Drugs in Dev. Genetic Disease
Phase I
Israel 
Lead Product(s) : MNV-201
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Minovia Gets FDA Nod for Phase II Trial of MNV-201 in Pearson Syndrome
Details : MNV-201, an autologous hematopoietic stem cell product augmented with allogeneic mitochondria. It is being evaluated for the treatment of pearson syndrome.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
April 03, 2025
Lead Product(s) : MNV-201
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Motixafortide
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : St. Jude Children’s Research Hospital
Deal Size : Undisclosed
Deal Type : Agreement
BioLineRx and St. Jude Evaluate Motixafortide for CD34+ HSC Mobilization
Details : The agreement aims to evaluate motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with sickle cell disease (SCD).
Product Name : Aphexda
Product Type : Peptide, Unconjugated
Upfront Cash : Undisclosed
May 30, 2024
Lead Product(s) : Motixafortide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : St. Jude Children’s Research Hospital
Deal Size : Undisclosed
Deal Type : Agreement
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Cystic Fibrosis Foundation
Deal Size : $8.5 million
Deal Type : Funding
CF Foundation Provides Up to $8.5M to SpliSense for Splicing Mutation Treatment Trial
Details : The funding will support SpliSense to continue the clinical development of an inhaled antisense oligonucleotide drug SPL84 for treating patients suffering from cystic fibrosis.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
April 03, 2024
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Cystic Fibrosis Foundation
Deal Size : $8.5 million
Deal Type : Funding
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
SpliSense Announces FDA Clearance for SPL84 Phase 2 Initiation for Cystic Fibrosis
Details : SPL84 is an antisense oligonucleotide product that binds specifically to the mutated CFTR RNA in the targeted sequence. It is being evaluated for the treatment of cystic fibrosis.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
April 03, 2024
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Safety, Tolerability, and Pharmacokinetics of SPL84 in Healthy Volunteers
Details : SPL84 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Cystic Fibrosis.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
January 23, 2024
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Motixafortide,Natalizumab
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : BioLineRx initiated Phase I clinical trial studies evaluating BL-8040 (motixafortide), CXCR4/SDF-1 inhibitor, as monotherapy and in combination with natalizumab for the treatment of sickle cell disease.
Product Name : Aphexda
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
December 21, 2023
Lead Product(s) : Motixafortide,Natalizumab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : SPL84, the Company's lead inhaled anti sense oligonucleotide (ASO) product for the treatment of patients with CF, carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
September 06, 2023
Lead Product(s) : SPL84
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Motixafortide,Natalizumab
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Washington University School of Medicine
Deal Size : Undisclosed
Deal Type : Collaboration
Details : The aim of the collaboration is to advance a Phase 1 clinical trial that will evaluate the safety and feasibility of BL-8040 (motixafortide) to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in sickle cell disease (SCD).
Product Name : Aphexda
Product Type : Peptide, Unconjugated
Upfront Cash : Undisclosed
March 06, 2023
Lead Product(s) : Motixafortide,Natalizumab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Washington University School of Medicine
Deal Size : Undisclosed
Deal Type : Collaboration
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