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Archive for the ‘Gene therapy’ Category

Scientists want human trials for gene therapy that could help battle addiction – Digital Trends

Sunday, January 12th, 2020

In recent years, new gene editing tools have been used for everything from genetic modification of plants to increase crop yields to, far more controversially, genetic tampering with human embryos. Could a form of gene therapy also be useful in helping treat cocaine addiction, a form of addiction that proves highly resistant to alternative approaches, such as conventional medical treatment and psychotherapy? Thats what researchers from the world-famous Mayo Clinic are hoping to prove.

They are seeking approval for the first-in-human studies of an innovative new single-dose gene therapy. Their approach involves the delivery of a gene coding for an enzyme, called AAV8-hCocH, which metabolizes cocaine in the body into harmless byproducts. In order to progress to this next step in their work, they first have to gain permission from the U.S. Food and Drug Administration (FDA) in the form of an Investigational New Drug Application.

The researchers have already demonstrated the safety of their approach in mice. In a prior experiment, they showed a complete lack of adverse effects in mice which had both been previously exposed to cocaine and those which had not.

Mice given one injection of AAV8-hCocH and regular daily injections of cocaine had far less tissue pathology than cocaine-injected mice with no vector treatment, the researchers wrote in the abstract for their paper describing the work. Biodistribution analysis showed the vector located almost exclusively in the liver. These results indicate that a liver-directed AAV8-hCocH gene transfer at reasonable dosage is safe, well-tolerated, and effective. Thus, gene transfer therapy emerges as a radically new approach to treat compulsive cocaine abuse.

This is not the first time similar work has been carried out. In February 2017, scientists at the University of British Columbia genetically engineered a mouse so as to be incapable of becoming addicted to cocaine. However, one of the researchers on the project told Digital Trends that transferring this work across to humans for possible treatment for addiction was not straightforward. Instead, that work was more focused on exploring the link between drug use and genetics and biochemistry.

Theres still a whole lot more research that needs to be done in this area. Even if the FDA grants the Mayo Clinic researchers permission for their human trials, well most likely be waiting a few years at least before this treatment could be rolled out to the general public. Its an exciting leap forward, nonetheless.

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At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease – The New York Times

Sunday, January 12th, 2020

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared. But doctors had given her blood transfusions while she regrew her own red blood cells, so it was not clear if the absence of symptoms was because of the gene therapy or the transfusions.

As she recovered, Helen returned to her passion: dancing. One day, she came back from her school dance group and told her mother, My legs hurt. It feels funny. Ms. Cintron smiled. Thats soreness, she explained. Helen laughed. She had only known pain from sickle cell.

Helen was scheduled for her six-month checkup on Dec. 16. By then, all the transfused cells were gone, leaving only blood made by stem cells in her own marrow. The doctors would finally tell her whether the therapy was working.

The day before, she and her parents visited the New England Aquarium in Boston. She was able to stay outside on a cold, blustery day, watching one seal bully the others, barking and fighting. When Helen mentioned that her hands were cold, Ms. Cintrons stomach clenched in fear. But it was just a normal thing to feel on a winter day.

The next morning, Dr. Esrick delivered the news. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved even with blood transfusions. She had no signs of sickle cell disease.

Now you are like me, her father told her. I jump in the pool, I run. Now you can do it, too!

Her family, accustomed to constant vigilance, is only now getting used to normal life.

On Dec. 23, Helen and her mother flew to the familys new home in Arizona.

Helen recently described her transformed outlook on Facebook.

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Generation Bio grabs a $110M round to ramp up work on next-gen gene therapies – FierceBiotech

Sunday, January 12th, 2020

In 2018, Generation Bio broke cover with a $25 million series A, swiftly followed by a meatier $100 million second funding round.

Now, just before the J.P. Morgan Healthcare Conference, it has grabbed its biggest yet, a $110 million series C, as it looks to go all in for IND-enabling studies for its leading programs: liver-targeted therapies for hemophilia A and phenylketonuria.

In addition to the liver, Generation Bio is also working on potential treatments for diseases of skeletal muscle and the eye.

The Art of Recognizing Clinical Supply Risk Factors and Applying Proactive Measures to Avoid Study Delays and Disruptions

No two studies are the same and each clinical supply project carries unique risks. But what characteristics are most likely to raise a flag that issues are ahead? Are there certain types of clinical sponsors and studies that are at greater risk of experiencing supply challenges? And how do clinical sponsors know what is important to focus on and what is not? Join us for this webinar as we attempt to answer these questions.

The early-stage Cambridge, Massachusetts-based biotech saw its major round led by T. Rowe Price with help from Farallon, Wellington Management and existing investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital and an entity associated with SVB Leerink.

Generation Bios platform is geared up to be gene therapy 2.0 and is designed to develop re-dosable, long-lasting, scalable gene therapies for severe diseases.

The company is developing gene therapies under the GeneWave banner that use closed-ended DNA rather than viruses to deliver therapeutic proteins, which could sidestep safety issues such as immune reactions

Our vision is to develop re-dosable, long-lasting gene therapies manufactured at a scale that leaves no patient or family behind, said Geoff McDonough, M.D., president and CEO of Generation Bio.

Since our founding we have had the support of high-quality investors who share our excitement about the potential of our platform to lead a new generation of gene therapy and about advancing our lead programs toward the clinic.

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Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track – Xconomy

Sunday, January 12th, 2020

XconomyBoston

Solid Biosciences is slashing its workforce, including two top executives, in order to devote the companys remaining resources to its experimental gene therapy for Duchenne muscular dystrophy.

The corporate restructuring announced Thursday comes two months after the FDA placed a hold on the study after safety problems emerged that were linked to the gene therapy, SGT-001. Cambridge, MA-based Solid Bio (NASDAQ: SLDB) says going forward it will focus on how to address the clinical hold and resume testing. With the corporate changes, Solid Bio says it has enough cash to last into next year. At the end of the third quarter of 2019, the company reported cash and other holdings totaling $105.7 million.

Following the announcement, Solid Bios stock price slid more than 17 percent to $3.66 per share in pre-market trading.

Solid Bio has been developing SGT-001 as a way to potentially address the genetic defect underpinning Duchenne. Patients who have the inherited disease dont make enough of the muscle protein dystrophin. The Solid Bio gene therapy uses an engineered virus to deliver genetic material intended to restore dystrophin production. But the company had also previously disclosed theres a chance that the dosing requirements of the gene therapy could increase the risk of side effects related to the virus used in the treatment.

The complications reported in the November clinical hold included an immune system reaction, a decrease in red blood cells, kidney injury, and blood circulation difficulties. Those problems are similar to ones cited in the FDAs 2018 clinical hold on tests of SGT-001. Months later, the agency allowed the study to resume but with additional safety measures.

Solid Bios board approved the corporate restructuring on Tuesday, according to a securities filing. In the first quarter of this year, the company expects to record a $2.1 million charge related to the layoffs, which will cut about one third of its workforce. Last years annual report states that the company had 111 full-time employees as of Dec. 31, 2018. Those leaving Solid Bio include Alvaro Amorrortu, the companys chief operating officer, and Jorge Quiroz, its chief medical officer. But both will continue to advise Solid Bio under consulting agreements.

Photo by Flickr user reynermedia via a Creative Commons license

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan [[at]] xconomy.com.

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At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens – Boston.com

Sunday, January 12th, 2020

BOSTON Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest.

The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.

Helen, who at 16 was the youngest person ever to undergo the therapy, was sound asleep for the big moment.

It was a critical moment in medical science.

For more than a half-century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the diseases horrifying hallmark: episodes of agony that begin in babyhood.

Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts said the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African American, often poor or of modest means.

The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.

This is the story of two quests for a sickle cell cure one by the Obando family and one by a determined scientist at Boston Childrens Hospital, Dr. Stuart Orkin, 73, who has labored against the disease since he was a medical resident in the 1970s.

Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee Obando. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood.

Scientists have been experimenting with gene therapy for two decades, with mixed success. And it will be years before they know if this new procedure is effective in the long term. But if it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA.

Four weeks after the infusion of stem cells, Helen was strong enough to be discharged. At home, in Lawrence, Massachusetts, on a sofa with her mother by her side, she put a hand over her eyes and started to sob. She and her family wondered: Would it work? Was her suffering really over?

A Familys Nightmare

Sheila Cintron, 35, and Byron Obando, 40, met when she was in the eighth grade and he was a high school senior. They fell in love. Haylee, their first child, was born in 2001, when Cintron was 17.

When a newborn screening test showed that Haylee had the disease, her father asked, Whats sickle cell?

They soon found out.

As the family gathered for her first birthday party, Haylee started screaming inconsolably. They rushed her to the hospital. It was the first of many pain crises.

Doctors warned the parents that if they had another baby, the odds were 1 in 4 that the child would have sickle cell, too. But they decided to take the chance.

Less than two years later, Helen was born. As bad as Haylees disease was, Helens was much worse. When she was 9 months old, a severe blockage of blood flow in her pelvis destroyed bone. At age 2, her spleen, which helps fight bacterial infections, became dangerously enlarged because of blocked blood flow. Doctors surgically removed the organ.

After Helen was born, her parents decided not to have any more children. But four years later, Cintron discovered she was pregnant again.

But they were lucky. Their third child, Ryan Obando, did not inherit the sickle cell mutation.

As Ryan grew up, Helens health worsened. When he was 9, Helens doctors suggested a drastic solution: If Ryan was a match for her, he might be able to cure her by giving her some of his bone marrow, though there would also be major risks for her, including death from severe infections or serious damage to organs if his immune system attacked her body.

As it turned out, Ryan matched not Helen but Haylee.

The transplant succeeded, but her parents asked themselves how they could stand by while one daughter was cured and the sicker one continued to suffer.

There was only one way to get a sibling donor for Helen: have another baby. In 2017, the couple embarked on another grueling medical journey.

Obando had a vasectomy, so doctors had to surgically extract his sperm from his testicles. Cintron had 75 eggs removed from her ovaries and fertilized with her husbands sperm. The result was more than 30 embryos.

Not a single embryo was both free of the sickle cell gene and a match for Helen.

So the family decided to move to Mesa, Arizona, from Lawrence, where the cold, which set off pain crises, kept Helen indoors all winter. The family had already sold their house when they heard that doctors at Boston Childrens were working on sickle cell gene therapy.

Cintron approached Dr. Erica Esrick, a principal investigator for the trial. But the trial wasnt yet open to children.

Figuring Out the Science

Nothing had prepared Orkin for the suffering he witnessed in his 30s as a medical resident in the pediatric hematology ward at Boston Childrens. It was the 1970s, and the beds were filled with children who had sickle cell crying in pain.

Orkin knew there was a solution to the puzzle of sickle cell, at least in theory: Fetuses make hemoglobin the oxygen-carrying molecules in blood cells with a different gene. Blood cells filled with fetal hemoglobin do not sickle. But the fetal gene is turned off after a baby is born, and an adult hemoglobin gene takes over. If the adult gene is mutated, red cells sickle.

Researchers had to figure out how to switch hemoglobin production to the fetal form. No one knew how to do that.

Orkin needed ideas. Supported by the National Institutes of Health and Howard Hughes Medical Institute, he kept looking.

The breakthrough came in 2008. The cost of gene sequencing was plummeting, and scientists were finding millions of genetic signposts on human DNA, allowing them to home in on small genetic differences among individuals. Researchers started doing large-scale DNA scans of populations, looking for tiny but significant changes in genes. They asked: Was there a molecular switch that flipped cells from making fetal to adult hemoglobin? And if there was, could the switch be flipped back?

They found a promising lead: an unprepossessing gene called BCL11A.

In a lab experiment, researchers blocked this gene and discovered that the blood cells in petri dishes started making fetal instead of adult hemoglobin.

Next they tried blocking the gene in mice genetically engineered to have human hemoglobin and sickle cell disease. Again, it worked.

Patients came next, in the gene therapy trial at Boston Childrens that began in 2018.

The trial run by Dr. David Williams, an expert in the biology of blood-forming stem cells at Boston Childrens, and Esrick has a straightforward goal: Were going to reeducate the blood cells and make them think they are still in the fetus, Williams said.

Doctors gave adult patients a drug that loosened stem cells immature cells that can turn into red blood cells from the bone marrow, their normal home, so they floated free in the bloodstream. Then they extracted those stem cells from whole blood drawn from the patient.

The researchers used a disabled genetically engineered AIDS virus to carry information into the stem cells, flipping on the fetal hemoglobin gene and turning off the adult gene. Then they infused the treated stem cells into patients veins. From there, the treated cells migrated into the patients bone marrow, where they began making healthy blood cells.

With the success in adults, the Food and Drug Administration said Boston Childrens could move on to teenagers.

When her mother told her about the gene therapy trial, Helen was frightened. But the more she thought about it, the more she was ready to take the risk.

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared.

Helen was scheduled for her six-month checkup Dec. 16. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved, even with blood transfusions. She had no signs of sickle cell disease.

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Novartis opens facility for innovative cell and gene therapies in Switzerland – Science Business

Sunday, January 12th, 2020

In the presence of Federal Councillor Alain Berset and other distinguished guests, Novartis inaugurated a new manufacturing facility for cell and gene therapies at Stein, Switzerland on November 28th.

Our site in Stein is vital for new launches of solid and liquid drugs, said Steffen Lang, Global Head of Novartis Technical Operations and member of the Novartis Executive Committee. "The construction of the new manufacturing facility is another investment in the production of breakthrough cell-based therapies that can potentially change the lives of patients.

In addition to manufacturing areas for novel CAR-T cell therapies, the new building also hosts the production of innovative, difficult-to-manufacture solid dosage forms such as tablets and capsules. In September 2019, the first clinical production of a cell and gene therapy batchwas successfully completed.

Unlike conventional drug production, cell and gene therapy asks for the manufacture of a personal dose for each patient. For this purpose, patients who have already undergone various therapies have a small amount of their own blood cells taken, which are then sent to Stein. "Here we enrich part of the white blood cells, the T cells, and genetically modify them so that they can recognize and fight the cancer cells in the patient's blood," says Dorothea Ledergerber, project manager of the Stein plant for cell and gene therapies. The altered cells are then sent back to hospital and administered to the patient by infusion. Novartis is doing pioneering work here: "We have the unique opportunity to offer patients for whom there have been no other therapeutic options a totally new perspective by using these novel CAR-T cell therapies," says Dorothea Ledergerber.

Read more in German

This release wasfirst publishedby Novartis.

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Global Gene Therapy Market is Expected to Reach US$ 5,609.9 Million by 2027, Growing at an Estimated CAGR of 8.2% Over the Forecast Period as…

Sunday, January 12th, 2020

PUNE, India, Jan. 8, 2020 /PRNewswire/ -- In terms of revenue, global gene therapy market was valued at US$ 919.6 million in 2018 and is anticipated to reach US$ 5,609.9 million by 2027, growing at a CAGR of 8.2% over the forecast period. Market participants are adopting partnerships or acquisition as their strategy to strengthen their foothold. For instance, Pfizer Inc. acquired Medivation, Inc. and Bamboo Therapeutics, Inc. to develop a focused product for the treatment of patients with rare diseases related to neuromuscular and central nervous system. Companies are building relationships with community and patients to understand the disease and design therapies accordingly.

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Lethal diseases like cancer can be treated using gene therapy by inserting the antisense strands to revert the effect of the oncogenes using bio engineered vectors. Recently, scientists developed bionic chip to transfer DNA to cells using electroporation technique. During the forecast period, nanoparticles will play an important role in gene delivery systems to increase the efficiency of transfection of the non-viral carriers, thereby, fuelling the gene therapy market.

Due to drastic shift in treatment patterns, gene therapy treatment is considered one of the reliable cures for lethal diseases. The vectors or the DNA carriers are safer and have improved in terms of carrying genes without rejection which help the companies to attract venture capitalists to invest more in gene therapy market. Most of the research companies are focusing on development of gene carriers for the successful gene delivery. One of the prominent used vectors among the gene vehicle family is adeno associated virus. Cancer and Sensory disorders are the major area of concern that need to be fixed and hence drug development related to these disease is driving the gene therapy market.

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The detailed research study provides qualitative and quantitative analysis of gene therapy market. The market has been analyzed from demand as well as supply side. The demand side analysis covers market revenue across regions and further across all the major countries. The supply side analysis covers the major market players and their regional and global presence and strategies. The geographical analysis done emphasizes on each of the major countries across North America, Europe, Asia Pacific, Middle East & Africa and Latin America.

Key Findings of the Report:

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Gene Therapy Market

By Geography

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Putting gene therapy in reach – University of California

Sunday, January 12th, 2020

Cellular engineering promises new treatments for cancer and other maladies. But most manufacturing processes propel the cost of these so-called living drugs into the stratosphere, far beyond reach of most people who need them.

A technology patented at the University of California, Riverside, and recently licensed to startup Basilard BioTech could bring these prices back down to earth.

The technology, developed by Masa Rao, an associate professor of mechanical engineering in the Marlon and Rosemary Bourns College of Engineering, minimizes damage to the cell in the manufacturing process. This enables both high gene delivery efficiency and cellular viability, a feat that most other approaches cannot match.

Basilard spun out of Raos laboratory earlier this year. The company has obtained an exclusive license to commercialize the technology, which they have branded SoloPore. Basilard is seeking to develop it as a disruptive new platform for engineering ex vivo cell and gene therapies for cancer specifically, as well as genetic disorders and degenerative diseases more broadly.

Basilards SoloPore technology is a differentiated solution that provides greater scalability, safety, efficiency, and versatility than prevailing gene delivery methods, said Basilard CEO Brynley Lee. This will allow us to reduce manufacturing cost, and therefore, bring these revolutionary therapies to more of those in need.

Basilard is raising seed capital and working to build a commercial prototype. The young company is the first biotech instrumentation company to emerge from UC Riversides EPIC entrepreneurship incubator, which guides innovators through the commercialization and entrepreneurial process and helps connect them with investors.

Within the span of less than a year, weve gone from a purely academic effort to the formation of a startup thats on the cusp securing its first venture capital funding, Rao said. UC Riversides Office of Technology Partnerships has been instrumental in this rapid ascent.

Weve worked hard for the past three years to accelerate technology translation and commercialization with entrepreneurial programs that have mentored more than 220 entrepreneurs and 120 startups in the Inland Empire since October 2016, said Rosibel Ochoa, associate vice chancellor for technology partnerships. Basilards quick rise is a sign that we are building a healthy entrepreneurial ecosystem that supports the growth of startups in our region.

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Local infant receives $2.1 million gene therapy infusion after initial Medicaid rejection – WTHR

Sunday, January 12th, 2020

INDIANAPOLIS (WTHR) Four-month-old Anthony Schmitz has spent his entire life on a ventilator in intensive care at Riley Hospital for Children. But Wednesday he received a gene therapy infusion that might save and change his life.

Zolgensma is a prescription gene therapy that costs $2.1 million for the one-time dose.

The drug has proven effective in treating children with spinal muscular atrophy (SMA) under the age of two.

Indiana Medicaid first rejected the treatment for Schmitz because he was on a ventilator but gave approval on appeal.

"Early diagnosis is key and don't give up, said Louise Johnson, Schmitzs mother. It's not a death sentence, so just keep fighting. It's a baby. Keep fighting."

"I think this was really a group decision that said, 'Yeah, medically this made sense for this child. So, the cost kind of fell by the wayside, said Dr. Larry Walsh, Riley Children's Health Pediatric Neurologist.

Zolgensma replaces the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene.

Without treatment, Anthony's life expectancy was about two years.

"No mom wants to bury their child, said Johnson, who is from Evansville. So, I just want to see him grow up with his brothers."

Schmitz received the treatment Wednesday morning.

The infusion took just over an hour. But it will be weeks, if not months, before doctors know if the medicine is working for him.

"Even if we can make some smaller difference where we do help his respiratory function, where he doesn't need to be on a ventilator - things like that - that would be a tremendous win I think for he and his family, said Dr. Walsh.

"The future is unknown, so I'm still nervous, said Johnson. But I'm more excited. I can't wait."

Indiana adopted newborn screening for SMA in 2018.

Schmitz is now part of a handful of babies to receive gene therapy infusion at Riley for the rare, progressive genetic disease.

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‘I decided to fight like a mother’: How one parent is battling to cure a disease so rare it has no name – CNBC

Sunday, January 12th, 2020

The only thing Amber Freed ever wanted was to be a mom.

Like a lot of people, she and her husband Mark had a hard time conceiving. But after two years of IVF treatments, the Denver couple got a double dose of good news: Amber was pregnant with twins.

Maxwell and Riley were born on March 27, 2017.

"They instantly changed my life and made me so happy," Amber said.

But while the twins came into the world together, they didn't develop at the same pace as they grew. When they were about four months old, Amber and Mark noticed the difference: Maxwell wasn't reaching for toys or his bottle like his sister did he didn't use his hands at all.

After six months of genetic testing, Maxwell was diagnosed with a disease so rare it doesn't even have a name. Instead, it's known by its genetic location: SLC6A1. At the time of Maxwell's diagnosis, there were only 50 known cases in the world.

"I just remember thinking that that wasn't the name of a disease. It was the name of a flight number," said Amber. "I could not understand what my perfect, beautiful little baby boy had, and neither could the doctors."

What they did know was that Maxwell's rare neurological condition would likely cause severe movement and speech disorders and intellectual disability. Between the ages of three and four, Maxwell is expected to develop a debilitating form of epilepsy and start to regress.

Mark and Amber Freed with their twins Riley and Maxwell

Amber Freed

Amber refused to just sit back and watch that happen. She quit her job as a financial analyst at Janus Henderson the day Maxwell was diagnosed, and dedicated herself to finding a cure.

"It was in that moment that there was no future for my most prized possession in the world, that I was not going to accept that answer for little Maxwell," she said. "And I decided to fight like a mother."

She asked the doctors what they would do if Maxwell were their child. They told her to "call scientists."

Working 80 hours a day, Amber became an expert in the biology of the disease and reached out to 140 scientists over the next three months. She founded a non-profit and in 10 months, between that and a GoFundMe campaign, has raised $1 million to fund the initial research into a cure.

Amber was told gene replacement therapy was Maxwell's best hope.

The Food and Drug Administration has already approved gene therapy for some other diseases, including a rare form of vision loss and for some leukemia patients. It involves introducing a new gene through a virus that doesn't make the patient sick. It targets the defective gene, replacing it with a good copy, altering the patient's DNA and - it's hoped- dramatically improving the disease with a single treatment.

At some point, Amber decided Dr. Steven Gray at the University of Texas Southwestern Medical Center in Dallas was the best person to help her son. But Gray was busy and hard to pin down. So Amber showed up at a conference where she knew he'd be speaking, and sat down next to him. After a four-hour dinner that night, they had a game plan.

Gray's team has advanced their research on SLC6A1 to the point where they're ready to start clinical trials.

But a phase one trial requires money. A lot of money. Amber needs another $3 million to-$6 million. And connections in the drug industry.

So she's joining the thousands of health industry investors and executives flying to San Francisco for the JPMorgan Healthcare Conference. You'll never find a place with a denser concentration of the people who fund drug development. She's hoping for donations or maybe to find a biotech company that would want to invest as a business opportunity.

But the Freed family is racing against the clock. Amber and Mark's little boy, who they call "Mr. Snuggles" because he loves hugging his sister and giving open mouth kisses, could start having debilitating seizures within the next year.

And even if she can get a clinical trial started, there's never a guarantee any patient, including Maxwell, will be admitted.

"The University of Texas Southwestern was very straightforward upfront that you may not be doing this for Maxwell," Amber explained. "There's a chance this may not be done in time for him, that you're doing it for every child that comes after him. And I lived with that fear and uncertainty for a very long time. And I understand and the way I make peace with it is thinking that there's no greater legacy in the world and doing the best you can to really impact a multitude of little lives."

She says her dream is that SLC6A1 will someday be part of a newborn screening panel, and that babies with the defect will be able to be treated and cured before they ever leave the hospital.

"They will never become symptomatic of this disease," she hopes. "There will never be another Maxwell Freed."

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'I decided to fight like a mother': How one parent is battling to cure a disease so rare it has no name - CNBC

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Type 2 Gaucher Trial of PR001 Gene Therapy Has Hold Lifted by FDA – Gaucher Disease News

Sunday, January 12th, 2020

The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on a Phase 1/2 trial designed to test the gene therapy candidate PR001 in patients with type 2 Gaucher disease.

The team atPrevail Therapeutics expects to initiate patient dosing in the first half of 2020.

Prevail was awaiting a decision by the FDA to test higher doses of PR001 than initially planned. This request was supported by preclinical evidence of greater efficacy with no safety issues at such dosages. The investigational new drug (IND) application of PR001, an essential step to opening a clinical study, had first been accepted in June 2019.

PR001 uses a modified, harmless version of an adeno-associated virus (AAV9) to deliver a fully working version of the GBA1 gene to nerve cells. Mutations in this gene cause Gaucher disease by producing a defective enzyme called beta-glucocerebrosidase, which leads to the accumulation of fatty molecules inside cells.

In type 2 Gaucher disease, called acute infantile neuronopathic Gaucher disease, these toxic fatty molecules build up in the patients brain from early infancy, resulting in neurological symptoms.

By restoring production of normal beta-glucocerebrosidase in affected brain cells, a single dose of PR001 is intended to ease Gaucher symptoms and modify disease course.

Work in mice and monkeys showed that PR001 now being developed in collaboration with Lonza Pharma & Biotech is well-tolerated, leads to the production of a functional enzyme in nerve cells, reduces the accumulation of fatty molecules, and improves motor function.

We are pleased to now have an active IND for PR001 for the nGD [neuronopathic Gaucher disease] indication and look forward to initiating a Phase 1/2 clinical trial in the first half of 2020, Asa Abeliovich, MD, PhD, Prevails founder and CEO, said in a press release.

Patients with nGD have the most severe form of Gaucher disease and a significant unmet need for therapies to treat their neurological manifestations. We believe PR001 has tremendous potential, he added.

In addition, the company plans to initiate another Phase 1/2 study in people with type 3 Gaucher later this year. Patients with this type also experience neurological symptoms, but they are milder and progress slower than those seen in patients with type 2 Gaucher.

Prevail is also developing PR001 for GBA1 mutation-related Parkinsons disease. Mutations in the GBA1 gene are one of the most common genetic risk factors for Parkinsons. A Phase 1/2 clinical trial (NCT04127578), called PROPEL, is currently recruiting participants with Parkinsons to test PR001 administered directly into the cerebrospinal fluid (the liquid surrounding the brain and spinal cord).

With over three years of experience in the medical communications business, Catarina holds a BSc. in Biomedical Sciences and a MSc. in Neurosciences. Apart from writing, she has been involved in patient-oriented translational and clinical research.

Total Posts: 24

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

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Type 2 Gaucher Trial of PR001 Gene Therapy Has Hold Lifted by FDA - Gaucher Disease News

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PACT Pharma Raises $75M in Oversubscribed Series C Financing to Develop Fully Personalized NeoTCR-T Cell Therapies for Patients with Cancer | DNA RNA…

Sunday, January 12th, 2020

DetailsCategory: DNA RNA and CellsPublished on Sunday, 12 January 2020 11:53Hits: 206

- The round was led by Vida Ventures, a next generation life science venture firm with industry-leading experience in cell and gene therapy

- The financing will be used to expand clinical & manufacturing development to deliver clinical data for patients with multiple solid tumor types

SOUTH SAN FRANCISCO, CA, USA I January 10, 2020 I PACT Pharma, in pursuit of its vision to eradicate solid tumors using transformational, first-in-class fully personalized NeoTCR-T cell therapies, today announced that it has closed an oversubscribed $75 million Series C financing. This round, led by Vida Ventures, a next generation life science venture firm with industry-leading experience in the cell and gene therapy, also included current investors of PACT.

Combined with proceeds from previous financings, PACT will use the Series C proceeds to expand the scope of its clinical plan to investigate NeoTCR-T cell products targeting multiple neoantigens for a spectrum of solid tumor types. In addition to clinical expansion, PACT will open in 2020 a next-gen GMP manufacturing facility in South San Francisco to support the end-to-end production and supply chain for the engineering of personalized neoantigen-targeted autologous T cells. Under the direction of industry veteran Tim Moore, President and Chief Technology Officer, PACT will leverage the new in-house manufacturing facility to automate manufacturing and analytic processes to reduce cycle time and manufacturing costs.

"PACT has grown from company launch to opening its first-in-kind clinical trial in two years. Our progress has been exhilarating and the support from our existing investors has made that progress possible," said Alex Franzusoff, PhD, Chief Executive Officer of PACT Pharma. "As we look to the next stage of our development and expansion of our clinical programs, we are excited to have interest from a new group of prominent investors who both understand the potential of NeoTCR-T cell therapy and have direct experience in the space. Vida Ventures stood out as a partner of choice, given their depth of operational experience in research, clinical development and manufacturing in cell therapy as well as their proven ability to guide companies like Kite and Allogene across key stages of development.

As part of the Series C financing, Helen S. Kim, Managing Director at Vida Ventures, will join the Company's Board of Directors. Ms. Kim brings over 25 years of biotechnology leadership experience and serves on the boards of Assembly Biosciences, Applied Molecular Transport, A2 Biotherapeutics and Exicure, Inc.

"Our investment in PACT Pharma represents our goal to fund scientific advances by embracing cutting edge innovation with the potential to make a meaningful difference in the lives of patients," said Kim. "PACT has developed a pioneering platform of personalized designer T cells with the potential to target some of the most elusive solid cancers facing society today."

ABOUT PACT Pharma

PACT Pharma is an independent, privately funded clinical stage company, based inSouth San Francisco, California, developing transformational personalized neoTCR-T cell therapies for the eradication of solid tumors and is now enrolling patients in its first-in-human Phase 1 clinical studies at several key academic centers of the CIRM-funded Alpha Clinic network, inCalifornia.

PACT Pharma's distinguised co-founders,David Baltimore(Nobel Laureate),Antoni Ribas,Jim Heath,Terry RosenandJuan Jaen launched the company in early 2017. The company is backed by GV (formerly Google Ventures), Canaan, Casdin Capital, Droia, Foresite Capital, Invus Opportunities, Pontifax and Wu Capital and is supported by investment from AbbVie Ventures and Taiho Ventures. PACT Pharma's technology is designed to individually program tumor-exclusive targeting into each patient's own immune system cells to eradicate their own cancer. The process, which is currently in Phase 1 clinical testing, involves taking a biopsy of a person's cancer tissue to assess the tumor-exclusive mutations with predictive algorithms, then to biologically verify the optimal targets by capturing T cells from blood that already recognize the mutations. Using the T cell receptor information from the captured T cells, together with proprietary, cutting edge, (non-viral) precision genome engineering technologies, fresh patient T cells are edited in one step to craft tumor-specific neoTCR-P1 cells. These private designer T cells have been shown to immediately kill mutation-expressing tumors in pre-clinical studies, and to create a deep reservoir of 'ready-to-go' neoTCR-P1 cells with the potential for long term persistence to prevent future cancer recurrence. These developments offer PACT exceptional prospects to leverage the potential of ideal tumor targets and biologically verified neoTCRs into clinical development of neoTCR-T adoptive cell therapies.

SOURCE: PACT Pharma

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Ori Biotech announces a $9.4M seed round to advance innovation in Cell and Gene Therapy manufacturing – BioSpace

Sunday, January 12th, 2020

January 9, 2020, London, UK and Philadelphia, USA - Ori Biotech Ltd (Ori), an innovator in Cell and Gene Therapy (CGT) manufacturing, today announced that they successfully closed a $9.4M (7M) seed round which will be used to bring their innovative manufacturing platform to market. The Ori platform will deliver scalable solutions to flexibly address the critical clinical and commercial manufacturing needs of CGT developers.

Founded by Dr. Farlan Veraitch and Prof. Chris Mason in 2015, Ori has designed a bespoke platform to specifically address the unique requirements of the new generation of personalised, living medicines. The investor syndicate is comprised of some of the UKs leading venture investors including Amadeus Capital Partners, Delin Ventures, Kindred Capital and a London-based family office, alongside a group of angel investors who have supported the company since inception.

Jason C. Foster, newly appointed CEO of Ori Biotech said: The successful financing underscores the potential of the Ori platform to fully automate cell and gene therapy manufacturing to increase throughput, improve quality and decrease costs. We look forward to collaborating with best-in-class suppliers, service providers and therapeutics developers to create next generation manufacturing solutions. We appreciate the support from our investors, and I am honored to join a company that has the potential to positively impact millions of lives by enabling patient access to these lifesaving treatments.

Hundreds of clinical trials and a few recently marketed products have shown the revolutionary potential of CGTs. But this potential will never be realised unless we can remove the current bottleneck around scalable manufacturing. Ori Biotech has developed an innovative platform technology to facilitate scalable manufacturing that could eventually enable millions of patients to get access to the next generation of personalised medicines, commented Dr Alan Barge, ex-Head of Oncology at AstraZeneca, Venture Partner at Delin Ventures and Non-Executive Director of Ori Biotech.

Dr Farlan Veraitch, Co-Founder and Chief Scientific Officer of Ori Biotech added, The challenges of providing high throughput, high quality and cost-effective CGT manufacturing are well documented in the industry and in publications by global regulatory authorities like the US FDA. By pioneering a completely novel hardware and software platform approach, we can help the CGT industry accelerate the delivery of these transformative therapies to patients in need.

Ori Biotech at JP Morgan Healthcare Conference, San Francisco

The Ori Biotech team will be at the 38th Annual J.P. Morgan Healthcare Conference on 13-16 January 2020 in San Francisco, California.

Please get in touch if you would like to set up a meeting, details below

About Ori Biotech

Ori Biotech is a London- and Philadelphia-based CGT manufacturing technology company. Ori has developed a proprietary, flexible manufacturing platform that closes, automates and standardises manufacturing allowing therapeutics developers to further develop and bring their products from pre-clinical process development to commercial scale manufacturing.

The mission of the Ori platform is to fully automate CGT manufacturing to increase throughput, improve quality and decrease costs in order to enable patient access to this new generation of lifesaving treatments. Founded by Dr. Farlan Veraitch and Prof. Chris Mason in 2015, the Company has brought together a seasoned Board and executive management team with over 80 years of pharmaceutical, cell therapy and venture building experience including CEO Jason C. Foster (Indivior) and CBO Jason Jones (Miltenyi Biotec) alongside industry-leading expert advisors like Bruce Levine and Anthony Davies.

For more information, contact:

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Ori Biotech announces a $9.4M seed round to advance innovation in Cell and Gene Therapy manufacturing - BioSpace

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Adverum Biotechnologies Reports Additional Clinical Data from First Cohort of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD at…

Sunday, January 12th, 2020

--44 week median follow up for patients (n=6)----Zero anti-VEGF rescue injections required following intravitreal ADVM-022; First patient has reached 52-weeks post treatment----Vision remains stable and anatomical improvements maintained--

MENLO PARK, Calif., Jan. 11, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced clinical data for the first cohort of patients (n=6) in the OPTIC phase 1 clinical trial of ADVM-022, the companys intravitreal injection gene therapy, in treatment-experienced patients with wet age-related macular degeneration (wet AMD). The data are being presented today by Charles C. Wykoff M.D., Ph.D., director of research, Retina Consultants of Houston, at the Atlantic Coast Retina Club Macula 20/20 Annual Meeting inNew York, NY.

A copy of the presentation is available on the Adverum corporate website under Events and Presentations in the Investors section, available here.

In October 2019, Adverum presented data from the first cohort in OPTIC at a median 34-week time point (28-44 week range). Today, additional data for the first cohort are being presented, including efficacy and safety data, with a median follow up of 44 weeks at a range of 40-52 weeks, and included:

As of December 1, 2019, ADVM-022 continues to be well-tolerated in the first cohort with no drug-related or procedure-related serious adverse events (SAEs), no drug-related systemic adverse events and no adverse events meeting the criteria for dose-limiting toxicities (DLTs). Low-grade inflammation was reported in all six patients and was generally mild to moderate and responsive to steroid eye drops. One ocular SAE, a retinal detachment, that was not related to ADVM-022 or the administration procedure was reported.

OPTIC Phase 1 Clinical Trial Data from Cohort 1 (n=6)

1 Best corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) (i.e., sight charts) 2 Central retinal thickness (CRT), also referred to as central subfield thickness (CST) assessed using Optical Coherence Tomography (OCT) imaging and measured by an independent Central Reading Center3 BCVA and CST values for patient with retinal detachment (unrelated to study treatment) used last observations prior to detachment 4 This event was deemed unrelated to ADVM-022 or any study procedure

These longer-term follow-up data demonstrate that patients in this first cohort of OPTIC are achieving sustained benefits from ADVM-022, a one-time intravitreal therapy, and have not required any anti-VEGF rescue injections through a median of 44 weeks while demonstrating impressive anatomic improvements, said Charles C. Wykoff M.D., Ph.D., director of research, Retina Consultants of Houston and associate professor of clinical ophthalmology, Blanton Eye Institute, Houston Methodist Hospital and Weill Cornell Medical College, Houston Texas. With a median follow-up period of 44 weeks, ADVM022 continues to control wet AMD disease activity in all 6 patients and the low-grade intraocular inflammation appears manageable with steroid eyedrops. Based on the data to date, ADVM-022 has the potential to be a meaningful and potentially transformative treatment for patients with wet AMD.

Aaron Osborne, MBBS, chief medical officer of Adverum, added, These new clinical data are promising as they continue to support the safety, efficacy, and durable clinical profile of ADVM-022 and this therapys potential to change the treatment paradigm for patients with wet AMD. Anti-VEGF injections, the current standard of care, carry a significant treatment burden and real-world outcomes data suggest that vision outcomes are suboptimal due to undertreatment. In the first cohort of OPTIC, we continue to see stable vision and anatomical improvements being maintained out to a median of 44 weeks after a single ADVM-022 injection in these difficult-to-treat patients who previously required frequent anti-VEGF injections. We look forward to presenting longer-term data from the first cohort and 24-week data from the second cohort of OPTIC on February 8 at the Angiogenesis, Exudation, and Degeneration 2020 symposium.

About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMDThe multi-center, open-label, Phase 1, dose-escalation trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. In the first cohort, patients (n=6) received ADVM-022 at a dose of 6 x 10^11 vg/eye and in the second cohort, patients (n=6) received ADVM-022 at a dose of 2 x 10^11 vg/eye. In the third cohort (n=9), patients also are receiving a dose of 2 x 10^11 vg/eye and in the fourth cohort (n=9), patients will receive a dose of 6x10^11 vg/eye. Patients in the third and fourth cohorts will receive prophylactic steroid eye drops instead of oral steroids which were used in the first and second cohorts. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as mean number of anti-VEGF rescue injections and percentage of patients needing anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.

Eight leading retinal centers acrossthe United States(U.S.) are participating in the OPTIC Phase 1 trial for ADVM-022. For more information on the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD, please visithttps://clinicaltrials.gov/ct2/show/NCT03748784.

About ADVM-022 Gene TherapyADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for wet AMD and diabetic retinopathy patients.

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for ADVM-022 for the treatment of this disease.

Adverum is currently evaluating ADVM-022 in the OPTIC Study, a Phase 1 clinical trial in patients 50 years and older with wet AMD. Additionally, Adverum plans to submit an Investigational New Drug Application for ADVM-022 for the treatment of diabetic retinopathy to the U.S. Food and Drug Administration in the first half of 2020.

About Wet Age-related Macular Degeneration (Wet AMD)Age-related macular degeneration (AMD) is a progressive disease affecting the macula, the region of the retina at the back of the eye responsible for central vision. In patients with wet AMD, an aggressive form of AMD, abnormal blood vessels grow underneath and into the retina. These abnormal blood vessels leak fluid and blood into and beneath the retina, causing vision loss.

Wet AMD is a leading cause of vision loss in patients over 60 years of age, with a prevalence of approximately 1.2 million individuals in the U.S. and 3 million worldwide. The incidence of new cases of wet AMD in the U.S. is approximately 150,000 to 200,000 annually, and this number is expected to grow significantly as the countrys population ages.

The current standard-of-care therapy for wet AMD is anti-VEGF intravitreal injections. These are effective but typically require eye injections every 4-12 weeks in order to maintain vision. Compliance with this regimen can be difficult for patients, caregivers, and healthcare systems, leading to undertreatment and resulting in loss of vision.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs for serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration. For more information, please visit http://www.adverum.com

Forward-looking StatementsStatements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to statements regarding: Adverums plans to report additional clinical data for ADVM-022 from the OPTIC trial and to advance ADVM-022, including Adverums plans to submit an Investigational New Drug Application for ADVM-022 for the treatment of diabetic retinopathy to the U.S. Food and Drug Administration in the first half of 2020, and the potential benefits of ADVM-022, all of which are based on certain assumptions made by Adverum on current conditions, expected future developments and other factors Adverum believes are appropriate in the circumstances. Adverum may not achieve any of these in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022; obtaining regulatory approval for gene therapy product candidates; enrolling patients in clinical trials; reliance on third parties for conducting the OPTIC trial and vector production; and ability to fund operations through completion of the OPTIC trial and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with the SEC on November 7, 2019 under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor and Media Inquiries:

Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com1-646-876-5196

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Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig’s Disease in Mice – BioSpace

Monday, December 30th, 2019

Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrigs disease, is a neurodegenerative disease affecting nerve cells in the brain and spinal cord. Researchers at the University of California San Diego School of Medicine published research describing a new way to deliver a gene-silencing vector to mice with ALS. The therapy resulted in long-term suppression of the disease if the treatment was given before the disease started. It also blocked disease progression in the mice if symptoms already appeared.

The study was published in the journal Nature Medicine.

At present, this therapeutic approach provides the most potent therapy ever demonstrated in mouse models of mutated SOD1 gene-linked ALS, said senior author Martin Marsala, professor in the Department of Anesthesiology at UC San Diego School of Medicine. In addition, effective spinal cord delivery of AAV9 vector in adult animals suggests that the use of this new delivery method will likely be effective in treatment of other hereditary forms of ALS or other spinal neurodegenerative disorders that require spinal parenchymal delivery of therapeutic gene(s) or mutated-gene silencing machinery, such as in C9orf72 gene mutation-linked ALS or in some forms of lysosomal storage disease.

ALS appears in two forms, sporadic and familial. The most common form is sporadic, responsible for 90 to 95% of all cases. Familial ALS makes up 5 to 10% of all cases in the U.S., and as the name suggests, is inherited. Studies have shown that a least 200 mutations of the SOD1 gene are linked to ALS.

In healthy individuals, the SOD1 gene provides instructions for an enzyme called superoxide dismutase. This enzyme is used to break down superoxide radicals, which are toxic oxygen molecules that are a byproduct of normal cellular processes. It is believed that the mutations in the gene cause ineffective removal of superoxide radicals or potentially cause other toxicities resulting in motor neuron cell death.

The new research involves injecting shRNA, an artificial RNA molecule that can turn off, or silence, a targeted gene. This delivers shRNA to cells by way of a harmless adeno-associated virus (AAV). In the research, they injected the viruses carrying shRNA into two locations in the spinal cord of adult mice expressing an ALS-causing mutation of the SOD1 gene. They were performed just before disease onset or after the laboratory animals started showing symptoms.

The researchers have tested the approach in adult pigs, whose have spinal cord dimensions closer to those in humans. They found that by using an injector developed for adult humans, the procedure could be performed without surgical complications and in a reliable fashion.

The next step will be more safety studies with a large animal model.

While no detectable side effects related to treatment were seen in mice more than one year after treatment, the definition of safety in large animal specimens more similar to humans is a critical step in advancing this treatment approach toward clinical testing, Marsala said.

About 5,000 people are diagnosed with ALS in the U.S. each year, with about 30,000 people living with the disease. There are symptomatic treatments, but no cure. Most patients die from the disease two to five years after diagnosis.

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First Alzheimer’s Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients – Benzinga

Monday, December 30th, 2019

Maximum Life Foundation ("MaxLife"), is rapidly transforming the way we treat aging diseases. MaxLife plans to use a promising gene therapy offered by Integrated Health Systems to give free access to ten (10) early to mid-stage Alzheimer's Disease (AD) patients. David Kekich, MaxLife's CEO, stated "MaxLife will grant 100% of the therapy costs to help bring pioneering gene therapy to cure this disease and make Alzheimer's Disease a thing of the past."

NEWPORT BEACH, Calif. (PRWEB) December 30, 2019

Cure Now Instead of Palliative Care

According to the Alzheimer's Association:

Alzheimer's costs Americans $277 billion a year and rising. Sharp increases in Alzheimer's disease cases, deaths and costs are stressing the U.S. healthcare system and caregivers. About 5.7 million Americans have Alzheimer's disease. To date, no one has survived it.

Improvements of AD symptoms and the recovery of normal brain functions have been demonstrated in-vivo in mouse experiments, and in-vitro in human cell experiments through the rejuvenation of microglia (the brain's first line of defense against infection) and neurons as well as stimulating mitochondrial function using the telomerase reverse transcriptase (TERT) protein.

One human patient received a lower dose therapy in August 2018 with no adverse side effects. To date, the patient's disease has not progressed. MaxLife hopes to see symptom reversals in the next patients.

"If we can prove a benefit to patients that have no other option now, we can potentially treat Alzheimer's Disease in people in early to mid-stage Alzheimer's, finally creating effective medicine at the cellular level," states Kekich. "If successful, this treatment could potentially be used on other diseases such as Parkinson's and ALS."

The unique difference is developing treatments against the cellular degeneration caused by aging as the root cause of most major diseases. Studies have proven aging is the leading risk factor for many life-threatening diseases, including Alzheimer's.

With a world class Scientific Advisory Board, MaxLife is ready to push forward into practical solutions. A gene therapy facilitator, Integrated Health Systems plans to treat other adult aging-related diseases with no previous cure such as Sarcopenia, Atherosclerosis, Chronic Kidney Disease (CKD) and even aging itself with gene therapies.

"This technology could halt many of the big age associated killers in industrialized countries'" states Kekich. "Compassionate care helps patients with no other option to get access to experimental therapies that may benefit both themselves and society as a whole."

MaxLife also seeks grants and donations for human gene therapy studies for atherosclerosis, sarcopenia and chronic kidney disease as well as for human aging. The protocols have already been developed. Please Click Here and scroll to the bottom of the page to see how to donate.

To apply for a free therapy or for more information, see http://www.maxlife.org/alzheimers-disease/ and https://maxlife.org/how-to-register-and-qualify-for-the-alzheimers-human-study/.

For Further Information, Contact: David Kekich, CEO Maximum Life Foundation.

Maximum Life Foundation is a 501(c)(3) Not-For-Profit corporation founded in 1999.

Tax I.D. #31-1656405. David A. Kekich Tel. #949-706-2468. Info@MaxLife.org

For the original version on PRWeb visit: https://www.prweb.com/releases/first_alzheimers_disease_gene_therapy_human_study_provided_by_maximum_life_foundation_offers_10_free_therapies_for_qualifying_patients/prweb16809113.htm

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BLA Submitted for Gene Therapy to Treat Hemophilia A – Monthly Prescribing Reference

Monday, December 30th, 2019

Home News Drugs in the Pipeline

BioMarin has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults. This is the first marketing application submission for a gene therapy product for any type of hemophilia.

Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy that is administered as a single infusion to produce clotting factor VIII. The BLA submission is supported by interim analysis of a phase 3 study and 3-year phase 1/2 data. Results from the ongoing phase 1/2 study showed that bleed rate control and reduction in factor VIII usage was maintained for a third year following a single administration of valoctocogene roxaparvovec.

The FDA previously granted Breakthrough Therapy and Orphan Drug designations to valoctocogene roxaparvovec. The Company anticipates the BLA review to commence in February 2020.

We look forward to working with the FDA as we seek marketing authorization for the potential first gene therapy for hemophilia A, said Hank Fuchs, MD, President, Global Research and Development at BioMarin. Our hope is one day very soon to deliver a transformative treatment that has the potential to change the way hemophilia A is treated.

For more information visit biomarin.com.

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Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research – News18

Monday, December 30th, 2019

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome. Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy. "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency." Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells. The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors." The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured. "This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting." In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden. For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs. The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself. "It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy. Scientists practising the technique insert a normal gene into cells containing a defective gene. It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient. Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union. They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster." Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period." "We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies. They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor. However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine. Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans. The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process. "That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations. "It's very easy to do if you don't care about the consequences," Musunuru added. Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability. There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US. The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online. "Not everyone is a biologist or scientist," she said. And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops? Charpentier thinks that technology generally tends to be used for the better. "I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18

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2019: The year gene therapy came of age – INQUIRER.net

Monday, December 30th, 2019

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the United States and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

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Gene Therapy Market 2020: New Innovative Solutions to Boost Global Growth with New Technology, Busin – PharmiWeb.com

Monday, December 30th, 2019

Global Gene Therapy MarketResearch Report 2020-2029 is a vast research database spread across various pages with numerous tables, charts, and figures in it, which provides a complete data on the Gene Therapy market including key components such as main players, size, SWOT analysis, business situation, and best patterns in the market. This analysis report contains different expectations identified with income, generation, CAGR, consumption, cost, and other generous elements. Further, the report determines the opportunities, its restraints as well as analysis of the technical barriers, other issues, and cost-effectiveness affecting the market during the forecast period from 2020 to 2029. It features historical & visionary cost, an overview with growth analysis, demand and supply data. Market trends by application global market based on technology, product type, application, and various processes are analyzed in Gene Therapy industry report.

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By Vector: Viral vector Retroviruses Lentiviruses Adenoviruses Adeno Associated Virus Herpes Simplex Virus Poxvirus Vaccinia Virus Non-viral vector Naked/Plasmid Vectors Gene Gun Electroporation Lipofection By Gene Therapy: Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Other By Application: Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

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Base Year: 2019 | Estimated Year: 2020 | Forecast Year: 2020 to 2029

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CONTINUE

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