Search Immortality Topics:

Page 102«..1020..101102103104..110120..»


Category Archives: Gene Medicine

AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome – Associated Press

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.

Media Contacts: AskBio Robin Fastenau Vice President, Communications +1 984.275.2705 rfastenau@askbio.com Angelman Syndrome Foundation Amanda Moore Chief Executive Officer +1 317.514.6918 amoore@angelman.org UNC Health | UNC School of Medicine Mark Derewicz Director, Research & News +1 984.974.1915 Mark.Derewicz@unchealth.unc.edu

The rest is here:
AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome - Associated Press

Posted in Gene Medicine | Comments Off on AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome – Associated Press

Fat Busting Proteins Produced In the Laboratory – Technology Networks

Triglycerides, those fats that seem to be the bane of any diet, remain a mystery for many researchers. Plenty of them are in Big Macs, deep pan pizza and the like, but some are a necessity to fuel the body for daily activities.Researchers Mark Castleberry, a doctoral student, and professor Sean Davidson, both in the UC College of Medicine, have found a way to produce in the laboratory a human protein produced in the liver known as Apolipoprotein A5 (APOA 5). It plays an important role in metabolizing and clearing excess levels of triglycerides from the bloodstream.

We are really interested in understanding triglycerides because hypertriglyceridemia too much fat in your blood is a big factor leading to cardiovascular disease, diabetes, obesity and other health concerns, explains Davidson, who holds appointments in UCs departments of Pathology and Laboratory Medicine and Molecular Genetics, Biochemistry and Microbiology. When you have a lot of fat that is hanging around in your circulation its important to clear as much of it out as soon as possible.

APOA5 is highly involved in how fast triglycerides get taken out of your circulation, says Davidson, who has a doctorate in biochemistry. The more APOA5 you have the faster the triglyceride is removed. Everybody agrees it is an important protein but scientists dont know much about its structure or how it does what it does. If we could figure out how it works we could come up with a drug that uses the same mechanism or trigger it to work better.

Castleberry says researchers inserted a human gene coded by DNA into bacteria genetically engineered to produce human proteins. Once those proteins were produced they were removed from the host and purified for use in studies at the lab bench and in mouse models.

We can quickly make a much greater amount of this protein using bacterial production than if we tried to isolate it from blood in humans, explains Castleberry. The mice in this study were basically fed a large bowl of fat and triglycerides.

We could analyze their blood after we fed them and observe the level of fat change as they digested the meal, said Castleberry. We were able to give our protein to the mice that had that fatty meal and rapidly clear the triglycerides that would have accumulated in their blood."ReferenceCastleberry et al. (2020) Functional recombinant apolipoprotein A5 that is stable at high concentrations at physiological pH. The Journal of Lipid Research. DOI: https://doi.org/10.1194/jlr.D119000103

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Read more:
Fat Busting Proteins Produced In the Laboratory - Technology Networks

Posted in Gene Medicine | Comments Off on Fat Busting Proteins Produced In the Laboratory – Technology Networks

AI Discovers Smell Genes Linked To Cancer Outcomes – Unite.AI

The AI community must collaborate with geneticists, in finding a treatment for those deemed most at risk of coronavirus. A potential treatment could involve removing a persons cells, editing the DNA and then injecting the cells back in, now hopefully armed with a successful immune response. This is currently being worked on for some other vaccines.

The first step would be sequencing the entire human genome from a sizeable segment of the human population.

Sequencing Human Genomes

Sequencing the first human genome cost $2.7 billion and took nearly 15 years to complete. The current cost of sequencing an entire human has dropped dramatically. As recent as 2015 the cost was $4000, now the cost is less than $1000 per person. This cost could drop a few percentage points more when economies of scale are taken into consideration.

We need to sequence the genome of two different types of patients:

It is impossible to predict which data point will be most valuable, but each sequenced genome would provide a dataset. The more data the more options there are to locate DNA variations which increase a bodys resistance to the disease vector.

Nations are currently losing trillions of dollars to this outbreak, the cost of $1000 a human genome is minor in comparison. A minimum of 1,000 volunteers for both segments of the population would arm researchers with significant volumes of big data. Should the trial increase in size by one order of magnitude, the AI would have even more training data which would increase the odds of success by several orders of magnitude. The more data the better, which is why a target of 10,000 volunteers should be aimed for.

Machine Learning

While multiple functionalities of machine learning would be present, deep learning would be used to find patterns in the data. For instance, there might be an observation that certain DNA variables correspond to a high immunity, while others correspond to a high mortality. At a minimum we would learn which segments of the human population are more susceptible and should be quarantined.

To decipher this data an Artificial Neural Network (ANN) would be located on the cloud, and sequenced human genomes from around the world would be uploaded. With time being of the essence, parallel computing will reduce the time required for the ANN to work its magic.

We could even take it one step further and use the output data sorted by the ANN,and feed it into a separate system called a Recurrent Neural Network (RNN). The RNN uses reinforcement learning to identify which gene selected by the initial ANN is most successful in a simulated environment. The reinforcement learning agent would gamify the entire process of creating a simulated setting, to test which DNA changes are more effective.

A simulated environment is like a virtual game environment, something many AI companies are well positioned to take advantage of based on their previous success in designing AI algorithms to win at esports. This includes companies such DeepMind and OpenAI.

These companies can use their underlying architecture optimized at mastering video games, to create a stimulated environment, test gene edits, and learn which edits lead to specific desired changes.

Once a gene is identified, another technology is used to make the edits.

CRISPR

Recently, the first ever study using CRISPR to edit DNA inside the human body was approved. This was to treat a rare type of genetic disorder that effects one of every 100,000 newborns. The condition can be caused by mutations in as many as 14 genes that play a role in the growth and operation of the retina. In this case, CRISPR sets out to carefully target DNA and to cause slight temporary damage to the DNA strand, causing the cell to repair itself. It is this restorative healing process which has the potential to restore eyesight.

While we are still waiting for results on if this treatment will work, the precedent of having CRISPR approved for trials in the human body is transformational. Potential disorders which can be treated include improving a bodys immune response to specific disease vectors.

Potentially, we can manipulate the bodys natural genetic resistance to a specific disease. The diseases that could potentially be targeted are diverse, but the community should be focusing on the treatment of the new global epidemic coronavirus. A threat that if unchecked could lead to a death sentence to a large percentage of our population.

FINAL THOUGHTS

While there are many potential options to achieving success, it will require that geneticists, epidemiologists, and machine learning specialists unify. A potential treatment option may be as described above, or may be revealed to be unimaginably different, the opportunity lies in the genome sequencing of a large segment of the population.

Deep learning is the best analysis tool that humans have ever created; we need to at a minimum attempt to use it to create a vaccine.

When we take into consideration what is currently at risk with this current epidemic, these three scientific communities need to come together to work on a cure.

Read the original here:
AI Discovers Smell Genes Linked To Cancer Outcomes - Unite.AI

Posted in Gene Medicine | Comments Off on AI Discovers Smell Genes Linked To Cancer Outcomes – Unite.AI

Coronavirus Treatment: Hundreds of Scientists Scramble to Find One – The New York Times

Working at a breakneck pace, a team of hundreds of scientists has identified 50 drugs that may be effective treatments for people infected with the coronavirus.

Many scientists are seeking drugs that attack the virus itself. But the Quantitative Biosciences Institute Coronavirus Research Group, based at the University of California, San Francisco, is testing an unusual new approach.

The researchers are looking for drugs that shield proteins in our own cells that the coronavirus depends on to thrive and reproduce.

Many of the candidate drugs are already approved to treat diseases, such as cancer, that would seem to have nothing to do with Covid-19, the illness caused by the coronavirus.

Scientists at Mount Sinai Hospital in New York and at the Pasteur Institute in Paris have already begun to test the drugs against the coronavirus growing in their labs. The far-flung research group is preparing to release its findings at the end of the week.

There is no antiviral drug proven to be effective against the virus. When people get infected, the best that doctors can offer is supportive care the patient is getting enough oxygen, managing fever and using a ventilator to push air into the lungs, if needed to give the immune system time to fight the infection.

If the research effort succeeds, it will be a significant scientific achievement: an antiviral identified in just months to treat a virus that no one knew existed until January.

Im really impressed at the speed and the scale at which theyre moving, said John Young, the global head of infectious diseases at Roche Pharma Research and Early Development, which is collaborating on some of the work.

We think this approach has real potential, he said.

Some researchers at the Q.B.I. began studying the coronavirus in January. But last month, the threat became more imminent: A woman in California was found to be infected although she had not recently traveled outside the country.

That finding suggested that the virus was already circulating in the community.

I got to the lab and said weve got to drop everything else, recalled Nevan Krogan, director of the Quantitative Biosciences Institute. Everybody has got to work around the clock on this.

Dr. Krogan and his colleagues set about finding proteins in our cells that the coronavirus uses to grow. Normally, such a project might take two years. But the working group, which includes 22 laboratories, completed it in a few weeks.

You have 30 scientists on a Zoom call its the most exhausting, amazing thing, Dr. Krogan said, referring to a teleconferencing service.

Viruses reproduce by injecting their genes inside a human cell. The cells own gene-reading machinery then manufactures viral proteins, which latch onto cellular proteins to create new viruses. They eventually escape the cell and infect others.

In 2011, Dr. Krogan and his colleagues developed a way to find all the human proteins that viruses use to manipulate our cells a map, as Dr. Krogan calls it. They created their first map for H.I.V.

That virus has 18 genes, each of which encodes a protein. The scientists eventually found that H.I.V. interacts, in one way or another, with 435 proteins in a human cell.

Dr. Krogan and his colleagues went on to make similar maps for viruses such as Ebola and dengue. Each pathogen hijacks its host cell by manipulating a different combination of proteins. Once scientists have a map, they can use it to search for new treatments.

In February, the research group synthesized genes from the coronavirus and injected them into cells. They uncovered over 400 human proteins that the virus seems to rely on.

The flulike symptoms observed in infected people are the result of the coronavirus attacking cells in the respiratory tract. The new map shows that the viruss proteins travel throughout the human cell, engaging even with proteins that do not seem to have anything to do with making new viruses.

One of the viral proteins, for example, latches onto BRD2, a human protein that tends to our DNA, switching genes on and off. Experts on proteins are now using the map to figure out why the coronavirus needs these molecules.

Kevan Shokat, a chemist at U.C.S.F., is poring through 20,000 drugs approved by the Food and Drug Administration for signs that they may interact with the proteins on the map created by Dr. Krogans lab.

Dr. Shokat and his colleagues have found 50 promising candidates. The protein BRD2, for example, can be targeted by a drug called JQ1. Researchers originally discovered JQ1 as a potential treatment for several types of cancer.

On Thursday, Dr. Shokat and his colleagues filled a box with the first 10 drugs on the list and shipped them overnight to New York to be tested against the living coronavirus.

The drugs arrived at the lab of Adolfo Garcia-Sastre, director of the Global Health and Emerging Pathogens Institute at Mount Sinai Hospital. Dr. Garcia-Sastre recently began growing the coronavirus in monkey cells.

Over the weekend, the team at the institute began treating infected cells with the drugs to see if any stop the viruses. We have started experiments, but it will take us a week to get the first data here, Dr. Garcia-Sastre said on Tuesday.

The researchers in San Francisco also sent the batch of drugs to the Pasteur Institute in Paris, where investigators also have begun testing them against coronaviruses.

If promising drugs are found, investigators plan to try them in an animal infected with the coronavirus perhaps ferrets, because theyre known to get SARS, an illness closely related to Covid-19.

Even if some of these drugs are effective treatments, scientists will still need to make sure they are safe for treating Covid-19. It may turn out, for example, that the dose needed to clear the virus from the body might also lead to dangerous side effects.

This collaboration is far from the only effort to find an antiviral drug effective against the coronavirus. One of the most closely watched efforts involves an antiviral called remdesivir.

In past studies on animals, remdesivir blocked a number of viruses. The drug works by preventing viruses from building new genes.

In February, a team of researchers found that remdesivir could eliminate the coronavirus from infected cells. Since then, five clinical trials have begun to see if the drug will be safe and effective against Covid-19 in people.

Other researchers have taken startling new approaches. On Saturday, Stanford University researchers reported using the gene-editing technology Crispr to destroy coronavirus genes in infected cells.

As the Bay Area went into lockdown on Monday, Dr. Krogan and his colleagues were finishing their map. They are now preparing a report to post online by the end of the week, while also submitting it to a journal for publication.

Their paper will include a list of drugs that the researchers consider prime candidates to treat people ill with the coronavirus.

Whoever is capable of trying them, please try them, Dr. Krogan said.

See the article here:
Coronavirus Treatment: Hundreds of Scientists Scramble to Find One - The New York Times

Posted in Gene Medicine | Comments Off on Coronavirus Treatment: Hundreds of Scientists Scramble to Find One – The New York Times

Abeona Therapeutics Announces First Patient Treated in Pivotal Phase III Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic…

Majority of potential study participants have been pre-screened

EB-101 successfully manufactured at Abeona and transplanted at Stanford University Medical Center

NEW YORK and CLEVELAND, March 17, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that investigators at Stanford University Medical Center have treated the first patient in the pivotal phase III VIITAL study evaluating EB-101, the Companys gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB).

Treating the first patient in our pivotal Phase III VIITAL study is an important achievement for the EB-101 program, now the most advanced gene therapy program in RDEB, said Joo Siffert, M.D., Chief Executive Officer. This achievement confirms that Abeona can deliver EB-101 in a study setting that closely parallels its potential real-world application. We remain confident that VIITALTM will replicate results from the Phase I/II trial demonstrating that EB-101 treatment resulted in sustained and durable wound healing with a favorable safety profile.

The VIITALPhase III study is a multi-center, randomized clinical trial assessing EB-101 in up to 15 RDEB patients, with approximately 30 large, chronic wound sites treated in total. The primary outcome measure is wound healing, comparing treated with untreated wound sites on the same patient.Secondary endpoints include the assessments of pain, as well as other patient reported outcomes. Investigators at Stanford University Medical Center are currently enrolling eligible patients into the VIITALTM study and preparations for an additional clinical site initiation are ongoing. Additional information about the trial is available at abeonatherapeutics.com/clinical-trials/rdeb.

Abeona is producing EB-101 for the VIITALTM study at the Elisa Linton Center for Rare Disease Therapies, its fully-functional gene and cell therapy manufacturing facility centrally-located in Cleveland, OH. The 26,000 ft2 center is housing large-scale cGMP capacity for AAV gene therapy and EB-101 cell therapy manufacturing, and state-of-the-art laboratories to support CMC development for process and analytics, all of which is validated and governed by comprehensive quality systems and overseen by experienced staff.

About EB-101EB-101 is an autologous, gene-corrected cell therapy in late-stage clinical development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patients own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Data from a Phase I/IIa clinical trial conducted by Stanford University evaluating EB-101 showed that the gene-corrected cell therapy provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.

About Recessive Dystrophic Epidermolysis BullosaRecessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Companys clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Companys portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). http://www.abeonatherapeutics.com

Forward Looking StatementThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Companys clinical trials, including the timing and success thereof; the Companys products and product candidates; EB-101 can provide durable healing in large, chronic wounds that afflict many RDEB patients; future regulatory interactions with regulatory authorities; and the Companys goals and objectives. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Companys Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise these forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

Investor Contact:Dan FerryLifeSci Advisors, LLC+1 (617) 535-7746daniel@lifesciadvisors.com

Media Contact:Scott SantiamoDirector, Corporate CommunicationsAbeona Therapeutics+1 (718) 344-5843ssantiamo@abeonatherapeutics.com

See more here:
Abeona Therapeutics Announces First Patient Treated in Pivotal Phase III Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic...

Posted in Gene Medicine | Comments Off on Abeona Therapeutics Announces First Patient Treated in Pivotal Phase III Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic…

Thinking out loud: IP strategies for gene therapy inventions – Med-Tech Innovation

Reuben Jacob and Fiona Kellas, Maucher Jenkins share their expertise on IP strategies and considerations for gene therapy inventions.

Gene therapy enables the treatment of a disorder or disease through the insertion of a gene into a patients cells instead of using drugs or surgery.This technique involves the introduction of genetic material into cells to compensate for abnormal genes in the patient or to make protein that will be beneficial to the patient.As an example, if a mutated gene causes a protein that is necessary for the correct functioning of cells to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.Gene therapy is understood to be useful in the treatment of a range of conditions such as cancer, cystic fibrosis, muscular dystrophy and Alzheimers disease.

UK role in gene therapy techR&D

Gene therapy is considered to be very important to the future of medicine and as such, many companies are focussing their research and development into gene therapy technologies.The UK is a growing industry for research into these areas and it is anticipated that by 2035 the UK industry around cell and gene therapy technologies will be worth in the region of 10 billion.Gene therapy research is still at an early stage.Due to this length of time and the associated costs involved in developing an effective gene therapy and taking it through to approval, it will be important for companies working in this area to put into place an effective IP strategy that will provide protection for their inventions and assist them in maintaining their market position.In addition, the competitive nature of the gene therapy industry means that will be important for a company to obtain patent protection for inventions being developed, as well as reviewing the patent landscape to check that the company is free to operate in their chosen area.

What makes something patentable?

In order for an invention to be patentable, it must be new, inventive and capable of industrial application.In addition to the requirement that an invention meets the above requirements of patentability, it is also important that the invention does not contain subject matter that is excluded from patentability.One of the challenges associated with obtaining patent protection for gene therapy inventions is that the European and US patent systems include a number of exceptions to patentability that are relevant to biological material and natural products.In Europe, it is not possible to obtain patent protection for a method of treatment or surgery of the human body.Thus, the removal of cells from a patient would not be considered to be patentable in Europe.In addition, inventions relating to stem cells that are derived from the destruction of human embryos are not patentable in Europe.In the US, recent case law (Molecular Pathology v Myriad Genetics, Inc, 2013) has meant that inventions relating to natural phenomena and natural products must show characteristics that are different to their natural counterpart(s).

However, despite the above challenges, there are a number of aspects of the gene therapy technology that may be eligible for patent protection.Typically, the gene therapy procedure can involve performing the required modification procedure on cells that have been removed from a patient before reintroducing the cells into the subject to produce their modified effect.The process of modifying the cells may be patentable if it fulfils the above requirements of patentability.In addition, it may be possible to obtain protection for the methods that are used to culture, manipulate or modify the cells that are used for gene therapy.

At Maucher Jenkins, we have a team of attorneys who can provide IP advice and assistance in the area of patenting inventions involving gene therapy, molecular biology and biochemistry.

by Fiona Kellas, Reuben Jacob

16 March 2020

14:20

Original post:
Thinking out loud: IP strategies for gene therapy inventions - Med-Tech Innovation

Posted in Gene Medicine | Comments Off on Thinking out loud: IP strategies for gene therapy inventions – Med-Tech Innovation