StemCell Therapy

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Charles River Laboratories will acquire Cognate BioServices, a cell and gene therapy contract development and manufacturing organization, in an $875 million cash deal that will significantly expand the companys capabilities in the high-growth cell and gene therapy sector.

In addition to expanding Charles Rivers capabilities in the cell and gene therapy space, the company said the acquisition of Memphis, Tenn.-based Cognate will also establish a comprehensive solution from discovery and non-clinical development through CGMP manufacturing in advanced drug modalities. Cognate has a global presence with more than 500 employees. In January, Cognate announced plans to significantly expand cell and gene therapy manufacturing capacity, laboratory space, warehousing capabilities, and increase office support at its facilities inthe United StatesandEurope.

The company has extensive experience producing various cell types and technologies used in cellular immunotherapy and immuno-oncology, regenerative medicine and advanced cell therapy. Its primary area of expertise is in CGMP cell therapy manufacturing, which processes a variety of cellular products and other starting materials to develop and produce allogeneic (donor-derived) and autologous (patient-derived) cell therapies. Cognate also produces plasmid DNA, which is a foundational tool used in the development of gene-modified cell therapies and gene therapies, as well as other CDMO inputs, Charles River said.

James C. Foster, president and chief executive officer of Charles River Labs, said Cognate provides a synergistic fit for Charles River. He said bringing Cognate under the companys umbrella presents a unique opportunity to expand the companys capabilities and also enhance its offerings to clients in emerging areas of scientific innovation.

Additionally, Charles River said the addition of Cognate will complement its existing Biologics Testing Solutions business. Cognates capabilities will enable clients to seamlessly conduct analytical testing, process development, and manufacturing for advanced modalities with the same scientific partner, enabling them to achieve their goal of driving greater efficiency, the company said.

The addition of Cognate is also expected to provide a significant financial boost to Charles River. Cognate is expected to generate annual revenue of approximately $140 million in 2021, and is expected to grow at least 25% annually over the next five years, the company said.

This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions and enabling us to achieve our goal of establishing a single scientific partner to provide biopharmaceutical clients with an integrated solution to help accelerate their cell and gene therapy programs from discovery and non-clinical development through commercialization, Foster said in a statement. Because of the synergistic fit with Charles River, the market growth potential, and the emerging role of advanced drug modalities as treatments for oncology and rare disease, we believe Cognate will meaningfully enhance our long-term revenue and earnings growth potential.

The acquisition is expected to move quickly. Charles River anticipates the closing of the deal by the end of the first quarter of 2021. Based on the anticipated completion of the acquisition by the end of the first quarter, Cognate is expected to add approximately $110 million to Charles Rivers 2021 consolidated revenue for the partial year.

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Charles River Dives Deep Into Cell and Gene Therapy With Cognate BioServices Acquisition - BioSpace

Two Kolon Life Science executives were acquitted Friday of involvement in the company's allegedly fraudulent report on a key ingredient in its gene therapy drug.

The Seoul Central District Court handed down the ruling to the drugmaker's two executives, surnamed Cho and Kim, who were indicted on charges that included fraud related to the company's suspected illegalities in the development and sales of Invossa, a cell and gene therapy for osteoarthritis.

But the court separately ordered Cho, a senior director at the company's clinical development team, to pay a fine of 5 million won ($4,500) for bribing an official at the Ministry of Food and Drug Safety in seeking favors in the company's drug development process.

They were suspected of having been involved in submitting fraudulent documents on purpose to the ministry to get approval for Invossa.

While acknowledging that the company submitted some false data, the court said the ministry seemed to have failed to perform due diligence in verifying the submitted documents.

The court also found them not guilty of fraud and other charges for having received a state subsidy of 8.2 billion won based on allegedly fraudulent documents.

Kolon Life Science, a unit of Kolon Group, initially received the approval for Invossa in 2017. But it was revoked in May 2019 after it was revealed that a key material used in Invossa came from a kidney cell, instead of from cartilage as stated in the document submitted for approval.

The company acknowledged that a substance in the joint pain treatment drug had been mislabeled, but claimed no one has suffered from any medical complications from the drug use.

Later in the day, however, the Seoul Administrative Court ruled against the drugmaker, dismissing its plea to reinstate the license for Invossa.

Having submitted false data was "a grave fault as drugs can have a significant effect on people's lives and health," the court said, even though it was hard to confirm with the evidence presented that the company intentionally committed any wrongdoing.

"There is no illegality with the ministry's decision to revoke the license since it did not seem to be aware of data that could raise suspicions about the drug's safety," the court ruled. (Yonhap)

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Kolon Life executives acquitted of falsifying data of gene therapy drug - The Korea Herald

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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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.

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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)

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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.

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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

While youre receiving treatment for cancer, some of the drugs you take can cause painful sores to develop inside your mouth. You can also get them if youve had a bone marrow (stem cell) transplant as part of your cancer care.

Although they often heal on their own, these mouth sores can make it uncomfortable to eat and talk. Well discuss what you can do to relieve the pain and prevent them from getting worse.

Mouth sores can be a common side effect of cancer treatment. The condition, known as stomatitis or mucositis, is an inflammation of the tissues inside your mouth.

Whitish, ulcer-like sores can form on your cheeks, gums, lips, tongue, or on the roof or floor of your mouth. Even if you dont develop mouth ulcers, you may have patches that feel inflamed and painful, as if theyve been burned.

Anyone who is receiving chemotherapy, radiation therapy, or a bone marrow (stem cell) transplant can develop mouth sores as a side effect of these treatments.

If you have dry mouth or gum disease, or if your teeth and gums are not well taken care of, you may be at a higher risk of getting mouth sores during your treatment. Women and people who smoke or drink alcohol are also at a higher risk, according to the Oral Cancer Foundation.

If youre receiving chemotherapy, the sores could begin forming anywhere from 5 days to 2 weeks after your treatment. Depending on the specific cause, the sores could go away on their own in a few weeks, or they could last longer.

Its important to find ways to manage your pain and to watch for signs of an infection. Cancer-related mouth sores can lead to weight loss, dehydration, and other serious complications.

Cancer cells can grow very quickly. The aim of cancer treatment is to stop or slow down that growth. The cells in the mucous membranes lining your mouth are also fast-growing cells, so cancer treatments affect them, too.

Cancer treatments also keep the cells in your mouth from being able to repair themselves efficiently when theyre damaged.

Radiation therapy can also damage the glands in your mouth that make saliva. A dry mouth is more susceptible to infections that cause mouth sores.

Chemotherapy and radiation can both change the microbiome in your mouth, upsetting the balance between good and bad bacteria. The growth of harmful bacteria in your mouth can also lead to mouth sores.

Sometimes cancer treatments suppress your immune system, which may make it more likely that youll get a bacterial, viral, or fungal infection that causes mouth sores. An older infection (such as the herpes simplex virus) can also suddenly flare up again.

If youve had a bone marrow (stem cell) transplant, sores may be a sign that youve developed a condition known as graft-versus-host disease (GVHD).

When this happens, the cells in your body are attacking the transplanted cells as though they were an unhealthy invader. According to research published in Journal of Clinical and Experimental Dentistry, short-term (acute) GVHD occurs in 50 to 70 percent of stem cell transplant cases and longer-term (chronic) GVHD is seen in 30 to 50 percent of cases.

The form of GVHD that causes mouth sores is usually mild, and doctors often treat it with corticosteroid medications.

Its important to talk with your doctor if you develop mouth sores after a stem cell transplant, as some kinds of GVHD can turn serious if left untreated.

There is a good chance that youll experience mouth sores at some point during your cancer treatment. Researchers estimate that 20 to 40 percent of those who have chemotherapy and 80 percent of those who have high-dose chemotherapy will develop mucositis afterward.

Still, there are steps you and your cancer care team can take to lower your risk, reduce the severity of the sores, and promote faster healing.

About a month before your cancer treatment begins, schedule an appointment with your dentist to make sure your teeth and gums are healthy. If you have cavities, broken teeth, or gum disease, its important to come up with a dental treatment plan to take care of these conditions so they dont lead to infections later, when your immune system may be vulnerable.

If you wear braces or dentures, ask your dentist to check the fit and remove any part of the device you dont need during your treatment.

Its very important to maintain good oral hygiene practices throughout your treatment to lower your risk of infection. Brush and floss gently but regularly, avoiding any painful areas. You can also ask your dentist whether a mouth rinse with fluoride is advisable in your case.

For certain kinds of chemotherapy (bolus 5fluorouracil chemotherapy and some high-dose therapies), your healthcare team may give you ice chips to chew for 30 minutes before your treatment. This type of cold therapy can lower your risk of getting mouth sores later.

During treatment of some blood cancers, doctors may give you injections of palifermin, also known as human keratinocyte growth factor-1 (KGF-1), to prevent mouth sores.

If youre scheduled to receive high-dose chemotherapy or radiotherapy, your cancer care team may prepare your mouth using low-level laser therapy beforehand to keep you from getting mouth sores.

For people who have radiation therapy for head and neck cancers, doctors may prescribe this medicated mouthwash to minimize mouth sores.

The length of time your mouth sores may last depends on the specific cancer treatment youve had. Here are some estimates broken down by treatment:

You may notice symptoms anywhere between a few days and a few weeks after your cancer treatment. Heres what you may see and feel as mucositis develops:

You may notice that the sores become slightly crusty as they heal. Its important to keep track of your symptoms and let your oncologist know if the sores arent healing on their own.

Contact your doctor right away if you:

Untreated mouth sores can lead to malnutrition, dehydration, and life-threatening infections.

There are a few different ways that you can help mouth sores heal and avoid prolonger pain or an infection.

While the sores are healing, its very important to keep the inside of your mouth clean to prevent an infection from developing.

The National Cancer Institute recommends that you gently clean your teeth every 4 hours and just before you go to sleep at night. Here are a few tips to consider:

If the pain from mouth sores is interfering with your ability to eat and drink, your doctor may treat the condition with a opioid mouthwash or one containing doxepin or lidocaine.

To ease discomfort and keep your mouth from feeling dry, you may want to try rinsing with a mild saltwater or baking soda solution. Heres how to make each of them:

Your cancer care team may recommend that you use a lubricating liquid (artificial saliva) to moisten the inside of your mouth if dryness is a problem. These liquids are usually gel-like. They coat your mouth with a thin film to help ease discomfort and promote healing.

Some people have found it useful to rinse with a blend of medications called the magic mouthwash. Formulas for this mouthwash vary, but most of them include a combination of medications to treat different symptoms, including:

Magic or miracle mouthwash solutions usually have to be prescribed by a doctor and prepared by a pharmacist, although some people mix up an over-the-counter version at home.

There isnt enough research to say for sure whether magic mouthwash works. If you think youd like to try it, talk with your oncologist or a healthcare professional about whether its a good idea for you.

Here are a few more things you can try at home that may help ease pain from mouth sores:

Mouth sores are one of the most common side effects of cancer treatment. Shortly after chemotherapy, radiation, or transplant treatments, painful, ulcer-like sores can form on the inside of your mouth.

These sores may go away on their own. If they dont, its important to seek medical treatment for them because they can lead to very serious complications.

Before you start cancer treatments, visit a dentist to make sure your teeth and gums are healthy. Keeping up good dental hygiene practices during and after cancer treatment will help limit mouth sores.

If the sores are keeping you from eating and drinking, talk with your oncologist about medications could relieve the pain and speed up the healing process, so you can enjoy a better quality of life during treatment.

Its really important to keep track of any sores in your mouth so you can reach out to your healthcare team if they dont improve. Sores that deepen or worsen can lead to serious even life-threatening complications.

See the article here:
Mouth Sores from Chemo: Symptoms, Causes, and Treatments - Healthline

By Ernie Mundell and Robert Preidt HealthDay Reporters

THURSDAY, Feb. 18, 2021 (HealthDay News) -- Diabetes is a big risk factor for a severe bout of COVID-19, and a new European study bears that out: It finds that 1 in every 5 hospitalized COVID-19 patients with diabetes die within 28 days of admission.

One U.S. expert wasn't surprised by that grim finding.

"Diabetic patients are clearly in a very high-risk category and should be among the first groups of people to get the vaccine," advised Dr. Mangala Narasimhan, who directs critical care services at Northwell Health in New Hyde Park, N.Y. She also advises people with diabetes to make sure they are taking control of their blood sugar levels and avoiding any complications of the disease.

Such steps "seem to really make a difference in terms of survival from COVID infection," said Narasimhan, who wasn't involved in the new study.

The research was led by Bertrand Cariou and Samy Hadjadj, diabetologists at University Hospital Nantes in France. In May of last year they had released preliminary findings that showed that 10% of COVID-19 patients with diabetes died within seven days of hospital admission.

The newer, updated results are from a larger number of patients -- close to 2,800 -- treated for COVID-19 at 68 hospitals across France. Their mean age was 70, nearly two-thirds were men, and many were overweight. About 40% were also experiencing various forms of complications from their diabetes.

During the 28 days after their admission to a hospital, 21% of patients died, the French team reported Feb. 17 in the journal Diabetologia.

Of those patients who survived at least one month, 50% were discharged from the hospital with a median stay of nine days; 12% were still hospitalized at day 28, and 17% had been transferred from their first hospital to another facility.

Younger age, routine diabetes therapy using the drug metformin, and having had symptoms longer prior to hospital admission were key factors associated with a higher likelihood of being discharged from the hospital, the researchers said.

Patients who regularly took insulin -- possibly indicating more advanced diabetes -- had a 44% higher risk of death than those who didn't take insulin, the investigators said. Long-term blood sugar control wasn't associated with patient outcomes, but a higher level of blood sugar at the time of hospital admission was a strong predictor of death and of a lower chance of discharge.

Dr. Barbara Keber directs family medicine at Glen Cove Hospital in Glen Cove, N.Y. Reading over the findings, she said they show "diabetes is clearly a significant risk factor for both need for ICU/ventilator care in the hospital as well as for death" within a month of admission.

Keber said it "makes sense" that people with complications from poorly controlled diabetes are at higher risk, since this creates a "pro-inflammatory state" that is similar to that seen in advanced COVID-19.

But Keber also cautioned that death rates may have improved for COVID-19 patients, including those with diabetes, over the past year.

"This study was done in the first wave of the pandemic, and many of the current treatment regimens and medications that were tried in the early phase have been found to not be beneficial and other treatment regimens have taken their place," she noted.

For example, "the current use of steroids for treatment may play a role in the [improved] prognosis of patients overall and especially for those with diabetes," Keber said.

More information

The American Diabetes Association has more on COVID-19.

SOURCES: Mangala Narasimhan, DO, director, critical care services, Northwell Health, New Hyde Park, N.Y.; Barbara Keber, MD, chair, family medicine, Glen Cove Hospital, Glen Cove, N.Y.; Diabetologia, news release, Feb. 17, 2021

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COVID Deaths High When Hospitalized With Diabetes - WebMD

Diabetic retinopathy is an eye disease that affects people living with diabetes. It develops when high blood sugar damages the tiny blood vessels in the retina. This causes a variety of symptoms like blurry vision and vision loss.

This progressive disease may lead to irreversible vision loss, so its important to have regular eye exams. A doctor can then diagnose the condition early and slow its progression.

Glucose, or blood sugar, is a main source of energy yet too much circulating in the blood can be harmful to the body.

Typically, the pancreas releases the hormone insulin, which helps cells absorb glucose for energy. In the case of diabetes, though, the body doesnt make enough insulin or doesnt use it properly. This causes glucose to accumulate in the blood.

Consistent levels of high blood sugar can affect different parts of the body, including the eyes.

Diabetic retinopathy doesnt only weaken or damage the blood vessels in the eye. It can also cause the development of new abnormal blood vessels in the retina.

Diabetic retinopathy is a progressive eye disease classified by two types and four stages.

The two types are nonproliferative and proliferative. Nonproliferative refers to early stages of the disease, while proliferative is an advanced form of the disease.

This is the earliest stage of diabetic retinopathy, characterized by tiny areas of swelling in the blood vessels of the retina. These areas of swelling are known as micro aneurysms.

Small amounts of fluid can leak into the retina at the stage, triggering swelling of the macula. This is an area near the center of the retina.

Increased swelling of tiny blood vessels starts to interfere with blood flow to the retina, preventing proper nourishment. This causes an accumulation of blood and other fluids in the macula.

A larger section of blood vessels in the retina become blocked, causing a significant decrease in blood flow to this area. At this point, the body receives signals to start growing new blood vessels in the retina.

This is an advanced stage of the disease, in which new blood vessels form in the retina. Since these blood vessels are often fragile, theres a higher risk of fluid leakage. This triggers different vision problems such as blurriness, reduced field of vision, and even blindness.

Diabetic retinopathy doesnt usually cause symptoms during the nonproliferative stages, so its possible to have it and not know it. This is because blood vessels dont always leak in these stages.

Many people dont have symptoms until the disease progresses to proliferative diabetic retinopathy.

However, an eye examination by an eye care specialist or ophthalmologist can detect diabetic retinopathy in its earlier stages, before symptoms become apparent.

Symptoms of proliferative diabetic retinopathy include:

Be mindful, too, that diabetic retinopathy symptoms usually affect both eyes at the same time.

To diagnose diabetic retinopathy, your doctor may complete a comprehensive eye examination. This involves measuring:

Your doctor will likely also dilate your eye to examine your optic nerve and retina using special eye drops.

Doctors can also diagnose diabetic retinopathy with fluorescein angiography, which checks for abnormal blood vessel growth or leakage.

Theyll inject a yellow dye into a vein in your arm, allowing the dye to travel through your blood vessels. A special camera takes images of the dye as it travels through the blood vessels in your retina.

Diabetic retinopathy may lead to irreversible vision loss, but it is treatable. Treatment starts with managing blood sugar and diabetes. This includes taking diabetes medication as directed, watching your diet, and increasing physical activity.

Keeping blood sugar within a healthy range can slow the progression of vision loss.

Other treatments will depend on the stage or extent of the disease. If caught very early before damage to the retina occurs blood sugar management might be the only necessary treatment. Your doctor will continue to monitor your eyes, though, to ensure the disease doesnt progress.

If youre in a nonproliferative stage but experience some eye damage, treatment options might include:

Preventing diabetic retinopathy starts with managing blood sugar.

This involves managing diabetes with medication, balanced eating habits, and regular physical activity. You should also monitor your blood sugar on a regular basis and speak with your doctor if your levels are difficult to manage.

A healthy diet consists of:

Diabetes management may also involve other changes. This can include managing your blood pressure and cholesterol and avoiding tobacco.

Diabetic retinopathy isnt the only complication of diabetes. Blood sugar levels outside of a healthy range can cause other long-term issues, such as:

It may also lead to other conditions involving significant vision loss or blindness, such as:

If you have diabetes, make an appointment to see an eye care specialist such as an ophthalmologist at least once a year, or as often as your doctor recommends.

You should also see your doctor if your glucose level remains high despite medication and other changes, or if you notice any changes in vision, even if they are subtle.

Diabetic retinopathy is a potentially serious eye disease that can result in permanent distorted vision or loss of vision. Any changes in vision, such as blurriness, poor night vision, and an increase of eye floaters, should prompt a trip to the eye doctor.

Speak with your eye care specialist to diagnose any possible eye conditions. Although diabetic retinopathy isnt reversible, it is treatable.

Read more:
Diabetic Retinopathy Stages: The 4 Stages and What to Do - Healthline

Managing diabetes and keeping your blood sugar within a healthy range doesnt only protect against heart attacks and stroke, it can also keep your feet healthy.

Diabetes is a condition where the body doesnt produce enough insulin or use insulin properly, causing sugar levels in the blood to rise above normal. Uncontrolled high blood sugar can reduce blood flow in your feet, leading to serious complications.

Paying attention to your foot healthwhich includes recognizing early signs of problemsand maintaining a healthy blood sugar lowers the risk for complications.

Prolonged high blood sugar can gradually damage your blood vessels, restricting blood flow to your organs and other parts of your body. Lack of blood flow can lead to heart disease, stroke, kidney problems, and even vision problems.

Blood vessel damage also affects blood flow to your feet, causing a number of foot health issues.

According to the Centers for Disease Control and Prevention (CDC), about half of people living with diabetes will develop some kind of diabetic neuropathy or nerve damage. This damage can occur anywhere in the body, but usually affects the nerves in the feet and the legs.

Nerve damage can cause a tingling sensation and pain in your feet. As your condition worsens, you might lose all feeling in your feet. This is when diabetic neuropathy becomes dangerous.

Pain is a warning that something isnt right in the body. It can alert you to cuts, sores, and blisters on your feet. But if you have diabetic neuropathy and lose feeling in your feet, a cut or blister could go unnoticed for an extended length of time. If you dont receive prompt treatment for these types of injuries, you could develop an infection.

Diabetic neuropathy can lead to other complications. Reduced blood flow to your feet means that sores or infections might not heal as easily. Infections that dont heal can progress to gangrene, which is death of tissue due to lack of blood flow.

If gangrene starts to affect other parts of your body, your doctor might have to amputate a toe, foot, or leg to stop its spread.

Diabetes can also cause a circulation disorder known as peripheral vascular disease. This cardiovascular disease results from limited blood flow to the legs and feet. A blockage or narrowing of blood vessels also restricts blood flow.

This condition can occur in anyone, but the risk is higher in people with diabetes, because blood vessel changes often prevent the smooth flow of blood. Plus, high blood sugar can thicken blood to the point where it doesnt flow easily.

Nerve damage from diabetes can also trigger a rare condition known as Charcot foot. This typically occurs when a person has an injury, such as a sprain or fracture, that goes unnoticed due to lack of sensation caused by peripheral neuropathy. As the person continues to walk on the injured foot, it causes trauma to the bone.

Deformity occurs when joints become dislocated and collapse. The arch of the foot will often collapse, too, causing a roundness on the bottom of feet.

Along with foot deformity, other signs of Charcot foot include swelling, and your feet might appear red and warm to the touch.

A round bottom on feet also raises the risk of sores due to friction. If you have diabetic neuropathy and lose feeling in your feet, an open sore can become infected. This puts you at risk for amputation.

Poor blood circulation and blood flow can slow the healing process of sores on your feet, putting you at risk for serious life-threatening complications.

Even if you havent lost feeling in your feet, bring the following symptoms to your doctors attention. Signs of feet issues include:

You can avoid serious diabetes complications by seeing your doctor and getting treatment early for conditions that affect your feet.

Unfortunately, theres no cure for diabetic neuropathy. But you can take steps to slow the progression of this disease. Your doctor will likely recommend pain medication to help alleviate nerve pain.

For mild nerve pain, you can take over-the-counter medications like acetaminophen or ibuprofen. For moderate or severe pain, prescription medications like anti-seizure drugs and antidepressants can help ease nerve pain and improve the quality of your life, too.

Maintaining a healthy weight and regular physical activity can also slow the progression of diabetic neuropathy.

If you develop peripheral vascular disease your doctor will also recommend treatment to slow disease progression and improve blood flow.

Regular exercise, eating a healthy, balanced diet, and losing weight can help improve blood flow, as does quitting smoking. Smoking restricts blood vessels.

Treatment might also involve medication to reduce blood clotting, lower your cholesterol, or reduce your blood pressure depending on the underlying cause of a blockage.

Good diabetes managementmedication, regular exercise, and a healthy dietcan also reduce symptoms of peripheral vascular disease.

In severe cases, you may need angioplasty for peripheral vascular disease. This is a surgical procedure to open up a blocked artery and restore blood flow.

Gangrene treatment involves antibiotics to kill bacteria and stop an infection, as well as surgery to remove damaged tissue. Treatment for Charcot foot involves preventing further deformity.

Wearing a cast to immobilize the foot and ankle can gradually strengthen these bones, as does wearing custom shoes or a brace. In severe cases, surgery can help correct a deformity.

One way to prevent foot issues with diabetes is to keep your blood sugar within a healthy range, so check your blood sugar on a regular basis. Also, take your diabetes medication as instructed. If youre unable to control your blood sugar, see your doctor.

Other tips to prevent foot issues include:

Not only should you take steps to keep your blood sugar within a healthy range, you should also take steps to keep your feet healthy. Heres how to protect your feet with diabetes:

Some diabetes foot complications are life-threatening, or they put you at risk for amputation. See a doctor if you have any concerns or notice unusual changes with your feet.

A seemingly minor issue like cracked skin on your feet, yellow toenails, athletes foot, or an ingrown nail can become a serious problem if left untreated. Also, see your doctor for any cuts or scrapes that dont heal to avoid an infection on your feet.

Although theres no cure for diabetes, a healthy diet, regular exercise, and taking your medication as instructed can lower your risk for complications.

Its very important to keep your feet healthy when you have diabetes. Check your feet daily for signs of injury or infection, and see your doctor right away if you notice any unusual symptoms.

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Diabetic Feet: Issues, Treatment, and Prevention - Healthline

United Nations Secretary-General, Antnio Guterres, and World Health Organization (WHO) Director-General, Dr Tedros Adhanom Ghebreyesus, have announced that a Global Diabetes Compact will be launched on 14 April 2021 to mark the 100-year anniversary of the discovery of insulin for the treatment of diabetes. The centenary offers a window of opportunity for the global diabetes community to come together to reflect on addressing barriers to accessing insulin and associated health technology products. It is an opportune time to forge a common vision among all stakeholders to develop a multisectoral plan of action to address these barriers and ensure that no person living with diabetes goes untreated.

The WHO Department of Noncommunicable Diseases (NCD), in collaboration with the Division of Medicines and Health Products, is convening a series of biannual dialogues with the private sector to define meaningful and effective contributions to the implementation of national responses for the prevention, management and control of NCDs and the attainment of related Sustainable Development Goal (SDG) targets. The dialogues will focus on mobilizing commitments and contributions by the private sector toward national NCD responses to achieve SDG targets 3.4, 3.8 and 3b by improving access to and affordability of safe, effective and quality-assured medicines and health technology products.

Improving access to medicines and health technology products for the diagnosis, management and treatment of diabetes is multi-faceted and part of a broader challenge of ensuring access to health care. It requires a robust health system which includes good leadership and governance, adequate financing, access to information and evidence, quality service delivery, a strong health workforce, and equitable access to essential medicines and health technology products of assured quality, safety, efficacy and cost-effectiveness. Effective interventions will require enhanced collaboration and commitment for greater impact at country-level. The first dialogue in the series, which is being held on 23-24 February 2021, will focus on improving access to human insulin and associated health technology products for diabetes as part of the Global Diabetes Compact. Subsequent dialogues will focus on other NCDs, such as cardiovascular disease, cancer, lung diseases, oral health, rehabilitation, sensory impairments and disability.

This first dialogue aims to encourage inputs, commitments, and contributions from the pharmaceutical and health technology product industries to support WHOs activities to improve access to medicines and health technology products for diabetes, including for the 14 April 2021 launch of the Global Diabetes Compact.

A summary report of this meeting will be available on this website after the meeting.

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Dialogue with the private sector on medicines and technologies for diabetes care, February 2021 - World Health Organization