CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia



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New Therapies Advance For Rare Blood Cancer Polycythemia Vera

Emerging treatments like rusfertide may reduce phlebotomies and improve life for patients with polycythemia vera, according to Dr. Aaron Gerds.

Dr. Aaron Gerds, assistant professor of medicine at Cleveland Clinic Taussig Cancer Institute, emphasized the growing momentum in treatment development for polycythemia vera, a rare blood cancer. While the disease may not receive widespread attention, he noted that researchers are actively pursuing new therapies, including rusfertide and other agents targeting the hepcidin pathway.

Rusfertide, in particular, may reduce or eliminate the need for phlebotomies, potentially improving patients' quality of life. Gerds highlighted that multiple new therapies are being studied — such as givinostat in a phase 3 trial — and stressed that even in rare diseases, progress is being made.

What would you like patients to take away from today's conversation?

There are a couple of important things to note. One is that we are developing — and continue to develop — new therapies for polycythemia vera. Even though it's a rare disease and not often highlighted in the broader pantheon of diseases presented at ASCO, it's still a very important condition, especially for the people who have it.

There is a group of us who are very invested in this disease and are working hard to develop new therapies for it. Rusfertide isn't the only one in development — there are also other drugs targeting the hepcidin pathway, as well as agents outside that pathway. For example, givinostat is currently in a randomized phase 3 trial as well as a registration-type study, also seeking approval.

Even though it's rare, we are working hard to develop better treatment options. That's the most important takeaway.

Specifically, with rusfertide, it's exciting because it's a new therapy that reduces or even eliminates the need for phlebotomies. That can really improve patients' quality of life, which is important. Having another tool in our arsenal to care for these patients is exciting and can offer a lot to our patients.

Transcript has been edited for clarity and conciseness.

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Disease Management Decisions In Patients With Low-Risk Polycythemia Vera

While treatment options may be fairly straightforward for high-risk patients with polycythemia vera, it's less clear for patients who are considered low risk, explained Jennifer Vaughn, MD.

Patients with polycythemia vera (PV) fall on a spectrum of risk for thrombotic events, which can be one of the factors that comes into play when choosing therapy, explained Jennifer Vaughn, MD, hematologist-oncologist and assistant professor in the Division of Hematology at The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute. However, management is less clear when the patient is considered low risk.

Vaughn recently spoke about the topic at The American Journal of Managed Care®'s Cleveland Regional Institute for Value-Based Care® (IVBM) meeting.

This transcript has been lightly edited for clarity.

Transcript

At the Cleveland IVBM, you discussed treatment options for polycythemia

vera and how a patient might choose between them. Can you discuss that decision making process and what they're weighing?

First and foremost, anytime you're meeting a patient with PV, you want to decide where they fall on the spectrum of risk for developing thrombotic events. We know that patients who fit into the high-risk category for PV—whether that be because they're over age 60 or because they've had a prior thrombotic event—they are recommended to receive some sort of cytoreductive therapy in addition to phlebotomy and aspirin and possibly even anticoagulation, if they've ever had a venous thrombotic event in the past. That's kind of the easy portion of that question, because we know that those patients are just a general higher risk, probably due to sort of the aging process, other comorbidities, etc, that warrants additional treatment.

However, what's really an evolving science is, how do we manage patients who've been into the low-risk category? Traditionally, those patients have been younger. They've never had a thrombotic event, and we have focused on treating them with phlebotomy and aspirin exactly as you said, because it's felt to be less invasive—I guess that's the wrong word—less expensive, for sure, and perhaps easier on the patient, but really, the goals of phlebotomy are to reduce the red blood cell count and help the blood to flow better, so that their risk of blood clotting is reduced. What we do with phlebotomy is we introduce a little bit of iron deficiency in those patients, so that you're basically depriving the bone marrow of the building blocks it needs to be able to make red blood cells.

That's all well and good if you can do that, it just in just the right way. But eventually patients will become symptomatic from having iron deficiency, and more and more attention is being given to the time that patients are required to spend seeking out therapeutic phlebotomy as a routine part of their treatment. If you're a younger patient, and you are working, you have children you have to take care of, just general things in life that you have to balance, then taking time to go to the clinic to receive phlebotomy is actually a detriment in many ways. I've actually had patients who have inquired about cytoreductive therapy because they felt like it was too burdensome for them to be able to take the time they needed to be able to get the routine phlebotomies and maintain that hematocrit below the goal level of 45%.

There's also a new and involving literature that early treatment with certain drugs, perhaps interferon therapies, could potentially lead to a reduction in the detectable JAK2 [Janus kinase 2] allele burden. And while we don't have really rigorous data to confirm this, the hope is that by reducing the detectable JAK2 allele burden—meaning we're reducing the detectable number of mutated cells in the blood—perhaps we are, in the long term, going to reduce that patient's risk of developing progression to a more serious hematologic illness in the future. I say that very, very cautiously, because it's really mostly an active area of research right now, because patients do live a really long time and take a long time to progress with polycythemia vera, if they progress at all. It's really hard to get that concrete data to sort of guide our management that way.

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Ionis Pharmaceuticals Partners With Japan-Based Ono ... - Benzinga

On Tuesday, Ionis Pharmaceuticals Inc IONS and Ono Pharmaceutical Co., Ltd entered into a license agreement in which Ono obtains exclusive global rights for the development and commercialization of sapablursen, an investigational RNA-targeted medicine for polycythemia vera (PV).

PV is a rare hematologic disease characterized by the overproduction of red blood cells, significantly increasing the risk of serious blood clots, especially in critical organs like the lungs, heart and brain.

Sapablursen is currently being evaluated in adults living with PV in the fully enrolled Phase 2 IMPRSSION study.

Also Read: Ionis Pharmaceuticals Q4 Revenue Growth Exceeds Guidance, Forecasts Over $600 Million In 2025 Sales

Sapablursen received FDA Fast Track designation and orphan drug designation in 2024.

Under the agreement, Ionis will receive a $280 million upfront payment, potentially earning up to $660 million in additional payments based on the achievement of development, regulatory, and sales milestones.

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  • Ionis is also eligible to earn royalties in the mid-teen percentage range on annual net sales of sapablursen.

    Ionis will be responsible for completing the ongoing Phase 2 IMPRSSION study, while Ono will be solely responsible for subsequent development, regulatory filings, and commercialization.

    "Ionis remains committed to advancing the wholly owned medicines we choose to commercialize ourselves, which includes our first independent launch currently underway and three additional anticipated launches in the next three years. Streamlining our Ionis-owned portfolio provides financial flexibility, supporting our commitment to invest in and focus on our near and mid-term commercial opportunities and generate substantial revenue growth," said Brett P. Monia, Ph.D., CEO of Ionis.

    Earlier this week, the European Union approved Ionis Pharmaceutical and AstraZeneca plc's AZN Wainzua (eplontersen) for hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN.

    Price Action: At the last check on Wednesday, IONS stock was up 1.24% at $33.54.

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