It’s time to build the infrastructure needed to scale up inhaled gene therapies targeting cystic fibrosis, and to bolster investment that supports several key program components.
In less than one year, ReCode Therapeutics has reeled in $200 million in financing. Big pharmaceutical companies are taking notice of the biotech startup’s technology, which enables lipid nanoparticles to go a wide range of organs and tissues, potentially broadening the reach of genetic medicines.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
During my 36-year-old brother’s terminal stay in the ICU, I discovered a dire need for a more comprehensive approach to critical care. The more he relied on this large, corporate hospital system to keep him healthy and happy, the clearer it became that no matter how highly-ranked or well-funded it was, this hospital did not possess the flexibility, agility, or progressive thinking it needed to confront cases like his.
ReCode Therapeutics closed an $80 million Series B round of financing that included Pfizer and Sanofi as investors. The startup’s technology uses lipid nanoparticles to deliver genetic medicines, and its two lead programs are for rare lung disorders.
Shares of the company rose 9 percent on the news that the FDA had approved Trikafta. The drug will be an option for patients using the company's other approved CF drugs.
Firm says blockchain and app will ease secure sharing of patients' medical records and post-marketing surveillance.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
The vest gathers patients’ airway clearance therapy session information, such as how many minutes the session lasted, duration of cough pauses, frequency settings and transmits it to the portal where physicians can see the data.
Genetic testing can play a critical role in helping potential parents understand their options if they are carriers for a wide array of genetic disorders, such as cystic fibrosis.
Vertex Pharmaceuticals, known for its line of cystic fibrosis drugs, has entered the gene editing space with a new $105 million deal with CRISPR Therapeutics.
Cystic fibrosis has become the poster child for precision medicine – most new therapies are carefully tailored to a CF patient’s genetic makeup. University of North Carolina spinout Spyryx Biosciences is proposing a one-size-fits-all approach to the disease: It’s developing a peptide-based therapy that, in theory, could be effective among all CF patients. This startup’s still pre-IND […]
We will highlight Build My Health's revenue practice management tools, which could help physician practices add up to $250,000 to their practices.
In this brave new world of drug development, we’re seeing philanthropic foundations intertwine with industry to bring meds faster to market. But this new model could unfold into a popularity contest, pitting underfunded disorders against media darlings or wealthy folks’ diseases (read: Ice Bucket Challenge). Case in point: Fifteen years ago, the Cystic Fibrosis Foundation invested some $150 million into a biotech […]
The concept of using protein homeostasis – or proteostasis – as a platform for drug development is a rather new one, but it makes a good amount of sense: Maintaining a proper balance of protein production in the body is critical to a person’s health. Proteostasis makes sure every protein within a cell either is folded […]
NEW YORK (Reuters) - A 10-year-old Pennsylvania girl who sparked a national debate about child access to organ donation took her first independent breath this week on a second set of donor lungs after her first transplant failed, the girl's family sai...
It’s been nearly two-and-a-half decades since the gene associated with the inherited disease cystic fibrosis was discovered, paving the way for disease-modifying drugs. In that time, only one such drug has been approved in the U.S. and Europe — Vertex’s Kalydeco. And that drug is only approved for the 4 percent of CF patients who […]
Friday’s U.S. Food and Drug Administration approval of the TOBI Podhaler was great news for drugmaker Novartis and for the cystic fibrosis community. It was also welcome news for Texas drug startup Savara Pharmaceuticals, whose CEO Rob Neville said he was “delighted that the FDA has approved a very necessary product.” Novartis’s drug is an […]