Gene edit­ing los­es its siz­zle. CRISPR Ther­a­peu­tics IPO de­buts be­neath the range

CRISPR Ther­a­peu­tics CEO Rodger No­vak

Some of the bloom has come off the rose of gene edit­ing. CRISPR Ther­a­peu­tics had to ac­cept $14 a share to launch its IPO, a bit be­low the range that it had set for it­self. And that had to be a dis­ap­point­ing de­but af­ter ri­vals Ed­i­tas and In­tel­lia were both able to fire off hot of­fer­ings ear­li­er in the year — though they have al­so been feel­ing the chill more re­cent­ly.

CRISPR Ther­a­peu­tics $CR­SP still came away with $56 mil­lion from the maid­en of­fer­ing, adding $35 mil­lion with a sale of 2.5 mil­lion shares to its big part­ner Bay­er un­der their ear­li­er pact. Add it up and the $91 mil­lion does drop in­to the same ball­park as the $94.4 mil­lion score by Ed­i­tas and the $108 mil­lion reaped by In­tel­lia in the spring. But it’s sig­nif­i­cant­ly less than what the biotech was an­gling for.

These com­pa­nies have been de­vel­op­ing pre­clin­i­cal pro­grams re­ly­ing on CRISPR-Cas9 gene edit­ing tech­nol­o­gy, adapt­ing what’s been wide­ly billed as a faster, cheap­er way to slice and splice DNA in pur­suit of new treat­ments. And each have plans to cau­tious­ly leap from an­i­mals in­to hu­mans, leav­ing the field full of promise but years away from any ac­tu­al prod­uct. More in­vestors may be wak­ing up to the fact that this is one rev­o­lu­tion that is like­ly to play out in slow mo­tion.

That may be why Ed­i­tas shares $ED­IT have swooned, slid­ing from a high of to $44 to yes­ter­day’s close of $13.81, be­low its de­but price. In­tel­lia $NT­LA has al­so been feel­ing the chill, drop­ping from $30.40 to yes­ter­day’s close of $12.02. Ear­li­er this year gene edit­ing had been one of the few bright spots among a sig­nif­i­cant­ly re­duced stream of biotech IPOs, as the big boom of 2013-2015 fad­ed.

The lat­est gene edit­ing IPO leaves the three top play­ers on full pub­lic dis­play, with a fourth, Cari­bou, still op­er­at­ing pri­vate­ly with ven­ture back­ing.

CRISPR Ther­a­peu­tics, head­ed by CEO Rodger No­vak and based in Switzer­land with R&D in Cam­bridge, MA, has been play­ing its cards close to its vest in re­cent months,  but it sent a clear sig­nal about the up­com­ing IPO in June, when it added a com­plete­ly un­need­ed $38 mil­lion to its last ven­ture round, tak­ing it to a whop­ping $140 mil­lion. Biotechs that go pub­lic these days need plen­ty of cash in the bank and in­sid­er in­vestors lin­ing up to buy shares.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

FDA pan­el large­ly op­pos­es ex­pand­ing use of Lil­ly, Boehringer's SGLT2 in­hibitor to type I di­a­bet­ics

Last week, Eli Lilly and partner Boehringer Ingelheim rejigged the terms of their 2011 diabetes pact that nurtured the development of their blockbuster Jardiance franchise. Akin to manufacturers of rival SGLT2 drugs, the companies are working to expanding the use of their type II diabetes drug to reach a broader group of patients. On Wednesday, an expert panel to the FDA resisted that effort by largely voting against their quest to market the drug in type I diabetics.

Amir Nashat, World Medical Innovation Forum via Youtube

Bay­er bets up to $100M on ex­plor­ing new bio­mol­e­c­u­lar con­den­sate ter­rain with a biotech up­start

In the Indiana Jones warehouse of genomic oddities, the millions of units of so-called “junk DNA” that create nothing but play a hand in tons of things have grabbed most of the attention. But there are other arks and Templar crosses out there.

Among them: the code for intrinsically disordered regions. Floating like boundless clumps of boiling spaghetti throughout the cell, these regions first appeared in scientific sketches at the turn of the century before vanishing from most cell diagrams, such as those in a high school textbook. Most organelles were neatly bound in membranes. These loose molecules resisted characterization. Scientists largely ignored them.

In­vestors could emerge from Neil Wood­ford de­ba­cle with £1B loss, in­ter­nal analy­sis re­veals

When Link Fund Solutions announced that it is closing Woodford Equity Income Fund permanently and kicking out Neil Woodford, it was implied that investors probably won’t get back everything they entrusted to the fund manager. But nobody knew just how much they would lose.

An internal analysis commissioned by Link suggested that the collective loss could amount to £1 billion — out of a fund last valued at £3.1 billion — Citywire has revealed.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

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The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.