Category Archives: Genetic medicine

LEXINGTON, Mass., May 10, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced the appointment of Andrea Paul as general counsel and corporate secretary and Janice Olson as senior vice president of strategy and portfolio management. Ms. Paul and Ms. Olson are both seasoned industry experts with extensive experience in the pharmaceutical and biotechnology industries including in the gene therapy and rare disease sectors.

"It is an exciting time at LogicBio with ongoing efforts to expand the reach of our GeneRide and sAAVy platforms validated by our recent collaboration deals with Daiichi Sankyo and CANbridge Pharmaceuticals. Adding key talent across the team is essential to our success and I look forward to working with Andrea and Janice, whose broad industry experience will be instrumental as we further strengthen our leadership position in the development of next-generation genetic medicines," said Frederic Chereau, president and chief executive officer of LogicBio Therapeutics.

Andrea Paul, General Counsel and Corporate Secretary

Ms. Paul joins LogicBio Therapeutics as general counsel and corporate secretary,effective May 17, 2021, bringing several years of experience in the pharmaceutical and biotechnology industries. She is currently at Akebia Therapeutics, Inc., where she has held roles of increasing responsibility, most recently serving as vice president, legal. While at Akebia, Ms. Paul was a key strategic legal partner in the company's merger with Keryx Biopharmaceuticals, Inc. as well as in the company's financing and business development transactions. Prior to Akebia, she served as senior corporate counsel at Momenta Pharmaceuticals, Inc. Before that, she was an associate at Mintz Levin and Sullivan & Cromwell LLP. She received her JD from Harvard Law School, where she served as the managing editor of the Harvard Law Review, and her BA from Columbia University (Columbia College). Ms. Paul currently serves as the co-chair of the Securities Law Committee of the Boston Bar Association.

"I am excited to be joining the executive team at LogicBio. The Company is deeply committed to patients, and I look forward to helping the team deliver the benefits of genetic medicine to the fight against early onset childhood diseases," said Ms. Paul.

Janice Olson, Senior Vice President, Strategy and Portfolio Management

Ms. Olson joins LogicBio Therapeutics as senior vice president, strategy and portfolio management,effective June 7, 2021. Previously, she spent more than 25 years at Genzyme (now Sanofi Genzyme) holding multiple roles, most recently serving as head of global medical affairs operations, starting in January 2016. Before that, Ms. Olson served as vice president of portfolio and program management for the Genzyme R&D center. During her time at Genzyme, she developed extensive experience managing and coordinating multi-disciplinary teams for numerous R&D projects from discovery through launch. She has also served as program lead for multiple gene therapy programs developed worldwide. Ms. Olson received her MBA and BS in biology from Northeastern University.

"I am thrilled to become part of LogicBio's amazing journey at such a transformative step of its development, and I am looking forward to helping the team and the Company's new strategic partners advance programs forward for patients with significant unmet needs," said Ms. Olson.

The Company also announced the resignation of Kyle Chiang, PhD, who will be stepping down as chief operating officer, effective May 28, 2021, to pursue an opportunityat a venture capital firm focusing on early-stage sustainability and life sciences focused ventures.Dr. Chiang will consult with the Company over the next several months to ensure a smooth transition.

"I would like to thank Kyle for his commitment over the early years of the Company and wish him the best as he enters a new field. With the additions announced today, we are further strengthening our leadership team, and I am confident we are well positioned to continue to expand our platforms and pipeline in the years ahead," added Mr. Chereau.

About LogicBio Therapeutics, Inc.

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVy capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit https://www.logicbio.com/.

Media Contacts:

Adam DaleyBerry & Company Public RelationsW: 212-253-8881C: 614-580-2048[emailprotected]

Jenna UrbanBerry & Company Public RelationsW: 212-253-8881C: 203-218-9180[emailprotected]

Investor Contacts:

Matt LaneGilmartin Group617-901-7698[emailprotected]

SOURCE LogicBio Therapeutics, Inc.

https://www.logicbio.com/

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LogicBio Therapeutics Strengthens Management Team with Key Appointments - PRNewswire

A gene calledGAS2plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in Penns Perelman School of Medicine.

The researchers, whose findings arepublished online inDevelopmental Cell, discovered that the protein encoded byGAS2is crucial for maintaining the structural stiffness of support cells in the inner ear that normally help amplify incoming sound waves. They showed that inner ear support cells lacking functionalGAS2lose their amplifier abilities, causing severe hearing impairment in mice. The researchers also identified people who haveGAS2mutations and severe hearing loss.

Anatomists 150 years ago took pains to draw these support cells with the details of their unique internal structures, but its only now, with this discovery aboutGAS2, that we understand the importance of those structures for normal hearing, says study senior authorDouglas J. Epstein, professor of genetics at Penn Medicine.

Two to three of every 1,000 children in the United States are born with hearing loss in one or both ears. About half of these cases are genetic. Although hearing aids and cochlear implants often can help, these devices seldom restore hearing to normal.

One of the main focuses of the Epstein laboratory at Penn Medicine is the study of genes that control the development and function of the inner eargenes that are often implicated in congenital hearing loss. The inner ear contains a complex, snail-shaped structure, the cochlea, that amplifies the vibrations from sound waves, transduces them into nerve signals, and sends those signals toward the auditory cortex of the brain.

Read more at Penn Medicine News.

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Discovery of a new genetic cause of hearing loss illuminates how inner ear works | Penn Today - Penn Today

Carlene Knight, 54, is one of the first patients in a landmark study designed to try to restore vision in those who have a rare genetic disease that causes blindness. Josh Andersen/Oregon Health & Science University hide caption

Carlene Knight, 54, is one of the first patients in a landmark study designed to try to restore vision in those who have a rare genetic disease that causes blindness.

Carlene Knight would love to do things that most people take for granted, such as read books, drive a car, ride a bike, gaze at animals in a zoo and watch movies. She also longs to see expressions on people's faces.

"To be able to see my granddaughter especially my granddaughter's face," said Knight, 54, who lives outside Portland, Ore. "It would be huge."

Michael Kalberer yearns to be able to read a computer screen so he could get back to work as a social worker. He also hopes to one day watch his nieces and nephews play soccer instead of just listening to them, and move around in the world without help. But that's not all.

"Maybe be able to as romantically poetic as this sounds see a sunset again, see a smile on somebody's face again. It's the little things that I miss," said Kalberer, 43, who lives on Long Island in New York.

Kalberer and Knight are two of the first patients treated in a landmark study designed to try to restore vision to patients such as them, who suffer from a rare genetic disease.

The study involves the revolutionary gene-editing technique called CRISPR, which allows scientists to make precise changes in DNA. Doctors think CRISPR could help patients fighting many diseases. It's already showing promise for blood disorders such as sickle cell disease and is being tested for several forms of cancer.

But in those experiments, doctors take cells out of the body, edit them in the lab and then infuse the genetically edited cells back into patients.

The experiment Knight and Kalberer volunteered for marks the first time scientists are using CRISPR to edit DNA when it's still inside patients' bodies.

"This is the very first time that anyone's ever actually tried to do gene-editing from inside the body," said Dr. Lisa Michaels, chief medical officer at the company sponsoring the study, Editas Medicine of Cambridge, Mass. "We're actually delivering the gene-editing apparatus to the part of the body where the disease takes place in order to correct it."

NPR is the first news organization to interview any of the patients participating in the unprecedented experiment.

"It's mind-boggling to me," Knight told NPR. "It's not an everyday subject where people can say, 'Oh, I had my genes altered.' "

The first stage of the study, which treated the first patient last year, was designed primarily to assess safety. And so far, the procedure appears to be safe. By the end of the year, the researchers said they expect to share the first data on whether the procedure restored any vision for the patients.

"We're optimistic," said Dr. Eric Pierce of Mass Eye and Ear, a member of Mass General Brigham in Boston, where Kalberer was treated.

Why researchers turned to CRISPR

Knight and Kalberer were born with a form of Leber congenital amaurosis, which destroys light-sensing cells in the retina, devastating vision. A defect in a gene called CEP290 causes the version of the disease with which Knight and Kalberer were born. It affects as many as about 1,500 people in the U.S. and about 15,000 to 30,000 people worldwide, according to Pierce.

"I have no peripheral vision whatsoever," Knight said. "That means I cannot see off the side at all. I can only see straight ahead about the size of a pencil lead."

And her eyes often jump around uncontrollably which can be a symptom of LCA making even that tiny window blurry.

Kalberer can only see through a tiny peephole, too. And his limited view can suddenly blur, so it's as if he's looking through a swimming goggle that's been flooded, further obscuring his vision.

Michael Kalberer, 43, was born with a form of Leber congenital amaurosis, which destroys light-sensing cells in the retina. He says he's hopeful the procedure will help restore his vision. Mass Eye and Ear hide caption

Michael Kalberer, 43, was born with a form of Leber congenital amaurosis, which destroys light-sensing cells in the retina. He says he's hopeful the procedure will help restore his vision.

Both Kalberer and Knight are legally blind.

"I do not have visual independence. I do not have visual autonomy," Kalberer said during the first of two interviews, which occurred in September just before undergoing the procedure. "The diagnosis could have broken me. And for a while it did."

Doctors decided to try using CRISPR to edit a gene inside their bodies because retinal cells are too fragile to remove, edit in a lab and try to return to the body. Traditional gene therapy is also impossible for this condition because a healthy version of the defective gene is too big to fit into the genetically modified viruses used to ferry new genes into people's bodies.

Instead, doctors made three small incisions in Kalberer's right eye and in Knight's left eye so they could infuse billions of copies of a harmless virus. Each virus had been engineered to carry genetic instructions to manufacture the CRISPR gene-editor inside their retinas.

"It was a bit scary," Knight said. "It was exciting and scary at the same time."

The hope was the CRISPR would act like a microscopic surgeon, literally slicing out the genetic mutation in cells in their retinas. That should trigger production of a protein that could restore the function of the light-sensing cells in their retinas, preventing any further loss of vision and possibly restoring at least some of their lost vision.

"It sounds a bit like science fiction to be injecting billions of tiny little virus particles under the retina so that they can go make spelling corrections of the gene inside a patient's own retinal cells," said Pierce of Mass Eye and Ear. "But it's really happening."

Kalberer visits with Dr. Jason Comander at Mass Eye and Ear in Boston. Doctors are comparing the vision of patients before and after the procedure, and between their treated and untreated eyes, to see if the procedure is helping them see better. Mass Eye and Ear hide caption

Kalberer visits with Dr. Jason Comander at Mass Eye and Ear in Boston. Doctors are comparing the vision of patients before and after the procedure, and between their treated and untreated eyes, to see if the procedure is helping them see better.

For patients, CRISPR's possibility is a "gift"

Researchers said the approach could offer a way to treat many other diseases where it's also not possible to take cells out of the body, including other eye disorders, brain diseases such as Huntington's, and muscle diseases such as muscular dystrophy.

"It's just so exciting to be on the cutting edge of science," said Dr. Mark Pennesi, an associate professor of ophthalmology at the Oregon Health & Science University in Portland, where Knight was treated.

As a safety precaution, doctors started by using the lowest number of viruses carrying the CRISPR instructions in older patients, who had the least to lose because their vision was already extensively damaged. The researchers also minimized risk by treating only one eye in each patient.

"No one had ever done direct treatment of gene-editing inside the human body," said Michaels of Editas Medicine. "And as a consequence, there [were] a lot of concerns whether by taking these patients who have limited vision we might have had risks such as disturbing the vision and even loss of the little bit they had."

The researchers have already treated four people and are hoping to add six more adults and eventually eight children. They are also testing gradually increased doses.

Doctors are comparing the patients' vision before and after the procedure, and between their treated and untreated eyes to see if the procedure is helping them see better.

"It's hard to put into words," Kalberer said. "You hope for it. You do the best you can. But to even have the possibility it's a gift."

Knight hopes it will help her, or at least help researchers learn things that could benefit others with the condition, especially children.

"The possibilities are wonderful," she said. "It makes me hopeful."

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Experiment Uses CRISPR To Edit Genes While They Are Still Inside A Person's Body : Shots - Health News - NPR

--Conference Session: Therapeutic Applications of Extracellular Vesicles: From Diagnostics to Drug Delivery--

LOS ANGELES, May 12, 2021 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that Linda Marbn, Ph.D., Capricors CEO, will present at the American Society of Gene and Cell Therapys (ASGCT) 24th annual meeting, being held virtually May 11-14, 2021.

We are honored to have been selected as an industry leader on exosome delivery for SARS-CoV-2 vaccinations. This premier international symposium is a key medical conference in our field and brings together the leading scientists and healthcare professionals focused on gene and cell therapies, said Linda Marbn, Ph.D. chief executive officer of Capricor.

Please visit the Investor Relations section of the Company website for archived webcasts and investor materials available athttp://capricor.com/news/events/.

About ASGCT

The mission of ASGCT is to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. ASGCTs strategic vision is to be a catalyst for bringing together scientists, physicians, patient advocates, and other stakeholders to transform the practice of medicine by incorporating the use of genetic and cellular therapies to control and cure human disease.

Details for the 2021 ASGCT virtual meeting are available on the meeting website athttps://annualmeeting.asgct.org.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell- and exosome-based therapeutics and vaccines for the treatment and prevention of a broad spectrum of diseases. Capricor's lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and the cytokine storm associated with COVID-19. Capricor is also developing our exosomes platform technology as a next-generation therapeutic platform. Our current focus is on the development of exosomes loaded with nucleic acids, including mRNA, to treat or prevent a variety of diseases. For more information, visitwww.capricor.comand follow the Company onFacebook,InstagramandTwitter.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2020 as filed with the Securities and Exchange Commission on March 15, 2021. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricors exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Media Contact:Caitlin Kasunich / Raquel ConaKCSA Strategic Communicationsckasunich@kcsa.com/rcona@kcsa.com212.896.1241 / 212.896.1204

Investor Contact:Joyce AllaireLifeSci Advisors, LLCjallaire@lifesciadvisors.com617.435.6602

Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com310.358.3200

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Capricor to Present at the American Society of Gene and Cell Therapy's 24th Annual Meeting - GlobeNewswire

Newswise Researchers at The Mehandru Center for Innovation in Nephrology at Hackensack Meridian Jersey Shore University Medical Center and other authors recently had their new case report article Metabolic Acidosis, Hyperkalemia, and Renal Unresponsiveness to Aldosterone Syndrome: Response to Treatment with Low-Potassium Diet, published in the Saudi Journal of Kidney Diseases and Transplantation.1

Led by Nephrologist and Professor of Medicine Sushil K. Mehandru, M.D., the article describes three cases of Mehandru Syndrome, a potentially life-threatening electrolyte disorder that occurs in the absence of indicators or conditions that can cause high potassium levels, such as kidney disease, diabetes or high potassium diets or supplements. In each case, the high blood potassium was managed successfully with a low-potassium diet.

Mehandru Syndrome is named after renowned Nephrologist and Professor of Medicine Dr. Mehandru, whose research led to its discovery. This condition of high potassium is caused by a presumed genetic metabolic resistance to aldosterone, the hormone that helps break down potassium in the blood, said Dr. Mehandru, professor, Hackensack Meridian School of Medicine and chief, Division of Nephrology and Hypertension, Jersey Shore University Medical Center. High potassium often has no symptoms, and if left untreated, it can lead to potentially fatal disturbances in heart rhythm.

The publication describes three cases of Mehandru Syndrome in patients who had metabolic acidosis, a condition where too much acid accumulates in the body; hyperkalemia, a high level of the electrolyte potassium in the blood; normal blood pressure; normal calcium excretion by the kidneys; and normal levels of renin, and aldosterone.

These patients did not respond to diuretics to attempt to reduce the high levels of potassium in their blood or to sodium restriction. Rather, management of their care with a low-potassium diet resulted in the normalization of potassium blood levels and correction of the metabolic acidosis.

The authors ruled out the possible diagnosis of Gordon syndrome in these patients. It is an autosomal dominant disease that usually presents in children and adolescents. Classical presentation of Gordon syndrome includes high potassium, severe hypertension, metabolic acidosis, low renin and aldosterone levels in the presence of normal renal function. The treatment of Gordon syndrome has conventionally included aggressive salt restriction and low-dose diuretics. In the published article, the authors report that the three cases they described did not have severe hypertension or elevated calcium in the urine, and did not respond to salt restriction or diuretic therapy.

The authors propose that Mehandru Syndrome be considered in the differential diagnosis of patients who present in the clinical setting with unexplained high levels of potassium and metabolic acidosis. The cases we presented in this paper have unusual clinical characteristics and as yet undefined genetic abnormalities, said Dr. Mehandru. Further genetic mutations need to be defined to understand the changes at the molecular level.

Dr. Mehandru has studied kidney disease and electrolyte abnormalities for many years, and is Director of The Mehandru Center for Innovation in Nephrology at Jersey Shore University Medical Center, recently named by Hackensack Meridian Health to recognize and honor Dr. Mehandrus contributions to kidney research and improving the health of patients with kidney disease and related conditions.

Reference

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Medical Journal Publishes New Case Reports from Mehandru Center for Innovation in Nephrology at Jersey Shore University Medical Center Describing...

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COLUMBUS, Ohio--(BUSINESS WIRE)--Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced today that David Dismuke, Ph.D., Chief Technical Officer, will present at the American Society of Gene and Cell Therapys (ASGCT) 24th annual meeting, being held virtually May 10-14, 2021.

The mission of ASGCT is to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease.

We are proud to be presenting our exciting data at this prominent global gathering of scientists, researchers and industry leaders committed to transforming genetic medicine and access to gene and cell therapies for patients, said Timothy J. Miller, Ph.D, CEO, President and Co-Founder of Forge Biologics. Dr. Dismuke is an authority in gene therapy manufacturing and vector designhis work on pEMBR, our improved adenovirus ad helper plasmid for AAV production, is one example of the unique technology he has developed at Forge to provide better manufacturing solutions for our clients.

Dr. Dismukes presentation, Development of pEMBRAn Improved Adenovirus Helper Plasmid for AAV Production, is scheduled for Friday, May 14, at 12:45 p.m. ET. It is Abstract Number 224, and it will be available on demand during the conference, while the presentation will be viewable for 30 days following the conclusion of the meeting.

I am honored to be sharing data on pEMBR at such a notable gene therapy conference. pEMBR was designed with the goal of creating an adenovirus helper plasmid that is easier to manufacture and safer, while still providing high productivity of recombinant AAV vectors, said David Dismuke, Ph.D.

Details for the 2021 ASGCT virtual meeting are available on the meeting website at https://annualmeeting.asgct.org.

About Forge Biologics

Forge Biologics is a hybrid gene therapy contract manufacturing and therapeutic development company. Forges mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, The Hearth, to serve as their headquarters. The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit http://www.forgebiologics.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210511005427/en/

Media Inquiries:Dan SalvoDirector of Communications and Community DevelopmentForge Biologics Inc.media@forgebiologics.com

Business DevelopmentMagdalena TyrpienVice President and Head of Business DevelopmentForge Biologics Inc.BD@forgebiologics.com

Investor RelationsChristina PerryVice President, Finance and OperationsForge Biologics Inc.Investors@forgebiologics.com

Patient, Pediatrician, Genetic Counselors & Family InquiriesDr. Maria EscolarChief Medical OfficerForge Biologics Inc.medicalaffairs@forgebiologics.com

Source: Forge Biologics Inc.

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Forge Biologics to Present at the American Society of Gene and Cell Therapy's 24th Annual Meeting - StreetInsider.com

Huntington's disease research suffered a double whammy after Swiss pharmaceutical giant Roche Holding AG halted late-stage trials of an experimental medicine and Wave Life Sciences Ltd. abandoned two drug studies for futility.

Roche stopped dosing patients with tominersen after an independent data monitoring committee questioned whether the drug's benefits outweighed its risks.

"Obviously, it's a very sad situation when we have to stop a pivotal program in a disease like Huntington's where there's such a high unmet need and such a terrible impact on these patients and their families," Bill Anderson, head of Roche's pharmaceuticals business, said on a call with reporters following Roche's first-quarter results. "It's a major setback for the field. But we will do everything we can to learn from the work that's been done. And if there's a way forward, we would certainly pursue it."

The research stumbles, while not uncommon in the notoriously complex world of central nervous system drug development, leave only a single experimental compound in the last stage of trials usually required to seek marketing approval: pridopidine from Naarden, Netherlands-based Prilenia Therapeutics BV.

First developed at Israel's Teva Pharmaceutical Industries Ltd., where Prilenia CEO Michael Hayden was global head of R&D and chief scientific officer until 2017, pridopidine is being studied in phase 3 trials that will probably read out by the third quarter of 2022. That makes it the most advanced experimental compound in development for the hereditary disease, which affects about 70,000 people in the U.S. and Europe, the biggest potential markets for treatments. No medicines have been approved to either delay onset or slow the disease's progression.

Roche is following up with the patients on the trials of tominersen to thoroughly assess the effect of therapy. The Basel, Switzerland-based pharma company intends to continue research into Huntington's in its early-stage neurology research groups, as well as possibly via gene therapy, Anderson said.

Regulators have expressed more support for Huntington's drug developers after the influence and investment brought to bear by Roche, Europe's largest pharmaceutical company by revenue.

"They know all about Huntington's disease," Prilenia's Hayden said in an interview with S&P Global Market Intelligence. "I'm very grateful to Roche in particular, that they brought their muscle and their weight and their resources to raising awareness [of the disease] all over the world."

A number of early studies are still ongoing, including one by Roche's crosstown rival Novartis AG, which is investigating its experimental spinal muscular atrophy treatment, branaplam, as a treatment for Huntington's. The once-a-week oral therapy is in phase 1 trials after securing orphan-drug designation from the U.S. Food and Drug Administration, with the first regulatory submissions planned for 2025.

Gene therapy?

Other early-stage approaches to treating the neurodegenerative disease include one-time gene therapies in trials with companies including uniQure NV and Voyager Therapeutics Inc., which had a clinical hold lifted by the FDA on April 26.

But gene therapy may not be the approach to take, Roche's Anderson said. Huntington's disease is caused by a single mistake in a particular gene that triggers the formation of a toxic protein that kills nerve cells. Gene therapy introduces genetic material into cells to make up for abnormal genes or to produce a necessary protein. The therapy also needs to be distributed throughout the brain, and that requires intracranial surgery, injections into the brain or both.

"There are different types of genetic disorders," Anderson said. "Some of them, you have a case where you're missing a protein. And it's a relatively easier thing to add a protein when you're missing a protein with gene therapy, than if you have a genetic defect that is causing a toxic protein to form. In which case, you have to knock that down. That's a harder task for gene therapy. It doesn't mean it's not possible. It's just a trickier thing to do."

Anderson said he was not referring to any research at Spark Therapeutics, the gene therapy company that Roche acquired in 2019.

Hayden, who is also a professor of medical genetics at the University of British Columbia, hopes to show the safety and tolerability already seen in more than 1,300 people to date. The phase 3 trial of the oral compound known as a sigma-1 receptor agonist is focused on the early Huntington's patient population, with a regulatory accepted endpoint of total functional capacity.

"We've learned deeply from the past. And we think we have it at least as close to right as we can now," Hayden said. "We believe that the study is quite de-risked, because we're not trying to look for new findings."

Pridopidine, which has patent protection until 2038, is easy to produce and available in tablet form. Hayden is keen to make the therapy readily available, should it gain approval, noting that the highest prevalence of the disease in the world was discovered in a remote tribe in Venezuela. Hayden also witnessed Huntington's impact at the start of his medical career in South Africa.

"I travelled the country and recognized that Huntington's disease was alive and well in Africa. These were [many] people who were disenfranchised by apartheid and disenfranchised by this disease," Hayden said.

While not wanting to raise unrealistic hopes, both Hayden and Anderson signaled that Huntington's researchers are not undeterred by recent setbacks.

"There are lots of things that we're doing these days that we didn't think we can do a few years ago," Anderson said. "And so we remain hopeful that we will find a solution."

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Huntington's disease setback for Roche shifts the research spotlight to Prilenia - S&P Global

Overall market sentiment has been high on Akouos Inc (AKUS) stock lately. AKUS receives a Bullish rating from InvestorsObserver's Stock Sentiment Indicator.

When making investment decisions, sentiment gives a good overview of what stocks investors currently favor. Sentiment incorporates short-term technical analysis into its score and does not encompass any fundamental analysis such as profitability of the company. This means that earnings updates and other news can greatly impact overall sentiment.

Recent trends are a good indicator of current market sentiments. In its most basic form, stocks that are trending up are desirable by investors while stocks currently falling must be unattractive.

InvestorsObserver's Sentimental Indicator tracks both changes in price and volume to analyze the most recent trends. Typically an increase in volume indicates ongoing trends are getting stronger, while a decrease in volume usually signals an end to the current trend.

Available options can also represent current sentiments for a given stock. Since investors are able to bet on future trends of stocks using options, we consider the ratio of calls to puts when analyzing market sentiments .

Akouos Inc (AKUS) stock has gained 1.08% while the S&P 500 has fallen -1.5% as of 12:11 PM on Wednesday, May 12. AKUS has risen $0.14 from the previous closing price of $12.98 on volume of 96,352 shares. Over the past year the S&P 500 has risen 35.92% while AKUS has fallen -40.36%. AKUS lost -$2.77 per share in the over the last 12 months.

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Akouos Inc is a genetic medicine company focused on developing gene therapies that restore, improve, and preserve hearing. The company is involved in developing potential genetic medicines for a variety of inner ear disorders.

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Should You Buy Akouos Inc (AKUS) Stock on Wednesday? - InvestorsObserver

SUZHOU, China and PALO ALTO, California, May 10, 2021 /PRNewswire/--Gracell Biotechnologies Inc. (NASDAQ: GRCL) ("Gracell"), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced the appointment of Dr. Jenny (Yajin) Ni as its Chief Technology Officer (CTO). In her role, Dr. Ni will be responsible for strategically leading CAR-T product development, Chemistry, Manufacturing, and Control (CMC), and supply chain management activities. Dr. Ni will lead the efforts to ensure the smooth technology transfer to Gracell's strategic collaborator, Lonza, for manufacturing of FasTCAR-enabled product candidates in the U.S.

Dr. Ni brings over 25 years of experience in process and product development for gene & cell therapies and vaccines to Gracell. Prior to joining Gracell, Dr. Ni served as Head of Process Development at both Pfizer and Allogene Therapeutics, where she served in senior leadership roles in technical development and operation functions, led allogeneic CAR-T product development, and advanced multiple first-in-human allogeneic CAR-T pipeline programs through preclinical to clinical development, including five IND approvals for hematological malignancy and solid tumor indications. Prior to that, Dr. Ni also served as Director of Tech Operations at VIRxSYS Inc., where she held roles of increasing responsibility across process and analytical development, technology transfer, as well as technical support for GMP manufacturing and QC testing. While at VIRxSYS, Dr. Ni was instrumental in bringing the first-ever lentiviral vector-modified autologous T cell product for HIV infection into clinical development. Dr. Ni holds a Ph.D. in Molecular Virology from Kyoto University in Japan and an M.D. in Internal Medicine from Kunming Medical University in China.

"We are delighted that Dr. Ni has joined Gracell as CTO," said Dr. William (Wei) Cao, Founder, Chairman and Chief Executive Officer of Gracell. "Manufacturing is often a critical bottleneck in developing and commercializing CAR-T therapies. With the establishment of an internal GMP-compliant manufacturing facility in Suzhou and a process development center in Shanghai, we are well-positioned to support our extensive pipeline. A key component of our manufacturing strategy is a fully-closed system that enables us to substantially reduce costs, improve productivity, and scale-up production of our autologous FasTCAR product candidates. With Dr. Ni's extensive knowledge and experience, I am confident that Gracell will further accelerate the expansion of its global manufacturing capability and enhance our leadership position within the cell and gene therapy field."

"Gracell has successfully built a GMP-compliant manufacturing facility and an R&D center in China, as well as started establishing a state-of-the-art cGMP process in the U.S. in collaboration with Lonza. Gracell's comprehensive manufacturing strategy serves as a great foundation for its preclinical and clinical development initiatives," said Dr. Jenny (Yajin) Ni. "As CTO, I am excited to lead our efforts to ensure the smooth transfer of our pioneering FasTCAR technology to Lonza, and continuously broaden our proprietary genetic engineering and cell manufacturing capabilities and advance the next generation of CAR-T cell therapies for the treatment of cancer."

About Gracell

Gracell Biotechnologies Inc.("Gracell") is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies. Leveraging its pioneering FasTCAR and TruUCAR technology platforms, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal production quality, high therapy cost and lack of effective CAR-T therapies for solid tumors. For more information on Gracell, please visit http://www.gracellbio.com. Follow @GracellBio on LinkedIn.

Cautionary Noted Regarding Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the expected trading commencement and closing date of the offering. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including factors discussed in the section entitled "Risk Factors" in Gracell's most recent annual report on Form 20-F as well as discussions of potential risks, uncertainties, and other important factors in Gracell's subsequent filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Gracell specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Readers should not rely upon the information on this page as current or accurate after its publication date.

Media contact Marvin Tang [emailprotected]

Investor contact Gracie Tong [emailprotected]

SOURCE Gracell Biotechnologies Inc.

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Gracell Biotechnologies Appoints Seasoned Gene & Cell Therapy Executive Dr. Jenny (Yajin) Ni as Chief Technology Officer - PRNewswire

Russian Geographical Society

When you hold a job like Defense Minister of Russia, you presumably have to be bold and think outside the box to protect your country from enemy advances. And with his latest strategic ideacloning an entire army of ancient warriorsSergei Shoigu is certainly taking a big swing.

In an online session of the Russian Geographical Society last month, Shoigu, a close ally of Russian President Vladimir Putin, suggested using the DNA of 3,000-year-old Scythian warriors to potentially bring them back to life. Yes, really.

First, some background: The Scythian people, who originally came from modern-day Iran, were nomads who traveled around Eurasia between the 9th and 2nd centuries B.C., building a powerful empire that endured for several centuries before finally being phased out by competitors. Two decades ago, archaeologists uncovered the well-preserved remains of the soldiers in a kurgan, or burial mound, in the Tuva region of Siberia.

Because of Tuvas position in southern Siberia, much of it is permafrost, meaning a form of soil or turf that always remains frozen. Its here where the Scythian warrior saga grows complex, because the frozen soil preserves biological matter better than other kinds of ground. Russian defense minister Sergei Shoigu knows this better than anyone, because hes from Tuva.

Of course, we would like very much to find the organic matter and I believe you understand what would follow that, Shoigu told the Russian Geographical Society. It would be possible to make something of it, if not Dolly the Sheep. In general, it will be very interesting.

Shoigu subtly suggested going through some kind of human cloning process. But is that even possible?

To date, no one has cloned a human being. But scientists have successfully executed the therapeutic cloning of individual kinds of cells and other specific gene-editing work, and of course, there are high-profile examples of cloning pretty complex animals. Earlier this year, for example, scientists cloned an endangered U.S. species for the first time: a black-footed ferret whose donor has been dead for more than 30 years.

So, why are humans still off the menu?

Blame a technical problem with the most common form of cloning, which is called nuclear transfer. In this process, a somatic cell (like a skin or organ cell, with a specific established purpose in the body) has its nucleus carefully lifted out, and this nucleus is deposited in an oocyte, or egg cell, with its nucleus carefully removed. Its like a blank template waiting to have a new nucleus swapped in.

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From a technical perspective, cloning humans and other primates is more difficult than in other mammals, the National Institutes of Healths (NIH) National Human Genome Research Institute says on its website:

You might remember spindle proteins from your mitosis diagrams back in high school biology. And while theres a relatively easy way around this problem, its almost moot when cloning humans is considered extremely taboo in most of the world. In some places, its also explicitly illegal.

We would like very much to find the organic matter and I believe you understand what would follow that.

Curiously, the U.S. hasnt banned the gene editing of embryos. But the NIH doesnt fund research on the practice, and places like in-vitro clinics arent allowed to do any non-U.S. Food and Drug Administration-approved manipulation of embryos under any circumstances.

That example starts to illustrate why the problem is so complexbecause a lot of cutting-edge genetic medicine is walking right up to the line without crossing it. Making laws that address full human embryo cloning, then, requires a jigsaw puzzle of careful language that doesnt rule out these kinds of therapeutic cloning.

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But lets say Russia ignores all legality in favor of Shoigus big plans. In that case, scientists would have to develop a way to lift out the human nucleus without damaging the cell beyond repair.

Scientists have cloned certain monkeys, so primates are at least hypothetically still in the mix, despite the spindle proteins. But the success rate even for non-primate clones is already very lowit took Dolly the sheeps research team 277 attempts to get a viable embryo.

And what if all of that went perfectly? Well, the Scythians were powerful warriors and gifted horsemen, but scientistsor the Kremlinmust carefully monitor a cloned baby version of a deceased adult warrior for illnesses and other prosaic childhood problems. Who will raise these children? Who will be legally responsible for their wellbeing?

Shoigu may envision a future race of extremely capable fighters, but ... thats at least 20 years away, with an added coin flip on nature versus nurture. After all, the Scythian warriors didnt have plumbing, let alone smartphones. This is a whole new world.

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Russia Is Going to Try to Clone an Army of 3,000-Year-Old Scythian Warriors - Popular Mechanics