Category Archives: Genetic Therapy

Discover how gene therapy can treat diseases caused by genetic mutations such as cystic fibrosisGene therapy seeks to repair genetic mutations through the introduction of healthy, working genes.Encyclopdia Britannica, Inc.See all videos for this article

Gene therapy, also called gene transfer therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype.

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cancer: Gene therapy

Knowledge about the genetic defects that lead to cancer suggests that cancer can be treated by fixing those altered genes. One strategy

Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome. Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germline gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If that is achieved, those cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germline gene therapy has been achieved experimentally in animals but not in humans.

Scientists have also explored the possibility of combining gene therapy with stem cell therapy. In a preliminary test of that approach, scientists collected skin cells from a patient with alpha-1 antitrypsin deficiency (an inherited disorder associated with certain types of lung and liver disease), reprogrammed the cells into stem cells, corrected the causative gene mutation, and then stimulated the cells to mature into liver cells. The reprogrammed, genetically corrected cells functioned normally.

Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. Few clinical trials of gene therapy in humans have satisfied all those conditions, often because the delivery system fails to reach cells or the genes are not expressed by cells. Improved gene therapy systems are being developed by using nanotechnology. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed.

Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Some objections to gene therapy are based on the view that humans should not play God and interfere in the natural order. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Benefits, however, would also be passed on indefinitely. There also has been concern that the use of somatic gene therapy may affect germ cells.

Although the successful use of somatic gene therapy has been reported, clinical trials have revealed risks. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. In 2000 researchers in France announced that they had successfully used gene therapy to treat infants who suffered from X-linked SCID (XSCID; an inherited disorder that affects males). The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Those outcomes highlight the difficulties foreseen in the use of viral vectors in somatic gene therapy. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response.

Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. While some have argued that gene therapy is eugenic, others claim that it is a treatment that can be adopted to avoid disability. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples hopes for new treatments that may never materialize.

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gene therapy | Description, Uses, Examples, & Safety ...

The Center for Gene Therapy at Nationwide Childrens Hospital is working to develop childrens gene therapy treatments. Officials say the gene therapy research and clinical trials there are starting to attract companies to central Ohio.

Nationwide Childrens Hospital is in the forefront of curing several genetic childhood diseases, transforming Columbus into a major medical hub, several gene therapy experts say.

The hospital's Center for Gene Therapy at the Abigail Wexner Research Institute is working to develop treatments for children, which is attracting patients and companies to Ohio, according to officials at Nationwide Childrens and JobsOhio, the state's economic development organization.

The illnesses that were making use of in gene therapy are devastating illnesses, said Dr. Kevin Flanigan, the director of Nationwide Childrens Center for Gene Therapy. These are ones we know that children would be significantly impaired for life or die because of the disease.

Gene therapy involves altering the genes inside the patient's cells in an effort to treat or stop disease. It gives doctors the chance to treat many previously untreatable rare and genetic diseases.

Gene therapy is currently available primarily in a research setting, with only four gene therapy products approved by the U.S. Food and Drug Administration for sale in the United States. One of the four, Zolgensma, started as a clinical trial for spinal muscular atrophy at Nationwide Childrens in 2014.

The hospital is working on a handful of gene therapy treatments for various childhood diseases that affect muscle, motor or mental functions, Flanigan said.

Gene therapy presents a tremendous opportunity for our medical system, and Columbus has been a huge part of that growth thanks to the work being done at Nationwide Childrens Hospital, Edith Pfister, chairwoman of the American Society of Gene & Cell Therapys communications committee, said in an email.

The FDA approved Zolgensma, a one-time treatment that intravenously delivers the gene that is missing in children with spinal muscular atrophy, on May 24.

SMA is a progressive childhood neuromuscular disease that is caused by a mutation in a single gene that attacks nerve cells. It causes major physical limitations including the inability to breathe, swallow, talk or sit up. Children born with SMA typically die or need permanent breathing assistance by the time they turn 2 years old.

Donovan Weisgarber was diagnosed with SMA type 1 at Nationwide Childrens in November 2015 when he was 5 weeks old. His parents, Matt and Laura Weisgarber, decided to participate in a clinical trial at the hospital and Donovan received Zolgensma.

Before the treatment, Donovan was unable to swallow and had difficulty breathing. Today, the 4-year-old has doubled his life expectancy and is able to talk, sit up, roll over and hold his head up on his own. He also attends the Early Childhood Education and Family Center on Johnstown Road on the East Side, which offers services from the Franklin County Board of Developmental Disabilities.

(Gene therapy) has given us an opportunity that we otherwise wouldnt have to love Donovan and experience him, said Matt Weisgarber, 33, of the Northeast Side.

A lot of people hear Ohio and think flyover state, but now Columbus is going to be a hub of the most groundbreaking science known to mankind and thats a really cool thing, he said.

Boston Childrens Hospital and Childrens Hospital of Philadelphia also have impressive gene therapy centers, but Columbus sets itself apart from those East Coast cities, said Severina Kraner, JobsOhios health care director.

The cost to operate, manufacture and live in Ohio is cheaper than Boston and Philadelphia, putting Ohio in a position to win cell and gene therapy companies, she said.

People are being priced out of these coastal cities, Kraner said.

One of the companies who has committed to building in Columbus is Sarepta Therapeutics, a Massachusetts-based biopharmaceutical company. Sarepta signed an agreement with Nationwide Childrens in May 2019, giving the company the licensing to a gene therapy treatment that came out of hospital research for limb-girdle muscular dystrophies, a group of diseases that cause weakness and wasting of the muscles in the arms and legs.

Sarepta is scheduled to open an 85,000-square-foot Gene Therapy Center of Excellence near Nationwide Childrens Hospital in the fall to do early research for all the companys gene therapy programs. A team of about 30 employees from Sarepta is currently working at a facility at Easton Town Center.

The region has every ingredient needed for a thriving gene therapy cluster: a strong academic foundation, world-renowned research hospitals, and, now, industry investment, Louise Rodino-Klapac, Sareptas senior vice president of gene therapy, said in an email. All of these contribute to creating a pipeline of talented people who will accelerate scientific advances that help patients.

Nationwide Childrens recently also announced it will be expanding its gene therapy research by creating Andelyn BioSciences, a new for-profit subsidiary that will manufacture gene therapy products for the biotechnology and pharmaceutical industries.

Were hoping, and we have a vision, that Andelyn can help capitalize a biotechnology hub in central Ohio focused on developing and advancing gene therapies, said Dr. Dennis Durbin, Nationwide Childrens chief science officer.

Andelyn BioSciences will launch this summer and operate out of the Abigail Wexner Research Institute, 575 Children's Crossroad. Nationwide Children's is trying to secure a permanent location for Andelyn and is looking at land on Ohio State Universitys West Campus.

Gene therapy treatment, however, comes at a high price.

The manufacturer set the price of Zolgensma at more than $2.1 million. Insurers can pay $425,000 a year for five years for one treatment.

Insurance companies are used to regular installment payments, but the single-dose nature of gene therapies are adding a level of uncertainty to health insurance structures, Pfister said in an email. A one-time administration gene therapy costs less overall, but it occurs in one upfront payment.

Pfister said she is hopeful the cost of gene therapy will go down.

Currently, most of the FDA-approved gene and cell therapies are tailored for the specific patient, but theres an incredible amount of research going into standardizing the components and delivery mechanisms behind gene therapy, Pfister said in an email.

Dr. Jerry Mendell helped usher in the era of gene therapy at Nationwide Childrens when he came to the hospital in 2004.

Nationwide Childrens first gene therapy trial was in 2006 for duchenne muscular dystrophy, a rare, inherited, degenerative muscle disorder that almost exclusively affects boys.

Things have really changed significantly in the gene therapy world because of the contributions weve made here, and its been a very gratifying experience, said Mendell, the principal investigator in Nationwide Childrens Center for Gene Therapy.

mhenry@dispatch.com

@megankhenry

Read more:
Nationwide Childrens among hospitals leading the way in gene therapy - The Columbus Dispatch

NextUp

Castle Creek Biosciences is developing several treatments that may provide relief for "Butterfly Children" and others living with rare genetic diseases.

John Maslowski is the chief executive officer of Castle Creek Biosciences. / Courtesy photo

NextUp is a weekly NextHealth PHL feature that highlights the local leaders, organizations and research shaping the Greater Philadelphia regions life sciences ecosystem. Email qmuse@phillymag.com with pitches for NextUp.

Who: New Jersey-based Castle Creek Pharmaceutical, a privately held company focused on developing treatments for rare genetic disorders, recently acquired Exton-based Fibrocell Science, a cell and gene therapy company focused on creating cell-based therapies for skin and connective tissue diseases.

The two companies combined to create Castle Creek Biosciences, a company working to advance a topical ointment and a new gene therapy platform to treat a range of rare genetic diseases and skin conditions.

What: Before the merger, both Castle Creek and Fibrocell had advanced investigational therapies for the treatment of epidermolysis bullosa (EB) into late-stage clinical research. EB is a group of rare genetic conditions that make the skin susceptible to easy blistering and scarring.

Castle Creek Biosciences most advanced product is the gene therapy FCX-007, a treatment that is designed to treat the life-threatening genetic disorder recessive dystrophic epidermolysis bullosa (RDEB). For children living with the RDEB, even the slightest touch, rub or scratch can lead to severe blistering or the removal of large areas of skin.

The company is currently conducting a pivotal Phase III clinical trial to test the efficacy of its novel gene therapy platform that involves collecting skin cells from each patient, genetically modifying the cells, and injecting the modified cells back directly into the patients blisters and wounds. In this process, the patients cells are genetically modified by a viral vector to introduce type seven collagen, an important protein that helps hold the layers of skin together. Castle Creek believes its gene therapy treatment can create the anchoring fibrils that hold the layers of skin together, preventing the characteristic blistering and scarring of EB.

Castle Creek also has a topical ointment in Phase II trials that is believed to block an important inflammatory signaling pathway in epidermolysis bullosa simplex (EBS), the most common form of epidermolysis bullosa. A third product in the companys pipeline is a gene therapy aimed at treating localized scleroderma, a chronic autoimmune disorder that causes excessive collagen deposits that result in thickening of the dermis and underlying tissue. It also causes pain, restricted motion, disfigurement and developmental issues.

The ultimate goal is to create a treatment platform for multiple EB patients and to become the leading EB treatment company in the United States and around the world, chief executive officer of Castle Creek Biosciences, John Maslowski, told NextHealth PHL.

When: In April 2019, Castle Creek and Fibrocell entered into a collaboration agreement to develop and commercialize what was then Fibrocells lead gene therapy candidate, FCX-007. Castle Creek officially acquired Fibrocell for $63.3 million in December 2019. Maslowski believes the merger will enable the combined company to play a more dominant role in developing treatments for EB and other rare diseases.

We really wanted to create a combined effort to treat the EB population in a more general way and really become a leader there, Maslowski said.

As we moved along in our partnership, it just seemed to make a lot of sense to us and Castle Creek that we really should be combined as one company because it creates a lot of synergies. We had a lot of experience in gene therapy, R&D, and manufacturing. They had a lot of clinical experience with EB patients and a lot of the commercial experience that we were missing. So that marriage really filled in a lot of the gaps and together, made us a more complete company.

Why: According to the National Institutes of Health, EB is estimated to affect one in 30,000 to 50,000 people in America. Patients living with EB are called butterfly children because their skin is as fragile as a butterflys wings. Any friction caused by simple daily acts, like putting on clothes or hugging, can result in mild to severe blistering and skin erosion.

If you can imagine, with just a little bit of lateral friction, EB patients get blisters all over their body. Its extremely painful, it leads to all sorts of other medical complications, and many EB patients tend not to live into their 30s, Maslowski explained.

There are currently no treatment options approved by the U.S. Food & Drug Administration for any form of EB.

What It Means: With its years of combined expertise in developing and commercializing new treatments for rare diseases, Castle Creek Biosciences has the potential to bring an EB treatment to the market within two to three years.

Read more:
NextUp: The Company Using Gene Therapy to Cure Rare Skin Conditions - Philadelphia magazine

Audentes Therapeutics, an Astellas company, announced it is building a gene therapy manufacturing plant in Sanford, North Carolina.

The company is investing $109 million in a new 135,000-square-feet facility, with the initial phase to take place over about 18 months. It is planned to go into operation in 2021. It is expected to create more than 200 new jobs in Lee County, North Carolina. Hiring is expected to start this year.

Our investment in large-scale manufacturing has always been a cornerstone of our strategy to develop and ultimately deliver our important genetic medicines to patients as rapidly as possible, said Natalie Holles, president and chief executive officer of Audentes. This new facility in Sanford will support the next phase of our growth as we establish a robust, global supply chain and expand our therapeutic and geographic scope as a part of the Astellas group of companies. We are excited to join the vibrant biopharmaceutical research and manufacturing community that the state of North Carolina has established.

Audentes is headquartered in San Francisco and focuses on gene therapy. It was acquired by Tokyo-based Astellas Pharma in January.

No specifics were given about what the site will manufacture. In October 2019, Audentes announced positive data from ASPIRO, the clinical trial of AT132 in patients with X-Linked Myotubular Myopathy (XLMTM). AT132 is an AAV8 vector that contains a functional copy of the MTM1 gene. XLMTM is a serious, life-threatening, rare neuromuscular disease marked by extreme muscle weakness, respiratory failure and early death.

The company indicates it hopes to submit a Biologics License Application (BLA) for AT132 to the U.S. Food and Drug Administration (FDA) later this year.

An announcement ceremony was held in the industrial shell building the company is buying in the Central Carolina Enterprise Park. It is about 45 miles southwest of Raleigh.

Gov. Roy Cooper stated, With our powerhouse research centers and highly skilled workforce, biotech pioneers recognize North Carolinas role as a leader in the life sciences. Lee County is a perfect fit for Audentes as they seek to become a global leader in genetic medicines.

The employees at the new factory are expected to earn an average salary of $83,900, which is a little over twice the Lee County average of $41,800. If Audentes hits hiring milestones, it will qualify for a state Job Development Investment Grant worth up to $3.7 million.

The county and the city of Sanford are also offering $5.7 million incentives, which includes almost $400,000 in training support from the North Carolina Community College System.

Audentes chose the location over California, Massachusetts and Colorado.

In every interaction, I was impressed with Audentes patient-centric approach to developing their AAV-based gene therapy to transform the lives of affected patients and families, said Laura Rowley, NCBiotechs director of life science economic development. She led the Centers outreach activity with Audentes. Their decision to grow in North Carolina reflects the Research Triangle regions specialized training capabilities and strengths in gene therapy and biomanufacturing. The passion and focus of the Audentes team makes me confident that they will be an outstanding addition to North Carolinas gene therapy community.

Audentes is acting as the Center of Excellence for Astellas newly founded Genetic Regulation Primary Focus.

Audentes Therapeutics is joining one of the nations top life science clusters, said Anthony M. Copeland, North Carolinas Commerce Secretary. North Carolina has the largest biomanufacturing workforce in the nation and a growing concentration of gene therapy scientists, researchers and workers.

The site of the new plant is quite close to Pfizers new gene therapy campus, which is under construction. That $600 million research and manufacturing facility has a 230-acre campus in Sanford and will employ 340 people.

More:
Audentes to Build $109 Million Gene Therapy Factory in North Carolina - BioSpace

Dive Brief:

Bluebird's commercial operations are just getting off the ground. In its latest earnings report, the Cambridge, Massachusetts-based biotech detailed how it has inked agreements with health insurers in Germany that should provide coverage for LentiGlobin, which is sold under the brand name Zyntegloin Europe, for up to 50% of eligible beta-thalassemia patients. Bluebird expects the first commercial patient to be treated before July.

Across the Atlantic, U.S. patients are looking at a longer timeline before LentiGlobin becomes available. Stifel analysts wrote in a note to clients that they don't foresee any stateside patients receiving the therapy commercially in 2020 "given what we anticipate will be a complicated negotiation process with payors."

Analysts at Raymond James, meanwhile, downgraded Bluebird to a "Market Perform" rating, writing that "execution issues on the regulatory, clinical and manufacturing side outweigh our support for the innovative drug products."

As Bluebird works through the latest delay in beta-thalassemia,it will also be preparing for an expanded research program in sickle cell. The company already intended to kick off a late-stage study in sickle cell patients with a history of vaso-occlusive crises in the first half of 2020. With Tuesday's earnings presentation, though, came plans to initiate a second late-stage study sometime this year, which will evaluate LentiGlobin's effects in about 18 children with sickle cell and elevated stroke risk.

A sickle cell approval, though a ways off, could boost Bluebird's bottom line. Beta-thalassemiais rarer in U.S. than other parts of the world, and certainly less common than sickle cell. According to estimates cited by the National Organization of Rare Disorders, roughly 3,300 U.S. patients have beta-thalassemiaversus the 100,000 who have sickle cell.

An expanded program could provide more evidence of LentiGlobin's benefit in this larger patient pool.Yet the updates don't seem to have alleviated investor concerns. Bluebird shares were down nearly 10% in late Wednesday morning, trading around $80 apiece.

"LentiGlobin in Sickle Cell Disease remains a bright spot, in our view, but with [late-stage studies] expected to get underway this year, we don't expect investor sentiment to change anytime soon," Stifel analysts wrote.

The investment bank models Zyntelgo bringing in $12 million worth of revenue in 2020 from the beta-thalassemia indication, increasing to $53 million in 2021 and $390 million by 2030. Conversely, it models $48 million in 2022 from the sickle cell indication, increasing to almost $2 billion by 2030.

Read more from the original source:
Bluebird's gene therapy hits another delay, this time in the US - BioPharma Dive

17th patient dosed in the ongoing phase 2/3 AAVance study

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 FDA's recognition of LYS-SAF302's 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 Lysogene's 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 Company's 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 Company's 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 Company's control that could cause the Company's 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 Company's 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200224005960/en/

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