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Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test – MedCity News

PlateletBio, a company developing a new class of cell therapies based on the biology of platelets, has raised $75.5 million to advance its drug pipeline, including a lead candidate for a rare bleeding disorder on track to reach the clinic next year.

Platelets are components of blood best known for their role forming clots that stop bleeding. But Watertown, Massachusetts-based PlateletBio notes that platelets have other properties, including a role delivering growth factors and proteins throughout the body. PlateletBio is developing therapies that take advantage of these properties, but rather than using platelets from a patient or healthy donors, the startup makes them.

In the body, platelets are formed in bone marrow. PlateletBio produces its platelet-like cells, or PLCs, inside a bioreactor that mimics bone marrow conditions. The source material for its PLCs are stem cells, which have the ability to become almost any cell or tissue in the body.

Platelets are technically not cells. They dont have a nucleus, but thats an advantage for therapeutic applications. Since a PlateletBio therapy wont introduce DNA into a patients body, the potential risks that come from introducing foreign genetic material are avoided. PlateletBio says it can produce PLCs with new features and therapeutic payloads that include antibodies, signaling proteins, therapeutic proteins, and nucleic acids.

PlateletBios lead cell therapy candidate is being developed to treat immune thrombocytopenia, a blood disorder in which the immune system mistakenly sees a patients platelets as foreign and destroys them. Immune thrombocytopenia patients have dangerously low platelet counts that make them susceptible to bleeding.

There is no FDA-approved treatment for the underlying cause of immune thrombocytopenia, but corticosteroids are used to try to dampen the immune systems attack on platelets. Platelet transfusions are another option, but the National Organization for Rare Disorders notes that these treatments are usually reserved for emergencies because the platelets are likely to be destroyed by antibodies produced by the patient.

Patients who have not responded to earlier treatments have two FDA-approved small molecule options: Tavalisse, from Rigel Pharmaceuticals, and the Swedish Orphan Biovitrum drug Doptelet. Sanofi aims to treat the disease with a small molecule called rilzabrutinib. That drug is designed to block Brutons tyrosine kinase, a protein that plays a role in the development of a B cells, a type of immune cell. Sanofi acquired the molecule last year via its $3.7 billion acquisition of Principia Biopharma.

The lead disease target for the Principia drug was multiple sclerosis. In September, Sanofi reported that rilzabrutinib failed that Phase 3 study. A separate Phase 3 test in immune thrombocytopenia is ongoing, as is a mid-stage clinical trial in another autoimmune condition called IgG4-related disease.

PlateletBio isnt the only company trying to turn a component of the blood into a new type of cell therapy. Cambridge, Massachusetts-based Rubius Therapeutics is developing cell therapies based on red blood cells. After disappointing early clinical trial results in the rare disease phenylketonuria last year, Rubius shifted its focus to cancer and immune system disorders. PlateletBios PLCs would represent an entirely new approach to treating immune thrombocytopenia. According to PlateletBios website, the company plans to file an investigational new drug application for its therapeutic candidate in the first half of next year.

PlateletBio is based on the research of Harvard University scientist Joseph Italiano, who co-founded the company under the name Platelet BioGenesis. When the startup emerged in 2017, it was developing platelets that could address the platelet shortage problem facing blood donation centers. Two years ago, the startup expanded its Series A round with $26 million in additional financing and plans to make its platelets into cell therapies. Besides immune thrombocytopenia, other diseases the biotech aims to treat include osteoarthritis and liver fibrosis.

PlateletBios latest financing, a Series B round, adds new investors SymBiosis, K2 HealthVentures, and Oxford Finance. Earlier investors Ziff Capital Partners and Qiming Venture Partners also participated in the new round.

This is a major milestone for PlateletBio, adding capital and resources needed to advance our innovative platelet-like cell therapy science and manufacturing platform and support key corporate initiatives over the next 18 to 24 months, Sam Rasty, the startups president and CEO, said in a prepared statement.

Photo by Flickr user Marco Verch via a Creative Commons license

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Cell therapy biotech PlateletBio reels in $75M as it looks ahead to first clinical test - MedCity News

VDH: Cases surge again to third-worst on record – Vermont Biz

Vermont Business MagazineThe Vermont Department of Healthis reporting today that COVID-19 cases surged again to 376 cases, the third highest on record. This was an increase of 114 from Saturday.Cases Friday were377 and there were 487 cases on Thursday, which was the all-time record. The 12worst days have all come since mid-September, following fromthe Delta variant surge that began in July. There were no additional COVID-related death, which stand at384 statewide.

Based on record high cases this past week, Governor Phil Scott issued a statement Thursday afternoon urging Vermonters to get vaccinated. He said in part, "The simple fact is, this pandemic is being driven by the unvaccinated, including 53,000 eligible adults. The three lowest vaccinated counties account for 25% of todays cases, yet they only make up 10% of our state population. Unvaccinated Vermonters are up to 5 times as likely to contract COVID in Vermont and they account for 70-85% of our hospitalizations and ICU stays. Unvaccinated adults are directly contributing to the strain on our hospital capacity. Enough is enough, its time to step up and get vaccinated something over 90% of your fellow Vermont adults have done."

Cases have been especially high in the Northeast Kingdom, which has had elevated case rates in recent weeks. Chittenden County had the most cases with 83 and Washington County had51.

Meanwhile, the CDC gave final approval late Tuesday for Pfizer vaccines for children 5-11 and parents could start signing up their kids starting Wednesday morning (for vaccine information or to registerCLICK HERE). Shots for 5-11 are available starting Friday at some state clinics and as early as Sunday at some pharmacies.

There are about 44,000 kids in the 5-11 age band in Vermont. Human Services Secretary Mike Smith said there will be plenty of first and second doses available for all who sign up. The doses are a third of the strength of the adult dose and like the adult Pfizer regimen will require a followup shot for full immunity.

See locations of vaccine clinics and case dashboards below.

There are 45 people hospitalized (up thrtee) with 12in the ICU (downtwo).Hospitalizations are high but have been stable.

Recent fatalities have all been amongVermonters 50 and older, with most in the oldest age band, which has been the case since the beginning of the pandemic.

There were 39 COVID-related deaths in Vermont in October, which is the third-worst month on record. There are nine so far in November.

The 11 worst days for cases have all come since mid-September,as the Delta variant has taken oversince early July.

Financial Regulation Commissioner Michael Pieciak said both the case counts and fatalities have not shown dramatic reductions, as they have in other parts of the nation, particularly the South, or even in southern New England.

While the health experts do not understand exactly why this is, given Vermont's high vaccination rate, Pieciak said the three Northern New England states are all showing a similar pattern.

In addition, Health Commissioner Mark Levine, MD, continues to urge Vermontersto get their booster dose, especially if they're over 65. The immunity appears to wane over time and older Vermonters and those immunocompromised were among the first vaccinated last winter.

The elderly have been especially vulnerable to COVID, with the vast majority of fatalities coming among those 80 and over (206total deaths, while having by far the fewest number of infections for any age band.). One death Saturday for 80+.

Thestate announced last Thursday nightthat boosters for Moderna (like already approved Pfizer, six months after second dose) and Johnson & Johnson (two months after first dose)would begin immediately. The CDC is also allowing people to change/ mix and match vaccines.

TheFDA Tuesdayapproved the low-dose Pfizer vaccine for children5-11. Human Services Secretary Mike Smith said if the CDC approves it tonight then the state will allow parents to sign up their kids as early as 8 am tomorrow morning with doses being available as early as Thursday.

He said that in addition to the usual vaccination clinics, the state will also be bringing the vaccine directly to 112 schools around the state, especially to those in outlying areas where transportation or fewer pharmacies could make it more difficult for kids to get their shots.

Dr Rebecca Bell, President, Vermont Chapter of the American Academy of Pediatrics, at the governor's press conference Tuesday, sought to alleviate parents' fears.

She said the efficacy of this lower dose children's vaccine is still 91 percent. And unlike with adults, children in the control group did not show any of the flu-like symptoms that some adults have experienced.

Parents should also take comfort that a vaccinated child would no longer have to quarantine if they were a close contact to someone that tested positive. This issue has kept many kids out of school even though they never contracted COVID.

The Northeast Kingdom has the highest case rate of any region of the state, with Orleans County having the highest county rate, with over 1,000 cases per 10,000 residents. Bennington has the second highest rate with over 900 per 10,000.

Smith said the state will reinstitutepop up vaccination sitesin the NEK to increase vaccination rates, which are the lowest in the state. See list of upcoming NEK clinicsHEREand see below for statewide list of clinics.

COVID cases reported today were high in southern Vermont, with Bennington County alone reporting 34 and Rutland County 24.

But with all three boosters now available, vaccines for kids available and cases apparently declining, Governor Scott said Tuesday,"I'm more hopeful today than I have been in weeks."

Education Secretary Dan French announced Tuesday that the state was once again delaying the roll out of the 80 percent rule for ending school mask mandates. It will not happen go into effect until January 18, 2022, when students return to school after the Martin Luther King Holiday break.

The governor and his staff reiterated that these are recommendations only, because there is no State of Emergency, not mandates. The local school districts, like local businesses and individuals, must make their own rules.

As the Delta variantcontinues to be active in Vermont, Governor Scottiscalling on all Vermonters to act responsibly.

The new school guidance reads:

"To allow school districts time to calculate the percentage of currently eligible students who have received two doses of a two-dose vaccine, schools should require universal masking for all students and staff when indoors until January 18, 2022.

"Currently, all Vermonters ages 12 and older are eligible to be vaccinated.

"After January 18, 2022, masks should no longer be required for all those eligible for vaccination when the vaccination rate (two doses of a two-dose vaccine) among students is equal to or greater than 80% of the schools currently eligible population.

"Masks should be required indoors for students younger than 12, who are not eligible to be vaccinated at this time.

"Masks, when required, may be removed when needed for instructional or operational purposes.

"Masks are currently required for all passengers on buses per federal regulation, regardless of age or vaccination status.

"Masks should not be required outdoors. Guidance will be updated when vaccine eligibility expands."

The state also released school sports guidance Tuesday.

See Vaccination & COVID-19 Dashboards & Vaccination Sites TableBelow

Addison County

New Cases:5

Recent Cases 14 days:91

Bennington County

New Cases:20

Recent Cases 14 days:352

Caledonia County

New Cases:30

Recent Cases 14 days:204

Chittenden County

New Cases:83

Recent Cases 14 days:651

Essex County

New Cases:8

Recent Cases 14 days:90

Franklin County

New Cases:48

Recent Cases 14 days:301

Grand Isle County

New Cases:2

Recent Cases 14 days:26

Lamoille County

New Cases:15

Recent Cases 14 days:134

Orange County

New Cases:14

Recent Cases 14 days:169

Orleans County

New Cases:42

Recent Cases 14 days:334

Pending Validation

New Cases:4

Recent Cases 14 days:6

Rutland County

New Cases:15

Recent Cases 14 days:412

Washington County

New Cases:51

Recent Cases 14 days:283

Windham County

New Cases:6

Recent Cases 14 days:176

Windsor County

New Cases:33

Recent Cases 14 days:261

Most cases in Vermont are in the younger age groups with the 20-29 reporting the most, with nearly 7,300 total cases out of 37,100+, but only one death. The over 79 demographic has the fewest cases (just over 1,200) but by far the most fatalities with 188, or more than half the state total.

Financial Commissioner Michael Pieciak said Tuesday (SEE HIS FULL SLIDE DECK HERE) cases across the nation are falling fast and that the seven-day and 14-day averages in Vermont and the Northeast also are falling but at a slower rate. Vermont's seven-day infection rate is down15 percent. For the 14-day average, while overall it is down 2 percent for those who are fully vaccinated, it's up 9percent for those who are not fully vaccinated (which includes the unvaccinated).

Cases in high vaccination regions of the country are not displaying the typical Delta variant pattern, as in India, of a spike followed by a steep drop off after a couple months.

In Vermont, Delta has shown slow growth and a long plateau. Health Commission Mark Levine suggested that the drop off here could be another couple of weeks off, but he frankly was not sure.

He and Governor Scott and Human Services Secretary Mike Smith all urged that everyone who is eligible to get a vaccine to get one now, to get a booster now and to wear a mask while at an indoor gathering.

Governor Scott said the data shows that virus transmission with Delta is occurring at things like weddings and baby showers and birthday parties, sort of small and medium events where people are gathered for a period of time. It is not happening while visiting a convenience store or other type place where you are in an out, he said. Nor is it happening at outdoor gathering events.

Pieciak,in his COVID-19 Modeling presentation, said deaths seem to have slowed down a little as Delta has taken its toll. There have been 23 in October so far (as of the 21st)

Still, September was the second worst month for COVID-related deaths in Vermont since the beginning of the pandemic, with 45.

December 2020 was the worst with 71 and April 2020 was third worst with 35.

Meanwhile, the state is ramping up antigen testing in schools to keep kids in school who otherwise would be sent home if there were a close contact of someone who tests positive. Children now have a higher rate of infection than adults.

Governor Scott and state officials are urging all those who are eligible now to get vaccinated or get a booster shot, to do so, in order to reduce community transmission of the novel coronavirus (see clinic sites below).

As of October 1, many more Vermonters can now schedule and receive their Pfizer vaccine booster shots. He said there is plenty of vaccine supply.

We know vaccines are safe and effective, and these additional doses add even more protection. So, I encourage anyone who is eligible to register for your booster today, said Governor Scott. At the same time, we continue to urge those who have not yet gotten their first dose to get vaccinated. The data shows we are now in a pandemic of the unvaccinated, and vaccines are the best way to protect yourself, friends and family, and to make sure we continue moving forward from the pandemic.

Governor Scott said that the state will take a very broad interpretation of eligibility.

"We've reflected on this," Scott said. "We'regoing to be quitelenientin terms of whoshould beincluded and if they'd like tohave a booster we'd like to find a wayfor them to have it. So Iexpect that number is substantiallyhigherat this point. So our interpretation ofthis will be,again, quite broad."

Addition of Rapid Testing Tools Will Help Keep More Kids in School

When a student or staff member with COVID-19 is present in school during their infectious period, the school may implement Test to Stay for unvaccinated close contacts.

Unvaccinated, asymptomatic students (ages 5 and up) and staffwho areclosecontacts of a positiveCOVID-19case.

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VDH: Cases surge again to third-worst on record - Vermont Biz

ElevateBio Supercharges Gene Editing and Therapeutic Product Development Capabilities Through Acquisition of Life Edit Therapeutics – Yahoo Finance

- Life Edit's genome editing capabilities to be fully integrated with ElevateBio's other cell and gene enabling technologies, including induced pluripotent stem cells (iPSCs), viral vector, and cellular engineering

- Brings a broad array of editing modalities, including deletion, insertion, base editing, and CRISPRa/CRISPRi

CAMBRIDGE, Mass., October 27, 2021--(BUSINESS WIRE)--ElevateBio, LLC (ElevateBio), a cell and gene therapy technology company focused on powering transformative cell and gene therapies, today announced that it has acquired all of AgBiome Delta, LLCs (AgBiome) shares of Life Edit Therapeutics, Inc. (Life Edit). Life Edit offers a powerful suite of gene editing technologies that have the potential for any genomic sequence of interest to be removed, added, or altered. Life Edit holds one of the world's largest and most diverse arrays of novel RNA-guided nucleases and base editors that offer greater specificity and broad genome access. These nucleases were derived from AgBiome's proprietary non-pathogenic microbe collection, which could potentially reduce immunogenicity risks.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211027005153/en/

"ElevateBios powerful suite of enabling technologies, which now includes Life Edits genome editing capabilities as well as our existing iPSC, viral vector, and cell engineering platforms, is designed to disrupt the rapidly advancing fields of cell and gene therapy," said David Hallal, Chairman and Chief Executive Officer of ElevateBio. "Our vision is to build a world-class center of excellence in genome engineering to push the boundaries of therapeutic development and drive innovation for our own therapeutic pipeline as well as provide access to these critical technologies to our growing number of industry partners."

"Genome editing is a central component of all cell and gene therapy development, and access to novel RNA-guided nucleases and base editors that offer specificity and broad genome coverage will be critical. We believe Life Edit's technology is one of the most versatile in the field, opening up enormous potential," Mitchell Finer, Chief Executive Officer of Life Edit and President, R&D of ElevateBio. "This integration will also enable Life Edit to have greater access to ElevateBios drug development and manufacturing capabilities as we build and advance the pipeline, which will initially focus on developing in vivo gene therapies to address neurologic conditions with high unmet need. In addition, by combining Life Edit's gene engineering capabilities with ElevateBios iPSC technology, our goal is to expand the number of therapeutic uses, including potentially making universal or hypoimmune cells that go undetected by the immune system."

Story continues

Life Edit was spun out of AgBiome in October 2020 and AgBiome continues to retain rights for gene editing outside of human therapeutics.

About Life Edit Therapeutics:Life Edit has one of the world's largest and most diverse arrays of novel RNA-guided nucleases and base editors active in mammalian cells. They were developed from a proprietary collection of non-pathogenic organisms and offer gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. Life Edit has a large and diverse library of RNA-guided nucleases, including Type II and Type V systems that encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with one of our proprietary deaminases. The company's nuclease collection has a broad range of Protospacer Adjacent Motifs (PAMs). These short sequences must accompany the DNA sequence for the enzyme to edit a gene, which offers unprecedented access to genomes. Life Edit has identified several classes of DNA modifying enzymes, including novel deaminases that can edit cytidine (C) or adenine (A). Many of the company's RGNs are smaller than widely used CRISPR-Cas systems, offering ease and flexibility for in vivo delivery and manufacturing.

Life Edit is headquartered in Morrisville, NC. Visit us at http://www.lifeeditinc.com, or follow Life Edit on LinkedIn and Twitter.

About ElevateBio:ElevateBio is a cell and gene therapy technology company built to power the development and manufacturing of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built world-class facilities, and integrated diverse technology platforms necessary for rapid innovation and commercialization of cell, gene, and regenerative therapies. The company has built an initial technology stack, including gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering, that can be leveraged across the entire portfolio and by strategic partners. At the center of the business model is ElevateBio BaseCamp, a centralized research and development (R&D) and manufacturing company that offers R&D, process development (PD), and Current Good Manufacturing Practice (CGMP) manufacturing capabilities. The company is focused on increasing long-term collaborations with industry partners while also developing its own highly innovative cell and gene therapies. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

ElevateBio is located in Waltham, MA. Visit us at http://www.elevate.bio, or follow ElevateBio on LinkedIn, Twitter, or Instagram.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211027005153/en/

Contacts

Investor: Catherine Hu chu@elevate.bio 646-535-8276

Media: Courtney Heath ScientPR Courtney@scientpr.com 617-872-2462

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ElevateBio Supercharges Gene Editing and Therapeutic Product Development Capabilities Through Acquisition of Life Edit Therapeutics - Yahoo Finance

ReNeuron enters cancer therapy collaboration with UCL – ShareCast

Cell-based therapeutic specialist ReNeuron has entered a collaboration agreement with University College London (UCL), it announced on Tuesday, to conduct research into the generation of immune cells from induced pluripotent stem cells (iPSCs), for anti-cancer cell therapies.

The AIM-traded firm said it would be working alongside Dr Claire Roddie, associate professor at the UCL Cancer Institute, and the team at the UCL CAR-T cell cancer therapy programme.

It said it would provide UCL with iPSCs from its CTX immortalised neural progenitor cell line to be assessed for their ability to differentiate into functional T cells and natural killer (NK) cells.

If this first objective was met, the CTX-iPSC cell line would be further used to generate chimeric antigen receptors (CAR) T cells, or CAR-NK cells.

ReNeuron explained that CARs allow T and NK cells to target receptors present on the surface of cancer cells, allowing them to recognise and specifically kill tumour cells.

Dr Roddie and her team had extensive expertise in generating CAR cells, the company said, adding that both groups would work collaboratively to generate haematopoietic stem cells, lymphoid progenitors and cytotoxic T cells from the CTX-iPSCs.

We are very much looking forward to working with Dr Roddie and her team at UCL in this exciting and rapidly growing research space, said chief scientific officer Dr Stefano Pluchino.

This collaboration is another strong development for ReNeuron which demonstrates the uniqueness and strong translational potential of our proprietary iPSC technology platform and will allow us to further expand into the oncology space.

If this initial research is successful, significantly large numbers of cancer patients can be treated with next generation alternate cancer therapies.

At 1243 BST, shares in ReNeuron Group were up 4.91% at 119.6p.

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ReNeuron enters cancer therapy collaboration with UCL - ShareCast

Shah on the Potential Utility of NK Cells in Multiple Myeloma – OncLive

Nina Shah, MD, discusses the potential utility of natural killer cells in multiple myeloma.

Nina Shah, MD, hematologist and oncologist, associate professor of medicine, Department of Medicine, University of California, San Francisco (UCSF) Helen Diller Family Comprehensive Cancer Center, discussesthepotential utility of natural killer (NK) cells in multiple myeloma.

NK cells are a potential type of allogeneic cellular therapy for patients with multiple myeloma, explains Shah. However, a challenge with NK cells is that there are not as many NK cells as other cells; therefore, NK cells need to be expanded and often engineered, Shah explains. One example of engineeringinducibleNK cells is being evaluated by Fate Therapeutics. The NK cells are engineered from a human induced pluripotent stem cellbank to then be cultured, Shah adds.

Moreover, the NK cells strongly express CD16 and Fc receptor on the cell surface. Additionally, CD38 is engineered so utilizing daratumumab (Darzalex) is an option with both engineering tactics, Shah continues. Additionally, the cells are engineered in the context of CAR so targeting BCMA is an option to kill cells. An IL-15 receptor fusion protein was also engineered to allow the NK cells to persist. Preclinical data have shown that the cells are active and persistent, Shah explains.

Overall, more data are anticipated, which will include patients with relapsed/refractory multiple myeloma who received NK cells with a monoclonal antibody, such as daratumumab, Shah explains. Moreover, this approach would be given off-the-shelf, which could open an accessible option to patients, Shah concludes.

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Shah on the Potential Utility of NK Cells in Multiple Myeloma - OncLive

Exacis Biotherapeutics Announces Strategic Partnership With CCRM For Specialty Manufacturing Of Services And Investment For Development Of…

Cambridge, MA, - Exacis Biotherapeutics, Inc., a development-stage immuno-oncology company working to harness the immune system to cure cancer, today announced initiation of a strategic partnership with Toronto-based Centre for Commercialization of Regenerative Medicine (CCRM) for specialty manufacturing services related to the development of Exacis innovative, iPSC-derived mRNA-engineered NK cell products to treat cancer. The partnership also includes a cash investment into Exacis by CCRM Enterprises Holdings Ltd., the for-profit venture investment arm of CCRM, which will be used to fund operations.

Exacis CEO Gregory Fiore, MD, commented, We welcome CCRM as a key partner to allow us to rapidly advance our virus-free manufacturing processes to make novel NK cell products that are engineered for performance and to avoid rejection. CCRM is a recognized leader in iPSC-derived cell therapy development and manufacturing and we are thrilled to have them as a partner. Their confidence in Exacis is evidenced by the accompanying investment, by CCRM Enterprises Holdings Ltd., underscoring the unique value proposition offered by Exacis differentiated platform and approach to cell therapies. We look forward to partnering with CCRMs CDMO experts to apply our mRNA based technologies to develop best-in-class products to treat challenging hematologic and solid tumors.

Cynthia Lavoie, PhD, President and CIO of CCRM Enterprises Inc. added, We are pleased to support Exacis by way of an investment, and with our sector expertise and specialized infrastructure. This is a successful model that we have employed in the past to support promising technologies and together we will develop leading cell therapy products that utilize the substantial potential of the Exacis platform as it advances its iPSC-derived cell programs.

About Exacis Biotherapeutics

Exacis is a development stage immuno-oncology company focused on harnessing the human immune system to cure cancer by engineering next generation off-the-shelf NK and T cell therapies aimed at hematologic malignancies and solid tumors. Exacis was founded in 2020 with an exclusive global license to a broad suite of patents covering the use of mRNA based cell reprogramming and gene editing technologies for use in generating engineered NK and T cells for oncology applications. These patents were developed and are owned by Factor Bioscience.

Exacis differentiated cell therapy platform avoids the use of DNA and viruses and uses instead a proprietary mRNA based technology. Exacis uses the technology to generate iPSCs and to edit their genomes to create stealthed, potent allogeneic cell products, termed ExaNK, ExaCAR-T or ExaCAR-NK cells.

About CCRM

CCRM is a global, public-private partnership headquartered in Toronto, Canada. It receives funding from the Government of Canada, the Province of Ontario, and leading academic and industry partners. CCRM supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding and infrastructure. CCRM is the commercialization partner of the University of Toronto's Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About CCRMEnterprises Inc.

CCRMEnterprises Inc. is the for-profit venture investment arm of the Centre for Commercialization of Regenerative Medicine (CCRM).CCRM Enterprises invests in early stage ventures and projects developing therapeutics and enabling technologies. Through an extensive network of investors, it can bring together risk capital to support these early-stage ventures as they scale up along the development pathway.By tapping into CCRMs manufacturing infrastructure and expertise, CCRM Enterprises provides capital-efficient support to accelerate and de-risk these high potential, early-stage ventures, further enabling the development of an advanced therapies ecosystem. CCRM Enterprises Holdings Ltd. is the associated entity that holds shares in CCRM portfolio companies. Learn more about our investing strategy here.

About T and Natural Killer (NK) Cell Therapies

T and NK cells are types of human immune cells that are able to recognize and destroy cancer cells and can be modified through genetic engineering to target specific tumors.

Contact Exacis Biotherapeutics for further information and media:

Exacis Biotherapeutics info@exacis.com

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Exacis Biotherapeutics Announces Strategic Partnership With CCRM For Specialty Manufacturing Of Services And Investment For Development Of...

Team finds way to enhance stem cell therapy for CNS injuries – BioPharma-Reporter.com

The scientists, Christopher Rathnam and colleagues, say they have designed a way of controlling the formation of 3D spheroids made from stem cells, while enhancing the spheroids ability to differentiate into functional neurons.

The technology led to an increase in stem cell survival and differentiation two challenges with existing stem cell therapy systems in a mouse model of spinal cord injury, noted the team in a paper published inScience Advances

We believe that our technology platform is an ideal candidate for improving many other types of cell therapies that require high cell survival and effective control of cell fate, making it useful not only for treating [spinal cord injuries] but also for various other diseases and disorders, said the authors.

Although stem cell therapy holds enormous potential for treating debilitating injuries and diseases of the CNS, the team outlined how low survival and inefficient differentiation have restricted its clinical applications.

Recently, 3D cell culture methods, such as stem cellbased spheroids and organoids, have demonstrated advantages by incorporating tissue-mimetic 3D cell-cell interactions, said the experts.

However, a lack of drug and nutrient diffusion, insufficient cell-matrix interactions, and tedious fabrication procedures have compromised their therapeutic effects in vivo, they added.

To address these issues, the Rathnam led team developed a method in which biodegradable manganese dioxide nanosheets guide the rapid assembly of neural stem cells, derived from human induced pluripotent stem cells (iPSCs), into 3D spheroids.

The technique also enables controlled drug release inside the core of the spheroids, which could help to improve cell survival and differentiation, they said.

To evaluate the efficacy of the structures, which they termed synthetic matrix-assisted and rapidly templated (SMART) spheroids, the researchers implanted them at injury sites in a mouse model of spinal cord injury.

As controls, they injected cell suspensions and conventional neurospheres, formed without the use of their novel nanosheets, at the spinal cord injury sites, with the same total number of cells per animal and at the same concentrations.

They found significantly higher cell survival and improved neuronal differentiation efficiency for the SMART neurospheres compared with the controls both 7 days and 1 month after injection.

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Team finds way to enhance stem cell therapy for CNS injuries - BioPharma-Reporter.com

Bacterial Infections Linked with Unapproved Stem Cell Treatments – Contagionlive.com

Unproven products marketed as stem cell therapies could be risky, according to a new study from the Centers for Disease Control and Prevention, which analyzed bacterial infections from unapproved products derived from umbilical cord blood.

The case series study, published in JAMA Network Open, examined 20 bacterial infections in eight states. It involved reviewing medical records, sterility testing of products and whole-genome sequences of patient and product isolates for participants who developed bacterial infections after receiving stem cell treatments between August 2017 and September 2018.

The findings of this investigation show that stem cell therapies that are not FDA-approved or that are not used for the approved medical conditions can pose serious health risks to patients with no benefit, Kiran Mayi Perkins, MD, lead investigator with the CDCs Outbreak and Response Team, told Contagion. Currently, the only stem cell products derived from umbilical cord blood that are FDA-approved for use in the United States are approved for use in patients with disorders that affect the production of blood, but they are not approved for other uses. However, these products are often illegally marketed by clinics as being safe and effective for treating a wide range of diseases or conditions. Therefore, patients should be aware of the unproven benefits and the potential risks to their health when using unapproved and unproven stem cell products for conditions that they have not been shown to effectively treat.

All but one of the patients in the study required hospitalization after receiving stem cell treatment for conditions including osteoarthritis, rheumatoid arthritis and injury. The CDC performed sterility testing on vials of product and compared bacterial isolates with those from the patients.

We were surprised by the magnitude of bacterial contamination found in the vials that we tested; over half of the vials of the stem cell product that we tested were contaminated with bacteria, and many of these vials had very high bacterial counts, Perkins said.

Unapproved stem cell products have been marketed for conditions such as joint diseases, sports injuries and chronic pain and have become more prevalent as people seek products to prevent and treat COVID-19, the study noted. However, these uses are not approved by the US Food and Drug Administration (FDA).

ReGen Series products processed by Genetech and distributed by Liveyon were recalled after bacterial infections were reported in Texas and Florida in 2018, and a national investigation was launched. Information was gathered about patients, product administration, infection control practices and product manufacturing and distribution.

The bottom line is that many stem cell clinics are offering unproven products that have the potential to be dangerous, Perkins said. There is good research that is being done on stem cell therapies, but there are also a lot of clinics that are selling stem cells for unproven uses. To date, the only stem cell treatments approved by the FDA are products made from a donors umbilical cord blood that are used to treat certain cancers and disorders of the blood and immune system. If the cells are being used to treat other conditions such as pain, orthopedic conditions, autism, anti-aging, or COVID-19, they are not approved and may not be safe. We urge all patients and health care practitioners considering stem cell therapies to ensure that the stem cell product is being used for the approved indication or under an Investigational New Drug Application (IND) and is on FDAs list of approved products.

The states in which confirmed bacterial infections were identified as of March 2021 are Texas, Florida, California, Arizona, Kansas, Maine, Colorado and Massachusetts.

The treatments were injected into the patients knees, shoulders, spine or digits or administered through intravenous infusion or as a nasal spray. Infections included 10 at the injection site, five bloodstream infections and five with both injection site and bloodstream infections. Most common bacteria were Escherichia coli and Enterobacter cloacae.

CDC will continue to investigate any reports that it receives that are concerning for infectious risks to patients associated with the receipt of stem cell products and will report these to FDA, the agency that has regulatory oversight for these types of therapies, Perkins said.

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Bacterial Infections Linked with Unapproved Stem Cell Treatments - Contagionlive.com

Stem Cells Used to Treat Avascular Necrosis of the Femoral Head – Yale School of Medicine

Collapsed femoral heads caused by osteonecrosisotherwise known as avascular necrosis unfortunately represent the root cause for approximately 10% of all hip replacements nationwide. Daniel Wiznia, MD, is utilizing a stem cell treatment at Yale School of Medicine and integrating new techniques along with 3D imaging technology as part of a joint-preservation procedure.

Occurring in more than 20,000 Americans each year, osteonecrosis of the femoral head is commonly diagnosed in patients in their 30s and 40s. The disease is caused by injury of the blood supply to the head of the femur, which is the ball portion of the hips ball and socket joint.

If unaddressed, this disease may ultimately lead to the collapse of the femoral head, requiring the patient to undergo a hip replacement. For patients in this age group, a total hip replacement is not ideal as it likely will wear out and the patient will require more surgery.

The goal for each case is clear: prevent the femoral head from collapsing and the need for a hip replacement. As part of a surgical procedure, Wiznia harvests bone marrow from the patients pelvis. The individuals own stem cells are then isolated from the marrow, concentrated, and injected into lesions within the avascular portion of the femura treatment that is only taking place at some of the nations largest medical centers.

The key in these instances is to discover the avascular necrosis before the head collapses, Wiznia said. Because the vascular injury is usually a painless event, patients are generally unaware of the specific point in time when the vascular injury occurred, which is why cases are rarely discovered in time. However, we do know that 80% of patients who have avascular necrosis on one side of the hip have it on the opposite side. We usually are able to catch that second asymptomatic side in those situations and conduct the core decompression with stem cell treatment before it collapses.

According to Wiznia, this treatment reduces the risk of the head of the femur from collapsing, and the stem cell therapy has shown promising results. Soon after the procedure, many patients with avascular necrosis experience rejuvenated blood supply to the area and the bone is repopulated with new cells. This can additionally alleviate the short-term need for a hip replacement.

This novel stem cell therapy has demonstrated improved pain and function, and the stem cells decrease the risk of the femoral head from collapsing, Wiznia said. This translates into fewer young patients requiring hip replacements, and subsequent surgeries in their later years.

As an engineer himself, Wiznia works closely with the Yale School of Engineering & Applied Sciences and the Integrated 3D Surgical Team to better tailor this treatment to each specific patient.

One of the challenges of orthopaedic surgery in the human body is that surgeons are operating in a three-dimensional space and are often reliant on two-dimensional imagery such as X-rays, Wiznia added. Through the use of computer modeling, we are able to customize those images and create models that are specific to each patient, which, in turn, enhances outcomes and overall post-operative success rates.

Enhanced models and 3D imaging enable surgeons like Wiznia to better locate and target both the lesions and necrotized bone in these instances. Effectively doing so regenerates the femoral head and stimulates new osteoblast growth, which will heal the region, maintain the integrity of the joint, and decrease the chance of femoral head collapse and need for a hip replacement.

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Stem Cells Used to Treat Avascular Necrosis of the Femoral Head - Yale School of Medicine

Stem cell therapy can help combat common symptoms of aging – The Mountaineer

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Stem cell therapy can help combat common symptoms of aging - The Mountaineer