Category Archives: Stem Cells

AgeX Therapeutics, Inc. ("AgeX"; NYSE American: AGE), a biotechnology company focused on developing therapeutics for human aging and regeneration, announced a research collaboration with the University of California, Irvine (UCI) using AgeXs PureStem technology to derive neural stem cells, with the goal of developing cellular therapies to treat neurological disorders and diseases for which there are no cures. The collaborations initial R&D work, expected to take approximately one year, will be conducted in the UCI laboratory of Leslie Thompson, PhD, Chancellors Professor of Psychiatry & Human Behavior and Neurobiology & Behavior, a leading researcher in the field of Huntingtons disease and other neurological disorders, under a Sponsored Research Agreement handled by the Industry Sponsored Research team at UCI Beall Applied Innovation. The initial focus will be on Huntingtons disease, while other potential targets may include Parkinsons, Alzheimers, and stroke.

The primary goal of the research will be to develop a robust method of deriving neural stem cells from pluripotent stem cells in sufficient quantity and with sufficient purity and identity for use in cell-based therapy. Professor Thompsons laboratory has already accumulated safety and efficacy animal data that may support an IND submission to the FDA as early as 2021 for the commencement of clinical trials to treat Huntingtons disease.

"We look forward to utilizing AgeXs cell derivation and manufacturing PureStem technology, with its many potential advantages, including industrial scalable manufacturing, lower cost of goods, and clonal cells with high purity and identity. Our goal is to have an improved neural stem cell production method ready within a year to move into clinical development," said Professor Thompson.

"We are absolutely delighted to start this exciting collaboration with Professor Thompson, who has worked tirelessly over her career to develop a neural stem cell product candidate for Huntingtons disease and who has already generated preclinical animal data that may support the initiation of clinical studies," said Dr. Nafees Malik, Chief Operating Officer of AgeX. "Moreover, we are very excited to be entering the field of neurology, which has huge clinical and commercial potential. Neural stem cells may be very useful in other neurological disorders that are common in aging demographics, such as Parkinsons, Alzheimers and stroke."

"This is an example of the kind of collaboration we will be seeking under our newly-unveiled collaboration and licensing strategy, which is to run parallel to our in-house product development," said Dr. Greg Bailey, Chair of AgeX. "We will be collaborating with a world leader in their field on a research project which is close to the clinic."

The collaboration includes an opportunity for AgeX to organize a company to be jointly owned with Professor Thompson and other researchers to pursue clinical development and commercialization of cell therapies derived using licensed inventions arising from the research program, as well as certain patent pending technology for neural stem cell derivation, and certain technical data, including animal data, to support IND submissions.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

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For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Further, in the case of AgeXs new neural stem cell program there can be no assurance that: (i) any new cell derivation methods will be invented in the sponsored research program, (ii) any derivation methods that may be developed will be sufficient to derive neural stem cells in quantities and of purity suitable for clinical use and commercialization, (iii) that any new inventions or existing technology will be licensed on commercially favorable terms, (iv) that any neural stem cells derived for therapeutic use will be shown to be safe and effective in clinical trials, and (v) that any neural stem cells derived for therapeutic use will be successfully commercialized even if clinical trials are successful. Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200203005261/en/

Contacts

Media Contact for AgeX: Bill Douglass Gotham Communications, LLC bill@gothamcomm.com (646) 504-0890

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AgeX Therapeutics to Collaborate with University of California, Irvine on Neural Stem Cell Research Program for Huntingtons Disease and Other...

Jasper Therapeutics has raised an additional $14.1 million to finance the development of its investigational conditioning therapy, JSP191, as a safer and more effective treatment to prepare patients for stem cell transplant.

The investment, led by Roche Venture Fund and sponsored by other investors, increases the company financing to more than $50 million, the company stated in a press release.

JSP191 (formerly AMG191) is designed to replace toxic chemotherapy and radiation therapy as conditioning regimens to prepare patients for stem cell transplants in a safer and more effective way.

A stem cell transplant involves two main steps: conditioning, which consists of removing the patients diseased or damaged hematopoietic stem cells (from which all blood and immune cells develop) and replacing them with healthy stem cells that will reconstitute healthy bone marrow.

During a stem cell transplant, patients can either receive healthy stem cells from a donor, known as allogeneic transplants, or the patients own stem cells corrected by gene therapy.This type of genetically-corrected autologous stem cell transplant can be used to cure genetic diseases caused by mutations in a single gene, as is the case in sickle cell disease.

Conditioning plays an important part in the success of a transplant. It clears the patients own ill cells in the bone marrow, leaving space for the transplanted healthy stem cells to effectively engraft and replace them. However, current regimens rely on toxic treatments using radiation or chemotherapy that damage cellular DNA and lead to short- and long-term side effects, including damage to organs and prolonged hospitalization. For this reason, many patients cannot undergo a stem cell transplant, so safer and more effective approaches are needed to bring this potentially curative treatment to more patients.

Jasper Therapeutics, joined by theCalifornia Institute for Regenerative Medicine, is developing JSP191 to meet that need.

JSP191 isa humanized antibody that binds to CD117 (also known as c-Kit), a protein receptor thats expressed on the surface of hematopoietic stem cells and required for stem cells to survive. By targeting CD117, JSP191 disrupts signals that are essential for these cells to survive, leading to their death.

Early studies have shown that JSP191 is able to inhibit the formation of new human blood cells and safely eliminates hematopoietic stem cells, both in cells cultured in the lab and in animal models.

Based on these promising data, Jasper advanced the agent to the first clinical tests in humans. According to the company, JSP191 has been evaluated in more than 80 healthy volunteers and patients.

A Phase 1/2 study (NCT02963064) is ongoing in patients with severe combined immunodeficiency (SCID), a rare disorder in which infection-fighting immune cells called lymphocytes fail to work. The trial is testing pre-treatment with JSP191 as a single therapy in SCID patients undergoing a donor stem cell transplant, the only curative treatment for the disease.

Initial results from the study presented at the 2019 American Society of Hematology Annual Meeting in December showed that JSP191 safely cleared patients bone marrow cells and facilitated the engraftment of donor stem cells, with minimal to no toxicity in four of six patients evaluated to date.

These results suggest that antibody conditioning for [hematopoietic cell transplantation] may be preferable to traditional chemo(radio)therapy conditioning, especially in patients with non-malignant diseases and/or increased risk of toxicities due to such agents, such as certain forms of SCID, Fanconi anemia and sickle cell disease, the researchers wrote.

Jasper Therapeutics plans to expand JSP191s clinical development beyond patients with SCID.

We expect to initiate clinical trials of JSP191 in 2020 to evaluate it as a conditioning agent in patients undergoing hematopoietic cell therapy for acute myeloid leukemia, myelodysplastic syndrome and Fanconi anemia, and IND [Investigational New Drug]-enabling studies for sickle cell disease and autoimmune indications, Judith Shizuru, MD, PhD, Jasper Therapeutics co-founder and member of the board of directors, said in a press release.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, during which she focused her research on molecular biology, epigenetics and infectious diseases.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Jasper Raises $50M for Its Stem Cell Transplant Conditioning Therapy - Sickle Cell Anemia News

DetailsCategory: DNA RNA and CellsPublished on Monday, 03 February 2020 15:31Hits: 134

License agreement to utilise Affimer proteins in next generation cell and gene therapies

CAMBRIDGE & WETHERBY, UK I February 03, 2020 I Avacta Group plc (AIM: AVCT), the developer of Affimer biotherapeutics and reagents, is pleased to announce that it has signed a collaboration and license agreement with AffyXell Therapeutics (AffyXell), the recently established joint venture with Daewoong Pharmaceutical (Daewoong), to develop Affimer proteins to be used by AffyXell for the generation of new cell and gene therapies.

Avacta and AffyXell will now work together to develop Affimer proteins against a range of targets which, when produced by mesenchymal stem cells (MSCs), are intended to inhibit inflammatory and autoimmune pathways and improve the overall efficacy of MSCs, creating a next generation of stem cell therapies.

The initial focus for AffyXell will be on inflammatory and autoimmune diseases. In the longer term, there is potential for AffyXell to address oncology uses for these Affimer-enabled cell and gene therapies.

Under the terms of the collaboration and license agreement, Avactas research and development costs associated with generation of the Affimer proteins will be funded by AffyXell. In addition, Avacta will retain the rights to commercialise the Affimer proteins outside of the field of cell therapies.

Dr Alastair Smith, Chief Executive Officer of Avacta Group, commented: The potential for AffyXells new class of cell therapies, which can be applied to a wide range of inflammatory and autoimmune diseases, is enormous. We are therefore very excited by the opportunity to be part of this new venture and to demonstrate the power of Affimer proteins in the field of engineered cell therapies.

Our objective is for these cell therapies to finally fully address diseases, such as inflammatory bowel diseases and multiple sclerosis, as well as other autoimmune diseases, such as chronic obstructive pulmonary disease. The potential to make a difference in the quality of life for millions of patients continues to inspires us, as we know it does our colleagues at Daewoong as well.

From a commercial perspective, the global stem cell market is expected to be worth USD16bn [1] by 2025. AffyXell has a unique opportunity to combine two world-class technologies, Avactas Affimer antibody mimetic platform and Daewoongs proprietary technology for generating off-the-shelf allogeneic MSC therapies to create the next generation of stem cell therapies. We believe that this has the potential to create substantial value for stakeholders in the near future.

Seng-ho Jeon, CEO of Daewoong Pharmaceutical, commented: We are very excited to contract this Collaboration and License Agreement followed by the successful establishment of an innovative joint venture, AffyXell. Daewoong and Avacta will make every effort to AffyXells growth and its development of distinguished and innovative cell and gene therapy treatment.

As announced at the J.P. Morgan Healthcare Conference this January, Daewoong has been inspired to partner global biotechnology companies like Avacta to create the best and the most comprehensive technology in new drug development field. Starting from our very first cell and gene therapy specialised joint company, we reaffirm our belief that our contract can not only further establish AffyXell to thrive as an leader of cell and gene therapy, but also, allow us to enhance quality of lives to those who suffer from rare and intractable conditions.

[1] https://www.grandviewresearch.com/press-release/global-stem-cells-market

SOURCE: Avacta

Excerpt from:
Avacta and Daewoong Agree Collaboration and License Agreement with AffyXell Therapeutics | DNA RNA and Cells | News Channels - PipelineReview.com

NASSAU, BAHAMAS The fate of Freeports pioneering stem cell therapy provider has now become the subject of legal dispute, with its principal creditor seeking the winding-up of the company.

According to court documents seen by Eyewitness News, Okyanos claims that damagessuffered toits nearly 15,000 sq feet rented Freeport facilities as a result of Hurricane Dorian in September 2019 left thepremisesunfit for use. As a consequence, all of thecompanysbusiness ceased. That claim however has beenfiercely disputed by its landlord First Commercial Ltd who is seeking to have its dispute arbitrated and block the removal of the companys equipment which could satisfy an award in those proceedings.

Okyanos is the first licensed stem cell therapy provider in The Bahamas. It has been treating patients with chronic diseases by using their own stem cells from fat tissue. It opened to patients in October 2014, providing treatment for those with autoimmune, orthopaedic, cardiovascular and neurological conditions.

According to details outlined in a wind-up petition by its primary creditor LS Enterprise Ltd, -a companyregisteredin the British Virgin Islands, Okyanos between August 2, 2017 and May 6 2019 entered into four loanfacilityagreementsfor working capital and generalcorporate amounting to some $15.9 million. Okyanos had ultimately sought additional loans from LS Enterprise Ltd following Hurricane Dorian however was informed that it was in default of its facility agreements having ceased to carry on its business and that all loans were immediately due and payable.

According to the wind-up petition an ex parte order obtained by Okyanoss landlord First Commercial Ltd had restrained the stem cell therapy provider fromremediatingthepremisesor removing its equipment. It is claimed that substantial amounts of water and wind had penetrated the companyspremises causing major property loss and damage.Further, a lack of consistent electricity and airconditioninghad caused mold contamination.

Thecompanyattemptedtomitigateloss and damage by movingequipmentto aclimatecontrolledstorage and and preparing thecompanysoperatingfacilitiesfor moldremediation butthat was hampered by the landlord whose servants oragentsinstructed thecompanys personnel to cease anddeistfrom such activities, LS Enterprise outlined in its petition.

It further noted that in a notice to the landlord dated October 25, 2019, Okyanos had exercised its right to terminate its leaseagreementwithin 60 daysfollowingthe storm due to the facilities being unfit for use or occupancy the hurricane. On October 30, First Commercial Ltd obtained an ex parte order restraining the company from anyfurtherremediation efforts as well as the removal or disposal of equipment, LS Enterprise has claimed. The company had filed for damages with its insurance provider but had not received the majority of any such insurance proceeds the petition station.

Accordingly it is claimed that the company has insufficient funds to secure a new lease forfacilitiesto treatpatients,arbitratewith landlord torecover itsequipment and supplies, or conduct amarketingcampaignto attract patients to The Bahamas.

However, an affidavit but attorney Andre Jay Feldman, president and a member of the Board of Directors of First Commercial Centre seeking to restrain Okyanos from breaching its lease disputed the companys damages claim.

According to Mr Feldmans affidavit, a copy of which was seen by Eyewitness News, he inspected thepremiseson September 4 and beyond some minimal damage due to a single window having opened in one of theclinicalrooms, there was no damagewhatsoeverthroughoutthe clinic and nosignificantdamage referred to by the company. Mr Feldman claimed that Steve Araiza, a Houston based attorney for Okyanos had written him on September 26, 2019 seeking a rent abatement and to end theleaseon thegrounds of hurricane damage.

According to Mr Feldman, flood water had entered parts of theground flooroccupiedby CIBC and on September 15, 2019, the bank had not onlyremediatedany damage but hadreopenedfor business. He further contended that on September 4, Okyanos had allowed the Rand Memorial Hospital to use itsfacilities for urgent patient care. Mr Feldman contends that there was no damage to make the facilities unfit for use. According to Mr Feldman, since September 1 there had been no payment towards rent or electricity with respect to air-conditioning consumption. He asserted that if Okyanos is allowed to remove its assets they would be shipped out of country and would not be available to satisfy any award from arbitration. The landlord is claiming it is owed $1,768,000 and that while it plans to claim on a $890,000 loss of rentinsurancethere is no guarantee that can be recovered.

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Freeport stem cell therapy provider Okyanos in wind-up petition battle - EyeWitness News

Kenneth Pettine's stem cell product to treat OA was tested on retired Navy SEALs

FORT COLLINS, CO / ACCESSWIRE / February 3, 2020 / Kenneth Pettine is proud to announce that his revolutionary mesenchymal stem cell product to treat osteoarthritis was recently tested on 33 former Navy SEALs (one is a medal of honor recipient).

Kenneth Pettine is co-founder of Paisley Laboratories and a co-developer of a bone marrow-derived mesenchymal stem cell active growth factor and exosome product that is anticipated to revolutionize regenerative medicine.

In this study, Extracellular Vesicle Isolate Product (EVIP) was injected into 33 retired Navy SEALs to assist with knee, shoulder, elbow, ankle, and wrist osteoarthritis. At three-month follow-up, the injection appeared both safe and effective, with improvements ranging from 40% to as high as 98%. The average improvement is over 70%.

"This is extremely promising and we are motivated to continue our clinical studies to improve the quality of life for patients," says Kenneth Pettine.

Kenneth Pettine notes in his study that over 50 million Americans require daily treatment for disability and pain associated with OA. Every year, over one million total hip and knee replacements are performed in the U.S. with direct costs of over $30 billion and indirect costs of over $200 billion, with these numbers expected to double in the next three years.

In addition to this trial, Kenneth Pettine has three additional clinical studies planned to evaluate his stem cell products to treat erectile dysfunction, chronic obstructive pulmonary disease (COPD), and chronic lower back pain from painful discs.

For more information, visit https://www.kenneth-pettine.com/

About Kenneth Pettine

Dr. Kenneth Pettine is a serial entrepreneur and published clinical researcher with over 30 years of experience as an orthopedic surgeon. He holds a medical degree from the University of Colorado School of Medicine and completed his master's degree in orthopedic surgery and residency at the Mayo Clinic in Rochester, Minnesota.

In 1991, Dr. Pettine founded the Rocky Mountain Associates in Orthopedic Medicine. Kenneth Pettine is also the founder of Paisley Laboratories and the co-founder of the Society for Ambulatory Spine Surgery. In addition, he co-invented the Prestige cervical artificial disc and the Maverick Artificial Disc. Dr. Pettine is the principal investigator of 18 FDA studies involving non-fusion implants, biologics, and stem cells. He holds the only two issued U.S. patents for performing stem cell joint and spinal injections and currently has 21 additional patents pending for bone marrow derived mesenchymal stem cell applications. Kenneth Pettine is also a philanthropist and currently has a scholarship program underway to help students fund their education.

For more information, visit https://www.kenneth-pettine.com/ or https://www.kennethpettinescholarship.com/

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info@kenneth-pettine.com

https://www.kenneth-pettine.com/

SOURCE: Kenneth Pettine

View source version on accesswire.com: https://www.accesswire.com/574987/Dr-Kenneth-Pettine-Announces-Verification-of-Clinical-Safety-Trial

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Dr. Kenneth Pettine Announces Verification of Clinical Safety Trial - Yahoo Finance

It involves milking snake venom by hand and injecting it into horses or other animals in small doses to evoke an immune response. The animal's blood is drawn and purified to obtain antibodies that act against the venom.

Producing antivenom in this way can get messy, not to mention dangerous. The process is error prone, laborious and the finished serum can result in serious side effects.

Experts have long called for better ways to treat snake bites, which kill some 200 people a day.

Now -- finally -- scientists are applying stem cell research and genome mapping to this long-ignored field of research. They hope it will bring antivenom production into the 21st Century and ultimately save thousands, if not hundreds of thousands, of lives each year.

Researchers in the Netherlands have created venom-producing glands from the Cape Coral Snake and eight other snake species in the lab, using stem cells. The toxins produced by the miniature 3-D replicas of snake glands are all but identical to the snake's venom, the team announced Thursday.

"They've really moved the game on," said Nick Cammack, head of the snakebite team at UK medical research charity Wellcome. "These are massive developments because it's bringing 2020 science into a field that's been neglected."

Hans Clevers, the principal investigator at the Hubrecht Institute for Developmental Biology and Stem Cell Research in Utrecht, never expected to be using his lab to make snake venom.

So why did he decide to culture a snake venom gland?

Clevers said it was essentially a whim of three PhD students working in his lab who'd grown bored of reproducing mouse and human kidneys, livers and guts. "I think they sat down and asked themselves what is the most iconic animal we can culture? Not human or mouse. They said it's got to be the snake. The snake venom gland."

"They assumed that snakes would have stem cells the same way mice and humans have stems cells but nobody had ever investigated this," said Clevers.

After sourcing some fertilized snake eggs from a dealer, the researchers found they were able to take a tiny chunk of snake tissue, containing stem cells, and nurture it in a dish with the same growth factor they used for human organoids -- albeit at a lower temperature -- to create the venom glands. And they found that these snake organoids -- tiny balls just one millimeter wide -- produced the same toxins as the snake venom.

The team compared their lab-made venom with the real thing at the genetic level and in terms of function, finding that muscle cells stopped firing when exposed to their synthetic venom.

The current antivenoms available to us, produced in horses not humans, trigger relatively high rates of adverse reactions, which can be mild, like rash and itch, or more serious, like anaphylaxis. It's also expensive stuff. Wellcome estimate that one vial of antivenom costs $160, and a full course usually requires multiple vials.

Even if the people who need it can afford it -- most snakebite victims live in rural Asia and Africa -- the world has less than half of the antivenom stock it needs, according to Wellcome. Plus antivenoms have been developed for only around 60% of the world's venomous snakes.

In this context, the new research could have far-reaching consequences, allowing scientists to create a biobank of snake gland organoids from the 600 or so venomous snake species that could be used to produce limitless amounts of snake venom in a lab, said Clevers.

"The next step is to take all that knowledge and start investigating new antivenoms that take a more molecular approach," said Clevers.

To create an antivenom, genetic information and organoid technology could be used to make the specific venom components that cause the most harm -- and from them produce monoclonal antibodies, which mimic the body's immune system, to fight the venom, a method already used in immunotherapy treatments for cancer and other diseases.

"It's a great new way to work with venom in terms of developing new treatments and developing antivenom. Snakes are very difficult to look after," Cammack said, who was not involved with the research.

Clevers said his lab now plans to make venom gland organoids from the world's 50 most venomous animals and they will share this biobank with researchers worldwide. At the moment, Clevers said they are able to produce the organoids at a rate of one a week.

But producing antivenom is not an area that pharmaceutical companies have traditionally been keen to invest in, Clevers said

Campaigners often describe snakebites as a hidden health crisis, with snakebites killing more people than prostrate cancer and cholera worldwide, Cammack said.

"There's no money in the countries that suffer. Don't underestimate how many people die. Sharks kill about 20 per year. Snakes kill 100,000 or 150,000," said Clevers.

"I'm a cancer researcher essentially and I am appalled by the difference in investment in cancer research and this research."

One challenge to making synthetic antivenom is the sheer complexity of how a snake disables its prey. Its venom contains several different components that have different effects.

Researchers in India have sequenced the genome of the Indian Cobra, in an attempt to decode the venom.

"It's the first time a very medically important snake has been mapped in such detail," said Somasekar Seshagiri, president of SciGenom Research Foundation, a nonprofit research center in India.

"It creates the blueprint of the snake and helps us get the information from the venom glands." Next, his team will map the genomes of the saw-scaled viper, the common krait and the Russell's viper -- the rest of India's "big four." This could help make antivenom from the glands as it will be easier to identify the right proteins.

In tandem, both breakthroughs will also make it easier to discover whether some of the potent molecules contained in snake venom are themselves worth prospecting as drugs -- allowing snakes to make their mark on human health in a different way to how nature intended -- by saving lives.

"As well as being scary, venom is amazingly useful," Seshagari said.

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Snake venom can now be made in a lab and that could save many lives - CNN

Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

Crystal Nguyen, now 20, was first diagnosed with acute myeloid leukemia at age 12. She found a stem-cell match for a needed bone-marrow transplant through the international cord blood bank.Handout

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

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Chinese New Year babys B.C. family gives gift of life in cord-blood donation - The Province

A team of researchers at Osaka University in Japan successfully transplanted cardiac muscle cells created from iPS into a patient, who is now recovering in the general ward of the hospital.

The team, led by Yoshiki Sawa, a professor in the university's cardiovascular surgery unit, created the cardiac muscle cells from iPS cells in a clinical trial to verify the safety and efficacy of this type of procedure. The researches want to transplant heart muscle cells into ten patients who have serious heart malfunctions because of ischemic cardiomyopathy over a three year period.

RELATED: RESEARCHERS ORGANIZE STEM CELLS BASED ON A COMPUTATIONAL MODEL

Instead of replacing the heart of patients, the researchers developed degradable sheets of heart muscle cells that were placed on the damaged areas of the heart.

To grow the heart muscle cells in the lab, the researchers turned to induced pluripotent stem cells otherwise known as iPS. Researchers are able to take those iPS cells and make them into any cell they want. In this case, it was heart muscle cells.If the clinical trials prove successful it could remove someday the need for heart transplants.

I hope that (the transplant) will become a medical technology that will save as many people as possible, as Ive seen many lives that I couldnt save, Sawa was quoted at a news conference reported the Japan Times.

As for the patient, the team plans to monitor him during the next year to ascertain how the heart muscle cells perform. According to the Japan Times, the researchers opted to conduct a clinical trial instead of a clinical study because they want approval from Japan's health ministry for clinical application as soon as possible.

The report noted that during the trial the researchers will look at risks, probabilities of cancer and the efficacy of transplanting 100 million cells for each patient that could include tumor cells.

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Heart Muscle Cells Made in the Lab Successfully Transplanted into Patient - Interesting Engineering

John T Lear,1 Axel Hauschild,2 Eggert Stockfleth,3 Nicholas Squittieri,4 Nicole Basset-Seguin,5 Reinhard Dummer6

1Manchester Royal Infirmary, Manchester, UK; 2Klinik Fr Dermatologie, Venerologie Und Allergologie Universittsklinikum Schleswig-Holstein, Kiel, Germany; 3Universittshautklinik Bochum, Bochum, Germany; 4Sun Pharmaceutical Industries, Inc., Princeton, NJ, USA; 5Department of Dermatology, Hpital Saint Louis, Paris, France; 6Skin Cancer Center University Hospital, Zrich, Switzerland

Correspondence: John T LearUniversity of Manchester, 46 Grafton Street, Manchester M13 9NT, UKTel +44 161 276 4173Fax +44 161 276 8881Email john.lear@srft.nhs.uk

Nevoid basal cell carcinoma syndrome (NBCCS), or Gorlin syndrome, is a rare hereditary disease characterized by the development of multiple cutaneous basal cell carcinomas (BCCs) from a young age.1 Loss-of-function germline mutations in the hedgehog-related patched 1 (PTCH1) tumor suppressor gene are the most common cause of NBCCS.1 The hedgehog signaling pathway plays a major role in embryonic development, and in adulthood, is involved in the renewal and maintenance of distinct tissues, including hair follicles, muscle stem cells, and gastric epithelium.2 Its abnormal activation is thought to drive the formation of both sporadic BCCs and those resulting from NBCCS.1 Patients with NBCCS inherit one inactive copy of PTCH1 and then acquire a second-hit mutation, resulting in hedgehog pathway activation and BCC formation.1 Mutations in Suppressor of fused (SUFU) or the PTCH1 homolog PTCH2 have also been found in a subset of patients meeting criteria for NBCCS.1,3

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20 January 2020

A new study suggests that rare harmful mutations in young healthy donors' stem cells can be passed on to recipients of stem cell transplants, potentially leading to health problems.

Stem cell transplants can be used to treat some blood disorders and cancers, such as acute myeloid leukaemia (AML), but can also have life-threatening complications such as cardiovascular problems and graft-versus-host disease (GvHD), where new immune cells from the donor attacks the patient's healthy cells.

'There have been suspicions that genetic errors in donor stem cells may be causing problems in cancer patients, but until now we didn't have a way to identify them because they are so rare,' said Dr Todd EDruley, Associate Professor of Paediatrics, Haematology and Oncology at Washington University School of Medicine, StLouis. 'This study raises concerns that even young, healthy donors' blood stem cells may have harmful mutations and provides strong evidence that we need to explore the potential effects of these mutations further.'Researchers analysed samples from patients with AML and their stem cell donors looking at 80 specific genes. The small pilot study identified at least one harmful genetic mutation in 11 of the 25 donors using an advanced sequencing technique. The donors ranged from 20 to 58 years old, with a median age of 26. Researchers later detected the harmful mutations present in donors within the recipients.

These extremely rare, harmful genetic mutations that are present in donors' stem cells do not cause any health problems to the donors, however, they may be passed on to the patients receiving stem cell transplants. Intense chemo- and radiation therapy is required prior to stem cell transplants and the immunosuppression given after the transplant unfortunately allows the rare mutation containing cells the opportunity to replicate quickly, which potentially can create health problems for the patients who receive them.

Co-author, Dr Sima TBhatt, Assistant Professor of Paediatrics, Haematology and Oncology also at Washington University, said 'Transplant physicians tend to seek younger donors because we assume this will lead to fewer complications. But we now see evidence that even young and healthy donors can have mutations that will have consequences for our patients. We need to understand what those consequences are if we are to find ways to modify them.'

The clinical implications of the findings need to be further studied. Dr Bhatt added: 'Now that we've also linked these mutations to GvHD and cardiovascular problems, we have a larger study planned that we hope will answer some of the questions posed by this one.'

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Novel mutations in stem cells of young donors can be passed to recipients - BioNews