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Hot Stocks
uniQure reports on progress made in developing AMT-260 » 11:02
11/29/22
11/29
11:02
11/29/22
11:02
QURE

uniQure

$27.42 /

+0.105 (+0.38%)

uniQure hosted a virtual…

uniQure hosted a virtual investor event focused on AMT-260, an AAV gene therapy for refractory temporal lobe epilepsy, or rTLE, and other focal epilepsies in an event that featured a presentation from rTLE clinical expert Ellen Bubrick, MD, associate chair of Neurology at Harvard Medical School and director of the Epilepsy Surgery Program at Brigham and Women's Hospital in Boston, MA. "The presentation highlighted preclinical data that supports the safety and tolerability of AMT-260 and plans for its clinical development, as well as uniQure's miQURE and linQURE technology platforms that allow the Company to use miRNAs to safely reduce the expression of genes in the brain. The company also highlighted its progress in developing a commercial-scale AAV manufacturing platform," the company stated. "One week after the landmark FDA approval of the uniQure-developed world's first gene therapy for hemophilia B, we are pleased to share the progress that we have made in developing AMT-260, a first-in-class gene therapy for temporal lobe epilepsy. Today's event presented the preclinical data and the innovative technologies that we hope will allow us to develop AMT-260 and other gene therapies that can locally regulate neuronal activity for diseases of the central nervous system. We look forward to continuing to work toward submitting an investigational new drug application for AMT-260 and advancing it into the clinic in 2023," stated Ricardo Dolmetsch, Ph.D., president of research & development at uniQure.

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QURE uniQure
$27.42 /

+0.105 (+0.38%)

QURE uniQure
$27.42 /

+0.105 (+0.38%)

11/23/22 Piper Sandler
FDA 'tea leaves' bode well for BioMarin's Roctavian, says Piper Sandler
11/23/22 Truist
uniQure price target raised to $65 from $51 at Truist
08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
QURE uniQure
$27.42 /

+0.105 (+0.38%)

QURE uniQure
$27.42 /

+0.105 (+0.38%)

Conference/Events
uniQure to hold a virtual event » 08:25
11/29/22
11/29
08:25
11/29/22
08:25
QURE

uniQure

$27.32 /

+0.42 (+1.56%)

Virtual Research &…

Virtual Research & Development Event to be held on November 29 at 8:30 am. Webcast Link

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QURE uniQure
$27.32 /

+0.42 (+1.56%)

QURE uniQure
$27.32 /

+0.42 (+1.56%)

11/23/22 Piper Sandler
FDA 'tea leaves' bode well for BioMarin's Roctavian, says Piper Sandler
11/23/22 Truist
uniQure price target raised to $65 from $51 at Truist
08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
QURE uniQure
$27.32 /

+0.42 (+1.56%)

QURE uniQure
$27.32 /

+0.42 (+1.56%)

Conference/Events
uniQure to hold a virtual event » 04:55
11/29/22
11/29
04:55
11/29/22
04:55
QURE

uniQure

$27.32 /

+0.42 (+1.56%)

Virtual Research &…

Virtual Research & Development Event to be held on November 29 at 8:30 am. Webcast Link

ShowHide Related Items >><<
QURE uniQure
$27.32 /

+0.42 (+1.56%)

QURE uniQure
$27.32 /

+0.42 (+1.56%)

11/23/22 Piper Sandler
FDA 'tea leaves' bode well for BioMarin's Roctavian, says Piper Sandler
11/23/22 Truist
uniQure price target raised to $65 from $51 at Truist
08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
QURE uniQure
$27.32 /

+0.42 (+1.56%)

QURE uniQure
$27.32 /

+0.42 (+1.56%)

Monday
Conference/Events
uniQure to hold a virtual event » 22:49
11/28/22
11/28
22:49
11/28/22
22:49
QURE

uniQure

$27.32 /

+0.42 (+1.56%)

Virtual Research &…

Virtual Research & Development Event to be held on November 29 at 8:30 am. Webcast Link

ShowHide Related Items >><<
QURE uniQure
$27.32 /

+0.42 (+1.56%)

QURE uniQure
$27.32 /

+0.42 (+1.56%)

11/23/22 Piper Sandler
FDA 'tea leaves' bode well for BioMarin's Roctavian, says Piper Sandler
11/23/22 Truist
uniQure price target raised to $65 from $51 at Truist
08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
QURE uniQure
$27.32 /

+0.42 (+1.56%)

QURE uniQure
$27.32 /

+0.42 (+1.56%)

Over a week ago
On The Fly
Biotech Alert: Searches spiking for these stocks today » 10:29
11/25/22
11/25
10:29
11/25/22
10:29
XOMA

Xoma

$25.00 /

+0.12 (+0.48%)

, VBLT

VBL Therapeutics

/

+

, BMRN

BioMarin

$97.09 /

-0.76 (-0.78%)

, QURE

uniQure

$27.40 /

+1.125 (+4.28%)

, MRTX

Mirati Therapeutics

$98.06 /

+2.24 (+2.34%)

, CSLLY

CSL

$100.10 /

+0.15 (+0.15%)

, OBSV

ObsEva

/

+

These names in the…

ShowHide Related Items >><<
XOMA Xoma
$25.00 /

+0.12 (+0.48%)

VBLT VBL Therapeutics
/

+

QURE uniQure
$27.40 /

+1.125 (+4.28%)

OBSV ObsEva
/

+

MRTX Mirati Therapeutics
$98.06 /

+2.24 (+2.34%)

CSLLY CSL
$100.10 /

+0.15 (+0.15%)

BMRN BioMarin
$97.09 /

-0.76 (-0.78%)

XOMA Xoma
$25.00 /

+0.12 (+0.48%)

VBLT VBL Therapeutics
/

+

07/20/22 Chardan
VBL Therapeutics downgraded to Neutral from Buy at Chardan
07/20/22 H.C. Wainwright
VBL Therapeutics downgraded to Neutral from Buy at H.C. Wainwright
07/20/22 Oppenheimer
VBL Therapeutics downgraded to Perform from Outperform at Oppenheimer
07/18/22 Oppenheimer
VBL Therapeutics assumed with an Outperform at Oppenheimer
BMRN BioMarin
$97.09 /

-0.76 (-0.78%)

11/25/22 Truist
FDA decision on BioMarin's Roctavian extremely positive, says Truist
11/23/22 Piper Sandler
FDA 'tea leaves' bode well for BioMarin's Roctavian, says Piper Sandler
11/23/22 Morgan Stanley
Approval of CSL's Hemgenix positive for BioMarin, says Morgan Stanley
11/14/22 Morgan Stanley
BioMarin Voxzogo concerns lowered by Ascendis data, says Morgan Stanley
QURE uniQure
$27.40 /

+1.125 (+4.28%)

11/23/22 Truist
uniQure price target raised to $65 from $51 at Truist
08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
MRTX Mirati Therapeutics
$98.06 /

+2.24 (+2.34%)

11/22/22 BMO Capital
BMO says Mirati could be valued at up to $200 per share in 'upside scenario'
11/16/22 Citi
Mirati Therapeutics price target lowered to $121 from $127 at Citi
10/28/22 B. Riley
Mirati Therapeutics initiated with a Neutral at B. Riley
10/20/22 Oppenheimer
Revolution Medicines initiated with an Outperform at Oppenheimer
CSLLY CSL
$100.10 /

+0.15 (+0.15%)

11/10/22 Credit Suisse
CSL upgraded to Outperform from Neutral at Credit Suisse
11/03/22 Citi
Arcturus Therapeutics upgraded to Buy at Citi after CSL deal
11/02/22 Barclays
Arcturus Therapeutics upgraded to Equal Weight from Underweight at Barclays
OBSV ObsEva
/

+

08/30/22 H.C. Wainwright
ObsEva downgraded to Neutral from Buy at H.C. Wainwright
07/29/22 Canaccord
ObsEva cut to Hold at Canaccord as company goes 'back to square one'
07/29/22 Canaccord
ObsEva downgraded to Hold from Buy at Canaccord
07/28/22 Cantor Fitzgerald
ObsEva downgraded to Neutral from Overweight at Cantor Fitzgerald
XOMA Xoma
$25.00 /

+0.12 (+0.48%)

VBLT VBL Therapeutics
/

+

QURE uniQure
$27.40 /

+1.125 (+4.28%)

MRTX Mirati Therapeutics
$98.06 /

+2.24 (+2.34%)

BMRN BioMarin
$97.09 /

-0.76 (-0.78%)

MRTX Mirati Therapeutics
$98.06 /

+2.24 (+2.34%)

CSLLY CSL
$100.10 /

+0.15 (+0.15%)

VBLT VBL Therapeutics
/

+

QURE uniQure
$27.40 /

+1.125 (+4.28%)

OBSV ObsEva
/

+

MRTX Mirati Therapeutics
$98.06 /

+2.24 (+2.34%)

CSLLY CSL
$100.10 /

+0.15 (+0.15%)

BMRN BioMarin
$97.09 /

-0.76 (-0.78%)

VBLT VBL Therapeutics
/

+

Recommendations
FDA 'tea leaves' bode well for BioMarin's Roctavian, says Piper Sandler » 10:32
11/23/22
11/23
10:32
11/23/22
10:32
BMRN

BioMarin

$98.35 /

+7.17 (+7.86%)

, QURE

uniQure

$24.22 /

+1.23 (+5.35%)

Piper Sandler analyst…

Piper Sandler analyst Christopher Raymond cautions that "reading FDA tea leaves can be a dangerous game," but with that said he thinks it is "reasonable to connect a couple of recent data points from FDA" that he thinks bode well for Roctavian's U.S. approval process, including yesterday's approval of uniQure's (QURE) hemophilia B gene therapy Hemgenix and today's announcement that the FDA no longer intends to conduct an AdCom to review BioMarin's (BMRN) Roctavian hemophilia A application. The analyst, who reminds investors that Roctavian's EU launch is currently underway and that his recent hematologist survey makes it appear there is "some degree of pent-up demand for the therapy," has an Overweight rating and $128 price target on BioMarin shares.

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QURE uniQure
$24.22 /

+1.23 (+5.35%)

BMRN BioMarin
$98.35 /

+7.17 (+7.86%)

BMRN BioMarin
$98.35 /

+7.17 (+7.86%)

11/23/22 Morgan Stanley
Approval of CSL's Hemgenix positive for BioMarin, says Morgan Stanley
11/14/22 Morgan Stanley
BioMarin Voxzogo concerns lowered by Ascendis data, says Morgan Stanley
11/14/22 Piper Sandler
BioMarin should rally 'meaningfully' after Ascendis data, says Piper Sandler
10/31/22 Oppenheimer
BioMarin upgraded to Outperform from Perform at Oppenheimer
QURE uniQure
$24.22 /

+1.23 (+5.35%)

11/23/22 Truist
uniQure price target raised to $65 from $51 at Truist
08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
08/08/22 Chardan
uniQure price target lowered to $60 from $65 at Chardan
QURE uniQure
$24.22 /

+1.23 (+5.35%)

BMRN BioMarin
$98.35 /

+7.17 (+7.86%)

QURE uniQure
$24.22 /

+1.23 (+5.35%)

BMRN BioMarin
$98.35 /

+7.17 (+7.86%)

Recommendations
uniQure price target raised to $65 from $51 at Truist » 08:12
11/23/22
11/23
08:12
11/23/22
08:12
QURE

uniQure

$23.03 /

+1.52 (+7.07%)

Truist analyst Robyn…

Truist analyst Robyn Karnauskas raised the firm's price target on uniQure to $65 from $51 and keeps a Buy rating on the shares. The analyst cites the company's announcement yesterday regarding the FDA approval of Hemgenix with a "clean label". Karnauskas adds that she is updating her model to reflect the $3.5M price in U.S., modeling a peak $800M in royalties vs. $600M previously.

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QURE uniQure
$23.03 /

+1.52 (+7.07%)

QURE uniQure
$23.03 /

+1.52 (+7.07%)

08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
08/08/22 Chardan
uniQure price target lowered to $60 from $65 at Chardan
08/08/22 Truist
uniQure's high-dose HD data still on track for 1H23, says Truist
QURE uniQure
$23.03 /

+1.52 (+7.07%)

QURE uniQure
$23.03 /

+1.52 (+7.07%)

Hot Stocks
uniQure announces FDA approval of gene therapy for adults with hemophilia B » 16:41
11/22/22
11/22
16:41
11/22/22
16:41
QURE

uniQure

$23.03 /

+1.52 (+7.07%)

uniQure announced that…

uniQure announced that its partner CSL has received approval from the U.S. Food and Drug Administration for HEMGENIX, a one-time gene therapy for the treatment of adults 18 years of age and older living with hemophilia B. The product is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. CSL licensed the exclusive global rights to HEMGENIX from uniQure in May 2021 and is now solely responsible for the further development, registration, and commercialization of the therapy. "Today's landmark approval represents a major milestone in the field of genomic medicine and ushers in a new treatment paradigm for patients living with hemophilia B," said Matt Kapusta, chief executive officer of uniQure. "This is uniQure's second internally-developed gene therapy to achieve approval and the world's first gene therapy for hemophilia B, an historic achievement based on more than a decade of research and clinical development, as well as the tireless dedication of our employees, clinicians, patients and their families. For nearly 25 years, uniQure has been driven by a singular mission: to transform peoples' lives by harnessing the power of gene therapy. This groundbreaking approval delivers on this promise by providing people with hemophilia B the possibility of being liberated from burdensome infusions and an ability to unlock the potential in their daily lives. We offer our sincere gratitude to the hemophilia community, without whose years of dedication and invaluable support this medical breakthrough could not have been achieved."

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QURE uniQure
$23.03 /

+1.52 (+7.07%)

QURE uniQure
$23.03 /

+1.52 (+7.07%)

08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
08/08/22 Chardan
uniQure price target lowered to $60 from $65 at Chardan
08/08/22 Truist
uniQure's high-dose HD data still on track for 1H23, says Truist
QURE uniQure
$23.03 /

+1.52 (+7.07%)

QURE uniQure
$23.03 /

+1.52 (+7.07%)

Over a month ago
Earnings
uniQure reports Q3 EPS ($1.02), consensus ($1.16) » 07:15
11/02/22
11/02
07:15
11/02/22
07:15
QURE

uniQure

$18.83 /

+0.21 (+1.13%)

Reports Q1 revenue $1.4M,…

Reports Q1 revenue $1.4M, consensus $3.48M. "We are pleased that following a comprehensive review of all available safety, biomarker and imaging data, the Data Safety Monitoring Board has recommended that we resume patient enrollment at the higher dose in the ongoing Phase Ib/II study evaluating AMT-130 in Huntington's disease," stated Matt Kapusta, chief executive officer of uniQure. "Patient safety will always be our utmost priority, and we are grateful for the close collaboration between our clinical team, study investigators and most importantly, the patients, all of whom were instrumental in completing our comprehensive investigations. We believe that AMT-130 has the potential to provide a positive impact for patients with this devastating disease for which there is no currently approved treatment. We look forward to finishing patient enrollment in the higher-dose cohort of the European study in the first half of 2023 and remain on track to announce data from the U.S. Phase I/II study in the second quarter of 2023. In the third quarter, the U.S. Food and Drug Administration and the European Medicines Agency completed site inspections of our Lexington, Massachusetts gene therapy manufacturing facility, and we have since received good manufacturing practice certification from the European authorities. This is an important milestone as we seek to establish industry-leading commercial gene therapy manufacturing capabilities. Our partner, CSL Behring, continues to interact with U.S. and European health authorities as they complete their review of the marketing applications for etranacogene dezaparvovec and, assuming approval, we look forward to partnering with our colleagues at CSL Behring to bring this potentially transformational treatment option to people living with hemophilia B."

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QURE uniQure
$18.83 /

+0.21 (+1.13%)

QURE uniQure
$18.83 /

+0.21 (+1.13%)

08/09/22 Wells Fargo
uniQure price target lowered to $24 from $34 at Wells Fargo
08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
08/08/22 Chardan
uniQure price target lowered to $60 from $65 at Chardan
08/08/22 Truist
uniQure's high-dose HD data still on track for 1H23, says Truist
QURE uniQure
$18.83 /

+0.21 (+1.13%)

QURE uniQure
$18.83 /

+0.21 (+1.13%)

Over a quarter ago
Recommendations
uniQure price target lowered to $24 from $34 at Wells Fargo » 06:53
08/09/22
08/09
06:53
08/09/22
06:53
QURE

uniQure

$18.60 /

-6.92 (-27.12%)

Wells Fargo analyst Jim…

Wells Fargo analyst Jim Birchenough lowered the firm's price target on uniQure to $24 from $34 following the clinical update to the AMT-130 program. The analyst keeps an Equal Weight rating on the shares.

ShowHide Related Items >><<
QURE uniQure
$18.60 /

-6.92 (-27.12%)

QURE uniQure
$18.60 /

-6.92 (-27.12%)

08/09/22 H.C. Wainwright
uniQure selloff brings attractive entry point, says H.C. Wainwright
08/08/22 Chardan
uniQure price target lowered to $60 from $65 at Chardan
08/08/22 Truist
uniQure's high-dose HD data still on track for 1H23, says Truist
05/05/22 Stifel
uniQure price target lowered to $37 from $66 at Stifel
QURE uniQure
$18.60 /

-6.92 (-27.12%)

QURE uniQure
$18.60 /

-6.92 (-27.12%)

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