Hemophilia News and Research

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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.

Subcutaneous injection of new protein helps treat hemophilia more effectively

Patients with hemophilia bleed spontaneously or following trauma. The most common sites of bleeding are into joints, but bleeds can also happen into any tissue in the body including the muscles, brain, or eyes.

31 Aug 2018

Promising data supports development of AAVhu37-based gene therapy for hemophilia A

Gene therapy using an optimized adeno-associated virus to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks in some animals.

21 Aug 2018

RCSI and Bayer collaborate to improve treatments for people with hemophilia

RCSI and Bayer have today announced a research collaboration that aims to improve treatments for people with severe haemophilia.

9 Aug 2018

Scientists uncover new strategy to kill tumors without harming healthy cells

Scientists at Scripps Research have uncovered a new strategy to kill tumors, including some triple-negative breast cancers, without harming healthy cells, a discovery that could lead to more ways to treat tumors while reducing side effects.

26 Jul 2018

Researchers use genome editing to reduce cholesterol levels in animal model

Using genome editing to inactivate a protein called PCSK9 effectively reduces cholesterol levels in rhesus macaques, a species of monkey, according to researchers from the Perelman School of Medicine at the University of Pennsylvania.

10 Jul 2018

Study finds that several existing anti-cancer drugs may work in part by binding to RNA

Bolstering the notion that RNA should be considered an important drug-discovery target, scientists at Scripps Research have found that several existing, FDA-approved anti-cancer drugs may work, in part, by binding tightly to RNA, the regulators of the basic activities of life within cells.

30 Jun 2018

New study finds way to help treat alcohol addiction and ease withdrawal symptoms

According to a new study by Scripps Research scientists, activating a receptor found in brain cells may help treat alcohol addiction and ease some withdrawal symptoms. The researchers studied the effects of activating the receptor in alcohol-addicted rats and reported the results today in the journal eNeuro.

27 Jun 2018

ABPI associated with NHS for 70 years in pharmaceutical research

In this special year, both the NHS and the Association of the British Pharmaceutical Industry are celebrating our respective 70th anniversaries. The histories of these two organizations have been inextricably linked since 1948 and our relationship with the NHS continues to go from strength to strength.

25 Jun 2018

Study provides clues to how cancer cells develop resistance to chemotherapies

About half of all drugs, ranging from morphine to penicillin, come from compounds that are from - or have been derived from - nature. This includes many cancer drugs, which are toxic enough to kill cancer cells.

22 Jun 2018

Chemists invent new method for remote chiral induction

Chemists at Scripps Research have addressed one of the most formidable challenges in synthetic chemistry by inventing a method for "enantioselective remote meta-CH activation," which enables the making of chiral molecules that were previously difficult or impossible to synthesize.

19 Jun 2018

Researchers use droplet-sized 'miniecosystems' to test therapeutic potential of molecules

Scripps Research scientists have solved a major problem in chemistry and drug development by using droplet-sized 'miniecosystems' to quickly see if a molecule can function as a potential therapeutic.

19 Jun 2018

Scientists could build better drugs by learning from bacteria-derived molecules

Bacteria found in soil may harbor a potential game-changer for drug design. A new study by Scripps Research, published today in Nature Communications, suggests scientists could build better drugs by learning from bacteria-derived molecules called thiocarboxylic acids.

19 Jun 2018

Study finds increased risk of mortality in people with cold agglutinin disease in five years after diagnosis

Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, today announced results from a new, population-based retrospective study that found a greater than twofold increased risk of mortality in people with cold agglutinin disease within the first five years after diagnosis.

18 Jun 2018

Scientists engineer bi-specific antibodies that attract immune killers directly to cancer cells

Our immune system's arsenal of defenses usually protects us from cancer. But sometimes, cancer cells overwhelm or evade this elaborate defense system.

30 May 2018

New RNA deletion tool could help create drugs that correct genetic diseases

As scientists gain insights into which genes drive diseases, they are pursuing the next logical question: Can gene editing technologies be developed to treat or even cure those diseases? Much of that effort has focused on developing technologies such as CRISPR-Cas9, a protein-based system.

30 May 2018

LogicBio team to present data on preclinical effectiveness of GeneRide platform

LogicBio Therapeutics, Inc., a genetic medicine company founded to develop safe medicines with lasting therapeutic benefit for children with life-threatening diseases, today announced it will be presenting data on the company's technology and robust preclinical pipeline at the annual meeting of the American Society of Gene & Cell Therapy at the Hilton Chicago, from May 16-19, 2018.

14 May 2018

Researchers devise neuronal cookbook for turning skin cells into different types of neurons

Scientists at The Scripps Research Institute have devised what they call a "neuronal cookbook" for turning skin cells into different types of neurons.

10 May 2018

Scripps Research professor wins 2018 RSC Bioorganic Chemistry Award

Floyd Romesberg, PhD, professor at The Scripps Research Institute, has won the 2018 Royal Society of Chemistry Bioorganic Chemistry Award for his ground-breaking contributions to the expansion of the genetic alphabet.

9 May 2018

Scientists discover how potential new drug targets spinal muscular atrophy

Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die before age two. To help these patients, doctors need therapies that target the genetic mutation and stop its progression.

8 May 2018

Scripps Research team receives $12 million grant to improve flu and malaria vaccines

Scientists at The Scripps Research Institute aim to improve flu and malaria vaccines with the support of a new $12 million grant from the Bill & Melinda Gates Foundation.

3 May 2018

Hemophilia News and Research

Further Reading

Subcutaneous injection of new protein helps treat hemophilia more effectively

Promising data supports development of AAVhu37-based gene therapy for hemophilia A

RCSI and Bayer collaborate to improve treatments for people with hemophilia

Scientists uncover new strategy to kill tumors without harming healthy cells

Researchers use genome editing to reduce cholesterol levels in animal model

Study finds that several existing anti-cancer drugs may work in part by binding to RNA

New study finds way to help treat alcohol addiction and ease withdrawal symptoms

ABPI associated with NHS for 70 years in pharmaceutical research

Study provides clues to how cancer cells develop resistance to chemotherapies

Chemists invent new method for remote chiral induction

Researchers use droplet-sized 'miniecosystems' to test therapeutic potential of molecules

Scientists could build better drugs by learning from bacteria-derived molecules

Study finds increased risk of mortality in people with cold agglutinin disease in five years after diagnosis

Scientists engineer bi-specific antibodies that attract immune killers directly to cancer cells

New RNA deletion tool could help create drugs that correct genetic diseases

LogicBio team to present data on preclinical effectiveness of GeneRide platform

Researchers devise neuronal cookbook for turning skin cells into different types of neurons

Scripps Research professor wins 2018 RSC Bioorganic Chemistry Award

Scientists discover how potential new drug targets spinal muscular atrophy

Scripps Research team receives $12 million grant to improve flu and malaria vaccines

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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