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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

J&J vaccine trial hits pause as US doubles down on antibody therapies

Johnson & Johnson (JNJ) announced late Monday it was pausing shots in its late-stage coronavirus vaccine trial due to an adverse event, but plans to continue enrolling patients and stick to the current manufacturing timeline.

It is still unknown if the participant was receiving a placebo or the vaccine, a point that will be determined by an independent advisory group, known as the Data Safety Monitoring Board (DSMB). Another vaccine using similar technology from AstraZeneca (AZN) is still on hold after a serious adverse event from a trial participant triggered a halt in September. The trial has continued in the U.K., but remains on hold in the U.S.

“Adverse events – illnesses, accidents, etc. – even those that are serious, are an expected part of any clinical study, especially large studies,” J&J said statement late Monday, reiterating that “studies may be paused if an unexpected serious adverse event (SAE)” occurs that may or may not be related to the trials. The company promised a “careful review of all of the medical information before deciding whether to restart the study.”

Mathai Mammen, head of research and development at Janssen, J&J’s pharmaceutical arm, said the information is kept confidential from the company until the DSMB reviews it.

“It will be a few days at minimum for the right set of information to be gathered and evaluated,” Mammen said during an investor call Tuesday.

The two frontrunner candidates in the U.S., Moderna (MRNA) and Pfizer (PFE) with BioNTech (BNTX), are using technology that has never been approved, but have so far not hit any significant adverse events.

Meanwhilel, Pfizer’s non-peer-reviewed data showed some side effects, but nothing that would trigger a halt. Pain at the point of injection and fatigue are considered normal vaccine side effects — seen frequently after flu shots.

Monoclonal antibodies

There are over 7.6 million cases in the U.S. (Graphic: David Foster/Yahoo Finance)
There are over 7.6 million cases in the U.S. (Graphic: David Foster/Yahoo Finance)

The experimental treatment used by President Donald Trump while he was at Walter Reed Military Medical Center is gaining momentum as the country awaits a vaccine by the end of the year.

Monoclonal antibody treatments, which are lab-produced antibodies from a sample of recovered patients, has been seen as a bridge between standard treatments and a vaccine, as well as an alternative for those who cannot be given a vaccine. That is because it can both treat and defend against the virus.

Trump received a high dose of Regeneron’s (REGN) antibody cocktail, which sparked the company and Eli Lilly (LLY), which is also developing an antibody treatment, to apply for emergency use authorizations (EUA). The U.S. Food and Drug Administration (FDA) has yet to provide an update on these filings from last week, but analysts have anticipated authorizations following Trump’s treatment.

Meanwhile, Operation Warp Speed announced a collaboration with AstraZeneca to test and produce a monoclonal antibody cocktail, which would be provided for free once authorized by the FDA.

Adding to the portfolio of candidates, the National Institutes of Health (NIH) announced Tuesday it

Trump extols drug therapies in COVID-19 video: ‘I want to get for you what I got’

President Trump said Wednesday it is important to speed monoclonal antibodies and other drug therapies to hospitals to help people overcome the coronavirus, saying it helped him rebound from infection.

“I wasn’t feeling so hot, and within a very short period of time, they gave me Regeneron. And other things too, but I think this was the key. But they gave me Regeneron and it was like unbelievable, I felt good immediately,” he said in a five-minute video from outside the West Wing.

The president, who is still recovering from the virus, is wearing a navy suit and red necktie and doesn’t appear to be suffering from symptoms.

Mr. Trump noted a similar drug is coming from Eli Lilly and Co. and the Food and Drug Administration may approve it on an emergency basis.

He said seniors, in particular, will get the drugs quickly and suggested they might be delivered at no cost.

“You’re gonna get better, you’re gonna get better really fast,” he said. “This is things that nobody even thought of even a few months ago.”

Mr. Trump went so far as to call his infection, confirmed last Thursday, a “blessing in disguise,” saying it was his suggestion to receive the Regeneron drug that was made available under “compassionate use” rules.

“I want to get for you what I got,” Mr. Trump said.

Mr. Trump complained, however, that FDA rules for emergency authorization of a vaccine will extend beyond Nov. 3.

“They want to play their games, it’s going to be right after the election,” Mr. Trump said.

The president also reiterated his anger with China, saying it was their fault the virus spread around the world after it leaped to humans in Wuhan in December.

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Prime 10 Complementary And Different Medicine Therapies That Work

Drinking plenty of water is at all times good, however a glass or two of Ouzo thins the blood and alleviates among the worst signs. The synthesis of sulfonamide started with the synthesis of protonsil of the hostile effects of sulfonamides was hypoglycemia, which led to the development of sulfonylurea drugs.

If uncovered to infrared radiation for a long time, the body can lose fluid or water and this can lead to dehydration. The remedy consisted of a surgeon making a number of cuts on the physique while the blood drained into a bowl. Understanding the Prednisone unwanted effects or indeed of taking any drug stays a alternative for the buyer, but typically such choices fade into insignificance towards the ravaging effects of the disease the drug’s taken to manage.

A great instance could be the place sufferers are largely educated and in a position to research their diseases over the web earlier than consulting the doctor, being able to suggest and seek the advice of medical therapies, and refuse or negotiate sure assessments or medications.

Hepatitis B virus has developed resistance to medication like interferon, lamivudine, fevoir and extra, in line with Dr. Steven-Huv Han. It is a disease that causes the body to provide too many pink blood cells or platelets. Take your straight edge and pencil and draw a rectangle approximately 10 inches by 2 inches on on the leather-based piece you’re utilizing to create your medicine bag.

Many individuals will get allergic downside with sulpha medication, so we ought to be very cautious once we use them. For example, the drugs used for the treatment of various illnesses resembling hypertension, malaria, tuberculosis or epilepsy may be grouped by adding the prefix -anti-.…

Prime 10 Complementary And Various Medicine Therapies That Work

Hepatitis B virus has developed resistance to drugs like interferon, lamivudine, fevoir and extra, in line with Dr. Steven-Huv Han. It’s a disease that causes the physique to provide too many red blood cells or platelets. Take your straight edge and pencil and draw a rectangle approximately 10 inches by 2 inches on on the leather piece you are using to create your medicine bag.

On the flipside, the consumerist model is the place shoppers have high control over their treatments while physicians have low control – think about sufferers now having the ability to seek second opinions, choose their doctors, have interaction in alternative and biomedical treatments at the same time.

This might seem crazy however I by no means had no problem with my hair until I used biotin each time I wash my hair I observed that I m losing some on the drain and my hair appears much less then it was.I’m stopping it and I am not going dangerous. For a long time conventional medicine snubbed this therapy.

An excellent instance could be the place sufferers are largely educated and capable of analysis their illnesses over the web before consulting the physician, with the ability to suggest and consult medical treatments, and refuse or negotiate sure assessments or medications.

Tens of millions of lives are saved every year due to one animal’s, outcomes to either a drug or new type of surgical procedure, and I consider as an alternative of believing that an animal’s life was a waste; I view it as an alternative as the following step in bettering people’s lives for tons of of years to return.…

Over The Counter Therapies For Arthritis In Dogs And Cats

These medicines block the transport of calcium into the graceful cells lining the coronary arteries and other arteries of the physique. handout written materials on a medication that patients are taking to include unwanted side effects and other drug interactions.

The playing cards that come with medicine card spread every has a number and an animal on its inside faceIf its quantity and face are the other way up the cardboard is alleged to be in reverse position. You’ll not know what to do with them till you have got read the hubs medicine cards and totem animals, later you possibly can learn medicine wheel and medicine cards spreads.

If uncovered to infrared radiation for a very long time, the body can lose fluid or water and this can result in dehydration. The therapy consisted of a surgeon making multiple cuts on the physique whereas the blood drained into a bowl. Understanding the Prednisone uncomfortable side effects or indeed of taking any drug remains a alternative for the buyer, yet usually such decisions fade into insignificance against the ravaging results of the disease the drug’s taken to control.

Sufferers are encouraged to drink a number of water to keep the physique’s water at an optimum level. In order for better medicines and treatments to be developed, there have to be a testing performed to guantee that the medicine is not dangerous or is not going to kill human.

Take a look at a number of the medical web sites and you can find that it’s troublesome to overdose on Biotin as a result of it’s water-soluble. Nicely i have to say that i COMPLETELY assume that animal cruelty is improper but even though i like animals, all the individuals on the earth would die if they tested it on humans.…

Over The Counter Therapies For Arthritis In Canines And Cats

Infrared in Health care – Digital Infrared Thermal Imaging (DITI) is a technique that’s used for diagnosis within the medical area. Freiberg’s illness, which is an osteochondritis of the metatarsal head, causes ache on weight bearing or compression. These anti-allergy medicines might be consumed within the form of tablets, syrups, powder and drops.

Nevertheless, hepatitis C could be transmitted by intravenous drug customers, health care givers, or people having tattoos. There may be hence a real problem of medical social management, as sufferers are subjected to various treatments whether they prefer it or not.

Authorities is hiding all of this from the individuals cause i guess if a video of animals being tortured was placed on TV people would feel dangerous and vote against animal cruelty unless they have no coronary heart. From thousands and thousands of virus in a Hepatitis B affected person the inhabitants of virus was decreased to solely hundreds after therapy with infusion chelation therapy.

Animal cruelty is unsuitable however what about all these people we’ve got locked up as a result of they’ve completed something wrong we must always use them for our resurch in stead of animals. Alternatively when medicine failed and the affected person died, their body was washed in anointed in scented oil before being laid to relaxation.

He was administered with blood transfusion but my mom requested that she be allowed to let him drink the boiled juice of Tawa-tawa. These are all body painkiller medicines. For many people, the blood thickens because the physique makes an attempt to cope with the intense warmth, and nosebleeds and swollen ankles are frequent for those unused to the Greek summer season.…

Top 10 Complementary And Various Medicine Therapies That Work

A very good instance would be where patients are largely educated and in a position to research their diseases over the internet before consulting the doctor, having the ability to suggest and consult medical therapies, and refuse or negotiate certain assessments or medicines.

Checks that are carried out on animals range from them being pressured to ingest totally different household cleaning merchandise and then monitored to see the outcomes and new cosmetics positioned on their skin to seek out out if there are any negative effects that would be dangerous to a human.

Elderly folks will be liable to low blood stress if uncovered to infrared radiation. The biomedical model of medicine is the dominant healthcare mannequin in many international locations. This powered form might be either consumed immediately or it may be added to boiled water and then the answer be drunk.

C.Geriatric dentistry :- Geriatric dentistry or Gerodontics is a branch offers with the older adults involving the diagnosis, prevention, and therapy of issues related to regular ageing and age-associated diseases as part of an interdisciplinary workforce with other health care professionals.

My household went to an emerald mine in North Carolina and the stones and crystals we used had been ones that we found, so we take a look at the stones as having extra positive power as a result of the household bond of the time spent attempting to find them.I have about an eighth to 1 / 4 cherokee and my kids have more cherokee and some blackfoot indian of their bloodline so we respect the earth and all of its plants and animals.…