Category Archives: Genetic Therapy

The frequency with whichspinal muscular atrophy (SMA) is found in newborns may be lower than previously thought, according to one-year data from an SMA screening program in the state of New York.

Based on expected disease frequency, the program anticipated that 20 to 38 of the more than 225,000 newborns screened would have tested positive for the neuromuscular disease, but only eight babies were identified as having SMA.

Results from other state screening programs underway will help to better assess SMAs actual frequency in the U.S., the researchers said.

The study, Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy, was published in the journal Nature: Genetics in Medicine.

Spinal muscular atrophy is the most common genetic cause of death in infants and children, with an estimated frequency of between 1 in every 6,000 and 1 in every 11,000 births.

In most cases (95%98% of all incidences), it is caused by the complete loss of exon 7 in theSMN1gene, which affects the production of SMN, a protein essential for muscle health. Exons are the sections of a gene that contain instructions for protein production.

The existence of a secondSMNgene, calledSMN2, partly compensates for the loss ofSMN1-produced SMN.Typically, the moreSMN2genecopies a person has, the less severe the disease.

SMA is inherited in anautosomal recessivemanner, meaning a child must acquire two mutated copies of theSMN1 gene one from the father and one from the mother to develop the disease. People with only one defectiveSMN1 copy will not have this disease but will be carriers, meaning they can pass the mutation to their children.

Given the approval of a first SMA treatment Spinraza, by Biogen in late 2016, the findings of a pilot SMA newborn screening study in New York state, and the importance of early detection and treatment in preventing lifelong disability, SMA was added to theU.S. Recommended Uniform Screening Panel(RUSP) for newborns in 2018.

As of March, 19 states were screening all newborns for SMA, and others either had legislation pending or were running pilot screening programs. To date, none of these states have reported on SMA frequency based on their screening programs.

Newborns are screened for SMA via a genetic test that checks for the absence of exon 7 in both SMN1 copies. In some states, such as New York, the program also includes assessingSMN2 copy number in infants screening positive for SMA.

Researchers looked at data from Oct. 1, 2018, through Sept. 30, 2019 the first full year of the New York states SMA newborn screening program.

Of the 225,093 infants screened, eight carried exon 7 deletions in both copies of SMN1. Three of these babies had two copies of SMN2 (likely to develop SMA type 1, a severe form), three had three copies (likely to develop type 2 or 3 disease), and two had at least four copies (likely to develop SMA type 3 or 4).

All infants were asymptomatic at diagnosis, and seven were treated with Zolgensma (byAveXis,a subsidiary of Novartis) an SMA gene therapy approved in May 2019 including two babies (with two and three SMN2 copies) initially treated with Spinraza.

All continued to show no symptoms of the disease at a last follow-up (ranging from two to 12 months). Oneinfant with at least four SMN2 copies was not treated, and is being carefully monitored long-term for signs predictive of disease onset, the researchers wrote.

None of the parents had reported knowing of a family history of SMA.

The team noted that, based on current SMA incidence estimates, 20 to 38 newborns were expected to screen positive for SMA among newborns in New York, a number far above the eight positive infants found.

The New York program had an SMA frequency of 1 in 28,137 births (range of 1 in every 14,259 to 55,525 births), which was between 2.6 and 4.7 times lower than expected (1 in 6,000 to 11,000 births).

Including data collected through the end of February 15 positive cases among some 314,000 newborns screened the resulting frequency was still low, about 1 in 21,000 births, the team noted.

This low SMA frequency, it added, cannot be solely attributed to the 2%5% of SMA patients missed by newborn screening, due to other SMA-causing mutations than exon 7 deletion.

Instead, this discrepancy is likely due to imprecise or biased estimates (most are based on small European populations that may not be representative of the U.S. population), as well as better informed reproductive decisions such as increased awareness, access to and uptake of carrier screening, genetic counseling, cascade testing, prenatal diagnosis, and advanced reproductive technologies, the researchers wrote.

Longer-term data from this and other screening programs will shed light on the true incidence of SMA in the U.S., they added.

They also recommended strongly that screening programs continue, and expand across states.

SMA newborn screening ensures equity in diagnosis of a common genetic condition in infants, such that affected children of families who cannot or choose not to undergo carrier screening are universally afforded the same benefit of new, life-saving treatments that are most effective when identified prior to symptom onset, the researchers wrote.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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SMA Less Common Than Expected Among Newborns Screened in NY - SMA News Today

Its reasonableto assume immunotherapies such as PD-1 inhibitors, which unleashthe bodys own immune system to target and destroy cancer, work best in hot tumors that are flooded with immune cells in their microenvironment. But a new study by scientists at the Dana-Farber Cancer Institute found that is not the case in kidney cancer.

The researchers discovered that in advanced clear cell renal cell carcinoma (ccRCC)the most common form of kidney cancertumors that were infiltrated with large numbers of CD8 T cells were less likely to respond to Bristol Myers Squibbs PD-1 inhibitor Opdivo than cold tumors were.

The findings, presented at the American Society of Clinical Oncology virtual event and published in Nature Medicine, provide critical insights that may help predict which patients are more likely to benefit from immuno-oncology agents, the researchers argued.

The Dana-Faber scientists examined 592 tumors collected from three Opdivo kidney cancer clinical trials in an attempt to draw a correlation between patient outcomes and immune and genomic biomarkers. They discovered that kidney cancer deviates from several well-known tenets of cancer treatment. Normally, tumors containing a large number of neoantigensproteins formed as a result of tumor mutations and therefore new to the immune systemare often more susceptible to immunotherapy. But that didnt affect ccRCC responsiveness to Opdivo, the team found.

Perhaps most surprisingly, hot tumors with high levels of CD8 T cells didnt respond well to Opdivo, either. But why?

The researchers found that these hot tumors were depleted of mutated PBRM1 genes, which are often associated with improved survival from PD-1 blockade. Instead, they had more of an unfavorable genetic featurethe loss of a chromosomal segment called 9p21.3. When found within hot tumors, deletion of 9p21.3 was associated with worse clinical benefit and survival after PD-1 treatment.

We believe that these two factors may explain why CD8 T cell infiltration of the tumors did not make them responsive to checkpoint blocker therapy, while other types of cancer that exhibited CD8 T cell infiltration but did not have those chromosomal changes did respond, explained co-authorSachet Shukla, Ph.D., chief of the computational group at the Dana-Farber Translational Immunogenomics Laboratory,in a statement.

RELATED:Could the anti-cancer gene p53 be a target in treating kidney cancer?

The Dana-Farber study offers clues to mechanisms that contribute to response and resistance to PD-1 drugs in ccRCC and possibly other types of tumors as well, the researchers suggested. It can help identify patients most suitable for these immuno-oncology drugs and provide fundamental information to aid in development of rational combination therapies to overcome resistance in the future, said study co-author Toni Choueiri, M.D., director of the Lank Center for Genitourinary Oncology at Dana-Farber.

The presence of high numbers oftumor-infiltrating immune cells isoften linked to better immunotherapy treatment outcomes. Thats why scientists are constantly looking for ways to turn coldtumors hot. In October, a Yale University team described a method for using gene-editing system CRISPR to make tough-to-spot tumors more visible to the immune system.

Another approach aimed at improving immuno-oncology in kidney cancer involves combining immune-boosting treatments. A combo of BMS' Yervoy with Opdivo was approved for first-line treatment of kidney cancer in 2018. In February of this year, the company unveiled new data showing 56% of patients taking the combo in a trial were still alive at 42 months, versus 47% of patients taking Pfizer's Sutent alone.

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Why 'hot' kidney tumors don't respond to immunotherapy with PD-1 blockers - FierceBiotech

Five years ago, as much of pharma began leaving neuroscience, three big-name scientists from Genentech and some A-list investors, including ARCH and Flagship, made a $217 million bet that new genetic insights and a reliance on biomarkers could bring them success. They called it Denali Therapeutics.

Still, Denali faced the problem that neuroscience developers have faced for decades: How do you get a large molecule across the blood-brain barrier, a natural defense evolved precisely to keep them out? Enzyme replacement therapy, for instance, would be a great candidate to treat several neurological disorders, but enzymes cant cross the barrier.

Now, Denali thinks theyve solved the problem, or at least part of it. In a pair of papers published inScience Translational Medicine,the South San Francisco biotech detailed the invention of a new transport vehicle to sneak large molecules past the brains gates. So far, its been used in mice and monkeys, but they wont wait long to bring it to patients: A clinical trial using it to replace an enzyme lost in people with Hunters syndrome is set to begin this year, with proof-of-concept data expected to come before 2021.

The blood-brain barrier consists in part of tightly packed endothelial cells. Since certain molecules, such as insulin, cross the barrier by first binding to receptors on these cells and then being allowed through, scientists have long tried to build antibodies that can similarly bind to these receptors and shuttle across a therapeutic cargo. But the results, over several decades, have been less than transformative.

CEO and founder Ryan Watts has been part of that search since his Genentech days. The research method he and Denalis scientists came up with began with a process called directed evolution in which a protein is induced to mutate repeatedly, until it gives rise to a protein with the qualities you want to build a protein, called an FC fragment, that binds to whats called a transferrin receptor, a node that normally imports iron into the brain. In theory, there are numerous drugs one could then hook onto that Fc fragment, but Denali first tested it with an antibody-targeting enzyme called beta-secretase. The enzyme is linked to the build-up of amyloid plaques in people with Alzheimers, and the researchers showed their vehicle reduced the amount of amyloid in mice and monkeys.

In a second study, the researchers attached an enzyme called iduronate-2-sulfatase, the critical protein that people with Hunters syndrome are missing. Without it, sugars called glycosaminoglycans build up in cells, causing abnormalities in several different organs. Shire gained approval for an enzyme replacement therapy in 2006, but it only works outside the brain (the companys erstwhile efforts to improve cognitive function yielded little promise). Using the transport vehicle, though, Denali was able to get significantly increased brain penetration of the enzyme and reduce the pathology in mice and monkeys.

Denali played up the potential versatility of their approach over other blood-brain-barrier-crossing proposals, such as bispecific antibodies, saying you can attach a greater range of therapies to their vehicle. The company has over a dozen programs including a Parkinsons one now in the clinic but the first test of the vehicle will be later this year, in 16 kids with a rare disease whose worst symptoms remain untreated.

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Denali unveils new way of crossing blood brain barrier as the big neuroscience bet enters its clinical years - Endpoints News

Others at the center, which coordinates research at eight universities, are reviewing past work on drugs that showed potential against the SARS epidemic in 2002 and the MERS outbreak in 2012. Both diseases are also caused by coronaviruses.

At some point, though, scientists at the center and across the country will need to focus on future threats and break new ground.

You can make a lot of movement fast, based on what you know, Satchell said. But at some point, you hit a wall where you have to discover new things.

Andy Mesecar needs a weekend off.

An expert in biochemistry and gene therapy, hes worked seven-day weeks for the last few months, racing to find a drug for patients with COVID-19 while teaching at Purdue University and submitting daily reports to the National Institutes of Health. The center at Northwestern has funded his work since 2018.

Mesecar has a manuscript under review in a scientific journal on how a drug approved to treat hepatitis C could be modified to potentially treat COVID-19. His lab is one of the leading centers studying coronaviruses, and he and his team have dedicated nearly two decades to the research. But that was still not enough to rapidly create an effective therapy specific to COVID-19.

With a background in biochemistry and structural biology, Mesecar started out as an assistant professor at the University of Chicago, studying enzymes that could fight cancer. He got into infectious diseases after the anthrax attacks in the weeks after 9/11. Then, he pivoted to studying enzymes that could be used against SARS when it surfaced in November 2002.

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Everything we did was to predict the next outbreak. Yet scientists werent prepared for COVID-19. Why? - Longview Daily News

INDIANAPOLIS A dime-size nanochip developed by a world-renowned researcher who recently relocated to Indianapolis could help transform the practice of medicine. It could also turn Indianapolis into a manufacturing and research hub for radically new disease and trauma treatment techniques.

It all began in August 2018, when Chandan Sen, one of the worlds leading experts in the nascent field of regenerative medicine, moved his lab from Ohio State University to the Indiana University School of Medicine. He brought along a team of about 30 researchers and $10 million in research grants, and now serves, among a myriad of other positions, as director of the newly formed Indiana Center for Regenerative Medicine and Engineering, to which IU pledged $20 million over its first five years.

IU recruited Sen away from Ohio State in part because of its desire not just to promote academic research in his field but also to help develop practical, commercial products and uses for his breakthroughs.

A scientist prefers to be in the lab and keep on making more discoveries, said Sen, 53.

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But I thought that, unless we participate in the workforce development process and the commercialization process, I dont think that the business people would be ready to do it all by themselves. Because its such a nascent field.

Its definitely new and its potential sounds like the stuff of science fiction.

Regenerative medicine, as its name hints, seeks to develop methods for replacing or reinvigorating damaged human organs, cells and tissues.

For instance, instead of giving a diabetic a lifetimes worth of insulin injections, some of his skin cells could be altered to produce insulin, curing him. Such techniques might also be used for everything from creating lab-grown replacement organs to, someday, regenerating severed limbs.

Regenerative medicine offers a form of medicine that is neither a pill nor a device, Sen said.

It is a completely new platform, where you dont necessarily depend on any given drug, but are instead modifying bodily functions.

A big, tiny breakthrough

Sen and his teams signal contribution to the field is a technique theyve dubbed tissue nanotransfection, or TNT. Put simply, it uses a nanotechnology-based chip infused with a special biological cargo that, when applied to the skin and given a brief electrical charge, can convert run-of-the-mill skin cells into other cell types. Potentially, the technique could be used for everything from regrowing blood vessels in burn-damaged tissue to creating insulin-secreting cells that could cure diabetics.

Obviously, such applications are still down the road a ways. But the technology is far enough along that some products are already making it to marketand investors, entrepreneurs and established companies are sniffing around for opportunities. According to the Alliance for Regenerative Medicine, more than 1,000 clinical trials worldwide are using regenerative medicine technologies.

Thousands of patients are already benefiting from early commercial products, and we expect that number will grow exponentially over the next few years, said Janet Lambert, the alliances CEO.

Lambert predicts that the number of approved gene therapies will double in the next one to two years. Last year, the U.S. Food and Drug Administration predicted it would be approving 10 to 20 cell and gene therapies each year by 2025.

These new techniques could do more than just revolutionize medicine. They could also upend the medical industry as we know it. And the IU School of Medicineand Indianapoliscould lead the way.

There are really only two or three places in the country that did the kind of comprehensive work that Dr. Sens group was doing, said Anantha Shekhar, executive associate dean for research at IU School of Medicine. And they were doing it from the lab all the way to the clinic, where they were already applying those technologies in patients.

So it was very attractive to think of starting with a bang bringing a comprehensive group here and creating a new center.

Ambitious goals

Instead of merely treating chronic conditions, regenerative medicine could end them, once and for all.

For instance, consider a car with an oil leak. The traditional medical approach might be to live with the chronic condition by pouring in a fresh quart of oil every few days. The regenerative medicine approach would fix the leak. Its good for the car, good for the cars owner but not necessarily good for the guy who was selling all those quarts of oil.

Which is why these new techniques, if they catch on, could cause turmoil in the medical industry.

Because regenerative medicine has the potential to durably treat the underlying cause of disease, rather than merely ameliorating the symptoms, this technology has the potential of being extremely disruptive to the current practice of medicine, Lambert said.

This has the potential to be hugely disruptive, Sen added, because so much of medicine today relies on huge industrial infrastructures to manage, not cure, chronic diseases and disabilities.

If such disruption comes to pass, the leaders of 16 Tech, a 50-acre innovation district northwest of downtown that aspires to house dozens of medical-related startups and established firms, would love to be its epicenter.

The Center for Regenerative Medicine will be one of the tenants of 16 Techs first building, a $30 million, 120,000-square-foot research and office building scheduled to open in June.

Regenerative medicine is probably one of the next major waves of medical innovation in the world, 16 Tech CEO Bob Coy said. To have him here doing this work gives Indianapolis and Indiana an opportunity to develop an industrial cluster in regenerative medicine.

Coy believes the most momentous early step on that road was the recent establishment by Sen of masters and doctoral programs in regenerative medicine at the IU School of Medicine. Its the first degree of its type in the country, earning IU and Indianapolis the enviable status of first mover.

I think, for example, of [Pittsburghs] Carnegie Mellon University, which, back in the late 1960s, created the first college of computer science in the country, Coy said. And now you know Carnegie Mellons reputation in computer science.

What isnt in place yet is a state or city program to promote development of a regenerative medicine hub.

We need to start doing that, Coy said. That means putting a lot of the infrastructure in place to support startups that are based on this technology, as well as recruiting companies that want to collaborate with Dr. Sen.

In spite of the lack of a coherent recruitment program, Coys phone has started to ring, thanks largely to Sens presence.

There have been a few meetings Ive had with people who already have relationships with him, who, when they come to town, have reached out to meet and talk about what were doing at 16 Tech, he said.

Fueling entrepreneurship

One of the first 16 Tech startups with designs on the regenerative medicine niche is Sexton Biotechnologies.

The company was groomed by Cook Regentec, a division of Bloomington-based Cook Group charged with incubating and accelerating technologies for regenerative medicine and the related field of cell gene therapy.

Any products that show promise are either folded into the company, turned into their own divisions or, as in Sextons case, spun off as an independent entity with Cook retaining a financial stake.

Its a measure of the newness of this field that Sextons 17 employees arent working on new medicines, but rather marketing basic tools needed to conduct research. The companys offerings include a vial for storing cell and gene products in liquid nitrogen, and a cell culture growth medium.

Theres a ready market for such tailor-made gear, because, for years, researchers in the regenerative medicine field had to make do with jury-rigged equipment.

What most of those companies did was repurpose things like tools from the blood banking industry, or tools from bio pharma, said Sean Werner, Sextons president.

So thats why a lot of newer companies are starting to build tools explicitly for the industry, as opposed to everybody just having to cobble together stuff that was already out there.

Werner said investors recognize the momentous opportunity in regenerative medicine and are flocking to the field.

Its not something you have to explain, he said. Companies and VC groups are trying to get a piece of it.

What has investors and medical researchers charged up is the almost unlimited range of potential applications, from healing burns to, perhaps someday, regenerating limbs.

I think it would be a huge revolution if were able to, for example, regenerate insulin-secreting cells in children who have become juvenile diabetics or have for whatever reason lost their pancreas, Shekhar said. Those are the kinds of things that will start to change the way we see certain diseases.

Lambert predicted that, as the science advances, so will the business case.

While early programs focused primarily on rare genetic diseases and blood cancers, were already seeing the field expand into more common age-related neurological disorders, such as Parkinsons and Alzheimers, she said.

I expect this trend to continue in the coming years, greatly increasing the number of patients poised to benefit from these therapies.

Werner said regenerative medicine also is seeking advancements in manufacturing technologies that will lower the cost of product development.

It all adds up to a huge opportunity the state is well-positioned to seize, Werner believes.

Indiana is a perfect place for this kind of thing to really ramp up, he said. Theres no reason we cant lead the field.

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IU team pursuing breathtaking advancements in regenerative medicine - The Republic

Gene therapy does more than treat genetic diseases it can cure them. A one-time dose of a non-replicative viral vector, such as commonly used recombinant adeno-associated virus (AAV), delivers a functional gene to replace or compensate for a dysfunctional version that is causing a patients disease (Figure 1). As a cutting-edge biopharmaceutical technology, there are multiple gene therapies now FDA approved; with hundreds more in clinical trials, were likely to see many more of these therapies on the market soon.1 However, to keep up with the rapid pace of clinical research, developers are working to streamline the manufacturing and quality control process to improve quality and lower the cost of bringing these important drugs to market.Developers use a multitude of analytical tests to develop gene therapies and optimize their manufacturing process. When developers get aberrant test results, they must be able to interpret where the problem lies. Did the manufacturing process produce an undesirable product, or is the analytical testing method unreliable? Analytical testing companies that have the infrastructure, personnel, and experience often partner with developers to tighten up analytical variability so that results of tests clearly indicate where there are opportunities to increase efficiency and product quality.

Figure 1. Gene delivery by recombinant viral vector.During gene therapy, viral capsids containing the therapeutic gene are taken up by the patients cells and the genetic material is delivered to the nucleus. There, the gene gets expressed as a protein necessary for the patients health. Credit: Avomeen.

Figure 2. A full AAV capsid and associated capsid impurities. Complete viral capsids have AAV are assembled from 60 capsid proteins, with a defined stoichiometry and shape and contain a therapeutic gene. AAV vector impurities include capsids that contain too many copies of the gene (overfilled), those that contain lower copy numbers or truncations of the gene (partially full), or empty capsids that contain no genetic material. Credit:Avomeen.

There are several ways to measure the empty/full capsid ratio, and as developers are establishing their chemistry, manufacturing and control (CMC) protocol, it is important that they choose an optimized method, as they must use that method for effective quality control from early process development to lot release and stability.3 Gene therapy developers may choose analytical ultracentrifugation to evaluate capsids, but while highly effective, this method is not as quantitative, robust or efficient as some newer methods. High-performance liquid chromatography (HPLC) using AAV full/empty analytical columns have been demonstrated to be highly effective at separating full, empty, and improperly filled capsids for robust quantification. Additionally, this method is higher throughput than ultracentrifugation, and requires less precious AAV sample to run.

Cellular potency is evaluated by transducing cells with the AAV product and then measuring a phenotypic or functional outcome due to the transduction. Developing these tests can be challenging because there is no one-size-fits-all test that will give developers the answers they need. Developers often draw on the experience of analytical labs to determine how to best evaluate their AAV products transduction efficiency.A gene therapy in development must also be tested to ensure that it is free of residual, process-related impurities such as polyethylenimine, iodixanol, poloxamer, and other excipients that must be removed in the final product to ensure safety. Few research and manufacturing facilities have the equipment and expertise necessary to perform this kind of testing, and it is advisable to find one that has experience testing polymers, extractables and leachables to examine if components of the manufacturing equipment or drugs packaging are not contaminating the final product.

As fast-paced as the gene therapy field is now, it stands to become a true race to the finish line to bring new gene therapies to market in the near future. Regulatory bodies are becoming more familiar with reviewing gene therapies, and the road to commercialization will move more quickly. There is no denying that gene therapies will bring incredible benefits to patients, but it will be crucial to improve manufacturing efficiency and lower costs to make gene therapies more accessible to the patients who need them.References

1. Colasante, W., Diesel, P., and Gerlovin, Lev. (2018). New Approaches To Market Access And Reimbursement For Gene And Cell Therapies. Cell & Gene. Retrieved from: https://www.cellandgene.com/doc/new-approaches-to-market-access-and-reimbursement-for-gene-and-cell-therapies-0001

2. Fraser Wright, J. (2014). Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment. Biomedicines, 2, 80-97; doi:10.3390/biomedicines2010080

3. U.S. Food & Drug Administration (2019). Guidance for Human Somatic Cell Therapy and Gene Therapy. Retrieved from: https://www.fda.gov/animal-veterinary/guidance-industry/chemistry-manufacturing-and-controls-cmc-guidances-industry-gfis

4. Stein, R. (2019). At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever. NPR. Retrieved from: https://www.npr.org/sections/health-shots/2019/05/24/725404168/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever

5. Cohen, J.T, Chambers, J. D., Silver, M. C., Lin, P., Neumann, P.J. (2019). Putting The Costs And Benefits Of New Gene Therapies Into Perspective. Health Affairs. Retrieved from: https://www.healthaffairs.org/do/10.1377/hblog20190827.553404/full/

6. ATCC (accessed May, 2020) ATCC Virus Reference Materials. Retrieved from: https://www.atcc.org/en/Standards/Standards_Programs/ATCC_Virus_Reference_Materials.aspx#

7. U.S. FDA (2020). FDA Details Policies on Gene Therapies in Seven Guidances. Retrieved from: https://www.fdanews.com/articles/195767-fda-details-policies-on-gene-therapies-in-seven-guidances

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Troubleshooting the Development of New Gene Therapies - Technology Networks