[{"company":"Cycle Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"US FDA Grants Once Daily Dosing Option for NITYR (nitisinone) Tablets for HT-1 Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Cycle Pharmaceuticals"},{"company":"Orchard Therapeutics","sponsor":"Genpharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Secures Partnerships to Broaden Access to LibmeldyTM for Eligible Patients in Middle East & Turkey","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"company":"Diurnal Group","sponsor":"Citrine","pharmaFlowCategory":"D","amount":"$13.2 million","upfrontCash":"$0.5 million","newsHeadline":"Citrine Medicine Announces Strategic Partnership with Diurnal To Bring Alkindi\u00ae To China For Use In Pediatric Congenital Adrenal Hyperplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"company":"Orchard Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces $150 Million Strategic Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Outlines Comprehensive Presence at 2021 WORLDSymposium\u2122","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"company":"Diurnal Group","sponsor":"Citrine Medicine","pharmaFlowCategory":"D","amount":"$2.2 million","upfrontCash":"$1.0 million","newsHeadline":"Diurnal Triggers $1.25m in Milestone Payments from Citrine Medicine","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Group plc - Diurnal receives European Commission approval for Efmody\u00ae","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Group launches Efmody in UK to Treat Patients with Rare Condition Congenital Adrenal Hyperplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"company":"Cycle Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cycle Pharmaceuticals Launches SAJAZIRTM (icatibant) Injection, A New Treatment Option in Hereditary Angioedema (HAE)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Cycle Pharmaceuticals"},{"company":"Orchard Therapeutics","sponsor":"NHS England","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Historic Agreement Making Libmeldy Available by NHS England for MLD Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"company":"Orchard Therapeutics","sponsor":"Italian Medicines Agency","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Reimbursement Agreement Making Libmeldy Available for All Eligible MLD Patients in Italy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"company":"Sharp","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alnylam Pharmaceuticals Partners with Sharp on Packaging RNA Interference (RNAi) Therapeutics for Europe","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sharp"},{"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces Multiple Presentations at 2022 SSIEM Annual Symposium Highlighting Neurometabolic Disease Portfolio","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"}]
Details:
After obtaining the first-ever European Marketing Authorisation for an RNAi therapeutic in 2018, in March 2020 Alnylam received European approval for its second RNAi product, Onpattro (Patisiran).
Lead Product(s):
Patisiran
Therapeutic Area: Genetic Disease
Product Name: Onpattro
Highest Development Status: Approved
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Alnylam Pharmaceuticals
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Partnership
October 01, 2020
Details:
Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children.
Lead Product(s):
Atidarsagene Autotemcel
Therapeutic Area: Genetic Disease
Product Name: Libmeldy
Highest Development Status: Approved
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 29, 2022
Details:
Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA.
Lead Product(s):
Atidarsagene Autotemcel
Therapeutic Area: Genetic Disease
Product Name: Libmeldy
Highest Development Status: Approved
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Italian Medicines Agency
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Agreement
April 11, 2022
Details:
The agreement coincides with publication of the positive National Institute for Health and Care Excellence’s evaluation determination, which recognized the clinical impact and economic benefit of Libmeldy according to the institute’s Highly Specialized Technologies process.
Lead Product(s):
Atidarsagene Autotemcel
Therapeutic Area: Genetic Disease
Product Name: Libmeldy
Highest Development Status: Approved
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
NHS England
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Agreement
February 04, 2022
Details:
SAJAZIR™ is a bradykinin B2 receptor antagonist indicated for treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older.
Lead Product(s):
Icatibant Acetate
Therapeutic Area: Genetic Disease
Product Name: Sajazir
Highest Development Status: Approved
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 28, 2021
Details:
Efmody® is a preparation of hydrocortisone that has been specifically designed for patients with CAH, a rare condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase.
Lead Product(s):
Hydrocortisone
Therapeutic Area: Genetic Disease
Product Name: Efmody
Highest Development Status: Approved
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 13, 2021
Details:
The $1.25 million milestone payment has been triggered as a result of the recent European Commission’s approval for Efmody® (hydrocortisone modified-release hard capsules) and the attainment by Citrine of certain development milestones in China for Alkindi.
Lead Product(s):
Hydrocortisone
Therapeutic Area: Genetic Disease
Product Name: Efmody
Highest Development Status: Approved
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Citrine Medicine
Deal Size: $2.2 million
Upfront Cash: $1.0 million
Deal Type: Partnership
June 01, 2021
Details:
Efmody® is a preparation of hydrocortisone that has been specifically designed for patients with CAH, an orphan condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase.
Lead Product(s):
Hydrocortisone
Therapeutic Area: Genetic Disease
Product Name: Efmody
Highest Development Status: Approved
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
May 28, 2021
Details:
Funds will support Orchard's growing commercial capabilities, including the launch of Libmeldy in Europe, advance its regulatory and clinical development activities and expand its hematopoietic stem cell gene therapy approach into larger indications.
Lead Product(s):
Autologous CD34+ cell enriched HSPC
Therapeutic Area: Genetic Disease
Product Name: Libmeldy
Highest Development Status: Approved
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
RA Capital Management
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Public Offering
February 05, 2021
Details:
Multiple abstracts highlighting clinical and real-world data for OTL-200 and Metachromatic Leukodystrophy (MLD) will be presented at the conference.
Lead Product(s):
Libmeldy
Therapeutic Area: Genetic Disease
Product Name: OTL-200
Highest Development Status: Approved
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
January 28, 2021
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