26 Apr

Treating and Curing Disease Through Human Genome Editing

DNA defines who we are. And like anything else, sometimes there are mistakes – mutations in genes that can cause life-lasting conditions. But what if broken genes could be repaired? How many patients could we help in the years ahead? This is the promise and possibility of human genome editing.

While DNA modification techniques have existed for decades, recent developments in genome editing technologies have provided scientists and researchers with far more precise and efficient tools than ever before. As a result, 2017 marked the first genome editing clinical trials in humans initiated in the United States which are targeting a metabolic disorder known as Hunter Syndrome. And research is currently underway on clinical applications of genome editing technologies to treat genetic disorders like sickle cell disease, cystic fibrosis, congenital blindness, hemophilia, amyloidosis, and lysosomal storage disorders.

As we look beyond human health, these products can be applied to animal and plant DNA, as well as many organisms that are used in basic biological research. Applications in agriculture and animal health have the potential to deliver major advances to help feed the world.

To learn more about human genome editing, head to our Toolkit for Advocates and check out our infographic and issue brief on this important topic.

26 Apr

DARPA Enlists Insects in R&D Effort to Protect the Food Supply

Farmers spend a lot of time and money trying to keep insects from damaging their crops. But the U.S. military sees some of these bugs as potential friends, not foes. A government-funded research project is studying how to use insects to deliver a targeted therapy to a crop following an outbreak of disease, a disaster, or even a terrorist strike.

The initiative comes from the Defense Advanced Research Projects Agency, the research arm of the military. According to Blake Bextine, program manager in the agency’s biological technologies office, protecting the food supply is a matter of national concern and insects can… Read more »

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25 Apr

Vaccines: Vital in fighting antimicrobial resistance

In recognition of BIO’s 25th Anniversary, each month we are celebrating historical milestones in biotech. This week marks World Immunization Week and we are pleased to share a guest post from Thomas Cueni, Director General of the IFPMA, and Chair of the AMR Industry Alliance on the role that immunization plays in fighting antimicrobial resistance.

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We have been lucky to live through a period of improving global public health, facilitated in part by innovation from and partnerships with the pharmaceutical industry. However we now find ourselves facing a new threat from an old foe – infectious disease through antimicrobial resistance (AMR). The challenge ahead is extensive but this World Immunization Week reminds us that we already have access to an underused AMR defense – vaccines.

AMR develops when an infectious microorganism (bacteria, virus, parasite and fungus) no longer responds to a drug to which it was originally sensitive. Drugs can lose their effect because the microbes change; either they mutate or acquire genetic information from other microbes to develop resistance. The phenomenon is accelerated by use, and especially misuse, of antimicrobial medicines whereby resistant strains survive and aggregate.

Antibiotic resistance has the power to transport us back to a time when minor infections were deadly. As an example, since the 1970s the typhoid bacteria has been evolving and typhoid, an otherwise vaccine-preventable infectious disease, is becoming increasingly resistant to antibiotics, resulting in the emergence of multi-drug resistant typhoid. AMR is responsible for around 700,000 deaths annually but this could rise to 10 million by 2050, surpassing the number of deaths expected from cancer, if major action isn’t taken soon. Beyond the tremendous increase in mortality, direct medical costs and loss in productivity could cause a huge financial burden of around 60–100 trillion US dollars a year.

Immunization has a crucial role to play in the reduction of AMR. Vaccines are one of the most cost-effective health tools; every dollar spent on immunization is estimated to provide returns of $44 in economic and social benefits[i]. In regards to AMR, vaccines can help to:

  • Reduce the use of antibiotics by preventing bacterial infections before they occur, thus removing any need to treat the infection. Increasing access to vaccines such as pneumococcal and meningococcal conjugate can decrease infection rates and consequently antibiotic use
  • Reduce the prevalence of viral infections, which are often inappropriately treated with antibiotics and can give rise to secondary infections that require antibiotic treatment. Immunization against viral influenza can reduce antibiotic use by as much as 64% in vaccinated individuals[ii]
  • Reduce the number of infections in the population through direct protection of vaccinated individuals and by reducing carriage (the infection of an individual without causing symptoms), thus limiting the spread of infections within a community (herd immunity)
  • Limit the spread of AMR organisms within and across communities by reducing the volume of visits to healthcare points of care, especially hospitals, which are themselves a source of infection

Vaccines can be utilized in the fight against AMR in three main ways. Firstly, current vaccination efforts through National Immunization Programs need to be encouraged and universal access to existing vaccines needs to be expanded. The global health efforts to eradicate smallpox show that this is a feasible prospect.

Secondly, immunization programs need to be offered to children and adults alike as part of a life-course approach to preventing disease, which stresses the importance of vaccination at all stages of life to help prevent illness. Adult immunization programs provide a cost-effective approach to promoting health and wellness in older populations (whose immune systems gradually weaken) and in people who have underlying health problems.

Finally, industry needs to prioritize the R&D of new vaccines – especially for diseases that pose the biggest threat to drug resistance (the WHO has a priority list of antibiotic-resistant bacteria). There are scientific and commercial challenges in developing new vaccines and more investments from both public and private sources, as well as economic and regulatory incentives, are needed to overcome these hurdles.

Expanding access to vaccines should be viewed as a key component to achieving Universal Health Coverage and most of the SDGs, but is it also a fundamental strategy in achieving other health priorities such as AMR. The scale of antimicrobial resistance has never been so large, and with that global attention is high. In 2016, over 100 companies signed the Industry Declaration on AMR and 193 countries committed to fighting AMR by signing a UN declaration on antimicrobial resistance.

The AMR Industry Alliance is an industry initiative that brings together over 100 companies breaking silos between different sectors such as biotechs, diagnostics, generics and R&D pharma. It ensures signatories deliver on specific commitments made in the Industry Declaration on AMR and the Industry Roadmap. One of the key pledges is to foster innovative approaches to using alternatives to antimicrobials and new technologies for diagnosis and vaccines. The Alliance calls for an integrated deployment of vaccines and medicines, diagnostics, antibiotics and other therapies to address the multiple challenges across the continuum of care – from prevention, monitoring and screening to treatment.

Universal vaccination and the successful development of new or improved vaccines are important measures in the long-haul fight against AMR. We are dedicated to working together as an industry to address AMR and partnering with world leaders and donors to ensure immunization can play its full role in achieving global health security.

[i] Ozawa S, et al. Return On Investment From Childhood Immunization In Low- And Middle-Income Countries, 2011–20. Health Affairs. 2016; 35(2)

[ii] Wilby K, Werry D. A review of the effect of immunization programs on antimicrobial utilization. Vaccine. 2012; 30(46): 6509-6514

25 Apr

SMA Day: AveXis Gene Therapy Creeps Forward as Biogen Drug’s Sales Slow

Just a few years ago, there were no drugs available for patients with the rare genetic disease spinal muscular atrophy. Now two cutting-edge therapies look headed for a commercial battle, with each data and revenue update a key moment in the race.

Biogen (NASDAQ: BIIB) was the first to bring an SMA drug to market, the RNA drug nusinersen (Spinraza), a chronic treatment that has shown an ability to help people with multiple forms of the progressive disease. But sales of the landmark drug are slowing, and there is pressure building from rival AveXis (NASDAQ: AVXS), the Chicago-based… Read more »

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25 Apr

CSA for Lighthouse Initiative Industry 4.E

[Source: http://ec.europa.eu/health/ageing/innovation/index_en.htm] Identifier: H2020-ECSEL-2018-3-CSA-Industry4E-one-stagePillar: Industrial LeadershipOpening Date: Deadline: Thu, 24 May 2018 17:00:00 (Brussels local time)Modification Date: Wed, 25 Apr 2018Latest information: The
ECSELJUWork Plan 2018
has been updated but none of the changes/updates affects this Call.

25 Apr

CALL FOR PROPOSALS FOR ACTION GRANTS UNDER 2018 RIGHTS, EQUALITY AND CITIZENSHIP WORK PROGRAMME

[Source: http://ec.europa.eu/health/ageing/innovation/index_en.htm] Identifier: REC-AG-2018Pillar: REC Programme 2014-2020Opening Date: Deadline: Thu, 26 Apr 2018 17:00:00 (Brussels local time)Modification Date: Wed, 25 Apr 2018Latest information: The submission session is now available for: REC-RRAC-HATE-AG-2018(REC-AG)

24 Apr

With TMC3, Texas Leaders Aim to Launch Houston as Top Biotech Hub

Houston-Texas political leaders, along with heads of the Texas Medical Center and its institutions, announced on Monday plans for a biotech-focused hub that could place Houston on the map of life sciences innovation centers.

The TMC Translational Research campus, or TMC3, is a planned $250 million research center in the shape of a double helix that would include restaurants, bars, retail, housing, and a conference center and hotel. Bond covenants prohibiting commercial activity at the TMC properties were removed for this specific project, and officials said they expect pharma and other businesses to join physicians and researchers at the TMC3… Read more »

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24 Apr

Call for proposals for multi programmes 2018 – Promotion of agricultural products

[Source: http://ec.europa.eu/health/ageing/innovation/index_en.htm] Identifier: AGRI-MULTI-2018Pillar: AGRIP-1-1Opening Date: Deadline: Thu, 19 Apr 2018 17:00:00 (Brussels local time)Modification Date: Tue, 24 Apr 2018Latest information: 36 proposals were submitted in response to the 2018 call for proposals formulti programmes for the promotion of agricultural products.

24 Apr

How prestigious/helpful is a internship at Genentech gRED?

Hello!

I am a current grad student in chemistry and was offered an research internship opportunity at Genentech. Here is my dilemma: I have fallen back on my research and was thinking about skipping on the internship to focus on finishing up my current research project. However, the more I read about the internship, the more it sounds like this would be a bad idea; it seems that the selection process is very competitive and it can quite helpful career-wise.

Has anyone done the internship? Was it helpful to your career? Should I just focus on my research instead?

Thank you all for your advice!

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24 Apr

Anthony Hopkins has the strangest and most wonderful Twitter account

But the true measure of Sir Anthony Hopkins’ genius is his beautiful, thoughtful, and fabulously bizarre Twitter account. If you have not been following Anthony Hopkins on Twitter, I am sorry, for you have been missing out on almost two years of strange selfies, psychedelic artwork, and motivational …