.............113.............
Also, we should learn the outcome from a large clinical trial of an infusion of broadly neutralizing antibodies (bnAbs) to prevent HIV infection. Encouraging results will motivate researchers to refine and improve the concept, especially now that the field has discovered more bnAbs and that we have the scientific capabilities to engineer next-generation HIV antibodies to be more potent and more long-lasting.
And while we’re hopeful that these vaccine candidates and antibodies will be efficacious, we need to plan now for making them affordable and accessible to the people who need them most. In 2020, we in the field must collaborate even at these early stages of development to ensure that these new products will be widely and promptly available once approved.
— Dr. Mark Feinberg, president and CEO,
International AIDS Vaccine Initiative
A ‘pivotal year’ for CRISPR-based therapies
2020 will be a pivotal year for engineered cell therapies driven by CRISPR in rare diseases and in oncology, and CRISPR will solidify its place in medicine. While 2019 showed us a glimpse of promising data in transfusion-dependent thalassemia and sickle cell disease, we will see additional data across a range of devastating diseases with significant patient need — including various heme malignancies. The other wave we will see is the use of CRISPR in regenerative medicine to develop implantable cells in a range of organs — whether pancreas, liver or other cells. Finally, we will see additional progress with in vivo approaches using CRISPR, due to the advances we are making in viral and non-viral delivery technologies. CRISPR’s promise continues to become a reality.
— Sam Kulkarni, CEO of CRISPR Therapeutics
Aiming to reduce worldwide deaths of under-5 children
Today, we are closer than ever before to bringing global under-5 child mortality below 5 million. But to get there, we need to provide a complete package to health workers to treat fever, which can kill children in just a few hours. A child under 5 in sub-Saharan Africa gets an average of six bouts of fever a year. A substantial percentage of these are likely to be due to malaria, but also bacterial and viral infections that show up as pneumonia and diarrhea. Undiagnosed and treated, pneumonia, diarrhea, and malaria can weaken and kill children in as quickly as 24 hours. And yet treatment and prevention for these three conditions are extremely cheap.
With the right programs, the right partners and the right levels of funding, we can make sure that a child who has fever gets adequate treatment, whether antimalarials, antibiotics, oral rehydration solution, or simply antipyretics. We have an extraordinary opportunity to drive this change and make 2020 a game-changing year for children – a year when we change the curve of child mortality and accelerate progress toward the Sustainable Development Goals. Millions of children and their families are counting on us.
— Henrietta Fore, executive director, UNICEF
Explosion in drugs targeted to patients using biomarkers
The Personalized Medicine Coalition is tracking an increase in the number of available drugs that are associated with biomarker-based targeting strategies. The coalition expects this number to exceed 230 in 2020, up from five in 2008 and 132 in 2016. In addition to this increased use of strategies that evaluate patients to determine which therapies are the best match for them, we are likely to see continued movement toward the development of drugs customized for individuals, like CAR T-cell therapies.
— Edward Abrahams, president, Personalized Medicine Coalition
Digital health culling will begin in earnest
I think 2020 will be the year that digital health consolidation and culling begins in earnest — we are already starting to see it. I doubt there will be much IPO activity outside of what are actually large services business that call themselves digital health but are primarily health services businesses. I think investment dollars may stay the same because a lot of money has been raised, but we will continue to see concentration on fewer companies, particularly those that have actually proven their financial value proposition to clients — the burden of proof is becoming much more onerous, and that is entirely appropriate for a market that ran up a lot on hype or hope, depending on how you look at it. But hope is not a strategy, as the saying goes, so the companies that will garner investment will be the ones committed to doing the hard work of proving clinical and financial ROI to their clients and, finally, a rational growth and profitability plan to their investors.
— Lisa Suennen, managing director and leader of Digital & Technology Group, Manatt
Flashmag January 2020 www.flashmag.net