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Highlight on Gene Therapy
Panel Recap: The promise, the reality, the future of Gene Therapy

A recent TIDO panel at Boston Children’s Hospital featuring David Williams, MD, Chief Scientific Officer of Boston Children's, Philip Reilly, MD, JD, of Third Rock Ventures, Philip Gregory, DPhil, Chief Scientific Officer of bluebird bio, discussed where gene therapy currently stands, which areas success is most anticipated, and the impact it will have on patient treatment. The panel delved into the technical challenges, the translational process, the complex regulatory paths, and how a therapy may be priced. 
Gene Therapy restores whisper-fine hearing, balance in Usher syndrome mice

Researchers at Boston Children's reported in Nature Biotechnology the ability to restore a high level of hearing in Ush1c mice, down to the equivalent of a whisper, by delivering a specific corrected gene using a synthetic vector in the cochlea. Previous vectors were able to safely penetrate inner hair cells. This new vector, Anc80, which was developed at Massachusetts Eye and Ear Institute, safely transferred genes to outer hair cells in addition to inner hair cells. Of 25 mice tested by measuring responses in auditory brain regions, 19 heard sounds quieter than 80 decibels.
News in STAT: One boy’s cure raises hopes and questions about gene therapy for sickle cell disease (David Williams, MD; Subscription needed)
Boston Children's Research Showcase and Networking Reception
We invite you to learn more about Boston Children’s research advances in rare diseases and hear from our researchers in hematology, oncology, pulmonary, cardiology, neurology and the Manton Center for Orphan Disease Research. A panel discussing strategies to bridge the gap between academic research and industry drug development will feature David Williams, MD, Chief Scientific Officer at BCH, Alan Beggs, PhD, Director of the Manton Center, Philip Reilly, MD, JD, Venture Partner, Third Rock Ventures, and Alvin Shih, MD, MBA, ‎CEO, Enzyvant. This event is the first of three scientific networking events organized by Boston Children’s Hospital and hosted by MassBio.

Highlighting Immunology Opportunities

Boston Children’s immunology research programs are led by the Division of Immunology, and the Program in Cellular and Molecular Medicine (PCMM), previously known as the Immune Disease Institute (IDI). BCH researchers in these departments have had success launching startups such as Moderna, Morphic Rock, and Scholar Rock. Below are some exciting research opportunities available for partnership: 

Reinvigorating the body’s immune system to fight chronic infection. Chronic viral infection, which affects hundreds of millions of patients worldwide, can lead to exhaustion of cytotoxic T cells, weakening of the patient’s immune system and higher associated mortality. The lab of Florian Winau, MD, has identified a critical role for lipid scramblase TMEM16F and showed that activation of TMEM16F is key for preventing over-activation of T cells and subsequent fatigue. Targeting TMEM16F may lead to improved recovery from chronic viral infections. This strategy alone or in combination with an immune checkpoint therapy may serve to treat cancer.

Broadly neutralizing antibodies against influenza variants. Every year, researchers produce flu vaccines targeting the likely influenza strains for the season; however these predictions are sometimes incorrect, as happened in the 2015-2016 season. The lab of Stephen Harrison, PhD, has discovered antibody configurations that are effective against numerous influenza strains spanning three decades of flu season, thereby providing a strategy to develop broadly neutralizing therapeutics against influenza.

Blocking NETs in the body to prevent multiple conditions. When immune cells named neutrophils die, they release neutrophil extracellular traps (NETs). These NETs may kill pathogens but can also cause severe damage to healthy tissue. The lab of Denisa Wagner, PhD, has found that stopping the formation of NETs prevents a number of specific health conditions, including deep vein thrombosis, blood clots in cancer, transfusion-related acute lung injury, and impaired wound healing in diabetic patients. Therefore, a strategy to develop NETS inhibiting therapeutics could prevent these conditions.

News in MedicalXpress: Cargo-carrying red blood cells alleviate autoimmune diseases in mice (Vijay Sankaran, MD, PhD)