Drugs in Dev. Genetic Disease
Phase III
Netherlands 
Lead Product(s) : Sonlicromanol
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Netherlands Enterprise Agency
Deal Size : $5.6 million
Deal Type : Funding
Khondrion gets $5M Credit Plus more Funds for Mitochondrial Trial
Details : The funding aims to advance the clinical development of Khondrion lead product product KH176 (sonlicromanol), which is being evaluated for the treatment of Primary mitochondrial Disease.
Product Name : KH176
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
May 06, 2025
Lead Product(s) : Sonlicromanol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Netherlands Enterprise Agency
Deal Size : $5.6 million
Deal Type : Funding
Lead Product(s) : Sonlicromanol
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : UMDF
Deal Size : Undisclosed
Deal Type : Funding
UMDF Supports Khondrion Drug Development for Primary Mitochondrial Disease
Details : The proceeds will be used for the development of KH176 (sonlicromanol), a first-in-class, brain-penetrant redox-modulator, for adults with primary mitochondrial disease due to the m.3243A>G mutation.
Product Name : KH176
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
November 21, 2024
Lead Product(s) : Sonlicromanol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : UMDF
Deal Size : Undisclosed
Deal Type : Funding
Lead Product(s) : Sonlicromanol
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Khondrion Gets FDA Clearance for Sonlicromanol Phase 3 in Mitochondrial Disease
Details : KH176 (sonlicromanol) is a mPGES-1 inhibitor small molecule drug candidate, which is being evaluated for the treatment of adult patients with PMD due to the m.3243A>G mutation.
Product Name : KH176
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 14, 2024
Lead Product(s) : Sonlicromanol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : CSL Behring
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable FIX expression, assessed at 18 months following a single dose of AMT-061(etranacogene dezaparvovec).
Product Name : Hemgenix
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
September 12, 2021
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : CSL Behring
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : CSL Behring
Deal Size : $2,050.0 million
Deal Type : Licensing Agreement
UniQure Announces Closing of Commercialization and License Agreement With CSL Behring
Details : uniQure announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec expired on May 5, 2021, and the agreement became fully effective on...
Product Name : Hemgenix
Product Type : Cell and Gene therapy
Upfront Cash : $450.0 million
June 05, 2021
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : CSL Behring
Deal Size : $2,050.0 million
Deal Type : Licensing Agreement
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
Details : UniQure's comprehensive investigation showed that AMT-061 (etranacogene dezaparvovec) is very unlikely to have contributed to the HCC in our patient. The company is looking forward to announcing top-line 52-week data from the HOPE-B pivotal trial later t...
Product Name : Hemgenix
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
April 26, 2021
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The number of bleeds requiring treatment decreased by 91 percent after the one-time administration of etranacogene dezaparvovec, with 87 percent of patients reporting no such bleeds after dosing.
Product Name : Hemgenix
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
August 12, 2020
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : CSL Behring
Deal Size : Undisclosed
Deal Type : Licensing Agreement
Details : The AMT-061 program, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.
Product Name : Hemgenix
Product Type : Cell and Gene therapy
Upfront Cash : $450.0 million
June 24, 2020
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : CSL Behring
Deal Size : Undisclosed
Deal Type : Licensing Agreement
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Company to present long-term follow-up data from its hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical...
Product Name : Hemgenix
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
April 11, 2020
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The targeted number of patients to be dosed per the clinical trial protocol was 50. In total, 54 patients have received the one-time dose of etranacogene dezaparvovec.
Product Name : Hemgenix
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
March 26, 2020
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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