[{"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis to Initiate SMART Phase 3b Global Study of Zolgensma in Children up to 21 kg, Building on Real-World Experience","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Novartis Pharmaceuticals Corporation"},{"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Announces Lift of Partial Clinical Trial Hold and Plans to Initiate a New, Pivotal Phase 3 Study of Intrathecal OAV-101 in Older Patients With SMA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Novartis Pharmaceuticals Corporation"},{"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche Trial of Ionis Huntington\u2019s Disease Drug Halted After Side Effects","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"F. Hoffmann-La Roche"},{"company":"F. Hoffmann-La Roche","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche Provides Update on Tominersen Programme in Manifest Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"F. Hoffmann-La Roche"},{"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Chugai In-Licenses Gene Therapy Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"F. Hoffmann-La Roche"},{"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa\u00ae (Idebenone) in Duchenne Muscular Dystrophy (DMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","amount":"$21.1 million","upfrontCash":"Undisclosed","newsHeadline":"Santhera Receives Financing Commitment of up to CHF 20 Million from Fund Managed by Highbridge Capital Management","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"UK\u2019s MHRA renews EAMS Scientific Opinion for Santhera\u2019s Idebenone in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of\n Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"company":"Idorsia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Idorsia Announces the Results of MODIFY, a Phase 3 Study of Lucerastat in Fabry disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Idorsia Pharmaceuticals"},{"company":"Idorsia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Idorsia to Further Characterize Lucerastat for The Treatment of Fabry Disease by Continuing the Open-Label Extension of the Phase 3 MODIFY Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Idorsia Pharmaceuticals"}]
Details:
Under the license agreement between Roche and Chugai, Chugai obtained exclusive marketing right in Japan for delandistrogene moxeparvovec (SRP-9001), an investigational gene therapy for Duchenne muscular dystrophy.
Lead Product(s):
Delandistrogene Moxeparvovec
Therapeutic Area: Genetic Disease
Product Name: SRP-9001
Highest Development Status: Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Chugai Pharmaceutical
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Licensing Agreement
December 16, 2021
Details:
Lucerastat demonstrated a substantial reduction in levels of the Fabry-disease biomarker plasma Gb3 after 6 months of treatment. The potential effect of lucerastat on kidney function over 6 months of treatment was evaluated at the interim analysis of the extension study.
Lead Product(s):
Lucerastat
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
December 13, 2021
Details:
The safety, tolerability, pharmacodynamics, and pharmacokinetics of oral lucerastat were evaluated in study in adult patients with Fabry disease. In this single-center, open-label, randomized study, 10 patients with Fabry disease were randomized to lucerastat for 12 weeks.
Lead Product(s):
Lucerastat
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 11, 2021
Details:
STEER will build upon the Phase 1/2 study which showed that treatment with OAV-101 IT led to significant increases in Hammersmith Functional Motor Scale-Expanded scores and a clinically meaningful response in older patients between ≥2 years and <5 years old with SMA Type 2.
Lead Product(s):
OAV-101
Therapeutic Area: Genetic Disease
Product Name: OAV-101
Highest Development Status: Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 03, 2021
Details:
New clinical study to evaluate safety and efficacy of Zolgensma in children up to 21 kg, adding to real-world experience and regulatory approvals in Europe and Canada.
Lead Product(s):
Onasemnogene abeparvovec
Therapeutic Area: Genetic Disease
Product Name: Zolgensma
Highest Development Status: Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
April 23, 2021
Details:
Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT.
Lead Product(s):
Tominersen
Therapeutic Area: Genetic Disease
Product Name: RG6042
Highest Development Status: Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Ionis Pharmaceuticals
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
March 22, 2021
Details:
Data from an interim analysis conducted by the independent Data and Safety Monitoring Board concluded that the Phase 3 SIDEROS study with Puldysa® (idebenone) was unlikely to meet its primary endpoint.
Lead Product(s):
Idebenone
Therapeutic Area: Genetic Disease
Product Name: Puldysa
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 06, 2020
Details:
Data from an interim analysis conducted by the (DSMB) concluded that the study was unlikely to meet its primary endpoint. Santhera will discontinue the study, withdraw the European marketing authorization application and end the global development program for Puldysa.
Lead Product(s):
Idebenone
Therapeutic Area: Genetic Disease
Product Name: Puldysa
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 06, 2020
Details:
Uk's Medicines and Healthcare products Regulatory Agency has renewed for a further year the Early Access to Medicines Scheme scientific opinion for idebenone for patients with Duchenne muscular dystrophy in respiratory function decline who are not taking glucocorticoids.
Lead Product(s):
Idebenone
Therapeutic Area: Genetic Disease
Product Name: Puldysa
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 23, 2020
Details:
In combination with Santhera's existing cash and cash equivalents, this financing will provide the Company with sufficient funding to complete regulatory work for Puldysa®, including the CHMP review, and with the achievement of Company milestones.
Lead Product(s):
Idebenone
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Highbridge Capital Management
Deal Size: $21.1 million
Upfront Cash: Undisclosed
Deal Type: Financing
June 04, 2020
Market Place
News Type
Upload your Marketing & Sales content on your company Virtual Booth, click HERE
