Category Archives: Gene Medicine

Yes, its been a challenging and often tragic year when it comes to health. But our hardest times often push us to our greatest achievements, and theres a lot of progress to be excited about in 2021like these seven advances.

Gene therapy for repairing sickle cell disease

The idea is simple: Take a defective gene, fix it, and cure a devastating genetic disease. Getting that to work has been tougher, but this year scientists at several research centers made remarkable progress with sickle cell disease. The conditioncaused by an inherited defect in the gene producing hemoglobin in the red blood cells that leads to deformed, crescent-shaped cells that cause pain, anemia, and organ damageaffects some 100,000 Americans, mostly Black and Hispanic.

Researchers at Boston Childrens Hospital are testing a gene therapy treatment that tricks the gene into reverting to a healthier version called fetal hemoglobin. In eight patients treated so far, they are seeing the fetal hemoglobin increase and hoping the change will last a lifetime, says Erica Esrick, M.D., a pediatric hematologist there. Several other research groups have reported similarly promising results using gene therapy and gene-editing techniques for patients with sickle cell disease. Experts believe these techniques may one day cure many genetic diseases, such as hemophilia and genetic immune deficiencies, as well as some cancers.

The first new birth control in decades

Women looking for a nonhormonal birth control option finally got a new one this fall. Phexxi is a prescription gel that makes the vagina more acidic so its less welcoming to sperm. Using it is simple: You squirt it from a prefilled applicator up to an hour before sex. Phexxi is not the most effective birth control method (its 86% effective, the manufacturer says, compared with 91% for the Pill and 99% for an IUD), and it doesnt protect against STDs, so you still need to use a condom, but its something to celebrate. The last time new options for women came on the marketthe patch, vaginal ring, and hormonal IUDwas two decades ago, says Elizabeth Watkins, Ph.D., a professor of the history of health sciences at the University of California, San Francisco. The more ways women have to take control of their reproductive futures, the better.

Telemedicine comes of age

Who would have thought so many of us would comfortably visit a doc in our PJs from home? But because of COVID-19, by summer nearly a quarter of us had used a computer, app, or phone for a telemedicine appointment (before the pandemic, only 14% of women said their doctors even offered this option, according to a federal survey). Were now virtually showing a rash to a dermatologist, demonstrating joint pain to a rheumatologist, and getting an antibiotic Rx for a nasty UTI.

Even more significantly, were treating our anxiety via online psychotherapyhugely helpful, especially in areas where therapists are in short supply. Will this tele-trend last? Experts say it wont continue to the same extent, as uneven insurance reimbursements, laws about practicing across state lines, and the belief that docs need to see us in the flesh have physicians eager for patients to return to their offices. But the shift toward at least a few appointments each year that dont require driving, taking a half day off from work, or even changing out of your sweats may be here to stay.

Finally, a focus on Black womens health

Its sad and horrifying that it took publicized deaths at the hands of police and disproportionate suffering from COVID-19 to bring this about, but Black womens health is finally getting the attention it deserves. In 2020, there was increased awareness of the impact of structural racism on health care: Black women suffer pregnancy-related death four times as often as white women, and their mortality rate is higher for everything from heart disease and cancer to asthma and flu.

A true fix will require hard actions that have yet to materialize, but people of all races are taking the crucial first step in asking how we can fix this, says health disparities researcher Courtney Denise Townsel, M.D., a maternal-fetal medicine physician at Michigan Medicine in Ann Arbor. An editorial in the prestigious medical journal The Lancet this summer finally termed racism a public health emergency of global concern. And Congress introduced the Black Maternal Health Momnibus Act to address the health disparities in pregnancy and birth and to inspire innovative solutions with a series of grants.

Super-convenient breast pumps

For decades, breast pumps were stuck in the Dark Ages, with bottles, tubes, and wiring requiring you to sit in one spot for up to half an hour, says Tanya Powell, a board-certified lactation consultant at Sutter California Pacific Medical Center in San Francisco. Thankfully, the past few years have seen miraculous advances, and even more improved in 2020. Today the most convenient pumps, such as Willows Generation 3, slip right into your bra, allowing you to pump as you move around or even as you sleep. The Elvie Pump is silent enough to let you stealthily pump during an online business meeting. Powell cautions, however, that this generation of pumps might not be optimal for establishing or building a milk supply. Consult a lactation consultant to help choose the pump thats best for you.

Disposable medical scopes

Scopes let doctors peer inside your body, but if these flexible lighted tubes with cameras arent thoroughly disinfected between patients, their use can lead to rare but serious bacterial infections. The duodenoscope, a device snaked down the throat to the top of the small intestine to diagnose and treat pancreas and bile duct diseases, has proven especially tough to clean. Now the first fully disposable duodenoscope has been approved by the FDA, which experts expect may finally end the recent spate of infections blamed on dirty scopes. Other similar tossable devices, including one for colonoscopies, are expected in the next few years, making potentially lifesaving exploratory procedures safer.

A novel way to detect Alzheimers

If a loved ones memory is fading, its important to know whether theyre suffering from Alzheimers or another type of dementia, since early detection means better treatment (even if there is as of yet no cure) and perhaps the chance to take part in a clinical trial. But today, a definitive diagnosis requires a clinical evaluation as well as brain imaging or a spinal tap.

So its huge news that scientists have discovered a simple blood test that has accurately diagnosed Alzheimers in early studies. If the test, which hunts for proteins indicative of the disease, performs well in additional studies, it will offer the first easy, early screen for this debilitating disease. For some people, knowing leads to better health behaviors, says Mary Sano, Ph.D., director of the Alzheimers Disease Research Center at Mount Sinai, in New York City. Some may find the knowledge anxiety-producing, but for those who want to get a jump on treatment, the new blood test could be a game changer.

Plus, the biggest health story of 2020

In 2019, no one knew anything about the virus that would later be linked to COVID-19. But in one year, remarkable progress has been made. Several vaccines are showing promise. Doctors are using steroids and high-flow oxygen to keep people alive. New diagnostic tests have emerged, including one using lines like on a pregnancy test. And a powerful way to reduce the spread has been found: wearing a simple cloth mask.

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Top Health Breakthroughs of 2020 Amazing Medical Innovations - Prevention.com

DUBLIN--(BUSINESS WIRE)--The "Growth Opportunities in Gene Therapy, Automated Bioanalytics, and Biomarker Platforms" report has been added to ResearchAndMarkets.com's offering.

The research provides technological insights across inflammation, infectious diseases, and microbiomics.

The Life Science, Health & Wellness TOE will feature disruptive technology advances in the global life sciences industry. The technologies and innovations profiled will encompass developments across genetic engineering, drug discovery and development, biomarkers, tissue engineering, synthetic biology, microbiome, disease management, as well as health and wellness among several other platforms.

The Health & Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare.

Innovations in Life Sciences, Health & Wellness from:

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

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2020 Report: Growth Opportunities in Gene Therapy, Automated Bioanalytics, and Biomarker Platforms - ResearchAndMarkets.com - Business Wire

This free webinar will be held at 6 p.m. ET on Dec. 1.

The experts at the MJH Life Sciences COVID-19 Coalition will turn their attention to the question of coronavirus testing in a new free webinar.

At 6 p.m. ET on Dec. 1, the coalition will discuss the questions of diagnostic vs. antibody tests, at-home vs. point-of-care, sensitivity vs. specificity, and all other facets of testing past or current COVID-19 infection.

Sign up for the free webinar, Innovative Testing Strategies for COVID-19, here.

The coalition sprang from MJHs mission to improve quality of life through healthcare communications, education, and research, the MJH Life Sciences COVID-19 Coalition was formed to help keep healthcare professionals up-to-date and informed on the science and latest learnings on COVID-19.Leveraging relationships with top thought leaders across a variety of key specialties, the Coalition generates the most accurate, up-to-the-minute information on the pandemics ever-evolving impact on healthcare professionals and the patients they treat. For more information on the MJH Life Sciences COVID-19 Coalition, visitmjhlifesciences.com/covid19-coalition.The speakers at this webinar will be:

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MJH Life Sciences COVID-19 Coalition to hold innovative testing strategies webinar - Medical Economics

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced the appointment of Kathy Reape, M.D., and Laura Sepp-Lorenzino, Ph.D., to the companys board of directors.

Drs. Reape and Sepp-Lorenzino bring significant gene therapy translational and development expertise to our board, said RA Session II, President, Founder and CEO of Taysha Gene Therapies. Their combined gene therapy experience across preclinical and clinical development will be invaluable as we continue to advance our broad portfolio into the clinic. Across all levels of the organization, we are building a team that has the passion, experience and talent to execute on our mission of eradicating monogenic CNS disease.

Dr. Reape was most recently Chief Medical Officer at Spark Therapeutics where she oversaw clinical development, pharmacovigilance and medical affairs activities and was a key member of the team responsible for the development and commercialization of the first FDA-approved in vivo gene therapy, LUXTURNA, for an inherited retinal disease caused by mutations in both copies of the RPE65 gene. She also oversaw the development of Sparks pipeline of gene therapies addressing CNS disease, hemophilia, metabolic disorders and inherited retinal dystrophies. She has over 18 years of experience in the pharmaceutical industry in clinical research and development and has been involved with approximately two dozen product approvals including small molecules, biologics, biosimilars and therapeutic devices. She received both her undergraduate and M.D. degrees from the University of Pennsylvania and completed her internship and residency at the University of Florida and University of Medicine and Dentistry of New Jersey.

Taysha has built a deep pipeline of potentially transformative gene therapies for patients with life-threatening CNS diseases, said Dr. Reape. Many of the conditions that Taysha is addressing have no therapeutic alternatives, are associated with a poor quality of life and often result in a shortened life expectancy. It is important that we rapidly advance these gene therapies into the clinic to serve patients so desperately in need.

Dr. Sepp-Lorenzino is currently the Chief Scientific Officer at Intellia Therapeutics and has held several senior positions over her extensive career. Most recently, she was Vice President and Head of Nucleic Acid Therapies at Vertex Pharmaceuticals. She previously served as Alnylams Vice President and Entrepreneur-in-Residence, where she led the hepatic infectious disease strategies therapeutic area and was a key figure in partnering and in-licensing activities. She spent 14 years at Merck & Co., including as Executive Director and Department Head of the RNA therapeutics discovery biology unit. Dr. Sepp-Lorenzino received her degree in biochemistry at the University of Buenos Aires, Argentina and her M.S. and Ph.D. in biochemistry from New York University.

Joining the Taysha board is a truly exceptional opportunity to contribute to the development of multiple innovative gene therapies, commented Dr. Sepp-Lorenzino. Taysha is taking a leadership position in the industry by combining decades of gene therapy experience with a portfolio of programs that have the potential to address the underlying biology of various CNS disorders in order create an engine for potential new treatments.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the conduct or timing of our clinical trials and our research, development and regulatory plans for our product candidates, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Adds Industry-Leading Gene Therapy Executives to Board of Directors - Business Wire

TipRanks

Expectations of good news on the near horizon are buoying markets right now. Over the past month, both the S&P 500 and the NASDAQ are up 11% to new record highs.Investors are excited at the prospect of a COVID vaccine coming before the winter is out. And the electoral results, that Democrat Joe Biden will ascend to the Presidency while the Republicans will emerge strengthened in Congress, promise the avoidance of extremes typical of divided government. In short, investors are looking forward to return to normal environment over the next several months. And that has them seeking stocks that are primed for gains. Against this backdrop, Goldman Sachs analysts are pounding the table on three stocks in particular, noting that each could surge over 40% in the year ahead. After running both tickers through TipRanks database, we found out that the rest of the Street is also standing squarely in the bull camp.Codiack BioSciences (CDAK)As we have all learned from coronavirus pandemic, some new thing in medical science can make huge impact on our world. Codiack aims to turn that principle to good. This research-oriented pharmaceutical aims to turn exosome therapeutics into a whole new class of medicines. Exosomes are the degradation mechanism RNA, and can transfer genetic material around a body.And therein lies the potential. Codiack has developed a design platform for the engineering of exosome proteins capable of carrying and protecting drug molecules through cell walls. In effect, the proteins will mimic the pathways used by viruses but are non-viral, and are designed to carry a payload of therapeutic agents. If successful, exosome therapy offers doctors the ability to design a drug that will deliver specific agents to specific cells to fight specific disease.Codiack is involved in all aspects of exosome therapeutics, from design to manufacturing, and currently has an active pipeline of agents seven, in all in various stages of discovery, preclinical testing, and the beginnings of Phase 1 trials.In the biosciences, success or failure is all about that pipeline, and in its diverse, active pipeline of agents in a new sector of biotechnological pharmaceuticals, Codiack has a fine resource to attract investors. To get those investors, the company went public this past October, selling 5.5 million shares at an opening price of $14.10 per share.Among the healthcare name's fans is Goldman Sachs analyst Graig Suvannavejh. The analyst wrote, Biopharma industry interest in exosomes has long been high, but engineering them for a specific function and manufacturing at scale have both proven challenging. Among a field of multiple competitors, CDAK has made the most significant progress on both fronts, and as such we view their technology platform as best-in-class."Given share underperformance (-37%) since the IPO, we find risk/reward highly compelling at current levels, and with key 2021 data sets to provide potential de-risking and positive share inflection," the analyst concluded.Suvannavejh rates CDAK a Buy, and his $29 price target shows the extent of his confidence it implies a 222% upside for the coming year. (To watch Suvannavejhs track record, click here)Overall, Codiack has a Strong Buy from the analyst consensus 3 reviewers have put up Buy ratings in recent weeks. The stock is selling for $8.90, and its $24 average price target implies a 166% one-year upside potential. (See CDAK stock analysis on TipRanks)Arcutis Biotherapeutics (ARQT)Acrutis is a pioneering researcher in the treatment of dermatological disease. Arcutis is involved in discovering the next generation of dermatological treatments an important niche, especially when one realizes that one common ailment, psoriasis, has not seen an FDA approval for a novel treatment in over two decades.The company is leveraging recent advances in immunology and inflammation to find new approaches to skin treatment. The goal is to make it easier for patients and doctors together to manage conditions like psoriasis, alopecia, atopic dermatitis, seborrheic dermatitis, and vitiligo, to name just a few.The company's lead candidate, ARQ-151 (roflumilast cream), is about to enter a phase 3 trial for atopic dermatitis, and is in an advanced phase 3 stage in Plaque Psoriasis. Arcutis has recently issued an update on positive data from the Phase 2 trials of ARQ-151 in atopic dermatitis. The drug is a once-daily treatment, and has demonstrated significant patient relief from symptoms, especially itching and itching-related sleep problems. This is another stock in Suvannavejhs coverage universe. The Goldman analyst is impressed by developments in the companys pipeline work, noting: ARQT provided an update on the outcome of its end-of-Phase 2 meetings with the FDA, following their Phase 2a trial of ARQ-151 in atopic dermatitis (AtD). Feedback from regulators was broadly encouraging, in particular, acknowledging the robust long-term safety data being generated by ARQT for ARQ-151 in plaque psoriasisAccordingly, Suvannavejh rates ARQT a Buy, and sets a $36 price target that indicates room for 40% upside growth in 2021. (To watch Suvannavejhs track record, click here)Arcutis has 2 recent Buy reviews, making the consensus rating a Moderate Buy. The stocks average price target is $37, suggesting a 44% upside from current levels. (See ARQT stock analysis on TipRanks)Oak Street Health (OSH)With the last stock, we move from medical research to medical care. Specifically, Oak Street Health is a primary care clinic operator, and part of the Medicare Network. The company has operations and clinics in Illinois, Indiana, Michigan, Pennsylvania, and Ohio, along with New York, North Carolina, Rhode Island, Tennessee, and Texas. It has been in operation for eight years, and went public this past summer, holding the IPO in August.In the third quarter, the companys first as a publicly traded entity, OSH brought in $217.9 million in revenue. The revenue number was up 56% from the year-ago quarter. Earnings per share matched expectations, at 15 cents.The companys expansion proceeds apace, and in October, Oak Street entered New York by opening, in Brooklyn, its 70th location. A planned expansion in Texas, involving a partnership with Walmart, is also proceeding as planned, and Oak Street has opened its first Walmart Community Clinic the Dallas-Fort Worth area city of Carrollton.Robert Jones, covering this stock for Goldman, set a $74 price target to back his Buy rating. At currently levels, this target implies an upside of ~58% in the next 12 months. (To watch Jones track record, click here)Results suggest operations are still on track, with few incremental updates since the 2Q call, where management noted a resumption of center openings, (pivoted) marketing efforts, and in-person visits despite COVID. In 3Q, OSH opened 13 new centers and is on track for 73-75 by end of year The company maintained that it is continuing to operate at a high level in places with elevated COVID case counts like Chicago and Detroit, Jones noted.All in all, the Strong Buy analyst consensus rating OSH is based on 8 reviews, breaking down to 7 Buys and just a single Hold. The stock is selling for $46.94, and its $61.29 average price target suggests it has a ~31% upside for the coming year. (See OSH stock analysis on TipRanks)To find good ideas for healthcare stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Sarepta Therapeutics to Present at the Evercore ISI 3rd Annual HealthCONx Virtual Conference - Yahoo Finance

A recap of biopharma companies thankful for more cash this week.

SR One

SR One officially moved out from parent company GlaxoSmithKlines house, closing its first independent fund of $500 million. The funds will be used to build elite biotechnology companies developing innovative medicines that address significant unmet needs. As with many parents, GSK remains the largest contributor to the fund but with backing from others as well. Since its birth in 1985, SR One has invested over $680 million in biotech groups, most recently supporting Decibel Therapeutics Series D to support gene therapy hoping to restore hearing in children with congenital deafness due to a gene deficiency.

RESILIENCE

With big plans and big-name execs behind it, RESILIENCE launched with $800 million in capital to revolutionize the world of biopharma manufacturing. The new company dreams of democratizing access to new, cutting edge therapies and medicines by overcoming the hangups of traditional manufacturing. By providing high-tech, end-to-end manufacturing solutions, RESILIENCE will maximize production capacity and free up their partners to focus on important R&D. COVID-19 has exposed critical vulnerabilities in medical supply chains, and todays manufacturing cant keep up with scientific innovation, medical discovery and the need to rapidly produce and distribute critically important drugs at scale. We are committed to tackling these huge problems with a whole new business model, said Robert Nelsen, RESILIENCE founder, Chairman of the Board, and managing director at ARCH Venture Partners.

Olema Oncology

Originally pitched as a $100 million IPO, Olemas entrance onto the Nasdaq ballooned into $240.4 million in aggregate gross proceeds. Olemas big push is drug development for womens cancer, with an estrogen receptor antagonist as the star of their pipeline. Preclinical studies have supported advancing OP-1250 into its current Phase 1/2 development stages for estrogen receptor-positive, HER2-negative metastatic breast cancer. Initial data is anticipated in the second half of 2021. With these proceeds, Olema helps to prove the efficacy of their drug, offering a better option for breast cancer patients.

Atai Life Sciences

Dissatisfied by the limited efficacy and side effects of existing SSRI therapies, this Berlin start-up is attempting to make psychedelic drugs to treat mental health disorders. Thanks to a $125 million Series C round, Atai is now poised to fund pre-clinical and clinical development of ATAIs existing mental health programs. The company anticipates the proceeds to provide the runway to key clinical milestones - a Phase II data readout for arketamine in treatment resistant depression at Perception Neuroscience and ibogaine in opioid use disorder at DemeRx.

Medable

The COVID-19 pandemic has pushed multiple industries to find innovative ways to increase accessibility with shifts to innovative contactless, socially distanced and digital options. The clinical trial field is no different. Medable is a decentralized and remote trial platform connecting patients, sites and clinical trial teams. An infusion of $91 million in new funding brings Medables total capital raise to more than $136 million to support its critical role in enabling clinical trials amidst a global pandemic. The pandemic has made the world aware of the importance of clinical drug development, saidDr. Michelle Longmire, CEO and co-founder of Medable. We need transformative technologies that break down critical barriers to improve patient access, experience and outcomes. This new funding will enable Medable to continue our aggressive pursuit of new technologies that improve clinical trials to benefit all patients.

Pharvaris

Following a $66 million financing round last fall, Pharvaris raised the bar with an oversubscribed $80 million Series C this week. The funds will go towards their pipeline of compounds for the treatment of hereditary angioedema (HAE) and other bradykinin-B2-receptor-mediated indications. The goal is completing Phase I assessments in healthy volunteers by the end of the year to announce the data in 2021. "Our team is committed to developing and delivering differentiated products to patients the oversubscription of our Series C highlights broad enthusiasm for our vision for HAE and beyond," saidBerndt Modig, Chief Executive Officer and co-founder of Pharvaris.

Imago Biosciences

DoublingitsSeries Braiselast year,clinical-stage pharmaceutical companyImago Biosciencesraked in$80 millionthis weekto take theirlead candidate through a Phase III trial. Bomedemstat is a lysine specific demethylase 1 inhibitor. The current Phase III study is for the treatment of myeloproliferative neoplasms blood cancers that occur when the body makes too many white or red blood cells or platelets. Theres also a Phase IIb trial investigating the drug for the treatment of myelofibrosis and essential thrombocythemia. Imago has hopes for an IPO in 2021 to bring in enough cash to take the second trial to Phase III. Full enrollment for both is anticipated by the middle of next year.

Elevation Oncology

Elevation Oncology is raising the bar on cancer treatment with individualized, purpose-built precision medicines. Elevations core belief is that patients deserve the right clinical team and the right genomics test to match the best therapeutics to the unique genomic profile of each tumor. Their precision medicine approach has a lead program in Phase II study to evaluate the HER3 monoclonal antibody seribantumab for the treatment of patients with tumors harboring an NRG1 gene fusion. Funds from the $65 millionSeries Bfinancing round will be used to complete enrollment on the study.

AliveCor

Reinforcing their cross-company collaboration, OMRON Corporation led the round of investing in this weeks $65 million Series E funding. "OMRON is committed to developing life-saving cardiovascular health technologies in pursuit of our 'Going for Zero' vision to eliminate heart attacks and strokes. It is a lofty goal and, as we said when we evolved our vision, we cannot do it alone. That is why we seek like-minded partners, like AliveCor, to advance our initiatives and work together to save lives," saidIsao Ogino, president and CEO of OMRON Healthcare.

Umoja Biopharma

Launching with $53 million, Umoja takes on a new approach to T-cell engineering immunotherapy. Rather than removing T-cells from patients, modifying them in the lab and returning them to the patient, Umoja delivers the genetic payload to T-cells directly in the patient to attack and destroy hematologic and solid, organ-based tumors. This allows the cells to expand and respond in a more natural manner, making it not only a faster and more accessible approach, but also safer and more effective. The company is slated for its first clinical trial to initiate in 2021 targeting pediatric osteosarcoma.

Eluminex Biosciences Limited

China-based Eluminex is committed to the discovery and development of first-in-class and best-in-class therapies in the ophthalmic field. With a $50 million Series A in their pocket, Eluminex is building an innovative pipeline to address critical unmet medical needs in vision-threatening diseases for patients worldwide. The company is also eyeing future plans for establishing a global clinical and registration center in the San Francisco Bay Area.

Catamaran Bio

Seizing an opportunity to take a different approach to the development of allogeneic cell therapies, Catamaran launched this week with $42 million to get them off the ground. Off-the-shelf therapies are the next frontier of immune therapies. Instead of using CAR T-cells, Catamaran is utilizing CAR-NK (natural killer) cells to develop potent therapies capable of reaching solid tumors. The companys proprietary TAILWIND platform programs their therapy with NK cell-specific CAR architectures and potency-boosting switches to neutralize the hostile tumor microenvironment and enable efficacy against diverse cancer types. Catamarans therapies are made using healthy donor cells that are engineered and manufactured for off-the-shelf use, unlike current CAR-T therapies that take multiple weeks to manufacture.

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Biopharma Money on the Move: November 18-24 - BioSpace

iCrowdNewswire Nov 25, 20202:28 PM ET

The approval of KYMIRAH, YESCARTA, Alofisel and Zyntelgo has increased the interest of pharma stakeholders in cell therapies; further, owing to the technical challenges in this field, outsourcing manufacturing operations has become a necessity

Roots Analysis has announced the addition of Cell Therapy Manufacturing Market (3rd Edition), 2019 2030 report to its list of offerings.

Owing to various reasons, the demand for cell therapies is anticipated to increase over the coming years. Therefore, both therapy developers and contract service providers may need to strengthen their capabilities and expand available capacity. In this context, automation is expected to be a key enabler within the cell therapy manufacturing and contract services industry.

To order this 550+ page report, which features 160+ figures and 250+ tables, please visit this link

Key Market Insights

More than 160 organizations claim to be engaged in cell therapy manufacturing

The market landscape is dominated by industry players, representing more than 60% of the total number of stakeholders. Amongst these, over 55 are large or mid-sized firms (having more than 50 employees).

100+ players focused on T-cell and stem cell therapies

Most of these players are focused on manufacturing T-cell therapies, including CART, TCR or TILs. It is worth highlighting that more than 35 organizations claim to have necessary capabilities for the manufacturing of both types of therapies.

Presently, 70+ companies have commercial scale capacity

As majority of the cell therapy products are in clinical trials, the demand is high at this scale. However, it is worth noting that several players (~50%) have already developed commercial scale capacity for cell therapies.

Europe is currently considered a current hub for cell therapy production

More than 220 manufacturing facilities have been established by various players, worldwide; of these, 35% are in Europe, followed by those based in North America. Other emerging regions include Australia, China, Japan, Singapore, South Korea and Israel.

50+ facility expansions reported between 2015-2019

More than 85% of the expansions are related to setting up of new facilities across different regions. Maximum expansion activity was observed in the US and in certain countries within the Asia Pacific regions.

20+ companies offer automated solutions to cell therapy developers

Players that claim to offer consultancy services related to automation include (in alphabetical order) Berkeley Lights, Cesca Therapeutics, Ferrologix, FluDesign Sonics, GE Healthcare and Terumo BCT. Further, we identified players,namely (in alphabetical order) Fraunhofer Institute for Manufacturing Engineering and Automation IPA, Invetech, KMC Systems, Mayo Clinic Center for Regenerative Medicine and RoosterBio, that offer consultancy solutions related to automation.

Partnership activity has grown at an annualized rate of 16%, between 2014 and 2018

More than 200 agreements have been inked in the last 5 years; majority of these were focused on the supply of cell-based therapy products for clinical trials. Other popular types of collaboration models include manufacturing process development agreements (16%), services agreements (12%) and acquisitions (10%).

By 2030, developed geographies will capture over 60% of the market share

Asia Pacific is anticipated to capture the major share (~36%) of the market by 2030. It is also important to highlight that financial resources, technical expertise and established infrastructure is likely to drive cell therapy manufacturing market in Europe, which is estimated to grow at a CAGR of ~26%.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 11 billion (by 2030) financial opportunity within the cell therapy manufacturing market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom the manufacturing of cell therapies is largely being outsourced due to exorbitant costs associated with the setting-up of in-house expertise. The report includes detailed transcripts of discussions held with the following experts:

The research covers profiles of key players (industry and non-industry) that offer manufacturing services for cell-based therapies, featuring a company overview, information on manufacturing facilities, and recent collaborations.

For additional details, please visit

https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html or email [emailprotected]

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Contact:Gaurav Chaudhary+1 (415) 800 3415[emailprotected]

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Contact:Gaurav Chaudhary[emailprotected]

Roots AnalysisA430, 4th Floor,Bestech Business Towers, Sector 66, Mohali, India[emailprotected]+1 (415) 800 3415+44 (122) 391 1091Web: https://www.rootsanalysis.com/LinkedIn: https://in.linkedin.com/company/roots-analysisTwitter: https://twitter.com/RootsAnalysis

Keywords:cell therapy manufacturing, car-t manufacturing, car t cell therapy cost, gene therapy manufacturing, Roots Analysis, Pharma, Analysis, Research, Report,

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Growing at an annualized rate of over 16.5%, the cell therapy manufacturing market is estimated to reach close to USD 11 Billion by 2030, claims Roots...

Having demonstrated the ability to offer improved treatment related outcomes and also enhance the quality of lives of patients suffering from a diverse range of clinical conditions, the demand for cell therapies is anticipated to increase in the near future

Roots Analysis has announced the addition of Cell Therapy Manufacturing Market (2nd Edition), 2018 2030 report to its list of offerings.

Given the commercial success of multiple cell therapies, such as RECELL (Avita Medical) and YESCARTA (Gilead Sciences), and an evolving clinical pipeline, the opportunity for contract development and manufacturing organizations (CDMOs) is anticipated to grow significantly. Over the years, many service providers have begun automating their operations, in order to eliminate chances of human error during manufacturing.

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Over 145 companies / organizations are actively involved in manufacturing cell-based therapiesThe market landscape is currently dominated by the presence of industry players, which represent more than 55% of the total number of players. Amongst these, over 45 are large or mid-sized firms (having more than 50 employees). It is also worth noting that this field has witnessed the entry of several start-ups.

50+ organizations claim to possess commercial manufacturing capabilities for cell therapiesAs most of the cell therapy products are under clinical evaluation, majority of the manufacturing facilities currently have the capacity to support clinical scale production requirements. At the same time, it is worth noting that several players (over 35%) have already developed / are developing commercial scale capacity for cell therapies.

Europe has emerged as a key region for the production of cell therapies with more than 40% of manufacturing facilitiesGlobally, 195 facilities have been established by various players for the manufacturing of the cell therapies; of these, 41% are located in Europe, followed by those based in North America (38%). Other emerging regions include Australia, China, Japan, Singapore, South Korea and Israel.

Close to 15 companies are presently offering automated solutions to cell therapy developersOver the years, automation has emerged as a key enabler of cell therapy manufacturing. Players that claim to offer consultancy services related to automation include (in alphabetical order) these include (in alphabetical order) Berkeley Lights, Cesca Therapeutics, Ferrologix, FluDesign Sonics, GE Healthcare and Terumo BCT. Further, we identified five players, namely (in alphabetical order) Fraunhofer Institute for Manufacturing Engineering and Automation IPA, Invetech, KMC Systems, Mayo Clinic Center for Regenerative Medicine and RoosterBio, that offer consultancy solutions related to automation.

150+ partnerships were inked between 2014 and 2018Of these, 32% were observed to be focused on the supply of cell-based therapy products for clinical trials. Other popular types of collaboration models implemented in this domain include additional services agreements (24%), joint ventures (9%) and acquisitions (3%).

North America anticipated to capture 50% of the cell therapy manufacturing market by 2030Owing to high venture capital funding and drug development activity, North America is anticipated to capture close to 50% of the total market share by 2030. It is also important to highlight that financial resources, technical expertise and established infrastructure is likely to drive cell therapy manufacturing market in Europe to grow at a CAGR of over 18%.

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The USD 10+ billion (by 2030) financial opportunity within the cell therapy manufacturing market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom manufacturing of cell therapies is largely being outsourced due to the exorbitant costs associated with setting-up in-house facilities. The report includes detailed transcripts of discussions held with the following experts:

The research covers profiles of key players (industry and non-industry) that offer contract manufacturing services for cell-based therapies, featuring an overview of the company, information on its manufacturing facilities, and recent collaborations.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing-market-2nd-edition-2018-2030/209.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Cell Therapy Manufacturing Market is projected to reach close to USD 11 Billion by 2030, growing at an annualized rate of 14.9%, claims Roots Analysis...

Revolutionizing the biopharma manufacturing worldfor the futurewould requirean amalgamationof brilliant, experienced minds from the pharmaceutical world.First-of-its-kind RESILIENCE launched todayseemingly up for the taskwithsome big names andbig plans for U.S.-led biopharmaceutical innovation.

The idea behind the new biopharma manufacturing and technology company is democratizing access to thenew, cutting-edge medicines and therapies without thehangupsof traditional manufacturing. RESILIENCE is a unique ecosystem that can provide high-tech, end-to-end manufacturing and development solutions, freeing up their partners to focus on important R&D.

RESILIENCE is investing heavily in powerful technologies to manufacture the complex drugs of the future including gene and cell therapies, viral vectors, vaccines and proteins. Manufacturing is one of the biggest challenges faced by those attempting to pioneer new treatment modalities.A partnership with RESILIENCE would give dramatic increased production capacity, getting more drugs to more patients in need.

We created RESILIENCE to reimagine biopharmaceutical manufacturing through unprecedented investment in technology and a best-in-class team to execute our vision, said Robert Nelsen, RESILIENCE founder, Chairman of the Board, and managing director at ARCH Venture Partners. COVID-19 has exposed critical vulnerabilities in medical supply chains, and todays manufacturing cant keep up with scientific innovation, medical discovery, and the need to rapidly produce and distribute critically important drugs at scale. We are committed to tackling these huge problems with a whole new business model.

Scientific advancement has accelerated far beyond the traditionalmanufacturing capabilities. Perhaps now its more evident than ever, as potential COVID-19 treatments and vaccines are approved but in harshly limited supply.RESILIENCE is offering faster, safer and more flexible manufacturing for partners, from pre-clinical development to commercial supply.

Our aim with RESILIENCE is to improve manufacturing of breakthrough medicines so that they are more accessible to patients and to foster scientific innovation that makes new modalities of medicine possible, said RESILIENCE co-founder and CEO Rahul Singhvi, Sc.D. By providing improved process platforms and the highest quality manufacturing network, RESILIENCE has been designed to help society meet the challenges of tomorrow, whether thats the next pandemic or high demand for a great new medicine. I am incredibly proud to lead a team of extraordinary professionals with deep and diverse talent to build a more resilient future.

The new company has already raised over $800 million in capital following a Series B led by ARCH Venture Partners and 8VC. Spanning the U.S., Canada and partner nations, RESILIENCEboasts ahardy roster of C-suite players from multiple pharma giants. The founding exec team is made up of CEO Rahul Singhvi,former Operating Partner atFlagship Pioneering,COO ofTakeda Vaccines, andCEO ofNovavax; President, COO & CFO SandyMahatme, former CFO atSarepta Therapeutics andSVP Finance &Corporate Development atCelgene; andVice ChairmanPatrick Yang,former EVP atJuno Therapeutics andEVP & Global Head of Technical Operations atRoche/Genentech.The board of directors is made up of even more big names.

Hailing out of San Diego and Boston, RESILIENCE has secured over 750 thousand square feet of operating space that should be up and running over the next year.

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New Manufacturing Innovator Launches to Change the Way Medicine is Made - BioSpace

Redwood City, Calif.-based Guardant Health is taking Roche-owned Foundation Medicine to court over a patent dispute for their liquid biopsy technology, further heating up the rivalry between the two cancer detection companies.

In newly released documents, Guardant claims Foundation is currently infringing on up to seven patents which are part of Guardants intellectual property for its liquid biopsy test. Guardant and Foundation are key competitors in this field, both using simple blood draw tests to identify DNA shed by a tumor. These liquid biopsy tests, which rely on next-generation DNA sequencing to better identify signs of cancer, have become increasingly popular in cancer diagnostics.

Guardants suit, filed by Weil, Gotshal & Manges LLP and Farnan LLP, claims Foundations FoundationOne Liquid product is nearly identical to Guardants Guardant360. In the suit, Gardant asks the court for monetary relief or unspecified damages, damages for lost sales, an injunction against Foundation from infringing the patents and legal costs.

This isnt the first time Guardant and Foundation have faced each other in court over patent infringement. Previously filed motions have also alleged false advertising claims, and only last year, Foundation urged the U.S. Patent and Trademark Office to review claims around a patent.

Investors have spent billions of dollars to develop sophisticated liquid biopsy setups over the past five years, gearing research and development funding to companies like Guardant, Foundation, Grail, and San Franciscos Freenome, among several others. Using this technology, companies hope they will offer more precise and easier-to-use diagnostics for cancer as well as tests that can detect subtle molecular changes to identify disease prior to the emergence of symptoms.

Guardants Guardant360 test and Foundations FoundationOne Liquid test share similar offerings, with both tests assisting clinicians in the identification and tracking of cancers. These tests, which have been sold under the U.S. Food and Drug Administrations radar, also help doctors determine which treatments to switch to based on changes in a patients tumor.

In August, Guardant was granted the first FDA approval for a comprehensive genomic profiling test. Last month, a study published in Nature Medicine suggested the Guardant360 accelerated clinical trial enrollment and detected more actionable gene alterations compared with tissue biopsy in patients with advanced gastrointestinal cancer, suggesting Guardants liquid biopsy test could prove useful for expediting cancer research.

Also, the FDA has recently granted an Emergency Use Authorization to Guardants newly developed Guardant-19 test which can be used for the detection of SARS-CoV-2, the novel coronavirus responsible for COVID-19. According to the company, the Guardant-19 test can provide detection results within a single day.

The reverse transcriptase polymerase chain reaction next-generation sequencing Guardant-19 test is so far being offered to Guardant employees as well as select partner organizations through the Guardants CLIA-certified clinical laboratory. Additionally, Guardant is currently using the test to help in the safe reopening of Delaware State University.

The FDA has also recently granted an EUA for Agena Biosciences high-throughput, low-cost SARS-CoV-2 panel. Agenas product offerings are slightly different than Guardant and Foundation, however, as this company focuses primarily on a mass spectrometry-based platform for research into cancer profiling for solid tumors and liquid biopsies.

As details of the lawsuit emerge, its unclear how the outcomes will affect recent FDA approvals for Foundations platform. Just last month, the FDA granted approval for Foundations FoundationOne Liquid CDx for three new companion diagnostic indications, including advanced ovarian, breast and non-small cell lung cancer.

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Guardant Health and Foundation Medicine Face Off in Another Legal Battle - BioSpace