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

Lilly Covid-19 antibody treatment trial paused. Experts say that’s normal

Drugmaker Eli Lilly and Co. said Tuesday that the government-sponsored clinical trial of its Covid-19 antibody treatment has been paused because of a safety concern.

“Out of an abundance of caution, the ACTIV-3 independent data safety monitoring board (DSMB) has recommended a pause in enrollment,” Lilly spokeswoman Molly McCully said in an emailed statement. “Lilly is supportive of the decision by the independent DSMB to cautiously ensure the safety of the patients participating in this study.”

The Lilly drug, called LY-CoV555, is similar to the Regeneron antibody treatment President Donald Trump received after his Covid-19 diagnosis. It is currently being tested in hospitalized patients.

Full coverage of the coronavirus outbreak

The pause in the Lilly trial comes a day after Johnson & Johnson paused its Covid-19 vaccine trial after a volunteer had an unexplained illness. J&J said the participant’s condition was being reviewed and evaluated by the Data Safety Monitoring Board. It’s unclear whether the volunteer who had an adverse event had received the actual vaccine or a placebo.

It’s not uncommon for late-stage trials to hit bumps.

“Pauses are not infrequent in a clinical trial, and in these studies, they are going to be probably a bit more frequent as the development line for a product is significantly truncated,” Dr. Carlos del Rio, infectious diseases expert at Emory University School of Medicine, said.

Just last week, Lilly requested emergency use authorization from the Food and Drug Administration for the antibody treatment, using data from a separate clinical trial — not the trial that was just paused.

No published or peer-reviewed results have been released about the treatment, but last month the company released partial results from a trial of the drug, which suggested it could help keep patients with mild to moderate forms of the illness from progressing to a point where they would need to be hospitalized.

The paused trial is sponsored by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. When the phase 3 trial was announced in August, Lilly had planned to enroll approximately 300 volunteers hospitalized with mild to moderate illness with fewer than 13 days of symptoms, according to the NIH. Participants were also given the antiviral remdesivir.

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U.S. pauses Eli Lilly’s trial of a coronavirus antibody treatment over safety concerns

  • Eli Lilly’s phase three trial of its ACTIV-3 monoclonal antibody treatment for the coronavirus has been paused due to potential safety concerns.
  • The ACTIV-3 trial is designed to test a monoclonal antibody developed by Eli Lilly in combination with remdesivir, an antiviral with emergency use authorization for the virus.
  • It’s one of several ongoing trials, as part of the National Institute of Health’s “Activ” program, designed to accelerate the development of vaccine treatments in partnership with the pharmaceutical industry.



a group of people standing around a plane: In this May 2020 photo provided by Eli Lilly, researchers prepare mammalian cells to produce possible COVID-19 antibodies for testing in a laboratory in Indianapolis.


© Provided by CNBC
In this May 2020 photo provided by Eli Lilly, researchers prepare mammalian cells to produce possible COVID-19 antibodies for testing in a laboratory in Indianapolis.

Eli Lilly’s late-stage trial of its leading monoclonal antibody treatment for the coronavirus has been paused by U.S. health regulators over potential safety concerns, the company confirmed to CNBC on Tuesday.

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“Safety is of the utmost importance to Lilly. We are aware that, out of an abundance of caution, the ACTIV-3 independent data safety monitoring board (DSMB) has recommended a pause in enrollment,” a spokeswoman Molly McCully told CNBC. “Lilly is supportive of the decision by the independent DSMB to cautiously ensure the safety of the patients participating in this study.”

The company’s shares fell by about 3% in afternoon trading after the news started to leak out over Twitter.

The news comes less than 24 hours after Johnson & Johnson confirmed that its late-stage coronavirus vaccine trial was paused after a participant reported an “adverse event” the day before.

Dr. Mathai Mammen, global head of Janssen research and development at J&J, told investors on a conference call Tuesday that the company still has “very little information” on the reason for the holdup, including if the patient received the vaccine or the placebo. Preliminary information has been sent to the data safety monitoring board for review, he added.

Medical experts note that pauses in large clinical trials are not uncommon. They added it’s possible the bad reaction could be result of an unrelated illness, and not the drug itself. The review from the data and safety monitoring board will help determine that.

Video: Dr. Scott Gottlieb on remdesivir Covid-19 treatment study: ‘Results were strong’ (CNBC)

Dr. Scott Gottlieb on remdesivir Covid-19 treatment study: ‘Results were strong’

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The ACTIV-3 trial is designed to test a monoclonal antibody developed by Eli Lilly in combination with Gilead Sciences’ remdesivir, an anti-viral with emergency use authorization for the virus. It’s one of several ongoing trials that are part of the National Institute of Health’s “Activ” program, which is designed to accelerate the development of Covid-19 vaccines and treatments. It is also backed by Operation Warp Speed, the Trump administration’s effort to manufacturer and distribute vaccines to fight Covid-19.

Eli Lilly’s drug is part of a class of treatments known as monoclonal antibodies, which are made to act as immune cells that scientists hope can fight the virus. The treatment was developed using a blood sample from one of the

Ionis’ inhaled antisense medicine demonstrates potential as a novel treatment for cystic fibrosis | News

CARLSBAD, Calif., Oct. 13, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that data from a clinical trial of IONIS-ENAC-2.5Rx demonstrated a significant decrease in the expression of epithelial sodium channel (ENaC) in subjects with cystic fibrosis (CF). The study showed a mean 55.6 percent decrease (p<0.05) in ENaC mRNA expression at the 75 mg dose in the multidose segment of the trial. The study represents the first time an antisense medicine delivered directly to the lung via a nebulizer has shown a significant reduction in ENaC messenger RNA levels. In preclinical studies, ENaC mRNA reductions of 40 percent or more resulted in significant improvement in mouse models of CF lung disease.

IONIS-ENAC-2.5Rx is an investigational antisense medicine designed to reduce the expression of ENaC in the lung. ENaC is believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene. Data from the Phase 1 study will be presented via e-poster at the 2020 North American Cystic Fibrosis Conference, which will hold virtual sessions and discussions Oct. 21-23.

Cystic fibrosis is a life-threatening disease affecting approximately 30,000 people in the U.S. and about 70,000 worldwide. Although CF is a multisystem disease, the main cause of morbidity and mortality is lung disease, characterized by small airway obstruction due to mucus accumulation, decreased mucus clearing and subsequent inflammation, infections and lung function decline.

“We are very encouraged by these data, which demonstrate attractive tolerability and safety for IONIS-ENAC-2.5Rx with substantial target reduction and the convenience of once weekly administration. The data also confirm our expectations for aerosol delivery of antisense medicines for lung diseases based on a wealth of preclinical data,” said Brett P. Monia, Ph.D., Ionis’ chief executive officer. “These results point to the exciting potential for aerosol delivery of other Ionis medicines that we plan to advance to the clinic, including treatments for chronic obstructive pulmonary disease, or COPD, and severe asthma.”

The company also plans to initiate a clinical study to evaluate IONIS-ENAC-2.5Rx in patients with COPD associated with chronic bronchitis starting later this year. IONIS-ENAC-2.5Rx is one of more than 20 potentially transformative antisense medicines in the growing Ionis-owned pipeline that the company is prioritizing and preparing for commercialization.

The primary endpoint of the study was evaluation of safety and pharmacokinetics of IONIS-ENAC-2.5Rx delivered via a Pari eFlow mesh nebulizer. In the single escalating dose study, 32 subjects in four cohorts received a single dose of 3, 10, 37.5, or 100 mg and were followed for 30 days. In the multiple ascending dose study, 24 subjects in three cohorts received four doses of 10, 37.5, or 75 mg once weekly, with an additional dose administered during the first week. An additional cohort of eight subjects received a 37.5 mg dose given thrice weekly for 10 doses. Subjects were followed for 13 weeks after dosing. Fiberoptic bronchoscopy including bronchial brushings and bronchoalveolar lavage was

Ionis’ inhaled antisense medicine demonstrates potential as a novel treatment for cystic fibrosis

CARLSBAD, Calif., Oct. 13, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that data from a clinical trial of IONIS-ENAC-2.5Rx demonstrated a significant decrease in the expression of epithelial sodium channel (ENaC) in subjects with cystic fibrosis (CF). The study showed a mean 55.6 percent decrease (p

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

IONIS-ENAC-2.5Rx is an investigational antisense medicine designed to reduce the expression of ENaC in the lung. ENaC is believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene. Data from the Phase 1 study will be presented via e-poster at the 2020 North American Cystic Fibrosis Conference, which will hold virtual sessions and discussions Oct. 21-23.

Cystic fibrosis is a life-threatening disease affecting approximately 30,000 people in the U.S. and about 70,000 worldwide. Although CF is a multisystem disease, the main cause of morbidity and mortality is lung disease, characterized by small airway obstruction due to mucus accumulation, decreased mucus clearing and subsequent inflammation, infections and lung function decline.

“We are very encouraged by these data, which demonstrate attractive tolerability and safety for IONIS-ENAC-2.5Rx with substantial target reduction and the convenience of once weekly administration. The data also confirm our expectations for aerosol delivery of antisense medicines for lung diseases based on a wealth of preclinical data,” said Brett P. Monia, Ph.D., Ionis’ chief executive officer. “These results point to the exciting potential for aerosol delivery of other Ionis medicines that we plan to advance to the clinic, including treatments for chronic obstructive pulmonary disease, or COPD, and severe asthma.”

The company also plans to initiate a clinical study to evaluate IONIS-ENAC-2.5Rx in patients with COPD associated with chronic bronchitis starting later this year. IONIS-ENAC-2.5Rx is one of more than 20 potentially transformative antisense medicines in the growing Ionis-owned pipeline that the company is prioritizing and preparing for commercialization.

The primary endpoint of the study was evaluation of safety and pharmacokinetics of IONIS-ENAC-2.5Rx delivered via a Pari eFlow mesh nebulizer. In the single escalating dose study, 32 subjects in four cohorts received a single dose of 3, 10, 37.5, or 100 mg and were followed for 30 days. In the multiple ascending dose study, 24 subjects in three cohorts received four doses of 10, 37.5, or 75 mg once weekly, with an additional dose administered during the first week. An additional cohort of eight subjects received a 37.5 mg dose given thrice weekly for 10 doses. Subjects were followed for 13 weeks after dosing. Fiberoptic bronchoscopy including bronchial brushings and bronchoalveolar lavage was performed during screening and after completion of dosing in the MAD cohorts. Quantitative RT-PCR was performed from the bronchial cell brushings to evaluate ENaC mRNA levels.

About Ionis Pharmaceuticals

As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our

Takeda group begins manufacturing COVID-19 plasma treatment ahead of approval

By Michael Erman

NEW YORK (Reuters) – The Takeda Pharmaceutical Co-led group that is developing a blood plasma treatment for COVID-19 has started manufacturing while the late-stage trial to determine whether it works is ongoing, Takeda Chief Executive Christophe Weber said on Monday.

The group, known as the CoVIg Plasma Alliance, enrolled its first patient in the Phase III trial on Friday after months of delays. It aims to enroll 500 adult patients from the United States, Mexico and 16 other countries and hope to have results by the end of the year.

“The likelihood it works is very high,” Weber said in an interview. “And that’s why we have launched a campaign in order to accelerate the donation of convalescent plasma to manufacture and produce this product.”

The alliance, which includes CSL Behring, Germany’s Biotest AG and other companies, is testing a hyperimmune globulin therapy, which is derived from blood plasma of people who have recovered from COVID-19. Hyperimmune globulin therapy offers a standardized dose of antibodies and does not need to be limited to patients with matching blood types.

That makes it more advanced and convenient than treatment with convalescent plasma drawn from recovered patients.

The manufacturing process is expensive.

Weber said the treatment could be slightly more costly to make than monoclonal antibody treatments like the ones Regeneron Pharmaceuticals Inc and Eli Lilly and Co have developed. The alliance does not intend to profit from the treatment, Weber said.

The Takeda CEO said he does not know how many doses of the treatment the group will be able to produce by the end of the year. That will depend on donations as well as the dosage size they decide to test in the clinical trial.

The trial will test the hyperimmune globulin therapy in combination with Gilead Sciences Inc’s antiviral drug remdesivir compared with patients who get remdesivir alone, he said.

(Reporting by Michael Erman; Editing by Bill Berkrot)

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DNA Dental Comprises the Best Dentist in Dallas, TX, Offering Cosmetic, General, and Restorative Dental Care and Treatment Solutions – Press Release

DNA Dental Comprises the Best Dentist in Dallas, TX, Offering Cosmetic, General, and Restorative Dental Care and Treatment Solutions

Dallas, TX – Dallas, TX residents who are in search of 5-star and premium dental care and treatment solutions can now rest assured that the team at DNA Dental brings this quality of services to them at affordable rates. The dental clinic in Dallas, TX boasts a team of experienced and professional dental specialists who are constantly improving on their dental practice to ensure that each patient is given the attention and care they deserve.

Focused on improving the oral health and wellness of the members of the community, DNA Dental has proudly expanded on its dentistry practice to offer a wide range of dental solutions, including the very best children’s dentistry solutions in the area. Children who come into the dental clinic will have access to the very best and experienced pediatric dentists who are committed to creating the best dental care and treatment experiences using the latest dental tools, technology, and procedures while also aiming for a pain-free dental care and treatment experience.

The entire team of Dentist at DNA Dental also focus on helping children to form the right dental foundations for their oral health and wellness needs. Added to this, the dental clinic boasts of the best Cosmetic Dentist in the Dallas, TX area. The professional cosmetic dentist offers a range of popular cosmetic dental services that are designed to help patients to reach the goal they have for a bright and beautiful smile.

Describing the dental solutions offered at the clinic, Dr. Darya Timin, the spokesperson for DNA Dental said, “Here at DNA Dental, we are committed to providing gentle care to every member of your family. We understand that you may feel nervous about your dental visits, and we are here to help you feel relaxed and comfortable. Our dentists and team are proud to provide quality, gentle dentistry solutions. We invite you to contact our friendly team today to learn more about our comprehensive services and to schedule your next appointment with one of our experienced dentists. Take your next step toward a healthy smile today!”

Serving patients in Dallas, TX, and the surrounding areas, DNA Dental also offers comprehensive restorative dentistry solutions including dental bridges, dental crowns, dental implants, dentures, and dental fillings.

Patients who visit the clinic will have access to the latest dental technologies, as well as a cutting-edge approach to dental care and treatment.

DNA Dental is located at 6162 E Mockingbird Ln Ste 205, Dallas, TX 75214, US. For top-quality dental care and treatment solutions, contact their team by calling +1 214-817-3113 to schedule an appointment. For additional information regarding their services, send an email to [email protected] or visit the dental clinic’s website.

Media Contact
Company Name: DNA Dental
Contact Person: Dr. Darya Timin
Email: Send Email
Phone: +1 214-817-3113
Address:6162 E Mockingbird Ln Ste 205
City: Dallas
State: TX
Country: United States
Website: https://dnadentaldallas.com/

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U.S., AstraZeneca strike deal for Covid-19 antibody treatment

ANDREW YATES | AFP | Getty Images

The U.S. government has awarded $486 million to AstraZeneca Plc to develop and secure supplies of up to 100,000 doses of Covid-19 antibody treatment, a similar class of drug that was used in treating President Donald Trump.

The agreement, under the Trump administration’s Operation Warp Speed, is for developing a monoclonal antibody cocktail that can prevent Covid-19, especially in high-risk population like those over 80 years old, the U.S. Department of Health and Human Services said.

The treatment has come under the spotlight after Trump was treated with Regeneron Pharmaceuticals’ antibody drug last week. The president has also released a video on Twitter touting its benefits.

In a call earlier on Friday, a top U.S. health official said the government was expecting to provide more than 1 million free doses of antibody treatments to Covid-19 patients, similar to the one that was administered to Trump.

Regeneron and Eli Lilly have both applied to the U.S. Food and Drug Administration for emergency use authorizations of their antibody treatments.

AstraZeneca said it was planning to supply up to 100,000 doses starting toward the end of 2020 and that the U.S. government could acquire up to an additional one million doses in 2021 under a separate agreement.

Regeneron signed a $450 million deal in July to sell Operation Warp Speed enough doses of its antibody treatment, REGN-COV2, to treat around 300,000 people.

Eli Lilly said on Friday it had not signed an agreement with Operation Warp Speed.   

AstraZeneca plans to evaluate the treatment, AZD7442, which is a cocktail of two monoclonal antibodies, in two studies.

One trial will evaluate the safety and efficacy of the experimental treatment to prevent infection for up to 12 months in about 5,000 participants, while the second will evaluate post-exposure preventative and pre-emptive treatment in roughly 1,100 participants.  

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Diamond Dental Associates LLC, Has The Best Flemington Dentist For Oral Healthcare And Treatment Needs

Diamond Dental Associates LLC, Has The Best Flemington Dentist For Oral Healthcare And Treatment Needs

Flemington, NJ – Members of the Flemington, NJ community who wish to sport the best smiles and enjoy the advantages of top oral health can rest assured that Diamond Dental Associates LLC has the best dentists in the area to deliver these premium services at the most affordable prices. At Diamond Dental Associates LLC, the dental team focuses on providing premium quality dental care and treatment solutions to patients while delivering the best dental experience in the process.

As the leading dental clinic in the area, Diamond Dental Associates LLC has a team of dentists with over three decades of experience who are on the ground to listen to oral health complaints from patients and address the problem. At Diamond Dental Associates LLC, the team employs the use of the latest dental technologies to establish diagnosis and craft treatment plans that are most suitable for the needs of each patient.

Promising the very best dental experience, the top Flemington Dentist at the clinic said: “Here at Diamond Dental Associates LLC, we pledge to provide the finest personal service using state of the art technology for our patients, who will always be welcomed to a warm and caring environment. At Diamond Dental Associates we change lives one smile at a time. We are committed to making every interaction with our patients a positive one. We respect and acknowledge your concerns about the dental treatment you will receive. We recognize that it is difficult for highly fearful people to visit a dental office for needed treatment and we offer services that help you to feel confident that the treatment we provide is in your best interests and fits in with your preferences, lifestyle, and monetary investment.”

As a dental clinic that is constantly working on new ways to address the dental care and treatment needs of patients, Diamond Dental Associates LLC – Flemington Dentist has expanded the services offered to include general dentistry practice which focuses on improving oral health and protecting the mouth from common debilitating conditions. As part of the general dentistry services offered, patients will get to enjoy routine dental examinations, oral cancer screening, teeth cleaning, dental sealants, and more.

Added to this, the dental team at Diamond Dental Associates LLC also offers a wide range of cosmetic dental solutions aimed at improving the appearance and appeal of the teeth. As part of the cosmetic dental solutions offered, patients are welcomed into the clinic to enjoy teeth whitening services aimed at brightening the teeth and smile; cosmetic bonding used to restore decayed teeth, strengthen a weak tooth, close spaces between the teeth, and alter the shape and/or size of the tooth.

The dentists also offer dental veneers that are used to cover up blemishes, chips, cracks, and tooth discoloration; dental implants for restoration of missing or lost teeth; as well as sports mouthguards and sleep apnea treatment.

Visit Diamond Dental Associates LLC at North, 334 NJ-31 #1, Flemington, NJ 08822, or call 908-838-0004. For more information, send an email

UW Medicine, Fred Hutch test experimental antibody treatment used on Trump for COVID-19

Regeneron Pharmaceuticals’ cocktail of two monoclonal antibodies was used to treat President Donald Trump for COVID-19.

SEATTLE — Researchers at UW Medicine are partnering with the Fred Hutchinson Cancer Research Center to lead a study of Regeneron Pharmaceuticals’ antibody cocktail as a way to prevent COVID-19 infections. 

The researchers are currently recruiting patients for the study. 

The same experimental Regeneron Pharmaceuticals cocktail of two monoclonal antibodies was used on President Donald Trump after he became infected.

“It’s the same antibody cocktail, that can be used for both prevention and treatment,” said Dr. Ruanne Barnabas, co-principal investigator and associate professor of Global Health and Allergy and Infectious Diseases at University of Washington School of Medicine. “But trials for both prevention and treatment are ongoing.”

The trial, which also includes 100 additional sites and plans to recruit 2,000 patients, is focused on helping people stay healthy after a close member in their own household becomes sick with COVID-19. The antibody cocktail is called REGN-COV2.

If that’s the case, why not use this antibody in lieu of vaccines, a number of which are in the final trial phases? 

Dr. Barnabas said, “These antibodies last for a short time, for a number of weeks. But a vaccine will teach our bodies to make these antibodies that will last for years. That’s the plan.”

The presumption is someone given the cocktail would have enough antibodies immediately to last long enough to get through their housemates’ infection.

“Monoclonal antibodies could help us achieve an end to the pandemic,” said Dr. Shelly Karuna with Fred Hutch in a statement. 

People wanting to participate in the study must have a confirmed household member test positive for COVID-19 to qualify. To learn more about getting involved call (206) 773-7129 or visit the UW Medicine website. 

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