GSK eyes robust immune response from RSV vaccine 

Cold viruses mostly do not get much attention in terms of R&D. They usually make humans feel miserable for a few days, and later than clear up. However, some cold viruses can hit vulnerable people much harder, which leads to pneumonia and hospitalizations. These are colds caused by the respiratory syncytial virus (RSV) in the elderly and children under 5, causing severe complications and can prove fatal sometimes.

In younger children, it can cause bronchiolitis and pneumonia in both infants and the elderly, and it is thought to cause about 3 million hospitalizations of children under five globally, with around 177,000 hospitalizations of older people in the U.S. There have been many attempts by Big Pharma at a vaccine, but there had been setbacks.

GlaxoSmithKline took a step closer to finding the vaccine, announcing data from several candidates at IDWeek 2020 from several midstage trials, as it currently plots a late-stage make-or-break series of tests. The RSV candidate vaccines were for two different groups: one for maternal immunization (GSK3888550A) and one for older adults (GSK3844766A). Top-line, GSK informed that both were well-tolerated and highly immunogenic in phase 1/2 clinical studies.

SparingVision raises USD 52.5 million for the development of mutation-agnostic gene therapy

SparingVision has raised USD 52.5 million for developing its mutation-agnostic gene therapy treatment for retinitis pigmentosa. SparingVision funds the clinical trials of an AAV gene therapy, which could stop vision worsening in the 2 million RP patients.

Gene therapies, including Luxturna of Spark Therapeutics, are designed to address genetic drivers that cause some patients to develop retinitis pigmentosa and suffer vision loss. However, with at least 65 distinct retinitis pigmentosa-causing mutations of three different types, the number of patients that can benefit from anyone gene-replacement therapy is relatively less. SparingVision, in contrast, is going after the whole retinitis pigmentosa market.  

The market potential is tremendous, unlike most of the gene therapies that are being developed, which address particular mutations. The commercial feasibility of those mutation-specific gene therapies is kind of questionable as per the new CEO of SparingVision, Stéphane Boissel. 

InCarda raises USD 30 Million to reimagine afib treatment with drug-device combo

Flecainide has been around for treating atrial fibrillation for the past four decades, but the CEO of InCarda Therapeutics contemplates that it requires a face-lift. Armed with USD 30 million in venture funding, InCarda expects to make the drug available as an at-home inhalable treatment for patients in the early stages of the disease.

Conventionally, flecainide is either delivered intravenously in a hospital setting or given as a pill, which has not been well understood for acute atrial fibrillation.

The patients in the early stages of paroxysmal atrial fibrillation (PAF) are often not treated as they face a few episodes of irregular heartbeats a year that go away within a week, as said by the CEO. These episodes are not comfortable, causing lightheadedness and a fluttering sensation in the chest, among other unpleasant symptoms. However, they are also dangerous: the longer the condition is left untreated, then it will likely develop into a chronic condition or cause a stroke.

BridgeBio looks out for more prospects in new academic deals

BridgeBio Pharma inked a pair of R&D pact with the University of Colorado and the Salk Institute. The deals are targeted at propelling research out of Salk and the University of Colorado’s Anschutz Medical Campus on genetically driven diseases, assuring that promising treatments do not end up overlooked in academia and get an opportunity at development. BridgeBio struck similar pacts this summer with the University of Florida and Johns Hopkins University.

Collaborations such as these are the lifeblood of BridgeBio that set up shop in 2015 to progress treatments for ignored rare diseases. Multiple players crowd into areas such as Duchenne muscular dystrophy while overlooking thousands of other rare diseases, which could use treatment.

Instead of searching for novel approaches for treating a particular disease or filling a gap in the pipeline, BridgeBio takes a more open-ended approach.