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05 Oct 2022 SyntheX and Bristol Myers Squibb Enter Into a Research Collaboration to Discover and Develop Targeted Protein Degradation (TPD) Therapeutics
05 Oct 2022 Voyager Therapeutics Announces Pfizer License of Next-Generation AAV Capsid for Rare Neurologic Disease Target
04 Oct 2022 Pfizer Announces Positive Topline Results from Phase 3 TALAPRO-2 Trial
04 Oct 2022 Incyte Announces Agreement to Acquire Medicxi-backed Villaris Therapeutics and Auremolimab (VM6), an Anti-IL-15Rβ Monoclonal Antibody
04 Oct 2022 Kezar Life Sciences Receives FDA Clearance of IND for Zetomipzomib for the Treatment of Autoimmune Hepatitis
04 Oct 2022 Kira Pharmaceuticals Receives FDA Clearance of IND Application for Phase 2 Evaluation of KP104 in Systemic Lupus Erythematosus Associated Thrombotic Microangiopathy (SLE-TMA)
04 Oct 2022 Astellas and Pantherna Enter into a New Technology Evaluation Agreement for Research with Expanded Target Organ to Generate mRNA-Based Regenerative Medicine Programs Using Direct Reprogramming
04 Oct 2022 Atsena Therapeutics Announces Positive Results from Phase I/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital Amaurosis (LCA1)
04 Oct 2022 Prometheus Biosciences Receives FDA Clearance of IND Application for its Second Precision Program, PRA052
04 Oct 2022 Y-mAbs Announces Pivotal Data for Omburtamab
03 Oct 2022 Alexion, AstraZeneca Rare Disease to Acquire LogicBio® Therapeutics to Accelerate Growth in Genomic Medicine
03 Oct 2022 Biogen and Denali Therapeutics Announce Initiation of the Phase 3 LIGHTHOUSE Study in Parkinson’s Disease Associated with LRRK2 Pathogenic Mutations
03 Oct 2022 Clene Reports Topline Results Demonstrating Survival Signal for CNM-Au8® in Healey ALS Platform Trial
03 Oct 2022 Late-Breaking Phase 1/2 Data Demonstrates Safety Profile of Investigational Gene Therapy Botaretigene Sparoparvovec (AAV-RPGR) and Sustained Vision Improvement in Patients with X-Linked Retinitis Pigmentosa (XLRP)
03 Oct 2022 Solid Biosciences Announces Acquisition of AavantiBio and Concurrent $75 Million Private Placement
03 Oct 2022 Santhera Submits Marketing Authorization Application to the European Medicines Agency for Vamorolone in Duchenne Muscular Dystrophy
01 Oct 2022 Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting
01 Oct 2022 FDA Approves Taiho's LYTGOBI® (futibatinib) Tablets for Previously Treated, Unresectable, Locally Advanced or Metastatic Intrahepatic Cholangiocarcinoma
30 Sep 2022 Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial for RP-A501 in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022
30 Sep 2022 Intercept Pharmaceuticals Announces REVERSE Phase 3 Study of Obeticholic Acid (OCA) in Compensated Cirrhosis due to NASH Did Not Meet its Primary Endpoint

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