Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation For EDIT-301 For The Treatment Of Severe Sickle Cell Disease
CAMBRIDGE, Mass., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to EDIT-301, an investigational, gene editing medicine, for the treatment of severe sickle cell disease (SCD).
"Sickle cell disease is a devastating disease that leads to anemia, pain crises, organ failure, and early death. Receiving RMAT designation for EDIT-301 for severe sickle cell disease highlights the urgent need for new treatment options for patients and supports our belief that EDIT-301 can provide life-changing clinical benefits to patients," Gilmore O'Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. "I would like to thank the participants, their families, clinicians, and colleagues at collaborating institutions that contribute to the RUBY trial. We look forward to sharing further clinical updates including additional data for the trial prior to year-end."
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising regenerative medicine therapies. An investigational cell therapy medicine or gene editing medicine is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the experimental medicine has the potential to address unmet medical needs for the disease or condition. Advantages of the RMAT designation include all the benefits of the fast track and breakthrough therapy designation programs, including but not limited to intensive FDA guidance on efficient and expedited drug development, possible rolling review, and priority review of the biologics license application (BLA), and FDA's organizational commitment involving senior managers.
The FDA previously granted Orphan Drug Designation and Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease and beta thalassemia.
About Sickle Cell DiseaseSickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin (HbS). In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape. The abnormal shape causes the red blood cells to have shortened lifespan and to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing the manifestation of sickling.
About EDIT-301EDIT-301 is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.
About the RUBY TrialThe RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available on www.Clinicaltrials.Gov (NCT#04853576).
About Editas MedicineAs a clinical-stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute's Cas12a patent estate and Broad Institute and Harvard University's Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.Editasmedicine.Com.
Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ''anticipate,'' ''believe,'' ''continue,'' ''could,'' ''estimate,'' ''expect,'' ''intend,'' ''may,'' ''plan,'' ''potential,'' ''predict,'' ''project,'' ''target,'' ''should,'' ''would,'' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company's expectation to provide further clinical data updates, including additional data for the RUBY trial by year-end. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials, including the RUBY trial, and clinical development of the Company's product candidates, including EDIT-301; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption "Risk Factors" included in the Company's most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company's subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.
St. Jude Donor Dr. Vernon Rayford Breaks Down Sickle Cell Advanced Research
Dr. Vernon Rayford, an internal medicine specialist and pediatrician from Tupelo, Miss., says St. Jude Children's Research Hospital is actively examining a cure for sickle cell anemia. As a St. Jude donor, Rayford has contributed thousands of dollars to active research for therapies such as bone marrow transplants and gene editing.
In light of spreading awareness of sickle cell disease, Rayford spoke to rolling out about how St. Jude is handling standard care for children and families.
In layman's terms, what is sickle cell disease?
Sickle cell disease is the most common inherited blood disease.
It's a problem where the blood, particularly the red blood cells, have a genetic mutation. [There's] a mutation in the genetic code of the DNA, and that mutation causes the red blood cell to look like a sickle or a sharp-edged [shape], instead of a lifesaver or a flotation device. As a result, people end up with complications that can include painful episodes, which are called crises, lung disease, increased risk of infections, a risk of stroke or a low blood count, hence the name anemia. Ultimately, the consequence of all this damage from the abnormal shape of red blood cells can lead to a lot of hospitalizations and unfortunately, a much shorter lifespan.
How is St. Jude bringing awareness to this disease?
Babies who are born with sickle cell across the United States, they're being identified within the first few days of life. With newborn screening and recognition of sickle cell disease, there is treatment.
Next, babies are referred to sickle cell specialists. St. Jude is a provider of sickle cell care to almost 1,000 children in a wide stretch from North Mississippi up to Southeastern Missouri. Once they're identified, there are certain medications that can be used to reduce the risk of infection and medications that can reduce the risk of having painful crises.
Ultimately, there are some cutting-edge therapies that involve bone marrow transplants and going in and making edits to the gene code. There's active research in changing the genes through a bone marrow transplant and that's another area that St. Jude excels at. They are setting the standard of care for almost 1,000 children and families with sickle cell disease, but there's cutting-edge research, particularly with gene editing. Twenty to thirty years ago we didn't think this was possible. How can you improve and potentially create a condition that's almost like a cure for sickle cell?
St. Jude is at the forefront of researching that. Not only offer it to the kids that they take care of in their service area but also apply it to the larger world.
Mum 'has To Pretend Sickle Cell Pain Disappears In Hospital As She Can't Get Right Care'
A young mother used to pretend her agony from sickle cell disease had gone so she could leave hospital - because she "couldn't get the right care" from doctors.
Kyra Campbell has been in and out of hospital more than 150 times throughout her life for pain caused by sickle cell disease, a debilitating inherited blood condition. Many of these occasions have been as a result of experiencing what she calls "a pain attack" - when her body shuts down and "goes into shock" due to immense agony caused by the condition.
When this has happened, Kyra, 25, has been rushed into A&E and had overnight stays to avoid seizures, passing out and further complications. However, the mother of one used to lie to her family and doctors and pretend her pain had gone because she wasn't getting the right help or medication. People with sickle cell disease, of which there are approximately 15,000 in the UK, tend to require daily medication, often including amitriptyline, and penicillin - and other drugs when they have "pain attacks".
Kyra Campbell is pictured in immense pain during a stay in hospital following a 'pain attack' (Image:
Supplied) Kyra often has to go to hospital due to problems caused by sickle cell disease (Image:
Supplied)But when Kyra, from West Norwood, South London, went into shock when she was heavily pregnant with son Logan, there wasn't an appropriate anaesthetist available in A&E to administer the right drugs - and she spent 12 hours in hospital before she received the right medication. Logan was born premature two weeks later via C-section, and Kyra believes her stress led to this.
"As you can imagine, when I go into a shock, I'm in need of very serious medication, whether that's codeine, morphine, ketamine or whatever. Of course somebody with the correct credentials has to be there to administer that medication but unfortunately, they weren't available," Kyra told Mirror amid Black History Month.
The mum, pictured with her son Logan, is one of around 15,000 people with sickle cell disease, which can be debilitating (Image:
Supplied)"That was a very scary moment. In all honesty, I wasn't scared for myself because I know what goes on and how I deal with it, but I was pregnant. I didn't know if he could feel the pain. I didn't know if he could feel how stressed I was so it was a very scary time. Ultimately, two weeks later, he had to do born via an emergency C section. So I do believe that a large part of the stress course came to him, as he was six weeks premature."
Sickle cell disease mostly affects people of Afro-Caribbean heritage and, figures released last month showed demand for black donors has surged by more than 50 per cent in five years. Those living with it have also campaigned recently for NHS to support them with more resources, such as more staff with the credentials to administer the right medication.
The mum used to pretend she felt well enough to leave hospital in order to get discharged (Image:
Supplied)Kyra, who has trained in health and social care but had to stop working due to debilitating nature of the condition, continued: "There have been so many campaigns for people with sickle cell to get the correct medical care when they're in A&E because it's a very life-changing moment. It can be detrimental if that person isn't seen to straightaway.
"They say there isn't enough funding but all it really takes is listening to the person that's coming to your A&E. In my condition, I'm in a significant amount of pain. Just because I'm not you know, crying and screaming doesn't mean I'm not in pain. It's kind of two different sides of the spectrum. It's either you go to A&E, and you're left because you're not crying and screaming and wailing and pain, and you're trying your best to deal with the situation. Or you can walk around and scream in pain, but instead they look at you that you're a drug addict. This is the point of view from people with sickle cell disease together by the way, as a collective, not just me. Those sort of feelings that are echoed in the campaign.
The 25-year-old woman, from South London, wants to raise awareness of the condition (Image:
Supplied) Logan was born premature via C-section and Kyra believes he suffered the same stress she did during her pregnancy (Image:
Supplied)"My family know me very well. So what I used to do, which they won't let me get away with anymore, is when I have been in hospital for a certain amount of time, say a week or two, I tended to pretend that the payment just disappeared. That was because it was always very hard to get my next dose of medications. You tend to have your doses of medication every few hours, but for some reason when it comes to somebody with sickle cell to do a whole evaluation every four hours, and it's very stressful, it's very stressful.
"It's like, 'you know why I'm here. Why am I having to explain myself to every couple of hours?' It's it's hard and it's hard to do when you're in that amount of pain. That's what I used to do, especially when I was younger, but my family know me now. I can't carry on getting away with that... I used to do that and say 'no, no. It's fine, I can go home and that was OK'. That's again, that's a sad reality, but it's just the truth."
When a 'pain attack' happens, Kyra's body goes into shock and she needs important medication (Image:
Supplied) Kyra shared details of the medication she needs every day and further tablets when she suffers an attack (Image:
Supplied)Some sickle cell disease patients undergo blood transfusions in hospital every six weeks but, again due to the lack of black donors, this opportunity is becoming less readily available. The process typically helps fresh blood flow through the body because, in sickle cell patients, red blood cells otherwise form into abnormal shapes and get stuck, causing life-threatening blockages and complications. In Kyra's case, her body started rejecting the transfusions when she was just nine. She will only undergo one in extremely rare and life-threatening episodes now.
When Logan was born, in September 2018, he was healthy. Tests were carried out to assess if he too had sickle cell disease but he is only a carrier, unlike his mother. Although he has just turned five, Logan is aware of his mum's journey and how his genes could impact his life and that of any future children.
Kyra said: "He will have to be very mindful about who he decides to bring up and marry and have kids with, because of course there is that chance that if he then meets a partner with sickle cell trait and they do decide to have children there will be that one in four chance that they have sickle cell.
"He's absolutely fantastic. The condition isn't affecting him at all because he only has the trait. So he's only he's slightly anaemic, so he takes a bit of insulin, but even at five years old, he completely understands and it's really lovely, and it's really warming actually."
Although Logan is a carrier of sickle cell, he is 'absolutely fantastic' with his mum (Image:
Supplied)But more than 300 babies are born with the condition each year. Only around 2 per cent of donors have the rare Ro subtype, so meeting the demand can be difficult. That's why the blood of black donors plays such a vital role in helping to save and improve the lives of those living with sickle cell disease in the UK today. For more information, visit NHS's website.
A Department of Health and Social Care spokesperson said: "We're working hard with NHS Blood and Transplant (NHSBT) to provide the best possible care to those living with sickle cell disease and boost blood donation numbers for sickle cell patients who need regular blood transfusions to stay alive.
"NHS England is also working with stakeholders and patient representatives to better support those with sickle cell disease in hospital settings, especially during acute episodes. Backed by almost £10 million of government funding NHSBT opened a state-of-the-art research centre to develop new gene and cell therapy – which uses living cells or genetic material such as DNA – to try and cure diseases such as sickle cell disease."
Sickle cell disease is the name for a group of inherited health conditions that affect the red blood cells. The most serious type is called sickle cell anaemia. Sickle cell disease is particularly common in people with an African or Caribbean family background. People with sickle cell disease produce unusually shaped red blood cells that can cause problems because they do not live as long as healthy blood cells and can block blood vessels. Sickle cell disease is a serious and lifelong health condition, although treatment can help manage many of the symptoms. People born with sickle cell disease tend to have problems from early childhood, although some children have few symptoms and lead normal lives most of the time. The main symptoms of sickle cell disease are: painful episodes called sickle cell crises, which can be very severe and last for days or weeks an increased risk of serious infections anaemia (where red blood cells cannot carry enough oxygen around the body), which can cause tiredness and shortness of breath Some people also experience other problems, such as delayed growth, strokes and lung problems. Source: NHSNHSBT referred Mirror to NHS England, which said National Institute for Health and Care Excellence (NICE) sets the guidelines NHS must follow. A NICE spokesperson said: "When we consulted on our guideline we heard from patients that care could vary from hospital to hospital including issues around pain relief and the time taken to receive treatment. As a result the NICE guideline for managing acute painful episodes in hospital offers useful and useable guidance on how to treat people with sickle cell who are having an acute painful episode and aims to reduce variation in how these are managed in hospital, focusing on effective, fast and safe pain relief.
"The guideline advises healthcare practitioners that acute episodes should be treated as an emergency to ensure they get the best care fast. Patients or their carers should also be considered an expert in their condition. The guideline highlights that there should be discussions about planned treatment for the episode, treatment received during previous episodes, any concerns they may have and any psychological and/or social support they may need.
"Pain relief should be offered within 30 minutes of arriving at the emergency department at hospital with an acute painful sickle cell episode. The guideline recommends assessing how well the pain relief is working every 30 minutes, and this should continue until satisfactory pain relief has been achieved. After that, clinical assessment should happen at least every four hours. Patients who need strong opioids to manage the pain should be clinically assessed every one hour for the first six hours, and at least every four hours after that.
"When exercising their judgement, professionals and practitioners are expected to take this guideline fully into account, alongside the individual needs, preferences and values of their patients or the people using their service. It is not mandatory to apply the recommendations, and the guideline does not override the responsibility to make decisions appropriate to the circumstances of the individual, in consultation with them and their families and carers or guardian. Local commissioners and providers of healthcare should also consider local and national priorities for funding and developing services.
"Opioids are controlled drugs, and this means they have been defined by the Misuse of Drugs Act 1971. They have to be closely regulated because they can be misused and can cause harm. As such they must be managed and used safely in all healthcare settings. For more information on this please look at the NICE guideline for Controlled drugs: safe use and management.
For support and advice on managing sickle cell disease, visit sicklecellsociety.Org, email [email protected] or call 020 8961 7795
