Top 5 Target	Count

COL7A1(Collagen alpha-1(VII) chain)	1
COL3A1 x elastin	1
IL-12(Interleukin-12)	1
CFTR(Cystic fibrosis transmembrane conductance regulator)	1
COL1A1(collagen type I alpha 1 chain)	1

Drugs associated with Krystal Biotech, Inc.

Beremagene geperpavec

Target

COL7A1

Mechanism

COL7A1 gene transference

Active Org.

Krystal Biotech, Inc.

Originator Org.

Krystal Biotech, Inc.

Active Indication

Epidermolysis Bullosa Dystrophica

Inactive Indication-

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

First Approval Date19 May 2023

KB-105

Target

TGM1

Mechanism

TGM1 replacements [+1]

Active Org.

Krystal Biotech, Inc.

Originator Org.

Krystal Biotech, Inc.

Active Indication

Autosomal recessive ichthyosis

Inactive Indication-

Drug Highest PhasePhase 2

First Approval Ctry. / Loc.-

First Approval Date-

KB-407

Target

CFTR

Mechanism

CFTR gene transference

Active Org.

Krystal Biotech, Inc.

Originator Org.

Krystal Biotech, Inc.

Active Indication

Cystic Fibrosis

Inactive Indication-

Drug Highest PhasePhase 1

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with Krystal Biotech, Inc.

NCT06563414 / RecruitingNot Applicable

A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)

This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Corneal abrasion symptomology, frequency, and outcomes will be evaluated for up to 6 months.

Start Date02 Aug 2024

Sponsor / Collaborator

Krystal Biotech, Inc.

NCT06228326 / RecruitingPhase 1

KB707-02: a Phase 1 Study of Inhaled KB707 in Patients with Advanced Solid Tumor Malignancies Affecting the Lungs

The Sponsor is developing KB707, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector that is designed to stimulate an anti-tumor immune response through the production of cytokines delivered to the airways of people with advanced solid tumor malignancies affecting the lungs via nebulization. This Phase 1, open-label, multicenter, dose escalation and expansion study is designed to evaluate the safety and tolerability of KB707 in adults with with advanced solid tumor malignancies affecting the lungs who have progressed on standard of care therapy, cannot tolerate standard of care therapy, or refused standard of care therapy. The study will include a dose escalation portion for single agent KB707 using a standard 3+3 design followed by an expansion portion to further evaluate single agent KB707 at a dose determined by preliminary data in the dose escalation phase. Subjects in both the dose escalation and dose expansion cohorts will receive KB707 via nebulization weekly for three weeks, then every three weeks for up to two years until tumor progression, death, unacceptable toxicity, symptomatic deterioration, achievement of maximal response, subject choice, Investigator decision to discontinue treatment, or the Sponsor determines to terminate the study.

Start Date17 Apr 2024

Sponsor / Collaborator

Krystal Biotech, Inc.

NCT06049082 / RecruitingPhase 1

A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI*ZZ or PI*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. At the high dose, two cohorts will be conducted in parallel to evaluate patients on and off IV augmentation therapy.

Start Date15 Feb 2024

Sponsor / Collaborator

Krystal Biotech, Inc.

100 Clinical Results associated with Krystal Biotech, Inc.

0 Patents (Medical) associated with Krystal Biotech, Inc.

Literatures (Medical) associated with Krystal Biotech, Inc.

01 Apr 2021·Journal of Investigative DermatologyQ1 · MEDICINE

Preclinical Evaluation of a Modified Herpes Simplex Virus Type 1 Vector Encoding Human TGM1 for the Treatment of Autosomal Recessive Congenital Ichthyosis

Q1 · MEDICINE

Article

Author: Krishnan, Suma ; Yogesha, S D ; Freedman, John C ; Parry, Trevor J ; Zhang, Peipei ; Majumdar, Avijit ; Ramasamy, Sureshkumar ; O'Malley, Mark ; Agarwal, Pooja ; Regula, Lauren K ; Coghlan, Sarah

Autosomal recessive congenital ichthyosis (ARCI) is a diverse group of cornification diseases associated with severe clinical complications and decreased quality of life. Germline mutations in the TGM1 gene, which encodes the enzyme TGM1, are the predominant cause of ARCI. These TGM1 mutations trigger the abnormal epidermal differentiation and impaired cutaneous barrier function observed in patients with ARCI. Unfortunately, current ARCI therapies focus solely on symptomatic relief. Thus, there is a significant unmet need for therapeutic strategies aimed at correcting the TGM1 deficiency underlying ARCI. In this study, we investigated the ability of KB105, a gene therapy vector encoding full-length human TGM1, to deliver functional human TGM1 to keratinocytes. In vitro, KB105 efficiently infected TGM1-deficient human keratinocytes, produced TGM1 protein, and rescued transglutaminase enzyme function. In vivo studies demonstrated that both single and repeated topical KB105 administration induced TGM1 protein expression in the target epidermal layer without triggering fibrosis, necrosis, or acute inflammation. Toxicity and biodistribution assessments on repeat dosing indicated that KB105 was well-tolerated and restricted to the dose site. Overall, our results demonstrate that rescuing TGM1 deficiency in patients with ARCI through topical KB105 application represents a promising strategy for safely and noninvasively treating this debilitating disease.

01 Jul 2019·Journal of Investigative DermatologyQ1 · MEDICINE

Thrombospondin-1 Is a Major Activator of TGF-β Signaling in Recessive Dystrophic Epidermolysis Bullosa Fibroblasts

Q1 · MEDICINE

Article

Author: Webster, Timothy G ; Russell, Rebecca J ; Fuentes, Ignacia ; Lim, Yok Zuan ; South, Andrew P ; Guttmann-Gruber, Christina ; Krishnan, Suma ; Cao, Qingqing ; Salas-Alanis, Julio C ; McGrath, John A ; Fertala, Andrzej ; Atanasova, Velina S ; Agarwal, Pooja

Mutations in the gene encoding collagen VII cause the devastating blistering disease recessive dystrophic epidermolysis bullosa (RDEB). RDEB is characterized by severe skin fragility and nonhealing wounds aggravated by scarring and fibrosis. We previously showed that TSP1 is increased in RDEB fibroblasts. Because transforming growth factor-β (TGF-β) signaling is also increased in RDEB, and TSP1 is known to activate TGF-β, we investigated the role of TSP1 in TGF-β signaling in RDEB patient cells. Knockdown of TSP1 reduced phosphorylation of smad3 (a downstream target of TGF-β signaling) in RDEB primary fibroblasts, whereas overexpression of collagen VII reduced phosphorylation of smad3. Furthermore, inhibition of TSP1 binding to the LAP/TGF-β complex decreased fibrosis in engineered extracellular matrix formed by RDEB fibroblasts, as evaluated by picrosirius red staining and analyses of birefringent collagen fibrillar deposits. We show that collagen VII binds TSP1, which could potentially limit TSP1-LAP association and subsequent TGF-β activation. Our study suggests a previously unreported mechanism for increased TGF-β signaling in the absence of collagen VII in RDEB patient skin. Moreover, these data identify TSP1 as a possible target for reducing fibrosis in the tumor-promoting dermal microenvironment of RDEB patients.

PLOS Neglected Tropical Diseases

Transmission of viable Haemophilus ducreyi by Musca domestica

Article

Author: Donlan, Rachel A ; McCandless, Kayla ; Humphreys, Tricia L ; Gaston, Jordan R ; Stabile, Haley D

Haemophilus ducreyi was historically known as the causative agent of chancroid, a sexually-transmitted disease causing painful genital ulcers endemic in many low/middle-income nations. In recent years the species has been implicated as the causative agent of nongenital cutaneous ulcers affecting children of the South Pacific Islands and West African countries. Much is still unknown about the mechanism of H. ducreyi transmission in these areas, and recent studies have identified local insect species, namely flies, as potential transmission vectors. H. ducreyi DNA has been detected on the surface and in homogenates of fly species sampled from Lihir Island, Papua New Guinea. The current study develops a model system using Musca domestica, the common house fly, as a model organism to demonstrate proof of concept that flies are a potential vector for the transmission of viable H. ducreyi. Utilizing a green fluorescent protein (GFP)-tagged strain of H. ducreyi and three separate exposure methods, we detected the transmission of viable H. ducreyi by 86.11% ± 22.53% of flies sampled. Additionally, the duration of H. ducreyi viability was found to be directly related to the bacterial concentration, and transmission of H. ducreyi was largely undetectable within one hour of initial exposure. Push testing, Gram staining, and PCR were used to confirm the identity and presence of GFP colonies as H. ducreyi. This study confirms that flies are capable of mechanically transmitting viable H. ducreyi, illuminating the importance of investigating insects as vectors of cutaneous ulcerative diseases.

107

News (Medical) associated with Krystal Biotech, Inc.

13 Sep 2024

·www.globenewswire.com

Krystal Biotech to Present at 2024 Cantor Global Healthcare Conference

PITTSBURGH, Sept. 13, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the Company will participate in the 2024 Cantor Global Healthcare Conference on September 18, 2024, in New York. Krish S. Krishnan, Chairman and Chief Executive Officer, will take part in a fireside chat scheduled at 1:20 pm ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 1:20 pm ET on Wednesday, September 18, 2024 and will be posted on the Investors section of the Company’s website. About Krystal Biotech, Inc.Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). CONTACTInvestors and Media:Stéphane PaquetteKrystal Biotechspaquette@krystalbio.com

Drug Approval

06 Sep 2024

·www.globenewswire.com

Krystal Biotech to Present at H.C. Wainwright 26th Annual Global Investment Conference

PITTSBURGH, Sept. 06, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the Company will participate in the H.C. Wainwright 26th Annual Global Investment Conference on September 10, 2024, in New York. Krish S. Krishnan, Chairman and Chief Executive Officer, is scheduled to give a company presentation at 12:30 pm ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 12:30 pm ET on Tuesday, September 10, 2024 and will be posted on the Investors section of the Company’s website. About Krystal Biotech, Inc. Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). CONTACTInvestors and Media:Stéphane PaquetteKrystal Biotechspaquette@krystalbio.com

Drug ApprovalGene Therapy

30 Aug 2024

·endpts.com

Ipsen sells priority review voucher for highest price since 2016

Ipsen Pharmaceuticals sold its priority review voucher to an unidentified “large global pharmaceutical company” for $158 million, it said this week, the highest publicly announced price for one of the vouchers in about eight years. Ipsen obtained the rare pediatric disease voucher in August 2023 thanks to the approval of Sohonos (palovarotene), which is intended to help adults and children with the rare disease known as fibrodysplasia ossificans progressiva. Priority review vouchers, or PRVs, can be acquired through the approval of rare pediatric disease or rare tropical disease drugs, and then can be sold or used by companies to speed the FDA’s reviews of any new drug. The $158 million sale is about $50 million more than any of the other PRV sales since last August, which includes sales of between $100 million and $108 million for Krystal Biotech, Valneva, X4 Pharmaceuticals and Day One Biopharmaceuticals. The last time a PRV sold for more than $158 million was in 2016, when PaxVax Bermuda sold its PRV, acquired via the approval of its cholera vaccine Vaxchora, for $290 million to an undisclosed buyer. PRVs have typically sold for about $100 million since the voucher programs began more than a decade ago, with higher prices of more than $150 million only coming between 2014 and 2016. “The sale of the PRV generates further firepower for our ongoing medicine launches and external-innovation strategy, which has seen multiple additions to our portfolio at every stage of development in 2024,” Philippe Lopes-Fernandes, chief business officer at Ipsen, said in a statement.

Priority ReviewDrug ApprovalAcquisition

100 Deals associated with Krystal Biotech, Inc.

100 Translational Medicine associated with Krystal Biotech, Inc.

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Pipeline

Pipeline Snapshot as of 24 Dec 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Preclinical

Phase 1 Clinical

Phase 2 Clinical

Approved

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

Beremagene geperpavec ( COL7A1 )	Epidermolysis Bullosa Dystrophica More	Approved
KB-105 ( TGM1 )	Autosomal recessive ichthyosis More	Phase 2
KB-408 ( A1AT )	Alpha 1-Antitrypsin Deficiency More	Phase 1
KB-407 ( CFTR )	Cystic Fibrosis More	Phase 1
KB-707 ( IL-12 x IL-2 )	Advanced Malignant Solid Neoplasm More	Phase 1

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