Archive for January, 2020

Researchers have developed cytochalasin B-induced membrane vesicles which they suggest could be a new form of cell-free therapy in regenerative medicine.

Work on extracellular microvesicles (ECMVs) derived from human mesenchymal stem cells (MSCs) has revealed a potential new form of cell-free therapy.

ECMVs are microstructures surrounded by a cytoplasm membrane; they have proven to be a prospective therapeutic tool in regenerative medicine due to their biocompatibility, miniature size, safety and regenerative properties. These can be used to circumvent the limitations of existing cell therapies without losing any effectiveness.

Cell therapies are grafts or implants of living tissue, such as bone marrow transplants, used to replace and regenerate damaged organ tissue. They currently have limited applications, as they work differently dependent on conditions and the environment they are placed into. They can also be rejected by the immune system.

A study at Kazan Federal University, Russia, has investigated cytochalasin B-induced membrane vesicles (CIMVs) which are also derived from MSCs and are very similar to natural ECMVs.

Proteome analysis of human MSCs and CIMVs-MSCs. Venn diagram of identified proteins MSCs and CIMVs-MSCs (A). Distribution of the identified proteins in organelles, percent of unique identified proteins (B) (credit: Kazan Federal University).

The scientists studied and characterised the biological activity of MSC-derived CIMVs. A number of biologically active molecules were found in CIMVs, such as growth factors, cytokines and chemokines; their immunophenotype was also classified.They also found that CIMVs could stimulate angiogenesis in the same way as stem cells.

The team came to the conclusion that human CIMVs-MSCs can be used for cell-free therapy of degenerative diseases. Induction of therapeutic angiogenesis is necessary for the treatment of ischemic tissue damage (eg, ischemic heart disease, hind limb ischemia, diabetic angiopathies and trophic ulcers) and neurodegenerative diseases (eg, multiple sclerosis and Alzheimers disease), as well as therapies for damage of peripheral nerves and spinal cord injury.

The group say they are continuing to research the therapeutic potential for artificial microvesicles for autoimmune diseases.

The study was published in Cells.

Follow this link:
Novel form of cell-free therapy revealed by researchers - Drug Target Review

Autologous stem cell and non-stem cell based therapies involve an individuals cell to be cultured and then re-introduced to the donors body. These therapies do not use foreign organism cells and are therefore free from HLA incompatibility, disease transmission, and immune reactions.Increasing demand for the new therapies in the field of regenerative medicine is directly facilitating the growth of autologous stem cell and non-stem cell based therapies market. Furthermore, since the risk to transplantation surgeries is significantly reduced in these therapies, they are increasingly being preferred for treatment of bone marrow diseases, aplastic anemia, multiple myeloma, non-Hodgkins lymphoma, Hodgkins lymphoma, Parkinsons disease, thalassemia, and diabetes.

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Moreover, rising incidents of cancer, diabetes and cardiovascular diseases along with growing geriatric population is another factor attributed for its high growth. However, side-effects of autologous stem cell and non-stem cell based therapies such as nausea, infection, hair loss, vomiting, diarrhea, etc. are expected to affect the market to an extent. High cost is another factor that can act as challenge to autologous stem cell and non-stem cell based therapies market. In spite of this, less risk post transplantation surgeries and favorable tax reimbursement policies are anticipated to reduce the impact of these limitation during the forecast period.Autologous stem cell and non-stem cell based therapies market can be segmented on the basis of application, end-user, and region.

In terms of application, the autologous stem cell and non-stem cell based therapies market can be segmented into blood pressure (BP) monitoring devices, intracranial pressure (ICP) monitoring devices, and pulmonary pressure monitoring devices. In terms of end-user, the market can be segmented into ambulatory surgical center and hospitals. By region, the market can be segmented into North America, Europe, Asia Pacific, Middle East and Africa and South America. Amongst all, Asia Pacific is anticipated to be the most attractive market owing to favorable reimbursement policies in the region.The players operating in autologous stem cell and non-stem cell based therapies market are limited.

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They are consistently involved in research and development activities for product development to keep up with the growing competition, thereby aiding the growth of autologous stem cell and non-stem cell based therapies market across the world.

The major players operating in autologous stem cell and non-stem cell based therapies market are Regennex, Antria(Cro), Bioheart, Orgenesis Inc., Virxys corporation , Dendreon Corporation, Tigenix, Georgia Health Sciences University, Neostem Inc, Genesis Biopharma, Brainstorm Cell Therapeutics, Tengion Inc., Fibrocell Science Inc., Opexa Therapeutics Inc, Regeneus Ltd, and Cytori Inc., among others.

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Autologous Stem Cell And Non Stem Cell Based Therapies Market Extracts Market, 2018-2026 by Segmentation Based on Product, Application and Region ...

Right now, your bodys stem cells are working hard replacing your skin every two weeks, creating new red and white blood cells and completing thousands of other tasks essential to life. They are your own personalized fountain of youth.

Scientists generally agree that a stem cell should be able to do both of the following:

One theory of ageing suggests that between the ages of 30 and 50, our stem cells reach a turning point and start to decline in number and function. This results in the typical features associated with ageing.

There does not seem to be a single discoverer of stem cells. Accounts date back to the 1800s and even further, but the first successful medical procedure was a bone marrow transfusion in 1939. Advances in immunology led to donor matching, initially via siblings and close relatives. Unrelated donor matching flourished in the 1970s, alongside donor registries.

In the 1980s, scientists identified embryonic stem cells in mice, leading to the 1997 cloning of Dolly the Sheep. This created immense interest for human and medical applications and a backlash in the US as federal R&D funding was essentially halted in 2001.

In 2012, a Nobel Prize was awarded for the earlier discovery of induced pluripotent stem cells (iPS). Essentially, they return potency and self-renewal properties to mature non-stem cells, essentially making them act like stem cells again.

In the decade between 2010 and 2019, the first wave of stem cell start-ups emerged, alongside R&D programmes at many large pharmaceutical companies, leading to innovation and the first human clinical trials for iPS and other related therapies.

According to Q3 2019 data from the Alliance for Regenerative Medicine, there are 959 regenerative medicine companies worldwide sponsoring 1,052 active clinical trials; 525 of these companies are in North America, 233 in Europe and Israel, and 166 in Asia. In aggregate, $7.4 billion has been invested in regenerative medicine companies in 2019; $5.6 billion of which has been dedicated to gene and gene-modified cell therapy, $3.3 billion in cell therapy, and $114 million in tissue engineering.

Overview of the cancer stem cells market

Perhaps most excitingly, curative therapies are hitting the market and the results are astonishing: 60% of Acute Lymphoblastic Leukemia patients taking Novartis Kymirah showed a complete response (no traces of cancer) and were declared in full remission. Meanwhile, 75% of patients with Transfusion-Dependent -Thalassaemia treated with bluebird bios Zynteglo achieved independence from transfusions. Perhaps most astonishingly, 93% of spinal muscular atrophy patients treated with Novartis Zolgensma were alive without permanent ventilation 24 months after treatment. We should expect more medical breakthroughs in the coming years.

New science, new start-ups: several companies in the sector have gone public or been acquired. These exits led to the recycling of talent and capital into new companies. Because the science and commercial systems have also advanced, the companies in the next wave are pursuing bigger challenges, driving innovation, with even greater resources.

Patients are eager: the current market for stem cell therapies is growing at 36% per year, though it will rapidly expand when a breakthrough occurs toward the treatment of a non-communicable disease (such as cancer, diabetes, heart disease) or a lifestyle factor (for example, growing hair in the correct places, expanding cognitive abilities or increasing healthy lifespan).

New R&D models: funding is flowing into the sector from large companies, VC funds, and institutions such as the California Institute for Regenerative Medicine (CIRM) and New York State Stem Cell Science programme (NYSTEM). Some of the leading university R&D platforms include the Center for the Commercialization of Regenerative Medicine in Toronto, the Stanford Institute for Stem Cell Biology and Regenerative Medicine, the Oxford Stem Cell Institute, and most notably, the Harvard Stem Cell Institute (HSCI).

Founded in 2004, HSCI has established a phenomenal track record. It provided the first $200,000 in funding to Derrick Rossis lab, which inspired the largest biotech IPO to date. HSCI scientists were also co-founders or principals in the three most prominent gene-editing companies (CRISPR Tx, Intellia and Editas), the combined $1.55-billion True North/iPierian acquisitions and the recent $950-million acquisition of Semma Tx, Frequency Tx, Fate Tx, Epizyme Inc., and Magenta Tx.

For the casual investor, Evercore ISI is building a Regenerative Medicine Index, which may be the simplest way to build a portfolio. For institutions and those with deeper pockets, regenerative medicine funds are forming, including the Boston-centric Hexagon Regenerative Medicine Fund, which aims to create companies out of the Harvard Stem Cell Institute.

Caveat emptor. Though patients needs are immediate, those seeking treatments should think very carefully about the risks. There are many dubious clinics touting expensive stem cell treatments and some patients have experienced horrifying complications. Dr. Paul Knoepfler of UC-Davis has written a practical and scientifically accurate guide, a strongly recommended read if you or a family member are considering treatment or a clinical trial.

The leading causes of death in 1900 were mostly infectious/communicable diseases. While the prevalence of most causes has diminished, the largest increases include heart disease (+40%) and cancer (+300%). Granted, this is partly due to doubling life expectancy and a lack of death from other causes. However, given time and resources, scientists and physicians may cure these challenging diseases.

Total disease burden by disease or injury

Today, six of the seven leading causes of death are non-communicable diseases (heart disease, stroke, lung diseases, cancer, Alzheimers disease and diabetes). Based on the early promise mentioned above, regenerative medicine may be our best hope to solve the great non-communicable diseases of our time, and perhaps the single most transformative medical innovation in a century.

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The views expressed in this article are those of the author alone and not the World Economic Forum.

Excerpt from:
Why stem cells could be the medical innovation of the century - World Economic Forum

SAN DIEGO--(BUSINESS WIRE)--Allele Biotechnology and Pharmaceuticals, Inc. (President and CEO: Jiwu Wang, Ph.D., Allele), a San Diego-based private company, and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., Astellas), through its Massachusetts-based subsidiary Astellas Institute for Regenerative Medicine (AIRM), entered into a licensing agreement to expand Astellas access to Alleles induced pluripotent stem cell (iPSC) technologies for various cell therapy programs.

Astellas, one of the largest pharmaceutical companies in Japan and already a leader in the development of cell-based therapeutics, has further dedicated to development of the field through its commitment to state-of-the-art iPS cell generation, modification, and manufacturing. iPSC lines can differentiate into all somatic tissue types, enabling a wide variety of therapeutic applications. The field of iPSC-derived cells has seen dramatic growth in clinical trials recently--the majority of the ~12 clinical trials around the world were initiated within the last 18 months and many more are upcoming.

Allele has been developing its core strength in reprogramming somatic cells into iPSCs with granted patents and the first commercial cGMP system it developed over the past 10 years. Allele also engages in more than a dozen different human tissue derivation activities through its own R&D efforts for internal programs and partnerships. To realize the unparalleled potential of iPSC, Alleles researchers and cGMP team are committed to setting up and validating cell assays for product quality control, genome analysis pipelines, closed-system automation for reprogramming, and machine learning in iPSC-related fields.

Under the terms of the new license agreement, Astellas will pay Allele upfront and milestones, product-based royalties, and potentially manufacture fees.

About AlleleAllele Biotechnology and Pharmaceuticals was founded in 1999. In 2015, the company completed an 18,000 square foot state-of-the-art facility in San Diego for the production of GMP-grade human iPSC lines. The facility also supports the production of tissue-specific cells differentiated from these iPSCs, including pancreatic beta cells, neural progenitor cells, and cardiomyocytes.

Excerpt from:
Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines - Business Wire

Today, Cobb-Vantress appointed Dr Mark Cooper as managing director of genetics to oversee the companys global genetic program. Effective immediately, Dr Cooper will continue work to achieve genetic gains and competitive advantage through alignment of Cobbs breeding program with its product strategy, developing a portfolio of products to meet growing global market needs. He will report to Dr Aldo Rossi, vice president of research and development (R&D).

In his new role, Dr Cooper will lead a global, multifunctional team, including Dr Rachel Hawken, senior director of genetics; Dr Manouchehr Katanbaf, senior geneticist; and Dr Sriram Krishna, senior geneticist. Prior to this appointment, Dr Cooper previously worked as director of product testing. Since joining Cobb, he has also served as pedigree geneticist responsible for male line development, European director of genetics, director of genetics for all of Cobbs breeding programs, and director of product management.

Cobb has been dedicated to genetic research and the responsible use of technology for over 100 years, said Dr Rossi. Dr Cooper has made a big impact in his nearly 20 years with Cobb, and were looking forward to the continued advancements we expect him to accomplish in this new position.

In his time at Cobb, Dr Coopers research has focused on technology development and implementation in the breeding program, welfare parameters and meat quality. He has also spent time with global business leaders and customers to understand and update the R&D team on the product portfolio needed for the future. Most recently, he led Cobbs product testing team, helping to evaluate the companys product performance and development.

Im honored to take on the position of managing director of genetics, said Dr Cooper. Im fortunate because Cobb invests a significant percentage revenue into research and development, allowing us to continue leading the way in genetic progress.

Dr Cooper earned a bachelors degree in poultry science from Texas A&M University, a masters degree in poultry genetics from the University of Georgia, and a PhD in poultry genetics from the University of Arkansas.

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Cobb-Vantress appoints genetics executive - The Poultry Site

Aicardi syndrome is a very rare condition that usually affects girls.

Depending on its severity, it can cause developmental delay, epilepsy, problems with vision, and a shortened life expectancy.

In this article, learn more about Aicardi syndrome, including its risk factors, symptoms, and treatments.

Aicardi syndrome is a rare condition that almost exclusively affects females, with doctors having reported only a few cases in males.

Another name for Aicardi syndrome is agenesis of corpus callosum, or ACC.

Experts think that the condition develops in an embryo during early pregnancy, when there is a change in the DNA of one or more genes.

Aicardi syndrome is not passed down through families. It occurs in people with no family history of the condition.

Doctors usually diagnose Aicardi syndrome in early infancy after the baby experiences seizures called infantile spasms.

Children with Aicardi syndrome may also have developmental delays, learning difficulties, and partial sight or blindness. They may also have a shortened life expectancy.

Aicardi syndrome is very rare, occurring in just 1 in 105,000167,000 babies in the United States. Around the world, there are likely about 4,000 people with the condition. Most of these people are female.

Researchers believe that Aicardi syndrome results from genetic mutations that happen while an embryo is forming. One change may involve the X chromosomes in affected females.

Female embryos have two X chromosomes, while males embryos have just one.

Research indicates that when the characteristic genetic mutations occur in one X chromosome, female embryos can survive because another, healthy X chromosome is present.

If these changes occur in the single X chromosome of a male embryo, it is unlikely to survive. This could explain why babies born with the syndrome are almost exclusively female.

However, very rarely, male babies have been born with Aicardi syndrome. Some boys with the condition have an extra X chromosome.

A mutation in the TEAD1 gene on chromosome 11 may also be responsible for some cases of Aicardi syndrome in boys and girls.

Scientists have yet to prove these theories definitively, and research into the causes of Aicardi syndrome is ongoing.

Infantile spasms are usually the first symptom of Aicardi syndrome. These are seizures that involve single jerks of the whole body.

The spasms often appear before 3 months of age, and they can occur several times a day.

Before a doctor can make a diagnosis of Aicardi syndrome, they need to conduct tests to rule out other possible causes of the symptoms. These alternate causes could include:

Children with Aicardi syndrome usually have some degree of developmental delay and learning difficulties.

Epilepsy is a feature of Aicardi syndrome, and one study found that those with more severe epilepsy had poorer cognitive skills, involving organization and memory.

Some people with Aicardi syndrome have milder symptoms and may not receive a diagnosis until they are adults.

A doctor can detect Aicardi syndrome's changes to the brain with an MRI scan. Some or all of the following features could be present:

People with Aicardi syndrome often have chorioretinal lacunae, which are round, whitish-yellow lesions in the retina the tissue that lines the back of the eye. An ophthalmologist can see these lesions with an ophthalmoscope.

A person with Aicardi syndrome may also have:

Sometimes, these symptoms cause partial-sightedness or blindness.

Also, some people with Aicardi syndrome have distinct facial features and other physical attributes, including:

Other health issues associated with Aicardi syndrome are:

Aicardi syndrome can cause different symptoms in different people, and the treatments also vary.

The aim of treatment is to manage the symptoms, and a doctor will tailor their approach to address each person's situation.

Some treatments focus on easing the severity and frequency of seizures. Others, such as physical, speech, and occupational therapies, can help people with Aicardi syndrome overcome developmental delays and problems relating to vision.

Having a rare disease or being the parent or caregiver of someone with this type of illness can be difficult. A person may feel isolated.

Support groups give people a space to voice their concerns and speak with others who face similar challenges.

The following groups may be useful for people with Aicardi syndrome and their loved ones:

Aicardi syndrome is a rare condition that can cause seizures, vision problems, and other symptoms. It mainly occurs in females.

Most experts think that Aicardi syndrome results from genetic mutation in embryos during very early pregnancy. It is not passed down through families.

As there is no cure for the condition, treatment aims to manage each individual's symptoms.

Read more here:
Aicardi syndrome: Definition, causes, symptoms, and more - Medical News Today

Researchers have uncovered new genetic evidence linked to anxiety in the largest study on the condition that included about 200,000 veterans.

By comparing the participants genomes, the researchers pinpointed six genetic regions related to anxiety and their ties with other psychiatric conditions.

Dr. Joel Gelernter, a senior co-author of the study, said the research provides molecular evidence of shared genetic risk for anxiety and other psychiatric conditions such as depression, which can help identify specific genes that affect risks for such disorders.

To the extent that it identifies genes that were not previously known to be associated biologically with these traits, it will help us understand the biology, and biology can lead to treatment strategies, said Dr. Gelernter, who is a Yale University professor and psychiatrist for the VA Connecticut Healthcare Center. So the ultimate hope is that this study and/or its successor studies will begin to lead us to understand novel biology, which can then lead us to novel treatments that are relevant to anxiety traits.

The study, published last week in the American Journal of Psychiatry, pulled data from the Million Veteran Program, one of the worlds largest biodata banks that includes genetic and medical information from U.S. military veterans.

About 40 million adults in the U.S., or 18%, live with anxiety disorders the most common mental illness, according to the Anxiety and Depression Disorder of America.

However, the studys researchers estimate that anxiety disorders affect 1 in 10 Americans each year.

Dr. *Elspeth Ritchie, a retired military psychiatrist and chief of psychiatry at MedStar Washington Hospital Center, said the differences in numbers could be due to how anxiety is defined, commenting on how post-traumatic stress disorder used to be medically categorized as an anxiety disorder.

It is estimated that only about a third of those with anxiety disorders receive treatment.

Although there are not immediate treatment implications for our findings, they do point us toward future treatments that may involve some of the biochemical pathways and systems identified by our research, said Dr. Murray Stein, a co-author of the study, University of California San Diego professor and staff psychiatrist for the VA San Diego Healthcare System.

He said genome-wide association studies, such as this one, examine millions of markers across the entire genome to see if each marker is more or less common among people with anxiety.

The research team found five genetic variants related to anxiety in European Americans and one in African Americans.

While previous similar studies examined traits among individuals of mostly European descent, this study also included DNA samples from African Americans, who are not always included in large genetic studies, said Dr. Gelernter.

The study discovered the first genome-wide significant findings on anxiety in African ancestry, according to a press release by the Department of Veterans Affairs.

About 18% of the enrollees in the Military Veteran Program, which has more than 800,000 participants, are African American.

One of the variants linked to anxiety the researchers identified occurs in an estrogen receptor. Women are more likely to have anxiety and depression traits, said Dr. Gelernter, but this variant was found in a primarily male study group. He said he would like to eventually test sex differences of this genetic variant.

People with anxiety disorders often experience intense, disproportionate concerns about anticipated events that lead to distress that can interfere with daily activities.

Anxiety is very common, and its also very common for it to be either untreated and undertreated. We do have decent treatments for it, said Dr. Ritchie. I think its important [to treat] because anxiety really gets in peoples way.

She noted there hasnt been much progress so far in the development of new psychiatric treatments based on genetic studies.

Some medications and types of psychotherapies such as cognitive behavioral therapy effectively treat anxiety disorders.

Genetic studies in other fields of medicine have led to precision medicine approaches for treating various diseases. The studys researchers hope more genetic insight into anxiety will lead to the development of tailored treatments for psychiatric patients.

The research team also found that genetic variants tied to anxiety overlapped with other psychiatric conditions such as neuroticism, schizophrenia and insomnia.

(* Correction: An earlier version of the story incorrectly stated Dr. Elspeth Ritchies name. The story has been updated.)

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Researchers uncover genetic links to anxiety in veterans - Washington Times

Learning Objectives

This module will update you on the role of genetics in breast and ovarian cancer, including:

Dr Marc Tischkowitz is a reader and consultant in medical genetics at the University of Cambridge and East Anglian Medical Genetic Service

There are a few key questions that can give an idea of whether family history needs to be explored further:

These four questions should identify the need for a more detailed investigation is required. It is crucial to ask about the paternal side. All the main cancer susceptibility genes can be passed on by either sex but as men rarely get breast cancer, the history can appear more distant on the male side. The cancer pattern can be masked if there are lots of males in a family, so it is important to ask about the male/female balance. If a woman has a paternal grandmother, aunt or cousins with breast or ovarian cancer this should be taken as seriously as a positive maternal history. Ask for this information directly, as it is often not volunteered. If I were only allowed one key message in this article, it would be always to ask about paternal history.

BRCA1/BRCA2variants are 10 times more common in those of Ashkenazi Jewish descent, so it is important to ask about this where appropriate.

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CPD: Key questions on breast and ovarian cancer genetics - Pulse

AUSTIN, Texas Although they have no way of identifying their biological fathers, male chimpanzees form intimate bonds with them, a finding that questions the idea of fatherhood in some of humanitys closest relatives, according to a study of wild chimpanzees in Uganda.

In adulthood, male chimpanzees form strong relationships with one another. These bonds can be mutually beneficial to relieve stress, protect one another and share food.In a new study examining when and with whom these bonds develop, researchers found chimpanzees most often bond with their maternal brothers and old males, including seemingly unbeknownst to the younger chimps their biological fathers.

Fatherhood is really a social relationship that happens to be linked to a genetic relationship. In humans, it is strongly correlated because humans tend to form pair bonds, said Aaron Sandel, assistant professor of anthropology at The University of Texas at Austin. Usually we think that pair bonds evolved in humans first, and then fathers came to play an active role. However, my findings suggest that elements of fatherhood may have arisen in a chimpanzee-like social system before mates formed pair bonds.

Sandel and his team studied the social relationships of 18 adolescent and young adult (12-21 years old) male chimpanzees at Ngogo in Kibale National Park, Uganda, over the course of one year.

The researchers found that by age 12, which is about the time chimpanzees begin to regularly travel independently from their mothers, male chimpanzees form strong friendships with other males in their communities. These relationships range in intimacy, from associating in the same subgroup, where they travel, rest and feed together; to spending time in proximity, a tighter-knit group that stays within about 15 feet of one another; to grooming, where they use their fingers to comb through each others hair.

For humans, you can imagine association, proximity and grooming as if you were at a coffee shop, Sandel said. Youre in association with everyone at the coffee shop. Youre in proximity to others at the same table or one table away. And if you have a private conversation with someone, thats like grooming.

Through observation and fecal sampling, researchers found that the younger males tended to associate and spend the most time in proximity to their maternal brothers. Many bonds were with distantly related or unrelated males. Some bonds were between peers. However, many of their closest relationships were with old, retired males, including their biological father.

Its as if chimpanzees have father figures and some of these are their actual father, Sandel said. This was surprising because chimpanzee females will mate with multiple males while ovulating, and theres no paternal care in a chimps rearing. So, there is no reason to think that she or the males know who the father of her offspring is, and vice versa.

But there are benefits to bonding with an older, somewhat retired male, the researchers noted. Older males are well connected in the community but are no longer competing for a position in the hierarchy. It could be that the chimps biological father was high-ranking in the past, making him more likely to reproduce with females within the same territory and more likely to be groomed by younger chimps looking to climb the social ladder, Sandel suggested.

The researchers proposed that future research should examine the mechanisms of how father-son relationships might develop. But the fact that there is a relationship at all suggests that such bonds can arise without a bond between parents and without the father caring for his offspring as an infant, two factors thought to be important for father-offspring relationships in humans.

This research, co-authored by researchers from Arizona State University and the University of Michigan, was published in the American Journal of Primatology and reviewed and approved by the Uganda National Council for Science and Technology, Uganda Wildlife Authority and the University Committee on Use and Care of Animals at the University of Michigan.

More:
Glimpses of Fatherhood in Non-Pair-Bonding Chimps - UT News | The University of Texas at Austin

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Even if they have no way of identifying them, male chimpanzees form intimate bonds with their fathers, according to a new study of wild chimpanzees in Uganda.

In adulthood, male chimpanzees form strong relationships with one another. These bonds can be mutually beneficialto relieve stress, protect one another, and share food.

In the new study, which examines when and with whom these bonds develop, researchers discovered that chimpanzees most often bond with their maternal brothers and old males, includingseemingly unbeknownst to the younger chimpstheir biological fathers.

Elements of fatherhood may have arisen in a chimpanzee-like social system before mates formed pair bonds.

Fatherhood is really a social relationship that happens to be linked to a genetic relationship. In humans, it is strongly correlated because humans tend to form pair bonds, says Aaron Sandel, assistant professor of anthropology at the University of Texas at Austin.

Usually we think that pair bonds evolved in humans first, and then fathers came to play an active role. However, my findings suggest that elements of fatherhood may have arisen in a chimpanzee-like social system before mates formed pair bonds.

For the study in the American Journal of Primatology, researchers studied the social relationships of 18 adolescent and young adult (12-21 years old) male chimpanzees at Ngogo in Kibale National Park, Uganda, over the course of one year.

The researchers found that by age 12, about the time chimpanzees begin to regularly travel independently from their mothers, male chimpanzees form strong friendships with other males in their communities.

These relationships range in intimacy, from associating in the same subgroup, where they travel, rest, and feed together; to spending time in proximity, a tighter-knit group that stays within about 15 feet of one another; to grooming, where they use their fingers to comb through each others hair.

For humans, you can imagine association, proximity, and grooming as if you were at a coffee shop, Sandel says. Youre in association with everyone at the coffee shop. Youre in proximity to others at the same table or one table away. And if you have a private conversation with someone, thats like grooming.

Through observation and fecal sampling, researchers found that the younger males tended to associate and spend the most time in proximity to their maternal brothers. They shared many bonds with distantly related or unrelated males. Some bonds existed between peers. However, many of their closest relationships were with old, retired males, including their biological father.

Its as if chimpanzees have father figures and some of these are their actual father, Sandel says. This was surprising because chimpanzee females will mate with multiple males while ovulating, and theres no paternal care in a chimps rearing. So, there is no reason to think that she or the males know who the father of her offspring is, and vice versa.

There are benefits to bonding with an older, somewhat retired male, the researchers say. Older males are well connected in the community but are no longer competing for a position in the hierarchy.

It could be that the chimps biological father was high-ranking in the past, making him more likely to reproduce with females within the same territory and more likely to receive grooming from younger chimps looking to climb the social ladder, Sandel suggests.

The researchers propose that future research should examine the mechanisms of how father-son relationships might develop. But the fact that there is a relationship at all suggests that such bonds arise without a bond between parents and without the father caring for his offspring as an infant, two factors thought to be important for father-offspring relationships in humans.

Additional coauthors are from the University of Michigan and Arizona State University.

Source: University of Texas at Austin

Read more:
Chimp sons like to hang with fathers and brothers - Futurity: Research News

LA SALLE On Tuesday morning, the barn at Coyote Ridge Ranch in Weld County served as the bovine equivalent of a hair salon.

Some of the ranchs top Hereford cattle were brought in for a bath and blow-dry. Outside, workers gave the rust and white-colored animals a final clipper trim, preparations for their impending closeups.

The National Western Stock Show is back for its 114th year in Denver and Coyote Ridge Ranch Herefords are right in the thick of it, as theyve been for three decades.

The Cornelius family, lead by Jane Evans and her son Hampton, founded the ranch in Boulder County, but for the past 25 years, its operated on a 1,000-acre spread south of La Salle. The barn/cattle hair salon there is 130 years old and may be the oldest structure in the Beebe Draw valley, according to the family.

Over the decades, the Corneliuses have established a reputation as one of the countrys if not the worlds preeminent producers of top quality Herefords and Hereford genetics. And the stock show is their biggest marketing opportunity of the year.

Denver is like a trade show for us. Were not there to win a ribbon. Its cool when you win one but were there to promote our genetics and make contacts, Jane said of the National Western show, where Coyote Ridge will be showing a pen of three heifers and a pen of three bulls this year.

Were showing our spring-born 2019 cattle, Hampton said. Everything is for sale. The idea is to drum up interest to get people to come back here and take a look at our other ones.

The business-first approach doesnt mean they arent proud of how Coyote Ridge Ranch has performed at National Western. Hampton rattles off the stack of honors the operation has come away with in years past. They include three grand champion pens, two bulls that won individual championships in competitions on the hill at National Western and countless individual class champions.

Its a validation of what youre doing, Jane said.

Coyote Ridge Ranch is what is known in the cattle business as a seedstock producer. The means its herd of 160 or so Herefords is being raised to further the genetics of the breed. It sells bulls, heifers, semen and embryos to commercial Hereford ranching operations that in turn produce steers for slaughter and sale to consumers.

The ranch dates back to when Hampton and his sisters Katie and Coleman, a former Denver Post staff writer, were kids raising cows and calves as part of 4-H and Future Farmers of America programs. The hobby blossomed into a passion and the family herd grew large enough to become a viable business.

The Corneliuses were drawn to Herefords because of the breeds disposition, its hardiness and the animals deep connection to ranching culture in the American West. Nowadays, Coyote Ridge is considered an elite Hereford seedstock producer with genetics from their animals spread across ranching operations in the U.S. and all over the world.

I would describe that family as just being committed to making really good cattle and breeding Herford cattle the way they need to be bred for the commercial industry, said Jack Ward, executive vice president of the American Hereford Association.

Ward and many other staffers from his association are in Denver this week for the stock show. Among the events Ward is organizing is the national Hereford junior heifer show on Wednesday morning and the Mile High Night Hereford Sale, which is expected to bring more than 1,000 people to the National Western Stadium Arena at 6:30 p.m. Friday, Ward said. Coyote Ridge will be represented at both events, of course, with Hampton Cornelius son, John, showing a heifer in the junior show.

Beyond their work furthering the Hereford breed, the Corneliuses have become ambassadors for the cattle industry in Colorado. With a ranch thats within an hours drive from downtown Denver and a willingness to open their operation to visitors, theyve hosted school groups, chefs, and delegations from countries including China, Japan and South Korea.

(Hamptons wife) Kay and Jane Evans are both so very well-spoken on so many segments of their industry, Colorado Beef Council marketing director Tami Arnold said. Just the typical consumer, we know we could take them out to their place and they would be able to really represent the beef industry well.

For Jane Evans, the most exciting visit came last summer when a Taiwanese trade delegation stopped at Coyote Ridge Ranch. It wasnt just because the visitors were so impressed by seeing a cattle operation where riders on horseback drive a herd across a pasture. She was pleased because the delegates were in Colorado to sign a letter of intent with Gov. Jared Polis to expand access in Taiwan for Colorado agricultural goods including beef.

It gets its moment in the sun in Denver every January during the National Western, but Jane is quick to point out the livestock industry is a major force in Colorados economy. Cattle operations alone generated $3.4 billion in cash receipts in the state last year, according to the University of Colorados 2020 business economic outlook.

Jane Evans, 78, has established a reputation of her own over the years. Cattle ranching has been a male-dominated industry, but she hasnt shied away from being at the center of it. In the mid-1990s, she became the first woman elected to the American Hereford Association board of directors.

She paved the way for women in the beef industry, specifically for leadership and we love her for that, said Arnold, who in addition to working with the Cornelius family through the Colorado Beef Council also ranches nearby and has known them most of her life.

Janes love for agriculture goes back to her childhood in Alabama, when her grandfather would let her tag along when he would assess farms and ranches as part of his work as a banker.

I was very lucky. In those days girls did one thing and boys did another, she said. My mother used to say, When Jane Evans grows up shes going to own a large cattle ranch in the West like Dale Evans, and I do. (Dale Evans was married to singing cowboy Roy Rogers and they had a popular TV show in the 1950s.)

The next week will be a busy one for the Cornelius family. But now that theyve settled into their pen in the National Western Centers evolving stock yards, they do expect to have a little fun.

These guys work alone a lot. When they get together they definitely will kick up their heels a little bit, she said of her family and her fellow ranchers at the stock show. You see people that you have a lot in common with, that you work with, that you swap genetics with. Even though there is stiff competition, there is an awful lot of camaraderie.

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Frog and lizard scientists add their voices to the call for climate change action to save species from extinction

By 2085 climate change could mean life is a 60-year long boys weekend for tuatara. Temperature can dictate whether tuatara eggs hatch as female or male. In a warming world maleswill briefly emerge from eggs as the lonely, functionally extinct winners.

It would be an ignominious end for a species whose roots go back to the dinosaur age.

Last week, almost a thousand attendees from around the globe descended on Dunedin for the 9th World Congress of Herpetology. Held every four years, the event is like the Olympics for those who study reptiles and amphibians.

The 600 pluspresentations included debate on the fragile state of the worlds amphibians and reptiles. Around 40 percent of amphibians are threatened with extinction. Reptile species are under similar pressure.

University of Otago Zoology Professor Phil Bishop, the congress director, said a recurring thread in presentations was the threat of climate change.

It is highly likely that we will observe many species going extinct in front of our very own eyes in the next decade.

New Zealand used to have seven frog species. Three are now extinct and the remaining four are threatened with, or at risk of, extinction.

Climate change is one of those topics where people generally throw their arms up and say theres nothing we can do but weve had enough of not doing anything. We really need to be pushing the people who can do something about it.

The congress released a declaration calling for all governments to acknowledge the accumulated evidence of global climate change and take immediate action to mitigate future impact. This includes increasing protection for biodiversity and wild places.

As a group, the congress also made a commitmentfor itsevents to become 100 percent carbon neutral in future and serve locally sourced food with limited meat and dairy.

Climate change adds pressure to the numerous issues facing amphibians and reptiles.

Bishop said the biggest issue facing amphibians worldwide wasloss of habitat.

Whether its habitat destruction, or habitat alteration by climate change, or habitat fragmentation, thats the main issue.

Frogs are cold-blooded and cant regulate their body temperature themselves, relying on theenvironment around them. There are fears the pace of warming temperatures might prove too much.

If we could just stop messing around with their habitat, a lot of amphibians would be safe.

The fires in Australia highlight another concern. With habitat burnt, any survivors of speciesare easy pickings for predators such as feral cats. Studies show they will travel some distance to hunt in sites which have been recently burnt. For those which escape the flames and predators and manage to establish a new population there's the issue of in-breeding.

Previous studies of Australian frog life after bushfire revealed genetic diversity within the population had decreased. With just a small number of adults surviving the fires, populations had become inbred meaning they were likely to bemore vulnerable to future threats.

Disease is a threat which has become a major concern.

Theyre getting a number of infectious diseases. I liken this to the thousand straws which break the camels back. With a whole number of stressors, amphibiansbeing subjected to emerging diseases, [is] the last nail in the coffin, just knocking them down so they become extinct.

One disease, chytrid fungus is well known. However, presentations at the conference highlighted another virus which may prove to be more of a danger than first thought.

Ranavirus: The new killer on the block

Skin ulcers, limbs dropping off, emaciation and haemorrhaging are all symptoms of ranavirus.

Once caught, theres no cure and the result is often fatal.

The diseaseaffects amphibians as well as reptiles and fish and has been found in the United Kingdom, United States and parts of Asia and Australia.

Research in the UK shows warming temperatures caused by climate change haveincreased the spread and severity of the virus. When the weather warms to 16C, outbreaks increase.

So far, ranavirus hasnt been found in New Zealand frogs. With an active exotic fish pet trade Bishop expects its just a matter of time before its found here.

With no cure available the hope seems to be for species to eventually develop immunity to the virus.

The suffocating fungus

One of the well known issues facing amphibians is the chytrid fungus. This affects how the creatures breathe through their skin and effectively suffocates them.

Referred to as an amphibian apocalypse the fungus is estimated to have caused the extinction of around 90 amphibian species as well as be responsible for a decline in numbers of around 500 different species.

Bishop said a strain of the fungus hit New Zealand in the mid 1990s to early 2000s. Hardest-hit were introduced species and the native Archeys frog.

Studies since then have shown our native frogs have an immunity to the strain present here. Frogs exposed to the pathogen show no signs of disease. Within 10 weeks the frogs are pathogen-free.

If we could close all our borders to make sure no other disease got into New Zealand, were safe from a chytrid fungus point of view because all our frogs seem to be able to cure themselves.

The concern is the arrival of a different strain of the fungus. The strain which is causing the most problems worldwide isnt here yet and Bishop worries every time he hears of any accidental frog or cane toad incursion.

Climate change is likely to alter where the fungus is found. Preferring cool, wet environments, its likely to move up mountains atthe same time amphibians beat an upward escape from warming temperatures.

"There seems to be a lot of emerging infectious diseases which were tolerated by amphibian populations that seem to not be tolerated anymore."

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When it comes to care and services for people with neurodevelopmental disabilities like fragile X, there is a long way to go. Protics published study found that medical professionals in Serbia know very little about fragile X, a problem she hopes to remedy through medical conferences and the countrys newly launched Fragile X Society the only one for the entire Balkan region. With funding support from Sacramentos Serbian immigrant community, Protic already has set up the countrys first fragile X testing site.

The X in fragile X syndrome refers to the X chromosome, where the mutated gene that causes the disorder is located. That gene, FMR1, is involved in making a protein important for the development of critical connections between nerve cells. When the cells cant make the protein, the nervous system can malfunction, leading to the signs and symptoms of fragile X syndrome. Fragile X affects one in 2,500 to 4,000 males and one in 7,000 to 8,000 females.

What the disorder looks like in children varies, but males are more severely affected. They are more likely to have intellectual disabilities and distinguishing physical characteristics like long, narrow faces, big ears, prominent jaw and forehead, unusually flexible fingers and flat feet. Affected individuals also frequently have speech and language delays, behavior challenges, ADHD and anxiety.

An estimated 50% of children with fragile X also have autism, and its the most common single-gene cause of autism, accounting for about up to 6% of cases. And while children with fragile X and autism may share many characteristics, children with fragile X can be more interested in other people, make friends more easily and do better in social settings, Randi Hagerman said.

Ive known many boys with fragile X who have been voted Homecoming King, she told the conference audience one of many hopeful anecdotes she shared during her talk.

As theyve been doing increasingly over the past several years, MIND Institute experts brought a treasure trove of information to the people gathered in Belgrade to advance the regions understanding of fragile X and help clinicians and others begin to address the needs of the affected population.

MIND Institute Director Leonard Abbeduto, a psychologist, whose work includes using digital communication technologies to help parents support their childrens language development, said the trip to Belgrade exemplifies the evolution of the MIND Institute, founded in 1998 to find and develop treatments for neurodevelopmental disabilities. The institutes International Training Program in Neurodevelopmental Disorders, which invites health care professionals and scientists from around the world to train at the MIND Institute, has had 24 participants from China, Philippines, Thailand, Turkey, Singapore, Spain, Ecuador, Colombia, Chile, Japan, Israel, South Korea and Serbia, including Protic. Many of them have taken their knowledge home to create their own programs.

The MIND is a research, treatment and training center created out of the efforts of families who wanted to make life better for their children and other families, Abbeduto said. We are really trying to build on what weve done to enhance our impact locally, nationally and internationally, which is why I was motivated to make the trip to Belgrade.

Joining colleagues from Johns Hopkins, Rush, Emory and other universities, MIND Institute speakers elaborated on the latest in clinical diagnosis and assessments for children with fragile X, family-focused language interventions, the latest in scientific discovery and DNA testing, new targeted treatments currently in use and others on the horizon, as well as the importance in involving patients and families in advocacy efforts.

I find that we have so much knowledge at the MIND Institute, I really want to share it, said Randi Hagerman. Sometimes, in many other countries the doctors have never even heard of fragile X syndrome.

Maija and Cristian Sukreski traveled from Croatia to the meeting in Belgrade in search of advice to help their 3-year-old son, Petar, diagnosed a year ago with fragile X. The tiny, blond Petar is rambunctious, nonverbal and increasingly aggressive, hitting his parents when they attempt to calm him. Its a trait his mother worries could become dangerous as she tearfully ponders the future.

Their trip, Maija Sukreski said, was to find help.

He is going to therapy now, and I hope he can receive other therapy that will be more effective to slow down the behavior problems and help him talk, she said.

In a small and drab clinic exam room, the couple awaited a chat with David Hessl, a MIND Institute psychologist and researcher known internationally for his work to refine psychological assessments for children with fragile X syndrome and other neurodevelopmental disorders. Petar stood, biting his hands then flapping them, a common autism characteristic referred to as stimming short for self-stimulating. They told Hessl about the boys worrisome behaviors.

The aggression is a form of communication, Hessl told the parents. Hes not angry with you. Hes just overwhelmed.

Hessl suggested that instead of punishing Petar for hitting, they should reinforce good behavior with rewards of things he wants, like bananas, or special toys.

When he hits, dont be emotional or reactive. Stay really neutral. Turn away, he said, adding: Put a lot of time in this now and you will save yourself a lot of problems later. When his speech comes, he will probably be less aggressive.

In another exam room, Hagerman and Protic were talking to the Cvijetics while Demetrije chewed his knuckles, played with his fathers iPhone and made frequent whimpering sounds.

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The first stranded orca whale in England and Wales for nearly two decades has been discovered.

The 15ft-long juvenile male died after becoming stranded in the salt marshes of The Wash, the bay and estuary where Norfolk and Lincolnshire meet.

Experts are investigating the incident - the first confirmed stranding the Cetacean Strandings Investigation Programme has had in England and Wales since 2001.

Orcas are a priority species for research by the Zoological Society of London (ZSL), which is a partner of the programme, as they are top predators that can absorb significant concentrations of marine pollutants such as chemicals known as PCBs which accumulate as they go up the food chain.

Blubber, liver, muscle and kidney samples were collected from the whale by ZSL's Rob Deauville and Matt Perkins.

Most of the marine mammal's internal organs were intact despite having died a week ago, meaning its skin has started decomposing.

The investigators will analyse samples for marine contaminants and use genetic analysis to determine which population the whale came from.

Its teeth have also been collected to accurately assess his age.

Experts said there was no evidence of recent feeding as its stomachs were largely empty.

The team found a large fragment of plastic in the first stomach but it had not killed the orca as the stomach was not blocked.

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First stranded orca in nearly 20 years discovered on England's east coast - Sky News

Ken Baker, Columnist Published 12:27 p.m. ET Jan. 14, 2020

Ken Baker and Cocoa(Photo: Submitted)

According to the Chinese Calendar, the 13 months from Jan.25, 2020, through Feb.21, 2021 will be the Year of the Ratthe Gold Rat.

Each of the 12 Chinese Zodiac signs (rat, ox, tiger, rabbit, dragon, snake, horse, goat, monkey, rooster, dog and pig) is associated with one of five elements: water, wood, fire, earth or metal (gold). So all told, it takes 60 years (12 x 5) to cycle through all possible combinations.

A person born under the sign of the Rat is purported to be quick-witted and resourceful, with a rich imagination though, perhaps, a bit shy in courage. Since Chinese culture attributes diligence and thriftiness to the rat, its expected those born in a Rat year will do pretty well for themselves.

Or so its said.

Setting aside such cultural personifications along with any aversion we might harbor towards the animal, what have we learned about the rats biology and the way it actually lives its life?

In our area and indeed throughout North America and Europe, the species youd be most likely to encounter today is the brown or Norway rat (Rattus norvegicus). But thats only been the case over the last several centuries.

The black rat (Rattus rattus), also known as the roof or ship rat, is thought to have invaded Europe from Southeast Asia sometime between the 4th and 2nd century BC and North America in the 16th century. The somewhat larger and more aggressive Norway rat appears to have reached Europe as stowaways on trading ships from Northern China during the 1500s and North American shores some 200 years later.

In cooler northern climates, the Norway has largely replaced the once abundant black rat, which is still the more common species in tropical areas.

The Chinese Zodiac calendar celebrates 2020 as the Year of the Rat, which gives recognition to this Norway rat.(Photo: Submitted)

It should be noted that there are many other species of larger-than-a-mouse rodents commonly referred to as rats. The genus Rattus alone has over 50 such species and there are interesting beasts called rats in several unrelated genera (Neotoma, Dipodomys and Bandicota, for example).

But the black and Norway are the two species that have played the largest role in human history. In fact to ask about the natural habitat of either mammal poses an interesting challenge. Both have been so closely associated with human habitations for so long that the best description of their normal habitat in nature might simply be wherever people live.

Which is not to say their biology is any less complex and interesting. Female Norway rats, for example, commonly live in colonies of six or so related individuals. Each female will have her own nest chamber within a shared (often underground) burrow. Intriguingly, members of the group will often nurse their young collectively.

While daughters commonly remain with the colony, males disperse soon after being weaned. If the population of rats in the area is relatively low, one adult male will typically dominate the colony, vigorously defending it against other males and mating with its females.

In dense populations, however, there will be too many intruders for him to maintain exclusive control of the colony and he will have to suffer the presence of other males seeking to mate with females when they come into breeding condition (about once every 4 -5 days if not impregnated).

In the wild (that is excluding rats kept as pets or in a scientific laboratory), the average lifespan of a Norway rat is probably less than one year. Studies of several European populations found about 95 percentannual mortality, with just a (very) few venerable old-timers making it to three years.

The rats perception of the world (its mvelt in the language of behavioral biologists) is very different from our own. Their eyesight is quite weak beyond a foot away, they can only detect large shapes and movement and, like most other mammals, they cannot detect the color red.

But this doesnt make them less effective in navigating their environment. Norway rats, which are primarily active at night, live in a world of textures, sounds and smells. When moving about, the Norways long whiskers whisk back and forth several dozen times per second, lightly touching all nearby objects.

They can hear (and communicate with) sounds much higher in pitch than we can detect, and its been estimated that over 1 percentof their genetic material is devoted to the detection of odors.

Finally a word on the connection between rats and the Bubonic Plague that swept through the Eastern Hemisphere in the mid-1300s, killing 25 to 60 percentof the human population of Europe. The so-called Black Death, caused by the bite of a flea carrying the Yersinia pestis bacterium, has long been blamed on the spread of flea-infested black rats.

However more recent studies have strongly suggested Yersinias initial invasion of Europe might be better pinned on gerbils, of all things, which unlike rats can carry the bacteria in their blood for some time without killing them.

Oh, and regardless of its name, the Norway rat has no special association with Scandinavia.

Ken Baker is a retired professor of biology and environmental studies. If you have a natural history topic you would like Dr. Baker to consider for an upcoming column, please email your idea to fre-newsdesk@gannett.com.

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An announcement from our friends at White River Growpro.

White River Growpro, a specialty gardening store located in downtown White River Junction, is currently running a cannabis pheno hunt and is giving away free cannabis seeds to anyone who would like to participate in the contest.

This is not a traditional cannabis cup in which the biggest and best buds win, its a challenge looking for a particular phenotype amongst many.

This contest, which was announced last spring and has been extended through July 1st, 2020, has already given out over 2500 free seeds and still has several thousand seeds available for those whod like to play. They are specifically searching for an old cannabis strain: East Coast Sour Diesel.

We have always felt that the original cut of East Coast Sour Diesel (ECSD) is one of the finest cannabis cultivars.

Here at Growpro, explains Growpro co-owner and cannabis breeder Kendall Smith, we have always felt that the original cut of East Coast Sour Diesel (ECSD) is one of the finest cannabis cultivars. With wide ranging appeal, this uplifting variety has smells of sour lemon skunk and earth. The problem is that the original is clone only and its become finicky to grow. Were trying to change that.

Ive been working on a breeding project with a 30-year-old cut of ECSD that is currently in the F2 stage, says Smith, meaning there will be a wide genetic expression throughout the seeds. Somewhere in these seeds will be a pheno that is as close to the original East Coast Sour Diesel as we can expect to find.

This crowd sourced breeding project is a creative way to take advantage of current law and share cannabis genetics with the Vermont community. Co-owner Stephanie Waterman says this project was inspired by a culture of sharing genetics amongst friends.

Vermont law only allows for 2 plants in flower, which means pheno hunting can take years and years.

Under current Vermont law, says Waterman, there is not a great way to obtain seeds other than through gifting, and we are limited by our plant counts. Vermont law only allows for 2 plants in flower, which means pheno hunting can take years and years. Thats why were enlisting an army of growers to help us find the pheno, injecting thousands of free seeds into the Vermont growing community, and giving out thousands of dollars in prizes to the winners! Its all in good fun.

Anyone whod like to participate can stop into White River Growpro and request a packet. Each pack contains 13 regular cannabis seeds, meaning they will produce both males and females.

Growers have until 7/1/2020 to submit a quarter ounce of finished flower for the judges.

If your flower is selected as a finalist, you will be asked to submit a clone of that plant that the judges will grow out to confirm the genetic is on point. The winner will be announced in December of 2020.

This contest is about having fun and sharing cannabis genetics with the greater Vermont community.

Anyone who completes the contest and submits finished flower will be able to get a cutting of the winning phenotype. In the end, says Waterman, this contest is about having fun and sharing cannabis genetics with the greater Vermont community. Weve seen some amazing phenotypes coming out of this contest and I love seeing the great work the growers are doing adds Kendall.

For more information about the contest, prizes, and the breeding project, please visit the White River Growpro website and click on Events & Classes.

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White River Growpro's "Hunt for the Pheno Contest" Gives Away Free Cannabis Seeds | Cannabis Events, Culture and News - Heady Vermont

Taylor Tran (left) and her mother Mai Nguyen. Taylor underwent cancer treatment when she was 2 years old, causing her to go into early menopause when she was just 16.

You pay the price for having cancer over and over again.

Mai Nguyens words are loaded with sorrow as she speaks about her 17-year-old daughter, Taylor Tran, who is dealing with fertility concerns more than a decade after she survived late-stage cancer.

Its easy to understand the exasperation Nguyen feels: Her daughter was diagnosed with stage 3 single-cell sarcoma of the kidney when she was 2 years old and was treated with intense chemotherapy and radiation. Now, the treatments that saved her life have put her into early menopause.

Its been traumatic, Nguyen said. Weve tried so hard to allow Taylor to have a normal childhood and this feels like one more thing cancer has taken from her.

Stories like Taylors inspired Seattle Childrens urologist Dr. Margarett Shnorhavorian to tackle a challenging area of research that was largely uncharted more than a decade ago. Since then, shes helped change perspectives and protocols for fertility preservation in childhood cancer survivors.

Stories like Taylors inspired Seattle Childrens urologist Dr. Margarett Shnorhavorian to tackle a challenging area of research that was largely uncharted when she started more than a decade ago.

Shnorhavorians passion stems from the encounters shes had with the families of childhood cancer patients since starting her practice 12 years ago. She often spoke with families whod just received a devastating diagnosis about the importance of fertility preservation.

One thought was that families wouldnt want to think about fertility when they were trying to deal with the chaos of cancer, Shnorhavorian said. But when I brought it up, I saw the tone in the room lift. They were grateful we were talking about their life after cancer, because it meant we believed they would have one. It was like glimpsing at the end of a rainbow.

At the time, there was little information providers could offer about how patients fertility might be affected by various cancer treatments. Families couldnt find the answers they desperately wanted.

Initially, I thought if we brought up sperm banking and an adolescent didnt want to talk about it, we should just end the conversation there, Shnorhavorian said.

That was until a study led by Shnorhavorian changed her mind. The study surveyed over 400 adolescent and young adult cancer patients finding that a significant portion had never discussed their fertility preservation options with their care team before starting treatment.

I learned not to take no for an answer when a young patient didnt want to think about it. We have an obligation to explore the subject and remind them that, even if they are not thinking about their future fertility, we are thinking about it for them, she said.

As Shnorhavorian set out to establish her research program, she was keenly aware of the hurdles shed need to overcome to study fertility in a pediatric population.

First, recruiting enough participants to study the effects of cancer treatment on fertility would be difficult. Childhood cancer is relatively rare, so there were fewer eligible patients to draw from.

Also, many providers were uncomfortable discussing fertility issues with patients and their families who were, understandably, focused on survival. If adolescent and young adult participants did enroll, it would be difficult keep track of them because they change addresses frequently.

Shnorhavorian also had to make study participants comfortable contributing specimens such as sperm samples.

The adolescent and young adult population is unique, so we developed research methods tailored to their needs, she said.

They created a data collection system where patients could participate wherever they were located. They could have blood drawn in their dorm room or collect their sperm sample at home and send it in the mail, rather than going to a clinic.

That was a game-changer in our field, Shnorhavorian said.

Today, thanks to her innovative research methods, Shnorhavorian is leading a multi-site study to investigate the effects of chemotherapy on boys and men who have survived osteosarcoma, a common type of bone cancer.

When I started, there was limited research on male fertility, mainly because everyone had been lulled into a false sense of security thinking boys can sperm bank. But that is not an option for pre-pubertal males, she said.

According to Shnorhavorian, there are still no options for young boys to preserve their fertility.

I chose osteosarcoma because it was a population of men who would not have fertility impairments due to other therapies, like radiation, or their diagnosis. We hope the lessons we learn studying this disease can be applied to other cancers.

Patients are being recruited for the study from 178 Childrens Oncology Group institutions in the U.S., Canada and Australia.

Shnorhavorian and her teammates in Seattle Childrens Center for Clinical and Translational Research are hoping to identify biomarkers of fertility risks and genetic susceptibility to fertility impairments and better understand of sperm development and how cancer therapies modify sperm DNA.

By studying these predicting factors, Shnorhavorian hopes to shed light on why some cancer survivors become infertile after treatment, eventually, leading to preventative interventions.

When Taylor learned she was going into early menopause, she decided to freeze her eggs. My cancer treatment gave me a second chance at life, but that doesnt mean I shouldnt get to have the same experiences others have.

When Taylor learned she was going into early menopause in February 2019, she decided to freeze her eggs. She feels grateful for the opportunity even though several aspects of the fertility treatment were difficult and she often felt isolated.

My cancer treatment gave me a second chance at life, but that doesnt mean I shouldnt get to have the same experiences others have, Taylor said.

Shnorhavorian hopes her research will continue to grow so providers can, one day, offer cancer patients new opportunities to preserve their fertility before treatment or treat their cancer without harming their reproductive health.

It is our obligation to give our patients hope, Shnorhavorian said. We have a long way to go to offer fertility preservation to every child, boy or girl, but from my standpoint, its no longer a question of Should we do this? but rather How do we do it?

Nguyen finds the idea exciting.

I want to see other kids, like Taylor, have the opportunity to raise their own genetic children, she said. It would be amazing if researchers can find a way to cure them without taking away that experience.

To learn more about the clinical trial Dr. Shnorhavorian is leading for osteosarcoma survivors, please visit the study page on our website. For more information on clinical trials at Seattle Childrens, please visit our current research studies page.

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From our Obsession

New thinking is required to serve an aging population.

Scientists know a lot about the hallmarks of different types of dementia. Alzheimers disease is characterized by buildups of amyloid and tau proteins. Vascular dementia is the result of gnarled and broken blood vessels that normally supply oxygen to the brain. Parkinsons disease and other Lewy Body dementias are caused by misshapen alpha-synuclein proteins in the brain.

Conventional wisdom has it that each of these dementias needs its own treatment. An anti-amyloid drug probably wouldnt work for someone who doesnt have amyloid buildups in their brain.

But to this day, there are no definitive treatmentsor preventive measuresfor any of the dozens of dementias out there. Which has led some researchers to take a more systematic approach: What if there were a single mechanism in the brain that, when faulty, leads to all kinds of dementias? And what if this mechanism, like a switch, could be flipped off?

Thats the thinking of Michael Fossel, the founder of the Michigan-based biotech startup Telocyte, which is developing treatments for Alzheimers. Today (Jan. 14), Fossel published a review articlepostulating that Alzheimers and other dementias are caused by a failing of a workhorse class of brain cells called glia. He also proposes that he and his colleagues at Telocyte, founded in 2015, have a solution: a gene therapy that could target these cells to keep dementia at bay.

The paper is theoreticalits a review, so its not presenting any original data. Its a new way of thinking, and a bold proposition. Its encouraging to see individuals like Dr. Fossel pulling together research and trying to come up with new theories, says Rebecca Edelmayer, the director of scientific engagement at the Alzheimers Association. The Alzheimers Association is a nonprofit and publisher of Alzheimers and Dementia, the journal in which Fossels review article appeared.

But while theories are important, Edelmayer says, they also need to be tested. Gene therapies are still relatively new. And theres reason to wonder about the safety of the gene the therapy would introduce: one that codes for the enzyme telomerase. Before scientists can even begin to test Fossels systematic theory of dementia, theyll need a lot of data demonstrating its safety.

Telomerase has been a focus of longevity research for years. Its an enzyme that lengthens telomeres, which are the genetic caps on the end of our chromosomes. Every time cells divide, telomeres shortenand when telomeres have been sufficiently shaved away, cells enter a state called senescence and stop dividing. Then, they self-destruct.

Shorter telomeres have been correlated with a whole host of age-related health issues: cancer, diabetes, and even forms of dementia. But its not the telomeres themselves that cause these issues, Fossel suggests. As my telomeres shorten, there are a lot of other things going on, too, he says.

The relative length of telomeres, we know, sends a signal to the rest of the cells DNA. As telomeres shorten with cell replication, cells change the way they carry out other genetic instructions, which can result in shoddy protein production. Its a process called the telomere positron effect (paywall), and scientists still dont fully understand it.

Fossel posits that when telomeres shrink in microglial cells, part of the brains immune system, other critical parts of their DNA degrade, tooand that genetic damage can result in many different dementias.

Telocytes gene therapy would aim to rebuild those glial telomeres. That would involve sending an active copy of the telomerase gene, TERT, into the cerebrospinal fluid, carried by a virus. The virus, which should be otherwise benign, isnt great at getting genetic material into specific cells: in mouse models, about 5% of the total therapy winds up in neurons, to no lasting effect, and about 1% winds up in microglial cells, Fossel says. But even with the TERT gene just floating around in the glial cell for a few weeks or months, it might be enough for telomerase to lengthen those end caps and trick the cell into expressing genes like it did in its younger days.

Usually, gene therapies work by introducing new genetic material that replaces a persons faulty or missing genetic code. Telocytes gene therapy, however, wouldnt be replacing a gene: Itd just be giving glial cells another copy of one they already have. All of our cells have the TERT gene embedded in their chromosomes. But the vast majority of cells (save for red blood cells, sperm or egg cells, and cells along parts of the digestive tract) have the gene switched permanently off.

Thats for good reason: Telomerase is active in most forms of cancer. Which is why many scientists fear that inserting a gene that codes for telomeraselike Telocytes gene therapyrisks causing cancer

My main concern is its safety, says Jue Lin, molecular biologist at the University of California San Francisco whose work focuses on studying telomere length and stress levels over time. We dont know whether the over-expression of telomerase will increase the risk of cancer. In the brain, particularly in the glial cells that Fossels proposed gene therapy would target, the cancer in question would likely be glioblastoma, a ravenously growing brain tumor.

Mouse models using telomerase gene therapy in the brain have been promising, with no notable incidence of cancerbut those experiments are imperfect. Mice express telomerase differently than humans do, Lin explains: They have a lot more telomerase, in more tissues than humans. Mice also dont live as long as we do, and cancer takes a long time to develop, Lin says.

And gene therapies carry the risk of a dangerous immune reaction to the virus carrying the therapeutic gene. The viruses used in gene therapy todayand the one Fossel proposes usingshould be safer than the ones used in the early days of gene therapy. Adeno-associated virus, or AAV for short, should elicit only the tiniest of immune responses. But scientists have recently voiced concerns about the long-term safety of gene therapies using AAV.

Given the risks, theres disagreement over whether the telomerase approach is worth pursuing. Its important and interesting to have an additional hypothesis, says Diego Forero, a researcher at the School of Health Sciences at the Fundacin Universitaria del rea Andina in Colombia. His work, which is independent of Fossels, focuses on exposing astrocytes, a type of glial cell in the brain, to telomerase, to see how theyll react. Hes found that telomerase is involved in other cellular functions, like a cells metabolism. In his opinion, its too early to say that Fossels theory should be tested.

Rather than focusing on the potential therapeutic application of telomerase in brain cells, Forero is interested in more basic, exploratory research. He thinks that applying it to a specific targetlike a cure for dementiawouldnt tell scientists enough about all the ways telomerase could affect brain cells.

Those calls for prudence can be frustrating for dementia patients facing a dearth of options. Even with no immediate plans to conduct clinical trials, Fossel says he has already had some 200 people with mild to moderate dementia reach out to him as willing participants. Theyre ineligible for most other clinical trials for dementia therapies, which tend to seek out participants who have risk factors of the disease but minimal symptomsor none at all.

People have faced terrible disease and said Im going to take my chances, says Arthur Caplan, a bioethicist at New York Universitys Langone Medical Center. With vulnerable populations desperate for treatment, peer review from independent scientists becomes even more important. Its critical that the data and research are conducted by parties that dont have a vested financial interest in a certain outcome.

These studies also need to have strong institutional review boards, Caplan says. These boards are required any time researchers are conducting experiments with human subjectsespecially when the risks are so high.

Libella Gene Therapeutics, a Kansas-based biotech startup, is beginning a clinical trial for a telomerase gene therapy to treat broad aging this year. However, its taken its work to Colombia, where the standards for institutional review boards arent as high as they are in the US. Its a tactic informally known as IRB shopping, and it raises eyebrows in the research community.

Were always open to new ideas and novel ways [to treat dementia, Edelmayer says. We have to leave no stone unturned. But, she continued, one of the biggest things we want to see is not just theories. We want to see them tested.

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Could a gene therapy cure dementia? - Quartz

SAN RAFAEL, Calif., Jan. 13, 2020 /PRNewswire/ --BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, announced today that both the U.S. Food and Drug Administration (FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. have granted the Company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

The Company expects to start dosing patients in PHEARLESS, a Phase 1/2 study, in the first quarter of 2020 with product made at commercial scale from its award-winning gene therapy manufacturing facility. The Company is actively preparing regulatory submissions to open additional clinical sites in other countries.BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Status.

BMN 307 follows BioMarin's first investigational gene therapy clinical program: valoctocogene roxaparvovec to treat severe hemophilia A, currently in Phase 3. Based on data from an interim analysis of the ongoing Phase 3 study, the European Medicines Agency validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec, and the review process has begun. The Company also has submitted a Biologics License Application to the FDA and anticipates the review to begin in February 2020.

"With BMN 307, we are joining together our expertise in PKU biology and the knowledge we have gained from developing the only two approved therapies for PKU with our understanding of gene therapy clinical development and manufacturing from our valoctocogene roxaparvovec experience," said Hank Fuchs, President, Worldwide Research and Development at BioMarin."BioMarin has stood with the PKU community for over 15 years and remains dedicated to continuing to increase the body of medical knowledge in this devastating disease."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

BioMarin's clinical program is composed of two key studies. PHEARLESS, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, PHENOM, which has already started enrolling patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has treated approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 40 clinical studies in PKU and has sponsored 38 external clinical studies. BioMarin researchers have authored 54 publications in medical and scientific journals on PKU and supported another 52 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307. Manufacturing capabilitiesare an essential driver for our gene therapy programs and allows us to control quality, capacity, costs and scheduling enabling rapid development. Manufacture of BMN 307 was performed with a commercial ready process at scale in this facility. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and will speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet both commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well in to the future.

About Phenylketonuria

PKU, or PAH deficiency, is a genetic disorder affecting approximately 50,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's BMN 307 program generally, BioMarin's planned submissions to regulatory authorities for BMN 307, BioMarin's gene therapy manufacturing capabilities, the impact of using material manufactured at commercial scale in a clinical trial, the timing and results of BioMarin's planned Phase 1/2 trial of BMN 307, and the review of marketing applications for valoctocogene roxaparvovec. 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:results and timing of current and planned preclinical studies and clinical trials of BMN 307; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; 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 September 30, 2019 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.

Contact:

Investors:

Media:

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

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BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy - PRNewswire

After a manufacturing specification delay, Bluebird Bio has officially launched Zynteglo, its first gene therapy, andtapped a new leader to run the operation.

Nicola Heffron, a former exec with Celgene, Shire and GlaxoSmithKline, has joined Bluebird as its European chief, Bloomberg reported, just as the company gets its rollout underway there. She is replacing Andrew Obenshain, whos moving up to the global leadership team.

Heffron is tasked with charting the course for Bluebirds first commercial launch of its first product, Zynteglo, to treatbeta thalassemia, a rare inherited disease marked by reduced production of oxygen-carrying hemoglobin in red blood cells.

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And Heffron knows a thing or two about blood disorders. Before Bluebird, she headed up global marketing for Celgenes myeloid portfolio, her LinkedIn profile shows.

Wednesday, Bluebird revealed that the $1.76 million-per-treatment gene therapy is now available in Germany. The University Hospitalof Heidelberg serves as the drugs first qualified treatment center inthe country, and Bluebird said its working with institutions that have expertise in stem cell transplant as well as in treating patients with [beta thalassemia] to establish more centers.

RELATED:Bluebird Bio readies Zynteglo launch as EU approves 'refined' manufacturing

During a presentation at the annual J.P. Morgan Healthcare Conference on Tuesday, Bluebird CEO Nick Leschly confirmed the companysvalue-based payment model for the costly onetime therapy. Under that arrangement,payments of 315,000 ($351,000) each are made in five installments over five years. Except for the first round of expenses, payers only pay the rest if Zynteglo delivers on its therapeutic promise.

So far, the reimbursement agreements Bluebird has penned using that model can coverabout half of Germanys patients, according to Leschly.

Novartis has also rolled out a similar program for its $2.1 million spinal muscular atrophy gene therapy Zolgensma.

Bluebird won its European nod for Zynteglo last year but delayed the launch after a manufacturing specification hiccup. The Cambridge, Massachusetts-based biotech has established a manufacturing network that includes both internal facilitiesand contract partners for itslentiviral vector and drug product, Leschly said Tuesday.

RELATED:ASH: Bluebird's multiple myeloma CAR-T follow-up shows promise in phase 1

In the U.S., Bluebird has started itsrolling submission for approval and is in talks with the FDA regarding the requirements and timing of the various components of the application. Its expecting to finish the process in the first half of 2020.

Outside of beta thalassemia, Bluebird is testing the same drug, also known as LentiGlobin, in sickle cell disease, which is also marked by an abnormality in hemoglobin.

According to phase 1/2 data presented at last years American Society of Hematology annual meeting in December, none of the 17 patients enrolled in group Cwhich used an improved stem cell harvest technique and a new manufacturing processrequired regular blood transfusions post-treatment. Nine patients had beenfollowed for at least six months at that data cutoff.

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Bluebird Bio taps ex-Celgene exec Heffron to lead its first gene therapy launch - FiercePharma

Precision BioSciences, a Durham-based genome editing company, has reached two regulatory milestones for its potential therapy against multiple myeloma, a chronic cancer of white blood cells.

The U.S Food and Drug Administration (FDA) has accepted the companys Investigational New Drug (IND) application and also granted the therapy Orphan Drug Designation, a status that gives companies tax reductions and other incentives to develop treatments for rare diseases.

The therapy, designated as PBCAR269A, is Precisions third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate.

FDA acceptance of the IND for PBCAR269A further underscores the ongoing progress in our allogeneic CAR T pipeline, said Matt Kane, co-founder and chief executive officer of Precision BioSciences. We have now moved three CAR T programs from preclinical to clinical stage development since April 2019, and we look forward to continuing to advance our allogeneic CAR T portfolio to bring these novel therapeutic candidates to patients.

Matthew Kane

The company plans to begin a Phase 1 clinical trial of the therapy this spring at multiple sites using material produced at its own manufacturing facility in Durham. About 48 patients are expected to be enrolled.

For more information about the trial, visitwww.clinicaltrials.gov, and enter study identifier number NCT04171843.

In preclinical disease models, PBCAR269A has demonstrated no evidence of graft-versus-host disease at doses that resulted in potent anti-tumor activity, said Chris Heery, M.D., chief medical officer of Precision BioSciences. There remains significant unmet need in the treatment of relapsed/refractory multiple myeloma, and we are excited to begin clinical trials with an off-the-shelf CAR T therapy candidate in this setting.

The IND for PBCAR269A builds on the initial clinical data Precision presented in late 2019 for its lead program, a CAR T therapy for treating non Hodgkins lymphoma and B-cellacute lymphoblastic leukemia, and the FDAs acceptance of an IND for another CAR T therapy for treating non-Hodgkins lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma.

Precision BioSciences harnesses T cells, a type of white blood cell that is vital to the adaptive immune systems ability to identify specific antigens and destroy pathogens. Through the companys CAR T technology, the T cells can be directed to kill cancer cells.

Precision produces the CAR T therapies by selecting T cells derived from healthy donors as starting material. Then, using its proprietary ARCUS genome-editing technology, the company modifies the donor T cells.

Scientists insert the CAR gene at the T cell receptor locus, enabling the T cell to target a specific marker on a cancer cell, while knocking out the T cell receptor to prevent the patients immune system from recognizing and attacking the T cells.

The company optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of certain types of CAR T cells throughout the manufacturing process and in the final product.

The process creates a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease, normally a major challenge when inserting foreign or altered cells or tissues into the body.

The company has posted a four-minutevideoon its website to explain CAR T therapy, using Samurai warriors as an analogy.

Last July Precision opened its Manufacturing Center for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor CAR T cell therapy products.

Precision Bio facility

Given the potential output of our platform, weve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients, Kane said when the facility opened. In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.

The facility can produce three different drug substances: allogeneic CAR T cells, messenger RNA and adeno-associated viral vectors. It was designed to meet regulatory requirements in the United States, Europe and Japan.

In addition to health care, Precisions ARCUS genome-editing platform has applications in food and agriculture.

In 2018 the company created a new name and brand identity, Elo Life Systems, for its food and agriculture business, previously known as Precision PlantSciences, based in Research Triangle Park.

Elo is using the ARCUS platform and other new technologies for applications in crop improvement, animal genetics, industrial biotechnology and sustainable agriculture.

Since it was spun out of Duke University in 2006, Precision raised about $300 million in venture capital, government grants and collaboration agreements. The company went public in March 2019, grossing $145.4 million in an initial public offering of stock.

The companys shares are listed on the Nasdaq Global Select Market under the ticker symbol DTIL, shorthand for the companys marketing tagline, Dedicated to improving life.

(C) N.C. Biotech Center

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Precision BioSciences hits two key FDA milestones in advancing gene therapy for cancer - WRAL Tech Wire

SAN FRANCISCO Pharmaceutical companies and their investors have grown accustomed to big news kicking off the year, specifically multibillion-dollar deals.

The last three J.P. Morgan Healthcare Conferences, considered a sort of Opening Day for the industry, were hallmarked by the acquisitions of Ariad Pharmaceuticals, Impact Biomedicines and Loxo Oncology. (And that's not including Bristol-Myers Squibb's $74 billion deal for Celgene the week before the meeting last year.)

In fact, Eli Lilly pressed Loxo for a quick buyout to have something flashy to announce at last year's conference. Such enthusiasm was noticeably absent this time around, though, resulting in a quieter first day than biotech shareholders had hoped for.

The Nasdaq Biotechnology Index fell almost 2%, with Sage Therapeutics, Clovis Oncology and other potential takeover targets trading down by market's close. Brad Loncar, a founder of biotech exchange-traded funds, noted on Twitter how even shares of MorphoSys fell despite the German drugmaker having the most positive news of the day.

While major M&A announcements seem unlikely for the rest of the week, industry experts still expect the challenges facing bigger companies will result in a healthy number of deals in 2020. In the meantime, biotechs will be busy trying to deliver on their development plans some of which were provided in more detail during Monday's presentations.

Connecticut-based Alexion Pharmaceuticals is best known for its high-priced rare disease drugs Soliris and Ultomiris. On Monday, the company gave an early look at full-year financials, reporting a top line revenue increase of more than 20% between 2018 and 2019. That growth correlates to, at the very least, roughly $4.96 billion in annual revenue, which would be slightly higher than the average analyst estimate.

For Stifel's Paul Matteis, more surprising than the revenue beat was Alexion's plan to treat four times as many U.S. neurology patients with Soliris and Ultomiris by 2025. If successful, the plan would create a "substantial upside" to revenue estimates, according to the analyst.

"This of course raises a number of natural questions," Matteis wrote in a note to investors, "such as where will this growth come from, and what does it assume (if anything) for additional neuro indications where Soliris/Ultomiris isn't derisked."

Shareholders, however, responded positively to the updates, sending Alexion shares up 4%.

Soliris is approved to treat several diseases, including a chronic neuromuscular illness known as gMG and a type of central nervous system inflammation abbreviated as NMOSD. Alexion says that, in less than two years time, these neurology indications have become its largest franchise by patient volume. By the end of 2019, almost 1,900 U.S. neurology patients were taking Soliris.

Ultomiris, a follow-on to Soliris, is under investigation as a treatment for gMG and NMOSD across a couple of late-stage studies. And on Tuesday, Alexion announced it will soon begin a Phase 3 study of the drug in ALS, with plans to enroll 350 adults in a 50-week trial.

Selling new paths to growth is particularly important for Alexion now, as the company has come under pressure from activist investor Elliott Advisors to seek a sale.

BioMarin could bring the first hemophilia gene therapy to market later this year. While waiting for regulators to confirm its approval application is under review, the California biotech announced Monday it has more than doubled capacity at a gene therapy plant. Altogether, the facility can make up to 10,000 doses each year of either the hemophilia treatment or a separate BioMarin gene therapy that's about to begin human testing.

That capacity level, according to executives, would allow the company to treat all U.S. hemophilia A patients in roughly two year's time. The update increases the competitive pressure on drugmakers with marketed products, such as Takeda and Novo Nordisk, as well as those working on rival hemophilia gene therapies. Swiss pharma giant Roche falls into both buckets, and could lose out on many patients because of BioMarin, according to a recent doctor survey from Citi Research.

Sarepta Therapeutics also had a manufacturing update, announcing that production for its experimental micro-dystrophin gene therapy is now large enough to be considered commercially viable.

RBC Capital Markets analyst Brian Abrahams called this a "critical manufacturing milestone" for Sarepta, one that shores up the timeline for a pivotal study scheduled to start sometime in the middle of the year.

Despite launching one of the industry's largest initial public offerings in 2018, Rubius Therapeutics has struggled out of the gate in getting its first clinical data. While its ambitions are large, the Flagship Pioneering-backed biotech failed to meet expectations it set for delivering early Phase 1 data from its lead asset, a PKU drug, by the end of 2019.

"We understand what we didn't do right in 2019, and we are doing it differently in 2020," CEO Pablo Cagnoni told a half-filled breakout room at the JPM conference. "We will deliver in 2020."

While Cagnoni and other executives emphasized cancer therapies set to enter clinical testing in 2020 and speedy progress on its own manufacturing plant, the biotech also admitted it still has not dosed a single PKU patient to date, calling into question the company's ability to execute.

Pressuring Rubius further is BioMarin's announcement that it will develop a PKU gene therapy, with plans to dose its first patient this quarter. BioMarin already sells two PKU drugs and could be a formidable competitor.

Shares in Rubius ticked down by about 5% Monday and have shed two-thirds of their value since the company went public.

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Alexion's neuro bet, Rubius' trial troubles and biotech's gene therapy milestones - BioPharma Dive

SAN FRANCISCO, Jan. 14, 2020 /PRNewswire/ --GenScript Biotech Corp., a leading global biotechnology group and a pioneer in the field of gene synthesis, today held its inaugural "Global Forum on Cell & Gene Therapy and the Booming China Market," during the JP Morgan Healthcare Conference week, attracting hundreds of industry leaders, investors and others to address the challenges and opportunities in this innovative field.

"As an industry, we are on the brink of achieving some extraordinary breakthroughs in cell and gene therapy for cancer and other diseases," said GenScript Biotech CEO Frank Zhang, PhD. "Four gene and cell therapies have recently been approved by the FDA, bringing new hope to patients, and this is only the beginning. Our vision is to make cancer a chronic or curable disease rather than a deadly one, and to transform the treatment of cancer, autoimmune and other diseases by leveraging the advantages of cell and gene therapy."

While significant advances are being made, the Forum also tackled some of the more pressing challenges, such as mitigating treatment side effects, improving treatment efficacy in solid tumors and scaling up manufacturing. Panelists from Kite Pharma, GE Healthcare Life Sciences, Ziopharm Oncology, Oxford Biomedica, Genethon, CARsgen Therapeutics, J&J Innovation Asia Pacific, the American Society of Gene & Cell Therapy, Loncar Investment, Lilly Asia Ventures, and many others participated in the event.

In the U.S. alone, the U.S. Food and Drug Administration is expected to approve 40-60 cell and gene therapies by 2030. During a panel discussion focused on regulatory issues, experts considered what regulators will need to do to keep up with the rapid pace of innovation, the new hospital-based regulatory pathway in China, how to ensure quality through the manufacturing process, and the challenges and opportunities that come with regulatory harmonization among different countries.

China continues to attract significant attention from industry and investors and is poised to grow even more. During his welcoming remarks, Zhang noted China's emergence as a global economic leader, with a projected $1.1 trillion spend on healthcare this year, as well as the growing disease burden in China. By 2030, an estimated 4.3 million Chinese will be diagnosed with one of the 14 major cancers, according to research from IMS Health. Panelists addressed issues such as the amount of capital required to achieve scale in China, and advantages of the market in China.

"The drug development business is changing rapidly and China is at the fore in a number of ways," Zhang said. "Biotech and pharma companies do not need or desire to have the infrastructure to scale their drugs through commercialization. With lower costs, China is a natural place for companies to contract out costly development and manufacturing to organizations that have the expertise and experience to collaborate with them through the entire discovery to development lifecycle."

For its part, GenScript has put significant resources into its Contract Development and Manufacturing Organization (CDMO) business to meet the increasing demand. In 2018, the company officially launched its biologics CDMO segment, and last year opened a new GMP compliant biologics research center. GenScript is also leading the way in cell therapy through its antibody discovery service and plasmid and virus production capabilities.

InDecember2017,LegendandJanssen, a Johnson & Johnson business,enteredintoaworldwidecollaborationandlicenseagreementtodevelop,manufactureandcommercializeLCARB38M(JNJ4528)inmultiplemyeloma. The companies are advancing this investigational B-cell maturation antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell therapy in previously treated patients with the disease. Clinical trials are ongoing in both the U.S. and China and the therapy has received orphan drug designation and breakthrough treatment designation from the FDA in the U.S. and PRIME designation from the European Medicines Agency.

GenScript is one of only a few Chinese companies to have a significant presence during JPM week. Based on the success of this year's Forum and the interest in cell and gene therapy, GenScript will host its second annual Forum with influential leaders on the topic again next year during JPM week at the San Francisco Grant Hyatt.

About the GenScript Biotech Global Forum

The GenScript Biotech Global Forum, with the theme "Cell & Gene Therapy and the Booming China Market," brings together industry, investors, government and academia to learn, collaborate and become inspired and engaged in the advancement of cell and gene therapy in China and across the globe. As the most exclusive annual gathering of global cell and gene therapy leaders during the JP Morgan week, the inaugural Forum in 2020 attracted more than 700 attendees gathered in San Francisco, representing leading biotech and pharma companies, cell and gene therapy innovators, top universities and government agencies. The forum aims to build a bridge between the cell therapy industry and capital community, accelerating mutual understanding and creating collaboration opportunities.

About GenScript Biotechnology

GenScript Biotech Corporation (Stock Code: 1548.HK) is a leading global biotechnology group and a pioneer in the field of gene synthesis. The companyoperates in four main categories: life sciences CRO services, biologics development and manufacturing, industrial biology and cell therapy.

Founded in 2002 and listed on the Hong Kong Stock Exchange in 2015, GenScript has established a global presence across Greater China, North America, the EU, and Asia Pacific. Today, over 100,000 customers from over 160 countries and regions around the world use GenScript's premier, convenient, and reliable products and services.

GenScript currently has more than 2,900 employees globally, 34 percent of whom hold master's and/or doctorate degrees. In addition, GenScript has a number of leading commercial products, more than 100 patents and 270 patent applications. As of June 2019, GenScript's products and services have been cited in 40,300 scientific papers worldwide.

GenScript is committed to being the most reliable biotech company in the world and to achieving our vision of making humankind and nature healthier through biotechnology.

For more information, please visit https://www.genscript.com/

Contact:

Corporate:Fiona CheCorporate Communications Manager, GenScript+86 -025-58897288-6321 Fiona.che@genscript.com

Media Susan ThomasPrincipal, Endpoint Communications(619) 540-9195susan@endpointcommunications.net

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GenScript Biotech Global Forum Highlights Advances in Cell and Gene Therapy and Opportunities in China - PRNewswire

In 2019 a Novartis drug called Zolgensma got approved and made huge headlines. The headlines were less about the fact that this one time treatment can treat spinal muscular atrophy (a condition occurring in 1 out of 10,000 newborns and limiting their life to often mere two years) but about it being the most expensive drug ever.

In the United States the list price of Zolgensma stands over $2 million. The one time treatment is a so-called gene therapy that injects a virus aimed at fixing the failed or mutated gene responsible for motoric muscle functions. While this treatment is good news for many patients, it also fueled the debate on the ethics of high drug prices.

I would like to provide a few fresh, not fully reflected, and hopefully provoking thoughts for the new year on what policy makers should keep in mind when aiming to control drug prices.

So why dont we just cap the prices per drug?

Two million dollars per patient is definitely a crazy high amount. The reason why it cant be offered much cheaper right now is that there are (fortunately) not that many patients that actually need this treatment. The drug manufacturer has a very small target audience they can charge for this treatment of a rare disease. From that small pool of people they need to recoup their development costs for this drug, make up for totally failed projects, and make shareholders happy with a profit margin.

This sounds cruel So why dont we have stronger laws on capping such excesses? If we legislate a maximum cap to how much a drug per patient is allowed to cost, we will end up with pharmaceutical research aimed at treating and curing common diseases and patients with rare diseases will have a bleak future.

Why are there so often drugs that are similar to already existing drugs and dont focus on curing diseases we cant cure or treat yet?

Medical research is unfortunately a shotgun. When starting to develop a molecule or target a genetic defect you never really know where you end up. On average, only one of every 5,000-10,000 substances synthesized in research facilities will make it successfully through all stages of product development to become an approved drug. Many projects and even entire biotech companies fail to bring even one product to the commercial stage. Investing in life sciences requires a very healthy appetite for risk, and hence an incentive scheme that rewards those able to create value with their inventions is required. By the time a medical drug reaches the regular patient, an average of 12.5 years will have elapsed since the first discovery of the new active substance. The total investments needed to get to one active substance that can be accessed by a patient is around two billion Euros.

Why is delinkage is a bad, very bad, idea?

There are big proponents within the World Health Organization and some southern European governments to de-link the research and production of medical drugs. Supporters of this so-called delinkage support publicly funded R&D of drugs and private companies merely charging for their production costs plus a modest markup. This semi-planned pharma economy should frighten all those who are still waiting for medical innovations to cure their or their loved ones from medical conditions. Medical innovation (or to be more precise the lack of it) in planned economies such as the Soviet Union is a solid benchmark of why this is a bad idea. Research funding has been politicized in such systems and only funneled into pet projects of apparatchiks or (in the best case) to very common diseases the majority of voters are interested in.

Side note: The Soviet Union put in a lot of effort to develop cutting-edge doping drugs for their Olympic athletes but neglected researching actual cures. An example is the story of a Russian scientist who smuggled in a drug with the help of Mayo Clinic practitioners to treat his daughters tuberculous meningitis was even sentenced to death for being in contact with an American scientist.

So how can things stop getting more expensive?

Critics of the innovative pharmaceutical industry often make the point that pharma has an incentive to not fully cure patients as they only make money with sick patients. The same argument can be made about hospitals and outpatient doctors. There are historic capitation examples in which doctors only get paid per healthy patient month (I was told by someone very smart that this used to be the case in ancient Chinese villages). These concepts obviously work much better in a super-low-tech health system or for the reimbursement of providers only. Adding pharmaceutical research into this equation makes things definitely hard; but not impossible.

So would it be possible to have fully integrated healthcare companies that are providers, payers, and pharma industry at the same time?

A rather crazy thought experiment: I just googledvertical integration pharmaceuticals and insuranceand surprisingly couldnt find much. My healthcare management textbook told me a decade ago that integrating (or collaborating) with pharma would be called a diagonal collaboration as pharma and med tech are often not even seen as a proper part of the health system. A vertically integrated healthcare company owning drug patents and having patent cross license agreements with similar companies would have a higher incentive to provide its patients/insurance population with all drugs it has access to. Given the growing importance of health data both in treating patients (patient value chain) but also finding cures could be massively leveraged in such an integrated organization. The main profits would be generated through insurance premiums and drug sales to markets the insurance is not active in or to competitors.

This could be an interesting market response to realigning incentives in private healthcare and healthcare in general. The biggest roadblocks and reasons why this has not happened yet are:

Will the next big thing come out of a garage lab?

I recently watched the very entertaining Netflix documentary seriesUnnatural Selectionthat shows both the significant advances of gene therapy by established pharma companies and some outlaw biohacker activists that try to democratize biotech research through their garage labs. While most of these biohackers seem to be overly optimistic of their chances to develop effective cures and genetic improvements, one should not reject the possibility of disruption within the biotech industry.

The big consumer-reaching advances of the computer and software industry happened eventually in Californian and New Mexican garages and not in the labs of IBM or GE. Gene editing techniques such as CRISPR bring a lot of hope to patients and families struggling with genetic diseases. And first advances with this technology in agriculture and animal health suggest that treatments can be developed in relatively small and inexpensive labs and research environments.

So theres a possibility that the competition density in the biotech industry will intensify and market entry barriers be lowered due to gene editing technologies. At the same time regulatory barriers and approval requirements need to adopt to these technologies, allow them, and take into account that too high barriers will keep competition low and hence prices for new treatments high.

Quick and easy one to save Billions in 2020:Scrap value added and all sales taxes from prescription medicine. In some countries this would lead to a reduction of drug prices by 25%! It is also more than sad that some developing countries still levy tariffs on imported innovative medicines.

Please feel free to chip in to this debate. It is important to keep an open mind and find new ways to fund innovative medicine. At the same time it is important to stick to the realities of medical research and innovation.

A less futuristic but more public policy oriented paper on The Consumer Case for Intellectual Property authored by me can be foundhere.

Fred Roeder has been working in the field of grassroots activism for over eight years. He is a Health Economist from Germany and has worked in healthcare reform and market access in North America, Europe, and several former Soviet Republics. One of his passions is to analyze how disruptive industries and technologies allow consumers more choice at a lower cost. Among many op-eds and media appearances, he has been published in the Frankfurter Allgemeine Zeitung, Wirtschaftswoche, Die Welt, the BBC, SunTV, ABC Portland News, Montreal Gazette, Handelsblatt, Huffington Post Germany, CityAM. LAgefi, and The Guardian. Follow him on Twitter @FredCyrusRoeder

A version of this article was originally published on Consumer Choice Centers website as For the new year: Some fresh thoughts on how to tackle high drug prices and has been republished here with permission.

Excerpt from:
Viewpoint: Pricey gene therapies fuel debate over drug pricesand why there is no easy fix - Genetic Literacy Project

International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials which account for 77% of the total 127 ongoing trials.

London UK, 15th January 2020 - The Cell and Gene Therapy Catapult (CGT Catapult) today released their 2019 UK Advanced Therapy Medicinal Products (ATMPs) clinical trials database showing that the 127 ongoing trials represent a ~45% increase compared to 2018, and account for 12% of the total global cell and gene therapy clinical studies.

The NHS and UK ecosystem are providing the right platforms to allow innovative therapies to progress through to the clinic in ever increasing numbers. Cell and gene therapies require novel approaches, systems and infrastructure to enable the delivery of these life changing, living medicines to patients. The favourable UK environment is being recognised internationally with the majority of commercially sponsored trials being backed by international organisations.

Cell and gene therapies continue to progress towards large scale commercialisation to add to the therapies already approved for use. The database shows that 77% of UK cell and gene therapy trials are now sponsored by commercial organisations compared to only 25% in 2013. This number reflects the continuous investment that companies are making in cell and gene therapies, and the transformative effects that these therapies are proving to have on patients lives.

The main indication for cell and gene therapies clinical trials remains oncology (39%) followed by ophthalmology (13%) and haematology (12%).

Health Minister Baroness Blackwood said:

These extraordinary figures show the UKs life sciences sector is leading the world in getting cutting-edge treatments to NHS patients as quickly as possible. As our research capabilities continue to grow, our exceptional NHS as a whole will be able to reap the benefits of not only more innovative medicines for patients, but investment from companies seeking outstanding expertise.

Ian Campbell, Interim Executive Chair, Innovate UK commented:

The UK is a world leader in developing innovative therapies. Through continued support from government, we are building a network of excellence which brings together research and commercial organisations. With the Cell and Gene Therapy Catapult playing a key role in advancing the development of these therapies, this will not only bring concrete benefits to patients but will also allow the UK sector to compete globally.

Keith Thompson, Chief Executive Officer, Cell and Gene Therapy Catapult stated:

The total number of cell and gene therapy clinical trials in the UK has been increasing consistently by an average of 25% year on year since 2013. This has been enabled by the development of the UKs fantastic ecosystem to support the development and clinical adoption of cell and gene therapies. The infrastructure and initiatives that have been put in place, with strong backing by the government, including the Advanced Therapy Treatment Centres network, are giving companies the confidence to setup and run their innovative clinical studies here. The result, is that we are now seeing therapies moving from academic projects towards becoming commercial products that can be delivered at scale by the NHS.

The report and database can be downloaded at ct.catapult.org.uk/clinical-trials-database

About Cell and Gene Therapy Catapult:

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 200 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

See the rest here:
The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. -...