Category Archives: Genetic Medicine

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC)

Maintains Orphan Drug Designation (ODD) in the EU Providing 10-years of Market Exclusivity

Significant BenefitOver Existing Therapies for Patients with Severe Hemophilia A in EU Based on EMA Determination of ODD

Conference Call and Webcast to be Held Wed., Aug. 24th at 8:00 pm Eastern

SAN RAFAEL, Calif., Aug. 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). The EC also endorsed EMA's recommendation for Roctavian to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity. The EMA recommendation noted that, even in light of existing treatments, Roctavian may potentially offer a significant benefit to those affected with severe Hemophilia A. The one-time infusion is the first approved gene therapy for hemophilia A and works by delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis, thus relieving patients of their treatment burden relative to currently available therapies. People with hemophilia A have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting.

It is estimated that more than 20,000 adults are affected by severe hemophilia A across more than 70 countries in Europe, the Middle East, and Africa. Of the 8,000 adults with severe hemophilia A in the 24 countries within BioMarin's footprint covered by today's EMA approval, there are an estimated 3,200 patients who will be indicated for Roctavian. BioMarin anticipates additional access to ROCTAVIAN for patients outside of the EU through named patient sales based on the European Medicines Agency (EMA) approval in countries in the Middle East, Africa and Latin America and expects additional market registrations to be facilitated by the EMA license.

"This approval in the EU represents a medical breakthrough in the treatment of patients with severe hemophilia A that expands the conversation between a patient and physician on treatment choices to now include a one-time infusion that protects from bleeds for several years," said Professor Johannes Oldenburg, Director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany. "It is exciting to imagine the possibilities of this approved gene therapy, which has demonstrated a substantial and sustained reduction in bleeding for patients, who potentially could be freed from the burden of regular infusions."

"Roctavian approval in Europe is a historic milestone in medicine and is built upon almost four decades of scientific discovery, innovation, and perseverance. We thank the European Commission for recognizing Roctavian's value as the first gene therapy for hemophilia A, a feat that we believe will transform how healthcare professionals and the patient community think about caring for bleeding disorders," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer of BioMarin. "We are grateful to the patients, investigators and community, who dedicated their time and effort to this achievement and whose aspirations provided the driving force behind making this one-time therapy a reality."

The EC based its decision on a significant body of data from the Roctavian clinical development program, the most extensively studied gene therapy for hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study. The GENEr8-1 Phase 3 study demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate. In addition, the data included five and four years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study. BioMarin has committed to continue working with the broader community and the EMA to monitor the long-term effects of treatment. The Product Information will be available shortly on the EMA website under the Medicines tab. Search for "ROCTAVIAN" and select "Human medicine European public assessment report (EPAR): Roctavian. Then select "Product Information" in the Table of Contents and then select "Roctavian: EPAR Product Information."

A Conditional Marketing Authorization (CMA) recognizes that the medicine fulfils an unmet medical need based on a positive benefit-risk assessment, and that the benefit to public health of the immediate availability on the market outweighs the uncertainties inherent to the fact that additional data are still required. BioMarin will provide further data from ongoing studies within defined timelines to confirm that the benefits continue to outweigh the risks, building on what already constitutes the largest clinical data package for gene therapy in hemophilia A. Conversion to a standard marketing authorization will be contingent on the provision of additional data from currently ongoing Roctavian clinical studies, including longer-term follow up of patients enrolled in the pivotal trial GENEr8-1, as well as a study investigating efficacy and safety of ROCTAVIAN with prophylactic use of corticosteroids (Study 270-303), for which enrollment is now complete.

Orphan drug designation is reserved for medicines treating rare (affecting not more than five in 10,000 people in the EU), life-threatening or chronically debilitating diseases. Authorized orphan medicines benefit from ten years of market exclusivity, protecting them from competition with similar medicines with the same therapeutic indication, which cannot be marketed during the exclusivity period.

BioMarin remains committed to bringing Roctavian to eligible patients with severe hemophilia A in the United States and is targeting a Biologics License Application (BLA) resubmission for Roctavian by the end of September 2022. Typically, BLA resubmissions are followed by a six-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.

Robust Clinical Program

BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3B, single arm, open-label study to evaluate the efficacy and safety of Roctavian at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A (Study 270-303). Also ongoing are a Phase 1/2 Study with the 6e13 vg/kg dose of Roctavian in people with hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and aa Phase 1/2 Study with the 6e13 vg/kg dose of Roctavian in people with hemophilia A with active or prior Factor VIII inhibitors (Study 270-205).

Safety Summary

Overall, single 6e13 vg/kg dose of Roctavian has been well tolerated with no delayed-onset treatment related adverse events. The most common adverse events (AE) associated with Roctavian occurred early and included transient infusion associated reactions and mild to moderate rise in liver enzymes with no long-lasting clinical sequelae. Alanine aminotransferase (ALT) elevation (113 participants, 80%), a laboratory test of liver function, remained the most common adverse drug reaction. Other adverse reactions included aspartate aminotransferase (AST) elevation (95 participants, 67%), nausea (52 participants, 37%), headache (50 participants, 35%), and fatigue (42 participants, 30%). No participants developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with Roctavian.

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (Factor VIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (Factor VIII 1-5%) or mild (Factor VIII 5-40%) disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week (100-150 infusions per year) or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month (12-48 infusions per year). Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.

Conference Call and Webcast to be Held Wed., Aug. 24th at 8:00 pm Eastern

BioMarin will host a conference call and webcast to discuss the EC approval today, Wed., Aug. 24th at 8:00 pm Eastern. This event can be accessed in the investor section of the BioMarin website at https://investors.biomarin.com/events-presentations.

U.S./Canada Dial-in Number: 800-831-4163

Replay Dial-in Number: 800-645-7964

International Dial-in Number: 213-992-4616

Replay International Dial-in Number: 757-849-6722

(No ID required for live call)

Playback ID: 9184

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visit http://www.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the number of adults across Europe, the Middle East, and Africa who are affected by severe hemophilia A; the number of adults in the countries within BioMarin's footprint covered by the EMA approval who have severe hemophilia A and are indicated for Roctavian; BioMarin anticipating additional access to Roctavian for patients outside of the EU through named patient sales based on the EMA approval in countries in the Middle East, Africa and Latin America and the expectation that additional market registrations will be facilitated by the EMA license; the potential for Roctavian to be a one-time infusion protecting patients from bleeds for several years and freeing them from the burden of regular infusions; Roctavian potentially offering a significant benefit to those affected with severe hemophilia A; Roctavian potentially transforming how healthcare professionals and the patient community think about caring for bleeding disorders; BioMarin's plans to provide further data from ongoing studies within defined timelines to confirm that the benefits of Roctavian continue to outweigh the risks; conversion of Roctavian's CMA to a standard marketing authorization; BioMarin's plans to re-submit a BLA for Roctavian to the FDA by the end of September 2022; and the duration of the FDA's review procedure of BioMarin's BLA resubmission for Roctavian. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results and timing of current and planned preclinical studies and clinical trials of Roctavian; additional data from the continuation of the clinical trials of Roctavian, any potential adverse events observed in the continuing monitoring of the participants in the clinical trials; the content and timing of decisions by the FDA, the EC and other regulatory authorities, including decisions to grant additional marketing registrations based on an EMA license; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture Roctavian for the clinical trials and commercially; our ability to provide the additional data from currently ongoing Roctavian clinical studies to support the conversion from a CMA to a standard marketing authorization; and those and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc and ROCTAVIAN is a trademark of BioMarin Pharmaceutical Inc.

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Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

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Here is the original post:
First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC) -...

Its an impressive thing, an absolute revolution for medicine, he says. Osvaldo Podhajesarmolecular biologist who integrates Leloir Institute who with their team are almost the only ones who investigate gene treatment in Argentina. These treatments are based on the concept of being able to modify a cell at the genetic level so that a disease can be reversed. Some examples that show how disruptive these treatments are are patients. Spinal Muscular Atrophy (SMA) those who sit or walk, those who progress to blindness from Alzheimers disease Labour and regained vision or those that were somehow cured leukemia, In that league, where science thins some fictional stories, it is this type of therapy at play that represents a unique window toward a new opportunity for thousands of people.

One of the possible techniques for performing this type of therapy is described by Hernan Martinoboss Scientific researcher from the University Hospital of Australias Pediatric Neurology and the Argentine Federation of Rare Diseases (Fedepof)Genetically modified is to administer genetic material to the patient by means of a viral vector. This modified virus, which also removed the possibility of being pathogenic, is the one that enters the cells and corrects the error.

What three criminal lines can child death investigators pursue in Crdoba?

for its part, Susanna Baldinimedical director of the Argentine Chamber of Medicinal Specialties (Caeme)who, among other topics, talked about gene therapies at the first meeting of media and pharmawhich took place in Mendoza, in which they participated Country, show that the nucleus of the cell contains chromosomes, which are formed by genes. With each chromosome having two pairs, it is possible that one or both are mutated. Dominant diseases require only one copy to be abnormal to develop in the individual, whereas recessive diseases require both copies to be mutated. And those errors or mutations are what this type of therapy tries to correct.

A little history

Podhajaser Explains that the first clinical trials of gene therapy took place in 1990 and involved genetically modifying the T lymphocytes of a girl who suffered from an immunodeficiency linked to the ADA (adenosine deaminase) gene. In boys who suffer from this disease, their immune system does not work properly and they have to stay in isolation. Since then, thousands of clinical studies have been conducted in this discipline, used in congenital metabolic diseases (where the mutated gene is known to be unable to produce normal proteins) and in more complex diseases such as cancer or neurodegenerative diseases. . ,

,Gene therapy has made remarkable progress And these children with mutations in the ADA gene can have gene therapy and can now lead normal lives with their reorganized immune systems. But advances in gene therapy have occurred not only in this disease in particular, but also extend to retinopathy, where people with blindness have regained their vision as if Leber congenital amaurosis. In this case, the RP65 gene is directly delivered to the retina. or with friends spinal muscular atrophy One who cannot sit can do so again after receiving specific gene therapy of the mutated gene which is also administered using viral vectors, he details. Podhajaser,

amartino Recalls a case of a patient in the late 1990s who was treated for a disease OTC, which had a very severe immune reaction to the vector and died. This delayed many other research related to gene therapy. However, later studies continued and today the results are generally very successful. Of course, he claims amartinoThere is still not enough time to know if these treatments will have any effect for long-term use.

two types of gene therapy

On the one hand, this description amartinothere are in vivo therapy, In this type of therapy, the viral vector that transfers the gene can be applied directly to the organ or tissue where the disease is most affected.

Instead, in ex vivo Stem cells are taken from the patient, we modify them and we insert a new gene into them. Then we re-infect the previously modified cells. Its Like an Autotransplant, For that you have to first give him chemo and remove all his white blood cells. Whether one type of therapy or the other is used will depend on the patients disease, although there are diseases for which both methods are investigated, says the expert.

An example of an ex vivo therapy is CAR-T. is used, It is a cellular gene therapy where cells are taken from the patient and they are genetically manipulated so that they can attack the malignant cells. So the patient kills his own cancer. For now this type of treatment is mainly used for certain types of leukemia., he argues baldini,

Podhajaser warns that the use of car-t It is a treatment that, although it is already used, is very complex. An appropriate laboratory is required to modify these cells with the gene of interest. After modification, the cells are kept in the laboratory for some time and reintroduced to the patient. The patient must be close to that laboratory and the cells cannot be shipped from Argentina to the United States because they will not arrive properly.

another problem of car-tadd Podhajaser Like conventional cancer therapy, the tumor has resistance over time. CAR-Ts are usually directed against a specific protein that they recognize and use to attack the malignant cell. Unfortunately, tumors can recur from cells that do not express this protein and thus survive treatment.

The third drawback is that car-t They do not work as a sole treatment in solid tumors, which are the most frequent tumors. And the reasons are simple: they work so well in hematopoietic tumors because they are cells that do not form a compact tumor tissue, unlike most cancers. And CAR-T just cant enter the tumor, he explains. Podhajaser,

Innovative, but too expensive

One of the issues with these treatments is cost. Millions of dollars are being invested in research and development for hundreds of rare diseases, but this high level of investment is inevitably going to make the treatments very expensive, he laments. express. amartino,

In Argentina, there was a case demonstrating the complexity of obtaining sufficient funding for this type of treatment. Emma, the child who suffered from SMA type 2 and required US$2,100,000 worth of medication from the Novartis laboratory. In order to increase that amount, the influential person Santiago Marata launched the campaign Everyone with Emmita.

,Gene therapy far too expensive, on the order of hundreds of thousands of dollars. Many of the accepted gene therapies either cure a person with a previously incurable disease, or significantly increase their quality of life. To address the payment for these treatments, what is being achieved is negotiations between developing companies and states, because being rare diseases, there are not so many patients who need these treatments, he says. . Podhajaser,

with your colleague, baldini Gives the example of Spain, where there is a shared risk scheme between companies and the state. If they give treatment to the patient and it is not successful, they do not pay for the said treatment.

See the original post here:
Walk Again Or Stop Blindness. How Gene Therapy Is Revolutionizing Medicine - Nation World News

Researchers from the Murdochs Children Research Institute (MCRI) are developing new treatments for congenital heart disease that could enable children born with birth defects can regenerate the damaged organ.

In 2011, Prof. Enzo Porello, who is nowhead of the Heart Regeneration Laboratory at the MCRI,demonstrated the regenerative properties of newborn mouse hearts at the University of Texas Southwestern Medical Centre. Prior to this research, the capacity of mammalian hearts to regenerate was a debated topic.

This sort of changed our thinking of what was possible in terms of stimulating the human heart to regenerate itself following damage, such as a heart attack, Porrello said, reported theAustralian. And I guess this also fuelled my own interest in my subsequent career in the area of regenerative medicine.

After hearing about cases where newborns recovered from massive heart attacks, Porello began to explore the regenerative properties of human newborn hearts.

In 2017, Porello and Prof. James Hudson manufactured living and beating heart tissues from stem cells in a laboratory at the University of Queensland.

Porello said that although other scientists had grown heart muscle cells from stem cells, nobody had grown the cells as miniature complex three-dimensional tissues. Additionally, they were not able to grow such tissues in a format compliant to drug development, he said.

And thats really the technological breakthrough that we were able to make.

According to the Australian Institute of Health and Welfare, approximately 9 out of every 1,000 babies born around the world will be born with congenital heart disease. In Australia, it is estimated that 2,400 babies are born with congenital heart disease annually, while in America, nearly one percent of all babies born are estimatedby the Centre For Disease Control to have the condition.

Porello said that, at the moment, if a child develops heart failure and doesnt respond to standard frontline therapies, a heart transplant is their only option. Children in this situation are put on a transplant waiting list, and whilst waiting for a heart to become available, they are put on mechanical support.

Heart transplantation is limited by organ donor availability, and its also limited by the need for lifelong immunosuppression in those patients, Porello said.

And so if were able to develop these bioengineered heart tissues from stem cells, this could potentially prevent or delay the need for heart transplantation in these very unwell individuals with end-stage heart failure.

Porello said that the ultimate goal of his research is to harness the self-repairing capacity of the newborn heart and to develop drugs that waken the hearts dormant regenerative abilities so that the organ may repair itself after damage.

I would say that based on recent studies in the field in the past 10 years since we first made our discovery in mice, we are certainly getting closer, he said.

There is sort of proof of concept that this is possible now, at least in mice, and the question is whether or not we can now make that a therapeutic reality in humans.

The first step in creating these complex heart tissues is attaching special molecules to stem cells; these molecules trigger the cells to morph into heart muscle tissue. The heart tissues are then developed in a plastic culture dish that consists of 96 tiny wells.

The geometry of the well is designed in such a way that the heart tissues spontaneously form when the heart muscle cells are inserted into the well, Porrello said.

He said that within each well of the device are tiny elastic micropillars; the pillars function as elastic cantilevers since they are attached to the dish at only one end and extend horizontally to the dish. The heart muscle cells condense around these cantilevers to produce tiny miniature beating heart tissues that contract around the micropillar; every time the tissue contracts, the micropillar within it deflects.

Porello said that the device enables researchers to measure the force that the tissues are generating, allowing them to observe how fast the tissues are beating and whether they display any irregularities in their heartbeat. These capabilities are useful for treatment testing because the effect that medication or genetic manipulations of stem cells have on the tissues heartbeat can be seen.

And so it serves as a pretty powerful platform for looking at drug responses, but also modelling genetic forms of heart disease.

Were actually now scaling up these tissues and growing very, very large bioengineered heart tissue patches that can be implanted onto the heart.

In an email to The Epoch Times, Porello said in the future that bioengineered heart tissue patches could be used to treat adults with heart failure, and alternative approaches are already being trialled.

Our bioengineered heart tissues could also be used to support the failing heart in adults with underlying heart disease.

Further studies are required to confirm that our bioengineered heart tissue patches are safe and effective in animal models before progressing to human trials. These pre-clinical safety and efficacy studies are underway.

He noted that although significant advances and a better understanding of the hearts regenerative mechanisms have been made in recent years, using this knowledge to develop a safe and effective drug is a slow process.

It typically takes 10 years and around $1 billion dollars to develop a new heart failure drug and take it all the way through to clinical approval. We are at the beginning of that journey.

We need to gain a better understanding of the fundamental biology underlying heart regeneration before we can develop effective treatments.

Porello is now applying his discoveries in a clinical context at theMCRIto reach his goal of regenerating human hearts. The regeneration research at the institute has two branches, the first focuses on studying diseases using lab-grown models of the heart muscle. The models are made using blood and tissue samples collected from sick children at the Royal Childrens Hospital in Melbourne.

He said that this branch of the research enables the team to model the genetic basis of the disease in any individual.

Were using this technology to model childhood heart disease, trying to understand its causes, and then using those genetic models of heart disease to test and develop therapeutic approaches to treat those conditions, he said.

Porello said that the second branch of the research performed at the MCRI explores the regenerative approach to growing the very, very large bioengineered heart tissue patches. The researchers plan is to eventuallyimplant the patches into a heart to function as a biological assistance device that supports the function of the heart.

If it works, it would be transformative, Porello said.

Stem cells have been used in medicine for more than fifty years, with the most common stem cell procedure currently beingbone marrow transplantsalso known as hematopoietic stem cell transplantsused to treat patients with blood cancers such asleukemiaand blood disorders such assickle cell diseaseandthalassemia.

More recently, skin grown from stem cells has been used to treat extensive burns, and stem cells from fat (adipose tissue) have been used as tissue fillers.

More here:
Regenerative Properties of the Newborn Heart Offers Hope for Those With Congenital Heart Disease - The Epoch Times

By performing a DNA comparison of two similar jellyfish species, researchers have found the genes that could stop and reverse ageing in immortal jellyfish

By Jason P. Dinh

One jellyfishs regeneration powers seem linked to key genetic changes.

Roy Ensink Photography

An immortal species of jellyfish has double copies of genes that protect and repair DNA. The finding could provide clues to human ageing and age-related conditions.

Jellyfish start their lives as drifting larvae. They eventually attach to the seafloor and develop into sprout-like polyps. The bottom-dwellers clone themselves, forming stacked, sedentary colonies that bud off into free-swimming umbrella-shaped medusas.

That stage is a dead end for most jellyfish but the immortal jellyfish (Turritopsis dohrnii) can reverse the cycle. When times get tough,like in harsh environments or after injury, they melt their bodies into amorphous cysts, reattach to the seafloor and regress into polyps. They can restart the cycle indefinitely to skirt death by old age.

To find out how the immortal jellyfish staves off aging, Maria Pascual-Torner at the University of Oviedo in Spain and her colleagues sequenced its genome its full set of genetic instructions and compared it to that of the related but mortal crimson jellyfish (Turritopsis rubra).

They found the immortal jellyfish had twice as many copies of genes associated with DNA repair and protection. These duplicates could produce greater amounts of protective and restorative proteins. The jellyfish also had unique mutations that stunted cell division and prevented telomeres chromosomes protective caps from deteriorating.

Then, to pinpoint how T. dohrnii reverts into polyp form, the scientists looked at which genes were active during this reverse metamorphosis. They found the jellies silenced developmental genes to return cells to a primordial state and activated other genes that allow the nascent cells to re-specialise once a new medusa buds off. Together, Pascual-Torner says, these genetic alterations shield the animal from the weathering of time.

But Maria Pia Miglietta at Texas A&M University at Galveston points out that the crimson jellyfish can also rejuvenate, just not as commonly as T. dohrnii. Using them for comparison might reveal differences in the degree of immortality rather than the key to immortality itself, she says.

Still, Pascual-Torner says the genes they identified could be relevant to human ageing. They could inspire regenerative medicine or provide insights into age-related diseases like cancer and neurodegeneration. The next step is to explore these gene variants in mice or in humans, she says.

Journal reference: Proceedings of the National Academy of Sciences, DOI: 10.1073/pnas.2118763119

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Immortal jellyfish genes identified that may explain their long lives - New Scientist

PITTSBURGH--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio) and the University of Pittsburgh today announced that they have entered into a long-term strategic partnership to accelerate the development of highly innovative cell and gene therapies. Through this agreement, ElevateBio will locate one of its next BaseCamp process development and Good Manufacturing Practice (GMP) manufacturing facilities in Pittsburgh, fully equipped with its enabling technologies, including gene editing, induced pluripotent stem cell (iPSC) and cell, vector, and protein engineering capabilities. The University of Pittsburgh has long been a research powerhouse and is consistently among the top U.S. institutions in National Institutes of Health research funding.

The Richard King Mellon Foundation announced a $100 million grant to the University of Pittsburgh in November 2021 to create the Pitt BioForge BioManufacturing Center at Hazelwood Green. The grant was the largest single-project grant in the Foundation's 75-year history. The University of Pittsburgh and ElevateBio BaseCamp intend to locate the new technology-enabled process development and GMP manufacturing facility at Pitt BioForge at Hazelwood Green to further innovation in the Pittsburgh region. The new facility is expected to generate more than 170 permanent full-time jobs, 900 construction jobs, and 360 off-site support jobs.

This announcement supports the region's rise as a leader in cell and gene therapy and advances our vision of bringing an entirely new commercial manufacturing sector to the area," says Patrick Gallagher, Chancellor of the University of Pittsburgh. "The University of Pittsburgh is proud to partner with ElevateBio in this work, which will see us leveraging lessons from the labin new and exciting waysfor the benefit of human health.

To realize our vision of transforming the cell and gene therapy field for decades to come, broadening our footprint across metropolitan areas is a key priority for us, and we are thrilled that the University of Pittsburgh will be home to one of our BaseCamp facilities, said David Hallal, Chairman and Chief Executive Officer of ElevateBio. Weve identified Pittsburgh as an ideal location to extend our BaseCamp presence as it sits at the intersection of science, technology, and talent. We are grateful for the support of the Governor and County Executive as we bring the first-of-its-kind offering we have built at ElevateBio BaseCamp to advance the work of the entire biopharmaceutical industry.

Pitt Senior Vice Chancellor for the Health Sciences, Dr. Anantha Shekhar, continued by saying, We have some exceptional emerging research coming out of the University of Pittsburgh. However, the missing ingredient has been access to high-quality process science and manufacturing capabilities. As we position ourselves to become the next global hub for life sciences and biotech, we were in search of the right partner to help us realize our vision, and ElevateBios expertise and reputation in cell and gene therapy made them the perfect partner to accelerate our ability to build our biomanufacturing center of excellence.

This partnership between two national life-science powerhouses the University of Pittsburgh and ElevateBio - is a consequential step forward in realizing our shared vision to make Pittsburgh a national and international biomanufacturing destination, said Sam Reiman, Director of the Richard King Mellon Foundation. Pitt BioForge is a generational opportunity to bring extraordinary economic-development benefits to our region, and life-changing cell and gene therapies to patients - distribution that will be accelerated and enhanced by Pitts partnership with UPMC. ElevateBio could have chosen to locate its next biomanufacturing hub anywhere in the world; the fact they are choosing to come to Pittsburgh is another powerful validation of our region, and the Pitt BioForge project at Hazelwood Green.

We are excited that Pitt, working with UPMC Enterprises, has attracted ElevateBio to this region, said Leslie Davis, President and Chief Executive Officer of UPMC (University of Pittsburgh Medical Center). The companys expertise and manufacturing capabilities, combined with Pitt research and UPMCs clinical excellence, are essential to delivering the life-changing therapies that people depend on UPMC to deliver.

In addition, the Commonwealth of Pennsylvania and the County of Allegheny have provided incentive grants to ElevateBio in support of this partnership to build a biomanufacturing center and establish Pittsburgh as a premier biomanufacturing destination.

This announcement is continued verification of Pittsburgh's ability to attract new and emerging companies that provide economic opportunities in the life sciences field. The University of Pittsburgh and its medical school are a magnet for that ecosystem and along with this region's quality of life and investment in innovation, we continue to see businesses choosing Pittsburgh, said County Executive Rich Fitzgerald. The creation of the Innovation District, and the many companies that call it home, continue to provide great opportunities for talent to fill jobs across the ecosystem's pipeline. We welcome ElevateBio to our region and look forward to all that you will do here as part of this great ecosystem.

About ElevateBio:

ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, BaseCamp in Waltham, MA, is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. It was designed to support diverse cell and gene therapy products, including autologous, allogeneic, and regenerative medicine cell products such as induced pluripotent stem cells, or iPSC, and viral vector manufacturing capabilities.

Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

For more information, visit us at http://www.elevate.bio, or follow ElevateBio on LinkedIn, Twitter, or Instagram.

About the University of Pittsburgh:

Founded in 1787, the University of Pittsburgh is an internationally renowned leader in health sciences learning and research. A top 10 recipient of NIH funding since 1998, Pitt has repeatedly been ranked as the best public university in the Northeast, per The Wall Street Journal/Times Higher Education. Pitt consists of a campus in Pittsburghhome to 16 undergraduate, graduate and professional schools and four regional campuses located throughout Western Pennsylvania. Pitt offers nearly 500 distinct degree programs, serves more than 33,000 students, employs more than 14,000 faculty and staff, and awards 9,000 degrees systemwide.

For more information, please visit http://www.pitt.edu and http://www.health.pitt.edu.

About the Richard King Mellon Foundation:

Founded in 1947, the Richard King Mellon Foundation is the largest foundation in Southwestern Pennsylvania, and one of the 50 largest in the world. The Foundations 2021 year-end net assets were $3.4 billion, and its Trustees in 2021 disbursed $152 million in grants and Program-Related Investments. The Foundation focuses its funding on six primary program areas, delineated in its 2021-2030 Strategic Plan.

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ElevateBio and the University of Pittsburgh Announce Creation of Pitt BioForge BioManufacturing Center at Hazelwood Green to Accelerate Cell and Gene...

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Molecular prevalence and genetic diversity of Bartonella spp. in stray cats of zmir, Turkey - Parasites & Vectors - Parasites & Vectors