Category Archives: Genetic Therapy

For most parents, their child's second birthday is one of the many exciting milestones of toddlerhood - a chance to share a toast with friends and family and look forward to a future filled with possibilities.

For Queensland parents Kellee and Jamie Clarkson, it's a date that fills them with dread.

Their 18-month-old daughter Wynter has spinal muscular atrophy (SMA), a degenerative genetic disorder that attacks the motor neurons in her spine, progressively weakening her muscles and shutting down movement.

Without treatment, children like Wynter with type 1, the most severe form of the disease, will never sit up, crawl or walk and won't live to see their second birthday.

A revolutionary new gene therapy called Zolgensma is Wynter's best chance at a life free of wheelchairs, breathing apparatus and thrice-yearly lumbar punctures that leave her screaming for help but she only has until she turns two.

So far, the gene therapy is only available through health care in the United States, with Japan on Wednesday becoming the second country to approve the drug for children under two.

"It's such a long shot it would be like winning the lottery," Wynter's mum Kellee told 9News.

"But Jamie and I won't give up we will pursue every avenue we can."

The couple, who live just outside of Toowoomba, embarked on a fundraising drive last November in a desperate bid to raise the money, but the $66,000 they've raised falls far short of the jaw-dropping sum they need.

Their best hope now lies in a global lottery-style draw announced by drug's developers Novartis in December, offering 100 doses of the drug for free in 2020 to children under two years old.

But with roughly 60,000 children diagnosed worldwide every year, the chances of being drawn remain slim.

The first names have already been selected in a series of fortnightly draws, but the drug company is remaining tight-lipped on where the patients are located and how many children have missed out.

Swiss pharmaceutical company Novartis has attracted heavy criticism from patient advocates and health advisors for the lottery-style format of its global "managed access scheme".

UK patient group TreatSMA applauded the company's effort to offer the drug for free, but said it was "yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease".

For parents, competing against other desperate families to gain treatment at the expense of others also comes at a psychological cost.

"It plays with your emotions," Kellee said.

"There are children worse off than Wynter and there are children better off. Wynter is older, she doesn't have as much time, so it would be hard to see a child who is much younger get it.

"It's so hard that there is such a big amount of money involved to give your child the best life, and it's just by chance that your child could get this drug."

Novartis' Director of Communications and Advocacy, Peter Murphy, defended the managed access program as "anchored in principles of fairness, clinical need and global accessibility".

He told 9News the company had sought advice from bioethicists as well as doctors and patient advocates before launching the program, and they had concluded this was the best way to ensure equity regardless of country of residence and capacity to pay.

With only one facility currently approved to manufacture the drug, they simply can't produce enough doses quickly enough to provide one to every child.

As they wait and hope for a miracle, life for the Clarksons centres around a gruelling schedule of daily physio exercises, fortnightly occupational therapy and speech therapy visits and the daily battle to keep Wynter from getting sick.

"Wynter can't sit on her own, she can't crawl, she obviously can't walk... She needs help with everything. I would do it to the day I die, but it's so hard to watch other kids be able to do those things," mum Kellee said.

As well as impacting gross motor skills, SMA can affect the muscles used for swallowing and breathing, meaning Wynter needs a BiPAP machine when she sleeps and finds the seemingly simple task of eating exhausting.

"We spend a lot of time trying to get her enough nutrition. It takes a lot out of her to eat it's like running a marathon," Kellee explained.

"Wynter at the moment is really struggling with her weight gain and it is a real possibility that she will have to have a gastric tube put in."

With SMA affecting Wynter's ability to cough and expel mucus, even going outside and playing with other children can have life-threatening implications.

"We're constantly worried about her getting sick. I'm constantly thinking about 'oh I don't want to go to the shopping centre because she could get a cold and die'," Kellee said.

That almost became a reality last year, when Wynter caught the common cold and ended up in ICU for two weeks fighting for life.

"She required lots of deep suctioning to the lungs, they put a catheter down her nose and mouth and she was on a BiPAP 24/7," Kellee said.

"It was the most traumatic, distressing experience of my life and my husband's."

Currently, the only medication available to treat SMA in Australia is Spinraza, a drug that has halted the devastating progression of Wynter's disease but has failed to give her back her compromised lung and swallowing functions.

It also means hospital visits every four months for the rest of her life.

"She's 17-months-old and she's had seven lumbar punctures," Kellee said.

"They can't give them any anaesthetic and she has to have it awake."

"It is absolutely horrendous to watch. As a parent, it just absolutely crushes you to see your child in pain and looking at you and wondering why you're allowing this to happen."

Patient advocacy group SMA Australia's Julie Cini said she understood the Clarkson's desperation to access Zolgensma, but the harsh reality was that some children would miss out while the drug was pending approval in Australia.

"It's like dangling a carrot in front of someone and then chucking it in the bin," she said.

Ms Cini is currently working to have the gene therapy approved under the Pharmaceutical Benefits Scheme, which would allow every child in Australia to access the drug.

Novartis applied to the Therapeutical Drug Administration in late 2019, but approval could still take many months, even years.

While paralysing her body, SMA has left Wynter's mind untouched, and Kellee says she remains a bright and bubbly toddler with a brilliant sense of humour.

"She has a very funny personality, she's very bright. A very happy little girl loves to dance, loves to laugh. She's at that stage where she's getting cheeky in a good way.

"She's got a powered wheelchair she drives around perfectly she doesn't know any different.

"To us, she's just perfect."

SMA advocate Ms Cini said it was important to remember just how far treatment had come.

She knows all too well the gut-wrenching trauma of watching your child's slowly but inevitable progress towards death.

"I look at Wynter every single day and I see what I couldn't see in my children," Ms Cini said.

"What she can do is phenomenal my kids couldn't do what she's doing. My outcome was death. The (Clarksons) have a chance to have a life with their child."

Ms Cini is optimistic that new treatments and early detection could see a future where those with SMA, even in its most severe form, could live long and independent lives.

A national newborn screening program to detect SMA before symptoms take hold vital in limiting the disease's progress - is currently being piloted in NSW.

"Hopefully, when drugs like Zolgensma get passed, we're able to treat our kids within the first two weeks of life," she said.

"I can see the future of SMA, I'm going to walk in to a room and I won't even know which kid has it."

For Kellee and Jamie Clarkson, that day can't come soon enough.

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Parents' fight to access life-changing $3.2m gene therapy for their daughter - 9News

Rare Disease Day is celebrated across the globe to raise awareness amongst the general public and policymakers about rare diseases and how they impact patients lives. The first Rare Disease Day was celebrated in 2008 on February 29 because of its rare date and since then, occurs on the last day in February each year, a month with a rare number of days.

Although rare suggests not many people are affected with a condition, collectively, 300 million people around the world live with a rare disease and they face similar challenges. The barrier to an accurate diagnosis means patients may doctor hop and spend years getting a host of tests done because no one is familiar with the condition and can diagnose it. Theres often frustration due to this lack of understanding from health care professionals, and living in the unknown.

Even with a diagnosis, more than 90% of rare diseases are still without an FDA approved treatment, according to the National Organization for Rare Disorders.

Some rare diseases may cause a multitude of different health problems that keep children from going to school or being able to socialize with others in the same way their peers can. Similarly, rare diseases may affect physical appearance and make children self-conscious or have low self-esteem.

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Michigan Medicine researchers are constantly working to better understand the mechanisms behind rare diseases. Education helps health care professionals make accurate diagnoses, create treatment methods and improve the quality of life for those that live with these conditions.

Heres a sampling of their research from the last year.

Systemic Scleroderma Treatments: Where Are We Now?A new and novel outcome measure is being used to determine effectiveness of new scleroderma treatments.

Gene Therapy Treatment Targets Rare Mutation Tied to BlindnessAdvances in gene therapy are yielding new options for treating inherited retinal degenerations, giving specialists new tools and new hope for patients and families.

A Mission to Improve Cystic Fibrosis Treatment Across the GlobeTo reach patients in need, one doctor has developed atraining program to improve testingand care available for those with thegenetic disease, starting intheMiddle East.

Accelerating Childrens Access to New Treatments for High Risk Brain TumorsMichigan Medicine joins an exclusive, global network that helps speed up the process of linking children with incurable brain cancer to promising clinical trials.

A New Clue in the Mystery of ALS, Frontotemporal DementiaMichigan Medicine researchers identify a potential therapeutic target for neurodegenerative conditions using animal models.

Drug Trial Seeking First Ever Treatment for Dangerous Side Effect of Prader-Willi SyndromeA worldwide research effort is underway for finding a treatment option for hyperphagia, the most common genetic cause of life threatening childhood obesity.

Arthritis Treatment Could Provide Relief for Lichen Planus Skin RashIts often difficult to manage patients with this skin inflammation, but new research identifies a target that existing medications may be able to address.

Approach Could Help in Treating Glioblastoma, Other Rare CancersMichigan led research presents a new way of uncovering predictive biomarkers when data from large randomized trials arent available.

Sickle Cell Disease Could Be Treated by Turning Back the ClockReactivating genes normally active before birth could prevent the harmful effects of this blood disorder with few treatment options.

Registry Helps Move Aortic Dissection Care Forward Diagnosis, treatments and outcomes for acute aortic dissection have evolved, with an international registry revealing trends and the power of using data.

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To learn more about Rare Disease Day, visit the National Organization for Rare Disorders website.

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10 Studies That Highlight the Importance of Rare Disease Research - Michigan Medicine

A Japanese health ministry panel meets in Tokyo on Feb. 26, 2020, to discuss whether or not to approve production and sales in Japan of Zolgensma, a gene therapy medication used to treat spinal muscular atrophy. (Kyodo)

TOKYO (Kyodo) -- A health ministry panel approved Wednesday the production and sale in Japan of a medication thought to be the most expensive in the world that is used to treat a rare childhood genetic disorder.

The drug, Zolgensma, to be produced and sold by Novartis Pharma K.K., the Tokyo-based unit of Swiss pharmaceutical giant Novartis AG, will get the official green light from the Ministry of Health, Labor and Welfare in March.

The medication carries a price tag of $2.1 million in the United States, where it was approved by the Food and Drug Administration in May last year.

It is a one-time therapy for spinal muscular atrophy, a genetic disorder that causes motor neuron loss and muscle wasting. The disorder affects about two infants out of every 100,000 and can lead to severe respiratory problems and early death.

It will be the second gene therapy drug approved in Japan. Once officially approved, the medication would be covered by national health insurance as early as May.

The drug will be used to treat children less than two years of age and Novartis calculates it will be administered to 15 to 20 individuals per year in Japan.

With side effects including heart damage and liver problems reported during clinical testing, the company will observe patients' health and check the drug's safety and effectiveness.

Following the revelation last year of data manipulation during animal testing, the health ministry said it had examined other experimental data to confirm the safety of the medication.

See the original post here:
Japan gov't panel OKs world's most expensive drug used in gene therapy - The Mainichi

Alia (2nd from left) and her father (left) with Dr Arif Khan (3rd from left), paediatric ophthalmologist and ocular geneticist at Cleveland Clinic Abu Dhabi, and other hospital doctors at a press conference in Abu Dhabi. Alia and her sister, Hessa, were the first to receive gene therapy in the UAE. Picture by Samihah Zaman, Image Credit:

Abu Dhabi: Two Emirati sisters from Abu Dhabi have become the first to receive gene therapy in the UAE, it was announced in the capital today.

The therapy was conducted with the aim of saving the vision of Alia, 13, and Hessa, 11, who suffered from retinal damage due to a defective gene.

The vision-saving surgery was performed at Cleveland Clinic Abu Dhabi in January (2020), and doctors said at a press conference that they are hopeful it will slowly improve the vision of both patients.

"The prospect of slowly losing one's vision from an untreatable condition is traumatic for both children and their parents. This genetic therapy means we can now replace the faulty gene in the eye, saving and even improving the vision of an individual who would otherwise have eventual irreversible blindness," said Dr Arif Khan, paediatric ophthalmologist and ocular geneticist at the hospital.

Only centre in region with procedure

Cleveland Clinic Abu Dhabi is only one of 10 centres worldwide, and the only one in the region, that is qualified to offer this procedure - the first gene therapy that was approved by the Food and Drug Administration in the United States, doctors said. But its availability opens the doors for more gene therapies to be offered to treat conditions as varied as diabetes and spinal muscular atrophy, they added.

Weve travelled abroad multiple times to find some treatment for our daughters, and this procedure has been a godsend. It has only been a month since the therapy however, and we are hoping for much more improvement to their vision over time, Fatima, the girls mother, told Gulf News.

The procedure

Alia and Hessa were born with RPE65-related retinal dystrophy, a genetic dystrophy in which the RPE65 protein is lacking because a child inherits two copies of the defective RPE65 from both parents. It is a recessive disease and therefore rare, and known to affect one in 200,000 people worldwide. But the condition is also more common in the region because of the limited genetic pool and cultural preferences for consanguineous marriages.

Patients with the condition face gradual damage to their retinas, the photosensitive layer at the back of the eye, and could eventually end up with irreversible blindness.

The gene therapy to treat the condition uses a vector - a bioengineered non-pathogenic virus to deliver normal copies of the RPE65 gene to the eye.

The procedure takes only about an hour, but it is very delicate. The retina can be considered an offshoot of the brain because its cells are very thin, like brain tissue. We use advanced microscopes to create openings in the white of the eye, then use a specialized canula to reach under the retina and inject the vector into the specific area, which can be as small as one-tenth of a millimeter, explained Dr Emad Abboud, chief of the department of the posterior segment at the hospitals Eye Institute.

Post-surgical recovery takes only about a week, but outcomes take a while to become obvious.

As Dr Khan explained, the main benefits for patients receiving the therapy is an improvement of vision in low-light conditions, and an improvement of the visual field.

Vision is a complex sense, involving photosensitivity, visual field, visual acuity, colour sensitivity, motion sensitivity, and the ability to navigate. But [at the least], this therapy prevents the progressive deterioration of the retina, he explained.

What the family said

My handwriting has definitely gotten better, Alia told Gulf News when asked about how the therapy has helped.

I cant tell what else will get better but I already feel that this has been life-changing, she added.

According to Fatima, she noticed her daughters had visual impairment when they were as young as two months old.

The doctor pointed out to me that they were not focusing or making eye contact. So we were aware of the condition. It definitely made life very challenging, especially as their development was delayed, the mother explained.

Alia and Hessa both crawled and walked late, and have needed assistance getting around. While they kept pace with their peers at school, it took Hercualean efforts from both girls, their parents, shadow teachers and at-home tutors, and required the use of Braille and visual aid resources.

Fatima and her husband, Mubarak, kept looking for treatments for both girls, travelling to Germany, India and Spain.

In 2016, we visited the Cleveland Clinic Abu Dhabi, and it was then that we heard of this revolutionary treatment. It was still not approved by the FDA, but we kept dreaming, she said.

It has been a month since the surgery and the doctors said they are more photosensitive now and that this is a good sign. Of course, as a mother, I would love for them to eventually have complete vision, Fatima added.

What is the therapy?

The UAE Ministry of Health and Prevention (MoHAP) approved the gene therapy for RPE65-related retinal dystrophy, Luxturna, in June 2019.

In a statement released at the time, the MoHAP said its registration of Luxturna was only the third global registration of the drug at the health authority level, and was aimed at paving the way for future gene-based therapies for complex conditions.

The United States Food and Drug Administration only approved Luxturna, developed by gene therapy developer, Spark Therapeutics, and the Childrens Hospital of Philadelphia, in late 2017.

The gene therapy to treat the condition uses a vector - a bioengineered non-pathogenic virus to deliver normal copies of the RPE65 gene to the eye with the defective gene.

While the Cleveland Clinic Abu Dhabi did not provide the cost of treatment, international media reports put it at $425,000 (Dh1.56 million) per eye.

Benefits of gene therapy

Dr Khan said at the press conference that us familiar with a few more families with the RPE65-related retinal disorder.

Over time, gene therapy could be used to treat many conditions. The eye is uniquely suited for these therapies because it is a self-contained organ. But at least five other genetic therapies are in development, some at the human trial level, he explained.

Consider gene delivery to be like providing a protein factory to the body. So for instance, some diabetic patients require regular insulin injections. If a gene could be engineered to make insulin, and it could be delivered to the patient, he wouldnt need these regular injections, Dr Khan added.

Excerpt from:
UAE's first gene therapy improves eyesight of two Emirati sisters - Gulf News

PARIS--(BUSINESS WIRE)--Regulatory News:

Lysogene (Paris:LYS) (FR0013233475 LYS), a pioneering Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its LYS-SAF302 program for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA). LYS-SAF302, a second-generation gene therapy, is designed to deliver a functional copy of the SGSH (N-sulfoglucosamine sulfohydrolase) gene to the brain through a one-time direct-to-CNS administration, and is being investigated in the international Phase 2/3 clinical trial AAVance (NCT03612869).

Karen Aiach, Founder and Chief Executive Officer said: MPS IIIA is a lethal neurological disease with debilitating symptoms for which there is currently no treatment. The FDAs recognition of LYS-SAF302s potential to improve neurocognitive deficits in children with MPS IIIA represents an important achievement for Lysogene and the patient community. Karen Aiach added: We are also pleased to announce the treatment of the 17th patient with LYS-SAF302, which once again demonstrates our capacity to execute with quality and speed. LYS-SAF302 is the flagship of our differentiated direct-to-CNS gene therapy platform, which has been validated not only by our partner Sarepta, but also by the scientific community. We are fully dedicated to continue the full speed development of LYS-SAF302, as well as all the other programs in our pipeline.

The Fast Track program facilitates the development and accelerates the review of new drugs for serious conditions, which have the potential to address unmet medical needs. The purpose is to expedite the availability of new treatment options for patients. A product that receives Fast Track designation is eligible for more frequent interactions with FDA, potential eligibility for accelerated approval, priority review, and rolling Biologics License Application (BLA) review.

This Fast Track designation demonstrates the regulators sustained interest in Lysogenes cutting edge in vivo gene therapy program. LYS-SAF302 has previously received Orphan Drug Designations for the treatment of MPS IIIA in the European Union in 2014 and in the US in 2015, as well as Rare Pediatric Disease Designation in the US, added Marie Deneux, Chief Regulatory Officer. In the complex field of gene therapy for neurodegenerative diseases, a continued communication with FDA is essential.

The AAVance Phase 2/3 clinical study is designed as an open-label, single-arm, multicenter study of LYS-SAF302 for the treatment of MPS IIIA. The study will include 20 patients with the severe classical form of MPS IIIA and has been extensively discussed upfront with key opinion leaders, regulators and health technology assessment bodies, as well as with patient representatives. As of today, 17 patients have been treated out of the total of 20. The primary objective is to assess the drug efficacy in improving the neurodevelopmental status of patients after 24 months, compared to the expected evolution based on natural history data. Safety, tolerability, effect on behavior, sleep and quality of life will also be collected as secondary endpoints. Lysogene has also set up the sub study, PROVide, collecting supportive video outcomes in the home environment.

About MPS IIIA

MPS IIIA is a rare inherited neurodegenerative lysosomal storage disorder affecting approximately 1 in 100,000 newborns. Inherited in an autosomal recessive pattern, it is characterized by intractable behavioral problems and developmental regression resulting in early death. It is caused by mutations in the SGSH gene, which encodes an enzyme called Heparan-N-sulfamidase necessary for heparan sulfate (HS) recycling in cells. The disrupted lysosomal degradation and resulting storage of HS and glycolipids such as gangliosides leads to severe neurodegeneration. There are currently no treatment options for patients.

About LYS-SAF302

LYS-SAF302 is an AAV-mediated gene therapy, the goal of which is to replace the faulty SGSH gene with a healthy copy of the gene. LYS-SAF302 employs the AAVrh10 virus, chosen for its ability to target the central nervous system. Proof-of-concept was established in MPS IIIA pre-clinical models demonstrating strong expression, broad distribution, and the ability of the product to correct lysosomal storage defects by producing the missing enzyme. Safety data from an IND-enabling toxicity and a biodistribution GLP study showed that, at any dose level evaluated, LYS-SAF302 was not associated with unexpected mortality, change in clinical signs, body weight, behavior or macroscopic findings in the brain. Sarepta holds exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene maintains commercial exclusivity of LYS-SAF302 in Europe.

About Lysogene

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/2 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

Forward Looking Statement

This press release may contain certain forward-looking statements, especially on the Companys progress of its phase 2-3 clinical trial and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2018 registration document (Document de rfrence), registered with the French Markets Authorities on April 29, 2019, under number R. 19-016, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA - Business Wire

The argument that excessive regulation adversely affects small and medium enterprises (SMEs) does not stand up to scrutiny, according to molecular geneticist Dr Michael Antoniou, head of the gene expression and therapy group at Kings College London.

The regulatory approval process for new biotech crop varieties is often said to be unduly slow and expensive, presenting an important barrier for biotech SMEs.

The chair of the agriculture committee (AGRI) MEP, Norbert Lins, recently told EURACTIV.com that it is much easier for larger companies to implement new GM legislation, but its the smaller ones that are most affected by the recent gene-editing ruling.

Likewise, the European Association for Bioindustries (EuropaBio) told EURACTIV that restrictive regulation is pushing out SMEs, encouraging them to instead focus their research efforts on other parts of the world.

It is much easier for larger companies to implement new GM legislation, but its the smaller ones that are most affected by the recent gene-editing ruling, the chair of the agriculture committee (AGRI) MEP, Norbert Lins, told EURACTIV.com at the sidelines of a recent plant breeding conference.

However, speaking at a recent conference on gene editing and seed rights, Antoniou said that, contrary to popular belief, excessive regulation is not the limiting factor for SMEs entering the gene editing market.

This event, organised by the European Coordination Via Campesina (ECVC) in collaboration with the European Economic and Social Committee, brought together academics, experts from the European patent office and farmers from Europe. They debated the impacts of patents on the seed markets, as well as the potential impacts in Europe if the legislative framework on GMOs may be changed.

Citing a report from industry consultancy firm Phillips McDougal, Antoniou highlighted that, of the average $136 million cost of bringing a GM trait to market between 2008 and 2012, only 25% was found to be related to regulatory issues.

The remaining 75% went towards research and development costs. In addition to these, he said that with CRISPR gene-edited plants, there will also be onerous, very expensive licencing arrangements and royalty fees paid to CRISPR gene editing patent holders.

Flawed financial strategies and failure to attract right investment, inexperienced management and mediocre science were instead named as the main reasons that the vast majority of biotech SMEs fail.

Antoniou therefore concluded that deregulation of gene editing would have a limited effect on SMEs, given that the development of gene-edited plants will take a similar input of resources and time, regardless of more lenient regulation.

The discussion over the future of biotechnology in Europe heated up after the EU Court ruled in July last year that gene editing should, in principle, fall under the GMO Directive. We focus on the future of biotechnology in Europe, the regulatory framework of the so-called new plant-breeding techniques, as well as GMOs.

Drawing on his personal experience in developing biotech products in both start-ups and larger companies, Antoniou also stressed that takeovers of SMEs by larger biotech companies are not only common but often actively sought after by SMEs as the quickest way to expand their portfolio and turn a profit.

When an SME, which is set up by venture capital, builds a good patent portfolio, it is frequently taken over by a larger company. This is exactly what happened to an SME that I worked with for many years, specialising in gene therapy and biomanufacturing applications, he told EURACTIV.

However, far from being a cause for lamentation, it was heralded as a success by all concerned: the venture capitalists obtained a quicker, profitable return on their investment and greater resources became available for further research and development.

He added that this is standard business practice in the SME biotechnology sector and ultimately results in the concentration of patent ownership and products in a few large companies.

Guy Kastler of the seeds working group of the ECVC concurred, telling EURACTIV that when a start-up registers a patent to a product from a new genetic engineering process, it has very little chance of survival; the most likely outcome is that it is bought out by a multinational company.

In fact, most small and medium-sized seed companies have disappeared from the market as a result.

He added that the deregulation of GMOs would aggravate this trend at the European level, and that the big losers will be farmers and consumers.

[Edited by Zoran Radosavljevic]

Excerpt from:
Gene-editing regulation not the biggest hurdle for SMEs in EU, says academic - EURACTIV