Recon: Mustang Bio Soars After Gene Therapy Appears to Cure ‘Bubble Boy’ Patients

• ‘Bubble Boy’ Biotech Mustang Bio Soars 250 Percent After Cure Announcement (Bloomberg) (Reuters) (NPR) (Endpoints) (NIH)
• Pricing is taking a toll on the reputation of the pharmaceutical industry (STAT)
• A biotech startup’s lofty goal: Kill cancer with pills that target RNA instead of proteins (STAT) (Xconomy)
• Drugmakers Reveal List Prices Online After Pressure From Trump (Bloomberg)
• When a new biotech’s drug pipeline is more of a ‘hypeline’ (STAT)
• CRISPR edits lung-disease gene in utero, hitting only the affected organ in a mouse study (STAT) (Endpoints)
• Prostate Drugs May Raise Diabetes Risk (NYTimes)
• IBM halting sales of Watson AI tool for drug discovery amid sluggish growth (STAT)
• High-Deductible Health Policies Linked To Delayed Diagnosis And Treatment (NPR)
• Scammers May Be Using DNA Testing to Defraud Medicare and Steal Identities (Bloomberg)
• Cummings accuses Oversight Republicans of obstructing drug price probe (The Hill)
• Newsom plans to take on Big Pharma over prescription drugs. LA County wants in (LA Times)

• UK Government Investigating Illumina's Proposed Acquisition of Pacific Biosciences (GenomeWeb)
• WHO Releases First Guideline on Digital Health Interventions (Focus)
• Health Canada Encourages RWE Submissions (Focus)
• Big pharma’s billion-dollar bets (Financial Times)
• 'Fingerprint database' could help pinpoint environmental cancer causes (PharmaTimes)
• NICE final guidance published for Cimzia (PharmaTimes)
• Lonza plagued by supply problems after China plant explosion (Fierce)

• The Lifesaving Power in Stem Cells (NYTimes)
• Pharmas That Own Their Markets (Forbes)
• HIV drugmakers ViiV, Gilead top 2018 pharma reputation survey (Fierce)
• Exploiting machine learning for end-to-end drug discovery and development (Nature)
• Serial biotech entrepreneur Michael Gilman takes his key to the $100M-plus club and goes all-in on drugging RNA (Endpoints)
• On a mission to make organ transplants safer and more durable, Blackstone gifts Talaris a new CEO, leads $100M round (Endpoints) (Xconomy)
• Gilead-partnered Vienna-based immunotherapy company Hookipa makes Nasdaq debut with downsized $84M IPO (Endpoints)
• Acticor's blood thinner cuts hemorrhage risk in human study, could offer hope for acute stroke (Fierce)
• How a Chicago Woman Fell Victim to Candida Auris, a Drug-Resistant Fungus (NYTimes)
• Wotton replaces Gilman as CEO of cell, gene therapy startup Obsidian (Fierce)
• Bispecific Antibodies: FDA Drafts Guidance for Developers (Focus)
• Two Gene Therapy Approvals Headline CBER’s FY 2018 Report (Focus)
• Aurolife Pharma, LLC, et al.; Withdrawal of Approval of 31 Abbreviated New Drug Applications (FDA)

• Genfit bags FDA breakthrough tag en route to phase 3 in PBC (Fierce)
• FDA Grants Ultragenyx Rare Pediatric Disease Designation for Experimental Therapy (RARE)
• Moleculin Receives FDA Approval of Fast Track Designation for Annamycin (Press)
• Viela Bio Receives U.S. FDA Breakthrough Therapy Designation for Inebilizumab for Treatment of Neuromyelitis Optica Spectrum Disorder (Press)
• Vaxiion Therapeutics Initiates Phase 1 Clinical Trial of VAX014 for the Treatment of Non-Muscle Invasive Bladder Cancer in the United States (Press)
• Indalo Therapeutics Initiates Dosing in Phase 1 Clinical Trial of Lead Antifibrotic Drug Candidate IDL-2965 (Press)
• Soligenix Achieves Significant Enrollment Milestone for its Pivotal Phase 3 Clinical Trial of SGX942 in the Treatment of Oral Mucositis in Head and Neck Cancer (Press)
• Talaris Therapeutics, Inc. Announces Promising Phase 2 Data of Novel, Allogeneic Cell Therapy in Living Donor Kidney Transplant Recipients (Press)
• Innovent Announces First Patient Dosed in a Phase I Clinical Trial of IBI302 for the Treatment of Wet AMD (Press)

• Abbott Has Midas Touch with Its 1Q19 Earnings (MDDI)
• Acelity registers for another IPO (MassDevice)
• Medline Industries buys Namic business from AngioDynamics (MassDevice)
• Cook Medical releases patient-level data from Zilver PTX paclitaxel-coated balloon study (Drug Delivery)

• Oral arguments in Title X lawsuits get underway (Politico)
• Celgene Wants Sun Pharma's Revlimid Generic Blocked (Law360-$)
• United Therapeutics teamed with pumpmaker to block Remodulin generics, Sandoz suit says (Fierce)
• We are cutting government regulation at the expense of patients' lives (The Hill)
• Medicare Part D must evolve to help people fight cancer (STAT)
• Neovasc settles final spat with Edwards unit CardiAQ Valve (MassDevice)
• J&J Dodges Pelvic Mesh Claims In Philadelphia Trial (Law360-$)
• Biologic Manufacturers Should Expect Antitrust Scrutiny (Law360-$)
• FSIS Proposes to Simplify Labeling Compliance by Removing the Requirement for Dual Declaration of Net Content (FDA Law Blog)
• Double Dose Of Delaware Summary Judgment (Drug & Device Law)

• FDA Advisory Committee Calendar

• European Parliament Passes SPC Waiver in Landslide Vote (Focus)
• MedTech Europe Warns Over ‘Untenable’ Transition to EU MDR/IVDR (Focus)
• EU Regulatory Roundup: Commission Outlines How EU Data Protection Rules Affect Clinical Trial Sponsors (Focus)
• France’s Sanofi contributes €10M to the restoration of Notre Dame (Endpoints)

• Lupin seeks more time from commerce ministry to complete Indore SEZ project (Economic Times)
• Low-cost Chinese APIs throng Indian market illegally, pose health risk to people (PharmaBiz)
• Granules India gets USFDA nod for pain relief drug (Economic Times)
• Regulator directs drug manufacturers to incorporate new recorded adverse effects in leaflets (Economic Times)
• Hair in one vail, Aurobindo recalls 88k Lidocaine injections from US (Economic Times)