Gene and cell-based treatment is promising solutions for the control and cure of some chronic and life-threatening diseases such as sickle-cell disease (SCD), haemophilia, blood cancers, and HIV. Most of the current gene therapy clinical trials on SCD and HIV are conducted in North America.The treatment is either by using someone elses cells or those of the patient. Gene therapy, also called genetic engineering, involves getting ones cells (a patient), improving them either by enhancing them to fight disease or as a replacement for the diseased cells and using them to treat the disease.

Unlike in agriculture where a lot of the genetic engineering is on seed, Dr Cissy Kityo, the executive director at Joint Clinical Research Centre (JCRC) says in medicine, the human seed (ova or sperm) or the embryo is not touched.Its not about engineering custom humans as this has no current ethical basis. Therefore, it presents a new treatment paradigm, Dr Kityo says.

Gene therapy is administered once in a lifetime. Therefore, for someone with HIV, that eliminates the burden of taking ARTs. It also has the potential to save the overall healthcare cost and increase the individuals productivity.Research is ongoing to ensure this treatment is effective, safe, and free from off-target effects and any contamination.

The processDr Francis Ssali, the deputy executive director in charge of clinical care and research at JCRC, says genetic modification involves a series of processes, the first of which is to collect specialised white blood cells called T-cells and blood-forming stem cells from the patients blood.These cells are then taken to a clean medical laboratory where they are counted, checked for viability, and purified. Thereafter, the gene to correct the disease is inserted into these cells and this is done by either using special enzymes called CRISPR or by the use of self-inactivating partial viruses called Lentiviral vectors. The lentiviral vector delivers the required gene into the cells without resulting in viral infection in the patients cells, he says.

The process of introducing the corrective gene into the patients cell is called transduction and it can take between four to seven days to perform in the laboratory. Once the cells have received this gene modification, they are checked for quality and safety before they are ready for reinfusion back into the patient.In some instances, the patient is given medical treatment to enable them to receive the gene therapy cells, he adds.However, Dr Ssali says the current approaches to gene-therapy cell manufacturing are labour intensive and take a relatively long time to prepare, and require a large clean laboratory space.

Thankfully, there are newer laboratory instruments that can automate this genetic engineering work in a single closed instrument, with efficiency, he says.Uganda has 1.4 million people living with HIV and 400,000 people living with sickle cells yet adherence to medicine is inconsistent for some.Some HIV-resistant viral variants have emerged which threaten the efficacy of the treatment programme. As such, genetic engineering will be a blessing.Globally, the first-generation cure trials for HIV were done, second-generation trials are coming up and there is hope that soon a short-term cure will be got.

Ugandan perspectiveIn Uganda, Dr Ssali says the hope is that by 2030, Uganda will have controlled HIV/Aids greatly and also contributed to finding a functional cure.Dr Kityo says JCRC hopes to start HIV gene therapy trials in Uganda in 2024.The other focus is technology transfer where these gene therapy products are produced where they are needed, more efficiently, and more cost-effectively. That is why there will be more compact systems rather than the large labs, she adds.

In Africa, Uganda ranks fifth among countries with sickle cell disease and whereas bone-marrow transplants can cure SCD, only 10 percent of the eligible patients can get a matched donor. Nonetheless, with gene therapy, this will not be an issue since the patients own cells are used.Thankfully, the current gene therapy treatment technologies for HIV are the same used in sickle cell cure research. That is why preparing to address HIV also works to tackle the sickle cell disease, Dr Kityo says.

The joint Clinical Research Centre is working towards building the research teams and creating the necessary infrastructure for this novel research and clinical care. Arthur Makara, the coordinator of Uganda Biotechnology and Biosafety Consortium, calls for several partnerships because even when JCRC creates these technologies, they need help to mass produce them for a bigger population. Gene therapy only works on an individual, not on the sperm or ovary. Therefore, Dr Kityo says even after treatment, a sickle cell patient will still have sickle cell gene but normal cells in their marrow and live a normal life.

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Gene therapy brings hope to people with sickle cell, HIV - Monitor

PARIS, Oct. 11, 2022 /PRNewswire/ -- Eligo Bioscience, a leading in vivo gene-editing company, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation for its oral drug candidate EB003, for the treatment of Shiga-toxin producing bacterial infection as it relates to the prevention of hemolytic uremic syndrome (HUS).

"Granting of Orphan Drug Designation and Rare Pediatric Disease designation for EB003 highlights the FDA's recognition of the potential of how our unique CRISPR-based modalities can be used to address devastating diseases driven by the expression of bacterial genes, such as hemolytic uremic syndrome," said Xavier Duportet, Ph.D., Chief Executive Officer of Eligo Bioscience. "We are grateful that the FDA is providing additional support for the development of therapies geared towards rare pediatric diseases, and encourages us in our mission to propose highly innovative solutions to patients in need."

Children under 5 years of age are particularly sensitive to the expression of Shiga toxins from E. coli (STEC) bacteria after their ingestion from contaminated foods. Toxin production in the gut triggers bloody diarrhea, and its translocation and accumulation in the systemic compartment can lead to HUS, a life-threatening clinical syndrome involving destruction of blood platelets, anemia and acute kidney injury. There are currently no approved therapies for this rare disease.

Strong preclinical data in multiple animal models support EB003's capacity to efficiently and precisely eliminate Shiga-toxin genes from patients' gut, leading to a rapid decrease in toxin levels and associated symptoms, and preventing their evolution towards HUS. EB003 is IND-enabled with a robust manufacturing process at the 100L-scale, full preclinical package and a clear regulatory path-to-clinic.

Incentives attached to the ODD and RPD designation support the development of EB003 and are further validations of the proprietarygene editing platform that Eligo is leveraging to builda pipeline of high-value drug candidates in immuno-inflammation and oncology.

About ODD and RPD

The FDA grants Orphan Drug Designation to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S. ODD provides Eligo Bioscience with development incentives including tax credits for clinical testing, prescription drug user fee exemptions, and seven-year marketing exclusivity in the event of regulatory approval.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases primarily affecting individuals ages 18 or younger and fewer than 200,000 individuals in the United States. If EB003 is approved for a Biologics License Application (BLA) by the FDA, Eligo Bioscience may be eligible to receive a priority review voucher (PRV), redemption of which will result in priority review for any subsequent marketing application.

About EB003

EB003 is a first-in-class microbiome gene therapy designed, built, and optimized to target STEC bacteria in the gut of infected patients, leveraging Eligo's unique expertise in synthetic biology, phage biology, genetic engineering, and bioinformatics. As opposed to antibiotics which unfortunately lead to Shiga toxin overproduction in the process of killing the STEC bacteria, Eligo's proprietary sequence-specific CRISPR technology mechanism of action leads to the disruption of the genes coding for the Shiga toxin, and therefore offers a unique approach to this unmet need.

EB003's mechanism of action relies on the in-situ delivery of a non-replicative DNA payload encoding a CRISPR-Cas nuclease guided towards stx virulence genes. In bacteria where such genes are present, the nuclease mediates a DNA double-strand break, which leads to the inactivation of the stx genes, preventing any production of Shiga toxin and also causing death of the bacteria as their DNA repair mechanisms are highly inefficient.

About Eligo

Eligo Bioscience is the world leader in microbiome in vivo gene editing therapy and is advancing a highly differentiated pipeline of precision medicines to address unmet medical needs in immuno-inflammation and oncology caused by the expression of specific deleterious bacterial genes by our microbiome.

Eligo was founded by scientists from The Rockefeller University and from MIT. Eligo was named a Technology Pioneer by the World Economic Forum and has received venture capital funding from Khosla Ventures and Seventure Partners, and non-dilutive funding from GlaxoSmithKline, the European Commission, CARB-X, and Bpifrance.

For more information about Eligo visit https://www.eligo.bio/.

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Contact: Xavier Duportet , [emailprotected]

SOURCE Eligo Bioscience

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Eligo Bioscience Receives FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) Designation for EB003 for the Prevention of Hemolytic...

The skin is a crucial part of the body and serves as a defense against external environmental elements such as exposure to sunlight, extreme heator cold, dust, and bacterial infection. Oxidative activity occurs during the metabolism of human tissues and is a natural and inevitable part of the aging process of the skin. Free radicals with one or more unpaired electrons and a reactive state are produced as a result of the oxidative process. The skin has its antioxidant defense against this oxidation process in the extracellular space, organelles, and subcellular compartments [1]. The use of donated skin from healthy homozygotic twins may help avoid these problems. Bauer published the first successful case of skin transplantation between homozygotic twins in 1927 [2]. One of the primary health problems that significantly affect many different groups of people and varies in age and intensity is burns. Despite improvements in nonsurgical and surgical burn treatments, the patient's look continues to be a public health concern. Skin transplantation is still regarded as the gold standard for surgical burn therapy. The availability of skin for grafting is one of the main challenges in burn surgery. Regarding nonsurgical treatment, a variety of skin dressings or alternatives are still an option [3].

Additionally, biologics have been used to treat kids with allergic skin conditions. Benralizumab and dupilumab are authorized for patients older than 12 years, whereas omalizumab and mepolizumab are authorized for youngsters as old as six years. Reslizumab is only permitted for patients older than 18 years. In eligible people, these identicalantibodies may be introduced if asthma or reactive skin conditions are not effectively controlled [4]. The expression of genes capable of immunoregulatory function may lessen allograft rejection. Recent research suggests that viral interleukin (IL)-10 is one of the most effective ways to prevent rejection since it can lower the immune response during allotransplantation[5].

Tissue donation is protected by the Medical (Therapy, Educational, and Research) Act in Singapore. Reviewing the demographic and psychosocial characteristics that may generate hesitancy or unwillingness among healthcare providers is the goal of this study. A questionnaire-based survey with 18 items was carried out at the National Heart Centre of Singapore and the Singapore General Hospital. A total of 521 people took part in the survey. There were descriptive statistics run for the participant's demographics, the motivating elements behind tissue donation, motivating factors for discussing tissue donation, and causes for doubt or reluctance to donate tissue to a close relative. Fisher's exact testand Pearson's chi-square testwere used to analyze any connections that may exist among various factors and the support for tissue donation [6].

The disease known as bacteremia, or the infection of bacteria in the blood, has a high mortality rate. High rates of morbidity are linked to it. The patient's age, underlying health, and aggressiveness of the infective organism all influence the prognosis. Transfusion-transmitted infections are a rare cause of bacteremia, notwithstanding how challenging it can be to pinpoint the origin of the condition. Between one per 100,000 and one per 1,000,000 pack red blood cells or between one per 900,000 and one per 100,000platelets are the expected incidences of bacterial spreading through donated blood. One in eight million red blood cells and one in 50,000 to 500,000 white blood cells result in fatalities. Because frozen platelets are thawed and kept at room temperature before being infused, there is a chance for any pathogens that may be present to grow before the substance is transfused, which is assumed to be the source of the greater rates of platelet transfusion. Making sure that blood used for transfusions is free of toxins is essential for further lowering infection rates. One method for accomplishing this is by meticulously preparing and washing a donor's skin at the location of the collection [7].

Across the world, skin allografts are used to temporarily replace missing or damaged skin. Skin contamination that occurs naturally might also be introduced during recovery or processing. The recipients of allografts may be at risk due to this contamination. Allografts must be cultured for bacteria and disinfected, although the specific procedures and methods are not required by standards. Twelve research publications that examined the bioburden reduction techniques of skin grafts were found in a comprehensive evaluation of the literature from three databases. The most commonly mentioned disinfection technique that demonstrated lower contamination rates was the utilization of broad-range antibiotics and antifungal medicines. It was found that using 0.1% peracetic acidor 25 kGy of mid-infraredirradiation at cooler temperatures resulted in the largest decrease in skin transplant contamination rates [8].

Skin, the uppermost organ that protects the human body, is the surface upon which different environmental signals have the most immediate impact [9]. The number, quality, and distribution of melanin pigments produced by melanocytes determine the color of human skin, eyes, and hair, as well as how well they shield the skin from harmful ultraviolet (UV) rays and oxidative stress caused by numerous environmental pollutants. Melanocyte stem cells in the region of the follicular bulge replace melanocytes, which are located in the skin's layer of the interfollicular epidermis. Skin inflammation is brought on by a variety of stressors, including eczema, microbial infection, UV light exposure, mechanical injury, and aging [10]. Skin surface lipid(SSL) composition primarily reflects sebaceous secretion in the skin regions with the highest intensity of sebum (forehead, chest, and dorsum), which also flows from those sites to regions with lower concentrations, where the participation of cellular molecules rich in linoleic and oleic acid becomes more important [11]. Surgically removed skin from individuals who underwent a body contouring procedure was combined with discarded skin from excess belt lipectomies, breast reductions, and body lifts. After applying traction to both ends of the excised section, meshing by 3:1 plates, and covering with Vaseline gauze coated in an antiseptic solution prepared for burn covering, it can be removed by a dermatome. All patients in group III received a skin allograft from a living first-degree family (father, mother, brother, or sister), as they share about 50% of their DNA [12].

The principal goal is to evaluate the results of skin care therapies, like emollients, for the primary prevention of food allergy and eczema in babies. A secondary goal is to determine whether characteristics of study populations, such as age, inherited risks, and adherence to interventions, are connected to the most beneficial or harmful treatment outcomes for both eczema and food allergies [13].

Vitamin C supports the skin's ability to scavenge free radicals and act as an infection barrier, possibly protecting against environmental oxidative stress. In phagocytic cells, such as neutrophils, an accumulation of vitamin C can encourage chemotaxis, phagocytosis, the generation of reactive oxygen species, and ultimately the death of microbes. Neutrophils eventually undergo apoptosis and are cleared by macrophages, resulting in the resolution of the inflammatory response. However, in chronic, non-healing wounds, such as those observed in diabetics, the neutrophils persist and instead undergo necrotic cell death, which can perpetuate the inflammatory response and hinder wound healing. Vitamin C's function in lymphocytes is less apparent; however, studies have indicated that it promotes B- and T-cell differentiation and proliferation, perhaps as a result of its gene-regulating properties. A lack of vitamin C lowers immunity and increases illness susceptibility [14]. The skin's distinctive form reflects the fact that its main purpose is to protect the body from the environment's irritants. The inner dermal layer, which ensures strength and suppleness, feeds the epidermis the nutrients, and also the outer epidermal layer, which is incredibly cellular and acts as a barrier, are the two layers that make up the skin. Normal skin contains high levels of vitamin C, which supports a variety of well-known and important activities, such as boosting collagen synthesis and helping the body's defense mechanisms against UV-induced photodamage. This information is occasionally used as support for introducing vitamin C to therapies; however, there is no evidence that doing so is more beneficial than just increasing dietary vitamin C intake [15].

Allograft donor selection has been affected by the worry that HIV could be transmitted through the skin of an allograft. To establish the potential presence of HIV at the period of donation, there is, however, no conclusive diagnostic test available. We examine the prevalence of HIV in human tissue, consider the potential for HIV transmission through the transplant of humanallograft skin, and talk about the validity of current HIV testing to uncover solutions to enhance skin banks' HIV donor screening procedures. The risk of HIV transmission to severely burned patients could be reduced by using the polymerase chain reactionsas a fast detection methodfor HIV, with skin biopsies in conjunction with standard regular HIV blood screening tests [16].

A total of 262 dead donor skin allograft contributions were made during the past 10 years. The response revealed a considerable improvement after the community received counseling. Most of the donors were over 70 years, and most of the recruitment was done at home. In 10 years, 165 patients received tissue allografts from 249 donors. With seven deaths out of 151 recipients who had burn injuries, the outcome was good [17]. An injury to the tissue caused by electrical, thermal,chemical, cold, or radiation stress is referred to as a "burn." The skin's ability to repair and regenerate itself is hampered by deep wounds that produce dermal damage. Skin autografting is currently the gold standard of care for burn excision, but if the patient lacks donor skin or the wound is not suitable for autografting, the use of temporary bandages or skin substitutes may be absolutely necessary to hasten wound healing, lessen discomfort, avoid infection, and minimize aberrant scarring. Among the options are xenografts, cultured epithelial cells, allografts from deceased donors, and bioartificial skin replacements [18].

In the "developed" world's burn units, "early closure" in burn wounds means removing the burned tissues and replacing them within the first "five" post-burn days with graft or their substitutes. Acceptability of this method, however, may be hampered by a general lack of education and a lack of health education among the citizens in "developing" countries. A lack of dedicated and well-trained burns surgeons might make things worse. One of the growing Gulf nations in the Middle East is the Sultanate of Oman, where in November 1997, the National Burns Center at Khoula Hospital debuted "early" surgery, which quickly became a standard technique for managing burn wounds [19]. Major burn wounds that are promptly excised heal faster, are less infectious, and have a higher chance of survival. The best way to permanently heal these wounds is with the immediate application of autograft skin. However, temporary closure using a number of treatments can assist lower evaporative loss, ward off infection, alleviate discomfort, and minimize metabolic stress when donor skin harvesting is not possible or wounds are not yet suitable for autografting. The gold for such closure is fresh cadaver allograft, although alternative materials are now available, including frozen cadaver tissue, xenografts, and a number of synthetic goods. This study examines the physiology, product categories, and applications [20].

Large burn wounds are challenging to treat and heal. To help with this procedure, several engineered skin replacements have been created. These alternatives were created with specific goals in mind, which define the situations in which they may and should be used to enhance healing or get the burn site ready for autograft closure in the end. This article analyses some of the current skin replacements in use and explores some of the justifications for their usage. According to current viewpoints, the usage of skin substitutes is still in the early stages, and it will take some time before it is evident how they should be used in therapeutic settings [21].

Each skin layer has a different width based on where in the body it is located due to differences within the thicknesses of the dermal and epidermal layers. The stratum lucidum, a second layer, is what gives the palms of the hand and the soles of the feet their thickest epidermis. Although it is thought that the upper back has the thickest dermis, histologically speaking, the upper back is regarded to just have "thin skin" since that lacks thestratum lucidum layer and has a thinner epidermis as hairless skin [22].

We provide a rare instance of an individual who underwent satisfactory allogeneic split-thickness skin graft (STSG) transplanting and had previously undergone a bone marrow stem cell transplant. Hodgkin's bone marrow transplant (BMT) had already been done on the patient because of the myelodysplasia and non-lymphoma. Human leukocyte antigen(HLA) typing performed prior to BMT allowed for the identification of the donor and recipient, who were siblings (not twins). We achieved complete donor chimerism. Scleroderma, ichthyosis-like dryness, and severe chronic graft-versus-host disease (cGvHD) were all present in the recipient. Scalp ulceration with full thickness resulted from folliculitis. An STSG was removed under local anesthesia from the donor sister's femoral area and then transplanted into the recipient's prepared scalp ulcer without any additional anesthesia [23]. We conducted an allogeneic donor skin transplant in seven adult patients following allogeneic hematopoietic stem transplant surgery for cGvHD-associated refractory skin ulcers. Serious cGvHD-related refractory skin ulcers continue to be linked with significant morbidity and mortality. While split skin grafts (SSG) were performed on four patients, a full-thickness skin transplant was performed on one patient for two tiny, refractory ankle ulcers, and one patient got in vitro extended donor keratinocyte grafts made from the original unrelated donor's hair roots. An extensive deep fascial defect of the lower leg was first filled with an autologous larger omentum-free graft in one more patient before being filled with an allogeneic SSG (Figure 1) [24].

Three skin grafting innovations led to significant improvements in the care for burn injuries. Firstly, it was discovered that the dermal layeris the most crucial component of graft in creating a new, durable, resilient surface. Secondly, it was shown that deep islands of hair follicles and sebaceous gland epithelium regrow at the donor site following the excision of a partial-thickness graft, allowing grafts to be cut thicker rather than as thin as feasible. The dermis might be transplanted without having to be as thin as feasible disrupting the areas of healing. When the grafts were thicker, it was possible to build tools for cutting bigger grafts. The split-thickness graftwas the name given to these bigger grafts, and for the first in terms of square feet, it took a long time to effectively resurface big regions instead of millimeters square [25]. Skin banking was introduced in 1994 by the Melbourne-based Donor Tissue Bank of Victoria (DTBV). It is still the only skin bank in operation in Australia, processing cadaveric skin that has been cryopreserved for use in treating burns. Since the program's creation, there has been a steady rise in the demand for transplanted skin in Australia. Several major incidents or calamities, in both Australia and overseas, required the bank to provide aid. Demand is always greater than supply, thus the DTBV had to come up with measures to enhance the availability of allograft skin on a national level since there were no other local skin banks [26]. The treatment of individuals with severe burns may benefit greatly from cadaveric allograft skin. Estimating the present popularity and levels of usage of transplant skin in the US, however, is challenging. In the American Burn Association's Directory of Burn Care Resources for North America 1991-1992, which lists 140 medical directors of US burn centers and 40 skin banks, a poll of these individuals was conducted. For skin bank and burn directors, respectively, the number of responses was 45% and 38%. At the participating burn centers, 12% of patients who were hospitalized received treatment with allograft skin. Although just 47% of skin banks could provide fresh cadaver skin, 69%of burn center directors opted to utilize fresh skin. This study, which was presented to a Tissue Bank Special Interest group at the American Burns Association annual meeting in 1993, tabulated survey results as well as a review and discussion of potential future directions of replacement andskin banking research [27].

A possible substitute for human cadaveric allografts (HCA)in the treatment of severely burned patients is pig xenografts that have undergone genetic engineering. However, if preservation and lengthy storage, without cellular viability loss, were possible, their therapeutic utility would be greatly increased. This study's goal was to determine the direct effects of cryopreservation and storage time on vital in vivo and in vitro characteristics that are required for an effective, perhaps equal replacement for HCA. In this study, viable porcine skin grafts that had been constantly frozen for more than seven years were contrasted with similarly prepared skin grafts that had been kept frozen for only 15 minutes [28]. When freshly collected allogeneic skin grafts are not available, it is thought that frozen humanallogeneic skin grafts are a viable substitute. However, there is little functional and histological knowledge on how cryopreservation affects allogeneic skin transplants, particularly those that overcome mismatched histocompatibility barriers. To compare fresh and frozen skin grafts across major and minor histocompatibility barriers, we used a small-scale pig model. Our findings are relevant to the existing clinical procedures requiring allogeneic grafting and they may enable future, transient wound treatments using frozen xenografts made of genetically engineered pig skin since porcine skin and human skin share several physical and immunological characteristics [29].

Peeling Skin Syndrome

The two types of peeling skin syndrome (PSS), i.e., acral PSS and generalized PSS, are uncommon autosomal recessive cutaneous genodermatoses. The general form now includes type A non-inflammatory, type B inflammatory, and type C. A single missense mutation in CHST8, the gene that codes for Golgi transmembrane N-acetylgalactosamine 4-O-sulphotransferase, results in PSS type A. As seen in our example, this mutation leads to the intracellular breakage of corneocytes, which results in asymptomatic skin peeling. Congenital ichthyosis or erythematous patches that migrate and have a peeling border are to blame for the clinical similarity between PSS type B and Netherton syndrome[30].

Chromhidrosis

Yonge described chromhidrosis for the first time in 1709. It is an uncommon disorder characterized by the discharge of colored sweat. There are three subtypes of chromhidrosis: apocrine, eccrine, and pseudochromhidrosis [31].

Necrobiosis Lipoidica

Necrobiosis lipoidica is a granulomaillness that frequently affects the lower limbs and manifests as indolent atrophic plaques. Several case studies detail various therapy options with varying degrees of effectiveness and propose potential correlations. Squamous cell carcinoma growth and ulceration are significant side effects. Despite therapy, the disease's course is frequently indolent and recurring [32].

Morgellons Disease

It is a stressful and debilitating illness to have Morgellons disease. Multiple cutaneous wounds that are not healing are a frequent presentation for patients. Patients frequently give samples to the doctor and blame the problem on protruding fibers or other things. The initial theories for the origin of this disorder ranged widely and were hotly contested, from infectious to mental [33].

Erythropoietic Protoporphyria

The final enzyme in the heme biosynthetic pathways and the cause of erythropoietic protoporphyria is ferrochelatase partial deficiency. After the first exposure to sunlight in early infancy or youth, photosensitivity develops inerythropoietic protoporphyria. There have been reports of erythropoietic protoporphyria all around the world; however, its epidemiology varies by locale. After age 10, it was discovered that 20% of the Japanese patients had erythropoietic protoporphyria symptoms [34].

Eruptive Xanthomas

Localized lipid deposits known as xanthomas are linked to lipid abnormalities and can be seen in the skin, tendons, and subcutaneous tissue. This disorder's hyperlipidemia may be brought on by a basic genetic flaw, a secondary condition, or perhaps both. Such a skin exanthem may be the initial indication of cardiovascular risk [35].

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Skin Grafting, Cryopreservation, and Diseases: A Review Article - Cureus

At around 7 p.m. on Dec. 19, 2020, three young adults and their teacher gathered for dinner at the restaurant of the swank 1880 club in Singapore. They ordered chicken and waffles and, on the side, chicken baos. History Made, proclaimed the menus, because those diners had eaten the worldsfirst portions of chicken meat manufactured from cells, rather than slain birds.

The location was unlikely, but no accident. After a California-based start-up, Eat Just, succeeded in cultivating chicken meat from cells, it chose Esco Aster, a Singapore-based synthetic biology (syn-bio) contract manufacturing company, to manufacture cultivated chicken nuggets andbreasts as well as shredded chicken. Then the Singapore Food Authority (SFA) gave Eat Justpermissiontoproduce small batches of cultured cells in Esco Asters food-safe bioreactors, and to sell the products locally once they had met its stringent food safety criteria. Thus, the SFAbecame theworlds first regulatory authorityto approve the sale of cultured chicken meat.

Unlikeother nations, Singapore is wooing syn-bio start-ups across the world to make the city their home base.In addition to cell-basedmeats, the government is catalyzing the manufacture of proteins from plants, algae, and fungi. Ithas set up aFuture Ready Food Safety Hubto help companies navigate its approvals process, and to speed up the launch of bio-engineered products.

Over two dozen syn-bio food companiessuch as Shiok Meats, which recently launchedthe worlds first lab-grown crab and shrimp meatshave set up shop in Singapore. Thus, the city-state, which has hardly any farmland or livestock, plans to scale new technologies tomeet its goal of producing30% of its food locally by 2030, and boost economic growth by turning intoone of the worlds firstand biggestcultivated meat exporters.

Singapore may be showing the way, but most countries, unaware of the potential of syn-bio, havent put the emergent industry at the top of their policy agendas. As a result, the syn-bio industrys growth may be getting stymied. For instance, several forecasts in 2020 suggested that cultivated meat was likely to grow into a$150 billion segmentby the end of this decade, and account for around 10% of the global meat market. Two years later, that seems unlikely, not because the technologys development has slowed but because governments have been slow to legislate, regulate, and foster the industry.

Its shocking because syn-bio products have several advantages over conventional ones. Theyre sustainable, using little, or no, water, land, or carbon-emitting materialsand much less that most traditional livestock. They promise to make humanhealth better, with new syn-bio therapies likely to vanquish many diseases. And novel products, such as soil-nourishing bacteria, will help boost agriculturalproduction manifold. In fact, the technology offers governments the ability todecouple economiesfrom global supply chains, andreduce their dependence on raw material imports.

Syn-bio is clearly the next growthfrontier, sodeveloping suitable policies will be critical to unlock its benefits. According to aBCG study,syn-bio technologiescould reshape industries that will account for nearly a third of global GDP by 2030 if governments develop the appropriate regulations and rules. Moreover, as Singapore has shown, creating the conditions in which syn-bio start-ups will flourish isnt solely the prerogative of large, industrialized countries.

Although eachnations starting point will differ, every government must tackle challenges on three fronts to benefit from syn-bio.

Governments must, first and foremost, invest in advancing nations and companies knowledge of synthetic biology, much of which is still uncharted territory. As theU.S. recently did, countries can orchestrate syn-bio research by announcing formal policies, creating budgets, and setting up national agencies to spearhead the process.

Policymakers should focus on gathering and synthesizing scientific and technical knowledge by funding basic research programs; creating R&D facilities; and catalyzing the creation of graduate and post-graduate education programs in universities and colleges. One key objective should be to create talent for applied areassuch as bioreactor builders and fermentation specialistsso that they develop efficient microorganisms that use second-generation feedstock, such as organic waste, rather than processed sugars. Another priority should be to create computing resources, in terms of people and processing power, because the amount of biological data available is fast outpacing countries processing capabilities.

Apart from creating national repositories of scientific knowledge that any individual or institution can access, governments must push for the development of open standards and protocols to facilitate knowledge dissemination. They must create trusted data-sharing platforms and partner with institutions such asiGEMandBioBricks, which have developed the Get & Give philosophy and established standards for syn-bio parts to ensure their interoperability. For instance, Googles DeepMind and its A.I.,Alpha Fold, in tandem with a European intergovernmental organization, recently made public the structures of nearly all the proteins known to science.

Nations that are starting out on syn-bio quests must harness international forums and open platforms to move up the learning curve. Syn-bio research is becoming global; in 2022, iGEMs well-known syn-bio competition saw46 countries participating, 50% of which were developing countriestwice as many as a decade ago.

Second, policymakers must support business scaling of syn-bio applications,stipulating design-to-cost milestones to ensure that the efforts develop applications that will make an impact. A recentBCG study, for instance, projected when different industries are likely to be affected by syn-bio technologies. Governments must monitor the maturity of these emerging technologies by tracking cost and scale tipping points, and develop funding roadmaps that will help grow them to commercial scale.

Co-ordination can maintain the design-to-cost focus from the get-go, and help overcome the hurdles in the way of the commercialization of syn-bio technologies. Dont forget, only a few microbes such asE. coliand common yeast have been produced at scale. Others, such as mammalian cells, havent reached that stageyet.

Because syn-bio technologies dont scale linearly, engineering and development will be crucial to make it possible. Governments must use multilateral forums to forge connections between local and global stakeholders, and use technical collaborations to reduce knowledge gaps.

Countries trying to catch up should nurture the capabilities to develop applications that have commercial precedents, such as bio-catalysts and bio-chemicals. They best ways of doing that are to both orchestrate cross-border joint ventures and technology transfers, and intensify research efforts at home. Governments would be wise to attract global investments in late-stage startups, so the latter can scale and wont need to be acquired by multinational giants.

In most countries, incubators and accelerators that have seed funds and innovative financing models will help translate research into commercial ventures, and plant the financial foundations of healthy syn-bio ecosystems. For instance, in 2014,Singapore piloted intellectual property valuations, which raised awareness about IPs use as collateral and helped create an effective syn-bio ecosystem in the city.

Finally, governments must balance the need to create a friendly regulatory environment for syn-bio ventures with the need to win a social license.People have deep suspicions about syn-bio applications, just as they have about organismswhose genetic makeup has been modified in a laboratory using genetic engineering or transgenic technology (GMOs).Policy-makersmust keep educating society about syn-bio technologys potential and risks, and gauge perceptions and acceptance of its applications, so they can make course corrections.

Stakeholders must be involved at every stage of the value chain, from lab to market, to ensure that consumers buy syn-bio products. Its smart to proactively discuss the intent of the new technology. For instance, DARPA quietly launchedInsect Allies, a $45 million project to test the ability of engineered virus-carrying insects to protect crops from pestilence, in 2016. After manyU.S. scientists criticizedthe projects intent, DARPA was forced todefend itselfby highlighting its benefits and describing the safeguards it had deployed.

Syn-bio ventures must ensure the equitable use of shared resources, such as water, if they are to retain the social license from stakeholders such as farmers and indigenous populations. When Amyris set up afermentation facility in Brazilrecently, for example, it sourced feedstock from local sugarcane farms that didnt contribute to deforestation; required minimal irrigation; and didnt suck up drinking water. Local regulators must ensure syn-bio firms adhere to rules and laws even as they engage with local communities to identify all their concerns.

Finally, governments must keep in mind that the same syn-bio products can be created in different ways, and so, the regulatory regimes will need to vary. For instance, startups such as Impossible Foods, Mosa Meat, and Meati all compete in the cultured meats market, but, because they use microbes, cells, and fungi, respectively, to develop products, they must be subject to different legal frameworks. That could create entry barriers if policy-makers dont streamline the regulatory landscape.

Just as the 1990s belonged to the Internet, the 2020s mark syn-bios coming of age. As the worlds knowledge and use of syn-bio technologies grow, governments have no choice but to develop policies that will allow the industry to flourish. Because the technology creates novel and sustainable offerings, policy-makers must come to grips with syn-bio if they wish to boost economic growth even as they safeguard the environment. Only policy-makers that seize this dual opportunity by enacting supportive policies will be able to build their nations competitive advantage for the Bio Age.

ReadotherFortunecolumns by Franois Candelon.

Franois Candelonisa managing director and senior partner at BCG and global director of the BCG Henderson Institute.

Maxime Courtauxis a project leader at BCG and ambassador at the BCG Henderson Institute.

Vinit Patelis a project leader at BCG and ambassador at the BCG Henderson Institute.

Some companies featured in this column are past or current clients of BCG.

See more here:
Cultured meat could help solve the climate crisis. Heres what it will take to move it from the lab to the dinner table - Fortune

The scariest time of the year is almost upon us.

By the time Election Day passes the amount of doomsday banter about how everything is in the toilet uttered by those trying to gain election or to pass or block propositions will be enough to turn the most hardcore Pollyanna into a pessimist.

Contrary to the sound bit drivel that passes as a serious conservation about politics and the state of the world today we are not about to enter the Dark Ages.

Take Americas main mode of transportation.

We are told it is driving us to a climate Armageddon, causing gridlock, sending food prices up by consuming crops that should feed people, and making life in cities dangerous and unpleasant.

Cars are evil, arent they? Our cities were much better off before the automobile, right?

Before you start getting nostalgic about the good old days consider this: The day when horses where the main source of transportation were no bed of roses. They were worse than anything you can possibly imagine today.

If you think that is a big pile of manure, youre right.

There were 100,000 horses in New York City alone at the dawn of the 20th century or one horse for every 34 humans. Horses on average produce 25 pounds of manure each day. In 1900 that meant New York City had to contend with close to 2.5 million pounds of manure.

The Currier & Ives style images of that era dont show horse manure piled along streets much like snow drifts. It doesnt show empty parcels being stacked dozens of feet high with manure.

Manure smells. Manure breeds flies. Flies spread diseases. When it rained the manure problem got even worse.

Lets not forget that horses are a big source of methane that just like the dairy cows in the San Joaquin Valley being vilified by environmental perfectionists contribute to the greenhouse gas effect that some believe will end civilization.

Statistically, you were more likely to get killed by a horse in New York City in 1900 than by an automobile in the Big Apple in 2022.

And lets not forget horse urine splashing the streets 24/7.

There are issues with gas powered vehicles. No doubt about it. But were not going to hell in a hand basket.

Everything about a modern city helps most of us live longer and healthier: Wastewater treatment systems, storm drainage, closed treated water systems, and garbage collection are things we all take for granted but they play key roles in average longevity.

People born in the United States in 1900 could expect to live an average of 46.3 years. People born in 2022 can expect to live 76.1 years.

Another biggie is safer and healthier food.

If youre an environmental perfectionist make sure you have a defibrillator handy before you continue reading: An adequate food supply that is safer and affordable as whole is thanks in a large part to genetic engineering and chemicals.

The list is endless. The things that we are being told we should loathe today would have been the envy of Americans a century ago.

So why do we bellyache so much?

The answer will flood the airwaves non-stop for the next 32 days.

We are pounded 24/7 not by political messages about what good someone is going to do but why their opponents are evil reincarnated.

And if the ad takes an aim at the incumbent, you are left believing we are teetering on the edge of widespread collapse of everything from our financial system, cities, safety net, schools, and military to our quality of life.

If youre the incumbent youre casting your opposition as a power crazed lunatic who makes Mr. Scrooge at the onset of A Christmas Carol look like Mother Teresa in comparison.

None of this implies that we dont have problems.

We do.

But from the perspective of the last 2,000 plus years of civilization not even a hiccup in the passage of time on a planet that is 4.543 billion years old it clearly isnt that bad.

Speaking of planets, exactly what corner of the world can 329.5 million of us or even the residents of the Westside flee to where it is nirvana?

Mexico, where they just had 18 people slaughtered at a city hall?

Europe, where energy prices make it seem like theyre giving electricity, natural gas and gasoline away in the United States?

Africa, where genocide and famine routinely plague the continent?

China, where the government would have interceded long ago to re-educate social media posters in local and national elections and would have pulled the plug on anything they viewed as decadent on YouTube, Facebook, Instagram, or TikTok?

Even searching out corners of the United States to escape California because people are fed up with the Golden State can backfire spectacularly

The rush to Idaho, Texas, and other places has driven housing prices upward faster than in California and has amplified problems to the point escape states from California and its 39.7 million residents are causing long-time residents to bemoan their world is going to hell.

Years ago, several people escaped the Central Valley to move to Montana citing the then once-a-week bust of meth labs as one of the prime reasons. Today, Montana is more of a hotbed of meth production than California.

The dirty little secret from all of this is that we make panic decisions spending money and resources on cures for our ills that arent either needed or are remedies that are excessive given how overstated our problems are in reality.

Weve never had it so good.

And thats not manure.

Continued here:
Ignore scary messages: We've never had it so good & that's not manure - West Side Index & Gustine Press-Standard

DUBLIN--(BUSINESS WIRE)--The "Phosphoramidite Market - A Global and Country Level Analysis: Focus on Type, End User, and Region - Analysis and Forecast, 2022-2032" report has been added to ResearchAndMarkets.com's offering.

The global phosphoramidite market is projected to reach $2,062.9 million by 2032 from $900.3 million in 2021, at a CAGR of 7.78% during the forecast period 2022-2032. The market growth can be ascribed to the rising demand for oligonucleotides, growing synthetic biology market, increasing partnerships and collaborations, continuous investment for research and development activities by private and public firms, and growing prevalence of several diseases creating an urgent need for novel therapeutic treatments.

Market Lifecycle Stage

The global phosphoramidite market is in the rapidly evolving and dynamic stage, which opens ample opportunities for life sciences companies. Also, companies that are already in the development phase for phosphoramidite are trying to bring diverse manufacturing options to the market to improve the quality of oligonucleotides. The companies operating in the global phosphoramidite market are now focusing more on high-quality and customized options. Also, major players such as Merck KGaA, Maravai LifeSciences Holdings, Inc, LGC Science Group Holdings Limited, and Thermo Fisher Scientific Inc. are investing heavily in research and development for their respective phosphoramidite products.

Impact

Before the discovery of phosphoramidites in 1981, deoxyribonucleic acid (DNA) synthesis techniques were restricted in the quality of DNA produced. Inefficient reactions generated truncated oligonucleotide sequences. Additionally, intermediates utilized in the synthesis process were highly unstable, predisposing the growing oligonucleotide sequence to damage. Together, several characteristics of phosphoramidites have enabled the chemistry to work well for a long time.

Phosphoramidite chemistry is of fundamental importance for producing synthetic oligonucleotides that can be further utilized in a range of different areas, for instance, as primers for a polymerase chain reaction, oligonucleotide-based therapies, and genetic engineering. Millions of synthetic oligonucleotides are employed in research laboratories, pharmaceutical industries, and hospitals.

The phosphoramidite chemistry is unlikely to be replaced by emerging technologies, as the demand for primers and short-length DNA sequences is burgeoning within pharmaceuticals, synthetic biology, and molecular diagnostic industries. Essentially, synthetic DNA is applied for the discovery and engineering of biological pathways, making DNA synthesis a breakthrough of the last century. Thus, the market is anticipated to grow significantly during the forecast period 2022-2032.

Market Segmentation

Segmentation 1: by Type

Segmentation 2: by End User

Segmentation 3: by Region

How can this report add value to an organization?

Innovation Strategy: The global phosphoramidite report can help the reader gain a holistic view of the current and future global phosphoramidite market, including the competitive landscape, market size, and factors impacting the industry.

Growth/Marketing Strategy: The global phosphoramidite market has seen major development by key players operating in the market, such as acquisitions, business expansion and funding, and other developments. The favored strategy for the companies has been business expansion activities along with acquisitions to strengthen their position in the market.

Competitive Strategy: Key players in the global phosphoramidite market have been analyzed and profiled in the study. Moreover, a detailed competitive benchmarking of players operating in the global phosphoramidite market has been done to help the reader understand how players stack against each other, presenting a clear market landscape. Additionally, comprehensive competitive strategies such as partnerships, agreements, and collaborations will aid the reader in understanding the untapped revenue pockets in the market.

Key Market Players

Market Dynamics

Market Drivers

Market Restraints

Market Opportunities

Key Topics Covered:

1 Market

2 Market Scope

3 Research Methodology

4 Market Overview

5 Industry Insights

6 Market Dynamics

7 Global Phosphoramidite Market, by Type, $Million, 2021-2032

8 Global Phosphoramidite Market, by End Users, $Million, 2021-2032

9 Global Phosphoramidite Market, by Region, $Million, 2021-2032

10 Competitive Insights

11 Market - Competitive Benchmarking & Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/ylrmt2

Read more:
Global Phosphoramidite Market Report 2022: Increasing Synthetic Nucleotide Applications in Therapeutics Drives Growth - ResearchAndMarkets.com -...