BASEL, Switzerland, Sept. 02, 2022 (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV), a biopharmaceutical company that aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy, today announced that David Marek, Chief Executive Officer of Myovant Sciences, Inc., and Uneek Mehra, Chief Financial and Business Officer, will participate in the following upcoming investor conferences:

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Myovant Sciences to Participate at Upcoming Investor Conferences

MENLO PARK, Calif. and SINGAPORE, Sept. 02, 2022 (GLOBE NEWSWIRE) -- ASLAN Pharmaceuticals (Nasdaq:ASLN), a clinical-stage, immunology focused biopharmaceutical company developing innovative treatments to transform the lives of patients, today announced Dr Carl Firth, CEO, is scheduled to give an in-person company presentation at the H.C. Wainwright 24th Annual Global Investment Conference on Monday, September 12, 2022, at 10:30 am ET. The conference will be held from September 12 to 14, 2022, virtually and in-person at the Lotte New York Palace Hotel.

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ASLAN Pharmaceuticals to Present at H.C. Wainwright 24th Annual Global Investment Conference

BOSTON and LAUSANNE, Switzerland, Sept. 02, 2022 (GLOBE NEWSWIRE) -- SOPHiA GENETICS SA (Nasdaq: SOPH), a cloud-native software company in the healthcare space, announced today Chief Executive Officer, Dr. Jurgi Camblong and Chief Financial Officer, Ross Muken will participate in a fireside chat at the 20th Annual Morgan Stanley Global Healthcare Conference on Monday, September 12, 2022, at 8:45 a.m. EDT.

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SOPHiA GENETICS to Participate in Fireside Chat at 20th Annual Morgan Stanley Global Healthcare Conference

MELBOURNE, Australia, Sept. 02, 2022 (GLOBE NEWSWIRE) -- Genetic Technologies Limited (ASX: GTG; NASDAQ: GENE, “Company”, “GTG”, “GENE”), a global leader in guideline driven genomics-based tests in health, wellness and serious disease, is pleased to report excellent commercial progress in the USA strategic operations with the following updates:

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Genetic Technologies Provides Update on US Operations and Payer Engagement

SEATTLE, Sept. 02, 2022 (GLOBE NEWSWIRE) -- Adaptive Biotechnologies Corporation (“Adaptive Biotechnologies”) (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, today announced it will be participating in the upcoming Morgan Stanley Global Healthcare Conference in New York, NY.

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Adaptive Biotechnologies to Participate in the Morgan Stanley Global Healthcare Conference

SAN DIEGO, Sept. 02, 2022 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals (Nasdaq: CRNX) today announced that Monica R. Gadelha, MD, PhD, professor of endocrinology at the Medical School of the Universidade Federal do Rio de Janeiro and a principal investigator in the Phase 2 ACROBAT program, will be presenting data from a planned two-year interim analysis from the ACROBAT Advance open label extension (OLE) study at the 35th Brazilian Congress of Endocrinology and Metabolism (CBEM) being held in São Paulo, Brazil from September 3-7, 2022.

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New Long-Term Safety and Efficacy Data for Investigational Compound Paltusotine to be Presented at the Brazilian Congress of Endocrinology and...

SAN DIEGO, Sept. 02, 2022 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (the “Company” or “Fate Therapeutics”) (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, today announced that the Company will participate in the following upcoming investor conferences:

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Fate Therapeutics to Participate at Upcoming September Investor Conferences

WESTLAKE VILLAGE, Calif., Sept. 02, 2022 (GLOBE NEWSWIRE) -- Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT), an early-stage commercial company focused on developing meaningful innovations in immuno-dermatology, today announced that Neha Krishnamohan has been appointed to the Arcutis Board of Directors and as a member of the audit committee. Following the appointment, the Board will be composed of 10 directors.

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Arcutis Announces Appointment of Neha Krishnamohan to Board of Directors

HAYWARD, Calif., Sept. 02, 2022 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “the Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its Fiscal Year ended June 30, 2022. The Company has filed its annual report on Form 10-K for the quarter ended June 30, 2022, with the U.S. Securities and Exchange Commission.

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Benitec Biopharma Releases Full Year 2022 Financial Results and Provides Operational Update

CAMBRIDGE, Mass., Sept. 02, 2022 (GLOBE NEWSWIRE) -- Immuneering Corporation (Nasdaq: IMRX), a biopharmaceutical company using translational bioinformatics to advance a pipeline of product candidates designed to benefit large populations of patients with cancer and other diseases, today announced it submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA). The IND application supports a Phase 1/2a clinical trial of IMM-1-104, an oral once daily small molecule in development for the treatment of advanced RAS mutant solid tumors. In contrast to the narrow approach of targeting specific mutations such as KRAS-G12C, IMM-1-104 is a third generation MEK inhibitor designed for broad pan-RAS activity as well as activity in other MAPK-activated tumors. Based on preclinical data to date, IMM-1-104 has demonstrated robust single-agent anti-tumor activity across a broad range of in vitro and in vivo tumor models driven by MAPK pathway activation events. This includes animal models of KRAS mutant pancreatic cancer, NRAS mutant melanoma, KRAS mutant colorectal cancer, and KRAS mutant lung cancer, regardless of the specific mutation upstream of MEK that drives activation of the MAPK pathway, and all while maintaining a well-tolerated safety profile in such models.

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Immuneering Announces Submission of IND Application to the FDA for Phase 1/2a Trial of IMM-1-104 to Treat Advanced Solid Tumors with RAS Mutations

ORION CORPORATION STOCK EXCHANGE RELEASE – OTHER INFORMATION DISCLOSED ACCORDING TO THE RULES OF THE EXCHANGE2 SEPTEMBER 2022 at 17.30 EEST

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Prosecutor appealed the district court's decision to dismiss the charges pressed against a member of Orion's Board of Directors for a suspected...

Company also retains B2i Digital to enhance engagement with the investor community

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Todos Medical Announce Kingcarlx as Brand Ambassador for the Tollovid #TolloUp Lifestyle Campaign

COPENHAGEN, Denmark, September 2, 2022 – Bavarian Nordic A/S (OMX: BAVA) announced today the dosing of the first subject in the Phase 3 clinical trial of ABNCoV2, a VLP-based, non-adjuvanted COVID-19 booster vaccine candidate.

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Bavarian Nordic Announces Initiation of a Global Phase 3 Clinical Trial of its COVID-19 Booster Vaccine Candidate

Montrouge, France, September 2, 2022

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DBV Technologies to Present at Upcoming Investor Conferences

Leuven, BELGIUM, Boston, MA, US – September 2, 2022 – CET 10:30PM – Oxurion NV (Euronext Brussels: OXUR) a biopharmaceutical company developing next generation standard of care ophthalmic therapies, with clinical stage assets in vascular retinal disorders, announced today that it has amended its mandatory convertible bonds issuance and subscription agreement announced on April 6, 2021 (“funding program”) with the Negma Group, a financial institution focused on supporting growth and capturing value through a multi-strategy approach. The proceeds will be devoted to Oxurion’s KALAHARI Phase 2, Part B clinical trial. The trial is currently underway and is evaluating THR-149, its novel therapeutic for second line therapy, against market leader aflibercept in the treatment of diabetic macular edema (DME) for the 40-50% of DME patients that get suboptimal response to standard of care anti-VEGF therapy, with top-line data expected mid next year.

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Oxurion Announces Amendment to Funding Program with Negma Group for EUR 6 Million in Funding

How do bone marrow transplants work, and what conditions do they treat?

A bone marrow transplant is actually a misnomer, as these procedures transplant stem cells, not the actual bones. Specifically, these procedures use hematopoietic stem cells (HSC), also known as blood-forming stem cells, to potentially cure an ever-expanding number of diseases.

There are three main cell types found inside a persons blood based on their function:

red blood cells: these cells carry oxygen throughout the body

platelets: these cells help form clots to stop bleeding

white blood cells: these cells lead the charge in fighting infections (also known as the immune system)

Each of these cell types, despite their different functions, shapes, sizes and lifespan, arise from the same source the hematopoietic stem cell, which constantly replenish each cell type. HSCs reside almost exclusively deep inside our bones in the center of the hard, protective shelter of calcium and other minerals. So, the marrow (soft, middle portion of our bones) can be thought of as the factory that supply each person with the blood cells needed to overcome infections, trauma, and to live a healthy, long life.

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When we perform a transplant, we are actually either using a patients own stem cells (autologous transplant) or stem cells from another human (allogeneic transplant), leading to the more appropriate name of hematopoietic stem cell transplant (HSCT). These transplants are most commonly used to treat and cure cancer.

Autologous transplants are used in the treatment of many types of solid tumors (such as brain tumors, germ cell tumors, neuroblastoma), where the tumor can only be effectively destroyed by giving very high doses of chemotherapy that also damage the patients own HSCs. Before giving a patient those high doses of chemotherapy, we collect his or her own HSCs with a process very similar to dialysis (we remove stem cells from their blood), and then freeze and store them in a specialized lab.

After the patient receives that high dose of chemotherapy, the treatment team then thaws the stem cells and infuses them back into the patient via a specialized catheter placed in his or her veins. The stem cells quickly return home and find the bone marrow space, and within 10 to 21 days, they will start making new white blood cells and platelets, followed by red blood cells.

Allogeneic transplants are performed for many types of leukemias or bone marrow failure syndromes (such as aplastic anemia or Fanconi anemia) where the patients own stem cells are broken and need to be replaced by a healthy humans stem cells. However, many other non-malignant conditions (not cancer) can be effectively cured with this procedure, as conditions that result from defects of different blood cell types (red blood cells, white blood cells or platelets) are corrected when the factory is replaced with a healthy donors stem cells.

This is an exciting time in the field of transplant, as we are now able to offer cures for many childhood diseases that historically are chronic and/or life-threatening. HSCT is now being offered to patients with sickle cell disease/thalassemia (red blood cells are defective), along with many conditions that are now called inborn errors of immunity (white blood cells are defective).

Among the more than 500 different genetic conditions that damage white blood cells include severe combined immunodeficiency (SCID), hemophagocytic lymphohistiocytosis (HLH), chronic granulomatous disease (CGD), and severe congenital neutropenia (SCN). Not only are the numbers of conditions potentially cured with HSCT rapidly growing, but the success rates and ability to prevent and treat complications of this procedure are improving exponentially as well. We are looking forward to offering these procedures to more children here at UVa Childrens.

To learn more about Dr. Roehrs and the care he provides, visit uvahealth.com/findadoctor/profile/philip-a-roehrs.

Dr. Philip Roehrs is the clinical director for pediatric stem cell transplant and cellular therapy at UVa Childrens and UVa Health.

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Ask the Expert: How do bone marrow transplants work, and what conditions do they treat? - The Daily Progress

City of Hope recently shared significant news at the 24th Annual AIDS Conference about a patient treated in 2019 whose HIV has been in remission. The man had been living with HIV for 31 years before coming to City of Hope with another grave diagnosisacute myeloid leukemia.One of the best hopes for long-term remission of acute myeloid leukemia (AML) is a stem cell transplant, and City of Hope has one of the nations leading transplant programs, having performed more than 17,000 transplants since 1976. In addition, the institution is at the forefront of using transplants to treat older adults with blood cancers, including increasing efficacy and safety in those over 60 and those with comorbidities, like the then 63-year-old City of Hope patient with HIV. The research was presented by Jana K. Dickter, M.D., City of Hope associate clinical professor in the Division of Infectious Diseases.

City of Hope hematologist Ahmed Aribi, M.D., assistant professor in the Division of Leukemia, prepared the patient for an allogeneic blood stem cell transplant with a chemotherapy-based, reduced-intensity regimen developed for treatment of older patients with blood cancers. Reduced-intensity chemotherapy makes the transplant more tolerable for older patients and reduces the potential for transplant-related complications from the procedure.

Aribi and his team worked with City of Hopes Unrelated Donor BMT Program directed by Monzr M. Al Malki, M.D. to find a donor who was a perfect match for the patient and had the rare genetic mutation, homozygous CCR5 Delta 32, which is found in just 1 to 2% of the general population.

People who have this mutation have a resistance to acquiring HIV. CCR5 is a receptor on CD4+ immune cells, and most strains of HIV use that receptor to enter and attack the immune system. But the CCR5 mutation blocks that pathway, which stops HIV from replicating.

After this successful transplant for both AML and HIV, the patient has been in remission for HIV since stopping ART in March 2021. While this outcome has happened in three other patients, the City of Hope patient was both the oldest to undergo a transplant with HIV and leukemia and go into remission for both. He had also lived with HIV the longest 31 years.

The City of Hope patient is another major advancement. It demonstrates that research and clinical care developed and led at City of Hope are changing the meaning of an HIV diagnosis for patients across the United States and the world, said John Zaia, M.D., director of City of Hopes Center for Gene Therapy, Aaron D. Miller and Edith Miller Chair for Gene Therapy and a leader in HIV research. City of Hope remains at the forefront of clinical research that changes peoples lives for the better.

When I was diagnosed with HIV in 1988, like many others, I thought it was a death sentence. I never thought I would live to see the day that I no longer have HIV. City of Hope made that possible, and I am beyond grateful. The City of Hope patient

The story above is one significant example of several important advances being made at City of Hope in the care of people with HIV. When many centers still treated patients with low-intensity, noncurative treatment approaches for HIV-related lymphoma, City of Hope challenged that paradigm by demonstrating that autologous transplantation could be used to cure patients who would otherwise die.

More recently, City of Hope is leveraging its leadership in CAR T cell therapya groundbreaking treatment currently used to rally the bodys natural defenses against cancer and exploring its potential in tandem with another advance, City of Hopes vaccine for cytomegalovirus (CMV).

In a proof-of-concept study, funded by theCalifornia Institute for Regenerative Medicine, lab models demonstrated that the combination therapy could recognize and eliminate HIV without serious toxicity to cells in the virus host. In cultured human cells, the CAR T cells killed cells tagged with the gp120 protein, and kept killing them, without significant signs of risking damage to healthy cells. In a mouse model for HIV/AIDS, high doses of the dual-action CAR T cells followed by the CMV vaccine were successful in controlling HIV, and even nestled into the bone marrow, indicating potential for treatment to keep working over the long term.

In addition to achieving breakthrough outcomes in cancer and HIV, City of Hope has been recognized as the seventh "Best Hospital" for cancer in the nation according to U.S. News & World Report's 2022-23 Best Hospitals: Specialty Ranking. This marks the first time the cancer treatment center has cracked the top 10 of the U.S. News & World Report annual rankings and the 16th consecutive year it has been distinguished as one of the nation's elite cancer hospitals. It was also rated as high performing in four cancer surgery specialties: lung, colon, prostate and ovarian cancers.

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From optimized stem cell transplants to CAR T cell therapy: Advancing options for cancer, HIV and more - City of Hope

So far, Bcells havent gotten the same attentionindeed, genetically engineered versions have never been tested in a human. Thats partly because engineering B cells is not that easy, says Xin Luo, a professor at Virginia Tech who in 2009 demonstrated how to generate B cells that have an added gene.

That early work, carried out at Caltech, explored whether the cells could be directed to make antibodies against HIV, perhaps becoming a new form of vaccination.

While that idea didnt pan out, now biotech companies like Immusoft, Be Biopharma, and Walking Fish Therapeutics want to harness the cells as molecular factories to treat serious rare diseases. These cells are powerhouses for secreting protein, so thats something they want to take advantage of, says Luo.

Immusoft licensed the Caltech technology and got an early investment from Peter Thiels biotech fund, Breakout Labs. Company founder Matthew Scholz, a software developer, boldly predicted in 2015 that a trial could start immediately. However, the technology the company terms immune-system programming didnt turn out to be as straightforward as coding a computer.

Ainsworth says Immusoft had to first spend several years working out reliable ways to add genes to B cells. Instead of using viruses or gene editing to make genetic changes, the company now employs a transposona molecule that likes to cut and paste DNA segments.

It also took time to convince the FDA to allow the trial. Thats because its known that if added DNA ends up near cancer-promoting genes, it can sometimes turn them on.

The FDA is concerned if you are doing this in a B cell, could you develop a leukemia situation? That is something that they are going to watch pretty closely, says Paul Orchard, the doctor at the University of Minnesota who will be recruiting patients and carrying out the study.

The first human test could resolve some open questions about the technology. One is whether the enhanced cells will take up long-term residence inside peoples bone marrow, where B cells typically live. In theory, the cells could survive decadeseven the entire life of the patient. Another question is whether theyll make enough of the missing enzyme to help stall MPS, which is a progressive disease.

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A new gene therapy based on antibody cells is about to be tested in humans - MIT Technology Review

North America Market Comprises Of 53.1% Market Share Due To Rising Number Of Spine-Related Disorders

Fact.MR A Market Research and Competitive Intelligence Provider: Theglobal bone grafts and substitutes marketreached a valuation ofUS$ 3.06 Bnin 2020. Moreover, sales of bone grafts and substitutes are slated to rise at a CAGR of4.9%to reachUS$ 4.44 Bnby the end of 2028.

Bone grafts and substitutes (BGS) are rapidly used common materials used mainly to replace missing bones or mend fractures. Moreover, it is commonly being used in the hip, foot, and ankle surgeries, as well as fractures and musculoskeletal injuries. Moreover, the primary goal of using bone grafts and substitutes is to aid in the healing of fractures and bone injuries, as well as to replace natural bone.

Moreover, surge in demand for synthetics and xenografts, rise in usage of bone graft substitutes in regenerative medicines, and the surge in the number of illnesses that necessitate their usage would propel the market for bone grafts and substitutes forward.

In addition to this, continuous R&D initiatives to upgrade product offerings are one of the most common trends in the market. This rise in R&D initiatives is driven by surge in need for bone graft substitutes for bone-related occurrences fractures and trauma. Researchers from across the globe are putting in efforts to find new ways to use bone grafts in regenerative medicines.

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Bone grafts manufacturers are constantly investing in the development of new products with improved bioactivity, biocompatibility, and mechanical qualities. Companies have a varied product portfolio that is technologically advanced, as well as a larger global presence. Key players in the market are putting emphasis on innovative products in various orthopedic application areas.

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Bone Growth Stimulator Market - Bone growth stimulator market was nearly worthUS$ 1.8Bn in 2020 and is anticipated to expand1.6xover the forecast period, anticipated to reach a valuation ofUS$ 3Bn by 2031. In the short-run, bone growth stimulators revenue is likely to topUS$ 1.9Bn by 2022.

Bone Marrow Processing Systems Market - A bone marrow processing system is a functionally closed, sterile system designed for automatically isolating and concentrating stem cells derived from donated bone marrow aspirate.

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Antibiotic-loaded Bone Cement Market - Infections are among the major issues encountered during various orthopedic surgeries, and antibiotic-loaded bone cement is commonly used to avoid any sorts of medical predicaments. To ensure the safety of patients undergoing orthopedic surgeries, the demand for antibiotic-loaded bone cement is increasing across the healthcare industry.

Injectable Bone Graft Substitutes Market - Growing instances of bone defects among individuals has fuelled demand for the bone grafting techniques in the healthcare industry. As the need to conduct trauma and orthopedic surgeries persist, manufacturers are developing a range of bone grafts or bone graft substitutes to stimulate insufficient or impaired bone regeneration.

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Bone Grafts And Substitutes Market Is Expected To Witness An Impressive CAGR Of 4.9% Due To Rise In Usage Of Bone Grafts And Substitutes For Healing...

By Giles Campion, EVP, head of R&D and chief medical officer, Silence Therapeutics

siRNA is a gene-silencing technology with great potential for treating a wide range of rare diseases, as I discussed in my previous article, but its promise doesnt end there. In this last article in the series, I examine siRNAs potential for treating not-so-rare and even quite common diseases.

Unlike rare diseases, which are often caused by pathological genetic mutations, common diseases may be associated with genetic variants that are not pathological and therefore do not dysregulate a biological process. For example, variants of the LPA or PCSK9 gene can increase a persons risk of cardiovascular disease by affecting cholesterol levels, but these variants do not directly cause cardiovascular disease by disrupting a fundamental biological process. This contrasts with, for example, mutations in the HBB gene that cause beta thalassemia and disrupt the mechanisms that protect the body from toxic iron buildup.

Nevertheless, the approach to treating rare and common diseases with siRNA therapies is similar: silence a gene that has little or no effect on phenotypes outside the disease, thereby maximizing safety. This is an important factor in rare diseases, which often begin early in life and require lifelong treatment. But it is equally important in common chronic diseases, such as hyperlipidemia, in which a patient has abnormally high levels of fats in the blood, where patients may live for decades before they experience any overt symptoms from their condition and are not likely to tolerate a therapy with even minor side effects that interfere with their quality of life.

At the forefront of common conditions being targeted by gene silencing is elevated lipoprotein (a), or Lp(a), a cholesterol-rich particle closely related to the well-known cardiovascular risk factor LDL. High levels of Lp(a) are associated with high risk of cardiovascular events, such as heart attacks and strokes; low levels of Lp(a) are associated with a low risk of these events.

Unlike other types of cholesterol-carrying particles, Lp(a) levels are not significantly modifiable by lifestyle factors; levels are genetically determined by the variant of the LPA gene, which encodes apolipoprotein (a) a major protein component of Lp(a) that a person has. Because these variants are not pathological mutations, the person may not experience disease symptoms for years and may even be unaware of their elevated Lp(a) levels. Yet the condition is common: One in five people have high levels of Lp(a), defined as 50 mg/dl or 120 nmol/L. Other cholesterol-reducing medicines, such as statins, have no effect on Lp(a) and can even increase levels; currently there are no approved Lp(a)-reducing therapies.

However, assessments of human genetic databases, such as the UK Biobank, have revealed that cardiovascular risk is the only phenotype associated with Lp(a) levels. Some individuals have zero levels of Lp(a), and the only known phenotype in them is a much-reduced incidence of cardiovascular events. This indicates that silencing LPA with a properly designed siRNA therapy, such as Silences clinical-stage asset SLN360, could reduce the risk of cardiovascular disease in people with elevated Lp(a) while minimizing the risk of any unwanted or unexpected side effects.

The PCSK9 gene is another example of an siRNA target for the common condition of hyperlipidemia. The PCSK9 protein negatively regulates the cellular uptake of low-density lipoprotein-cholesterol (LDL-C) in the bloodstream by reducing the number of LDL receptors on the surface of cells. This means that high levels of PCSK9 decrease cellular uptake of LDL-C, leaving more of it in circulation.

High LDL-C levels in blood are associated with coronary artery disease (CAD). While not entirely determined by genetics, as Lp(a) levels are, some variants of the PCSK9 gene are associated with low levels of LDL-C and a reduced incidence of cardiovascular disease. Similar to the LPA gene, this suggests that silencing PCSK9 with an siRNA could reduce LDL-C levels in the blood to treat hyperlipidemia and reduce the risk of CAD. Indeed, the siRNA therapy inclisiran, which silences PCSK9, was approved by the European Union in December 2020 and in the United States in December 2021 for use in people with atherosclerotic cardiovascular disease (ASCVD), ASCVD risk equivalents, and heterozygous familial hypercholesterolemia (HeFH), in conjunction with lifestyle changes and other cholesterol-lowering medicines.

An important feature of siRNA therapies in the treatment of common chronic conditions such as elevated Lp(a) and elevated LDL-C is that they have long-lasting effects, and thus they require less frequent dosing than statins and other small molecule drugs, which must be taken daily. This in turn should increase patients compliance with the therapeutic regimen and thereby improve outcomes. In fact, a 2018 retrospective study found that hyperlipidemia patients who were prescribed the right intensity (level) of statin treatment and complied 100% with their therapy had a 40% lower risk of cardiovascular events than patients who received low-intensity statin treatment and had 5% compliance.1The study concluded that an optimal therapy could reduce the risk of cardiovascular events by 30% in three years.

Though published before any siRNA therapy was approved for hyperlipidemia, the studys implications are clear: Therapeutic intensity and patient compliance are important factors in saving peoples lives. With siRNA therapies, the intensity is known, and the compliance issues are likely to be less of an issue compared with oral drugs. This is just one aspect of siRNA that makes it as well-suited for treating common diseases as rare diseases.

siRNA also has the potential to improve outcomes in hematopoietic stem cell transplantation (HSCT). Though not a disease per se, HCST is a procedure commonly used to treat a range of blood cancers and, with increasing frequency, certain autoimmune disorders.

HCST involves ablating the existing bone marrow to make way for a healthy stem cell graft to repopulate the marrow. This ablation shifts an enormous load of dead iron-laden blood cells into the circulation. Retrospective studies suggest this acute release of toxic iron from ablated cells can adversely affect the survival of the stem cell graft and increase the risk of potentially lethal infections in HSCT patients.

As in the rare disease examples I mentioned previously, silencing TMPRSS6 with an siRNA could increase hepcidin to reduce iron levels in HSCT patients, potentially improving their survival and engraftment outcomes.

I am passionate about RNA technology and the benefits that targeted, precision siRNA medicines can bring to patients with rare diseases and not-so-rare diseases who need new therapeutic options. As both a physician and drug developer, I find it rewarding and exciting to witness this technology finally coming into its own, with the promise of delivering even greater benefits in the coming years.

Reference

About The Author:

Giles Campion, MD, joined Silence Therapeutics as head of R&D and chief medical officer in 2019 and was appointed as an executive director in 2020. He is an expert in translational medicine and an experienced biotech and pharmaceutical professional across many therapeutic areas, most recently in orphan neuromuscular disorders. He has held senior global R&D roles in several large pharma, diagnostics, and biotech companies, including as group vice president of the neuromuscular franchise at BioMarin Pharmaceutical Inc., and chief medical officer and senior vice president of R&D at Prosensa. He is also a co-founder of PepGen Ltd. He earned his bachelors and doctorate degrees in medicine from the University of Bristol and is listed on the General Medical Council (UK) Specialist Register (Rheumatology).

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The Promise Of Gene Silencing To Treat Not-So-Rare Diseases - BioProcess Online