Neurenati Announces its First Scientific and Clinical Advisory Board Meeting with KOLs to Advance the Development of NEU-001, a Promising Cure for Hirschsprung Disease
Article content
MONTRÉAL — Neurenati, an innovative biotechnology company dedicated to developing cutting-edge therapies for unmet medical needs in rare pediatric diseases, is excited to announce the successful convening of its inaugural Scientific and Clinical Advisory Board (SCAB) meeting. This pivotal meeting recently brought together leading pediatric surgeons, gastroenterologists, and industry experts in regulatory affairs and drug development to collaboratively shape a strategic roadmap for advancing NEU-001, a fixed combination of glial cell derived growth factor (GDNF) and butyrate, into clinical stage.
Article content
The SCAB of Neurenati is composed of Dr Jacob Langer (pediatric surgeon, previously at SickKids Hospital, Toronto), Dr Tomas Wester (pediatric surgeon, Karolinska University Hospital, Stockholm), Dr Anne Dariel (pediatric surgeon, La Timone Hospital, Marseille), Dr Kattayoun Kordy (pediatric gastroenterologist, Founder and CEO Lucien Bio), and Dr Andrew Mulberg (pediatric gastroenterologist and former division deputy director of gastroenterology and inborn errors products, Center for Drug Evaluation and Research, US FDA).
Article content
The meeting served as a valuable platform for transatlantic dialogue, to share insights on the preclinical prospects of direct benefit, clinical trial design, and regulatory pathways for NEU-001. The diversity of expertise provided Neurenati with meaningful guidance on best clinical practices in both North America and Europe, a deeper understanding of the patient journey, and—most importantly—confirmed the urgent need to advance therapeutic solutions for individuals affected by Hirschsprung disease. 'Hirschsprung disease is an uncommon condition that affects approximately one in 5,000 children. The only effective treatment at this time is a major surgical operation. The innovative work being done by Neurenati could revolutionize the treatment of this disease and could potentially avoid the need for surgery in these tiny children,' said Dr Langer. 'Novel non-operative treatment options for Hirschsprung disease would have a significant impact on future management of these neonates,' confirmed Dr Wester.
Article content
Looking ahead, Neurenati is committed to leverage a strong collaboration with its scientific and clinical advisory board members as it navigates the path towards IND filing, ensuring that NEU-001 meets the highest standards of safety and efficacy. 'We are thrilled to have gathered such an esteemed group of thought leaders who are passionate about advancing the field of medicine,' said Meriam Kabbaj, DPharm, PhD, Chief Development Officer of Neurenati. 'Their contributions will be instrumental in shaping Neurenati's approach as it prepares for the next phase of its clinical program, with a strong focus on delivering a safe and effective therapy to pediatric patients in need.'
Article content
Article content
Article content
Article content
Article content
Contacts
Article content
Media Contact:
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
CBC
35 minutes ago
- CBC
Nunavik's tuberculosis outbreaks are a result of decades of colonial neglect, professor says
Nunavik is on track to set another record number of tuberculosis cases, which one scholar says is unacceptable in this day and age. There have been 56 cases so far this year of tuberculosis reported in the region, according to Quebec's health department. On Monday, the region's 14 mayors released a letter, calling on the Quebec government to declare a public health emergency over the rates of tuberculosis. The Nunavik Regional Board of Health and Social Services has said the incidence of tuberculosis in the region is 1,000 times higher than among non-Indigenous people born in Canada. Health Minister Christian Dubé told CBC News he "will continue to follow the recommendations of public health experts on the subject," though he stopped short of committing to a declaration. Natasha MacDonald, a McGill University professor from Kuujjuaraapik who researches culturally responsive care for tuberculosis in Nunavik, said she doesn't believe Quebec is treating the tuberculosis situation in Nunavik with the same urgency as elsewhere in the province. This interview has been edited for length and clarity. What do you make of this call from the mayors to declare a public health emergency over the tuberculosis situation? It's unfortunate that we are in a time and place where we have to make such calls to action. Under a number of international, national and provincial jurisdictions, including the Viens Commission, the Quebec government has a responsibility to ensure that health care in our communities is adequate and is on par with those of non-Indigenous populations. Because of the urgent situation that Nunavik is in right now, the mayors have had no choice but to unite together as one voice to demand that the government do its job. Why do you think we're seeing this upward trajectory in cases of tuberculosis, in 2025? What's unbelievable is that we are a first world nation in Canada, and we have a third world disease within Canada. It exists in Nunavik in our Inuit communities, and it's the same strain that has been in Nunavik since 1928. Quebec made efforts with the City of Montreal, not that long ago, to stop the spread of tuberculosis. Cases were found, contact tracing was done, people were isolated and medicated. In Nunavik, it has been left to grow and we have outbreaks in six of our 14 communities. This is unacceptable, and it should not happen. We are part of Quebec, we are part of Canada. You've alluded to the damage that tuberculosis has wreaked on families historically, with Inuit being sent south for care. Given that history, what do you believe we, as a society, still don't understand treating tuberculosis in Nunavik? What's happening in Nunavik is a result of decades of systemic colonial neglect. Inuit are expected to adapt to those systems, rather than the government adapting those systems to meet the needs of Inuit. The way the regional health board has been implanted by the provincial government makes it a very program-centred health-care system and not a person-centred health-care program. One of the infectious disease nurses said at one point, in all of Nunavik, they ran out of sputum testing kits. So she had to beg and plead from the province of Manitoba to send over 40,000 sputum testing kits. Quebec didn't have any and it wouldn't procure more. There aren't even X-rays in most of our communities, much less X-ray technicians in those communities. And Inuit feel uncomfortable going to the clinic or hospital because of decades of mistrust in medical care and the systemic and individual racism that exists. One of the calls to action in the mayors' letter is about Inuit health sovereignty. What does that look like? For things to change in our communities, Inuit need to be the ones who determine how our programs and systems run, so that they are tailored to the way we think and work. Inuit are being expected to move around the calendars and schedules of health-care workers, and they are workers are told they should not be going house to house for testing. If it were Inuit leading our organizations, we would have a better understanding on how to do more screening. It's not somebody from Quebec City who should be dictating how much money should be going toward basic equipment, We should be. We're the ones in our communities. We're the ones that know that the X-ray machine is broken in this one town, or that we have a new graduate who's just finished their radiography course who could be hired into a position within their community. What we are talking about here is structural change. Can you compare what the system looks like now with Nunavik's health organizations, to that ideal vision you've just talked about? Systemic change would come when Inuit are able to create a new system altogether, through self-determination or self-government. Or if we can appropriate the system and have an Inuk lead who can hire resources, because we know best on how to allocate our resources and where the needs are. The midwifery program in Nunavik is a perfect example of that. This is a system where Inuit have designed how our mothers want to give birth in a community with support, in an Inuit way, and it is unbelievably successful. This is not rocket science. We also understand that translators are as key as any doctor or nurse. Right now, you have French-speaking nurses and doctors that come into our community, and English is often the lingua franca because most Inuit in Nunavik still speak Inuktitut. When you're translating, for example, with a term like tuberculosis, you have to be very careful to make the difference between tuberculosis, which is a disease that can kill you, and something like bronchitis. You can't just say it's a lung problem. I understand that we have to collaborate with the Ministry of Health in Quebec, but at the same time, they have to understand we are not just another region within Quebec. We are distinct and we have needs that have not been met for decades. It's also an example of systemic racism because Inuit in Nunavik have been ignored, have been left behind, have been left to die for decades, and this is unacceptable. It was unacceptable after the first TB case. It was unacceptable after the first death. It's unacceptable now when our numbers are record high. We have nurses who are burnt out. And if this were anywhere else in Quebec, there would be an outcry and the government would be immediately addressing this, so we are expecting the same.
CTV News
36 minutes ago
- CTV News
Vancouver woman with MS asks why B.C.'s the only province that doesn't cover her medication
Documentary filmmaker Teresa Alfeld, 37, is pushing for the B.C. government to cover multiple sclerosis medication that is covered in every other province. Teresa Alfeld is a documentary film director from Vancouver who was diagnosed with relapsing-remitting multiple sclerosis in September 2024. As a Type 1 diabetic, the 37-year-old can't take steroid medications regularly prescribed for MS. 'In conversation with my doctor, we both agreed I needed a really strong drug to make sure that the lesions didn't continue progressing, and so our choice was Kesimpta,' said Alfeld. Kesimpta is a once-a-month injectable that costs $26,000 a year. 'Sadly, that's not something that myself and my family can cover,' Alfeld said. She secured six doses of the medication from the manufacturer while she looked into long-term coverage. 'I have two private plans, my own and my husbands, which we went through first and then found out that neither plan covered it. And so then it was on to PharmaCare. And that was when we found out, shockingly, that they were not going to cover it,' said Alfeld. She learned British Columbia is the only province in Canada that doesn't cover the cost of Kesimpta for MS patients. 'We call it the postal code lottery at MS Canada because your access to medication really depends on where you live,' said Alanah Duffy with MS Canada. 'I would have thought that OK, I'm a special case because I can't use the steroids. Surely they'll cover just me in this case. But I've been rejected multiple times,' Alfeld said. Duffy said MS Canada has been petitioning the B.C. government to cover the medication for years, with no success. 'What we've continuously heard from the B.C. government is that these treatments are not cost effective. But our argument is that funding these treatments would save other health-care costs because Kesimpta is a highly efficacious treatment,' Duffy said. In an email, the Ministry of Health said it engaged in price negotiations with the company that makes Kesimpta, but the manufacturer was unable to address the province's concerns with regards to cost-effectiveness and value for money. The statement went on to say: 'B.C. covers many drugs that other provinces do not. Numerous treatment options for relapsing-remitting multiple sclerosis (RRMS), with the same clinical effectiveness as ofatumumab (Kesimpta) and ocrelizumab (Ocrevus), are covered by PharmaCare. We encourage patients to discuss treatment options with their physician.' Alfeld said she can't take the alternative drugs the ministry is suggesting because they contain steroids, which are not advisable for people with Type 1 diabetes. Older drugs that are covered are also far less effective, she said. 'The drug that I have been taking is rated at about 99 per cent efficacy at stopping the progression of lesions, which is why my neurologist and I chose it for myself. The legacy drugs that I've been offered are rated 50 per cent,' said Alfeld. 'So this is a huge gamble that I'm not comfortable taking.' The doses of Kesimpta that Alfeld got from the drug manufacturer have now run out. 'I am due for my next shot on June 30th, and I don't have that shot,' she said, adding she has been working with her MLA and petitioning PharmaCare and the Health Ministry to provide the medication. But as the days tick down until she's due for her next shot, Alfeld is worried. 'I just focus on taking action, because I can't really allow myself to think about what's going to happen.'
CTV News
an hour ago
- CTV News
Salami being recalled due to Salmonella outbreak
Salami sold in Alberta is being recalled due to a Salmonella outbreak. (Supplied) Some brands of salami are being recalled due to a salmonella outbreak in Alberta. There are currently 44 confirmed cases in the province. The recall also affects Ontario, which has 13 confirmed cases as of Wednesday afternoon. The brands being recalled in the two provinces are: Rea brand Genoa Salami Sweet; Rea brand Genoa Salami Hot; and Bona brand Mild Genova Salami. According to officials, the salami may have been sold in sandwiches from deli counters and the product was distributed to grocery stores, specialty markets, restaurants, cafes, delis, and butcher shops. Recalled items should not be consumed and should be thrown out or returned to where they were purchased. Symptoms of salmonellosis develop six to 72 hours after eating products infected with the bacteria and can include chills, fever, nausea, diarrhea, vomiting and stomach cramps. Most people will recover on their own but a serious illness may require treatment at a hospital, or lead to long-lasting health effects or death. An outbreak investigation is ongoing. Updates and more information can be found online.
