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All-female breast health team uplifts patients, one another - Huntington Herald Dispatch

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The Girls Lifting Organization has grown quickly since becoming an official on-campus organization. Credit: Logo created by Elizabeth Thompson

At a Big Ten school like Ohio State, the opportunities for exercise and gym attendance seem endless, but a disproportionate number of female students attend these facilities to work out. The Girls Lifting Organization hopes to change that.

Within only one month of being an active and official on-campus organization, the Girls Lifting Organization has already accumulated over 250 members and made strides towards making on-campus gyms more female-populated, Linzy Malcolm, a third-year in molecular genetics, said.

The concept originated in February 2022 from Malcolm and Elizabeth Thompson, a third-year in linguistics, while reflecting on the differences in their experiences working out at home and at Ohio State, they said.

Elizabeth and I both went to the same all-girls school. So, going and working out was pretty simple, as our gyms were pretty much only females, Malcolm said. We just noticed how different going to the gym is here on campus, and we decided to make an organization to make girls feel more comfortable using the spaces here.

Co-presidents of GLO and friends since preschool, Malcolm and Thompson relate in their desire to create a safe, designated work-out space for women, Thompson said.

Thompson said women are often a minority in gyms due to societys gender roles and contrasting expectations for men and women in physical appearances.

On a macro level, I think that society places a lot of emphasis on men being strong, Thompson said. I think that women, our body ideals, are more, I dont want to say emphasized on being weak, but theres definitely a stigma around bulking up or things like that. So, I think thats just right out the gate, something that women have to overcome. But within campus, all the gyms, theyre very clearly male-dominated.

Thompson said the facilities having a majority-male makeup is not a bad thing, but it can be intimidating when there isnt a strong female presence.

With the establishment of GLO as an on-campus organization, members can receive weekly workouts and accompanying playlists both created by Malcolm and Thompson to do on their own or alongside a group once a week at the RPAC, Malcolm said.

GLO is an organization for girls on campus who are interested in fitness, but are intimidated in getting started and going to our on-campus recreation facilities, Malcolm said. Its for the type of girl who is a beginner lifter, or an experienced and advanced lifter who wants more of a community to feel safer in those spaces.

The co-presidents have many long-term goals for their organization, Thompson said, including initiating conversations about establishing a female-only workout space on-campus.

I think our general attitude towards the club is like, shoot for the moon and kind of land amongst the stars or something like that, Thompson said. Thats really cheesy, but thats kind of what were going for.

Malcolm said she is most excited to continue seeing friendships and confidence develop amongst the members.

The club doesnt have a focus on increasing your max reps or max weight, it has a focus on making sure that you feel comfortable in the skin and the space that you are in, Malcolm said.

The Girls Lifting Organization accepts members year-round. More information on joining the organization can be found on its Instagram.

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The "GLOwing" future of the Girls' Lifting Organization: Making on-campus gyms feel safer for female students - OSU - The Lantern

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ABU DHABI -

A strong presence of women is marking the 19th Abu Dhabi International Hunting and Equestrian Exhibition (ADIHEX 2022), being held from September 26 to October 2.

Organised by the Emirates Falconers Club, this year's event has recorded a significant attendance by female visitors and participants, reporters told The Arab Weekly.

ADIHEX 2022, reporters said, is actually turning into a family festival, appealing to all ages and community members.

Over the past few years, the UAE has seen an increase in demand among women, when it comes to the purchase of captive-bred falcons. Moreover, during past ADIHEX shows, there has been a significant demand among girls and women to buy licensed hunting weapons.

This comes following government efforts to ease procedures for licensing hunting weapons, in accordance with current legislation in the country.

During this year's ADIHEX, the role of women is being highlighted through multiple participants, including by the Emirates Falconers Club, the Mohammed bin Zayed Falconry and Desert Physiognomy School and the European Foundation for Falconry & Conservation, the International Council for Game and Wildlife Conservation (CIC) and the European Federation for Hunting and Conservation (FACE).

Women are also taking part in the showing of prey birds at Al Ain Zoo, as well as seminars and workshops, organised on the sidelines of the exhibition.

The Japanese pavilion is also celebrating the role of women in falconry, with the participation of expert Japanese female falconers.

When it comes to equestrian sports, ADIHEX is expected to boast a strong presence of horsewomen from the UAE and various other countries.

A seminar on the Sustainability Platform: Mechanism of Horse Registration Services at the Emirates Arabian Horse Association (EAHA) will be presented by Ms Lola al-Mansouri.

Meaniwhile, researcher Sariya al-Marzouq will give a lecture on the genetics of horses and the most important qualities of Arabian horses from an economic point of view, including the standards of stamina, performance and appearance, which are related to the features and qualities of equine beauty.

Tina Al Qubaisi, a member of the Fatima Bint Mubarak Ladies Sports Academy (FBMA) and the Dhabian Equestrian Club, will deliver a workshop on the "Connect with Nature, Connect with Animals" platform entitled "Equestrian Sports Training". She will also join in special events in the live performances arena that showcases the skills needed to train horses including their jumping.

Amna al-Jasmi, the Emirati horsewoman is another prominent participant in the live performances arena.

Being an impressive model for the Emirati young woman, especially with her pioneering role in spreading the sport among younger generations, Jasmi will also present performances of archery with the traditional bow and arrow on horseback and demonstrate some types of historical martial arts (fencing with the traditional Arab sword) at this year's exhibition.

On the Outdoor Adventure platform, Emirati athlete and mountaineer Nayla Nasser al-Balushi will share her experience with the ADIHEX visitors, along with her athlete husband, Saeed Khamis al-Maamari. They are the first Emirati couple to climb Mount Everest, the world's highest mountain.

Meanwhile Dr Fatima Sajwani will deliver an important workshop entitled Motivating Arab Women to Practice Exciting Outdoor Sports Activities.

The Abu Dhabi Hunting and Equestrian Exhibition will also see the participation of so-called female heritage guardians. These Emirati women will introduce the public to traditional handicrafts, such as the Sadu, Khous and other crafts that have been passed on by Emirati women from one generation to another.

In the field of arts, women are also expected to record a significant presence. From the Fujairah Fine Arts Academy (FFAA), Fatima Alamiri will deliver a unique workshop on Thread Drawing.

Fatima al-Dhanhani, another female artist, will present a workshop on Arabic calligraphy, while Sally al-Azzawi will give a presentation on the Basics of Decoration and Illumination and Ms Shurooq Azzam on Pottery Formation.

Projects run by women are also set to dominate the current show, offering various items, including traditional handicrafts, accessories, cosmetics, perfumes, incense among many other products.

The renowned Emirati artist Azza Al Qubaisi will hold a special workshop entitled "Make your own Jewellery."

Additionally, many companies seem to be keen to offer women-specific products to attract the ADIHEX female visitors. This comes as falconry activities, organised by the UAE throughout the year, have encouraged girls and young women to learn the arts, skills and science of falconry, fishing and hunting, thus paving the way for them to participate in falconry competitions, as with the equestrian and other traditional sports.

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Strong female participation marks new ADIHEX show | | AW - The Arab Weekly

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TFP Rep: Scott Dirk

Date of Sale: 09/19/2022

Location: Sale held at the ranch near LaGrange, Wyo.

Auctioneer: Wes Tiemann

Sales Manager: CK6 Consulting

Averages:

88 Open Heifer calves avg. $1,892

101 Bred Cows avg. $2,415

17 Spring pairs avg. $3,125

Comments:

Due to several years of drought and some lingering health issues, Ken Haas Angus opened the gates to their cow herd in the form of a cow herd reduction sale. Ken and Heather along with daughter Kendall offered a top set of females from the heart of the herd for this sale. There was over 45 years of top genetics and breeding in this sale offering calving ease, feed efficiency and excellent carcass merit.

Top selling female was lot 180, KCH Eileen 263, Feb. 2022 heifer calf sired by GB Fireball 672 selling to Express Ranches, Yukon, OK for $7,500.

Lot 179, KCH Erica 2111, Jan. 2022 heifer calf sired by GB Fireball 672 sold to Express Ranches, Yukon, OK for $5,000.

Lot 204, KCH Mandy 295, Feb. 2022 heifer calf sired by EXAR Monumental 6056B to Sydenstricker Genetics, Mexico, MO for $4,000.

There were no extreme tops to the sale, but the cattle sold steady all day long with several volume buyers adding these great genetics to their herds.

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Ken Haas Angus Cow Herd Reduction Sale - The Fence Post

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Iron status and the risk of sepsis and severe COVID-19: a two-sample Mendelian randomization study | Scientific Reports - Nature.com

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Animals

Large white domestic crossbred pigs (Sus scrofa) were used in this study. An atlas focused on pig cardiovascular development was previously published using these same animals6. All animal work was humanely conducted under an approved University of Missouri IACUC protocol and according to ARRIVE guidelines. All wildtype animals studied, including both fetuses and newborns, were generated by Landrace Large White cross parent gilt with semen from Choice USA Genetics (Choice USA, West Des Moines, IA). All pigs used for this study were raised on an approved farm facility and then moved into a University of Missouri animal facility for sample collection. All facilities are approved for biomedical pigs by the University of Missouri Animal Care and Use Committee and followed the Guide for the Care and Use of Laboratory Animals and the program is AAALAC accredited. The Sap130 mutant pig used in this study was generated previously with RRID, NSRRC:00816.

Breeding and harvesting of fetal and newborn pigs was carried out as previously described6. Briefly, wild type gilts were bred by artificial insemination with wild type semen. Day 0 of gestation was classified as the first day of detectable estrus, and pregnant female pigs were humanely euthanized on day 20, 26, 30, 35, 42, 64, 90 or 115 of gestation (referred to as D20, D26, D30, D35, D42, D64, D90, D115). Specimens up to 35 GA are referred to as embryos as they are largely indistinguishable between mouse, pig, human, but those at 42days GA and beyond are referred to as fetuses, as at these stages they have craniofacial and limb features distinct for the pig. The stages selected for the present study are based on our earlier analysis of the temporal profile of cardiovascular development in the pig, with D20 corresponding to early heart development comprising the looped heart tube developmental stage6. At this stage, neither ventricular, atrial, nor outflow septation has occurred. As the heart is the first organ system to form, this provided a reasonable starting point for profiling development of the abdominal organs, which are initiated after formation of the heart. From our previous study, we had determined spacing at~56day intervals can maximize what can be learned regarding the developmental progression of organogenesis at these early stages. However, the exact day of collection varied by a day or two, determined by availability of staff for collection of the specimen. Beyond day 35, we collected and analyzed fetuses at increasingly larger intervals that spanned Day 42, Day 64, Day 90 and Day 115 (newborn). By Day 42, most abdominal organs are fully formed, except for the gonads whose development continues at Day 64, but is completed by day 90.

For the embryo/fetus collection, the uterus was opened on the antimesometrial side and fetuses were removed. The whole fetus from each stage was then drop-fixed in 4% paraformaldehyde at room temperature. For fetuses at D42, D64 and D90, a small opening on the side of each fetus was introduced to allow fixative to permeate the chest cavity. Newborn piglets were kept on ice until dissection of all organs. Collected organs were photographed and placed in fixative. Embryos/fetuses were fixed in 4% PFA or 10% buffered formalin for 25days. At minimum, three embryos/fetuses per stage were analyzed. D20 to D42 embryos/fetuses were necropsied by using a stereomicroscope with digital images captured using the Kontron Progres digital camera. MRI scans were conducted followed by histological reconstructions using episcopic confocal microscopy. Newborn pigs (D115) and 2-day old pigs were analyzed by gross dissections and individual organs were separated and further analyzed by MRI. All animal work was humanely conducted under an approved University of Missouri IACUC Protocol.

The SAP130 mutant piglet was generated by a het x het mating of male 243 and female 244. The male (243) contains a 7bp deletion and the female (244) contains a 4bp deletion in SAP130. Six piglets were born. The piglets were genotyped in the same manner as Gabriel et al.6. After the piglets were genotyped, it was identified that one of the founder pigs was mosaic and contained a second modified allele, a 6bp deletion with 2bp mutation. It is not clear, which founder pig contained the third allele. Only one SAP130 mutant piglet was born from this litter containing an allele with a 4bp deletion and an allele with a 7bp deletion which resulted in a SAP130 null genotype. The mutant piglet was identified by external phenotype and euthanized for analysis at term.

For embryos at D20, D26, D30, and D35, following necropsy and MRI, the whole fetus or only the abdominal section of the fetus was embedded in paraffin for episcopic confocal microscopy (ECM). Paraffin embedded samples were sectioned using a Leica SM2500 sledge microtome and serial confocal images of the block face were captured using a Leica LSI scanning confocal macroscope mounted above the sample block as previously described6. The 2D serial image stacks collected were visualized using the OsiriX Dicom viewer11 (https://www.osirix-viewer.com). These image stacks could be digitally re-sectioned in multiple imaging planes and 3D reconstructed for optimal viewing of the abdominal organs.

Prior to MRI scanning, embryos/fetuses were fixed and stained with a gadolinium (Gd)-based contrast agent to shorten the tissue T1. Briefly, after fixation embryos/fetuses were immersed in 1:200 MultiHance23 (gadobenate dimeglumine, 529mg/ml, Bracco Diagnostic, Inc. Monroe Twp, NJ) diluted with phosphate-buffered saline (PBS) at 40C for at least 48h. After staining, small embryos were secured on a tongue depressor (McKesson Medical-Surgical, Irving, TX) with Webglue surgical adhesive (n-butyl cyanoacrylate, Patterson Veterinary, Devens, MA). The embryos/fetuses were then immersed in Fomblin Y (perfluoropolyether, Sigma-Aldrich Millipore) to eliminate the susceptibility artifact at the tissue-air interface and to avoid dehydration during imaging.

MRI was carried out as previously described, with special emphases on abdominal structures, using a Bruker Biospec 7T/30 system (Bruker Biospin MRI, Billerica,MA) with a 35-mm or 72-mm quadrature coil for both transmission and reception6. 3D MRI was acquired with a fast spin echo sequence, the Rapid Acquisition with Refocusing Echoes (RARE), with the following parameters: effective echo time (TE) 24.69ms,RARE factor 8, repetition time (TR) 900ms. We used RARE also known as Fast Spin-Echo (FSE) or Turbo Spin-Echo (TSE) pulse sequence for high-resolution 3D imaging with T2-weighted contrast. It generates similar T2-weighted contrast as the Half-Fourier-Acquired Single-shot Turbo spin Echo (HASTE), a Turbo spin-echo technique that is used for sequential acquisition of high-resolution T2-weighted images. However, the strategy for fast spin echo is different. Our RARE condition with RARE factor 8 uses 8 echoes as 8 phase-encoded k-space lines to accelerate acquisition; whereas HASTE uses a single-shot technique or segmented multiple shorts to cover sufficient k-space from a single TR. HASTE although commonly used in human scanners, it is not available in the Bruker preclinical scanner used in this study. RARE provides flexible T2-weighting conditions by changing RARE factors depending on the tissue types of interest. We have tested various RARE factors, TE, and TR combinations to optimize the contrast, signal to noise ratio (SNR), and scan time used in this study.

The field of view (FOV), acquisition matrix and voxel sizes varied based on the sample size. The typical spatial resolution for D26, D30, and D35 embryos ranged from 39m to 46m, that of D42, D64 and D90 fetuses ranged from 45m to 62m. The FOV, matrix, resolution, echo time, RARE factor, and other MR parameters used for imaging at the different GAs are provided in Supplemental Spread Sheet 1. The 3D MRI imaging stacks were exported with DICOM format and could be re-oriented to any viewing angle with Horos Dicom Viewer (Horosproject.org).

Necropsies were performed as previously described on D42, D64, D90, D105, D115 and 2-day old wildtype normal pigs which showed no external malformations6. Briefly the thoracic, abdominal, and pelvic viscera were examined in situ for malformations, the heart, great vessels, and lungs were removed as a block and examined using the sequential segmental analytical method24,25. Following examination of thoracic organs, the abdominal-pelvic visceral blocks were removed as a block and dissected and examined from behind (dorsal in the pig). Because pigs are quadrupeds, structures, which in bipedal mammals are described as inferior in pigs are described as posterior or caudal, for example the inferior caval vein can be referred to as the posterior or caudal caval vein in the pig. However, to better align the pig to the bipedal mammal, we have chosen, like others, to describe the abdominal organs of pigs as in bipedal mammals26. The abdominal organs in mammals obtain their basic gross appearance before term but continue to develop after birth by increasing in size or length as well as at the cellular and biochemical levels. In addition, in the very early embryo the organs begin by cell differentiation, and they lack the expected configuration that is seen in the fetus. In this study we focus on the assessment of the basic gross appearance of the organs before term.

See the rest here:
Profiling development of abdominal organs in the pig | Scientific Reports - Nature.com

Recommendation and review posted by Bethany Smith

Maya, the worlds first cloned arctic wolf, was born in a lab in China(Picture: Twitter/China Science)

A Beijing-based genetics company has successfully cloned an Arctic wolf, in a ground-breaking move which could help preserve endangered species.

26 years after the birth of Dolly the Sheep, Sinogene Biotechnology unveiled the new wolf pup, named Maya to the world.

She had been born back in June, but the firm wanted to wait until she was 100 days old before they announced her to ensure the clone was in good health, Global Times reports.

Scientists created the pup by taking a donor cell from a wild female Arctic wolf which had been introduced from Canada and combining it with an embryo grown inside a beagle, which shares genetic ancestry with ancient wolves.

The process, called somatic cell nuclear transfer, is the same process which birthed Dolly the Sheep back in 1996.

Arctic wolves, also known as white or polar wolves, are not endangered like other wolf breeds because they live in isolation, although Singogen hopes this process can be used in future to save other species at risk of extinction.

The newly born wolf has the same genome as the original wolf, but the cloned wolf hasnt lived with other wolves, but with a dog, Zhao Jianping, the deputy general manager of Sinogene, told EuroNews.

In fact, for cloned pet dogs and cats, there is also a problem of early socialisation. The earlier the socialisation, the more beneficial for its future development, he added.

Some concern has been raised by activists over the suffering of animals required to go through surgery in order to be implanted with cloned donor cells and embryos.

Others says there is a moral imperative against playing God by creating life from artificial sources without fertilisation.

Regardless, Mayas birth is regarded as a landmark in the application of cloning technology, and Mr Jianping is confident that refining the process could be the only way to help save some species of animals from extinction.

Well continue to work in this field, he added to EuroNews.

In the next step, we may clone rare wild animals other than canines or cats and it will be more difficult.

Although Dolly had to be euthanised at six years old after she was found to have a lung tumour, Maya is currently exhibiting all the signs of a normal, healthy arctic pup.

Unfortunately Maya will likely have to spend her life in captivity due to her lack of socialisation, although she has been kept in good company by the beagle who birthed her who acts as her surrogate mother and playmate.

Mayas birth continues the life of a wild female arctic wolf also named Maya, who was introduced from Canada in 2006 and died of old age in 2021.

Another male cloned arctic wolf is expected to be delivered on Thursday.

Get in touch with our news team by emailing us at webnews@metro.co.uk.

For more stories like this, check our news page.

See original here:
World's first cloned Arctic wolf born 26 years after Dolly the sheep - Metro.co.uk

Recommendation and review posted by Bethany Smith

At Casa Cattle Company in Corinna, there are plenty of cows and calves making up a successful belted Galloway breeding program but not many bulls to be seen.

Instead, there are two cryogenic tanks in the grain room, each containing 150 vials of bull semen and 30 fertilized embryos containing genetic material from around the country.

Rather than deal with the expense and work required to maintain enough bulls to ensure the genetic diversity Ashton Caron is looking for in his herd, he has opted to instead purchase or save semen from his own prized stock to do the job.

For Caron, saving the semen means big savings and a high-tech way to weather the current economic crisis. Swapping out a bull for artificial insemination technology allows Caron to keep producing high-quality calves that can be sold at a profit while minimizing costs.

It has become very essential, Caron said. With rising grain and hay costs due to COVID, we need to be able to maximize profits and minimize expenses.

It may not be the romantic Hollywood image of the cows and bulls doing what comes naturally out on the range. But for Caron it just makes good economic sense to dip into a vial, often referred to as a straw, of semen rather than deal with an old fashioned cattle roundup when it comes time to breed a cow.

Most people who do this us included can drastically increase the value of their cattle in a short time, Caron said. I can take a bull from the other side of the world and breed it to my cow and have those genetics without having to bring them together, which would not really be feasible.

A healthy breeding bull can cost thousands even tens of thousands of dollars. Then there are the expenses of feeding, housing and keeping the animal healthy. Over the course of a year Caron said that it can run upward of $2,000 for just one animal.

A vial of semen, on the other hand, from a bull of the same quality costs Caron between $25 and $30.

Caron and his family raise Belted Gallowaycattle, a traditional Scottish breed that has adapted to living on poor upland pastures and the windswept moors of southwest Scotland. They are distinctive with their jet black front and hind ends and white middle. The coloration has earned them the nickname Oreo cows.

Selective breeding using artificial insemination is more common when it comes to dairy livestock, according to Caron. Farmers are able to selectively breed for cows that are able to produce more milk than previous generations.

It costs maybe $300 a year to keep the tanks full of liquid nitrogen, said Caron, who began saving semen and embryos eight years ago. We run around 100 cows here, and its easy to breed without a bull, so why would we have one?

Carons cattle have won numerous awards at fairs and livestock shows, something that represents years of breeding on the farm. Thanks to using purchased or saved semen and embryos, the breeding program has shown these champion results at a fraction of what it would have cost to purchase and raise the bulls needed to keep the herd going.

Currently tucked safely away in liquid nitrogen keeping them at a steady minus 300-degrees Fahrenheit in the two tanks each insured for $25,000 theres semen from 50 different bulls, Caron said.

High-quality semen means high demand and that influences the price, he said. We do have some semen worth $600 a straw, but that is from a prize bull that is now deceased, so it is a very limited quantity.

The semen can be planted directly into a female or it can be combined with the egg of a cow in a laboratory as in vitro fertilization.

Most of the breeding cows on the farm serve as surrogate mothers. But a few of those cows are of such high quality that, once they are impregnated, their embryos are harvested to use for in vitro fertilization.

This Simmental cow is a surrogate pregnant with a calf from Knockouts embryo and Loverboys semen, as marked on her ear tag. Credit: Linda Coan OKresik / BDN

Among those special cows there is one that stands above the rest with genetic material with connections to old Maine money.

I was a freshman in high school in 2017 when the Hudson Pines herd owned by David Rockerfeller on Mount Desert Island was sold, Caron said. Me and a friend bought her for $4,000 and shes the best cow on the farm.

The best part of all of this, Caron said, is you do not need a high quality cow to produce a high quality calf. All you need is the embryo from two prize parents.

There is currently not a huge market for selling his bulls semen or any embryos, but Caron said the demand is slowly growing.

In terms of the semen, we had a bull that was born and raised here and before we got rid of him in 2019 we got 300 straws of semen from him, Caron said. Now we are down to less than 50 straws and the cool thing is 90 percent of those customers came were west of Idaho [and] its cool to see our [bulls] progeny on these farms out west.

More articles from the BDN

Continue reading here:
Artificial insemination is keeping this Maine farm in business - Bangor Daily News

Recommendation and review posted by Bethany Smith

Beef + Lamb New Zealand award winners. Photo / Supplied

The winners in the inaugural Beef + Lamb New Zealand (B+LNZ) Awards were announced at a gala dinner at the Napier War Memorial Centre last night.

The awards aim to celebrate the people, innovation, technologies and management systems of New Zealand's grass-based red meat industry.

Andrew Morrison, chairman of B+LNZ reflected on the achievements of the sector over the last couple of years and its resilience in maintaining strong exports in light of Covid-19.

"Environmentally, our sheep and beef production systems are amongst the most sustainable in the world with around 24 per cent of New Zealand's native vegetation flourishing on our sheep and beef farms, and one of the world's lowest carbon footprints."

Throughout the evening, the finalists of the eight award categories were introduced and winners announced. The ceremony concluded with the presentation of the Regional Leadership Award.

Science and Research Award

Lincoln University's Dryland Pastures Research Group won the Ballance Agri-Nutrients Science and Research Award.

For the past 20 years, the Dryland Pastures Research Group has supplied the science that underpins the agronomic guidance it provides to transform sheep and beef farms on hill country throughout New Zealand.

The group's work has transformed thousands of hectares of east coast hill country from Central Otago to Gisborne; while its message has been to use legume dominance to address low nitrogen the main impediment to production in farm systems.

In selecting the winner of this category, the judges commented that the Dryland Pastures Research Group had produced "a huge and important body of work," tackling multiple soil types and farming environments with positive outcomes for productivity, resilience and environmental sustainability.

Livestock Technology Award

The Datamars Livestock Technology Award was won by software decision support-tool Farmax.

Widely used by New Zealand's pastoral farmers to help balance financial, environmental and production goals, Farmax encourages a holistic approach to farm planning.

Judges remarked that this decision support tool had evolved over 30 years and continued to innovate to drive farm profitability, productivity and sustainability.

They noted that as science-based software, Farmax generated a good return on investment and was a leader in what it provided farmers and the industry, in terms of exploring future scenarios.

Innovative Farming Award

Canterbury-based calf rearing business Maatua Hou won the Gallagher Innovative Farming Award.

Creating a viable business on a small land holding, while demonstrating an alternative calf rearing model that reduces bobby calf wastage, is at the heart of the Maatua Hou business.

Set up by four couples who saw an opportunity to think outside of the square, Maatua Hou owns a 34ha drystock block at Burnham, around 40kms outside of Christchurch.

They established what they described as "an alternative calf-rearing model" - one where the supplier cashflows calf-rearing costs and profits are shared.

Judges commented that Maatua Hou provided an innovative and scaleable solution to the bobby calf problem and they believed that, as a business model, it demonstrated an opportunity to build a dairy support industry.

They said Maatua Hou was an interesting innovation which had its strengths in the partnership between producers and finishers and the sharing of benefits and returns.

Market Leader Award

The Silver Fern Farms Market Leader Award was won by Coastal Lamb Ltd.

Richard and Suze Redmayne launched the Coastal Spring Lamb brand in 2010 as a way to better understand their lambs' end-consumer.

Having a background in commerce and marketing before going farming, Richard wanted to build a connection between his family and the consumer through their brand, initially targeting New Zealand's domestic market.

Today Coastal Lamb Ltd involves 17 family-owned supply farms throughout the country.

Critical to Coastal Lambs' success is connecting the producers with the consumers (including chefs), many of whom had never had the opportunity to meet a farmer before.

The judges described Coastal Lamb Ltd as "a true and innovative example of market leadership".

Having forged a new path to market, Coastal Lamb Ltd had demonstrated a clear understanding of the market and in-depth communication with its customers, judges said.

Emerging Achiever Award

The AgResearch Emerging Achiever Award was won by Cambridge-based Estee Browne.

Browne is the breeding programme manager for Browne Pastoral Enterprise's sheep milking unit.

She oversees the selection of genetics and replacement ewes for the company's 1400 ewe dairy unit and rears 2400-plus lambs to weaning, after which they are either finished or retained as replacements.

Judges said Browne had "broken stereotypes" by achieving in a male-dominated area of the agricultural industry.

She had created "good, workable systems" within the sheep milking industry and had clear goals while maintaining a work-life balance, they said.

People and Development Award

The Rabobank People and Development Award was won by the Agri-Women's Development Trust (AWDT).

AWDT aims to empower women to accelerate progress and change in both the primary sector and rural communities.

Over the past 11 years, the charitable trust has helped almost 5000 people with confidence, purpose, leadership and influence from the farm to the boardroom.

The judges commented on AWDT's proven track record of successfully developing people.

They said the organisation had identified a gap in the market and developed programmes targeting female partners in farming businesses.

This had helped to build confidence and resilience in the whole red meat sector, they said.

Rural Champion Award

The FMG Rural Champion Award was won by Tairwhiti-based agribusiness professional Sandra Matthews.

Matthews was instrumental in setting up Farming Women Tairwhiti (FWT) which now has a membership of over 850 primary industry women.

Matthews believed the success of FWT was partially due to the region's isolation, with no other organisation offering farming women the support, connectivity and educational opportunities they were craving.

Since stepping back from FWT last year, Matthews took on several national governance and consulting roles and gained more time to focus on her own business coaching enterprise.

Judges said Matthews' work in establishing, leading and growing Farming Women Tairwhiti was "immeasurable".

As well as setting up FWT, Matthews has been involved in establishing a number of industry initiatives and played an often-understated role in championing the industry.

Alliance Significant Contribution Award

AgResearch scientist David Stevens, based at the Invermay research institute near Mosgiel, won the Alliance Significant Contribution Award.

Stevens' 37-year career has spanned one of the more tumultuous periods in New Zealand's agricultural history.

He has seen the removal of subsidies and the rapid shift from a focus on stocking rates and wool production to meat and productivity.

The farm system's scientist, who has a background in agronomy, was initially involved in plant breeding but this changed in the early 1990s when farmers found that what they were producing was no longer fit for market.

They needed forages that would deliver in terms of animal performance - so Stevens and his colleagues began doing animal production trials alongside agronomic trials - and this morphed into farm systems work.

He said the most rewarding part of his work was having the opportunity to work one on one with farmers through a huge range of projects.

Judges said Stevens was instrumental in creating the foundation of modern sheep and beef farming systems which delivered prosperity to many farmers.

Stevens had a long involvement with the sector and had contributed to the development of a number of resources which had significantly benefited farmers throughout the country, judges said.

Regional Leadership Award

The winner of the B+LNZ Regional Leadership Award, which recognised an outstanding individual, organisation or business in the sheep, beef and dairy beef sector, went to the East Coast Rural Support Trust.

The Trust was represented by Hawke's Bay farmers Mark Barham and Jane Tylee and Wairarapa-based former farmer and Anglican priest Steven Thomson.

The winner of this award was identified by the farmer council in the award's host region.

The judges commented that these three Rural Support Trust members in particular had carried out "outstanding work in the region," particularly during and after the drought.

Much of their work was confidential, and often went unrecognised, but judges said the Regional Leadership Award was an acknowledgement of everything they do to support rural communities and the people within them.

The rest is here:
Winners announced in inaugural Beef + Lamb New Zealand Awards - New Zealand Herald

Recommendation and review posted by Bethany Smith

Press Release

So, you want to grow some sweet cannabis seeds but you have no clue where to start?

Or worse, youve tried growing your own but you got 90% male cannabis plants and 10% (dead) females?

Well, Im supposed to help you with that

But since you may be doing something illegal, Im not sure if I will.

First, tell me where you live and then Ill give you a hand (maybe).

(Im listening)

Hum, okay, thats fine.

Thats a pretty terrible place to live in, so you do deserve a hand (just kidding).

Ive spent weeks comparing the 10 best seed banks that ship to the USA from germination guarantees to pricing to potency to genetics and tons of boring stuff

All this so that you dear newbie wouldnt have the same **** (less-than-ideal) experience I had my first time around.

(And the second time)

(And the third but lets not dwell on the past!)

So, weed on! (Sorry, I couldnt resist)

Thailand Disclaimer: As of June 9th 2022, cannabis will be removed from the Category 5 Narcotics list. Despite this, to import cannabis and hemp seeds into Thailand, you will need to seek permission according to the 1964 Plant Quarantine Act and the 1975 Plants Act.

Pros

Cons

Accepted Payment Methods

Robert Bergman founded I Love Growing Marijuana in 2012 as a small blog sharing growing tips. When the blog became a hit, readers began asking where they could buy seeds.

But since most pre-legalization seed banks were credit card scams

Bergman decided to partner with breeders and start the ILGM seed bank. Fast forward 10 years and ILGM is one of the most reputable seed banks in the world.

As such, it offers a 100% germination guarantee. If a seed doesnt sprout, contact the support and they will send you new ones for free.

Speaking about free stuff

ILGM offers free shipping to the USA as well as frequent Buy 10 Get 10 Free deals on popular strains such as White Widow and Super Skunk. If youre serious about growing cannabis, we recommend checking those out first.

By the way, how do you find the right seeds?

Well, ILGM has pretty good categories and search filters. As such, you can filter cannabis seeds by THC levels, price, climate, height, yield, difficulty, flowering period, and more. You can also check customer reviews on most strains to see if the description matches real-life experiences.

And since were talking about customer reviews, its also worth mentioning that I Love Growing Marijuana has received over 27,000 of them, with a good 8.9 overall rating

>> Check ILGMs Buy 10 Get 10 deals on the official site (free US shipping) <<

Cons

Accepted Payment Methods

Crop King wont die and neither will its marijuana seeds.

Crop King Seeds is a Canadian seed bank founded in the early 2000s. It had to shut down its operations in 2005 and re-established itself in the United States due to murky cannabis legality.

The company grew its operations due to the legalization of cannabis in some US states and finally returned to Calgary in 2013 making it one of the longest-running seed banks in North America.

But what male and female seeds does it stock today?

Well, the seed bank has over 500 cannabis strains, including high-THC and high-yielding seeds like Gorilla Glue and Alaskan Thunder Fuck, respectively.

Like ILGM, Crop King Seeds provides a germination guarantee, although it only covers an 80% rate instead of 100%. That said, its still much better than most cannabis seed banks online that provide zero guarantees.

Speaking about guarantees

Crop King has very helpful customer support that you can reach 24/7 via its live chat feature. If you have any problems with your seeds or even if you just want a recommendation, theyre quite knowledgeable and care about their customers.

>> Check out Crop Kind Seeds 500+ strains on the official website <<

Pros

Cons

Accepted Payment Methods

Seedsman is one of the oldest seed banks to buy from, being in business since 2002.

But perhaps more importantly, the company stocks over 4,000 cannabis strains, including everything you can think of high-CBD, high-THC, high-yielding, pest-resistant, and compact plants, just to name a few.

Besides, Seedsman is actively campaigning for the legalization of marijuana, not just within the UK but all over the world.

Good to know: Although other websites also contain guides and instructional materials, Seedsman takes their website to another level with detailed histories of their famous strains. This might be interesting to cannabis enthusiasts who want to learn about the strains they are growing.

But as to what you really care about

Seedsman has several promotions to make buying cannabis seeds online affordable. Besides a few BOGO deals, you can also get a 10% discount when you buy over $200 worth of seeds, as well as up to 6 free cannabis seeds.

>>Check out Seedsmans BOGO deals and get 10% off bulk orders (official site) <<

Pros

Cons

Accepted Payment Methods

Rocket Seeds is a seed bank that is also a partner with other cannabis seed banks and breeders. They carry high-quality seeds from the likes of Crop King Seeds and Beaver Seeds.

Its basically a one-stop shop for comparing the best seed banks online.

This means that growers might be able to shop for popular strains from different cannabis seed banks and breeders in just one site. This can help them save time when looking for a new strain to grow.

During our research, one of the common compliments that Rocket Seeds received is how fast and reliable their shipping is.

>> Check out the best seed banks on Rocket Seeds official website <<

Pros

Cons

Accepted Payment Methods

Quebec Cannabis Seeds has been in operation for more than 15 years. The company tries to come up with strains that are less vulnerable to pests and diseases.

Although QCS doesnt produce as many new strains as other online cannabis seed banks, they make up for it by introducing new strains resistant to diseases and pests.

If you are a grower whose plants are always getting damaged, Quebec Cannabis Seeds strains might be the ones you are looking for particularly their auto-flowering cannabis seeds.

Aside from the usual phone number, Quebec Cannabis Seeds customer service is also available via email so you can send them a detailed inquiry if you have one.

Its website also has a live chat that customers can use although it is only available from Mondays through Fridays from 10:00 AM to 6:00 PM.

>> Check out current deals on Quebec Cannabis Seeds official website <<

Pros

Cons

Accepted Payment Methods

Beaver Seeds started in 2009 and is one of the best cannabis seed banks to buy from in Canada. The company claims that they consciously limited the number of strains they sell to avoid selling inferior strains.

This Canada-based seed bank only carries popular strains to limit its selection.

The company has what they call a seed library. It has detailed descriptions of high-quality weed seeds (including feminized seeds) and information on the proper care for each strain. This might help growers since they can have as they talk a lot about the best water levels, climate, and fertilizer.

Beaver Seeds provides customer support 24/7 which might help growers who need assistance, especially those who are just starting. This can be a good sign for buyers located in other countries since they dont have to wait for Canadian business hours to get assistance.

>> Try Beaver Seeds amazing customer support on the official website <<

Pros

Cons

Accepted Payment Methods

Like other best seed banks, Sun West Genetics has been in operation for over a decade. They sell auto-flowering, medical, and feminized seeds to customers.

Sun West Genetics can be a great source of premium marijuana seeds with over 500 different strains in its roster of high-quality cannabis seeds online.

The wide variety of strains can also be appealing to those who want to open their dispensary since they can offer more strains to their end-users.

Sun West Genetics has a breeding program that continuously tries to discover new breeds for customers. This can be great for customers looking for new breeds that might be more suited to growing in their location.

>> Check out the amazing seed genetics on Sun West Genetics official website <<

Pros

Cons

Accepted Payment Methods

Sonoma Seeds is one of the best marijuana seed banks based on the US West Coast. It sells strains sourced from all over the world. The company also ships weed seeds to customers globally.

Sonoma Seeds customers have a wide array of payment options that they can choose from. Customers can benefit from this since they wont encounter any difficulties looking for a payment option that will work best.

Sonoma Seeds also provides free shipping for orders exceeding $200. However, to maintain accurate shipping, the company charges a nominal $30 International Shipping with a Tracking fee. This fee can benefit customers since theyll track their shipping as it makes its way to the customers country.

The seed bank ships seeds worldwide which might make them a favourite among buyers outside of the United States.

>> Get worldwide shipping on Sonoma Seeds official website <<

Pros

Cons

Accepted Payment Methods

Marijuana Seeds Canada is a seed bank that started operations in 2009. It holds offices in Vancouver and promises to deliver weed seeds with high germination rates.

This company offers affordable medical marijuana seeds on its website. Based on our research, their medicinal seeds are cheaper by around $5 than most seed banks online, and you get free seeds on bulk orders.

MJ Seeds Canada provides customers with an 80% Germination Guarantee. Growers may find this attractive since the marijuana seed bank guarantees that at least 8 out of 10 seeds will sprout and be ready for planting.

While doing our research, one of the most common customer comments we came across is how responsive MJ Seeds Canadas customer service is. You might find this useful since it means that the seed bank values its customers time and tries to attend to their inquiries promptly.

>> Get high-quality auto-flowering seeds on MJ Seeds official website <<

Pros

Cons

Follow this link:
10 Best Seed Banks that Ship Cannabis Seeds Discreetly to You (Free US Shipping) - Thaiger

Recommendation and review posted by Bethany Smith

Theres a handful of reasons why the Philadelphia region has been (perhaps unfortunately) dubbed Cellicon Valley in the last few years, and a new report from the Chamber of Commerce for Greater Philadelphia and economic consulting firm Econsult Solutions has IDd them all.

In the report, which looked at 14 large cell and gene therapy hubs in the US, Philadelphia ranked as the runner up, just behind Boston, as the top spot for research and innovation in this space. Other metro areas such as New York and San Francisco scored the third and fourth spots on the list. The report shouts out early local work, including the first FDA-approved gene (Luxturna) and cell (Kymriah) therapies developed here at Spark Therapeutics and the University of Pennsylvania, respectively.

The Philadelphia region is increasingly attracting new and expanding cell and gene therapy companies because it checks all the boxes, but its the regions research infrastructure as defined by NIH-funded cell and gene therapy research and its large number of research institutions that give it the edge, said Claire Marrazzo Greenwood, executive director and CEO of Council for Growth and SVP of economic competitiveness for the Chamber, in a statement.

The study compared cell and gene therapy hubs for their research infrastructure, human capital, innovation output, commercial activity and value proposition. Heres why Philly ranked high:

Because Philly is home to four Tier 1 universities, 93 higher ed institutions, and tons of hospitals and research institutions, it scored second in research infrastructure. The region scored first for most National Health Institute funding, and the report said 302 gene or cell therapy patents had been approved in the last decade. In 2021, the region was home to 15,400 jobs in pharmaceutical manufacturing, and it pulled in $4.2 billion in venture capital funding since 2018.

The talent coming from the high number of universities and colleges and more than 450,000 students in the region also ranked the region high for human capital. Of this, a whopping 54% stay in the region. R&D jobs in the field have also increased more than 100% in the last five years.

Philadelphia also scored high for its innovation output, meaning the region produces a large amount of intellectual property in the cell and gene therapy space. As the birthplace of the industry, the report says, the region is currently home to 302 granted patent and 130 clinical trials now underway.

The large amount of attention cell and gene therapy has gotten from investors in the last four years also ranked the region high in commercial activity. Within the past few years, two local cell and gene therapy companies Passage Bio and Cabaletta Bio have also completed IPOs, raising more than $260 million combined. Cell and gene therapy companies also make up a significant portion of Phillys commercial real estate, leasing about 12 million square feet, with about 9 million planned in construction projects.

And Philadelphias value proposition, or cost to do business, helped the region rank so highly, the report said. The region attracts families and talent with cultural institutions, culinary scene and schools. Plus, life sciences office space rentals (averaging about $58 per square foot) were very affordable next to cities like San Francisco (at $78 per square foot).

Greater Philadelphia is an extremely livable region, boasting some of the worlds best museums, top-notch restaurants, and large open spaces at a comparatively affordable price, Econsult said in its summary.

Go here to see the original:
Why Philly ranks #2 among best cell and gene therapy hubs in the US - Technical.ly

Recommendation and review posted by Bethany Smith

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced the dosing with ATA-100 of a first patient in a phase 1/2 clinical study in FRKP-related limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

This is an exciting milestone for our company but most importantly, if this clinical trial is successful, it could have a life-changing impact for patients affected by LGMD-R9, said Stephane Degove, Chief Executive Officer and Co-Founder of Atamyo Therapeutics.

This clinical trial (EudraCT 2021-004276-33, NCT05224505) is a multicenter, Phase 1/2 study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of intravenous ATA-100, a single-dose Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.

This study will consist of 2 phases: an open-label dose escalation phase (Stage 1) and a double-blind placebo controlled, randomized phase (Stage 2).

LGMD-R9 is a severe progressive and debilitating disease with no approved treatment, said Pr. John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, in Copenhagen, where the first patient was dosed, and principal investigator of this trial. This experimental treatment represents a new hope for the patients. It is a great motivation to know that the work we are doing here has the potential to make a life-changing difference.

After the first patient dosed in Copenhagen, we are now expecting recruitments at the two other approved clinical sites (Paris, FR, and Newcastle, UK) to complete enrollment of the dose escalation phase (Stage 1) of the study. For Stage 2 (after dose selection), we plan to open additional clinical sites in Europe and in the United States, said Dr. Sophie Olivier, Chief Medical Officer of Atamyo.

About the LGMD-R9 program ATA-100

ATA-100 is a one-time gene replacement therapy for LGMD-R9/2I based on the research of Dr. Isabelle Richard, who heads the Progressive Muscular Dystrophies Laboratory at Genethon (UMR 951 INSERM/Genethon/UEVE). ATA-100 has been awarded Orphan Drug Designation status by the U.S. Food and Drug Administration and the European Medicines Agency.

LGMD2I/R9 is a rare genetic disease caused by mutations in the gene that produces fukutin-related protein (FKRP). It affects an estimated 5,000 people in the US and Europe. Symptoms appear around late childhood or early adulthood. Patients suffer from progressive muscular weakness leading to loss of ambulation. They also are prone to respiratory impairment and myocardial dysfunction. There are currently no curative treatments for LGMDR9.

About Atamyo Therapeutics

Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyos most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD). The name of the company is derived from two words: Celtic Atao which means Always or Forever and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit http://www.atamyo.com

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Atamyo Therapeutics Announces First Patient Dosed with ATA-100 Gene Therapy in LGMD-R9 Clinical Trial - Business Wire

Recommendation and review posted by Bethany Smith

Gene Therapy Cell Culture Media Market Research ReportThe global Gene Therapy Cell Culture Media industry research report provides an in-depth and methodical assessment of regional and global markets, as well as the most current service and product innovations and the global markets predicted size. The Gene Therapy Cell Culture Media research does a complete market analysis to find the major suppliers by integrating all relevant products and services in order to understand the roles of the top industry players in the Gene Therapy Cell Culture Media segment. The global Gene Therapy Cell Culture Media market also provides a thorough analysis of cutting-edge competitor research and new industry advancements, as well as market dynamics, challenges, restrictions, and opportunities, in order to give precise insights and the latest scenarios for appropriate judgments.

The gene therapy cell culture media market was valued at 152.67 million in 2019 and is expected to record a CAGR of 10.87% during the forecast period, 20202029.

Get FREE PDF Sample Copy of the Report @ https://straitsresearch.com/report/gene-therapy-cell-culture-media-market/request-sample

This report centers about the top players in global Gene Therapy Cell Culture Media marketplace:Lonza, HiMedia, GE Healthcare, FUJIFILM Irvine Scientific, R&D Systems, Inc. (Bio-Techne), Thermo Fisher Scientific and Sartorius AG.

This research study contains a SWOT analysis, significant trends, and a financial evaluation of the Gene Therapy Cell Culture Media and the global markets major competitors. Additionally, the Gene Therapy Cell Culture Media study provides a complete perspective of the Gene Therapy Cell Culture Media market and assists organizations in generating sales by providing a better knowledge of the leading competitors growth plans and competitive environment. This report includes a deep investigation of PEST and the industrys overall dynamics during the anticipated term. The research includes essential results as well as highlights of guidance and significant industry changes in the Gene Therapy Cell Culture Media industry, supporting market leaders in developing new tactics to increase income.

Top key industry segmentsBy Type: Lysogeny Broth, Chemically defined media, Serum free media, Specialty media, Stem cell media, Custom media, OthersBy End User: Pharmaceutical & Biotechnology companies, Academic institutions, Research laboratories, Others

The global Gene Therapy Cell Culture Media study also looks at industry trends, size, cost structure, revenue, potential, market share, drivers, opportunities, competitive environment, market challenges, and market forecast. This study also includes a complete and general review of the Gene Therapy Cell Culture Media industry, as well as in-depth industry variables that affect market growth. In addition to supply chain characteristics, key players current market conditions, and a generally discussed market pricing study, the Gene Therapy Cell Culture Media research contains insights on supply chain features, key players recent market situations, and a widely talked market price study. Aside from the acceptance rate, the global Gene Therapy Cell Culture Media market study shows the entire quantity of technical progress produced in recent years. It does a complete study of the Gene Therapy Cell Culture Media market using SWOT analysis.

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The Gene Therapy Cell Culture Media market analysis covers many of the important device developments that are now being used in the global sector. The end-user is primarily concerned with the production of global Gene Therapy Cell Culture Media market items, and market prices reflect this. Global Gene Therapy Cell Culture Media market operators, including regional and global companies, place work orders with global Gene Therapy Cell Culture Media market manufacturers. As a consequence, demand numbers for the global Gene Therapy Cell Culture Media market are derived from the perspective of end-users, based on their orders.

About Us:StraitsResearch is a leading research and intelligence organization, specializing in research, analytics, and advisory services along with providing business insights & research reports.Contact Us:Email: sales@straitsresearch.comTel: +1 6464807505, +44 203 318 2846

Other Reports:https://www.globenewswire.com/en/news-release/2022/07/26/2486248/0/en/Battery-Recycling-Market-Size-is-projected-to-reach-USD-18-96-Billion-by-2030-growing-at-a-CAGR-of-7-12-Straits-Research.htmlhttps://www.digitaljournal.com/pr/airbag-control-unit-market-share-to-witness-significant-revenue-growth-during-the-forecast-period-2026https://www.digitaljournal.com/pr/light-fidelity-li-fi-devices-market-study-by-latest-research-trends-and-revenue-till-2026-top-key-players-signify-holding-philips-n-v-netherlands-purelifi-uk-oledcomm-francehttps://www.digitaljournal.com/pr/application-modernization-services-industry-report-global-market-manufacturers-outlook-growth-and-forecast-2029

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Gene Therapy Cell Culture Media Market Research Report is Likely to grow at a higher CAGR during the Forecast Period The Colby Echo News - The Colby...

Recommendation and review posted by Bethany Smith

Sanofi will partner with the Californian biotechnology company Scribe Therapeutics in a deal that extends its exploration of new ways to build cancer cell therapies.

Under a partnership announced Tuesday, Sanofi will pay Scribe $25 million upfront to gain access to the five-year-old startups gene editing technology. The pharmaceutical company is also promising more than $1 billion in additional payments based on unspecified development and commercial milestones, although that money may never be paid out.

In return, Sanofi gets non-exclusive rights to use Scribes CRISPR-based gene editing technology to develop cancer treatments constructed from modified natural killer, or NK, cells. A type of immune defender, NK cells have drawn increasing interest from cancer drugmakers looking for alternatives to the T cells used in CAR-T treatments for leukemia, lymphoma and multiple myeloma.

This collaboration with Scribe complements our robust research efforts across the NK cell therapy spectrum and offers our scientists unique access to engineered CRISPR-based technologies as they strive to deliver off-the-shelf NK cell therapies and novel combination approaches that improve upon the first generation of cell therapies, said Frank Nestle, Sanofis head of research and chief scientific officer, in a statement.

Sanofi missed the first wave of cancer cell therapy development, which companies like Novartis, Gilead and, more recently, Bristol Myers Squibb have led. But it appears interested in making up ground with bets on newer technologies.

In November 2020, Sanofi bought Kiadis Pharma and its pipeline of donor-derived NK cell therapies. Five months later, the company acquired Tidal Therapeutics, which was attempting to use messenger RNA to reprogram immune cells in the body to attack cancers.

While a much smaller financial commitment, the partnership with Scribe could help Sanofi better develop NK cells therapies. Scribes gene editing technology relies on the CRISPR framework pioneered by its cofounder Jennifer Doudna, but the company has developed its own DNA-cutting enzymes, too.

Scribe raised $100 million in a Series B round last spring and in March hired ex-Barclays banker David Parrot as its chief financial officer. In an interview with CFO Dive, Parrot said he had been brought on to help eventually launch an initial public offering, but noted the company would focus first on inking partnerships as public markets remain cool to IPOs.

The deal with Sanofi is the second Scribe has disclosed publicly. Its also working with Biogen on a research collaboration focused on ALS and another undisclosed disease.

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Sanofi partners with Scribe to gain gene editing tools for cell therapy work - BioPharma Dive

Recommendation and review posted by Bethany Smith

BLA Includes Substantial Body of Data from Pivotal Phase 3 and Ongoing Phase 1/2 Studies

If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A

SAN RAFAEL, Calif., Sept. 29, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Company resubmitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The resubmission incorporates the Company's response to the FDA Complete Response (CR) Letter for valoctocogene roxaparvovec gene therapy issued on August 18, 2020, and subsequent feedback, including two-year outcomes from the global GENEr8-1 Phase 3 study and supportive data from five years of follow-up from the ongoing Phase 1/2 dose escalation study.

BioMarin anticipates an FDA response by the end of October on whether the BLA resubmission is complete and acceptable for review. Typically, BLA resubmissions are followed by a six-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure. If approved, valoctocogene roxaparvovec would be the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017.

In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN on August 24, 2022 and endorsed the recommendation from the European Medicines Agency (EMA) to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity in the European Union.

"We are pleased to reach this point in the development program for valoctocogene roxaparvovec and look forward to working with the FDA with the goal of bringing a potentially transformative therapy to people with severe hemophilia A in the United States," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "This large and robust data set provided in this BLA resubmission shows an encouraging efficacy profile. We remain committed to sharing these data with the public, along with even longer-term data generated through our ongoing clinical trials and any post-approval studies, to further our understanding of AAV gene therapy in severe hemophilia A and of gene therapies more broadly."

The resubmission includes a substantial body of data from the valoctocogene roxaparvovec clinical development program, the most extensively studied gene therapy for severe hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study. The GENEr8-1 Phase 3 study demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate. In addition, the data package included supportive evidence from five years of follow-up from the 6e13 vg/kg dose cohort in the ongoing Phase 1/2 dose escalation study. The resubmission alsoincludesaproposedlong-term extension studyfollowingall clinicaltrialparticipantsfor up to 15years, as well astwo post-approval registry studies.

Robust Clinical Program

BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A (Study 270-303). Also ongoing are a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors (Study 270-205).

Safety Summary

Overall, to date, a single 6e13 vg/kg dose of valoctocogene roxaparvovec has been well tolerated with no delayed-onset treatment related adverse events. The most common adverse events (AE) associated with valoctocogene roxaparvovec have occurred early and included transient infusion associated reactions and mild to moderate rise in liver enzymes with no long-lasting clinical sequelae. Alanine aminotransferase (ALT) elevation, a laboratory test of liver function, has remained the most common adverse drug reaction. Other adverse reactions have included aspartate aminotransferase (AST) elevation (101 participants, 63%), nausea (55 participants, 34%), headache (54 participants, 34%), and fatigue (44 participants, 28%). No participants have developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with valoctocogene roxaparvovec.

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (Factor VIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (Factor VIII 1-5%) or mild (Factor VIII 5-40%) disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week (100-150 infusions per year) or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month (12-48 injections or shots per year). Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visitwww.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin anticipating an FDA response by the end of October on whether the BLA resubmission is complete and acceptable for review, BioMarin's expectations regarding the duration of the review procedure, valoctocogene roxaparvovec being the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A, if approved, BioMarin's commitment to sharing longer-term data generated through its ongoing clinical trials and any post-approval studies. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec; additional data from the continuation of the clinical trials of valoctocogene roxaparvovec, any potential adverse events observed in the continuing monitoring of the participants in the clinical trials; the content and timing of decisions by the FDA and other regulatory authorities, including decisions to grant additional marketing registrations based on an EMA license; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec for the clinical trials and commercially; and those and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc and ROCTAVIAN is a trademark of BioMarin Pharmaceutical Inc.

Contacts:

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Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

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BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA - PR...

Recommendation and review posted by Bethany Smith

ReportLinker

INTRODUCTION With the increasing number of cell and gene therapies being developed and launched for a wide range of therapeutic areas, these modalities are on their way to become one of the highest valued markets in the biopharmaceutical domain.

New York, Sept. 29, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application Area, Therapeutic Area, and Geographical Regions : Industry Trends and Global Forecasts, 2022-2035" - https://www.reportlinker.com/p06323417/?utm_source=GNW In fact, in 2021, cell and gene therapy developers raised capital worth more than USD 20 billion, registering an increase of 19% from the amount raised in 2020 (~USD 17 billion). It is worth highlighting that, in February 2022, the USFDA approved second CAR-T therapy, CARVYKTI, developed by Johnson and Johnson, which can be used for the treatment of relapsed or refractory multiple myeloma. Additionally, close to 1,500 clinical trials are being conducted, globally, for the evaluation of cell and gene therapies. Over time, it has been observed that the clinical success of these therapies relies on the design and type of gene delivery vector used (in therapy development and / or administration). At present, several innovator companies are actively engaged in the development / production of viral vectors and / or non-viral vectors for cell and gene therapies. In this context, it is worth mentioning that, over the past few years, multiple viral vector and non-viral vector based vaccine candidates have been developed against COVID-19 (caused by novel coronavirus, SARS-CoV-2) and oncological disorders; this is indicative of lucrative opportunities for companies that have the required capabilities to manufacture vectors and gene therapies.

The viral and non-viral vector manufacturing landscape features a mix of industry players (well-established companies, mid-sized firms and start-ups / small companies), as well as several academic institutes. It is worth highlighting that several companies that have the required capabilities and facilities to manufacturing vectors for both in-house requirements and offer contract services (primarily to ensure the optimum use of their resources and open up additional revenue generation opportunities) have emerged in this domain. Further, in order to produce more effective and affordable vectors, several stakeholders are integrating various novel technologies; these technologies are likely to improve the scalability and quality of the resultant therapy. In addition, this industry has also witnessed a significant increase in the partnership and expansion activities over the past few years, with several companies having been acquired by the larger firms. Given the growing demand for interventions that require genetic modification, the vector and gene therapy manufacturing market is poised to witness substantial growth in the foreseen future.

SCOPE OF THE REPORTThe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (5th Edition) by Scale of Operation (Preclinical, Clinical and Commercial), Type of Vector (AAV Vector, Adenoviral Vector, Lentiviral Vector, Retroviral Vector, Plasmid DNA and Others), Application Area (Gene Therapy, Cell Therapy and Vaccine), Therapeutic Area (Oncological Disorders, Rare Disorders, Neurological Disorders, Sensory Disorders, Metabolic Disorders, Musco-skeletal Disorders, Blood Disorders, Immunological Diseases, and Others), and Geographical Regions (North America, Europe, Asia Pacific, MENA, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the rapidly growing market of vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies having in-house manufacturing facilities. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe. Amongst other elements, the report includes:An overview of the current status of the market with respect to the players engaged (both industry and non-industry) in the manufacturing of viral, non-viral and other novel types of vectors and gene therapies. It features information on the year of establishment, company size, location of headquarters, type of product manufactured (vector and gene therapy / cell therapy / vaccine), location of manufacturing facilities, type of manufacturers (in-house and contract services), scale of operation (preclinical, clinical and commercial), type of vector manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others) and application area (gene therapy, cell therapy, vaccine and others).An analysis of the technologies offered / developed by the companies enagaged in this domain, based on the type of technology (viral vector related platform, non-viral vector related platform and others), type of manufacturer (vector manufacturing, gene delivery, product manufacturing, transduction / transfection, vector packaging and other), scale of operation (preclinical, clinical and commercial), type of vector involved (AAV, adenoviral, lentiviral, retroviral, non-viral and other viral vectors), application area (gene therapy, cell therapy, vcaccine and others). It also highlights the most prominent players within this domain, in terms of number of technologies.A region-wise, company competitiveness analysis, highlighting key players engaged in the manufacturing of vectors and gene therapies, across key geographical areas, featuring a four-dimensional bubble representation, taking into consideration supplier strength (based on experience in this field), manufacturing strength (type of product manufactured, number of manufacturing facilites and number of application areas), service strength (scale of operation, number of vectors manufactured and geographical reach) and company size (small, mid-sized and large).Elaborate profiles of key players based in North America, Europe and Asia-Pacific (shortlisted based on proprietary criterion). Each profile features an overview of the company / organization, its financial performance (if available), information related to its manufacturing facilities, vector manufacturing technology and an informed future outlook.Tabulated profiles of the other key players headquartered in different regions across the globe (shortlisted based on proprietary criterion). Each profile features an overview of the company, its financial performance (if available), information related to its manufacturing capabilities, and an informed future outlook.An analysis of partnerships and collaborations established in this domain since 2015; it includes details of deals that were / are focused on the manufacturing of vectors, which were analyzed on the basis of year of partnership, type of partnership (manufacturing agreement, product / technology licensing, product development, merger / acqusition, research and development agreement, process development / optimization, service alliance, production asset / facility acquisition, supply agreement and others), scale of operation (preclinical, clinical and commercial), type of vector involved (AAV, adenoviral, lentiviral, retroviral, plasmid and others), region and most active players (in terms of number of partnerships).An analysis of the expansions related to viral vector and non-viral vector manufacturing, which have been undertaken since 2015, based on several parameters, such as year of expansion, type of expansion (new facility / plant establishment, facility expansion, technology installation / expansion, capacity expansion, service expansion and others), type of vector (AAV, adenoviral, lentiviral, retroviral, plasmid and others), application area (gene therapy, cell therapy, vaccine and others) and geographical location of the expansion.An analysis evaluating the potential strategic partners (comparing vector based therapy developers and vector purification product developers) for vector and gene therapy product manufacturers, based on several parameters, such as developer strength, product strength, type of vector, therapeutic area, pipeline strength (preclinical and clinical).An overview of other viral / non-viral gene delivery approaches that are currently being researched for the development of therapies involving genetic modification.An in-depth analysis of viral vector and plasmid DNA manufacturers, featuring three schematic representations, a three dimensional grid analysis, representing the distribution of vector manufacturers (on the basis of type of vector) across various scales of operation and type of manufacturer (in-house operations and contract manufacturing services), a heat map of viral vector and plasmid DNA manufacturers based on the type of vector (AAV, adenoviral vector, lentiviral vector, retroviral vector and plasmid DNA) and type of organization (industry (small, mid-sized and large) and non-industry), and a schematic world map representation, highlighting the headquarters and geographical location of key vector manufacturing hubs.An analysis of the various factors that are likely to influence the pricing of vectors, featuring different models / approaches that may be adopted by product developers / manufacturers in order to decide the prices of proprietary vectors.An estimate of the overall, installed vector manufacturing capacity of industry players based on the information available in the public domain, and insights generated via both secondary and primary research. The analysis also highlights the distribution of the global capacity by company size (small, mid-sized and large), scale of operation (clinical and commercial), type of vector (viral vector and plasmid DNA) and region (North America, Europe, Asia Pacific and the rest of the world).An informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.A discussion on the factors driving the market and various challenges associated with the vector production process.A qualitative analysis, highlighting the five competitive forces prevalent in this domain, including threats for new entrants, bargaining power of drug developers, bargaining power of vector and gene therapy manufacturers, threats of substitute technologies and rivalry among existing competitors.

One of the key objectives of this report was to evaluate the current market size and the future opportunity associated with the vector and gene therapy manufacturing market, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies, anticipated growth in target patient population, existing price variations across different types of vectors, and the anticipated success of gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2022-2035. In order to provide a detailed future outlook, our projections have been segmented on the basis of scale of operation (preclinical, clinical and commercial), type of vector (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), application area (gene therapy, cell therapy and vaccine), therapeutic area (oncological disorders, rare disorders, neurological disorders, sensory disorders, metabolic disorders, musco-skeletal disorders, blood disorders, immunological diseases, and others) and geographical region (North America, Europe, Asia Pacific, MENA, Latin America and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. For the purpose of the study, we invited over 300 stakeholders to participate in a survey to solicit their opinions on upcoming opportunities and challenges that must be considered for a more inclusive growth. The opinions and insights presented in this study were also influenced by discussions held with senior stakeholders in the industry. The report features detailed transcripts of interviews held with the following industry and non-industry players:Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences)Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals)Cedric Szpirer (Former Executive & Scientific Director, Delphi Genetics)Olivier Boisteau, (Co-Founder / President, Clean Cells), Laurent Ciavatti (Former Business Development Manager, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells)Alain Lamproye (Former President of Biopharma Business Unit, Novasep)Joost van den Berg (Former Director, Amsterdam BioTherapeutics Unit)Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital)Eduard Ayuso, DVM, PhD (Scientific Director, Translational Vector Core, University of Nantes)Colin Lee Novick (Managing Director, CJ Partners)Semyon Rubinchik (Scientific Director, ACGT)Astrid Brammer (Senior Manager Business Development, Richter-Helm)Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Former Marketing Manager, Plasmid Factory)Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing)Beatrice Araud (ATMP Key Account Manager, EFS-West Biotherapy)Nicolas Grandchamp (R&D Leader, GEG Tech)Graldine Gurin-Peyrou (Director of Marketing and Technical Support, Polypus Transfection)Naiara Tejados, Head of Marketing and Technology Development, VIVEBiotech)Jeffery Hung (Independent Consultant)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGYThe data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market may evolve across different regions and technology segments. Wherever possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include:Annual reportsInvestor presentationsSEC filingsIndustry databasesNews releases from company websitesGovernment policy documentsIndustry analysts views

While the focus has been on forecasting the market over the period 2022-2035, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWEREDWho are the leading players (contract service providers and in-house manufacturers) engaged in the development of vectors and gene therapies?Which regions are the current manufacturing hubs for vectors and gene therapies?Which type of vector related technologies are presently offered / being developed by the stakeholders engaged in this domain?Which companies are likely to partner with viral and non-viral vector contract manufacturing service providers?Which partnership models are commonly adopted by stakeholders engaged in this industry?What type of expansion initiatives are being undertaken by players in this domain?What are the various emerging viral and non-viral vectors used by players for the manufacturing of genetically modified therapies?What are the strengths and threats for the stakeholders engaged in this industry?What is the current, global demand for viral and non-viral vector, and gene therapies?How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 is an executive summary of the insights captured in our research. It offers a high-level view on the likely evolution of the vector and gene therapy manufacturing market in the short to mid-term, and long term.

Chapter 3 is a general introduction to the various types of viral and non-viral vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of the currently available gene delivery vehicles. The chapter also features the clinical and approved pipeline of genetically modified therapies. Further, it includes a review of the latest trends and innovations in the contemporary vector manufacturing market.

Chapter 4 provides a detailed overview of close to 150 companies, featuring both contract service providers and in-house manufacturers that are actively involved in the production of viral vectors and / or gene therapies utilizing viral vectors. The chapter provides details on the year of establishment, company size, location of headquarters, type of product manufactured (vector and gene therapy / cell therapy / vaccine), location of manufacturing facilities, type of manufacturer (in-house and contract services), scale of operation (preclinical, clinical and commercial), type of vector manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 5 provides an overview of close to 70 industry players that are actively involved in the production of plasmid DNA and other non-viral vectors and / or gene therapies utilizing non-viral vectors. The chapter provides details on the the year of establishment, company size, location of headquarters, type of product manufactured (vector and gene therapy / cell therapy / vaccine), location of plasmid DNA manufacturing facilities, type of manufacturer (in-house and contract services), scale of operation (preclinical, clinical and commercial) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 6 provides an overview of close to 90 non-industry players (academia and research institutes) that are actively involved in the production of vectors (both viral and non-viral) and / or gene therapies. The chapter provides details on the year of establishment, type of manufacturer (in-house and contract services), scale of operation (preclinical, clinical and commercial), location of headquarters, type of vector manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 7 features an in-depth analysis of the technologies offered / developed by the companies engaged in this domain, based on the type of technology (viral vector and non-viral vector related platform), purpose of technology (vector manufacturing, gene delivery, product manufacturing, transduction / transfection, vector packaging and other), scale of operation (preclinical, clinical and commerical), type of vector involved (AAV, adenoviral, lentiviral, retroviral, non-viral and other viral vectors), application area (gene therapy, cell therapy, vaccine and others) and leading technology providers.

Chapter 8 presents a detailed competitiveness analysis of vector manufacturers across key geographical areas, featuring a four-dimensional bubble representation, taking into consideration supplier strength (based on its experience in this field), manufacturing strength (type of product manufactured, number of manufacturing facilities and number of application area), service strength (scale of operation, number of vectors manufactured and geographical reach) and company size (small, mid-sized and large).

Chapter 9 features detailed profiles of some of the key players that have the capability to manufacture viral vectors / plasmid DNA in North America. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 10 features detailed profiles of some of the key players that have the capability to manufacture viral vectors / plasmid DNA in Europe. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 11 features detailed profiles of some of the key players that have the capability to manufacture viral vectors / plasmid DNA in Asia-Pacific. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 12 features tabulated profiles of the other key players that have the capability to manufacture viral vectors / plasmid DNA. Each profile features an overview of the company, its financial performance (if available), information related to its manufacturing capabilities, and an informed future outlook.

Chapter 13 features in-depth analysis and discussion of the various partnerships inked between the players in this market, during the period, 2015-2022, covering analysis based on parameters such as year of partnership, type of partnership(manufacturing agreement, product / technology licensing, product development, merger / acquisition, research and development agreement, process development / optimization, service alliance, production asset / facility acquisition, supply agreement and others), scale of operation (preclinical, clinical and commercial) and type of vector (AAV, adenoviral, lentiviral, retroviral, plasmid and others) most active players (in terms of number of partnerships).

Chapter 14 features an elaborate discussion and analysis of the various expansions that have been undertaken, since 2015. Further, the expansion activities in this domain have been analyzed on the basis of year of expansion, type of expansion (new facility / plant establishment, facility expansion, technology installation / expansion, capacity expansion, service expansion and others), geographical location of the facility, type of vector (AAV, adenoviral, lentiviral, retroviral, plasmid and others) and application area (gene therapy, cell therapy, vaccine and others).

Chapter 15 highlights potential strategic partners (vector based therapy developers and vector purification product developers) for vector and gene therapy product manufacturers, based on several parameters, such as developer strength, product strength, type of vector, therapeutic area, pipeline strength (clinical and preclinical). The analysis aims to provide the necessary inputs to the product developers, enabling them to make the right decisions to collaborate with industry stakeholders with relatively more initiatives in the domain.

Chapter 16 provides detailed information on other viral / non-viral vectors. These include alphavirus vectors, Bifidobacterium longum vectors, Listeria monocytogenes vectors, myxoma virus based vectors, Sendai virus based vectors, self-complementary vectors (improved versions of AAV), minicircle DNA and Sleeping Beauty transposon vectors (non-viral gene delivery approach) and chimeric vectors, that are currently being utilized by pharmaceutical players to develop gene therapies, T-cell therapies and certain vaccines, as well. This chapter presents overview on all the aforementioned types of vectors, along with examples of companies that use them in their proprietary products. It also includes examples of companies that are utilizing specific technology platforms for the development / manufacturing of some of these novel vectors.

Chapter 17 presents a collection of key insights derived from the study. It includes a grid analysis, highlighting the distribution of viral vectors and plasmid DNA manufacturers on the basis of their scale of operation and type of manufacturer (fulfilling in-house requirement / contract service provider). In addition, it consists of a heat map of viral vector and plasmid DNA manufacturers based on the type of vector (AAV, adenoviral vector, lentiviral vector, retroviral vector and plasmid DNA) and type of organization (industry (small, mid-sized and large) and non-industry). The chapter also consists of six world map representations of manufacturers of viral / non-viral vectors (AAV, adenoviral, lentiviral, retroviral vectors, and plasmid DNA), depicting the most active geographies in terms of the presence of the organizations. Furthermore, we have provided a schematic world map representation to highlight the geographical locations of key vector manufacturing hubs across different continents.

Chapter 18 highlights our views on the various factors that may be taken into consideration while pricing viral vectors / plasmid DNA. It features discussions on different pricing models / approaches that manufacturers may choose to adopt to decide the prices of their proprietary products.Chapter 19 features an informed analysis of the overall installed capacity of the vectors and gene therapy manufacturers. The analysis is based on meticulously collected data (via both secondary and primary research) on reported capacities of various small, mid-sized and large companies, distributed across their respective facilities. The results of this analysis were used to establish an informed opinion on the vector production capabilities of the organizations by company size (small, mid-sized and large), scale of operation (clinical and commercial), type of vector (viral vector and plasmid DNA) and region (North America, Europe, Asia Pacific and the rest of the world).

Chapter 20 features an informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies. This section offers an opinion on the required scale of supply (in terms of vector manufacturing services) in this market. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analyzed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.

Chapter 21 presents a comprehensive market forecast analysis, highlighting the likely growth of vector and gene therapy manufacturing market till the year 2030. We have segmented the financial opportunity on the basis of type of vector (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), application area (gene therapy, cell therapy and vaccine), therapeutic area (oncological disorders, rare disorders, neurological disorders, sensory disorders, metabolic disorders, musco-skeletal disorders, blood disorders, immunological diseases, and others), scale of operation (preclinical, clinical and commercial) and geography (North America, Europe, Asia Pacific, MENA, Latin America and rest of the world). Due to the uncertain nature of the market, we have presented three different growth tracks outlined as the conservative, base and optimistic scenarios.

Chapter 22 highlights the qualitative analysis on the five competitive forces prevalent in this domain, including threats for new entrants, bargaining power of drug developers, bargaining power of vector and gene therapy manufacturers, threats of substitute technologies and rivalry among existing competitors.

Chapter 23 provides details on the various factors associated with popular viral vectors and plasmid DNA that act as market drivers and the various challenges associated with the production process. This information has been validated by soliciting the opinions of several industry stakeholders active in this domain.

Chapter 24 presents insights from the survey conducted on over 300 stakeholders involved in the development of different types of gene therapy vectors. The participants, who were primarily Director / CXO level representatives of their respective companies, helped us develop a deeper understanding on the nature of their services and the associated commercial potential.

Chapter 25 summarizes the entire report, highlighting various facts related to contemporary market trend and the likely evolution of the viral vector, non-viral vector and gene therapy manufacturing market.

Chapter 26 is a collection of transcripts of the interviews conducted with representatives from renowned organizations that are engaged in the vector and gene therapy manufacturing domain. In this study, we spoke to Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences), Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals), Cedric Szpirer (Former Executive & Scientific Director, Delphi Genetics), Olivier Boisteau, (Co-Founder / President, Clean Cells), Laurent Ciavatti (Former Business Development Manager, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells), Alain Lamproye (Former President of Biopharma Business Unit, Novasep), Joost van den Berg (Former Director, Amsterdam BioTherapeutics Unit), Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital), Eduard Ayuso, DVM, PhD (Scientific Director, Translational Vector Core, University of Nantes), Colin Lee Novick (Managing Director, CJ Partners), Semyon Rubinchik (Scientific Director, ACGT), Astrid Brammer (Senior Manager Business Development, Richter-Helm), Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Former Marketing Manager, Plasmid Factory), Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing), Beatrice Araud (ATMP Key Account Manager, EFS-West Biotherapy), Nicolas Grandchamp (R&D Leader, GEG Tech), Graldine Gurin-Peyrou (Director of Marketing and Technical Support, Polypus Transfection), Naiara Tejados, Head of Marketing and Technology Development, VIVEBiotech) and Jeffery Hung (Independent Consultant)

Chapter 27 is an appendix, which provides tabulated data and numbers for all the figures in the report.

Chapter 28 is an appendix that provides the list of companies and organizations that have been mentioned in the report.Read the full report: https://www.reportlinker.com/p06323417/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application...

Recommendation and review posted by Bethany Smith

Ryan Cawood, chief scientific officer, WuXi Advanced Therapies.

Cawood began by noting that WuXi Advanced Therapies supports clients throughout their journeys toward developing cell and gene therapies. With the acquisition of the UK-based contract research organization (CRO) Oxgene and its adenoassociated virus (AAV) and lentivirus platforms (known as the TESSA and XLenti platforms/technologies, respectively), WuXi Advanced Therapies now can scale processes up to good manufacturing practice (GMP) manufacturing through to commercial supply.

After describing the work that takes place in different company locations, Cawood focused on the TESSA technology, which provides a plasmid-free alternative for large-scale clinical manufacturing. Most AAV manufacturing is based on plasmid transfection. Not only are such processes expensive, but transfection can occur only at a certain cell density. Adenovirus can be used to manufacture AAV, and such processes are easy to scale up. However, they yield as much adenovirus as AAV, creating major downstream purification issues and raising product-contamination concerns. The goal of the TESSA technology is to use an adenoviral vector to manufacture AAV without contaminating the preparation. In the early phase of the adenoviral life cycle, genes in the AAV helper plasmid are expressed by cells to manufacture both AAV and adenovirus. But in late phases of the cycle, those genes induce cells to make unwanted adenovirus. The TESSA technology is designed to close down all of those late genes.

Cawood described how TESSA technology regulates the promoter that drives expression of structural proteins. The adenovirus titer is determined by the promoter that it represses, so the more the virus tries to make itself, the more it cripples itself. WuXi Advanced Therapies is working on different models of the technology and now has made TESSA rep-cap genes for all of the main serotypes that people work with. Production titers for one particular construct yielded 1 1012 gc/mL. TESSA rep-cap 1, 2, 4, and 5 showed significant improvements in productivity per cell in a suspension-based process for all the serotypes that the company has worked on generally 10-fold more than what is produced using a plasmid system. He also described how data from a cell line developed at WuXi AppTec showed improvement in packaging efficiencies.

Cawood noted that the US Food and Drug Administration (FDA) is increasing pressure on manufacturers to ensure absence of residual contaminants and confirm efficacy. WuXi Advanced Therapies has tested a number of different serotypes and compared the ability of those AAV particles to infect cells with AAVs made from the plasmid process. He illustrated work showing that in some cases, the particles were >10-fold more infectious when produced by TESSA technology than when induced by the plasmid-based process. In an example of scaling up the TESSA technology for AAV6 to 50 L, every cell in the population contained the adenoviral DNA after three days. Around 3% of the particles were able to infect a cell compared with 0.5%0.7% with the plasmid system. Before any purification, 66% of full particles were obtained in the bioreactor. Following purification, the number came to about 102%, and using analytical ultracentrifugation yielded 94.4% full capsids of pure AAV. In an example of scaling up the technology for AAV2, the yields were lower than in the bioreactor but still 20 higher than what was produced by the plasmid-based equivalent. In an alternative model, the company introduced the AAV genome into the chromosome of an HEK293 cell line. The cells were infected with just one TESSA vector, after which AAV was removed with purification.

Other examples illustrated how the technology increased AAV particle yields for all serotypes tested. It increased particle infectivity for a number of the serotypes, is safe and efficient, and removes dependency on transfection. It allows for a number of different operations in bioreactors that simply cant be done in a transfection-based process.

Cawood concluded by noting that the company provides materials that clients can access through evaluation in their own laboratories. WuXi Advanced Therapies also can construct specific TESSA vectors for clients.

Find More OnlineWatch the complete presentation online at https://bioprocessintl.com/sponsored-content/the-future-of-aav-gene-therapy-is-scalable.

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The Future of AAV Gene Therapy Is Scalable - BioProcess Insider

Recommendation and review posted by Bethany Smith

A single star may adorn the Texas state flag but the life sciences ecosystem is comprised of a galaxy of brightly shining companies such as FUJIFILM Diosynth Biotechnologies, Taysha Gene Therapies and Veravas developing innovative new medicines, diagnostics and medical devices.

The growth of the quickly rising Lone Star Bio life sciences hub is fueled by financial support from the state government in the form of the multi-billion dollar Cancer Prevention Research Institute of Texas fund and the drug discovery research coming out of the states universities. Baylor College of Medicine and Texas A&M headline the list of academic powerhouses.

Thats where it all begins. Most research that results in drug discovery generally starts at universities, said Andrew Strong, a partner at Houston-based Hogan Lovells, a legal firm representing biotech clients across Texas and the United States, in an interview with BioSpace.

Barry Burgdorf, also an attorney with Hogan Lovells, echoed Strongs statement. There is a ton of great IP spinning out of the universities, he said. That research is fueling a number of startups across the state, as well as legacy pharma companies that are licensing the developmental programs. That, in turn, is strengthening the ecosystem, Burgdorf noted.

Although the slowing economy is reducing expenditures of venture capital, Burgdorf and Strong agreed that in Texas, there has been no slowdown of new technologies being developed in the universities.

Texas is still very focused on growth [and] recruiting companies, Burgdorf said.

Real estate prices are also attractive to companies hoping to set up shop in Texas, especially when compared to other major U.S. hubs such as Boston and San Francisco.

Lower Cost Real Estate

The per-square-foot cost for space in the Boston area is approximately $95.57, according to Pete Briskman, executive managing director and co-lead for JLLs Mid-Atlantic life sciences practice. In the Bay Area, the per-square-foot cost is $82.41. This compares to $22 per square foot In Houston. Briskman said the difference is critical to companies as it can help them build out space and hire new employees.

He said companies used to have a mantra that the real estate costs were less significant than the science. When it comes to a place like Texas where a company is saving tens of millions of dollars, however, it becomes a real consideration.

Strong agreed with that assessment. He relayed that the CEO of a Boston area biotech told him real estate needs were taking up seven percent of its annual budget. The money can go much farther in Texas, he said.

Strong pointed to the August decision of Cellipont Bioservices to relocate to Texas from San Diego. Cellipont, a cell therapy contract development and manufacturing organization, plans to build a 76,000-square-foot facility in the state.

Strong was the founding chief executive officer of Kalon Biotherapeutics, a startup biotech spun out of the A&M system. He sold the company to FUJIFILM.

Strong pointed to the significant investments FUJIFILM has made. Those expenses are having a positive ripple effect across the region. For every one of the 1,000 FUJIFILM jobs in Texas with a salary of more than $80,000, six additional jobs have been created because of these investments, he said.

FUJIFILMs Ever-Expanding Footprint

Since its 2014 arrival in Texas, FUJIFILM Diosynth Biotechnologies (FDB)s contract manufacturing operations in College Station have rapidly expanded and changed the landscape of the ecosystem across the Brazos Valley region. The Japan-based company has invested hundreds of millions of dollars into its facility, expanding its offerings to bolster the development of gene therapies. The College Station facility has become the largest single-use CDMO production campus in the United States.

In 2019, FUJIFILM established anew Gene Therapy Innovation Center. One year later, the federal government selected the College Station facility to support COVID-19 vaccine candidate manufacturing at its Flexible Biomanufacturing Facility.

In December 2021, FUJIFILM announced another investment to expand its services in Texas and in June, it provided additional finances to expand its continuous processing technologies.

Gerry Farrell, chief operating officer at FDB Texas, told BioSpace thecompany's expansions within Texas have been supported by a strong partnership with state and local governments, as well as the universities.

Tayshas Gene Therapies for Rare and Orphan Diseases

Dallas-based Taysha started the year with disappointing news from its experimental gene therapy for Sandhoff and Tay-Sachs diseases, two forms of GM2 gangliosidosis. A patient treated with TSHA-101, a bicistronic vector, died. However, the patient did not succumb to complications from the gene therapy. They died after contracting a hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection while being treated for COVID-19.

Although the death was related to the infection, the independent review board determined a review of the data was warranted.

In addition to TSHA-101, Taysha is also developing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations. In its quarterly financial report issued in August, the companyhighlighted positive momentum with its gene therapy for giant axonal neuropathy.

Taysha received orphan drug and rare pediatric disease designations from the FDA and orphan drug designation from the European Commission for TSHA-120, an AAV9 gene therapy. Data showed GAN patients treated with TSHA-120 have seen durable improvement and recoverability of sensory nerve amplitude potential (SNAP), a definitive clinical endpoint, the company noted in its announcement.

Taysha is also developing a gene therapy for Rett Syndrome. TSHA-102 is the first-and-only gene therapy in clinical development for Rett. It has also received orphan drug and rare pediatric disease designations from the FDA and has been granted orphan drug designation from the European Commission.

Veravas Antibody Detection Platform

Based in Austin, Veravas launched in 2017. The company has developed the VeraPrep Antibody Detection Platform. The platform uses proprietary magnetic beads to pre-analytically clean samples to remove problematic heterophilic and autoantibody interference, according to the company. The clean sample allows for better capture and measurement of targeted IgA, IgG and IgM immunoglobulins.

During the COVID-19 pandemic, Veravas used its platform to develop an antibody test for SARS-CoV-2.

Baylor Genetics' Pandemic Contributions

A pioneer in genetic testing, Houston-based Baylor Genetics offers a range of diagnostic sequencing and analysis. The company provides a full spectrum of cost-effective, genetic testing it claims leads to clinically relevant solutions.

Baylor offers whole exome and genome sequencing services that provide data for point mutations, insertions and deletions. Oncology testing can find mutation panels through next-generation sequencing.

The company also provides prenatal diagnostics, molecular diagnostics and cytogenetics.

During the height of the COVID-19 pandemic, Baylor launched a combination test for the SARS-CoV-2 virus, as well as influenza A and B.

Asuragen

Also based in Austin, molecular diagnostics company Asuragen, a Bio-Techne brand, provides streamlined solutions for genetics, oncology, controls and companion diagnostic needs.

One of Asuragens products is AmplideX, a genetic test for Fragile X syndrome, whichcauses mild to severe intellectual disability and is associated with autism spectrum disorder.

Beyond Fragile X, Asuragen offers a chronic myeloid leukemia monitoring kit.

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5 Life Science Companies Drive Innovation in Lone Star Bio - BioSpace

Recommendation and review posted by Bethany Smith

SHANGHAI, Sept. 29, 2022 /PRNewswire/ -- NeuShen Therapeutics, Inc., a biotechnology company focusing on developing innovative treatments for central nervous system (CNS) disorders with dual platforms of AAV-based gene therapy and small molecule discovery, announced today the closure of ~$20 million Series pre-A financing led by LAPAM, a China based venture capital. NeuShen was founded by a group of industrial executives who have extensive global experiences in central nervous system (CNS) drug development. The new capital will be used to expand the team and catalyze in-house CNS drug discovery in both the US and China.

"The successful completion of this fundraising is a testimony to our team's ability to accomplish CNS drug development and jump starts our discovery engine to build a pipeline with AAV-based gene therapy and small molecule programs," said Joan Shen, M.D., Ph.D., chief executive officer and founder of NeuShen. "CNS disease is an area with huge unmet needs. Our company has had a very clear goal from Day 1, which is to develop novel therapies to relieve the burdens of patients with CNS disorders. In the past few months, we have developed an achievable R&D strategy and built-up a substantial core team with experienced CNS drug hunters. Significant progress has been made in building the internal small molecule pipeline and new AAV gene therapy programs. In addition, multiple collaborations and partnerships have been discussed and established."

CNS disorders are increasingly recognized as major causes of death and disability worldwide while the diagnosis and treatments have largely lagged. Urgent measures are needed to tackle the growing challenges. "Bringing breakthroughs and learnings from other disease targets such as ophthalmology, oncology and hematology, we believe AAV-based gene therapy represents a new opportunity in the treatment of CNS disorders. My colleagues and I at Horae Gene Therapy Center are looking forward to working with Neushen to explore these treatment opportunities. The experiences of NeuShen team in neurosciences will be critical to make this happen," noted Dr. Guangping Gao, Professor, Director, Horae Gene Therapy Center, UMass Chan Medical School. The collaborations between NeuShen and UMass are currently in discussion, which will include multiple projects in CNS gene therapy.

"We are very excited to partner with NeuShen from the beginning. Lapam Capital has a strong commitment to healthcare, and we believe the CNS therapeutic area will attract more investment, considering the huge unmet needs and scientific advancements in the field. We highly value Dr. Joan Shen and her management team for their expertise. Lampam Capital is confident with NeuShen's ability to be a top player in developing innovative therapies for CNS diseases," said Mr. Zhihua Yu, Managing Director of Lapam Capital.

"Dr. Shen has assembled a pre-eminent group of scientists, clinicians and drug developers to build a global biotech developing novel medicines for unmet needs in CNS therapeutic areas. TTM Capital are excited to support Neushen to build its multi-modality pipelines to help patients worldwide," said Ms. Lilly Zhang, founding and managing partner at TTM Capital.

About NeuShen Therapeutics

NeuShen Therapeutics is a biotechnology company focusing on innovative drug research and development to address CNS disorders, applying dual research platforms, including AAV-based gene therapy and small molecule discovery. With operations both in Shanghai, China and Boston, MA, NeuShen has a world-class team and is honored to be advised by an outstanding Board of Directors and Scientific Advisory Board.

https://www.neushen.com/

About Lapam Capital

Headquartered in Beijing, Lapam Capital is a leading healthcare venture capital firm in China. Lapam is currently managing five RMB funds and one USD fund, with more than 10 billion RMB under management. Lapam Capital focuses on investments in early to middle stage fast-growing companies that have innovative pharmaceuticals and medical devices. It has invested in about 60 biopharmaceutical companies and 10 medical device companies to date, including Betta Pharma, RemeGen Co. Ltd., Clover Biopharmaceuticals, Yahong Meditech, Stemirna Therapeutics, Binhui Biotech, ImmuneOnco Biopharmaceuticals, Biostar Pharmaceuticals, Aibo Medical Robot Co. Ltd. and many other companies with great potential. Lapam Capital has a professional investment team with more than 20 years' international and domestic biopharmaceutical industry R&D and management experience and can provide comprehensive value-added supports for the invested companies.

About TTM Capital

TTM Capital is an investment firm that specializes in China and worldwide healthcare industry. We focus on early and growth stage companies including pharmaceutical, biotech and medical technology sub-sectors.

TTM Capital's team consists of experienced global vision investment professionals with extensive industry experience, who work together to achieve superior and consistent returns for the firm's investors. We are committed to accumulating industry experience over time, with the aim to develop an ecosystem of expertise to create transformative healthcare businesses.

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NeuShen Therapeutics Closes Pre-A Financing with ~$20M - BioSpace

Recommendation and review posted by Bethany Smith

Duchenne muscular dystrophy (DMD) was first described by the French neurologist Guillaume Benjamin Amand Duchenne in the 1860s, though it took until 1986 for researchers to identify a particular gene flaw that leads to the condition. The identification of the dystrophin gene by Louis Kunkel and Jerry Louis opened the door for disease-modifying therapies such as exon-skipping, stop codon readthrough, gene therapy, and CRISPR/cas9 mediated gene editing that focus in on dystrophin restoration.

Currently, there are 4 drugs approved in the United States for mutations amenable to skipping of exons 51, 53, and 45, which are applicable to about 30% of patients total with DMD. Each of these were approved through the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases. At the recently concluded 2022 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) annual meeting, September 21-24, in Nashville, Tennessee, Emma Ciafaloni, MD, gave the Reiner Lecture to a crowd of a few hundred clinicians, highlighting new treatments for DMD.

In her talk, she summarized the expanding pipeline of agents for DMD, how each differs mechanistically, and whether any are more advantageous than another. Ciafaloni, a professor of neurology and pediatrics at the University of Rochester Medical Center, also discussed how to translate new treatments from trials to clinics, the need to improve clinical trial design and process, and how researchers can build on previous successes. Prior to her presentation, as part of a new NeuroVoices, Ciafaloni provided commentary on several topics regarding the DMD pipeline, including the differences and advantages each approach brings, as well as ways to overcome complexities with conducting clinical trials.

Emma Ciafaloni, MD: The exciting research development in the field of Duchenne muscular dystrophy is extraordinary. Many years after understanding the pathophysiology of Duchennewhich the gene wasnt discovered until the late 1980sall that knowledge is finally paying off and opening a window on therapeutic strategies that have to do with disease-modifying gene editing. There are many different approaches now, some like exon skipping, which are already used in the clinics. Some are different stages of development, such as gene therapy in phase three trials. I would be surprised if we didnt have a gene therapy drug in the clinic in the near future. And then CRISPR, which has not been used yet in humans, but has made major milestones and proof of concept in animal models that are highly promising. These are all strategies that are advancing very rapidly, I think that the field is moving much faster than in the past because of the collaboration between pharma and academia, and the patients and the families. There are many clinical trials in Duchenne, and it's a very exciting time.

Also, there has never been a time before in muscular dystrophies in general, not just Duchenne, where there were so many different, new ideas, as well as old ideas that finally started working in humans. The second part of my talk briefly covered other treatments, ideas and strategies that are not directed to restoration of dystrophin. They're not genetic treatments, but they work more on the downstream pathology of muscle degeneration into Duchenne, like the fibrosis, inflammation, and regeneration. There are some interesting drugs out there, probably a few that are going to be approved soon. We're looking at probably a multifactorial type of treatment, it may be a combination treatment. It's never been a richer time in terms of treatments for Duchenne. Also, it's exciting because some of the lessons learn, for example, with the genetic treatments, are extremely helpful for the larger field of neuromuscular diseases and even neurology. The learning has been fantastic.

With spinal muscular atrophy leading the way, we're moving into more muscle-based diseases [with gene therapy], but the lessons learned are still very valuable. Additionally, we have seen this collaboration between different sponsors, pharmaceuticals, and academias to share the learning, because that's just going to help move things faster and better and in a safer way. That is a positive phenomenon that is unprecedented, and it's helping to accelerate the science in a safe and effective way.

There are still many questions that remain. All these genetic modification approaches have been exon skipping, or gene therapy replacement. They don't replace the full-length dystrophin because it's a very large gene. It's a biologically modified type of dystrophin, so there is no doubt that it will have a profound benefit, but I think that there is plenty of room for improvement. Obviously, gene therapy is not approved yet, so remains to be seen in terms of clinical improvement. But even in the exon skipping, I think that we're going to see much more exciting next generation, exon-skipping that people are currently working on very hard on. The field of science and medicine always evolves. What we have now is only going to be much better down the road in a few years. I have no doubt, and the community of Duchenne is working very hard to make even the drugs that we have now, better.

Sometimes, for the more general neurologist or certainly for the general public, it's important to remember that when we talk about Duchenne muscular dystrophy, or many of our neuromuscular diseases that we discuss here at AANEM, these are also rare diseases. The definition from the FDA for a rare disease is less than 200,000 total patients in the United States. For Duchenne, for example, we're talking about maybe around 12,000 patients. This is not [multiple sclerosis], or Parkinson disease or Alzheimer disease. There are challenges in clinical trial designs that are unique, and they need to be understood. Some of the accelerated approval for some of these drugs is part of that challenge and difference. For example, especially with the genetic approach, some of these genetic approaches like exon skipping, only target a specific mutation in maybe 10% to 13% of patients. Now you're taking a subgroup of an ultra-rare disease that is only 10% of that population. Then you need to run clinical trials that are going to have a chance to prove a difference, and so, you restrict the inclusion criteria to a specific age. Then you're really challenged to find enough patients to do well in a placebo-controlled trial. It's important to keep that in mind that there is plenty of room for improvement in making our rare disease clinical trial design more effective, less time consuming for patients, and improving the approval path.

I also want to say that in Duchenne, the amount of data that has been produced in the past several years in terms of motor endpoints, natural history, the six-minute walk test, the North Star [Ambulatory Assessment], etc. These outcome measure prospective cohorts have been incredibly invaluable. This is just to recognize the incredible amount of work that researchers and families and patients have done in the past several years that is helping the field immensely. We are at a different time, its an exhilarating, exciting time. I think that the community of rare diseases like Duchenne have been incredibly, hard-working in a good, cohesive way to advance the field forward, which is very refreshing.

Transcript edited for clarity. Click here for more NeuroVoices.

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NeuroVoices: Emma Ciafaloni, MD, on the Vast Expansion of Innovative Approaches to Duchenne Muscular Dystrophy - Neurology Live

Recommendation and review posted by Bethany Smith

Presently, there are no effective therapies for spinal cord damage. Physical therapy can help patients regain some movement, but the outcomes are heavily limited in severe cases due to the inability of spinal neurons to repair organically after injury.

However, in a study published in the journal PLOS Biology, Simone Di Giovanni and his research team at Imperial College London (United Kingdom) have looked to change this roadblock. The team displayed how weekly treatments that contain an epigenetic activator can help the regeneration of motor and sensory neurons in the spinal cord, when administered to mice 12 weeks after a serious injury.

Using a small molecule known asTTK21, theresearchers were able to trigger genetic programming that stimulates axon regeneration in neurons. TTK21 affects gene epigenetics via activating the CBP/p300 family of coactivator proteins.

TTK21 therapy was investigated in micemodels where the specimen had experiencedsevere spinal cord damage. The mice were raised in an enriching environment that allowed them to be physically active, as is recommended for human patients.

The treatment started 12 weeks after the severe spinal cord damage and lasted 10 weeks. Researchers discovered numerous improvements following TTK21 therapy when compared to the control treatment. Increased neuron sprouting in the spinal cord was the most noticeable effect. The researchers additionally found that motor axon retraction above the site of injury stopped and sensory axon development sharply increased. These changes were most likely caused by the observed increase in gene expression associated with regeneration. The next stage will be to further increase these effects to stimulate the regenerated axons to reconnect with the rest of the nervous system, so that the mice regain the capability to may move freely again.

Di Giovanni goes on to emphasize, This work shows that a drug called TTK21 that is administered systemically once/week after a chronic spinal cord injury in animals can promote neuronal regrowth and an increase in synapses that are needed for neuronal transmission. This is important because chronic spinal cord injury is a condition without a cure where neuronal regrowth and repair fail. We are now exploring the combination of this drug with strategies that bridge the spinal cord gap such as biomaterials as possible avenues to improve disability in spinal cord injury patients.

Press release:https://www.eurekalert.org/news-releases/964425

See more here:
Epigenetic therapy promotes spinal cord regeneration in mice following injury - RegMedNet

Recommendation and review posted by Bethany Smith

NORTH BETHESDA, Md., Sept. 29, 2022 /PRNewswire/ -- The Foundation for the National Institutes of Health (FNIH) and the National Heart Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH) are launching a new partnership to investigate the syndrome of heart failure with preserved ejection fraction (HFpEF). Developing precision treatment strategies for HFpEF is more critical than ever, as the world's population continues to live longer, resulting in an increase in HFpEF cases. Utilizing cutting edge technologies, including digital measurements and artificial intelligence analytic methods, the Accelerating Medicines Partnership Heart Failure (AMP HF) Program is designed to find novel proteins or genes that could mitigate this disease when altered by therapeutics.

"We know that treatments that target the biological changes that drive disease are often most effective, but the challenge faced by researchers is finding the right targets," said Lawrence A. Tabak, D.D.S., Ph.D., who is performing the duties of the NIH director. "AMP Heart Failure aims to improve the odds of hitting the mark earlier and faster."

Although compelling progress has been made in the treatment of many forms of heart disease, death due to heart failure continues to rise nationally. The AMP HF Program aims to alter the landscape of heart failure treatment, improving the outlook for millions of patients around the world.

"The promise of precision treatments for heart failure is that we will have the opportunity to diagnose individuals much earlier and intervene, changing the course of this disease," said NHLBI Director Dr. Gary H. Gibbons. "The AMP Heart Failure program -- and the high caliber of the partnership at its core -- will help us better understand and treat this common syndrome with the goal of ultimately benefitting millions."

In the United States, heart failure directly contributes to about 45% of all cardiovascular disease deaths.1 HFpEF is a common form of heart failure in which the ejection fractionthe percentage of blood ejected from the left ventricle with each heartbeatis within the normal range. HFpEF is difficult to detect, because the left ventricle appears to be functioning normally, and is often deadly, with a five-year survival rate of just 35-40%. In addition to a high risk for mortality, patients with HFpEF live with declining quality of life and poor capacity to perform tasks of daily living.

The AMP HF Program, a public-private partnership facilitated by the FNIH, will advance our understanding of heart failure with preserved ejection fraction using two complementary and integrated research components: analyzing existing HFpEF datasets, sourced from public and private sector funded studies, and initiating a new clinical trial to confirm retrospective findings in an observational cohort with a goal to develop a framework for new precision treatments.

"HFpEF is clearly a major cause of heart failure hospitalizations and diminished quality of life for older patients. Up until now, developing effective therapeutic strategies to identify and treat HFpEF has eluded us. Through AMP HF, we will harness the valuable perspectives and expertise that collaborations bring to biomedical research, paving the way for a more hopeful outlook," said Dr. Julie Gerberding, Chief Executive Officer at the FNIH.

"Roughly half of all heart failure patients suffer from HFpEF. Understanding what it is, when it happens, and how to treat it remains the single largest unmet need in cardiovascular health.2 The AMP HF Program aims to close this gap in understanding, and ultimately improve the lives of patients everywhere," said Dr. Norman Stockbridge, Director of the Division of Cardiology and Nephrology at the Office of Cardiology, Hematology, Endocrinology and Nephrology at the U.S. Food and Drug Administration (FDA).

AMP Heart Failure is the latest initiative to emerge from the AMP Program, a set of public-private collaborations that coalesce the collective knowledge of the NIH, the U.S. FDA, the biotech and pharmaceutical industry, and patient organizations to speed drug development across different diseases. AMP HF brings together the resources of 8 partner organizations spanning the public and private sectors, with combined commitments totaling over $37 million. The FNIH will provide project management for the effort over the next 5 years.

NIH Institutes and Centers involved include:National Heart, Lung, and Blood Institute

Private partners include:American Society of Echocardiography (ASE)Bayer USCytokinetics, Inc.Ionis Pharmaceuticals, Inc.Novartis AGUltromics

Support also provided by the American Heart Association

For more information about the program, click here. To read what our partners and supporters are saying about the program, click here.

About the Accelerating Medicines Partnership Program: AMP Heart Failure joins other AMP programs expediting discovery around Alzheimer's disease, Parkinson's disease, Schizophrenia, Rheumatoid Arthritis and Lupus, Type II Diabetes, Common Metabolic Diseases, Autoimmune and Immune-Mediated Diseases, and the Bespoke Gene Therapy Consortium, all coordinated by the FNIH since the 2014 launch of the large-scale initiative. The AMP partnerships use cutting-edge scientific approaches to bring new medicines to patients by enhancing validation of novel, clinically relevant therapeutic targets and biomarkers. To learn more about AMP, visithttps://fnih.org/AMP.

About the Foundation for the National Institutes of Health: The Foundation for the National Institutes of Health creates and manages alliances with public and private institutions to support the NIH, the world's premier medical research agency. FNIH works with its partners to accelerate biomedical advances and therapies targeting diseases in the United States and across the globe. The FNIH organizes and administers research projects; supports education and training of new researchers; and holds educational events focused on areas of unmet medical need worldwide. Established by Congress in 1990, the FNIH is a not-for-profit 501(c)(3) charitable organization. For additional information about the FNIH, please visit https://fnih.org.

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

ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.

CONTACT:Katherine Thompson[emailprotected]

SOURCE Foundation For The National Institutes of Health, Inc.

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The FNIH Announces New Research Initiative to Identify More Precise Treatment Strategies for Patients Suffering from Heart Failure - PR Newswire

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PITTSBURGH A collaborative group of neuroscientists from the University of Pittsburgh School of Medicine and Carnegie Mellon University received a $6.8 million grant from the National Institutes of Health Brain Research Through Advancing Innovative Neurotechologies (BRAIN) Initiative to create an ultra-high resolution molecular atlas of the brain and develop brain cell type-specific strategies for effective and precise gene delivery.

The research will leverage genetic information resolved with single-cell precision to establish a comprehensive database of cell types and neural circuits comprising the brains cognitive and reward systems. In combination with ultra-high-resolution magnetic resonance imaging (MRI), the researchers intend to build brain atlases of marmosets and macaque monkeys and make them available to other neuroscientists across the world, free of charge.

This award enables cross-disciplinary collaboration between experts in neural imaging, gene therapy, machine learning, and molecular biology to advance our understanding of single-cell level organization of the brains essential systems, said project principal investigator Dr. William Stauffer, assistant professor of neurobiology at Pitt. We hope this unmatched degree of precision will eventually pave the way for the development of effective and precise gene editing technologies that might revolutionize treatment of previously fatal diseases, such as Alzheimers or Parkinsons.

The recently launched BioForge Initiative, backed by Pitt Senior Vice Chancellor for the Health Sciences, Dr. Anantha Shekhar, will be used to advance the wide-scale production and commercialization of the gene delivery vectors identified with the grant support.

We are excited that the services of a state-of-the-art biomanufacturing facility will soon be available in Pittsburgh to help make the lofty goal of delivering new and improved medical treatments for brain disorders a reality, said Shekhar. It feels very special to participate in a program that will not only bring life-saving treatments to our patients but also facilitate the dissemination of Pitt-developed technologies to research labs around the world and take a big step toward creating products with economic impact on the region.

The BRAIN Initiative was announced in 2013 to deepen understanding of the inner workings of the human mind and over the years has grown to prioritize the expansion of molecular cell-type profiling and data analysis, enabling genetic and non-genetic access to cell types across multiple species. The multi-year NIH grant was awarded as part of the Armamentarium for Precision Brain Cell Access, a large-scale NIH BRAIN Initiative project.

Delivery technologies for specific brain cell types are revolutionizing experimental neuroscience by allowing researchers to probe the cells and circuits underlying complex behaviors, said Dr. John Ngai, director of the NIH BRAIN Initiative. An expanded toolkit of precision brain cell access tools supported by the first phase of the Armamentarium project could ultimately inform cell- and circuit-specific therapies for human patients, for example, those with epilepsy, neurodevelopmental diseases, or mood disorders.

Projects like the one led by Stauffer, who is interested in defining how different cell types contribute to behavior, as well as investigating cell type-specific disease processes, are essential to the Initiatives mission. Stauffer and his close collaborators, Leah Byrne, Ph.D., assistant professor of ophthalmology at Pitt, and Andreas Pfenning, Ph.D., assistant professor of computational biology at CMU, were awarded a BRAIN Initiative grant in 2018 to begin defining the molecular profiles of different neuron types.

Even a small piece of brain tissue contains dozens of different subtypes of neurons, each performing different functions during different behaviors, said Pfenning, who is a part of CMUs Neuroscience Institute. The ability to target these populations using viruses could accelerate basic research and also pave the way for targeted therapeutics.

Pfennings group will use custom-made machine learning models and evolutionary theory to identify sequences that are most likely to label subpopulations of neurons. His laboratory will also test the ability of those sequences to target specific cell types in the mouse brain.

Further building on the molecular profiling data, scientists at Pitts Brain Institute intend to identify cell type-specific drivers of gene expression in the forebrain and the frontal lobe and develop ready-to-use, specific and efficient gene delivery vectors, including adeno-associated viruses (AAVs). To develop novel AAVs, they will use scAAVengr, the single cell AAV engineering pipeline developed by Byrne. The team will combine scAAVengr-optimized AAV viral shells with newly identified cell type-specific enhancers, and the combination of these elements will generate viral vectors capable of delivering highly efficient and cell type-specific gene therapies. Afonso Silva, Ph.D., professor of neurobiology who holds an endowed chair in translational neuroimaging at Pitt and also a member of the Brain Institute, joins Stauffer, Byrne and Pfenning on the project team. The Silva lab will create an ultra-high resolution MRI atlas of the rhesus monkey brain. That MRI-based atlas will provide the framework for detailing how viral vector expression is controlled in a brain-wide fashion.

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Pittsburgh Project to Pave Way for Technology to Revolutionize Treatment of Fatal Brain Diseases - timesobserver.com

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Healthcare Contract Research Outsourcing Market: Introduction

According to the report, the global healthcare contract research outsourcing market was valued at US$ 38.04 Bn in 2020 and is projected to expand at a CAGR of 6.6% from 2021 to 2028. A contract research organization (CRO) is a company that provides clinical trial management services for the pharmaceutical, biotech, and medical device companies.

Read Report Overview https://www.transparencymarketresearch.com/hcro-market.html

The different types of CRO services are regulatory affairs, site selection & activation, recruitment support, clinical monitoring, data management, trial logistics, pharmacovigilance, biostatistics, medical writing, and project management.

Broad and Expanding Product Pipeline: Key Driver

Rise in demand for new drug and novel therapies for treatment of life-threatening diseases such as cancer & immunological disorders and evolving research & development process have led to an increase in the number of compounds in pipeline in the last decade.

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According to the Congressional Budget Office (CBO), in 2019, there was an increase of 60% of new drugs approved for sale compared to that in 2018.

In the past few years, development of biosimilar products, cellular therapy, and immune-biological has contributed toward the advancing pipeline and need of stringent clinical trials. This drives the global healthcare contract research outsourcing market.

Funding for Emerging Healthcare Companies: Major Driver

Increase in funding for small to mid-sized pharmaceutical, biotechnology, and medical devices companies has induced large companies to opt for CRO services with focus on niche market. Currently, expenditure in the biotechnology sector is the highest due to recent advancements in research on monoclonal antibodies, immunotherapy, gene therapy, and cancer vaccines. Small pharmaceutical companies focus on advancement in product development through CRO services due to rise in demand for innovative products and patent expiration.

The need to improve adoption and value proposition in the market has induced medical devices companies to outsource clinical trial services.

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Clinical Trial Services Segment to Dominate Global Market

The clinical trial services segment is expected to dominate the global market during the forecast period. Optimization of R&D costs and shorter drug development timeline resulting from outsourcing of clinical trial services to CROs are likely to contribute to the segments large market share in the near future. However, the segment is projected to lose market share to regulatory services and clinical data management & biometrics segments by 2028 due to stringent regulatory guidelines.

North America to Lead Healthcare Contract Research Outsourcing Market

In terms of region, the global healthcare contract research outsourcing market has been segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America accounted for a major share of 46.59% of the global market in 2020. The market in the region is estimated to reach US$ 27.19 Bn in 2028, expanding at a CAGR of 5.6% from 2021 to 2028. This can be attributed to the presence of world renowned research experts in CNS, rare disease, oncology, immunology, and stem cells, advanced infrastructure of clinical research sites, and effective government incentive programs.

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Key Players in Global Market

Major players operating in the global healthcare contract research outsourcing market include Syneos Health, PAREXEL International, ICON plc, PRA Health Sciences, Inc., Charles River, Laboratory Corporation of America Holdings (Covance), IQVIA, Medpace, and Pharmaceutical Product Development, LLC.

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Healthcare Contract Research Outsourcing Market to Reach the Value of US$ 63.09 Bn by 2028 - Digital Journal

Recommendation and review posted by Bethany Smith

Only companies that have strong platforms will be able to meet specific timelines, says Bristol-Myers Squibbs Jim Xu.

As COVID-19 transitions into an endemic, delegates at Biotech Week Boston discussed the challenges concerning the upstream process development process. Jianlin (Jim) Xu, scientific director, biologics development at BMS, explained told a packed room that monoclonal antibodies (mAbs) are difficult to produce with complex glycosylation profile, process related impurities, and amino acid oxidation.

Xu also outlined other challenges, which included bioreactor operation scalability, upstream processes lasting over a month for one run from vial thaw to harvest, competitive development of similar mAb products in the industry, and the length of time it takes to create stable monoclonal CHO lines with desirable characteristics.

To solve said challenges, Xu maintained that a strong platform is the number one solution, and that a robust platform can help a firm meet particular timelines. He said that a platform approach has numerous benefits, from decreasing costs, shortening development timelines, and making it possible to have a robust production process from clinical supply through to commercial use.

Xu claimed that in order to develop upstream platform technology, the individual and/or company must be able to demonstrate the benefit and proof-of-concept of an upstream technology in the first instance. Additionally, he told delegates that the technology should be successfully applied to various mAb products, and this will provide a good starting point for future mAb manufacture process development.

In other platform related news at Biotech Week Boston, PerkinElmer launched its Cellaca PLX system, saying that it is the cell analysis solution to streamline cell and gene therapy research and production.

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BMS: Platforms are pivotal to pandemic speed - BioProcess Insider - BioProcess Insider

Recommendation and review posted by Bethany Smith