Stem Cell Clinic

MDA Awards Venture Philanthropy Funding of More Than $1M to AavantiBio to Develop Gene-Targeting Therapy for Friedreich’s Ataxia – P&T Community

By Gene Therapy Clinics

NEW YORK and GAINESVILLE, Fla., Jan. 13, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) and AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich's ataxia (FA), today announced the award of MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio's phase 2 clinical trial of a gene-replacement therapy for the disease.

MVP is MDA's drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular disorders. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics.

FA is a rare, hereditary disease that causes damage to parts of the spinal cord and brain, and there is currently no cure. AavantiBio was founded with the aim of developing an effective treatment for the disease and improving the lives of FA patients.

"With the approval of several first-time gene-targeting therapies for neuromuscular diseases over the past several years, including the first-ever gene-replacement therapy for spinal muscular atrophy, our community should have much to hope for as more and more therapies continue to be developed," says Lynn O'Connor Vos, MDA's president and CEO. "MDA is thrilled to be a part of the quest to help further develop the first gene-targeting therapy for the treatment of Friedreich's ataxia. By partnering with AavantiBio, together we can address the unmet need faced by patients who live with this genetic disease, for which there are still no treatments and no cures."

Co-founders Manuela Corti, PT, PhD, assistant professor of Pediatrics at the University of Florida, Gainesville, and Barry Byrne, MD, PhD, associate chair of Pediatrics and director of the Powell Gene Therapy Center at UF, started working with the FA community five years ago and are thrilled about their new partnership with MDA.

"This is a great opportunity for AavantiBio, and we're thankful to the MDA for their generous contribution," Dr. Corti says. "We hope to strengthen our collaboration as we work together on this project."

Dr. Corti and Dr. Byrne aim to include both adults and children who have FA in their clinical trials, paving the way for new solutions.

"We're delighted to take the next step in fulfilling our commitment to patients and families living with FA based on this investment from MDA," Dr. Byrne says. "We're looking forward to initiating screening for the first clinical study and a pivotal study in FA before the end of the year. The endorsement and investment from the MDA will be key to our programmatic growth."

MDA's investment will help accelerate AavantiBio's mission and begin production of the clinical gene vector for its therapy program. Clinical trials are expected to begin in 2020.

Dr. Corti was previously awarded an MDA research grant to develop and test a gene-replacement therapy in a mouse model of FA. With the current funding, AavantiBio will sponsor a study at the University of Florida led by Sub Subramony, MD, professor of Neurology. The clinical trial will assess changes in neurological and cardiac function in patients with FA treated with both intravenous (systemic) and intrathecal (into-the-spine) injections of the company's gene-replacement therapy for the mutated FXN gene.

About Friedreich's ataxia

FA is a mitochondrial disease. Mutations in the frataxin gene (FXN) lead to decreased production of the frataxin protein, resulting in diminished energy production in cells, including those of the nervous system and heart. FA's major neurological symptoms include muscle weakness and ataxia, or a loss of balance and coordination. FA mostly affects the spinal cord and the peripheral nervesthat connect the spinal cord to the body's muscles and sensory organs, but it can also affect the cerebellum (causing ataxia) and heart. The prevalence of FA has been estimated at 1 in every 50,000 individuals worldwide. Symptoms typically begin between the ages of 5 and 15 years, and the rate of progression varies from person to person. There currently are no effective cures or treatments for FA.

About AavantiBio

Founded in 2017 and based inGainesville, Fla., AavantiBio is a biotechnology companyfounded on the vision of creating the first effective treatment for FA. The company's gene-replacement therapy approach uses an adeno-associated virus (AAV) vector to deliver a functional copy of the FXN gene to a patient's cells.

The co-founders bring more than 30 years of research experience in the field of neuromuscular disease, including their current work focusing on FA. Dr. Corti has more than 10 years of research experience in gene therapy approaches for the treatment of Duchenne muscular dystrophy and Pompe disease. Dr. Byrne has made significant contributions to theunderstanding and treatment of Pompe disease. He was previously awarded nearly $2 million in MDA funding to conduct foundational research in developing and testing AAV vectors in animal models of muscular dystrophy, and he was the first to show that AAV vectors are able to effectively express therapeutic genes in striated muscle cells.

About the Muscular Dystrophy Association

MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to approved, life-changing therapies across multiple neuromuscular diseases. We support the largest network of multidisciplinary clinics providing best-in-class care at more than 150 of the nation's top medical institutions, and each year thousands of children and young adults learn vital life skills and gain independence at MDA Summer Camp and through recreational programs. For more information visitmda.org.

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SOURCE Muscular Dystrophy Association

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MDA Awards Venture Philanthropy Funding of More Than $1M to AavantiBio to Develop Gene-Targeting Therapy for Friedreich's Ataxia - P&T Community

Oncocyte Announces the Commercial Availability of DetermaRx, the First Test for Chemotherapy Benefit Prediction in Patients with Early Stage Non-Small…

By Gene Therapy Clinics

Leonard Cancer Institute at Mission Hospital, California and Florida Precision Oncology sign up as early access sites for the test

Canadian regulatory approval received allowing for path to reimbursement

IRVINE, Calif., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Oncocyte Corporation (NYSE American: OCX), a molecular diagnostics company with a mission to provide actionable answers at critical decision points across the lung cancer care continuum, today announced that DetermaRx, formerly known as the Razor treatment stratification test, is now commercially available in the United States. Additionally, Oncocyte has received regulatory approval in Canada to begin distribution of DetermaRx in that country. DetermaRx enables the identification of early-stage lung cancer patients who may benefit from adjuvant chemotherapy post surgical resection. In a clinical study, high-risk patients identified by this test post-surgery and treated with adjuvant chemotherapy had a significant increase in survival rates.

Florida Precision Oncology and the Leonard Cancer Institute at Mission Hospital in Mission Viejo, California have signed up for early access to the test. The Leonard Cancer Institute at Mission Hospital is a brand new, state of the art regional cancer center and a part of the Providence St. Joseph Health network which consists of 119,000-plus caregivers and employees, serving 51 hospitals and more than 800 clinics delivering a comprehensive range of health and social services across Alaska, California, Montana, New Mexico, Oregon, Texas and Washington. Florida Precision Oncology, with locations in Aventura, Miramar and Boca Raton, is focused on delivering multidisciplinary cancer care in the community setting where most cancer is treated.

We are thrilled to officially transform Oncocyte into a commercial-stage company as we make DetermaRx available to lung cancer patients in the U.S., and in the near future, Canada, who are in need of additional clarity when making treatment decisions after surgery, said Ron Andrews, Chief Executive Officer of Oncocyte. Under the current standard of care, approximately 30%-50% of stage I IIA patients who have undergone surgery to remove lung tumors recur and die within five years of surgery. This is unacceptable. We believe DetermaRx, which has been extensively validated and published in top tier peer-reviewed publications, will address this critically underserved treatment decision point, helping physicians and patients make the right treatment decisions at the right time. We are also very pleased that Medicare, which covers ~70% of eligible patients, has proposed positive coverage for this test. This will promote broad access for patients who may stand to benefit from DetermaRx.

Dr. Samer Kanaan, medical director for the lung program at Mission Hospitals Leonard Cancer Institute added, Mission Hospital is honored to have been invited to serve as one of Oncocytes early access partners. This new diagnostic tool will allow us to further enhance our comprehensive lung cancer treatment program and optimize post-surgical treatment decisions. This test serves an important unmet medical need, and I look forward to making it available to patients across the Mission Hospital and Providence St. Joseph networks.

Dr. Edgardo S. Santos, Founding Partner of Florida Precision Oncology, further commented, There is no question that DetermaRx addresses an unmet need in thoracic oncology that we have had for decades. Lung cancer has an incidence of approximately of 225,000 patients per year; if we focus on those adenocarcinoma patients who have stage I and IIA disease, we are talking about 40,000 patients per year with a poor or suboptimal 5-year survival rate. If I have a tool such as DetermaRx which can identify those patients with early pathological stage (supposedly cured) carrying a high-risk for recurrence (based on their genomic profile), there is no question in mind that it will significantly impact the survival of my patients. We also must keep in mind that with the advent of lung cancer screening, we will be able to identify more patients at early stages. Hence, we must be ready to have a discussion regarding therapy post-operatively. Besides poor features from the tumor that we have used for years to decide for or against postoperative chemotherapy, now DetermaRx brings molecular analysis on board which from my standpoint is a kind of personalized management. For example, not all stages IA are the same; some carry higher risk for recurrence than others. We must target them.

Story continues

In bringing DetermaRx to market, Oncocyte will initially prioritize those cancer centers and physicians who manage the highest number of early stage lung cancer patients. Starting the first quarter of 2020, the Company will deploy an experienced salesforce which has a proven track record driving market leadership of novel high-volume molecular diagnostics.

About DetermaRx

DetermaRx is a molecular diagnostic test that enables the identification of early-stage lung cancer patients who may benefit from chemotherapy following surgery, allowing them to be treated when their cancer may be more responsive to adjuvant chemotherapy. The test utilizes a gene expression analysis of 14 specific genes from a patients tumor and a proprietary algorithm to stratify early stage NSCLC patients into one of two groups, one that may benefit from chemotherapy because of high risk of recurrence, and another that may avoid chemotherapy because of low risk of recurrence. DetermaRx is extensively validated and published with independent, blinded global studies in over 1,500 patients and seven publications in prestigious journals including the Lancet and JAMA.

About Oncocyte Corporation

Oncocyte is a molecular diagnostics company whose mission is to provide actionable answers at critical decision points across the lung cancer care continuum, with the goal of improving patient outcomes by accelerating and optimizing diagnosis and treatment. The Company is currently preparing to launch DetermaRx, a treatment stratification test that enables the identification of early-stage lung cancer patients at high risk for recurrence post-resection, allowing them to be treated when their cancer may be more responsive to adjuvant chemotherapy. DetermaDx, the companys liquid biopsy test in development, utilizes a proprietary immune system interrogation approach to clarify if a patients lung nodules are benign, which may enable them to avoid potentially risky invasive diagnostic procedures.

DetermaDx and DetermaRx are trademarks of Oncocyte Corporation.

Oncocyte Forward Looking Statements

Any statements that are not historical fact (including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates and similar expressions) are forward-looking statements. These statements include those pertaining to the time to complete and the results of the Companys ongoing Clinical Validation study for DetermaDx, implementation and results of research, development, clinical trials and studies, commercialization plans, future financial and/or operating results, and future opportunities for Oncocyte, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential diagnostic tests or products, uncertainty in the results of clinical trials or regulatory approvals, the capacity of our third-party supplied blood sample analytic system to provide consistent and precise analytic results on a commercial scale, the need and ability to obtain future capital, maintenance of intellectual property rights, and the need to obtain third party reimbursement for patients use of any diagnostic tests we commercialize. Actual results may differ materially from the results anticipated in these forward-looking statements and accordingly such statements should be evaluated together with the many uncertainties that affect the business of Oncocyte, particularly those mentioned in the Risk Factors and other cautionary statements found in Oncocytes Securities and Exchange Commission filings, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Oncocyte undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Investor ContactBob YedidLifeSci Advisors, LLC646-597-6989bob@lifesciadvisors.com

Media ContactCait Williamson, Ph.D.LifeSci Public Relations, LLC646-751-4366cait@lifescicomms.com

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Oncocyte Announces the Commercial Availability of DetermaRx, the First Test for Chemotherapy Benefit Prediction in Patients with Early Stage Non-Small...

Scientists Have Built The First-Ever Robots Constructed Entirely Out of Living Cells – ScienceAlert

By Embryonic Stem Cells

In another lifetime, if they had been allowed to follow their natural development, the stem cells taken from embryonic frogs would have turned into skin and heart tissue within living, breathing animals.

Instead, in configurations designed by algorithms and constructed by humans, those cells have been assembled into something new: the first-ever robots constructed entirely out of living cells.

The creators have called them xenobots; tiny, submillimetre-sized blobs containing between 500 and 1,000 cells that have been able to scoot across a petri dish, self-organise, and even transport minute payloads. These xenobots are unlike any living organism or organ we've encountered or created to date.

The possibilities for custom living machines designed for a variety of purposes, from targeted drug delivery to environmental remediation, are pretty mind-blowing.

"These are novel living machines," said computer scientist and roboticist Joshua Bongard of the University of Vermont.

"They're neither a traditional robot nor a known species of animal. It's a new class of artifact: a living, programmable organism."

Designing the xenobots required the use of a supercomputer, and an algorithm that could virtually put together a few hundred frog heart and skin cells in different configurations (somewhat like LEGO bricks), and simulate the results.

The scientists would assign a desired outcome - such as locomotion - and the algorithm would create candidate designs aimed to produce that outcome. Thousands of configurations of cells were designed by the algorithm, with varying levels of success.

The least successful configurations of cells were tossed out, and the most successful were kept and refined, until they were about as good as they were going to get.

Then, the team selected the most promising designs to physically build out of cells harvested from embryonic African clawed frogs (Xenopus laevis).This was painstaking work, using microscopic forceps and an electrode.

When they were finally put together, the configurations were actually able to move around, as per the simulations. The skin cells act as a sort of scaffolding to hold everything together, while the contractions of the heart cell muscles are put to work to propel the xenobots.

These machines moved about an aqueous environment for up to a week without the need for additional nutrients, powered by their own 'pre-loaded' energy stores in the form of lipids and proteins.

One design had a hole through the middle in an attempt to reduce drag. This hole could be exapted into a pouch for transporting objects, the team found; as they evolved the design, they incorporated the pouch and transported an object in a simulation.

(Kriegman et al., PNAS, 2019)

The xenobots moved objects around in the real world, too. When their environment was scattered with particulates, the xenobots spontaneously worked together, moving in a circular motion to push the particulates into one spot.

It's fascinating work. According to the researchers, their efforts can provide invaluable insight into how cells communicate and work together.

"You look at the cells we've been building our xenobots with, and, genomically, they're frogs. It's 100 percent frog DNA - but these are not frogs. Then you ask, well, what else are these cells capable of building?" said biologist Michael Levin of Tufts University.

"As we've shown, these frog cells can be coaxed to make interesting living forms that are completely different from what their default anatomy would be."

Although the team calls them 'living', that may well depend on how you define living creatures. These xenobots are not able to evolve on their own, there are no reproductive organs, and they are unable to multiply.

When the cells run out of nutrients, the xenobots simply become a small clump of dead cells. (This also means they are biodegradable, which gives them another advantage over metal and plastic robots.)

Although the current state of the xenobots is relatively harmless, there is the potential for future work to incorporate nervous system cells, or develop them into bioweapons. As this field of research grows, regulation and ethics guidelines will need to be written, applied and adhered to.

But there is plenty of potential good, too.

"We can imagine many useful applications of these living robots that other machines can't do," Levin said, "like searching out nasty compounds or radioactive contamination, gathering microplastic in the oceans, travelling in arteries to scrape out plaque."

The research has been published in PNAS, and the team has made their source code freely available on Github.

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Scientists Have Built The First-Ever Robots Constructed Entirely Out of Living Cells - ScienceAlert

Koepka Ready to Get Back to Work After Nearly 3 Months Off – The New York Times

By Stem Cell Treatment

HONOLULU Brooks Koepka hasn't been the same since he tied for third in the FedEx Cup final in August, and hasn't felt entirely healthy since last March.

He wouldn't rate himself full strength now.

Koepka returns to competition this week in the Abu Dhabi HSBC Championship, his first tournament since he reinjured his left knee in South Korea at the CJ Cup. He said Tuesday his left knee doesn't feel the same as my right.

It probably won't for a while, but it does feel stable, Koepka said. Leaving Korea and all the way up to about a month ago, it just didn't feel stable. It felt like it could either way. It could go left, out, back.

Koepka says he's had issues since March and just dealt with them. He still managed to win the PGA Championship for the second straight year and pick up his first World Golf Championship. During his short offseason, Koepka had stem cell treatment on his left knee because the patella tendon was partially torn.

Then, he was walking off a tee when he slipped on a wet piece of cement, went to brace himself from falling and reinjured the knee. He said his knee cap moved into the fat pad, which he described as excruciating.

He had physical therapy in San Diego for most of December and says he started hitting balls right before Christmas. Koepka said he wouldn't have flown to the United Arab Emirates if he didn't feel healthy, and that his speed and everything else about his game were the same as before he was hurt at the CJ Cup.

"From that moment on, after a couple days of hitting balls and not feeling pain, it was like, 'OK, I could get back here and do this and finally play,'" he said.

A NAME FROM THE PAST

The first player of note from an emerging golf nation is not always the best one. As Li Haotong of China was making his debut at the Presidents Cup, Guan Tianlang was preparing to qualify for the PGA Tour Series-China.

Guan, who won the Asia-Pacific Amateur and then made the cut at the Masters and Zurich Classic when he was 14, made it through. Despite closing with a 79, he tied for 10th last week to earn full status for the season in China.

Guan is a sophomore at Arizona and is still an amateur.

"I think I will turn pro soon," he said, adding there was a "good chance" he would play China's opening tournament. But I still need some time to think about everything. I might also balance school and play professional events.

Guan says he expected some highs and lows after his Masters performance. "I think that I'm trending in the right direction now," he said.

G-MAC STYLE

Graeme McDowell can add his name to the list of players who went searching for distance and lost sight of their game.

McDowell was enthusiastic at the Sony Open, and that was before he closed with rounds of 67-64 for the best weekend score at wet Waialae. It gave him a tie for fourth, his best finish since winning in the Dominican Republic last spring.

He attributes that to getting back to his normal flight with irons.

McDowell started working in August with Kevin Kirk, also the swing coach for Patrick Reed.

"The first thing I said was I've got to start hitting it lower again," McDowell said. I'm not playing the wind anymore. That was my bread and butter.

He still works with Pete Cowen, but McDowell said their schedules didn't mesh as much with McDowell out of the top 50 and not playing in all the majors or World Golf Championships.

Where did he lose his way?

"It probably came from trying to launch the drive too high in a little search for a wee bit of distance," he said. I got an iron in my hand, it was vertical. That's not me. I need to hit the ball back down to a good window.

For two weeks in the Hawaii wind, he said he was back to G-Mac style.

SHORT ROAD, LONG SHOT

For the host country of the Tokyo Olympics, Hideki Matsuyama (No. 21) and Shugo Imahira (No. 33) are the leading candidates to represent Japan.

Next in line is Ryo Ishikawa at No. 83. Ishikawa showed signs of getting back to form last year when he won three times on the Japan Golf Tour, his first titles since 2016 and his biggest year in Japan since 2010. The problem facing him now is a schedule.

Ishikawa is part of a solid field this week in the Singapore Open, co-sanctioned by Japan. Among those playing are Justin Rose, Henrik Stenson and Matt Kuchar. Otherwise, the Japan Golf Tour season doesn't start until a week after the Masters. That would leave Ishikawa only six events on his home tour before the cutoff for the Olympics.

Ishikawa is looking for sponsor exemptions, with his eye on the Genesis Invitational at Riviera and perhaps the Arnold Palmer Invitational at Bay Hill. He made his U.S. debut at Riviera in 2009 when he was 17.

POLICY BOARD

Six new players were selected for the 16-member Player Advisory Council this year, the group tasked with listening to players and conveying their thoughts to the four members of the PGA Tour's policy board. The newcomers include Russell Knox and Harry Higgs.

More telling was who was put up for election as PAC chairman, who next year would join the policy board Justin Thomas, Charley Hoffman and Peter Malnati. That assures a streak that probably should have ended long ago. No foreign-born player has ever been on the policy board.

Last year, 48 of the 125 players who qualified for the FedEx Cup postseason were international players, including 12 of the 30 who reached the Tour Championship.

The others on the PAC: Ryan Armour, Paul Casey, Zach Johnson, Anirban Lahiri, Rory McIlroy, Jon Rahm and Harold Varner III all served last year. Also new for this year are David Hearn, Billy Horschel, Ryan Palmer and Kevin Streelman.

Missing from the list is Bryson DeChambeau. Last year at The Northern Trust, when he was criticized for how long it took him to play a shot, DeChambeau said, I've asked to be on the PAC committee for three years, and it takes time to get on there.

Higgs is a rookie, although the PAC is evenly populated by players young and old, high and low in the FedEx Cup.

The election for PAC Chairman ends on Feb. 7.

DIVOTS

Collin Morikawa's three-putt from 4 feet on the final hole of the Sony Open took him from a potential four-way tie for ninth to a seven-way tie for 21st. Perhaps more than a difference of $108,900 if he had made the short birdie, Morikawa would have moved to No. 50 in the world. He's No. 53. Morikawa needs to be in the top 50 a week before the Masters to get an invitation. ... Inbee Park is returning to Australia for the first time in six years. Park, voted the LPGA Tour's best player of the last decade, plans to play the Vic Open and the Australian Ladies Masters in February. ... The last seven rounds on the PGA Tour in Hawaii were played under lift, clean and place rules.

STAT OF THE WEEK

Lanto Griffin was 7-under par on the 18th hole of the Sony Open. He played the other 68 holes in 1 under.

FINAL WORD

I think we know all four tournaments that I'm looking forward to. I think that's pretty obvious. Brooks Koepka. Four of his seven victories since 2017 have been majors.

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Koepka Ready to Get Back to Work After Nearly 3 Months Off - The New York Times

Astellas teams with Adaptimmune to create CAR-T, TCR therapies – FierceBiotech

By Stem Cell Treatment

Astellas has teamed up with Adaptimmune to develop allogeneic chimeric antigen receptor T-cell (CAR-T) and T-cell receptor (TCR) therapies. The agreement sees Astellas pay $50 million (45 million) upfront and commit to many times as much in milestones to work with Adaptimmune to identify targets and develop cell therapies against them.

Through the collaboration, Adaptimmune will use its target identification and validation capabilities to help pick out up to three targets that are outside of the scope of its other projects. Astellas and Adaptimmune will then develop treatments against the targets, respectively contributing universal donor cell and cell therapy technologies.

This new collaboration may encompass both CAR-T and TCR t-cell approaches, Helen Tayton-Martin, Adaptimmunes chief business officer, said in a statement. It brings together highly complementary skills and expertise across the two organizations, and will enable the accelerated development of new, off-the-shelf T-cell therapy products for people with cancer.Adaptimmune will pocket $50 million and $7.5 million a year in research funding, plus development and sales milestones that could bring the total deal value up toward $900 million.

The Art of Recognizing Clinical Supply Risk Factors and Applying Proactive Measures to Avoid Study Delays and Disruptions

No two studies are the same and each clinical supply project carries unique risks. But what characteristics are most likely to raise a flag that issues are ahead? Are there certain types of clinical sponsors and studies that are at greater risk of experiencing supply challenges? And how do clinical sponsors know what is important to focus on and what is not? Join us for this webinar as we attempt to answer these questions.

Astellas will fund work up to the end of phase 1. Beyond that, Astellas and Adaptimmune will decide whether to develop and commercialize an asset together or let one company take it forward alone. If only one company takes a candidate forward, the other partner will receive milestones and royalties. Astellas could receive more than $500 million if it opts out of developing any of the therapies.

The deal tightens Adaptimmunes ties to Universal Cells, a stem cell biotech Astellas bought in 2018. Astellas entered into the deal with Adaptimmune through its Universal Cells subsidiary, which has worked with the transatlantic cell therapy specialist on gene-edited induced pluripotent stem cell lines since 2015.

Landing the Astellas deal also provided another fillip to Adaptimmunes share price, which rose 200% Monday on the back of news of partial responses in patients who received its T-cell therapy. The sharp stock rise provided a timely boost for Adaptimmune.

In November, the cell therapy biotech warned investors of substantial doubts about its ability to continue as a going concern for more than one year. With its share price now up at levels last seen in the summer, Adaptimmune is better placed to raise the money needed to lessen those doubts.

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Astellas teams with Adaptimmune to create CAR-T, TCR therapies - FierceBiotech

FROM THE LABS: There is a new player in adult bone healing – Baylor College of Medicine News

By Stem Cell Treatment

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized. Bone stem cells have been found both in the bone marrow and in the outer layer of tissue, called periosteum, that envelopes the bone. Of the two, periosteal stem cells are the least understood.

Having a better understanding of how adult bones heal could reveal new ways of repair fractures faster and help find novel treatments for osteoporosis. Dr. Dongsu Park and his colleagues at Baylor College of Medicine investigate adult bone healing and recently uncovered a new mechanism that has potential therapeutic applications.

Previous studies have shown that bone marrow and periosteal stem cells, although they share many characteristics, also have unique functions and specific regulatory mechanisms, said Park, who is assistant professor of molecular and human genetics and of pathology and immunology at Baylor.

It is known that these two types of bone stem cells comprise a heterogeneous population that can contribute to bone thickness, shaping and fracture repair, but scientists had not been able to distinguish between different subtypes of bone stem cells and study how their different functions are regulated.

In the current study, Park and his colleagues developed a method to identify different subpopulations of periosteal stem cells, define their contribution to bone fracture repair in live mouse models and identify specific factors that regulate their migration and proliferation under physiological conditions.

The researchers discovered specific markers for periosteal stem cells in mice. The markers identified a distinct subset of stem cells that showed to be a part of life-long adult bone regeneration.

We also found that periosteal stem cells respond to mechanical injury by engaging in bone healing, Park said. They are important for healing bone fractures in the adult mice and, interestingly, they contribute more to bone regeneration than bone marrow stem cells do.

In addition, the researchers found that periosteal stem cells also respond to inflammatory molecules called chemokines, which are usually produced during bone injury. In particular, they responded to chemokine CCL5.

Periosteal stem cells have receptors molecules on their cell surface called CCR5 that bind to CCL5, which sends a signal to the cells to migrate toward the injured bone and repair it. Deleting the CCL5 or the CCR5 gene in mouse models resulted in marked defects or delayed healing. When the researchers supplied CCL5 to CCL5-deficient mice, bone healing was accelerated.

The findings suggested potential therapeutic applications. For instance, in individuals with diabetes or osteoporosis in which bone healing is slow and may lead to other complications resulting from limited mobility, accelerating bone healing may reduce hospital stay and improve prognosis.

Our findings contribute to a better understanding of how adult bones heal. We think this is one of the first studies to show that bone stem cells are heterogeneous, and that different subtypes have unique properties regulated by specific mechanisms, Park said. We have identified markers that enable us to tell bone stem cell subtypes apart and study what each subtype contributes to bone health. Understanding how bone stem cell functions are regulated offers the possibility to develop novel therapeutic strategies to treat adult bone injuries.

Find all the details of this study in the journal journal Cell Stem Cell.

Other contributors to this work include Laura C. Ortinau, Hamilton Wang, Kevin Lei, Lorenzo Deveza, Youngjae Jeong, Yannis Hara, Ingo Grafe, Scott Rosenfeld, Dongjun Lee, Brendan Lee and David T. Scadden. The authors are affiliated with one of the following institutions: Baylor College of Medicine, Texas Childrens Hospital, Pusan National University School of Medicine and Harvard University.

This study was supported by the Bone Disease Program of Texas Award and The CarolineWiess Law Fund Award, the NIAMS of the National Institutes of Health under award numbers 1K01AR061434 and 1R01AR072018 and U54 AR068069 and the NIDDK of the NIH.

By Ana Mara Rodrguez, Ph.D.

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FROM THE LABS: There is a new player in adult bone healing - Baylor College of Medicine News

Allogeneic Stem Cells Market Expected to Grow with a CAGR of 12% Due to New Product Approvals, 2020-2024 – ResearchAndMarkets.com – Business Wire

By Stem Cell Treatment

DUBLIN--(BUSINESS WIRE)--The "Allogeneic Stem Cells Market by Application and Geography - Forecast and Analysis 2020-2024" report has been added to ResearchAndMarkets.com's offering.

Global Allogeneic Stem Cells Market: About this market

The allogeneic stem cells market analysis considers sales from regenerative therapy and drug discovery and development applications. Our study also finds the sales of allogeneic stem cells in Asia, Europe, North America, and ROW. In 2019, the regenerative therapy segment had a significant market share, and this trend is expected to continue over the forecast period. Factors such as functional restoration of tissues will play a significant role in the regenerative therapy segment to maintain its market position. Also, our global allogeneic stem cells market report looks at factors such as new product approvals, increasing strategic alliances in the field of regenerative medicines, and investments in the field of regenerative medicines. However, stringent regulations, high cost of allogeneic stem cell therapies, and serious complications associated with stem cell therapies may hamper the growth of the allogeneic stem cell industry over the forecast period.

Global Allogeneic Stem Cells Market: Overview

New product approvals

The new product approvals and special drug designations are anticipated to boost the growth of the market. Based on the application, the allogeneic stem cells market has been segmented into regenerative therapy and drug discovery and development. Manufacturers are increasingly emphasizing innovations and improvisation in the development of regenerative therapies. Many of the regenerative therapeutic candidates have obtained approval for clinical trials in the US, Europe, and APAC due to the efficacy of allogeneic stem cell therapeutics. This is encouraging market players to launch new product lines to stimulate the overall product demand for stem or regenerative therapy using allogeneic stem cell therapeutics and provide better options for their customers. Thus, new product approvals will lead to the expansion of the global allogeneic stem cells market at a CAGR of over 12% during the forecast period.

Special drug designations

Research in the field of stem cell focuses mainly on developing new treatments for deadly diseases, which have negligible treatment using traditional treatment options. Thus, therapeutic candidates, which are currently under development, have been awarded special drug designations by regulatory bodies considering their proven efficacy. Many drugs received designations such as the breakthrough drug designation and the orphan drug designation from regulatory bodies such as the US FDA and the EMA. Drug designations enhance the research and enable drugs to reach the market and provides strong incentives, which in turn, encourages vendors to expedite R&D on novel therapies such as allogeneic stem cell therapy. This development is expected to have a positive impact on the overall market growth.

Key Topics Covered:

PART 01: EXECUTIVE SUMMARY

PART 02: SCOPE OF THE REPORT

PART 03: MARKET LANDSCAPE

PART 04: MARKET SIZING

PART 05: FIVE FORCES ANALYSIS

PART 06: MARKET SEGMENTATION BY APPLICATION

PART 07: CUSTOMER LANDSCAPE

PART 08: GEOGRAPHIC LANDSCAPE

PART 09: DECISION FRAMEWORK

PART 10: DRIVERS AND CHALLENGES

PART 11: MARKET TRENDS

PART 12: VENDOR LANDSCAPE

PART 13: VENDOR ANALYSIS

For more information about this report visit https://www.researchandmarkets.com/r/phsh0a

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Allogeneic Stem Cells Market Expected to Grow with a CAGR of 12% Due to New Product Approvals, 2020-2024 - ResearchAndMarkets.com - Business Wire

Global Stem Cell Market Is Projected to Grow at a CAGR of 11.9% During the Forecast Period – Press Release – Digital Journal

By Stem Cell Treatment

A recent report published by Infinium Global Research on stem cell market provides an in-depth analysis of segments and sub-segments in the global as well as regional stem cell market

This press release was orginally distributed by SBWire

Pune, India -- (SBWIRE) -- 01/13/2020 -- Infinium Global Research has recently published a trending report on the Global Stem Cell Market (Type - Adult Stem Cell, Induced Pluripotent Stem Cell, Human Embryonic Stem Cell, and Other Types; Application - Regenerative Medicine, Neurology, Oncology, Cardiology, and Other Application; End User - Therapeutics Companies, Cell and Tissue Banks, Tools and Reagents Companies, and Service Companies): Global Industry Analysis, Trends, Size, Share and Forecasts to 2025. A stem cell has a unique ability to develop into specialized cell types in the body depending on the source of stem cells and their biological plasticity. Owing to their ability to transform into the new cell, they can be used to replace cells and tissues that have been damaged or lost due to disease. Stem cell therapy is a unique technique that can be used in tissue regeneration, cardiovascular disease treatment, brain disease treatment, and blood cell regeneration treatment. According to the report, the global stem cell market is projected to grow at a CAGR of 11.9% over the forecast period of 2019-2025.

For More Details Get FREE Sample Pages of this Premium Global Report@ https://www.infiniumglobalresearch.com/reports/sample-request/13006

Research Projects are Set to Offer New Opportunities for the Growth of the Stem Cell Market

A multi-disciplinary research team, based at the Wellcome-MRC Cambridge Stem Cell Institute (University of Cambridge), studied young and old rat brains to understand the impact of age-related brain stiffening on the function of oligodendrocyte progenitor cells (OPCs).

"MS is relentless, painful, and disabling, and treatments that can slow and prevent the accumulation of disability over time are desperately needed. The Cambridge team's discoveries on how brain stem cells age and how this process might be reversed have important implications for future treatment because it gives us a new target to address issues associated with aging and MS, including how to potentially regain lost function in the brain."- Dr. Susan Kohlhaas, Director of research at the MS Society

Adult Stem Cell Segment is Expected to Maintain its Dominance Over the Forecast Period

According to the analyst at Infinium Global Research, the scope of the market is analyzed on the basis of type, application, and end-user. Based on the type the study includes adult stem cells, induced pluripotent stem cell, human embryonic stem cell, and other types. Based on the application, the study includes regenerative medicine, neurology, oncology, cardiology, and other application. Based on the end-user, the study includes therapeutics companies, cell and tissue banks, tools and reagents companies, and service companies.

Based on the type of adult stem cell segment is expected to dominate the market due to factors such as minimal ethical issues, lower rejection rates, and long-term renewal property associated with stem cell utility has contributed to the dominance of this segment.

Ask Discount for the Latest Research Report @ https://www.infiniumglobalresearch.com/reports/request-discount/13006

Regional Analysis

North America Dominates the Stem Cell Market and Anticipated to Stay on Top During the Forecast Period

Advanced healthcare facilities and increasing funding for research and development are and well-established reimbursement policies and the presence of major market players are supporting the growth of the market in this region.FDA published four guidance documents in November 2017 that supplement existing statutes and together form its regenerative medicine regulatory framework. With these guidance documents, the FDA sought to clarify the distinctions between products that are subject to full drug approval requirements and those that are not.

Competitive Analysis

The key players profiled in the report are Cellular Engineering Technologies Inc., Advanced Cell Technology, Inc., STEMCELL Technologies Inc., BIOTIME, Inc., Astellas Pharma Inc., BrainStorm Cell Therapeutics, Celgene Corporation, CellGenix GmbH, Genea BioCells, Lonza Group AG, and Other companies.

In 2019, STEMCELL Technologies Win Deloitte Best Managed Companies' Award

STEMCELL Technologies is Canada's largest biotechnology company that develops specialized cell culture media, cell isolation systems, accessory products and scientific services that are used by life sciences researchers working in cell therapy, cancer research, and regenerative medicine fields.

In November 2019,BrainStorm Cell Therapeutics Inc.announced the publication of "NurOwn Phase 2 Randomized Clinical Trial in ALS: Safety, Clinical and BioMarker Results," in the international, peer-reviewed journalNeurology.

Browse Complete Global Report and Detailed TOC: https://www.infiniumglobalresearch.com/healthcare-medical-devices/global-stem-cell-market

About usThe Infinium Global research comprises of a team of well-experienced analysts who have qualified in generating incisive reports. The stem cell report offers trends, opportunities, challenges, market size, and forecast for major geographical regions and key countries. Moreover, The IGR-Growth Matrix analysis given in the report brings an insight into the investment areas that existing or new market players can consider. The report provides insights into the market using analytical tools such as Porter's five forces analysis and DRO analysis of the stem cell market.

For more information on this press release visit: http://www.sbwire.com/press-releases/global-stem-cell-market-is-projected-to-grow-at-a-cagr-of-119-during-the-forecast-period-1270550.htm

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Global Stem Cell Market Is Projected to Grow at a CAGR of 11.9% During the Forecast Period - Press Release - Digital Journal

Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers -…

By Stem Cell Treatment

NEW YORK, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, today announced preliminary net revenues for 2019, as well as outlined key BPDCN market successes and upcoming commercial and clinical milestones.

Unaudited preliminary 2019 results include:

The above financial information is based on preliminary unaudited information, is subject to adjustment, and does not present all information necessary for an understanding of the Companys full-year and fourth quarter financial results for 2019. Stemline expects to report complete audited 2019 financial results on or before March 16, 2020.

Robert Francomano, Chief Commercial Officer of Stemline, stated, We are very pleased with the solid uptake seen in the first year of the ELZONRIS launch, as we continue to successfully create, penetrate and grow a new market in BPDCN. Given the orphan nature and unique features of this disease, we believe patient starts were subject to significant quarterly variance a phenomena that will likely continue throughout 2020. We are actively implementing a host of tactics to expand and further penetrate this emerging market.

Ivan Bergstein, CEO of Stemline, commented, 2019 was a transformational year for Stemline as we launched ELZONRIS, the first and only CD123 targeted agent and first agent ever approved for patients with BPDCN. We continue to pursue growth opportunities not only in BPDCN but also in a number of malignancies where targeting CD123 could provide therapeutic benefit. We look forward to data readouts in CMML, MF, and AML, including in patient subsets with high CD123, later this year and on into next year. Given our continued commercial and clinical progress, we look forward to a productive 2020 and beyond.

Corporate Highlights and Key Commercial and Clinical Milestones

BPDCN

Chronic Myelomonocytic Leukemia (CMML)

Myelofibrosis (MF)

Acute Myeloid Leukemia (AML)

About ELZONRISELZONRIS(tagraxofusp), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit http://www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at http://www.bpdcninfo.com.

About CD123CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkins lymphoma (HL), and certain Non-Hodgkins lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

About Stemline Therapeutics Stemline Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics. ELZONRIS(tagraxofusp), a targeted therapy directed to CD123, is FDA-approved and commercially available in the U.S. for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML). Additional pipeline candidates include: felezonexor (SL-801) (XPO1 inhibitor; Phase 1 in advanced solid tumor patients ongoing), SL-1001 (novel RET kinase inhibitor, IND-enabling studies ongoing), SL-701 (immunotherapeutic; Phase 2 in glioblastoma patients completed), and SL-901 (novel kinase inhibitor; prior abbreviated European Phase 1, IND-enabling studies ongoing). For more information, please visit the companys website at http://www.stemline.com.

Forward-Looking StatementsSome of the statements included in this press release may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The factors that could cause our actual results to differ materially include: the risk that our actual revenue for the fourth quarter and year endedDecember 31, 2019may differ materially from our estimated results for these periods as a result of the completion of year-end closing procedures or the audit of our financial statements; the success of our U.S. launch and commercialization; the success of our MAA submission to the EMA and potential launch in Europe; the success and timing of our clinical trials and preclinical studies for our product and product candidates, including ELZONRIS in additional indications and our other pipeline candidates, including site initiation, institutional review board approval, scientific review committee approval, patient accrual, safety, tolerability and efficacy data observed, and input from regulatory authorities including the risk that the FDA, EMA, or other ex-U.S. national drug authority ultimately does not agree with our data, find our data supportive of approval, or approve any of our product candidates; the possibility that results of clinical trials are not predictive of safety and efficacy results of our product candidates in broader patient populations or of our products if approved; our plans to develop and commercialize our product candidates, including, but not limited to delays in arranging satisfactory manufacturing capabilities and establishing commercial infrastructure for ELZONRIS; product efficacy or safety concerns resulting in product recalls or regulatory action; the risk that estimates regarding the number of patients with the diseases that our product and product candidates may treat are inaccurate; inadequate market penetration of our products; our products not gaining acceptance among patients (and providers or third party payors) for certain indications (due to cost or otherwise); the risk that third party payors (including governmental agencies) will not reimburse for the use of ELZONRIS at acceptable rates or at all; the companys ability to produce, maintain or increase sales of ELZONRIS; the companys ability to develop and/or commercialize ELZONRIS; the adequacy of our pharmacovigilance and drug safety reporting processes; our available cash and investments; our ability to obtain and maintain intellectual property protection for our product and product candidates; delays, interruptions, or failures in the manufacture and supply of our product and product candidates; the performance of third-party businesses, including, but not limited to, manufacturers, clinical research organizations, clinical trial sponsors and clinical trial investigators; and other risk factors identified from time to time in our reports filed with the SEC. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not intend to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof.

Contact: Investor RelationsStemline Therapeutics, Inc.750 Lexington AvenueEleventh FloorNew York, NY 10022Tel: 646-502-2307Email: investorrelations@stemline.com

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Stemline Therapeutics Announces Preliminary 2019 Net Revenues for ELZONRIS (tagraxofusp) and Highlights Commercial and Clinical Growth Drivers -...

Magenta Therapeutics Advances Conditioning Platform and Clinical Programs, Highlights Recent Milestones and 2020 Goals – Yahoo Finance

By Stem Cell Clinic

Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today highlighted recent progress across several programs and outlined goals for 2020. These updates will be discussed during a webcast presentation at the 38th annual J.P. Morgan Healthcare Conference on Wednesday, January 15th at 11:30 a.m. PT (2:30 p.m. ET).

"In 2019 we generated landmark data from our ADC-based targeted patient preparation platform, which is delivering a new class of antibody-drug conjugates (ADCs) that have the power to bring one-time treatment to more patients with autoimmune diseases, blood cancers and genetic diseases. We also presented clinical data for our first-line stem cell mobilization program, MGTA-145, which we are developing as the new standard of care for stem cell mobilization with the potential to benefit all of the transplant-eligible patients each year," said Jason Gardner, D. Phil., President and Chief Executive Officer, Magenta. "As we begin 2020, we are particularly excited to unveil our MGTA-117 clinical candidate for targeted patient preparation for stem cell transplant or gene therapy. New results announced today highlight the potency, safety and broad therapeutic index of MGTA-117, well above that of currently approved ADCs. We believe that MGTA-117 is the optimal agent for depleting stem cells to enable safe immune reset. We look forward to moving this program into the clinic with initial clinical data expected in 2021."

Targeted Patient Preparation Programs

Current methods to condition patients before transplant and gene therapy are dependent on toxic, non-specific chemotherapy or radiation. These pre-transplant treatments are associated with significant side effects, including infertility, cancer, organ damage and death. Magenta is developing targeted, disease-modifying ADCs that are designed to precisely and rapidly remove the disease-causing cells in the body and enable immune system reset without the need for chemotherapy or radiation.

CD117-ADC Recent Progress

Data presented at the American Society of Hematology (ASH) annual meeting in December 2019, showed the first-ever successful transplant of gene-modified cells in non-human primates using a CD117-targeted, single-agent ADC from Magenta, without the use of chemotherapy or radiation. These unprecedented results validate and advance Magentas conditioning platform.

Building on this work, Magentas new clinical candidate, MGTA-117, is a CD117 antibody conjugated to amanitin. Results published today in an abstract for the Transplant and Cellular Therapy annual meeting show that MGTA-117 potently depleted stem and progenitor cells and demonstrated a wide tolerability: potency ratio of 30 fold (therapeutic index; typical range for approved ADCs at this stage is two to six fold). This program is advancing to the clinic and further validates Magentas antibody drug conjugate-based conditioning platform. MGTA-117 was developed under a partnership with Heidelberg Pharma that grants Magenta exclusive worldwide development and marketing rights for ADCs using an amanitin payload and targeting CD117.

MGTA-117 in 2020

Magenta is scaling up manufacturing of MGTA-117 and completing IND-enabling studies in 2020. The Company intends to move this new product candidate into the clinic with initial clinical data expected in 2021.

CD45-ADC Recent Progress

Current standard treatment for patients with multiple sclerosis involves years of chronic dosing of medications that do not halt the progression of the disease. For patients with systemic sclerosis, a potentially fatal autoimmune disease, there are no approved therapies. Immune reset through stem cell transplant has demonstrated durable remissions in thousands of patients with autoimmune diseases such as multiple sclerosis and systemic sclerosis, and it is recommended by the European League Against Rheumatism (EULAR) in treatment guidelines for systemic sclerosis. The immune reset process involves two main steps: removing the disease-causing cells and replacing them with healthy cells to rebuild the immune system to a healthy state.

Magenta is developing targeted ADCs designed to precisely remove the disease-causing cells in the body without the need for chemotherapy or radiation. Magentas CD45-ADC program targets CD45, a protein expressed on immune cells and stem cells and is designed to remove the cells that cause autoimmune diseases in order to enable curative immune reset.

Story continues

Data presented at the American College of Rheumatology (ACR) meeting in November 2019 showed that a single dose of CD45-ADC removed disease-causing reactive T cells, enabled successful immune reset and rebuild of the immune system and was well tolerated in three models of autoimmune disease, including the EAE model, the most reliable murine model of multiple sclerosis. Further, a single dose of CD45-ADC significantly reduced disease incidence and delayed disease onset in this model that has successfully provided preclinical proof of concept for many clinically validated standard-of-care therapies.

CD45-ADC in 2020

Magenta has identified a lead antibody and has progressed this program into IND-enabling studies, which the Company plans to further advance in 2020.

MGTA-145 First-Line Stem Cell Mobilization Therapy

MGTA-145 Recent Progress

Magenta is developing MGTA-145 as the new first-line standard of care for stem cell mobilization in a broad range of diseases, including autoimmune diseases, blood cancers and genetic diseases. MGTA-145, a CXCR2 agonist, works in combination with plerixafor, a CXCR4 antagonist, to harness the physiological mechanism of stem cell mobilization.

Magenta is currently studying MGTA-145 and plerixafor in a Phase 1 study in healthy volunteers. Data from the Phase 1 study presented at the ASH annual meeting in December 2019 showed that MGTA-145 in combination with plerixafor successfully enables safe, same-day dosing, mobilization and collection of sufficient high-quality hematopoietic stem cells for transplant. Further, when cells collected from the first two apheresis subjects were transplanted into humanized mice, the cells engrafted more rapidly and at a five-fold higher level than cells from G-CSF-mobilized peripheral blood.

MGTA-145 in 2020

Magenta intends to complete the Phase 1 study and move this program into multiple Phase 2 studies in patients in 2020. The Phase 2 studies will include both allogeneic and autologous transplant settings and will evaluate mobilization and collection of high-quality cells and engraftment of the cells after transplant.

MGTA-456 Cell Therapy

MGTA-456 Recent Progress

MGTA-456 is a cell therapy designed to provide a high dose of stem cells that are well matched to the patient to enable safe immune and blood system rebuild and durable remissions in patients with blood cancers. In September, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for MGTA-456 for the treatment of multiple inherited metabolic disorders.

Magenta is currently studying MGTA-456 in a Phase 2 study in patients with inherited metabolic disorders, including cerebral adrenoleukodystrophy (cALD) and Hurler syndrome. These are rare, rapidly progressive neurologic disorders that are fatal when left untreated. Results in the first two evaluable patients with cALD updated in December 2019 showed early and durable resolution of the disease at 12 months follow-up. The Loes score and NFS score, which measure progress of the disease, remained stable, suggesting that progress of the disease has been halted in these patients. The early and durable resolution of disease with MGTA-456 is not consistently seen with other therapies, including standard stem cell transplant, gene therapy or enzyme replacement therapy.

MGTA-456 in 2020

Magenta intends to complete enrollment in the Phase 2 in 2020 and continue dialogue with the FDA under the RMAT designation, and to discuss with the European Medicines Agency (EMA) for development in Europe

About Magenta Therapeutics

Headquartered in Cambridge, Mass., Magenta Therapeutics is a clinical-stage biotechnology company developing novel medicines for patients with autoimmune diseases, blood cancers and genetic diseases. By creating a platform focused on critical areas of unmet need, Magenta Therapeutics is pioneering an integrated approach to allow more patients to receive one-time, curative therapies by making the process more effective, safer and easier.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could", "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavor," "potential," "continue" or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption "Risk Factors" in Magentas Annual Report on Form 10-K, as updated by Magentas most recent Quarterly Reports on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200113005308/en/

Contacts

Magenta Therapeutics:Manisha Pai, Vice President, Communications & Investor Relations617-510-9193mpai@magentatx.com

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Magenta Therapeutics Advances Conditioning Platform and Clinical Programs, Highlights Recent Milestones and 2020 Goals - Yahoo Finance

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