Category Archives: Genetic Therapy

Boston Children's Hospital researchers used an investigational gene therapy to treat heart failure in a mouse model of Barth syndrome. Barth syndrome is a rare genetic disorder in boys that results in life-threatening heart failure. It also causes weakness of the skeletal muscles and the immune system. The disease is caused by a mutation of a gene known as tafazzin or TAZ.

In 2014, William Pu and researchers at Boston Childrens Hospital collaborated with the Wyss Institute to develop a beating heart on a chip model of Barth syndrome. It used heart-muscle cells with the TAZ mutation that came from patients own skin cells. This was able to prove that TAZ was the cause of the cardiac problems. The heart muscle cells did not organize normally and the mitochondria, the cells energy engines, were disorganized, resulting in the heart muscle contracting weakly. By adding healthy TAZ genes, the cells behaved more normally.

The next step was an animal model. The results of the research were published in the journal Circulation Research.

The animal model was a hurdle in the field for a long time, Pu said. Pu is director of Basic and Translational Cardiovascular Research at Boston Childrens and a member of the Harvard Stem Cell Institute. Efforts to make a mouse model using traditional methods had been unsuccessful.

Douglas Strathdees research team at the Beatson Institute for Cancer Research in the UK recently developed animal models of Barth syndrome. Pu, research fellow Suya Wang, and colleagues characterized the knockout mice into two types. One had the TAZ gene deleted throughout the body; the other had the TAZ gene deleted just in the heart.

Most of the mice that had TAZ deleted throughout their whole bodies died before birth, likely from skeletal muscle weakness. Of those that survived, they developed progressive cardiomyopathy, where the heart muscle enlarges and is less able to pump blood. The heart also showed signs of scarring similar to humans with dilated cardiomyopathy, where the hearts left ventricle is dilated and thin-walled.

The mice that lacked TAZ only in their heart tissue that survived to birth had the same features. Electron microscopy indicated that the heart muscle cells and mitochondria were poorly organized.

Pu and Wang and their team then used gene therapy to replace TAZ in the newborn mice and in older mice, using slightly different techniques. In the newborn mice the engineered virus was injected under the skin; in the older mice it was injected intravenously. The mice who had no TAZ in their bodies and received the gene therapy survived to adulthood.

In the newborn mice receiving the gene therapy, the therapy prevented cardiac dysfunction and scarring. In the older mice receiving the therapy, it reversed the cardiac dysfunction.

The study also showed that TAZ gene therapy offered durable treatment of the cardiomyocytes and skeletal muscle cells, but only when at least 70% of the heart muscle cells had taken up the gene via the therapy. Which the researchers point out that when the therapy is developed for humans, that will be the most challenging problem. You cant just scale up the dose because of inflammatory immune responses, and multiple doses wont work either because the body develops an immune response. Maintaining the gene-corrected cell is also a problem. In the heart muscles of the treated mice, the corrected TAZ gene stayed relatively stable, but slowly dropped in skeletal muscles.

The biggest takeaway was that the gene therapy was highly effective, Pu said. We have some things to think about to maximize the percentage of muscle cell transduction, and to make sure the gene therapy is durable, particularly in skeletal muscle.

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Gene Therapy Reverses Heart Failure in Animal Model of Barth Syndrome - BioSpace

RESEARCH TRIANGLE PARK, N.C. and CHAPEL HILL, N.C., March 18, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a leading clinical-stage adeno-associated virus (AAV) gene therapy company, today announced that it has entered into a research collaboration and licensing agreement with the University of North Carolina at Chapel Hill (UNC) for the development and commercialization of gene therapy for Angelman syndrome.

This collaboration allows us to leverage groundbreaking research from UNC and apply our AAV development capabilities to find a gene therapy treatment for Angelman syndrome, said Sheila Mikhail, JD, MBA, AskBio Chief Executive Officer and co-founder. We look forward to advancing this program together.

Angelman syndrome is a rare neurogenetic disorder caused by the loss of function of the UBE3A gene. The disorder occurs in approximately one in 15,000 people, or about 500,000 individuals worldwide, and there is currently no cure. In addition to life-altering symptoms such as speech and motor deficits, more than 80 percent of Angelman syndrome patients experience epilepsy, which typically does not respond well to standard anti-seizure medications.

A UNC School of Medicine team, led by Mark Zylka, PhD, and Ben Philpot, PhD, has generated preclinical evidence that gene therapy may help individuals with Angelman syndrome by improving seizure and motor outcomes.

Individuals with Angelman syndrome face lifelong challenges, and our gene therapy approaches hold the potential to correct this disorder at its genetic roots. We are incredibly excited to partner with AskBio, as they have been vanguards of clinical gene therapies for rare diseases, said Mark Zylka, PhD, Director of the UNC Neuroscience Center. Ben Philpot, PhD, Associate Director of the UNC Neuroscience Center added, We look forward to advancing this transformative treatment to the clinic and potentially improving the lives of individuals with Angelman syndrome.

The partnership between AskBio and UNC could transform the lives of people living with Angelman syndrome by providing them with a potential therapy for this rare disease, said Amanda Moore, Angelman Syndrome Foundation CEO. The Angelman Syndrome Foundation has long been proud to support the work of UNC researchers, Drs. Ben Philpot and Mark Zylka, and invest in science that positively affects the Angelman syndrome community. The collaboration between UNC and AskBio brings us a step closer to delivering a viable gene therapy to the people and families we serve.

The financial terms of the agreement were not disclosed.

More about Angelman SyndromeDeletion of the maternally inherited copy of the UBE3A gene causes Angelman syndrome. Symptoms include microcephaly (small head circumference), severe intellectual disability, seizures, balance and movement problems (ataxia), lack of speech, and sleep problems. Behavioral symptoms include frequent laughing, smiling and excitability. Angelman syndrome was first described in 1965, yet no treatment options have been approved in the 55 years since. While individuals with the disorder have a normal lifespan, they require life-long care and are not able to live independently.

About Angelman Syndrome FoundationThe mission of the Angelman Syndrome Foundation is to advance the awareness and treatment of Angelman syndrome through education and information, research and support for individuals with Angelman syndrome, their families and other concerned parties. We exist to give them a reason to smile, with the ultimate goal of finding a cure. To learn more, visit https://www.angelman.org.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10 and an extensive adeno-associated virus (AAV) capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). Learn more at https://www.askbio.com or follow us on LinkedIn.

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AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome - GlobeNewswire

Law360 (March 18, 2020, 4:34 PM EDT) -- There have been remarkable advances over the last several years in the development of cell and gene therapies, or CGTs. These therapies represent truly groundbreaking approaches to the treatment and prevention of diseases, many of which have proven resistant to traditional drugs or therapies.

Cell therapy generally refers to the transfer of live cells into a patient to treat a disease. The cells may originate from the patient (autologous therapy), where they are extracted, modified and reinfused into the patient, or from a donor (allogeneic therapy). Gene therapy involves a change in the genetic code of a patient by inserting or...

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Cell And Gene Therapies Are Driving M&A Deal Activity - Law360

People with mesothelioma dont have many treatment options.

A combination of surgery, chemotherapy and radiation is the standard of care. For years, chemotherapy was the only method with approval from the United States Food and Drug Administration.

In 2019, that changed. Now, in 2020 and looking forward, theres a lot of potential for another unorthodox treatment to emerge.

Last May, the FDA approved using the NovoTTF-100L System on a limited basis for pleural mesothelioma. Patients can use the treatment as a first-line option with chemotherapy and only if they arent candidates for surgery.

The NovoTTF-100L became the first mesothelioma treatment option in 15 years to receive FDA approval. In 2004, pemetrexed was approved for treating this cancer.

Hopefully, we wont have to wait another 15 years for the next approval. Here are three classes of cancer treatment that could break through soon and get approval from the FDA.

If youre interested in accessing any of these cancer treatment techniques, please email our registered nurse, Jenna Campagna. She can help you connect with a mesothelioma specialist and look into experimental treatments. Please email her at jenna@mesotheliomaguide.com.

Immunotherapy is at the forefront of many clinical studies involving mesothelioma. The treatment uses drugs to enhance the bodys immune system. The theory is that the body could better fight mesothelioma with just a little outside help.

There are a few variations of immunotherapy. The one used the most in experiments is checkpoint inhibitors. This type of immunotherapy drug targets the relationship between two proteins: PD-1 and PD-L1.

PD-1 is a protein on T-cells, which are the immune systems fighter cells against diseases like mesothelioma. PD-L1 is a protein in mesothelioma cells.

When the two interact, the T-cells cannot detect mesothelioma cells as dangerous to the body. Basically, the PD-1/PD-L1 connection is a mask for mesothelioma.

Checkpoint inhibitors block this connection, which allows the immune system to detect and attack infected cells. The two most common drugs are nivolumab (brand name Opdivo) and pembrolizumab (brand name Keytruda).

Dr. Patrick Forde and his team are testing immunotherapy with surgery and chemotherapy for pleural mesothelioma. Dr. Forde is a thoracic oncologist at Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

The clinical trial involves two stages of two immunotherapy drugs: nivolumab and ipilimumab. Patients receive three doses of nivolumab before surgery and one dose of ipilimumab. Then patients can receive further immunotherapy treatment after surgery.

Immunotherapys versatility is one of the treatments best qualities. It can be used before or after surgery, in conjunction with chemotherapy, and even paired with other emerging therapies.

Dr. Forde, who specializes in treating pleural mesothelioma, said immunotherapy can reduce the size of tumors and how widespread they are in the body. This regression can help surgeons remove the diseased cells during operation.

There are numerous forms of gene therapy. One type involves restructuring the genetic characteristics of infected cells. Another type one called suicide gene therapy uses genes to engage the immune system and attack the tumors.

Gene therapy usually isnt sufficient on its own. Instead, its used in conjunction with other mesothelioma treatment options.

The purpose of gene therapy is to repair or weaken infected cells. Repairing can reduce the number of dangerous mesothelioma cells in the body. Weakening can increase the effectiveness of other treatment methods, such as surgery, chemotherapy or immunotherapy.

A Phase 3 clinical trial involving gene therapy might be the most promising one available to pleural mesothelioma patients. Dr. Daniel Sterman is the director of the Multidisciplinary Pulmonary Oncology Program at New York University Langone Health. He is the lead investigator in the study, which combines gene therapy with chemotherapy.

The trial uses TR002, which is adenovirus-delivered Interferon Alpha-2B. The treatment is a gene therapy transported by a virus into the body. Interferon Alpha-2B is a gene therapy that causes tumors to produce interferon alpha, which is a cancer-fighting protein.

The increased production of this protein alerts the immune system of danger. The T-cells recognize that the cells are producing this protein and begin attacking them.

The Phase 2 results were astonishing in a positive way. The disease control rate meaning the cancer either was stagnant or reduced in size was 87.5% for enrolled patients. Additionally, the survival time for patients who received this gene therapy treatment nearly doubled compared to those who didnt.

Virotherapy involves using programmed viruses to either attack mesothelioma cells or kickstart the immune system. Virotherapy also is used as a vessel to transfer other forms of therapy into the body.

Its another outside-the-box method to help the body stop mesotheliomas progression. There are three types of virotherapy used in experimental mesothelioma treatment:

Oncolytic viruses are modified viruses that focus on cancer cells and ignore healthy ones. This form of virotherapy explicitly targets mesothelioma, and its the most common type of the treatment.

Viral vectors are transports for other forms of treatment. Viral vectors could deliver modified genes or an immune system enhancer.

Viral immunotherapy involves using viruses to activate the immune system. Since the viruses are considered intruders, the immune system senses danger and looks for these viruses. Doctors often program the virotherapy similar to the genetic structure of mesothelioma cells. Doing so allows the immune system to identify harmful cells based on these genetic characteristics.

A new clinical trial involving virotherapy and immunotherapy has potential. The study uses ONCOS-102, which is a modified virus that transports immune system stimulants (cytokines).

ONCOS-102 is a combination of viral immunotherapy and oncolytic viruses. The virus aspect breaks apart the mesothelioma cells, and the cytokines send danger signals to the immune systems T-cells. When the mesothelioma cells break apart, they release antigens that the T-cells recognize as cancerous.

This two-pronged process involving virotherapy and immunotherapy has helped patients. Those receiving ONCOS-102 plus chemotherapy had a progression-free survival of around nine months.

Progression-free survival means the patient didnt die and the disease didnt spread further in the body. By comparison, patients who only received chemotherapy had a progression-free survival of around seven months.

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3 Mesothelioma Treatment Options on the Rise - Mesothelioma Guide

- Research for novel cancer therapeutics pool in massive investments in red biotechnology market

- Player leverage AI to extract potential of red biotechnology in preserving health and controlling diseases

ALBANY, New York, March 18, 2020 /PRNewswire/ -- A wide assortment of applications of genetic engineering technology, vaccine research, and biologics have helped expand the potential of the red biotechnology market, which was worth US$314.2 billion in 2018. Growing application of biotechnology in medicine has unlocked promising prospects particularly in chronic and rare diseases treatment.

Future Outlook

Emerging applications of gene therapy, pharmacogenomics, and genetic testing in the preservation of health, notably in oncology, are shaping the future growth trajectories in the red biotechnology market. By 2027-end, the revenues are projected to reach US$512 bn, clocking CAGR of ~6% from 2019 to 2027. In developing countries, many new collaborations are likely to be forged, defining future initiatives on cloud-based technology and AI.

Analysts' Viewpoint

"Staying abreast with standardized regulations and regulatory norms will enable biotechnology and pharmaceutical companies to chart new growth avenues in the red biotechnology market," note the analysts. Marked prevalence of cancer world over expands scope for market stakeholders, they further opine.

Key Takeaways of Red Biotechnology Market Study

Explore 176 pages of top-notch research, incisive insights, and detailed country-level projections. Gain business intelligence on Red Biotechnology Market (Application: Biopharmaceutical Production, Gene Therapy, Pharmacogenomics, and Genetic Testing; End User: Biopharmaceutical Industry, CMOs & CROs, Research Institutes, and Others) - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2019 - 2027 at https://www.transparencymarketresearch.com/report-toc/2063

Red Biotechnology Market: Key Driving Factors and Promising Avenues

A few striking trends in investments in healthcare sector shape the evolution of the red biotechnology market. Growing number of clinical trials in cancer research and incessant efforts of biotechnology players to find therapies for rare diseases are boosting the pace of new drug approvals. A few statistics support research and developments in aforementioned realms.

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Red Biotechnology Market: Regional Outlook

Among the various regional markets, North America leads the pack, and is expected to retain its dominance throughout the assessment period. Spate of investments in clinical trials for chronic and rare diseases and massive investments in developing cancer drug pipeline have helped the North America red biotechnology market to reach dominant position. Stakeholders are harnessing artificial intelligence will continue new prospects in the regional market. Prominently, relentless efforts of biopharmaceutical players in the U.S. test new drugs and vaccines form crucial trend in the growth of this regional market.

On the other hand, a growing numbers of players have shifted their attention to Asian economies to tap into the vast latent potential in personalized healthcare. The region will see new strategic collaborations among healthcare companies and biopharmaceutical players.

Red Biotechnology Market Competition Landscape

Growing demand for biologics and biosimilars in developing economies has been crucial to the expansion of red biotechnology prospects, globally. Most notably, Asia Pacific has been the key focus for players to gain competitive edge over their peers and rivals.

Biopharmaceutical players have begun inking new partnership deals and distribution agreements in the region, particularly in on oncology therapeutics and development. Focus on novel therapeutics and disease pathways for bettering human health has been one of the key winning imperatives for companies in the red biotechnology market. To consolidate their positions, top players are aiming to expedite product approvals for rare and chronic diseases, and have increased their stakes in advanced stages of clinical trials.

A handful of big pharmaceutical and biotech players, and multinational healthcare companies jointly contribute half of the global revenues. These include Pfizer Inc., Gilead Sciences Inc., Amgen Inc., and F. Hoffmann-La Roche.

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The red biotechnology market is segmented on the basis of application, end user, and region.

Explore Transparency Market Research's award-winning coverage of the global Healthcare industry:

Bioinformatics Market- The global bioinformatics market is projected to grow at a significant CAGR during the forecast period and is likely to touch a valuation of US$9.1 bn by 2018.

Gene Therapy Market -Expanding at a stellar, double-digits CAGR (Compound Annual Growth Rate) of 40% over the forecast period of 2018 to 2026, the global gene therapy market is a dizzying trajectory, marking out a rosy landscape for players operating in the playfield. As per a Transparency Market Research report, based on extensive primary and secondary research, states that over the period states, the market would accrue a worth of USD 5164.03 million a steep and impressive increase from the USD 17 million worth noted in 2017.

Biosimilars Market- Advancements in oncology settings are improving patient quality of life. Due to effective biosimilars, healthcare providers are able to receive better outcomes in several cancer patients who can opt for convenient treatments such as a painless injections rather than receiving a lengthy chemotherapy session. Patent expiry of biologic drugs for the treatment of cancer are creating a scope for incremental opportunities. As such, oncology indication segment of the biosimilars market is estimated to reach a value of~US$ 21.1 Bnby the end of2027.

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Exploring Avenues in Personalized Medical Care Defines Advances in Red Biotechnology Market: Valuation to Touch Massive US$512 Bn By 2027, Finds...

Yousuf Khan is PhD student in molecular and cellular physiology at Stanford School of Medicine. He is the first author on a recent article published in BMC Genetics which outlines the discovery of a novel overlapping coding sequence in the gene POLG. His work, combined with other research in the field, may suggest that there is an abundance of overlapping open reading frames.Technology Networks recently spoke with Khan to learn more about the research study, how genetics can be likened to cooking up a recipe, and an accidental discovery that could lead to an exciting collaboration in this field.

Molly Campbell (MC): For our readers that may be unfamiliar with genomics, open reading frames and bioinformatics, can you tell us about the background of your research?Yousuf Khan (YK): Lets compare the expression of a genetic sequence with cooking a meal. TheDNAis our motherscookbook, it contains every single possible recipe that we would ever need. But when we want to prepare a meal, we dont want to get our precious cookbook dirty. So instead, we store the cookbook in a different location (genomic DNA in eukaryotes is inside the nucleus). When we want to make something, we make aphotocopyof a page in the cookbook (the cell creates amessenger RNA, a temporary copy, of a portion of DNA).

This process of copying a portion of theDNAintomRNAis called transcription. We then take oursingle sheet of our photocopied recipe(ourmRNA) and take it back into the kitchen (thecytoplasmof the cell).

In thekitchen(cytoplasm), we read thephotocopied recipeone step at a time. By reading thephotocopied recipefrom thefirst step to the last step(the open reading frame), we convert the instructions into our finished,delicious meal. In a similar manner,mRNAis read and translated into aproteinby a machine called a ribosome.In the traditional way we understand biology, cells that want to create different proteinsjust alternatively splice differentmRNAsto be translated. This would be the equivalent of photocopyingrecipesfor scrambled eggs, pancakes, and bacon on Monday morning and then photocopying a different set ofrecipes(e.g. mashed potatoes, steak, and salad) for dinner.

MC: Can you expand on your recently published study? Have you essentially discovery a "gene within a gene"? If so, does this point towards a potentially "hidden" genome?YK: In our article, we found that the gene POLG creates an mRNA that contains a very long overlapping open reading frame. Imagine youre following the instructions of a recipe to make lasagna; you start with step one and you complete every step until you reach the last step. But if you started at step two and then completed steps three, four, five, and six, you would create a hamburger instead. So encoded within a single photocopied recipe, there are multiple meals that can be made.

This study and others that have been published previously may suggest that there are an abundance of these overlapping open reading frames. The real effort is finding them and characterizing them!

MC: What were the key challenges you encountered in this research?YK: The real challenge is finding these overlapping sequences. Theyre tricky to detect and it would not have been possible to do this work without the support of the amazing researchers at Ensembl.

MC: What are your next steps in this space?YK: I think there are more of these "hidden genes" to be found. The two important questions are i) where exactly are they? and ii) how are they read?

MC: On Twitter, you said "This finding was also made by another group at the exact same time, whose manuscript will be up shortly as well" Will your research group be looking to collaborate?YK: This is actually a funny story. I was at a conference in Germany last September and I was sitting in the audience listening to a talk. As my focus began to wane, I started leafing through the abstract book seeing what other research was going to be presented at the conference. Right in front of me on a completely random page I turned to was almost exactly my research. The only difference was that it was done by a completely different group. I panicked. However, after an email of advice from adviser, I decided to approach the group and we ended up agreeing on trying to coordinate our submissions. My work was accepted to a journal faster and hence they uploaded their manuscript to a preprint server after I told them my paper was up. The link for their work is here.

Yousuf Khan, PhD researcher at Stanford School of Medicine, was speaking to Molly Campbell, Science Writer, Technology Networks.

Reference: Khan et al. (2020). Evidence for a novel overlapping coding sequence in POLG initiated at a CUG start codon. BMC Genetics. DOI: https://doi.org/10.1186/s12863-020-0828-7.

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Novel Discovery of "Hidden" Gene Within a Gene in Mammals - Technology Networks