Monthly Archives: August 2021

Calidi Biotherapeutics Announces Exclusive License Agreement with City of Hope and the University of Chicago for Novel Oncolytic Virotherapy…

Posted: August 18, 2021 at 1:53 am

DetailsCategory: DNA RNA and CellsPublished on Monday, 16 August 2021 17:11Hits: 496

LA JOLLA, CA, USA I August 16, 2021 I Calidi Biotherapeutics, Inc., a clinical-stage biotechnology company with novel allogeneic stem cell platforms for delivery of oncolytic viruses, together with the University of Chicago and City of Hope, a world renowned NCI-Designated Comprehensive Cancer Center, based in Duarte, California, have entered into an exclusive worldwide licensing agreement for patents covering cutting edge therapies using an oncolytic adenovirus in combination with a clinical grade allogeneic neural stem cell line.

City of Hope (COH) scientists, led by Dr. Karen Aboody in collaboration with Dr. Maciej Lesniak's team at University of Chicago, and later Northwestern University, have used COHs exclusive GMP grade immortalized, clonal human neural stem cell line, to selectively deliver an oncolytic adenovirus to tumor sites. Dr. Aboody and Dr. Lesniak, together with Dr. Rachael Mooney at COH, have spent 13 years in a passionate effort to translate promising pre-clinical results into the clinic, attaining FDA approval for commencing a first-in-human Phase-1 trial in recurrent glioma patients.

We are very excited about the partnership and collaboration with Calidi Biotherapeutics. Their deep understanding and expertise using allogeneic stem cells as a delivery platform to protect, deliver, amplify, and potentiate oncolytic virotherapy, can potentially result in a significantly more effective treatment for cancer patients with invasive tumors, commented Dr. Karen Aboody, Professor, Department of Developmental and Stem Cell Biology, City of Hope National Medical Center & Beckman Research Institute.

The first wave of Oncolytic Viruses were novel, but lacked the ability to efficiently deliver the virus to tumor sites, due to the human complement immune system inactivating the viruses, usually within one hour of patient injection, thus resulting in a lack of efficacy, stated Allan Camaisa, Co-Founder, Chairman and CEO of Calidi Biotherapeutics. We believe this collaboration with City of Hope will allow us to implement Calidis proprietary techniques together with City of Hopes novel approach to glioblastoma and other malignant tumors, using neural stem cells combined with an oncolytic adenovirus. This FDA approved Investigational New Drug (IND), planned for patient trials in the first quarter of 2022, increases Calidis drug pipeline and gives our company a tumor-tropic stem cell line to use for oncolytic virus delivery in cancer patients.

This exclusive license agreement, which was executed by the University of Chicagos Polsky Center for Entrepeneurship and Innovation, transferred the COH/University of Chicago IND to Calidi for the commercial development of a licensed product. The agreement grants to Calidi commercial exclusivity in using neural stem cells with the adenovirus known as CRAd-pk-S-7 for oncolytic virotherapy.

Calidis scientific and medical teams are very excited to contribute in the development of this promising technology that has significant potential to help many patients with advanced tumors, said Boris Minev, MD, President, Medical and Scientific Affairs at Calidi Biotherapeutics. We are delighted to collaborate with the outstanding researchers and clinicians who developed this novel oncolytic virotherapy approach.

About Calidi Biotherapeutics

Calidi Biotherapeutics is a clinical-stage immuno-oncology company with proprietary technology that is revolutionizing the effective delivery of oncolytic viruses protected by stem cells for targeted therapy against difficult-to-treat cancers. Calidi Biotherapeutics is advancing a potent allogeneic stem cell and oncolytic virus combination for use in multiple oncology indications. Calidis off-the-shelf, universal cell-based delivery platform is designed to protect, amplify, and potentiate oncolytic viruses currently in development leading to enhanced efficacy and improved patient safety. Calidi Biotherapeutics is headquartered in San Diego, California. For more information, please visit http://www.calidibio.com.

About University of Chicago

The University of Chicago is a leading academic and research institution that has driven new ways of thinking since its founding in 1890. As an intellectual destination, the University draws scholars and students from around the world to its campuses and centers around the globe. The University provides a distinctive educational experience and research environment, empowering individuals to challenge conventional thinking and pursue field-defining research that produces new understanding and breakthroughs with global impact.

The Polsky Center for Entrepreneurship and Innovation applies world-class business expertise from the University of Chicago Booth School of Business to bring new ideas and breakthrough innovations to market. Home of the Universitys technology transfer office, the Polsky Centers dedicated team of professionals with deep technical expertise enabling technology commercialization perform market analysis, manage intellectual property, identify partners, and negotiate partnerships and licenses for discoveries and inventions developed by faculty, researchers, and staff. Learn more at polsky.uchicago.edu and follow us on Twitter @polskycenter.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin, monoclonal antibodies, and numerous breakthrough cancer drugs are based on technology developed at the institution. Translational Genomic research (TGen) became a part of City of Hope in 2016. AccessHope, a wholly owned subsidiary, was launched in 2019, dedicated to serving employers and their health care partners by providing access to City of Hopes exceptional cancer expertise. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is ranked among the nations Best Hospitals by U.S. News & World Report. Its main campus is located in Pasadena, California, near Los Angeles, with additional locations throughout Southern California and in Arizona. For more information about City of Hope, follow us on Facebook, Twitter, YouTube, or Instagram.

SOURCE: Calidi Biotherapeutics

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Calidi Biotherapeutics Announces Exclusive License Agreement with City of Hope and the University of Chicago for Novel Oncolytic Virotherapy...

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Dont ignore those stomach woes – The New Indian Express

Posted: August 18, 2021 at 1:52 am

Express News Service

BENGALURU:Almost everyone has experienced pain in the stomach at some point of time or the other. Whether from eating too much junk food or acquiring virus, we all know what it is like to suddenly be affected with stomach problems. Its also understood that not all stomach pain is the same. Stomach is an important part of the human body and is significantly linked to overall health and disease.

Stomach pain could be a sign of a wide range of health issues, and this could also be due to multiple reasons and can be the cause of concern if it continues. It could be due to a simple infection or development of cancerous cell in the stomach. Hence such pain should never be neglected.

Stomach cancer mostly affects the elderly and two-thirds of those who have it are over the age of 65. It is an abnormal growth of cells that begins in the stomach and spread to other organs of the body like the liver, lungs, and bones. Since stomach cancer usually doesnt cause any early symptoms, it often goes undiagnosed until it spreads to other parts of the body. Weight loss and continued abdominal pain are the most common symptoms at initial diagnosis.

Stomach cancer typically begins as precancerous that can develop into cancer slowly over time. Though some of these changes are evident in diagnostic procedures such as endoscopy, they usually dont cause any symptoms until the cancer progresses. The symptoms include:

Although genetic predisposition can impact the risk of developing stomach cancer, certain lifestyle factors also play a key role. One may be more likely to be diagnosed with stomach cancer if you consume a lot of processed foods and meat and are obese. Lifestyle changes like losing weight, avoiding large meals, reducing the intake of salty and processed foods, following a diet rich in fruits, vegetables, lean proteins and avoiding tobacco use can help in decreasing the risk of stomach cancer.

TreatmentTreatment depends upon the stage of tumour and fitness of the patient to undergo surgical procedure. Treatment can be broadly categorised into curative and palliative. Curative treatment intends to eradicate the disease. To get the best results in advanced tumours, chemotherapy, radiotherapy, and surgery are combined in an approach called multimodal treatment. Depending upon the extent of tumour, chemotherapy can be given before surgery or after surgery.

Suffering from indigestion or heartburn after a meal doesnt mean you have cancer. But if symptoms persist or gets worse, it is better to consult your health care professional and get checked immediately.

(The writer is a consultant surgical oncology, HCG Cancer Hospital Bengaluru)

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People and places at Penn | Penn Today – Penn Today – Penn Today

Posted: August 18, 2021 at 1:51 am

For many students, attending college marks the first time away from home, the first time managing a budget, even the first time doing laundry. New spaces, new classes, new friends, new routines. This year, its not just new for the freshmen. For many, late August will mark their first return to campus since March 2019. In anticipation of Move-In, Penn Today asked six undergraduate students in the Philadelphia area this summer to divulge their favorite spots.

Ariana Jimnez found her niche on an exploratory walk, while Steven Chen and Quinn Gruber found theirs through special interests, and Lucas Monroe gravitates to athletic history. Francisco Barrera chose a study spaceand where he goes to decompress. Dorms, libraries, and outside spaces are all on the list. Morgan Bacon is most looking forward to Hey Day to kick off this semester. Its nerve-wracking to be declared a senior, but also very exciting. Ive enjoyed my time at Penn so much, she says. I can't wait to reconnect with my friends and just hang out on the grass.

Steven Chens first engagement with Penn was through the Netter Center for Community Partnerships, and as aPenn Program for Public Service intern, the biology major from Warminster, Pennsylvania now works through Netter Centers OurSpace to host weekly meetings with a group of West Philadelphia and Penn students at the LGBT Center, one of his favorite campus spots. Its a very accepting space, Chen says, a place for them to have fun and express themselves.

The sophomore is currently collaborating with the LGBT Center and the Netter Center to develop aqueer sexual health education program that utilizes peer-assisted learning. A pre-med student, Chen is interested in providing health care access to queer people and people of color. You have to be healthy to pursue your other dreams, he says. Health is the foundation.

I like a good puzzle or challenge, says Ariana Jimnez, who plans to investigate white-collar crime as an FBI agent. This requires business-industry experience, so the sophomore from Plainfield, Illinois is enrolled in the Wharton School, concentrating on finance and social impact.

She passed the benches in the engineering quad on a walk one day, and which has since become a favorite place to chill and listen to music, Jimnez says. Currently Im bingeing Taylor Swift before her new album comes out.

Jimnez is looking forward to having the opportunity to engage with people face-to-face this fall, she says. Shes also looking forward to living in New College House West. You already know Im going to be exploring that as soon as I get in there, she says. While on routine walks, Im always trying to look in to see if it looks cool, Jimnez says. And it does look cool.

Francisco Barrera of Miami, Florida is really interested in the energy problem. Barrera wants to part of the solution, so he applied to Penns Vagelos Integrated Program in Energy Research, where the senior is studying engineering and physics. Barrera spent his summer doing research with Deep Jariwala of the Device Research and Engineering Laboratory, looking into new materials and new configurations for maximizing light absorbance and efficiency for the next generation of solar cells, he says.

Barreras favorite place to study is the Fisher Fine Arts Library. Im always surrounded by STEM. The Fine Arts Library is a little bit of an escape from that, he says. I was always totally inspired by how beautiful the inside was. Its so quiet compared to other study spaces.

When not working, Barrera heads to the beach volleyball court, a great stress reliever, he says, especially during freshman year when the Miami student was adjusting to college life. The sandy court makes it more fun and athletic, Barrera says, Im more willing to throw myself down to dive for a ball.

A double major in English and Italian from Cortlandt Manor, New York, Quinn Gruber loves the Student Projects Space at the Kelly Writers House. The space is special for me because its the home of the Zine Library, a student-run collection of zines (small, self-published books/pamphlets/print objects) that Ive helped curate for three years now, with Alyson del Pino (C21) and now Victoria Garcia (C23), says Gruber, currently a senior. I love hanging out on the green beanbag with a hot cup of tea and reading the amazing zines that we have in the library, and appreciate the space as a great resource for students who make art,since we have supplies,books, and printing available for everybody.

Gruber also plays violin in a Penn Chamber Quartet, an important reprieve from the pressures of work and school, they say. Gruber enjoys visiting the Eugene Ormandy Music & Media Center to borrow whatever piece Im working on, whether for Chamber or myself, and to discover new music by just wandering through the stacks, they say.

I was always really passionate about nutrition, and more specifically childhood nutrition, says Morgan Bacon. Growing up in Philadelphia, Bacon was acutely aware of food deserts, and began working with the Food Trust. Along with social determinants, she realized how big of a role nutrition played in heath, and applied to Penn Nursing.

The Annenberg Center is one of Bacons favorite places, because the senior attended a performing arts camp here as a child, which piqued my interest in college, she says. It all seemed like a glamorous life.

Once on campus, Bacon spent two years living at Lauder College House. I just loved it so much, she says. The courtyard area was a space where the house could come together.

Im on the basketball team, so thats an easy choice for me, says Lucas Monroe of Abington, Pennsylvania, of why he chose the Palestra as one of his favorite campus spots. But Im also a huge basketball nerd and the Palestra is the cathedral. Its Mecca. More games have been played there than in any other college arena, and thats why its so historic. I like to sit at the top, he says. Ill do homework up there; Ill read up there.

Monroe, a junior majoring in political science with a minor in Africana Studies, also likes to work on the hill outside of Van Pelt-Dietrich Library. Its a peaceful spot, he says, but also one thats good for people watching. You have the library to the left of you, College Hall diagonal. The Franklin statue and the Button are all right there; theres a lot of cool things you can look at.

Homepage image: Clockwise from top left: Morgan Bacon, Ariana Jimnez, Francisco Barrera,Quinn Gruber, Lucas Monroe, and Stephen Chen introduce their favorite places at Penn.

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FDA Puts APB-102 Gene Therapy for SOD1 ALS on Fast Track – ALS News Today

Posted: August 5, 2021 at 2:51 am

The U.S. Food and Drug Administration (FDA) has granted fast track status to Apic Bios experimental therapy APB-102, designed for amyotrophic lateral sclerosis (ALS) patients who carry mutations in the SOD1 gene that lead to misfolded proteins in cells.

This designation accelerates the development of investigational therapies that address unmet medical needs in serious or life-threatening conditions. It makes Apic Bio eligible for more frequent meetings with the FDA and discussions about APB-102s development plan.

We are pleased that the FDA recognizes the significant unmet need for treatments for SOD1 ALS, an always fatal neurogenerative disorder, where mutations in the SOD1 gene account for approximately one-fifth of all inherited forms of the disease, Jorge Quiroz, MD, executive vice president and chief medical officer of Apic Bio, said in a press release.

About 1520% of people with familial ALS and 12% of those with sporadic ALS carry mutations in the SOD1 gene. These mutations result in the production of neurotoxic forms of the SOD1 protein, an enzyme responsible for the removal of free radicals, which is important for cellular health.

APB-102 is a gene therapy designed to address ALS cases caused by SOD1 mutations. It consists of a microRNA a small RNA molecule that latches onto intermediate RNAs carrying the genetic instructions for protein production that binds to a certain portion of the SOD1 RNA sequence and prevents the SOD1 protein from being produced.

This microRNA molecule is contained inside a harmless adeno-associated virus and delivered into the spinal canal via a direct (intrathecal) injection. In a proof-of-concept study involving two ALS patients, APB-102 lowered SOD1 levels in their brain and spinal cord.

The FDA recently cleared a Phase 1/2 clinical trial to investigate APB-102s safety, tolerability, and efficacy in ALS patients with SOD1 mutations. The study is scheduled to begin early next year, and will be conducted in three parts.

In part 1, study participants will receive single but ascending doses of APB-102 to determine an optimal treatment dose. In the second part, patients will be randomly assigned to receive either a placebo or APB-102, at the dose determined in part 1. Part 3 will consist of an extended follow-up.

APB-102 received orphan drug status from the FDA in July 2019, a designation intended to promote the development of investigational therapies for rare and serious diseases. It offers several benefits, including exemption from FDA application fees and seven years of market exclusivity upon approval.

We believe in the therapeutic potential of our gene therapy candidate APB-102 that targets the underlying pathophysiology of the disease, and we remain on track to initiate our Phase 1/2 study of APB-102 in early 2022, said Quiroz.

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Ultragenyx to Participate in Gene Therapy Panel at Wedbush PacGrow Healthcare Conference – Yahoo Finance

Posted: August 5, 2021 at 2:51 am

NOVATO, Calif., Aug. 04, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President will participate in a panel titled Miss Con-GENE-iality - Updates in Gene Tx on Wednesday, August 11, 2021 at the Wedbush PacGrow Healthcare Conference at 12:00 PM ET.

The live and archived webcast of the presentation will be accessible from the companys website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: http://www.ultragenyx.com.

Contact Ultragenyx Pharmaceutical Inc.Investors & MediaJoshua Higa415-475-6370

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Global Cell & Gene Therapy Mfg. Market Could Reach $57.4B By 2028 – Contract Pharma

Posted: August 5, 2021 at 2:51 am

According to ResearchAndMarkets.com's new report, Cell And Gene Therapy Manufacturing Market Size, Share & Trends Analysis Report by Therapy Type, by Scale (R&D, Commercial), by Mode, by Workflow (Vector Production, Cell Banking), by Region, and Segment Forecasts, 2021 - 2028", the global cell and gene therapy manufacturing market size is expected to reach $57.4 billion by 2028.The market is estimated to grow at a CAGR of 20.3% from 2021 to 2028 due to an exponential rise in clinical pipeline along with a rising number of regulatory approvals for advanced therapies.With this growth are promising opportunities for contract development of cell and gene therapies. Sponsors are forming strategic alliances with contract manufacturers to help accelerate R&D of their candidate programs, increasing demand for CMOs/CDMOs services.Several new methods are being introduced to help advance cell and gene therapy manufacturing, including the potential of single-use technology in production workflows. This method is increasingly gaining attention to help speed the development process while reducing the overall cost and production timeline. Such technological advancements are expected to further drive market growth over the next few years.

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Global Cell & Gene Therapy Mfg. Market Could Reach $57.4B By 2028 - Contract Pharma

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CRISPR advancements and the ethics of gene editing – Vanguard – Psuvanguard.com

Posted: August 5, 2021 at 2:51 am

As of late June 2021, it was announced that researchers have effectively treated a genetic disorder inside humans using CRISPR therapy placed into patients bloodstreams. CRISPR is an acronym for clustered regularly interspaced short palindromic repeats, a naturally-occurring genome editing system in bacteria, repurposed by scientists.

The news has made shockwaves around the medical field as the technology has the potential to dramatically improve human health.

Genetic mutations account for more than 6000 human diseases. Curing these have been extremely difficult and expensive, and not all diseases are curable with even the most recent advancements within gene therapy.

Currently, there are two main ways gene therapy is conducted: somatic cell gene editing and germ-line gene editing.

Somatic cell gene editing involves editing matured cells within a human body. This kind of editing would not become hereditary, but does cure diseases for the afflicted individual. This is the only method to alter cells within a developed human body.

Germ-line gene editing involves editing cells such as sperm, eggs or embryos. This technique would allow any genetic modifications to be passed down to any future generations. This means that the editing process only needs to occur among a few independent cells, rather than trillions of cells found within an adult human.

Before the recent breakthrough in gene editing, CRISPR was used by removing affected cells from a patient, editing out the mutations in a lab and placing them back into the body to replicate. An example of this is curing sickle cell anemia through editing and infusing bone marrow cells.

The alternative method is to use a process known as Adeno Associated Virus, or AAV gene therapy. This method actually takes an artificially created virus with the healthy genes implanted inside, so that the virus itself infects and delivers the healthy genes to a patients cells. This is the current method for curing diseases such as Spinal Muscular Atrophy.

What makes this new advancement in CRISPR technology so novel is the method of deployment. This process injects CRISPR therapy directly into the bloodstream, so that it can make edits directly to the affected cells without invasive surgery or using AAV gene therapy.

The difficulty with direct CRISPR therapy inserted into the bloodstream has been attempting to figure out how CRISPR can correctly target and edit only the affected cells necessary. This new medical trial was successfully able to inject CRISPR into the patients bloodstream to target and edit the affected cells in their body from a genetic disorder, paving way to what could be an entirely new process to cure genetic diseases.

The ability to genetically edit human individuals is at the core of ethics within the medical industries, and, while the recent advancement is something to be celebrated, there are concerns about approaches to ethical gene editing and deploying such technology in the right hands for the right reasons.

One end of an extreme involved a scandal involving He Jiankui, a Chinese researcher who made claims of a successful birth with the first gene-edited twin girls. He has since been jailed and received international condemnation for the dangerous and unethical research experiment.

Another concern involving gene editing revolves around self-proclaimed biohackers, individuals without prior educational experience who perform genetic editing techniques either on themselves or on other living species, such as plants.

Both situations signify the most important question to consider with gene editing: regulation of use.

Dr. Bryan Cwik, a bioethics philosophy professor at Portland State University, spoke with Vanguard to discuss the ways scientists can approach gene editing in an ethical way.

The first thing to ask is, which ways work best? Cwik said. Are there advantages or disadvantages when treating this class of disease? These are ways to think about responsible gene editing.

Advancements in gene therapy are still extremely elementary, and cannot cure all types of genetic diseases as we currently understand them. Cwik explains that diseases with no deterministic link and complex etiologies should not be considered for this kind of research.

Deterministic link is a technical term used by the medical industry to explain the types of connections certain genes have with diseases. Spinal Muscular Atrophy is an example of a disease with a deterministic link; if an individual has a specific genome inside their cells, they will have the disease. These types of genetic disorders are ones suited for current advancements within gene therapy treatments.

Other diseases, however, have complex ways with which they might be constructed in the body, or the ways in which a disorder is afflicted within an individual, known as etiology. Schizophrenia is a prime example of a disease that has genetic roots, but has complicated factors; scientists dont fully understand what triggers it.

These advancements mean great news for medicine and individuals who suffer from genetic disorders such as Huntingtons disease. Others still require much more research before enough can be done. If we are to advance in gene therapies, we must be extremely cautious as to how research should be continuously conducted, and who has access to tools that constitute gene therapies.

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Taysha Gene Therapies Inc (TSHA) falls 2.00% for August 4 – Equities.com

Posted: August 5, 2021 at 2:51 am

Last Price$ Last TradeChange$ Change Percent %Open$ Prev Close$ High$ low$ 52 Week High$ 52 Week Low$ Market CapPE RatioVolumeExchange

TSHA - Market Data & News

Today, Taysha Gene Therapies Inc Incs (NASDAQ: TSHA) stock fell $0.36, accounting for a 2.00% decrease. Taysha Gene Therapies opened at $17.77 before trading between $18.11 and $17.38 throughout Wednesdays session. The activity saw Taysha Gene Therapiess market cap fall to $668,485,049 on 76,140 shares -below their 30-day average of 164,130.

Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, Taysha aims to rapidly translate our treatments from bench to bedside. Taysha Gene Therapies has combined its teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, Taysha Gene Therapies leverages its fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives.

Visit Taysha Gene Therapies Inc's profile for more information.

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To get more information on Taysha Gene Therapies Inc and to follow the company's latest updates, you can visit the company's profile page here: Taysha Gene Therapies Inc's Profile. For more news on the financial markets be sure to visit Equities News. Also, don't forget to sign-up for the Daily Fix to receive the best stories to your inbox 5 days a week.

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Guangping Gao and Dan Wang partner with ASC Therapeutics to develop novel gene therapy for maple syrup urine disease – UMass Medical School

Posted: August 5, 2021 at 2:51 am

UMass Medical School researchers Guangping Gao, PhD, and Dan Wang, PhD, are working with ASC Therapeutics, a privately held biopharmaceutical company developing in-vivo gene replacement, gene editing and allogeneic cell therapies, to bring a gene therapy for maple syrup urine disease (MSUD) to the clinic.

Our significant progress developing an AAV-based therapy for MSUD will strongly benefit from this partnership by accelerating the progression of this much-needed treatment into the clinic. ASC Therapeutics IND clearance by the FDA of a novel gene therapy for hemophilia A confirms their excellence, commitment and expertise in translating preclinical discoveries into clinical programs, said Dr. Gao, thePenelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research.

Maple syrup urine disease is a rare genetic disorder affecting degradation of the branched-chain amino acids (BCAA) leucine, isoleucine and valine and their ketoacid derivatives. MSUD is caused when a mutated form of the BCKDHA, BCKDHB or DBT gene is inherited from both parents. As a result of this mutation, the body is unable to breakdown certain parts of proteins. This leads to the build-up of toxic substances that can cause organ and brain damage. There are several forms of MSUD. The most common is the classic or infantile form. Symptoms of the classic form of MSUD start in early infancy and include poor feeding, irritability, extra sleepiness and muscle spasms. If untreated, respiratory failure may occur. The symptoms of other forms of MSUD start in adolescence or adulthood.

A protein restricted diet and supplements are the mainstay of treatment but have insufficient efficacy and afford no protection against episodic and life-threatening encephalopathic crises.

In some cases, liver transplantation may be considered and is considered a cure.

Often diagnosed based on the results of a newborn screening test, MSUD gets its name because the urine of infants with MSUD smells like maple syrup.

Under the agreement, the research group at UMMS, led by Gao and Dr. Wang, assistant professor of RNA therapeutics, will develop multiple animal models for MSUD, as well as conduct pre-clinical testing using such animal models and proprietary AAV constructs. ASC Therapeutics receives an exclusive option on the selected constructs and intellectual property resulting from this partnership. ASC Therapeutics and UMMS will jointly conduct further IND-enabling studies, regulatory and manufacturing activities to obtain IND clearance and advance this program into clinical stage.

We look forward to working with the UMMS team led by Dr. Gao and Dr. Wang, leading experts for the development and application of AAV technology. This partnership solidifies our gene therapy pipeline and will provide novel genetic therapies to patients with severe unmet medical needs worldwide, said Ruhong Jiang, PhD, CEO for ASC Therapeutics.

Related stories on UMassMed News:UMMS establishes gene therapy collaborative research agreement with PfizerH-ABC Foundation partners with UMass Medical School and Yale to study gene therapy for H-ABCGuangping Gao makes list of Nature Biotechnology Top 20 translational researchers

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Taysha Gene Therapies to Participate in Upcoming August Investor Healthcare Conferences – Yahoo Finance

Posted: August 5, 2021 at 2:51 am

BTIG Virtual Biotechnology Conference on August 9, 2021 at 11:30 am ET

2021 Wedbush Pacgrow Healthcare Virtual Conference on August 11, 2021 at 8:35 am ET

DALLAS, August 02, 2021--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivitol-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a fireside chat for the BTIG Virtual Biotechnology Conference and a panel for the 2021 Wedbush Pacgrow Healthcare Virtual Conference.

Conferences Details:

Event:

BTIG Virtual Biotechnology Conference

Date:

Monday August 9, 2021

Time:

11:30 am ET

Format:

Fireside chat

Participants:

RA Session II, President, Founder and CEO

Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

Kamran Alam, Chief Financial Officer

Event:

2021 Wedbush Pacgrow Healthcare Virtual Conference

Date:

Wednesday August 11, 2021

Time:

8:35 am ET

Format:

Panel discussion

Participants:

RA Session II, President, Founder and CEO

Webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

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View source version on businesswire.com: https://www.businesswire.com/news/home/20210802005344/en/

Contacts

Company Contact: Kimberly Lee, D.O.SVP, Corporate Communications and Investor RelationsTaysha Gene Therapiesklee@tayshagtx.com

Media Contact: Carolyn HawleyCanale Communicationscarolyn.hawley@canalecomm.com

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Taysha Gene Therapies to Participate in Upcoming August Investor Healthcare Conferences - Yahoo Finance

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