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Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome

Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis

TSHA-102 anticipated to submit Investigational New Drug application in 2021

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.

Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 cases in the U.S. and European Union (EU). It is an X-linked disease that primarily occurs in females, but it can be seen very rarely in males. It is usually recognized in children between six to 18 months of age as they begin to miss developmental milestones or lose abilities they had developed. Individuals with Rett syndrome also show symptoms that include loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments, breathing issues and sleep disturbances.

“Patients with Rett syndrome are currently managed with symptomatic treatments as there are no therapies approved to treat the underlying cause of disease,” said Berge Minassian, M.D., Chief Medical Advisor of Taysha and Chief of Pediatric Neurology at the University of Texas Southwestern Medical Center (UT Southwestern). Dr. Minassian is credited with describing the CNS isoform of the MECP2 gene which is responsible for neuronal and synaptic function throughout the brain. “Gene therapy offers a potentially curative option for patients suffering with Rett syndrome.”

Rett syndrome is caused by mutations in the MECP2 gene. TSHA-102 is designed to deliver a healthy version of the MECP2 gene as well as the miRNA-Responsive Auto-Regulatory Element, miRARE, platform technology to control the level of MECP2 expression. “TSHA-102 represents an important step forward in the field of gene therapy, where we are leveraging a novel regulatory platform called miRARE to prevent the overexpression of MECP2,” said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “In collaboration with Sarah Sinnett, Ph.D. to develop miRARE, our goal was to design a regulated construct that allowed us to control MECP2 expression to potentially avoid adverse events that are typically seen with unregulated gene therapies.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are

Reflow Medical Receives Approval in Japan for the Wingman Catheter to Cross Chronic Total Occlusions (CTOs) in Peripheral Artery Disease

Reflow Medical, Inc., a California-based medical device company, announced that Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has approved the Wingman™ CTO Catheter. Reflow Medical has partnered with Century Medical, Inc. (CMI), a leading medical device distributor based in Tokyo, to introduce the Wingman CTO Catheter in Japan.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201008005741/en/

Wingman CTO Catheter (Photo: Business Wire)

The Wingman Catheter crosses peripheral CTOs using an extendable beveled tip. The physician controls the advancement and activation of the tip to create a channel to help penetrate, or cross, the occlusion with a guidewire, enabling further treatment of the lesion with therapeutic devices. The catheter is compatible with the physician’s preferred guidewire and procedural technique.

Approval by Japan’s PMDA follows the completion of the Wing-IT CTO clinical trial, a prospective, international, multicenter study that treated 85 patients and followed them for 30 days. The Wingman Catheter was able to demonstrate a 90% crossing rate when up to two previous guidewires could not cross the CTOs, meeting its primary safety and efficacy endpoints.

“We are very excited to launch the Wingman CTO catheter in the Japanese market,” said CMI President and CEO Takahiko Motani. “This product should have a significant impact on the Japanese peripheral market by helping physicians address specific treatment challenges within a growing patient population. We look forward to providing this new and unique device in partnership with Reflow Medical, Inc.”

“Reflow Medical focuses their efforts on developing technologies based on physicians’ needs,” said John R. Laird, MD, of Adventist Heart and Vascular Institute in St. Helena, California. Dr. Laird was the principal investigator for the study. “We rely on Reflow for real clinical evidence when utilizing the technology.”

“It is an honor for our device to have gained approval by Japan’s PMDA,” noted Reflow Medical Co-founder and CEO Isa Rizk. “We are grateful for the opportunity to partner with a fantastic organization like Century Medical.”

Reflow’s Wingman Crossing Catheter received clearance from the U.S. Food and Drug Administration for the expanded CTO indication for the Wingman Crossing Catheter in March 2020 after completing the Wing-IT clinical trial.

About Reflow Medical, Inc.

Reflow Medical, Inc. is a private company focused on empowering physicians through the design and development of innovative and effective technologies for cardiovascular disease. Reflow is developing a family of products to treat cardiovascular disease.

About Century Medical, Inc.

Century Medical, Inc. (CMI) is one of the largest independent medical device distributors in Japan, with more than 40 years of experience marketing medical devices. CMI has been a pioneering force, successfully introducing many new technologies to the Japanese healthcare community. Century Medical, Inc, is a wholly owned subsidiary of ITOCHU Corporation.

About Chronic Total Occlusions (CTO) in Peripheral Artery Disease (PAD)

Chronic total occlusions (CTO) of the lower extremities are found in 40% of symptomatic peripheral artery disease (PAD) patients. Endovascular options for the treatment of a CTO can be complex; conventional wire and catheter technique have a

Colorado Medical Waste Receives Environmental Leadership Award

An Environmental Leader in the State of Colorado going above and beyond environmental compliance

The Colorado Department of Public Health and Environment (CDPHE) Division of Environmental Health and Sustainability awards Colorado Medical Waste with the Environmental Leadership Program Silver Award. The ELP is a statewide environmental recognition and reward program for facilities that voluntarily go above and beyond compliance of state and federal regulations and are committed to continual environmental improvement for their business and communities as well.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201007005268/en/

The ELP logo stencil was sprayed at their facility in Aurora along with a photo taken of available employees. (Photo: Business Wire)

A video by Colorado Governor Jared Polis was presented to virtually celebrate the program’s new members. The ELP logo stencil was sprayed at their facility in Aurora along with a photo taken of available employees.

Inclusion in the CDPHE Environmental Leadership Program was awarded to Colorado Medical Waste for demonstrating its commitment to:

Using the natural oxidizing power of ozone, electricity, and an industrial shredder, waste volume is reduced by 90% to a sterile confetti residual with “ZERO” emissions as ozone reverts back to simple oxygen. Tons of medical waste streams are diverted from landfills, incinerators, and hazardous waste facilities. State of the art processes and technologies reduce the public health effects and environmental impact of traditional autoclave and incineration technologies. Efficacy tests prove ozone is 100x more effective than steam and an environmental alternative to incineration. Reduction of landfilled medical waste and incineration decreases methane greenhouse gas emissions and their contribution to global warming and climate change. Colorado Medical Waste and ozone processing bring medical waste management into the 21st century.

Beverly Hanstrom, the company CEO and owner says, “We are leading the industry and are at the forefront of environmental stewardship to reduce the carbon footprint of healthcare and medical waste. Our leadership raises awareness and exemplifies our passion and commitment to make a difference.”

Find us on:
Facebook: https://www.facebook.com/ColoradoMedicalWaste
Twitter: https://twitter.com/COmedwaste

Video – https://www.youtube.com/watch?v=6e9TxcTAO0c
Photos – https://www.coloradomedicalwaste.com/press-media/

Related Links:

Environmental Leadership Program: https://www.colorado.gov/cdphe/environmental-leadership-program
Environmental Leadership Brochure: https://drive.google.com/file/d/1RQq1EHIippch2lVgsn0C95ZDzHyll7wi/view?usp=sharing

View source version on businesswire.com: https://www.businesswire.com/news/home/20201007005268/en/

Contacts

Beverly Hanstrom, CEO/Owner
Colorado Medical Waste, Inc.
3131 Oakland St.
Aurora, CO 80010
303-794-5716
(303) 763-2339 Fax
Website: www.coloradomedicalwaste.com
Email: [email protected]
Links: Brochure | Video | LinkedIn

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Saint Francis Service Dog in training receives rare root canal

ROANOKE, Va. – A Southwest Virginia dentist operated on a very unusual patient on Friday.

Walker the labrador retriever received a root canal in preparation for his work as a Saint Francis Service Dog.

The procedure is rare, but Saint Francis Service Dogs executive director Caball Youell said it was essential to Walker’s future career.

“His tooth was starting to cause him problems,” Youell said. “He needs to be able to pick things up comfortably, carry things for someone, or retrieve items.”

Walker got his canine tooth repaired at the Roanoke Animal Hospital, but none of the veterinarians there had the capacity to treat a root canal.

“We needed some special equipment and materials to do the root canal, and the dental community stepped up,” said Dr. Mark Finkler of Roanoke Animal Hospital.

Instead, Dr. Grant Throckmorton of Wythe Family Dentistry drove an hour from Wytheville to operate on Walker in Roanoke.

“The tooth is about twice as long as you’ll ever see a human tooth,” Throckmorton said. “They’re just massive. Some of my instruments don’t even work on dog teeth.”

The procedure went well, and Throckmorton finished the job in about an hour.

Youell said she is in the process of finding Walker’s permanent home, and she’s relieved his tooth will no longer cause him trouble.

“He’s going to want to go pick things up and bring them to you,” Youell said. “Whenever you see Walker, he looks around and says, ‘what present can I bring you?’”

Copyright 2020 by WSLS 10 – All rights reserved.

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Trump receives steroid treatment for Covid-19

The White House physician said Sunday that President Donald Trump is now taking another drug for Covid-19, adding to his growing list of treatments for his illness.

Dr. Sean Conley said the president was given a steroid called dexamethasone following “two episodes of transient drops in his oxygen saturation,” meaning his oxygen levels dropped too low.

Full coverage of the coronavirus outbreak

“We debated the reasons for this and whether we even intervene,” Conley said during a press conference at Walter Reed National Military Medical Center in Bethesda, Maryland. “As a determination of the team, based predominantly on the timeline from the initial diagnosis, that we initiate dexamethasone.”

Trump received the first dose of the steroid on Saturday and will be on it for “the time being,” Conley said.

Dexamethasone has shown to be beneficial in those with severe Covid-19 because it can stop the immune system from going into overdrive. When this happens, the immune system can do more harm than good, attacking the body in what’s called a cytokine storm.

However, the drug is not recommended for more mild cases of the disease.

“The fact that he got the steroid sets up a bit of a red flag that there’s something going on here,” NBC News senior medical correspondent Dr. John Torres told Kate Snow on Sunday. “I think they might be painting a little bit of a rosy picture for everyone.”

The treatment was first shown to be helpful in June in clinical trials in the U.K. Preliminary results of that trial published in the New England Journal of Medicine found that the steroid reduced the risk of death in patients with severe Covid-19 who need supplemental oxygen. But for mild cases, the treatment can be harmful.

“However, there was no evidence that dexamethasone provided any benefit among patients who were not receiving respiratory support at randomization, and the results were consistent with possible harm in this subgroup,” the researchers wrote.

Download the NBC News app for breaking news about the president’s health

The National Institutes of Health guidelines for the drug reflect those findings, stating that dexamethasone should only be given to patients who are on ventilators or require supplemental oxygen. They do not recommend using it for those with less serious illness because of the potential for harm.

“We do not recommend giving it to patients who are not on supplemental oxygen or ventilated because in that case, it’s going to suppress the immune system and it won’t be able to fight off the Covid in those early stages,” Torres said.

Dr. Michael Saag, an infectious disease specialist at the University of Alabama at Birmingham, said the fact that doctors gave him the drug makes him suspect the president might have taken a sudden turn Thursday night into Friday morning.

“The disease course of Covid-19 is very unpredictable and waxes and wanes throughout the course of illness,” Saag told NBC News. “I would expect for the president, and every other symptomatic person with

KA Imaging’s Portable Dual-energy X-ray Receives Canadian Medical Device Licence from Health Canada

Canadian market comprises more than 1,000 hospitals. Number of medical and diagnostic laboratories (which include x-ray) surpasses 3,000.

A Canadian Medical Device Licence was issued September 24 by Health Canada for Reveal 35C™, created by KA Imaging – a University of Waterloo spin-off company.

Reveal is a portable dual-energy X-ray detector that enables bone and soft-tissue differentiation without motion artifacts in a single X-ray exposure. The detector simultaneously delivers DR, bone, and tissue images in one shot. Reveal can be taken to the bedside of vulnerable patients in hospital and used easily to aid emergency procedures. It is also affordable and comes at a quarter of the price of many fixed dual-energy systems.

“Getting this Health Canada licence is a major step forward for KA Imaging, especially coupled with the recent FDA clearance. As a Canadian company we are thrilled to have received Health Canada’s approval and eagerly anticipate providing our solution to the Canadian market for advance in the field of X-ray,” said Amol Karnick, President and CEO of KA Imaging.

Statistics from the Industry Canada database from 2019 show that there are more than 1,000 hospitals (including general and specialized facilities) in the country, and more than 3,000 medical and diagnostic laboratories – including X-ray services. In addition, because the detector is portable – it is the only portable dual-energy solution on the market – less obvious sectors can also benefit from the technology.

“At long-term care facilities, which care for vulnerable populations that can suffer from mobility limitations or health concerns, taking the detector to the patient can be very beneficial,” said Dr. Karim S. Karim, CTO of KA Imaging.

Reveal is already being used in clinical trials with promising results. The detector is being tested on patients suspected of having lung cancer at Grand River Hospital in Kitchener, and for detection of pneumonia (including COVID-19) in a Toronto-based hospital.

“Early triaging is an essential countermeasure to prevent the spread of COVID-19 infection, and we are confident that our technology can make a significant contribution,” said Karim.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200930005044/en/

Contacts

Fernanda Fraga
[email protected]
226 215 9897

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