Groundbreaking gene therapy treatments have tremendous potential to provide treatment to the around one in ten Americans living with rare diseases. But these game-changing therapies also bring with them new challenges, including how the individuals who need them most will get access to and be able to pay for these expensive treatments.

To tackle this issue, we convened a panel of health leaders with ample experience in rare disease treatment and gene therapy to talk through how our health system can approach paying for gene therapy. In our latest 5 Slides Were Watching conversation, Jennifer Hodge, PhD, US Rare Neurology Medical Team lead at Pfizer, Angela Ramirez Holmes, founder and president of Cal Rare, and Ryan Fischer, chief advocacy officer at Parent Project Muscular Dystrophy, discuss potential solutions to this complex issue.

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Hodge brought a slide that illustrated the benefits of gene therapy, including the potential for one-time treatments that can eliminate the need for ongoing therapies and reduce the long-term economic impact of treating rare diseases. She also listed some of the challenges to adopting widespread use of gene therapy, such as the need for rare disease specialists to deliver care and ensuring patients get appropriate and timely care. She said 95% of individuals living with rare diseases have limited or no approved treatment options.

It really makes the rare disease population, collectively, one of the most underserved communities in medicine today. And that, frankly, is unacceptable to me.

Holmes focused on Medi-Cals role in rare disease treatment. Her slide cited that 51% of births in California are funded by Medi-Cal and 53% of the states children under five receive Medi-Cal coverage. She said Medi-Cals significant share of state health care coverage indicates the state needs to prioritize policies that make treatment accessible for Medi-Cal recipients with rare diseases.

With nearly four million rare disease patients in California, we know that policy solutions will be needed. And if we dont address the people that are covered by Medi-Cal, we will leave behind that piece of access for patients.

Fischer shared a slide that detailed ways California can advance rare disease treatments: continuing to engage with rare disease experts, leveraging investments in data collection, and continuing patient-focused drug development (PFDD).

[Its important] that we recognize that were really part of one drug development ecosystem. Its essentially a community of stakeholders living in conjunction with other components and their environment, all interacting as one system. We have to recognize that and recognize the needs of all stakeholders within that ecosystem.

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Video: Gene therapy and the promise for rare disease - State of Reform

COVID-19 disrupted cell and gene therapy trials as well as the contractors and suppliers that support them, according to experts.

SARS-CoV-2, the virus that causes COVID-19, impacted all parts of biopharma. For example, the focus on vaccines highlighted manufacturing capacity limitations while increased demand for consumables and raw materials put pressure on suppliers.

The pandemic also disrupted R&D to the extent that some developers put projects on hold according to Amelie Boulais, head of market entry strategy at Sartorius, who said during the BioProcess Insider State of the Industry webinar that the problems fed back up the supply chain.

The entire supply chain for the industry has been stretched by the pandemicwe try to mitigate any risk of shortages or unanticipated disruptions, we monitor our stock levels during daily operations.

But all our strategies rely on forecasts from our customers and, with COVID, suddenly, some customers working, for example, in the gene therapy field, just stopped production because the trials were on hold.

The disruption of supply chains had less of an impact on the cell therapy industry according to Patrick Lucey, CEO of CDMO Lykan Bioscience.

We actually have avoided a lot of these delays in the cell therapy space. Obviously, the amount of materials we use is reasonably small scale were not looking for major demand of excipients, or media components or buffer components or disposables.

So weve been able to navigate this in a couple of ways. First, you know, our supply chain team was pretty forward looking. So there are common materials across all biologics manufacturers, cleaning materials, and things like this. So we moved quickly and stockpiled a lot of these cleaning materials and things to be prepared for changeover and all that work.

And then on the on the client or processing material side, again, cell therapy is reasonably small scale, so not a huge demand for common raw materials, we do use a shared raw material approach across clients, so they have a security of supply there.

But the cell therapy sector has not escaped the disruption according to Lucey who told delegates clinical trial activities were impacted.

Where COVID did impact cell therapy is a lot of these therapies in terms of clinical trials require access to ICU for side effects. And certainly at the peak of COVID we saw ICUs in the US and globally overwhelmed with COVID patients.

So the availability for the proper resources from that perspective, were constrained, and therefore, clinical trials were delayed, Lucey said.

The positive news is that many trials halted during the early months of the pandemic have resumed. Partly this is because trial sites have reopened or sponsors have switched to decentralized models. In addition supply chains have recovered or been modified.

Vectors are an exception according to Lucey, who says that, despite commitments to increase production capacity, sourcing is still a challenge for the cell and gene therapy space.

The one area that continues to dog the cell and gene therapy segment is really the access to lentiviral, or viral vectors in general. Weve seen a lot of capacity go up for viral vector production, but yet we still see some of our clients challenged with sourcing.

And so thats certainly an area that thats still challenging from a capacity perspective. But otherwise, I think we are we are getting back to normal in the cell therapy side.

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COVID hit cell and gene therapy trials and suppliers - BioProcess Insider - BioProcess Insider

When Misty Lovelace was a baby, her eyes were drawn to the light.

She could not focus on faces, only sources of light. Her grandmother Cynthia Lovelace, who would become her main caretaker, suspected vision problems.

By age three, Misty was diagnosed as legally blind. School systems struggled with how to handle her. She was intelligent and intuitive, but people would treat her as if she had a learning disability.

As she got older, Misty started carrying a lamp with her at school. She would put her lunch under it to see what she was about to eat. She learned Braille and used a cane to navigate. When she visited the doctor for checkups, her prognosis seemed to get worse.

"[The doctor] would take her little face and he'd put his hands on her face and say, 'Misty, I'm so sorry, there's nothing more we can do for you, honey. You're going to wake up in the dark one day,'" Lovelace recalled.

"It'd be like looking through a tunnel. And all of a sudden that tunnel goes out."

Misty has Leber congenital amaurosis, or LCA, a genetic disorder that often manifests at a young age, causing vision loss. In Misty's case, and for approximately 1,000 to 2,000 other people in the U.S., the disease is caused by mutations in a gene called RPE65.

Misty Lovelace, age 4

Courtesy of Misty Lovelace

What Misty didn't know as her vision got darker was that a scientist and doctor duo at the Children's Hospital of Philadelphia had already spent years working on a gene therapy for her disease.

The gene therapy, which would eventually become known as Luxturna, was not an overnight success. Decades of research and setbacks preceded the landmark U.S. approval of Luxturna four years ago, the first the Food and Drug Administration had ever granted to a gene therapy for an inherited disease. While Luxturna is not a cure for blindness, treatment has brought sustained improvements in sight, particularly in lower light, for several patients who spoke with BioPharma Dive. As a result, they've needed less help in educational and social environments, and have more independence.

Their experience with Luxturna is proof of gene therapy's potential as well as its limitations. As the first gene therapy of its kind, Luxturna also holds lessons for a field that's grown dramatically since its December 2017 approval.

Lovelace said she never stopped trying to find a way for Misty to regain her sight. The possibility gave her hope as she watched her granddaughter adjust to a life that, for her, was almost in total darkness.

A call from Jean Bennett was a lifeline.

Bennett and her husband, Albert Maguire, met at Harvard Medical School in the early 1980s. The two began researching gene therapy together, attempting to treat blindness in mice. Soon they were testing their approach on Briard dogs with the same defective RPE65 gene that causes LCA in humans.

By 2007, their gene therapy was ready to be tested in people a high-stakes proposition for a field that had largely been shut down nearly a decade before. After 18-year-old Jesse Gelsinger died during a 1999 gene therapy study, many questioned whether such research was safe. The success Bennett and Maguire had with Luxturna was a large part of gene therapy's journey back to the forefront of biomedical research, aided by improvements in how such treatments are designed and delivered.

Testing began at the Children's Hospital of Philadelphia, where Misty was recruited as a study participant. At age 12, she took her first flight out of Kentucky and received the gene therapy in both eyes, starting with the one with worse vision.

"We didn't know if I was going to get worse, stay the same or get better," she said. "But in my mind, I was going to be completely blind by 18, so what's knocking a couple years off?"

The improvements were almost immediate, however. Lovelace recalls her granddaughter commenting on her wrinkles as soon as the eye patches from the procedure were removed. Misty could make out the fine hairs on the manes of horses, her favorite animal and hobby. Rainbows and stars, though, she found underwhelming.

More than eight years later, Misty says she's grateful she "took the leap," attributing to Luxturna her independence and ability to pursue a career as a horse trainer.

Misty Lovelace

Courtesy of Misty Lovelace

Results from early participants like Misty led to the formation of Spark Therapeutics and a larger clinical trial in Pennsylvania and at the University of Iowa that gave the biotech company the evidence needed to approach the FDA.

On Oct. 12, 2017, a panel of scientists and FDA advisers unanimously endorsed the gene therapy, with Misty one of several individuals who shared their stories. The FDA followed with an approval on Dec. 18, a gene therapy milestone.

"For many of us, this is exactly the type of disease that we hoped that gene therapy would someday treat," Wilson Bryan, director of an FDA office tasked with reviewing Luxturna, said at the time. The next year, Luxturna was also approved in Europe.

It's unclear how many people have received Luxturna since. A Spark spokesperson told BioPharma Dive the company does not disclose that information. In 2019, the company told the Philadelphia Business Journal it had shipped 75 vials of the gene therapy in its first year post-approval. (One vial is used per eye.)

Spark is now owned by the Swiss pharmaceutical company Roche, which does not disclose sales of Luxturna. In February, however, Roche reduced the accounting value of Luxturna, citing "reduced sales expectations."

Luxturna consists of one hundred and fifty billion copies of the corrected RPE65 gene encoded into modified viruses, which are delivered into the eye via about 0.3 milliliters of liquid. Those few drops are injected underneath the retina and, over the course of a week, the viral particles shuttle the functional gene into the patient's eye cells. Once inside, the gene instructs the cells to produce a protein that's otherwise missing, helping restore visual function.

Vials of Luxturna

Spark Therapeutics

"This is not a cure," said Jason Comander, a physician at Massachusetts Eye and Ear in Boston who has administered Luxturna. "It will not make your vision normal," he added, "and there's a small chance that it could hurt your vision." Comander consults with other drugmakers and in 2019 received a nominal amount from Spark.

Luxturna also benefits each patient differently. Comander said the vast majority gain some night vision, while others report improvements in central or side vision. Some see more substantial improvements one of his patients was able to see in up to one thousand times dimmer light than in pre-surgery exams. Many have been able to walk without canes and read without using Braille after surgery.

Their vision isn't perfect, however. Some recipients, Misty included, are still considered legally blind and unable to drive. How long the benefit of gene therapy treatment will last is still unclear, though a recent study co-authored by Maguire and Bennett indicated "improvements were maintained up to 3 to 4 years" after Luxturna.

Comander, who was in his residency while Luxturna was tested, said seeing Maguire administer the therapy affirmed his decision to go into the practice. Now, Comander has done close to a dozen surgeries; his youngest patient was 4 years old at the time of treatment and his oldest was in their 30s. While younger patients saw greater improvements, each patient's eyes functioned better in lower light following treatment.

For Comander, Luxturna was an inspiration, one that he said has helped fuel greater interest in gene therapy. "Many careers have been dedicated to expanding on the success of Luxturna, and it's made a huge difference in the field," he said.

Since Luxturna's clearance, Novartis won FDA approval in May 2019 for a spinal muscular atrophy treatment known as Zolgensma, making it the second gene therapy for an inherited disease available in the U.S. A handful of other gene therapies are in late-stage testing and, behind them, are an expanding pipeline of experimental medicines for a constellation of genetic conditions. In 2020 alone, the FDA received more than 230 applications from cell and gene therapy developers to begin clinical trials, the head of the agency's biologic drugs division said earlier this year.

Gordon "Creed" Pettit was one of the kids who couldn't get into clinical trials for Luxturna. His mother, Sarah St. Pierre-Pettit, brought him from Florida to the University of Iowa a number of times. But he couldn't get through the tests needed to qualify him for treatment.

From there, it was a waiting game until Luxturna's approval. Soon after the FDA's decision, Pierre-Pettit brought Creed to Audina Berrocal at the Bascom Palmer Eye Institute in Miami.

Gordon "Creed" Pettit and Audina Berrocal, the surgeon who administered Luxturna to him.

Photo courtesy of Sarah Pierre-Pettit

Creed was Berrocal's first Luxturna patient. As a pediatric retina specialist, Berrocal said Spark sought her out in the fall of 2017. To date, she's performed a dozen surgeries, all of which have yielded positive results.

"Of all the things I've done in my career, this has been the most amazing and the most rewarding in the sense that we are changing the genetics, the DNA of a person, and we're allowing them to do things that before they couldn't do," Berrocal said. Berrocal consults with other drugmakers and has contributed to published research on Luxturna. In 2018 and 2019, she received nominal payments from Spark.

But treatment, even when positive, can come with adjustments, too. In Creed's case, he was overwhelmed by the sudden change, at first telling his mother he wished he had his old eyes back.

With time, however, Creed has started challenging himself more. "I think most of the gains were at the beginning," Pierre-Pettit said. "Whatever Luxturna did is done. But now that he finally feels confident with himself, he's putting Luxturna to the test now."

For Creed, that means being more social and inquisitive about the world around him. Now 12 years old, he hasn't mentioned wanting his old eyes back for years.

"It's still almost like a new kid every day, like a new baby that sees something new," his mother said.

From a young age, Luke Ward told his mother, Stephanie Joachim, about his dream of playing soccer. But the sport as well as many other daily tasks seemed out of reach.

His vision problems were apparent from birth. While his twin sister could track people with her eyes, Luke stared only at sources of light. When he started walking, he needed to put his hands out to stop himself from running into walls.

Genetic testing revealed Luke had LCA. His doctor said he'd be legally blind by kindergarten. Around the same time, Joachim read an article about Luxturna, but was too late to get Luke enrolled in clinical testing. By the time the FDA approved the therapy, the family had already decided that Luke was getting Luxturna.

Luke Ward with his twin sister, Leia.

Courtesy of Stephanie Joachim

But Joachim was anxious after learning Luxturna's price tag of $425,000 per eye. "I was just flabbergasted and I was like, 'You know what, it's fine. We have the best health insurance,'" she said.

To the family's disappointment, and as other Luxturna patients have experienced, insurance denied the request and cited the therapy's then "newness" as a reason.

At some point in the process, however, Luke's file crossed the desk of an anonymous person who was "so moved from Luke's story and from Luke's pictures, he volunteered to pay for Luke's surgery," Joachim said.

Luxturna's cost was criticized when the therapy was approved and has remained an issue within the patient community since. Shortly after the FDA gave its OK, Spark announced a program with health insurer Harvard Pilgrim and affiliates of Express Scripts, through which the company agreed to pay rebates if the drug doesn't help patients meet certain thresholds.

In a statement to BioPharma Dive, Spark said it offers a "range of patient services and payment models to help navigate and support access" to Luxturna, but did not respond to questions on the number of times rebates have been paid.

Luke Ward

Courtesy of Stephanie Joachim

"Parents shouldn't be paying for this out of pocket," Berrocal, who was also Luke's surgeon, said.

Berrocal told Luke he's the "poster child for Luxturna," Joachim said. He can play sports with his twin sister, including soccer and tee-ball. He started kindergarten this year and has no issues seeing the whiteboard. He still has visual impairments, though, including his peripheral vision. His mother says they keep their shoes tucked out of the way in the house to prevent Luke from tripping.

Four years after its approval, Luxturna continues to be sought out by patients. Joachim says she's received messages from people in Spain, South Africa and the U.K. inquiring about Luke and his progress.

And as Luxturna keeps working, other drugmakers hope to replicate its success. The eye, in particular, is the focus of many gene therapy developers, as it's easy to access and targeting it doesn't carry as many safety risks as other organs. Novartis, which sells Luxturna in Europe, AbbVie, Biogen and Johnson & Johnson are all exploring gene therapies for the eye.

Research into gene editing is advancing as well. In September, Editas Medicine shared preliminary results from the first trial testing a CRISPR gene editing treatment that does its work inside the body. Treatment appeared safe, although the efficacy results were mixed, with several patients experiencing little improvement in vision. The treatment uses CRISPR editing to restore the function of eye cells in people with another form of LCA known as type 10.

Berrocal believes Luxturna represents the beginning of what genetic medicine can offer to patients with many inherited diseases, not only those of the eye.

"20 years from now, we could look back and say, 'Oh my god, that was so rudimentary. Look how much you have advanced,'" she said. "But we have to start somewhere, right? And in 2021, this is what we have, and it's working."

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Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy - BioPharma Dive

AavantiBio has poachedanother bluebird bio leader to bring its lead gene therapy program into clinical trials. Bluebird's vice president of preclinical and translational development is leaving to become chief scientific officer of the one-year-old biotech.

Jenny Marlowe, Ph.D., departs bluebird two months after the companyaskedthe FDA to approve itsgene therapybetibeglogene autotemcel (approvedin the E.U. as Zynteglo)for a severe genetic blood disorder. Marlowe was the leader of that program through2021 and had been at bluebird since 2017 after a nearly nine-year venture at Novartis Institutes for BioMedical Research.

Now, she becomes the latest executive hire at AavantiBio, the Sarepta Therapeutics-backed biotech thatlaunchedlast October with $107 million in financing.

The startup's pipeline is led by a gene transfer therapy for Friedreich's ataxia, a rare genetic disease impacting the spinal cord and nerves, which leads to loss of body movement control.That asset is still preclinical, and three other undisclosed programs are in earlier stages.

RELATED:Bluebird bio gets FDA green light to restart sickle cell gene therapy trials after rocky few months

Another former bluebird VP,Jessie Hanrahan, Ph.D., was hiredby AavantiBio as chief regulatory officer in May.

Marlowe and Hanrahan are two of multiple C-suite additions this year.

Other executive hiresinclude former Sarepta EVP Ty Howton as chief operating officer and general counsel,Paul Herzichas chief technology officer,Jared Simonsas VP of manufacturing,Douglas Swirskyas chief financial officerand ex-Cyclerion Therapeutics Chief Medical OfficerChristopher Wright, M.D., Ph.D., as chief medical officer.

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AavantiBio snags another bluebird bio VP with gene therapy chops as 6th executive hire this year - FierceBiotech

A life-saving gene therapy with a reported list price of 1.79m per dose became available on the National Health Service in March after NHS England struck a confidential pricing deal with Novartis for whats said to be the worlds most expensive drug.

Zolgensma treats spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness, progressive movement loss and paralysis. Severely affected babies have a life expectancy of twoyears.

What makes this therapy so exciting is that its delivered in a single dose. Containing a replica of the missing SMN1 gene, it helps babies to breathe without the help of a ventilator, sit up on their own and crawl. By contrast, many traditional drugs for rare diseases need to be taken permanently, often several times a week. Some have painful side effects. Moreover, they treat only thesymptoms.

More than three-quarters of the approximately 7,000 rare diseases known to science are linked with just one faulty or missing gene, according to the US National Center for Advancing Translational Sciences. The hope is that many such disorders can be cured with one-off treatments.

But the typical costs incurred in producing gene therapies are astronomical. The University of Californias Innovative Genomics Institute estimates that developing one treatment can require an investment of up to $5bn (3.7bn) more than five times the R&D cost of the average conventional drug which puts Zolgensmas price tag into perspective. SMA is thought to affect up to 10,000 babies annually worldwide, but the drug is unlikely to be affordable in many countries outside England, where about 80 babies are born with the disorder eachyear.

Because people like me were under-treated for haemophilia as children, we developed significant physical disabilities. Yet children are still being treated suboptimally today, with the same inevitable badoutcomes

Patients with lipoprotein lipase deficiency, an inherited disorder affecting about one person in a million and causing severe pancreatitis, have already been denied the drug Glybera. In 2012, it became the first gene therapy to be approved in the EU, but was withdrawn in 2017 because it was unprofitable, even with a price tag of $1m.

Cost isnt the only problem.In the high-risk game of pharma roulette, there are many more losers than winners. The 50-year history of gene therapy has been marked by failure and controversy. For instance, of several hundred trials that were started before 2002, not a single one was completed successfully, according to research published in Value in Health, the journal of the US Professional Society for Health Economics andOutcomes.

In 1999, studies in the field were nearly ended in the USafter the death of Jesse Gelsinger, an 18-year-old high-school student in Tuscon, Arizona. He had a rare metabolic disorder called ornithine transcarbamylase deficiency syndrome, which causes ammonia to reach dangerous levels in the body.Having managed his condition on a low-protein diet and nearly 50 pills a day, Gelsinger joined a gene therapy trial. Previous participants had experienced flu-like symptoms after taking the treatment, but he developed an intense inflammatory response that provedfatal.

Gelsingers tragic case illustrates some of the risks of gene therapy. Genes cannot be inserted directly into patients cells, so they are usually delivered using a vector. The most common vectors are viruses with the original bad genes substituted by healthy ones. Such treatments could target the wrong cells and lead to other illnesses, such as cancer; generate infections if the virus recovers its disease-causing abilities; or prompt the patients immune system to overreact in attacking thevirus.

Nonetheless, there is increasing optimism that gene therapy will become part of mainstream care. In October, the first patient to undergo such treatment at Great Ormond Street Hospital for Children (GOSH) celebrated his 21st birthday. Rhys Evans, from Cardiff, was born with severe combined immunodeficiency, a rare condition leaving him vulnerable to even the smallest infection. He was a year old when his parents made the brave decision to write him into medical history. Evans is one of more than 100 young patientsto have received gene therapy at GOSH, a leading international research centre. Without it, many would have died before their secondbirthday.

Paul Gissen, professor of metabolic medicine and head of the genetics and genomic medicine department at GOSH, says: The original research was sponsored by academic grants from bodies such as the Medical Research Council and charities. The success of these has led to an explosion of industry-sponsored trials by companies such as Alexis and Spark Therapeutics.

More than 120 clinical trials testing cell and gene therapy 10% of the global total are ongoing in the UK. Any success stories arising from these will inevitably raise the hopes of the 500,000-plus Britons who are thought to be living with a geneticdisorder.

Laurence Woollard, 32, has haemophilia A, caused by defects to the F8 gene. Haemophilia, which impairs the bloods ability to clot, can cause arthritis and joint damage as well as painful internal bleeding. He has extensive joint damage even though he injects himself up to five times a week with synthetic Factor VIII, the clotting protein encoded byF8.

In England, we have one of the cheapest Factor VIII products in the developed world, says Woollard, who is the founder and director of On the Pulse, a consultancy providing guidance on the management of rare diseases. Because people like me were under-treated for haemophilia as children, we developed significant physical disabilities. Yet children are still being treated suboptimally today, with the same inevitable badoutcomes.

Gene therapy could free Woollard from a lifetime of painful maintenance therapy and even save the NHS money in the long term, despite speculation that the cost of treating haemophilia this way could exceed 1.8m per patient. Research published in 2017 estimated that the combined annual cost of looking after people with severe haemophilia in France, Germany, Italy, Spain and the UK was 1.4bn (1.2bn), equating to an average of just under 200,000 perpatient.

Health economists are proposing alternative payment models for gene therapy. One suggestion is that the health service or insurer should pay annuities to meet the costs, as long as the treatment works, until these have been met infull.

The UK continues to invest heavily in the field. In March, for instance, the Medical Research Council, the LifeArc charity and the Biotechnology and Biological Sciences Research Council announced an 18m programme to create gene therapy innovation hubs at NHS Blood and Transplant in Bristol, the University of Sheffield and Kings CollegeLondon.

But Woollard and others with haemophilia fear that increasing optimism about gene therapy may encourage people to join trials without fully understanding the risks. The current process through which a patient gives informed consent is inadequate, they say, arguing that it should involve an independent adviser, not a one-off discussion with a researcher.

Thats a radical proposal, but not nearly as revolutionary as gene therapy itself, which could transform the treatment not only of many rare conditions, but also of several other diseases, including some cancers. As James Watson, one of the scientists who discovered the molecular structure of DNA, put it: We used to think that our fate was in the stars. But now we know that, in large measure, our fate is in ourgenes.

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The role of gene therapy in curing rare diseases - Raconteur

SEATTLE, Nov. 18, 2021 /PRNewswire/ -- Five patients with mild to moderate dementia received a proprietaryintranasal dual gene therapy composed of hTERT and Klotho. The treatment demonstrated an excellent safety profile with no complications. Efficacy data was also collected and tracked, including cognitive testing, immune system response, physical exams, telomere length, blood analyses, and brain imaging.

All patients saw sustained improvement on the Folstein exam, a test used to measure cognitive impairment. Improved Folstein scoring corresponds to improved memory and cognition.

"This is a remarkable study considering that dementia patients' cognitive function declines predictably every year. Without therapy, a patient's score on the Folstein test declines approximately 3-points-per-year after diagnosis," stated Patrick Sewell, BioViva's Director of Clinical Affairs.

Systemic telomere lengthening was observed in all patients. Longer telomeres have been linked with a reduced risk for a myriad of age-related diseases and all-cause mortality. Telomere lengthening leads to longer replicative cell life and is associated with a younger biological age.

"Telomere shortening and the reduced expression of Klotho directly correlate with the diseases of aging," BioViva CEO Elizabeth Parrish stated, "this study demonstrates the potential to successfully address aging at the cellular level. Despite decades of effort and billions of dollars devoted to dementia research, we have seen very little progress...until now. Anyone who has lost a loved one to dementia understands the importance of this work."

The patent-pending study was carried out by Integrated Health Systems and was sponsored by Maximum Life Foundation, a 501(c)(3) organization; BioViva USA Inc. evaluated the results.

The study's findings were published this month in the peer-reviewed Journal of Regenerative Biology and Medicine in Volume 3; Issue 6 (2021).

About BioViva USA Inc.

BioViva USA Inc. is a company devoted to evaluating and developing gene therapies and new viral vectors to treat aging-associated diseases.

Contact: info@bioviva-science.com

About Integrated Health Systems Ltd.

IHS is an international consortium of medical specialists skilled at creating and delivering cutting-edge and tailored regenerative therapies. Contact info@integrated-health-systems.com

Supporting Video: Human Dementia Study: BioViva Analyzes the Data - YouTube

View original content to download multimedia:https://www.prnewswire.com/news-releases/dementia-patients-receive-dual-gene-therapy-show-cognitive-improvements-301427335.html

SOURCE BioViva

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Dementia Patients Receive Dual Gene Therapy, Show Cognitive Improvements - KHQ Right Now

SK Group's logo at it headquarters in central Seoul. (Yonhap)

SK said it plans to sign a formal deal soon with the Center for Breakthrough Medicines (CBM), a Philadelphia-based contract development manufacturing organization (CDMO), before the end of this year.

Details on the terms of the investment were not available.

With the investment in CBM, SK is taking "a step closer to achieving its goal of establishing a value chain of synthetic and bio pharmaceuticals in the US, Europe and Asia by 2025," Lee Dong-hoon, head of SK Inc.'s bio investment center, said in an online presentation.

When finalized, the deal will come about eight months after SK's takeover of Yposkesi, a French developer and manufacturer of gene and cell therapy products, helping SK further tighten its grip in the burgeoning bio manufacturing markets in America and Europe.

A gene and cell therapy CDMO provides development and manufacturing services for the advanced therapeutic technology. CBM is known for production of plasmid DNA -- a key raw material in the technology -- and viral vectors, which are a tool used to inject the genetic material into cells.

With the investment from SK, CBM will expand its facility, located in the famous gene and cell therapy bio cluster of Cellicon Valley, to a size stretching 65,000 square meters by 2025, according to SK.

SK Inc. has been ramping up investment in the bio business, one of the four core areas it has set eyes on as future growth drivers under a blueprint to grow into a global investment firm with 140 trillion won ($118.7 billion) in market cap by 2025. (Yonhap)

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SK in final talks for investment in US gene therapy firm - The Korea Herald

Intellia Therapeutics (NASDAQ:NTLA) and CRISPR Therapeutics (NASDAQ:CRSP) are pioneers in developing CRISPR gene-editing therapies.In this Motley Fool Live video recorded on Nov. 10, 2021, Motley Fool contributors Keith Speights and Brian Orelli address a viewer's question about a potentially major safety risk that both of these companies face.

Keith Speights: The next question, Brian, Mark asked a question concerning potential technical risks posed by double-stranded breaks made in DNA specifically as related to Intellia Therapeutics, also is CRISPR Therapeutics at risk? What's your take on this?

Brian Orelli: I guess this is a question about whether CRISPR, the technique, makes a break at the site of where it's supposed to, based on the sequence that you put into the CRISPR. Then it makes a break and then it gets put back together and that creates a mutation.

Usually, CRISPR is most useful for knocking out a gene. If you make a mutation and then now the gene longer works. Like for instance, there are CRISPR Therapeutics drug for beta-thalassemia and sickle cell disease, knocks out the gene that represses fetal hemoglobin. By knocking that out, then you express fetal hemoglobin and then that helps the patients since they are lacking adult hemoglobin.

The issue, I think, is probably not necessarily at that site, but it can also potentially make mutations at other sites. I think that's a risk of CRISPR, and they have to go through and make sure that the number of additional mutations that they're making is minimal.

The gene therapy companies have the same issues on the DNA that you put in for gene therapy to express a protein, can potentially integrate into some places. That can cause potential to cause cancers and stuff like that. I think it's definitely a risk, and the fact that we've only had a few patients, probably makes it harder.

Keith Speights: Yeah. To my knowledge, Brian, so far, neither Intellia nor CRISPR Therapeutics have reported significant issues with this. I'm not aware of any anyway.

Brian Orelli: Right, but they've only added a few patients, too, so we don't know.

Keith Speights: Right, it's still early.

Brian Orelli: I think it's a little hard to know exactly what the rate might be until you get into the hundreds of patients or even more if it's a low likelihood.

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A lengthy video posted on TikTok that makes a host of unfounded claims about COVID-19 vaccines all of which have been repeatedly debunked originated with InfoWars, a website renowned for sowing conspiracy theories.

The viral video claims that COVID-19 vaccines "failed miserably" in animal trials and are "a type of gene therapy that several top scientists warn will kill you." It includes hashtags like #truthcomesout and #firefauci.

The video appeared on Facebook, where it was flagged as part of Facebooks efforts to combat false news and misinformation on its News Feed. (Read more about our partnership with Facebook.)

COVID-19 vaccines did not fail in animal trials or result in the death of the animals tested. The vaccines are not a form of gene therapy, which modifies a persons genes to replace or fix mutations that lead to diseases. The scientists cited in the video have spread misinformation about the vaccines.

The footage is an excerpt from a longer video posted online Oct. 13 called "Kill Shot," by Greg Reese, an editor and producer for the website InfoWars, which has spread other vaccine misinformation. Most recently, InfoWars was in the news because its founder and host, Alex Jones, was found liable for defamation against the families of victims from the 2012 Sandy Hook Elementary School shooting, which Jones has portrayed as a hoax.

In the TikTok video, a narrator says that 22 months into the COVID-19 pandemic, "We have all the information needed to paint a clear picture of whats going on." The narrator then recites a laundry list of false claims about Dr. Anthony Fauci, PCR tests, the ingredients of vaccines and more.

The narrator says, "The COVID vaccines are not vaccines but rather highly controversial mRNA tech that failed miserably on its animal trials, a type of gene therapy that several top scientists warn will kill you," a statement that contains multiple falsehoods.

First, COVID-19 vaccines did not fail animal trials. Fact checkers have debunked this claim, noting that the two vaccines most widely used in the U.S. Pfizer and Moderna produced desirable outcomes in animal testing. Results from Modernas animal testing were published in the New England Journal of Medicine after monkeys had a robust immune response to the vaccine.

Animals also did not die during the vaccine trials. Full Fact reported that had any animals died, human trials that were running concurrently would have been halted, which they were not.

Next, COVID-19 vaccines are not a type of gene therapy; PolitiFact and others have reported that the claim is false. Gene therapy is a process of modifying genes to replace or fix mutations that lead to diseases, according to PolitiFact.

Thats different from mRNA vaccines, which send instructions to the bodys cells to make a piece of spike protein, which is also found on the surface of the virus that causes COVID-19, so that the immune system can respond to it.

Finally, the videos narrator says "several top scientists warn" that the COVID-19 vaccines "will kill you," and names Dr. Ryan Cole and Dr. Nathan Thompson. Cole, who is licensed to practice medicine in several states and is under investigation by the Washington Medical Commission, falsely claimed in the spring that mRNA vaccines cause cancer and autoimmune diseases. He was rebuked by the author of the medical paper he cited as evidence for the claim.

The other doctor identified, Thompson, has claimed that COVID-19 vaccines weaken the immune system, which PolitiFact rated False.

Our ruling

A TikTok video posted on Facebook says COVID-19 vaccines "failed miserably" in animal trials and are "a type of gene therapy that several top scientists warn will kill you." The video originated on InfoWars, known for spreading conspiracy theories.

The vaccines did not fail in animal trials or result in the death of the animals tested.

COVID-19 vaccines are not a type of gene therapy, which involves modifying genes to replace or fix mutations that lead to diseases. The mRNA vaccines do not change a persons genetic makeup and never enter the part of the cell that hosts DNA.

The scientists cited in the video have spread misinformation about the vaccines.

We rate this claim False.

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A video that originated on InfoWars is filled with falsehoods about COVID-19 vaccines - PolitiFact

NEW YORK and CLEVELAND, Nov. 17, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that information about the companys ABO-102 pivotal Transpher A study in Sanfilippo syndrome type A (MPS IIIA) will be presented at the 14th International Congress of Inborn Errors of Metabolism (ICIEM). The meeting will take place virtually and at the Hilton Sydney in Sydney, Australia on November 21-23, 2021.

The oral presentation titled, Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA), will be presented by Dr. Nicholas Smith, Department of Neurology and Clinical Neurophysiology, Womens and Childrens Hospital. The presentation includes new data characterizing the positive correlational relationship between the Bayley Scales of Infant and Toddler Development (BSITD) and the Mullen Scales of Early Learning (MSEL), standardized measures of non-verbal cognitive functioning for young children, and previously announced efficacy and safety data on Abeonas AAV-based gene therapy, ABO-102. The presentation will take place during a session on Tuesday, November 23, 2021 from 11:45 am-1:15 pm Australian Eastern Daylight Time (AEDT).

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include: EB-101, its investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development; novel investigational AAV-based gene therapies ABO-102 in the pivotal Transpher A study for Sanfilippo syndrome type A (MPS IIIA) and ABO-101 in the Phase 1/2 Transpher B study for Sanfilippo syndrome type B (MPS IIIB). The Companys development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeonas novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeonas fully integrated gene and cell therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and planned commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

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Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Companys most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

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Abeona Therapeutics Announces Presentation on Pivotal Transpher A Study of ABO-102 in MPS IIIA at the 14th ICIEM Conference - Yahoo Finance