Makana In The News

On a Mission to Revolutionize Transplantation

At the heart of Makana’s mission lies a deep-rooted commitment to addressing one of the most pressing challenges in healthcare: the shortage of donor organs. For decades, patients facing organ failure have been confined to lengthy waiting lists, their lives hanging in the balance as they await a transplant that may never come. When you consider the waiting list is over 100,000 deep in the United States alone, the hurdle becomes even more critical to overcome. Dr. Joe Tector, the visionary mind behind Makana, recognized the urgency of this issue and embarked on a quest to find a solution. Drawing inspiration from his experiences as a transplant surgeon and fueled by the memory of landmark transplant cases, Dr. Tector set out to pioneer a new approach to organ transplantation—one that would minimize the need for human donors and provide a limitless supply of organs to those in need.

A Pioneering Approach to Xenotransplantation:

The journey of Makana began in the halls of academia, where Dr. Tector’s academic lab at Indiana University became the birthplace of groundbreaking research in xenotransplantation. Collaborating with a team of brilliant scientists and researchers, Dr. Tector delved into the complexities of transplant biology and genetic engineering, seeking to unlock the secrets of cross-species organ compatibility with pig kidneys. Through years of tireless experimentation and innovation, Makana’s team made significant strides in overcoming the immunological barriers that had long hindered the success of xenotransplantation. Just three genes stood in the way of success. By harnessing the power of genetic engineering, Makana succeeded in developing non-human pig organs that could successfully integrate with the human body, offering new hope to patients who had previously been deemed untreatable. Decades of research resulted in this groundbreaking negative cross-match with human recipients— a sign that the transplant could be successful in the human host. Specifically, Makana’s triple knockout pig represents a significant breakthrough, offering hope to countless individuals languishing on transplant waiting lists.

Having overcome numerous hurdles in the past, many remain on the road ahead. Makana and its team are driven to achieve these goals, while operating on a ‘no Plan B’ approach which adds an extra element of pressure to goal achievement. Aiding in this progress is the research team in Miami, which provides the perfect testbed for groundbreaking xenotransplantation.

Makana Leading Miami’s Emerging Hub for Biotech Innovation

Nestled in the vibrant ecosystem of Miami, Makana epitomizes Florida’s burgeoning prominence in the biotech landscape. The Daytona Examiner sat down with Mark Platt, President and CEO of Makana Therapeutics, to discuss the company’s recent strides. Our chat with Mark underscored the strategic decision to establish Makana’s headquarters in Miami, leveraging proximity to the esteemed Miami Transplant Institute and the University of Miami.

“We’re a scrappy biotech company that has a lot of room for growth, obviously, but that’s why we’re in Florida, and it’s been a great environment down here in Miami.” said Mark Platt.

This strategic positioning facilitates collaboration with leading experts and institutions in the field, fostering a dynamic environment conducive to innovation and growth. Makana’s presence in Miami not only accelerates the pace of research and development but also augurs well for the region’s emergence as a hub for biotech innovation. As the company continues to chart new frontiers in organ transplantation, its footprint in Miami solidifies the city’s standing as a beacon of scientific advancement and economic prosperity.

Makana’s continued push towards the boundaries of medical science is the realization of latent biotech power. Given Makana’s proximity to their xenotransplantation goals, it is easy to imagine the growth and notoriety the brand will pick up, but also its dominance in the market for non-human organ supply. This impact reverberates far beyond the confines of the laboratory. Through strategic partnerships with esteemed institutions such as the Miami Transplant Institute and the University of Miami, Makana has cemented its position as a leader in the burgeoning biotech landscape of Miami. This strategic positioning not only accelerates the pace of research and development but also underscores Miami’s emergence as a hub for biotech innovation. As Makana’s team of scientists, researchers, and clinicians work tirelessly to advance the field of organ transplantation, they are not only changing the lives of individual patients but also reshaping the future of healthcare on a global scale.

Ethical Considerations and Regulatory Frameworks

While Makana’s advancements in xenotransplantation hold immense promise, they also raise important ethical considerations and regulatory challenges. The concept of transplanting organs from non-human species into humans prompts discussions about animal welfare, potential risks of disease transmission, and the long-term implications for both recipients and society as a whole. Makana recognizes the importance of addressing these concerns responsibly and transparently. The company collaborates closely with regulatory authorities, bioethicists, and other stakeholders to ensure that its research and clinical trials adhere to the highest ethical standards and safety protocols, while offering superb conditions for animals that go far beyond the regulatory standards. By fostering open dialogue and engaging with diverse perspectives, Makana aims to navigate the complex ethical landscape surrounding xenotransplantation while maximizing the potential benefits for patients in need.

A Singular Mission: Saving Lives Through Innovation

At the heart of Makana’s endeavors lies a singular mission: to restore hope and transform lives through groundbreaking innovation. Dr. Joe Tector and Mark Platt’s unwavering commitment to this mission reflects Makana’s ethos of perseverance and purpose. With such a focus on delivering life-saving therapies to patients in need, Makana navigates the complex landscape of regulatory hurdles and scientific challenges with steadfast resolve. Every milestone achieved in the company’s journey brings renewed hope to individuals grappling with organ failure, underscoring the profound impact of Makana’s pioneering work.

When you consider the sheer number of people on the transplant list domestically, and even abroad, it becomes hard to quantify the impact of Makana successfully commercializing this product. Assuming success in moving to a clinical trial and eventually into widespread use, hundreds of thousands of lives can be saved— lives that will go on to change the world, leaving legacies that too will shape the future of humanity. No words can effectively underscore and define this enduring impact. As the company prepares to embark on clinical trials and bring its transformative therapies to fruition, Makana stands poised to usher in a new era of hope and healing for patients worldwide.

Makana is leading the charge towards a future where organ transplantation is no longer a last resort but a readily available option for those in need. Offering a preview into the future, Mark discussed subsequent organ product expansions into the even-rarer human organs that too have long waiting lists. All of these life changing, and life extending organs are predicated on the success of their pig kidney xenotransplantation moving to a human clinical trial. As the world watches with bated breath, Makana continues to push the boundaries of what’s possible, reaffirming its position at the vanguard of medical innovation and inspiring hope for a brighter, healthier future for all.

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Dr. Joseph Tector has spent decades working on solving the organ shortage problem. For the past 30 years, he has been making a seemingly impossible dream possible through his groundbreaking work in xenotransplantation, transplanting organs from animals to humans. As a surgeon at the Miami Transplant Institute and the founder of Makana Therapeutics, Dr. Tector is raising gene-edited pigs to provide an abundant supply of organs with lower rates of organ rejection to be transplanted into humans in need. He is determined to revolutionize the field of transplant medicine and give hope to millions of families around the world.

Surgeons are sometimes seen as the mechanics of the medical profession, so confident in their craft that there is no room to deviate from a well-trodden path. Dr. Tector has a surgeon’s telltale confidence. Unlike many surgeons, however, Dr. Tector’s tolerance for the unknown is a rare specimen in the medical field. To advance a vision like xenotransplantation means having the stamina to pursue an end goal that will be at least forty years in the making, if indeed it happens at all. I was inspired to chat with him how a surgeon’s frustration led him to dedicate his life to a solution that is redefining transplant care.

Dr. Tector, you are an experienced organ transplant surgeon. Tell me what led you to start a company focused on the pioneering research of xenotransplantation?

A transplant is enormously successful for patients, if they can get it. The reality is that the shortage of organs is a major barrier. Many people who need it don’t receive it. The technology of creating pigs as organ donors is a solution, and we started off by learning how to make those pigs. And then we learned that it wasn’t going to be feasible to get organs out of a university lab to those who need them, so we founded Makana Therapeutics.

As you’ve built this company and pursued xenotransplantation research at the Miami Transplant Institute. Talk to me about the hurdles you have had to overcome. What were the challenges you faced early on and how has that evolved as you’ve made progress?

As a surgeon, going through training, we are told to get a good mentor and follow a path. I was more project driven, I wanted to make xenotransplantation, this particular thing, happen. People would say that this is so far into the future, this is never going to happen. Then, the next obstacle was how we’d pay for it. Overall, the first barrier is to take down preconceived notions like these.

It was daunting for a surgeon. But, after making progress, it goes from nobody understanding what needs to happen to everyone understanding, and then people want to tell you what to do next. It can be very distracting, so you need to remain focused. A surgeon is focused on outcomes, so maybe that was my advantage. It’s a lot of work. And it never gets to the point where I say “this is easy, so now I can pick up some hobbies.” It takes a lot of persistence and focus.

We know that in organ transplants, waitlists are too long for everyone but women are waiting longer on average than men. Women are nearly 9 percent more likely to die when waiting for a kidneytransplant, 24 percent more likely while waiting for a liver transplant and 50 percent more likely when waiting for a lung transplant. Have you seen the human side of this gender disparity firsthand as a surgeon?

Those facts are real and upsetting. There are a couple of challenges that make it worse. Women are generally smaller than men. When a donor becomes available, if it is a large male, it can’t be transplanted to a small female. We don’t account for the time it takes for the next available organ for a specific individual when thinking about transplant lists. Xenotransplantation can eliminate this problem – because pigs come in all sizes.

Also, there is a very telling statistic on the scarcity from human donors today. On average, out of 1,000 deaths of people who have signed a donor card, only about 3 will have died in a manner that makes them suitable donors. We’ll never have enough organs from human donors even if everyone pledged to donate. Xenotransplantation is the logical solution.

I’ve read that pigs share 98% of genes with humans, which makes them potentially ideal animals for donating organs to humans. But have you seen resistance from a societal point of view to the notion of pigs being used in this way?

To the contrary, I’ve been impressed with how well people have received this. In the late 2000s, there was almost a frenzy around xenotransplantation. We have been coming along slowly on the scientific side, and people have seen the incremental steps. Surgeons have already been using submucosa pig tissue for meshes and heart valves with great success for years. It’s already a reality.

Explain to me in lay terms what the gene editing process is about and why it is such a big part of the research you have been doing.

It is driven by reducing pig organ rejection in the human body. If you look at cross-species transplantation, what has kept us from doing this successfully are antibodies in the host species that bind to cells from the transplanted organ. We are taking out the “gene scissors” and we are editing the pig’s DNA, so that the pig cells appear more similar to a human. One major advance in the past 10-12 years is the development of these so-called “gene scissors”. The creation of CRISPRS have allowed us to make gene edits in a very efficient manner. Our pigs have 3 genes knocked out, so there is a lot less antibody binding and this should mean greater success for the patient.

So, this gene editing process not only makes the organs more compatible but may help with some of the other concerns around communicable diseases. What else are you doing to ensure the health and wellness of future patients?

If there was a big concern, it is possible you can use gene editing to help with that. As it pertains to pigs and humans, pigs present a very low risk. Gene editing is in its infancy. As we introduce this into the clinic, there are many things that are considered. For regulatory concerns, the FDA (Food and Drug Administration) needs to get comfortable. You don’t want an organ that doesn’t have any immune capabilities. You need the organ to participate in the immune process.

As we’ve seen time and again with companies pushing boundaries in any field, regulators and lawmakers are often playing catch up. Have you been working with regulators like the FDA as you’ve been building and researching?

We have been working with the FDA. We presented our basic approach with the pig we intend to use, and what pathogen testing we are going to do to prevent any infections. They evaluated the barrier facility, immunosuppression we are going to do, and our preclinical data. They gave us one additional study to do before we can start a pilot trial.

That’s exciting to hear you are so close to clinical trials.

Yes, we are.

So, your passion is getting to the day where transplant surgeons can schedule a xenotransplant for a patient with kidney failure, or liver failure, or heart failure, well in advance, with adequate preparation and plenty of perfected organs ready to use. How far off is that day, in your best estimate?

As I said, we are very close to clinical implementation. This is 40 years in the making. I do think that the situation you described will happen in my lifetime. If you were to ask me: do you have any idea what your next pig is after this triple knock out? I’d say, yes. It is going to be a slow entry, but if we are methodical about the future, the progress will speed up rapidly.

How do you stay committed to your mission? What motivates you?

It has possessed my entire being. I am married and I have 5 kids. But this has been soul possessing. If you see what transplant can do for an individual and for families, it is transformative. My dad was my role model and gave me so much, but having been a surgeon has been a huge driver for me. There are parts of this journey that people would tell me about, bad things, that I just didn’t see. My inability to see those bad things allowed me to keep going. Ultimately, there is a scientific goal and a solution for patients here, and that’s been the focus.

Talk to me about being a surgeon and a founder. How did you manage? Both jobs are extremely demanding.

That has been an evolution, There were times when it was unbelievably hard to do both. But ultimately my clinical practice informed what we were doing which drove our key decisions. We have a fantastic CEO at Makana, Mark Platt. In the areas that I am helpful, he wants my input, and then I am cognizant that there are parts of the business that I shouldn’t be involved in. My team in Miami is incredible and they are supportive. They can clearly see how we are changing things for the better. That’s what you want in a team.

For more information on Dr. Joseph Tector’s xenotransplantation research at Makana Therapeutics, click here.

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