Category Archives: Gene therapy

The bioindustry will require more cell and gene therapy plants, says an expert, who says the facilities of the future must be automated, scalable, and flexible.

The number of cell and gene therapies entering clinical development has increased significantly in recent years. According to the Alliance for Regenerative Medicines (ARM) there are 1,066 such therapies in trials at present1, which is a 32% increase on the number of studies in 2014. But the surge in clinical activity has not been matched by an increase in production capacity says Darren Dasburg, a cell and gene therapy-focused consultant.

Hundreds of facilities will be needed to manufacture the treatments that are in play now, he said, adding that if you factor in the plants needed to make viral vectors that could exceed a thousand facilities.

The good news, Dasburg says, is that these facilities are more like labs than traditional large biopharmaceutical plants.

Viral vector capacity is critical to the cell and gene therapy sector. Vectors are hollow viruses used to insert genetic material into cells, both cells used in protein expression and cells used therapeutically. Various organisations have voiced concerns about industry capacity to make vectors. In 2018, for example, the Alliance for Advanced Biomedical Engineering said the scarcity of viral vectors could hamper expansion2. Since then the situation has improved, but it has not been resolved3. While viral vector production capacity in the contract services sector has increased, the expansion is still falling short of demand.

Partly this is because of the complexity of making the vectors, according to Dasburg.

Most viral vectors are produced using adherent manufacturing technologies which are expensive to operate, he explains. A vial of just 20 million cells can cost $2030K because it is so challenging to make.

To bring down costs, vector capacity still needs to increase, continues Dasburg, who predicted that biopharma will continue to rely on CDMOs for the foreseeable future.

Cell and gene therapy manufacturing is still a young industry. Biopharma is still figuring out what the ideal production facility should look like.

Building for flexibility and multipurpose manufacturing is important, Dasburg says, noting that explaining CDMOs and IP holders need to understand they are attacking rare genetic diseases and ailments where the therapy might be a third-line treatment. The numbers are often quite lower, and the treatments can be one and done. All meaning the companies of the future will be attacking many more areas of need.

In terms of technology, all cell and gene therapy facilities should feature sufficient isolator capacity, Dasburg says. Isolators are probably the number one investment to make. Too many people are trying to work five people in full dress in a small room attempting to manufacture in a hands-on traditional way when isolation and automation could help immensely.

Dasburg pointed to benchtop platforms capable of processing a single CAR-T patients treatment as an example of an innovative approach being used. These can be arranged in an array within a single ballroom-like facility providing 100% containment going from leukapheresis bag to treatment bag without any human intervention.

References1. alliancerm.org/wp-content/uploads/2020/02/CBX-Meeting-7-Feb-2020-FINAL.pdf2. aabme.asme.org/posts/virus-shortage-for-cell-therapies-creates-engineering-opportunity3. http://www.genengnews.com/insights/gene-therapy-dollar-is-waiting-on-viral-vector-dime/

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More Cell and Gene Therapy Facilities in the Hundreds are Needed - Genetic Engineering & Biotechnology News

COLLEGE STATION, Texas, Feb. 28, 2020 /PRNewswire/ --FUJIFILM Diosynth Biotechnologies U.S.A., Inc. (FDB), a world leading cGMP Contract Development and Manufacturing Organization (CDMO) supporting its partners in the biopharmaceutical industry with the development and manufacture of their biologics, vaccines and gene therapieshas announced that it has commenced the planned capacity expansion of its "Flexible Biomanufacturing Facility" at its College Station, Texas location. The Flexible Biomanufacturing Facility is designed to support late phase and commercial manufacturing of gene therapies and other advanced therapies. The Flexible Biomanufacturing Facility complements the extensive development and early phase manufacturing capabilities provided by the company from the National Centre for Therapeutic Manufacture also located in College Station, Texas.

The expansion project will include the addition of cell culture and high throughput manufacturing suites. The expanded facility will house multiple 500L and 2000L bioreactors to support the production of gene therapy products that have high production demands. This $35M USD expansion is part of the previously announced capital investment of approximately 13 billion yen (approx. $120 million USD) in the gene therapy field by FUJIFILM Corporation.

"The capacity increase to our Flexible Manufacturing Facility will allow us to support the growing demand for commercial-ready, high volume production of gene therapy products, many of which are in development to treat rare diseases, which makes this announcement even more significant, as today we honor rare disease patients on Rare Disease Day," said Gerry Farrell, chief operating officer, FUJIFILM Diosynth Biotechnologies, Texas.

"These investments are part of FUJIFILM Corporation's long term strategy to provide leading, future proofed end-to-end gene therapy solutions, from pre-clinical to commercial launch," said Martin Meeson, president and chief operating officer, FUJIFILM Diosynth Biotechnologies, U.S.A.

FDB expects the expansion of its Flexible Biomanufacturing Facility to complete by Fall 2020.

About Fujifilm

FUJIFILM Diosynth Biotechnologies an industry-leading Biologics Contract Development and Manufacturing Organization (CDMO) with locations in Teesside, UK, RTP, North Carolina, College Station, Texas and Hillerod, Denmark. FUJIFILM Diosynth Biotechnologies has over thirty years of experience in the development and manufacturing of recombinant proteins, vaccines, monoclonal antibodies, among other large molecules, viral products and medical countermeasures expressed in a wide array of microbial, mammalian, and host/virus systems. The company offers a comprehensive list of services from cell line development using its proprietary pAVEway microbial and Apollo cell line systems to process development, analytical development, clinical and FDA-approved commercial manufacturing. FUJIFILM Diosynth Biotechnologies is a partnership between FUJIFILM Corporation and Mitsubishi Corporation. For more information, go to: http://www.fujifilmdiosynth.com

FUJIFILM Holdings Corporation, Tokyo, Japan, brings cutting edge solutions to a broad range of global industries by leveraging its depth of knowledge and fundamental technologies developed in its relentless pursuit of innovation. Its proprietary core technologies contribute to the various fields including healthcare, graphic systems, highly functional materials, optical devices, digital imaging and document products. These products and services are based on its extensive portfolio of chemical, mechanical, optical, electronic and imaging technologies. For the year ended March 31, 2019, the company had global revenues of $22 billion, at an exchange rate of 111 yen to the dollar. Fujifilm is committed to responsible environmental stewardship and good corporate citizenship. For more information, please visit: http://www.fujifilmholdings.com.

All product and company names herein may be trademarks of their registered owners.

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FUJIFILM Diosynth Biotechnologies Expands Its Gene Therapy Manufacturing Capacity At Its College Station, Texas Location - Tyler Morning Telegraph

24 Feb 2020

Another gene therapy for a central nervous system disorder has posted positive results. The Phase 1/2 trial enrolled 18 men with X-linked retinitis pigmentosa (RP), a disease of retinal degeneration that leads to blindness. Robert MacLaren of the University of Oxford led the study, published February 24 in Nature Medicine.

The dose-escalation trialtested six doses; the therapy consisted of an adeno-associated virus 8 expressing a normal copy of the RP GTPase gene. Seven patients saw improvements in their vision that endured for the six-month duration of the trial. Inflammation within the eye cropped up at the higher doses, which may have temporarily blurred therapeutic effects in some patients.

Interest in using gene-based therapy for AD and other neurodegenerative disorders has grown ever since such a therapy was approved for treating spinal muscular atrophy (SMA) in babies and toddlers, and the field is now looking to learn from trials of other nervous system disorders (Nov 2019 news;Dec 2019 news).

X-linked RP is caused by mutations in the RP GTPase regulator (RPGR) gene. The mutations trigger degeneration of photoreceptors starting in childhood. No treatments exist. Recent approval of a gene therapy for another retinal disorderRPE65-related retinal degenerationsuggests retinal gene therapy could work (Russell et al., 2017).

However, the RPGR gene has confounded scientists, as it contains a repetitive, purine-rich stretch that undergoes alternative splicing. Tinkering with the sequence has boosted fidelity and stability of the gene, and conferred therapeutic benefits in animal models of the disease (Fischer et al., 2017).

In this trial, each of the 18 men, who were between 22 and 50 years old, had severe retinal degeneration. They were recruited in six cohorts of three patients each, who received increasing doses of the viral therapy. The participants received an injection of the virus into their subretinal space; they were then monitored for safetythe trials primary outcomeand tested for vision and retinal sensitivity for six months.

Across the cohorts, 55 adverse events occurred, all mild. Seven out of nine patients on the three highest doses experienced mild retinal inflammation, which was corrected by oral corticosteroids. In all, the trial met its primary safety endpoint.

Seven out of 12 patients receiving one of the top four doses had visual gains in the treated eye. This was gauged by retinal microperimetry, a map of the quality of light perceived across the retina. The improvements started at one month and continued at the six-month follow-up. The researchers proposed that inflammation in the top three dose cohorts may have offset visual improvements in some patients.

The trial was not designed to draw conclusions about efficacy. The researchers speculated that the therapys effectiveness will boil down to the stage of retinal degeneration, vector dose, and any interfering effects of inflammation. The second phase of the study will compare two doses to placebo.

Though the eyes are not strictly the brain, retinal therapy can be considered as part of the revitalization of gene-therapy approaches for neurodegenerative diseases. AAV-based expression of survival motor neuron 1 has improved life for those with SMA, but the tricky aspects of RPGE gene expression, and the inflammatory response that cropped up at higher doses, point to potential challenges other gene therapies may have to overcome (Nov 2016 news; Mendell et al., 2017).Jessica Shugart

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Retinal Gene Therapy Trial Posts Positive Result - Alzforum

In London, the firm Ori Biotech is developing an automated way to manufacture gene and cell therapies, a bottleneck for many biotech companies.

Mission: To develop a robotic system to automate the labor-intensive tasks involved in manufacturing cell and gene therapies, such as gene transduction and cell expansion.

Gene and cell therapies are maturing as a field, as advances in genomics and data analysis open up the way for ever more advanced therapies. One limitation of this trend is that much of the manufacturing process is currently manual, making it expensive and hard to produce consistent results.

The first generation of cell and gene therapies have demonstrated the revolutionary potential of these products but also the challenge of making them available to patients at scale, Ori Biotechs co-founder and CSO, Farlan Veraitch, told me.

Ori Biotech aims to address this manufacturing bottleneck by making robots take care of simple, but time-consuming tasks. For example, adding or taking away cells from cell culture containers in current practice is laborious, and also can also introduce contamination into the batch. Part of Oris approach is to make adjustable containers that reduce the need for moving cells between containers and exposing them to contamination.

There are many companies similarly developing methods for improving the manufacture of gene and cell therapies. For example, the Belgian company Univercells recently raised 50M to fund manufacturing technology to make viral vector production cheaper in gene therapies. Synthace is another company automating lab protocols involved in the quality control process of manufacturing. According to Veraitch, Ori stands out from other companies because it was set up by experts in the gene therapy field.

Many of them are startups innovating in one unit operation, others are part of established service providers that arent really technology companies and still others are repurposing technologies from other uses to try to find a way to fit a square peg to into a round hole, he told me.

Ori Biotech has a number of partners involved, such as Hitachi Chemical, and the UK innovation center Cell and Gene Therapy Catapult. The company aims to test its system in early 2020 in its partners labs. Ori is funding the development with an 8.6M seed round that it raised last month.

What we think:

With the current difficulties in manufacturing cell and gene therapies, they are expensive to make and arent meeting their full mainstream potential. With companies like Ori Biotech working on it, this may not be the case for long.

With the amazing clinical data coming through and more than 900 companies running more than 1,000 clinical trials in cell and gene therapy, the industry is poised at a tipping point, Veraitch said. We are seeing cures for previously untreatable types of cancer and terminal rare disease being studied and approved so there is plenty of reason to be hopeful.

In addition, Veraitch believes that overcoming manufacturing obstacles will also lead to the treatments becoming more accessible to healthcare systems and patients. At present, gene therapies are among the most expensive drugs in the world, such as Novartis spinal muscular atrophy treatment Zolgensma, which is priced at over 1.8M per person.

The industry is currently at a similar stage to Henry Ford building one Model-T at a time in his workshop and needs a move to the equivalent of assembly-line manufacturing, which would increase throughput, decrease costs, and increase quality, concluded Veraitch.

Images from Shutterstock

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This Startup Is Automating the Manufacture of Gene and Cell... - Labiotech.eu

A health ministry panel approved Wednesday the production and sale of a gene therapy for treating spinal muscular atrophy, a rare incurable disease that destroys muscle function through a decrease in motor neuron cells.

The health minister is expected to formally approve the drug, Zolgensma, by the end of March, with public health insurance coverage seen starting this summer.

The one-time-only gene therapy, developed by Swiss drug giant Novartis AG, will cover patients below 2 years old with abnormalities in their genes to make proteins necessary for maintaining motor nerves. Novartis estimates 15 to 20 children a year are likely to receive treatment using the drug in Japan.

In the therapy, proteins are created after the genes, carried by adeno-associated viruses, are delivered to motor nerves through intravenous drips. In an overseas clinical trial, all of the 15 severe SMA patients who used the therapy were able to live without an artificial ventilator. If no treatment is offered, about 75 percent of such patients are expected to die or need an artificial ventilator before they reach the age of 13.6 months.

The therapy, which has already been approved in the United States, is expensive, costing the dollar equivalent of 230 million for a one-time infusion.

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Japanese government panel OKs expensive gene therapy for rare disease - The Japan Times

The excitement about cell and gene therapies is almost tangible within the biotech and pharma industry. Over 950 companies are actively developing advanced therapies, which are expected to make exceptional improvements to peoples lives in the next decade. Although hopes are high, the industry still faces a number of challenges in cell and gene therapy manufacturing, mainly around being able to deliver these often difficult to make, complex treatments at the scale needed to meet patient demand.

The unprecedented growth of the industry, alongside the need to develop scalable manufacturing strategies, has led to a number of challenges that need to be addressed urgently. Previously, patient numbers were so small that processes were highly manual and required numerous skilled operators. However, the recent success of early gene therapy trials means upscaling now needs to be considered right from the start.

In the early days the aim was just to get to the clinic, said Lindsey Clarke, Head of Cell and Gene Therapy EMEA at Bio-Techne. Scale didnt come into it so much, but now the conversations we are having focus much more on making these complex therapies at a scale needed for a commercial medicine. There are increased efforts on finding solutions that dont just work for trials with 10 patients, but will still work at 1000 times that scale.

Life science tools and technology provider, Bio-Techne, has made it its mission to further support the cell and gene therapy industry by channeling its expertise into developing technologies that can help to scale manufacturing processes. The companys commitment is highlighted by its recent investment into a new good manufacturing practice (GMP) manufacturing facility in St Paul, Minnesota, US, that will focus on producing raw materials for use in cell and gene therapy applications.

We have realized that if all our customers are to be successful with their therapies then there will be a huge demand for raw materials, Clarke explained. So weve started building that capability, ahead of time. But its not just about supply, we are also innovating, from simple things like looking closely at the format our products come in and making them more compatible with large-scale manufacturing to whole new product ranges.

Bio-Technes investment in the new GMP manufacturing facility is a solution to meet the growing demand for raw materials needed for cell and gene therapy manufacturing. But its just one piece of a large puzzle: cell and gene therapy developers also need to consider the complex logistics required to deliver their therapies to the clinic, particularly when its an autologous therapy.

The process from the patient to the clinician, to the apheresis collection, to the manufacturing site, then the complex manufacturing process and then delivery back to the patient is highly complex.

Another key challenge closely related to upscaling is the great risk of human error in manual processes. Many of the cell and gene manufacturing processes currently in place have been developed with small patient numbers in mind and involve manual steps.

Humans are an excellent source of variability and risk, explained Clarke. When youre manufacturing in a GMP environment, you need highly-skilled, trained operators and there is a shortage of them out there. Automation is going to be key to address this issue. Not only does it reduce the manpower that is required, but it can also streamline the processes and make them less risky, more scalable, and reproducible as well, Clarke added.

With cell and gene therapy products, various analytical methods are used to assess critical quality attributes during development and manufacturing. These reflect the identity, potency, purity, safety, and stability of the product. However, such methods are frequently complex, non-standardized, time-consuming, and performed manually by trained operators.

Organizations such as Cell and Gene Therapy Catapult have called for the development of new analytical solutions for quality testing of advanced therapies throughout the manufacturing process. More automated analytical technologies have the potential to increase facility throughput and make quality control (QC) faster, less error-prone, more reproducible, and more GMP compliant.

Although Bio-Techne has a long-standing history of developing quality proteins, antibodies, small molecules, and immunoassays, it has expanded into automated protein analytical technologies in recent years.

For viral and non-viral vectors, Bio-Technes ProteinSimple branded platforms are rapidly being adopted by cell and gene therapy developers for assessment of vector identity, purity, and stability. Compared to traditional methods like Western blot, SDS-PAGE, and ELISA, ProteinSimples technology platform is based on capillary electrophoresis and microfluidics and provides a fully automated and accurate quantitative analysis of vectors.

We are also seeing Micro-Flow Imaging (MFI), a more common image-based analytical platform in biologics, used to characterize subvisible particles for quality control of cell and gene therapy products, explained Kamar Johnson, Commercial Development Manager in Cell and Gene Therapy at Bio-Techne. These robust automated platforms offer ease of use, rapid time to result, and software that meets GMP requirements.

Collaboration lies at the heart of successful innovation. It is especially important at the interface between process development and manufacturing, said Johnson.

Not everyone is an expert in everything, we all have our particular niches of expertise, added Clarke. We believe that we need to collaborate to get the innovation that will help change the way we manufacture cell and gene therapies. Collaboration is the key to solving the challenges of the cell and gene therapy industry.

On that note, Bio-Techne recently partnered with Fresenius Kabi and Wilson Wolf to form a new joint venture that provides manufacturing technologies and processes for the development and commercialization of new cell and gene therapies.

The collaboration combines Bio-Technes expertise of proteins, reagents, media, and gene editing technologies with Fresenius Kabis Lovo cell processing system and the bioreactor expertise from Wilson Wolf with its G-Rex technology that is designed as a scalable platform for personalized cell therapies.

As processes develop and technologies evolve, the cell and gene therapy space will be confronted with new challenges. At Bio-Techne, the team is keeping an eye out for interesting trends that might affect the industry.

I see the induced pluripotent stem cell (iPSC) therapy field continuing to grow with more allogeneic cell therapies being developed, says Johnson. Allogeneic manufacturing is potentially less complicated than autologous manufacturing due to the ability to provide off-the-shelf products when patients need them.

Although the challenges in cell and gene therapy manufacturing remain a problem, companies like Bio-Techne are establishing quicker, simpler, and more automated options within quality control, manufacturing, and process development.

Wherever we go, we see newer technologies supporting cell and gene therapy manufacturing, says Clarke. Within our industry, changes come so rapidly and the treatments have shown so much promise that there is a lot of focus on cell and gene therapies. This puts a lot of pressure on us as an industry to provide these treatments. I believe that collaboration is the key to tackling this problem.

To learn more about the challenges in cell and gene therapy manufacturing and how to solve them, visit Bio-Technes website or get in touch with the experts here!

Images via Shutterstock.com

Author: Larissa Warneck

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Tackling the Challenges in Cell and Gene Therapy... - Labiotech.eu

For the cell and gene therapy revolution to be fullyrealized, physicians, research scientists, biomanufacturing experts, advocacygroups, regulatory bodies like the Food & Drug Administration (FDA) andother key stakeholders have to Think Different, as Apple famously encouraged.

The vein-to-vein, one batch to one patientformula of personalized medicine is radically and rapidly forcing changes onbiomanufacturing where cell and gene therapy best practices are not yetcalcified and are changing as we speak. Personalized medicine supply chainchallenges are emerging and still being worked through and the high cost ofthese therapies remains a daunting challenge for life science companies andpatients.

Cell and gene therapy challenges across R&D, manufacturing, commercialization, and supply chain as well as bioethical challenges yet to be fully confronted or resolved have been well documented and discussed by experts across the BioHealth Capital Region (BHCR).

One company Jeeva InformaticsSolutions, Inc. (Jeeva) in Herndon, Virginia is developing BigData solutions for a less publicized but equally urgent cell and gene therapychallenge: How to manage a radically new form of clinical trial that could spandecades. Founder and CEO of Jeeva, Harsha K. Rajasimha, is building a companythat leverages Big Data, AI and mobile tech to decentralize clinical trials.Jeevas goal is to make it easier for biotech companies to collect, aggregate,analyze and report required clinical trial data while helping patients stayenrolled and compliant over longer periods of time with little travel from theconvenience of their homes.

The FDA recently passed newindustry guidelines requiring long-term follow up (LTFU) periods ofup to 15 years for gene and cell therapies, representing a sea change for howclinical trials will operate in the future.

The cell and gene therapy space provides significant hope for cures that can reverse genetic mutations. This space is growing. There are more than 900 clinical trials ongoing. Hundreds if not thousands of genetic diseases are likely to have therapies for the first time in our lifetime, stated Rajasimha.

At Jeeva, we are trying to solve a number ofissues but have focused on one particular problem that needs to be addressed ifthese cell and gene therapies are going to be delivered to patients: No oneknows the long term implications of these therapies because they are a one anddone type treatment. If a patient receives therapy today, they are done, and sothe FDA has mandated that recipients need to be monitored for up to 15 years.We are looking to address this issue with digital health technologies and AI,he added.

Jeeva believes that these long-term monitoringchallenges can be managed by making clinical trial participation easier viamobile applications, video conferencing consultations and centralizedscheduling, to name just a few of Jeevas product features. Leveraging digitalhealth tech can reduce the need for travel and eliminate inconveniences thatmight cause a patient to become non-compliant or, worse, drop out of a trialaltogether.

Decentralized clinical trials eliminate the heavy burden of patient travel and makes the process simpler and more efficient. Utilizing a Bring-Your-Own-Device (BYOD) approach and an eVisit consultation model to create decentralized trials can reduce brick and mortar visits by 20% to 80%, according to Jeeva.

Thenew FDA guidelines is the latest attempt to grapple with unchartedsafety protocols for cell and gene therapies. Clinical trial challenges are notnew to the biotechnology industry, however; rather, the approach to clinicaltrials has been inefficient and static for decades, leading to industry-wideproblems with clinical trial enrollment and recruitment that has a dominoeffect that lengthens the commercialization process and increases drugdevelopment costs.

We want to be a catalyst for accelerating thedrug development and delivery process. Patient recruitment is a huge barrierand has made the biopharmaceutical industry unsustainable. The average cost ofbringing a drug to market is $2.5B and takes 10-15 years to get to market. Wefeel that by educating and informing the global community about clinical trialsand enrollment opportunities using AI and digital health tech, we can help allstakeholders in getting people earlier access to treatments and getting thetreatments to markets faster, Rajasimha said.

Jeeva not only can help small to midsize biotechs improve trial recruitment and longer-term safety monitoring, but the company uses AI to improve clinical trial operations. By using AI and high tech tools, biotech companies can leverage historical trial data to shape new trials while empowering real-time adjustments to trials based on real-time monitoring to improve overall success rates.

Rajasimha continued, We have been building AItools to solve our customers specific needs, not just for AIs sake. Wesurveyed our customers last year and they told us Every single clinical trialseems like the first trial ever conducted by mankind. Even companies likePfizer and Novartis, which have been conducting hundreds of trials for decades,feel that when they launch a new trialit is no more efficient than theprevious trial.

So, we have been building an AI assistant that learns from past clinical trials data to make the next trial more efficient, he added. Rajasimha quickly reinforced patient centricity by saying it is not something you fix with an all technology solution, unless the robustly tested technology solution is combined with the human elements and focus on patients perspectives. Having been a global patient advocate in the U.S. over the past six years has given me a unique perspective on how to integrate technology in the lives of patients and caregivers. Moreover, a growing number of trials are recruiting patients from multiple countries and reducing international travel burden on patients over extended durations will be critical to achieve enrollment.

While the concept of virtual clinical trialsmight seem futuristic, Rajasimha and the Jeeva team believe the market is readyfor change.

A number of pilot projects or proof of concept clinical trials, about 20 of them, have been published where patients didnt go to the clinic at all. The feasibility of conducting such remote patients studies has been validated multiple times by the industry now. The tipping point has arrived. One of the key barriers for widespread adoption of decentralized clinical trials was a lack of FDA guidelines. Now the FDA has clarified its expectations about how the industry and stakeholders can share the responsibility to reduce the burden on patients. Enough validation and regulatory guidelines have put us in a position to give our customers what they need, stated Rajasimha.

Rajasimha sees partnering with smaller to midsize biotechs early on in the drug development process meaning well before the start of Phase II or III trials as an inflection point where it can deliver the greatest impact. In addition, we are seeing some initial interest from the Medical Cannabis industry, opioid crisis intervention for chronic pain management, and patient advocacy groups, where patients often live in remote, rural areas, can also benefit from decentralized, hybrid virtual clinical trials. Finally, real-world evidence studies, or longitudinal cohort studies, is also a growing market because companies need to collect and manage patients across longer time horizons, which is Jeevas sweet spot.

Rajasimha and Jeeva are starting to see this growing market interest manifest itself in new funding partnerships. Jeeva recently announced that CIT GAP Funds had invested in the company. Jeeva is currently in an early-stage investment round and the company is in active product development with a validated prototype. Jeeva is seeking new customer pilot projects to add to its ongoing pilots, which include chronic pain, medical cannabis, oncology and cell, and gene therapy products. Later this year, the company plans to complete multiple pilot projects and have validation in Good Clinical Practices (GCP) settings.

Rajasimha and his Jeeva team are certainlyembracing a think different approach to the future of clinical trials. Jeevaand its AI-driven, virtual clinical trial model is poised to help biotechcompanies thrive and meet the unmet medical needs of more patients across theglobe.

You can listen to Rajasimhas interviewwith podcast host Daniel Levine earlier this month on iheart radio here.

Team Jeeva is seeking customer pilot projectsand strategic partners to join the journey and will be exhibiting at the NationalInstitutes of Health Rare Disease Day event on Feb 28, 2020.Rajasimha will also be delivering a keynote speech on AI in rare diseases atthe BIO-IT World West Conference at San Franciscoon March 3rd, 2020.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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This Startup is on a Mission to Decentralize Cell and Gene Therapy Clinical Trials - BioBuzz

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them or one of the couple of Americans who arrived later got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

We dont have too much time, Andre Kalil, the trials lead investigator, told Endpoints News. Everythings moving really fast.

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Biogen touts new evidence from the gene therapy company it wagered $800M on - Endpoints News

AffyXell Therapeutics (South Korea), the recently established joint venture between biotherapeutics and reagents supplier Avacta and Korean pharmaceutical company Daewoong, (Jan 8) is now working under a collaboration and licensing agreement with Avacta for the generation of new cell and gene therapies.

The two companies will develop Avactas Affimer proteins against a range of targets which, when produced by mesenchymal stem cells (MSCs), are intended to inhibit inflammatory and autoimmune pathways and improve the overall efficacy of MSCs, creating a next generation of stem cell therapies.

The initial focus for AffyXell will be on inflammatory and autoimmune diseases. In the longer term, there is potential for AffyXell to address oncology uses for these Affimer-enabled cell and gene therapies.

Dr Alastair Smith, Chief Executive Officer of Avacta Group, commented: The potential for AffyXells new class of cell therapies, which can be applied to a wide range of inflammatory and autoimmune diseases, is enormous. We are therefore very excited by the opportunity to be part of this new venture and to demonstrate the power of Affimer proteins in the field of engineered cell therapies.

Seng-ho Jeon, CEO of Daewoong Pharmaceutical, commented: We are very excited to contract this Collaboration and License Agreement followed by the successful establishment of an innovative joint venture, AffyXell. Daewoong and Avacta will make every effort to (help) AffyXells growth and its development of distinguished and innovative cell and gene therapy treatment.

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Gene Therapies to Incorporate Enhanced Immune-Modulatory Effects - Labmate Online

For most parents, their child's second birthday is one of the many exciting milestones of toddlerhood - a chance to share a toast with friends and family and look forward to a future filled with possibilities.

For Queensland parents Kellee and Jamie Clarkson, it's a date that fills them with dread.

Their 18-month-old daughter Wynter has spinal muscular atrophy (SMA), a degenerative genetic disorder that attacks the motor neurons in her spine, progressively weakening her muscles and shutting down movement.

Without treatment, children like Wynter with type 1, the most severe form of the disease, will never sit up, crawl or walk and won't live to see their second birthday.

A revolutionary new gene therapy called Zolgensma is Wynter's best chance at a life free of wheelchairs, breathing apparatus and thrice-yearly lumbar punctures that leave her screaming for help but she only has until she turns two.

So far, the gene therapy is only available through health care in the United States, with Japan on Wednesday becoming the second country to approve the drug for children under two.

"It's such a long shot it would be like winning the lottery," Wynter's mum Kellee told 9News.

"But Jamie and I won't give up we will pursue every avenue we can."

The couple, who live just outside of Toowoomba, embarked on a fundraising drive last November in a desperate bid to raise the money, but the $66,000 they've raised falls far short of the jaw-dropping sum they need.

Their best hope now lies in a global lottery-style draw announced by drug's developers Novartis in December, offering 100 doses of the drug for free in 2020 to children under two years old.

But with roughly 60,000 children diagnosed worldwide every year, the chances of being drawn remain slim.

The first names have already been selected in a series of fortnightly draws, but the drug company is remaining tight-lipped on where the patients are located and how many children have missed out.

Swiss pharmaceutical company Novartis has attracted heavy criticism from patient advocates and health advisors for the lottery-style format of its global "managed access scheme".

UK patient group TreatSMA applauded the company's effort to offer the drug for free, but said it was "yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease".

For parents, competing against other desperate families to gain treatment at the expense of others also comes at a psychological cost.

"It plays with your emotions," Kellee said.

"There are children worse off than Wynter and there are children better off. Wynter is older, she doesn't have as much time, so it would be hard to see a child who is much younger get it.

"It's so hard that there is such a big amount of money involved to give your child the best life, and it's just by chance that your child could get this drug."

Novartis' Director of Communications and Advocacy, Peter Murphy, defended the managed access program as "anchored in principles of fairness, clinical need and global accessibility".

He told 9News the company had sought advice from bioethicists as well as doctors and patient advocates before launching the program, and they had concluded this was the best way to ensure equity regardless of country of residence and capacity to pay.

With only one facility currently approved to manufacture the drug, they simply can't produce enough doses quickly enough to provide one to every child.

As they wait and hope for a miracle, life for the Clarksons centres around a gruelling schedule of daily physio exercises, fortnightly occupational therapy and speech therapy visits and the daily battle to keep Wynter from getting sick.

"Wynter can't sit on her own, she can't crawl, she obviously can't walk... She needs help with everything. I would do it to the day I die, but it's so hard to watch other kids be able to do those things," mum Kellee said.

As well as impacting gross motor skills, SMA can affect the muscles used for swallowing and breathing, meaning Wynter needs a BiPAP machine when she sleeps and finds the seemingly simple task of eating exhausting.

"We spend a lot of time trying to get her enough nutrition. It takes a lot out of her to eat it's like running a marathon," Kellee explained.

"Wynter at the moment is really struggling with her weight gain and it is a real possibility that she will have to have a gastric tube put in."

With SMA affecting Wynter's ability to cough and expel mucus, even going outside and playing with other children can have life-threatening implications.

"We're constantly worried about her getting sick. I'm constantly thinking about 'oh I don't want to go to the shopping centre because she could get a cold and die'," Kellee said.

That almost became a reality last year, when Wynter caught the common cold and ended up in ICU for two weeks fighting for life.

"She required lots of deep suctioning to the lungs, they put a catheter down her nose and mouth and she was on a BiPAP 24/7," Kellee said.

"It was the most traumatic, distressing experience of my life and my husband's."

Currently, the only medication available to treat SMA in Australia is Spinraza, a drug that has halted the devastating progression of Wynter's disease but has failed to give her back her compromised lung and swallowing functions.

It also means hospital visits every four months for the rest of her life.

"She's 17-months-old and she's had seven lumbar punctures," Kellee said.

"They can't give them any anaesthetic and she has to have it awake."

"It is absolutely horrendous to watch. As a parent, it just absolutely crushes you to see your child in pain and looking at you and wondering why you're allowing this to happen."

Patient advocacy group SMA Australia's Julie Cini said she understood the Clarkson's desperation to access Zolgensma, but the harsh reality was that some children would miss out while the drug was pending approval in Australia.

"It's like dangling a carrot in front of someone and then chucking it in the bin," she said.

Ms Cini is currently working to have the gene therapy approved under the Pharmaceutical Benefits Scheme, which would allow every child in Australia to access the drug.

Novartis applied to the Therapeutical Drug Administration in late 2019, but approval could still take many months, even years.

While paralysing her body, SMA has left Wynter's mind untouched, and Kellee says she remains a bright and bubbly toddler with a brilliant sense of humour.

"She has a very funny personality, she's very bright. A very happy little girl loves to dance, loves to laugh. She's at that stage where she's getting cheeky in a good way.

"She's got a powered wheelchair she drives around perfectly she doesn't know any different.

"To us, she's just perfect."

SMA advocate Ms Cini said it was important to remember just how far treatment had come.

She knows all too well the gut-wrenching trauma of watching your child's slowly but inevitable progress towards death.

"I look at Wynter every single day and I see what I couldn't see in my children," Ms Cini said.

"What she can do is phenomenal my kids couldn't do what she's doing. My outcome was death. The (Clarksons) have a chance to have a life with their child."

Ms Cini is optimistic that new treatments and early detection could see a future where those with SMA, even in its most severe form, could live long and independent lives.

A national newborn screening program to detect SMA before symptoms take hold vital in limiting the disease's progress - is currently being piloted in NSW.

"Hopefully, when drugs like Zolgensma get passed, we're able to treat our kids within the first two weeks of life," she said.

"I can see the future of SMA, I'm going to walk in to a room and I won't even know which kid has it."

For Kellee and Jamie Clarkson, that day can't come soon enough.

Read more from the original source:
Parents' fight to access life-changing $3.2m gene therapy for their daughter - 9News