Vertex Pharmaceuticals opens expanded San Diego research center with focus on cystic fibrosis

By Bradley J. Fikes

Vertex Pharmaceuticals opened its new San Diego research center Monday, starting a new chapter in a decades-long quest to not only treat but cure cystic fibrosis.

In 18 years, three drugs for the lung-ravaging disease have emerged from Vertex’s San Diego center and more are in the pipeline.

The first, Kalydeco, was approved in 2012. It is the first drug that treats the underlying cause of the disease. The second, Orkambi, was approved three years later. And the third, Symdeko, was approved in February.

These drugs can benefit about half of all patients with the incurable disease. In the next several years, Boston-based Vertex hopes its drugs can help nearly all patients live longer, healthier lives.

Cystic fibrosis is caused by a genetic defect that allows a buildup of thick mucus in the lungs, and other internal organs. This mucus clogs airways and promotes the growth of bacteria. The average lifespan of patients is 37 years, up from 20 years in 1980. Treatments include antibiotics to fight lung infections and mucus-thinning drugs.

The new 170,000 square-foot building on Torrey Pines Mesa more than doubles the company’s space. The center includes cell culturing equipment to grow lung cells from patients, to be used for drug screening. A 4,000 square-foot incubator suite will serve outside collaborators.

Asides from cystic fibrosis, the staff will work on other serious diseases.

Among the speakers Monday morning was a veteran in the fight against cystic fibrosis: Jennifer Ferguson, who has two children with the disease, Ashton and Lola. Both her children are taking Vertex drugs, and both were present with her at the event.

With these drugs and the promise of better therapies ahead, she says Ashton and Lola have a good chance of growing up and leading their own lives. She urged all Vertex employees to think of themselves as part of a team to cure the disease.

Ferguson, of San Diego, found out about the work from the Cystic Fibrosis Foundation. The foundation had invested $30 million in startup Aurora Biosciences to find therapies.

In 2001, Vertex purchased Aurora for $592 million in stock, the same year Ashton was diagnosed. The research went on under Vertex, and Ferguson became quite familiar with the research team.

“The Cystic Fibrosis Foundation asked me to come speak, to show them what it’s like to have a little child with CF,” she said. “So I came here about 17 years ago with him as a 6-month-old.”

At that time, many cystic fibrosis patients never reached adulthood.

“I had a hard time keeping it together,” Ferguson told the audience of that long-ago visit.

“But I looked in the staff’s faces — and some of you are still here — and I thought, I’m going to put my faith and trust in your hands, in your brains. And I was able to let go of my worry, because you were on the case.”

Ferguson started visiting every few years to check on what progress was being made, first with Ashton, and later including Lola. She also raises money for the Cystic Fibrosis Foundation.

Both her children have shown improvement since starting the Vertex drugs, Ferguson said. But they still need to go through a daily regimen of clearing out their lungs.

From medications, the research frontier has advanced to investigations into a cure. That means fixing the genetic defect, which can come in several variations, inside living patients.

That cure might come from the hot new gene editing technology called CRISR. In 2015, Vertex allied with startup CRISPR Therapeutics to develop curative therapies.

This post was originally published on The San Diego Union-Tribune

A Drug Costs $272,000 a Year. Not So Fast, Says New York State.

From The New York Times:

A Drug Costs $272,000 a Year. Not So Fast, Says New York State.

New York’s Medicaid program says Orkambi, a new drug to treat cystic fibrosis, is not worth the price. The case is being closely watched around the country.

A wave of breakthrough drugs is transforming the medical world, offering hope for people with deadly diseases despite their dizzying price tags.

But what if it turns out that some of these expensive new drugs don’t work that well?

That’s the quandary over Orkambi, a drug that was approved in 2015 for cystic fibrosis and was only the second ever to address the underlying cause of the genetic disease. Orkambi, which is sold by Vertex Pharmaceuticals, costs $272,000 a year, but has been shown to only modestly help patients.

Now, in a case that is being closely watched around the country, New York state health officials have said Orkambi is not worth its price, and are demanding that Vertex give a steeper discount to the state’s Medicaid program. The case is the first test of a new law aimed at reining in skyrocketing drug costs in New York’s Medicaid program.

The high price of prescription drugs has ignited a populist furor, and in May, the Trump administration unveiled a set of proposals to address the issue. But while the ideas at the federal level are still mostly theoretical, some states have begun tackling the issue themselves. Earlier this year, Massachusetts asked the federal government for permission to limit its coverage of drugs in an effort to secure larger discounts from drug makers. Other states, like California and Vermont, have passed laws requiring drug companies to turn over certain financial details if they raise prices significantly.

Advertisement

“There’s a number of states that are really trying to push forward and say, we need to be thinking very differently about how we’re paying for drugs,” said Matt Salo, the executive director of the National Association of Medicaid Directors. “We need the ability to say that there are some drugs that are just not priced in a rational way.”

Orkambi held great promise for people with cystic fibrosis when it was approved three years ago. A similar drug, Kalydeco, approved in 2012, was viewed as groundbreaking because it was the first to try to counteract the genetic defect that causes cystic fibrosis. The disease, which affects about 30,000 Americans, leads to a buildup of sticky mucus in the lungs and can lead to death by respiratory failure by the time many people are 40.

But while Kalydeco, also known as ivacaftor, was found to be effective, it was only approved for a sliver of patients with the disease — those who had certain genetic mutations. Orkambi, which combines ivacaftor and another drug, lumacaftor, was approved for mutations that covered nearly half of cystic fibrosis patients, but studies showed it was not as effective as Kalydeco.

Since Orkambi’s approval, several countries have balked at paying for it, including Britain, France and Canada.

In the United States, private insurers and Medicare plans have generally covered Orkambi. Medicaid programs, which cover health insurance for the poor, are required to cover all drugs.

Still, many insurers require patients to pay thousands of dollars out of pocket, and even though Vertex offers assistance, not everyone qualifies.

Lora Moser, 40, is covered by Medicare because she is disabled, and said she had to stop taking Orkambi in January because she could not afford the first month’s payment of more than $3,000 required by her insurer, Humana. A spokeswoman for Humana said that for high-cost drugs like Orkambi, the insurer helps patients identify outside assistance programs to cover out-of-pocket costs.

Image
Ms. Moser said a nonprofit group that had provided financial assistance declined to renew her grant because, she said she was told, her annual household income was too high.CreditTamir Kalifa for The New York Times

A nonprofit group that had provided assistance the previous year to Ms. Moser declined to renew her grant because, she said she was told, her annual household income was too high. She said her income is about $600 above their limit.

“I’ve never felt more destitute and hopeless as I do right now, from a medical standpoint,” Ms. Moser said.

A spokeswoman for Vertex, Heather Nichols, said more than 99 percent of cystic fibrosis patients who are eligible to take Orkambi in the United States have “broad access” to the drug.

“Vertex has a longstanding commitment to supporting access for all eligible patients, and we will continue to oppose any attempts to restrict patient access to these transformative medicines,” Ms. Nichols said.

Despite its lukewarm reception, Orkambi has been a boon for Vertex. In 2017, the drug was its top-selling product, bringing in about $1.3 billion in sales, a considerable sum for a product that is only approved to treat about 28,000 people worldwide.

Dr. Steven D. Pearson, the president of the Institute for Clinical and Economic Review, which evaluates the cost-effectiveness of drugs, said the problem is that in the United States, drug companies control the prices, especially in the case of newly approved drugs like Orkambi.

“Our system is set up not to distinguish very well between those drugs that are fairly priced and those that are not,” he said. Dr. Pearson’s institute concluded that Vertex’s cystic-fibrosis drugs should be discounted by as much as 77 percent. “That gives the incentive to the company to overreach, and that’s part of why our system is so out of whack,” he said.

In April, Orkambi became the test case for the New York law when a state board ruled that the drug was not worth its cost, recommending that it be discounted from the list price by roughly 70 percent — an amount that was influenced by work done by Dr. Pearson’s institute. New York’s law, passed in 2017, allows the state to ask manufacturers for a deeper discount if the state’s Medicaid drug budget exceeds a certain amount.

Under federal law, state Medicaid programs get a rebate of at least 23 percent. New York officials said that they identified 30 drugs this year that were priced too high, and that those products’ manufacturers agreed to deeper discounts, resulting in about $60 million in annual savings. Vertex, which is based in Boston, was the only company that refused, the state said. New York officials did not identify the manufacturers that agreed to steeper discounts.

For now, at least, Vertex appears to have the upper hand because federal law requires the state to cover Orkambi, although the state can limit its use. Under its new law, New York could also demand that Vertex disclose details about how it sets its price, including how much goes toward research and development or to other areas, like marketing. But even if Vertex complied, that information would not be made public because it is considered proprietary.

Ms. Nichols, the Vertex spokeswoman, said the company had no plans to agree to a discount below the 23 percent required by law.

And Donna Frescatore, the director of New York’s Medicaid program, said she was reluctant to limit the use of Orkambi for those who need it. “It’s certainly a balance with our ability to get fair pricing for this medication,” she said.

Image

Ms. Moser prepares to administer medication through a nebulizer at her home in Leander, Tex. CreditTamir Kalifa for The New York Times

But despite the impasse, Mr. Salo said big states like New York are major buyers of prescription drugs, and companies may see an interest in taking those states seriously. “I see this as being of very, very widespread interest,” he said. “A lot of other states are kind of watching and saying, ‘How is that going to work?’”

The debate over Orkambi may soon become moot — earlier this year, the Food and Drug Administration approved a new cystic fibrosis drug, also made by Vertex. Symdeko, as the drug is called, treats a similar population as Orkambi, but has been proven to be more effective. It carries a list price of $292,000 a year, and some analysts, including Geoffrey Porges, of Leerink, say they believe Symdeko will eventually replace Orkambi.

Given the arrival of Symdeko, some analysts said New York would be smart to negotiate a package deal for all three of Vertex’s cystic fibrosis drugs, similar to a deal recently made with Ireland. Ms. Frescatore said that’s an approach that she would consider.

“You don’t want a patient being forced to take Orkambi because it’s cheaper,” Mr. Porges said. “You want the right patient to get the right medicine.”

Katie Thomas covers the business of health care, with a focus on the drug industry. She started at The Times in 2008 as a sports reporter. @katie_thomas

Vertex Employees Donate $1M to CF and Other Communities via Matching Gift Program

By Carolina Henriques

Vertex Pharmaceuticals employees have raised more million $1 million  using  the Vertex Foundation‘s matching gift program in a show of commitment to causes that include the cystic fibrosis (CF) community, a company press release states.

The dollar-for-dollar matching gift program is being run through the nonprofit Vertex Foundation, established by the company in November 2017 as part of it’s charitable giving goal of donating $500 million to qualified nonprofits and other causes worldwide over 10 years.

To date, more than 500 Vertex employees have used the program to support 753 charities around the globe working to advance work in areas that include healthcare, human services, education, and disaster relief.

Vertex’s charitable commitment has four primary goals: supporting CF patients and caregivers worldwide, including enabling access to Vertex’s medicines; helping underserved students and young women with STEAM (science, technology, engineering, arts and math) education; supporting young doctors and scientists; and strengthening and fostering innovation in local communities through health and wellness programs.

“Giving back is in our DNA at Vertex, and our employees have a long history of going the extra mile to improve the lives of patients, students and their neighbors,” Jeffrey Leiden, president, chairman and chief executive officer of Vertex, said in the release. “I’m proud that The Vertex Foundation is able to help extend the impact of our employees’ giving and look forward to seeing the reach of these investments in the causes they care about most.”

Also as part of its 10-year commitment, Vertex awarded $400,000 in scholarships to eligible CF patients and their family members in May as part of its second “All in for CF” scholarship program. In total, 80 scholarships worth $5,000 each were awarded for the upcoming academic year.

Vertex, which specializes in cystic fibrosis, has three approved CF therapies: Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor).

The company is also testing potential triple combination treatments for CF.

For the rest of this article, click here.

Antioxidant Supplement Helps Cystic Fibrosis Patients, Study Finds

By Carolina Henriques

A special formulation of an antioxidant-enriched multivitamin helped decrease the frequency of pulmonary exacerbations in patients with cystic fibrosis (CF), according to a new study.

The findings, by researchers at Children’s Hospital Colorado and the University of Colorado School of Medicine, were published in the American Journal of Respiratory and Critical Care Medicine under the title “Effects of an Antioxidant-enriched Multivitamin in Cystic Fibrosis: Randomized, Controlled, Multicenter Trial.”

Inflammation is an important contributor to lung damage and to progressive lung function decline in CF. In the study, researchers looked at the effects of a “cocktail” of multiple antioxidants on inflammation and health outcomes in CF patients.

“Single oral antioxidant formulations have been tested previously in CF with mixed results. However, there had not been a well-designed, randomized controlled trial of an antioxidant ‘cocktail’ that included multiple antioxidants in a single formulation,” Scott Sagel, MD, PhD, said in a University of Colorado news story. Sagel is the study’s first author and a pediatric pulmonologist at Children’s Colorado.

The 16-week study (NCT01859390) was conducted from September 2013 to October 2015 at 15 U.S. CF centers affiliated with the CF Foundation’s Therapeutics Development Network.

The study included 73 pancreatic-insufficient CF patients, 10 years and older (average age 22), who could not adequately absorb important dietary antioxidants such as beta-carotene, coenzyme Q10 (CoQ10), tocopherols (vitamin E), and selenium, which help neutralize inflammation in the body.

Participants received either capsules of antioxidant-enriched multivitamins, or control multivitamins without antioxidant enrichment. The capsules were designed specifically for people with difficulties in absorbing fats and proteins, like CF patients.

The investigational antioxidant-enriched multivitamin supplement, called AquADEKs-2, contains standard amounts of fat-soluble vitamins (A, D, E, K) plus several antioxidants, including beta-carotene, mixed tocopherols, CoQ10, mixed carotenoids (lutein, lycopene and zeaxanthin), and the minerals zinc and selenium.

Sagel and his team found that the antioxidant-enriched multivitamin supplement increased the concentration of antioxidants in the bloodstream, and temporarily reduced inflammation at four weeks, even though these results were not sustained through the end of the 16 weeks of the study.

In addition, antioxidant supplementation was found safe and well-tolerated by study participants.

Researchers also observed that antioxidant treatment appeared to prolong the time to the first pulmonary exacerbation requiring antibiotics, and also to reduce the frequency of pulmonary exacerbations altogether.

For the full article, please visit CF News Today.

CF Foundation ‘Venture Philanthropy’ Model Crucial to CF Breakthroughs

By Larry Luxner

When the Cystic Fibrosis Foundation (CFF) was established in 1955, most people with cystic fibrosis (CF) didn’t make it to their sixth birthday. Today, the average life expectancy of a CF patient is 47 years.

To date, the U.S. Food and Drug Administration has approved 12 CF therapies. Three of them are CFTR modulators that treat the basic disease-causing defect, benefiting 60 percent of all patients, and more therapies are on the way.

Preston W. Campbell III, the CFF’s president and CEO, directly attributes this dramatic improvement to the foundation’s philosophy of “venture philanthropy.”

“We are now in Phase 3 CFTR trials that, if successful, will mean that as early as next year, more than 90 percent of all individuals with CF will have a highly effective therapy targeting CF’s basic defect,” he said. “More therapies that treat the complications of CF are in the pipeline than ever before.

“It begs the question: how did all of this happen?”

Campbell answered that during his March 26 presentation, “Patient advocates taking a real stand in drug development: How the CFF worked with biotech and pharma to find a cure,” at the 2018 World Orphan Drug Congress USA in Oxon Hill, Maryland.

Back in 1960, the Bethesda, Maryland-based foundation broke ground by establishing a Care Center Network to provide multidisciplinary care. Within five more years, it had formed a patient registry.

With only $400,000 in the bank, it would also commit $11 million to research, Campbell said. “Five years later, in 1985, the basic CF defect was identified, and in 1989, the CFTR gene was discovered. That opened the floodgates,” he added.

Campbell’s predecessor, Robert J. Beall, created the Therapeutics Development Program — now called its Venture Philanthropy Model — in 1998 to entice industry to focus on CF, and specifically on CFTR as a target. Its three components were financial assistance, research tools and scientific advice, and a clinical trials network.

“We would lower the risk for industry to come into the CF space. We also made our research tools and scientific advice freely available, and we also embedded the best scientists in the world in these industry programs,” said Campbell, who took over from Beall as head of the CFF in January 2016. “Finally, in order to make sure clinical trials were safely and efficiently done, we created a clinical trials network that originally had seven centers and now has 89.”

In the beginning, CFF’s investments were typically in the $1.5 million range. Ultimately, the foundation invested more than $100 million in Aurora and its successor, Vertex Pharmaceuticals, whose headquarters are in Boston.

To date, the FDA has approved three Vertex CFTR modulators: Kalydeco (ivacaftor) for patients with the G551D mutation in the CFTR gene (2012); Orkambi (lumacaftor/ivacaftor)for patients who are homozygous for F508del, the most common mutation in the CFTR gene (2015); and Symdeko (tezacaftor/ivacaftor) for homozygous F508del patients as well as others (2018).

“Payments are milestone-based, so we pay for success,” Campbell said. “A scientific advisory committee determines if milestones are met and if the project should continue. Successful programs offer a return on our investment, so if the program is foundering, we shake hands and walk away.”

To continue to full article, please click here.

A Dutch Company on the Quest Against Cystic Fibrosis

An interview by:  Clara Rodríguez Fernández

Daniel de Boer founded ProQR in 2012 following a strong determination to improve the lives of people with cystic fibrosis. We started ProQR Therapeutics for a very personal reason,” he told me. “Eight years ago, my son was born, and diagnosed with cystic fibrosis. At that time, I was a serial entrepreneur in IT. I decided to make a career switch and start a company to develop drugs for cystic fibrosis, but then also for other genetic diseases.”

One would think that a person without a background in biotech might have it difficult to succeed, but de Boer is not the only to have so far successfully undertaken this endeavor. Over in France Karen Aiach built Lysogene to treat her daughter’s rare genetic disorder, while in the US the story of John Crawley and his company Amicus Therapeutics, founded to help his two children’s diagnosis, went so far as to inspire a movie. The determination and motivation of these parents seem to overdrive any challenges they might have faced because of their limited experience.

De Boer set out to create a business plan for his new company and found out that there was already quite a lot of activity, especially in approaches using small molecules or gene therapy.“We decided that we really wanted to add something new to the space, and take a completely novel approach.”

So he started looking for a new technology, and he found it. “Around that time, I met for the first time with some people in biotech, including the CEO at Alnylam, John Maraganore, and we talked about how they used RNA approaches for genetic diseases,” says de Boer.

Technologies targeting RNA are quite new compared to those that target DNA such as gene therapy. But RNA-based treatments have started to gain traction in the last few years. There are multiple ways that RNA can be used as a therapeutic, but its distinctive advantage over gene therapies and the likes is that it does not permanently change our genetic makeup, making it possible to reverse its effects.

Today, RNA technology is being tested in multiple rare diseases caused by genetic mutations, such as hemophilia, porphyria, or iron overload disorders. I thought, ‘if you can do that for all these other genetic diseases, why not for cystic fibrosis?’” says the Boer. “With that in mind, we started ProQR.”

For the rest of this article, please click here.

Potential Nitric Oxide Treatment for Resistant Bacterial Infections Gets Patent

A possible inhalable treatment for antibiotic-resistant bacterial infections in people with cystic fibrosis due to Pseudomonas aeruginosa now has a U.S. patent and is being readied for a first clinical trial, Novoclem Therapeutics announced.

The patent (No. 9,850,322) was issued to the University of North Carolina (UNC) at Chapel Hill where the potential therapy, BIOC51, was discovered, and covers a technology known as water-soluble polyglucosamine compositions that release nitric oxideContinue reading Potential Nitric Oxide Treatment for Resistant Bacterial Infections Gets Patent

List of CF Patient Assistance Programs

Patient Assistance Programs

AbbVie 
CREON® CFCareForward Patient Support Program offers nutritional services to eligible patients, as well as financial and educational support for patients and families.
http://www.creon.com/CFCareForward
1-855-227-3493

Chiesi USA 
Chiesi USA offers prescription access support, financial assitance, and product counseling for patients taking BETHKIS® (Tobramycin Inhalation Solution) and PERTZYE® (pancrelipase) 
ttp://bethkis.com/support-services/
http://www.pertzyecf.com/patient/free-support-and-savings/r
1-888-865-1222

Genentech, Inc.
Genentech Access Solutions 
http://www.genentechaccesssolutions.com/portal/site/AS/
1-866-4-ACCESS
Pulmozyme® Access Solutions Co-Pay Card Program 
https://www.activatethecard.com/pulmozyme/welcome.html
1-877-794-8723

Gilead
Cayston® Access Program
http://www.gilead.com/responsibility/us-patient-access/cayston%20access%20program
1-877-722-9786

Novartis Pharmaceuticals
Patient Assistance Now (Spanish Speaking Services) 
http://www.pharma.us.novartis.com/info/patient-assistance/patient-assistance.jsp?usertrack.filter_applied=true&NovaId=2935377019348182802
1-800-245-5356

Vertex Pharmaceuticals
Vertex GPS: Guidance & Patient Support (Kalydeco® or ORKAMBI™)
http://www.vertexgps.com/
1-877-752-5933

RespirTech
Customer financial assistance program for patients using inCourage® airway clearance therapy.
http://www.respirtech.com/reimbursement-incourage-airway-clearance-therapy/patient-financial-resources
1-800-793-1261

Live 2 Thrive 
Live 2 Thrive Offers copay assistance, free vitamins and supplements, and nutritional information for eligible patients. 
https://www.live2thrive.org/
1-888-936-7371

OTHER RESOURCES

Foundation Care 
List of company assistance programs 
http://www.foundcare.com/fc-patients/reimbursement-help/

Cystic Fibrosis Patient Assistance Foundation
Assistance for affording medications and devices for managing CF
https://www.pparx.org/prescription_assistance_programs/cystic_fibrosis_patient_assistance_foundation

Cystic Fibrosis Services
Additional patient assistance programs for those without insurance coverage 
http://www.walgreens.com/topic/pharmacy/cystic-fibrosis-services.jsp

Boomer Esiason Foundation
Links to assistance programs for Tobradex, Creon, Aceon, Estratest HS, Prometrium, EstroGel, Pulmozyme, Advair, and Cipro
http://www.esiason.org/what-is-cf/resources/patient-assitance

HospitalBillHelp.org 
Guidance for Californians facing hefty hospital bills 
http://www.hospitalbillhelp.org

NeedyMeds.org 
Additional Patient Assistance Programs
http://www.needymeds.org

Partnership for Prescription Assistance
Database of Patient Assistance Programs
(Search by drug, company or program name) 

http://www.pparx.org/prescription_assistance_programs/list_of_participating_programs

Patient Advocate Foundation
Mediation and arbitration services for patients with debilitating and life-threatening illnesses.
http://www.patientadvocate.org/

Introduction of New Therapies Affects Pregnancy Rates in Women With CF

The overall rate at which women with cystic fibrosis are becoming pregnant dropped slightly in recent years — coinciding with the introduction of CFTR modulators and the clinical trials that led to their approval as CF therapies — but appears to be rising again to pre-trial levels, a study reports.  Continue reading Introduction of New Therapies Affects Pregnancy Rates in Women With CF

A Breath of Fresh Air for Biotechs Working on Cystic Fibrosis Therapies

Researchers from the University of Zurich have determined the structure of a chloride channel, which could be a target for new drugs to treat cystic fibrosis.

Researchers at the University of Zurich have found a new target for future cystic fibrosis treatments. The study, published in Nature, has uncovered the structure of a protein that could help to correct the mechanism underlying the buildup of sticky mucus in patients’ lungs. This could give rise to a new wave of therapeutics for the condition, which at the moment lacks disease-modifying treatments.

Cystic fibrosis is a severe genetic disease affecting the lungs, for which there is currently no cure. It is caused by a malfunctioning chloride channel, CFTR, which prevents the secretion of chloride by cells, leading to the production of thick, sticky mucus in the lung. The condition affects around 70,000 people worldwide, who suffer from chronic infections and require daily physiotherapy.

However, one potential approach to treat cystic fibrosis is to activate the calcium-activated chloride channel, TMEM16A, as an alternative route for chloride efflux. As TMEM16A is located within the same epithelium as CFTR, its activation could rehydrate the mucus layer. The research group used cryo-electron microscopy to decipher the structure of TMEM16A, which is part of a protein family that facilitates the flow of negatively charged ions or lipids across the cell membrane.

The changes that occur in the lungs of cystic fibrosis patients.

TMEM16A is found in many of our organs, playing a key role in muscle contraction and pain perception, as well as in the lungs. It forms an hourglass-shaped protein-enclosed channel, which when bound by positively charged calcium ions, opens to let chloride ions to pass through the membrane.

Current treatments for cystic fibrosis include bronchodilators, mucus thinners, antibiotics, and physiotherapy, which only control symptoms. However, biotechs around Europe are beginning to make progress, with ProQR completing a Phase Ib trial and Galapagos and Abbvie’s triple combination therapy entering Phase I. Antabio has also received €7.6M from CARB-X to develop a new antibiotic against Pseudomonas infections.

The identification of a new target provides patients and biotechs alike with renewed hope of new and effective cystic fibrosis treatments, or even a cure. It will be interesting to see whether small molecules or gene therapy specialists could take advantage of this information.

Original article: https://labiotech.eu/cystic-fibrosis-treatment-target/