Quotient Sciences Quotient Sciences

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[{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed First to Advance Cannabinol (CBN) into Therapeutic Clinical Trials","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inovio Publishes Patient Benefit Results From A Pilot Clinical Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completed Enrollment in Phase 1 Clinical Trial of INM-755 CBN","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Submits Clinical Trial Application to Evaluate INM-755 in Second Phase 1 Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completion of Initial Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Science Translational Medicine Publication of its Program in Danon Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Dosing Initiation in 2nd Phase 1 Trial with INM-755","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Receives FDA Fast Track Designation for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces First Patient Treated in Higher Dose Cohort in Phase 1 Clinical Trial of RP-A501 for Danon Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completion of Subject Treatments in Second Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants AT-007 Pediatric Rare Disease Designation and Orphan Designation for Treatment of PMM2-CDG","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Applied Therapeutics"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Roth Capital Partners","pharmaFlowCategory":"D","amount":"$8.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces the Closing of US$8 Million Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","amount":"$3.7 million","upfrontCash":"Undisclosed","newsHeadline":"Rocket Pharma Gets Funding from the California Institute for Regenerative Medicine for Phase 1 Clinical Trial of RP-L401 for Infantile Malignant Osteopetrosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$4.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Announces Closing of US$4.5 Million Private Placement","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Precigen","sponsor":"National Institutes of Health","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Precigen Receives FDA Orphan Drug Designation for PRGN-2012 AdenoVerse\u2122 Immunotherapy for Recurrent Respiratory Papillomatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Vaccine","graph2":"Precigen"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharma Submits Clinical Trial Applications to Evaluate INM-755 (Cannabinol) Cream in a Phase 2 Trial for Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Initiates Phase 1 Clinical Trial of SAN711","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Beckley Psytech","sponsor":"Integrated","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"Beckley Psytech Completes Oversubscribed $80m (\u00a358m) Fundraise to Develop Portfolio of Psychedelic Medicine Breakthroughs","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Beckley Psytech"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Initiates Multiple Ascending Dose Stage of SAN711 Phase 1 Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Xentria","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xentria to Present Initial Findings from Preclinical Study of XTMAB-16 at ATS 2022 International Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Xentria"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge CAN106 Phase 1 Data Presented at the European Hematology Association 2022 Congress","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acer Therapeutics and Relief Therapeutics Announce ACER-001 IND Submission for the Treatment of Maple Syrup Urine Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Acer Therapeutics"},{"orgOrder":0,"company":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to Omeros\u2019 MASP-3 Inhibitor OMS906 for Treatment of Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Omeros"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics Announces IRB Approval and Initiation of an Investigator Initiated Trial of Nexodyn for Epidermolysis Bullosa at Ann & Robert H. Lurie Children's Hospital of Chicago","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Relief Therapeutics"},{"orgOrder":0,"company":"Rallybio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rallybio Announces Positive Phase 1 Single Ascending Dose Results for RLYB116, an Innovative Subcutaneously Injected Inhibitor of Complement Component 5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Rallybio"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Frontera Therapeutics Doses First Patient in Phase 1 Clinical Trial for Gene Therapy FT-001 for the Treatment of Leber Congenital Amaurosis-2","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Frontera Therapeutics"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Benitec Biopharma Enrolls First OPMD Subject into the Clinical Development Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Benitec Biopharma"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics Announces Enrollment of First Three Patients in Proof-of-Concept Clinical Trial of RLF-TD011 for the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Relief Therapeutics"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Trial of Novel Disease-Modifying Genetic Medicine, RCT1100 for the Treatment of Primary Ciliary Dyskinesia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ReCode Therapeutics"},{"orgOrder":0,"company":"BPGbio","sponsor":"Debra of America","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BPGbio Announces Expanded Partnership with debra of America for BPM 31510 for Epidermolysis Bullosa Phase II\/III Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"BPGbio"},{"orgOrder":0,"company":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Omeros Corporation Reports Interim Data with Alternative Pathway Inhibitor OMS906 as Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Omeros"},{"orgOrder":0,"company":"Calico Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Calico Life Sciences Announces First Participant Dosed in Phase 1b Clinical Study Evaluating ABBV-CLS-7262 for the Treatment of Vanishing White Matter Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Calico Life Sciences"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Receives Regulatory Clearance from FDA to Commence Phase 2 STARBORN-1 Trial of TARA-002 in Pediatric Patients with Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Protara Therapeutics"},{"orgOrder":0,"company":"TWi Biotechnology","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"TWi Biotechnology Exploring Promising Treatment for Granuloma Annulare (GA)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"TAIWAN","productType":"Small molecule","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"TWi Biotechnology"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NovelMed Phase I Clinical Trial Shows Inhibition of the Alternative Pathway and Preservation of the Classical Pathway A Long-Acting Anti-Properdin Monoclonal Antibody NM3086 for PNH Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","amount":"$7.5 million","upfrontCash":"Undisclosed","newsHeadline":"Azitra, Inc. Announces Pricing of Initial Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Azitra"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","amount":"$7.5 million","upfrontCash":"Undisclosed","newsHeadline":"Azitra, Inc. Announces Closing of Initial Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Azitra"},{"orgOrder":0,"company":"MyoPax","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MyoPax Receives Rare Pediatric Disease Designation from FDA for Breakthrough Regenerative Cell Product in Exstrophy-Epispadias Complex","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"GERMANY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"MyoPax"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"Benitec Biopharma Inc. Announces Closing of $30 Million Underwritten Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Benitec Biopharma"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Bioluminescence Ventures","pharmaFlowCategory":"D","amount":"$260.0 million","upfrontCash":"Undisclosed","newsHeadline":"ReCode Therapeutics Announces Closing of Extension to Series B Financing","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ReCode Therapeutics"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio Phase Ib trial of AB-1005 Gene Therapy in Patients with Parkinson\u2019s Disease Meets Primary Endpoint","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"AskBio"},{"orgOrder":0,"company":"Drug Farm","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Drug Farm Receives Rare Pediatric Disease Designation from the U.S. FDA for DF-003 to Treat ROSAH Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Drug Farm"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode Therapeutics Doses First Patient in Phase 1 Clinical Study of Novel mRNA-based Therapy for the Treatment of Primary Ciliary Dyskinesia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"ReCode Therapeutics"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"Azitra, Inc. 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Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development

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            The net proceeds will support the development of AV078, a first-in-class CNS penetrant selective mTORC1 inhibitor and prepare for the start of a subsequent Phase 2 clinical trial in adult and pediatric patients with TSC refractory epilepsy.

            Lead Product(s): AV078

            Therapeutic Area: Rare Diseases and Disorders Product Name: AV078

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Hevolution

            Deal Size: $50.0 million Upfront Cash: Undisclosed

            Deal Type: Financing March 28, 2024

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            The net proceeds will be used for the development of ATR12-351 (ATR-12), a novel strain of S. epidermidis that has been genetically modified to express and secrete an active fragment of the full-length protein LEKTI. which is evaluated for the treatment of Netherton syndrome.

            Lead Product(s): ATR-12

            Therapeutic Area: Rare Diseases and Disorders Product Name: ATR12-351

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: ThinkEquity

            Deal Size: $5.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 16, 2024

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            The company intends to use the net proceeds for clinical trials and product development of ATR12-351 (ATR-12) for the treatment of Netherton Syndrome.

            Lead Product(s): ATR-12

            Therapeutic Area: Rare Diseases and Disorders Product Name: ATR12-351

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: ThinkEquity

            Deal Size: $5.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 13, 2024

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            DF-003 is a potent small molecule inhibitor of ALPK1, which plays an important role in immunity and inflammation. It is under phase 1 clinical development for the treatment of ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) Syndrome.

            Lead Product(s): 8-Bromo-pet-cyclic gmps

            Therapeutic Area: Rare Diseases and Disorders Product Name: DF-003

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 13, 2024

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            AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene. It is under phase 1 clinical development for the treatment of Parkinson's disease.

            Lead Product(s): AAV2-GDNF

            Therapeutic Area: Rare Diseases and Disorders Product Name: AB-1005

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Bayer AG

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 04, 2024

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            RCT1100 is an inhaled mRNA therapy designed to deliver and express DNAI1 mRNA in target cells. It is under phase 1 clinical development for the treatment of Primary Ciliary Dyskinesia.

            Lead Product(s): RCT1100

            Therapeutic Area: Rare Diseases and Disorders Product Name: RCT1100

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 03, 2024

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            SGX945 (dusquetide) is a peptide inhibitor administered intravenously via infusion, which is being investigated for treating Aphthous Ulcers in Behçet's Disease.

            Lead Product(s): Dusquetide

            Therapeutic Area: Rare Diseases and Disorders Product Name: SGX945

            Highest Development Status: Phase I Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 30, 2023

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            LP659 is a S1P1/5 receptor Modulator, small molecule drug, which is currently being investigated for the treatment of rare neuroinflammatory conditions.

            Lead Product(s): LP659

            Therapeutic Area: Rare Diseases and Disorders Product Name: LP659

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 29, 2023

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            AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene. It is under phase 1 clinical development for the treatment of Parkinson's disease.

            Lead Product(s): AB-1005

            Therapeutic Area: Rare Diseases and Disorders Product Name: AB-1005

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 17, 2023

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            The proceeds will advance ReCode’s proprietary Selective Organ Targeting lipid nanoparticle pipeline, including Phase 1 trial of RCT1100 for primary ciliary dyskinesia and RCT2100, its cystic fibrosis candidate.

            Lead Product(s): RCT1100

            Therapeutic Area: Rare Diseases and Disorders Product Name: RCT1100

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Bioluminescence Ventures

            Deal Size: $260.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing September 19, 2023

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