the OTOF gene mutation.Around 20,000 people across the US, UK, Germany, France, Spain and Italy are thought to have auditory neuropathy due to OTOF mutations, which shows the potential significance of a successful treatment. Patients are being enrolled in the study in the US, UK and Spain.

Addenbrookes Hospital in Cambridge is participating in the trial. Read more on the Cambridge University Hospitals website.

Photo of Opal Sandy taken byCambridge University Hospitals

More:
Baby born deaf can hear after breakthrough gene therapy - National Institute for Health Research

The companys presentation at ASGCT includes preliminary data results for a child who received the gene therapy.

Regeneron presented results from an ongoing Phase I/II clinical trial for its investigational gene therapy, DB-OTO, at the annual American Society of GeneandCell Therapy (ASGCT) meeting, being held in Baltimore, Md. from May 711. DB-OTO, a gene therapy for genetic deafness, improved hearing in one child, treated at 11 months old to normal levels within 24 weeks. A second child, treated at 4 years old, also showed hearing improvements at a six-week assessment, according to the company (1). Both children were born with genetic deafness due to variants of the otoferlin gene.

The results are from the ongoing Phase I/II CHORD (NCT# 05788536) first-in-human, multicenter, open-label trial. The trial evaluates the safety, tolerability, and preliminary efficacy of DB-OTO in infants, children, and adolescents with otoferlin (OTOF) variants in the United States, United Kingdom, and Spain. CHORD is a two-part study where participants will receive a single intracochlear injection of DB-OTO in one ear in Part A of the study. In Part B, participants receive simultaneous single intracochlear injections of DB-OTO in both ears at the selected dose from Part A.

Congenital deafness impacts approximately 1.7 out of every 1000 children born in the United States, according to Regeneron (1).Otoferlin-related hearing loss is especially rare and caused by variants in the otoferlin gene, which impairs the production of theOTOFprotein that is critical for the communication between the sensory cells of the inner ear and the auditory nerve.

Study data presented at ASGCT showsat the 24-week assessment the first participant, a 16-month-old child, had improvement of hearing to normal levelsacross key speech frequencies, with an average 84 dB improvement from baseline and one frequency measure reaching 10 dB in hearing level per PTA. Across all tested frequencies, an average 80 dB improvement from baseline was observed. There was also positive ABR responses, with best frequency reaching 45 dB.

The second participant, a four-year-old child, experienced consistent results to the first participant, including initial improvement of hearing with responses to loud sounds,which was observed across key speech frequencies, with an average 19 dB improvement from baseline and one frequency measure reaching 80 dB in hearing level per PTA. Across all tested frequencies, an average 16 dB improvement from baseline was observed. Positive ABR responses had best frequency reaching 75 dB.

The opportunity of providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime, saidLawrence R. Lustig, MD, chair of Columbia UniversitysDepartment of OtolaryngologyHead & Neck Surgery and a clinical trial investigator, in a press release. These impressive results showcase the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related deafness, and we are excited to see how this translates into an individuals development, especially [because] early intervention is associated with better outcomes for speech development. With the DB-OTO CHORD trial now enrolling participants in sites across theUS andEurope, were part of the beginning of a new era of gene therapy research that looks to create treatment options that address the root cause of profound genetic deafness.

1. Regeneron. Latest DB-OTO Results Show Dramatically Improved Hearing to Normal Levels in a Child with Profound Genetic Deafness Within 24 Weeks and Initial Hearing Improvements in a Second Child at 6 Weeks. Press Release. May 8, 2024.

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Regeneron Presents Update on Gene Therapy for Genetic Deafness at ASGCT - Pharmaceutical Technology Magazine

A new study published in New England Journal of Medicine has described the effect of gene editing in a group of patients with Leber congenital amaurosis (LCA).

The clinical trial involved two children and 12 adults with the genetic eye condition. Participants received an experimental gene editing therapy called Edit-101.

Outcomes used to evaluate the clinical trial were: visual acuity; full-field test results; the ability of participants to navigate a research maze containing obstacles and varied lighting levels; and self-reported improvements in quality of life.

The researchers found that 11 of the participants (79%) showed improvements in at least one of the four outcome measures, while 6 participants (43%) showed improvement in two or more outcomes. Four patients (29%) experienced improvements in visual acuity.

The scientists reported that there were no serious adverse effects linked to the treatment.

Dr Mark Pennesi, of Oregon Health & Science University, shared how rewarding it was to hear about vision improvements among patients who took part in the trial.

One of our trial participants has shared several examples, including being able to find their phone after misplacing it and knowing that their coffee machine is working by seeing its small lights, he said.

While these types of tasks might seem trivial to those who are normally sighted, such improvements can have a huge impact on quality of life for those with low vision, he added.

More:
Gene therapy improves vision in Leber congenital amaurosis patients - Association of Optometrists (AOP)

00;00;00;25 - 00;00;01;13

At four

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weeks, when we initially heard her

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turning to clapping, that was like

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I was mind-blown that had worked.

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Id sat behind Opal countless times

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when she's had, to us, ridiculously

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loud noises

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blared through speakers in a sound booth

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and she's never once turned, so to

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see that happen is mind-blowing.

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Good girl! Yippee!

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Clever girl!

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So this particular trial was for children

00;00;36;10 - 00;00;38;05

who have the OTOF gene variant

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that does not produce

00;00;39;05 - 00;00;40;09

a particular protein

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that they need for hearing.

00;00;41;21 - 00;00;43;06

And the surgery involves

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approaching the ear

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like a cochlear implant, infusing a virus

00;00;46;22 - 00;00;48;11

that makes the cells

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produce the protein that they're missing.

00;00;50;27 - 00;00;52;02

So one thing that we've been

00;00;52;02 - 00;00;53;11

really excited about here

00;00;53;11 - 00;00;54;22

is that we've been able to use

00;00;54;22 - 00;00;57;04

a really small dose of gene therapy

00;00;57;04 - 00;00;59;07

delivered directly to the cochlear,

00;00;59;07 - 00;01;00;06

and what that means is

00;01;00;06 - 00;01;01;12

that we're not delivering

00;01;01;12 - 00;01;02;25

a large dose of gene therapy

00;01;02;25 - 00;01;04;01

to the rest of the body.

00;01;04;01 - 00;01;06;11

So it means that we see

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fewer side effects.

00;01;07;13 - 00;01;08;10

And for Opal,

00;01;08;10 - 00;01;09;20

this is a very big change,

00;01;09;20 - 00;01;11;23

because before the gene therapy,

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she had profound hearing loss.

00;01;13;06 - 00;01;14;04

That meant she couldn't

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hear any sounds in that ear at all,

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even when they were very loud.

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And now she can hear

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sounds at a very soft level,

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almost in the normal range for children

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her age.

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Can I have a kiss?

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Aw, good girl, daddy kiss, mummy kiss.

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Yeah, if she doesn't have her implant

00;01;35;11 - 00;01;38;07

on, she acts exactly the same

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as if she did have it on,

00;01;39;09 - 00;01;42;05

because she can hear so well with this

Continued here:
Baby born deaf can hear after breakthrough gene therapy - Cambridge University Hospitals

Revolutionizing Treatment: ASGCTs Clinical Trials Spotlight on Immunotherapy, Cancer Vaccines, and Auditory Diseases

At this years ASGCTs 27th Annual Meeting in Baltimore, groundbreaking clinical trials took center stage, illuminating the latest advancements in gene therapy research. These trials presented innovative approaches ranging from targeted cell and gene therapies to advancements in AAV gene therapy and novel findings in cell therapy. Here we dive deeper into immunotherapy and cancer vaccines, alongside novel treatments for auditory diseases. With a focus on cutting-edge treatments and promising outcomes, these presentations underscore the transformative potential of gene and cell therapy in addressing diverse medical challenges.

Generating Antitumor T Cells: Personalized Neoantigen Vaccine and Pembrolizumab in Advanced Hepatocellular Carcinoma

Customized cancer vaccines targeting neoantigens from a patients tumor may boost the effectiveness of PD-1 inhibitors by stimulating tumor-specific immune responses. Findings from a single-arm Phase Ib/2a study investigate the use of a personalized therapeutic cancer vaccine (PTCV), designed to target specific mutations in an individuals tumor, in combination with pembrolizumab, a PD-1 inhibitor, for the treatment of advanced hepatocellular carcinoma (HCC). The results indicate that this combination therapy induces a robust immune response, involving both CD8+ and CD4+ T cells targeting tumor-specific neoantigens. These findings suggest a potential mechanism for the observed clinical activity and provide insight into improving treatment outcomes for patients with advanced HCC.

Long-Term Safety and Integration Site Analysis of T Cells Modified with Lentiviral or Gammaretroviral Gene Addition

No adverse events related to insertional mutagenesis were detected in a large patient cohort treated with CART therapy. Here, the long-term safety of CAR T cell therapy was assessed by analyzing data from 780 patients treated between 2001 and 2023. While 21 patients developed secondary malignancies, none were directly linked to the CAR T cell product. Integration site sequence analysis revealed insights into the behavior of transduced cell clones, with no evidence of pathogenic insertional mutagenesis. Overall, the study provides reassurance regarding the safety of CAR T cell therapy, despite recent concerns about secondary T cell malignancies.

CHORD Trial: DB-OTO Gene Therapy for Pediatric Hearing Loss

This trial highlights the significance of biallelic otoferlin gene mutations in causing severe-to-profound sensorineural hearing loss, emphasizing the potential for OTOF gene replacement therapy to restore physiological hearing. Subsequently, this investigation introduces DB-OTO, a novel adeno-associated virus vector designed for intracochlear delivery of the OTOF gene, and outlines the objectives of the CHORD trial (NCT05788536) evaluating its safety and efficacy in pediatric patients with profound hearing loss due to OTOF mutations. Initial results from the trial demonstrate promising outcomes, with no dose-limiting toxicities or DB-OTO-related adverse events reported. Significant improvements in hearing, as indicated by behavioral pure tone audiogram thresholds and auditory brainstem response, were observed in the DB-OTO-treated ear. Additionally, parental reports and auditory skills assessments further support the positive impact of DB-OTO on auditory function and development. These findings underscore the potential of DB-OTO gene therapy as a viable treatment option for patients with profound hearing loss caused by OTOF mutations, warranting further investigation in larger patient cohorts.

In conclusion, the presentations from the ASGCT annual conference in Baltimore showcase significant strides in immunotherapy, gene therapy, and treatments for ophthalmic and auditory diseases. The studies on personalized neoantigen vaccines, CAR T cell therapy safety, and DB-OTO gene therapy offer promising insights into improving outcomes for patients with advanced hepatocellular carcinoma, hematologic malignancies, and hearing loss due to genetic mutations. These findings underscore the potential of innovative cell and gene therapies in addressing unmet medical needs and enhance patient care. Moving forward, continued research and development in these areas will be instrumental in advancing precision medicine and improving the lives of individuals affected by these conditions. ASGCT 2024, gene therapy, gene therapy definition, cell and gene therapy, AAV gene therapy, Cell Reports impact factor, cell therapy, American, Baltimore, therapy, cell

Advancing Health: ASGCTs Clinical Trials Spotlight on Cell Therapy and Cell-Based Gene Therapy

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Profluent Achieves Human Genome Editing Milestone Using OpenCRISPR-1: The First AI-Generated, Open-Source Gene Editor

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The Gene & Cell Therapy Landscape: Recent Approvals and Upcoming Therapeutics of Interest

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Revolutionizing Treatment: ASGCT's Clinical Trials Spotlight on Immunotherapy, Cancer Vaccines, and Auditory ... - geneonline