StemCell Therapy

DORVAL, QC, Oct. 5, 2020 /CNW Telbec/ - Novartis Pharmaceuticals Canada Inc. (Novartis) is pleased to announce that Beovu (brolucizumab injection) is now available in Canada for the treatment of neovascular (wet) age-related macular degeneration (AMD). The next-generation anti-VEGF therapy for wet AMD, offers the option for eligible patients to start on three-month dosing intervals after the loading phase2,3. Beovu has been reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) and has received a positive recommendation for reimbursement by participating public health plans1.

With wet AMD, which can be aggressive in its progression, abnormal blood vessels form and grow under the central retina (macula) and cause it to swell up, distorting central vision. These abnormal blood vessels may leak fluid or blood in the eye, interfering with the macula's function and causing increasingly severe central vision loss.2,4

"Novartis has a deep history in ophthalmology and an ongoing commitment to eliminating preventable blindness by bringing innovative treatments to patients with serious eye diseases. We are dedicated to continuing to invest in research and as well as programs that improve the lives of people with wet AMD and are very proud to bring this latest advancement to Canadians," said Andrea Marazzi, General Manager, Novartis Pharmaceuticals Canada Inc. "We are pleased that the clinical value and benefit of Beovu have been recognized by CADTH and look forward to collaborating with decision makers to ensure access to Canadians in a timely manner."

AMD is the leading cause of vision loss in people over the age of 50, affecting more than a million Canadians. Symptoms of AMD involve loss of central vision, which is needed for recognizing faces, reading, driving and other daily activities. AMD comes in both dry and wet forms, with wet AMD being rarer and much more severe.4

"It's so easy to attribute a change in eyesight to getting older. We have been working hand in hand with Canadian vision organizations to drive the message across that any changes in vision should be immediately addressed. We know just how profound the impact of untreated wet AMD can be on a person's quality of life. Keeping up with injections may be challenging on older adults who are less inclined to want to visit clinics and hospitals. This is truer today than ever before," said Louise Gillis, President, Canadian Council of the Blind. "We are pleased that Beovu is available in Canada. It's a new option that offers the potential for Canadians diagnosed with the leading cause of blindness to maintain their vision and not have to worry about frequent visits for their injections. Missing out on treatment could potentially be catastrophic for someone with wet AMD."

Anti-vascular endothelial growth factor (anti-VEGF) therapy is widely regarded as the standard of care for patients with wet AMD1. Approved by Health Canada in March 2020, Beovu is the latest available anti-VEGF therapeutic advance, addressing the strong need for a treatment that reduces the need for frequent follow up.

Beovu (brolucizumab injection) Important Safety InformationThe full prescribing information for Beovu can be found at: http://www.novartis.ca.

About Novartis in ophthalmologyAt Novartis, our mission is to discover new ways to improve and extend people's lives. In ophthalmology, we develop and deliver life-changing medicines and therapies for diseases and conditions from front to back of the eye, enabled by data and transformative technologies. Our ophthalmic solutions reach more than 150 million people per year, from premature infants to the elderly.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consultwww.novartis.ca.

About NovartisNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 105,000 people of more than 140 nationalities work at Novartis around the world. Find out more at http://www.novartis.com.

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Lori Bogdanis, Novartis Pharmaceuticals Communications, +1 514 708 4801, E-mail: [emailprotected]

http://www.novartis.ca

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Next-generation wet AMD therapy Beovu, now available in Canada, receives positive recommendation from CADTH Drug Expert Committee - Canada NewsWire

When Jim OConnell joined the University of Florida as the assistant vice president of technology commercialization in 2016, UF Innovate | Ventures was part of his overall plan. He secured the money for the venture fund, recruited a seasoned director, and made Ventures a reality last September. The goal: quality UF startups.

Startups play a significant role in economic growth creating jobs, introducing new ideas and solutions to the marketplace, said OConnell, but without proper leadership, direction, and funding, the odds are against them. Ventures seeks to support UF startups in those areas.

Toward this aim, Ventures hired four Entrepreneurs in Residence, selected an investment committee and advisory council, invested in four different startups, created a network of investors on speed-dial, and helped new startups form and strengthen.

The EIRs have provided valuable insight and connections to investors and entrepreneurs across the state of Florida, said Jackson Streeter, MD, director of Ventures. Before joining UF Innovate, Streeter was the head of the Florida Technology Seed Capital Fund. He also served as CEO for Banyan Biomarkers, a UF startup.

Meet the entrepreneurs in residence

The four Entrepreneurs in Residence turn early business concepts into fundable companies, help companies find capital and strategic resources. One of the four said his biggest contribution to Ventures is offering warm introductions to his ties to companies, investor groups, successful entrepreneurs, and other talent that might provide value to the program.

Its been a pleasure working with the other Entrepreneurs in Residence from throughout the state of Florida to assist Jim OConnell and Dr. Jackson Streeter and the entire UF family, said Dr. Richard Munassi, an Entrepreneur in Residence located in the Tampa/St. Petersburg area. Being able to support the university, researchers, and students has been incredibly rewarding.

Munassi is a seasoned entrepreneur who has been working with tech startups for more than a decade; his primary focus is on healthcare technology, medical devices, and health IT. Entrepreneur in Residence Chris Carter of Ponte Vedra Beach is an angel investor and has served in a variety of mentorship, advisory, consulting, and venture development roles in support of entrepreneurs.

Robert Mino, a triple Gator living in Palm Beach County, is a corporate and patent attorney with broad commercial and research experience beyond the practice of law. Kelly Murray of Orlando centers her work on bridging health innovation and affordable housing with private investment and collaboration in both the public and private sectors.

Though our backgrounds have similarities at some point, we also augment each other by providing value in different ways with regard to the entire life sciences, health tech, and biotech industries, said Munassi. I think its a really strong group top to bottom. Jim and Jackson did a great job of choosing the Entrepreneurs in Residence, and I feel lucky to be a part of that.

Meet the investment committee and advisory council members

In addition to the four Entrepreneurs in Residence, Ventures enlisted the help of Investment Committee and Advisory Council members who independently review and approve investments made by UF Innovate | Ventures and provide strategic advice to the fund and the portfolio companies.

It is very exciting that UF has for the first time created a venture fund, said Streeter. Most leading universities have associated venture funds, and now UF has as well.

Two members of the Investment Committee and Advisory Council, Bill Gurley and Rodney Rogers, are UF graduates. Gurley has spent 20 years at Benchmark as a venture capitalist working with such companies as GrubHub, OpenTable, Stitch Fix, Uber, and Zillow. Rogers has more than 30 years in the tech services industry and is a noted serial entrepreneur and expert technologist known for his leadership contributions to startups that achieved billion-dollar valuations.

Bob Crutchfield is the managing director of Hotspot Ventures. He has also served on the boards for companies such as Shipt, Wellcentive, nContact, and Novamin. Randy Scott is a partner and co-founder of HealthQuest Capital, a venture capital firm investing in companies that transform the value of healthcare. Chris Walker is a private equity expert with HarbourVest Partners who has focused most of his efforts on U.S. venture capital and buyout fund investments.

Companies funded in FY20

The group selected four companies to benefit from the $1 million venture fund Ventures can invest each fiscal year. FY20s recipients included:

An example as to how Ventures works

In addition to investing in those existing UF startups, Ventures also helps form new startups by evaluation of market opportunities, strategic planning, recruiting management and board members, and finding investors. This year, Ventures helped Agricultural Intelligence,Rain Neuromorphics, Adjuvolt, and others.

Adjuvolts story shows how Ventures works. It began in the UF vet school, which encounters numerous instances of osteosarcoma, bone cancer. Osteosarcoma is the most common form of bone cancer. It is highly metastatic. If it is located in a dogs leg, it requires amputation and chemotherapy, which is only moderately effective.

In interactions with the vet school, Ventures leader Streeter heard about a technology developed byRowan Milner, a cancer vaccine to treat osteosarcoma in dogs. This isnt a vaccine given to prevent a dog from developing cancer; its a vaccine given to a dog to treat cancer once a tumor has developed.

Developing effective cancer vaccines has proven challenging due to the difficulty of eliciting a robust and consistent immune response against self antigens. Dr. Milner has found a way to formulate a vaccine that targets an antigen specific to osteosarcomas. Hes used it hundreds of times in dogs with success.

Humans and dogs have shared histopathologic and genetic features. In humans, osteosarcoma is often found in children. Could this technology extend to human care?

Ventures explores the technology, decides it has value and potential. It then identifies management for a potential startup: Richard Currie, Ph.D., BVM&S. Currie is a veterinarian and an entrepreneur, has experience in the multinational pharmaceutical and diagnostic sectors.

A company is born. Ventures will continue to help guide Adjuvolt, connect it to investors. The hope is that Adjuvolt can take a vaccine created for dogs in a lab at UFs vet school and, eventually, create vaccines for humans. The sequence might save the leg of a small human suffering from osteosarcoma. And many more.

Sara Dagen October 5, 2020

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UF Innovate | Ventures celebrates first year successes - University of Florida

TEL AVIV, Israel, Oct. 5, 2020 /PRNewswire/ --Tarsius Pharma, a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat patients with blinding ocular diseases, announced today that the GADOT 20/20 trial of its novel TRS01 drug for treating Active Anterior Non-Infectious Uveitis, demonstrated significant improvement in critical measures such as ACC (Anterior Chamber Cells), pain reduction and increased visual acuity.

The GADOT 20/20 trial was a dose-ranging, randomized, double-masked, controlled phase I/II clinical trial that evaluated the safety and efficacy of TRS01 in 16 subjects with active non-infectious anterior uveitis in the U.S. The study compared two different doses of TRS01.

Results showed a statistically significant improvement in the signs and symptoms of the disease, with the high-dose usage being superior to low-dose in a few important measures. Firstly, a consistent clearing of the anterior chamber cells (ACC) from baseline was achieved. Secondly, prompt resolution of pain was present, alongside increase of visual acuity. Also, compared to rescued subjects that received steroids, a reduction of IOP (intraocular pressure) was demonstrated in patients of both treatment arms.

"The results of this clinical trial with TRS01 are truly impressive, and suggest meaningful progress towards the development of a novel and clinically important class of ocular therapeutics topical anti-inflammatory agents with efficacy that is at least equal to corticosteroids, but with none of the known corticosteroid-associated side-effects," stated Dr. Ron Neumann, Tarsius Pharma's CMO.

Dr. Daphne Haim-Langford, Tarsius' CEO, said: "The results of this study bring us one big step forward in our journey to save the eye-sight of patients with uveitic glaucoma, who have no approved treatment available today for this devastating condition. Completing this trial during the COVID-19 pandemic, proves once again the emergency nature of uveitis flare-ups. On behalf of our entire Tarsius team, I thank the investigators, volunteers, and all study team members for contributing to the professional execution and evaluation of the clinical process, and to the understanding of TRS01 in humans."

About Ocular Inflammatory Diseases

Ocular inflammatory diseases impose a significant medical and economic burden on society, affecting hundreds of million people worldwide and posing severe risks of vision loss and blindness.

In more than 50 years, corticosteroids are the most common method used to treat ocular non-infectious inflammation of any kind. Rather than treating the disease itself, however, steroids merely relieve its symptoms, and long-term use can result in life-changing consequences.

The TRS Platform Technology has the potential to effectively treat a broad array of autoimmune and inflammatory ocular diseases. After a successful PoC which demonstrated TRS01's safety and efficacy, Tarsius Pharma is well-positioned to advance its novel platform technology in other sight threatening ocular diseases.

About Tarsius Pharma

Tarsius Pharma was established in 2016and is focused on developing TRS, a breakthrough, bio-inspired platform technology for the treatment of blinding ocular diseases.

This project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement No. 879598.

Contact:info@tarsiuspharma.comTel: +1-914-3392556

Photo - https://mma.prnewswire.com/media/1307023/Tarsius_Pharma.jpg

View original content:http://www.prnewswire.com/news-releases/tarsius-pharma-achieves-positive-results-in-proof-of-concept-study-for-trs01-targeting-uveitis-301145540.html

SOURCE Tarsius Pharma

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Tarsius Pharma Achieves Positive Results in Proof of Concept Study for TRS01 Targeting Uveitis - BioSpace

Ministry of Health is close to declaring Wadelai (Pakwach District) and West Nile (Koboko and Yumbe) foci as River Blindness Free

Ministry of Health is close to declaring Wadelai (Pakwach District) and West Nile (Koboko and Yumbe) foci as River Blindness Free

Uganda has stopped interventions in 2019 among 608,219 people in Nyagak Bondo focus previously treated for river blindness (Onchocerciasis), based on the current World Health Organization (WHO) guidelines, bringing the country closer to achieving its goal of eliminating river blindness nationwide. River blindness has been known as a public health problem affecting a significant population of Uganda since early 1950's.

The Government of Uganda declared a nationwide elimination policy in 2007. River blindness is a vector borne disease that is transmitted by the black flies which breed in fast flowing rivers. It causes serious eye and skin disease that may result in blindness. The disease was originally endemic in 40 districts in Uganda with about 4.6 million people at risk, excluding districts in the Victoria Nile focus that eliminated the disease in the early 1970's. CLICK HERE FOR MORE ON THIS NOTICE

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Notice From Ministry Of Health - New Vision

The global intraocular lens market size was estimated at USD 3.95 billion in 2019 and projected to reach USD 6.40 billion at a CAGR of 5.5% over the forecast period. Different informative opinions and markets on the global intraocular lens market from different global C-level professionals and industry leaders have been embedded in the report. Different top-level key players across the global regions have been profiled to get better insights into the global intraocular market. Additionally, it offers a detailed elaboration on various aspects such as global market policies, rules, and regulations imposed by governments to set standards for global businesses. Furthermore, different global competitors and new entrants are also presented in the report for understanding the degree of competition globally. The global market research report has been presented in a clear and professional chapter-wise format for understanding the subject matter.

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The following factors are encouraging the global intraocular lens market growth over the forecast period:

Alchemy Market Research Profiling the Leading Companies Functioning In the Intraocular Lens Market:

Driving Factors:

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The global market intelligence report has been prepared with the help of the following pointers:

Key Questions Answered Through This Global Market Research Report:

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Intraocular Lens Market is projected to reach USD 6.40 billion at a CAGR of 5.5% - Credence Turbine

In this day and age, corporations are really able to do virtually anything and everything they could want. If they wish to open a new office or launch a new division, they are often able to do so. Why then, are so many companies turning to philanthropic measures? Let's take a look.

Public Pressure

Nowadays, brand loyalty can often mean nothing to customers. While you once would have picked one company and stayed with them for your entire life, this is not the case any longer. If a brand does something that is not approved of by many of their customers, there is a chance that they will leave and go to a competitor.

As a result, many corporations have turned to philanthropic pursuits. This is a great way for them to respond to the concerns of their customers while also acknowledging some of the major events and issues currently facing us worldwide. For example, many corporations are making a commitment to going carbon-neutral within their business dealings. While this will require an awful lot of work on their part, and might also involve some serious redirection within the business, it is a fantastic move that will satisfy old clients and potentially attract new ones too.

Caring for Causes

Of course, it is not just the public who have control over these philanthropic pursuits. Company directors and CEOs are also directing their corporations towards issues and areas that they personally care about.

Two great examples here are Tej Kohli and Bill Gates. Tej Kohli is the founder of the philanthropic Tej Kohli Foundation which is engaged in several missions around the world. In particular, they are committed to trying to eliminate corneal blindness, one of the most common types of blindness in the world. Bill Gates and his wife Melinda set up the Bill and Melinda Gates Foundation. They have been engaged in healthcare philanthropy to help improve the living conditions for many thousands of people all across the world.

Uplifting Others

As there continues to be a growing wealth gap between the top percentiles of our society, corporations are beginning to look into ways they can offer help to some of those lower down. This could be in the form of a scholarship to attend higher education, or it could be through the form of work experience within the company.

It could even be as simple as constructing a local community centre for everyone to use. There are many small ways that a corporation can provide better opportunities to those in poorer areas. We never know what someone can achieve when given the right tools for success.

This is a trend that is most definitely not going away. With more and more corporations turning to philanthropic efforts, we are likely to see a lot of good come out of this world in the future. Take a look at some of your favourite companies to see what they are doing, or even look at some of the paths that you could adopt as a business owner.

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Why Corporations Are Turning To Philanthropy - International Business Times UK

The National Research & Training Center on Blindness & Low Vision (NRTC) is proud to announce our receipt of another competitive national grant. This grant, Independent Living Services for Older Individuals Who Are Blind Training and Technical Assistance, is sponsored by the Rehabilitation Services Administration under the U.S. Department of Education. This 5-year grant, which provides approximately $3 million in funding, begins October 1, 2020.

Sylvia Stinson-Perez is the project director for this grant known as the OIB-TAC. She is excited to take the strong foundation from the last five years of the NRTCs work on this grant and add new and innovative projects. There are three major products related to this grant:

Michele McDonnall, NRTC Director, is happy that the funding will allow the NRTC to continue to improve the provision of services to older individuals who are blind.

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The NRTC Awarded Second Major National Grant | The National Research and Training Center on Blindness and Low Vision - Mississippi State Newsroom

Avrobio has licensed a Hunter syndrome lentiviral gene therapy from the University of Manchester in the U.K. The deal positions Avrobio to join Regenxbio and Sangamo Therapeutics in the race to develop gene therapies for use in patients with the rare disorder.

Hunter syndrome, also known as mucopolysaccharidosis Type II, is driven by a mutation that limits a patients ability to break down sugar molecules generated as a byproduct of cell activity. The buildup of the molecules causes progressive damage to organs such as the brain, affecting the development of children with the lysosomal disorder.

Takedas approved drug Elaprase gives Hunter syndrome patients the enzyme they need to break the molecules down, but its inability to cross the blood-brain barrier renders it ineffective against the CNS manifestations of the disease.

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Avrobio has become the latest company to identify gene therapy as a potentially better way to treat Hunter syndrome. The biotech, which is active in other lysosomal disorders, has agreed to pay the University of Manchester $8 million upfront for the global rights to a stem cell gene therapy that is set to enter the clinic in the second half of next year.

Brian Bigger, Ph.D., a professor of cell and gene therapy at the U.K. university, developed the candidate and co-authored a paper about it in 2018. The paper describes work on a braintargeted hematopoietic stem cell gene therapy designed to give patients the lysosomal enzyme iduronate-2-sulfatase (IDS).

Avrobio will make the therapy, now called AVR-RD-05, by modifying a patients own hematopoietic stem cells with a transgene for IDS expression and a protein tag intended to improve stability of the enzyme. If AVR-RD-05 works as hoped, the cells will engraft in the bone marrow and make copies of themselves that also carry the transgene.

The differentiation of the cells into components of the immune system could enable AVR-RD-05 to cause a lasting, body-wide increase in the presence of the enzyme at the root of Hunter syndrome. Avrobio will fund a 9.1 million ($11.8 millon) investigator-sponsored phase 1/2 trial to assess whether the gene therapy performs as hoped. The biotech is also on the hook for up to $80 million in milestones.

Avrobio will face competition if it gets AVR-RD-05 all the way to market. Takeda is the incumbent, Regenxbio and Sangamo have gene therapies in the clinicalthough the latter has underwhelmed so farand Denali Therapeutics is developing an IDS enzyme designed to cross the blood-brain barrier.

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Avrobio bags gene therapy to join Regenxbio and Sangamo in race - FierceBiotech

Professor Robert MacLaren on the exciting potential of CRISPR gene editing and patients seeing stars for the first-time following treatment

Professor Robert MacLaren, of Oxford University, is leading work to develop gene therapies for inherited retinal conditions through Nightstar Therapeutics. The company was acquired in 2019 by US biotechnology company, Biogen, for $US 877 million.

One night I was at my computer and a patient emailed me to say he could see a night star. This was a positive thing. I said, If you are still there can you go and check which eye it was. He came back and said, It is definitely the eye that I had the gene therapy in.

Subsequently, when we developed the gene therapy company, the name NightStar was an obvious one because since then several of our patients have described seeing night stars after gene therapy. It symbolizes looking up at the heavens and hope for the future, but there is also that practical element of achieving better vision at night.

Although I am an ophthalmologist treating patients, when we are looking at the mechanisms of the disease, we get right down to the molecular level, looking at how the DNA is assembled and how it is processed.

It is very exciting as a physician in any specialty to be able to treat disease at the genetic level. Ultimately if the disease occurs at the genetic level and you treat it there, you are treating the origin of the disease itself. If you are just giving drugs to try and correct the genetic disease or compensate for it, it may help but that is not a real treatment. It provides insight into the mechanisms of life itself seeing how DNA is processed and how the genes evolve.

You can treat potentially any disease with that, whereas gene replacement therapy is limited to small genes. This is still good but ideally if you correct the mutation it is a better treatment than replacing the whole gene.

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Gene therapy: It provides insight into the mechanisms of life itself - AOP

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

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Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

Link:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

ALDERSON, W.Va. (WVVA) A Greenbrier County teen is about to become one of the first five patients in history to receive an experimental gene therapy for Hurler's Syndrome.

The Alderson native is in many ways your typical 13-year-old. "I like to help my dad feed cows....and donkeys," said Kendra Goins.

But life hasn't always been easy for Kendra. The extremely rare condition makes it impossible for her body to breakdown certain sugars. In addition to causing damage to her organs, the condition makes it difficult for her body to grow.

But whenever anyone has anything to say about it, her sister, Kiristen is always the first time stand up.

"Me with my big head is always the one to jump in," said Goins, who said she has gotten into quite a few quarrels over the issue at school in defense of her sister.

She worries though that she won't be able to jump in when Kendra heads to California soon for a clinical trial. Kendra is heading into the treatment with her first bone marrow transplant nearing the end of its course.

"I've spoken with experts across the country from the chemists who made the drug to the doctor who has used it. This looks like her golden ticket," said Kendra's mother Sheryl Goins.

Administered through the brain, the goal of the gene therapy is to help her body produce the enzymes she needs to survive.

While the cost of the clinical trial and airfare is covered, the family said they need help with expenses they will incur during their three-month stay.

To learn more about how you can help, visit https://www.gofundme.com/f/kendra039s-gene-therapy?utm_source=facebook&utm_medium=social&utm_campaign=p_cp%20share-sheet&fbclid=IwAR3yzHCexch5a_awjaYy06ijB28zMXJ-72WkfQ-SPEwMQgy5s8x_carlz34

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Greenbrier County teen to be among first in nation to receive gene therapy for Hurler's Syndrome - WVVA TV

Oct. 5, 2020 08:09 UTC

HARLOW, England--(BUSINESS WIRE)-- Diamond Pharma Services (Diamond), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics (GenSight), including the preparation, authoring support, agency communication and submission of GenSights first Marketing Authorisation Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ. The EMA decision is expected in H2 2021.

LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the US annually.

Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercialising innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege.

This submission adds to Diamonds broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programmes at various stages of development, and two MAA approvals - Glybera and Yescarta.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201005005339/en/

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Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ -...

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy candidate, PF-06939926 (Pfizer), for the treatment of Duchenne muscular dystrophy (DMD).

PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter. The Company has chosen the rAAV9 capsid due to its potential to target muscle tissue.

The designation was based on data from an ongoing phase 1b study evaluating the safety and tolerability of a single intravenous infusion of PF-06939926 in 9 ambulatory boys with DMD aged 6 to 12 years. Preliminary results showed that PF-06939926 was well tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The Company plans to launch a double-blind, placebo-controlled phase 3 study to evaluate the efficacy and safety of PF-06939926 in boys with DMD. The study will include patients who are at least 4 years old and less than 8 years old; all participants will need to be on a daily dose of glucocorticoids for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. The primary outcome of the study (change from baseline in North Star Ambulatory Assessment) will be assessed at 52 weeks; patients will be followed for 5 years after treatment.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. We are working to advance our planned phase 3 program as quickly as possible.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit pfizer.com.

Pfizer receives FDA Fast Track designation for Duchenne muscular dystrophy investigational gene therapy. https://www.businesswire.com/news/home/20201001005382/en/Pfizer-Receives-FDA-Fast-Track-Designation-for-Duchenne-Muscular-Dystrophy-Investigational-Gene-Therapy. Accessed October 2, 2020.

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Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference

NEW YORK, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the Company will present at upcoming conferences and will announce six-month safety and efficacy data from the second cohort of its AXO-Lenti-PD program the morning of Tuesday, October 6, 2020. Additionally, as previously announced the Company will hold a Parkinsons Disease R&D Day on Friday, October 30.

Information on the upcoming presentations can be found below:

Chardan 4th Annual Genetic Medicines Conference

2020 ARM Virtual Cell and Gene Meeting on the Mesa

AXO-Lenti-PD Parkinsons Disease R&D Day

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. Replays will be available for approximately 30 days following the conferences.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

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Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - BioSpace

Global Cancer Gene Therapy Market: Overview

Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

Read Report Overview https://www.transparencymarketresearch.com/cancer-gene-therapy-market.html

Radiation and chemotherapy therapy have consistent and reliable effects to decrease cancerous cells in the body. Recently, immunotherapy for hematological cancers has experienced a recognition and is of interest for many researchers Scientists have developed methods to isolate, replicate, and develop cancer-destroying cells from the patients blood cancer and injecting those cells back for the destruction of their cancers, with durable remissions.

New options for the treatment is needed to be developed if order to achieve elimination of cancer suffering and death by 2020. According to NCI, 5-year survival rate for cancers such as lung (15%), glioblastoma (5%), pancreatic (4%), and liver (7%) remains very low. Current available treatments have several side effects, the systemic toxicity due to chemotherapy results in nausea, mild cognitive impairments, and mouth ulcerations, in addition to long-term side effects such as increasing risk of developing other types of cancers. Therefore, new and innovative treatment methods are required to reduce the suffering of cancer patients.

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Global Cancer Gene Therapy Market: Drivers and Restraints

The emerging field of cancer Gene Therapy offers varied potential treatments. Gene therapy involves a range of treatment types, which use genetic material to alter cells (either in vivo or in vitro) to help cure the disease. Cancer Gene Therapy shown efficacy in various in vitro and preclinical testing. Preclinical testing for cancer gene therapy has been performed on glioma, pancreatic cancer, liver cancer, and many other cancers.

Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market. According to NCBI researchers, development of genetically-modified T-cell therapies for treatment of cancer has had maximum clinical impact among other gene therapies. However, high treatment cost is a major limitation in the cancer gene therapy market. The reason behind the huge cost for cancer gene therapy is the necessity of rigorous, exhaustive clinical trials; also treatment by cancer gene therapy differs from person to person depending upon the genetic acceptance of every patient, unlike other drugs thereby limiting the market growth.

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Global Cancer Gene Therapy Market: Key Segments

Based on type, the cancer gene therapy market is segmented into gene transfer immunotherapy and oncolytic virotherapy. Immunotherapy uses genetically modified cells and viral particles to stimulate the immune system to destroy cancer cells. Immunotherapy include treatment with either cytokine gene delivery or tumor antigen gene delivery. Oncolytic virotherapy uses viral particles, which replicate within the cancer cell causing the death of the cell. It is an emerging treatment modality that is expected to shows great promise, particularly in metastatic cancer treatment. It includes treatment with adenovirus, retrovirus, lentivirus, herpes simplex virus, adeno-associated virus, simian virus, alphavirus, and vaccinia virus. Gene transfer is the newest treatment modality that is expected to introduce new modified genes into cancerous cell or associated tissue for destruction of cell or to slow down cancer growth. This technique is flexible as a wide variety of vectors and genes are used for clinical trials with positive outcomes. As gene therapy advance, they could be used alone or in combination with other treatments to control the disease. Gene transfer or gene replacement is performed using naked/plasmid vectors, electroporation, sonoporation, magnetofection, and gene gun.

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Based on region, the global cancer gene therapy market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America is anticipated to hold the largest market share. The U.S. dominates the cancer gene therapy market owing to its increase in funding for research & development and other government initiatives. Key players in the biotech industry are engaging in research & development of gene therapy products. Moreover, rising demand for DNA vaccines and growing interest of venture capitalists to investment in commercialization of gene-based cancer therapies are likely to propel the market. The cancer gene therapy market in Asia Pacific is anticipated to expand at a rapid pace as in China cancer gene therapy is anticipated to attribute for largest revenue, due to the recent launch of Gendicine and rising healthcare expenditure with strong R&D facilities.

Global Cancer Gene Therapy Market: Key Players

Key players operating in the global cancer gene therapy market are Adaptimmune, ZioPharm Oncology Altor Bioscience, MolMed, bluebird bio, Shanghai Sunway Biotech company limited , MultiVir, Shenzhen SiBiono GeneTech, Corporation.

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Cancer Gene Therapy Market is Expected to Expand at an Impressive Rate by 2025 - The Daily Chronicle

CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Phillip Samayoa, vice president of strategy and portfolio development, will present a company overview at the annual Cell & Gene Meeting on the Mesa. The presentation will be available for registered participants to view on demand throughout the conference, to be held virtually Oct. 12-16, at meetingonthemesa.com.

Geoff McDonough, M.D., Generation Bios president and chief executive officer, will participate in a panel on the future of gene delivery. That discussion will be available to view on demand on the conference website starting Oct. 13.

About Generation Bio

Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.For more information, please visitgenerationbio.com.

Contact:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

Researchers at the Massachusetts Institute of Technologys (MIT) research enterprise Critical Analytics for Manufacturing Personalized Medicine (CAMP) have been awarded Intra-CREATE grants from the National Research Foundation (NRF).The grants will support research on retinal biometrics for glaucoma progression and neural cell implantation therapy for spinal cord injuries.

Krystyn Van Vliet, co-lead Principal Investigator at Singapore-MIT Alliance for Research and Technology (SMART) CAMP, and Professor of Materials Science and Engineering at MIT, said: Singapores well-established biopharmaceutical ecosystem brings with it a thriving research ecosystem that is supported by skilled talents and strong manufacturing capabilities. We are excited to collaborate with our partners in Singapore, bringing together an interdisciplinary group of experts from MIT and Singapore, for new research areas at SMART.

In addition to our existing research on our three flagship projects, we hope to develop breakthroughs in manufacturing other cell therapy platforms that will enable better medical treatments and outcomes for society.

Hosted by SMART CAMP, the first research project, Retinal Analytics via Machine learning aiding Physics (RAMP), brings together an interdisciplinary group of ophthalmologists, data scientists, and optical scientists from SMART, Singapore Eye Research Institute (SERI), Agency for Science, Technology and Research (A*STAR), Duke-NUS Medical School, Massachusetts Institute of Technology (MIT), and National University of Singapore (NUS). The team will seek to establish first principles-founded, and statistically confident models of, glaucoma progression in patients, which will enable rapid and reliable forecast of the rate and trajectory of glaucoma progression, leading to better targeted treatments.

MIT Mechanical Engineering Professor Barbastathis, co-leader of the research and Principal Investigator at SMART CAMP said: We look forward to leveraging the ideas fostered in SMART CAMP to build data analytics and optical imaging capabilities for this pressing medical challenge of glaucoma prediction.

The second research project, Engineering Scaffold-Mediated Neural Cell Therapy for Spinal Cord Injury Treatment (ScaNCellS), gathers an interdisciplinary group of engineers, cell biologists, and clinician scientists from SMART, Nanyang Technological University (NTU), NUS, IMCB A*STAR, A*STAR, French National Centre for Scientific Research (CNRS), University of Cambridge, and MIT.

The team will seek to design a combined scaffold and neural cell implantation therapy for spinal cord injury treatment that is safe, efficacious, and reproducible, paving the way forward for similar neural cell therapies for other neurological disorders. The project aims to achieve its goals through an enhanced biological understanding of the regeneration process of nerve tissue and optimised engineering methods to prepare cells and biomaterials for treatment.

Chew Sing Yian, Principal Investigator at SMART CAMP and Associate Professor of the School of Chemical and Biomedical Engineering, and Lee Kong Chian School of Medicine, at NTU said: Our earlier SMART and NTU scientific collaborations on progenitor cells in the central nervous system are now being extended to cell therapy translation. This helps us address SCI in a new way, and connect to the methods of quality analysis for cells developed in SMART CAMP.

Cell therapy, one of the fastest-growing areas of research, will provide patients with access to more options that will prevent and treat illnesses, some of which are currently incurable. Glaucoma and spinal cord injuries affect many, said Hanry Yu, co-lead Principal Investigator at SMART CAMP.

Our research will seek to plug current gaps and deliver valuable impact to cell therapy research and medical treatments for both conditions. With a good foundation to work on, we will be able to pave the way for future exciting research for further breakthroughs that will benefit the healthcare industry and society.

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New support for personalised medicine and cell therapy - Health Europa

Addressing gaps in testing and education within precision oncology can assist in improving access to patients of color and other underserved populations.

Addressing gaps in testing and education within precision oncology can assist in improving access to patients of color and other underserved populations, said Karen Winkfield, MD, PhD, incoming executive director, Meharry-Vanderbilt Alliance.

Transcript

AJMC: In the era of precision medicine, what can oncologists do to ensure that newer therapies are the right choice for minority patients?

Dr Winkfield: Precision oncology has really come a long way, and I do think that oftentimes people think about it as just genetic testing or genomic testing. That's important, and we do know that there's a gap, even in terms of testing, for some things as simple as an oncotype for breast cancer patients. We know that Black patients are not getting the oncotype test at the same rate as other racial and ethnic groups.

So, really, just making sure that there's access is one thing that can be important. Whether it be partnering with some of the testing facilities to make sure that there is opportunity for individuals who may be underinsured or uninsured to gain access to some of these precision oncology tests, that would be wonderful.

The other thing is making sure people understand, again, community engagement. I'm going to keep coming back to that, because patients need to understand the importance of it and know that they're not being a guinea pig, etc, but that we want to make sure that their treatment is personalized. The personalization is not just to them as an individual, but also to their tumor and what their tumor might be doing. So, that communication is important as well.

So, sometimes it may require a second biopsy. So, you can imagine, if a person has gone through therapy and then their tumor is not responding, and you say, "Oh, we need to get some more tissue," there can be some misunderstandings about that. So, really being open with your patients about the rationale and the reason for getting additional tissue, but, again, making sure that patients don't have very high out-of-pocket cost is one of the things that I think will go a long way to improving access to patients of color and other underserved populations to precision oncology.

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Dr Karen Winkfield on Improving Minority Access, Education for Precision Oncology - AJMC.com Managed Markets Network

OncoHost, announced on 5th October that Professor Yuval Shaked, co-founder and Chief Scientific Advisor at OncoHost, and Professor of Cell Biology and Cancer Science at the Technion Israel Institute of Technology, will deliver a presentation titled A Proteomics-Based Platform for Predicting Response to Immunotherapy and Personalizing Treatment Plans at the MAP 2020 Virtual Congress - ESMO this Friday, October 9th at 17:20 PM CEST.

The presentation will show how through the analysis of host response profiles (i.e. the patients reaction), oncologists may be able to harness this information to better predict clinical outcomes and suggest the ideal combination treatment with immunotherapy.

Despite major clinical success, immunotherapy treatments have demonstrated efficacy in only a small proportion of patients with non-small cell lung cancer (NSCLC), said Prof. Yuval Shaked. OncoHosts studies have indicated that individual host response to anti-cancer treatment can generate pro-tumorigenic activities and support tumor re-growth and spread. It is therefore vital to analyze and earlier predict host response to treatment in order to improve outcomes and reduce unnecessary side effects experienced by so many patients.

Using machine learning (ML)-based analysis and algorithms to identify highly predictive cohort-based proteomic signatures in patients, personalized response prediction can be used to improve precision medicine in oncology. Personalized treatment plans can then be created through information based on cohort-based statistical analysis, personalized adaption and finally, translation to clinically relevant targets.

Prof. Shakeds presentation will include a real-life case study on whom a Host Response profile analysis was performed, and will be followed by a live Q&A.

MAP is the leading congress in precision medicine in oncology in Europe, established by key opinion leaders in the field. The name of the Congress was recently changed into Molecular Analysis for Precision Oncology to better reflect its aims of focusing on science, translational research, education and collaboration. This years event will be held virtually on October 9-10.

Excerpt from:
OncoHost Data on Host Immunotherapy Response to be Presented This Week - Technology Networks