Novozymes delivered flat organic sales growth in the 2020 financial year and an EBIT margin of 26.1%. In 2021 the company expects 2-6% organic growth supported by innovation, stronger commercial presence, and gradual industry recovery. EBIT margin is expected at a solid 25-26%.
LONDON and RALEIGH, N.C., Feb. 02, 2021 (GLOBE NEWSWIRE) -- In a release issued under the same headline earlier today by Verona Pharma plc (Nasdaq: VRNA), please note that all instances of the unit "µg" were mistakenly represented as "mg." The corrected release follows:
Go here to see the original:
CORRECTING and REPLACING -- Verona Pharma Reports Positive Phase 2 Results with pMDI Formulation of Ensifentrine in COPD
NEW YORK, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Immunovant (Nasdaq: IMVT), a clinical-stage biopharmaceutical company focused on enabling normal lives for patients with autoimmune diseases, today announced a voluntary pause of dosing in its ongoing clinical trials for IMVT-1401.
Link:
Immunovant Announces Voluntary Pause in Clinical Dosing of IMVT-1401
If you asked Freda Miller 10 years ago if stem cells could be harnessed to repair brain injuries and disease, she would have said it was too early to tell.
Today, she describes the progress that she and other regenerative medicine experts have madein understanding what regulates populations of stem cells cells with the potential to turn into many different cell typesand the rapid advances those discoveries have driven.
The approaches were using allow us to find so much information on things we could only dream of before.
Miller, who is also a professor at the University of British Columbia, is leading a Medicine by Design-funded team with expertise in computational biology, neurobiology, bioengineering and stem cell biology that is investigating multiple strategies to recruit stem cells to promote self-repair in the brain and in muscle. If it succeeds, the research could improve treatments for diseases such as multiple sclerosis (MS) and cerebral palsy, as well as brain injury.
Millers team is one of 11 at U of T and its partner hospitals that are sharing nearly $21 million in funding from Medicine by Design over three years. Funded by a $114-million grant from the Canada First Research Excellence Fund, Medicine by Design is a strategic research initiative that is working at the convergence of engineering, medicine and science to catalyze transformative discoveries in regenerative medicine and accelerate them toward clinical impact.
This is the second round of large-scale, collaborative team projects that Medicine by Design has funded. The support builds on the progressmade in the first round of projects (2016-2019) and is spurring further innovation to push regenerative medicine forward. It alsoled to a 2017 publicationby many of the same researchers on Millers current project in Cell Reports that essentially provided a roadmap for how brain stem cells build the brain developmentally, and then persist to function in the adult brain.
Miller, a neuroscientist, has always been fascinated by the brain and neurons, the network of billions of nerve cells in the brain. Around 15 years ago, when she started to take an interest in the potential regenerative capabilities of stem cells, she began to wonder if she could use stem cells to treat brain injury or disease. Though too little was known about stem cells at the time, she knew that it was a question worth investigating. But she also realized that making and integrating new nerve cells, which are the working parts of brain circuits, would be a daunting task.
Even if you can convince the stem cells to make more neurons, those neurons then have to survive and they have to integrate into this really complex circuitry, says Miller. It just made sense to me that if were really going to test this idea of self-repair in the brain, we should go after something thats more achievable biologically.
So, Miller turned her attention to a substance called myelin, which covers nerves and allows nerve impulses to travel easily. In many nervous system diseases MS is a well-known example and brain injuries, damage to and loss of myelin is a main factor in debilitating symptoms. Thanks in part to the team project award from Medicine by Design, Miller leads a team that has a focus on recruiting stem cells to promote the generation of myelin.
Miller says repairing myelin, also called remyelination, will eventually help to better understand the effects of the target disease or injury, possibly even leading scientists to discover how to reverse it. Boosting myelin is a promising area of research, she adds, because its not an all-or-nothing situation.
Even a little bit of remyelination could have a big impact. You dont have to win the whole lottery; you dont have to have 100 per cent remyelination to have a measurable outcome.
The teams work is not limited to generating myelin to treat nervous system diseases or brain injury. They are also looking at how they could recruit stem cells to generate more muscle. They are specifically looking at muscular dystrophy, but Miller says the applications from that work can be used in other diseases or situations where damage to muscles has occurred, such as age-related disorders.
Millers team includes experts from diverse fields: Gary Bader, a professor at the Donnelly Centre for Cellular and Biomolecular Research and a computational biologist; bioengineers Alison McGuigan, a professor in the department of chemical engineering and applied chemistry in the Faculty of Applied Science & Engineering, and Penney Gilbert, an associate professor at the Institute of Biomedical Engineering; Sid Goyal, a professor at the department of physics in the Faculty of Arts & Science; ProfessorDavid Kaplan and Assistant ProfessorYun Li, both in the Temerty Faculty of Medicine and a senior scientist and a scientist, respectively, at SickKids; stem cell biologist Cindi Morshead, a professor and chair of the division of anatomy in the department of surgery in the Temerty Faculty of Medicine; and Peter Zandstra, a University Professor in the Faculty of Applied Science & Engineering and director of Michael Smith Laboratories at the University of British Columbia.
Miller says Medicine by Designs contribution in bringing teams like hers together is immeasurable.
There are tangible results you can measure like publications and other grants and clinical trials, Miller says. But there are a lot of intangible things Medicine by Design brings to the table like developing a culture of people from very diverse places and allowing them to do science together at a time when the biggest breakthroughs are going to be made by combining technological and biological approaches. Its hard to do that if youre on your own.
This large, interdisciplinary team effort combines data and computer modelling to look at individual stem cells in the brain and predict their behaviours. Through experimentation, they can then test if the cells behave the way they predicted, which Miller says they have had great success with. From there, the team casts a wide net, testing various ways to try to control cells behaviour with the end goal of convincing the stem cells to turn into cells that aid in healing and repair.
One approach they use is testing already approved pharmaceuticals to see if they have the desired effect on the stem cells behaviour. This approach has had success. In summer 2020, Morshead, Miller and their collaborators, led by Donald Mabbott, a SickKids senior scientist and professor in the department of psychology in the Faculty of Arts & Science, published a paper in Nature Medicine that showed that metformin, a common diabetes drug, has the potential to reverse brain injury in children who had had cranial radiation as a curative therapy for brain tumours.
Miller says that, to her knowledge, this is the first paper that demonstrates that this type of brain repair is possible in humans.
Continue reading here:
Medicine by Design researchers focus on promoting self-repair of the brain - News@UofT
The market has been high on Brainstorm Cell Therapeutics Inc (BCLI) stock recently. BCLI gets a Bullish score from InvestorsObserver's Stock Sentiment Indicator.
Sentiment uses short term technical analysis to gauge whether a stock is desired by investors. As a technical indicator, it focuses on recent trends as opposed to the long term health of the underlying company. Updates for the company such as a earnings release can move the stock away from current trends.
Price action is generally the best indicator of sentiment. For a stock to go up, investors must feel good about it. Similarly, a stock that is in a downtrend must be out of favor.
InvestorsObservers Sentiment Indicator considers price action and recent trends in volume. Increasing volumes often mean that a trend is strengthening, while decreasing volumes can signal that a reversal could come soon.
The options market is another place to get signals about sentiment. Since options allow investors to place bets on the price of a stock, we consider the ratio of calls and puts for stocks where options are available.
Brainstorm Cell Therapeutics Inc (BCLI) stock is up 7.94% while the S&P 500 is lower by -0.12% as of 12:02 PM on Tuesday, Jan 26. BCLI has risen $0.46 from the previous closing price of $5.79 on volume of 3,810,664 shares. Over the past year the S&P 500 is up 18.72% while BCLI has risen 49.88%. BCLI lost -$1.02 per share in the over the last 12 months.
To screen for more stocks like Brainstorm Cell Therapeutics Inc click here.
Brainstorm Cell Therapeutics Inc is a biotechnology company. The company is developing novel adult stem cell therapies for debilitating neurodegenerative disorders such as Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig's disease), Progressive Multiple Sclerosis (PMS), and Parkinson's disease (PD). Brainstorm's NurOwn, its proprietary process for the propagation of Mesenchymal Stem Cells (MSC) and differentiation into neurotrophic factor-(NTF) secreting cells (MSC-NTF), and their transplantation at, or near, the site of damage, offers the hope of more effectively treating neurodegenerative diseases.
Click Here to get the full Stock Score Report on Brainstorm Cell Therapeutics Inc (BCLI) Stock.
Subscribe to our daily morning update newsletter and never miss out on the need-to-know market news, movements, and more.
Read the original here:
Should You Buy Brainstorm Cell Therapeutics Inc (BCLI) Stock on Tuesday? - InvestorsObserver
The US Food and Drug Administrations Center for Biologics Evaluation and Research (FDAs CBER) has issued untitled letters to a firm marketing regenerative medicine products and to a company selling extracts for immunotherapy. On 25 January, CBER warned the owner of a California-based company called The Body Building, Inc. for marketing BioGenix regenerative medicine products to treat a variety of conditions including autoimmune disease, diabetes, Parkinsons disease, autism, and cardiovascular disease. Among the claims cited by CBER is an assertion from The Body Buildings Facebook page that the stem cells are highly anti inflammatory and immediately dissipate ANY PAIN [emphasis original] in the body in any area desired. They help modulate the functioning of your immune system which helps reverse the effects of auto immune disease. The products, which the firm claims contain primitive undifferentiated stem cells, do not carry a valid biologics license from FDA, notes the regulator, who referred the letters recipient to FDAs suite of four guidance documents setting the policy framework for human cellular or tissue-based products. CBER also noted that the website is advertising an exosome product, also subject to premarket review as a drug or biological product. The agency requests a response within 30 days. In other action, CBER warned Edge Pharma, LLC, based in Colchester, VT for marketing patient specific immunotherapy vials and custom mix allergy immunotherapy vials without a biologics license. Edge Pharma had not submitted an investigational new drug application for these products; neither had the firm applied for a biologics license. The warning letter also took issue with Edge Pharmas characterization of itself as a registered 503B outsourcing facility. Wrote CBER, Please be advised that biological products subject to licensure under section 351 of the PHS Act are not eligible for the exemptions for compounded drugs under sections 503A and 503B of the FD&C Act. Edge Pharma was referred to a January 2018 guidance on mixing, diluting or repackaging biologics outside the scope of an approved BLA, and asked to respond within 20 days. CDER warns Texas firm for multiple violations Separately, FDAs Center for Drug Evaluation and Research (CDER) warned a compounding center for multiple and significant violations, including receipt of drugs from active pharmaceutical ingredient (API) suppliers who themselves have been the subject of regulatory action. Houston-based Professional Compounding Centers of America (PCCA) garnered the letter after a week-long FDA inspection in October 2019. The heavily redacted warning letter begins by detailing the agencys concerns about API sourcing. We note that an inspection of records at your facility and a review of FDA import data demonstrated that FDA has, in the past, taken regulatory action against at least 23 of your other API suppliers for the manufacture of adulterated drugs under section 501 of the FD&C Act by either listing their drugs on import alert and/or issuing them warning letters, wrote FDA in the 27 January warning letter. A 23-point numbered list of the warning letters issued to individual API suppliers follows, though suppliers identity and other key details are redacted. Enough substance was left in the list, however, to ascertain that many of the listed firms had denied FDAs attempts to schedule inspections, and some evidently had issues found on inspection such as leaving undesirable material on the floors of the facilities, failure to investigate failed test results and failing to maintain complete data. The letter tells PPCA to provide lot numbers and dates of distribution for all of its products, and more: Considering that FDA has found a pattern of drug manufacturers with serious CGMP or other adulteration violations in your supply chain, in response to this letter, also provide a detailed plan to ensure you do not receive or deliver adulterated drugs in interstate commerce, in violation of section 301(c) of the FD&C Act, 21 U.S.C. 331(c). Other violations mentioned in the warning letter include serious problems with PCCAs glycerin testing program, which leaves open the potential for contamination with DEG. This known glycerin contaminant has caused fatalities in glycerin-containing medicines such as cough syrup, noted FDA in the letter. Finally, PCCA uses its name on the labels of some repackaged APIs without clearly indicating that these drugs were produced by other firms, a fact that became apparent during the October 2019 Inspection. FDA is calling for a regulatory meeting with PCCA to review the violations.
The rest is here:
Stem cell and allergy clinics receive CBER untitled letters - Regulatory Focus
Last year, Ricki Lake revealed her battle with hair loss on social media after posting an image of herself with a newly-shaved head. The actress and former talk show host had been struggling for more than 30 years with hair damage caused by heavy extensions, years of over styling and yo-yo dieting habits that would trigger yet more hair loss.She says it all started with her iconic role as Tracey Turnblad in the 1988 classic Hairspray.
Although the experience of going public with her condition was freeing for the star, she also worried that her hair wouldnt ever grow back. Lake says prior to shaving it off, she had gone through almost every treatment option, including Rogaine, supplements, spironolactone, steroid shots and platelet-rich plasma injections.
Now, a year later, she is sharing the latest part of her journey that includes a full head of healthy grey hair thanks to the Scandinavian brand Harklinikken, which specializes in hair loss. Here, the star talks about whats happened since she went public and the hair-care routine that has given her a new look and a new hope.
NewBeauty:Before going public with your hair loss journey, what did your day-to-day hair-care routine look like? Ricki Lake:I was wearing a hairpiece, like an extension with bangs in the front. I was living in London for a couple months for a job and the wear and tear of this piece being attached to my hair pulled it and caused it to get more and more frail. I had been on a diet and lost all this weight, so all my hair was shedding and it was just getting heavier on my head. I was supposed to take it off after one month, but I would leave it in for like four or five months because I didnt want to deal with the realityI was in denial.
When the piece came off it was bad. It was right around the holidays, so I bought a wig for $5,000 and only wore it for 15 minutes. I just couldnt wear it. After that, I confided in my boyfriend at the time and shared what was going on. I wouldnt ever let him run his hands through my hair, I was so self-conscious. So, that was the moment for me and I decided to shave it on New Years Day and document it with a photographer friend. I had my closest friends with meand I know it sounds dramatic because its just hair but it was like everything. It was my identity and my femininity, so it was really huge leap to just surrender and take this into my own hands.
NB: What was it like to shave your head. Did you learn anything about yourself or your scalp from the experience? RL:I mean, showering was such a giftI called it orgasmic earlier which is really funny. I hated showering so much before that. I hated getting my head massaged because they would touch me underneath and I was afraid they would feel what was happening. So, to not have to worry about it and to just the positive response I recieved from people that loved how I looked or people who could also relateit just was overwhelmingly positive even though it was about something I was so frightened of. It was really liberating and Ive never looked back. Its been a huge gift of growth for me and Im grateful for it, even the pain.
NB: How has Harklinikken helpedyou regrow your hair? RL:I was skeptical because I tried literally everything. Nine weeks after starting Harklinikken, I went back and we did a before and after of my hair and it was not only growing back, the quality of my hair has improved so much. I have something called androgenetic alopecia, which is so common for so many women. Its basically genetic diffused thinning of my hair through aging and through stress, hormonal birth control, and all the things. And, you know, I can honestly say my hair is in the best shape its ever been. I no longer color my hair. Im embracing my natural gray and silver and Ive been given my life back. It sounds dramatic, but thats truly how I feel.
NB: Yes, its so crazy sometimes how that works. The thing you fear the most, once youve gone through it, its sometimes the thing that helps you progress the most. RL:Yes, and its been true in other areas of my life, like you know I lost my husband almost four years ago to suicide and mental illness, and that was a gift too. Obviously losing my partner was the worst thing that ever happened to me, but then its through time and through the process of healing that you see the gifts that come out of that, like loving myself in the way that he loved me. For a long time, I didnt value myself enough. Losing him and knowing how much he loved me, I now do value myself and that has led me to the relationship Im in now. So, all of it is like this journey that we go on and these trials and tribulations that lead us to a better place hopefully.
NB: Is there any advice you would give to a younger you if you could? RL:I wouldnt have taken hormonal birth control. I would have stopped the yo-yo dieting and the self-loathing. I mean I just I beat myself up so much. I think so much of this has to come with age. You know people often say it and I didnt believe it to be true, but now I do see that when you turn 50 you dont really care what people think. What people think of me is none of my business. So, I definitely would have been kinder to my younger self if I had the opportunity.
NB: How has your approach to skin care and anti-aging changed from decade to decade? Is there anything youre doing now that you swear by? RL:Not really, I mean I get a lot of sleep I take really good care of myself. I eat well. I have a really active sex life. I mean I feel like Im in my sexual prime and my partner would agree. Im lucky that I do not have wrinkles! Im 52 and I think its purely genetic. I would get a facial every couple of months, but I dont anymore because of COVID. I used to get fake eyelashes and I dont even do that anymore!
At NewBeauty, we get the most trusted information from the beauty authority delivered right to your inbox
Find a NewBeauty "Top Beauty Doctor" Near you
Read the original post:
Ricki Lake Opens Up About Her Stunning Hair Transformation and How Sex Is the Best Anti-Ager - NewBeauty Magazine
For many women, salon closures and stay-at-home orders meant embracing their gray strands after decades of dye; for others, it meant hours of research about at-home hair color, root cover-ups, and lots of really cute headbands.
Lets get into the science behind it: As hair is being formed, melanocytes inject pigment into keratinocytesthe cells containing keratinwhich is the protein making up hair, skin and nails, says Wayne, NJ plastic surgeon and hair specialist Jeffrey B. Wise, MD. Over time, melanocytes continue to inject pigment into the hairs keratin, which is where hair gets its color. In the aging process, melanocytes slow down and eventually stop secreting melanin, which causes a lack of pigment, and the hair turns gray.
According to Chicago dermatologist Dr. Quenby Erickson, going gray is programmed in our genetic code, which means we can get clues as to how extensively and when it will happen by looking at our parents. However, a 2020 study published in Science Daily shows there may also be a link between stress and gray hair. When testing on mice, researchers found that the type of nerve involved in the fight-or-flight response causes permanent damage to the pigment-regenerating stem cells in the hair follicle. The study makes perfect sense, says Dr. Wise. Stress is a huge factor in premature aging, as well as hair thinning. Naturally, it should also affect hair graying as well. There is also a lot of evidence that shows smoking cigarettes plays a role in making hair go gray earlier.
Color isnt always the only factor either; textural changes can ensue as well. Some people are blessed with gorgeous gray hair, but for most of us, the gray is accompanied with thinning and rougher texture that leave our hair finer and harder to style, Dr. Erickson says. There are no proven ways to prevent hair from turning gray, but both Drs. Erickson and Wise have seen some promising results from platelet-rich plasma (PRP) injections. Because these treatments are aimed at waking up your own stem cells, they could potentially reinvigorate melanocyte production as well, explains Dr. Wise. We have seen growth of darker, thicker hairs on some of our stem cell therapy patients, even though the original goal was to combat thinning. Treatment results are dependent on the patients individual conditions, so realistic expectations should be set by your doctor.
Celebrity colorists Chad Kenyon and Rita Hazan say none of their clients embraced their grays during quarantine, or they tried, but caved eventually. For those in camp cover them up, topical dyes and root concealers can help camouflage. The process to cover gray hair is the same on both blonds and darker shades, but my clients with lighter hair can go longer in between touch-ups because gray hairs blend with blond hairs more easily, says Kenyon. Celebrity colorist Aura Friedman often suggests adding a darker pepper tone to silver hair for people who feel more comfortable being darker, but dont want the two-, three- or four-week regrowth touch-up thats needed.
For those who want to permanently cover their grays at home, Nikki Lee, celebrity colorist and cofounder of Nine Zero One Salon, recommends Garnier Nutrisse Nourishing Color Creme ($8). There are more than 75 shades and you can easily find your match using a virtual shade selector, she says. If DIY color makes you nervous, temporary root sprays are great to use in between salon appointments.
At NewBeauty, we get the most trusted information from the beauty authority delivered right to your inbox
Find a NewBeauty "Top Beauty Doctor" Near you
Read more:
Why Hair Goes Gray, and How to Cover It If You Want To - NewBeauty Magazine
Global Healthcare Inventory Management Market: Overview
Global Healthcare Inventory Management Market: Notable Developments
Some of the notable developments in the global healthcare inventory management market include:
Request Brochure of Report - https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=70833
Global Healthcare Inventory Management: Drivers and Restraints
The following are the factors that act as drivers and restraints in the global healthcare inventory management.
Request for Analysis of COVID-19 Impact on Healthcare Inventory Management Market - https://www.transparencymarketresearch.com/sample/sample.php?flag=covid19&rep_id=70833
Optimum Efficiency to Promote Global Healthcare Inventory Management
Inventory management saves time in tracking devices and equipment that are needed for patient. This results in optimum efficiency and more time for patient care, owing in the expansion of the global healthcare inventory management market. The tacking system uses GPS and RFID (Radio Frequency Identification) technologies to identify the inventory.
Additionally, if the equipment, devices, and medicines are not available on time, this could be fatal at times. Therefore, the demand for the inventory management is high due to large pool of patients seeking medical care; this is leading to the growth opportunities in the global healthcare inventory management market.
Request for Custom Research - https://www.transparencymarketresearch.com/sample/sample.php?flag=CR&rep_id=70833
Minimal Investment to Bolster Demand
Surgical devices and equipment are of high value and are not replaced often. If not managed properly it may go missing while handling them and result in high investment to acquire new equipppment. However, this is where inventory management comes handy in maintaining them, pushing the growth of the global healthcare inventory management market in upcoming years.
Further, the inventory management helps in stock taking and stock piling. It also helps in keeping a track of expired or obsolete stock. Additionally, it helps in minimizing the spreading of infections and diseases by stocking and sterilizing of devices and medical equipment. This reduces the chances of spreading of infection and diseases, aiding in the grandiose growth of the global healthcare inventory management over the forecast period.
Rapid technological advancements and rising research and development are also anticipated to drive the inventory management market. Mobile devices with longer battery life are expected to speed up the tracking process. Further, inventory tracking can reduce the cost of labor, aiding in the expansion of the global healthcare inventory management market.
Pre Book Healthcare Inventory Management Market Report at https://www.transparencymarketresearch.com/checkout.php?rep_id=70833<ype=S
Global Healthcare Inventory Management Market: Geographical Analysis
Previously, North America dominated the global healthcare inventory management market. The growth can be due to the presence of technological advancement, better medical infrastructure, and presence of large number of hospitals and research labs.
Asia Pacific is projected to witness an impressive boost in the global healthcare inventory management market. The growth in this region can be attributed to factors such as rapid industrialization and urbanization, improvement in medical infrastructure, and technological advancement.
Browse More Reports by Transparency Market Research:
Stool Management Systems Market: https://www.transparencymarketresearch.com/stool-management-systems-market.html
Adipose Derived Stem Cell Therapy Market: https://www.transparencymarketresearch.com/adipose-derived-stem-cell-therapy-market.html
Severe Combined Immunodeficiency Treatment Market: https://www.transparencymarketresearch.com/severe-combined-immunodeficiency-treatment-market.html
About Us
Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.
Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through ad hoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.
TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.
Contact Mr. Rohit Bhisey Transparency Market Research State Tower, 90 State Street, Suite 700, Albany NY - 12207 United States USA - Canada Toll Free: 866-552-3453 Email: sales@transparencymarketresearch.com Website: https://www.transparencymarketresearch.com/
See more here:
Healthcare Inventory Management Market: Rising Research and Development is Anticipated to Drive the Market - BioSpace
With the rising demand for cell and gene therapies, the need for manufacturing innovation has never been higher. A surge of deals and expansions in the last year is fuelling the push to truly make these therapies widely available and affordable.
Cell and gene therapies offer huge potential to treat a wide range of diseases including cancer, neurological, and genetic diseases. They have even shown promise to treat the symptoms of Covid-19.
The amount of academic and early-stage biotech research in this area has skyrocketed over the last few years. According to the Alliance for Regenerative Medicine, there are currently 1,220 ongoing clinical trials in this space, 152 of which are at phase III. Despite the global pandemic, investment in this area is also at a record high around the world, with the equivalent of 15.7B invested in 2020, a figure double that of 2019.
But research alone cannot get these complex treatments to patients. The sharp discrepancy between the high number of products in early-stage development and the still very small number that have made it onto the market, as well as their cost, speaks to the impact and importance of cost-effective and scalable manufacturing, Ryan Cawood, CEO of Oxgene (previously Oxford Genetics), told me. Oxgene is a UK biotech aiming to improve manufacturing for cell and gene therapies.
To meet this challenge, cell and gene therapy producers are exploding into motion. With 2021 only just getting started, weve seen manufacturing deals between Vigeneron and Daiichi Sankyo, Sirion Biotech and Cellectis, and Cevec and Biogen. The giant Thermo Fisher Scientific absorbed the Belgian viral vector producer Henogen for 724M. And CDMO heavyweights like Cognate BioServices and Polyplus Transfection have announced expansions to their manufacturing capacity.
Thedifficulties with manufacturing the recently approved Covid-19 mRNA vaccines in high enough quantities has really highlighted the importance of having a solid manufacturing strategy in place. This lesson applies equally to companies trying to take cell and gene therapies to market.
Stuck in the first generation
Despite the huge increase in development of cell and gene therapies over the past couple of years, manufacturing technology for these therapies is largely still at the first-generation stage. This can make scaling up a challenge.
Often cell and gene therapy manufacturing processes are highly manual stemming from the early academic or process development stage and, without adequate technology solutions available currently, these processes often remain this way through clinical trials and then into commercial manufacturing, said Jason Foster, CEO of Ori Biotech, a London- and New Jersey-based company focusing on cell and gene therapy manufacturing.
These first-generation processes cause manufacturing to be expensive, highly variable and low-throughput, which reduces the ability of patients to access these potentially life-saving therapies.
Another problem common to all bio-based therapeutics is that any product sourced from a live cell or a component of one is subject to a lot more variation than a simpler pharmaceutical product.
Most gene therapies are built on viruses found in nature. They have not evolved for very high productivity in a large-scale, animal component-free bioreactor, said Cawood.
The more complicated the biologic becomes, the more parts of it require optimization, and the more analytics you require.
According to Kevin Alessandri, the cofounder and CEO/CTO of the French company TreeFrog Therapeutics, there is also a lot of waste in cell therapy manufacturing.
Yields are impaired by high cell death at every passage, and genetic alterations inevitably arise, said Alessandri. When it comes to producing commercial batches to treat thousands of patients, scaling out 2D cell culture processes is far too expensive and poses batch-to-batch reproducibility issues.
While many in the industry are now turning to bioreactors to produce cells on a bigger scale, this is also not without problems. Impeller-induced shear stress is damaging the cells, thus negatively impacting cell viability and triggering undesired genetic mutations, explained Alessandri.
Taking manufacturing up a gear
What are companies in this space doing to make scaling up cell and gene therapies easier, quicker, and cheaper?
Ori Biotech raised24.8M in Series A funding in October last year to develop an automated and robotic manufacturing system to minimize the number of manual steps needed to produce a given cell or gene therapy. This speeds up the process as well as making it more accurate. Another advantage of the technology is that it can tailor the production capacity according to demand.
This is impossible to do in most current processes, which involve manual tube welding and transfers from flask to bag to bigger bag to bioreactor, said Foster, adding that this increases cost and variability while constraining throughput. Oris technology, in contrast, could take years off the production timeline and cut costs by as much as 80%.
London-based Synthace is one of several companies trying to improve advanced therapeutic manufacturing by developing software and computer systems to optimize the process, rather than industrial machinery.
Peter Crane, Corporate Strategy Manager for the company, said that in-depth data analysis and planning before starting the manufacturing process can make a big difference to outcomes, and that connected software can help make this task easier.
The best way to remove some of the risk associated with biomanufacturing of these products is to solve as many problems as possible before manufacturing.
In addition to making the process quicker, cheaper, and more accurate, computing tools can also help with quality control and tracking. In cell therapy manufacturing, especially autologous products, line of sight around electronic batch records, as well as the vein-to-vein supply chain, is incredibly important, emphasized Crane.
Another company specifically focusing on logistics and quality control is the Cardiff- and San Francisco-based TrakCel, which nailed deals with Ori Biotech in February and the UKs National Health Service in November.
The company TreeFrog Therapeutics works with cell encapsulation technology to improve quality and reduce waste, albeit from a more mechanical viewpoint. The company launched an industrial demonstration plant in June last year, followed by two co-development deals with undisclosed big pharma partners.
Encapsulated stem cells spontaneously self-organize in an in vivo-like 3D conformation promoting fast and homogeneous growth, as well as genomic stability, said Alessandri. The resulting 3D stem cell colony can then be differentiated in the capsule into functional microtissues ready for transplantation.
With our technology, which is based on high-throughput microfluidics capable of generating over 1,000 capsules per second, it becomes possible to expand and differentiate stem cells at a large scale, in industrial bioreactors, with best-in-class cell quality and reduced operating costs.
Oxgene has a focus on scaling up production for manufacturers. In September, the company launched a technology to scale up manufacturing of viral vector production with less contamination and a 40-fold improvement in yield compared to current methods. Oxgenes expertise with viral vectors also prompted a collaboration deal in April with the CDMO Fujifilm Diosynth Biotechnologies.
Innovation in new manufacturing technologies just hasnt kept pace with the level of discovery around genetic disease and potential avenues open to treat them, or even development of the viral vectors themselves, said Cawood. This is definitely changing though.
Enter the second generation of manufacturing
Cell and gene therapy manufacturing is definitely hot right now, boosted by increased needs from biotech and pharma companies developing Covid-19 vaccines and therapies, and by notable increases in investment.
Huge advances in gene and cell therapies over the last few years, such as the approval of the eye gene therapy Luxturna and the first CAR T-cell therapies, mean the demand for new manufacturing technologies has also increased exponentially.
A lot of very promising programs are now in the pipeline, and patients are waiting for their approval, said Alessandri. Industry urgently needs robust manufacturing technology, capable of serving millions of patients.
European biotechs are busy developing second-generation technologies to allow easier and cheaper scale up, producing higher quality products with less waste. They could start to phase out first-generation methods very soon.
The realm of cell manufacturing in industrial and food biotech is also likely to see big breakthroughs in the coming years. Earlier this month, for instance, the nutrition and health giant Royal DSM set up a lab in the Netherlands dedicated to applying artificial intelligence (AI) to the challenge of growing microbial strains at a commercial scale.
Rapid improvements in advanced computing options such as AI and machine learning technology, as well as robotics, are already having an effect on the industry, but this will only get bigger as time goes on.
Cover image from Elena Resko. Body text image from Shutterstock
More:
Cell and Gene Therapy Firms Gear up to Revolutionize Manufacturing - Labiotech.eu