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Research Roundup: Why mRNA Vaccines are So Good Against Severe COVID-19 and More – BioSpace

Posted: January 5, 2022 at 2:10 am

Not surprisingly, theres still plenty of interesting research coming out about COVID-19, but the end of 2021 also provided exciting science in other areas. Heres a look.

Scientists out ofWashington University School of Medicinewanted to understandwhy the mRNA vaccines by Pfizer-BioNTech and Moderna are so effective at preventing severe disease. Even in the face of Omicron, which is particularly good at evading immune protection, people vaccinated with the two mRNA vaccines appear to be strongly protected against hospitalization and death from COVID-19. The researchers, along withSt. Jude Childrens Research Hospital,found that the Pfizer-BioNTech strongly and persistently activated a specific type of helper immune cell known as T follicular helper cells. These immune cells help antibody-producing cells to create large amounts of increasingly powerful antibodies and drives development of some forms of immune memory. They published their research in the journalCell.

The longer the T follicular helper cells provide help, the better the antibodies are and the more likely you are to have a good memory response, said Dr. Philip Mudd, co-corresponding author and assistant professor of emergency medicine at Washington University. In this study, we found that these T follicular helper cell responses just keep going and going. And whats more, some of them are responding to one part of the viruss spike protein that has very little variation in it. With the variants, especially Delta and now Omicron, weve been seeing some breakthrough infections, but the vaccines have held up very nicely in terms of preventing severe disease and death. I think this strong T follicular helper response is part of the reason why the mRNA vaccines continue to be so protective.

Generally, the first antibodies generated in response to an infection or vaccine arent very high quality. The researchers say B cells need to go through a sort of boot camp in the lymph nodes before they can generate very powerful antibodies. T follicular helper cells, they note, are the drill sergeants of the boot camps. The helper cells give instructions to the antibody-producing cells on how to make even better antibodies and then encourage the best to multiple and sometimes become long-lived memory B cells.

More Data Omicron Evades Immune Protection

The growing body of evidence that the Omicron variant of SARS-CoV-2, the virus that causes COVID-19, can evade immunity created with vaccines and natural infection gained yet more support. Research out ofColumbia University Irving Medical Centerand theUniversity of Hong Kongtested antibodiesgenerated by vaccination and their ability to neutralize Omicron in laboratory assays using live viruses and pseudoviruses that mimic Omicron. They found that antibodies from people double-vaccinated by the Moderna, Pfizer-BioNTech, AstraZeneca-Oxford and Johnson & Johnson vaccines were significantly less effective against Omicron compared to the wildtype Wuhan strain. And the antibodies from people who were naturally infected were even less effective. The booster shots helped, but still showed decreased neutralizing activity.

Microorganism Helps Understand Cancer Resistance

Scientists atArizona State Universitydescribethe ability of a microorganism,Trichoplax adhaerens, to repair its DNA, even from significant radiation exposure. It also can extrude injured cells, which then die. The research provides insights into natural cancer-suppression mechanisms in a wide range of lifeforms.T. adhaerensis the simplest multicellular organism on Earth and is native to the Red Sea and other warm waters. In addition, its complete genome has been sequenced. No cancer has ever been seen in the organism. They can withstand radiation by increasing the expression of particular genes involved in DNA repair and genes linked with apoptosis (cell death). Their ability to extrude damaged cells, such as precancerous cells, may also explain their ability to fend off cancer.

Epigenetics of Microglia in the Brain

Epigenetics is the study of how the environment or behaviors change the ways genes work. In other words, although genes are sometimes turned on and sometimes turned off, epigenetics is the study of what turns them on or off and any in between states. Microglia are a type of immune cell found in the brain and central nervous system. They were thought for a long time to be activated or inactivated, and their effects were either pro-inflammatory or neuroprotective. But researchers at theIcahn School of Medicine at Mount Sinai, led by Fatemeh Haghighi, Professor of Neuroscience and Psychiatry,isolatedmicroglia cells from post-mortem human brain tissue from 22 people. The patients had a variety of illnesses while alive: one with schizophrenia, 13 with mood disorders, and 8 with no psychiatric disorders. The researchers used genome-scale methylation microarrays to analyze the microglia. Methylation is one form of epigenetic control of genes. They found that microglia demonstrated DNA methylation profiles distinct from other CNS cells, which was expected. But they also found differences in the methylation levels of microglia individually, which suggested that microglial methylation may play a role in a variety of psychiatric disorders.

Antibiotic-Antioxidant Combo Slows Dementia in Mice

Dementia, such as Alzheimers, is believed to be caused by an accumulation of proteins called beta-amyloid, tau and alpha-synuclein, which collect in the brain and form oligomers. Researchers atOsaka City University Graduate School of Medicinehad previouslydescribedthe use of the antibiotic rifampicin to remove oligomers from the brain, which improved cognitive function. But rifampicin can cause liver damage and other side effects. Resveratrol is a naturally occurring plant antioxidant that is used as a supplement in the U.S. and Europe. The researchers thought they could combine the positive effects of rifampicin while fighting its negative effects with resveratrol. They used a fixed dose combination intranasally five days a week for four weeks on mice models of Alzheimers, frontotemporal dementia, and dementia with Lewy bodies. The drugs improved cognition, inhibited oligomer accumulation, and restored synaptophysin levels, which facilitate synapses. In addition, the blood levels of liver enzymes that typically increase with rifampicin stayed normal. A bonus was they observed increased levels of brain-derived neurotrophic factor (BNDF) expression in the hippocampus, which was not typically seen with only rifampicin.

Severe COVID-19 Negatively Affects B-Cell Memory

Researchers at theUniversity of Texas Health Science Centerat San Antoniofoundthat patients who recovered from less-severe cases of COVID-19 had B cells that had better immune memory of the viruss spike protein compared to patients who recovered from severe COVID-19. The researchers analyzed blood samples a month after symptom onset and five months post-onset. At the one-month mark, a significant percentage of spike-specific B cells were active. But in eight people who recovered from less-severe disease, they had increased expression of markers linked with durable B-cell memory compared to people who recovered from severe disease. The markers included T-bet and FcRL5.

The increased percentage of B cells associated with long-lived immunity in non-severe COVID-19 patients may have consequences for long-term immunity against SARS-CoV-2 re-infection or severity of the resulting disease, the authors wrote.

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Give the Gift of Life During the Evan’s Journey Blood Drive with the Red Cross January 21st – wkdq.com

Posted: January 5, 2022 at 2:06 am

The new year is underway. If one of your resolutions is a bigger commitment to helping those who need it, there's no better way than giving the gift of life with a blood donation. The Southwestern Indiana Red Cross is giving you the opportunity to do just that during their upcoming Evan's Journey Blood Drive onFriday, January 21st from 1:00 until 7:00 PMat St. Paul's Lutheran Church in Evansville.

Due to thecontinuing COVID-19 pandemic, as well as the numerous devastating weather events taking place around the country, including the tornadoes that ripped through western Kentucky in December, the Red Cross was forced to cancel a number of blood drives in 2021 which has led toits worst blood shortage in over a decade. According to the Red Cross, someone requires a blood product somewhere in the countryevery two seconds, which is why making a donation is so important.

The Evan's Journey Blood Drive on January 21st is named in honor of Reitz High School student, Evan Meyer who is currently battling lymphoma and knows all too wellhow important the need for a consistent blood supply is. Evan's brother, Wes, is currently donating stem cells as part of Evan's treatment. The family, along with the Red Cross, have set a goal of 75 units of blood for the drive.

Appointments aren't required for the drive but arehighly encouraged. You can make an appointment now by visitingRedCrossBlood.orgor by calling 1-800-RED-CROSS.

The Red Cross also strongly encourages individuals withO+, O-, A-, and B- are to sign up for a Power Reddonation. This donation takes a little longer to complete than a standard donation (approximately 90 minutes) as it separates your red blood cells from the plasma and platelets and then returns the latter back to you through a saline solution. This type of donation doubles the impact of your donation for the patients who need it.

The entire month of January is National Blood Donor Month. In an effort to encourage donations, not just during the Evans Journey Blood Drive, but throughout the month, the Red Cross has teamed up with the NFL to give one lucky donor a trip to this year's Super Bowl in Los Angeles which includes:

Plus, another donor during the month of January will win a home theater package for the ultimate at-home Super Bowl experience which includes:

All you have to do to enter is make a blood donation anytime between now and January 31st. Visit RedCrossBlood.org to make your appointment now for either the Evan's Journey Blood Drive or any other drive taking place near you this month.

Read on to learn the average life expectancy in each state.

The 100 Best Places to Live in the Midwest

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Top 10 stories of 2021 – The Tribune | The Tribune – Ironton Tribune

Posted: January 5, 2022 at 2:06 am

By HEATH HARRISON and MARK SHAFFER

1. COVID-19Everyone wishes it wasnt so, but, unfortunately, the COVID-19 pandemic still dominated headlines this year.

The Lawrence County Health Department worked diligently all year to administer vaccines to the population, hosting clinics at a main location at the South Point Board of Education office, as well as at the health department, in schools and at events throughout the region.

The vaccine was first given to older and at-risk groups, then, by spring, was available to all adults. In the fall, booster shots were available, as well as vaccines for children.

While things seemed to be clearing up in the spring, and the Ironton-Lawrence County Memorial Day Parade and the Lawrence County Fair returned as cases numbers dropped, as the year progressed, both the Delta and Omicron variants of the virus kept the county in the red for high transmission.

Schools struggled with the situation, with the pandemic continuing to impact both the classroom and sports.

All but one school district in the county, Ironton, began the academic year with no mask requirements but, within weeks and cases on the rise, districts changed course and all in the county adopted a policy mandating facial coverings for students and staff.

As the Christmas holiday began, the health department reported that Lawrence County has seen 11,269 cases of COVID-19 since the pandemic began, 721 hospitalizations and 182 deaths.

Ohio Gov. Mike DeWine tours parts of Lawrence County that were hardest hit by Februarys three winter storms. (Submitted photo)

2. Winter stormsThe Tri-State area was brought to a standstill in February, with three winter storms hitting the region hard.

The first brought ice, the second, the most severe, had ice and snow, and a third consisted of only snow.

Residents throughout the area listened as the sounds of buckling trees and branches filled the night during the first two storms. Thousands were left without power and many roads were blocked and impassible.

Restoration of power was a long wait for many in rural parts of the county, with remote lines and poles presenting a challenge for crews from AEP and Buckeye Rural.

In the immediate aftermath of the storms, Gov. Mike DeWine visited Lawrence County, hosting a press conference at South Point High School before touring the hardest hit parts of the county. DeWine activated the National Guard to assist the county in its cleanup.

The impact of the storm lingered into late spring, with volunteer groups, such as teens from Maumee United Methodist Church, located in northern Ohio, continuing to help with cleanup on private properties.

3. New Lawrence County JailBuilt in 1974, the Lawrence County Jail was state of the art at the time but as the years passed, the demand for more cells increased and there wasnt a workable solution until October, when Gov. Mike DeWine announced the county will be getting $16.8 million to fund a new jail facility. Details about total costs, location and when construction could possibly start are still being worked out. DeWine was supposed to make an appearance in Lawrence County but canceled after being exposed to someone with COVID-19.

4. Chesapeake upheaval

New Chesapeake Mayor Nate Ittig and the newly appointed members of village council, along with incumbent council member Paul Hart, at Novembers regular meeting. (The Ironton Tribune | Heath Harrison)

2021 was a chaotic year for Chesapeake. It began with the village council removing Randy Thompson as police chief in February, replacing him with Steven Woodyard.

Then, in the summer, a recall petition against Mayor Kim Oldaker was filed, but ruled invalid by the county board of elections.

In October, the village was left with little of a government when Oldaker and four council members abruptly resigned, citing disagreements with council member Paul Hart, as well as those who were running unopposed for three seats in the November election.

With the resignations, council member Nate Ittig became acting mayor, leaving Hart as the sole member of council.

At Novembers council meeting, Ittig, now mayor, moved to fill the vacancies, naming the three candidates, Lisa Blake, Jacob Wells and Drew Griffin who were set to start their terms in January, to immediately fill those seats. They were joined by Katie Bentley and Marvin Henson, who Ittig chose for the other two vacancies, one of which was his former seat.

Following the appointments, at the same meeting, the council then voted not to retain Woodyard as police chief. This was met with the resignation of two officers.

Sgt. Josh Sammons, the highest ranking of the remaining officers, was named as interim chief.

The year also saw the resignation of street commissioner Danny Burd and fiscal officers Theresa Lawless and Lennie Abrams. Abrams returned when he was named as interim fiscal officer in December, in order to continue to help the village prepare for an audit from the state.

5. Grocery store changesIn April came surprising news that the Bartram and Son grocery store was being closed and the staff was being merged with those at a new Food Fair grocery store in the Ironton Hills Plaza, the site of the former Pick n Save, which closed in the spring.

Bartram and Son was bought by Forth Foods Inc., which owns 16 stores in the area. Tim Forth, the owner and president of Forth Foods Inc. and owner of Ironton Food Fair, said the two companies had worked together for many years and they ran with the Food Fair program, although they chose to run it under the Bartram and Son name.

Maddie Cogan and Robbie Brown are owners of the Ironton Shake Shoppe. (Mark Shaffer | The Ironton Tribune)

6. Shake Shoppe lawsuitOne of Irontons most popular businesses found itself sued in federal court. Maddie Cogan and Robbie Brown, owners of the Shake Shoppe in Ironton since 2019, were sued by Shake Shoppe LLC, the company of the Gallipolis Shake Shoppe, which has been owned by three generations of the Snedaker family since the 1950s.

In the 1950s, six Dairy Queens in southern Ohio, located in Ironton, Gallipolis, New Boston, Jackson, Logan and Portsmouth, saw the patent on their soft serve machine with the company expire. As a result, the six businesses decided to rebrand as the Shake Shoppe, forming a business agreement. They used the same signage, but were individually owned. Of the remaining three locations, only the Gallipolis is run by the same family who originally owned it.

In the suit, Shake Shoppe LLC cited slogans and imagery, which they said were drawn by hand by original owner Marge Snedaker and used as early as 1956. Shake Shoppe LLC had those items trademarked with the state and federal government.

The logos and designs were made into digital renderings and have been used by Shake Shoppe LLC since at least 2015 on social media sites and to make clothing items. The use of the logos on social media, on the Ironton shop and on T-shirts is the issue in the suit. The name and imagery have been used by the Ironton location since the 1950s, under its original owners, the Salisbury family, then two successors, before Cogan and Brown bought the location in 2019.

News of the lawsuit saw a surge of support for the Ironton location, with posts on social media, supportive T-shirt sales and an increase in traffic by those who wanted to back the location.

7. Steve Dodgion retires

New Collins Career Technical Center superintendent Adam Pittis, left, took over after Steve Dodgion, right, retired in the summer. (Submitted photo)

One of the most significant figures in education in Lawrence County wound down his career in 2021, when Steve Dodgion, the superintendent of Collins Career Technical Center for 19 years, retired in July.

Dodgion worked in Lawrence County schools for 47 years, with all but five of those being at CCTC, where he also served as assistant superintendent before taking the top job.

He was hailed by faculty for his work expanding CCTCs offerings and overseeing a growth in its student body, with the school serving more students than any other vocational institution in Ohio.

In addition to being hailed for developing CCTCs health programs, Dodgion said he was most proud of launching the Project Lead the Way STEM program 14 years ago, one of the first in the state to do so.

As Dodgion departs, the PLTW program is set to expand with the addition of an aviation program.

He was succeeded by Adam Pittis as superintendent.

8. Live events returnIn 2020, most local live events were downsized or canceled altogether because of state orders about large groups gathering in an effort to curtail the spread of COVID-19.

After the COVID-19 vaccine roll out in the spring, many events returned in full force like the annual Ironton-Lawrence County Memorial Day Parade and its associated events, the Lawrence County Fair, Ironton Wizardfest and the Ohio River Revival.

Brad Bear, one of the organizers of the Wizardfest, said crowds were happy to be back in Ironton and take it to the next level.

Its going really, really good. Its excellent, its fantastic, he said. Everybody came back to town and is having a great time.

Academic events also saw a return in 2021, with the county spelling bee and Quiz Bowl events making a comeback and schools returning to more traditional graduation ceremonies.

9. InfrastructureThere was a lot of infrastructure work in the area in 2021.

The new Russell, Kentucky viaduct bridge was completed in November and Kentucky Gov. Andy Beshear made a surprise appearance to open it up.In South Point, the Solida Road roundabout is being expanded by the Ohio Department of Transportation.

And ODOT announced a proposed roundabout for the intersection of U.S. 52 and State Route 93 in Ironton in an effort to reduce the number of crashes in the area. The plan calls for the construction of two single-lane roundabouts on State Route 93, one on the north and south sides of the U.S. 52 interchange, replacing signalized intersections. These would be designed in 2022, with construction expected to start in May 2023 and wrap up that October.

The Tower House in Ironton. (Mark Shaffer | The Ironton Tribune)

10. Tower House on TVThanks to an Instagram post, one local house gathered a lot of attention on social media and even ended up featured on national television.

Irontons Tower House was featured on HGTVs Cheap Old Houses, which follows Instagram stars Ethan and Elizabeth Finkelstein as the tour the country looking at houses that sell for under $150,000. The show is based on the Finkelsteins Instagram page, also called Cheap Old Houses, which has 1.4 million followers.

The Tower House, a gray four-story house with a tower that leans slightly, was built in 1874 by ironmaster Frederick Norton, the owner of the Norton and Belfont Iron works. Architectural details include a cameo etched into the front door knob, fretwork molding and the floors are made with yellow poplar, the same type of wood used in the iron blast furnaces.

The whole media sensation began with Heather Williams, a realtor with the Proctorville-based Bunch Real Estate Associates. She was in the process of selling The Tower House and had posted it online and then the Finkelsteins saw it.

I guess they saw the Tower House after I posted it on Historical Houses of America, Williams said. They contacted me and they had chosen, I think five houses in Ohio, and this was one of them.

In our upcoming Wednesday edition of The Ironton Tribune, we will look at notable residents of the Tri-State who passed in 2021.

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TCR2 Therapeutics, Arbor Biotechnologies Partner on TRuC-T Cell Therapies – Contract Pharma

Posted: January 5, 2022 at 2:02 am

TCR2Therapeutics Inc., a clinical-stage Cell therapy company with a pipeline of novel T cell therapies for cancer patients suffering from solid tumors, and Arbor Biotechnologies, a Biotechnology company discovering and developing genetic medicines, have entered into a strategic research collaboration and non-exclusive license agreement focused on the further development of a defined set of allogeneic TRuC-T cell therapies.The collaboration leverages Arbors proprietary CRISPR Gene -editing technology, which is tailored to address the underlying pathology of genetic diseases, and TCR2s TRuC platform, which has demonstrated clinical activity in multiple treatment-refractory mesothelin-expressing solid tumor indications with its Lead autologous program gavo-cel.Our autologous TRuC-T cells have already established clinical activity in multiple difficult-to-treat solid tumors without being dependent on HLA, thus allowing our therapies to be used on the broadest patient population. We believe allogeneic TRuC-T cell therapies will further extend this impact by accelerating patient access while reducing manufacturing cost, said Garry Menzel, Ph.D., president and chief executive officer of TCR2 Therapeutics . The collaboration with Arbor enables us to evaluate multiple allogeneic candidates with novel enhancements and advance a lead candidate in 2022, consistent with our vision of continuing to innovate novel therapies for cancer patients suffering with solid tumors."The collaboration with TCR2allows us to leverage Arbor's proprietary discovery engine and gene editing technologies with an established Cell Therapy leader developing life-changing treatments for serious cancers, said Devyn Smith, Ph.D., chief executive officer of Arbor. This agreement reinforces the versatility and strength of Arbors platform and furthers our strategic vision of expanding the impact of Arbors gene editors through partnerships with leading organizations developing engineered cell therapies.Under the terms of the agreement, Arbor will receive an upfront cash payment and is also eligible to receive additional milestone payments based upon the successful achievement of development, regulatory and commercial milestones across a selected number of programs. In addition, TCR2will pay tiered royalties on future net sales on any products that may result from this collaboration.

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Dystrogen Therapeutics Announces First in Man Dosing of Novel Chimeric Cell Therapy for Duchenne Muscular Dystrophy and Reports 6-week Clinical…

Posted: January 5, 2022 at 2:02 am

MIAMI, Jan. 4, 2022 /PRNewswire/ --Dystrogen Therapeutics Corp. a clinical-stage company developing chimeric cell therapeutics to modify the course of rare diseases, today announced dosing of the first patient in its Phase1 Human PilotClinical Study of DT-DEC01, Dystrophin Expressing Chimeric cells, or DEC, for the treatment of Duchenne Muscular Dystrophy (DMD).

The Phase1 Human PilotStudy is a non-randomized trial enrolling boys between the ages of 5 and 18. The trial investigates the safety, tolerability, and efficacy of DT-DEC01. The pilot trial will enroll a total of 10 patients with DMD across 3 escalating doses. The study will also assess NorthStar Ambulatory Assessment (NSAA), range of motion (ROM), performance of upper limb (PUL), 6MWD, Muscle strength by myometry and 5-grade Lovett scale, and cardiac muscle evaluation by ECHO.

The first patient, a 6-year-old boy with deletion of exons 3-12, received an infusion of DT-DEC01 on November 26th, 2021. The patient tolerated the procedure well and was discharged home. At day 7, day 28, and 6 week follow up, the patient demonstrated no side effects from the treatment. Functional outcomes, including ECHO, demonstrated a non-clinically significant improvement when compared to pretreatment baseline. Subjective parent reported assessment of post - treatment daily activity level demonstrated a significant improvement when compared to pretreatment baseline at 6 weeks. Subjective therapist reported assessment of post - treatment quality of motion as well as patient performance during functional assessment demonstrated a significant improvement when compared to pretreatment baseline at 6 weeks. At 6 weeks, laboratory data demonstrated no signs of inflammation, infection, antibody formation, or other adverse systemic effects on end organs.

Screening and enrollment of patients is expected to continue over the course of the next 12 months in the European Union, with data from this trial expected once patients have been evaluated for one full year post-treatment.

"We are very pleased to begin dosing patients in our pilotstudy. This is the first time we will assess our chimeric cell therapy in Duchenne patients, which will provide us not only with safety data but could also give us an early look into the potential efficacy of this treatment in possibly halting disease progression and even partly restoring functional loss in boys with muscular dystrophy", saidKris Siemionow, MD, PhD Founder and Chief Executive Officer of Dystrogen Therapeutics.

"This is an important step toward bringing the promise of this novel technology to patients with serious diseases like DMD, and we are thrilled to be leading the effort in what we believe may be a fundamental change in the treatment of debilitating disorders", saidMaria Siemionow, MD, PhD Founder and Chief Scientific Officer of Dystrogen Therapeutics.

About DT-DEC01

DT-DEC01 is a chimeric cell therapy. The advanced therapeutic medicinal product (ATMP) is made using Dystrogen's proprietary cell engineering technology which creates a DEC cell. Clinically, DEC cells have been shown to express CD56 at significantly higher levels than myoblasts from Duchenne patients. DEC cells express favorable HLA characteristics which carries multiple advantages. In preclinical studies, DEC cells have also been shown to express clinically significant levels of dystrophin when compared to controls. DEC cell therapy demonstrated significant functional improvement in cardiac, diaphragm, and other skeletal muscle strength and associated function in preclinical trials. Because DEC therapy is designed to prevent triggering an immune system response, a major advantage of DEC therapy is that it does not require immunosuppression. The therapy is not associated with any genetic manipulation and therefore involves no risk of off target mutation, does not use viral vectors, and its use is not dependent on the genetic mutation of the DMD patient, thus making DEC a universal therapy for all DMD patients.

About Dystrogen Therapeutics Corp

Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing therapies for rare genetic diseases. The company was founded based on the pioneering work of Prof. Maria Siemionow, a world-renowned scientist and surgeon who led the team that performed the first near-total face transplantation in the United States. Professor Siemionow's research focused initially on the creation of chimeric cells which have a role in modulation of the immune system's response to a transplant. This led to the development of Dystrophin Expressing Chimeric (DEC) cell therapy that is designed to prevent the immune system from attacking the chimeric cells. DECs are engineered cells and belong to a family of therapeutic technologies called Advanced Therapy Medicinal Products (ATMP). Using Dystrogen's patented cell engineering technology, DECs are made by combining a malfunctioning cell of the Duchenne patient with a normal, working cell from a healthy donor. This novel chimeric cell is composed of both the donor's and the recipient's cell structures but looks to the patient's immune system like his own cell and thus does not trigger an immune response while it functions (i.e. produces dystrophin) like the patients' normal cell. This offers a unique advantage and allows the patient's body and immune system to accept the chimeric cell without rejection. In such a way, Dystrogen has created dystrophin producing cells that can engraft in the patient's muscles (such as heart, diaphragm, skeletal muscles) and, as demonstrated in our research and related peer-reviewed publications, increase their dystrophin levels. Increased dystrophin levels have been shown to correlate with improved functional outcomes which was confirmed in preclinical studies of DEC.

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Erbi Biosystems Raises $4M to Provide Small-Scale Perfusion Bioreactors for Development and Manufacture of Cell Therapies – PRNewswire

Posted: January 5, 2022 at 2:02 am

CAMBRIDGE, Mass., Jan. 4, 2022 /PRNewswire/ -- Erbi Biosystems, developer of innovative, small-scale, automated, and high-performance cell culture systems, has closed a private round of funding totaling $4M. This latest financing will enable the company to expand operations to meet the growing demand for its Breez perfusion bioreactor product and develop extensions to enhance cell therapy applications.

The Erbi Breez bioreactor provides continuous media addition, cell retention and bubble-free gas diffusion through its True Perfusion technology, which is unique among existing small-scale bioreactors that can only approximate perfusion with intermittent media exchange. The Breez's fully automated, sterile, single-use system also includes online sensing and controls that are combined with easy-to-use software, delivering sophisticated feeding strategies, cell concentration, and media exchanges, resulting in performance that can exceed stirred tank and rocking bag reactors.

"Erbi has developed a compelling platform for small-scale cell culture," said Michael Chambers, Erbi investor and founding CEO of Aldevron. "In addition to its capabilities in bioprocessing, their platform enables testing of multiple replicates from a single sample of scarce patient cells - a unique and powerful capability that will accelerate cell therapy development."

"The Breez product has proven to be an effective scale-down tool for process development across a range of cell types," said Dr. Martin Madaus, former Chairman and CEO of Millipore. "The small volume, single-use, sterile and fully-automated format makes this an ideal platform for cell therapy manufacturing."

In 2021, Erbi Biosystems doubled its staff and relocated to a larger facility north of Cambridge,equipped with biolabs and cleanroom manufacturing space.

"We're growing quickly and expect to double again in 2022," said Dr. Michael Chiu, CEO of Erbi Biosystems. "Revenue from our growing list of Breez customers in the US, Europe and Asia is fueling our growth. This additional funding allows us to accelerate our pipeline of technology and product development projects which are focused on the needs of the cell therapy process development and GMP manufacturing."

Mr. Chambers and Dr. Madaus are both investors in the latest private round.

About Erbi Biosystems

Erbi Biosystems supports therapeutic drug manufacturers with tools to accelerate the developmentof their processes and therapies. Erbi's technology platform includes single-use, mL-scale cell culture, microfluidics, optical sensing, and integrated controls.

The Breez bioreactor is used across a range of applications and cell types, including media and cell-line development; intensified process and n-1; CAR-T process development; and non-mammalian cell culture. The small size, single-use device, and fully automated operation drastically reduce the time, media consumption and lab space required for process development. The mL-scale working volume and closed-sterile consumable format make it ideal when working with scarce and highly variable source materials, such as autologous therapy development.

The Erbi Breez system is available worldwide with sales and distribution in the US, EU, and China.

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Erbi Biosystems Raises $4M to Provide Small-Scale Perfusion Bioreactors for Development and Manufacture of Cell Therapies - PRNewswire

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Cell therapy, AI drug discovery highlight 2021’s record year in biotech IPOs – MedCity News

Posted: January 5, 2022 at 2:02 am

The biotech IPO market has been hot for the past few years, but it set all sorts of new records in 2021. A total of 399 companies priced IPOs in 2021, raising $142.5 billion, according to IPO research firm Renaissance Capital. Those figures made this year the busiest by deal count since 2000, and the biggest ever in terms of proceeds raised.

Healthcare, which includes biotech, was the busiest sector, accounting for 36% of the IPO activity, Renaissance said in its 2021 annual review. The biggest biotech IPO of the year belongs to Seattle cell therapy developer Sana Biotechnology, which was able to upsize an IPO that raised $588 million.

While Sanas IPO total is eye-catching, the sum is also notable because its the largest ever IPO for a company that has yet to reach the clinic. Not so long ago, biotech companies typically waited until they had clinical data before turning to the public markets. Even early dosing data reduces the investment risks.

Sana still doesnt have clinical data. What it does have is a cell and gene therapy technology and leadership from two former Juno Therapeutics executives who steered that company to a $9 billion acquisition by Celgene in 2018. Sana said it is preparing to file multiple investigational new drug applications in 2022 and 2023. That means investors must wait another year or more before the company produces data that could move the companys stock price.

Other cell therapy developers that went public this year include Lyell Immunopharma, Caribou Biosciences, Instil Bio, Talaris Therapeutics, and Achilles Therapeutics. Most of the biotechs in this group had reached at least early clinical testing at the time of their IPOs.

Recursion Pharmaceuticals had the second biggest biotech IPO of 2021. The Salt Lake City, Utah-based company raised $436.4 million to continue clinical development of drug candidates discovered by its AI platform. Similar to Recursion, Exscientia has an AI-based drug discovery platform that it uses to develop drug candidates for partners as well as its internal pipeline. The Oxford, U.K.-based company raised $304.7 million from its IPO. Absci, which uses its AI platform to produce proteins that become drug candidates for its pharmaceutical industry partners, raised $200 million in its stock market debut.

According to Renaissance, IPO filings increased for the third straight year; the 495 IPOs that were filed in 2021 through Dec. 20 is a more than 94% increase compared to the same period last year. The $142.5 billion raised is an 82.2% increase over last years total.

The year was also marked by a surge in companies going public by merging with special purpose acquisition companies (SPACs). Renaissance counted nearly 200 companies such deals. Biotech companies that went public in 2021 via SPAC deals include Nautilus Bio, Humacyte, Ginkgo Bioworks, and Pardes Biosciences.

As we head into a new year, the hot IPO market appears to be cooling off. While the market wont turn completely cold this winter, Renaissance doesnt think investors should expect a repeat of 2021s IPO activity.

Looking forward, we believe the 2022 IPO market will have a slow start and fail to match the past years record pace, but ample IPO candidates are ready to take the leap once conditions improve, the firm said.

Photo: Spencer Platt, Getty Images

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Avalere Analyst Weighs in On How Value Assessments Will Influence Gene and Cell Therapy Pricing – AJMC.com Managed Markets Network

Posted: January 5, 2022 at 2:02 am

Megan Olsen, MPH, principal at Avalere, discusses the role that value assessments are expected to play in the future pricing of gene and cell therapies.

Megan Olsen, MPH, principal at Avalere, discusses how the impact of small patient populations and other limitations may influence payer conversations about gene and cell therapies.

Transcript

What role will value assessments play in payer decisions on pricing for cell and gene therapies?

It's going to play an important role. And I think it already is today, to some extent. We've done some survey research with payers at Avalere and found that most payers are already doing their own valuations for products in this space. We've also seen other entities like [the Institute for Clinical and Economic Review] be referenced as a key stakeholder at informing valuation of various products in this space. When you have an introduction of a high cost potentially million dollar therapy, with the potential for the benefit over many years, it's going to get a lot of attention you'll want to understand what is the true value of the product relative to price. So, there are a lot of different frameworks under consideration for how to measure that and how to adapt various methodologies or framework specific to the cell and gene therapies, given the uniqueness of the asset.

There's a lot of evolving in the value-based space in the private market, but also in the policy sphere as well. We're seeing the new Biden administration emerge with its priorities in health care and I think value-based care is going to be at the top and we've heard references in the drug pricing arena on interest in tying prices to value in some way or leveraging or standing up a value assessment entity to really measure value and potentially have a role in informing price as well. So, I think a lot more to come there. And this whole swirling interest in innovative financing models is based on value and wanting to determine net pricing based on the value that the product is delivering to the patient, to the health care system, to the payer, etc. So, needing that data to to make those determinations but also using the data that is derived from these arrangements to inform future value conversations as well.

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BioLife Solutions Collaborates with Seattle Children’s to Improve Viral Vector and Cell and Gene Therapy Manufacturing – Yahoo Finance

Posted: January 5, 2022 at 2:02 am

Sexton Cell Processing tools and CryoStor cGMP Freeze Media Incorporated in Cell and Gene Therapy Workflows

BOTHELL, Wash., Jan. 4, 2022 /PRNewswire/ -- BioLife Solutions, Inc. (NASDAQ: BLFS), a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy and broader biopharma markets, today announced an extended collaboration with Seattle Children's Therapeutics, a venture at Seattle Children's, bringing cutting edge, curative technologies and therapies to defeat pediatric cancer and other diseases that impact children. The collaboration will focus on establishing best practices in biopreservation and closed-system manufacturing with the introduction and integration of Sexton's AF-500 for closed-system processing for cell therapy manufacturing and viral vector delivery that will be used in Seattle Children's Therapeutics new lentiviral vector manufacturing facility called VectorWorks.

BioLife Solutions New Logo 2021 (PRNewsfoto/BioLife Solutions, Inc.)

"This expanded collaboration is an exciting step for Seattle Children's Therapeutics as we work towards developing closed-system processes for cell therapy manufacturing," said Matt Selley, Director of GMP Manufacturing at Seattle Children's. "Lentiviral vector manufacturing is a critical component of the cell production process."

Seattle Children's has utilized BioLife's CryoStor cGMP freeze media for several years, as an optimized excipient for improved post-thaw viability and functional recovery of cells used in clinical applications and trials. As cell and gene therapy manufacturers move toward closed-system processing, it is vital that upstream critical excipients and ancillary materials, such as viral vectors, are manufactured and packaged in containers suitable for closed-system integration. While some upstream bioprocesses have been successfully automated, the final steps of downstream bioprocess, namely fill-finish, are often performed manually in open systems with associated risks of contamination and user error. Furthermore, current packaging for viral vector intermediates demands that therapy developers operate in higher grade environments due to the open nature of this manufacturing step.

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This continued collaboration will combine the Sexton off-the-shelf automated fill-finish system, AF-500, with Seattle Children's Therapeutics' expertise in viral vector manufacturing. Sexton's rapidly deployable AF-500 is capable of filling and sealing up to 560 CellSeal vials in 90 minutes. The goal is to utilize the AF-500 to initiate high throughput fill-finish of vector intermediates. Vectors will be filled into Sexton's proprietary vials, CellSeal and CellSeal Connect. CellSeal Connect builds on the original CellSeal cryogenic storage vial, which has been incorporated as the final drug packaging in commercial cell therapy products. The new version allows closed-system retrieval of intermediate products, such as viral vectors, thereby negating the need for therapy developers to operate in higher grade manufacturing suites for delivery of vectors and cargo.

The collaboration will result in detailed workflows, demonstrating the suitability of the CellSeal platform as a packaging container and closed system automation for viral vectors. It will include an assessment of the vial and fill system's usability and compatibility with high throughput fill-finish of viral vectors. In addition, post-fill activities such as high-density storage, shipping, distribution, thawing, and closed system retrieval will be assessed. As Seattle Children's Therapeutics will be the end-user of the viral vector product, the workflow resulting from the collaboration will cover the movement of viral vectors from the point of packaging to the point of transduction and patient administration, when applicable.

"The development of these closed connection processes in small volume aliquots has the potential to streamline this element of cell therapy manufacturing," said Sean Werner PhD, Chief Technology Officer, Cell Processing at Biolife Solutions. "Developing tools to meet the unique needs of this emerging industry is the best way to bring these life changing therapies to patients."

About BioLife SolutionsBioLife Solutions is a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy and broader biopharma markets. Our tools portfolio includes our proprietary CryoStor and HypoThermosol biopreservation media for shipping and storage, the ThawSTAR family of automated, water-free thawing products, evo cold chain management system, high capacity cryogenic storage freezers, Stirling Ultracold mechanical freezers, SciSafe biologic storage services, and Sexton Biotechnologies cell processing tools. For more information, please visit http://www.biolifesolutions.com, http://www.scisafe.com, http://www.stirlingultracold.com, or http://www.sextonbio.com and follow BioLife on Twitter.

About Seattle Children's Seattle Children's mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children's Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top children's hospitals in the country by U.S. News & World Report, Seattle Children's serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho the largest region of any children's hospital in the country. As one of the nation's top five pediatric research centers, Seattle Children's Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Children's Foundation works with the Seattle Children's Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. Join Seattle Children's bold initiative It Starts With Yes: The Campaign for Seattle Children's to transform children's health for generations to come.

For more information, visit seattlechildrens.org or follow us on Twitter, Facebook, Instagram or on our On the Pulse blog.

Cautions Regarding Forward Looking Statements

Except for historical information contained herein, this press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, statements concerning our extended collaboration with Seattle Children's and the results of such collaboration. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements, including among other things, those factors described in our risk factors set forth in our filings with the Securities and Exchange Commission from time to time, including our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We undertake no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.

At the CompanyTroy WichtermanChief Financial Officer(425) 402-1400twichterman@biolifesolutions.com

InvestorsLHA Investor RelationsJody Cain(310) 691-7100jcain@lhai.com

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GC Cell Inks First Licensing Agreement for Immuncell-LC With Rivaara Immune Private Limited in India – BioSpace

Posted: January 5, 2022 at 2:02 am

- GC Cell seeks to expand global availability of Immuncell-LC for liver cancer

- Rivaara Immune to lead development and commercialization in India

YONGIN, South Korea & MUMBAI, India--(BUSINESS WIRE)-- GC Cell (KOSDAQ:144510) and Rivaara Immune Private Limited today announced that they have entered into an exclusive licensing agreement, pursuant to which Rivaara Immune has agreed to develop and commercialize Immuncell-LC in India, Sri Lanka, and Bangladeshi. Developed by GC Cell, Immuncell-LC is a patient-derived autologous T-cell immunotherapy for liver cancer, which was approved by the Korean MFDS back in 2007.

Under the terms of the agreement, Rivaara Immune has obtained the exclusive right to develop and commercialize Immuncell-LC within India for liver cancer. In return, GC Cell is entitled to receive an undisclosed upfront payment, as well as payments for the achievement of commercial milestones, with royalties based upon the sales.

In addition, GC Cell will also receive a partial stake of Rivaara Immune and exclusive rights to supply media which is essential for the production of Immuncell-LC.

The number of cancer patients in India is more than 1.3 million, about 6 times that of Korea, and India's liver cancer market is 2.3 times that of Koreas. Notably, 5-year liver cancer survival rate is 4%, which is very low compared to 37% of Korea, so new treatments are urgently needed.

Immuncell-LC's overseas expansion is based on its domestic success, pushing for technology transfer by country, and signing contract with Rivaara Immune in India is the first outcome, said Dae-woo Park, CEO of GC Cell. Both companies will cooperate in a win-win strategic partnership, and are also closely discussing with a number of partners in China and other countries in the Middle East.

Rivaara Immune is pleased to partner with GC Cell, a leader in Immune Cell Therapy in Korea. With this partnership we will be among the first, to bring to the Indian market, customized solutions to cater to the unmet needs of the liver cancer sufferers, said Syd Daftary, Director of Rivaara Immune.

About Immuncell-LC

Immuncell-LC is a customized anti-cancer drug that is made from a patients blood. Professionals extract a sample of a patients T-cells from the blood and genetically modify it into a robust immune cell that has maximized anticancer functions and is incubated for about two weeks before they infuse it into the patient. Immuncell-LC received approval for the liver cancer immunotherapy from the Korean MFDS in 2007. It also received FDA Orphan Drug Designation for the Treatment of liver cancer, brain tumor, and pancreatic cancer. More than 7800 individuals have been treated with Immuncell-LC which recorded the highest selling of anti-cancer immune cell therapy in Korea.

About GC Cell

GC Cell is an integrated corporation created through the recent merger of Green Cross Labcell and Green Cross Cell, and is focusing on the development and production of treatments using immune cells and stem cells. In particular, it has a variety of pipelines for autologous and allogeneic cell therapies. It also has global competitiveness based on platform technology and experience throughout the entire period from the initial research stage to the commercialization stage.

About Rivaara Immune Private Limited

Rivaara Immune is a part of the Bharat Daftary group of healthcare companies and aims to be the leading and trusted provider of specialised cell therapy/Immunotherapy for patients with cancers and offer them an improved quality of life.

This press release may contain forward-looking statements, which express the current beliefs and expectations of GC Cell's management. Such statements do not represent any guarantee by GC Cell or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Cell undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220103005504/en/

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