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Category Archives: Gene therapy
Tweaking synonymous sites for gene therapy and vaccines – Drug Target Review
Posted: December 1, 2020 at 12:57 pm
Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.
With 61 codons specifying 20 amino acids, some can be encoded by more than one codon and it is often presumed that it does not matter which one a gene uses. When I first studied genetics, some books I read taught that mutations between such alternative codons (eg, GGA->GGC, both giving glycine) were called synonymous mutations, while others referred to them as silent mutations. However, are synonymous mutations really silent meaning they are identical in terms of fitness and function? Although they may specify the same amino acid, does that mean they are all the same?
Figure 1: Intronless GFP transgene expression is higher for variants of GFP with higher GC content at synonymous sites5
Perhaps one of the biggest surprises over recent years has been the discovery that versions of the same gene, differing only at synonymous sites, can not only have different properties, but effects that are not modest.1-5 For example, two versions of green fluorescent protein (GFP) differing only at synonymous sites can have orders of magnitude differences in their expression level.4 We similarly recently discovered that for an intronless transgene to express in human cell lines it needs to be GC rich, which can be achieved by altering the synonymous sites,5 as seen in Figure 1. It is no accident, we suggest, that the well-expressed endogenous intronless genes in humans (such as histones) are all GC rich and that our functional retrogenes tend to be richer in GC content than their parental genes.
The realisation that synonymous sites matter has clear relevance to the design of transgenes or other artificial genes, be these for experiments, gene therapy, protein production (eg, in bacteria) or for vaccine design. In the case of vaccines, we might wish to modulate a viral protein to be effectively expressed in human cells to illicit a strong and robust immune response.6 Conversely to the design of attenuated vaccines, we seek to produce a tuned down version of the virus that can function but is weak.7
The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered. One approach is mass randomisation try many alternatives and see what works.4,8,9 In principle this is fine, but this approach requires many randomisations, which is still technically difficult for long attenuated viruses. An alternative strategy that we have been exploring is to let nature tell us; we can apply tools and ideas from population genetics to better understand what natural selection favours and disfavours and in turn to estimate the strength of selection.
it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus
Estimation of the strength of selection is possible from knowledge of the site frequency spectrum, (ie, how common variants are) from which we can infer the distribution of fitness effects (DFE). If a site is under strong purifying selection, then mutations may occur in the population but these are rapidly eliminated, so variants are always rare. By contrast, if they are selectively neutral, we expect some variants to be quite common. We recently applied this methodology to show that synonymous mutations in human genes that disrupt exonic splice enhancer motifs are often under strong selection and affect many synonymous sites in our genes.10 This has implications for both diagnostics and for transgene design for gene therapy, as we often remove introns in heterologous genes, so freeing up these residues from their role in specifying exons ceases.11
The same DFE methodology cannot easily be applied to viruses, as the methods assume free recombination (ie, we assume one mutation does not impact the fate of others in the same genome). However, other population genetical tools can still be applied. Recently, we examined SARS-CoV-2 and identified the profile of mutations that we see at four-fold degenerate sites.12 From this profile we could estimate what the synonymous site composition would be, assuming that the only forces are mutational biases and neutral evolution (ie, no selection). We observed that in this genome there is a strikingly strong C->U mutation bias and a G->U one. In the raw data this is not so obvious as G and C are quite rare. However, the mutability of the sites per occurrence of the site reveals the underlying patterns.
Figure 2: The rate of mutational flux from one dinucleotide to another in the coding sequence of SARS-CoV-2. The direction of flux is indicated by the indentation of the connecting links: the inner layer represents flux out while the outermost layer represents flux into the node. The frequency of the flux exchange is represented by the width of any given link where it meets the outer axis. Dinucleotide nodes are coloured according to their GC-content. Hence, it is evident that there is high flux away from GC-rich dinucleotides whereas AU-rich dinucleotides are largely conserved.12
With knowledge of the mutational bias we then asked what the equilibrium frequency of the four nucleotides would be using four simultaneous equations. This is the nucleotide content at which for every mutation changing a particular base there is an equal and opposite one creating the same base somewhere else in the genome, ensuring overall unchanged nucleotide content. Given the strong C->U and G->U mutational biases, it is no surprise that the equilibrium content is very U rich (we estimate equilibrium U content should be about 65 percent). However, while the four-fold sites are indeed U rich, they are not that U rich, being closer to 50 percent. A clue as to why the mutation bias is so skewed to generating U comes from analysis of equilibrium UU content: UU residues are predicted to be very common, with CU residues being particularly mutable generating UU (Figure 2) this is expected due to human APOBEC proteins attacking and mutating/editing the virus.13
One probable explanation for this difference between predicted and observed nucleotide content is selection against U content. There may be many U residues appearing in the population, but many are pushed out of the population owing to purification selection, ie, because of the deleterious effects of the mutations. That such selection is happening in the SARS-CoV-2 genome is also clear from the sequence data. We estimate that for every 10 mutations that appear in the sequence databases, another six are lost because of selection prior to genome sequencing. Indeed, UU content is about a quarter of that predicted (Figure 3).
Figure 3: The predicted (under neutral mutational equilibrium) and observed dinucleotide content of SARS-CoV-2. Note the very high predicted levels of UU given the strong mutational flux to UU residues (see Figure 2) and the net underrepresentation in actual sequence.9
This leaves two problems: why is selection operating on SARS-CoV-2 and what can we do with this information? In some cases, we have a good idea as to why: many mutations to U at codon sites generate stop codons. However, we have observed that U destabilises the transcripts and is associated with lower-reported transcript levels;12 a full explanation of the causes of selection on nucleotide content therefore requires manipulation of the sequences.
The second question, what to do with this information, is perhaps more urgent. It has previously been noted that nucleotide content manipulation is a viable means to attenuate viruses.7 Currently there are three groups investigating this route to make a vaccine for SARS-CoV-2: Indian Immunologicals Ltd/Griffith University, Codagenix/Serum Institute of India and Acbadem Labmed Health Services/Mehmet Ali Aydinlar University. In prior attempts, attention has been paid to CpG levels and UpA levels (which we find to be correlated between SARS genes and between different viruses).12 CpGs attract the attention of zinc antiviral protein (ZAP) and UpA attracts an RNAase L. Not surprisingly, some viruses, including SARS-CoV-2, therefore have low levels of both dinucleotide pairs given the levels of the underlying nucleotides.
The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered
In the past, attenuation strategies have focused on modulating synonymous sites to increase CpG and UpA, making the virus more visible to antiviral proteins.14 We in turn suggest a general strategy to utilise this method and to increase U content as well.12 Given the evidence that selection on the virus is to reduce U content, while our antiviral proteins are mutating it to increase U content, it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus. This is an unusual circumstance in which we predict that we should build in more of the already most common synonymous site nucleotides (U in this case) to degrade the virus. More generally, it is assumed that the most used codons are those that tend to increase the fitness of the organism. In the face of such a severe mutation bias, however, this simpler logic no longer holds.
Laurence D Hurst is Professor of Evolutionary Genetics and Director of the Milner Centre for Evolution at the University of Bath. He is currently also the President of the Genetics Society. He completed his D.Phil in Oxford, after which he won a research fellowship and then moved to Cambridge University as a Royal Society Research Fellow. While on the fellowship he assumed his current Chair at Bath University. In 2015 he was elected a Fellow of the Academy of Medical Sciences and a Fellow of the Royal Society. He is a recipient of the Genetics Society Medal and the Scientific Medal of the Zoological Society of London.
Related topicsDisease research, DNA, Gene Therapy, Genetic analysis, Genomics, Protein, Proteogenomics, Proteomics, Research & Development, RNAs, Vaccine
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Tweaking synonymous sites for gene therapy and vaccines - Drug Target Review
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Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan – PRNewswire
Posted: December 1, 2020 at 12:57 pm
"The need for reproducible, scalable, and economical production of cell and gene therapies is creating a demand for digital bioprocessing technologies," said Nitin Naik, Global Life Sciences Vice President at Frost & Sullivan. "These technologies are critical to realize the true commercial potential of cell and gene therapies in the next two to three years and serve as a conduit to improve market access and control the total cost of therapy."
Naik added: "From a market segment perspective, while the stem cell market is lucrative, the highest growth is expected to be in gene-modified cell therapies, with a pipeline of 269 products,* followed by gene therapies, which account for 182 assets in the pipeline.* Further, although allogeneic stem cell therapies dominate the marketed product catalogs, interest in disease-modifying CAR-T therapies, which are largely autologous, is driving demand for the evolution of manufacturing technologies, models, and capacity expansion investment by CDMOs." (*as of August 2020)
To tap into the growth prospects exposed by the CGT market, companies must focus on:
Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026 is the latest addition to Frost & Sullivan's Healthcare research and analyses available through the Frost & Sullivan Leadership Council, which helps organizations identify a continuous flow of growth opportunities to succeed in an unpredictable future.
About Frost & Sullivan
For six decades, Frost & Sullivan has been world-renowned for its role in helping investors, corporate leaders and governments navigate economic changes and identify disruptive technologies, Mega Trends, new business models and companies to action, resulting in a continuous flow of growth opportunities to drive future success.Contact us: Start the discussion.
Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026
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Contact:Mariana Fernandez Corporate Communications P: +1 210 348 10 12 E: [emailprotected] http://ww2.frost.com
SOURCE Frost & Sullivan
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Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan - PRNewswire
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BioLife Solutions & Casdin Capital Innovation Accelerator Announce New Investments in Cell & Gene Therapy Bioproduction Tools – goskagit.com
Posted: December 1, 2020 at 12:57 pm
BOTHELL, Wash., Dec. 1, 2020 /PRNewswire/ --BioLife Solutions, Inc. (NASDAQ: BLFS)("BioLife" or the "Company"), a leading developer and supplier of a portfolio of class-defining bioproduction products and services for cell and gene therapies, today announced two new co-investments with innovation accelerator partner Casdin Capital. The first is a re-investment in iVexSol, a vector manufacturing company founded on a proprietary, next-generation, stable lentiviral vector production process. BioLife Solutions invested $1 million and Casdin invested $4 million in a $15.2 million Series A financing round which was led by a third undisclosed strategic investor. BioLife and Casdin also each converted their respective previous $1.1 million debt into equity in this round.
In a new joint investment, BioLife and Casdin also each agreed to invest $1 million in privately held PanTHERA CryoSolutions,a Canadian startup company that is developing next generation cryopreservation solutions incorporating ice recrystallization inhibitor (IRI) intellectual property. Subject to closing conditions, BioLife will execute a development and license agreement with PanTHERA, under which, BioLife will make milestone development payments up to $2 million over the next 24 months in exchange for exclusive, perpetual, worldwide marketing and distribution rights to the technology for use in cell and gene therapy applications.
Mike Rice, BioLife's CEO, remarked, "Drs. Rod Rietze and Michael Greene and the iVexSol team continue to make progress scaling iVexSol's proprietary manufacturing process. iVexSol has the real potential to disrupt the current viral vector manufacturing process which today results in lower yield and high cost, helping to accelerate the development of novel, life-saving cell and gene therapies. We also secured access to PanTHERA's novel and potentially disruptive IRI technology, which may form the basis of a next generation freeze media product line. We've known Drs. Jason Acker and Robert Ben of PanTHERA for many years and respect the rigor of their work."
"Crossing the early stage chasm with next generation technology is particularly difficult in this field." Eli Casdin, Managing Director at Casdin Capital, commented, "It's exciting to see the innovation accelerator build momentum, connecting entrepreneurs with investment capital, operational experience and commercial distribution. We look forward to making more investments in the months and years ahead."
Aby J. Mathew, PhD, Executive Vice President and Chief Scientific Officer at BioLife and Shaun Rodriguez, Director of Life Science Research at Casdin will join the PanTHERA board of directors at closing. Casdin also has a board observer seat at iVexSol.
About iVexSol
iVexSol is a startup vector manufacturing company founded on a proprietary, next-generation, stable lentiviral vector production process that transforms the way these essential gene-delivery vehicles are made. Its technology will greatly reduce the complexity, cost and development time of these critical reagents, thereby accelerating the development and enabling greater access to life-changing cell and gene therapies. For more information visitwww.ivexsol.com.
About PanTHERA CryoSolutions
PanTHERA CryoSolutions is a Canadian corporation that designs and manufactures patented ice recrystallization inhibitors for use in the cryopreservation of cells, tissues and organs. Launched out of a scientific collaboration between Dr. Robert Ben (University of Ottawa) and Dr. Jason Acker (University of Alberta), PanTHERA aims to enhance the cryopreservation process to improve both research tools and clinical therapy products. For more information visit http://www.pantheracryo.com.
About Casdin Capital
Casdin Capital, LLC is an investment firm focused on disruptive businesses. The firm is positioned to capitalize off an underappreciated, disruptive technology shift now unfolding in the life sciences and healthcare industry. Investment opportunities stretch the entire healthcare continuum and into sectors such as agriculture, industrial manufacturing and traditional information technology. For more information please visit http://www.casdincapital.com.
About BioLife Solutions
BioLife Solutions is a leading supplier of class-defining cell and gene therapy bioproduction tools and services. Our tools portfolio includes our proprietaryCryoStorfreeze media and HypoThermosolshipping and storage media, ThawSTARfamily of automated, water-free thawing products, evocold chain management system,Custom Biogenic Systemshigh capacity storage freezers and SciSafe biologic storage services. For more information, please visit http://www.biolifesolutions.com, and follow BioLife on Twitter.
Cautions Regarding Forward Looking Statements
Except for historical information contained herein, this press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, statements about the Company's expectations regarding the success of iVexSol or Panthera products or processes. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements, including risks and uncertainties related to market conditions, and those other factors described in our risk factors set forth in our filings with the Securities and Exchange Commission from time to time, including our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We undertake no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.
Media & Investor RelationsRoderick de GreefChief Financial Officer & Chief Operating Officer(425) 686-6002rdegreef@biolifesolutions.com
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BioLife Solutions & Casdin Capital Innovation Accelerator Announce New Investments in Cell & Gene Therapy Bioproduction Tools - goskagit.com
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2020 Report: Growth Opportunities in Gene Therapy, Automated Bioanalytics, and Biomarker Platforms – ResearchAndMarkets.com – Business Wire
Posted: December 1, 2020 at 12:57 pm
DUBLIN--(BUSINESS WIRE)--The "Growth Opportunities in Gene Therapy, Automated Bioanalytics, and Biomarker Platforms" report has been added to ResearchAndMarkets.com's offering.
The research provides technological insights across inflammation, infectious diseases, and microbiomics.
The Life Science, Health & Wellness TOE will feature disruptive technology advances in the global life sciences industry. The technologies and innovations profiled will encompass developments across genetic engineering, drug discovery and development, biomarkers, tissue engineering, synthetic biology, microbiome, disease management, as well as health and wellness among several other platforms.
The Health & Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare.
Innovations in Life Sciences, Health & Wellness from:
For more information about this report visit https://www.researchandmarkets.com/r/tkufmb
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2020 Report: Growth Opportunities in Gene Therapy, Automated Bioanalytics, and Biomarker Platforms - ResearchAndMarkets.com - Business Wire
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BioStock: Status report CombiGene – In the front line of gene therapy – marketscreener.com
Posted: December 1, 2020 at 12:57 pm
In recent years, gene therapies have emerged as one of the hottest areas within life science and the temperature is rising as the first treatments are now available to patients in the US and Europe. During the 2020s, several new therapies are expected to be marketed, a breakthrough period for these therapies that through one or a few treatments have the potential not only to relieve but to cure diseases. The attention focused on gene therapies has lately resulted in several acquisitions and licensing deals. On the Swedish stock market, there is only one listed gene therapy company, CombiGene. BioStock has now published a status report of the company, which can be downloaded below.
Read the full CombiGene status report at biostock.se:
https://www.biostock.se/en/status-report-combigene-in-the-front-line-of-gene-therapy/
This is a pressrelease from BioStock - Connecting Innovation & Capital.https://www.biostock.se/
(c) 2020 Cision. All rights reserved., source Press Releases - English
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Irish scientists develop gene therapy for inherited vision loss disorder – The Irish Times
Posted: December 1, 2020 at 12:57 pm
A team of Irish scientists have developed a gene therapy that could potentially lead to a new treatment for an eye disease causing progressive loss of vision, which affects many thousands of people across the globe.
The breakthrough therapy, which offers hope for people with dominant optic atrophy (DOA), was devised by researchers at Trinity College Dublin in collaboration with clinicians at the Royal Victoria Eye and Ear Hospital and the Mater hospital in Dublin.
It also has implications for a much wider suite of neurological disorders associated with ageing which collectively affect millions of people worldwide.
DOA is an inherited disorder characterised by degeneration of optic nerves. It typically starts during the first decade of life. Affected people usually develop moderate visual loss and colour vision defects but severity varies, symptoms can worsen over time and some people become blind. At present, there is no way to prevent or cure DOA.
The scientists published their results in Frontiers in Neuroscience on Thursday.
A gene (OPA1) provides instructions for making a protein found in cells and tissues throughout the body that is pivotal to the proper function of mitochondria, the energy generators in cells.
Without the protein, mitochondrial function is sub-optimal; the normally well-interconnected mitochondrial network in healthy cells becomes highly disrupted. For those with DOA, it is mutations in OPA1 and the dysfunctional mitochondria that are responsible for onset and progression of the disorder.
Led by research fellow Dr Daniel Maloney and Prof Jane Farrar from TCD School of Genetics and Microbiology, the gene therapy successfully protected the visual function of mice who were treated with a chemical targeting the mitochondria and were consequently living with dysfunctional mitochondria.
The scientists also found the gene therapy improved mitochondrial performance in human cells that contained mutations in the OPA1 gene.
We used a clever lab technique that allows scientists to provide a specific gene to cells that need it using specially engineered non-harmful viruses, Dr Maloney said.
This allowed us to directly alter the functioning of the mitochondria in the cells we treated, boosting their ability to produce energy which, in turn, helps protect them from cell damage.
Excitingly, our results demonstrate that this OPA1-based gene therapy can potentially provide benefit for diseases like DOA, which are due to OPA1 mutations, and also possibly for a wider array of diseases involving mitochondrial dysfunction.
Mitochondrial dysfunction causes problems in neurological disorders such as Alzheimers and Parkinsons disease. The impacts gradually build up over time, which is why many may associate such disorders with ageing.
Prof Farrar added: We are very excited by the prospect of this new gene therapy strategy, although it is important to highlight that there is still a long journey to complete from a research and development perspective before this therapeutic approach may one day be available as a treatment.
Because mitochondrial dysfunction was implicated in so many neurological disorders, there was great potential for this type of therapeutic strategy to make a major societal impact, she said.
The research was supported by Science Foundation Ireland, the Health Research Board of Ireland, Fighting Blindness Ireland, and Health Research Charities Ireland.
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New gene therapy could help treat bowel cancer that reaches the liver – 9News
Posted: December 1, 2020 at 12:57 pm
Gene therapy has shown promise for the first time to help treatbowelcancerthat has spread to the liver.
Adelaide researchers showed the novel approach, which uses a modified virus to infect the liver, was able to shrink tumours in mice.
"We're very excited by these results," Dr Susan Woods, one of the investigators in the study that was published in the Gastroenterology journal, said.
Further testing will be carried out to see whether the therapy could work for othercancers that travel to the liver such as tumours of the breast, lung and pancreas.
The modified virus exclusively targets the liver and introduces a copy of a gene that instructs the organ to make more cells called fibroblasts which are known to be good and slowcancergrowth.
The team from SAHMRI and the University of Adelaide have been investigating why normal cells that surround thecancerare good while others are corrupted and promote tumour growth.
"Inbowelcancer, we know that patients with the poorest prognosis have a lot of these corrupted or bad tumour supporting fibroblasts," Dr Susan Woods said.
This type of gene therapy that uses a modified virus to enter the liver is currently being used on patients with blood disorders.
"This is the first sign that we could use this to treatcancerthat has spread to the liver," Dr Woods said.
Bowelcancersurvivor Hannah Devereux is heartened by the research and said there needs to be more treatment options for people who are diagnosed with the disease late, when it has already spread.
Hannah was only 34 when she was diagnosed withbowelcancer, soon after her second child was born.
"Had the baby, he was 10 days old, and they found two tumours. My world came crashing down," she said.
Hannah had complained about digestive symptoms during the pregnancy.
"The doctor just thought it was pregnancy related," she said.
Hannah required intensive treatment for a year including six months of chemotherapy, radiotherapy and two major surgeries.
She has now reached the five-yearcancer-free milestone and is the ambassador of the Jodi Lee Foundation to preventbowelcancer.
Bowelcanceraffects more than 15,000 Australians each year and fewer than 50 per cent of cases are detected early.
More than 100 Australians each week die frombowelcancer.
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New gene therapy could help treat bowel cancer that reaches the liver - 9News
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Navigating the challenge of covering breakthrough therapies at MedCity INVEST Precision Medicine – MedCity News
Posted: December 1, 2020 at 12:57 pm
Our virtualMedCity INVEST Precision Medicine conference, December 9-11, will highlight aspects of precision medicine from the biopharma companies developing cell and gene therapies to diagnostics and the data sharing initiatives to support precision and personalized medicine.
One vexing part of any conversation on precision medicine and breakthrough therapies that have been developed and are continuing to work their way through clinical trials for cancer to rare disease is how to address the practical challenge of their high price tags. Although there are several models to cover the cost of these therapies such as Cignas Embarc Benefit Protection program,what happens when someone changes insurers when they change their job? What is fair for patients and whats fair for companies? When do these conversations even start?
The panel, Reimbursement Models for Cell and Gene Therapies, will highlight some of the pros and cons of different models that are coming to market. Panelists include Laura Okpala, Director, Reimbursement Policy, Gilead Sciences, and Mark Trusheim, Strategic Director, NEWDIGS initiative at the MIT Center for Biomedical Innovation.
Heres a preview of some of the sessions. Click here to see the agenda.
Interoperability Progress Report
How far have we progressed with sharing patient medical records? Patient data is key in unlocking riddles of medical science but interoperability is necessary to facilitate this. What companies are making an impact on a regional and national scale? What milestones are on the horizon? What obstacles continue to vex further advancement? How can we make medical records more accessible to patients?
Moderator: Elise Reuter, Senior Reporter, MedCity NewsSpeakers:Kevin Chaney, Senior Program Manager, Office of the National Coordinator for Health ITIda Sim, M.D., Ph.D., Professor of Medicine, University of California, San FranciscoNiko Skievaski, Co-Founder and President, Redox
What It Takes To Build A Successful, Regional BioInnovation HubPhiladelphia is one of many cities seeking to support the continued growth of cell and gene therapy and connected health industries. What do cities need to do to address education, training and other needs to support these sectors? This session will be held as part ofVenture Cafe Philadelphia.
(sponsored by IBX)
Lisa Dalton, Chief People Officer,Spark TherapeuticsAudrey Greenberg, Executive Managing Director,The Discovery LabsTiffany Wilson, President & CEO,University City Science Center
Moderator:Michelle Histand, Director of Innovation, Independence Blue Cross
Register nowand be part of the conversation at INVEST Precision Medicine.
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A new gene therapy against eye diseases – Phone Mantra
Posted: December 1, 2020 at 12:57 pm
Mitochondria degeneration
Characterized by degeneration of the optic nerves, the dominant optic atrophy usually begins to cause symptoms in patients in early adulthood: moderate vision loss and some color vision defects, although their severity varies.Symptoms can also worsen over time and lead to total blindness in some patients.There is currently no way to prevent or cure AOD.
Its occurrence is due to mutations in the OPA1 gene, essential for the proper functioning of the mitochondria, which are the energy producers in cells.Without the protein made by OPA1, mitochondrial function is suboptimal and the mitochondrial network, which is well interconnected in healthy cells, is severely disrupted, which can lead to the onset and then the progression of optic atrophy. dominant.
This new gene therapy has been successfully tested in mice treated with a chemical targeting the mitochondria and therefore living with dysfunctional mitochondria.It also improved the performance of mitochondria in human cells that contained mutations in the OPA1 gene, which gives hope that it could be effective in humans.
The scientists also found that their gene therapy improved the performance of mitochondria in human cells that contained mutations in the OPA1 gene, raising hopes that it might be effective in humans.
Our results fascinatingly demonstrate that this OPA1-based gene therapy has the potential to provide benefits for diseases like ODA, which are due to OPA1 mutations, and possibly for a wider range as well. of diseases involving mitochondrial dysfunction,says Dr. Daniel Maloney, lead author of the study.This includesother neurodegenerative diseases like Parkinsons and Alzheimers, which are linked to aging and the progressive dysfunction of mitochondria.
We are very enthusiastic about the idea of this new gene therapy strategy, explains Professor Jane Farrar, co-author of the work.Even if, she adds,there is still a long way to go from the point of view of research and development before this therapeutic approach can one day be available as a treatment.
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Avrobio tracks improvements in first patient treated with Gaucher gene therapy – FierceBiotech
Posted: November 19, 2020 at 10:58 am
Avrobio has shared data on the first Gaucher disease patient to receive its gene therapy AVR-RD-02. The patient, who was stable on enzyme replacement therapy at baseline, experienced a 22% drop in a toxic metabolite after receiving AVR-RD-02 and stopping taking the standard of care.
Gaucher, like the Fabry disease targeted by Avrobios lead prospect, is currently treated using enzyme replacement therapies sold by Sanofi and Takeda, which entered the market through its takeover of Shire. However, a significant minority of patients experience physical limitations despite treatment. Negative outcomes include bone pain and spleen enlargement. Johnson & Johnsons Zavesca offers an oral alternative, but there remain unmet medical needs.
Avrobio is developing AVR-RD-02 to address those needs. The data shared as part of Avrobios R&D day mark the start of the effort to show AVR-RD-02 performs as hoped in the clinic.
The first patient to receive AVR-RD-02 discontinued enzyme replacement therapy one month before taking the gene therapy. Three months after receiving the gene therapy, levels of Gaucher biomarker lyso-Gb1 had fallen 22%. The patients level of plasma chitotriosidase, a biomarker of cells associated with severe organ damage, was down 17%. Hemoglobin and platelets were in the normal range.
AVR-RD-02 triggered those changes without causing serious adverse events. The data drop offers an early indication that Avrobio may be able to improve outcomes by harvesting hematopoietic stem cells, adding a gene that encodes for glucocerebrosidase and reinfusing the cells back into the same patient. With enzyme replacement therapies costing healthcare systems up to $400,000 a year per patient, there is scope for AVR-RD-02 to cut the cost of treating Gaucher disease.
Avrobio shared the early look at clinical data on AVR-RD-02 alongside updates about other assets. There is now more than three years of data on some Fabry patients treated with Avrobios lead asset, putting the company in a position to plot a path to accelerated approval. Avrobio plans to submit its briefing book to the FDA by the end of the year to align on an accelerated approval strategy.
The update also covered cystinosis candidate AVR-RD-04. The first patient to receive the candidate is off oral and eye drop cysteamine 12 months after receiving the gene therapy. The number of crystals in the patients skin are down 56%, leading Avrobio to posit they may have gained the ability to make their own functional cystinosin protein.
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Avrobio tracks improvements in first patient treated with Gaucher gene therapy - FierceBiotech
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