StemCell Therapy

HOUSTON, Feb. 16, 2021 /PRNewswire/ -- Avance Biosciences Inc., a leading CRO providing GLP/GMP-compliant assay development, assay validation, and sample testing services supporting biological drug development and manufacturing, announced today that its Houston facility, which successfully passed an inspection by the U.S. Food and Drug Administration in Oct 2018, is undergoing major expansion to handle rapidly growing demand for their services.

The new facility, expected to be completed by Q3 2021, is located adjacent to the current facility and will expand the Houston campus by an additional 5,500 square feet. The new facility will be devoted to cell-based assay services and enable Avance to better address the specific needs of their GMP clients. Additionally, Avance is expanding their mammalian cell culture related assay capabilities including: mycoplasma testing, adventitious agents testing, sterility, potency, and others.

As a provider of genomics and biological testing services, Avance Biosciences offers a broad range of molecular biology and microbiology assays in compliance with current Good Manufacturing Practices (21 CFR Parts 210 & 211) and Good Laboratory Practices (21 CFR Part 58) to support its clients' regulatory submissions.

Avance's CEO, Dr. Xuening Huang commented, "We take a partnership approach with our clients and that means an extended relationship; from discovery to development to clinical testing and on to manufacturing. Our most recent expansions will ensure that we can keep pace with our customer's increased needs when ramping up development and manufacturing activities. Our primary goals are to deliver world-class service and complete customer satisfaction."

Avance's Vice President of Sales and Marketing, Cal Froberg commented, "It's clear there is tremendous growth in the development of cell and gene therapies and we're proactively managing resources to handle increased market demand for related support services. The industry is expanding rapidly and Avance is positioned well to address the specific needs of these customers."

This most recent expansion comes on the heels of another 7,500 square foot expansion completed in 2020 which has significantly increased Avance's NGS and ddPCR capabilities. This facility has been pivotal in addressing gene therapy development support needs such as: edited gene testing, gene integration assays, and DNA/RNA biodistribution studies.

Recently, Avance Biosciences was recognized as a top 10 Genomics Solutions Company for 2020. Current and future expansion plans will serve to solidify this position among the premier providers in this space.

About Avance Biosciences

Avance offers cGMP/GLP compliant genomics biological testing services in support of drug development and manufacturing. Its leading scientists have designed, validated, and tested thousands of assays under cGMP/GLP regulations for the FDA, EPA, and European and Japanese regulatory agencies. Avance's team has extensive knowledge and experience working with scientists, QA/QC professionals and project managers from over 100 pharmaceutical and biotechnology companies and organizations throughout the world.

Contact

Xuening Huangxuening.huang@avancebio.com877-909-52109770 West Little York RoadHouston, TX 77040 USA

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SOURCE Avance Biosciences

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Avance Biosciences Expanding Houston Campus in Support of Cell and Gene Therapy Drug Development - BioSpace

Theres news today that Bluebird has suspended its gene therapy work on sickle cell disease because of two cases of cancer in its treatment population. Another had been reported in 2018, so that takes us to two cases of myelodysplastic syndrome and one case of myeloid leukemia (which can be a sequel of MDS in some cases). This isnt good. Youll note that all of these are diseases of the bone marrow, and the marrow is where a good deal of the action in this sort of gene therapy takes place.

There are several companies working in this space, and its no coincidence. Sickle cell anemia is the absolute prototype of a genetically linked disorder, famously first identified in 1949 by Linus Pauling and co-workers. That paper termed it a molecular disease, and Pauling certainly deserves the credit he gets as a founder of molecular biology. Both sickle cell and the conceptually related beta-thalassemias are defects in the production of hemoglobin, and it has been obvious for decades that if you could somehow yank the defective gene out of the patients and replace it with a normal sequence that they simply wouldnt have these conditions any more.

There are by now plenty of other genetic disorders that fall into the same category, but these blood-cell based ones have a unique feature that has put them into the forefront of actual attempts at gene therapy. In these cases, all the relevant cells come from the same tissue, the bone marrow. And we actually have ways to kill that off and to swap in new tissue of our choosing: a bone marrow transplant. It is a tough procedure to go through, for sure, but not as tough as living a life of acute sickle cell attacks (or being killed off early by rampaging leukemia, to pick another application).

Contrast that with so many other gene-linked disorders take Huntingtons, for example. We know the gene for that one, and the protein it codes for, and it is equally obvious that if you could magically yank out that gene from a patient and insert the normal one for the Htt protein that they would no longer have the disease. But there is no analogous procedure for killing off the basal ganglia of the brain and replacing it with new neuronal tissue. Not quite. No, bone marrow based disorders are a unique opportunity, and thats why so much effort has gone into this area.

Its a similar situation to the way that therapeutic RNAs have been aimed at liver disorders. In that case, youre not wiping out the old cell population but rather trying to overwhelm it in situ, and the liver is chosen because we dont really know how to make i.v. dosed RNA species accumulate anywhere else. So we make do with what we have and turn the Liver Problem into the Liver Advantage. If we ever get to the point of treating Huntingtons at a genetic level, its surely going to be via a similar rework-things-in-situ method as well, but figuring out to do that in only the desired regions of the brain without causing trouble elsewhere is quite a challenge youve lost the Liver Advantage.

Now, Bluebird. They have been using a lentivirus vector to rewrite the bone marrow transplant tissue, and theres a solid reason for that. Lentiviruses (of which HIV is the most famous/infamous example) insert their genetic payloads into the host cells DNA. Its their key step, and they can do it even on non-dividing cells. Now, when a person hears viral vector these days, the thought is immediately of vaccines, and that takes us to the worry that the vaccines aimed at the COVID-19 pandemic will do things to our DNA. But were not using lentiviruses for the viral-vector vaccines were using adenoviruses, because those explicitly do not work by inserting genes into host DNA. Thats also a feature of the mRNA vaccines: messenger RNA is not incorporated into our DNA. Those two species are constantly working in close proximity in living cells and theres a huge pile of optimized protein machinery to keep them from getting crossed in that fashion. Nor does a messenger RNA sequence get turned back into DNA and inserted that way. Every cell has hundreds of thousands of mRNA molecules in it at any given time, and things would come to a catastrophic halt if these started getting reversed back into DNA sequences. (Our cells do have some RNA-to-DNA machinery in them, but it doesnt work like that).

But for gene therapy, the opposite considerations apply you most certainly want to insert new genes into human DNA, and you want it done quickly, efficiently, and right where you tell it to go. That last part is always the worry with any gene-insertion technique, be it some variety of CRISPR, zinc-finger nucleases, lentivirus vectors or what have you. This is one of the main reasons the human-editing experiment in China was so amazingly irresponsible, because our control over such things in a human embryo is just not acceptable yet. Not even close.

In fact, its tricky enough just in the stem cells pulled out of bone marrow. Thats one possibility for what Bluebird is seeing that when they treated the patients extracted cells with their lentivirus vector, that some of the hemoglobin genetic data got mishandled and plopped into the wrong stretch of DNA, demolishing some other important genes function in the process. You can be sure that theyre sequencing the abnormal blood cells from these patients now to see if this shows up. The MDS patient from 2018 turned out not to have this problem, so its possible that these two just reported dont, either. So whats the problem, if not that?

Well, as mentioned, bone marrow transplantation is a grueling process no matter what. The process of (either mostly or completely) wiping out a persons bone marrow stem cells involves severe treatments mixing chemotherapy with radiation, and one of the compounds used (and used by Bluebird) is called busulfan. The organic chemists in the crowd will find that one interesting: its the bis-mesylate of 1,4-butanediol, nothing more and nothing less, and if the thought of taking a reactive small molecule like that intravenously gives you the shivers, well, welcome to chemotherapy and get ready for some stuff thats even worse. The thing is, busulfan itself is a Class I carcinogen (as one would expect from its structure). Many older chemotherapy agents are. They are destructive to cells, and the only way you would take any of them is if you have a population of cells that you actually want to see destroyed, and you are willing to take your chances that you can bear up under the collateral damage of doing that. So its certainly possible that the leukemia seen in Bluebirds patients is at least partly driven by the bone marrow transplantation procedure rather than the gene alteration part. In case youre wondering, this could well be happening with some bone marrow transplant patients who undergo this whole procedure to treat leukemia itself, in which case it lands silently in the relapsed category. No, you only do bone marrow transplants when theres no alternative.

As that first link in todays post (Adam Feuerstein at STAT) mentions, though, theres ongoing research to make that part of the process less risky. Survival rates for bone marrow transplants in general have steadily improved over the years, and everyone knows that one of the rough parts is the pre-treatment. But that problem might or might not get solved in time to help out Bluebird (or to quell the worries that other gene-therapy outfits might have who are also targeting that hematopoietic tissue). If indeed its the problem in the first place. . .

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Cancer and Gene Therapy | In the Pipeline - Science Magazine

The new gene therapy target,GJB2 coding for the Connexin 26 protein, has been added to Sensorions development portfolio: with the target the third candidate to emerge from the R&D collaboration with Institut Pasteur. It represents the largest gene therapy opportunity for the French biotech to date.

The GJB2 program will focus on major new markets with an estimated patient population of 300,000 children and adults in Europe and the US alone.

Sensorion, a French clinical-stage biotech based in Montpellier, was founded in 2009 to develop novel therapies to restore, treat and prevent hearing loss disorders.

The GJB2 program draws on new research from Institut Pasteur which shows that the same genes that underly congenital deafness are also involved in severe forms of presbycusis (age-related hearing loss). These forms of presbycusis appearing to be monogenic types of hearing loss that can be potentially treated by gene therapy.

Although the types ofGJB2mutations in children and adults may differ, Sensorion says gene therapy could potentially provide a solution to both.

Mutations inGJB2are believed to alter a gap junction protein widely expressed in the inner ear, disturbing intercellular exchanges of molecules and leading to hearing loss that is severe-to-profound in a majority of cases.

Institut Pasteur research now shows three pathologies related to GJB2 mutations: congenital deafness;age-related hearing loss in adults; and progressive forms of hearing loss in children. Sensorion will prioritize the latter two forms, saying it is the first company to address these needs and offering the potential of large market opportunities.

The emergence of a new gene therapy target candidate validates our conviction that long-term solutions for restoring hereditary hearing loss will arise from an in-depth analysis of the "genetic landscape" of hearing loss," saidNawal Ouzren, CEO of Sensorion.

"It was clear that mutations in the GJB2 gene are important in severe to profound childhood hearing loss. However, the new discovery made by our collaborators at Institut Pasteur shows that alteration of this gene in adults offers new opportunities for Sensorion. It marks significant potential expansion of our pipeline and supports our goal of becoming a global leader in the field of gene therapies for hearing loss disorders.

Sensorions collaboration with Institut Pasteur initiated in 2019 has already led to gene therapy candidate programs in two other indications. Its USHER-CT gene therapy development program aims to restore inner ear function for patients suffering from Usher Syndrome Type 1 by providing a healthy copy of the USH1G gene coding for the SANS protein.

Meanwhile, the OTOF-GT gene therapy development program seeks to restore hearing in people with Otoferlin deficiency, one of the most common forms of congenital deafness.

Both of these have been proved in concept in preclinical studies.

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Sensorion and Institut Pasteur announce new gene therapy collaboration - BioPharma-Reporter.com

New data from GenSight Biologics showed promising results for lenadogene nolparvovec (LUMVOQ), an intravitreal gene therapy for leber hereditary optic neuropathy (LHON) caused by mutations in the mitochondrial ND4 gene.

Overall, the therapy was well-tolerated in patients, had a favorable safety profile, and was shown that it may lead to clinically meaningful improvements.

A team, led by Catherine Vignal-Clermont, MD, Rothschild Foundation Hospital, Paris, France, conducted an open-label, single-center, dose-escalation study that primarily assessed safety and tolerability of the gene therapy among 15 patients with LHON.

Therapeutic options for adolescent and adult patients with LHON are currently limited to idebenone (Raxone), a synthetic analog of coenzyme Q10, which is approved only in Europe under exceptional circumstances for treatment of LHON, Vignal-Clermont and team wrote.

They further acknowledged that no approved treatment exists in the United States.

REVEAL A Phase I/IIa Study

Among the exclusion criteria were vision loss in the fellow eye, glaucoma, diabetic retinopathy, macula edema, vitreoretinal disease, pathology of the retina or the optic nerve, retinal vein occlusion, narrow angles, optic neuropathy for other causes, or any other disease that would have an effect on visual function.

Eligible patients were divided into a dosing cohort to receive a single injection and then were followed-up immediately at day 3 for safety and efficacy assessments.

The investigators pursued further follow-up at weeks 1, 2, 4, 8, 12, 24, 36, and 48 post-treatment. Additional follow-up was performed at years 1.5, 2, 2.5, 3, 4, and 5.

The studys primary endpoint was the overall incidences of adverse events up to 5 years post-treatments for each dosing level and for the treatment as a whole.

Secondary endpoints included best corrected visual acuity (BCVA; calculated as logarithm of the minimal angle of resolution [LogMAR]), among other efficacy measurements.

Results

Throughout the follow-up period, the investigators noted no serious adverse events that were considered related to treatments.

Furthermore, patients did not experience unexpected adverse events nor grades 3 or 4 Common Terminology Criteria for Adverse Events.

Anterior chamber inflammation and vitritis were mostly managed with topical steroids, and ocular inflammation was considered to be dose limiting by the independent data safety monitoring board based on the benefits/risks for the subjects, the investigators wrote.

In terms of efficacy, the team reported that analysis of the LogMAR BCVA in both treated and untreated eyes showed clinically relevant and durable improvements compared with baseline.

As such, the mean improvement for the treated eye was -0.44 LogMAR and for the untreated eye was -0.49.

Thus, at 5 years post-treatment, the final value of LogMAR was +1.96 and +1.85, respectively, for the treated and untreated eyes.

As for those treated with the optimal dose level of 9 1010 viral genomes/eye (n = 6), the mean visual acuity improvement from baseline was 0.68 LogMAR for treated eyes and 0.64 LogMAR for untreated eyes.

The final mean value for the treated and untreated eyes were LogMAR +1.77 and +1.78, respectively.

While there was a meaningful improvement in visual acuity for REVEAL subjects, the final visual acuity was less favorable than that seen in the two subsequent pivotal phase III studies in which subjects were treated earlier during the course of their disease, Vignal-Clermont and colleagues wrote.

Nevertheless, the team acknowledged that these findings are a promising prelude to the Phase III RESCUE and REVERSE studies, which are running in tandem and currently assessing the efficacy of the single injection of the gene therapy in a larger population.

The study, "Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study," was published online in BioDrugs.

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Gene Therapy GS010 Safe, Well-Tolerated for LHON Patients - MD Magazine

Its back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance.

NICE is assessing Zynteglo (betibeglogene autotemcel), a one-off gene therapy for the condition, which can have life-threatening consequences and is associated with a curtailed life expectancy.

There is a curative treatment for people who rely on blood transfusions to survive and maintain their levels of red blood cells.

But haematopoietic stem cell transplantation is only possible when a donor with a matching human leukocyte antigen signature, within the correct age range, is available.

In this first draft guidance NICE raised a series of issues with Zynteglo, which bluebird has already agreed to supply at a confidential discount from its hefty price tag, which is around 1.57 million in Europe.

NICE said that data came from a small sample of patients and is using its standard discount rate of 3.5% to calculate the long-term benefits of the treatment.

The company has unsuccessfully pushed for a rate of 1.5%, which would attach more value to the long-term benefits of the therapy over a patients lifetime.

There was also a long list of other technical issues raised by NICE that count against Zynteglo in the assessment, including costs of fertility preservation and the number of simulated profiles in bluebirds data.

Nicola Redfern, bluebird bios UK general manager, said the first step is to present a new analysis of data addressing issues raised by NICE before there are any discussions about lowering the price again.

She pointed out that the dossier presented to NICE was compiled in 2019 and the company now has six years worth of follow-up data.

Redfern also added that this is the first time that NICE had assessed a gene therapy using its single technology assessment process, which is used for medicines likely to be used more widely on the NHS.

However Redfern was still surprised the rejection given the discussions with NICE so far in the process.

She said: Some of the specifics we thought we had covered off with them and discussed. The thing that baffled me most was the lack of understanding of this disease upon the people living with it.

The UK Thalassaemia Society noted NICEs citation of a UK patient reference report stating that 37% of respondents would immediately accept a referral to a transplant specialist and betibeglogene autotemcel if offered it.

Romaine Maharaj, executive director at UKTS, said: Most of our members are very excited about the new therapy developments and are keen to explore these treatment options.

Bone marrow transplant is only an option for a very small proportion of people with thalassaemia and so gene therapy offers a real potential alternative as a one-off resolution to this life-limiting condition.

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bluebird bio 'baffled' after NICE rejects beta-thalassaemia gene therapy - - pharmaphorum

Novartis is teaming up with the Gates Foundation to develop a gene therapy for sickle cell disease; the World Health Organization reports a drop in global cases of coronavirus disease 2019 (COVID-19); storms hinder vaccine rollout across the United States.

The Bill and Melinda Gates Foundation and Novartis are collaborating to discover and develop a one-step, one-time gene therapy treatment to cure sickle cell disease, STAT News reports. Although the cause of sickle cell disease is understood and the people it affects are known, a cure has been difficult to pin down. Through a 3-year partnership, the companies aim to create a treatment that is affordable and simple enough to treat individuals anywhere in the world, including sub-Saharan Africa, where disease prevalence is high. Current gene therapy approaches are complex and expensive, and they create treatments for patients one at a time. With an initial funding amount of $7.28 million, the companies ultimately hope to create an off-the-shelf product.

In the past week, the rate of new coronavirus disease 2019 (COVID-19) cases has declined by 16% around the world, according to the World Health Organization. This decline comes even as more virulent strains of COVID-19 have caused new outbreaks in some regions, The Washington Post reports. In addition to the decline in cases, over the past week there has been a 10% reduction in the number of deaths worldwide, with the greatest drop in cases seen in Europe and the Americas. In the United States alone, the number of new infections decreased by nearly 24% in the past week.

Deadly storms across the country hampered COVID-19 vaccination efforts on Tuesday, forcing appointment cancellations and delaying vaccine deliveries, according to the Associated Press. The surge of bad weather comes as the federal government rolled out new vaccination sites targeting hard-hit communities and seeks to boost the amount of vaccine sent to states to 13.5 million doses per week. Currently, the United States administers an average of 1.7 million doses per day, but the bad weather halted vaccinations in Pennsylvania, Illinois, Tennessee, and Missouri and is expected to disrupt shipments from facilities in Tennessee and Kentucky.

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What We're Reading: Sickle Cell Gene Therapy Partnership; Global COVID-19 Cases on Decline; Storms Slow Vaccine Rollout - AJMC.com Managed Markets...

WATERTOWN, Mass., and Research Triangle Park, N.C., Feb. 17, 2021 /PRNewswire/ --Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced the initiation of a Phase 1 dose-escalation trial of SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) containing no DNA combined with ImmTOR. The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.

"We are pleased to further evaluate ImmTOR's ability to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing by having initiated this dose-escalation study of SEL-399," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "This trial builds upon our strong preclinical data in non-human primates and marks the first time that ImmTOR in conjunction with an AAV capsid has been dosed in humans, which is a significant milestone. Data from this study will inform the design of future clinical trials in patients as we seek to unlock the full potential of gene therapy."

The dose-escalation trial of SEL-399 is designed to evaluate the safety and preliminary efficacyof ImmTOR in gene therapy. The study, being conducted in healthy volunteers at the SGS Life Sciences Clinical Pharmacology Unit in Antwerp, Belgium, plans to enroll up to 45 subjects to investigate increasing doses of ImmTOR and EMC-101. Subjects will be randomized in a 3:1 ratio of ImmTOR plus empty AAV8 capsid to empty capsid alone. Preliminary efficacy will be measured by assessing levels of AAV8-specific neutralizing antibodies.

Jude Samulski, Ph.D., chief scientific officer and co-founder of AskBio said, "By determining the dose at which ImmTOR is able to inhibit the formation of AAV-specific antibodies,this study could be a significant first step toward overcoming some of the unwanted immune responses associated with gene therapies. We look forward to using these findings to inform future studies as we work to develop strategies for repetitive dosing of AAV, thus extending durability of expression."

Selecta and AskBio expect to report initial results from this clinical trial in the fourth quarter of 2021.

AboutSelecta Biosciences, Inc.Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the body's natural self-tolerance in autoimmune diseases. The company's first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visitwww.selectabio.com.

About AskBioAsklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020,is a fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more atwww.askbio.comor follow us onLinkedIn.

About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2019, the Group employed around 104,000 people and had sales of43.5 billion euros. Capital expenditures amounted to2.9 billion euros, R&D expenses to5.3 billion euros. For more information, visit http://www.bayer.com.

AskBio Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding AskBio's pipeline of development candidates; AskBio's collaboration with Selecta; AskBio's clinical trials, including its ability to enroll subjects, the timing of any such trials and any potential side effects; whether ImmTOR will be able to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing; the timing of and results from the SEL-399/101 trial; whether the SEL-399/101 study could be a significant first step in overcoming the immunogenicity concerns associated with gene therapies; AskBio's strategies for repetitive dosing of AAV, extending durability of expression; AskBio's goal of developing life-saving medicines aimed at curing genetic diseases; and the potential benefits of AskBio's development candidates to patients. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio's business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects ofSelecta Biosciences, Inc.("the company"), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy, the ability of the Company and AskBio to develop gene therapy products using ImmTOR and AskBio's technology, the novelty of treatment paradigms that the Company is able to develop, whether the observations made in non-human primate study subjects will translate to studies performed with human beings, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the company's plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the company's intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the company's ability to grow its strategic partnerships, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "hypothesize," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human primates, the unproven approach of the company's ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the company's product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the company's inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the company's recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the "Risk Factors" section of the company's most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with theSecurities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the company's views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

SOURCE Asklepios BioPharmaceutical, Inc.

http://www.askbio.com

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Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR in Gene Therapy - PRNewswire

GenSight Biologics has reported that results from the Phase I/IIa REVEAL clinical trial of LUMEVOQ (lenadogene nolparvovec) gene therapy demonstrated a favourable safety profile in individuals with ND4 Leber hereditary optic neuropathy (LHON).

The trial also determined the dose used in the Phase III RESCUE and REVERSE trials.

Launched in 2014, the open-label, single-centre, dose escalation study analysed the safety and tolerability of LUMEVOQ in 15 participants with ND4 LHON who were followed for up to five years after administering a single intravitreal injection to their worst-affected eye.

Participants were enrolled in four cohorts of three subjects each, with each cohort given increasing doses of the gene therapy.

Dose escalation continued only after a safety evaluation by an independent data safety monitoring board (DSMB). A final extension cohort received the dose that the DSMB determined to have the best benefit-risk ratio among those administered to the four previous cohorts.

Data showed that LUMEVOQ was well-tolerated over the follow-up period of five years, with no serious adverse events noted.

These results are the first to show the favourable safety profile of the gene therapy while hinting at the efficacy analysed in the Phase III trials.

This safety profile was then affirmed in the Phase III RESCUE and REVERSE trials.

GenSight co-founder Dr Jos-Alain Sahel said: This study confirms the gene therapys favourable long-term safety and further demonstrates that the trends that were initially observed have been maintained for at least five years.

GlobalData's TMT Themes 2021 Report tells you everything you need to know about disruptive tech themes and which companies are best placed to help you digitally transform your business.

The company noted that REVEAL trial data and analyses were main components of the evidence package submitted to the European Medicines Agency (EMA) last September.

The submission was made seeking Marketing Authorisation Application (MAA) for LUMEVOQ for treating patients with visual loss due to ND4 LHON. The EMA decision is expected in the fourth quarter of this year.

Software, Consulting and Workshops for Data Analysis and Model-Based Decision Support

28 Aug 2020

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GenSight Biologics' gene therapy proves safe in LHON trial - Clinical Trials Arena

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimers disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

The protein, called brain-derived neurotrophic factor or BDNF, is part of a family of growth factors found in the brain and central nervous system that support the survival of existing neurons and promote growth and differentiation of new neurons and synapses. BDNF is particularly important in brain regions susceptible to degeneration in AD.

In previous published research, principal investigator Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine, and colleagues described the prevention and reversal of brain cell degeneration and death in animal models.

Mark TuszynskiMark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine.

We found that delivering BDNF to the part of the brain that is affected earliest in Alzheimers disease the entorhinal cortex and hippocampus was able to reverse the loss of connections and to protect from ongoing cell degeneration, said Tuszynski. These benefits were observed in aged rats, aged monkeys and amyloid mice.

Amyloid mice are genetically engineered to inherit a mutation in the gene encoding the amyloid precursor protein, and as a result develop amyloid plaques aggregates of misfolded proteins in the brain that are considered a hallmark characteristic of AD.

BDNF is normally produced throughout life in the entorhinal cortex, an important memory center in the brain and one of the first places where the effects of AD typically appear in the form of short-term memory loss. Persons with AD have diminished levels of BDNF.

But BDNF is not easy to work with. It is a large molecule and cannot pass through the blood-brain barrier. As a result, researchers will use gene therapy in which a harmless adeno-associated virus (AAV2) is modified to carry the BDNF gene and injected directly into targeted regions of the brain, where researchers hope it will prompt production of therapeutic BDNF in nearby cells.

The injections are precisely controlled to contain exposure to surrounding degenerating neurons since freely circulating BDNF can cause adverse effects, such as seizures.

The three-year-long trial will recruit 12 participants with either diagnosed AD or MCI to receive AAV2-BDNF treatment, with another 12 persons serving as comparative controls over that period.

This is the first safety and efficacy assessment of AAV2-BDNF in humans. A previous gene therapy trial from 2001 to 2012 using AAV2 and a different protein called nerve growth factor (NGF) found heightened growth, axonal sprouting and activation of functional markers in the brains of participants.

The BDNF gene therapy trial in AD represents an advance over the earlier NGF trial, said Tuszynski. BDNF is a more potent growth factor than NGF for neural circuits that degenerate in AD. In addition, new methods for delivering BDNF will more effectively deliver and distribute it into the entorhinal cortex and hippocampus.

Despite billions of dollars of research investment and decades of effort, there are only two symptomatic treatments for AD. There is no cure or approved way to slow or stop progression of the neurological disorder that afflicts more than 5 million Americans and is the sixth leading cause of death in the United States.

Numerous clinical trials are ongoing to assess pharmaceutical remedies. Tuszynski said gene therapy, which debuted in 1980 and has been tested on multiple diseases and conditions, represents a different approach to a disease that requires new ways of thinking about the disease and new attempts at treatments.

We hope to build on recent successes of gene therapy in other diseases, including a breakthrough success in the treatment of congenital weakness in infants (spinal muscular atrophy) and blindness (Leber Hereditary Optic Neuropathy, a form of retinitis pigmentosa), Tuszynski said.

BDNF gene therapy has the potential, unlike other AD therapies currently under development, to rebuild brain circuits, slow cell loss and stimulate cell function. We are looking forward to observing the effects of this new effort in patients with AD and MCI.

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UC San Diego: First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimers Disease - India Education Diary

HACKENSACK, N.J., Feb. 18, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to endDuchenne muscular dystrophy (Duchenne), andDuchenne UK, a UK-based patient organization,are pleased to announce ProfessorKanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of their Joint Research Grant Call of 2020. The full title of the research project is "Targeting the innate immune system to block acute inflammatory and chronic immune response to transgene and AAV vector in DMD".Professor Nagaraju's research will receive funding from the organizations in the amount of $350,000.

These are promising times for research into Duchenne muscular dystrophy (Duchenne). Several companies are now testing an approach that uses a shortened dystrophin gene to replace the faulty dystrophin gene in Duchenne. This is known as gene transfer using micro-dystrophin, or more commonly, gene therapy. The companies are using viruses known as AAVs (adeno-associated viruses) to deliver the therapy.

However, challenges exist in getting this treatment to the entire Duchenne population. This is mainly because of immune responses: some patients have pre-existing antibodies to the AAVs. This means they will not, currently, be able to have the treatment because their bodies will recognize the virus and stop it from delivering the micro-dystrophin to the cells. In addition, as gene therapy is a new treatment, it is not yet clear if another dose will be required at a later stage, and it is not currently possible to re-dose with the same AAV.

This is why Duchenne UK & PPMD launched a call for projects last year that would specifically address this challenge.

The organizations received a large number of proposals, and three were taken forward for final review from a panel of highly qualified, specialized scientists. They looked at a wide variety of factors, including significance to the Duchenne community, and the ability to translate the research into treatments for patients.

Professor Nagaraju's research is looking at blocking the mechanism by which the body is able to recognise an AAV virus and mount an immune response to it. Importantly, he is using medicines that are already in use in humans, in an approach known as repurposing.

If this approach were successful, it would allow more micro-dystrophin to get to the cells, potentially requiring a lower dose of the AAV than is currently being administered in the trials. It may also allow patients who have already been dosed with gene therapy to receive further doses. Further to this, by using repurposed drugs, this treatment should be more easily transferable to patients. Professor Nagaraju believes that "targeting initial immune recognition pathways is one way to improve efficacy and safety profiles of AAV mediated gene therapy".

PPMD's Founding President & CEO, Pat Furlong, and Duchenne UK's CEO, Emily Crossley explained in a joint statement:"Supporting patients and accelerating innovative research is at the heart of what we do at Duchenne UK and PPMD. We are pleased to partner with each other and award this grant. Gene therapy is offering great promise, but there are challenges associated with the immune response which are limiting the rate of progress and a barrier to ensuring all patients can have access to these potentially transformative therapies.We would like to thank all those who participated and supported our Joint Grant Call and are very much looking forward to working with Professor Nagaraju on this vitally important project for the Duchenne community."

To learn more about PPMD's innovative research agenda and our investment portfolio, visit PPMD's website.

About Parent Project Muscular Dystrophy

Duchenneis a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won four FDA approvals.

Everything we doand everything we have done since our founding in 1994helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.

About Duchenne UK

Duchenne Muscular Dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of Muscular Dystrophy. Diagnosed in childhood, it mainly affects boys. There is currently no cure. Started by families affected by the disease, Duchenne UK has one clear aim to end Duchenne.

Duchenne UK are funding research that's focused on getting treatments to those affected now as well as pushing for an effective treatment in the future.

Duchenne UK connects leading researchers with industry, the NHS and patients to challenge every stage of drug development, from research to clinical trials to drug approval. They connect families with each other to create a network of mutual support and to pool resources, knowledge and experience.

For more information about Duchenne UK: visit http://www.duchenneuk.org.

SOURCE Parent Project Muscular Dystrophy (PPMD)

More:
Duchenne UK and Parent Project Muscular Dystrophy Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne Muscular Dystrophy -...

Dublin, Feb. 15, 2021 (GLOBE NEWSWIRE) -- The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Europe Cell and Gene Therapy Market Report

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand.

In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields.

The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures.

Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patient's quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving.

CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

KEY QUESTIONS ANSWERED

1. What is the Europe cell and gene therapy market size and growth rate during the forecast period?2. What are the factors driving the demand for CAR-T therapy in the European region?3. How are strategic acquisitions aiding in market growth of cell and gene therapy products?4. Which segments are expected to generate the highest revenues during the forecast period?5. Who are the leading vendors in the European cell and gene therapy market?

Market DynamicsMarket Opportunities & Trends

Market Growth Enablers

Market Restraints

Prominent Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

For more information about this report visit https://www.researchandmarkets.com/r/qm1hjg

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Europe Cell and Gene Therapy Market Report 2021-2026: Prominent Players are Novartis, Spark Therapeutics, Amgen, Gilead Sciences & Organogenesis -...

Betibeglogene autotemcel (beti-cel), a one-time gene therapy, enabled durable transfusion independence in most patients with transfusion-dependent -thalassemia (TDT) who were treated across 4 clinical studies.

Of 60 patients enrolled overall, 17 of 22 (77%) treated in the 2 phase 1/2 studies were able to stop packed red blood cell transfusions. In the 2 phase 3 studies, which used a refined manufacturing process resulting in improved beti-cel characteristics, 89% (n = 31/35) of patients with at least 6 months of follow-up achieved transfusion independence for more than 6 months,1 reported Suradej Hongeng, MD, during the virtual 2021 Transplantation & Cellular Therapy Meetings.

The median follow-up after beti-cel infusion in the 4 studies has been 24.8 months (range, 1.1-71.8).

With up to 6 years of follow-up, 1-time beti-cel gene therapy enabled durable transfusion independence in the majority of patients, said Hongeng, from Ramathibodi Hospital of Mahidol University, in Bangkok, Thailand.

Patients who achieved transfusion independence experienced a 38% median reduction in liver iron concentration (LIC) from baseline to month 48. The median reduction in LIC was 59% in patients with a baseline LIC more than 15 mg/g dw. A total of 21 of 37 (57%) patients who achieved transfusion independence have stopped iron chelation for 6 months or longer, with a median duration of 18.5 months from stopping iron chelation to last follow-up.

Erythropoiesis as determined by soluble transferrin receptor level was also improved in transfusion-independent patients. Bone marrow biopsies showed improvement in the myeloid:erythroid ratio.

Beti-cel adds functional copies of a modified form of the -globin (A-T87Q-globin) gene into a patients own hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells using a BB305 lentiviral vector. Following single-agent busulfan myeloablative conditioning, beti-cel is infused, after which the transduced HSCs engraft and reconstitute red blood cells containing functional adult hemoglobin derived from the gene therapy.

Of the 60 patients treated, 43 were genotype non-/ and 17 were / . The median age at consent was 20 years in the phase 1/2 trials and 15 years in the phase 3 trials. Median LIC at baseline was 7.1 and 5.5 mg Fe/g dw, respectively, and median cardiac T2 was 34 and 37 msec, respectively. The vector copy number was 0.8 in the phase 1/2 trial and 3.0 in the phase 3 study. Additionally, 32t and 78t CD34+ cells were transduced, respectively.

The phase 1/2 studies showed promising results but lower achievement of transfusion independence in patients with the / genotype, leading to a refinement in the manufacturing process, which resulted in a higher number of transduced cells and a higher number of vector copy number, said Hongeng.

The median time to neutrophil engraftment was 22.5 days and the median time to platelet engraftment was 44 days. Lymphocyte subsets were generally within the normal range after beti-cel infusion, which is different from allogeneic stem cell [transplantation], which is probably around 6 months to a year to get complete recovery of immune reconstitution, he said. The median duration of hospitalization was 42 days.

All patients were alive at the last follow-up (March 3, 2020). Eleven of 60 (18%) of patients experienced at least 1 adverse event (AE) considered related or possibly related to beti-cel, the most common being abdominal pain (8%) and thrombocytopenia (5%). Serious AEs were those expected after myeloablative conditioning: veno-occlusive liver disease (8%), neutropenia (5%), pyrexia (5%), thrombocytopenia (5%), and appendicitis, febrile neutropenia, major depression, and stomatitis (3% each).

Of the 7 patients experiencing veno-occlusive liver disease, 3 were of grade 4 and 2 were of grade 3. Two other patients had grade 2 veno-occlusive disease. There were no cases of insertional oncogenesis.

Persistent vector-positive hematopoietic cells and durable HbaT87Q levels supported stable total hemoglobin over time. In phase 3 trials, the median peripheral blood vector copy number was 1.2 c/dg at month 12 and 2.0 c/dg at month 24, and the median total hemoglobin was 11.5 g/dL at month 12 and 12.9 g/dL at month 24.

The weighted average of hemoglobin during transfusion independence in the phase 1/2 trials was 10.4 g/dL, and patients were transfusion-independent for a median of 51.2 months. In the phase 3 studies, the weighted average of hemoglobin during transfusion independence was 11.9 g/dL, and patients were transfusion-independent for a medium 17.7 months.

Hongeng S, Thompson AA, Kwiatkowski JL, et al. Efficacy and safety of betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy in 60 patients with transfusion-dependent -thalassemia (TDT) followed for up to 6 years post-infusion. Presented at: 2021 Transplantation & Cellular Therapy Meetings; February 8-12, 2021; virtual. Abstract 1.

More:
Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions - OncLive

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, announced today that the company will present at the 10th Annual SVB Leerink Global Healthcare Conference taking place February 22-26, 2021. Details on the presentation can be found below.

Company management will also participate in one-on-one investor meetings at the conference.

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Contacts:

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

Continued here:
Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference - GlobeNewswire

As the world charges to vaccinate the population against the coronavirus, gene therapy developers are locked in a counterintuitive race. Instead of training the immune system to recognize and combat a virus, theyre trying to do the opposite: designing viruses the body has never seen, and cant fight back against.

Its OK, really: These are adeno-associated viruses, which are common and rarely cause symptoms. That makes them the perfect vehicle for gene therapies, which aim to treat hereditary conditions caused by a single faulty gene. But they introduce a unique challenge: Because these viruses already circulate widely, patients immune systems may recognize the engineered vectors and clobber them into submission before they can do their job.

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Scientists use machine learning to tackle a big challenge in gene therapy - STAT

The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Europe Cell and Gene Therapy Market Report

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually.

Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes.

There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields.

The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Story continues

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies.

While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe.

Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patient's quality of life. Cell and gene therapies for certain types of cancers have been promising results.

The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer.

Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

KEY QUESTIONS ANSWERED

1. What is the Europe cell and gene therapy market size and growth rate during the forecast period?

2. What are the factors driving the demand for CAR-T therapy in the European region?

3. How are strategic acquisitions aiding in market growth of cell and gene therapy products?

4. Which segments are expected to generate the highest revenues during the forecast period?

5. Who are the leading vendors in the European cell and gene therapy market?

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors.

While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors

Novartis

Spark Therapeutics

Amgen

Gilead Sciences

Organogenesis

Other Prominent Vendors

APAC Biotech

AVITA Medical

bluebird bio

CHIESI Farmaceutici

CollPlant

CO.DON

Human Stem Cells Institute PJSC (HSCI)

Medipost

NuVasive

Nipro

Orchard Therapeutics

RMS Regenerative Medical System

Orthocell

Osiris Therapeutics

Sibino GeneTech

Shanghai Sunway Biotech

Takeda Pharmaceutical Company

Terumo

Vericel

Emerging Investigational Vendors In Europe

Adaptimmune Therapeutics

AgenTus Therapeutics

Autolus

Cellecits

Celyad

CombiGene

EUKARYS

Freeline Therapeutics

Innoskel

PsiOxus Therapeutics Ltd

SparingVision

uniQure

For more information about this report visit https://www.researchandmarkets.com/r/6gqw7e

View source version on businesswire.com: https://www.businesswire.com/news/home/20210212005462/en/

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Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -...

Close to a year after Maryland-based CDMO Vigene Biosciences cut the ribbon on its headquarters, spiking demand for cell and gene therapy has prompted the company to lay out a major manufacturing upgrade in its home state.

Vigene is picking up a lease for 52,000-square-feet of manufacturing space in Montgomery County, Maryland, situated near its existing headquarters in Rockville. The expansion is set to bring the company's total lab and production space up to 110,000 square feet and, by 2025, will see up to 245 new hires join Vigene's current workforce of 125.

The new facility, located at 14200 Shady Grove Road, will complement existing R&D and manufacturing operations at Vigene's home base as the company faces growing demand for its cell and gene therapy products. Vigene's expansion has snared some financial perks from the state, too, including a $1,225,000 loan from the Maryland Department of Commerce, which is contingent on job creation and capital investment.

Innovation in Rare Disease: Making Progress with Cell & Gene Therapies A Webinar Series from the Rare Disease Innovations Institute and Syneos Health

Join patients and their families, legislators, industry experts, advocates and sponsors to discuss education initiatives, recent advancements and the future promise of cell and gene medicine, and current patient experiences with these therapies.

RELATED: Cognate beefs up cell, gene therapy manufacturing with new plants in U.S., EU

The company is keeping its own spending on the site under wraps, Jeffrey Hung, Ph.D., chief commercial officer of Vigene, said over email. "It suffices to say that we are going to invest heavily on the facility to qualify and commission it for commercial production purpose," he added.

With the new site, Vigene will add five more GMP suites to the 10 it operates now, Hung said. Specifically, the company plans to commission and set up two 2,000-liter single-use bioreactor suites, where upstream and downstream production trains will be located on the same floor for commercial viral vector production. Another floor will house multiple large-scale fermenters for commercial plasma production, he said.

Formed in 2012, Vigene specializes in gene therapies for patients with cancers and serious genetic disorders. It develops, manufactures and distributes adeno-associated viruses, lentiviruses, retroviruses, adenoviruses and plasmid viral vectors for gene delivery.

RELATED: Fujifilm continues CMDO expansion spree with $76M in funding for new Boston site

The company has checked into the COVID-19 fight, too, signing on to produce clinical materials for Maryland compatriot Altimmune's nasal vaccine candidate. Vigene in July agreed to churn out both drug substance and drug product for studies on the vaccine, which registered for a phase 1 trial in late December.

On Dec. 23, Altimmune revealed the FDA had slapped the investigational new drug application for its vaccine, AdCOVID, with a clinical hold, citing the need for protocol modifications and additional chemistry, manufacturing and control data. The company responded to the hold and, at the time, said it didn't expect the move to significantly disrupt its clinical timeline.

Altimmune has also added Swiss CDMO Lonza as a production partner on its nasal vaccine, and it previously set the goal to crank out at least 100 million AdCOVID doses in 2021.

Meanwhile, Vigene's expansion comes shortly after the christening of its Rockville HQ. It was just a year ago that we cut the ribbon at Vigenes new custom-built headquarters and already the growing demand for its gene and cellular therapy products requires additional physical expansion, Benjamin Wu, CEO and president of the company, said in a release.

See the article here:
CDMO Vigene plots cell and gene therapy manufacturing expansion, adding 245 new jobs along the way - FiercePharma

LONDON and LAUPHEIM, Germany, Feb. 11, 2021 (GLOBE NEWSWIRE) -- The Cell and Gene Therapy Catapult (CGT Catapult), an independent centre of excellence in innovation advancing the UKs cell and gene therapy industry, and Rentschler Biopharma SE, a leading global contract development and manufacturing organisation (CDMO) for biopharmaceuticals, have announced today that Rentschler Biopharma will establish their manufacturing capability in Advanced Therapy Medicinal Products (ATMPs), including Adeno-Associated Virus (AAV) Vectors for clinical trial supply, at the CGT Catapult site in Stevenage.

Under the terms of the agreement, Rentschler Biopharma will make a significant investment at the site over the next five years to set up their manufacturing capabilities, benefitting from the expertise and unique collaborative model provided by the CGT Catapult. The companys investment is expected to make a major contribution to meeting the demand from UK and international researchers for suitable manufacturing capability. This development will further strengthen the UK ecosystem through the addition of Rentschler Biopharmas more than 40 years of experience and solid reputation in the development and manufacturing of biologics for both clinical and commercial supply. The company will leverage the CGT Catapults expertise in ATMP manufacturing setup and technology development, as well as the cell and gene therapy cluster and ecosystem that has developed around Stevenage and across the UK.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said:We are excited to take this next big step in our evolution and address the growing industry demand for ATMP manufacturing capacity and viral vector supply. With the largest industry cluster for cell and gene therapies outside the US, the UK is an ideal location for us to establish our Center of Excellence for cell and gene therapy. We look forward to working with the CGT Catapult as we invest in this growing field. They are well established in this important market, enabling us to immediately tap into the organisations network and utilisethe UKs strong expertise and supply chain in cell and gene therapy manufacturing.

Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, commented:We are very pleased that Rentschler Biopharma, a global CDMO, has chosen to build their ATMP capacity in the UK, bringing in their expertise and investment. This will build new capacity to benefit the international ATMP supply chain and meet growing academic and commercial demand across the industry. As more companies from around the globe come to the UK, it demonstrates and enhances the attractiveness of its cell and gene therapy ecosystem as a place to develop new technologies and capabilities.

The investment in the UK cell and gene therapy industry announced today is expected to further accelerate the development of the vital infrastructure and skilled jobs needed to meet the rising demand for manufacturing capacity in the UK and globally, as well as streamline the supply chain for these advanced therapies. Currently, 27% of European ATMP companies are operating in the UK, and there are more than 90 advanced therapy developers. The last year has also seen a 50% increase in the number of ATMP clinical trials being run in the UK, accounting for 12% of global ATMP clinical trials, and these numbers are predicted to increase further.

The CGT Catapult manufacturing centre has been backed by over 75m of funding, including investment from the UK Governments Industrial Strategy Challenge Fund, the Department for Business, Energy and Industrial Strategy, Innovate UK and from the European Regional Development Fund. Since it was announced, there has been over 1.1bn of investment in the ATMP industry in its vicinity.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany, with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK.

For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the European Regional Development Fund

This project has received 3.36m of funding from the England European Regional Development Fund as part of the European Structural and Investment Funds Growth Programme 2014-2020. The Ministry of Housing, Communities and Local Government (and in London the intermediate body Greater London Authority) is the Managing Authority for European Regional Development Fund. Established by the European Union, the European Regional Development Fund helps local areas stimulate their economic development by investing in projects which will support innovation, businesses, create jobs and local community regenerations. For more information visit https://www.gov.uk/european-growth-funding.

About the Industrial Strategy Challenge Fund

This project has received 12m of funding from the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The Industrial Strategy Challenge Fund is a four-year, 1 billion investment in cutting-edge technology designed to create jobs and improve living standards, built on guidance from business and the academic community. Healthcare and Medicine is one of three core areas for investment under the programme.

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Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace

WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, has signed a partnership agreement with Genethon, a pioneering research center in the field of gene therapy.The alliance will harness the power of artificial intelligence to accelerate development of innovative gene therapies.

As part of this partnership, Genethon teams will use WhiteLab Genomics CatalystTM platform to develop new capsids, or vectors, which are essential components for gene therapy products.

While several gene therapy products have already obtained market authorization for the treatment of rare and common diseases, which demonstrates the efficacy of this approach for conditions considered to be incurable, development of these complex therapies continues to face major scientific and technical hurdles. Many vectors used in gene therapy are derivatives of adeno-associated viruses (AAV). Their use has limitations: natural immunization of 30% to 40% of the population and difficulty targeting a specific tissue. As a result, extremely large quantities of vectors are necessary. In this context, the use of artificial intelligence solutions stands out as a deciding factor to overcome these obstacles and produce optimized vectors that better target the relevant tissues, thus making it possible to inject smaller quantities of product while maximizing the effect of the therapy.

Turning to AI for faster development of optimized vectors

Genethons teams will use WhiteLab Genomics CatalystTM platform to accelerate select research programs.

Thanks to its Machine Learning algorithms, the WhiteLab Genomics platform will help researchers develop next-generation gene therapy vectors, with a view to enhancing their precision with regard to the tissues to be treated while reducing their immunogenic qualities.

"The tools developed by WhiteLab will make it possible for us to review thousands of sequences and devise new and innovative combinations. We aim to develop a new generation of more specific AAV vectors, contributing to the emergence of original treatments for neuromuscular disorders," said Dr. Ronzitti, who is managing the collaboration for Genethon.

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"We are thrilled to be working together with worldwide trailblazers and experts in the area of gene therapy," stated David Del Bourgo, CEO and co-founder of WhiteLab Genomics. "France is a prime source of innovation in this field, and we look forward to helping research teams, in France and abroad, to make practical use of these extremely complex biological datasets, while also providing assistance to accelerate the development of optimized products."

About White Lab Genomics

Founded in 2019 by David Del Bourgo, Julien Cottineau and Lucia Cinque, WhiteLab Genomics is a French start-up specializing in artificial intelligence solutions dedicated to biotherapies, such as gene and cell therapies. The companys proprietary technology allows for multi-parameter analysis of complex biological data to optimize these treatments while reducing development costs. WhiteLab Genomics provides this unique technology to its clients via the Catalyst platform, available in SaaS mode. Today, the start-up has locations at the Evry Gnopole Frances first biocluster and at Station F. WhiteLab Genomics was recently ranked among Station Fs "Future 40," an index of the 40 most promising companies within Europes largest start-up incubator. https://www.whitelabgx.com

About Genethon

Created by AFM-Telethon, Genethon is a non-profit research and development center dedicated to creating gene therapy products for rare diseases, from initial research to clinical validation. Genethon has several programs underway, in clinical, pre-clinical and research phases, to treat rare muscular, blood, immune system and liver disorders. Today, a product incorporating technologies developed thanks to Genethons pioneering research is available on the market in the United States, Europe and Japan to treat spinal muscular atrophy. Ten other products created through Genethon R&D, alone or in collaboration with partners, are at the clinical trial stage, while many more are slated to begin clinical trials in 2021 and 2022. Genethon.fr

View source version on businesswire.com: https://www.businesswire.com/news/home/20210210005707/en/

Contacts

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Beyond Communication Najette Chaib nchaib@beyondcom.fr +33 6 18 38 11 14

WhiteLab Genomics David Del Bourgo - contact@whitelabgx.com

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Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence - Yahoo Finance

TipRanks

We are indeed living in interesting times and in many ways, thats a good thing. Take the automotive industry, for example. Technology is changing a rapid pace, and when it settles, it will dramatically change the way we drive. In 2030, our concept of car will likely be unrecognizable to drivers from 1980. The biggest changes are coming from power systems and artificial intelligence. AI will bring autonomous tech to our cars, making self-driving vehicles a reality. But the power systems changes will hit us first. In fact, electric-drive vehicles are already on our roads, and electric vehicle (EV) companies are proliferating rapidly. For the moment, there are several roads to potential success in the EV market. Companies are working to position themselves as leaders in battery tech, or electric power trains, or to maximize their range and performance per charge. Its a fact-paced industry environment, offering both opportunity and excitement for investors. Smart investors will look for companies capable of meeting scaling demands, once they have settled on marketable models. Investment firm Morgan Stanley has been watching the EV industry, seeking out innovative new design and production companies that are positioning themselves for gains as the market matures. The firms automotive analyst, Adam Jonas, has selected two stocks that investors should seriously consider buying into, saying As we survey the EV/battery startup landscape, we are prioritizing highly differentiated technology and/or business models with a path to scale at a reasonable level of risk. Opening up the TipRanks database, weve pulled up the details on both of Jonas picks to see whether they could be a good fit for your portfolio. Fisker (FSR) First up, Fisker, is based in Southern California, the epicenter of so much of our ground-breaking tech industries. Fiskers focus is on solid-state battery tech, a growing alternative to the lithium-ion batteries that most EVs depend on. While more expensive that the older lithium-based systems, solid state batteries are safer and offer higher energy densities. Fisker has been busy patenting its moves into solid-state batteries, a sound strategy to lock in its advances in this field. For EVs, solid-state batteries offer faster charging times, longer range per charge, and potentially lower battery weight all important factors in vehicle performance. Every car company needs a flagship model, and Fisker has the Ocean an EV SUV with a mid-range price ($37,499) and a long-range power system (up to 300 miles). The vehicle features stylish design and room mounted solar panels to supplement the charging system, and is scheduled to enter serial production for the markets in 2022. The stylish design reflects the sensibilities of the companys founder, Henrik Fisker, known for his work on the BMW Z8 and the Aston Martin DB9. Fisker entered the public markets through a SPAC merger agreement last fall. Since completing the SPAC transaction on October 29, shares in FSR are up 112%. Morgan Stanleys Jonas is impressed by this company, describing the value proposition of Fisker as design, time to market, clean sheet user experience and management expertise, and saying that the 4Q22 launch schedule for the Ocean is likely to be met. Fisker is specifically targeting the personal owned/passenger car business as opposed to commercial oriented end markets, where emotive design and user experience matter more. Additionally, the company wants to create an all-digital experience from the website to the app to the HMI in the car and continued customer engagement through its flexible lease product, Jonas added. In line with his upbeat outlook on the company (and the car), Jonas rates Fisker an Overweight (i.e. Buy), and sets a $27 price target suggesting an upside of 42% for the coming year. (To watch Jonas track record, click here) Turning to the TipRanks data, weve found that Wall Streets analysts hold a range of views on Fisker. The stock has a Moderate Buy analyst consensus rating, based on 7 reviews, including 4 Buys, 2 Holds, and 1 Sell. Shares are currently priced at $18.99, and the $21.20 average price target implies a one-year upside of ~12%. (See FSR stock analysis on TipRanks) QuantumScape (QS) Where Fisker is working on solid-state batteries in the context of vehicle production, QuantumScape is setting itself up as a leader in EV battery technology and a potential supplier of the next generation of battery and power systems for the EV market. QuantumScape designs and builds solid-state lithium-metal batteries, the highest energy density battery system currently available. The key advantages of the technology are in safety, lifespan, and charging times. Solid-state batteries are non-flammable; they last longer than lithium-ion batteries, with less capacity loss at the anode interface; and their composition allows faster charging, of 15 minutes or less to reach 80% capacity. QuantumScape is betting that these advantages will outweigh the technologys current higher cost, and create a new standard in EV power systems. The companys strongest tie to the EV production field is its connection with Volkswagen. The German auto giant put $100 million into QuantumScape in 2018, and an additional $200 million in 2020. The two companies are using their partnership to prepare for mass-scale development and production of solid-state batteries. Like Fisker, QuantumScape went public through a SPAC agreement late last year. The agreement, which closed on November 27, put the QS ticker in the public markets where it promptly surged above $130 per share. While the stock has since slipped, it remains up 47% from its NYSE opening. For Morgan Stanleys Jonas, involvement in QS stock comes with high risk, but also high potential reward. In fact, the analyst calls it, "The Biotech of Battery Development." "We believe their solid state technology addresses a very big impediment in battery science (energy density) that, if successful, can create extremely high value to a wide range of customers in the auto industry and beyond. The risks of moving from a single layer cell to a production car are high, but we think these are balanced by the commercial potential and the role of Volkswagen to help underwrite the early manufacturing ramp," Jonas explained. Noting that QS is a stock for the long haul, Jonas rates the shares an Overweight (i.e. Buy), and his $70 price target indicates confidence in an upside of 28% for one-year time horizon. Granted, not everyone is as enthusiastic about QS as Morgan Stanly. QS's Hold consensus rating is based on an even split between Buy, Hold, and Sell reviews. The shares are priced at $54.64 and their recent appreciation has pushed them well above the $46.67 average price target. (See QS stock analysis on TipRanks) To find good ideas for EV stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analyst. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Gene Therapy Partnering Deals Terms and Agreements Directory 2010-2020: Company A-Z, Deal Value, Phase of Development, Deal Type, and Therapy...

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

Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announces that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LYS-GM101, the companys gene therapy candidate for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease. LYS-GM101 builds on Lysogenes extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.

The IND clearance follows the recent clinical trial authorization granted by the MHRA in the United Kingdom. Lysogene intends to initiate its global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The clinical trial will include a safety phase and a confirmatory efficacy phase. The company intends to dose a total of 16 patients, with dosage of the first patient expected in the first half of 2021.

We are very pleased to receive this IND clearance for LYS-GM101 which completes the MHRA approval received a few weeks ago. It represents a major milestone that marks our second CNS gene-therapy program to enter into a global clinical trial said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene. This IND clearance once again demonstrates our quality and timely execution, and our strong determination to bring new therapeutic solutions for diseases that currently have no treatment.

Christine Waggoner, President and Co-Founder of Cure GM1 Foundation added: Children with GM1 gangliosidosis represent a clear unmet medical need and we are thrilled to see a new therapeutic option entering the clinic, as it brings tremendous hope to families and the entire GM1 gangliosidosis community.

LYS-GM101 (adeno-associated viral vector serotype rh.10 expressing beta-galactosidase) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as Rare Pediatric Disease designation in the US in 2016.

Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors in infantile and juvenile GM1 gangliosidosis (NCT04310163).

About LysogeneLysogene 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 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. An adaptative 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 StatementThis press release may contain certain forward-looking statements, especially on the Companys progress of its clinical trials 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 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, 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 Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101 for the...