Hemophilia | Stock Gumshoe

April 17, 2014 / DrKSSMDPhD

In A Brutal Biotech Market, A Reason to Be Excited: Introducing Regado Biosciences

[ed note: Longtime commenter and reader Dr. KSS, a medical doctor and biotech investor, has agreed to share his thoughts with the Irregulars as one of our guest authors, educating us on health and medicine and sharing some of his biotech investing ideas. He has agreed to the same basic trading restrictions as other paid […]

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rmackintosh on Looking into Jeff Brown’s “The ‘God Key’: The Most Revolutionary Biotechnology Since Antibiotics…” / Feb 15, 8:27pm

that is a great question, and I suspect fixing bad genes in cancers will be difficult; maybe fixing bad genes in heredit...

kfoehr on Looking into Jeff Brown’s “The ‘God Key’: The Most Revolutionary Biotechnology Since Antibiotics…” / Feb 15, 3:46pm

Can't say which tech is better, but SGMO appears to be much farther ahead in research. "In 2017, Sangamo is conducting ...

danmcco on The Twelve Biotechs of Christmas 2018: Balthasar / Jan 29, 10:22am

$SNY $BIIV = crooks to pay fines... why don't they ever go to jail?Regulators at the SEC are sounding an alarm fo...

arbrnrngr on written by reader

In A Brutal Biotech Market, A Reason to Be Excited: Introducing Regado Biosciences / Dec 15, 9:33pm

$BioMarin Interesting UK study Hemophilia A success. Small study but quite the patient response....

SoGiAm on Special Comment: Update from $ARTH CEO Terrence Norchi, MD, MBA / Dec 11, 10:06am

#ASH17 NEWS http://e.endpointsnews.com/t/ViewEmail/t/16F7237A4D7B4F15/2F80028A944F69C925D77A725F39070E The BCMA-targ...

DrKSSMDPhD on Oncotutorial / Aug 23, 1:11pm

Imagine having hemophilia or von Willebrand disease and not bleeding all over creation when you scrape yourself. I HAVE ...

mtpoulsen on Long Idea of the Year / Jun 21, 8:04am

$Rubius Therapeutics Just secured $120M in funding from Flagship. Torben Straight Nissen, president at Rubies and partn...

smanuelm on Long Idea of the Year / May 16, 7:36am

$SGMO np: Receives Fast Track Designation From The FDA For SB-525 Investigational Hemophilia A Gene Therapy https://fi...

Cleveland on Long Idea of the Year / May 10, 5:04pm

$SGMO Amazing. Always been great at raising large amounts of capital and having numerous irons in the fire. I ha...

DrKSSMDPhD on The Twelve Biotechs of Christmas: One / Dec 22, 7:56am

At-heart coagulationist that I am, I'm pretty amped about Genentech/Roche's phase 3 success today with emicizumab. This ...

SoGiAm on

BIotech/Medical Catalyst Calender / Aug 26, 8:02am

$QURE rpts Q2: Add. data on AMT-060 in Hemophilia B Later this YrData fr Sanfilippo B & Parkinson's Dis. in Q1'17 h...

eyedoc on The State of Things: Ruction at Regulus / Jul 29, 6:00pm

to be more precise, in paragraph 1 "I don't see a gene therapy for Hemophilia B".......

DrKSSMDPhD on The State of Things: Ruction at Regulus / Jul 29, 5:59pm

Hi eyedoc! Thanks for doing some ferreting. In actual fact, Baxalta is recruiting now for its in-house factor IX gene th...

eyedoc on The State of Things: Ruction at Regulus / Jul 29, 4:41pm

Doc, I don't see a gene therapy for either Baxalta (which has a recombinant Factor IX replacement therapy and BAX 888 in...

AC/DC on The State of Things: Ruction at Regulus / Jul 29, 4:25pm

I think the answer is (b) to the best of my knowledge as Biomarin developing a gene therapy for Hemophilia A....

DrKSSMDPhD on The State of Things: Ruction at Regulus / Jul 29, 8:20am

Question of the Day:Which ONE of the following companies is NOT developing a gene therapy for factor IX deficiency (...

SoGiAm on The State of Things: Ruction at Regulus / Jul 28, 6:27am

$ALNY, $BMRN, $PFE, $QURE, $SHPG Gene Therapy and Other New Hemophilia Products https://twitter.com/Liquid_Biopsy_/st...

SoGiAm on Conflict Diamonds / Jul 21, 7:37am

$ONCE $PFE Spark Therapeutics and Pfizer Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment...

SoGiAm on Conflict Diamonds / Jul 11, 7:24pm

$QURE Announces Presentation of Updated Data for AMT-060 at the World Federation of Hemophilia World Congress 7/27 http...

newby3867 on Quickster: Introducing ScyNexis ($SCYX), a New Antimicrobial Play / Jun 20, 12:11am

Spark Therapeutics (ONCE) doing some good things with gene therapy treating hemophilia.https://www.technologyreview....

DrKSSMDPhD on The Bedlam in Young Goliath / May 19, 1:19pm

Lovely pop today in $ONCE, the firm treating hemophilia B with factor IX gene therapy. ONCE is attaining 30 percent norm...

DrKSSMDPhD on Special Commentary: Turbulence for Esperion / Mar 14, 11:16am

Ben: You were asking about Versartis $VSAR. In no particular order:(1) the company share price is in a perpetual dow...

newby3867 on Will February Bring a Swing Trade in Regulus ($RGLS)? / Feb 14, 12:22am

A few months old article, but a good article on the gene therapy race and competition to possibly cure Hemophilia.ht...

newby3867 on Will February Bring a Swing Trade in Regulus ($RGLS)? / Feb 13, 11:10pm

A promising looking gene therapy company Dimension Therapeutics (DMTX) looks interesting trading a bit over 6.00. It is ...

SoGiAm on The Manchurian Candida / Jan 6, 4:36pm

TLOG- TetraLogic Pharmaceuticals Announces Data on Lead Clinical Programs: http://globenewswire.com/news-release/2016/...

SoGiAm on The Manchurian Candida / Nov 29, 1:37pm

TGTX-TGTX Phase I/Ib Study Evaluating the Efficacy/Safety of TGR-1202 Est Study Completion Dec 2015 BXLT-PDUFA Adnovate...

newby3867 on The Tao of Recro / Oct 15, 11:25pm

Baxalta (BXLT) and Obizur get Health Canada approval for acquired Hemophilia A.http://finance.yahoo.com/news/baxalta...

DrKSSMDPhD on The Tao of Recro / Oct 10, 10:05am

Thanks, bird-dog Ben. The insider selling troubles me in that the stock is well off its highs. I have a long position in...

DrKSSMDPhD on Cisco, Chang & Charlie, Part Two / Jul 12, 10:01am

Don, yes, AC5 would definitely have a role in helping the hemophiliac pt. They come in several flavors: A, which confuse...

donbarrett on Cisco, Chang & Charlie, Part Two / Jul 11, 9:10pm

I have no idea of the current prevalence of Hemophilia or how crises are treated, but wonder if AC5 could be a home use ...

DrKSSMDPhD on The Last Samurai Biotech / May 15, 2:30pm

I have begun drafting a gene therapy primer for you. I think the ophthalmology community may be waking up to Molecular P...

Frenchy on The Last Samurai Biotech / May 2, 6:12pm

Well here's another M&A rumor from SA regarding PFE...-According to Reuters, Pfizer (PFE +0.2%) is negotiating a tak...

DrKSSMDPhD on The Great Gallic Foie Gras Hack / Apr 26, 12:46pm

Hi don: we have pretty vigorously discussed Spark $ONCE in prior threads. You might do Control F searching to find those...

simong156 on written by reader

Gumshoe BioDimSum II: Corralling the “A” Biotechs Plus a New Long Selection / Mar 20, 1:05pm

In a report released yesterday, Cory Kasimov from J.P. Morgan reiterated an Overweight rating on Spark Therapeutics (NAS...

DrKSSMDPhD on written by reader

The Zed Files / Mar 11, 9:59am

Larry, I don't personally think it competes with ARTH because this product's huge downside is that it makes recipients t...

DrKSSMDPhD on The Long Case for Receptos / Feb 4, 5:08am

Someone commented recently on $OPKO's recombinant factor VIIa-CTP product, soon to enter phase 2a. I wrote something abo...

adadfarm on The Long Case for Receptos / Jan 29, 4:44pm

Thank you Dr. KSS. I have re-reviewed your comments on prior thread at post # 74 and 251; and have also noted your comm...

DrKSSMDPhD on The Long Case for Receptos / Jan 29, 2:31pm

A good synopsis today from John Carroll, the editor at Fierce.$ONCE has competition in hemophilia B, BUT has a heads...

DrKSSMDPhD on The 2015 Stock Gumshoe Biotechnology Awards Show! / Jan 24, 10:28pm

Rae: it's a good company in a quite unusual position: it hits the street having completed proof of concept in a set of v...

DrKSSMDPhD on The Finale is Grand: The Gain from Pain Mainly Explained / Jan 4, 4:18pm

I am keen on Spark. I hope they will push ahead with IPO'ing. I know Kathy High, MD, the CEO. I would describe her, and ...

DrKSSMDPhD on written by reader

Ebola: To Parse, Perchance to Parlay / Nov 1, 5:05pm

To ScorpioRising and Kris Prasad: sorry for being a latecomer to the discussion about AAV-8. I appreciate the references...

DrKSSMDPhD on written by reader

Yellow Eyes, White Powders, and an Unknown Nordic Biotech That May Green Things Up for Investors / Jul 9, 2:55pm

Dan: the dose in question here is 4 x 10 to the tenth virus genomes per kg. And that is given by peripheral iv.By co...

omcdac1 on written by reader

Hepatitis with a C: Right Now Someone You Know Has It (and Doesn’t Know) / Jun 2, 1:21pm

Dr. KSS, Any thoughts about this - Uniqure - (QURE ) http://www.uniqure.com/ What is the future of the company ? A...

DrKSSMDPhD on written by reader

NASH: An All-Too-Common Liver Disease and a Company Aiming to Treat It / Mar 17, 6:01pm

Gary: Thanks for posting and I appreciate your comments. Alnylam certainly has a future, though by now it is big. I don'...

biocqr on “This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug” / Feb 9, 5:41pm

Dr. KSS, re: # 419..... you said "A number can be x times greater than predicted. It cannot be x times less than predict...

karmaswimswami on “This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug” / Feb 7, 11:54am

Siva and biocqr: As to the objections raised regarding TT-034, AAV8 and liver transfection/transduction efficacy, I have...

biocqr on “This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug” / Feb 7, 10:35am

Thanks Siva,Regardless of how the Nature AAV8 trial was run, the fact remains that Dr. Kay was surprised that in the...

biocqr on “This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug” / Feb 5, 9:31pm

Dr. K, I’m still somewhat confused about the viability of using AAV8 for the successful transduction of TT-034. Accor...

karmaswimswami on “This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug” / Feb 5, 12:16pm

I meant also to point out that while this is a global/multinational $109 billion company that controls the markets for i...

karmaswimswami on “This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug” / Feb 4, 3:06pm

This 2011 NEJM paper should truly lay to rest any concerns that AAV8, as Benitec is using in the TT-034, can succesfully...

sivapann on “This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug” / Feb 4, 11:22am

Below is a piece from http://rnaitherapeutics.blogspot.com. Note that the author is pro Tekmira. Dr KSS - I would like t...

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In A Brutal Biotech Market, A Reason to Be Excited: Introducing Regado Biosciences

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