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The most important health innovations of the past decade – The Hill

The 2010s are coming to an end, and looking back there have been some pretty amazing advances and innovations in health and science.

Advances in prosthetic limbs

Prosthetic limbs have been around since ancient times. In Egypt, a prosthetic wooden toe was found on a mummy dating back 3,000 years. By the Dark Ages, inventors could incorporate hinges on prosthetic arms used by knights. In modern times, the field of prosthetics has turned to incorporating more technology into physical stand-ins for limbs. In the last several years, theres been a boom in advances that have led to the best and most useful prosthetics weve ever seen.

Reports from the early 2010s talked about the potential for new technology to allow people to control prosthetics with their minds and to receive sensory information from their devices. It may have been a reach in the early part of the decade, but now it is literally within grasp. There are new prosthetic hands being tested that give the user the ability to grab objects with their thoughts and even to sense the texture of what they are touching. New bionic hands allow the user to feel again by sending signals back to the brain about the things they are touching, like whether its hard or soft. Other research groups have been working on bionic arms that can move based on the users thoughts through a brain-computer interface. While these have demonstrated its possible to accomplish these goals in the lab, theres still more to be done before people can use these devices outside in the real world.

Many of these advanced prosthetics are still prototypes and may not reach the general population for a while. Luckily, cheaper 3D printers have made simple prosthetics more accessible. These are important because a prosthetic device can improve the quality of life for people. For example, this person has been printing prosthetic hands and arms for people in Africa after watching an online tutorial. New materials that go into 3D printers are cheaper than they used to be and are being used in prosthetics to provide a more affordable option for patients.

Although prosthetics have been around for ages in some form or another, they arent always used. One variable to consider is the social acceptance of having a prosthetic. Theres still a lot of stigma around disabilities and many people may reject prosthetics even if they are available. In 2012, an athlete with both feet amputated competed in the mens 400 meter race at the Olympics in London. There was some controversy over whether the runner with a prosthetic foot should be allowed to run in races with people who dont have prosthetics or if they should only be allowed in competitions specifically for people who have them. Prosthetics also need to be comfortable and usable in order to be successfully adopted. In one study, about 4.5 percent of people rejected prosthetics and 13.4 percent stopped using their prosthetics. As the new prosthetics that are more natural and intuitive to use come to market, hopefully more people will benefit, and the social barriers to acceptance will disappear.

CRISPR

The genome modification technique called Clustered Regularly Interspaced Short Palindromic Repeats, aka CRISPR, was a culmination of a few decades of work by scientists, and major studies explaining the method were published in 2013. The version of it called CRISPR-associated protein 9 or CRIPSR-Cas9 is what most researchers are specifically using in most cases. It involves a regular gene editing mechanism that happens in bacteria. The bacteria can take sections of DNA from attacking viruses and essentially use that to remember the viruses if they return. When the virus is back, the bacteria can target the matching sections of DNA in the virus, cut it and disable the virus.

Though 2013 was only six years ago, as far as science goes, CRISPR has been moving at lightning speed towards practical applications. Using CRISPR to edit a gene sequence, researchers can now add, delete or modify DNA segments more quickly and accurately than ever before. Since the technique was developed, researchers have used CRISPR to target diseases caused by a single gene like cystic fibrosis or sickle cell disease.

Probably the most infamous use of CRISPR are the CRISPR babies. In late 2018, a Chinese researcher, He Jiankui, claimed to have used CRISPR to modify the genomes of two babies to include a mutated version of a gene that protects against HIV. This case was and is highly controversial for the ethical concerns with genetically modifying a human genome at the embryo level, or germline, meaning it can be passed down to future generations and has not been done before in humans. Recently, MIT Technology Review obtained excerpts from Hes research, and experts say that the report and data may be untrustworthy. This means it is still unclear if He and collaborators actually successfully modified the babies genomes. The scientific community overall condemns this way of using CRISPR to edit a human germline genome and has called for an international moratorium on it until a framework can be agreed on.The researcher has been sentenced to three years in prison in Shenzhen, China.

As fraught with controversy as the CRISPR babies may be, CRISPR technology still holds a lot of promise and can be used responsibly, supporters say. For example, researchers are using it to target cancer cells by taking a patients immune cells, modifying them using CRISPR and then infusing the patient with the modified cells. For blood diseases, a patient with sickle cell disease is reported to be responding well to a CRISPR treatment that has allowed her body to produce a crucial protein.

Another area that has boomed this decade partly because of CRISPR technology is stem cell therapy, which well get into in the next section.

Stem cell therapy

Technically, the only Federal Drug Administration (FDA)-approved stem cell therapies are blood-forming stem cells derived from umbilical cord blood. Blood-forming stem cells are used to treat patients with cancer after chemotherapy has depleted blood cells, as well as patients with blood disorders like leukemia whose bone marrow tissues are damaged. These types of treatments have been around for about 30 years, but in the 2010s weve seen potential for more uses of stem cells in health care.

The main idea behind stem cell therapy is that because the cells are pluripotent meaning they can become many other types of cells they can be introduced into parts of the body that are damaged and need new cells. On top of that, researchers can now extract some types of stem cells from a persons body, so no need for umbilical cords. This opens up the possibilities for highly personalized treatment where one person can be treated with stem cells from their own body.

Researchers are exploring how stem cells can be used to treat liver disease, cerebral palsy, stroke, brain injury and others. There are many ongoing research-backed clinical trials for stem cell therapy. A quick search for stem cell therapy on the governments clinical trial database turns up 5,638 results. And because of the work necessary to even get to the clinical trial stage, theres likely an order of magnitude more stem cell therapy studies in the pre-clinical trial stages.

Stem cell therapy is also being offered in for-profit clinics around the U.S. In these cases, the clinics are typically taking fat tissue from a patient, isolating the stem cells and then administering the stem cells back to the patient. In some cases, the treatments may lead to health complications, like blindness in a few extreme cases, and the FDA warns that such treatments are unapproved and potentially harmful. The FDA is ramping up regulation of stem cell clinics and earlier this year took a specific clinic in Florida to court.

Although there are many stem cell clinics offering unproven stem cell therapies, its not all hype. Granted that its difficult to pass the clinical trial stage to get FDA approval, stem cell research may lead to new treatments for several health conditions that could completely change the health care landscape.

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The most important health innovations of the past decade - The Hill

Hydrogel controls inflammation to speed healing – Futurity: Research News

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Researchers have established a baseline set of injectable hydrogels that show promise to help heal wounds, deliver drugs, and treat cancer.

Critically, theyve analyzed how the chemically distinct hydrogels provoke the bodys inflammatory responseor not.

The researchers designed the hydrogels to be injectable and create a mimic of cellular scaffolds in a desired location. They serve as placeholders while the body naturally feeds new blood vessels and cells into the scaffold, which degrades over time to leave natural tissue in its place. Hydrogels can also carry chemical or biological prompts that determine the scaffolds structure or affinity to the surrounding tissue.

We dont want zero inflammation; we want appropriate inflammation.

The study demonstrates it should be possible to tune multidomain peptide hydrogels to produce appropriate inflammatory response for what theyre treating.

Weve been working on peptide-based hydrogels for a number of years and have produced about 100 different types, says Jeffrey Hartgerink, a chemist and bioengineer at Rice University. In this paper, we wanted to back up a bit and understand some of the fundamental ways in which they modify biological environments.

The researchers wanted to know specifically how synthetic hydrogels influence the environments inflammatory response. The two-year study offered the first opportunity to test a variety of biocompatible hydrogels for the levels of inflammatory response they trigger.

Usually, we think of inflammation as bad, Hartgerink says. Thats because inflammation is sometimes associated with pain, and nobody likes pain. But the inflammatory response is also extremely important for wound healing and in clearing infection.

We dont want zero inflammation; we want appropriate inflammation, he says. If we want to heal wounds, inflammation is good because it starts the process of rebuilding vasculature. It recruits all kinds of cells that are regenerative to that site.

The labs tested four basic hydrogel typestwo with positive charge and two negativeto see what kind of inflammation they would trigger. They discovered that positively charged hydrogels triggered a much stronger inflammatory response than negatively charged ones.

Among the positive materials, depending on the chemistry generating that charge, we can either generate a strong or a moderate inflammatory response, Hartgerink says. If youre going for wound-healing, you really want a moderate response, and we saw that in one of the four materials.

But if you want to go for a cancer treatment, the higher inflammatory response might be more effective, he says. For something like drug delivery, where inflammation is not helpful, one of the negatively charged materials might be better.

Basically, were laying the groundwork to understand how to develop materials around the inflammatory responses these materials provoke. That will give us our best chance of success.

Researchers at Texas Heart Institute (THI) helped analyze the cellular response to the hydrogels through multidimensional flow cytometry.

The results of this work lay the groundwork for specifically tailoring delivery of a therapeutic by a delivery vehicle that is functionally relevant and predictable, says Darren Woodside, vice president for research and director of the flow cytometry and imaging core at THI. Aside from delivering drugs, these hydrogels are also compatible with a variety of cell types.

One of the problems with stem cell therapies at present is that adoptively transferred cells dont necessarily stay in high numbers at the site of injection, he says. Mixing these relatively inert, negatively charged hydrogels with stem cells before injection may overcome this limitation.

Hartgerink says the work is foundational, rather than geared toward a specific application, but is important to the long-term goal of bringing synthetic hydrogels to the clinic.

We have been speculating about a lot of the things we think are good and true about this material, and we now have more of a sound mechanistic understanding of why they are, in fact, true, Hartgerink says.

The research appears in Biomaterials.

Additional coauthors are from Rice and the Texas Heart Institute. The National Institutes of Health, the Welch Foundation, the Mexican National Council for Science and Technology, the National Science Foundation, and a Stauffer-Rothrock Fellowship supported the research.

Source: Rice University

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Hydrogel controls inflammation to speed healing - Futurity: Research News

AgeX Therapeutics Announces Drawdown of Second Tranche of Loan Facility from Juvenescence Ltd. – BioSpace

Juvenescence is pleased to continue its commitment to AgeX through this additional drawdown under the loan facility, commented Gregory Bailey, MD, Chairman of AgeX and CEO of Juvenescence. Juvenescence remains committed to funding the future development plans of AgeX through further advancements under the loan facility or otherwise. Since Juvenescences initial investment in AgeX in June 2018, AgeX has been an important element in the Juvenescence mission and strategy. Juvenescence is also investing its time and personnel to support AgeXs business development initiatives which have impressive potential. We look forward to AgeX announcing its plans for 2020 as it pursues tissue regeneration in Reverse Bioengineering, while advancing the development of BAT and VASC 1, the coupling of HLA-G with PureStem-derived cells for transplant therapies, and exploring partnerships with third parties.

This round of funding will allow us to continue to execute on our strategic plan to provide therapies for certain chronic and degenerative diseases through cellular regeneration and replacement, commented AgeXs founder and CEO Michael D. West, PhD.

As announced in the companys news release on August 14, 2019, AgeX has obtained a $2 million credit facility from Juvenescence to finance AgeXs operations and advance its product development programs.

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.

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). 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.

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AgeX Therapeutics Announces Drawdown of Second Tranche of Loan Facility from Juvenescence Ltd. - BioSpace

Preventive Healthcare For The Joints – Economic Times

At Stone Clinic were often asked, What can I do to preserve my knees? The answer: dont get hurt. Knees can go forever, providing their cartilage and ligaments stay intact. But how do I not get hurt?

Knees and ankles take one to three million steps per year, supporting up to five times our body weight. The bearing surface, called articular cartilage, has a remarkable ability to absorb force and provide nearly frictionless lubrication up to five times as slick as ice on ice. Unless it is injured or attacked by an inflammatory disease, articular cartilage can last foreverno matter how much running or other reasonable forces are applied. Once injured, however, its repair capacity is hampered by a low storehouse of reparative cells.

If the meniscus in the knee joint is injured, or a portion removed, the forces are concentrated on a smaller area of the articular cartilage. Thats when wear, down to the bone, occurs. If the ligaments of the knee are damaged, the joint motions are abnormal. As happens with a car out of alignment, the surfaces wear rapidly.

So our first answerdont get injuredis correct. And if your knee is injured? Repair, regenerate, and replace the damaged structures immediately, with the goal of restoring normal anatomy.

To avoid getting injured in the first place, the key is to choose wisely. Most often we make the choices that lead to injury. Here are a few ways to avoid the costly ones.

First, keep your head in the game. Most errors we see are mental errors. The athlete was out of control, got distracted, or landed poorly from a jump. They lunged when they shouldnt have, stuck their leg out to block an opponent when a different move would have been smarterthe list goes on. Here, the point: Focus in sports is not just on your opponent. Its on you. Your ability to maintain body awareness and make great choices determines whether or not you end up in my office.

Second, optimize your health. This means everything from mental health to fitness, including your diet and body weight. If you dont want to get injured, dont start out injured. Self-inflicted injury used to be thought of as smoking cigarettes and drinking too much alcohol. It is now clear that every meal, every choice, every decision about whether to train or not, determines how healthy we are. It appears that dietary choices heavily biased toward plant-based diets may make the largest difference in total body inflammation and disease development. And while supplements such as glucosamine appear to help joint lubrication and cartilage health, not many others have been shown to be at all helpful. An addiction to the testosterone, pheromones, adrenaline, and endorphins produced during daily exercise is the best possible addiction to have. Its also important to enjoy yourself. It can be fun to make a game out of optimizing your health. But its a painful chore if you see it as a chore. Healthy people get injured less often.

Third, partner up. Playing and training with a partner dramatically increases the efficacy and efficiency of your fitness program. The family that plays together, stays together was a rule we heard growing up. It remains a great guideline today.

Fourth, recognize injuriesminor and major onesearly, and treat them aggressively. We are developing the entire field of anabolic therapies for joint and tissue injuries where we inject bioactive factors that boost stem cell recruitment to the site of injury. We dont know yet whether or not this will truly accelerate healing or prevent arthritis, but the data is encouraging so far.

Fifth, sleep more. We often cut our sleep short. Then we work and play with fatigue, and wonder why we get hurt. The volume of data suggesting that a minimum of seven to eight hours of sleep per night dramatically affects our testosterone levels, our fitness, our decision making, and our memory storage, is overwhelming. Those last two hours of deep REM sleepwhen we imbed the lessons we learned through the dayare the key hours. Without them, we repeat our mistakes and leave ourselves open to avoidable errors of judgment and action.

So, how do you not get hurt? Be smart. Live smart. Your knees, and everything connected to them, will thank you for it.

DISCLAIMER : Views expressed above are the author's own.

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Preventive Healthcare For The Joints - Economic Times

Skip the mall this Christmas. Your organ-donation consent is the best gift you can give – The Globe and Mail

Margaret Lynch is an MFA student in the creative non-fiction program at the University of Kings College in Halifax.

'Tis the season for gift shopping. That time of year when we flock online, a Pavlovian response to slick advertising. We search shopping malls and stalk special one-day-only sale racks, spending hours seeking the perfect gift. But what if we didnt have to search? What if we already carried the perfect gift within us?

Less than one-quarter of Canadians have registered to donate organs, but 90 per cent of Canadians say they support organ donation. Presumed consent is a potential solution to increase donors, meaning that people need to opt out if they dont want their organs donated. Nova Scotia has already passed this legislation, to take effect in 2020. Alberta, Quebec and Prince Edward Island seem poised to follow suit.

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The decision to donate is complicated, but all I can think about are the eight people whose lives I might save.

I was 30 years old in January, 1988. One minute, I was in my office on a frigid day where skyscraper vents exhaled plumes of white smoke that rearranged themselves against a wintry-grey Toronto sky. The next, I was at a walk-in clinic listening to the young doctor with long, blonde hair speak the words that forever changed my life.

You have acute leukemia, she said. You need to go to a hospital today. At least I had an answer for my sluggish performance on the squash court.

Within an hour, I was admitted to Toronto General Hospital, wheeled into a room with two single beds and hooked up to a bag of blood, my first transfusion. Leukemia is a blood cancer. Abnormal cells produced in the bone marrow suppress the production of normal blood cells. Transfusions are critical for people with this and other blood disorders.

I watched winter unfold like a silent movie projected through the windows of my room. Swirling snow and howling winds raged outside. Inside, there was a sameness to the days. Blood tests and transfusions, doctors and drugs.

From January to June that year, I was transfused almost every second day. Months of chemotherapy decimated my bone marrow, causing collateral damage to my cells. I learned a lot about biology that year. Mustard-yellow platelets helped my blood to clot, so I wouldnt hemorrhage while I waited for my own to recover. Crimson-coloured packed cells increased my hemoglobin and iron levels to improve my bodys oxygenation. Greenish-yellow plasma transported essential nutrients, hormones and proteins throughout my body.

I received 157 units of packed red blood cells, platelets, fresh frozen plasma and albumin. Each unit represented a blood donation from someone I did not know. I am beyond grateful to each and every person who generously donated their blood to me.

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Though blood transfusions kept me alive, clinical trials and chemotherapy had failed to put me in remission. In May, 1988, I received another type of transfusion. On that day, my sisters ruby-red liquid bone marrow hung from an IV pole. Its contents dripped into a central vein in my chest through a Hickman line. I watched it and felt something I hadnt felt in four months: hope.

A bone-marrow transplant is the stuff of science fiction. Bone marrow is the soft tissue inside bones that produces blood-forming stem cells. The stem cells retrieved from my sisters marrow navigated into my own marrow where they regenerated as healthy blood cells. Im always reminded of the submarine crew in the 1966 movie, Fantastic Voyage, who were shrunk to microscopic size and ventured into the body of an injured scientist to repair damage.

Incredibly, the procedure cured my leukemia. To my sister, Mary: Thank you for giving me back my life.

I was lucky in 1988, still early days for the science of bone-marrow transplantation. Much has changed in the intervening years. Today, we know that stem-cell transplants can treat more than eighty diseases and disorders.

Three decades ago, I could only obtain a transplant because Id sourced my own matching sibling donor. Today, less than 25 per cent of people who require transplants will find a matching donor within their family. The rest rely on the generosity of unrelated donors. Canada participates in an international network of stem-cell registries with access to 36 million potential donors worldwide.

Because of my medical history, Im not an eligible blood donor, nor can I register for the stem-cell registry. But I can donate my organs, knowing my gift will be perfect for the person who needs it.

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This year, I skipped the mall. Instead, I registered my consent to help save lives by becoming an organ and tissue donor.

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Skip the mall this Christmas. Your organ-donation consent is the best gift you can give - The Globe and Mail

BrainStorm Cell Therapeutics Wins 2020 ‘Buzz of BIO’ Award – Multiple Sclerosis News Today

For its promising investigational therapeutic approach to neurodegenerative diseases, including progressive multiple sclerosis (MS), BrainStorm Cell Therapeutics is theBuzz of BIO 2020 winnerin the Public Therapeutic Biotech category.

The Buzz of BIO contest identifies U.S. companies with groundbreaking, early-stage potential to improve lives. The event also is anopportunity to make investor connections that could take products to the next phase.

Ten biotechnology companies are nominated in each of the three categories of Buzz of BIO: Public Therapeutic Biotech, Private Therapeutic Biotech, and Diagnostics and Beyond. In the Public Therapeutic Biotech category that BrainStorm won, nominated companies must be actively developing a publicly traded human treatment intended for review by theU.S. Food and Drug Administration.

As a developer of autologous cellular therapies for debilitating neurodegenerative diseases, BrainStorm is testing its investigational therapy,NurOwn, in progressive MS patients, for whom treatment options are limited.

The therapy is based on patients own bone marrow-derived mesenchymal stem cells that are engineered to secrete growth factors. Such factors are thought to protect nerves from damage, promote the repair of myelin (the protective coat of neurons that is destroyed in MS), and ultimately slow or stabilize disease progression.

BrainStorms current open-label Phase 2 clinical study (NCT03799718) is enrolling up to 20 adults with either secondary progressive or primary progressive MS at three U.S. sites:theKeck School of Medicine of USC, the Stanford School of Medicine, and theCleveland Clinic. After undergoing a bone marrow aspiration to collect cells, each participant will receive three intrathecal (injected into the spinal cord) NurOwn cell transplants within 16 weeks, and will be tracked for at least another 12 weeks to assess safety and effectiveness. Contact information for the trial centers is available here.

Thanks to everyone who voted for BrainStorm during the Buzz of BIO competition,Chaim Lebovits, BrainStorm president and CEO, said in a press release.

As the winner of the contest, BrainStorm also was invited to givea presentation at theBio CEO & Investor Conference, to be held Feb. 1011 in New York City.

The entire management team at BrainStorm was very pleased with the results of this competition, and we look forward to presenting to an audience of accredited investors who may benefit from the companys story, said Lebovits. We thank the BIO[Biotechnology Innovation Organization] team for singling out BrainStorms NurOwn as a key technology with the potential to improve lives.

NurOwn cells also are being tested in a Phase 3 trial (NCT03280056) in patients with amyotrophic lateral sclerosis (ALS).

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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BrainStorm Cell Therapeutics Wins 2020 'Buzz of BIO' Award - Multiple Sclerosis News Today

Former Kansas congressman assists with release of U.S. prisoner in Iran – The Garden City Telegram

Former U.S. Rep. Jim Slattery parlayed decades of international relationship-building to assist with gaining freedom of a Chinese-American captive and the release of an Iranian accused of violating trade sanctions.

Slattery, who served an eastern Kansas district in the U.S. House for more than a decade, worked as an attorney and consultant after leaving Congress. He forged relationships along the way that included Majid Ravanchi, a former University of Kansas student now serving as Irans ambassador to the United Nations. In an interview Monday, Slattery said he also was familiar with Iran Foreign Minister Mohammad Javad Zarif.

He contributed to dialogue that led to exchange of Xiyue Wang, a Princeton University doctoral student arrested in Tehran during a research trip, accused of being a spy and incarcerated the past three years, for Massoud Soleimani, an Iranian researcher held since 2018 in the United States on charges of breaking U.S. trade sanctions.

"Bottom line is two families are reunited," Slattery said. "One in Iran and one in Princeton, New Jersey."

On Saturday, the administration of President Donald Trump confirmed the brokered agreement would result in release of Soleimani and Wang. The president said in a statement freeing Americans was of vital importance to his administration and "we will continue to work hard to bring home all our citizens wrongfully held captive overseas."

Slattery, a Democrat engaged for 15 years in outreach projects in Iran, said he was invited to participate by Wang's family attorney. Slattery said he worked pro bono on the project, and told Zarif, the Iranian foreign minister, during a September meeting in New York there was no credible evidence Wang was engaged in espionage.

"I was completely convinced he was not a spy and completely convinced he was being held on bogus charges," Slattery said.

Wang was taken into custody in 2016, convicted on two counts of espionage and sentenced to 10 years in prison in Tehran.

The former Kansas congressman also concluded the U.S. Department of Justice had a weak case against Soleimani, a stem-cell researcher arrested after invited by the Mayo Clinic to come to Minnesota.

The case against him involved exportation of a growth factor used in research to Iran, but Slattery said Soleimani's lawyers could have convincingly argued in court the material was covered by a medical exception to the trade ban.

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Former Kansas congressman assists with release of U.S. prisoner in Iran - The Garden City Telegram

Drugs, Biologics, and Regenerative Medicine in 2019 – The National Law Review

Friday, December 13, 2019

Following up on our first post in this year-end series that discussed medical device regulatory activities at the Food and Drug Administration (FDA), the Mintz FDA teams second year-end post will provide an overview of 2019 with a focus on the drug, biologic, and regenerative medicine programs at the agency. In many ways, the past year could be called a business as usual year for the FDAs drugs and biologics centers in that they continued to make progress on all of large-scale programs and priorities initiated by former-Commissioner Scott Gottlieb, who left the agency in April. FDA has been under the leadership of an Acting Commissioner since that time, although Texas radiation oncologist Dr. Stephen Hahn will be taking the reins soon following his confirmation by the full Senate in a 72-18 vote on December 12, 2019. (The Senate HELP Committee advanced the nominee on December 3, 2019; see our blog post just prior to that committee vote here.)

At the same time, however, the final months of 2019 have exposed several challenges for various FDA programs that operate under the extensive drug and biologic authorities contained in the Food Drug & Cosmetic Act (FD&C Act) and the Public Health Service Act (PHS Act), respectively. The agency will be forced to grapple with many of these issues directly and deliberately in 2020as a result of deadlines of the agencys own making as well as external pressures coming from other parts of D.C. and from the rapidly changing nature of the U.S. health care system.

According to data presented by the Office of New Drugs in early December (see ONDs slides here), FDA had another extremely productive year when it comes to its approval of new molecular entities that address a unique blend of therapeutic areas. The agency approved 45 new molecular entities in FY 2019 (October1, 2018 to September30, 2019), of which 71%, or 32 products, received priority review status and 23 were designated as orphan drugs intended to treat rare diseases. As OND emphasized in the presentation, several of those new product approvals are notable for their uniqueness and therefore, in the agencys view, 2019 reflects not only quantity but [also] quality. The OND presentation also highlights a significant amount of other information on new molecular entity approvals and may be of interest to those readers who want to take a deeper dive into the data.

In addition to advancing important new drugs and biologics to market, former Commissioner Gottlieb is well-known for having spearheaded to development of a Drug Competition Action Plan (DCAP) and a Biosimilars Action Plan (BAP) during his nearly two-year tenure as head of the agency. Some of our prior coverage of the DCAP and BAP is available here. In general terms, the DCAP encourages market competition for generic drugs and helps to bring greater efficiency and transparency to the generic drug review process; the BAP aims to achieve similar goals for biosimilar products as the agency continues its implementation of the 2010 Biologics Price Competition and Innovation Act (BPCIA), including the critical drug-to-biologic transition that will occur by operation of law in March of 2020.

FDA continued to make progress on its various goals under these two initiatives during 2019. Some examples of this progress can be captured with these two data points:

FDA approved 1,171 generic drugs (935 full approvals and 236 tentative approvals) in the last fiscal year. Notably, 125 drug approvals were for first generics of medicines that had no direct generic competition. The agency released a report in October 2019 noting that generic drug approvals reached a record high in FY 2019 and also that several complex generic drug products had been approved for the first time. Under the DCAP, the agency has prioritized getting complex generic drug products to market with the goal of increasing competition and thereby reducing commercial prices for such products, which may be expensive due to difficult manufacturing processes.

On December 6, 2019, FDA licensed its 26th biosimilar product since the BPCIA was enacted and the 10th biosimilar this year (Amgens Avsola (infliximab-axxq), which is a biosimilar to Remicade and the fourth Remicade biosimilar approved by the agency to date). Although thirteen of the previous 25 approved biosimilars have yet to launch due to ongoing patent litigation or for business reasons, the agency has continued to focus on positive developments in the still-nascent U.S. biosimilars market, including with the issuance of a public statement on November 15 in conjunction with approval of the 25th biosimilar (see here).

With all of these wins, however, FDA still faces its fair share of challenges related to its expedited drug and biologic programs, especially as it appears to have accelerated its review of products intended for diseases with an unmet need to breakneck speed. Indeed, the OND presentation from early December also added that for FY 2020 and as of November 21, 2019, the agency had already approved 13 such drugs, suggesting that next year could be a record-breaking one in terms of innovative drug product approvals. A recent Bloomberg Law article (available here) used the phrase breakneck speed to describe the agencys actions in this space based on several recent FDA approvals of new molecular entities that have come months in advance of their assigned target dates. The Bloomberg Law article highlights that in response to FDAs speediness in reaching approval decisions on new drugs and biologics for diseases with unmet needs, patient advocates and, increasingly, insurers that have to pay for those treatments are starting to raise concerns that these products lack sufficient safety or effectiveness data.

Relatedly, there has been increasing pressure on FDA to remove certain accelerated approval drugs from the market following a failure by the drug product sponsor to confirm the efficacy or clinical benefits of the product in the required post-marketing confirmatory clinical trial. The most visible example of this regulatory challenge came in October 2019 when FDA convened an advisory committee to recommend whether it should withdraw accelerated approval from hydroxyprogesterone caproate injection (marketed under the brand name Makena for the prevention of preterm birth in pregnant women). FDAs Bone, Reproductive and Urologic Drugs Advisory Committee voted 9-7 to withdraw approval, with the dissenters favoring leaving Makena on the market while requiring the sponsor to conduct a new confirmatory trial. (Notably, no one voted for the option of leaving it on the market without requiring a new confirmatory trial.)Among the concerns of some members who voted to leave the product on the market with a new clinical trial obligation was that the drugs withdrawal would leave no safe treatment options for pregnant women at high risk of preterm birth. FDA will have to make a final decision regarding what to do about Makena in 2020, and it undoubtedly will face intense criticism (and potentially legal challenge) no matter what route it chooses to take for this public health quandary in which it finds itself.

In a similar vein, FDA official Dr. Richard Pazdur participated in a Senate briefing on December 10, 2019, in which he and other speakers defended the Breakthrough Therapy Designation program. FDA insisted again that the designation was intended to let the agency have earlier interactions with drug sponsorsand that it was not meant to be an early rating system for drugs or a signal of how they might do commercially. Given that the Breakthrough program was created in 2012 and is considered to be wildly successful, some speakers at the briefing expressed surprise that there was still any confusion about its purpose and function. Whether Congress picks up any of these emerging areas for consideration as part of FDAs 2022 user fee reauthorization packages remains to be seenas those negotiations will begin in earnest after the New Year, but the issues certainly are complex enough to allow for robust policy discussions to occur.

Finally, there are expected to be bumps in the road with the upcoming March 2020 transition of proteins previously approved under New Drug Applications (NDAs) to Biologics License Applications (BLAs)for which FDA only finalized its guidance for industry last year. The March 2020 transition date was established under the BPCIA and the agency does not have discretion in getting the transition done (only in how it handles the logistical and administrative issues created by transitioning approved products in this way). Check out our prior blog post on the final deemed to be a license transition guidance.

FDA guidance documents for all regulated product categories continued to be released on a regular basis this year, including several related to areas of agency priorities under the DCAP and BAP including the final biosimilar interchangeability guidance issued in May (see our blog post here) and a draft guidance on insulin interchangeability issued in November 2019. The latter also relates to the March 2020 NDA-to-BLA transition, as insulins are one of the largest class of products that will be transitioning into regulation as biologics, making them open to what is expected to be more efficient competition through the BPCIAs biosimilar pathway than what was possible in the past as insulin NDAs.

Despite the accelerated pace of the issuance of Agency guidance, however, FDA is beginning to face more challenges related to its decision-making and the scope of its exercise of agency discretion. In particular, a significant District Court for the District of Columbia ruling issued on December 6, 2019, Genus Medical Technologies, LLC v. FDA, provides hints of a potential shift in judicial deference to certain agency actions. The court vacated FDAs classification of a medical imaging liquid as a drug rather than as a device after determining that FDA did not have discretion to decide how to regulate a product merely because the definitions of drug and device overlap in the FD&C Act.

According to the district court judge, FDA was not interpreting the drug/device definitions in the statute properly, and Congress did not intend to allow the agency unfettered discretion to pick between the two categories. Rather, the court found that the text of the definitions are clear and do not create a gap or any ambiguity for FDA to fill with an exercise of agency discretion. This recently issued decision may indicate a potential shift in how courts are going to apply long-standing precedents related to judicial deference to agency decisions. If FDA decides to appeal the Genus ruling, it may end up at the Supreme Court as one of many expected challenges to the doctrines that established our current framework for judicial deference of an administrative agencys interpretation of an ambiguous statute.

One of the first FDA press releases for 2019 was co-authored by former Commissioner Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks and was focused on the agencys new policies aiming to advance the development of safe and effective cell and gene therapies. In the press release, the agency leaders predicted that by 2020, FDA would receive more than 200 Investigational New Drug Applications (INDs) for cell and gene therapies each year. The agency has continued to work diligently to increase its staff in CBER to conduct clinical reviews for such INDs and to try to keep pace with the industrys development of these innovative technologies.

As we discussed in our update on FDAs Comprehensive Regenerative Medicine Policy Framework earlier this year, the agency is prioritizing two parallel goals: (1) clarifying the regulatory criteria for product marketing and providing support and guidance to legitimate product developers; and (2) removing unapproved, unproven, and potentially unsafe products from the U.S. market. The second prong of this comprehensive plan for regenerative medicine products was the topic of one of Dr. Gottliebs very last statements as Commissioner before he left the agency, issued on April 3, 2019 in conjunction with CBER Director Dr. Marks, indicating how important this area is to the agencys current public health priorities.

In the April 2019 statement, Drs. Gottlieb and Marks acknowledged FDAs challenges and efforts to stop stem cell clinics and manufacturers from marketing unapproved products that put patients at risk, citing several Warning Letters issued to manufacturers that violated current good manufacturing practices (CGMPs) for human cells and tissue products. They noted that it was of particular concern given that the industry was nearly halfway through the period during which the FDA intends to exercise enforcement discretion for certain regenerative medicine products with respect to INDs and premarket approval requirements. Now that it is December 2019, that deadline is even closer with less than one year left. November 2020 is the end of the three-year period of enforcement discretion announced by FDA when it first articulated the policies and goals of this comprehensive framework in 2017. See our prior posts on the topic here and here.

Under the Comprehensive Regenerative Medicine Policy Framework, FDA appears to have stepped up the pace of issuing Warning and Untitled Letters to sellers of unapproved stem cell products during the second half of 2019. In conjunction with a Warning Letter issued on December 5, 2019 to two related companies for processing and marketing unapproved umbilical cord blood-derived cellular products, Dr. Marks of CBER reiterated the agencys concerns about safety and reminded the public of the upcoming compliance deadline: As evidenced by the number of actions that the agency has taken this month alone, there are still many companies that have failed to come into compliance with the [FD&C Act] and FDAs regulations.

Dr. Marks was referring to two Untitled Letters that were issued to stem cell product distributors on November 20 and November 25, 2019, respectively. The press release cited above also added that the agency had also recently sent 20 letters to manufacturers and health care providers noting that it has come to [FDAs] attention that they may be offering unapproved stem cell products, reiterating the FDAs compliance and enforcement policy.

FDA also prevailed this year in the U.S. District Court of the Southern District of Florida against a stem cell clinic charged with violating the FD&C Act and the PHS Act. In June 2019, the court held that the defendants adulterated and misbranded a stem cell drug product made from a patients adipose tissue without FDA approval and for significant deviations from CGMPs, issuing a permanent injunction as requested by the Department of Justice on FDAs behalf. The agencys statement on that important court win by the government is available here.

Lastly, on December 6, 2019, FDA issued a Public Safety Notification on Exosome Products. The safety notification informed the public of multiple recent reports of serious adverse events experienced by patients in Nebraska who were treated with unapproved products marketed as containing exosomes, which came to FDAs attention through the Centers for Disease Control and Prevention, the Nebraska Department of Health and Human Services, and others. There are currently no FDA-approved exosome products and, to be honest, we are not even sure what such a product would be since an exome consists of all the sequenced exons within a single human genome after the introns are removed. (So were the clinics administering complete exomes to patients? that seems unlikely.)But what we found noteworthy about this public safety notice is the forceful and direct language FDA used when describing the unscrupulous conduct of the sellers of these products:

Certain clinics across the country, including some that manufacture or market violative stem cell products, are now also offering exosome products to patients. They deceive patients with unsubstantiated claims about the potential for these products to prevent, treat or cure various diseases or conditions. They may claim that they these products do not fall under the regulatory provisions for drugs and biological products that is simply untrue. As a general matter, exosomes used to treat diseases and conditions in humans are regulated as drugs and biological products under the [PHS Act] and the [FD&C Act] and are subject to premarket review and approval requirements.

The clinics currently offering these products outside of FDAs review process are taking advantage of patients and flouting federal statutes and FDA regulations. This ultimately puts at risk the very patients that these clinics claim to want to help, by either delaying treatment with legitimate and scientifically sound treatment options, or worse, posing harm to patients, as evidenced by these recent reports of adverse events.

As we enter the final year of FDAs enforcement discretion period, perhaps these public notices and Warning/Untitled Letters will become even more frequent and the agency will become even more frustrated by the ongoing violations and medical practitioners who flout federal law. This area will see substantial activity in 2020 and we will be watching closely to see what changes, if anything, about FDAs approach in November when the deadline to come into compliance ends. Will there be widespread FBI raids on stem cell clinics engaged in this kind of bad behavior? Only time will tell.

Although we have highlighted what we view as some important challenges for the FDA to address in the coming months, other areas continue to be business as usual without anything very new to report. FDA continues to invest significant resources into improving the quality of compounded drugs and ensuring compliance with Sections 503A and 503B of the FD&C Act. Drug compounding was another topic of one of Dr. Gottliebs very last statements as Commissioner on April 3, 2019, in which he laid out the 2019 compounding priorities that included maintaining quality manufacturing and compliance and regulating compounding from bulk drug substances.

Notwithstanding all the efforts by FDA and State regulators in this area over the past several years, the agency continues to see concerning activity when it comes to compounded drugs, such as problems related to the condition under which compounded sterile medicines are made, which raisesignificant risks to patients. As a result, FDA has made it an intense area of focus to take enforcement actions against compounders who fail to produce sterile drugs in compliance with the law. During the past year, for example, FDA has won at least four permanent injunctions against various compounders after the agency identified behavior that posed a significant risk to public health and safety.

In addition, in 2019 FDA also increased its activities towards reducing and mitigating the impact of drug shortages on the health care system. See our prior blog post on Drug Shortages.

Finally, after a fairly slow year of enforcement in the prescription drug advertising space, the last two months of 2019, at least as of December 12th, have given us three (!) letters two untitled and one warning from the FDAs Office of Prescription Drug Promotion (OPDP). Most interestingly, the Warning Letter issued on December 2, 2019 for omitting warnings about the most serious risks associated with [a medication-assisted treatment] drug from promotional materials was announced to the public via FDA press release, which is not a typical action for normal-course OPDP letters to industry. The drug in question, approved for the prevention of relapse to opioid dependence following opioid detoxification, is associated with several significant risks including potential opioid overdose. Given the countrys public health emergency that is the opioid epidemic, FDA appears to have felt the need to make the deficiencies in the advertisement and those risks more widely publicized. So another thing we will be watching for in the New Year is whether this OPDP action represents the beginning of a new trend by the agency to publicize these Warning Letters more directly, or whether its advertising enforcement activities may be picking up due to industrys evolving approaches to promoting therapeutic products.

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Drugs, Biologics, and Regenerative Medicine in 2019 - The National Law Review

Jasper Therapeutics Launches with $35 Million Series A Financing to Develop and Commercialize Innovative Conditioning Agents and Therapies to…

PALO ALTO, Calif.--(BUSINESS WIRE)-- Jasper Therapeutics, Inc., a new biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies, today announced the launch of the company with a $35 million total Series A financing. Abingworth LLP and Qiming Venture Partners USA served as lead investors, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC. The proceeds will be used to advance the clinical development of the companys lead product candidate, JSP191, which is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplant.

Jaspers development of JSP191 is also supported by a collaboration with the California Institute for Regenerative Medicine (CIRM), which has been funding the program and is committed to providing a total of $23 million in grant support. As part of the Series A financing, Amgen, which discovered JSP191 (formerly AMG191), has licensed worldwide rights to Jasper that also include translational science and materials from Stanford University.

Jasper was co-founded by Judith Shizuru, M.D., Ph.D., a hematopoietic stem cell transplant expert at Stanford University, and Susan Prohaska, Ph.D., a Stanford University-trained immunologist, stem cell biologist and early-stage drug development professional. Dr Shizurus CIRM-funded lab advanced the understanding of the ability of anti-CD117 to impact hematopoietic stem cells and, together with the Lucile Packard Childrens Hospital Stanford and University of California, San Francisco (UCSF) pediatric transplant teams, was the first to study an anti-CD117 antibody in the clinic as a conditioning agent. That humanized antibody, now called JSP191, was first studied for conditioning for transplant in immune-deficient patients in collaboration with Amgen, UCSF and CIRM.

Stem cell transplantation is a potential curative therapy for people with hematologic cancers, autoimmune diseases, and debilitating genetic diseases. However, the pre-transplant conditioning required to prepare patients for transplant involves highly toxic chemotherapy, which can be life-threatening and limits the number of people who are able to benefit, said Dr. Shizuru, co-founder and member of the Board of Directors of Jasper Therapeutics. JSP191 is the only anti-CD117 antibody to demonstrate safety and efficacy in severely ill patients receiving stem cell transplant in the clinic. We plan to expand clinical development to patients receiving transplants for acute myeloid leukemia/ myelodysplastic syndrome or autoimmune diseases and to patients receiving stem cell-directed gene therapies.

Dr. Shizuru added, With an experienced executive team of biotech veterans and a strong syndicate of healthcare-focused investors, Jasper Therapeutics is well positioned to achieve our vision of building a leading biotech company starting with JSP191 and expanding to other novel therapies for immune modulation, graft engineering and cell and gene therapies.

JSP191 is currently being evaluated in an ongoing Phase 1 clinical trial as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that failed. This severe genetic immune disorder leaves patients without a functioning immune system. Interim results of the study will be presented in an oral presentation (abstract #800) on Monday, December 9, at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Fla. Clinical studies to evaluate the safety and efficacy of JSP191 as a conditioning agent in patients undergoing hematopoietic cell therapy for hematologic cancers are planned for 2020.

Founding Management Team

Dr. Shizuru and Mr. Lis are joined on the Jasper Therapeutics Board of Directors by Kurt von Emster, Managing Partner of Abingworth LLP, and Anna French, Ph.D., Principal at Qiming Venture Partners USA. Dr. Prohaska is a Board observer.

With our investment in this program, were able to realize our mission of fast-tracking stem cell treatments by helping academic researchers rapidly advance the most promising discoveries in the lab into the clinics and to drug development with commercialization partners, said Maria T. Millan, M.D., President and CEO of CIRM. Jaspers two co-founders took a novel antibody with unique properties and moved it from the bench to the bedside relatively quickly, and were thrilled to partner with this talented team to potentially impact a broad group of people who could benefit from stem cell therapy.

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow is currently achieved by subjecting patients to toxic doses of radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191

JSP191 (formerly AMG191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1 dose-escalation trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

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Jasper Therapeutics Launches with $35 Million Series A Financing to Develop and Commercialize Innovative Conditioning Agents and Therapies to...

One of Wall Street’s most high-profile hedge funds pushes Alexion’s CEO to the auction block and he’s not budging – Endpoints News

Exhilarating. A major advance. A milestone achievement. If one had just tuned into the panel comments on Biogens presentation at CTAD, it would seem that the biotech had an impressive, disease-modifying Alzheimers drug in aducanumab.

But off the stage, reactions to their admittedly complicated dataset and the biotechs explanation for resurrecting a drug that failed its futility analysis were a lot more mixed, with analysts continuing to question whether the evidence is substantial enough to warrant an FDA approval and raising new doubts on the safety side.

In an investor call later in the day, execs noted that they are not planning another study and stood by their intention, publicized in October to much surprise, to submit regulatory filings based on what they have.

We dont file willy nilly, said Al Sandrock, head of R&D. We only go to filing when we believe that there is a benefit-risk argument based on science, based on data. And if you look at our history, we havent done filings right and left without good reason.

Biogen had a theory going into the Clinical Trials on Alzheimers Disease meeting.

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One of Wall Street's most high-profile hedge funds pushes Alexion's CEO to the auction block and he's not budging - Endpoints News