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Veterinary Hospital | School of Veterinary Medicine

September 20th, 2022 2:02 am

The UC Davis VMTH client portal allows online access for clients to handle all aspects of their pets appointments and invoices. Register today for our new UC Davis VMTH client portal!

Once registered, VMTH clients can easily:

Please log into theClient Portalto create your account

Welcome to the William R. Pritchard Veterinary Medical Teaching Hospital at the University of California,Davis, a unit of the #1 ranked School of Veterinary Medicine. The hospital is equipped with cutting-edge, modern technology and services, allowing our board-certified faculty veterinarians to provide the best care possible and continue to be innovators of the some of the latest advancements in veterinary medicine, all while training the next generation of general practitioners and veterinary specialists.

The UC Davis veterinary hospital is accredited by theAmerican Animal Hospital Association, the only organization to accredit companion animal veterinary hospitals. AAHA-accredited hospitals are recognized among the finest in the industry because they voluntarily choose to be evaluated on more than 900 quality standards that go above and beyond state regulations, ranging from patient care and pain management to staff training and advanced diagnostic services. Less than 15% of animal hospitals in the United States and Canada achieve AAHA accreditation.

Explore jobs available at the UC Davis Veterinary Medical Teaching Hospital

Beyond the main hospital on the Davis campus, the SVM also has two satellite clinical facilities:

UC Veterinary Medical Center - San Diego

UC Veterinary Medicine Teaching & Research Center - Tulare

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Veterinary Hospital | School of Veterinary Medicine

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AAFP, EveryCat release guidelines on diagnosis of feline infectious peritonitis – American Veterinary Medical Association

September 20th, 2022 2:02 am

The American Association of Feline Practitioners and EveryCat Health Foundation announced on Sept. 1 that they have released the 2022 AAFP/EveryCat Feline Infectious Peritonitis Diagnosis Guidelines.

These guidelines, published in the September issue of the Journal of Feline Medicine and Surgery, provide veterinarians with the information necessary to diagnose FIP in cats.

First recognized over 50 years ago, feline infectious peritonitis has been one of the most important infectious diseases and causes of death in cats, especially affecting young cats less than two years old, said Dr. Vicki Thayer, co-chair of the guidelines task force, in an announcement about the new resource. Further, FIP can be challenging to diagnose in some cases and is often considered an enigma by the veterinary profession. Today, diagnosis relies upon evidence from signalment, history, physical examination findings, and diagnostic testing. The 2022 AAFP/EveryCat Feline Infectious Peritonitis Diagnosis Guidelines serve as a critical resource for veterinary practitioners diagnosing FIP in their cat patients.

According to the guidelines: FIP was once considered a terminal diagnosis. Research has demonstrated efficacy of new antivirals in FIP treatment, but these products are not legally available in many countries at this time, which includes the United States. The guidelines encourage veterinarians to review the literature and stay informed on clinical trials and new drug approvals.

Given the fact that FIP is fatal when untreated and nearly every small animal veterinary practitioner will see FIP cases, the ability to obtain a correct diagnosis is critical. FIP can be challenging to diagnose because of the lack of clinical signs or laboratory changes. The guidelines provide veterinarians with information to assist their ability to recognize cats presenting with FIP.

These Guidelines were written with the intent of providing the most current knowledge available in one comprehensive format combined with extensive supplemental resources all in one location, said Dr. Susan Gogolski, co-chair of the task force, in the announcement. The Guidelines will be an invaluable resource to veterinary teams around the world as a clinician builds the index of suspicion of FIP brick by brick.

The 2022 AAFP/EveryCat Feline Infectious Peritonitis Diagnosis Guidelines were developed by a task force of experts in feline clinical medicine. Tips, clinical images and tables, and algorithms are included throughout the document. In addition, the guidelines feature 16 supplemental online resources, such as videos, figures, instructions, and a client questionnaire.

The FIP diagnosis guidelines and supplemental resources are available here. Resources for cat caregivers can be found here.

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AAFP, EveryCat release guidelines on diagnosis of feline infectious peritonitis - American Veterinary Medical Association

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Don’t Fall Behind on Your Continuing Education this Autumn! – DVM 360

September 20th, 2022 2:02 am

Specialty: Dermatology

Webcast: Chronic Proliferative Otitis Prevention, Not Surgery!

Date/Time: September 29 a 7:00 PM EST

Sponsor: Nextmune

Missed last months live webinars? These are available on-demand via dvm360 Flex

Webcast: Urine Culture Shock! - Updates on the Diagnosis and Treatment of UTI in Dogs and Cats

We will take a uropathogen centered approach to gain a better appreciation for the bacteria that cause UTI and the drugs that can kill them! A review of the results of a study entitled Comparison of a Chromogenic Urine Culture Plate System (UTid+) and Conventional Urine Culture for Canine and Feline Specimens will also be included.Faculty: Stephen Cole VMD, MS, DACVM

Sponsor: Vetrimax

Webcast: Phosphate Binders: The Good, The Bound and the Ugly

This presentation will focus on the history of Phosphate Binder Medication development, dating back to the late 1960s, with an overview of each class of Phosphate Binder product manufactured, as to the designed commitment of its efficacy in the control of Hyperphosphatemia, and its ability to match the clinical assignment of Secondary Hyperparathyroidism regulation.

Faculty: Jerry A. Thornhill, DVM, DACVIM

Sponsor: Nutramax

Webcast: Debunking Pet Insurance

Recommending pet insurance for your clients can be overwhelming for both you and your client. Learn the various types of pet insurance providers, policy types, and benefits for pet owners and the veterinary practice, including the resources you need to make the best recommendation for your clients.

Faculty: Matthew McGlasson DVM, CVPM

Sponsor: Nationwide

Webcast: Non-antibiotic Therapy in Diarrhea

Antibiotics are no longer commonly used for treatment of acute and chronic diarrhea due to their adverse effects on the gut microbiome and risks for propagating antimicrobial resistance. We will focus on causes for acute/chronic diarrhea and the use of diet, probiotics/synbiotics, immunosuppressive therapy (with intestinal biopsy), as well as other novel treatment options in managing these patients.

Faculty: Karin Allenspach, DVM, FVH, PhD, Dipl. ECVIM-CA, FHEA, AGAF

Professor of Internal Medicine and Translational Health, Iowa State University College of Veterinary Medicine

Sponsor: Vetoquinol

Join us this October 10-12, 2022, in Atlantic City, NJ for the Atlantic Coast Veterinary Conference. Hear from our inspiring keynotes, Craig Clifford, DVM, MS, DACVIM (Oncology), Niccole Bruno, DVM BLEND Founder, and Fred Wininger, VMD, MS, DACVIM (Neurology).Choose from over 146 continuing education credits in 38 tracks of practical veterinary medicine, including companion animal, exotics, hands-on labs, and technician CEs. Plus learn more about the latest products from over 100 exhibitors and so much more. During your stay, network and unwind during the evenings at the casino shows and restaurants, the Boardwalk, or Absecon Lighthouse all within walking distance. Register early for the best rates.

Watch our latest dvm360 Live! episode!

Spectrum of care and meeting client expectations with empathy

Ryan E. Englar, DVM, DABVP joins this segment ofdvm360 Live!to explain how empathy and "unconditional positive regard" can help clinicians better serve patients when they cannot afford the gold standard of care for their pets. She shares her personal experiences with Adam Christman, DVM, MBA and discusses the concept of "spectrum of care.

A new sedative for dogs, endangered antelope born, and more

Check out the latest news and trending headlines, brought to you by Adam Christman, DVM, MBA.

Tune in to your favorite podcast channel and listen to a variety of these animal health care topics!

Have ideas for future continuing education opportunities or wish to educate fellow colleagues as one of our faculty? Tell us more at rlewis@mjhlifesciences.com.

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Using Acupuncture to Pinpoint The Source Of A Horse’s Problem – Lancaster Farming

September 20th, 2022 2:02 am

Although Sarah S le Jeune trained in Western veterinary medicine, she added acupuncture to her practice after experiencing the benefits for her own back pain.

Acupuncture had amazing effects on me, so I decided to explore how it might have application for my horses, said le Jeune, a board-certified equine sports medicine and rehabilitation specialist and surgeon with the University of California Davis.

She studied and earned certification in acupuncture through Chi University in Florida and began integrating it with conventional modalities to treat a range of conditions, often in conjunction with chiropractic.

The International Veterinary Acupuncture Society describes acupuncture as the insertion of fine needles into specific points on the body to produce a healing response. There are about 150 acupoints on a horse in areas associated with a high density of free nerve endings, mast cells, which are part of the immune system, small arterioles, which help regulate blood flow and pressure, and lymphatic vessels.

Typically, needles are inserted in five to 50 acupoints during a single session and rotated manually or stimulated by a weak electrical current sent by attaching electrodes, le Jeune said. Depending on where the needles are placed, they stimulate nerves, improve circulation, relieve muscle spasms, and trigger the release of hormones, including endorphins, one of the bodys pain control chemicals, and cortisol, a natural steroid.

Acupuncture has diagnostic as well as therapeutic value, le Jeune said, and is especially helpful in relieving pain.

Sarah S le Jeune believes acupuncture has diagnostic and therapeutic value and helps relieve pain in horses.

Any time pain is a component, acupuncture can be an adjunct and I stress adjunct modality, she said. It basically modifies the perception of pain, in how the nervous system processes pain. I use it in horses with back, neck and sacroiliac problems, any kind of muscular pain, and chronic lamenesses. It also can lower blood pressure, improve gastrointestinal motility, address endocrine and immune conditions, and reduce inflammation. They all seem to benefit from this modality.

Although equine acupuncture dates to ancient China, where horses were used in agriculture and battle, it has been practiced in the United States since the 1970s, and has become increasingly popular in recent years. While the earliest acupuncture likely was performed using sharp stones and more akin to acupressure, le Jeune said, modern acupuncture uses ultra-fine needles, 0.5 to 3 inches long and made of flexible stainless steel.

Trainers like acupuncture because they are limited in the drugs that can be given to horses that compete, and because they see results with very few negative side effects. Back pain is a fact of life for hunter jumpers and dressage, said le Jeune. For them, acupuncture and chiropractic work together fantastically. Ill treat horses with both in-between shows.

For older and retired equines, acupuncture can be an effective component of palliative care. If a horse has chronic, degenerative joint disease that causes pain, nothing will make the joints appear normal, but if you address the pain, quality of life improves, le Jeune said, noting that acupuncture can string out the time between more invasive interventions.

When used after surgery, acupuncture can speed healing by promoting blood flow and reducing inflammation.

Acupuncture has a calming effect because it triggers the release of beta-endorphins, serotonin and other neurotransmitters, le Jeune said. Its similar to the release we feel when we take a hot bath, listen to music, or eat chocolate.

Although it generally is considered safe, because acupuncture is an invasive procedure, it must be performed by a licensed veterinarian board-certified in acupuncture. As part of the process, practitioners typically will begin by examining the horse, palpating the body to locate areas of discomfort, and then gauging the horses sensitivity.

Acupuncture is very individually tailored, so you care very much how sensitive the individual is on a scale of one to five, said le Jeune. The more sensitive the horse, the less stimulation is needed; the more stoic, the more stimulation required.

Most, but not all, equines accept acupuncture, she said. You have to gain their trust. But if a horse is needle shy, it might not be the right modality for them. If the horse resists the procedure, I back off.

Le Jeune avoids using sedation during treatment because it would inhibit interacting with the horse and monitoring its responses, she said.

Acupuncture is also not usually a one-time treatment. The more chronic and severe the condition, the more acupuncture is needed, she said. Ill space it out to every other day and then weekly.

Veterinarian Carlos Jimenez of Complete Equine Health Service in Coatesville, Pennsylvania has practiced acupuncture for 30 years, having been introduced to the modality when one of his own horses a young hunter-jumper prospect developed problems during training.

He was being trained near Pittsburgh, and when we went to visit him, we saw that he wasnt moving right, Jimenez said. A veterinarian at the University of Pennsylvania New Bolton referred Jimenez for corrective shoeing to a well-known farrier who, in turn, suggested that Jimenez consult with a veterinarian certified in acupuncture and chiropractic.

The difference was night and day, Jimenez said. The horse went from not being able to turn his neck to the right to being able to touch his back hip with his nose after both the chiropractic adjustments and the acupuncture. Thats what got me started in integrative therapies in my own practice.

Acupuncture and chiropractic are almost exclusively what Jimenez practices now, with patients ranging from Amish buggy horses to dressage and racehorses.

Besides effectively treating sports-related injuries, acupuncture, especially electro-acupuncture, can help horses with colic, he said. Ill put needles into horses on either side of the spinal cord where they will innervate the intestines. By the end of treatment, I will start to hear gut sounds where there were none before. If the horse has a displaced large intestine, acupuncture may prevent it from turning into a volvulus that would require surgery.

Jimenez has used acupuncture on dying horses to help them peacefully transition, he said.

Kristin Edwards, of Dallas, Pennsylvania, is a small-animal veterinarian and horse owner who added acupuncture to her practice 23 years ago. She became interested when a client gave her a book on the modality, and she realized a calling to pursue training and certification.

Her equine acupuncture patients range from retired performance horses to endurance athletes.

Professional trainer Stephanie Kleinbauer of Laceyville, Pennsylvania, has Edwards perform acupuncture on her four horses once a month to stay ahead of, and even diagnose, problems.

If multiple visits show recurring pain in a particular spot it can be an indication that an injection or some other treatment might be needed, she said. Kristin helped find a problem with Quest, my 23 year old Norwegian Fjord gelding. He seemed so stiff when I went to ride him it was as if his whole body was involved. Through acupuncture, Kristin was able to narrow it down to a tear in his right pectoral muscle, and recommended massage therapy.

Kleinbauer is typically present when Edwards treats her horses and said the release of tension is often visible.

Helping horses to let go of anxiety and stress associated with pain or discomfort is one of acupunctures most fascinating benefits, Edwards said.

I love looking at the emotional component of disease in animals, and acupuncture lets me get into their emotions and help them release whatever issues they are holding onto. Acupuncture enables owners to see the emotions their horse is expressing. It helps them to have a closer relationship.

Horses respond quickly, which is gratifying to her as a practitioner, she said. Their eyes will soften and theyll lick and chew. They want to be helped and feel better, which makes it fun for me.

Lancaster Farmings Mid-Atlantic Horse tells the stories of horses and their people. Big and small horses; fast, slow, harness, carriage and farm horses; wild horses, donkeys, mules, mustangs and more. Mid-Atlantic Horse covers the wide world of the genus Equus. And for every horse story, there are many more about the people who live so closely with their horses.

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Stalking Allegations Against Retired Oregon City Veterinarian Lead to $245,000 Lawsuit – Willamette Week

September 20th, 2022 2:02 am

The harassment began five years ago with a piece of hate mail, sent to Kenneth Fandrichs Oregon City home. It was disguised as a letter from his union, the International Brotherhood of Electrical Workers.

Then, someone broke into Fandrichs truck and left a condom wrapper under a pair of his wifes work gloves.

By the time the Clackamas County bomb squad arrived to remove a large suspicious device from underneath his truck, Fandrich, 56, claims he was well aware of the identity of the culprit.

He filed for a series of stalking orders against 55-year-old Steve Milner, an animal surgeon. In court filings, Fandrich says Milner is responsible for all the incidents, which followed the end of an affair Milner was having with Fandrichs wife.

Fandrichs attorney filed a lawsuit in Multnomah County Circuit Court onSept. 12 for $245,000, and police have filed criminal charges against Milner in Clackamas County for allegedly planting a GPS tracker on Fandrichs truck, again, and violating the stalking order.

WW could not reach either man for comment. Milners attorney declined to speak about the case. Michael Fuller, who represents Fandrich in the civil lawsuit, provided WW with legal documents outlining both sides arguments. They were filed in court following Fandrichs request for a stalking order.

The bizarre saga is noteworthy because of the reluctance of police to intervene. According to the documents, the latest legal actions are the culmination of years of threats by both menand futile pleas for law enforcement to step in.

As recently as 2016, Fandrichs wife worked with Milner at his Oregon City veterinary hospital. (Milner recently retired.) There, the two had an affair, according to a statement Fandrich made to police. It was documented in a report that was included in the legal filings. That affair ended, Fandrich said, and the stalking began.

That police report was a result of a 911 call made by Fandrich on March 2, 2022, after Milner allegedly followed him all the way from Oregon City to Cornelius Pass Road in Hillsboro.

After being pulled over by police, Milner admitted to following Fandrich, according to the police report. Milner told the officer that Fandrich beat his wife, and he wanted to talk with him about it. Milner was taking the issue into his own hands because the police werent doing anything about it, he told the officer.

The officer then talked to Fandrich, who sounded terrified, according to the report. Milner, he said, was going to cut me up into little pieces because he is a surgeon. Milner is a doctor of veterinary medicine and operates on pets. Fandrich told the officer that he and his wife did have marital problems, but that she had been arrested for domestic violence, not him.

Fandrich told the officer that hed been trying to get the police to do something about Milners stalking for years, but they hadnt. He claimed, according to the report, that police [had] advised it was not against the law to place a GPS tracker on someone elses vehicle. (It is, if the owner does not consent.)

Neither the Oregon City Police Department nor the Clackamas County Sheriffs Office immediately responded to a request for comment.

The Hillsboro police officer noted that Milner did not seem receptive to the officers warnings that hed end up in jail if he kept up the harassment. Milner was not cited or arrested.

A few weeks later, Fandrich applied for a stalking order against Milner, who he said is trying to kidnap me and possibly kill me or disfigure me, Fandrich wrote in his application.

Milner unsuccessfully fought the order in court. His attorney, Ross Denison, filed a legal document arguing that the conduct was neither malicious nor undertaken in bad faith. Milner, Denison argued, engaged in the conduct with the sole motivation of protecting his intimate friends[Fandrichs] wifesphysical safety.

Denison played a recording for the court in which Fandrich threatened Milner. He used multiple hateful racial slurs and said he would put a bullet in his head, according to a legal filing that described the recording.

Even with the stalking order, the harassment did not end, prosecutors allege. Last month, Milner was finally arrested for an incident that prosecutors say happened in April. Milner was charged with violating the stalking order for unlawful use of a global positioning system device.

The complaint in Fandrichs recent civil lawsuit includes a still image from a video, allegedly showing Milner placing a tracking device on Fandrichs truck. The lawsuit accuses Milner of invasion of privacy, intentional infliction of emotional harm, trespassing and negligence.

That video, reviewed by WW, shows someone crawling under the vehicle and then running away in the night.

Milner has been released from custody pending a Clackamas County court date next week. In the meantime, hes been ordered to have no contact with Fandrich or Fandrichs wife.

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Stalking Allegations Against Retired Oregon City Veterinarian Lead to $245,000 Lawsuit - Willamette Week

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TheraVet Announces Its Participation in the 2022 ESVOT Congress With 3 Scientific Communications – Yahoo Finance

September 20th, 2022 2:02 am

GOSSELIES, Wallonia, Belgium, September 19, 2022--(BUSINESS WIRE)--Regulatory News:

TheraVet (ISIN: BE0974387194 - ticker: ALVET) (Paris:ALVET), a pioneering company in the management of osteoarticular diseases in pets, announced today its participation in the annual congress of the European Society of Veterinary Orthopaedics and Traumatology (ESVOT) which will be held from 21 to 24 September at the Acropolis in Nice, France.

With nearly 1000 participants, this event is the largest specialised congress in veterinary orthopaedics and traumatology in Europe, bringing together high-quality international speakers and therefore represents a prime showcase for the Company. ESVOT is chaired by Pr. Marc Balligand, who is also the President of the Scientific Advisory Board of TheraVet.

The Company will be particularly well represented with 3 scientific communications. The results obtained through close collaborations with renowned veterinarians will be presented during this conference, proof of the growing interest of the veterinary community in the Company's products:

"Percutaneous cementoplasty as a palliative treatment for dogs with osteosarcoma using a new self-setting bone substitute"Thursday 22 September 2022 at 8:00 pmPoster presentation by Dr. A. Villamonte Chevalier (DVM, PhD), Vet Technical Manager at TheraVet

"Microwave ablation as part of limb sparing multimodal therapy in dog with appendicular osteosarcoma"Saturday 24 September 2022 at 12:10 pmOral presentation by Dr. D. Jacques (DVM, DIPL. ECVS) Clinique Vtrinaire Occitanie, France

"Efficacy and safety assessment of a self-setting bone substitute (alpha-TCP) as an efficient alternative to autografts"Saturday 24 September 2022 at 5:30 pmOral presentation by Dr. G. Ragetly (DVM, PhD, Dipl. ACVS, DIPL. ECVS), CHV Frgis, France

The Company will also have a strong commercial presence for its BIOCERA-VET products, including:

- Drylab sessions allowing veterinarians to test and evaluate the performance of the products,- Videos demonstrating the use of the products in real clinical cases,- Coupons, promotional documents

Story continues

About TheraVet SATheraVet is a veterinary biotechnology company specialising in osteoarticular treatments for companion animals. The Company develops targeted, safe and effective treatments to improve the quality of life of pets suffering from joint and bone diseases. For pet owners, the health of their pets is a major concern and TheraVets mission is to address the need for innovative and curative treatments. TheraVet works closely with international opinion leaders in order to provide a more effective response to ever-growing needs in the field of veterinary medicine. TheraVet is listed on Euronext Growth Paris and Brussels, has its head office in Belgium (Gosselies) with a US subsidiary in Texas.For more information, visit the TheraVet website or follow us on LinkedIn / Facebook / Twitter

About BIOCERA-VETIn close collaboration with an international scientific board, THERAVET has developed a new line of calcium-phosphate and biological bone substitutes, BIOCERA-VET. BIOCERA-VET is a full range of innovative, easy-to-use, efficient & cost-effective bone substitutes indicated in bone surgeries where a bone graft is required and as a palliative alternative in the management of canine osteosarcoma. Based on extremely promising clinical results, this line offers the possibility of a better, more convenient and more efficient orthopedic surgery.

BIOCERA-VET is declined in different lines:

BIOCERA-VET BONE SURGERY RTU, ready-to-use highly injectable self-hardening calcium-phosphate cement

BIOCERA-VET SMARTGRAFT, a naturally osteoconductive bone graft

BIOCERA-VET GRANULES, an affordable biocompatible calcium-phosphate bone substitute

BIOCERA-VET OSTEOSARCOMA RTU, a ready-to-use highly injectable calcium-phosphate bone substitute for cementoplasty

For more information, visit BIOCERA-VET website.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220918005014/en/

Contacts

TheraVet Chief Operating OfficerSabrina Enainvestors@thera.vet Tel: +32 (0) 71 96 00 43

Chief Corporate OfficerJulie Winandinvestors@thera.vet

NewCap Investor Relations and Financial CommunicationsTho Martin / Hugo Willeferttheravet@newcap.eu Tel: +33 (0)1 44 71 94 94

Press RelationsArthur Rouilltheravet@newcap.eu Tel: +33 (0)1 44 71 00 15

NewCap Belgique Press RelationsLaure-Eve Monfortlemonfort@thera.vet Tel: + 32 (0) 489 57 76 52

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TheraVet Announces Its Participation in the 2022 ESVOT Congress With 3 Scientific Communications - Yahoo Finance

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Senate committee sets nomination hearing for top food safety official – Food Safety News

September 20th, 2022 2:02 am

A committee in the U.S. Senate has finally set a nomination hearing to consider Jose Emilio Esteban for the U.S. Department of Agricultures top food safety job.

Nominated on Nov. 12, 2021, by President Joe Biden, Esteban is scheduled to go before the Senate Committee on Agriculture, Nutrition, and Forestry on Sept. 22. The committee has not offered any reason why the nomination hearing has been delayed so long, except for a comment from Sen. Debbie Stabenow, D-MI. At one point she said there was paperwork needed from the White House.

Also set to testify before the Ag Committee are Alexis Taylor, the long-awaited nominee for undersecretary for trade and foreign agriculture affairs at USDA, and Vincent Garfield Logan who is nominated to serve on the Farm Credit Administrations board.

One nominee not scheduled for a confirmation hearing before the committee is Stacy Dean, who is nominated to be the undersecretary of food, nutrition and consumer services. Republicans have kept Dean under scrutiny for her handling of the Thrifty Food Plan reauthorization, which increased SNAP benefits, previously known as the food stamp program. Dean headed up that initiative after it was authorized in the 2018 farm bill.

As for Esteban, he has been waiting for confirmation while working at USDAs Food Safety and Inspection Service (FSIS) as the agencys chief scientist. There he provides scientific advice to support agency policies, including microbiology, chemistry and pathology.

It is his fourth position at FSIS, all within the Office of Public Health Science. Before his current assignment, he was Executive Associate for Laboratory Services, the Scientific Advisor for Laboratory Services and Research Coordination, and the Laboratory Director for the Western Laboratory.

Before joining the USDA, Esteban worked at the Centers for Disease Control and Prevention as an Epidemic Intelligence Service Officer, Staff Epidemiologist, and Assistant Director of the Food Safety Office.

Outside the U.S. federal government, Esteban is the Chair for the Codex Alimentarius Commission Committee on Food Hygiene. This committee sets definitions for international food hygiene standards for international trade.

Esteban was trained as a veterinarian in Mexico and supplemented his training with an MBA, a masters degree in preventive veterinary medicine, and a Ph.D. in epidemiology from the University of California-Davis.

To fill the gap while Esteban has been waiting for his confirmation, the White House named Sandra Eskin on March 17 as Deputy Under Secretary for Food Safety. Deputy jobs do not require Senate confirmation and Eskin went to work shortly after being named to the No. 2 food safety job.

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Senate committee sets nomination hearing for top food safety official - Food Safety News

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Helping osteoarthritic cats live their best lives – DVM 360

September 20th, 2022 2:02 am

Experts offer advice on identifying and managing feline OA and discuss the latest treatments for this painful condition

As veterinary medicine improves and cats live longer, they experience more complications associated with old age, including osteoarthritis (OA), a progressive degenerative joint disease. The median age of cats with OA is 10.2 years,1 and approximately 90% of those over 12 have been found to have OA.2

In an interview with dvm360, Elizabeth Colleran, DVM, DABVP, medical director at Chico Hospital for Cats in Chico, California, offered veterinarians and clients tips for detecting and managing the disease in cats.

According to Colleran, identifying osteoarthritis can be more difficult in cats than in dogs. Cats are secretive, she says, because hiding their emotions is to their advantage. As solitary hunters, they dont have [a] great repertoire of emotional signs on their face...but they do show what they think and how they feel.

One should look for a cat whose lifestyle [has] changed, she explained; for instance, a cat that used to sleep with the owner... [but] isnt sleeping with the owner anymore, that used to climb up to a high point on a cat tree...[but] doesnt do that anymore, or even a cat that is sleeping more than usual.

At the April 2022 Fetch dvm360 conference, Colleran mentioned other signs of OA: avoidance of other household members, increased grumpiness, decreased grooming, restlessness, changes in elimination behavior, clumsiness, and reluctance to jump.3

Pet parents can also use the Feline Musculoskeletal Pain Index, a validated pain score, to check their pets more closely. The Index asks questions about specific indicators of OA that reveal the intensity of a cats pain.

A visual assessment of the cats gait is important, but the environment mustbe carefully controlled. According to Colleran, a quiet, low-stress atmosphere is essential, and the cat should be given time to acclimate to the room. The veterinarian should then gently palpate the joints, even though cats not experiencing joint pain will also be averse to this.

Because OA is complex and usually affects various joints, it can be challenging to stage. Staging is best accomplished by examining the overall impact on the cat. Based on activity and mobility, OA stages are as follows.

When it comes to addressing the disease, its important to tell clients that it cant be treated, only managed to improve mobility and quality of life. A multimodal approach is the most beneficial, Colleran pointed out, because it combines traditional medication, adjunctive therapies, and environmental modification.

On the pharmacological side, nonsteroidal anti-inflammatory drugs can be used. There are also some really helpful adjunctive therapies, like laser therapy [and] acupuncture, she said. Assisi Loop is a favorite of mine because the cats dont mind it at all, she added, and then there are...supplements like omega-3 fatty acids.

Environmental modification includes making the cats space more comfortable so that it can get around more easily and providing it with a warm bed to soothe its joints.

Environmental modification means that were changing the environment to accommodate...something thats going on with the cat. For example...a lot of my clients will build stairs or ramps for the cats to get up to really high places where they like to sit and watch the world go by or watch the birds outside.

At the 2022 convention of the American Veterinary Medical Association,4 Alonso Guedes, DVM, PhD, an associate professor of anesthesia at the University of Minnesota College of Veterinary Medicine, talked about the newest kid[s] on the block: anti-nerve growth factor monoclonal antibodies.

Guedes explained that nerve growth factor (NGF) is synthesized and active on many types of cells: various cells secreteand are affected byit. NGF signaling is upregulated during the chronic inflammation associated with OA, which results in central and peripheral sensitization and hyperalgesia. In patients with OA, the secretion of NGF increases, sensitizing the periphery and central terminal of the neurons in the spinal cord.

The NGF also will activate immune cells, and these...cells will then secrete their mediators, [which]...will then amplify inflammation...so it seems like a good idea...[to use] a nerve growth factor neutralizing antibody to block NGF's many effects, and then the nervous system is able to return to its normal [state]. It is [a] pretty nice concept, Guedes added.

References

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Research shows what is driving shelter overpopulation – Vet Candy

September 20th, 2022 2:02 am

Progress made over decades to control overpopulation of dogs and cats through high-volume spay-neuter surgeries is at risk thanks to the ongoing COVID-19 pandemic, a team of UF researchers conclude in a new study.

The impact felt both at community shelters and veterinary clinics includes sharp declines in spay-neuter surgeries after the initial pandemic-triggered lockdowns, followed by staffing shortages in clinics and shelters, overcrowding and lagging pet adoption rates. All of these problems are compounded by a nationwide shortage of veterinarians, which has been felt even more acutely in shelters and spay-neuter clinics, the researchers say in a study that appears today (Sept. 13) in Frontiers of Veterinary Science.

Progress made over decades to control overpopulation of dogs and cats through high-volume spay-neuter surgeries is at risk thanks to the ongoing COVID-19 pandemic, a team of UF researchers conclude in a new study.

The study focused on the effect of the COVID-19 pandemic on the volume of surgical procedures performed by spay-neuter clinics, said Simone Guerios, D.V.M., Ph.D., a clinical assistant professor of shelter medicine at UF and the studys lead author.

The team drew its research from 212 clinics nationally, all of which make use of the cloud-based clinic management software program Clinic HQ, which is specifically designed for facilities that focus on spay-neuter and preventive health care services.

The high level of spay-neuter achieved over the past five decades is the single most important driver of reduced pet overpopulation and euthanasia in animal shelters, Guerios said. The rise in subsidized spay-neuter access helped drive the euthanasia of shelter pets in the United States from an estimated 13.5 million in 1973 to 1.5 million in 2019.

Using 2019 as a baseline, the UF team aimed to determine the impact of the pandemic on the volume of spay-neuter procedures performed in 2020-2021 at the 212 clinics, which collectively performed more than 1 million surgeries per year and were on track to increase surgeries by 5% over the previous year.

But in the 24 months from January 2020 through December 2021, 190,818 fewer surgeries were performed at the clinics studied than would be expected had 2019 levels been maintained, the researchers found.

If a similar pattern was experienced by other spay-neuter programs in the United States, it would suggest there is a deficit of more than 2.7 million spay-neuter surgeries that animal welfare organizations have yet to address, said co-author Julie Levy, D.V.M., Ph.D., the Fran Marino Endowed Distinguished Professor of Shelter Medicine Education at UFs College of Veterinary Medicine.

All the impacts of the pandemic combined have the potential to undermine progress made in controlling pet populations and euthanasia in shelters, Levy added.

Currently, shelters are in crisis mode, with overcrowding and lagging adoptions, Guerios said. Pet overpopulation seems to be increasing, leading to increased shelter euthanasia for the first time in many years.

The UF College of Veterinary Medicine is responding to societal needs by increasing class size and remodeling its surgical training facility to enhance surgical skills development. The college also offers four courses and clerkships specifically designed to provide students with real-world spay-neuter experience, Levy said.

As part of these hands-on learning opportunities, UF veterinary students spay and neuter thousands of cats and dogs in their local communities, she added.

Through our recent expansion of class size to meet the increasing demand for veterinary graduates, along with unique certificate programs and shelter medicine internships, our college is taking proactive action to address these disturbing trends in animal healthcare and well-being, said Christopher Adin, D.V.M., chair of UFs department of small animal clinical sciences, which oversees the colleges shelter medicine program.

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Undergrad publishes theory on immune dysfunction in space | Cornell Chronicle – Cornell Chronicle

September 20th, 2022 2:02 am

Its been known for decades that astronauts immune systems become suppressed in space, leaving them vulnerable to disease, but the exact mechanisms of immune dysfunction have remained a mystery now a Cornell undergraduate has found a potential solution.

A biological and mechanical engineering double major in the College of Engineering, Rocky An 23 published his theory, MRTF May be the Missing Link in a Multiscale Mechanobiology Approach toward Macrophage Dysfunction in Space, Sept. 12 in Frontiers in Cell and Developmental Biology.

An reviewed the last 20 years of literature on the behavior of macrophages key cells in the bodys immune response in space and recent research about how macrophages respond to forces in normal gravity, identifying a transcription factor that could prove to be the missing piece of the puzzle.

I just kept asking questions about how the data is presented, An said. There are these two really important papers, in particular, one a review of how macrophages are suppressed in microgravity, and another about the mechanobiology of macrophages. I was able to connect these two papers, and that's when the idea came to me. I was really excited, as it was kind of a eureka moment for me.

In space, the lack of gravity changes the shape of the immune cell, and scientists have suspected that changes to the cytoskeleton, the filamented infrastructure of the cell, were involved in immune dysfunction. Recent studies in normal gravity have shown that disturbing the cytoskeleton of macrophages reduces the transport of a particular protein, a transcription factor important for immune response, to the nucleus.

By comparing the studies of cells in microgravity and analyzing the modes of study and associated timescales whether macrophages were actually studied in space, or on a parabolic airplane, or in a simulation of microgravity in the lab An was able to point to this protein, Myocardin-Related Transcription Factor (MRTF), as a probable culprit in immune system dysfunction.

I think its a pretty convincing argument that MRTF is a big part of the problem, An said. I hope it will inspire future studies that really focus on that one protein and the cytoskeleton, and maybe it could be the first step towards a spaceflight immune treatment.

The paper suggests that MRTF could be implicated in the stress on the cardiovascular health of astronauts as well. An also points to other factors that may play a role in immune dysfunction and notes that further research is needed to understand how MRTF interacts with the macrophage nucleus in microgravity.

While An worked independently on the publication, he credits his Cornell professors (including Mingming Wu, professor, and Minglin Ma, associate professor, both of biological and environmental engineering in the College of Agriculture and Life Sciences; Brian J. Kirby, the Meinig Family Professor of Engineering; and Donna Cassidy Hanley, senior research associate in the College of Veterinary Medicine, among many other teachers) for modeling an interdisciplinary approach and encouraging inquiry.

Even before enrolling at Cornell, An had contact with the lab of Theodore Clark, professor of microbiology and immunology in the College of Veterinary Medicine, where An has engaged in research since his freshman year. He also credits his experience on the Cornell iGEM (Genetically Engineered Machines) Project Team, with advising from Jan Lammerding, professor in the Meinig School of Biomedical Engineering, as helping him develop as a scientist.

The biggest help has been the professors and the way theyve taught my classes, An said. And then with research in the lab and my project team, there have been a lot of opportunities to learn independently and ask our own questions.

An also gained valuable experience in summer internships. In 2021, he was chosen as a research associate for NASAs Space Life Sciences Training Program, where he studied the impact of microgravity on cells and co-authored his first paper, an optimization of a modeling framework for studying cells in microgravity.

An then spent the summer of 2022 as an Amgen Scholar at Harvard Universitys Wyss Institute, where he worked in the area of mechanical immunotherapy, exploring therapies that work by manipulating cells structure.

Ive always been interested in cells, but also the mechanics, how cells react to forces, An said. I like this approach because its somewhat new, and I think it's very different from what you generally learn in biology, where everything's a series of chemical reactions. I really enjoy the interaction between the two fields.

Support for Ans publication came from the Cornell Open Access Publication Fund.

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Tuskegee University is named HBCU Institutional Leader by Fulbright Program – Tuskegee University

September 20th, 2022 2:02 am

September 19, 2022

Contact:Thonnia Lee, Office of Communications, Public Relations and Marketing

TUSKEGEE, Alabama-- Tuskegee University has been named a Fulbright Historically Black College and University (HBCU) Institutional Leader for 2022.

For the third consecutive year, the U.S. Department of States Bureau of Educational and Cultural Affairs (ECA) is recognizing selected HBCUs strong engagement with the Fulbright Program, the U.S. government's flagship international academic exchange program. Fulbright HBCU Institutional Leaders have demonstrated noteworthy support for Fulbright exchange participants during the 2020-2021 and 2021-2022 academic years and have promoted Fulbright program opportunities on campus. Tuskegee University is new to the list this year.

The announcement of the 19 Fulbright HBCU Institutional Leaders was made by the State Department as HBCU leaders prepare to gather in Washington, D.C., and virtually for the White House Initiative on HBCUs National HBCU Week Conference, and Fulbright opportunities will be highlighted in events such as the career and recruitment fair during this week.

Assistant Secretary of State for Educational and Cultural Affairs Lee Satterfield commended the HBCUs receiving the Fulbright HBCU Institutional Leader designation this year, noting that HBCUs are an important part of the American and global higher education communities, providing life-changing exchange opportunities for American and international students, faculty, and administrators alike. I hope that these institutions success encourages all HBCUs to engage further with Fulbright and with the State Department.

This is an exceptional note of distinction, said President Charlotte P. Morris. We have always known the caliber of students and faculty whose vigorous academic work influences our society in a variety of areas. This acknowledgment will help our students and the broader community see the enormous value our university brings.

On Nov. 3, a Fulbright HBCU Virtual Workshop will feature representatives of Fulbright HBCU Institutional Leaders sharing best practices for HBCUs to leverage Fulbright Program engagement to support students and faculty, increase campus internationalization, and build global networks. This event is open to the public and is specifically designed for HBCU faculty, staff, and stakeholders.

Our goals for academic excellence mapped out in our Strategic Plan are confirmed once again by this acknowledgment, said Provost Dr. S. Keith Hargrove. We are providing academic tools for scholars and researchers to develop and contribute their best work. By supporting this kind of academic brilliance, it confirms our commitment to remain an HBCU that attracts and maintains a strong community of global scholars.

The Fulbright Program is the U.S. government's flagship international academic exchange program. Since its inception over 75 years ago, the Fulbright Program has given over 400,000 talented and accomplished students, scholars, teachers, artists, and professionals of all backgrounds and fields the opportunity to study, teach, and conduct research abroad, exchanging ideas, and contributing to finding solutions to important international problems.

Each year, the U.S. Congress appropriates funds to the U.S. Department of State to sponsor the Fulbright Program. Many foreign governments contribute substantially as well. Additional funding and in-kind funding is provided by U.S. and foreign host institutions, non-governmental organizations, private organizations, corporate partnerships, and individual donors.

Over the years, we have had faculty and student Fulbright awardees representing Tuskegee around the globe, said Dr. Rhonda Collier, director of the TU Global Office, Fulbright Faculty Liaison and Fulbright Program Advisor. Im so proud of the work were doing to support their study and research.

Over many years the Fulbright Program has designed and implemented a wide range of initiatives to increase participant diversity and inclusion. The program strives to ensure that its participants reflect the diversity of U.S. society and societies abroad. Fulbrighters come from all backgrounds and are selected through an open, merit-based competition, regardless of their race, color, national origin, sex, age, religion, geographic location, socio-economic status, disability, sexual orientation, or gender identity. Information on the Fulbright Programs diversity and inclusion initiatives is detailed on the Fulbright U.S. Student Program website.

For more information on the Fulbright Program, visit http://eca.state.gov/fulbright or contact the Bureau of Educational and Cultural Affairs Press Office by e-mail ECA-Press@state.gov.

Stories about the positive impact of the Fulbright Program over its first 75 years can be found at: https://fulbright75.org

Follow the Fulbright Programs social media accounts and websites for highlights on HBCUs and Fulbright:

2022 Tuskegee University

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NIH researchers develop gene therapy for rare ciliopathy – National Institutes of Health (.gov)

September 12th, 2022 2:11 am

News Release

Thursday, September 8, 2022

Gene augmentation rescues cilia defects in light-sensing cells derived from patients with blinding disease.

Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood. Using patient-derived retina organoids (also known as retinas-in-a-dish), the researchers discovered that a type of LCA caused by mutations in the NPHP5 (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body. The findings not only shed light on the function of NPHP5 protein in the primary cilium, but also led to a potential treatment for this blinding condition. NEI is part of the National Institutes of Health.

Its so sad to see little kids going blind from early onset LCA. NPHP5 deficiency causes early blindness in its milder form, and in more severe forms, many patients also exhibit kidney disease along with retinal degeneration, said the studys lead investigator, Anand Swaroop, Ph.D., senior investigator at the NEI Neurobiology Neurodegeneration and Repair Laboratory. Weve designed a gene therapy approach that could help prevent blindness in children with this disease and one that, with additional research, could perhaps even help treat other effects of the disease.

LCA is a rare genetic disease that leads to degeneration of the light-sensing retina at the back of the eye. Defects in at least 25 different genes can cause LCA. While there is an available gene therapy treatment for one form of LCA, all other forms of the disease have no treatment. The type of LCA caused by mutations in NPHP5 is relatively rare. It causes blindness in all cases, and in many cases it can also lead to failure of the kidneys, a condition called Senior-Lken Syndrome.

Three post-doctoral fellows, Kamil Kruczek, Ph.D., Zepeng Qu, Ph.D., and Emily Welby, Ph.D., together with other members in the research team collected stem cell samples from two patients with NPHP5 deficiency at the NIH Clinical Center. These stem cell samples were used to generate retinal organoids, cultured tissue clusters that possess many of the structural and functional features of actual, native retina. Patient-derived retinal organoids are particularly valuable because they closely mimic the genotype and retinal disease presentation in actual patients and provide a human-like tissue environment for testing therapeutic interventions, including gene therapies. As in the patients, these retinal organoids showed defects in the photoreceptors, including loss of the portion of the photoreceptor called outer segments.

In a healthy retina, photoreceptor outer segments contain light-sensing molecules called opsins. When the outer segment is exposed to light, the photoreceptor initiates a nerve signal that travels to the brain and mediates vision. The photoreceptor outer segment is a special type of primary cilium, an ancient structure found in nearly all animal cells.

In a healthy eye, NPHP5 protein is believed to sit at a gate-like structure at the base of the primary cilium that helps filter proteins that enter the cilium. Previous studies in mice have shown that NPHP5 is involved in the cilium, but researchers dont yet know the exact role of NPHP5 in the photoreceptor cilium, nor is it clear exactly how mutations affect the proteins function.

In the present study, researchers found reduced levels of NPHP5 protein within the patient-derived retinal organoid cells, as well as reduced levels of another protein called CEP-290, which interacts with NPHP5 and forms the primary cilium gate. (Mutations in CEP-290 constitute the most common cause of LCA.) In addition, photoreceptor outer segments in the retinal organoids were completely missing and the opsin protein that should have been localized to the outer segments was instead found elsewhere in the photoreceptor cell body.

When the researchers introduced an adeno-associated viral (AAV) vector containing a functional version of NPHP5 as a gene therapy vehicle, the retinal organoids showed a significant restoration of opsin protein concentrated in the proper location in outer segments. The findings also suggest that functional NPHP5 may have stabilized the primary cilium gate.

The study was funded by the NEI Intramural program. Patient samples were collected at the NIH Clinical Center.

NEI leads the federal governments efforts to eliminate vision loss and improve quality of life through vision researchdriving innovation, fostering collaboration, expanding the vision workforce, and educating the public and key stakeholders. NEI supports basic and clinical science programs to develop sight-saving treatments and to broaden opportunities for people with vision impairment. For more information, visit https://www.nei.nih.gov.

About the NIH Clinical Center:The NIH Clinical Center is the worlds largest hospital entirely devoted to clinical research. It is a national resource that makes it possible to rapidly translate scientific observations and laboratory discoveries into new approaches for diagnosing, treating, and preventing disease. Over 1,600 clinical research studies are conducted at the NIH Clinical Center, including those focused on cancer, infectious diseases, blood disorders, heart disease, lung disease, alcoholism and drug abuse. For more information about the Clinical Center, visit:https://www.cc.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Kruczek K, Qu Z, Welby E, et al. In vitro modeling and rescue of ciliopathy associated with IQCB1/NPHP5 mutations using patient-derived cells. Stem Cell Reports. Sept 8, 2022.

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Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |… – ALS News Today

September 12th, 2022 2:11 am

Repeated muscle injections with Engensis (VM202), Helixmiths investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial.

While the sample size was too small to determine the therapys efficacy, muscle biopsies were collected and will be examined to further evaluate the underlying mechanisms of Engensis.

These data suggest that high dose, repeated treatments of Engensis, were safe and well tolerated, providing a great deal of flexibility in designing dosing schemes for future clinical studies, Helixmith stated in a company press release.

Trial analysis will continue once the full dataset is available, and the company plans to present such findings at a future conference. The next steps for Engensis development will be determined at that time.

Engensis is a non-viral gene therapy that uses Helixmiths proprietary small circular DNA molecule to deliver the hepatocyte growth factor (HGF) gene to cells in the muscle environment.

HGF provides instructions to produce a protein of the same name that helps the body form new blood vessels, prevents muscle loss, and promotes the growth and survival of nerve cells. The therapyis delivered via intramuscular (into-the-muscle) injections.

Helixsmith believes that by increasing HGF production, Engensis has the potential to promote nerve cell and muscle regeneration, thereby countering the progressive loss of motor control that characterizes ALS.

The therapy has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration, both of which are intended to speed its clinical development and regulatory review.

A previous open-label Phase 1/2 trial (NCT02039401) found that four once-weekly intramuscular injections of Engensis (to a total dose of 64 mg) were safe and well-tolerated among 18 ALS patients. Signs that the therapy could slow disease progression were also observed.

These promising findings prompted the launch of a placebo-controlled Phase 2a trial, called REViVALS-1A (NCT04632225), which began patient enrollment last year. A total of 18 ALS patients experiencing motor symptoms in their limbs for four years or less were recruited at four sites in the U.S. and one in Korea.

Participants were randomized in a 2:1 ratio to receive three cycles of either Engensis or a placebo: at studys start, at two months, and at four months. Each cycle consisted of two days of injections to upper and lower limb target muscles, spaced two weeks apart (64 mg total of Engensis or a placebo).

This meant that Engensis-treated patients received a total of 192 mg of medication over the four-month period. All participants were monitored for six months from the studys start.

The trials main goal was to assess the safety and tolerability of Engensis, while efficacy measures were included as exploratory outcomes. These included changes in disability, muscle and lung function, survival, ALS-specific health-related quality of life, and the levels of muscle shrinkage biomarkers.

Top-line data showed that the investigational treatment was generally safe and well-tolerated, with no difference in the frequency of adverse events observed between the Engensis and placebo groups (83% for each).

One case of bronchitis a condition characterized by inflammation in the main airways of the lungs due to infection was observed in the Engensis group but was determined unrelated to treatment.

Injection site reactions were reported by 50% of Engensis-treated patients and 66.7% of those in the placebo group. Most of these reactions were mild or moderate in severity and temporary; no participant discontinued treatment due to the number of injections.

According to Helixmith, efficacy was unable to be evaluated due to the fact that four participants dropped out early from the small study.

Still, muscle tissue biopsies were obtained from injection sites to undergo analyses of muscle atrophy (shrinkage) biomarkers and others.

Since data on Engensis underlying mechanisms have been largely based on animal models, these results are expected to provide valuable information on the understanding of the mechanisms of actions of Engensis, and its effects on the [activity] of human genes, which will greatly help in the development of innovative medicines, the company stated in the release.

Helixmith greatly appreciates the generous and eager participation of the ALS patients, the company added.

Engensis is also being investigated across a range of conditions associated with deficits in circulation, and nerve and/or muscle damage, such as diabetic neuropathy, coronary artery disease, and Charcot-Marie-Tooth disease.

More than 500 patients have been treated with Engensis to date across 10 clinical trials and six different diseases, according to Helixmith. Data from these studies have also supported the therapys favorable safety profile and its ability to increase HGF production.

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Global Cancer Gene Therapy Market Report 2022: Benefits of Gene Therapy Over Conventional Therapies Driving Adoption – ResearchAndMarkets.com -…

September 12th, 2022 2:11 am

DUBLIN--(BUSINESS WIRE)--The "Cancer Gene Therapy Market By Therapy, By End User: Global Opportunity Analysis and Industry Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Cancer Gene Therapy Market was valued at $1,389.42 million in 2020 and is estimated to reach $11,359.35 million by 2030, registering a CAGR of 23.3% from 2021 to 2030.

Cancer gene therapy is a technique used for the treatment of cancer where therapeutic DNA is being introduced into the gene of the patient with cancer. Owing to the high success rate during the preclinical and clinical trials, cancer gene therapy has gained popularity.

Many techniques are used for cancer gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of the gene whose function is abnormal. Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells.

The rise in the prevalence of cancer, the benefits of cancer gene therapy over conventional cancer therapies, and the advancement in this field are the major factors that drive the market growth.

In addition, the surge in government support, ethical acceptance of gene therapy for cancer treatment, and rise in biotechnological funding encouraging the R&D activities for cancer gene therapy and thus fuel the growth of the cancer gene therapy market.

In addition rise in awareness regarding cancer gene therapy is a major factor that drives the global cancer gene therapy market growth.

In addition, an increase in government support for research in gene therapy, ethical acceptance of gene therapy for cancer treatment, and a rise in the prevalence of cancer boost the growth of the cancer gene therapy market. However, the high cost associated with the treatment and unwanted immune responses is expected to restrain the market growth.

Key Benefits For Stakeholders

Key Market Segments

By Therapy

By End User

By Region

Key Market Players

Key findings of the Study

Key Topics Covered:

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: CANCER GENE THERAPY MARKET, BY THERAPY

CHAPTER 5: CANCER GENE THERAPY MARKET, BY END USER

CHAPTER 6: CANCER GENE THERAPY MARKET, BY REGION

CHAPTER 7: COMPANY LANDSCAPE

CHAPTER 8: COMPANY PROFILES

For more information about this report visit https://www.researchandmarkets.com/r/ipoqor

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As Philly becomes a hub for life sciences, a new program will train workers for jobs in the field – The Philadelphia Inquirer

September 12th, 2022 2:11 am

Hopes run high in Philadelphia that the region the scientific home of two of the first cell and gene therapies approved by the FDA will remain a major player as the cutting-edge treatments assume a bigger role in medicine.

To make that happen, Philadelphias life sciences industry will need not just scientists, management, and money, but also skilled workers to help laboratories run smoothly at an ever-growing number of biotech companies in the region and eventually to manufacture cures and treatments for rare diseases and elusive types of cancer.

To help build that skilled workforce, the Wistar Institute, the University City Districts West Philadelphia Skills Initiative, and partners have launched a new biomedical technician training program.

It will enroll 18 students in a 12-week paid training program at Wistar, potentially followed by an additional 10 weeks of hands-on work at Iovance Biotherapeutics Inc. in the Navy Yard and then a $23-an-hour manufacturing job. Iovance, which now employs 150 people in Philadelphia, is developing cancer treatments using cell therapy.

Iovance did not say how many of the trainees it would hire. Iovance officials will interview them after they complete the Wistar part of the training.

We expect to have a number of opportunities to which program participants can apply, Tracy Winton, Iovances senior vice president for human resources, said in a statement.

Cell and gene therapies are still in the early stages of development, but Philadelphia scientists have long played a central part. Luxturna, a gene therapy cure for a rare form of congenital blindness, and Kymriah, a cell therapy treatment for some forms of leukemia, are based on the work of Philadelphia scientists. Both received FDA approval in 2017.

Cell therapy uses modified cells to carry treatment into the body. Gene therapy involves the replacement of defective genes that cause what are typically rare diseases.

The new training effort, scheduled to start Sept. 22, builds on one started in 2000 at Wistar, a nonprofit biomedical research institute in University City, in partnership with Community College of Philadelphia. The original Wistar program, which provided general preparation for work in biotech and until this year was spread over two summers for each cohort, has graduated 196 students.

Recruitment for the new program, which Wistar designed to specifically prepare individuals for jobs at Iovance, started Aug. 23 and runs through Friday. As of last Friday morning, 263 people had applied, according to the West Philadelphia Skills Initiative (WPSI), which for a decade has been training Philadelphians for specific jobs at individual employers, such as Childrens Hospital of Philadelphia and SEPTA.

WPSI is handling recruitment selection for the Iovance training. The selection process for the 18 open spots includes an assessment of mathematical ability and an interview, said Cait Garozzo, managing director of WPSI.

Some folks, obviously, are very desperate for a job, any job, and were not trying to connect people that just want any job to this opportunity. Were trying to connect people that want a career in this industry to this opportunity, Garozzo said.

This is the first time WPSI and Wistar have worked together. Other supporters are the Chamber of Commerce of Philadelphia and the Philadelphia Industrial Development Corp.

If this is successful, we really think this could be a game changer for this region, said Kristy Shuda McGuire, dean for biomedical studies at Wistar. We think this is something we could repeat. We could have more cohorts each year if there are single employers who are interested in this and have a lab-based position and would be interested in taking a whole cohort.

The total budget for the training program was not disclosed.

Wistars original training program which expanded this year to include Montgomery County Community College and will be open to students at Bucks County Community College and Camden County College next year typically sends graduates into biotech jobs or on to further education, McGuire said.

Among the graduates of the Wistar program that have gone on to build careers in life sciences is Lois Tovinsky, 36, who completed the program in 2013 and is now laboratory operations manager for Chimeron Bio, a biotech start-up in the Curtis Building that is working on RNA therapeutics against cancer.

Tovinsky graduated from college with a degree in political science in 2008, when the economy collapsed and jobs were hard to find. She heard about the Wistar program in a science class at Community College of Philadelphia and saw it as a chance to fulfill her interest in science and leap from her job as a dog walker into a science career.

I came to the program with no practical skills in the lab, and my knowledge of science was really just the few courses I had taken and my own interest and enthusiasm that I had for it, said Tovinsky, who now mentors students in the Wistar program.

Tylier Driscoll, 21, a biology major at Community College of Philadelphia, was one of 15 students in the Wistar training cohort that finished early last month.

I definitely wanted to do something over the summer that wasnt working at Aldi, Driscoll said. Before this, I hadnt had any lab experience and I really wanted to get a feel for what it was like to work in a lab. I was working at a supermarket at the time. This is the perfect opportunity for me to get into my field.

As part of his training, he spent five weeks working at BioAnalysis LLC, a contract research organization in Kensington that performs quality analysis on the viruses used in gene therapy.

Now, Driscoll has a part-time job at BioAnalysis that he starts Tuesday, the same day he goes back to CCP for the fall semester. He plans to finish his associate degree in the spring and then attend either Drexel University or Temple University for his bachelors degree.

Lake Paul, the president and founder of BioAnalysis, which he called a minority-owned biotech, said the Wistar program is an awesome opportunity and one that reminds him of his own experience. Paul said he grew up in the hood in Miami and wouldnt have obtained his doctorate at Purdue University without the Upward Bound programs that helped him pursue education.

It is a wonderful, exciting, and unique opportunity for these students, both underrepresented folks and regular folks. And to give them actual training like this is unparalleled, said Paul.

The Philadelphia Inquirer is one of more than 20 news organizations producing Broke in Philly, a collaborative reporting project on solutions to poverty and the citys push toward economic justice. See all of our reporting at brokeinphilly.org.

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Charles River and Cure AP-4 Announce Gene Therapy Manufacturing Collaboration – Business Wire

September 12th, 2022 2:11 am

ALDERLEY PARK, England--(BUSINESS WIRE)--Charles River Laboratories International, Inc. (NYSE: CRL) and Cure AP-4, a non-profit foundation dedicated to raising funds and awareness about Adapter-Protein 4 Hereditary Spastic Paraplegia (AP-4 HSP), today announced a manufacturing collaboration. Charles River, a contract research and development manufacturing organization (CRO/CDMO), will provide High Quality (HQ) plasmid DNA for Cure AP-4s Phase I/II gene therapy trials against AP-4 HSP.

Founded in 2016 by the families of two newly diagnosed AP-4 HSP (SPG47) patients, Molly Duffy and Robbie Edwards, Cure AP-4s gene therapy treatment will look to address the root cause of AP-4 HSP, a rare neurodegenerative disorder, and is intended as a one-time, curative treatment for the patient.

What is AP-4 HSP? AP-4 HSP, also known as AP-4 Deficiency Syndrome, includes four sub-types of HSP: SPG47, SPG50, SPG51 and SPG52. Each of these HSP sub-types is associated with a defective autosomal recessive gene which causes a failure in the AP-4 Adaptor Complex. The phenotype and prognosis for each sub-type is extremely similar. Patients afflicted with any of the AP-4 HSP genetic disorders generally present with symptoms including global developmental delay, microcephaly, seizures, brain malformation, and hypotonia (low-muscle tone). The few patients who learn to walk independently tend to lose that ability a few months or few years later as they develop hypertonia (high-muscle tone) and muscle spasticity. Of the 249 currently confirmed global AP-4 HSP cases, most patients experience mobility in some or all extremities as the disorder progresses and are severely intellectually challenged.

Plasmid DNA Manufacturing ServicesThe collaboration will leverage Charles Rivers market leading expertise in plasmid DNA production, specifically HQ plasmid, which combines key features of GMP manufacture with rapid turnaround times to accelerate the timeline to clinic. DNA plasmids are a critical starting material for many cell and gene therapy therapeutics and demand continues to outstrip supply. In response to this, Charles River recently announced the opening of a state-of-the-art HQ plasmid manufacturing center of excellence to address these supply shortages and support the growing needs of the cell and gene therapy field.

Charles River, with the acquisitions of Cognate BioServices, Cobra Biologics, and Vigene Biosciences in 2021, has extended its comprehensive cell and gene therapy portfolio to include CDMO capabilities spanning viral vector, plasmid DNA and cellular therapy production for clinical through to commercial supply.

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About Cure AP-4Cure AP-4, originally known as Cure SPG47, was founded in 2016 by the families of two newly diagnosed SPG47 patients, Molly Duffy and Robbie Edwards. At the time there were only nine other documented cases worldwide, and due to the extreme rarity of the disorder there are no known treatments or cures.

About Charles RiverCharles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit http://www.criver.com.

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Charles River and Cure AP-4 Announce Gene Therapy Manufacturing Collaboration - Business Wire

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Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European…

September 12th, 2022 2:11 am

WAKEFIELD, Mass.--(BUSINESS WIRE)--Myrtelle Inc., (Myrtelle or the Company), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has classified the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal Product (ATMP), specifically a Gene Therapy Medicinal Product (GTMP). ATMP classification, which is determined by the Committee for Advanced Therapies (CAT), was established to regulate cell and gene therapy and tissue engineered medicinal products, support development of these products, and provide a benchmark for the level of quality compliance for pharmaceutical practices. As a designated GTMP product, rAAV-Olig001-ASPA will follow the Centralized Procedure through the EMA and benefit from a single evaluation and authorization process. Additional benefits established through the ATMP regulation include pathways for Scientific Advice and significant fee reductions for such advice.

rAAV-Olig001 is a novel vector from a class of recombinant AAVs (rAAVs) that selectively target oligodendrocytes the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons and makes up the brains white matter. The Companys lead program is in Phase 1/2 clinical development for Canavan disease (CD) a fatal childhood genetic disorder characterized by the degeneration of the white matter in the brain. The production of myelin is affected in CD due to a mutation in the Aspartoacylase gene (ASPA) leading to deficiency in Aspartoacylase enzyme (ASPA). The oligodendrocyte-targeted gene therapy using the rAAV-Olig001 vector is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartic Acid (NAA), a neurochemical abundant in the brain, and supporting myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.

In addition to ATMP classification, rAAV-Olig001-ASPA has been granted US Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA which support the Companys mission to provide treatments for patients with CD. "The designation by the EMA of rAAV-Olig001-ASPA as a Gene Therapy Medicinal Product as a potential treatment for patients with Canavan disease provides important benefits in the development of this innovative therapy. The ATMP classification will facilitate discussions with the EMA as part of our strategy to seek product registration in the EU," said Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle.

ABOUT MYRTELLE

Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer for its lead program in Canavan disease. For more information, please visit the Companys website at: http://www.myrtellegtx.com.

ABOUT CANAVAN DISEASE

Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. CD patients are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD and only palliative treatments are available. More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.

Forward-Looking Statements

This press release contains forward-looking statements. Words such as may, believe, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding rAAV-Olig001-ASPA as a potential treatment for patients with Canavan disease. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelles program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelles expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

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Myrtelle's rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European...

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Urovant Sciences Receives Best in Category Award for Abstract Highlighting Investigational Novel Gene Therapy, URO-902, Presented at 2022…

September 12th, 2022 2:11 am

IRVINE, Calif. & BASEL, Switzerland--(BUSINESS WIRE)--Urovant Sciences, a wholly owned subsidiary of Sumitovant Biopharma Ltd., receives coveted Best in Category award for an interim 12-week analysis from the ongoing Phase 2a trial of an investigational novel gene therapy product, URO-902 (plasmid human cDNA encoding maxi-K channel). The award-winning abstract was presented at the 2022 International Continence Society annual meeting on September 8, 2022. The 2022 ICS Annual meeting is being held September 7-10, 2022, in a hybrid format with both online and in person participation (Vienna, Austria).

According to ICS, this honor is awarded to the highest-scoring abstract in each category. Scores are awarded by the ICS scientific committee members, external reviewers, and scientific session chairs. Abstracts are judged based on criteria of scientific merit, originality/topicality, and clinical relevance. Review the full 2022 Abstract Awards List here.

The podium presentation at ICS 2022 took place on Thursday, September 8, at 10:20 Central European Time (CET). Presentation #6 in Scientific Podium Session S1, Best Urology, was titled, Efficacy and Safety of a Novel Gene Therapy (URO-902; PVAX/HSLO) in Female Patients with Overactive Bladder Syndrome and Urge Urinary Incontinence: Results from a Phase 2A Trial. The presentation described a prespecified, 12-week interim analysis of a 48-week multicenter, randomized, double-blind, placebo-controlled, dose-escalation study (NCT04211831). URO-902 was administered using direct intradetrusor injections via cystoscopy under local anesthesia. The presenting author was Kenneth Peters, M.D., Principal Investigator, and Chief of the Department of Urology at Beaumont Hospital, Royal Oak; Medical Director of the Beaumont Womens Urology and Pelvic Health Center; and Professor and Chair of Urology of the Oakland University William Beaumont School of Medicine in Rochester, Mich.

We are delighted that this presentation has received the Best in Category Prize: Overactive Bladder, reflecting the high-quality scientific research involved, said Dr. Peters. The promising interim safety and efficacy findings from this prespecified analysis indicate that URO-902 has potential as a therapeutic option for overactive bladder patients who have failed oral pharmacologic therapy.

At week 12, both URO-902 24 mg and 48 mg were associated with clinically relevant improvement in mean daily micturition (urination), urgency episodes, UUI episodes, OAB questionnaire symptom bother score, and proportion of patient global impression of change responders. Treatment-emergent adverse events occurred in 45.5% of patients receiving URO-902 24 mg, 46.2% receiving 48 mg, and 50.0% receiving placebo. The most commonly occurring adverse event was urinary tract infection (0% in individuals receiving the 24 mg dose of URO-902; 15.4% in those receiving the 48 mg dose; and 3.8% in those receiving placebo). One patient in the 48 mg arm of the study had asymptomatic elevated post-void residual urine volume at week 2; this resolved spontaneously and did not require catheterization.

URO-902 is a unique potential treatment for OAB. It brings together the accessibility of the anatomy of the condition with a new innovative approach to therapy, said Sef Kurstjens, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Urovant Sciences. Later this year, Urovant anticipates 48-week data from the Phase 2a trial, at that point, well have a greater sense of the durability of the therapy and our proposed next steps.

The data were first presented earlier this year at the 2022 annual meeting of the American Urological Association (AUA2022) in New Orleans, La., from May 13-16, 2022.

About Overactive Bladder

Overactive bladder (OAB) is a clinical condition that occurs when the bladder muscle contracts involuntarily. Symptoms may include urinary urgency (the sudden urge to urinate that is difficult to control), urgency incontinence (unintentional loss of urine immediately after an urgent need to urinate), frequent urination (usually eight or more times in 24 hours), and nocturia (waking up more than two times in the night to urinate).1

While 33 million US adults experience the bothersome symptoms of OAB, approximately 546 million people 20 years are affected by OAB worldwide. 1,2

About the Phase 2a Study of URO-902

The 48-week multicenter study was a randomized, double-blind, placebo-controlled trial to evaluate the efficacy, safety, and tolerability of a single physician administered dose of URO-902, a novel gene therapy being developed for patients with OAB who have not been adequately managed with oral or transdermal pharmacologic therapy. URO-902 is administered via direct intradetrusor injections into the bladder wall under local anesthesia in patients who are experiencing OAB symptoms and urge urinary incontinence (UUI).

The Phase 2a trial enrolled 80 female patients in two cohorts: the first cohort received either a single administration of 24 mg of URO-902 or matching placebo, and the second cohort received 48 mg of URO-902 or matching placebo into the bladder wall. Multiple outcome measures were explored, including the effect on the number of micturitions, urgency episodes, and quality-of-life indicators compared to placebo, 12 weeks post-administration, as well as an assessment of the safety and tolerability of this potential new therapy. Patients were followed for up to 48 weeks after initial administration.

About URO-902

URO-902 (plasmid human cDNA encoding maxi-K channel) has the potential to be the first gene therapy for patients with OAB. If approved, this innovative treatment has the potential to address an unmet need for patients who have failed oral pharmacologic therapies.

References: 1. Irwin DE, Kopp ZS, Agatep B, Milsom I, Abrams P. Worldwide prevalence estimates of lower urinary tract symptoms, overactive bladder, urinary incontinence and bladder outlet obstruction. BJU Int. 2011;108(7):1132-1138. doi:10.1111/j.1464-410X.2010.09993.x

2. Leron E, Weintraub AY, Mastrolia SA, Schwarzman P. Overactive bladder syndrome: evaluation and management. Curr Urol. 2017;11:117-125. doi:10.1159/000447205

About Urovant Sciences

Urovant Sciences is a biopharmaceutical company focused on developing and commercializing innovative therapies for areas of unmet need, with a dedicated focus in Urology. The Companys second product candidate, URO-902, is a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., intends to bring innovation to patients in need in urology and other areas of unmet need.

About Sumitovant Biopharma

Sumitovant is a technology-driven biopharmaceutical company accelerating development of new potential therapies for patients with high unmet medical need. Through our subsidiary portfolio and use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovants subsidiary portfolio includes wholly-owned Enzyvant, Urovant, Spirovant, and Altavant, and one majority-owned subsidiary that is publicly listed: Myovant (NYSE: MYOV). Sumitomo Pharma is Sumitovants parent company. For more information, please visit http://www.sumitovant.com.

UROVANT, UROVANT SCIENCES, the UROVANT SCIENCES logo are trademarks of Urovant Sciences GmbH, registered in the U.S. and in other countries. All other trademarks are the property of their respective owners. 2022 Urovant Sciences. All rights reserved.

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The gene therapy that could transform the lives of millions – ABC News

September 12th, 2022 2:11 am

Tegan Taylor: There are a few things in life that are just inevitable; death, taxes, the genes you're born with. At least, that has been the case for pretty much every generation up until now. Gene therapies have the potential to change the trajectory of disease, and I've been talking to two people on the frontline of that shift.

Until about three years ago, Robert Lamberth had a disease that was incurable. I mean, it was literally in his genes.

Robert Lamberth: Not as a newborn, but yes, very, very young when I had my first bleed. Three, I think it might have been for me, back in the early '80s it was, a long time ago now.

Tegan Taylor: When he was born, he inherited a certain recessive gene that stopped his body from producing one of the essential factors you need for your blood to clot.

Robert Lamberth: Bleeding internally into my major weight-bearing joints, so ankles and knees. And as I got older, I'd have more odd bleeding into muscles in my legs and parts of my stomach and those sorts of things, so it was a little bit more serious when you have large muscle bleeds. The pressure of the bleeding can affect your organs, so that's quite serious.

Tegan Taylor: Managing haemophilia A is miles easier than it was a couple of decades ago. When he was little, Robert needed intravenous injections of his missing clotting factor, given in a hospital. When he got older, he didn't need to go to hospital anymore. Regular injections of the clotting factor were a feature of his life all the way through into his 30s. But not anymore.

John Rasko: We dream of cures in gene therapy but hesitate to use the word

Tegan Taylor: For decades, John Rasko has been chasing ways to change people's fates.

John Rasko: For the last 20-plus years we've been doing clinical trials using viral vectors to transfer a gene into humans for a therapeutic purpose.

Tegan Taylor: Professor Rasko is a haematologist and pathologist who spent much of his career studying genes, stem cells and basically how to hack processes inside the human body. And he is one of many scientists around the world trying to figure out ways of swapping out disease-causing genes in a way that, in time, could be used for pretty much any genetic disease.

John Rasko: When we reflect on rare diseases, it's often worth remarking and reminding ourselves that rare diseases of course by definition are rare, usually less than one in 5,000 or 10,000 people, but collectively rare diseases are very common when you add them all up because there are many thousands of them, lead to a burden of disease such as the commonality of diabetes or even some forms of cancer. So the problem is that of all the rare diseases, which some people say are more than 4,000 affecting humans, 80% of those rare diseases have a genetic basis. And of those diseases, only 5% have a specific therapy. So this is an incredible unmet need in human health.

Tegan Taylor: And the solution he and his colleagues have come up with might sound a bit familiar. It works in a similar way to the Covid vaccine made by AstraZeneca. It uses a harmless virus to take a genetic message into the body.

John Rasko: And that vector system is used to then ferry that genetic payload intravenously to the liver where it takes up residence, and hopefully after a single injection, corrects that person's genetic abnormality for the rest of their life. It's unimaginable, but a single injection can alter the course of a genetic disease that would otherwise affect a person from birth to death.

Tegan Taylor: Robert was part of the clinical trial Professor Rasko was involved in, testing the gene therapy.

Robert Lamberth: It would be three years ago now in May 2019 when I had that one single dose of the good stuff, and then that clearly worked its magic and now I'm growing my own factor VIII. I've had one breakthrough bleed.

Gene therapy for me, Tegan, has been quite revolutionary, so from a position of having 0.5% of clotting factor in my blood, I'm now growing my own factor VIII in my liver and I'm at about 15% clotting factor, which is an extraordinary growth.

Tegan Taylor: In August, Europe granted conditional approval for a haemophilia A therapy like the one Robert received. It hasn't been approved in Australia yet, although we do use gene therapy for other conditions, like spinal muscular atrophy, and genetic causes of blindness.

John Rasko: We are only at the very start of this genetic revolution. There are thousands of genetic diseases that affect humans, and we've only just started scratching the surface of where we can go with these gene-based therapeutics.

Tegan Taylor: Because Robert got the gene therapy as an adult, he's still living with the damage haemophilia A had already done to his body, but that doesn't mean it hasn't been transformative.

Robert Lamberth: I can just do so much more. I can be out there doing everything that I love at work and at play and going to the gym, without fear of having a micro-bleed the next day and being cross and crotchety and painful and grumpy at work, and then it turning into a more major bleed and then having to go and seek therapy, which means even more down-time. The sooner that we could roll out some gene therapy for younger people would be great.

Tegan Taylor: Robert Lamberth, who received gene therapy for haemophilia A, finishing us off there. And we also heard from Professor John Rasko from Royal Prince Alfred Hospital and the Centenary Institute at the University of Sydney.

Norman Swan: It's interesting how things have advanced there, Tegan. A few years ago, not so long ago, gene therapy could have been quite toxic because of the virus that they were using to carry the gene in, and you've got to hit the target, it can't be wasteful, and sometimes the virus did harm in its own right. So it's taken a long time to get that right, but the potential, as John Rasko says, is huge and it goes from cancer through to these inborn errors that you get such as haemophilia A.

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The gene therapy that could transform the lives of millions - ABC News

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Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market – Business Wire

September 12th, 2022 2:11 am

GRENOBLE, France--(BUSINESS WIRE)--Carroucell, the microcarrier supplier for cell culture in bioreactor, announced today that it has raised a total of 1.5 million. The funding includes the closing of a Series A financing, led by the Novalis Biotech Acceleration fund and with participation of Crdit Agricole des Savoie (CADS), as well as support from Bpifrance. The funding will be used to accelerate corporate growth through industrialization of the companys platform technology and ramping up of mass production processes to GMP standards.

Carroucell has developed a disruptive technological platform that offers unique flat shape microcarriers with a glass xenofree composition for cell culture in bioreactors. Unlike existing technologies, the combination of these novel microcarriers combined with the flexibility of the production process enables a faster, more optimized scale-up of the clinical phases. This more cost-effective process could provide customers with a more accelerated time and pathway to market.

For the first time, microcarrier customization and a more customer-oriented service are available for the development of the new applications into the cell culture and bioproduction market. There are many challenges with biomanufacturing performance. We believe our unique microcarrier technology and ability to address customer specific needs will overcome most challenges and stimulate a revolution in the sector moving forward, said Tarek Fathallah, Founder and President of Carroucell.

Carroucell is creating a new standard in biomanufacturing, which could help to facilitate patient access to many more innovations in cell and gene therapy in the future, said Jan Van den Berghe, co-founder and managing director of Novalis Biotech, who has also been appointed to the board of directors. When customers adopt Carroucell's technology platform, they are able to optimize the yield and the quality of the cell culture, solving the low-performance problem in bioproduction we see today.

The complex environment of cell culture in bioreactors and the increasing number of new applications requires an innovative approach to guarantee the balance of the system. Carroucells microcarrier plays the role of regulator of this system by ensuring its optimization, said Takis Breyiannis, CEO of Carroucell.

About Novalis BiotechNovalis Biotech (Ghent, Belgium) is an early-stage venture capital investor in technologies that revolutionize healthcare. The companys core competence lies in digitalization in the life sciences with a focus on bioinformatics, genomics and diagnostics. Novalis strongly believes in applying innovative enabling technology to advance the prevention, diagnosis, or treatment of a disease. For more information, please visit http://www.noval.is.

About CarroucellCarroucell is disrupting the biomanufacturing sector with its patented, innovative microcarrier and flexible process solution for customers. The microcarriers are based on a major innovation in the field of sol-gel process, which allows the production of bioactive microstructures not achievable by existing technologies. In the bioreactor, cells can cling and multiply in "3D" and allows cultivation of a large quantity of cells in a restricted volume. Carroucell has a partnership with Etablissement Franais du Sang (EFS), which enabled the validation of its microcarriers and facilitated first commercial orders. Carroucell was founded in 2016 by Tarek Fathallah. For more information, visit http://www.carroucell.com.

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Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market - Business Wire

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