The life-changing phone call came at 4:30 in the morning on Christmas Eve.

All of a sudden, Ashley Peddle went from making final preparations for the holiday season to planning cancer treatments that had to start within days.

The East Greenwich resident, 37, had been experiencing fatigue, shortness of breath and headaches for some time, but when her husband Ryan encouraged her to get checked out, she chalked it up to being a busy mother of four kids 10 and younger. Finally, in the week leading up to Christmas, her symptoms became too much to bear as she could not even climb the steps in her house and was falling asleep on the couch around dinnertime.

She saw a doctor and later some troubling results of blood tests led to the early-morning phone call to get to the hospital immediately.

We rushed right to the emergency room and in about an hour or two, our world was kind of rocked with the diagnosis of leukemia, Ryan Peddle says. (Penn Medicine) was great and started her treatment right away because the type of leukemia she has is very aggressive, so the sooner she started, the better.

Two months and two rounds of chemotherapy after being told she has acute myeloid leukemia, Peddles prognosis is good, her husband said, but she is not out of the woods yet. She recently returned home after a six-week hospital stay but will soon go back to receive a stem cell transplant, also known as a bone marrow transplant, which will help restore her bone marrow, produce healthy blood cells and strengthen her immune system. In turn, she will be better suited to fighting the leukemia and preventing a reoccurrence down the line.

The best donors for stem cell transplants are usually a family member such as a sibling, but if they are not a match, an unrelated volunteer whose tissue type matches that of the patient may be used. Not only have Ashley and Ryan become educated about the process throughout their ordeal, but so have a group of Ashleys friends, who decided to take action when they learned how badly donors are needed.

Shawn Keating, also of East Greenwich, took the lead and helped organize a drive-thru bone marrow registration event in Peddles honor, which will be held this Saturday, Feb. 20, from 10 a.m. until 2 p.m., at Samuel Mickle School, 559 Kings Highway, in Mickleton. Sponsored by Be The Match which runs the largest and most diverse bone marrow registry in the world to help people battling blood cancers like leukemia and lymphoma the event will allow adults between the ages of 18 and 44 to join the national list of potential donors.

Ashley Peddle was diagnosed with leukemia and is in need of a bone marrow transplant. A registration event is being held Feb. 20 from 10 a.m. until 2 p.m. at Samuel Mickle School, 559 Kings Highway, in Mickleton.

In addition to supporting Peddle, who she has become friends with through their childrens sports and school activities, Keating had two other reasons for getting involved.

Her kids are the same age as mine and when you see this happen to somebody, you realize it could happen to anybody, she said. The other thing was, I went to sign myself up for Be The Match and I realized that at one point I had already requested the kit and in the busyness of things, I must have forgotten to send it back. So I reached out to them, got to talking and thought this was the best option, especially for busy people. Its as easy as driving through, getting swabbed and being done. I just thought it would be an easier way to get as many people on the registry as possible.

Bree Amborn works for an organization called The Icla da Silva Foundation, which is a recruitment center for Be The Match, and she will be running Saturdays event. She said people can expect a simple process to getting registered, as they just need to fill out some forms on their phone and do a swab in each cheek for 10 seconds without even leaving their car.

Those who cannot attend but still want to join Be The Match can text PeddleStrong to the number 61474 or visit join.bethematch.org/PeddleStrong and have a kit mailed to their home. Amborn added that it is especially important to increase the numbers of donors in the African-American community, as Black patients chances of finding a match are only 23%, compared to 77% for white patients.

The need is super, super high to add more people and to work in diverse communities and increase those populations on the registry as well, she said, so if a patient is a person of color they have the same chances of finding their match as if the patient is white.

Amborn will be able to answer any questions people have on Saturday. She was actually a donor herself while in college; after signing up she found out she was a match for a 50-year-old woman battling myelofibrosis.

It was an extremely easy process and Be The Match was awesome, she said. They pay for everything, they organize everything and they schedule everything, and as the donor you just show up. Youre literally able to change someones life.

I think people have a misconception of how the donation process works, but 80% of the time the donation is actually taken from your bloodstream. It looks very similar to donating platelets, where you have a needle in each arm, they take blood out of one arm, they separate your stem cells from your blood and give you your blood back in the other arm. Theres a couple more steps to the process but thats really the basics. Youre awake the whole time and its not a surgery.

Keating is hopeful that when people hear that, their fears will disappear and they will be eager to register. She doesnt know what to expect for Saturdays turnout, predicting, we could get 50 people or we could get 300. But already the response in East Greenwich has been impressive, with more than 50 volunteers slated to be in attendance.

Some, like Keating and Jacqueline DAngelis, are bringing their teenaged children to help as well.

Like I told my daughter, we cant cure cancer, but we can certainly help others by collecting these swabs, said DAngelis, a neighbor of the Peddles who has known them for seven years.

Theres going to be a lot of people from our community out there this weekend to support Ashley and support this mission to increase that number. Ashley is such a wonderful part of the community and its nice to see how everyone can band together and try to make something good out of this difficult and trying situation for her family. Its nice to see so many families parents and their teenagers coming out to volunteer in the cold in the middle of a pandemic to do something nice.

Although the Peddles have been quarantining since Ashley returned home because her immune system is compromised, Ryan plans on stopping by the event and showing his appreciation for the volunteers and those joining the list.

I didnt know anything about the registry beforehand or else Id have been on it, he said. Thats one of the reasons were pushing this drive, to get as many people on the registry as we can. They may not help Ashley but theyll help someone just like her.

He said his wifes spirits have remained high during her treatment. One of the toughest parts was when she was in the hospital and could not get in-person visits from their daughter and three sons, ages 10, 8, 7 and 4. They were able to FaceTime frequently, however, for much-needed emotional support.

The family is also grateful for the outpouring of assistance from those around them.

Were honestly overwhelmed by the support weve received, Ryan said. Our kids are pretty active in sports and other activities in the township, and through that weve made a lot of really good friendships and met a lot of really great people. So many of them are stepping up, not only for this drive but to give blood, to donate platelets, to cook meals, to drop off little things for the kids like Valentines. Its just been overwhelming and we couldnt be more proud of the town we live in.

Follow this link:
N.J. mom of 4 has leukemia. A bone marrow registry is being held Saturday. - nj.com

SASKATOON -- An effort to increase stem cell donors within Black communities across Canada is being driven by a group of women whove had difficulty finding full genetic matches themselves.

Genetic matches are crucial for patients in need of stem cell transplants, such as those with leukemia and lymphoma, and matches are more commonly found within their own racial, ethnic and ancestral groups.

But the new Black Donors Save Lives campaign notes that fewer than two per cent of those in the Canadian Blood Services stem cell donor registry are Black.

And that decreases their chance of finding a match, campaign lead Sylvia Okonofua told CTVNews.ca in a phone interview. It becomes a numbers game for Black people on the stem cell waiting list, where its like finding a needle in a hay stack for them.

The recent University of Regina biochemistry graduate, with sights on becoming a hematologist, timed the virtual campaign to kick off during Black History Month.

It was overall frustrating to know that a patient from my community is so much less likely than other patients to be helped, she told CTVNews.ca. When you see that your people have a really, really low chance of being helped out, it takes you aback.

Okonofua noted part of the campaign uses TikToks, shareable infographics, and even an original song to get the message out and reach a wide audience.

And she said part of the outreach involves having Black stem cell recipients talk about their experiences with the health-care system and speak to the historical mistrust the Black community has towards the medical community.

She founded her campus chapter of Stem Cell Club, a non-profit organization with chapters across Canada which recruits Canadians as potential stem cell donors.

Registration for Black Donors Save Lives can be done online, where participants between the ages of 17 to 35 can fill out a questionnaire and have a swab kit mailed to their address. After they swab the inside of their cheeks and send the sample back, if there is a person in need, 90 per cent of donors will be asked to donate stem cells very similar to the way a person would be giving blood.

But a big difference is the donor is given a growth hormone a week before donation in order to increase the number of stem cells, as well as the process taking four to six hours.

Alternatively, one out of 10 donors will be asked if theyd like to donate stem cells via bone marrow surgery, which can take place over a day.

In 2017, Reve Agyepong experienced firsthand the lack of Black stem cell donors, to treat her sickle cell disease, which involve red blood cells becoming misshapen, which can block blood vessels and lead to damage to bones, brain, kidneys, and lungs, and can ultimately be fatal.

But Agyepong, who was born in Edmonton to Ghanaian parents, was fortunate to receive a stem cell transplant from her sister.

It is such a blessing to have a match within your own family because the percentages are just so low, she told CTVNews.ca by email. I am so fortunate to have found a match in my family or else transplant would have been off the table for me.

In fact, only one in four patients who need a stem cell transplant are able to find a matched donor within their family, with Black patients being less than half as likely as white patients to find a unrelated person they match with on a donor registry, according to the campaign.

For Jamaican-Canadian Dorothy Vernon-Brown, who helped inspire this months campaign, the current efforts are deeply personal. In 2013, she was diagnosed with acute myeloid leukaemia and was heartbroken to discover there were no stem cell matches in Canada's registry or internationally.

She ultimately received stem cells from her sister, who was a half-match, and has been spreading information to Black Canadians ever since, through her own advocacy group, Donor Drive for Dorothy.

Stem cell transplantation is a miracle for patients, and I wish people knew how easy it is to be a stem donor, she recounted on a Twitter thread for another stem cell awareness campaign. You could give someone an opportunity like my sister gave me, to be around and live the life I want. People want to live, so if that gift is in your hands, I appeal to you to see it as something significant to do in your life.

Okonofua and Vernon-Browns efforts are being aided by Dr. Warren Fingrut, a hematologist whos the director of the aforementioned Stem Cell Club.

He told CTVNews.ca in an email hes seen firsthand far too many patients from ethnic and racial minority groups in situations where they dont have fully-matched donors and are forced to seek other treatments.

I find this heart wrenching and I am very motivated to work to address this, Fingrut said.

That led to him founding his non-profit a decade ago, which has gone on to recruit more than 20,000 Canadians as stem cell donors, with more than 55 per cent being non-white. But in cases such as Vernon-Brown and others, those figures need to be much higher.

We started running national campaigns last year, focused on the recruitment of diverse peoples as donors, as well as males who are also preferred by transplant physicians (all else being equal) as they are associated with better outcomes for patients, Fingrut explained.

The campaign is also being done in partnership with several other groups, including the Katelyn Bedard Bone Marrow Association, Black Physicians of Canada, Black Medical Students Association of Canada and the National Black Law Students Association of Canada.

This campaign is one example of an initiative in the health-care sector, which seeks to address racial disparity impacting the care of Black patients, he wrote, noting Black people face many such disparities in access to care, and we want to see others in the health-care sector working with Black Canadians to tackle these issues and address them, in collaboration with Black communities.

Okonofua hopes next Black History Month, theyll be able to have in-person swabbing events in places of worship, community hubs, and cultural gatherings to show how easy it is.

Fingrut said this the first time his group has specifically engaged with one racial group and hopes to expand it to other ethnic and racial communities including South Asians, Indigenous peoples, and those of mixed ancestry in the near future.

Follow this link:
Meet the women hoping to recruit more stem cells donors from Black communities - CTV News

In people with polycythemia vera (PV), the bone marrow produces too many blood cells. This overproduction can lead to complications, such as abnormal blood clotting, unusual bleeding, and an enlarged spleen.

In rare cases, scar tissue may replace the bone marrow. When this happens, the bone marrow can no longer produce enough healthy blood cells. Experts refer to this condition, which is a type of chronic leukemia, as myelofibrosis (MF). It can sometimes lead to acute myeloid leukemia, though this is rare.

People with PV have a shorter-than-average life expectancy. Some of the possible complications of the disease can be life threatening.

Getting treatment can help reduce the risk of certain complications from PV, including blood clots. As a result, a person will likely lead a longer and healthier life with this disease if they receive treatment.

According to an article in Blood Cancer Journal, the median survival time for people with PV is 14 years after diagnosis. The authors take this survival time from a study in which half of the participants were still alive 14 years after diagnosis.

Younger people tend to live for longer with the disease. Research suggests that the median survival time for those under 60 years of age is 24 years following diagnosis.

Multiple factors affect the outlook and life expectancy of people with PV, including:

Blood clots are the most common cause of death in people with PV. When blood clots form in blood vessels, they can block the flow of blood to vital organs. This can lead to life threatening complications, such as stroke, heart attack, and venous thrombosis.

Treatment for PV can help relieve symptoms and lower the risk of blood clots. In this way, it also reduces a persons risk of life threatening complications.

In most cases, healthcare providers prescribe regular blood draws to treat PV. Blood draws reduce the number of blood cells in the body, which may help improve blood flow.

Healthcare providers may also prescribe low dose aspirin to help prevent the formation of blood clots. Additionally, they may prescribe other medications, such as hydroxyurea (Hydrea) or busulfan (Myleran).

If a person develops MF as a complication of PV, their healthcare provider may prescribe one or more of the following treatments:

These treatments may help improve symptoms, increase life expectancy, or both.

For example, scientists have found that stem cell transplants may help improve long-term survival in people with MF. However, this treatment comes with a high risk of life threatening side effects. It is especially risky for older adults and people with other health conditions. As a result, healthcare providers often avoid prescribing this treatment.

Some studies have found that treatment with JAK inhibitors may also improve survival rates in people with MF. However, when scientists reviewed the available evidence on Jakafi and Inrebic, they found that the quality of evidence on survival rates is limited. More research is necessary to confirm how these treatments affect life expectancy.

Early research involving people with PV found that the median survival time for those who did not receive treatment was less than 2 years after diagnosis. This research took place before the medical community recognized blood draws as a treatment option, and it reflects the high risk of blood clots in people not receiving treatment.

People with PV who do not receive treatment are more likely to develop blood clots. According to the Leukemia & Lymphoma Society, 4060% of people with untreated PV may develop blood clots within 10 years of diagnosis.

Scientists have not yet developed a cure for PV. However, healthcare providers may prescribe blood draws, medications, or other treatments to help manage symptoms, reduce the risk of complications, and increase life expectancy in people with this disease.

Researchers are also continuing to develop and test potential new treatments for PV, such as the anticancer drug imatinib mesylate (Gleevec) and novel types of JAK inhibitors.

In some cases, a persons healthcare provider may encourage them to take part in a clinical trial. In this type of study, participants receive an experimental treatment. People interested in learning more about the potential benefits and risks of taking part in a clinical trial can talk with their healthcare provider or the researchers running the study.

When a person receives a diagnosis of PV, getting treatment is important. Treatment may help minimize symptoms, lower the risk of complications, and improve life expectancy.

A persons recommended treatment plan for PV will depend on many factors, including their age, overall health, and whether they have developed certain complications.

People with PV who wish to learn more about their treatment options and outlook should talk with their healthcare provider.

Read the original here:
Polycythemia vera life expectancy: With treatment and more - Medical News Today

Taljat David/Shutterstock

Novartis and the Bill & Melinda Gates Foundation have partnered on a single-dose, in vivo gene therapy for sickle cell disease (SCD). The Foundation will offer funding for development of the therapy.

Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease, said Jay Bradner, a hematologist and president of the Novartis Institutes for BioMedical Research (NIBR). This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill & Melinda Gates Foundation in addressing this global unmet medical need.

The announcement comes only a day after bluebird bio announced that it has placed its Phase I/II and Phase III trial of LentiGlobin gene therapy for sickle cell disease (SCD) on temporary suspension. The cause is a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML).

HGB-206 is the companys ongoing Phase I/II trial of LentiGlobin for SCD. It includes three cohorts, A, B and C. In Group C, a refined manufacturing process designed to increase vector copy number was used.

Group C also received LentiGlobin for SCD manufactured from hematopoietic (blood) stem cells (HSCs) collected from peripheral blood after mobilization with plerixafor, instead of by way of bone marrow harvest, which was the method used in Groups A and B.

HGB-210 is their ongoing Phase III single-arm open-label trial. It is evaluating efficacy and safety of LentiGlobin for SCD in patients between two years and 50 years of age with sickle cell disease.

Which underlines that even though gene therapy is making headway, it is still a cutting-edge technology.

SCD is a hereditary blood disease that affects millions of people globally, with more than 300,000 born with it each year. It primarily affects people of African descent, and sub-Saharan Africa bears about 80% of the disease burden. It affects the structure of red blood cells, causing a distinct sickle shape, which decreases the ability of red blood cells to transport oxygen efficiently.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, said Trevor Mundel, president of Global Health at the Gates Foundation. Whats exciting about this project is that it brings ambitious science to bear on that challenge. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought. It also holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.

Novartis also announced today that the U.S. Food and Drug Administration (FDA) approved the expanded indication for Entresto (sacubitril/valsartan) to decrease the risk of cardiovascular death and hospitalization for heart failure in adults with chronic heart failure. The biggest benefit was for patients with left ventricular ejection fraction (LVEF) below normal.

The expansion was based on data in the PARAGON-HF Phase III trial.

This approval is a significant advancement, providing a treatment to many patients who were not eligible for treatment before because their ejection fraction was above the region we normally considered reduced, said Scott Solomon, professor of Medicine at Harvard Medical School and Brigham and Womens Hospital, and PARAGON-HF Executive Committee co-chair. We can now offer a treatment to a wider range of patients who have an LVEF below normal.

Featured Jobs on BioSpace

Visit link:
Novartis and Gates Foundation Team Up To Deliver Affordable Sickle Cell Gene Therapy - BioSpace

A desperate family is facing a nervous wait after a stem cell donor finally found to give their four-year-old daughter a new chance at life fell ill.

Little Adeline Davidson has been waiting more than two years for the procedure to treat a rare form of blood cancer, and several arrangements with donors have fallen through during that time.

The Alness youngster had been due to go under the knife in Glasgow this week after it appeared that the search for a match had eventually come to an end.

But the family encountered yet another setback as the procedure was cancelled when the stem cell donor fell ill.

Adelines parents Steph, 26, and Jordan, 28, say their daughters transplant now hangs in the balance as they face an agonising wait to find out what is wrong with the donor.

They say the plans could be thrown into disarray with top level talks and a possible world-first procedure required if tests show that the illness is Covid-related.

The family will only be able to find out the nature of the donors condition after he is operated on and the cells removed.

If the donor has coronavirus, a team of international surgeons will assemble to debate whether it would be safe for Adeline to undergo the transplant.

Mrs Davidson said: We have been waiting more than two whole years for our ill child to get a bone marrow transplant.

The hospital have told me that the donor has to donate, and then they release the information on the cause of his illness.

If it is Covid, that would mean they would have to ask international doctors and surgeons if they could go ahead.

They have never given a child thats Covid-negative marrow from someone who is Covid-positive.

If they decide not to proceed, we are back to looking for someone else to begin the search again, which is just a crazy, horrible thought. I dont even want to think about it.

Mrs Davidson added that she would consider going ahead even if the cells have come from someone with coronavirus.

She said: I think we have to go with the doctors word, but Id be so frightened.

We wouldnt have another choice though, unfortunately.

If they, as professionals, believe doing it would outweigh the risks, we would just have to believe that too.

Over the last two years, Adeline has endured around 85 blood transfusions, one anaphylactic shock and emergency helicopter and ambulance transfers to hospital.

Mrs Davidosn added: There is potential for even worse news but we just hope that it isnt Covid he has.

If it isnt Covid, then everything moves along as it was meant to be.

We are aware that on the register there was no-one else, so we were lucky this guy popped up.

If all goes well, Adeline will receive her transplant in four weeks.

The latest setback comes almost two years to the day since her transplant journey began.

In December, the family were dealt a devastating blow as health officials postponed her procedure, scheduled to take place in January, due to Brexit complications.

The four-year-old requires a specific type off marrow, processed by a centre outwith the UK, which is then brought to the country by road.

Life-saving transplant for Highland youngster postponed due to delays caused by Brexit

Early last year, the family were forced to turn to the register and launch a public appeal in search for multiple new donors due to an array of complications.

Mrs Davidson praised Adelines resilience but admits it breaks her heart to not be able to see her daughter progress onto school in August.

She said: The first year I was so positive lets get on with it, this needs done and I never thought why us?

I just thought we have so much to be grateful for and thankful for.

However, the whole of the second year, Im just thinking is someone messing with us because thats what it feels like.

She added: Adelines been so good. She hardly complains and I just think its because she has no idea whats shes missing, which is sort of a good thing but sad.

She is lucky thats shes an outgoing kid. She is behind, she has not socialised and although she is switched on, shes probably not as far on as her peers.

Even now, she should have been staring primary one in August this year but shes not even been to nursery. Its hellish.

See more here:
Heartbreak for family of cancer-stricken four-year-old as stem cell donor falls ill at last minute - Press and Journal

CAMBRIDGE, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

Original post:
Fulcrum Therapeutics to Participate in Upcoming Investor Conferences

BOSTON, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, announced today that it will present findings from its drug discovery and development programs at the 44th Annual MidWinter Meeting of the Association for Research in Otolaryngology (ARO), which will be held virtually February 20–24, 2021.

See the rest here:
Decibel Therapeutics to Present at the 44th Annual Association for Research in Otolaryngology (ARO) Conference

SANTA MONICA, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- BioVie Inc. (NASDAQ: BIVI) ("BioVie" or "Company"), a clinical-stage company developing innovative drug therapies for liver disease, today announced an interview with Terren Peizer, Chairman and Chief Executive Officer, will air on The RedChip Money Report® on Bloomberg TV in the US on Saturday, February 20, at 7 p.m. local time in 73M homes across the United States. The RedChip Money Report® also airs on Bloomberg International in Europe in 100M homes at 6 pm local time on Sundays.

See original here:
BioVie Interview to Air on Bloomberg TV in the US on the RedChip Money Report®

SALT LAKE CITY, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Clene Inc. (NASDAQ: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company, today announced that it will present updated interim data from its REPAIR-MS study and updated blinded interim data from its VISIONARY-MS study in poster presentations at the Americas Committee for Treatment and Research in Multiple Sclerosis’ ACTRIMS Forum 2021 (“ACTRIMS”) taking place virtually from February 25-27, 2021.

Go here to read the rest:
Clene Nanomedicine to Present Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021

Study Led by Gene Therapy Program at the University of Pennsylvania Published in Molecular Therapy Study Led by Gene Therapy Program at the University of Pennsylvania Published in Molecular Therapy

Read more:
Precision Announces 3-Year Pre-clinical Study Results Showing Long-term Durability and Safety of ARCUS In Vivo Gene Editing to Cut LDL Cholesterol...

BOSTON, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Imara Inc. (Nasdaq: IMRA), a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin, today announced that Rahul Ballal, Ph.D., President and Chief Executive Officer, will participate in a Fireside Chat at the SVB Leerink 10th Annual Global Healthcare Conference on Friday, February 26, from 1:40-2:10 p.m. ET.

Follow this link:
Imara to Present at SVB Leerink 10th Annual Global Healthcare Conference

NEW YORK and MAINZ, GERMANY, February 19, 2021 (GLOBE NEWSWIRE) — Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced the submission of new data to the U.S. Food and Drug Administration (FDA) demonstrating the stability of their COVID-19 vaccine when stored at -25°C to -15°C (-13°F to 5°F), temperatures more commonly found in pharmaceutical freezers and refrigerators. The data have been submitted to the FDA to support a proposed update to the U.S. Emergency Use Authorization (EUA) Prescribing Information, which would allow for vaccine vials to be stored at these temperatures for a total of two weeks as an alternative or complement to storage in an ultra-low temperature freezer.

Visit link:
Pfizer and BioNTech Submit COVID-19 Vaccine Stability Data at Standard Freezer Temperature to the U.S. FDA

SAN FRANCISCO, Feb. 19, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO),?a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers,?and affiliate ML Bio Solutions today announced that the first patient has been dosed in a Phase 2 trial of BBP-418 in patients with LGMD2i. BridgeBio and ML Bio’s BBP-418 is the first-ever oral disease-modifying investigational treatment for LGMD2i. BBP-418 was granted Orphan Drug Designation for LGMD2i by the US Food and Drug Administration (FDA) in 2019, and for LGMD by the European Medicines Agency (EMA) in 2020.

Original post:
BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i...

Partnerships will use Vaccinex’s ActivMAb® platform for viral display of complex antigens to enable antibody discovery Partnerships will use Vaccinex’s ActivMAb® platform for viral display of complex antigens to enable antibody discovery

See the rest here:
Vaccinex Announces Signing of Two Multi-Project Deals with Leading Pharmaceutical Companies Focused on Leveraging Its ActivMAb® Platform

CAMBRIDGE, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, today announced that it will host a conference call and webcast on Friday, February 26, 2021 at 8:00 a.m. ET to report financial results for the fourth quarter and year ended December 31, 2020 and provide business updates.

Read more:
Mersana Therapeutics to Host Conference Call Announcing Fourth Quarter and Year End 2020 Financial Results and Business Updates

NEW YORK, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that Jerry Durso, President and Chief Executive Officer, and Sandip Kapadia, Chief Financial Officer of Intercept, will present at the SVB Leerink 10th Annual Global Healthcare Conference on Friday, February 26, 2021 at 1:00 p.m. ET, and at the Cowen 41st Annual Healthcare Conference on Tuesday, March 2, 2021 at 10:30 a.m. ET.

See the original post:
Intercept to Present at Upcoming Investor Conferences

SOUTH SAN FRANCISCO, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases, today announced that Brian Wong, M.D., Ph.D., President and Chief Executive Officer, will present at the 10th Annual SVB Leerink Global Healthcare Conference on Friday, February 26, 2021 at 12:00 pm Eastern Time.

See more here:
RAPT Therapeutics to Present at the 10th Annual SVB Leerink Global Healthcare Conference

SOUTH SAN FRANCISCO, Calif., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a clinical-stage biotechnology company developing innovative therapeutics targeting hepatitis B virus (HBV), today announced two upcoming investor events:

Read the original post:
Assembly Bio Announces Upcoming Investor Events

WOBURN, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Yield10 Bioscience, Inc. (Nasdaq:YTEN), an agricultural bioscience company, today announced that it has received proceeds of approximately $3.8 million from the exercise of 472,099 warrants exercised between January 1, 2021 and February 16, 2021, and the Company has issued to investors 472,099 shares of common stock in exchange for the exercised warrants. The warrants were issued to investors pursuant to a public offering that closed on November 19, 2019. Since November 19, 2019, a total of 679,395 warrants issued in the public offering have been exercised for total proceeds of $5.4 million. The Series A and Series B warrants issued in the November 2019 public offering are exercisable at $8.00 per share and have expiration dates of May 19, 2022 and May 19, 2027, respectively.

View original post here:
Yield10 Bioscience Announces Warrant Exercises for Proceeds of $3.8 million

Chugai obtains exclusive development and marketing rights for AT-527 in Japan from Roche, who has ex-US rights for the treatment of COVID-19 Chugai obtains exclusive development and marketing rights for AT-527 in Japan from Roche, who has ex-US rights for the treatment of COVID-19

See original here:
Atea Pharmaceuticals Announces Chugai In-License of AT-527 from Roche for the Treatment of COVID-19 in Japan